WEEK OF SEPTEMBER 25, 2017

5 CLINICAL NEWSRegulatory, Clinical Results, Clinical Status

1 COMPANY NEWSDeals, Sales & Marketing, Other News

16 FINANCIAL NEWSCompleted Offerings, Other Financial News

COMPANY NEWS

DEALSAcceleron Pharma Inc. (NASDAQ:XLRN), Cambridge, Mass.Celgene Corp. (NASDAQ:CELG), Summit, N.J.

Business: Cancer, Hematology, CardiovascularAcceleron Pharma Inc. (NASDAQ:XLRN) gained worldwide rights to develop and commercialize sotatercept to treat pulmonary hypertension, amending a deal with partner Celgene Corp.(NASDAQ:CELG). Acceleron expects to begin a Phase II trial of sotatercept to treat pulmonary arterial hypertension (PAH) in 1H18.In 2008, Acceleron and Celgene partnered to co-develop and commercialize Acceleron's sotatercept for all indications, with an initial focus on cancer, including breast and ovarian cancers, and cancer-related bone loss. At the time, Acceleron received $50 million up front, including a $5 million equity investment by Celgene, and was eligible for up to $510 million in milestones, plus tiered royalties (see BioCentury, Feb. 25, 2008).Under the amended deal, Celgene is now eligible for low-twenty percent royalties for pulmonary hypertension indications. Acceleron spokesperson Brad Miles told BioCentury that Celgene and other investigators are evaluating sotatercept in multiple myeloma (MM) and Diamond-Blackfan syndrome. According to ClinicalTrials.gov, sotatercept completed a Phase II trial in MM and is in Phase I/II testing for Diamond-Blackfan syndrome.Sotatercept is a soluble fusion protein consisting of the extracellular domain of activin receptor type 2A (ACVR2A) linked to the Fc protein of human IgGI.Acceleron and Celgene are also partnered under a 2011 deal to co-develop and commercialize luspatercept (ACE-536). Top-

line data are expected in mid-2018 from Phase III trials to treat myelodysplastic syndromes (MDS) and beta-thalassemia (see BioCentury, Aug. 8, 2011).Luspatercept is a modified ACVR2B fusion protein that inhibits several ligands in the transforming growth factor (TGF) beta superfamily.

Arcus Biosciences, Hayward, Calif.Taiho Pharmaceutical Co. Ltd., Tokyo, Japan

Business: CancerCancer company Arcus Biosciences (Hayward, Calif.) granted Taiho Pharmaceutical Co. Ltd. (Tokyo, Japan) an option to develop and commercialize compounds in Japan and other Asian territories excluding China. Arcus said the deal includes anything listed on its pipeline chart.Arcus will receive $35 million over the first three years of the five-year agreement and is eligible to receive up to $275 million in milestones for each molecule, plus high-single to mid-double-digit royalties. Arcus declined to disclose additional financial terms.According to its website, Arcus expects to begin Phase I trials of AB928 next quarter and AB154 in 2Q18. AB928 is a dual adenosine A2A receptor (ADORA2A) and ADORA2B antagonist, and AB154 is a mAb that inhibits T cell immunoreceptor with Ig and ITIM domains (TIGIT).

CritiTech Inc., Lawrence, Kan.DFB Pharmaceuticals LLC, Fort Worth, TexasNanOlogy LLC, Fort Worth, TexasU.S. Biotest Inc., San Luis Obispo, Calif.

Business: Cancer, Drug delivery

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DFB Pharmaceuticals LLC (Fort Worth, Texas), CritiTech Inc. (Lawrence, Kan.) and U.S. Biotest Inc. (San Luis Obispo, Calif.) formed NanOlogy LLC (Fort Worth, Texas) to develop nanoparticle drug-delivery technology to treat cancer.The newco's pipeline includes NanoPac, a nanoparticle form of paclitaxel, in Phase II testing to treat ovarian, prostate and pancreatic cancer as well as mucinous cystic pancreatic neoplasms. Data from the prostate and two pancreatic trials are expected next year. Data are also expected early next year from a Phase II trial of SOR007, a topical ointment of uncoated nanoparticle paclitaxel, to treat actinic keratosis lesions. A Phase II study of SOR007 to treat cutaneous metastases from non-melanoma cancer is expected to begin next quarter. DFB's affiliate DFB Soria LLC granted NanOlogy rights to SOR007 for cancer indications.NanOlogy's pipeline is being developed under section 505(b)(2) of the Food, Drug and Cosmetic Act, which allows sponsors to reference data on safety and efficacy from the scientific literature or from previously approved products.

Dimension Therapeutics Inc. (NASDAQ:DMTX), Cambridge, Mass.Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), Novato, Calif.RegenxBio Inc. (NASDAQ:RGNX), Rockville, Md.

Business: Gene/cell therapy, Endocrine/MetabolicOn Sept. 18, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) offered to acquire gene therapy play Dimension Therapeutics Inc. (NASDAQ:DMTX) for $5.50 per share in cash, or about $138 million. In August, RegenxBio Inc. (NASDAQ:RGNX) agreed to acquire Dimension in a stock deal with an implied value of $3.41 per Dimension share.Dimension said its board will review and consider Ultragenyx's unsolicited offer, which is a 358% premium to Dimension's close of $1.20 on Aug. 24, the day before it announced the RegenxBio deal. On Sept. 19, Dimension said Ultragenyx's offer could reasonably be expected to lead to a "Superior Proposal" as defined in Dimension’s merger agreement with RegenxBio, which would legally allow Dimension to sit down at the table with Ultragenyx.Ultragenyx is developing biologics and small molecules to treat rare genetic diseases. The company believes it can offer its "clinical and regulatory expertise" and "growing rare metabolic disease commercial infrastructure to advance Dimension’s rare disease focused gene therapies through the clinic and to maximize their reach with patients." Ultragenyx said it would maintain a gene therapy development unit at Dimension's Massachusetts facilities.Under the terms of RegenxBio's deal, Dimension would become a RegenxBio subsidiary and Dimension shareholders would own about 10.9% of the combined company. In August, the companies said they expect the deal to close by year end. Dimension would have to pay RegenxBio a $2.9 million fee to terminate the deal, according to an SEC filing. RegenxBio declined to comment.

Dimension's most advanced gene therapy product, DTX301, is in Phase I/II testing to treat ornithine transcarbamylase (OTC; OTCD) deficiency. DTX301 is an adeno-associated virus 8 (AAV8) vector that delivers the OTC gene to liver cells. Dimension plans to submit an IND early next year for its next lead candidate DTX401, an AAV vector that delivers glucose-6-phosphatase catalytic subunit-related protein (G6PC; G6Pase), to treat glycogen storage disease type Ia (GSDIa). Both products have Orphan Drug designation in the U.S. and EU.Dimension developed all of its candidates using RegenxBio's NAV technology, to which Dimension has a license.Ultragenyx's recombinant human glucuronidase beta (rhGUS) is under FDA and EMA review to treat mucopolysaccharidosis VII (MPS VII; Sly syndrome). The BLA's PDUFA date is Nov. 16. In August, the company submitted a BLA to FDA for burosumab (KRN23, UX023) to treat X-linked hypophosphatemia (XLH). An MAA for burosumab to treat XLH is under EMA review with a CHMP opinion expected around year end Burosumab is a recombinant human IgG1 mAb against fibroblast growth factor 23 (FGF23).

Fred Hutchinson Cancer Research Center, Seattle, Wash.Fortress Biotech Inc. (NASDAQ:FBIO), New York, N.Y.

Business: Gene/Cell therapy, Cancer, HematologyFred Hutchinson Cancer Research Center (Seattle, Wash.) granted the Mustang Bio Inc. (NASDAQ:MBIO) subsidiary of Fortress Biotech Inc. (NASDAQ:FBIO) exclusive, worldwide rights to a third-generation CD20-specific chimeric antigen receptor (CAR) T therapy that incorporates tumor necrosis factor (TNF) receptor superfamily member 9 (4-1BB; TNFRSF9; CD137) and CD28 co-stimulatory domains. Mustang will partially fund a Phase I/II trial to evaluate the candidate in patients with relapsed or refractory B cell non-Hodgkin’s lymphoma (NHL). The trial is expected to begin at the center next quarter.The center and Mustang declined to provide further details.

Immunocore Ltd., Abingdon, U.K.Bill & Melinda Gates Foundation, Seattle, Wash.

Business: InfectiousT cell receptor (TCR) company Immunocore Ltd. (Abingdon, U.K.) received a $40 million equity investment from the Bill & Melinda Gates Foundation (Seattle, Wash.) as part of a collaboration to develop new therapies to treat infectious diseases.Immunocore said the new capital will be used to accelerate the development of the biotech's two infectious disease platforms: immune mobilizing monoclonal TCRs against virus (ImmTAV) and immune mobilizing monoclonal TCRs against bacteria (ImmTAB).The ImmTAV and ImmTAB technologies are similar in design and function to Immunocore's ImmTAC platform for cancer. Each is a bispecific biologic that combines an affinity-enhanced T cell

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receptor with an anti-CD3 single chain variable fragment (scFv) effector function to activate a T cell response against the target cell.Under the collaboration, Immunocore's first infectious disease programs will target tuberculosis and HIV.Most of Immunocore's pipeline is focused on cancer, with the lead program, IMCgp100, in a pivotal Phase II trial to treat advanced uveal melanoma.In 2015, Immunocore raised $320 million in a series A round, which at the time was the largest ever venture round for a European biotech (see BioCentury, July 20, 2015).

Inovalon Holdings Inc. (NASDAQ:INOV), Bowie, Md.Daiichi Sankyo Co. Ltd. (Tokyo:4568), Tokyo, Japan

Business: Neurology, BioinformaticsDaiichi Sankyo Co. Ltd. (Tokyo:4568) partnered with Inovalon Holdings Inc. (NASDAQ:INOV) to use Inovalon’s MORE2 Registry and Inovalon ONE platform to identify outcomes for abuse-deterrent opioids that would be valuable to health plans and then design outcomes-based contracts based on their findings. Inovalon declined to provide financial terms.The Medical Outcomes Research for Effectiveness and Economics (MORE2) Registry houses longitudinal demographic and medical data from 230 million patients. The Inovalon ONE cloud-based platform combines more than 70 data tool sets including technologies for data integrity analysis, predictive analysis, clinical data optical character recognition, natural language processing, decision support and large-scale transactional computation. Dan Mendelson, President of Inovalon’s Avalere Health LLC subsidiary, told BioCentury that the company’s analysis will seek to determine the outcomes which would be the “most compelling” to health plan providers. Daiichi did not respond to inquiries.In the U.S., Daiichi markets MorphaBond ER, an extended-release morphine formulation, and RoxyBond, an immediate-release oral oxycodone, to treat pain. The company has rights to the products from Inspirion Delivery Sciences LLC (Morristown, N.J.), on whose SentryBond abuse-deterrent technologies the products are based.

Johnson & Johnson (NYSE:JNJ), New Brunswick, N.J.Provention Bio Inc., Lebanon, N.J.

Business: Autoimmune, InflammationProvention Bio Inc. (Lebanon, N.J.), a newco pursuing autoimmune and inflammatory disease therapies, disclosed details regarding two in-licensed programs from the Janssen unit of Johnson & Johnson (NYSE:JNJ).Provention holds exclusive, worldwide rights to PRV-6527 (JNJ-40346527), an oral colony-stimulating factor 1 receptor (CSF1R; C-FMS; CD115) inhibitor, to treat inflammatory bowel disease (IBD) and to PRV-300 (JNJ-42915925), a toll-like receptor 3 (TLR3) mAb, for all indications.

In 1H18, Provention plans to begin a Phase IIa proof-of-concept study of PRV-6527 to treat Crohn's disease and a Phase I/II study of PRV-300 to treat ulcerative colitis.J&J spokesperson Brian Kenney said Janssen out-licensed PRV-6527 and PRV-300 to Provention after "considering the depth of our portfolio" and said Provention has the "translational and clinical expertise" needed to progress the assets.Provention also has exclusive, worldwide rights to a preclinical enterovirus vaccine against coxsackievirus B (CVB) infection from Vactech Oy (Tampere, Finland), which it is developing to prevent or delay the onset of Type I diabetes.The terms of the deals with Janssen and Vactech are undisclosed.Provention launched in June with all three assets and a $28.4 million series A round from the J&J's Johnson & Johnson Innovation-JJDC Inc. arm, the JDRF T1D fund and undisclosed private investors (see BioCentury, July 6).

Synpromics Ltd., Edinburgh, U.K.Solid Biosciences LLC, Cambridge, Mass.

Business: Musculoskeletal, Gene/Cell therapySynpromics Ltd. (Edinburgh, U.K.) will provide Solid Biosciences LLC (Cambridge, Mass.) access to muscle-selective synthetic DNA transcription promoter candidates based on Synpromics' PromPT platform which will be used to develop therapies for Duchenne muscular dystrophy (DMD).The companies said the promoters will be evaluated in vivo and that they will “enhance” Solid’s adeno-associated virus (AAV) program. The companies declined to provide terms of the deal, but Solid spokesperson Courtney Heath told BioCentury that "Synpromics' technology has the potential to help enhance specificity, expression and gene delivery for new gene therapy candidates as the company continues to build its portfolio."By year end, Solid plans to begin a Phase I/II trial of its preclinical AAV vector candidate, SGT-001, to treat DMD. The gene therapy delivered via an AAV vector is designed to stabilize the dystrophin glycoprotein complex by delivering microdystrophin, a shortened but functional copy of dystrophin. Heath added that promoter candidates identified under the deal will not be applied to Solid's existing candidates, including SGT-001.

COMPANY NEWS

SALES & MARKETINGBristol-Myers Squibb Co. (NYSE:BMY), New York, N.Y.

Business: CancerThe U.K.'s NICE issued a final appraisal determination (FAD) recommending the use of Opdivo nivolumab from Bristol-Myers Squibb Co. (NYSE:BMY) to treat squamous and non-squamous non-small cell lung cancer (NSCLC). The committee recommended

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the PD-1 inhibitor after BMS presented additional data and agreed to an arrangement through the country's Cancer Drugs Fund. Approximately 950 squamous and 350 non-squamous patients will be treated within the CDF agreement.In 2016, NICE issued draft guidance recommending against the use of Opdivo for the indication, but asked BMS to make a case for the inclusion of Opdivo in the CDF. At the time, NICE said BMS's CDF proposal should describe its plans for collecting data to address clinical uncertainties, including Opdivo's effectiveness in patients with high PD-L1 expression, and "demonstrate a plausible potential for cost effectiveness" (see BioCentury, Oct. 17, 2016).Under the CDF arrangement, five-year follow-up data will become available in June 2019 from the the Phase III CheckMate -017 trial to treat squamous NSCLC and the Phase III CheckMate -057 trial to treat non-squamous NSCLC. Both trials are evaluating Opdivo vs. docetaxel.The committee said the most plausible incremental cost-effectiveness ratio (ICER) would be £50,014 ($67,779) and £49,160 ($66,622) per quality-adjusted life year (QALY) gained for squamous and non-squamous NSCLC, respectively. The list price of Opdivo is £5,268 ($7,140) per month for a 73 kg patient.In the EU, Opdivo is approved to treat NSCLC, renal cell carcinoma (RCC), classical Hodgkin's lymphoma, squamous cell carcinoma of the head and neck (SCCHN) and urothelial carcinoma.

COMPANY NEWS

OTHER NEWSPfizer Inc. (NYSE:PFE), New York, N.Y.Johnson & Johnson (NYSE:JNJ), New Brunswick, N.J.

Business: Autoimmune, BiosimilarsPfizer Inc. (NYSE:PFE) filed a lawsuit alleging that Johnson & Johnson (NYSE:JNJ) violated antitrust laws by engaging in anticompetitive practices aiming at blocking sales of Pfizer’s biosimilar version of autoimmune drug Remicade infliximab.Pfizer's antitrust action takes a different approach than other biosimilar lawsuits, which typically allege patent infringement.In its complaint, filed in the U.S. District Court for the Eastern District of Pennsylvania, Pfizer says J&J threatened to withhold significant rebates unless insurers agreed to “biosimilar-exclusion” contracts that effectively block coverage for Pfizer’s Inflectra infliximab-dyyb and other infliximab biosimilars. Pfizer also alleged that J&J offered providers anticompetitive contracts that gave discounts on Remicade in exchange for not purchasing biosimilars to Remicade.Pfizer claims that those exclusionary contracts have caused insurers to deny coverage for Inflectra, even though the biosimilar has a lower cost. Inflectra's wholesale acquisition cost (WAC) of $946.28 per

package is about a 19% discount to Remicade's WAC of $1,167.82 per package.The complaint also argues that by "inappropriately excluding biosimilar competitors," J&J also undermines the principal goals of the Biologics Price Competition and Innovation Act (BPCIA).In a statement to BioCentury, Scott White, president of J&J's Janssen Biotech Inc. unit, said the lawsuit has no merit. "We are effectively competing on value and price and to date, Pfizer has failed to demonstrate sufficient value to patients, providers, payers and employers," he added.Inflectra posted $172 million in 1H17 sales, while Remicade generated 1H17 revenues of $3.2 billion.Pfizer launched Inflectra in the U.S. last November.In August 2016, a federal judge invalidated a patent covering Remicade, removing an obstacle to biosimilar competition. J&J filed the suit in 2015 seeking to prevent Pfizer from marketing Inflectra (see BioCentury, Aug. 22, 2016).Pfizer has U.S. rights to Inflectra from Celltrion Inc. (KOSDAQ:068270). The companies both market the biosimilar in the EU, where they share co-exclusive rights. Celltrion markets it as Remsima.Remicade is a humanized mAb against TNF alpha.In April, FDA approved a second Remicade biosimilar, Renflexis infliximab-abda, from Merck & Co. Inc. (NYSE:MRK) and Samsung Bioepis, a JV between Biogen Inc. (NASDAQ:BIIB) and Samsung BioLogics Co. Ltd. (KOSDAQ:20740) (see BioCentury, July 28).

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CLINICAL NEWS

REGULATORYAB Science S.A. (Euronext:AB), Paris, France

Product: Masipro masitinib (Masiviera, Masican, AB1010)Business: DermatologyEMA's CHMP for the second time recommended against approval of a resubmitted MAA from AB Science S.A. (Euronext:AB) for Masipro masitinib (Masiviera, Masican, AB1010) to treat systemic mastocytosis in adults. CHMP's concerns echoed those of a May negative opinion for the application, citing GCP failings, changes to the study design while the study was ongoing and limited safety data (see BioCentury, May 19). The committee was also concerned about side effects, including neutropenia and harmful effects on the skin and liver. AB Science said it plans to initiate a confirmatory study in response. Masitinib is a stem cell factor (SCF) receptor tyrosine kinase (c-Kit; KIT; CD117) inhibitor.

Biogen Inc. (NASDAQ:BIIB), Cambridge, Mass.Samsung BioLogics Co. Ltd. (KOSDAQ:207940), Seoul, South Korea

Product: Ontruzant biosimilar trastuzumab (SB3)Business: CancerEMA’s CHMP backed approval of Ontruzant (SB3) from Samsung Bioepis Co. Ltd. The product is a biosimilar of Herceptin trastuzumab from Roche (SIX:ROG; OTCQX:RHHBY) and its Genentech Inc. unit. The biosimilar is recommended to treat HER2-positive metastatic breast cancer, HER2-positive early breast cancer and HER2-positive metastatic gastric cancer. Ontruzant is the first trastuzumab biosimilar recommended for approval by CHMP.Herceptin is a humanized mAb against HER2. Samsung Bioepis, a JV between Biogen Inc. (NASDAQ:BIIB) and Samsung BioLogics Co. Ltd. (KOSDAQ:207940), launched in 2012 to develop biosimilars.

Boehringer Ingelheim GmbH, Ingelheim, Germany

Product: Cyltezo adalimumab (BI 695501)Business: AutoimmuneEMA’s CHMP recommended approval of Cyltezo adalimumab (BI 695501) from Boehringer Ingelheim GmbH (Ingelheim, Germany). The product is a biosimilar of Humira adalimumab from AbbVie Inc. (NYSE:ABBV). The biosimilar is recommended to treat rheumatoid arthritis (RA), juvenile idiopathic arthritis, axial spondyloarthritis, psoriatic arthritis, psoriasis, pediatric plaque psoriasis, hidradenitis suppurativa (HS), Crohn’s disease (CD), ulcerative colitis (UC) and uveitis. FDA approved Cyltezo in August. Humira is a mAb against tumor necrosis factor (TNF) alpha (see BioCentury, Sept. 1).

Braeburn Pharmaceuticals Inc., Princeton, N.J.Camurus AB (SSE:CAMX), Lund, Sweden

Product: CAM2038

Business: NeurologyBraeburn Pharmaceuticals Inc. (Princeton, N.J.) said FDA accepted and granted Priority Review to an NDA for weekly and monthly buprenorphine injection depot (CAM2038) to treat opioid use disorder. Its PDUFA date is Jan. 19, 2018.Braeburn has exclusive, North American rights as well as option rights in Japan, Korea, Taiwan and China to CAM2038 under an expanded 2014 deal with Camurus (see BioCentury, Dec. 8, 2014 & Oct. 31, 2016).CAM2038 is a ready-to-use prefilled syringe containing a lipid solution of buprenorphine with Camurus' FluidCrystal injection depot technology.

Bristol-Myers Squibb Co. (NYSE:BMY), New York, N.Y.Ono Pharmaceutical Co. Ltd. (Tokyo:4528), Osaka, Japan

Product: Opdivo nivolumab (BMS-936558, MDX-1106, ONO-4538)Business: CancerEMA's CHMP announced that in July Bristol-Myers Squibb Co. (NYSE:BMY) withdrew an MAA seeking to expand label for Opdivo nivolumab (BMS-936558, MDX-1106, ONO-4538) to include treatment of hepatocellular carcinoma in adults previously treated with Nexavar sorafenib. The committee said that at the time of the withdrawal, it was of the provisional opinion that the data BMS provided were insufficient to support approval because the study did not compare Opdivo directly with other treatments. CHMP also said there was insufficient information about the patients enrolled in the study.The human IgG4 mAb against PD-1 is approved in the EU to treat NSCLC, renal cell carcinoma (RCC), classical Hodgkin's lymphoma, squamous cell carcinoma of the head and neck (SCCHN) and urothelial carcinoma.BMS has worldwide rights to Opdivo from Ono Pharmaceutical Co. Ltd. (Tokyo:4528), except in Japan, South Korea and Taiwan, where Ono and BMS are partnered for the product (see BioCentury, July 28, 2014).

CSL Ltd. (ASX:CSL), Melbourne, Australia

Product: PrivigenBusiness: NeurologyCSL Ltd. (ASX:CSL) said FDA approved Privigen to treat chronic inflammatory demyelinating polyneuropathy (CIDP). The 10% formulation of IV immunoglobulin is approved in the U.S. to treat primary immunodeficiency (PI) and immune thrombocytopenic purpura (ITP).

GlaxoSmithKline plc (LSE:GSK; NYSE:GSK), London, U.K.Innoviva Inc. (NASDAQ:INVA), South San Francisco, Calif.

Product: Trelegy Ellipta fluticasone furoate/umeclidinium/vilanterolBusiness: Pulmonary

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FDA approved an NDA from GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) for Trelegy Ellipta fluticasone furoate/umeclidinium/vilanterol for maintenance treatment of chronic obstructive pulmonary disease. GSK said Trelegy Ellipta is the first once-daily single inhaler triple therapy approved for COPD in the U.S.Trelegy Ellipta is specifically indicated for COPD patients already receiving fixed-dose Breo Ellipta fluticasone furoate/vilanterol with or without Incruse Ellipta umeclidinium.On Sept. 19, GSK spokesperson Juan Carlos Molina told BioCentury that GSK will launch Trelegy Ellipta in the U.S. "shortly" at a wholesale acquisition cost (WAC) of $530 for a 30-day supply. The pharma will pay tiered royalties of 6.5-10% on annual net sales of the product to Innoviva Inc. (NASDAQ:INVA) under a deal to develop inhaled long-acting adrenergic receptor beta 2 agonist (LABA) products.FDA partly based the approval on data from the Phase III 200109 and 200110 trials, which showed that adding umeclidinium to fluticasone furoate/vilanterol significantly improved lung function in COPD patients.Trelegy Ellipta is a fixed-dose combination of 100 µg fluticasone furoate, an inhaled corticosteroid; 62.5 µg umeclidinium bromide, a long-acting muscarinic antagonist (LAMA); and 25 µg vilanterol, a LABA, delivered with GSK's Ellipta dry powder inhaler.On Sept. 15, EMA's CHMP recommended approval of Trelegy Ellipta for maintenance treatment of COPD.

Intercept Pharmaceuticals Inc. (NASDAQ:ICPT), New York, N.Y.Sumitomo Dainippon Pharma Co. Ltd. (Tokyo:4506), Osaka, Japan

Product: Ocaliva obeticholic acid (DSP-1747)Business: HepaticOn Sept. 21, FDA issued a drug safety communication warning of serious liver injury in some patients incorrectly dosed with Ocaliva obeticholic acid (DSP-1747) from Intercept Pharmaceuticals Inc. (NASDAQ:ICPT).Ocaliva is an oral farnesoid X receptor (FXR; NR1H4) agonist that has accelerated approval in the U.S. to treat primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. The drug also has conditional approval in the EU for the indication.Earlier this month, Intercept issued a "Dear Health Care Provider" letter warning that liver injury, liver decompensation, liver failure and death have been reported in PBC patients with moderate to severe hepatic impairment when Ocaliva was dosed more frequently than recommended on its label (see BioCentury, Sept. 15).In its announcement, FDA warned that Ocaliva is being incorrectly dosed in some patients with moderate to severe decreases in liver function, leading to an increased risk of serious liver injury and death. The agency recommended that healthcare professionals

determine a patient’s baseline liver function prior to starting Ocaliva, monitor patients frequently for disease progression, and reduce dosing frequency for patients who progress to moderate or severe liver impairment.Through the FDA Adverse Event Reporting System (FAERS), the agency said it received reports of 19 deaths and 11 cases of serious liver injury associated with Ocaliva in the 13 months since the drug was approved in May 2016. Of the 8 cases that provided information about the patient's cause of death, the cause was reported to be worsening of PBC in 7 cases and cardiovascular disease in the other case. Seven deaths and 6 cases of serious liver injury described patients with moderate to severe decreases in liver function who were dosed more frequently with Ocaliva than the drug's FDA label recommends.Spokesperson Christopher Frates told BioCentury it is reviewing FDA's warning and will provide a company statement "as soon as possible."Ocaliva is also in Phase III testing for non-alcoholic steatohepatitis (NASH) and Phase II testing for primary sclerosing cholangitis (PSC) and biliary atresia.Sumitomo Dainippon Pharma Co. Ltd. (Tokyo:4506) has exclusive rights in Japan and China from Intercept to develop and commercialize the product for chronic liver disease (see BioCentury, April 4, 2011).

Ipsen Group (Euronext:IPN; Pink:IPSEY), Boulogne-Billancourt, FranceTeijin Pharma Ltd., Tokyo, Japan

Product: Somatuline Autogel (Somatuline Depot) lanreotide acetate (ITM-014)Business: CancerIpsen Group (Euronext:IPN; Pink:IPSEY) said FDA approved Somatuline Depot lanreotide acetate (ITM-014) to treat carcinoid syndrome.The extended-release formulation of somatostatin analog lanreotide is already approved in the U.S. to treat acromegaly and gastroenteropancreatic neuroendocrine tumors (GEP-NETs). The product is approved to treat acromegaly and neuroendocrine tumors in 70 countries, and is marketed outside the U.S. as Somatuline Autogel.Teijin Pharma Ltd. (Tokyo, Japan) and Ipsen have a joint development and marketing deal for the compound in Japan.

Merck KGaA (Xetra:MRK), Darmstadt, GermanyPfizer Inc. (NYSE:PFE), New York, N.Y.

Product: Bavencio avelumab (MSB0010718C, PF-06834635)Business: CancerThe European Commission approved Bavencio avelumab (MSB0010718C, PF-06834635) from Merck KGaA (Xetra:MRK) and Pfizer Inc. (NYSE:PFE) to treat metastatic Merkel cell

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carcinoma. The companies expect to initially launch the human IgG1 mAb against PD-L1 in Germany and the U.K. as early as October.Bavencio has accelerated approval in the U.S. to treat metastatic Merkel cell carcinoma and locally advanced or metastatic urothelial carcinoma. Merck and Pfizer share Bavencio's rights under a 2014 deal (see BioCentury, Nov. 24, 2014).

MorphoSys AG (Xetra:MOR; Pink:MPSYY), Martinsried, GermanyJohnson & Johnson (NYSE:JNJ), New Brunswick, N.J.

Product: Tremfya guselkumab (CNTO 1959)Business: AutoimmuneEMA’s CHMP recommended approval of Tremfya guselkumab (CNTO 1959) from Johnson & Johnson (NYSE:JNJ) to treat moderate to severe plaque psoriasis in patients who are candidates for systemic therapy. The human HuCAL mAb targeting the p19 subunit of IL-23 was approved by FDA in July (see BioCentury, July 14).Under a 2000 deal, MorphoSys AG (Xetra:MOR; Pink:MPSYY) used its HuCAL human combinatorial antibody library to generate antibodies, including guselkumab, for J&J. The discovery portion of the deal expired in 2007 and MorphoSys is eligible for milestones and royalties (see BioCentury, Jan. 8, 2001).

Pfizer Inc. (NYSE:PFE), New York, N.Y.

Product: Sutent sunitinibBusiness: CancerFDA’s Oncologic Drugs Advisory Committee voted 6-6 on Sept. 19 that the benefit-risk profile of Sutent sunitinib from Pfizer Inc. (NYSE:PFE) is acceptable for the adjuvant treatment of patients at high risk of recurrent renal cell carcinoma following nephrectomy. The drug, which is already approved in the U.S. to treat advanced RCC, has a January PDUFA date for the indication.In briefing documents released ahead of the meeting, FDA asked the panel to discuss whether Sutent showed an improvement on the primary endpoint of disease-free survival (DFS) in the Phase III S-TRAC trial and whether DFS as defined in S-TRAC is a clinically relevant endpoint. The briefing documents noted that there is no widely agreed upon definition of DFS in RCC as there is in breast cancer.S-TRAC enrolled 615 treatment-naïve patients at high risk of recurrent RCC following surgery and showed that adjuvant treatment with Sutent led to a median DFS as determined by an independent radiology committee of 6.8 years vs. 5.6 years for placebo (HR=0.76, 95% CI: 0.59, 0.98, p=0.03). The 5-year DFS rate was 59% for Sutent vs. 51% for placebo (see BioCentury, Oct. 17, 2016).According to Pfizer, about 15% of patients with primary resected RCC are at high risk for recurrence and about 60% of these high-risk patients will have recurrence and develop metastatic disease within 5 years.

The small molecule that inhibits multiple tyrosine kinases is also approved in the U.S. to treat gastrointestinal stromal tumors (GIST) and advanced pancreatic neuroendocrine tumors. The company reported 2016 Sutent sales of $1.1 billion, representing 24% of its oncology revenue and 2% of its total revenue that year.

Santhera Pharmaceuticals Holding AG (SIX:SANN), Liestal, Switzerland

Product: Raxone idebenone (SNT-MC17)Business: MusculoskeletalEMA's CHMP recommended against approving an MAA for Raxone idebenone (SNT-MC17) from Santhera Pharmaceuticals Holding AG (SIX:SANN) to slow down the worsening of breathing function in Duchenne muscular dystrophy (DMD) patients who are not taking corticosteroids. CHMP said the data Santhera provided were insufficient because while Raxone did improve peak expiratory flow compared to placebo, the data showed "no clear improvement in other indicators of breathing function or in muscle strength, motor function or quality of life." The committee also said it had concerns about the way the study was conducted and analyzed, but did not provide details. Santhera said it plans to appeal the recommendation.Raxone, a short-chain benzoquinone, is approved in Europe to treat visual impairment in adolescents and adults with Leber’s hereditary optic neuropathy (LHON).

Tesaro Inc. (NASDAQ:TSRO), Waltham, Mass.Merck & Co. Inc. (NYSE:MRK), Kenilworth, N.J.

Product: Zejula niraparib (MK-4827, ZL-2306)Business: CancerEMA’S CHMP recommended approval of Zejula niraparib (MK-4827, ZL-2306) from Tesaro Inc. (NASDAQ:TSRO) as monotherapy for the maintenance treatment of adult patients with platinum-sensitive, relapsed, high-grade serous epithelial ovarian, fallopian tube or primary peritoneal cancer who are in response to platinum-based chemotherapy.FDA approved the oral PARP inhibitor in March (see BioCentury, March 30). In 2012, Merck & Co. Inc. (NYSE:MRK) granted Tesaro exclusive, worldwide rights to Zejula (see BioCentury, June 11, 2012).

ViiV Healthcare Ltd., Brentford, U.K.

Product: Triumeq dolutegravir/abacavir/lamivudine (Triumec, 572-Trii, S-349572/S-265744/S-247303)Business: InfectiousChina FDA approved Triumeq dolutegravir/abacavir/lamivudine (Triumec, 572-Trii, S-349572/S-265744/S-247303) from ViiV Healthcare Ltd. (Brentford, U.K.) to treat HIV/AIDS.Triumeq is a once-daily tablet that comprises Tivicay dolutegravir, an HIV integrase inhibitor, plus the nucleoside reverse transcriptase inhibitors (NRTIs) abacavir and lamivudine. It is approved in >70 countries, including the U.S. and EU, to treat HIV-1 infection.

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GlaxoSmithKline plc (LSE:GSK; NYSE:GSK), Pfizer Inc. (NYSE:PFE) and Shionogi & Co. Ltd. (Tokyo:4507) own ViiV.

XBiotech Inc. (NASDAQ:XBIT), Austin, Texas

Product: Hutruo (Xilonix, CV-18C3, T2-18C3, RA-18C3, MABp1, CA-18C3) Business: CancerEMA's CHMP reiterated its May negative opinion regarding an MAA from XBiotech Inc. (NASDAQ:XBIT) for Hutruo (Xilonix, CV-18C3, T2-18C3, RA-18C3, MABp1, CA-18C3) to treat symptoms of advanced colorectal cancer, including cachexia, saying the data provided did not show clear improvements in either lean body mass or quality of life (see BioCentury, April 28). The product is a human IgG1k mAb against IL-1 alpha.

CLINICAL NEWS

CLINICAL RESULTSAlnylam Pharmaceuticals Inc. (NASDAQ:ALNY), Cambridge, Mass.Sanofi (Euronext:SAN; NYSE:SNY), Paris, France

Product: Patisiran (ALN-TTR02, GENZ438027, GZ438027, SAR438027)Business: Endocrine/MetabolicMolecular target: Transthyretin (TTR)Description: IV RNAi therapeutic targeting the TTR gene using second-generation lipid nanoparticle (LNP) technologyIndication: Treat familial amyloidotic polyneuropathy (FAP)Endpoint: Change from baseline in modified Neuropathy Impairment Score+7 (mNIS+7) at 18 months; Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) score, NIS-Weakness subdomain of the mNIS+7, Rasch-built Overall Disability Scale (R-ODS), 10-meter walk test, modified BMI, and COMPASS-31 questionnaireStatus: Phase III dataMilestone: Additional Phase III data (11/2/2017); submit NDA (year end 2017); submit MAA (early 2018)Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) and the Sanofi Genzyme unit of Sanofi (Euronext:SAN; NYSE:SNY) reported top-line data showing that patisiran (ALN-TTR02) met the primary and all secondary endpoints in the Phase III APOLLO trial in 225 patients with familial amyloidotic polyneuropathy (FAP), a form of transthyretin (TTR) amyloidosis (ATTR). Late this year, Alnylam plans to submit an NDA to FDA for the IV RNAi therapeutic targeting the TTR gene, with an MAA submission to EMA expected early next year.On the primary endpoint, 0.3 mg/kg IV patisiran every 3 weeks significantly improved mean modified Neuropathy Impairment Score+7 (mNIS+7) from baseline to 18 months vs. placebo (p<0.00001). Patisiran also met the key secondary endpoint of improving the Norfolk Quality of Life Questionnaire-Diabetic

Neuropathy (Norfolk QOL-DN) score vs. placebo (p<0.00001), as well as the 5 other secondary endpoints of improving motor strength on the NIS-Weakness subdomain of the mNIS+7, disability on the Rasch-built Overall Disability Scale (R-ODS), gait speed as measured by the 10-meter walk test, nutritional status as measured by modified BMI, and autonomic symptoms as measured by the COMPASS-31 questionnaire vs. placebo (p<0.001 for all).The frequency of adverse events (96.6% vs. 97.4%) and deaths (4.7% vs. 7.8%) was similar between the patisiran and placebo arms. Additionally, there were fewer discontinuations from treatment (7.4% vs. 37.7%) and fewer discontinuations from treatment due to adverse events (4.7% vs. 14.3%) in the patisiran arm compared to the placebo arm. Peripheral edema occurred in 29.7% of patients in the patisiran arm compared to 22.1% of patients in the placebo arm.Full data from the double-blind, international trial will be presented at the European ATTR Amyloidosis Meeting for Patients and Doctors in Paris on Nov. 2.Sanofi Genzyme is also preparing regulatory applications for patisiran in Japan, Brazil and other countries, with submissions slated to begin in 1H18.Sanofi Genzyme has exclusive rights to patisiran from Alnylam outside of the U.S., Canada and Western Europe under a 2014 expansion of a 2012 RNAi deal (see BioCentury, Oct. 29, 2012 & Jan. 20, 2014). Patisiran uses second-generation lipid nanoparticle (LNP) technology from Arbutus Biopharma Corp. (NASDAQ:ABUS), which is eligible for milestones and royalties from Alnylam (see BioCentury, Nov. 19, 2012).

Amunix Operating Inc., Mountain View, Calif.Versartis Inc. (NASDAQ:VSAR), Redwood City, Calif.Teijin Pharma Ltd., Tokyo, Japan

Product: Somavaratan (VRS-317)Business: Endocrine/MetabolicMolecular target: NADescription: Long-acting recombinant human growth hormone (rhGH)Indication: Treat pediatric growth hormone deficiency (GHD)Endpoint: Annual height velocity at 12 months; safety and pharmacokineticsStatus: Phase III dataMilestone: NAVersartis Inc. (NASDAQ:VSAR) said somavaratan (VRS-317) missed the primary endpoint of non-inferiority to Genotropin somatropin in the Phase III VELOCITY trial to treat pediatric growth hormone deficiency (GHD).In the intent-to-treat (ITT) population, twice-monthly subcutaneous somavaratan failed to show non-inferiority to once-daily Genotropin in annual height velocity at 12 months (9.44 vs. 10.7 cm). Non-inferiority was defined as a lower bound of the 2-sided 95% CI for the difference in height velocity between treatment groups

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of greater than or equal to -2.0 cm/year. In the ITT population, the lower bound of the 2-sided 95% CI was -2.3 cm/year.Versartis said somavaratan did show non-inferiority to Genotropin on the primary endpoint in the per protocol (PP) population (9.71 vs. 10.63 cm). Secondary endpoints in the open-label, international trial included safety and pharmacokinetics.The company said it will continue to evaluate the data and provide an update on the program later this year. Versartis has rights to the long-acting recombinant human growth hormone (rhGH) from Amunix Operating Inc. (Mountain View, Calif.). Teijin Pharma Ltd. (Tokyo, Japan) has exclusive, Japanese rights to develop and commercialize somavaratan from Versartis (see BioCentury, Aug. 15, 2016).

Eli Lilly and Co. (NYSE:LLY), Indianapolis, Ind.

Product: Abemaciclib (LY2835219)Business: CancerMolecular target: Cyclin dependent kinase 4 (CDK4); CDK6Description: Selective dual inhibitor of CDK4 and CDK6Indication: Treat hormone receptor-positive, HER2-negative advanced breast cancerEndpoint: Progression-free survival (PFS); objective response rate (ORR), overall survival (OS), duration of response, disease control rate and pharmacokineticsStatus: Additional Phase III dataMilestone: PDUFA date (1Q18)Eli Lilly and Co. (NYSE:LLY) reported additional data from the Phase III MONARCH 3 trial in 493 postmenopausal women with hormone receptor-positive, HER2-negative, locoregionally recurrent or metastatic breast cancer showing that median progression-free survival, the primary endpoint, was not reached in patients receiving twice-daily 150 mg oral abemaciclib (LY2835219) plus anastrozole or letrozole vs. 14.7 months for anastrozole or letrozole alone (HR=0.54, 95% CI: 0.41, 0.72, p=0.000021). Abemaciclib plus anastrozole or letrozole led to an ORR, a secondary endpoint, of 59% vs. 44% for anastrozole or letrozole alone (p=0.04). Data were presented at the European Society for Medical Oncology meeting in Madrid.Lilly previously reported that abemaciclib plus anastrozole or letrozole met the PFS and ORR endpoints (see BioCentury, April 27). Additional secondary endpoints in the double-blind, placebo-controlled, international trial include overall survival (OS), duration of response, disease control rate (DCR), clinical benefit rate and pharmacokinetics.In July, FDA accepted and granted Priority Review to an NDA for abemaciclib to treat advanced breast cancer, with a PDUFA date in 1Q18. Specifically, abemaciclib is under review as monotherapy to treat hormone receptor-positive, HER2-negative advanced breast cancer in patients who had prior endocrine therapy and chemotherapy for metastatic disease; and in combination with Faslodex fulvestrant to treat hormone receptor-positive, HER2-

negative advanced breast cancer in women who had disease progression following endocrine therapy (see BioCentury, July 12).Abemaciclib is a selective dual inhibitor of cyclin dependent kinase 4 (CDK4) and CDK6. It has breakthrough therapy designation in the U.S. for refractory, hormone receptor-positive advanced or metastatic breast cancer.

GeNeuro S.A. (Euronext:GNRO), Geneva, Switzerland

Product: GNbAC1Business: AutoimmuneMolecular target: Endogenous retrovirus group W member 1 (HERVW; ERVW-1)Description: Humanized IgG4 mAb targeting the envelope protein of human HERVWIndication: Treat relapsing-remitting multiple sclerosis (RRMS)Endpoint: Cumulative number of gadolinium-enhancing T1 lesions on brain MRI at 6 months; MRI measures of neurodegeneration, clinical parameters at 6 and 12 months and biomarkersStatus: Phase IIb dataMilestone: Final Phase IIb data (1Q18)GeNeuro S.A. (Euronext:GNRO) and Servier (Neuilly-sur-Seine, France) reported data from the 12-month Phase IIb CHANGE-MS trial in 270 patients with relapsing-remitting multiple sclerosis (RRMS) showing that once-monthly IV GNbAC1 missed the primary endpoint of reducing the cumulative number of gadolinium-enhancing T1 lesions on brain MRI at 6 months vs. placebo. The companies also said there was no significant difference between treatment groups on other MRI measures of neuroinflammation. GNbAC1 was well tolerated. Final 12-month data from the double-blind, European trial are expected in 1Q18. GeNeuro said the study will continue as planned to explore "potential benefits" of the compound on MRI and clinical measures, including remyelination properties.GNbAC1 is a humanized IgG4 mAb targeting the envelope protein of human endogenous retrovirus group W member 1 (HERVW; ERVW-1). Data from a Phase IIa trial of GNbAC1 to treat Type I diabetes are expected in 3Q18.Servier has an option to license exclusive rights to develop and commercialize GNbAC1 worldwide, excluding the U.S. and Japan, after CHANGE-MS is completed (see BioCentury, Dec. 8, 2014 & Jan. 11, 2016).

GlaxoSmithKline plc (LSE:GSK; NYSE:GSK), London, U.K.

Product: Nucala mepolizumab (SB-240563)Business: PulmonaryMolecular target: IL-5Description: Humanized IgG mAb against IL-5Indication: Treat chronic obstructive pulmonary disease (COPD)

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Endpoint: Annual rate of moderate or severe exacerbations; time to first moderate or severe exacerbation, annual rate of exacerbations requiring hospitalization, St. George’s Respiratory Questionnaire (SGRQ) score and COPD assessment scoreStatus: Phase III dataMilestone: Submit regulatory application (2H17)GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) plans to submit regulatory applications this half for Nucala mepolizumab (SB-240563) to treat chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype after reporting mixed data from the double-blind, international Phase III METREX and METREO trials. The trials enrolled COPD patients at high risk of exacerbations despite the use of optimal standard of care (SOC) background therapy consisting of an inhaled corticosteroid (ICS), long-acting adrenergic receptor beta 2 agonist (LABA) and long-acting muscarinic receptor antagonist (LAMA).In the 836-patient METREX (Study 117106) trial, 100 mg subcutaneous mepolizumab every 4 weeks met the primary endpoint of reducing the mean annual rate of moderate or severe exacerbations in patients with a blood eosinophil count of >150 cells/µL at study entry or >300 cells/µL within the past year (n=462) vs. placebo (1.4 vs. 1.71 exacerbations per year, p=0.036). Mepolizumab also met the secondary endpoint of increasing the time to first moderate and severe exacerbation vs. placebo (192 vs. 141 days, p=0.04), but missed the secondary endpoints of reducing the annual rate of exacerbations requiring hospitalization, St. George’s Respiratory Questionnaire (SGRQ) score and COPD assessment score vs. placebo.In the 674-patient METREO (Study 117113) trial, 100 and 300 mg doses of mepolizumab every 4 weeks each missed the primary endpoint of reducing the mean annual rate of moderate or severe exacerbations in patients with a blood eosinophil count of >150 cells/µL at study entry or >300 cells/µL within the past year (n=674) vs. placebo (1.19 and 1.27 exacerbations per year, respectively, vs. 1.49 exacerbations per year, p=0.068 and p=0.14). Both doses of mepolizumab also missed all secondary endpoints vs. placebo.GSK said a prespecified meta-analysis of patients with an eosinophilic phenotype treated with mepolizumab in both trials showed that the rate of exacerbation reduction increased as baseline blood eosinophil counts increased, suggesting that blood eosinophil levels could be used as a biomarker to identify COPD patients most likely to respond to mepolizumab.Data were presented at the European Respiratory Society meeting in Milan and published in the New England Journal of Medicine. GSK reported preliminary data from METREX and METREO in May.GSK markets the humanized IgG mAb against IL-5 as Nucala as an add-on maintenance therapy to treat severe asthma in patients ages ≥12 with an eosinophilic phenotype in the U.S., and for severe eosinophilic asthma in the EU and Japan. An sBLA for Nucala is under FDA review to treat eosinophilic granulomatosis with polyangiitis.

Innoviva Inc. (NASDAQ:INVA), South San Francisco, Calif.GlaxoSmithKline plc (LSE:GSK; NYSE:GSK), London, U.K.

Product: Trelegy Ellipta fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI)Business: PulmonaryMolecular target: Muscarinic receptor; Adrenergic receptor beta 2 (ADRB2)Description: Fixed-dose combination of fluticasone furoate, an inhaled corticosteroid; umeclidinium bromide, a long-acting muscarinic receptor antagonist (LAMA); and vilanterol, a long-acting ADRB2 agonist (LABA)Indication: Treat chronic obstructive pulmonary disease (COPD)Endpoint: Annual rate of on-treatment moderate and severe exacerbations; change from baseline in trough forced expiratory volume in 1 second (FEV1), St. George's Respiratory Questionnaire (SGRQ) score and time to first on-treatment moderate or severe exacerbationStatus: Phase III dataMilestone: NAGlaxoSmithKline plc (LSE:GSK; NYSE:GSK) and Innoviva Inc. (NASDAQ:INVA) reported data from the Phase III IMPACT trial in 10,355 chronic obstructive pulmonary disease (COPD) patients showing that once-daily Trelegy Ellipta fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI) met the primary endpoint of reducing the annual rate of on-treatment moderate and severe exacerbations compared to both fluticasone furoate/vilanterol (0.91 vs. 1.07 exacerbations per year, p<0.001) and umeclidinium bromide/vilanterol (0.91 vs. 1.21 exacerbations per year, p<0.001). The double-blind, international trial enrolled patients with moderate to very severe symptomatic COPD and a history of exacerbation in the previous 12 months.Trelegy Ellipta also met the secondary endpoints of improving trough forced expiratory volume in 1 second (FEV1) from baseline to week 52, St. George’s Respiratory Questionnaire (SGRQ) score and time to first on-treatment moderate or severe exacerbation compared to both fluticasone furoate/vilanterol and umeclidinium bromide/vilanterol (p<0.001 for all).On Sept. 18, FDA approved an NDA for Trelegy Ellipta for maintenance treatment of COPD. GSK said Trelegy Ellipta is the first once-daily single inhaler triple therapy approved for COPD in the U.S. Trelegy Ellipta is specifically indicated for COPD patients already receiving fixed-dose Breo Ellipta fluticasone furoate/vilanterol with or without Incruse Ellipta umeclidinium. EMA's CHMP has recommended approval of Trelegy Ellipta for maintenance treatment of COPD.In 2Q18, GSK plans to begin submission of global regulatory applications for Trelegy Ellipta based on the IMPACT data.Trelegy Ellipta is a fixed-dose combination of 100 µg fluticasone furoate, an inhaled corticosteroid; 62.5 µg umeclidinium bromide, a long-acting muscarinic receptor antagonist (LAMA); and 25 µg

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vilanterol, a long-acting adrenergic receptor beta 2 agonist (LABA), delivered with GSK's Ellipta dry powder inhaler.GSK and are partnered under a deal to develop inhaled LABA products.GSK's fluticasone furoate/vilanterol is approved for COPD and asthma as Breo Ellipta in the U.S. and as Relvar Ellipta in Europe. The pharma's umeclidinium bromide/vilanterol is approved as Anoro Ellipta in the U.S. and EU for COPD.

Merck & Co. Inc. (NYSE:MRK), Kenilworth, N.J.

Product: Keytruda pembrolizumab (MK-3475)Business: CancerMolecular target: PD-1Description: Humanized IgG4 mAb against PD-1Indication: Treat unresectable or metastatic non-small cell lung cancer (NSCLC)Endpoint: Recommended Phase II dose (Phase I) and objective response rate (ORR) (Phase II); overall survival (OS), progression-free survival (PFS) and duration of responseStatus: Updated Phase I/II dataMilestone: N/AMerck & Co. Inc. (NYSE:MRK) reported updated data from 123 chemotherapy-naive patients with metastatic, non-squamous EGFR- and anaplastic lymphoma kinase (ALK)-negative non-small cell lung cancer (NSCLC) in cohort G of the Phase II portion of the Phase I/II KEYNOTE-021 trial showing that first-line treatment with 200 mg IV Keytruda pembrolizumab (MK-3475) every 3 weeks plus Alimta pemetrexed and carboplatin continued to improve progression-free survival (PFS) and overall response rate (ORR) with an additional 5 months of follow-up.In cohort G, Keytruda plus Alimta and carboplatin led to a median PFS of 19 months vs. 8.9 months for Alimta and carboplatin alone (HR=0.54, 95% CI: 0.33, 0.88, p=0.0067). Keytruda plus Alimta and carboplatin also improved the PFS rate at 12 (57% vs. 37%) and 18 (52% vs. 29%) months vs. Alimta and carboplatin alone. Keytruda plus Alimta and carboplatin led to an ORR of 56.7% vs. 31.7% for Alimta and carboplatin alone (p=0.0029). At a median follow-up of 18.7 months, median duration of response has not been reached in either arm.Median overall survival (OS) has not yet been reached in the Keytruda arm compared to 20.9 months for Alimta and carboplatin alone. Despite the trial's crossover design, Merck also said there was a trend toward improvement in OS for the Keytruda arm compared to Alimta and carboplatin alone (HR=0.59, 95% CI: 0.34, 1.05, p=0.03). In the 63 patients randomized to receive Alimta and carboplatin alone, 40 went on to receive subsequent anti-PD-1 or PD-L1 therapy, including 25 who received Keytruda as part of the study crossover. Data were presented at the European Society for Medical Oncology meeting in Madrid.

Merck has previously reported data from open-label, crossover, international KEYNOTE-021 trial (see BioCentury, May 25, 2015; May 23, 2016 & Oct. 17, 2016).Based on data from KEYNOTE-021, FDA granted accelerated approval in May to Keytruda in combination with carboplatin and Alimta as a first-line treatment of NSCLC, regardless of PD-L1 expression. Keytruda already had approval as a monotherapy for first-line treatment of NSCLC in patients with high PD-L1 expression with no EGFR or ALK genomic tumor aberrations, and for NSCLC patients whose tumors express PD-L1 with disease progression on or after platinum-containing chemotherapy (see BioCentury, May 12).The humanized IgG4 mAb against PD-1 also has full or accelerated approval to treat microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) cancer regardless of tissue of origin, melanoma, squamous cell carcinoma of the head and neck (SCCHN), classical Hodgkin's lymphoma and urothelial carcinoma.

Mirati Therapeutics Inc. (NASDAQ:MRTX), San Diego, Calif.

Product: Sitravatinib (MGCD516) (formerly MG516)Business: CancerMolecular target: Ret proto-oncogene (RET); TYRO3 protein tyrosine kinase (TYRO3; SKY)Description: Spectrum-selective kinase inhibitor that inhibits multiple receptor tyrosine kinases (RTKs) including RET, TYRO3, AXL receptor tyrosine kinase (AXL; UFO), c-Mer proto-oncogene tyrosine kinase (MERTK), VEGF receptor 2 (VEGFR-2; KDR/Flk-1) and stem cell factor (SCF) receptor tyrosine kinase (c-Kit; KIT; CD117)Indication: Treat advanced or metastatic non-squamous non-small cell lung cancer (NSCLC)Endpoint: Number of patients with tumor size reduction; safety and pharmacokineticsStatus: Phase II dataMilestone: NAMirati Therapeutics Inc. (NASDAQ:MRTX) reported preliminary data from a Phase II trial showing that sitravatinib (MGCD516) in combination with PD-1 mAb Opdivo nivolumab led to 3 confirmed partial responses in 11 evaluable patients with non-squamous non-small cell lung cancer (NSCLC) who progressed on or after treatment with checkpoint inhibitor therapy. The company said the predefined criteria for expansion from stage 1 to stage 2 of the open-label, U.S. trial, which will enroll a combined total of 34 patients, have been met. Data were presented at the International Association for the Study of Lung Cancer (IASLC) Multidisciplinary Symposium in Thoracic Oncology in Chicago.Mirati also presented a case study from a heavily pretreated NSCLC patient harboring an inactivating Cbl proto-oncogene (CBL) mutation in a Phase Ib trial evaluating sitravatinib as monotherapy. The company said the patient achieved a confirmed partial response with a maximum reduction in target lesions of 77%.

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Sitravatinib is a spectrum-selective kinase inhibitor that inhibits multiple receptor tyrosine kinases (RTKs) including Ret proto-oncogene (RET), TYRO3 protein tyrosine kinase (TYRO3; SKY), AXL receptor tyrosine kinase (AXL; UFO), c-Mer proto-oncogene tyrosine kinase (MERTK), VEGF receptor 2 (VEGFR-2; KDR/Flk-1) and stem cell factor (SCF) receptor tyrosine kinase (c-Kit; KIT; CD117).

MorphoSys AG (Xetra:MOR; Pink:MPSYF), Martinsried, GermanyJohnson & Johnson (NYSE:JNJ), New Brunswick, N.J.

Product: Tremfya guselkumab (CNTO 1959)Business: AutoimmuneMolecular target: IL-23Description: Human HuCAL mAb targeting the p19 subunit of IL-23Indication: Treat moderate to severe plaque psoriasisEndpoint: Proportion of patients with an Investigator’s Global Assessment (IGA) score of 0 or 1 point and who achieve a Psoriasis Area and Severity Index (PASI) 90 response at week 16 compared to placebo; proportion of patients with an IGA score of 0 or 1 point and PASI 90 response rates at week 16, 24 and 48 compared to Humira adalimumab and Dermatology Life Quality Index (DLQI) scoreStatus: Additional Phase III dataMilestone: NAThe Janssen Research & Development LLC unit of Johnson & Johnson (NYSE:JNJ) reported 2-year data from the open-label extension of the double-blind, international Phase III VOYAGE 1 trial of Tremfya guselkumab (CNTO 1959) showing that the drug maintained long-term efficacy in patients with moderate to severe plaque psoriasis.In the 837-patient trial, 82.1% of patients who received subcutaneous Tremfya at weeks 0 and 4 and then every 8 weeks for 100 weeks had 90% skin clearance as measured by the Psoriasis Area and Severity Index (PASI 90). Additionally, 82.4% of patients achieved an Investigator’s Global Assessment (IGA) score of cleared or minimal disease at week 100.Among patients initially randomized to receive Humira adalimumab from AbbVie Inc. (NYSE:ABBV) and then transitioned at week 52 to receive Tremfya every 8 weeks, the proportion of patients achieving PASI 90 increased from 50.5% at week 52 to 81.1% at week 100. Among this group, the proportion of patients who achieved an IGA score of cleared or minimal disease increased from 60.4% at week 52 to 84% at week 100. Janssen also said that patients initially randomized to receive placebo and crossed over to Tremfya at weeks 16 and 20 showed "consistent levels" of skin clearance at weeks 52 and 100. Data were presented at the European Academy of Dermatology and Venereology meeting in Geneva.Last year, Janssen reported that the human HuCAL mAb targeting the p19 subunit of IL-23 met VOYAGE 1's co-primary endpoints of improving PASI 90 (73.3% vs. 2.9%, p<0.001) and IGA success

(85.1% vs. 6.9%, p<0.001) response rates at week 16 vs. placebo (see BioCentury, Oct. 10, 2016).FDA approved Tremfya in July to treat moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy. An MAA for Tremfya is under EMA review with a decision expected by year end (see BioCentury, July 14).Under a 2000 deal, MorphoSys used its HuCAL human combinatorial antibody library to generate antibodies, including Tremfya, for J&J. The discovery portion of the deal expired in 2007 and MorphoSys is eligible for milestones and royalties (see BioCentury, Jan. 8, 2001).

Nabriva Therapeutics plc (NASDAQ:NBRV), Dublin, Ireland

Product: Lefamulin (BC-3781)Business: InfectiousMolecular target: Ribosomal 50S subunitDescription: Semi-synthetic derivative of the antibiotic pleuromutilin that binds the peptidyl transferase center on the ribosomeIndication: Treat moderate to severe community-acquired bacterial pneumonia (CABP)Endpoint: Early clinical response at 72-120 hours after the start of treatment; Investigator’s Assessment of Clinical Response at the test-of-cure (TOC) visit 5-10 days after the end of treatment in the modified intent-to-treat (mITT) and clinically evaluable (CE) populationsStatus: Phase III dataMilestone: Phase III data (2Q17); submit NDA (2H18)Nabriva Therapeutics plc (NASDAQ:NBRV) reported that IV-to-oral lefamulin (BC-3781) met the FDA- and EMA-defined primary endpoints in the double-blind, international Phase III LEAP 1 trial to treat moderate to severe community-acquired bacterial pneumonia (CABP). Top-line data from the Phase III LEAP 2 trial evaluating oral lefamulin to treat CABP are expected next spring, with an NDA submission to FDA to follow in 2H18. The company plans to submit an MAA to EMA “within a few months” after its NDA submission.In LEAP 1's 551-patient intent-to-treat (ITT) population, 150 mg lefamulin every 12 hours was non-inferior to moxifloxacin with or without adjunctive Zyvox linezolid on the FDA-defined primary endpoint of early clinical response at 72-120 hours after the start of treatment (87.3% vs. 90.2%). The non-inferiority margin was 12.5%. Early clinical response was defined as improvement in ≥2 of the 4 cardinal symptoms of CABP -- difficulty breathing, cough, production of purulent sputum and chest pain -- with no worsening in any of those symptoms or use of a concomitant antibiotic (other than linezolid). Investigators could switch patients to 600 mg oral lefamulin every 12 hours after 3 days of IV treatment.Lefamulin was also non-inferior to moxifloxacin with or without adjunctive linezolid on the EMA-defined primary endpoints of Investigator’s Assessment of Clinical Response at the test-of-cure (TOC) visit 5-10 days after the end of treatment in the modified

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ITT (mITT) (81.7% vs. 84.2%) and clinically evaluable (CE) (86.9% vs. 89.4%) populations. The non-inferiority margin for both EMA endpoints was 10%.Lefamulin is a semi-synthetic derivative of the antibiotic pleuromutilin that binds the peptidyl transferase center on the ribosome. The product has Qualified Infectious Disease Product (QIDP) and Fast Track designations in the U.S. to treat CABP and acute bacterial skin and skin structure infections (ABSSSIs).

Vanda Pharmaceuticals Inc. (NASDAQ:VNDA), Washington, D.C.Eli Lilly and Co. (NYSE:LLY), Indianapolis, Ind.

Product: Tradipitant (formerly LY686017, VLY-686)Business: DermatologyMolecular target: Tachykinin receptor (TACR1; NK1R)Description: TACR1 antagonistIndication: Treat chronic pruritus associated with atopic dermatitisEndpoint: Chronic pruritus as measured by itch visual analog scale (VAS) score; worst itch VAS, total Scoring Atopic Dermatitis Index (SCORAD), Clinical Global Impression of Change (CGI-C) and quality of life (QOL)Status: Phase II dataMilestone: NAVanda Pharmaceuticals Inc. (NASDAQ:VNDA) reported data from the double-blind, U.S. Phase II Study 2102 in 168 chronic pruritus patients with mild to severe atopic dermatitis showing that twice-daily 85 mg oral tradipitant for 8 weeks missed the primary endpoint of improving mean itch visual analog scale (VAS) score vs. placebo (reductions of 41.5 vs. 35.8 points, p=0.306). Tradipitant did meet the secondary endpoints of improving worst itch VAS score (reductions of 44.2 vs. 30.6 points, p=0.019), total Scoring Atopic Dermatitis Index (SCORAD) scale score (reductions of 21.3 vs. 13.6 points, p=0.008), Clinical Global Impression of Change (CGI-C) (2.6 vs. 3.3 points, p=0.007) and quality of life (QOL) as measured by the Patient Benefit Index (PBI) (p=0.038) vs. placebo.Vanda said it plans to meet with FDA "in the near future to further define and confirm the clinical development path towards registration" of tradipitant for atopic dermatitis.In 2012, Eli Lilly and Co. (NYSE:LLY) granted Vanda exclusive, worldwide rights to develop and commercialize the tachykinin receptor (TACR1; NK1R) antagonist (see BioCentury, April 23, 2012).

CLINICAL NEWS

CLINICAL STATUSFate Therapeutics Inc. (NASDAQ:FATE), San Diego, Calif.

Product: ProTmuneBusiness: TransplantMolecular Target: NA

Description: Donor-sourced mobilized peripheral blood modulated ex vivo with dexamethasone (FT4145) and 16,16-dimethyl prostaglandin E2 (PGE2) (FT1050)Indication: Prevent graft-versus-host disease (GvHD)Endpoint: Safety and incidence of grade B-D acute GvHD by Day 100 post-HCT; For CMV-positive patients, incidence of patients with CMV viremia or who initiate anti-viral therapy for CMV by Day 100 post-HCTStatus: Phase II startedMilestone: Phase I data (12/2017)Fate Therapeutics Inc. (NASDAQ:FATE) began enrollment in the double-blind Phase II portion of the U.S and European Phase I/II PROTECT trial of ProTmune to prevent acute graft-versus-host disease (GvHD) in patients with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation (HCT) in accordance with a recommendation by an independent DMC.Fate said the DMC's review showed all 7 patients who received ProTmune in the open-label Phase I portion met the day 28 safety objectives of neutrophil engraftment and survival with no graft failure events or treatment-related serious adverse events. The company said the median time to neutrophil engraftment was 18 days. The Phase II portion of the trial will enroll 60 patients to receive ProTmune or non-programmed mobilized peripheral blood cells (MPBC).The trial's primary endpoints are safety and incidence of Grade B-D acute GvHD by Day 100 post-HCT. The secondary endpoint is incidence of cytomegalovirus (CMV) viremia or initiation of CMV antiviral therapy in CMV-positive patients.Fate expects to present data from the trial's Phase I portion at the American Society of Hematology meeting in December.ProTmune comprises donor-sourced mobilized peripheral blood modulated ex vivo with dexamethasone (FT4145) and 16,16-dimethyl prostaglandin E2 (PGE2) (FT1050). It has Orphan Drug designation in the U.S. and EU and Fast Track designation in the U.S.

Madrigal Pharmaceuticals Inc. (NASDAQ:MDGL), West Conshohocken, Pa.Roche (SIX:ROG; OTCQX:RHHBY), Basel, Switzerland

Product: MGL-3196, VIA-3196Business: Endocrine/MetabolicMolecular target: Thyroid hormone receptor betaDescription: Selective agonist of thyroid hormone receptor betaIndication: Treat heterozygous familial hypercholesterolemia (HeFH)Endpoint: Mean percent change from baseline in LDL-C; mean percent change from baseline in non-HDL-C, triglycerides, lipoprotein(a) (LPA), apolipoprotein B (APOB), total cholesterol/HDL-C ratio, apolipoprotein A-1 (APOA1; apo(a))/APOB ratio and lipoprotein particle assessment and safetyStatus: Completed Phase II enrollmentMilestone: Phase II data (early 2018)

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Madrigal Pharmaceuticals Inc. (NASDAQ:MDGL) said it has enrolled 113 patients in a Phase II trial of MGL-3196 (VIA-3196) to treat heterozygous familial hypercholesterolemia (HeFH), exceeding its target enrollment of 105. The company said it would continue enrollment "for the next several days."The double-blind, placebo-controlled, European trial is evaluating MGL-3196 for 12 weeks in combination with patients' current drug regimens, including high-dose statins and/or the cholesterol absorption inhibitor Zetia ezetimibe from Merck & Co. Inc. (NYSE:MRK). The primary endpoint is the mean percent change from baseline in LDL-C. Secondary endpoints include mean percent change from baseline in non-HDL-C, triglycerides, lipoprotein(a) (LPA), apolipoprotein B (APOB), total cholesterol/HDL-C ratio, apolipoprotein A-1 (APOA1; apo(a))/APOB ratio and lipoprotein particle assessment and safety.Madrigal also said an independent DSMB recommended the trial, as well as another Phase II trial of MGL-3196 in non-alcoholic steatohepatitis (NASH), continue without modification based on its second review.Madrigal has exclusive, worldwide rights to MGL-3196 from Roche under a 2008 deal. The company expects top-line data from the HeFH trial in early 2018.

Novartis AG (NYSE:NVS; SIX:NOVN), Basel, SwitzerlandMedicines for Malaria Venture, Geneva, Switzerland

Product: KAF156Business: InfectiousMolecular target: NADescription: Imidazolopiperazine derivativeIndication: Treat malariaEndpoint: PCR-corrected adequate clinical and parasitological response (ACPR) at 28 days post-dose; safety, PCR-uncorrected and-corrected ACPR at 14 and 42 post-dose, PCR-uncorrected ACPR at 28 days post-dose, incidence of recrudescence and reinfection at 14, 28 and 42 days post-dose, parasite and fever clearance times, proportion of patients with parasitemia 12, 24 and 48 hours after treatment and pharmacokineticsStatus: Phase IIb startedMilestone: NANovartis AG (NYSE:NVS; SIX:NOVN) and Medicines for Malaria Venture (Geneva, Switzerland) began a Phase IIb trial of KAF156 plus a "new, improved formulation" of the antimalarial lumefantrine to treat malaria.The single-blind, African and Asian trial will enroll about 512 adults, adolescents and children to receive Novartis' Coartem artemether/lumefantrine or lumefantrine in combination with a single dose of 200, 400 or 800 mg KAF156; 400 mg KAF156 once daily for 2 days; or 200 or 400 mg KAF156 once daily for 3 days. The primary endpoint is PCR-corrected adequate clinical and parasitological response (ACPR) at 28 days post-dose. Secondary endpoints include safety, PCR-uncorrected and -corrected ACPR

at 14 and 42 post-dose, PCR-uncorrected ACPR at 28 days post-dose, incidence of recrudescence and reinfection at 14, 28 and 42 days post-dose, parasite and fever clearance times, proportion of patients with parasitemia 12, 24 and 48 hours after treatment and pharmacokinetics.KAF156 is an imidazolopiperazine derivative. Coartem is a fixed-dose artemisinin-based combination treatment.

Oncolytics Biotech Inc. (TSX:ONC; OTCQX:ONCYF), Calgary, Alberta

Product: Reolysin pelareorepBusiness: CancerMolecular target: NADescription: Formulation of human reovirus type 3, an oncolytic virusIndication: Treat relapsed or refractory multiple myeloma (MM)Endpoint: Dose-limiting toxicities (DLTs) and maximum tolerated dose (MTD); safety, response rate, maximum response rate within 6 cycles of therapy, time to maximum response, progression-free survival (PFS) and overall survival (OS)Status: Phase Ib startedMilestone: Preliminary Phase Ib data (1Q18)Oncolytics Biotech Inc. (TSX:ONC; OTCQX:ONCYF) said the first patient has been dosed in the Phase Ib MUK eleven trial of Reolysin pelareorep in combination with Revlimid lenalidomide or Imnovid pomalidomide from Celgene Corp. (NASDAQ:CELG) as a rescue treatment in relapsing or refractory multiple myeloma (MM) patients who are progressing despite treatment with 1 of those immunomodulatory agents (IMiDs).The open-label, dose-escalation, U.K. trial is evaluating IV Reolysin on days 1, 8, 15 and 22 of each 28-day cycle at a starting dose of 3x1010 50% tissue culture infective dose (TCID50) in about 44 patients. The primary endpoints are dose-limiting toxicities (DLTs) and maximum tolerated dose (MTD). Secondary endpoints include safety, response rate, maximum response rate within 6 cycles of therapy, time to maximum response, progression-free survival (PFS) and overall survival (OS).Reolysin is a formulation of human reovirus type 3, an oncolytic virus.Preliminary data from the trial are expected in 1Q18.In March, Myeloma UK partnered with Oncolytics and Celgene to conduct the trial (see BioCentury, March 24).

ViroMed Co. Ltd. (KOSDAQ:084990), Seoul, South Korea

Product: VM202Business: DermatologyMolecular target: NADescription: Plasmid DNA carrying the hepatocyte growth factor (HGF) geneIndication: Treat non-healing diabetic foot ulcers and concomitant peripheral artery disease (PAD)

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Endpoint: Proportion of patients with confirmed target wound closure by the 4-month follow-up; changes in ankle-brachial index and toe-brachial index, time to complete wound closure, proportion of patients with confirmed target wound closure at ≤7 months, percent change in wound volume, area and depth and proportion of patients with new ulcer formation on the target foot at 2, 3, 4 and 7 months, time to major and minor amputations and change in each Cardiff Wound Impact Questionnaire (CWIQ) domain score from baseline to months 4 and 7Status: Phase III startedMilestone: NAVM BioPharma, the U.S. division of ViroMed Co. Ltd. (KOSDAQ:084990), began a Phase III trial of VM202 to treat non-healing diabetic foot ulcers and concomitant peripheral artery disease (PAD). The double-blind, placebo-controlled, U.S. study will enroll 300 patients. The primary endpoint is the proportion of patients with confirmed target wound closure by the 4-month follow-up. Secondary endpoints include changes in ankle-brachial index and toe-brachial index; time to complete wound closure; proportion of patients with confirmed target wound closure at ≤7 months; percent change in wound volume, area and depth and proportion of patients with new ulcer formation on the target foot at 2, 3, 4 and 7 months; time to major and minor amputations and change in each Cardiff Wound Impact Questionnaire (CWIQ) domain score from baseline to months 4 and 7.The candidate is a plasmid DNA carrying the hepatocyte growth factor (HGF) gene.

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COMPLETED OFFERINGSAbClon Inc. (KOSDAQ:174900), Seoul, South Korea

On Sept. 18, AbClon Inc. (KOSDAQ:174900) raised W6.8 billion ($6.1 million) through the sale of 680,741 shares at W10,000 in an IPO.Director Kyu-Tae Kim told BioCentury that AbClon's lead candidate is AC101, a mAb targeting epidermal growth factor (EGF) that is in preclinical testing to treat gastric and breast cancer. In 2016, AbClon granted Shanghai Henlius Biotech Inc. (Shanghai, China), a JV between Shanghai Fosun Pharmaceutical Group Co. Ltd. (Shanghai:600196; HKSE:2196) and Henlius Biotech Co. Ltd. (Shanghai, China), exclusive, Chinese rights to AC101.

Acceleron Pharma Inc. (NASDAQ:XLRN), Cambridge, Mass.

On Sept. 20, Acceleron Pharma Inc. (NASDAQ:XLRN) raised $200 million through the sale of 5.4 million shares at $37 in a bumped-up follow-on underwritten by JPMorgan, Citigroup and Leerink Partners. The price is a 5% discount to Acceleron's close of $38.97 on Sept. 19, when it proposed after trading hours to raise $150 million.Acceleron expects top-line data in mid-2018 from Phase III studies of lead candidate luspatercept (ACE-536) to treat myelodysplastic syndromes (MDS) and beta-thalassemia. Celgene Corp. (NASDAQ:CELG) shares rights to luspatercept, a modified activin receptor type 2B (ACVR2B) fusion protein that inhibits several ligands in the TGF beta superfamily.On Sept. 18, Acceleron gained worldwide rights to develop and commercialize sotatercept to treat pulmonary hypertension, amending a deal with Celgene that originally granted Acceleron co-development rights. Acceleron expects to begin a Phase II trial of the candidate to treat pulmonary arterial hypertension (PAH) in 1H18. Sotatercept is a soluble ACVR2A fusion protein linked to the Fc protein of human IgGI.

Aldeyra Therapeutics Inc. (NASDAQ:ALDX), Lexington, Mass.

Aldeyra Therapeutics Inc. (NASDAQ:ALDX), which focuses on diseases related to aldehydes, raised $28.8 million through the sale of 4 million shares at $7.25 in a follow-on underwritten by Cantor Fitzgerald and Laidlaw. The price is a 20% discount to Aldeyra’s close of $9.10 on Sept. 18 when it proposed the offering after market hours. The figures include the sale of a 517,500 overallotment on Sept. 21.Earlier this month, the company reported data from a Phase IIa trial showing that ADX-102 ophthalmic solution significantly improved multiple signs and symptoms of dry eye disease. The product is a topical eye drop formulation of a small molecule that covalently binds and traps free aldehydes (see BioCentury, Sept. 14).

Array BioPharma Inc. (NASDAQ:ARRY), Boulder, Colo.Marinus Pharmaceuticals Inc. (NASDAQ:MRNS), Radnor, Pa.T2 Biosystems Inc. (NASDAQ:TTOO), Lexington, Mass.

Cancer company Array BioPharma Inc. (NASDAQ:ARRY), neurology company Marinus Pharmaceuticals Inc. (NASDAQ:MRNS), and diagnostics play T2 Biosystems Inc. (NASDAQ:TTOO) each priced follow-on offerings, raising more than $270 million.Array raised $258.8 million through the sale of 24.1 million shares at $10.75 in a bumped-up follow-on underwritten by JPMorgan, Cowen, Piper Jaffray, Stifel, Wells Fargo and SunTrust Robinson Humphrey. The price is a 2% increase to Array's close of $10.53 on Sept. 13 when it proposed the offering after trading hours. The figures include the sale of a 3.1 million share overallotment on Sept. 19.On Sept. 12, FDA accepted for review NDAs for binimetinib and encorafenib in combination to treat BRAF-mutant advanced, unresectable or metastatic melanoma. Binimetinib is a small molecule selective MAP kinase kinase 1 (MAP2K1; MEK1) and MEK2 inhibitor, and encorafenib is a small molecule BRAF inhibitor.Marinus raised $40.3 million through the sale of 10.7 million shares at $3.75 in a follow-on underwritten by JMP Securities. The price is a 6% discount to Marinus' close of $4.01 on Sept. 14 when it proposed the offering after trading hours. The figures include the sale of a 1.4 million share overallotment on Sept. 19.On Sept. 11, Marinus reported top-line data from the intent-to-treat population of a Phase II study in children with cyclin dependent kinase like 5 (CDKL5) epilepsy showing that oral ganaxolone (CCD-1042) reduced median 28-day seizure frequency by 43% from baseline, the trial's primary endpoint. Ganaxolone is a synthetic analog of the endogenous neurosteroid allopregnanolone, a GABA A receptor neuromodulator.T2 raised $17.5 million on Sept. 15 through the sale of 4.4 million shares at $4 in a follow-on underwritten by Canaccord Genuity, Cantor Fitzgerald, Janney Montgomery Scott, JonesTrading Services and WBB Securities. The price is a 34% discount to T2's close of $6.08 on Sept. 14 when it proposed the offering after trading hours. The company received CE Mark approval in July for its T2Bacteria panel, which identifies six sepsis-causing bacteria from a blood sample.

BiondVax Pharmaceuticals Ltd. (Tel Aviv:BVXV; NASDAQ:BVXV), Ness Ziona, Israel

Flu vaccine company BiondVax Pharmaceuticals Ltd. (Tel Aviv:BVXV; NASDAQ:BVXV) raised $10 million through the sale of 1.7 million American Depositary Shares (ADS) at $6 in a follow-on underwritten by Joseph Gunnar. Each ADS represents 40 ordinary shares. The figures include the sale of a 166,667 share overallotment on Sept. 18.

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Celcuity Inc. (NASDAQ:CELC), Minneapolis, Minn.

On Sept. 20, cancer diagnostic company Celcuity Inc. (NASDAQ:CELC) raised $22.8 million through the sale of 2.4 million shares at $9.50 in an IPO underwritten by Craig-Hallum Capital.

Cofactor Genomics Inc., San Francisco, Calif.

On Sept. 18, precision medicine company Cofactor Genomics Inc. (San Francisco, Calif.) raised $18 million in an oversubscribed series A round led by Menlo Ventures. Data Collective, Ascension Ventures, iSelect, Y Combinator, Wilson Sonsini Goodrich & Rosati, and Stanford also participated.Cofactor Genomics' CLIA-validated Pinnacle assay measures the expression of nearly 600 RNA biomarkers known to be associated with response to targeted therapeutics. The company's Paragon test is an RNA-based immunophenotyping assay that profiles the cellular components of the tumor microenvironment.Menlo's Mark Siegel, Data Collective's Matt Ocko and Ascension Venture's John Kuelper joined Cofactor Genomics' board.

Disarm Therapeutics Inc., Cambridge, Mass.

On Sept. 19, Disarm Therapeutics Inc. (Cambridge, Mass.) raised $30 million in a series A round led by existing investor Atlas Venture. New investors Lightstone Ventures and AbbVie Ventures also participated.Disarm is developing small molecule inhibitors of sterile alpha and TIR motif containing 1 (SARM1) to prevent axonal degeneration in a range of neurological disorders, including multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), glaucoma and peripheral neuropathies.Chairman and acting CEO Jason Rhodes said the series A will enable Disarm to bring at least one SARM1 inhibitor to the clinic "in a few years."Rhodes, who is also a partner at Atlas, co-founded Disarm in 2016 with CSO Rajesh Devraj and Washington University in St. Louis professors Jeffrey Milbrandt and Aaron DiAntonio. The company exclusively licensed IP covering its SARM1 inhibitors from Washington University for undisclosed terms.

DNA Script, Paris, France

On Sept. 12, synthetic DNA manufacturer DNA Script (Paris, France) said it raised €11 million ($13 million) in a series A round led by Illumina Ventures. Merck Ventures BV participated along with existing investors Sofinnova Partners, Kurma Partners and Idinvest Partners.DNA Script spokesperson Justin Jackson told BioCentury the company will use the funding, which it expects to cover the next two years, to develop its lab-based enzymatic technology and nucleotide chemistry platform for industrial scale-up.

DNA Script raised €2.5 million ($2.7 million) in 2016 in a seed funding round led by Sofinnova Partners.

Ennovabio, Shanghai, China

Oncology company Ennovabio (Shanghai, China) said it closed a pre-series A round, led by HighLight Capital with participation from Shanghai Zhangjiang Leading Venture Capital Co. Ltd.Ennovabio's founder Lei Jiang declined to disclose financial details, but said the company expects to raise a series A in mid- to late-2018.

Gilead Sciences Inc. (NASDAQ:GILD), Foster City, Calif.

On Sept. 14, Gilead Sciences Inc. (NASDAQ:GILD) raised $3 billion in an offering of senior unsecured notes underwritten by Merrill Lynch, Pierce, Fenner & Smith and Wells Fargo. The biotech will use the funds for its acquisition of Kite Pharma Inc. (NASDAQ:KITE).In August, Gilead agreed to acquire Kite at $180 per share in cash, which the companies say values Kite at about $11.9 billion. Gilead said it would finance the deal with a combination of cash, bank debt and senior unsecured notes. The deal, which has been approved by both companies' boards, is expected to close next quarter (see BioCentury, Sept. 1).The offering comprises $1 billion in notes that bear interest at 1.85%; the remainder have floating rates.Kite's lead chimeric antigen receptor T cell (CAR T) therapy axicabtagene ciloleucel (KTE-C19) is under FDA Priority Review to treat refractory aggressive non-Hodgkin's lymphoma (NHL), with a PDUFA date of Nov. 29. The therapy comprises autologous T cells genetically modified to express a CAR targeting CD19.

Iovance Biotherapeutics Inc. (NASDAQ:IOVA), San Carlos, Calif.

On Sept. 20, Iovance Biotherapeutics Inc. (NASDAQ:IOVA) raised $50 million through the sale of 7.7 million shares at $6.50 in a follow-on underwritten by Jefferies, Wells Fargo, Oppenheimer, H.C. Wainwright and Chardan. The price is a 24% discount to Iovance's close of $8.55 on Sept. 19, when it proposed the offering after market hours.The biotech's lead compound, LN-144, is an autologous T cell therapy that utilizes tumor infiltrating lymphocytes (TILs) derived from the patient's tumor. The candidate is in Phase II testing to treat metastatic melanoma.

Kaleido Biosciences Inc., Bedford, Mass.

On Sept. 18, Flagship Pioneering announced Kaleido Biosciences Inc. (Bedford, Mass.), a biopharmaceuticals company that uses its chemistry platform to discover and develop products that modulate the metabolic profile of the microbiome in order to treat and prevent severe diseases. Kaleido has been operating in stealth since it was founded by the life science venture firm in 2015.Geoffrey von Maltzahn, partner at Flagship Pioneering, said Kaleido has raised an undisclosed funding round. Kaleido also named Mike Bonney CEO and chairman. He was CEO of Cubist Pharmaceuticals Inc., which Merck & Co. Inc. (NYSE:MRK) acquired.

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Maltzahn and Bonney declined to disclose financial details, but said the latest round brings Kaleido's total amount raised to $65 million. Flagship led the latest round with participation from several undisclosed investors, including Bonney. Maltzahn and Bonney said the latest funds will be used to advance Kaleido's pipeline into additional human studies.They added that Kaleido is planning an additional round of funding "soon."The company's pipeline includes clinical-stage compounds for rare genetic disorders, metabolic disease, oncology and infectious disease, with preclinical efficacy demonstrated in five other indications. It has three lead products in first-in-human studies.

Krystal Biotech Inc. (NASDAQ:KRYS), Pittsburgh, Pa.

Krystal Biotech Inc. (NASDAQ:KRYS) raised $39.6 million after market close Sept. 19 through the sale of 4 million shares at $10 in an IPO underwritten by Ladenburg Thalmann. Shares began trading on Sept. 20.The company's lead product, KB103, is in preclinical testing to treat dystrophic epidermolysis bullosa. In 1Q18, Krystal plans to submit an IND to FDA for KB103, which is a replication-defective, non-integrating viral vector that delivers functional human collagen type VII (COL7A1) genes directly to the patients' dividing and non-dividing skin cells.

Landos Biopharma Inc., Blacksburg, Va.

On Sept. 21, Landos Biopharma Inc. (Blacksburg, Va.) spun out of life sciences accelerator Xontogeny LLC (Boston, Mass) with the first $5 million tranche of a $10 million series A round from Perceptive Advisors.CEO Josep Bassaganya-Riera said the final $5 million tranche will be triggered after the company files an IND for lead candidate BT-11 next year.BT-11 is an oral LanC like 2 (LANCL2) inhibitor in preclinical development to treat Crohn's disease and ulcerative colitis.Bassaganya-Riera said the A round will enable Landos to complete Phase I testing of BT-11 for Crohn's disease by early 2019.Landos is the first newco to spin out of Xontogeny, which closed a $15 million tranche of a $25 million series A from Perceptive in May (see BioCentury, May 19).Xontogeny's other portfolio companies are pursuing preclinical candidates in oncology, inflammation, infectious disease and cardiovascular disease. CEO Christopher Garabedian declined to disclose the number of companies in the accelerator. Garabedian joined Landos’ board.

LifeMine Therapeutics Inc., Cambridge, Mass.

On Sept. 18, LifeMine Therapeutics Inc. (Cambridge, Mass.) raised $55 million in a series A round led by WuXi Healthcare Ventures. Foresite Capital, GV, Arch Ventures Partners, Boyu Capital, Blue

Pool Capital, Merck & Co. Inc. (NYSE:MRK) and Alexandria Venture Investments also participated.The company's drug discovery platform combines genomics with artificial intelligence and synthetic biology to identify small molecules from fungi. LifeMine told BioCentury the company plans to use the funds to build out its drug discovery engine and identify the first targets, with plans to enter the clinic within the next four years. The company added that its goal is to focus on oncology "for the foreseeable future."In conjunction with the series A, WuXi's Edward Hu, Foresite's James Tananbaum, GV's Krishna Yeshwant and Boyu's Yanling Cao joined LifeMine's board. Richard Klausner, who is founder and director of Juno Therapeutics Inc. (NASDAQ:JUNO) and Grail Inc. (Menlo Park, Calif.), also joined the company's board and will serve as its chairman.

MediWound Ltd. (NASDAQ:MDWD), Yavne, Israel

On Sept. 19, dermatology company MediWound Ltd. (NASDAQ:MDWD) raised $22 million through the sale of 4.4 million shares at $5 in a follow-on underwritten by Cowen, Wells Fargo, Oppenheimer, SunTrust Robinson Humphrey and Aegis Capital.

Nabriva Therapeutics plc (NASDAQ:NBRV), Dublin, Ireland

Nabriva Therapeutics plc (NASDAQ:NBRV) raised $80 million on Sept. 19 through the sale of 9.4 million shares at $8.50 in a follow-on underwritten by Morgan Stanley, BofA Merrill Lynch, SunTrust Robinson Humphrey, Needham and Wedbush. The price is a 4% discount to Nabriva's close of $8.87 on Sept. 18 when it proposed the offering after market hours.On Sept. 18, Nabriva gained 29% after reporting that IV-to-oral lefamulin (BC-3781) met the primary endpoints in the Phase III LEAP 1 trial to treat moderate to severe community-acquired bacterial pneumonia (CABP). Top-line data from the Phase III LEAP 2 trial evaluating oral lefamulin to treat CABP are expected next spring, with an NDA submission to FDA to follow in 2H18. Lefamulin is a semi-synthetic derivative of the antibiotic pleuromutilin that binds the peptidyl transferase center on the ribosome.

Realm Therapeutics plc (LSE:RLM), Malvern, Pa.

On Sept. 21, dermatology and ophthalmology company Realm Therapeutics plc (LSE:RLM) raised £19.3 million ($26.2 million) through the sale of 66.4 million units at 29p in a private placement to new and existing investors. New investors included OrbiMed, BVF Partners, RA Capital Management, Abingworth and Polar Capital. CEO Alex Martin and Charles Spicer, who is non-executive chairman of Realm, also participated in the placement. Each unit

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comprises a share and a two-and-a-half year warrant to purchase 0.4 shares, with each whole warrant exercisable at 58p.By year end, Realm plans to start a pair of Phase II trials evaluating PR022 to treat atopic dermatitis and PR013 to treat allergic conjunctivitis, respectively. Top-line data from both trials are expected in mid-2018. PR022 and PR013 are topical gel and topical ophthalmic solution formulations, respectively, containing a high concentration of hypochlorous acid.

Rodin Therapeutics Inc., Cambridge, Mass.

On Sept. 18, Rodin Therapeutics Inc. (Cambridge, Mass.) raised $27 million in a tranched series B round from existing investor Atlas Venture and new investors GV, Hatteras Venture Partners, Remeditex Ventures and Third Point Ventures. Details regarding the tranches are not disclosed.Rodin is developing selective HDAC2 inhibitors to treat neurological disorders. The company plans to select a lead candidate this year and bring it into the clinic in 2018 to treat cognitive impairment in Alzheimer's disease (AD).In late 2016, Rodin and Biogen Inc. (NASDAQ:BIIB) mutually terminated a collaboration and option agreement. Under a deal in January of that year, Rodin and Biogen had partnered exclusively to research neuronal epigenetics and develop HDAC2 inhibitors for neurology indications, and Biogen held an option to acquire Rodin for up to $485 million in upfront and milestone payments.President and CEO Adam Rosenberg said Rodin did not receive any upfront or milestone payments from Biogen before the deal was terminated."We think Biogen could certainly be a natural partner for this going forward, but at this point, we want to take on the translational work ourselves," Rosenberg told BioCentury.He said it made sense for both companies to end the deal, but declined to elaborate.Rodin raised $17.3 million in a series A extension round from Atlas Venture and Biogen last year, and $12.9 million in a series A round from Atlas and Johnson & Johnson Development Corp. (JJDC) in 2014.In conjunction with the series B, Hatteras' Clay Thorp, Remeditex's John Creecy and Third Point's Jason Hong joined Rodin's board.

XW Laboratories Inc., Wuhan, China

On Sept. 20, XW Laboratories Inc. (Wuhan, China) said it raised $17.5 million in a series B round led by Elements Capital and WI Harper. New Korean investor KTB Network and all existing investors comprised of Kleiner Perkins Caufield & Byers China, Johnson & Johnson Innovation - JJDC Inc., and WuXi Venture participated.XW Labs said it has raised a total of $23 million between the series B and a 2014 series A led by KPCB China.

The company said at least one of its programs will enter the clinic next year.In March, the University of Pittsburgh granted XW Labs an exclusive license to a class of novel mitochondria targeted bis-nitroxide compounds, with potential opportunities in CNS diseases and cancer indications. XW is also developing existing drugs for new indications using technologies which "improve the pharmacokinetics and safety profiles of existing drugs" (see BioCentury, March 24).

Zai Lab Ltd. (NASDAQ:ZLAB), Shanghai, China

On Sept. 20, Zai Lab Ltd. (NASDAQ:ZLAB) raised $150 million through the sale of 8.3 million American Depository Shares (ADSs) at $18 per share in an upsized IPO on NASDAQ underwritten by JPMorgan, Citigroup and Leerink Partners. The price values the Shanghai cancer, autoimmune and infectious disease company at $888.9 million. Zai had previously proposed to sell 5.9 million shares at $16-$18 per share, and subsequently increased the number of shares to 8 million.Zai launched in 2014 to in-license therapeutics that target diseases prevalent in China and greater Asia. The company has a pipeline of five clinical candidates in-licensed from Western biopharma companies, the most advanced of which is niraparib. This year, the PARP inhibitor is expected to start a Chinese Phase III trial to treat ovarian cancer. Zai has exclusive Chinese rights to the molecule from Tesaro Inc. (NASDAQ:TSRO), which markets the drug as Zejula in the U.S.Zai has increasingly focused on internal discovery and development of biologics for oncology and autoimmune disease. In 2015, Zai opened an R&D center in Shanghai and announced a discovery and development partnership with Tsinghua University Immunology Institute.Last year, Zai expanded its business model further when it decided to use some of its VC-raised cash to invest in other life science companies (see BioCentury, Dec. 14, 2016).Prior to going public, Zai raised $164.5 million in three venture rounds, including $30 million in a series C round in June led by existing investor OrbiMed Advisors. The crossover round included participation from new investors Vivo Capital, Cormorant Asset Management and Rock Springs Capital.Zai is led by Chairman and CEO Samantha Du, who was managing director for Sequoia Capital China, where she was responsible for the firm’s healthcare investments in China. Before that, she was founder and CEO of Hutchison MediPharma Ltd., one of China’s first innovator biotechs and a subsidiary of Hutchison China MediTech Ltd. (LSE:HCM; NASDAQ:HCM).

FINANCIAL NEWS

OTHER FINANCIAL NEWSFlagship Pioneering, Cambridge, Mass.

Life science venture firm Flagship Pioneering set a $500 million

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20 WEEK OF SEPTEMBER 25, 2017

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target for its Flagship Pioneering Fund VI, according to an SEC filing on Sept. 14. The firm declined to disclose details.Flagship closed its fifth fund at $537 million in 2015.The firm's portfolio includes gene editing play Editas Medicine Inc. (NASDAQ:EDIT), mRNA therapeutics company Moderna Therapeutics Inc. (Cambridge, Mass.) and microbiome companies Seres Therapeutics Inc. (NASDAQ:MCRB) and Kaleido Biosciences Inc. (Bedford, Mass.).

Lightstone Ventures, Menlo Park, Calif.

On Sept. 20, Lightstone Ventures (Menlo Park, Calif.) closed Lightstone Ventures II at $250 million. Lightstone said the fund will invest $15-$20 million in each of about 15 early stage biotech and medical device companies. The fund has invested in one undisclosed company and participated in a $30 million series A round for Disarm Therapeutics Inc. (Cambridge, Mass.).In 2014, Lightstone closed its first fund at $172 million with investments including rare disease and lipid disorders company Catabasis Pharmaceuticals Inc. (NASDAQ:CATB), neurological diseases company Flex Pharma Inc. (NASDAQ:FLKS) and peptide therapeutics company Ra Pharmaceuticals Inc. (NASDAQ:RARX). Last year, the firm closed Lightstone Singapore, a $50 million fund to invest in and create Singapore-based life sciences companies (see BioCentury, May 5, 2014 & Feb. 22, 2016).

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21 WEEK OF SEPTEMBER 25, 2017

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