asia pacific’s leader in deployment of ... - cell therapies · essential infrastructure enabling...
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CONFIDENTIAL to Cell Therapies Pty Ltd Ground Floor, 10 St Andrews Place, East Melbourne, VIC, Australia T: +61 3 9656 5804 www.celltherapies.com.au Disclaimer: This document is a Small Scale Offer Document and is intended for sophisticated investors only (v10 11 Nov 2015)
Asia Pacific’s leader in deployment of cellular therapies, the
essential infrastructure enabling a new industry
Cell Therapies Pty Ltd (“CTPL”) Investment Highlights
Potential to create >A$100m revenue Asia Pacific centre of excellence in cell therapies
Need A$25m capital: $2m rights issue now; Series A $5-7m H1 2016; balance 2017
High growth cellular therapies market: the emerging fourth pillar of modern medicine
Growth from risk-reward projects (commercial manufacturing and in-licensing); facilities underwritten by fee-for-service contract manufacturing
Advanced pipeline of projects in immunotherapy, gene therapy, regen medicine
Pan-Asia facility network well advanced (Australia, Japan, Malaysia)
Unique ‘needle-to-needle’ small batch manufacturing and distribution capability: essential enabling infrastructure for the industry; CTPL’s competitive advantage
Established regional leader: internationally experienced/recognised team; world class clinical, research linkages; 12 years’ experience in TGA regulated cGMP supply
Attractive risk profile: portfolio of projects (uncorrelated risk); limited exposure to product development risk; multiple plays on breakthrough technology such as CAR-T
The market opportunity
Cellular therapies utilise stem cells and the body’s latent ability to repair itself to treat and even cure diseases and injuries largely untreatable by current medicine. Potential healthcare savings in the US alone from regenerative medicine treatments exceed $250b per year.1 Sector profile in 2014:
517 companies developing therapeutic products
$6.3b in capital raised (all sources)
66 approved products, 375 clinical trials
Breakthrough results in gene and immunotherapy
Needle-to-needle manufacturing/delivery: CTPL’s unique expertise Cellular therapies require a new paradigm for product delivery:
cGMP process control from cell collection at patient/donor through manipulation to delivery back to patient for transplant
Short shelf-life (often <48h) and patient specific preparation = scheduling, logistics and cryopreservation challenges
Small batch size, even for allogeneic products = scale-out rather than scale-up challenges and bespoke automation
CTPL is the essential infrastructure required for product developers to reach the market
“At its core, manufacturing will be a differentiating factor” Jason Kolbert, Maxim Group LLC at Phacilitate, Washington 2014
Needle-to-needle cGMP manufacturing +
Location close to 650m patients + = CTPL’s significant competitive advantage
World class clinical & research infrastructure
1 Alliance for Regenerative Medicine Annual Report 2014 and State of the Industry Report 2015
CONFIDENTIAL to Cell Therapies Pty Ltd Ground Floor, 10 St Andrews Place, East Melbourne, VIC, Australia T: +61 3 9656 5804 www.celltherapies.com.au Disclaimer: This document is a Small Scale Offer Document and is intended for sophisticated investors only (v10 11 Nov 2015)
CTPL is ideally positioned to participate
12 years’ experience distributing and manufacturing cellular therapies
Delivered Orthogen’s Cartogen® first generation ACI product for 10 years in Australia
cGMP and PIC/S compliant facility at Peter MacCallum Cancer Centre (“PMCC”), Melbourne, Australia (operating lease to CTPL)
Sterling quality track record: 18 TGA quality audits in the past 12 years
Global reputation and impact, deep regional experience: 7 manufacturing processes transferred to US, EU and Japan for late stage clinical trials; brought facilities to cGMP in Japan, Malaysia and Indonesia
Synergistic relationship with Founding Partner PMCC’s clinical, research organisations
Process development IP and know-how includes quality systems, cell tracking, essential participant in CRC for Cellular Therapy Manufacturing
Deep and broad experience across cell types and manipulations
Robust growth pipeline supports expansion to ASEAN and beyond Portfolio of haem/oncology and musculo-skeletal risk-reward projects at advanced stages of contracting; potential for >$100m sales in 2020 in ANZ/ASEAN; limited pre-approval investment
In-licensing: DMY-03 (gene therapy); BEA-01 (immunotherapy for melanoma); FIG-01 (knee cartilage repair in OA), KSJ-01 (traumatic cartilage injury), GRO-01 (urinary incontinence), BMI-01 (osteonecrosis and non-union fractures)
Commercial manufacturing (build network for share of future revenue): JET-01 (immunotherapy renal cancer); MIC-04 (CAR-T); DMY-04 (gene therapy EU/Asia)
Robust pipeline of further projects under CDA
10/30/13 Leukemia Patients Remain in Remission More Than Two Years After Receiving Genetically Engineered T Cell Therapy
www.uphs.upenn.edu/news/news_releases/2012/12/tcell/print.html 1/2
December 9, 2012
CONTACT:
Holly Auer215-349-[email protected]
This release is available online athttp://www.uphs.upenn.edu/news/News_Releases/2012/12/tcell/
Leukemia Patients Remain in Remission More Than Two Years
After Receiving Genetically Engineered T Cell Therapy
University of Pennsylvania Researchers Report on Results of Trial in 12 Patients, Including TwoChildren
ATLANTA — Nine of twelve leukemia patients who received infusions of their own T cells after the cells had beengenetically engineered to attack the patients’ tumors responded to the therapy, which was pioneered by scientistsin the Perelman School of Medicine at the University of Pennsylvania. Penn Medicine researchers will presentthe latest results of the trial today at the American Society of Hematology’s Annual Meeting and Exposition.
The clinical trial participants, all of whom had advanced cancers, included 10 adult patients with chroniclymphocytic leukemia treated at the Hospital of the University of Pennsylvania (HUP) and two children withacute lymphoblastic leukemia treated at the Children’s Hospital of Philadelphia. Two of the first three patientstreated with the protocol at HUP – whose cases were detailed in the New England Journal of Medicine andScience Translational Medicine in August 2011 – remain healthy and in full remissions more than two years aftertheir treatment, with the engineered cells still circulating in their bodies. The findings reveal the first successfuland sustained demonstration of the use of gene transfer therapy to turn the body’s own immune cells intoweapons aimed at cancerous tumors.
“Our results show that chimeric antigen receptor modified T cells have great promise to improve the treatment ofleukemia and lymphoma,” says the trial’s leader, Carl June, MD, the Richard W. Vague Professor inImmunotherapy in the department of Pathology and Laboratory Medicine and director of Translational Researchin Penn’s Abramson Cancer Center. “It is possible that in the future, this approach may reduce or replace theneed for bone marrow transplantation.”
The results pave the way for a potential paradigm shift in the treatment of these types of blood cancers, which inadvanced stages have the possibility of a cure only with bone marrow transplants. That procedure requires alengthy hospitalization and carries at least a 20 percent mortality risk -- and even then offers only a limitedchance of cure for patients whose disease has not responded to other treatments.
Three abstracts about the new research will be presented during the ASH meeting. David Porter, MD, director ofBlood and Marrow Transplantation in the Abramson Cancer Center, will give an oral presentation of Abstract#717 on Monday, Dec. 10, at 5 PM in the Thomas Murphy Ballroom 4, Level 5, Building B of the Georgia WorldCongress Center. Michael Kalos, PhD, director of the Translational and Correlative Studies Laboratory at Penn,will give an oral presentation on Abstract #756 on Monday, Dec. 10, at 5:45 PM in C208-C210, Level 2, BuildingC. Stephan Grupp, MD, PhD, director of Translational Research in the Center for Childhood Cancer Research atthe Children's Hospital of Philadelphia, will present a poster of Abstract #2604 on Sunday, Dec. 9, at 6 PM in HallB1-B2, Level 1, Building B.
The protocol for the new treatment involves removing patients' cells through an apheresis process similar toblood donation, and modifying them in Penn's cell and vaccine production facility. Scientists there reprogram thepatients’ T cells to target tumor cells through a gene modification technique using a HIV-derived lentivirus vector.The vector encodes an antibody-like protein, called a chimeric antigen receptor (CAR), which is expressed on thesurface of the T cells and designed to bind to a protein called CD19.
The modified cells are then infused back into the patient's body following lymphodepleting chemotherapy. Oncethe T cells start expressing the CAR, they focus all of their killing activity on cells that express CD19, whichincludes CLL and ALL tumor cells, and normal B cells. All of the other cells in the patient that do not expressCD19 are ignored by the modified T cells, which limits systemic side effects typically experienced during
Vision: essential enabler of clinical and commercial scale cellular therapy manufacturing and deployment in Asia Pacific
Melbourne - home base
Adelaide - Research and translation
Japan – Strategic AllianceMalaysia -
“Autologous Production for the
Future”
Reach of managed apheresis/tissue collection site network
“Manufacturing will be a competitive advantage”Jason Kolbert
CONFIDENTIAL to Cell Therapies Pty Ltd Ground Floor, 10 St Andrews Place, East Melbourne, VIC, Australia T: +61 3 9656 5804 www.celltherapies.com.au Disclaimer: This document is a Small Scale Offer Document and is intended for sophisticated investors only (v10 11 Nov 2015)
Contract manufacturing pipeline underwrites basic infrastructure costs during facility expansion; upside potential as products approach approval or projects convert to risk-reward and capacity needs expand by order of magnitude
Cancer immunotherapy (CAR-T): MIC-01, EVE-01
“Autologous Production for Future” commercial scale gene therapy feasibility: DMY-01
Apheresis network management: MIC-02, DMY-02, ORG-01
Robust pipeline of additional CMO opportunities Regional facility expansion plan in hand to enable single gateway access market of more than 750 m under a single, harmonised quality system and project management system
Melbourne: capacity to double from 2016 at PMCC’s new home at Victorian Comprehensive Cancer Centre (“VCCC”) ($12m Victorian Government commitment)
Nextcell, Adelaide: JV with UniSA for small scale translational manufacturing; manufacturing agreement for CRC for Cell Therapy Manufacturing
Pharmabio, Japan: strategic alliance with significant capacity expansion 2017
Malaysia: BioNexus status for subsidiary (tax incentives); establishing central manufacturing hub by 2017
Executing the business plan: operating plan
0-4 yrs to sales 2-6 yrs to sales >4 yrs to sales
E Melbourne Parkville
Adelaide Malaysia 1
Large scale Malaysia 2
Japan US/EU network partners
Facilities network
Rev
enu
e
A. Fee-for-service projects• Historical business model since 2003 continues: payment for activity• Expand to provide pan-Asia capability and service• Examples: Le-Y, Prima Biomed, Mesoblast, MIC
B. Risk-reward products• Investment in products for share of commercial profit• Products in pivotal trials; SE Asia scale manufacturing hub• Examples: JET (manufacturing), DMY (in-licensing)
C. Blue sky potential• EU/US expansion• Own product development• Vertical integration/M&A eg JP
CONFIDENTIAL to Cell Therapies Pty Ltd Ground Floor, 10 St Andrews Place, East Melbourne, VIC, Australia T: +61 3 9656 5804 www.celltherapies.com.au Disclaimer: This document is a Small Scale Offer Document and is intended for sophisticated investors only (v10 11 Nov 2015)
A$25m expansion capital needed to ignite growth
Current A$5m business is profitable but cannot self-generate cash for growth
51% shareholder PMCC prepared for dilution and full spin-off
A$25m capital required to secure and launch first three products, secure ASEAN operating footprint, expand capabilities: attractive valuations for early investors
Multiple add-ons possible: Additional products (~A$5-10m each to secure and launch), cell processing players for geographic expansion, distribution chain assets (cryo-preservation, apheresis/collection, etc), automation solutions
Multiple exits possible: merger with EU or US equivalent business; trade sale; IPO
Contact: Tim Oldham PhD, CEO, Cell Therapies Pty Ltd, +61 403 446 665
Executing the business plan: financing plan
0-4 yrs to sales 2-6 yrs to sales >4 yrs to sales
Re
ven
ue
A. Fee-for-service projects• Low margin, cash self-sufficient on average over time; leased facilities• Historically funded via working capital: cyclicality means frequent calls for financial
assistance unless total business is properly capitalised
B. Risk-reward products• Successive rounds of new equity investment (per project)• Project finance later projects; debt finance facilities• Provides temporary working capital relief for base business
C. Blue sky potential• Exits: trade sale or IPO
Series A round to ignite this
business