unlocking healthcare potential for patients in rare … · unlocking healthcare potential for...

Denise Scots-Knight – CEO10 January 2017

UNLOCKING HEALTHCAREPOTENTIAL FOR PATIENTS IN

RARE AND SPECIALTY DISEASES

DISCLAIMER

This document has been prepared in good faith and with reasonable care, however the information, statements and opinions contained in it have not been verified by or on behalf of Mereo BioPharmaGroup plc (the Company). No representation or warranty, express or implied, is given by or on behalf the Company, its subsidiaries or any of their respective shareholders, directors, officers, advisers, agents of other persons as to the accuracy, fairness, completeness or sufficiency of the information, projections, forecasts, opinions or statements contained in the presentation. In particular, the market data in this document has been sourced from third parties and has not been verified. Any reliance the recipient places on this document is at his sole risk, and to the fullest extent permitted by law and save in the case of fraud by or on behalf of the Company, no liability is accepted for this document and any errors, omissions, misstatements or inaccuracies (negligent or otherwise) in any of the information or opinions in this document.

Certain information contained herein constitutes “forward-looking statements”, which can be identified by the use of terms such as “may”, “will”, “should”, “expect”, “anticipate”, “project”, “intend”, “continue”, “target” or “believe” (or the negatives thereof) or other variations thereon or words of similar meaning. Due to various risks, uncertainties and assumptions, actual events, results or performance of the Company may differ materially from those reflected or contemplated in such forward-looking statements. Without prejudice to the generality of the preceding paragraph, no reliance should be placed on such forward-looking statements.

Mereo BioPharma January 2017 1

OVERVIEW AND2016 HIGHLIGHTS

Initial portfolioof three Phase 2 products acquired from Novartis with robust packages

BUSINESS OVERVIEW


UK-basedpublic specialty biopharmacompany

Developing innovative medicines in rare and specialty disease areas

Strategy to acquire clinical-stage products with flexibility to commercialise or out-license £90m

raised sinceJuly 2015

Plan to commercialise orphan products

Long term goal of 5-7 products from large pharmaceutical or biotechnology companies

($112m)

DIVERSIFIED PIPELINE ADDRESSING SIGNIFICANT END MARKETS


Product candidate Indication Highlights

Clinical development

Phase 1 Phase 2 Phase 3

BPS-804

OsteogenesisImperfecta (OI)

Antibody –Sclerostin inhibitor

BGS-649Hypogonadotropic Hypogonadism (HH)in obese men

Weekly, oral aromatase inhibitor

AcumapimodAcute Exacerbationsof Chronic Obstructive Pulmonary Disease (AECOPD)

Oral p38 MAPkinase inhibitor

Phase 2b/Pivotal dose ranging - 1H2017

Phase 2b trial ongoing

Phase 2 trial ongoing

OPERATIONAL HIGHLIGHTS – 2016

BPS-804

• Granted Orphan Drug Designation in US and EU

• Successful discussion with FDA on endpoints for initialphase 2b/pivotal study

• Discussions initiated in EU

BGS-649

• Commenced Phase 2bfor the treatment of hypogonadal hypogonadism (HH) in obese men

• Blinded interim to drop any ineffective doses, Q1 2017

• Six month safety extensionstudy initiated in Q4 2016

ACUMAPIMOD

• Opened US IND Q1 2016

• Commenced Phase 2 forthe treatment of acute exacerbations of COPD (AECOPD)


IP strengthened across all three programmes

STRATEGY

DELIVERING ON THE COMPANY STRATEGY

ADVANCE PIPELINE

Phase 2b/Pivotal trialfor BPS-804 in OI

Phase 2b dose confirmation for BGS-649 in HH

in obese men

Phase 2 dose ranging foracumapimod in AECOPD

OPTIMISE VALUE

Control allcommercialisation rights

Out-license, sell, commercialise or combine

various strategies tomaximise value

Intention to commercialise orphan products

EXPAND PORTFOLIO

Focus on product candidates in orphan and specialty diseases with clinically

meaningful data

Continue to evaluate additional potential new

products


Clinicallymeaningful data

Clear clinical &regulatory strategy

Clinical trials to reachvalue creating milestone

Optionality aroundfurther value creation

Indication with unmetmedical need

Sourced from largepharma companies

Scientific rationale & robust data package

Favourable competitivelandscape

ROBUST PRODUCT CANDIDATE SELECTION CRITERIA


SelectionCriteria

INITIAL PRODUCTPORTFOLIO

OSTEOGENESIS IMPERFECTA (OI)


An orphan genetic chronic bone disorder characterised by fragile bones that break easily

6.3OI cases per 100,000 population in the US 1

7.5OI cases per 100,000 population in the EU 2

Prevalence:

85% - 95%linked to a gene mutation

that produces abnormal type 1 collagen 1, 2

72% - 77%of total OI population1

Symptoms:

• Frequent bone fractures and loose joints• Early hearing loss• Respiratory problems • Brittle teeth

No approved therapies toreduce fractures in OI patients

OI types I, III and IV occur in

1) Based on Osteogenesis Imperfecta Foundation estimates2) Based on Orphanet estimates3) Shapiro J (2014) Osteogenesis Imperfecta: A Translational Approach to Brittle Bone Disease. Academic Press. Chapter 2: p15-22

BPS-804 (OI)


Clinical studies to date:• 83 patients have received BPS-804

• Statistically significant improvement in BMD and bone biomarkers in OI patients (P1NP, P1CP, BSAP and OC)

• Down regulation of bone resorption biomarker CTX-1 in OI patients

• Safe and well tolerated in thetarget population

Day 141 BPS-804 Placebo

Parameter N Ratio Geometric mean to baseline

P value NRatio Geometric mean to baseline

P value

Bonemineral density

9 1.04 0.038* 4 1.01 0.138

Fully human monoclonal antibody inhibiting sclerostin

Bone Biomarkers: Procollagen I N-terminal propeptide (P1NP), procollagen C terminal propeptide( P1CP), bone-specific alkaline phosphatase (BSAP), osteocalcin (OC), Carboxy-terminal telopeptide (CTX-1)

* Statistical significance

Note: Trial performed on 14 patients, 9 received BPS-804 and 5 received placebo

Statistically significant increase in BMD in OI patients

BPS-804 CLINICAL DEVELOPMENT


Estimated enrolment:

Trial arms: Study duration:

120 OI PatientsTypes I, III and IV

Study start:

1H 2017

Expected completion: Initial 6 month data

1H 2018

Three different doses of BPS-804Vs

PlaceboRandomised

52Weeks

Analysis at26 and 52 weeks

Primaryendpoints

Compare effects on trabecular volumetric BMD by HRpQCT at 6 months

Secondaryendpoints

• Effects on trabecular volumetric BMD by HRpQCT at 12 months

• Change in all HRpQCT parameters

• Effects on bone biomarkers

• PK/PD

• Effects on PRO and quality of life

HYPOGONADOTROPIC HYPOGONADISM (HH) IN OBESE MEN


A clinical syndrome that results from inadequate levels of testosterone

32.6%Adult males in the

US are obese 1

21.5%Adult males in the

EU are obese 1

Prevalence:

15.8%HH prevalencein obese men 2

10 million*obese men with HH in the US and the EU

Symptoms:

• Reduced or loss of libido• Erectile dysfunction • Fatigue• Impaired physical endurance and strength• Loss of vitality/motivation

Treatment rates:

<13%in the US andlower in Europe 3

1) Based on 2014 WHO estimates2) Hofstra et al (2008) Netherlands J. Med, 66 p103-1093) Update on Hypogonadism and Testosterone Replacement Therapy (2011) Chapter in Practicing Clinical Exchange p1-15

*estimate

BGS-649


Clinical studies to date:

• 130 patients have received BGS-649

• Statistically significant rise in testosterone from day 4 (P=0.012) to normal levels, with once weekly dosing

• Normalisation of testosterone levels was accompanied by rises in LH and FSH

• Safe and well tolerated in the target population

A Novel Aromatase inhibitor

Statistically significant rise in testosterone from day 4 (P=0.012 at day 4)

BGS-649 (HH): POSITIVE TREATMENT EFFECTS ON GONADOTROPHINS


Rises in gonadotrophins against placebo, in contrast to commontestosterone therapies which suppress LH and FSH, threatening fertility

FSH LH

BGS-649: CLINICAL DEVELOPMENT



268Obese men with HH

BMI > 30 kg/m2

Study start:

H1 2016

Expected completion: H2 2017

Primaryendpoints

Normalisation of testosterone300 – 1000 ng/dl in 75% of patients

Secondaryendpoint

• Change in LH and FSH

• Body composition

• Semen analysis

• Three PROs: IIEF, PROMIS and BFI

International Index of Erectile Function (IIEF), patient-reported outcomes measurement information system (PROMIS) and the Brief Fatigue Inventory (BFI)

Trial arms: Study duration:

Three differentdoses VsRandomised Placebo

24Weeks

Blinded interim analysis at 4 weeks

ACUTE EXACERBATIONS OF CHRONIC OBSTRUCTIVE PULMONARY DISEASE (AECOPD)


12mCOPD cases

diagnosed in the US1

13mCOPD cases

diagnosed in the EU1

Prevalence:

>1.5mHospital visits per year2

COPD includes chronic bronchitis, emphysema and some forms of bronchiectasis

Symptoms:AECOPDs occur when a patient with COPDexperiences a sustained increase in cough,sputum production or dyspnoea (shortnessof breath)

Each episode poses significant risk to the patient, including hospitalisation and an increased risk of death

Healthcare costs:

62.5%of all hospital admissions related to COPD are AECOPD patients 4

The yearly cost of COPD is approximately 5

$50bn(US total costs)

$38bn(EU direct costs)

AECOPDs account for the greatest proportion of COPD costs

1) National Heart, Lung and Blood Institute (accessed in Feb 2016)2) COPD Coalition3) Mannino et al (2002) MMWR Survell Summ 51: p1-6

4) Wier et al (2011) AHRQ, HCUP, Statistical Brief #106 p1-115) Global Strategy for the Diagnosis, Management and Prevention of COPD, Global Initiative for Chronic Obstructive Lung

Disease (GOLD) 2016

ACUMAPIMOD – LPS CHALLENGE



• 24 patient placebo controlledstudy challenged in vivo with lipopolysaccharide to assess thedrug’s anti-inflammatory properties

• Statistically significant reduction of TNFα versus placebo, demonstrating acumapimod’s ability to suppress acute inflammatory pathways relevant to AECOPD in man

• No food effect detected

p38 MAPK inhibitorLPS challenge data in healthy volunteers

Hours post dose

TNF

–al

ph

a co

nce

ntr

atio

ns

(pg

/mL)

ACUMAPIMOD – IMPROVEMENT IN FEV1



• 260 patients have received acumapimod

• Dosed at 75mg x 2 – AUC over exacerbation period (14 days) shows a statistically significant improvement in FEV1 vs placebo and prednisolone (P=0.0198 and 0.0102)

• Safe and well tolerated in the target population

Clinically meaningful improvement in FEV1 (>100ml) overexacerbation period

p38 MAPK inhibitor

ACUMAPIMOD: CLINICAL DEVELOPMENT



270AECOPD patients

Study start:

1H 2016

Expected completion: 2H 2017

Weeks

Primaryendpoints Change in FEV1 from baseline at 7 days

Secondaryendpoint

• Assessment of AUC of FEV1 over time

• Respiratory rate

• Time to normalisation of spirometry parameters

• EXACT-PRO

• GOLD stage II to IV• Requiring hospitalisation for treatment

Trial arms: Study duration:Two differentdosing regimens VsPlaceboRandomisedon top of SoC

26Weeks

FINANCIAL HIGHLIGHTS


Shares admittedto the AIM market

Financing activities:

£90 million

Net cash outflow from operating activities:

£10.5 million

Cash and cash equivalentsAt 30 November 2016:

£57.9 million

£14.7 million

Cash sufficient to fund through three key clinical milestones

09 June 2016

($112 million) Since July 2015

For 6 months ended 30 June 2016For 6 months ended 30 June 2015

Loss after tax:

EXPECTED NEWS FLOW AND CATALYSTS


H1 2017 H2 2017 H1 2018

BPS-804

BGS-649

Acumapimod

New Products

FPI*

6 months interim data

Multiple product evaluations

Interim analysis Phase 2b data

Extension study data

Phase 2b data

*FPI = First Patient In

SUMMARY


Well positioned to continue to deliver on clinical and business development objectives

Key milestones delivered for all three programmes

Two Phase 2 studies commencedwithin nine months of product acquisition

Orphan Drug Designation achieved inUS and EU for BPS-804 in OI

BPS-804 – potential pivotal study design agreed

Total funds raised >£90 million

Business development activities ongoing

THANK YOU

Mereo BioPharma Group plc

One Cavendish PlaceLondon, W1G 0QF

UK

+44 (0)333 0237 300

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