Mereo BioPharma Group plc · NASDAQ:MREO
Mereo BioPharma — Investor Presentation (March 2023)
26 slides from Mereo BioPharma's investor presentation, March 2023
Slide 1: Unlocking The Potential Of Novel Targets For Rare Diseases
Unlocking The Potential Of Novel Targets For Rare Diseases March 2023 Mereo BioPharma Group plc NASDAQ: MREO
Slide 2: Disclaimer
Disclaimer This presentation has been prepared by Mereo BioPharma Group plc (the “Company”) solely for your information and for the purpose of providing background information on the Company, its business and the industry in which it operates or any particular aspect thereof. For the purposes of thi
Slide 3: Mereo BioPharma – A Rare Disease Company
Mereo BioPharma – A Rare Disease Company Our Mission To improve the lives of patients with rare diseases Strategic principles guiding us on our journey… Acquire and develop programs in rare Focus on our core competencies and Partner programs where it makes diseases – partner of choice for in- experi
Slide 4: Mereo BioPharma – A Mid-Late-Stage Company with Validating Partnerships
Mereo BioPharma – A Mid-Late-Stage Company with Validating Partnerships • Pipeline of 3 programs in rare disease/rare oncology with advanced clinical data o Setrusumab for Osteogenesis Imperfecta (OI) under the Ultragenyx partnership, entered a registrational trial in Q2 2022 o Alvelestat for Alpha-
Slide 5: Setrusumab – In Late-Stage Development for the Treatment of OI
Setrusumab – In Late-Stage Development for the Treatment of OI Setrusumab Overview £1.65bn • Anti-sclerostin antibody under investigation for the treatment of Osteogenesis Imperfecta (OI), a rare genetic bone disease with high unmet need and no FDA or EMA approved therapies Peak Market Opportunity (
Slide 6: OI – A Rare Genetic Bone Condition with No Approved Standard of Care
OI – A Rare Genetic Bone Condition with No Approved Standard of Care Osteogenesis Imperfecta High Unmet Need • Signs and symptoms include frequent bone fractures, skeletal deformities, • A rare genetic bone disease, mostly linked pain, respiratory and gastric problems to a mutation in Type I collage
Slide 7: Summary of Phase 2b ASTEROID Results in Adults with OI Types I, III and IV
Summary of Phase 2b ASTEROID Results in Adults with OI Types I, III and IV Statistically Significant Dose-Dependent Increases in Areal BMD by DXA Following 12 Months of Setrusumab Therapy Lumbar Spine Mean (SEM) Change From Baseline at 12 *** Total Hip Femoral Neck 10 6 * *** Month 12, % 8 ** 4 ** 6
Slide 8: Phase 2b ASTEROID Study - Positive Impact on Bone Strength and Reducing Fractures
Phase 2b ASTEROID Study - Positive Impact on Bone Strength and Reducing Fractures Mean % Δ from baseline 20mg/kg 8mg/kg 2mg/kg 12 months (n=28) (n=22) (n=25) Radius Total vBMD 1.88 0.86 0.04 (p<0.01) Radius FE Failure Load 3.17 2.33 0.057 (p<0.01) Annualized Total Fracture Rate 0.4 Rate of Fracture
Slide 9: Ultragenyx – Mereo Partnership and Long Term Plan
Ultragenyx – Mereo Partnership and Long Term Plan Mereo – Ultragenyx Partnership Clinical Development Overview Mereo Territories & Focus • Ultragenyx funding and leading • Phase 2/3 Orbit* study in patients 5 - • Mereo’s current focus is laying the global development plan in 25 years old initiated i
Slide 10: Alvelestat – In Development for the Oral Treatment of AATD-LD and Bronchiolitis Obliterans Syndrome
Alvelestat – In Development for the Oral Treatment of AATD-LD and Bronchiolitis Obliterans Syndrome Alvelestat Overview £1bn • Neutrophil elastase (NE) is a protease enzyme that unopposed is believed to drive lung tissue destruction in Peak Market Opportunity (2035)(2) lead indication, AATD-LD and,
Slide 11: AATD-LD – A Rare Progressive Lung Disease with High Unmet Need
AATD-LD – A Rare Progressive Lung Disease with High Unmet Need Alpha-1 antitrypsin (AAT) inhibits the action of neutrophil elastase. Individuals who lack AAT or produce misfolded inactive AAT are at risk from progressive lung damage and early onset emphysema. AATD-LD High Unmet Need Significant Mark
Slide 12: Primary Endpoint: Blood NE Activity – Week 12
Primary Endpoint: Blood NE Activity – Week 12 P value % CFB compared to placebo Week 120 mg 240mg 4 <0.001 <0.001 8 0.012 <0.001 12 0.023 <0.001 Significant suppression at both doses > Sustained 90% suppression of blood neutrophil elastase activity at the 240mg dose 12
Slide 13: Aa-Val360 and Desmosine Disease Activity Biomarkers in AATD-LD
Aa-Val360 and Desmosine Disease Activity Biomarkers in AATD-LD Aa-Val360 Desmosine • “Footprint” of NE activity • “Footprint” of elastin breakdown • Elevated in blood and lung in AATD-LD • Elevated in AATD • Correlates with measures of AATD-LD disease • Correlates with measures of AATD-LD disease se
Slide 14: Primary Endpoint: Disease Activity Biomarkers % Change from Baseline at Week 12
Primary Endpoint: Disease Activity Biomarkers % Change from Baseline at Week 12 Aα-Val360 Desmosine Statistically significant decrease in desmosine and Aα-Val360 14 from baseline and vs. placebo with 240mg dose only
Slide 15: Association of Biomarker Responses with St. George's Respiratory Questionnaire Activity Domain
Association of Biomarker (Dual Aα-val360 and Desmosine) Responses with St. George’s Respiratory Questionnaire Activity Domain in Alvelestat Alvelestat Combined Groups Placebo 240mg dose = 12 Responders / 13 Non-responders Placebo = 3 Responders / 25 Non-responders 120mg dose = 3 Responders / 11 Non-
Slide 16: Clinical Data Support Progression into Phase 3 with Single Dose
Clinical Data Support Progression into Phase 3 with Single Dose • Meaningful impact of alvelestat on disease-activity biomarkers: • Consistent and progressive suppression at 240 mg • Extent of change in biomarkers compares favorably with augmentation • Association of biomarker response with clinical
Slide 17: Proposed Phase 3 Study Design Following End-of-Phase 2 Meetings
Proposed Phase 3 Study Design Following End-of-Phase 2 Meetings • Both FDA and EMA have demonstrated flexibility and openness to collaborate in their approach to approval in AATD-LD • Proposing one global trial for full approval in US and EU – no need for a long confirmatory study • Global Phase 3 w
Slide 18: Linking ASTRAEUS Change in Desmosine to CT-density Decline through RAPID Data
Linking ASTRAEUS Change in Desmosine to CT-density Decline through RAPID Data Correlation of change from baseline in desmosine at 2 years and lung density decline (RAPID study) Alvelestat month 3 -0.028 Source: Ma et al. The Effect of Alpha-1 Proteinase Inhibitor on Biomarkers of Elastin Degradation
Slide 19: SGRQ Activity Domain Detects Symptoms most Disruptive for Patients with AATD
SGRQ Activity Domain Detects Symptoms most Disruptive for Patients with AATD: ASTRAEUS findings consistent with published literature MPH966-2-01 Baseline SGRQ Scores Published Studies with AATD 19
Slide 20: Etigilimab – An Anti-TIGIT Antibody, in Development in Combination with an Anti-PD-1 Antibody
Etigilimab – An Anti-TIGIT Antibody, in Development in Combination with an Anti- PD-1 Antibody Etigilimab Summary • An IgG1 monoclonal antibody designed to balance affinity and ADCC characteristics while limiting ~£3.0bn+ side effects Peak Market Opportunity (2035)(4) • Pharmacodynamic changes consi
Slide 21: ACTIVATE Trial – Preliminary Efficacy Data
ACTIVATE Trial – Preliminary Efficacy Data Response summary, as of a data cut-off date of 4/20/2022 (efficacy analysis set; n=38)* Objective Cohort Responses by RECIST A C E F (Rare) F (Rare) F (Rare) H Total Endometrial Cervical TMB-H/MSS Uveal Sarcoma GCT Ovarian Cancer Cancer (HGSOC) (CPI-naïve)
Slide 22: Etigilimab Shows Robust Target Engagement in Patients and Exploratory Biomarker Correlation
Etigilimab Shows Robust Target Engagement in Patients and Exploratory Biomarker Correlation with Clinical Response Baseline Tumor Expression: Correlation with Etigilimab Shows Robust Target Engagement Reductions in Target Lesions CD226/CD8 Co-expression % Change From Baseline PVR Sarikonda et al, ES
Slide 23: Partnerships Overview
Partnerships Overview Current Partnerships • Ultragenyx leads global development of setrusumab, Mereo retains rights to setrusumab in Europe and the UK • Received $50 million upfront, up to $254 million in additional milestones plus royalties • Registrational trial initiated • Navicixizumab out-lice
Slide 24: Upcoming Key Milestones & Opportunities
Upcoming Key Milestones & Opportunities Upcoming Milestone For Core Programs Product 2022 2023 2024 Next Milestone Candidate H1 H2 H1 H2 H1 H2 Phase 2b/3 ORBIT (5 - 25 years) Phase 2/3 dosing update and Phase 3 Setrusumab transition OI Pediatric study ( <5 years ) Study initiation Alvelestat Phase 2
Slide 25: Roadmap for Growth
Roadmap for Growth Maintain A Strong • Cash runway into 2026: £67.5 million ($74.8 million) as of Sept. 30, 2022. 1 Balance Sheet • Active cost management – runway through key inflection points. • Focus on high value rare disease programs. Allocate R&D capital to key value inflection points. • Setru
Slide 26: Mereo BioPharma Group plc
Mereo BioPharma Group plc One Cavendish Place London W1G 0QF United Kingdom +44 (0)333 0237 300
Mereo BioPharma Group — Investor Presentation (March 2023) on 6ix