Mereo BioPharma Group plc · NASDAQ:MREO
Mereo BioPharma — Investor Presentation (January 2025)
50 slides from Mereo BioPharma's investor presentation, January 2025
Slide 1: Corporate presentation
Corporate presentation January 2025
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: Unlocking the potential of novel targets for rare diseases
Unlocking the potential of novel targets for rare diseases Our mission is to improve the lives of people living with rare diseases
Slide 4: Strategic principles guide our journey
Strategic principles guide our journey • Acquire and develop programs in rare diseases with high prevalence – partner of choice for in-licensing • Focus on our core competencies and experience in rare diseases • Develop pipeline of rare disease programs which have already received significant invest
Slide 5: A late-stage rare disease company with a capital efficient model
A late-stage rare disease company with a capital efficient model Achievements and fundamentals Two rare disease programs in-licensed and progressed to pivotal stage: • Setrusumab for Osteogenesis Imperfecta (OI) in Phase 3 under a partnership with rare disease leader Ultragenyx • Alvelestat for Alph
Slide 6: Track record of value-creating partnerships
Track record of value-creating partnerships Potential to provide future milestone payments and royalties • Both late-stage rare disease programs from large pharma following significant investment with success-based returns • Setrusumab acquired from Novartis • Alvelestat in-licensed from AstraZeneca
Slide 7: Addressing patient populations with high unmet needs & significant market opportunities
Addressing patient populations with high unmet needs & significant market opportunities of >$1Bn1 Osteogenesis Imperfecta Alpha-1 Antitrypsin Deficiency Rare genetic bone condition leading to Rare genetic progressive lung disease Disease Background problems including frequent fractures and character
Slide 8: Late-stage clinical pipeline with two rare disease programs
Late-stage clinical pipeline with two rare disease programs Product candidate Phase 1 Phase 2 Phase 3 2025 Milestones Setrusumab Orbit (5 - 25 yrs old) Phase 3 data Osteogenesis Imperfecta Cosmic (2 - 6 yrs old) Potential partnership Alvelestat* Potential Phase 3 initiation AATD-Lung Disease *Invest
Slide 9: We have achieved key designations available for rare diseases
We have achieved key designations available for rare diseases Setrusumab Alvelestat for Osteogenesis Imperfecta for AATD-associated Lung Disease Orphan Drug Designation - Breakthrough Ultragenyx Designation achieved in 2024 Fast-track Designation - PRV designation Not relevant Orphan Designation - P
Slide 10: Mereo is in a strong position to execute through 2025
Mereo is in a strong position to execute through 2025 Financial discipline delivers cash runway into 2027. Mereo is in a strong position to execute through 2025, including critical pre-commercialization activities for setrusumab. Setrusumab Alvelestat AND CLINICAL CORPORATE Interim Analysis 2 (p<0.0
Slide 11: Patient community quote
“It’s always a pleasure to come and speak with people who are actually making a difference on the ground and making a difference for people like myself and for others in the community. Because it is what you do that helps us to live the lives that we want and that we deserve.” Thines Ganeshamoorthy,
Slide 12: Setrusumab (UGX143)
OIFE Topical Meeting Setrusumab (UGX143) June 2023 Osteogenesis Imperfecta: a rare genetic bone condition with no FDA or EMA approved therapy
Slide 13: Rare genetic bone condition represents a >$1Bn market opportunity
Rare genetic bone condition represents a >$1Bn market opportunity A serious, but not Clear need for Established community mysterious condition treatment options • 80-90% linked to a • Well-established • No FDA / EMA approved mutation in Type I Community groups therapy collagen2,3 (Type I, III (OIFE
Slide 14: Setrusumab – a well-defined Mechanism of Action
Setrusumab – a well-defined Mechanism of Action Increased BMD and bone strength, decreased fracture rate 14
Slide 15: Phase 2b ASTEROID study in adults with OI Types I, III and IV
Phase 2b ASTEROID study 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 12 6 Lumbar Spine Total Hip Femoral Neck Mean (SEM) Change From Baseline at Month 12, % *** * 10 8 *** 4 ** ** * 6 109 patien
Slide 16: Orbit Phase 2 – BMD and Z-score mean increase through month 12
Orbit Phase 2 – BMD and Z-score mean increase through month 121 Improvements consistent across all OI Types studied Lumbar Spine BMD1 Lumbar Spine BMD1 % Change from Baseline Z-Score Change from Baseline 1.25 ± 0.17 22.25 ± 2.71 p<0.0001 p<0.0001 0.85 ± 0.13 14.19 ± 2.15 0.58 ± 0.10 9.16 ± 1.43 Chan
Slide 17: Orbit Phase 2 – increase in BMD observed in all age groups
Orbit Phase 2 – increase in BMD observed in all age groups,1,2 Greatest increase in patients 5-12 years of age Lumbar Spine BMD by Age Group % Change from Baseline to Month 12 Data consistent with ASTEROID Phase 2 data in adults2 Age Groups 1. Data as of June 2024; 2. Lewiecki EM et al. Evaluating S
Slide 18: Setrusumab resulted in a 67% reduction in annualized fracture rate
Treatment with setrusumab (mean duration of 16 months) resulted in a 67% reduction in annualized fracture rate (AFR) compared to pre-treatment AFR 6 y/o male patient with Type IV OI, increased mobility after 17 months on study 18
Slide 19: Safety evaluation shows setrusumab is well tolerated
Safety evaluation at 14 months shows setrusumab is well tolerated No No unexpected No subject No drug-related treatment-related adverse events or discontinued treatment hypersensitivity SAEs safety concerns for any adverse event reactions Most common adverse events (AEs) reported at 6 months*1 Adver
Slide 20: Orbit & Cosmic – Phase 3 studies are fully enrolled
Orbit* & Cosmic** – Phase 3 studies are fully enrolled Setrusumab vs. bisphosphonates 1:1 Setrusumab vs. placebo 2:1 randomization Objective randomization Double blind Open label 158 subjects ages 5 to 25 years with 69 subjects ages 2 to 6 years with Enrollment OI Types I, III, or IV OI Types I, III
Slide 21: Laying the foundation for a successful setrusumab launch in Europe
Laying the foundation for a successful setrusumab launch in Europe Targeted resourcing with rare disease Capturing & expertise articulating value to • Defined number of Partnering with healthcare systems expert centers – peak physician community 65-70 field force • Early engagement with to be delive
Slide 22: Evidence Generation: building and delivering our case in Europe
Evidence Generation: building and delivering our case in Europe Setting the baseline: Impact / Regulatory scientific advice & Validated “library” of data Burden of Disease in OI in Adult HTA & Payor advice sources to answer and Pediatric patients across authorities’ questions: at time Mereo European
Slide 23: Learning from the launch of Crysvita in Europe
Learning from the launch of Crysvita in Europe Kyowa Kirin reported EMEA revenues for Crysvita1, $M, 2018-2024 In its 7th year of launch, EMEA COVID-19 impacted revenues are still growing at ~30% EMA approved Crysvita commercialization for use in adults EMA approval for Oct 2020 328 328 pediatrics F
Slide 24: The Ultragenyx partnership, a highly effective collaboration
The Ultragenyx partnership, a highly effective collaboration • Ultragenyx leads and funds the global development plan, including CMC (Dec 2020) • Mereo retains European rights (including UK) and Ultragenyx has the USA and Rest of the World rights • Mereo received $50M upfront and a $9m milestone wit
Slide 25: The Ultragenyx partnership - potential attractive cash flows
The Ultragenyx partnership - potential attractive cash flows ROW European revenue revenue Flat royalty on EU sales % of proceeds from Ultragenyx (ROW)* Tiered royalty ROW sales Mereo Novartis Ultragenyx ≤ $245M in milestones BioPharma Tiered royalties on European revenue Share of PRV proceeds * Subj
Slide 26: Alvelestat (MPH966)
Alpha 1 Support Group UK Alvelestat (MPH966) Information Day September 2023 Alpha-1 Antitrypsin Deficiency-associated Lung Disease: a rare progressive lung disease with high unmet need
Slide 27: Alvelestat, a potential >$1bn market opportunity in AATD-LD
Alvelestat, a potential >$1bn market opportunity in AATD-LD A rare progressive Alvelestat targets root Two Phase 2 trials in Significant market disease with high cause of lung damage AATD-LD opportunity unmet need • Presents age 20 to 50 • Lack of AAT → risk of ASTRAEUS • Augmentation revenues with
Slide 28: Alvelestat supported by promising efficacy and safety data in >1000 subjects
Alvelestat’s potential role in lung disease is supported by promising efficacy and safety data in >1000 subjects Relevant Phase 2 studies Reduction in biomarkers of NE-driven connective tissue breakdown AATD-LD | ASTRAEUS study N=99 (desmosine and A-val360) in PI*ZZ patients not on augmentation the
Slide 29: Short-term symptomatic changes expected to evolve to long-term disease modification
Alvelestat results in short-term symptomatic changes that we expect will evolve to long-term disease modification Short-term effect of alvelestat Chronic effect of alvelestat Alvelestat shows >90% Timeframe 1-3 months 12-18 months inhibition of serum NE from wk 41 Impact Systemic NE inhibition NE in
Slide 30: Two AATD Phase 2 studies demonstrated good overall safety vs. placebo
Data from two AATD Phase 2 studies, demonstrated good overall safety vs. placebo and builds on extensive safety database Alvelestat 240 mg Alvelestat 120 mg Placebo N=40 (%) N=54 (%) N=67 (%) SAE 3 (7.5) 1 (1.9) 0 (0) Adverse Events of 11 (27.5) 10 (18.5) 18 (26.9) Special Interest Infections requir
Slide 31: Preliminary data support a protective effect on acute exacerbations
Preliminary data support a protective effect of alvelestat on acute exacerbations • Reduction in acute exacerbations observed in % patients with exacerbations by week 16 ATALANTa + ASTRAEUS combined, all doses N=161 Phase 2 program o Effect observed across all levels of GOLD Placebo 25% severity1 Al
Slide 32: Alvelestat treatment association with improvement in SGRQ, an FDA recognized PROM
Association of alvelestat treatment with improvement in Respiratory Health Status (SGRQ), an FDA recognized PROM* • St. George's Respiratory Questionnaire – Patient reported outcome in COPD recognized by FDA o Total score = Activity, Symptoms and Impacts domains o Activity domain most impacted in pa
Slide 33: Alvelestat expected to be long-term disease-modifying therapy beyond augmentation
Alvelestat is expected to be a long-term disease-modifying therapy going above & beyond augmentation therapy Long-term effect of alvelestat Reduction in desmosine for 240 mg alvelestat at 12 weeks superior to augmentation therapy Desmosine levels have been shown to Augmentation Alvelestat (240 signi
Slide 34: Well-defined plan for Phase 3 registrational trial in AATD-LD
Well-defined plan for Phase 3 registrational trial in AATD-LD • Earlier stage severe PI*ZZ patients observed to have Clinical greater response in SGRQ (Total and activity) Broader Data • Earlier stage patients (higher FEV1) may be more likely to show spirometry benefit population maximizes • Early →
Slide 35: Other programs, milestones and financials
Other programs, milestones and financials
Slide 36: Key milestones for core programs
Key milestones for core programs Product 2023 2024 2025 Next milestone candidate H1 H2 H1 H2 H1 H2 Setrusumab Phase 2 Orbit (5 - <26 yrs old) OLE OI Phase 3 Orbit (5 - 25 yrs old) Phase 3 Interim analysis Phase 3 Interim analysis Phase 3 Cosmic (2 - <7 yrs old) Alvelestat Potential partnering, Phase
Slide 37: Other programs
Other programs Other current partnerships Navicixizumab – global rights out-licensed to Feng Biosciences for further development in ovarian cancer • Payments of up to $300 million in milestones plus royalties Leflutrozole – global rights out-licensed to ReproNovo for further development • ReproNovo
Slide 38: Financial highlights
Financial highlights Cap Table ADSs (September 2024) (in thousands) Shareholders > 2% holding 86,026 Shareholders < 2% holding 68,709 Cash runway into 2027 Share capital – Issued and outstanding $80.5 million as of as of September 30, 20241 154,734 September 30, 2024 Potential Future Dilution: Warra
Slide 39: Thank you
Thank you With a special thank you to members of our community, who generously agreed to be featured in this presentation.
Slide 40: Appendix
APPENDIX Mereo BioPharma
Slide 41: Setrusumab (BPS-804)
Rare Disease Day Setrusumab (BPS-804) February 2023 Mouse models and HR-pQCT
Slide 42: Brittle mouse model – treatment with BPS-804 (setrusumab)
Brittle mouse model (Brtl/+)– treatment with BPS-804 (setrusumab) Mature Btrl control Mature WT Control Mature Brtl treated Mature WT Treated Brtl – brittle mouse model WT – wild type 42
Slide 43: HR-pQCT scans of patients with OI and controls
HR-pQCT scans of patients with OI and controls 43
Slide 44: The OFLEY STUDY and HR-pQCT
The OFLEY STUDY and HR-pQCT • Prospective study investigating the prediction of fracture (Fx) by bone microarchitecture assessed by HR-pQCT in postmenopausal women • HR-pQCT used to measure microarchitecture at the distal radius and tibia in 589 women (mean 68 years old) • During 9 year follow up 13
Slide 45: Alvelestat (MPH966) Additional data
Alpha 1 Support Group UK Alvelestat (MPH966) Information Day September 2023 Additional data
Slide 46: Alvelestat is highly effective in an Animal Model of AATD Lung Disease
Alvelestat is highly effective in an Animal Model of AATD Lung Disease Six-month Chronic Smoke (CS)-exposed guinea-pig model of AATD emphysema1 • CS inactivates AAT • Progressive inflammation, airspace enlargement and airway remodeling At clinically relevant doses, alvelestat, prophylactically (P) a
Slide 47: Earlier stage lung disease patients show greater SGRQ response
Earlier stage lung disease patients show greater SGRQ response ATALANTa study (non-augmentation • ATALANTa study – Non-augmentation subgroup (median subgroup) – Change in SGRQ Total Score FEV1 89.3%). Between group changes at week 12: • SGRQ Total = 4.7-point improvement (p=0.10) • SGRQ Activity = 1
Slide 48: Biomarkers and PK modelling confirm 240 mg dose for progression
Biomarkers and PK modelling confirm 240 mg dose for progression ASTRAEUS (Primary Endpoints) ASTRAEUS • 240 mg – progressive reduction in Aα-Val360 Desmosine biomarkers of NE-driven protein breakdown (A-val360 ) and elastin turnover (desmosine) • 120 mg – no effect on desmosine ATALANTa • Consisten
Slide 49: Mereo's approach to Phase 3
Historical augmentation studies Mereo’s approach to Phase 3 Limited to FEV1 <70% or <80% No upper FEV1 limit (Average FEV1 in RAPID, EXACTLE ~50%) • FEV1 – weighting towards patients >75% • Patients earlier in their disease not eligible • More patients eligible, including those not eligible • Interv
Slide 50: Mereo BioPharma Group plc
Mereo BioPharma Group plc 4th Floor, One Cavendish Place, London, W1G 0QF, UK investors@mereobiopharma.com www.mereobiopharma.com +44(0)333 023 7300
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