Biophytis SA · EURONEXT:ALBPS
Biophytis SA — Investor Presentation (September 2019)
27 slides from Biophytis SA's investor presentation, September 2019
Slide 1: Live Healthier Longer
LIVE HEALTHIER LONGER ae EPA: ALB ps
Slide 2: Forward Looking Statements
Forward Looking Statements All statements pertaining to future financial and/or operating results, future growth in research, clinical development, and potential opportunities for Biophytis SA and its subsidiaries (the “Company”) and its products, along with other statements about the future expecta
Slide 3: Overview
Overview Sarconeos (BIO101) for sarcopenia Sarconeos (BIO101) for DMD Macuneos (BIO201) for dry AMD 3
Slide 4: A clinical-stage biotechnology company in age-related diseases
A clinical-stage biotechnology company in age-related diseases Our goal Neuromuscular diseases Retinal diseases Improve functional Our lead drug candidate Our second drug candidate outcomes (mobility, strength Sarconeos (BIO101) is in Macuneos (BIO201) is in and vision) and healthspan clinical devel
Slide 5: Aging and biological resilience pathways
Aging and biological resilience pathways The global elderly (≥60) • As we age, our physical (mobility and strength) and visual population is projected to more performances decline due in part to the accumulation of than double by 20502 multiple stresses we are exposed to during our lifetime 2050 • W
Slide 6: Our clinical pipeline
Our clinical pipeline Candidates Indication Program Preclinical Phase 1 i Phase 2 , Phase 3. Sarconeos Sarcopenia SARA | E> ey — (BIO101) DMD MYODA f (Direct to Phase) P| Macuneos Dry AMD MACA (BIO201) Stargardt blophytis
Slide 7: Sarconeos (BIO101) for sarcopenia
Overview Sarconeos (BIO101) for sarcopenia Sarconeos (BIO101) for DMD Macuneos (BIO201) for dry AMD 7
Slide 8: Sarcopenia is an unmet medical need with no approved drugs
Sarcopenia is an unmet medical need with no approved drugs • Age-related degeneration of skeletal muscle characterized United States Europe by a loss of muscle mass, strength and the ability to stand Total Population 1: Total Population 3: and/or walk 324.2M 511.5M • A major cause of mobility disabi
Slide 9: Sarconeos (BIO101) potential mechanism of action: Activates the MAS receptor
Sarconeos (BIO101) potential mechanism of action: Activates the MAS receptor • MAS is a key component of the renin-angiotensin system (RAS) • We believe MAS activation triggers three downstream pathways which may preserve muscle strength, function and mobility in various age-related and muscular was
Slide 10: Sarconeos (BIO101) preclinical data in animal models for sarcopenia
Sarconeos (BIO101) preclinical data in animal models for sarcopenia suggests improvements in mobility and strength Beneficial effect on mobility Preservation of muscle strength in aged mice fed with high fat diet1 in immobilized mice Administration of 50 mg/kg/day of Administration of 50 mg/kg/day o
Slide 11: SARA-PK: Phase 1 trial completed in 2017
SARA-PK: Phase 1 trial completed in 2017 • Single and multiple ascending doses tested in 54 healthy adult and elderly volunteers • Determined the two active doses (175 & 350 mg b.i.d.) for our ongoing SARA-INT Phase 2b clinical trial P1 TRIAL SAFETY RESULTS PD SUMMARY IN MAD PHASE SAD: Dose range be
Slide 12: SARA-INT: Ongoing Phase 2b clinical trial
SARA-INT: Ongoing Phase 2b clinical trial Global, multicenter, double-blind, randomized, placebo-controlled trial in 334 elderly patients with sarcopenia at risk of mobility disability Objectives Key Endpoints Subpopulation Analysis • Assess safety and efficacy of Primary • Very low walking speed tw
Slide 13: SARA-INT: Recruiting patients at risk of mobility disability
SARA-INT: Recruiting patients at risk of mobility disability Inclusion Criteria Recruitment Recruitment • Age (≥65 or over) • Recruiting in U.S. and Belgium with 15 sites currently open; expanding to 22+ sites • Low mobility measured by Short Performance Physical Battery (SPPB) ≤8 out of 12 • Awaiti
Slide 14: Sarconeos (BIO101) for DMD
Overview Sarconeos (BIO101) for sarcopenia Sarconeos (BIO101) for DMD Macuneos (BIO201) for dry AMD 14
Slide 15: DMD is an unmet medical need with no cure and limited treatment options
DMD is an unmet medical need with no cure and limited treatment options Proportion of ambulatory class • Rare, genetic neuromuscular disease in male children in DMD1 characterized by accelerated degeneration of muscles, responsible for loss of mobility, respiratory failure and 100% cardiomyopathy, l
Slide 16: Proof of concept in mdx mice models of DMD
Proof of concept in mdx mice models of DMD: Improvements in mobility, strength and respiration Improved muscle strength, as Ameliorates the time-dependent Improved mobility as measured by measured by four-limb grip-test degradation of respiratory running distance1 force1 function2 C57BL10-mdx mice t
Slide 17: MYODA-INT: Seamless clinical trial design
MYODA-INT: Seamless clinical trial design (subject to regulatory approval) To test an oral pediatric drug formulation of Sarconeos (BIO101) for DMD patients Design Patients Regulatory Status • Global, multicenter, double-blind, • Ambulatory and non-ambulatory • Pre-IND correspondence with placebo-co
Slide 18: MYODA-INT: Potential composite score based on function
MYODA-INT: Potential composite score based on function • Composite endpoints adapted to the stage of severity of the disease in each patient • Clinical relevance endpoints Seamless Design Strength Composite Endpoints Mobility Strength Respiratory function 18
Slide 19: Macuneos (BIO201) for dry AMD
Overview Sarconeos (BIO101) for sarcopenia Sarconeos (BIO101) for DMD Macuneos (BIO201) for dry AMD 19
Slide 20: Dry AMD is an unmet medical need with no approved drugs
Dry AMD is an unmet medical need with no approved drugs Projection of AMD prevalence in • AMD is a common eye disorder among people over 50 that affects the North America (in M, mean projection)1 central part of the retina, known as the macula 25 • Can impair functions such as reading, driving, and
Slide 21: Macuneos (BIO201) potential mechanism of action: Non-canonical activation of PPARs
Macuneos (BIO201) potential mechanism of action: Non-canonical activation of PPARs • We believe Macuneos (BIO201) potentially counteracts the phototoxic effects of A2E by selective non-canonical activation of the transrepressive activity of PPAR⍺ and PPAR/ in the retina • Most other PPAR ligands m
Slide 22: Macuneos (BIO201) proof of concept in rodent models of dry AMD
Macuneos (BIO201) proof of concept in rodent models of dry AMD: Protects the retina • We are preparing for clinical development through our MACA program and plan to seek regulatory advice in 2H 2019 from European regulatory agencies for a Phase 1 clinical trial Preservation of Reduced A2E Dose-depen
Slide 23: Key milestones in clinical development of BIO101 in neuromuscular diseases
Key milestones in clinical development of BIO101 in neuromuscular diseases ✓ ❑ SARA-OBS completed enrollment of 218 patients in Oct. 2018 ✓ ❑ SARA-INT (Phase2b) enrolled first patient in May 2018 ❑ SARA-INT (Phase2b) expected to complete patient enrollment in 2020 ✓ ❑ MYODA-INT received orphan drug
Slide 24: Executive team
Executive team Samuel Agus - CMO Stanislas Veillet - Founder & CEO • MD, PhD, Board-certified Neurologist • PhD in genetics, AgroParisTech • 15+ years pharma/biotech experience • 25+ years in biotech; Pharmacia-Monsanto, including Abbott, Shire and Teva Danone Group Pharmaceuticals René Lafont - Co-
Slide 25: Scientific advisory board
Scientific advisory board Pr. Jean Mariani Dr. Roger Fielding • Professor of neuroscience and biology of aging • Professor of Medicine, Tufts University School of and Director of Charles Foix Institute of Medicine Longevity at Sorbonne University • Director and Sr. Scientist Jean Mayer USDA Human Nu
Slide 26: Board of directors
Board of directors Stanislas Veillet - Founder & CEO Eric Rowinsky - Independent Board Member • PhD in genetics, AgroParisTech • President of Rgenix and CSO at Clearpath Development Co; • 25+ years in biotech; Pharmacia-Monsanto, Danone board member of Biogen, Fortress Biotech and Verastem Group • 2
Slide 27: Thank you
Thank you Investor relations : investors@biophytis.com 28