Slide 1: Disclaimer
Disclaimer This presentation includes forward-looking statements. All statements contained in this presentation other than statements of historical facts, including statements regarding future results of operations and financial position of Millendo Therapeutics, Inc. (“Millendo,” “we,” “us” or “our
Slide 2: Millendo Therapeutics: Leading orphan endocrine disease company
Millendo Therapeutics Leading orphan endocrine disease company Clinical-stage company focused on orphan endocrine diseases • Clinically-validated molecules with differentiated mechanisms • High unmet medical needs in orphan indications A LEADER IN ORPHAN ENDOCRINE DISEASES Livoletide: Pivotal-stage
Slide 3: Millendo Executive Leadership
Millendo Executive Leadership Relevant and successful drug development and commercialization expertise JEFF BRINZA Chief Administrative Officer & General Counsel JULIA OWENS, PhD Chief Executive Officer PHARIS MOHIDEEN, MD Chief Medical Officer THOMAS HOOVER SVP Commercial Strategy RYAN ZEIDAN, PhD
Slide 4: Prader-Willi Syndrome (PWS)
Livoletide Nevanimibe Prader-Willi Syndrome (PWS) Well-characterized genetic orphan endocrine disease Complex disorder attributed primarily to spontaneous genetic error on CHROMOSOME 15 Prader-Willi syndrome is characterized by: Hyperphagia: Chronic feeling of insatiable hunger Starts at 5-8 years o
Slide 5: PWS Patients & Families Have Significant Needs
Livoletide Nevanimibe PWS Patients & Families Have Significant Needs Almost all Prader-Willi syndrome patients suffer from hyperphagia PWS, what specific things would you 60% with PWS DIABETES 7 Adults>50% BMI >40TYPE 2 “Assuming there is no complete cure for look for in an ideal treatment for PWS?”
Slide 6: Critical Need for PWS Treatments
Livoletide Nevanimibe Critical Need for PWS Treatments Livoletide is a compelling late-stage development drug candidate P Livoletide is ready for a pivotal study Zafgen’s beloranib Phase 3 Well-defined development and regulatory path program is useful precedent Novel compound and mechanism for PWS B
Slide 7: Livoletide for Treatment of Hyperphagia in PWS
Livoletide Nevanimibe Livoletide for Treatment of Hyperphagia in PWS Analogue of unacylated ghrelin etide • Once-daily injectable cyclic peptide vivo PK 9 Acylated Ghrelin (AG) AG is the most potent known appetite-stimulating hormone Unacylated Ghrelin (UAG) Livol (8mer) • Comparable in vitro and in
Slide 8: UAG is a Biologically Active Hormone
Livoletide Nevanimibe UAG is a Biologically Active Hormone Pharmacology of UAG is distinct from that of AG exhibit distinct 1GHSR1a = growth hormone secretagogue receptor, or ghrelin receptor 10 Numerous studies have shown that UAG is biologically active • Activates neurons in brain hunger centers •
Slide 9: Low UAG Levels Associated with PWS Hyperphagia
Livoletide Nevanimibe Low UAG Levels Associated with PWS Hyperphagia UAG inhibits AG-related effects Induced Food Intake in Rats as Hyperphagia Develops 350 8 2 Ph1aase Ph1abse Ph2aase Ph2abse Pha3se 3 Healthy Obese 0 0 UAG livoletide Acylated Ghrelin (AG) meal presentation meal presentation Adapted
Slide 10: 2-week Placebo-Controlled Phase 2 PWS Clinical Trial
Livoletide Nevanimibe 2-week Placebo-Controlled Phase 2 PWS Clinical Trial Robust, meaningful decreases in Hyperphagia Questionnaire (HQ) 0 0 0 baseline baseline baseline Allas et al. 2018 1 Home residents excludes subjects residing in hospital setting; analysis was pre-specified 12 All SubjectsHome
Slide 11: Phase 2 Safety Summary
Livoletide Nevanimibe Livoletide Phase 2 Safety Summary Well tolerated, no safety signals P Safety events well balanced relative to placebo • Subjects reporting any adverse event (AE) – – Livoletide: 60.9% Placebo: 58.3% • Most commonly reported AE in both groups were related injection site with pla
Slide 12: PWS Clinical Development Plan
Livoletide Nevanimibe Livoletide PWS Clinical Development Plan Single, pivotal Phase 2b/3 protocol 3 month endpoint Placebo crossover / 9-month extension Phase 2b Placebo crossover / 6-month extension Phase 3 6 month endpoint Operational efficiencies gained with a single protocol Robust dataset: ran
Slide 13: Nevanimibe (ATR-101) for Orphan Adrenal Diseases
Nevanimibe (ATR-101) for Orphan Adrenal Diseases
Slide 14: Nevanimibe for Orphan Adrenal Diseases
Livoletide Nevanimibe Nevanimibe for Orphan Adrenal Diseases Novel MOA has potential to address serious adrenal diseases P CAH Ph2b CS Ph2 ACAT1 Target Data to Date Clinical Trial Status Orphan Adrenal Diseases Clinical proof-of-concept from open-label Phase 2 clinical trial in CAH Adrenal-selective
Slide 15: Congenital Adrenal Hyperplasia (CAH)
Livoletide Nevanimibe Congenital Adrenal Hyperplasia (CAH) Rare Genetic Endocrine Disease Resulting in Inability to Synthesize Cortisol Exogenous cortisol treatment is required for life Monogenic deficiency of 21-HYDROXYLASE ENZYME Complications result from chronic high dose cortisol 90-95% • Higher
Slide 16: Nevanimibe Proof of Concept Established in CAH
Livoletide Nevanimibe Nevanimibe Proof of Concept Established in CAH Activity demonstrated through reductions in 17-OHP P Biological effect demonstrated through reduction in key steroids and steroid precursors • Mean reductions in 17-OHP were observed at all nevanimibe doses while mean increases wer
Slide 17: Millendo is a Leading Company in Rare Endocrine Diseases
Millendo is a Leading Company in Rare Endocrine Diseases Novel agents in orphan diseases with unmet needs and little innovation Rapidly and efficiently advance development of, obtain approval for, and commercialize livoletide for the treatment of PWS Pursue development of, obtain approval for, and c
Slide 18: Millendo Therapeutics–OvaScience Merger
Millendo Therapeutics–OvaScience Merger Definitive agreement for all-stock merger announced on August 9, 2018 Expected to be completed in 4Q 2018; new company will trade on Nasdaq under MLND Requires OvaScience shareholder approval among other customary conditions Concurrent financing from Millendo
Slide 19: Millendo Therapeutics-OvaScience Merger
Millendo Therapeutics-OvaScience Merger S% MILLENDO THERAPEUTICS. S~ MILLENDO \e “ar Definitive pec teenlnt for all-stock merger 2 announced on n August 9, 2018 Expected to be completed in 140 2018; new company will trade on Nastia u under MLND. Requires Ovasci nce shareholder approval among other c
Slide 20: ~ MILLENDO SON SOE Or
~ MILLENDO SON SOE Or J™ TRANSFORMING FERTILITY THERAPEUTICS