Tempest Therapeutics, Inc. · NASDAQ:TPST
Tempest Therapeutics — Millendo Therapeutics — Investor Presentation (January 2019)
25 slides from Tempest Therapeutics's investor presentation, Jan 7, 2019
Slide 1: Cautionary Statement Regarding Forward-Looking Statements
2 Cautionary Statement Regarding Forward-Looking Statements Certain statements contained in this presentation regarding matters that are not historical facts, are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securiti
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 Proceeds of $85M from merger with OvaScience and associated financin
Slide 3: Millendo Executive Leadership
JULIA OWENS, PhD Chief Executive Officer LOUIS ARCUDI III Chief Financial Officer JEFF BRINZA Chief Administrative Officer & General Counsel PHARIS MOHIDEEN, MD Chief Medical Officer THOMAS HOOVER SVP Commercial Strategy Ryan Zeidan, PhD SVP Development ANDREW SPENCER, PhD SVP Preclinical R&D Millen
Slide 4: Compelling Orphan Endocrine Disease Pipeline
Product Candidate Indication Preclinical Phase 1 Phase 2 Phase 2b Phase 3 Livoletide (AZP-531) Prader-Willi Syndrome (PWS) Nevanimibe (ATR-101) Congenital Adrenal Hyperplasia (CAH) Endogenous Cushing’s Syndrome (CS) Compelling Orphan Endocrine Disease Pipeline Multiple late-stage programs in areas o
Slide 5: Livoletide (AZP-531) for Prader-Willi Syndrome
Livoletide (AZP-531) for Prader-Willi Syndrome
Slide 6: Prader-Willi Syndrome (PWS)
Prader-Willi Syndrome (PWS) Well-characterized genetic orphan endocrine disease Prader-Willi syndrome is characterized by: Hyperphagia: Chronic feeling of insatiable hunger Starts at 5-8 years old and usually lasts until death Hallmark of PWS Intellectual/emotional disabilities and psychiatric disor
Slide 7: PWS Patients and Families Have Significant Needs
8 PWS Patients and Families Have Significant Needs Almost all Prader-Willi syndrome patients suffer from hyperphagia Livoletide Nevanimibe Hyperphagia is root cause of morbidity and mortality Abnormally high appetite leading to overeating and eventual metabolic issues Leads to patient accidents, whi
Slide 8: Critical Need for PWS Treatments
Livoletide Nevanimibe P Critical Need for PWS Treatments Livoletide is a compelling late-stage development drug candidate Well-defined development and regulatory path Livoletide is ready for a pivotal study initiating 1Q19 Beloranib Phase 3 program is useful precedent Novel compound and mechanism fo
Slide 9: Livoletide for Treatment of Hyperphagia in PWS
10 Livoletide for Treatment of Hyperphagia in PWS Pharmacology of UAG is distinct from that of AG Livoletide Nevanimibe Livoletide Unacylated Ghrelin (UAG) Acylated Ghrelin (AG) Once-daily injectable cyclic peptide Comparable in vitro and in vivo pharmacological profiles to UAG More stable than line
Slide 10: Lower Relative UAG Levels Associated with PWS Hyperphagia
11 Lower Relative UAG Levels Associated with PWS Hyperphagia Livoletide Nevanimibe Median UAG and AG Fall as Hyperphagia Develops AG and UAG in PWS Nutritional Phases Adapted from Kuppens, 2015 pg/mL Nutritional Phase Nutritional Phase Characteristics: Phase 1a: Hypotonia with difficulty feeding Pha
Slide 11: Robust Development Program
P Phase 1 study Single ascending dose (N=44) in healthy volunteers Multiple ascending dose (N=32) in overweight / obese adults Decreases in glucose and body weight Multiple ascending dose (N=36) in Type 2 diabetes patients Double-blind, placebo-controlled Phase 2 clinical trial in PWS completed (7 E
Slide 12: 2-week Placebo-Controlled Phase 2 PWS Clinical Trial
13 2-week Placebo-Controlled Phase 2 PWS Clinical Trial Robust, meaningful decreases in Hyperphagia Questionnaire (HQ) p = 0.097 24 23 20 18 12 14 p = 0.034 p = 0.019 BL = baseline HQ score (adjusted for 0-36 scale) 1 Home residents excludes subjects residing in hospital setting; analysis was pre-sp
Slide 13: Livoletide Demonstrated Similar Effect to Beloranib
14 Livoletide Demonstrated Similar Effect to Beloranib Similar HQ decreases observed with livoletide Livoletide Phase 2 Results (2 weeks) Beloranib Phase 3 Results (4 weeks) McCandless et al., Diabetes Obes Metab (2017) 19:1751-61 *1.8 mg for week 1-4; then 2.4 mg for week 5-26 Inclusion criteria of
Slide 14: Livoletide PWS Pivotal Study
Livoletide PWS Pivotal Study 15 Single, pivotal Phase 2b/3 protocol Placebo (n~40) livoletide (n~40) Phase 2b Phase 3 Placebo crossover / 9-month extension Placebo crossover / 6-month extension 3 month endpoint 6 month endpoint Livoletide Nevanimibe Placebo (n=50) 60 mg/kg livoletide* (n=50) 120 mg/
Slide 15: Nevanimibe (ATR-101) for Orphan Adrenal Diseases
Nevanimibe (ATR-101) for Orphan Adrenal Diseases
Slide 16: Nevanimibe for Congenital Adrenal Hyperplasia
Clinical Proof of Concept Clinical proof-of-concept from open-label Phase 2 clinical trial in CAH Favorable safety profile in preclinical and clinical studies Well-defined mechanism of action Clinical Trials Underway Phase 2b clinical trial in CAH started in 3Q18 Orphan drug designation granted by U
Slide 17: Nevanimibe Targets Starting Point of Adrenal Steroid Synthesis
18 Nevanimibe Targets Starting Point of Adrenal Steroid Synthesis Able to inhibit all steroidogenic pathways Livoletide Nevanimibe Congenital Adrenal Hyperplasia (CAH): Inactive 21-hydroxylase leading to elevated androgens and precursors X X
Slide 18: CAH Has Lifelong Impacts
19 CAH Has Lifelong Impacts Results from excess androgens or excess cortisol caused by over or undertreatment Arlt, Endocrine Research, 2010 Jenkin-Jones, European Journal of Endocrinology, 2018 Hagenfeldt, Human Reproduction, 2008 Finkielstain, Endocrine Care, 2012 Falhammar, European Journal of En
Slide 19: Nevanimibe Proof of Concept Established in CAH
20 Nevanimibe Proof of Concept Established in CAH Activity demonstrated through reductions in 17-OHP Livoletide Nevanimibe P P Mean change in 17-OHP by Study Visit (V) 125 mg BID 250 mg BID 500 mg BID 750 mg BID 1000 mg BID Visits 1-4 n=10; Visits 5-7 n=9; Visits 8-13 n=8 17-OHP (ng/dL) nevanimibe p
Slide 20: Nevanimibe CAH Phase 2b Study Overview
Phase 2b CAH study includes further dose ranging and 3 month continuous dosing Study initiated in 3Q18 21 Nevanimibe CAH Phase 2b Study Overview Intrasubject dose escalation with 3 months continuous dosing Livoletide Nevanimibe 3 MONTHS OF CONTINUOUS nevanimibe DOSING Primary Endpoint 17-OHP < 2x UL
Slide 21: Nevanimibe CAH Phase 2b Study Overview
Nevanimibe CAH Phase 2b Study Overview Intrasubject dose escalation with 3 months continuous dosing Primary n=20-24 +. 8 Mons oF continuous —______», Endpoint NEVANIMIBE DOSING ~10 sites 1000 mg BID, 1500 mg BID and 2000 mg BID ‘Dose tration based on 17-OHP levels Phase 2b CAH study includes further
Slide 22: Significant Potential Value Creation
3Q18 Nevanimibe Phase 2b CAH study initiation 4Q18 Public listing (MLND) and financing with total proceeds of $85M 1Q19 Livoletide pivotal Phase 2b/3 PWS study initiation 2Q19 Establish commercial organization in Boston area 2H19 Nevanimibe Phase 2b CAH study update 1H20 Nevanimibe Phase 2b CAH stud
Slide 23: Millendo is a Leading Company in Rare Endocrine Diseases
Pursue development of, obtain approval for, and commercialize nevanimibe for the treatment of CAH Continue to expand our pipeline by leveraging our expertise in in-licensing and acquiring product candidates Build a specialized sales and marketing organization in the United States targeting endocrino
Slide 24: Millendo is a Leading Company in Rare Endocrine Diseases
=r Millendo is a Leading Company in Rare Endocrine Diseases mid Novel agents in orphan diseases with significant unmet needs Rapidly and efficiently advance development of, obtain approval for, and ‘commercialize livoletide for the treatment of PWS Pursue development of, obtain approval for, and com
Slide 25: Millendo Therapeutics
»< YY MILLENDO THERAPEUTICS
Millendo Therapeutics — Investor Presentation (January 2019) on 6ix