Soleno Therapeutics, Inc. · NASDAQ:SLNO
Soleno Therapeutics — Investor Presentation (July 2017)
23 slides from Soleno Therapeutics's investor presentation, Jul 10, 2017
Slide 1: Corporate Presentation July 2017
Corporate Presentation July 2017 | Soleno Therapeutics
Slide 2: Certain Notices and Disclaimers
Certain Notices and Disclaimers Forward-Looking Statements This presentation contains forward-looking statements that are subject to many risks and uncertainties. Forward looking statements appear in a number of places throughout this presentation and include statements regarding our intentions, bel
Slide 3: Investment Highlights
Investment Highlights | © 2017 Soleno Therapeutics 3 Capnia and Essentialis Merger Compelling product profile Addresses hallmark symptoms of PWS, including hyperphagia Phase III ready Established, decades-long safety profile Strong IP Financing concurrent with completed merger Adequate to advance DC
Slide 4: Leadership Team
Leadership Team • Anish Bhatnagar, M.D. Chief Executive Officer • David O’Toole Senior VP, Chief Financial Officer • Neil Cowen, Ph.D. Senior VP, Drug Development • Kristen Yen, M.S. VP of Clinical Operations • Patricia Hirano, M.P.H. Head of Regulatory Affairs & Quality | © 2017 Soleno Therapeutics
Slide 5: Prader-Willi Syndrome (PWS)
Prader-Willi Syndrome (PWS) • Complex genetic neurobehavioral/metabolic disorder due to the loss or lack of expression of a set of genes on chromosome 15 • Afflicts about 1:15,000-1:25,000 individuals • Elevated mortality rates • Highest unmet needs – Hyperphagia – Aggressive behaviors – Body compos
Slide 6: Diazoxide – Long History of Safe Use; DCCR – Extensive Pre-Clinical and Clinical Data
Diazoxide – Long History of Safe Use DCCR – Extensive Pre-Clinical and Clinical Data • Diazoxide I.V., Oral suspension and Capsule – KATP channel agonist approved in 1976 – Previously used as IV treatment for malignant hypertension – BID/TID oral suspension for the treatment of hypoglycemia due to h
Slide 7: Mechanism of Action
Mechanism of Action • Appetite controlled by 2 sets of neurons in the hypothalamus – NPY/AgRP – secrete NPY and AgRP, appetite stimulatory neuropeptides – POMC – secretes POMC, an appetite suppressive neuropeptide – Express KATP channels • NPY expression is markedly elevated in PWS – Loss of SNORD11
Slide 8: PC025 Pilot Study in PWS
PC025 Pilot Study in PWS • Randomized, Withdrawal, Single-Center Study of DCCR in overweight or obese, genetically-confirmed PWS patients between 10 and 22 years | © 2017 Soleno Therapeutics 8 6 months Open-label extension N=5 Screening Open Label Treatment Phase N=13 Double-Blind, Placebo-Controlle
Slide 9: DCCR: Significant Hyperphagia Response
DCCR: Significant Hyperphagia Response | © 2017 Soleno Therapeutics 9 Hyperphagia was measured using a standard (Modified Dykens) questionnaire which assessed a range of PWS specific food-related behaviors Presented by Essentialis at the Annual Meeting of the Foundation for Prader-Willi Research on
Slide 10: DCCR Impacts Fat/Lean Body Mass
DCCR Impacts Fat/Lean Body Mass | © 2017 Soleno Therapeutics 10 Presented by Essentialis at the Annual Meeting of the Foundation for Prader-Willi Research on October 29, 2016 -7.5% -2.5% 2.5% 7.5% 12.5% 17.5% DEXA Mean Change from Baseline at 10 weeks Overall 4.2 mg/kg dose 0.0% Fat Mass Lean Body M
Slide 11: DCCR Reduces Aggressive Behaviors
DCCR Reduces Aggressive Behaviors • Based on the Behavioral Assessment Questionnaire from the Prader-Willi Syndrome Natural History Study • Aggressive and destructive behaviors – 70% of subjects at Baseline – 30% of subjects at 10 weeks (p=0.006) | © 2017 Soleno Therapeutics 11 “From a family standp
Slide 12: DCCR Impacts CV Risk Factors
DCCR Impacts CV Risk Factors -45% -35% -25% -15% -5% 5% 15% 25% Percent Change From Baseline Open-Label Treatment Phase TG Tot-C HDL-C LDL-C Non- HDL-C p=0.08 p=0.04 p=0.24 p=0.03 p=0.02 -45% -35% -25% -15% -5% 5% 15% 25% Percent Change from Baseline TG Tot-C HDL-C LDL-C Non- HDL-C Double-Blind Trea
Slide 13: Diazoxide – Long History of Safe Use; DCCR – Extensive Pre-Clinical and Clinical Data
Diazoxide – Long History of Safe Use DCCR – Extensive Pre-Clinical and Clinical Data • The safety profile of Proglycem in chronic use is well- known • In the development of DCCR, there have been no new safety findings • The doses of DCCR that will continue in development are at the low end or below
Slide 14: FDA Guidance Meeting (May 2017)
FDA Guidance Meeting (May 2017) • The FDA supported change in hyperphagia score (without a change in weight) compared to placebo as the primary endpoint for the planned Phase III study. • The dosing paradigm proposed for the Phase III study was acceptable. • The FDA proposed and Soleno agreed that t
Slide 15: Phase III Proposed Study Design
Phase III Proposed Study Design • Patients will be randomized in a 2:1 ratio to DCCR or placebo • Study start Q4 2017, 9 - 12 months duration • Primary endpoint – change in hyperphagia compared to placebo • All patients completing C601 are eligible to enroll in C602 | © 2017 Soleno Therapeutics 15 3
Slide 16: DCCR Estimated Development Timeline
DCCR Estimated Development Timeline | © 2017 Soleno Therapeutics 16 2017 2018 2019 CMC, Pharm/Tox, Clin Pharm Phase III study Open label Extension FDA guidance meeting NDA Submission Topline data from Phase III
Slide 17: Extensive IP Protection
Extensive IP Protection • Issued/Granted Patents – US: 3; EU: 3; JP: 1; – Also in China, India, Canada and Australia – Several pending applications – Expire in 2026 to 2029 – Covers composition of matter, formulations, combinations, method of use and method of manufacture • Protection in PWS – In ad
Slide 18: Pipeline – Orphan Opportunities
Pipeline – Orphan Opportunities | © 2017 Soleno Therapeutics 18 Product Indication US Population Estimate Estimated Timing to NDA DCCR Prader-Willi syndrome 12,500 – 21,000a 2019 Upside Opportunities for DCCR DCCR Hypothalamic Obesity 3,750 – 9,700b 2021 DCCR Smith-Magenis Syndrome 12,500 – 21,000c
Slide 19: Hypothalamic Obesity
Hypothalamic Obesity • Intractable weight gain and endocrine complications following damage to the hypothalamus • Most frequently follows excision of a cranial tumor, particularly craniopharyngioma • Often evident within 1-2 months of surgery • Dramatically reduced resting and voluntary energy expen
Slide 20: Smith-Magenis Syndrome (SMS)
Smith-Magenis Syndrome (SMS) • Complex genetic neurobehavioral / metabolic disorder due to haploinsufficiency of the retinoic acid-induced 1 (RAI1) gene on chromosome 17p11.2 • Key aspects of the natural history parallels PWS • Behavioral complications more prominent • Highest unmet needs: aggressiv
Slide 21: 2017 Priorities / Milestones
2017 Priorities / Milestones • 1Q17 – Closed merger transaction with Essentialis; completed concurrent $10M financing • May 2017 – Name change to Soleno Therapeutics • May 2017 – Complete FDA guidance meeting for DCCR • 2H17 – Initiate Phase III clinical study evaluating DCCR for the treatment
Slide 22: Financial Highlights
Financial Highlights 1 Does not include holdback shares of 900 thousand to be issued after 1 year and milestone shares of 4.6 million to be issued upon start of Phase II/III clinical trial Cash runway to value creating milestones (millions) 3/31/17 Cash $10.5 Debt $0 Shares outstanding: Common 47.51
Slide 23: Investment Highlights
Investment Highlights Capnia and Essentialis Merger Compelling product profile Addresses hallmark symptoms of PWS, including hyperphagia Phase III ready Established, decades-long safety profile Strong IP Financing concurrent with completed merger Adequate to advance DCCR through top-line data Multip
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