Soleno Therapeutics, Inc. · NASDAQ:SLNO
Soleno Therapeutics — Investor Presentation (September 2017)
29 slides from Soleno Therapeutics's investor presentation, Sep 26, 2017
Slide 1: Corporate Presentation September 2017
Corporate Presentation September 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 • Jonathan Wolter Interim Chief Financial Officer • Neil M. Cowen, Ph.D. Senior VP, Drug Development • Kristen Yen, M.S. VP of Clinical Operations • Patricia C. Hirano, M.P.H. Head of Regulatory Affairs & Quality | © 2017 Soleno Therape
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 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: Proglycem Challenges in the Treatment of PWS
Proglycem Challenges in the Treatment of PWS • Proglycem has not been approved for use in PWS – Use in PWS blocked by allowed DCCR patent claims • Proglycem product is a TID/BID oral suspension with a long bitter aftertaste – There are challenges with the uniformity of dosing with the oral suspensio
Slide 8: Mechanism of Action
Orexigenic Signals: Ghrelin Anorexigenic Signals: Leptin, Insulin KATP Channels Snord116 Deletion • Increased activation of orexigenic pathways • Reduced activation of anorexic pathways Mechanism of Action 8 • Appetite controlled by 2 sets of neurons in the hypothalamus that express KATP channels –
Slide 9: 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 ages 10 to 22 years – Included subjects with mild hyperphagia (Baseline hyperphagia score < 13) and with moderate-to-severe hyperphagia (Baseline hyperphagia scor
Slide 10: Hyperphagia Response During Open-Label Treatment
Hyperphagia Response During Open-Label Treatment Greater at Highest Dose and Moderate-Severe Hyperphagia | © 2017 Soleno Therapeutics 10 Presented by Soleno 10th International Meeting of Pediatric Endocrinology 14 Sep 2017 -10 -8 -6 -4 -2 0 Overall (n=11) Moderate - Severe Hyperphagia (n=6) Dose ≥ 4
Slide 11: Placebo Reverses DCCR Treatment Effect
Placebo Reverses DCCR Treatment Effect Patients with Moderate-Severe Hyperphagia | © 2017 Soleno Therapeutics 11 -40% -30% -20% -10% 0% 0 14 28 42 56 70 84 98 DCCR (n = 3) Placebo (n = 3) Study Day M e d ia n P e rce n t Ch a n g e f ro m B a s e lin e Visit 8 Visit 7
Slide 12: Hyperphagia Questionnaires
Hyperphagia Questionnaires Comparison of HQ-CT to Modified Dykens Used in PC025 | © 2017 Soleno Therapeutics 12 Question How upset did the person generally become when denied a desired food? How often did the person try to bargain or manipulate to get more food at meals? How often did the person for
Slide 13: Response to Common Hyperphagia Questions
Response to 6 Common Hyperphagia Questions 2 Doses of Beloranib (ZAF-311) and DCCR (PC025) | © 2017 Soleno Therapeutics 13 -2.0 -1.5 -1.0 -0.5 0.0 Upset when denied food Bargain for food Forage in trash Up at night Persistent asking for food Steal food Response by HQ-CT question DCCR ≥ 4.2 mg BL ≥ 1
Slide 14: DCCR Impacts Body Fat and Lean Body Mass
DCCR Impacts Body Fat and Lean Body Mass | © 2017 Soleno Therapeutics 14 Presented by Soleno 10th International Meeting of Pediatric Endocrinology 14 Sep 2017 -2500 -1250 0 1250 2500 3750 M e a n Ch a n g e f ro m B a s e lin e ( g ) Overall (n=11) Dose ≥ 4.2 mg/kg (n=4) p=0.02 p=0.003 p=0.059 p=0.0
Slide 15: Waist Circumference
Waist Circumference Significant Reduction from Baseline-V7 | © 2017 Soleno Therapeutics 15 • Reduction consistent with a loss of visceral fat. 106 108 110 112 114 Baseline (n = 11) Visit 7 (n = 11) Waist Circumference (cm) - 3.45 cm (p=0.006) Presented by Soleno 10th International Meeting of Pediatr
Slide 16: DCCR Reduces Aggressive Behaviors
DCCR Reduces Aggressive Behaviors | © 2017 Soleno Therapeutics 16 Presented by Soleno 10th International Meeting of Pediatric Endocrinology 14 Sep 2017 0 2 4 6 8 Screaming/Yelling Throwing Objects Aggressive/Violent Actions Foul Language Destructive Threatens to Hurt Others OVERALL Baseline Visit 7
Slide 17: DCCR Impacts CV Risk Factors
DCCR Impacts CV Risk Factors | © 2017 Soleno Therapeutics 17 -0.6 -0.4 -0.2 0 0.2 Triglycerides Total-C LDL-C HDL-C Non-HDL-C Mean Change from Baseline to V7 (mmol/L) p=0.039 p=0.244 p=0.030 p=0.088 p=0.169 Presented by Soleno 10th International Meeting of Pediatric Endocrinology 14 Sep 2017
Slide 18: DCCR Safety
DCCR Safety Consistent with Long History of Safe Use of Diazoxide • Safety profile of Proglycem in chronic use is well-known • In the development of DCCR, there have been no new safety findings – Most common adverse events include hyperglycemia and peripheral edema • Doses of DCCR that will continue
Slide 19: FDA Guidance Meeting (May 2017)
FDA Guidance Meeting (May 2017) • FDA supported change in hyperphagia score (without a change in weight) compared to placebo as the primary endpoint for the planned Phase III study. • Dosing paradigm proposed for the Phase III study was acceptable. • FDA proposed and Soleno agreed that the duration
Slide 20: EMA Scientific Advice (September 2017)
EMA Scientific Advice (September 2017) • EMA support change in hyperphagia score compared to placebo as the primary endpoint for the Phase III program • Single Phase III study acceptable • Treatment of children with hyperphagia acceptable without additional toxicology study(ies) • Dosing paradigm pr
Slide 21: Phase III Proposed Study Design
Phase III Proposed Study Design • Patients will be randomized in a 2:1 ratio to DCCR or placebo – Mod-severe hyperphagia, target dose ~4.2 mg/kg • Expected study start Q4 2017, 9 - 12 months duration • Primary endpoint – change in hyperphagia compared to placebo • All patients completing C601 are el
Slide 22: DCCR Estimated Development Timeline
DCCR Estimated Development Timeline | © 2017 Soleno Therapeutics 22 2017 2018 2019 CMC, Pharm/Tox, Clin Pharm Phase III study Open label Extension FDA guidance meeting NDA Submission Topline data from Phase III EMA Scientific Advice
Slide 23: Extensive IP Protection
Extensive IP Protection • Issued/Granted Patents – US: 5; EU: 3; JP: 1; – Also in China, India, Canada and Australia – Several pending applications – Expire in 2026 to 2035 – Covers composition of matter, formulations, combinations, method of use and method of manufacture • Protection in PWS – In ad
Slide 24: Pipeline – Orphan Opportunities
Pipeline – Orphan Opportunities | © 2017 Soleno Therapeutics 24 Product Indication US Population Estimate DCCR Prader-Willi syndrome 12,500 – 21,000a Potential Upside Opportunities for DCCR DCCR Hypothalamic Obesity 3,750 – 9,700b DCCR Smith-Magenis Syndrome 12,500 – 21,000c a Pediatrics 2011 127:19
Slide 25: 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 26: 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 27: 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 • August 2017 – Complete EMA Scientific Advice • 2H17 – Initiate Phase III
Slide 28: 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 III clinical trial Cash runway to value creating milestones (millions) 6/30/2017 Cash $7.5 Debt $0 Shares outstanding: Common 47.51 F
Slide 29: Investment Highlights
Investment Highlights | © 2017 Soleno Therapeutics 29 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 D
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