Soleno Therapeutics, Inc. · NASDAQ:SLNO
Soleno Therapeutics — Investor Presentation (July 2023)
33 slides from Soleno Therapeutics's investor presentation, Jul 10, 2023
Slide 1: Corporate Presentation July 2023
Exhibit 99.1 Corporate Presentation July 2023 | 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: Strategic Highlights
Soleno Therapeutics (NASDAQ: SLNO) Strategic Highlights Orphan asset in Phase 3 Orphan Drug Financed by >$1bn PWS Program for and IP protection to leading global market Prader-Willi mid-2030s Fast Track healthcare opportunity Designations investors syndrome (PWS) Topline data Protected by Orphan Add
Slide 4: Leadership Team
Leadership Team • Anish Bhatnagar, M.D. • Jim Mackaness Chief Executive Officer Chief Financial Officer • Neil M. Cowen, Ph.D. • Scott Madsen Senior VP, Drug Development VP, CMC • Patricia C. Hirano, M.P.H. • Charles Horn VP, Regulatory Affairs VP, Quality • Kristen Yen, M.S. VP, Clinical Operations
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 • Birth incidence ~1:15,000 births • Elevated mortality rates with mean age of death ~21 - 30 years • Highest unmet needs – Hyperphagia – Low lean
Slide 6: DCCR Was Developed to Facilitate Once Daily Dosing and Improve Response
DCCR Was Developed to Facilitate Once Daily Dosing and Improve Response § Choline salt chosen to improve § Titration and dosing optimized to solubility safely reach target dose and § Formulation developed to extend maintain therapeutic response absorption throughout the GI tract 30 25 § DCCR dosed o
Slide 7: Mechanism of Action in PWS
Mechanism of Action in PWS Genes, 11(4), 450. https://doi.org/10.3390/genes11040450. 7 | © 2023 Soleno Therapeutics
Slide 8: DCCR Phase 3 Clinical Program Design
DCCR Phase 3 Clinical Program Design • C601 (DESTINY PWS): Multi-center, randomized, double-blind, placebo- controlled, parallel arm study in patients with PWS (Phase 3) • C602: Open-label safety extension study C601 2-week C602 13-Week Double-blind Run-in Open-label Extension Treatment DCCR Complet
Slide 9: C601 Primary and Key Secondary Endpoints
C601 Primary and Key Secondary Endpoints All Data Data through March 1, 2020 DCCR Placebo DCCR Placebo Primary Endpoint (N = 82) (N = 42) (N = 82) (N = 42) Mean (SE) Change from Baseline in Hyperphagia at -5.94 -4.27 -6.64 -3.51 Visit 7 (0.88) (1.15) (1.00) (1.28) LS Mean Difference [DCCR-Placebo] (
Slide 10: C601 Behavioral Endpoints
C601 Behavioral Endpoints DCCR vs Placebo PWSP Domain p-value Aggressive Behaviors 0.048 Anxiety 0.018 Rigidity, Irritability 0.003 Compulsivity 0.008 Depression 0.185 Disordered Thinking 0.011 DBC-2 Total Score 0.009 Communication Disturbance 0.003 Social Relating 0.008 Observed values through Marc
Slide 11: C601 Key Hormonal and Metabolic Markers
C601 Key Hormonal and Metabolic Markers DCCR vs Placebo p-value Change from Baseline at Week 13 Decreased Acylated Ghrelin (active form) 0.0182 Decreased Leptin <0.0001 Decreased Insulin 0.0110 Increased Adiponectin <0.0001 11 | © 2023 Soleno Therapeutics
Slide 12: Long-term Data DCCR Interim Results and Comparison to PATH for PWS
Long-term Data DCCR 12 MONTHS INTERIM RESULTS AND COMPARISON TO PATH FOR PWS
Slide 13: C601/C602 and PATH for PWS (PATH)
C601/C602 and PATH for PWS (PATH) • C602 is an ongoing, open-label extension study of DCCR in subjects who completed DESTINY PWS successfully • PATH is an ongoing study evaluating the natural history of subjects with PWS – Sponsored by FPWR – ~ 650 active participants – Completion of several questio
Slide 14: C601/C602 Hyperphagia Change from Baseline
C601/C602 Hyperphagia Change from Baseline Weeks of Exposure to DCCR 0 13 26 39 52 0 -1 -2 -3 -4 * p < 0.0001 -5 -6 -6.5 * -7 -8 * -8.8 * -9 -9.8 * -9.9 -10 -11 -12 14 | © 2023 Soleno Therapeutics LSMean (±SE) Change from Baseline in HQ-CT Score
Slide 15: Change in Hyperphagia with DCCR Compared to PATH
Change in Hyperphagia with DCCR Compared to PATH 26 Weeks 52 Weeks PATH DCCR PATH DCCR (N = 229) (N = 108) (N = 138) (N = 106) 0 -2 -4 -6 -8 -10 p < 0.001 p < 0.001 -12 15 | © 2023 Soleno Therapeutics LS Mean (± SE) Change from Baseline
Slide 16: C601/C602 PWS Profile Behavioral Change Results after One Year of DCCR
C601/C602 PWS Profile Behavioral Change Results after One Year of DCCR Domain p-value Aggressive Behaviors <0.0001 Anxiety <0.0001 Compulsivity <0.0001 Depression <0.0001 Disordered Thinking <0.0001 Rigidity Irritability <0.0001 16 | © 2023 Soleno Therapeutics
Slide 17: C601/C602 Comparison to PATH – Change in Behaviors at Week 52
C601/C602 Comparison to PATH – LS Mean Change in Behaviors from Baseline at Week 52 Aggressive Rigidity/ Disordered Behavior Anxiety Irritability Compulsivity Depression Thinking (p<0.001) (p<0.001) (p<0.001) (p<0.001) (p=0.003) (p<0.001) 0.3 0.0 -0.1 -0.2 -0.3 -0.3 -1.0 -1.4 -2.0 -3.0 -3.0 -3.1 PAT
Slide 18: Endocrine and Hormonal Parameters After One-Year of DCCR
Endocrine and Hormonal Parameters After One-Year of DCCR Mean change from Baseline at 1 Year p-value Decreased Leptin <0.0001 Decreased Insulin 0.0004 Decreased HOMA-IR 0.0033 Increased Adiponectin <0.0001 18 | © 2023 Soleno Therapeutics
Slide 19: Impact of DCCR
Impact of DCCR • Photos provided with consent of the DCCR study participant’s caregiver through University of Florida, USA • Changes not representative of all participants • Changes occurred over 12 or more months of DCCR once daily 19 | © 2023 Soleno Therapeutics
Slide 20: DCCR Safety Profile
DCCR Safety Profile • ~100 patients treated for more than one year • Safety profile generally consistent with the known profile of diazoxide and prior experience with DCCR • The most common adverse events reported were hypertrichosis, peripheral edema and hyperglycemia • Most events were Grade 1 or
Slide 21: Regulatory Status and C602 Randomized Withdrawal Period
REGULATORY STATUS AND C602 RANDOMIZED WITHDRAWAL PERIOD
Slide 22: DCCR Regulatory Status
DCCR Regulatory Status • As a result of DESTINY PWS (C601) not meeting its primary endpoint in the overall analysis, FDA stated that additional controlled data are necessary to support an NDA submission – In June 2023, the FDA acknowledged that data from a proposed randomized withdrawal period of C6
Slide 23: DCCR Phase 3 Updated Clinical Program
DCCR Phase 3 Updated Clinical Program • C601 (DESTINY PWS): Multi-center, randomized, double-blind, placebo-controlled, parallel arm study in patients with PWS (Phase 3) • C602: Study with open-label extension (OLE) and randomized withdrawal (RW) periods • C614: Open-label safety extension study C60
Slide 24: Randomized Withdrawal Status
Randomized Withdrawal Status • Randomization completed May 2023 – 77/83 (93% of participants) randomized – 6 of 83 total participants in the study (6%) declined to participate, in line with our expectation of non- participation • Top-line data continue to be expected in third quarter 2023 24 | © 202
Slide 25: Scientific Outreach & Community Engagement
Scientific Outreach & Community Engagement Increasing levels of engagement with PWS community, physicians and advocacy groups Posters and Independent FPWR Independent town presentations at and PWSA| hall meetings with Growing body of medical and USA petition signed study participants clinical eviden
Slide 26: Running for Research Funded, Investigator-Sponsored Study in Early Phase PWS
Running for Research Funded, Investigator- Sponsored Study in Early Phase PWS • Rationale for the research study – Biomarker changes in Soleno Phase 3 program suggest DCCR may delay or prevent progression of PWS from early phases to hyperphagic phase • Study exclusively funded by Running for Researc
Slide 27: Extensive IP Protection
Extensive IP Protection Three families of patents being prosecuted in all major pharma markets – primary cases on all three issued Pharmaceutical formulations of Salts of K channel activators Methods to treat PWS ATP K channel activators and uses and uses thereof ATP Specific claims to behavioral th
Slide 28: Commercial Supply Chain
Commercial Supply Chain Establishing supply chain to deliver DCCR to patients • Preparing for validation of commercial scale for drug product • Production at established, GMP-compliant CMOs • Third-party Specialty Pharmacy and Hub Services being established Finished Drug Specialty Pharmacy / Bulk Ma
Slide 29: Financial Information
FINANCIAL INFORMATION S4soLeno Ine ‘
Slide 30: Up to $60m financing commitment
Up to $60m financing commitment • Leading Healthcare Investors - Nantahala, Abingworth and Vivo Capital – $10m RECEIVED May 2023 upon completion of enrollment in RW study – $15m at positive top line data - Tranche A ~8.6m at $1.75 – $35m after FDA approval - Tranche B 14m at $2.50 • Total $60m for 2
Slide 31: Financial Highlights
Financial Highlights Pro Forma Cash and Equity Cash $ million Shares Outstanding million* June 30, 2023* 19.4 18.0 Positive top line data** +15.0 26.6 FDA approval*** +35.0 40.6 * 11.2m common outstanding, March 2022 prefunded warrants + options and other warrants ** Includes exercise of 8.6m Tranch
Slide 32: Pipeline – Other Opportunities for DCCR
Pipeline – Other Opportunities for DCCR Potential Upside Opportunities for DCCR Estimated US Prevalence SH2B1 deficiency obesity 2,255 – 3,376 SIM1 deficiency obesity 1,747 – 2,572 Early onset morbid obesity associated with PCSK1 deficiency < 350 Chronic Hyperinsulinism 820 – 1,100 Glycogen Storage
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Corporate Presentation July 2023| Soleno Therapeutics
Soleno Therapeutics — Investor Presentation (July 2023) on 6ix