Corporate Presentation
March 2024 | Soleno Therapeutics
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, beliefs, projections, outlook, analyses or current expectations concerning, among other things, our ongoing and planned product development and clinical trials; the timing of, and our ability to make, regulatory filings and obtain and maintain regulatory approvals for our product candidates; our intellectual property position; the degree of clinical utility of our products, particularly in specific patient populations; our ability to develop commercial functions; expectations regarding product launch and revenue; our results of operations, cash needs, and spending of the proceeds from this offering; financial condition, liquidity, prospects, growth and strategies; the industry in which we operate; and the trends that may affect the industry or us.
We may, in some cases, use terms such as "believes," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should," "approximately" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Although we believe that we have a reasonable basis for each forward-looking statement contained in this presentation, we caution you that forward-looking statements are not guarantees of future performance and that our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate may differ materially from the forward-looking statements contained in this presentation.
You should also read carefully the factors described in the "Risk Factors" sections and other parts of our Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q, available at www.sec.gov, in order to better understand the risks and uncertainties inherent in our business and underlying any forward-looking statements. As a result of these factors, we cannot assure you that the forward-looking statements in this presentation will prove to be accurate. Furthermore, if our forward-looking statements prove to be inaccurate, the inaccuracy may be material. In light of the significant uncertainties in these forward-looking statements, you should not regard these statements as a representation or warranty by us or any other person that we will achieve our objectives and plans in any specified timeframe, or at all. Any forward-looking statements that we make in this presentation speak only as of the date of such statement, and we undertake no obligation to update such statements to reflect events or circumstances after the date of this presentation or to reflect the occurrence of unanticipated events.
Analyses in this presentation are preliminary and may be subject to change
2 | © 2024 Soleno Therapeutics
Soleno Therapeutics (NASDAQ: SLNO)
Strategic Highlights
NDA submission for DCCR1 in PWS2 planned mid-'24
Topline data from
randomized
withdrawal period
reported in Sep 2023
Met primary endpoint with significant improvements in hyperphagia
Decades-long safety
profile of parent molecule
IP protection to
mid-2030s
Protected by
multiple layers of
granted and
pending patents
Provides composition of
matter protection, as well as protection of formulations, and method of use
Potential for substantial patent term extension
Orphan Drug | |
and | >$2B PWS |
Fast Track | market |
Designations for | opportunity |
PWS | |
Orphan | Addresses |
designation in | the hallmark |
US and EU. Fast | symptoms |
Track granted in US | of PWS |
Significant upside | Significant commercial |
potential in other | potential in PWS, |
indications | an orphan indication with |
Orphan designation | high unmet need. |
granted for GSD1a in | No approved treatments |
US | for hyperphagia, the |
hallmark symptom of PWS |
Strong balance
sheet
Cash runway extends
beyond potential launch of DCCR
Oct 2023 ~$170M
financing
1DCCR (Diazoxide Choline) Extended-Release tablets
2Prader-Willi syndrome
3 | © 2024 Soleno Therapeutics
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 body mass/increased fat mass
- PWS-relatedbehaviors
- Families with a child with PWS have low quality of life
- Caregiver burden higher for caregivers of people with PWS than those with Alzheimer's
- Burden of care is highest after onset of hyperphagia
- Require supervised care for life
Butler MG, et al., Genet Med. 2017 Jun;19(6):635-642.
Bellis SA, et al., The Eur J Med Genet. 2022 Jan;65(1):104379. Kayadjanian N et al., PLoS One 2018 Mar 26; 12(3): e0194655.
4 | © 2024 Soleno Therapeutics
DCCR Was Developed to Facilitate Once Daily Dosing and Improve Response
- Choline salt chosen to improve solubility
- Formulation developed to extend absorption throughout the GI tract
- DCCR dosed once daily to achieve stable intraday circulating drug levels
- Strong relationship between circulating drug levels with DCCR and therapeutic responses in PWS
- Titration and dosing optimized to safely reach target dose and maintain therapeutic response
of | 30 | ||||||||||||
Circulatingconcentration diazoxide(µg/mL) | 25 | ||||||||||||
0 | |||||||||||||
20 | |||||||||||||
15 | |||||||||||||
10 | |||||||||||||
5 | |||||||||||||
0 | 2 | 4 | 6 | 8 | 10 | 12 | 14 | 16 | 18 | 20 | 22 | 24 | |
Hours Post-dose at Steady State PK015 |
5 | © 2024 Soleno Therapeutics
Mechanism of Action in PWS
Genes, 11(4), 450. https://doi.org/10.3390/genes11040450.
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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
2-week
Run-in
C601
13-WeekDouble-blind
Treatment
C602
Open-label Extension
N = 181
Screened
Enrolled N = 158
2:1 Randomization N = 127
DCCR N = 85
Placebo
N = 42
Completed
n = 120
Early
Termination
n = 7
Enrolled N = 115
As of October 2022
N=83
Mean age = 13.5 years
44.4% M / 55.6% F
20.2% UK / 79.8% US
61.3% Deletion / 37.9% Non-deletion
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C601 Primary and Key Secondary Endpoints
All Data | Observed Data through | |||||
March 1, 2020 | ||||||
Primary Endpoint | DCCR | Placebo | DCCR | Placebo | ||
(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] (SE) | -1.67 (1.29) | -3.13 (1.48) | ||||
p-value | 0.198 | 0.037 | ||||
Key Secondary Endpoints | p-value | p-value | ||||
Clinical Global Impression of Improvement at Visit 7 | 0.03 | 0.015 | ||||
(CGI-I) | ||||||
Mean Change From Baseline in Body Fat Mass (DXA) | 0.023 | 0.003 | ||||
at Visit 7 | ||||||
Caregiver Global Impression of Change at Visit 7 | 0.41 | 0.031 | ||||
(Caregiver GI-C) | ||||||
8 | © 2024 Soleno Therapeutics
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 March 1, 2020
9 | © 2024 Soleno Therapeutics
C601 Key Hormonal and Metabolic Markers
DCCR vs Placebo | |
Change from Baseline at Week 13 | p-value |
Decreased Acylated Ghrelin (active form) | 0.0182 |
Decreased Leptin | <0.0001 |
Decreased Insulin | 0.0110 |
Increased Adiponectin | <0.0001 |
10 | © 2024 Soleno Therapeutics
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Soleno Therapeutics Inc. published this content on 04 March 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 04 March 2024 14:15:19 UTC.