Saniona’s focus is on the discovery, development and delivery of proprietary product candidates for the treatment of rare diseases with high unmet medical need.
INVESTMENT RATIONALE
1. Tesomet: Phase 2b trials for two rare disorders voluntarily paused, subject to additional funding
Hypothalamic obesity (HO) – Impacts <65,000 in US and Europe; no approved treatments
Prader-Willi syndrome (PWS) – Impacts < 84,000 U.S. and Europe; no treatments for hyperphagia
2. Proprietary ion-channel drug discovery engine driving pipeline
SAN711: For rare neuropathic disorders, in Phase 1
SAN903: For rare inflammatory/fibrotic disorders (e.g. IPF), in preclinical
Library: 20,000 proprietary ion channel modifiers
3. Validation from multiple strategic partnerships
 |  |  |
| Medix: Tesofensine for obesity | Novartis: CAD-1883 for movement disorders | Boehringer Ingelheim: Novel target for schizophrenia |
3. Validation from multiple strategic partnerships
|
|
Boehringer Ingelheim: Novel target for schizophrenia |
Why rare diseases?
We made the strategic decision to focus on rare diseases because of the tremendous unmet need: there are an estimated 7,000 rare diseases, and less than 10% have FDA-approved treatments. Additionally, clinical trials and regulatory reviews of medicines for rare diseases can potentially require less time and/or less financial investment than in more common disorders, and the commercial infrastructure required to serve rare patient populations is generally smaller, making rare diseases better aligned with Saniona’s capabilities.