Financing for rare disease

Country

Switzerland

Switzerland-based Vaderis Therapeutics AG has raised $152 million from a Series B financing round to support development of a small molecule drug, engasertib, for a rare vascular disease for which no treatments are currently approved. The disease is hereditary haemorrhagic telangiectasia (HHT), a genetic disorder affecting the vasculature, which manifests in nose bleeds, anaemia, and visceral arteriovenous malformations (AVMs) AVMs are tangles of blood vessels that can cause internal bleeding and organ damage.

According to the journal, Genomic and Precision Medicine, the prevalence of HHT is about one in every 5,000 persons globally and therefore the disease is classified as rare. However scientists suspect that it is underdiagnosed.

The Series B financing was co-led by Life Sciences at Goldman Sachs Alternatives and TCGX, a global investment group located in California and New York, US. It will be used to advance engasertib into Phase 3 and for subsequent regulatory submissions. The US Food and Drug Administration gave engasertib a ‘fast track’ designation in late 2024, signalling the drug’s potential as a treatment for a debilitating genetic disease.

Engasertib is an allosteric inhibitor of the AKT1 and AKT2 protein kinases. Dysregulated AKT1 and AKT2 kinases disrupt normal cell growth and metabolism. The financing and initiation of the Phase 3 trial follow publication of a proof-of-concept study in the 18 February 2026 issue of The New England Journal of Medicine in which patients with HHT and treated with engasertib, showed improvement across multiple measures of the disease. 

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