Vaderis Therapeutics Raises $152M Series B to Advance AKT-Targeting HHT Drug

Vaderis Therapeutics Raises $152 Million Series B Funding

Vaderis Therapeutics, a biotechnology company developing treatments for hereditary hemorrhagic telangiectasia (HHT), has raised $152 million in Series B funding to advance its experimental drug engasertib.

The financing comes alongside the start of late-stage testing of engasertib in people with HHT, a rare inherited disorder that causes abnormal blood vessel formation.

The round was co-led by Life Sciences at Goldman Sachs Alternatives and TCGX, with participation from Omega Funds, EQT Life Sciences, Medicxi, and other investors.

Developing a Targeted Treatment for HHT

HHT is an inherited condition in which genetic mutations cause abnormal blood vessels to develop.

The condition can lead to:

• Persistent nosebleeds

• Chronic anemia

• Abnormal blood vessels on the skin

• Potential heart complications

There are currently no curative therapies for HHT. Existing approaches generally focus on managing symptoms or treating malformed blood vessels through medical procedures.

Vaderis estimates that HHT affects approximately 1 in 3,800 people in the United States.

Engasertib Targets AKT

Vaderis is developing engasertib as a targeted treatment for HHT.

The drug is designed to inhibit AKT, an enzyme involved in cell survival and growth that the company believes is hyperactive in HHT.

Engasertib specifically targets two forms of the enzyme, AKT1 and AKT2.

The company believes this more selective approach could provide therapeutic benefits while potentially avoiding some tolerability issues associated with broader AKT inhibition.

Advancing to Phase 3 Testing

Vaderis has begun a Phase 3 clinical trial evaluating engasertib in people with HHT.

According to the federal clinical trial database cited by BioPharma Dive, initial results could be available in 2028.

The company said the new funding provides sufficient capital to execute its development plans through 2029 and potentially commercialize engasertib independently if the drug succeeds in clinical testing and reaches the market.

Funding to Advance Clinical Development

The $152 million Series B will support the continued development of engasertib as Vaderis advances its late-stage clinical program.

The company is also evaluating its strategic options as the drug progresses through development.

Leadership

Vaderis Therapeutics is led by CEO Azmi Nabulsi.

Pierre Saint-Mezard serves as chief scientific officer and has described AKT1 and AKT2 as key targets for developing a potentially chronic treatment for HHT.

The company is focused on developing a more targeted therapeutic approach for a rare disease with significant unmet medical needs.