Medicxi backed by Novartis veteran to launch rare blood disease biotech with novel class of drugs used in cancer – Endpoint News

In 2017, two Novartis clinicians, Pierre Saint-Mézard and Damien Picard, approached Nicolas Benedict, who retired from Novartis in 2007 and became head of a series of biotechs. For rare diseases that cause vascular malformations.

The three founded Vaderis Therapeutics in Basel, Switzerland in 2019, raising CHF 18 million (just under US$19 million) with support from Medicxi Ventures. A clinic for a proof-of-concept study of 80 patients.

Benedict, who started the company at the height of the pandemic, remembered his last business trip before lockdown took him to London and secured funding for Vaderis from Medicxi. They closed the deal in April, making Vaderis Medicxi’s “first pandemic investment,” said Medicxi partner Giovanni Mariggi.

Akt is central to many important cellular functions including survival, growth and death. In cancer, Akt inhibitors block that pathway, causing tumor cells to stop growing and instead cause the cells to die. Vadelis’ trio hope to apply the concept to rare vascular diseases, starting with hereditary hemorrhagic telangiectasia (HHT for short).

Patients with HHT have abnormally grown blood vessels and may lack necessary capillaries, causing excessive bleeding and anemia. Current treatments are primarily focused on treating the symptoms associated with this disease. Vaderis hopes that Akt inhibitors can be used to address the abnormal vascular overgrowth associated with this disease.

But when looking for an Akt inhibitor for HHT, Benedict said he didn’t want to repurpose cancer drugs. “What we discovered quite early on was that many, if not most, of the currently available or currently investigated Akt inhibitors are not suitable for repurposing and have non-oncological indications. The toxicity they have,” Benedict said.

That led them to Almac Discovery, from which they acquired a new portfolio of Akt inhibitors.

Marigi repeated the safety point. He noted other points in the pathway where Akt might be blocked, but added that these treatments are associated with significant side effects. “If you’re an HHT patient and you have a relatively long life ahead of you, that’s unacceptable,” said Mariggi, referring to the side effects.

However, using an Akt inhibitor “was much better tolerated than using a PI3 kinase inhibitor or an mTOR inhibitor, so we felt it was better suited for chronic use.”

In parallel with the launch from Stealth, Vaderis will initiate a clinical trial to test the safety of its drug, called VAD044, in 80 patients in the United States, Canada and Europe. Benedict said the biotechnology has enough funding to complete clinical trials that are expected to begin in 2024, according to

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