Backed by Medicxi, Novartis vets launch rare blood disorder biotech with fresh take on drug class used in cancers
22 Aug 2022
In 2017, two Novartis clinicians — Pierre Saint-Mezard and Damien Picard — approached Nicholas Benedict, who’d left Novartis in 2007 and went on to head a series of biotechs, with an idea: use Akt inhibitors, typically used to treat cancers, for rare diseases that cause blood vessel malformations.
The three of them founded Vaderis Therapeutics in 2019 out of Basel, Switzerland, with CHF 18 million (just under $19 million USD) in backing from Medicxi Ventures, and are launching from stealth today as they take their idea, now in drug form, into the clinic for an 80-patient proof-of-concept study.
Having started their company right at the cusp of the pandemic, Benedict recalls his last business trip before lockdown was to London, to secure funding for Vaderis from Medicxi. They finalized the deal in April, making Vaderis Medicxi’s “first pandemic investment,” Medicxi partner Giovanni Mariggi said.
Akt sits at the center of a number of important cell functions, including survival, growth and death. In cancer, AktAkt inhibitors put a block in that pathway, stopping tumor cells from multiplying and causing them instead to die. The Vaderis trio wants to apply that concept to rare blood vessel diseases, starting out with hereditary hemorrhagic telangiectasia, or HHT for short.
Patients with HHT have blood vessels that grow abnormally, sometimes without the necessary capillaries, leading to excessive bleeding episodes and anemia, and current treatments focus mainly on treating the symptoms associated with the disease. With an Akt inhibitor, Vaderis hopes it can address abnormal blood vessel overgrowth associated with the disease.
However, when looking for an Akt inhibitor for HHT, Benedict said that they didn’t want to repurpose a cancer drug. “What we found fairly early in was that many, if not most, of the Akt inhibitors that are available or are being studied right now are not suitable for repurposing, and they’re not suitable for non-oncological indications, largely because of the toxicities that they hold,” Benedict said.
That led them to Almac Discovery, from which they acquired a portfolio of new Akt inhibitors.
Mariggi echoed the safety point. He noted other points on the pathway that Akt is part of that could be blocked, but added that those treatments come with serious side effects. “When you’re an HHT patient that hopefully has a relatively long life ahead of you, they’re just unacceptable,” Mariggi said, referencing the side effects.
But with an Akt inhibitor, “we felt that it was going to have a much better tolerability and therefore be more suited to chronic use than using a PI3 kinase inhibitor or an mTOR inhibitormTOR inhibitor,” Mariggi said.
Alongside its launch from stealth, Vaderis will begin a clinical trial to test the safety of its drug, dubbed VAD044, in 80 patients across the US, Canada, and Europe. Benedict says the biotech has enough funding to see it through the clinical trial, which is set to read out in 2024, according to clinicaltrials.gov.
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