Aug 31, 2022

Lei Lei Wu News Reporter 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. Keep reading Endpoints with a free subscription Unlock this story instantly and join 149,100+ biopharma pros reading Endpoints daily — and it's free. SIGN UP Paul Schloesser Associate Editor Whenever research and development into new Parkinson’s drugs are brought up, people tend to think of failure — for good reason. There have been years upon years of clinical trial flops, and existing therapies and brain stimulation devices bring only moderate relief. While some investors wrote off Parkinson’s as a lost cause, companies, researchers and outside observers are more optimistic. In recent years, the field gained a better understanding of the genetic basis of the disease, and drugs targeting new pathways advanced into clinical trials, including one program in late-stage testing. The need for treatments is acute, with a surge in worldwide diagnosis for Parkinson’s, a disease marked by symptoms like tremors and slowed movement, in what researchers bill as an epidemic. Premium subscription required Zachary Brennan Senior Editor As part of an attempt to halt a likely new surge of hospitalizations and deaths from Omicron’s BA.5 subvariant this fall, the FDA on Wednesday authorized both Pfizer/BioNTech and Moderna’s bivalent boosters, which aim to attack this latest version of the deadly coronavirus, which has taken more than one million lives since March 2020. But the authorizations of these updated boosters for those 12 and older — which partly includes an mRNA component of the original coronavirus strain from Wuhan, China, and also an mRNA component from the BA.4 and BA.5 lineages — didn’t come with any new human data, leaving critics to question if the new shots are actually more effective at preventing hospitalization or death than the original vaccines from both companies. Keep reading Endpoints with a free subscription Unlock this story instantly and join 149,100+ biopharma pros reading Endpoints daily — and it's free. SIGN UP Max Gelman Senior Editor Looking to build out a case for its drugs that can better penetrate the blood-brain barrier, Denali Therapeutics touted new interim Hunter syndrome data Wednesday morning. And execs are sharpening their regulatory pitch. The South San Francisco biotech reported longer-term results from a Phase I/II study evaluating DNL310, saying the safety profile remained similar to the current standard of care — enzyme replacement therapy — after 85 weeks. Additionally, a larger group of patients gives the company more confidence that early biomarker results from July 2021 indicate the drug is positively affecting Hunter syndrome patients. Keep reading Endpoints with a free subscription Unlock this story instantly and join 149,100+ biopharma pros reading Endpoints daily — and it's free. SIGN UP John Carroll Editor & Founder A microcap biotech player operating under a “going concern” alert is touting a tie-up with Bayer’s BlueRock, a high-profile outfit exploring the frontiers of cell therapy R&D. Sunnyvale, CA-based BioCardia says it nabbed a “sizable” upfront in the pact, which will put a catheter-based system it devised to deliver a patient’s bone marrow cells to heart muscle to use in BlueRock’s cardio work. BioCardia reported it won a breakthrough device award from the FDA on that. Keep reading Endpoints with a free subscription Unlock this story instantly and join 149,100+ biopharma pros reading Endpoints daily — and it's free. SIGN UP Amber Tong Senior Editor Lumakras, Amgen’s groundbreaking KRAS drug, was approved on data from an open-label, single-arm study suggesting, among other things, median progression-free survival of 6.8 months for patients with KRASG12C-mutated non-small cell lung cancer. Now, Amgen says it has the first proof that Lumakras beats chemotherapy — the current standard of care — on that metric. Further analyses are still ongoing and data will be reserved for future medical conferences, Amgen said, remaining tight-lipped. What it will say is that the drug, formerly known as sotorasib, met the primary endpoint of PFS in the CodeBreaK 200 trial, where it went head to head with intravenous docetaxel. Among 345 patients, Lumakras demonstrated “statistical significance and superiority” over the chemo. Keep reading Endpoints with a free subscription Unlock this story instantly and join 149,100+ biopharma pros reading Endpoints daily — and it's free. SIGN UP