Non-opioid performs, Califf discusses Aduhelm, & ARPA-H gets a home

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Sponsored by        Meghana here. Plenty of news from this week's STAT Breakthrough Summit. We talk Aduhelm, we talk CRISPR, and more.  CRISPR companies shrug off IP battles Harvard and the Broad Institute emerged victorious last month when the U.S. Patent and Trademark Office deemed them the rightful owners of intellectual property key to developing CRISPR-based human therapies. But the acrimonious and years-long CRISPR patent battle has left industry players a bit ... unfazed. “I think it’s really a topic for people interested in IP law,” an executive from CRISPR Therapeutics said during the STAT Breakthrough Science Summit yesterday. “At the end of the day, there might be a modest economic impact one way or another, but when you zoom out there’s so much exciting science and innovation going on that’s going to improve people’s lives. It’s just not something to focus on, in my opinion.” Leaders from Editas Medicine and Intellia Therapeutics agreed — although intellectual property around CRISPR is growing increasingly complex. More companies from around the world are working on their own CRISPR-based therapies, and are challenging the ownership of early, foundational CRISPR patents. So a lot still remains in the air. Read more. Califf admits Aduhelm has rattled faith in FDA FDA chief Robert Califf admitted that the controversy around Aduhelm's approval has tarnished the agency’s standing among experts — though he believes the general public’s opinion of the agency remains intact. “It’s pretty clear that the controversy around this has temporarily impacted the trust in the FDA by people who pay attention these things,” Califf said during the STAT Breakthrough Science Summit. He didn’t elaborate on how the FDA plans to handle the fallout of Aduhelm’s approval under his leadership — but described the Alzheimer’s drug controversy as an opportunity to reveal systemic issues. He also spoke extensively about the accelerated approval pathway, which was used to approve the Biogen drug, emphasizing that drugmakers need to wrap up post-market studies faster than they are now. Read more. Sponsor content by Genentech The power of perseverance in neuroscience For decades, conditions of the nervous system have been some of the most complex to understand and treat. Through scientific rigor and unwavering perseverance, one biotech company is asking the tough questions of themselves, the industry and society to push the boundaries of scientific understanding and discovery. Learn more about Genentech and Roche’s commitment to scientific advancement in neuroscience. ARPA-H to be housed in NIH ARPA-H, the new biomedical research agency championed by President Biden, will remain snugly within the NIH, STAT’s Lev Facher reports. But instead of reporting to the NIH director, the new agency’s leader will instead report to the head of HHS. The decision ends a long debate over whether ARPA-H would become an independent entity. Although Biden’s team thinks that ARPA-H would work best attached to the NIH, many researchers — and Democratic lawmakers — have argued that it would be more effective if it were smaller and more lithe, like DARPA. “I have consistently urged the administration to set up ARPA-H for success on Day 1 by creating an independent, empowered, risk-taking agency,” one congresswoman said in a statement. “Regrettably, the HHS secretary has chosen the opposite by relying on a bureaucratic structure, rather than a transformational one.” Read more.   Vertex non-opioid painkiller performs well mid-stage A non-opioid, novel pain medicine made by Vertex Pharmaceuticals met its goals in two mid-stage trials. The drug, called VX-548, was tested against placebo and hydrocodone in reducing post-surgical pain. The highest dose showed a statistically significant benefit over placebo, and numerically similar over hydrocodone, in patient-reported pain scores within 48 hours of surgery. Vertex now wants to advance the drug into pivotal studies later this year, should the FDA sanction it. Drugmakers have been looking for potent, non-addictive pain meds that can be used in lieu of opioids. Companies like Biogen, Amgen, and Genentech have all developed similar medicines but have run into hurdles. Read more. More reads Targeting GSK and AstraZeneca, Clovis lifts sinking Rubraca with broad ovarian cancer win. (FiercePharma) The complete sequence of a human genome. (Science) Thanks for reading! Until next week, @megkesh Continue reading the latest health & science news with the STAT app Download on the App Store or get it on Google Play Friday, April 1, 2022       1 Exchange Pl, Suite 201, Boston, MA 02109 ©2022, All Rights Reserved. I no longer wish to receive STAT emails Update Email Preferences | Contact Us | View In Browser