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Good morning, and welcome back to Endpoints Weekly. Some of our biggest news this week came out of DC, where Pfizer CEO and incoming PhRMA chair Albert Bourla spoke in support of the Trump administration. We’ve got more details on his comments below, and Endpoints News’ Max Bayer has the latest on the postponement of a meeting for the CDC’s Advisory Committee on Immunization Practices. During the meeting, advisors were scheduled to discuss at least 10 vaccines against Lyme disease, RSV, flu and Covid-19.
Senior Biopharma Correspondent Andrew Dunn tracked several announcements from the AI field this week, including the release of a new AI biology model from Nvidia and the Arc Institute, and Atomwise’s hiring of a new CEO.
If your weather has been as cold as Boston’s, it’s the perfect opportunity to grab a cup of tea and spend some time with Ryan Cross’ deep dive on the ups and downs of the CRISPR field. It’s the first story in an ongoing series about the future of CRISPR, so stay tuned for more. |
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Nicole DeFeudis |
Editor, Endpoints News |
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Bourla praises Trump as RFK Jr. preps vaccine review |
🗣️Pfizer CEO Albert Bourla, the incoming PhRMA chair, made some of the industry’s strongest comments in favor of President Donald Trump’s new administration this week. Bourla described Trump as someone who will bring change and be more in favor of industry than former President Joe Biden. Bourla made his comments at the PhRMA Forum, a gathering of industry professionals brought together by the lobbying group, in Washington, DC.
“I truly believe the opportunities clearly outweigh the risks, because this is an administration, to start with, that they believe that strong business, strong private sector, strong entrepreneurship makes America great,” Bourla said.
There was also much left unsaid at the event, and not just from Bourla. There were no mentions of the mass firings taking place at HHS, FDA, CDC and other health agencies, the controversial attempt to slash NIH overhead payments for research, or HHS Secretary Robert F. Kennedy, Jr.’s long history of anti-vaccine views and actions. The wave of departures extended to Celia Witten, the deputy director of CBER, whose exit was reported Friday afternoon.
Before this week, much of the biopharma industry had remained silent on the topic of Trump, and it remains to be seen whether Bourla’s comments will prompt others to do so. But former FDA Commissioner Rob Califf slammed the cuts across HHS as “haphazard, poorly thought-out.” Califf became one of the highest-profile individuals to criticize the layoffs, against the backdrop of largely quiet drugmakers. Califf returned to Duke earlier this month working as a cardiologist in the university’s health system.
Elsewhere in Washington, Kennedy is getting ready to launch a review of the childhood vaccine schedule, despite pledging during his confirmation hearings that he would not dig into vaccine safety or alter CDC recommendations. Sen. Bill Cassidy (R-LA), who was considered one of the biggest Republican swing votes, assented to Kennedy’s confirmation after receiving certain pledges that he wouldn’t change existing recommendations from the ACIP committee. But ACIP’s first meeting of Kennedy’s tenure, scheduled for this week, was postponed. |
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Will CRISPR matter? |
🔬Gene editing companies are struggling. Despite a landmark approval for the first CRISPR therapy in 2023, gene editing companies are still dealing with commercial challenges, scientific hurdles and limited interest from investors. Startups have found it difficult to raise money, and companies across the sector have cut staff and trimmed pipelines, Endpoints’ Ryan Cross reported this week as part of an ongoing series.
Gene editing’s tough year was evidenced by multiple shutdowns, including MIT-spinout Tome Biosciences and SalioGen Therapeutics. The latest is Spotlight Therapeutics, a startup based on science from UC Berkeley and UCSF that was devoted to delivering CRISPR therapies to the right target tissues.
Is gene editing stuck in the “trough of disillusionment?” Some experts think so, referring to a model commonly used to describe how breakthroughs progress. According to a recent report by the investment bank William Blair, gene editing stocks fell 56% last year.
But companies like CRISPR Therapeutics remain confident in gene editing’s potential to treat a range of diseases, from cardiovascular conditions to cancer. At least seven companies are developing therapies that could permanently lower cholesterol or harmful fats.
“This is just the early innings of CRISPR,” CRISPR Therapeutics’ CEO Samarth Kulkarni told Endpoints. He said CRISPR and Casgevy could be a “seminal moment that kicked off an entire new area of medicine.” |
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Atomwise hires new CEO as Nvidia and the Arc Institute drop AI bio model |
🖥️Atomwise hired Steve Worland, an industry veteran with over 35 years of biotech experience, as its next CEO. The San Francisco-based company is best known for its bold talk about its AI technology, AtomNet. But after years of raising capital and signing partnerships, the company has yet to put a drug in the clinic.
Worland wants to change that, and he’ll have help from a previously unreported $45 million Series C round that the company closed last March. Atomwise has a TYK2 inhibitor in IND-enabling studies, but Worland declined to provide a timeline to start human testing.
In other AI news, Nvidia and the Arc Institute dropped what they’ve said is the largest publicly available AI biology model to date. The model, called Evo 2, was trained on roughly nine trillion base pairs of DNA from over 128,000 different species of bacteria, archaea, and eukaryotes.
“From predicting how single DNA mutations affect a protein’s function to designing genetic elements that behave differently in different cell types, as we continue to refine the model and researchers begin using it in creative ways, we expect to see beneficiary uses for Evo 2 we haven’t even imagined yet,” Arc Institute’s chief technology officer Dave Burke said in a statement.
Nvidia’s VC arm is backing Achira, a startup that launched this week with a $33 million seed round. Achira is looking to build the next generation of computational tools for drug discovery, combining AI- and physics-based methods. |
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Biogen bets on Dravet syndrome |
🤝Biogen paid $165 million upfront for a drug from Stoke Therapeutics this week, an antisense oligonucleotide called zorevunersen that’s expected to enter a Phase 3 study before the end of June. If everything goes according to plan, Stoke could get up to $385 million in milestones and royalties on the drug’s sales. The deal came about a month after Biogen said it would focus its research efforts primarily on “external opportunities.”
Dravet syndrome is a rare, genetic form of epilepsy that is extremely severe and usually manifests before a patient turns one year old. Zorevunersen’s biggest competitor is likely to come from Lundbeck, which picked up a 5-HT2C superagonist in late-stage development in its acquisition of Longboard Pharmaceuticals last October. Zorevunersen takes a much different approach, however, targeting the SCN1A gene.
Others recently exited the space: Takeda scrapped its Ovid-partnered program last month after it failed a Phase 3 trial, and Eisai axed a pivotal study last November. The FDA also lifted a partial clinical hold on zorevunersen back in August. |
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Has Roche cracked DNA sequencing? |
🧬That’s the question being asked after the Swiss drugmaker detailed a new DNA sequencer that it hopes can complement other diagnostic offerings. Roche is still at least a year away from a commercial release and would have to compete with market-leader Illumina, who has historically outperformed the bigger company in this market. Illumina, though, is facing its own challenges after being blacklisted by the Chinese government in retaliation for the Biosecure Act and President Trump’s tariffs. Roche said further details will be released next week.
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