The Broad Institute, Inc.

Welcome back to another edition of Endpoints Weekly. Our Saturday newsletter will be off next week for Thanksgiving, so let us be the first to wish you all a happy and healthy holiday! I’m personally most excited to have my family’s homemade pumpkin pie. 😋 Things have slowed down ahead of Thanksgiving, but we still had some important news this week. FDA Commissioner Marty Makary made headlines with his comments about China trial data and how the agency is thinking about adopting President Donald Trump’s “America First” motto regarding user fees. We also had another buyout this week from Johnson & Johnson, a new gene editing paper from David Liu, and a recap of the Jefferies conference in London. And if you missed it this week, our Drew Armstrong and Andrew Dunn sat down with biopharma’s Mr. Reality Check, Derek Lowe, for an episode of Post-Hoc Live. You can watch the conversation here . See you in December! — Max Gelman FDA Commissioner Marty Makary floated the idea of higher user fees for companies operating outside the US, embracing the Trump administration’s “America first” mantra, Endpoints’ Zachary Brennan reported. “If your Phase 1 trial is not in the United States, maybe you should pay a higher user fee,” Makary said at a supply chain resiliency meeting at the National Academies of Sciences, Engineering, and Medicine late last month. The next round of user fees will be renegotiated under PDUFA VIII, which will run from 2028 through 2032. When asked whether the FDA will call for higher ex-US user fees in the negotiations over the next year, an FDA spokesperson told Endpoints that the administration “is prioritizing an America first approach to all activities, including drug development and research.” Makary also questioned the trustworthiness of Phase 3 trials coming from China. “I’m not sure we can trust a Phase 3 trial done in China. Do you trust the GDP numbers that come out of China? Why would you trust a Phase 3 clinical trial done in China?” he said at the event. Read more from Zach here. 🤝This week continued a recent uptick in mergers and acquisitions, with Johnson & Johnson agreeing to buy out Halda Therapeutics for $3.05 billion in cash. It’s J&J’s first acquisition since taking out Intra-Cellular Therapies earlier this year. Halda is working on small-molecule cancer drugs, which it calls regulated induced proximity targeting chimeras, or RIPTACs. The deal comes just a month after Halda released its first batch of clinical data in prostate cancer for its lead drug. The Halda buyout followed Merck’s announcement last Friday that it would acquire Cidara Therapeutics, which is developing flu antiviral treatments, for $9.2 billion. This week, Merck CEO Rob Davis fielded questions from analysts about the deal, saying he’s on the hunt for more . “We look forward to a future with a far more diversified set of growth drivers,” Davis said on the call. While Merck has relied on its cancer immunotherapy Keytruda to drive sales, it’s expected to fall off-patent in 2028. AstraZeneca also made a small deal for an obesity startup called SixPeaks Bio, tucking the announcement into its third-quarter earnings report earlier this month. Biotech correspondent Kyle LaHucik spoke with SixPeaks CEO Philip Just Larsen about the $170 million deal, with Larsen noting the company’s application for its first clinical trial is “imminent.” Read more here . 🧬Researchers led by the Broad Institute’s David Liu have come up with a new use for prime editing that could have potential for a range of diseases with the same root cause, Lei Lei Wu wrote . But like most promising new science, it comes with challenges. The research, published this week in Nature, describes how to use prime editing to address diseases caused by gene mutations that cut off the formation of proteins in the body. The genetic errors, called nonsense mutations, account for 24% of all disease-causing variants, according to the paper. The technique is focused on transfer RNAs, or tRNAs, which are a key molecule in the protein-making process. One major appeal of the approach is that it could use the same general solution for many different diseases. But it has yet to be studied in humans. There’s also the question of just how to deliver the therapy to the right place in the body. Fyodor Urnov, a molecular therapeutics professor at the University of California, Berkeley who was not involved in the study, called the work a “step change.” He told Lei Lei that Liu’s lab “came up with a new kind of engine, but in order for it to truly change the way we move around, whether by flight or by cars, we need to build different planes and different cars.” 💰Aspen Neuroscience locked down the $115 million Series C this week with plans to enter Phase 3 next year . The biotech is developing an autologous cell therapy treatment for Parkinson’s disease, re-engineering a patient’s own cells to fight the disease. Aspen anticipates recruiting next year and then dosing the first patient in 2027, seeking to enroll about 100 in total. Aspen got funding from OrbiMed, ARCH Venture Partners and Gilead’s Kite, among other prominent investors. The most likely competitor is Bayer-owned BlueRock Therapeutics. Aspen CEO Damien McDevitt believes his company can compete because it is developing an autologous treatment, versus BlueRock’s allogeneic method that requires immunosuppressants to help the body handle foreign cells. 🇬🇧 Check out this dispatch from our team members who attended the Jefferies Global Healthcare Conference in London. They covered Alkermes’ comments on how it views the strategic value for Avadel, Light Chain’s rationale for betting on CD47 as a promising approach in oncology, WuXi’s thoughts on building in the US, and more.

The US Food and Drug Administration has approved the first cancer drug based on discoveries from Broad Institute scientists. The drug, sevabertinib, was developed by Bayer Healthcare Pharmaceuticals in close collaboration with the Broad, and is the first FDA-approved medicine from the long-standing Broad-Bayer oncology research alliance. Sevabertinib is a new oral treatment option for a type of non-small-cell lung cancer, the most common type of lung cancer. The medicine is a pill that patients can take at home and is approved for adult patients with certain HER2 mutations in their tumors and who have already received chemotherapy or immunotherapy. This translates to roughly 4,000 to 8,000 lung cancer patients in the US every year who could potentially benefit from this new drug. Many of these patients are women, including younger women who have never smoked. The drug's approval is based on data from Bayer’s Phase I/II clinical trial, which found that over 70 percent of the patients studied in one cohort saw their tumors shrink or disappear. Many patients experienced profound and durable responses. This achievement is the result of discoveries made by Broad scientists two decades ago and of the collaboration between Broad and Bayer Pharmaceuticals, which began in 2013 . The team's clinical success demonstrates that long-term academic-industry partnerships can close the gap between biological discoveries and new medicines that improve health. "The whole idea of the Broad Institute when it was founded more than 20 years ago was to bring the power of human genome science and high-throughput research to solving human disease," said Matthew Meyerson , a lead cancer biologist on the project, institute member at the Broad, the Charles A. Dana Chair in Human Cancer Genetics at Dana-Farber Cancer Institute, and professor of genetics and medicine at Dana-Farber and Harvard Medical School. "Today, with our industry partners at Bayer, we're now able to see the fruits of these efforts in a new drug that we hope can help many patients by extending their lifespan and improving their quality of life." New option for lung cancer patients Lung cancer is the leading cause of cancer-related deaths in the US, and non-small-cell lung cancer (NSCLC) accounts for more than 85 percent of lung cancer cases. Up to 4 percent of NSCLC cases carry a HER2 gene mutation, which causes the HER2 protein to become overactive and spur abnormal cell growth. Sevabertinib halts the activity of HER2 proteins with certain mutations, inhibiting the growth of tumors. Approved as a second-line treatment, it may serve a patient population that until recently had no remaining options after chemotherapy or immunotherapy for treating their cancer. The FDA granted sevabertinib a Breakthrough Therapy designation in 2024, signifying that the compound may provide substantial improvement over existing therapies in an area of high unmet medical need. In 2025, sevabertinib was additionally granted Priority Review status , meaning the FDA would aim to take action on an application within six months (compared to ten months under standard review). A research success story This milestone began with work first published in 2005 led by Meyerson and Heidi Greulich , an institute scientist and senior group leader in the Cancer Program at the Broad. In 2005, the Broad team reported that some lung cancer patients who didn't respond to existing drugs had a mutation in their tumors known as an “exon 20 insertion” — an extra bit of DNA tucked in at a specific location in the gene EGFR . Developing a drug to target this mutation was one of the first projects for the Broad-Bayer alliance when it launched in 2013, and the work soon evolved into creating a drug to target a similar mutation in HER2 , a gene closely related to EGFR that’s involved in lung and other cancers. "We've been collaborating closely with the Bayer team on sevabertinib and its precursor compounds for 12 years now,” said Greulich. “It really shows how the power of persistence can be brought to bear on difficult problems to help patients." A Phase III clinical trial is continuing to evaluate sevabertinib as a first-line treatment option for patients with NSCLC carrying HER2 mutations. The drug is also being studied in patients with other metastatic or unresectable solid tumors, beyond lung cancer, that have HER2 mutations. "We're in an extraordinary time for the development of new medicines to treat cancer patients," said Meyerson. "And I think that, with our industry partners, we have a huge opportunity to repeat this success not once, not twice, but many, many times over." Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Contributions to this project were also made by colleagues in the Broad’s technology platforms, including the Genetic Perturbation Platform , the Center for the Development of Therapeutics , and the Proteomics Platform , and by colleagues in the Broad’s Cancer Program . What were some of the challenges you faced in developing this new medicine? HG: We encountered many challenges during the course of this project, so it wasn't smooth sailing all the way through. In fact, when we started the project, there were no cellular models available so we had to come up with a cellular assay that we could use to test early compounds. Our initial cell assay was unstable, but heroic efforts from senior research associate Bethany Kaplan helped to stabilize the assay so that it was robust enough for compound testing. In addition, our chemistry colleagues at Bayer worked for years to improve the potency, selectivity, and stability of the compounds to eventually develop sevabertinib. How did two decades of basic cancer research contribute to the development of the new drug? MM: The development of sevabertinib is a real testament to the power of long-term collaborative partnerships. Heidi and I have been working together on EGFR and HER2 for 22 years now, and we've developed a lot of knowledge of these molecules and how they behave. Before that, Bill Sellers and I worked to explore alterations of tyrosine kinase enzymes in human cancer that led to the EGFR discovery. And going back even further, Bill and Broad Institute director Todd Golub and I worked to understand what happens in the genomes of human cancers more broadly. These long-term scientific collaborations and all the biological insight that came from them, along with our deep and continued commitment to solving the same problems, have really helped us to get to where we are today with the development of sevabertinib. What’s next for sevabertinib and similar efforts? MM: It is an extraordinary time to make many new treatments for patients with cancer and other diseases as well. The success of sevabertinib is a real proof-of-concept that we can do this again. If we can keep targeting the genomic causes of cancer in a disciplined and persistent way, we will continue making further advances in cancer treatment, so it’s important that we keep this momentum going forward. Here at the Broad, with our industry partners, we could do this again, not just once, not just twice, but many, many more times, and I’m looking forward to our opportunities to do that. Seeing this effort come to fruition after so many years of hard work, what are your reflections on this milestone? MM: As a cancer researcher, it's just an incredible honor and privilege to be part of a team that has brought forward a new medicine that can help people with the terrible disease of lung cancer. HG: Not all academic scientists have the opportunity to initiate and execute a drug discovery project that directly benefits patients, so we’ve been incredibly fortunate in this respect. The development of sevabertinib is also really meaningful for me personally. Both my parents passed away from cancer, and my father from lung cancer in particular. So the fact that I've made a contribution to help other lung cancer patients resonates with me and makes me really glad I come to work every day at the Broad. Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.