Massachusetts Institute of Technology

Willkommen, bienvenue, welcome back to another edition of Endpoints Weekly. We’re supposed to get a Nor’easter this weekend on the East Coast after a mostly dry summer. The rain is needed, but hopefully everyone can stay dry and safe! We had a well-rounded mix of events, news and features this week. Leading the way was our annual Women in Biopharma story, marking the seventh time we’ve highlighted trailblazing women in the industry. Along with that, Andrew Dunn continued his salary analysis series, showing women are still underrepresented in the upper echelons of biopharma. Be sure to read both pieces if you haven’t already! We also learned this week that former CBER leader Peter Marks will start a new job at Eli Lilly, and Bob Langer started a new biotech. On the feature side, we have great stories across the board about Amgen and the push for lower-cost gene therapies. Have a great weekend! — Max Gelman 👩‍🔬We unveiled our seventh annual Women in Biopharma project this week, highlighting 20 women shaping the future of biopharma. This year’s list includes: the CEO behind one of the largest Series B rounds of the last few years, a chemist who helped invent a new class of cancer-targeting drugs, a tech leader focused on streamlining clinical trials, and more. Click here for the full list of honorees. If you missed our virtual event, you’ll be able to watch it on demand here next week. Women remain underrepresented in the highest rank of the pharma industry, according to a report by senior biopharma correspondent Andrew Dunn. His analysis of 695 publicly traded life sciences companies found that women hold 10.8% of CEO positions. Of 57 biopharma companies worth over $10 billion, five are run by women, or 8.8%. Andrew breaks down the data here. Hello, Michael Hale! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Michael Hale! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Michael Hale! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Michael Hale! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Michael Hale! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Michael Hale! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Michael Hale! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Michael Hale. You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Former CBER chief Peter Marks said this week that he’s joining Eli Lilly following his departure from the FDA earlier this year. In his resignation letter, Marks criticized HHS Secretary Robert F. Kennedy Jr.’s stance on vaccine safety. Marks oversaw the regulation of vaccines, as well as cell and gene therapies, in his role as CBER director. At Lilly, Marks will be the senior vice president for molecule discovery and head of infectious diseases. Marks’ move to Lilly may drive criticism of the supposed “revolving door” between the FDA and big drugmakers. Marks is the second high-profile former FDA leader who joined a biopharma company this year, after former CDER director Patrizia Cavazzoni took a job at Pfizer. He’s also the second ex-CBER official to jump to Lilly in as many months: Rachael Anatol, who was second in command in the FDA’s Office of Therapeutic Products, announced Lilly had hired her last month. 🍽️ Biotech correspondent Kyle LaHucik has the dish on Soufflé Therapeutics, a genetic medicines biotech created by Moderna co-founder Bob Langer. The biotech emerged from stealth on Tuesday and said it has “executed over $3.5 billion in capital raised and collaboration agreements,” including a $200 million Series A. MIT professors Daniel Anderson and Brad Pentelute and former Alnylam executive Victor Kotelianksi are also listed as co-founders. Soufflé said it’s working on targeted nucleic acids, including siRNA and other “cell-specific therapies.” And it has some of the biggest names in pharma as its partners and investors, including AbbVie, Bayer, Novo Nordisk, Polaris Partners, ARCH Venture Partners and Vida Ventures. The company is exploring treatments for muscular dystrophies, heart failure and metabolic disorders, and is specifically looking at developing a therapy for facioscapulohumeral muscular dystrophy. Pfizer’s buyout of Metsera last month put the big drugmaker firmly back in the GLP-1 race. But Metsera initially wanted more than Pfizer paid, according to background documents of the deal released this week . Metsera had a rival offer of as much as $91 per share, but that offer was seen as less certain to close. Pfizer ended up paying $47.50 per share in cash (worth $4.9 billion) and $22.50 per share in CVRs, potentially bringing the deal up to $7.3 billion. Metsera also pushed Pfizer for $35 in CVRs, which the drugmaker declined to give. In the end, Metsera had two other competitive bidders. The aforementioned first bidder had an offer of $56.50 per share in cash, plus up to $34.50 in CVRs. Meanwhile, the second bidder offered an even higher $47.50 per share in stock and $47.50 in CVRs, but backed out in early September. Ultimately, Metsera made the decision to go with Pfizer because the other offer came with “a variety of risks” that could have prevented the deal from closing for up to two years. 🖥️How is Amgen’s AI strategy playing out? Chief Technology Officer David Reese and other leaders at the company gave Endpoints’ Andrew Dunn an exclusive on Amgen’s efforts to put AI models at the heart of more and more of the drug discovery and development process. Reese left his position as head of Amgen’s R&D to lead the change. “The prize is getting undruggable targets even earlier in the process. The prize is understanding disease biology that we don’t yet understand, where we don’t even have a target or we don’t have tractable ways of going at a disease,” he said. Read more here.

Soufflé Therapeutics\' roster of partners includes several Big Pharma names including AbbVie, Amgen, Bayer and Novo Nordisk.\n A new RNA biotech has risen with a $200 million series A in the tank and a team of seasoned biotech chefs ready to light the burners. Soufflé Therapeutics will cook up siRNA medicines that are targeted and cell-specific, the newly hatched company said in an Oct. 7 LinkedIn post.Soufflé is a Langer family affair, with Moderna maverick Robert Langer co-founding the new company and his daughter Susan Langer serving as founding president and chief business officer. Langer’s son Michael, who recently launched a new biotech venture capital firm with support from his father, confirmed Soufflé’s $200 million series A in a separate LinkedIn post.In total, Soufflé has secured more than $3.5 billion in fundraising and partnerships, according to the company’s post.That roster of partners includes several Big Pharma names including AbbVie, Amgen, Bayer and Novo Nordisk, according to Soufflé’s website. Investors include Bessemer Venture Partners, Arch Venture Partners, Vida Ventures and Polaris Partners, among others.Soufflé plans to hit the clinic in 2026, with lead programs focusing on metabolism, facioscapulohumeral muscular dystrophy and cardiomyopathies caused by mutations in the phospholamban gene alongside a suite of other preclinical programs.“Proud to lift the lid today on the amazing work we are doing at Soufflé Therapeutics,” CEO Amir Nashat, Ph.D., an experienced biotech leader and veteran of Langer’s MIT lab, wrote in his own LinkedIn post. “We share a bold vision that potent, precise and safe medicines can be designed and developed for all diseases. I am honored to lead this talented team alongside my colleagues.” Rounding out Soufflé’s kitchen are Vadim Dudkin, Ph.D., chief technology officer and a practiced RNA medicine developer formerly with Merck and Johnson & Johnson; Roman Bogorad, Ph.D., as senior vice president of discovery biology; and Third Rock Ventures vet Mark Angelino, Ph.D., as chief strategy and operating officer, among others.Robert Langer’s fellow Soufflé founders, as well as the biotech\'s investors, are some of the same scientists and backers who helped create Alnylam Therapeutics and CRISPR Therapeutics, including Victor Kotelianski, M.D., Ph.D., who previously served as founding SVP of research at Alnylam.

Besides supporting work on Kernal\'s KR-402, the money will also be used to help expand Kernal’s mRNA 2.0 platform.\n The U.S. government has awarded Kernal Bio and three other sub-awardees up to $48 million to help advance the biotech’s in vivo mRNA-encoded CAR T-cell program. In an interesting—if not somewhat confusing—twist, the Advanced Research Projects Agency for Health (ARPA-H) is supporting Kernal’s KR-402, a next-generation cell therapy designed using the biotech’s “mRNA 2.0” platform, according to an Oct. 7 release. Recently, Trump administration officials have distanced themselves from mRNA vaccine work, with the Biomedical Advanced Research and Development Authority calling off $500 million in funding for such projects. Leaders in the administration cited different reasons for the pullback, including concerns over efficacy and “public distrust.” While the previous cuts impacted mRNA vaccines—and some projects that didn’t even include work in mRNA or vaccines—the main differentiator at issue may be the vaccine delivery approach versus the mRNA science itself. As of publication, the Department of Health and Human Services and ARPA-H had not responded to Fierce’s requests for comment.   With the newly announced project, ARPA-H is hoping the Boston biotech’s science can be used to create less expensive in vivo CAR T-cell therapies for hematological cancers and autoimmune diseases, according to the release. The funding is delivered via ARPA-H’s Engineering of Immune Cells Inside the Body (EMBODY) program, which is led by Daria Fedyukina, Ph.D. The financing will help Kernal advance the early-stage program through clinical development in multiple sclerosis and B-cell malignancies. The biotech’s science is built to improve therapeutic precision by using a two-pronged strategy: first, by utilizing selective mRNA that only translates in specific cells, and, second, by delivering RNA with a targeted lipid nanoparticle vehicle covered with antibodies so it can narrow in directly on target T cells. The money will also be used to help expand Kernal’s mRNA 2.0 platform, according to the release. Some government funding is also going to Kernal collaborators the Stanford University School of Medicine, the Dana-Farber Cancer Institute and the Jackson Laboratory, which are all working to engineer targeted, mRNA-encoded CARs and to create new manufacturing strategies for the potential therapies.  “Current CAR-T therapies heralded a true revolution in cancer treatment,” Kernal CEO and co-founder Yusuf Erkul, M.D., said in the release. “Yet, they have their limitations, including a three-week vein-to-vein turnaround time, tumor resistance leading to relapse, and side effects such as cytokine release syndrome or secondary T-cell malignancies.” “At Kernal Bio, we believe that we have the tools to evolve the CAR-T modality towards in vivo therapies,” Erkul concluded. The Hummingbird- and Amgen-backed biotech was formed by industry vets across Harvard, Merck & Co., Bristol Myers Squibb and MIT.