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Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here. This week brought some high-profile shakeups, including the resignation of Lykos CEO Amy Emerson following an FDA rejection last month, Laurie Glimcher’s announced departure from the Dana-Farber Cancer Institute, and more details on BioMarin’s reorganization and business strategy after recruiting prominent dealmaker James Sabry. We’ve got more information in today’s report. Lykos CEO steps down… Amy Emerson resigned as CEO of Lykos Therapeutics, following the FDA’s rejection last month of its MDMA-assisted therapy for PTSD. Emerson sat down with Endpoints News’ Max Bayer last month to discuss the rejection and “changes at all levels of the organization,” though she and the company did not disclose any C-suite changes. …and so does Laurie Glimcher  Glimcher will relinquish her duties as head of the Dana-Farber Cancer Institute at the end of the month and will be replaced by Benjamin Ebert, chair of the institute’s Department of Medical Oncology. Glimcher has for years been rumored as a potential pick to lead the NIH, and she even emerged as a wild-card pick for President Joe Biden’s FDA commissioner. But her future, as of now, remains unclear. BioMarin restructures BioMarin detailed its progress this week on a “cost transformation program” that’s expected to continue into next year and save $500 million in operating expenses. Executives also laid out a new corporate strategy that relies on three key business units: skeletal conditions, enzyme therapies and the hemophilia A gene therapy Roctavian. After recruiting well-known dealmaker dealmaker James Sabry, the company said it’s on the hunt for new opportunities. Recursion’s mixed data In its first of many expected clinical trial readouts over the next 18 months, Recursion reported data for a rare neurological disorder that can cause strokes, seizures and partial paralysis. But the results were decidedly mixed, particularly as investors and analysts had viewed the data as a potential “moment of truth.” Eli Lilly’s $1 billion bet Lilly is teaming up with the “dark genome” biotech called HAYA Therapeutics, putting down a 10-figure wager on a company that’s only raised a seed round. The upfront and equity investments remained undisclosed. Lilly’s deal follows a tie-up between Bayer and NextRNA last week on the “dark genome” focused on treating cancer. SPOTLIGHT Duke spinout launches in hopes of prevailing with brain cancer bispecific where Amgen, Roche did not Three Dyne executives leave as company reports new Duchenne drug data Astellas CEO talks future beyond Xtandi as Japanese drugmaker puts roots in Boston R&D On Thursday, Eli Lilly dropped more data for its once-weekly insulin candidate in adults with type 2 diabetes. Lilly and Novo Nordisk are racing to get a once-weekly insulin to many markets, but Novo hit a roadblock last month when the FDA turned down its weekly insulin candidate. IN8bio pauses glioblastoma program, halves workforce as it prioritizes leukemia asset Arrowhead spotlights key pancreatitis figures from Phase 3 lipid disease study to differentiate itself from Ionis Vaxcyte surges on Phase 1/2 data for 31-valent pneumococcal vaccine GSK’s Nucala cuts exacerbations in Phase 3 COPD trial, but numbers remain under wraps Athira’s small molecule drug for Alzheimer’s fails in mid-stage study Moderna’s mpox vaccine outperforms approved version in preclinical study ProKidney axes one Phase 3 study, aims for ‘expedited approval pathway’ using another Praxis’ epilepsy drug reduces seizures by 46% in pediatric patients in Phase 2 study Researchers seek answers as they spell out Phase 3 failure of Bayer’s next-gen anticoagulant PHARMA Amid the brouhaha over Alnylam’s disappointing and perhaps confusing HELIOS-B data presentation last week, competitor BridgeBio is — at least publicly — attempting to stay above the fray and stick to its commercialization plans. Federal judge rejects pharma industry’s request to halt Maryland’s 340B law J&J increases talc settlement by $1.1B after negotiating with plaintiffs, lawyers say Court orders J&J to pay $1B following buyout of surgical robotics company Court’s ruling on Illumina-Grail deal could signal a limit to European antitrust powers Higher dose of Spinraza helped spinal muscular atrophy patients, Biogen says Sanofi’s BTK inhibitor goes 1-for-3 in key MS trials, slows disability progression Roche unveils more mid-stage data for BTK inhibitor in relapsing multiple sclerosis Pfizer and Valneva say second Lyme booster led to ‘strong’ responses, target 2026 FDA submission Bayer’s Kerendia cuts heart death, failure and hospitalization by 16% in Phase 3 trial STARTUPS ZipBio has raised $4 million in seed funding to explore a new use for existing AI systems, it told Endpoints exclusively. The idea involves compressing large proteins to better package them in gene therapies or deliver them to hard-to-reach places like the back of the eye. Non-viral delivery startup Vesigen Therapeutics trims staff as it evaluates ‘strategic options’ Exclusive: Lilly-backed OrsoBio raises $67M for slate of obesity drugs as combinations take root Bicara, Zenas outline plans for $180M IPOs amid restored optimism for biotech debuts Little-known startup, backed by Foresite and Samsara, swings $60M China I&I deal FDA+ The FDA has raised concerns about potential “inappropriate use” of Iterum’s oral candidate for uncomplicated urinary tract infections ahead of a Monday advisory committee meeting. The agency is asking the committee to discuss the benefits and risks of sulopenem and considerations to ensure appropriate use. Stay tuned. The General Court of the European Union temporarily suspended the European Commission’s decision to revoke the conditional marketing authorization for Ocaliva. The drug will be available “until further notice,” Advanz Pharma said Thursday. CBER chief forecasts better global regulatory convergence around cell and gene therapies FDA grants full approval to Travere’s Filspari for IgAN  Milestone’s arrhythmia drug on track for China filing with new Phase 3 data FINANCING Arsenal Biosciences secured a $325 million Series C this week, while also reprioritizing its pipeline. The company’s lead program, AB-1015, will no longer advance past a Phase 1 trial in ovarian cancer. More news is expected before the year ends. Transplant biotech eGenesis snags $191M after first use of gene-edited pig kidney BioAge files for IPO, riding momentum for weight loss drugs MANUFACTURING Novartis will invest $200 million in its radioligand manufacturing efforts, planning to open a new site in Carlsbad, CA, and beef up its capabilities in Indianapolis.  The new California site will be the company’s third radioligand manufacturing site in the US. Novo Nordisk continues to grapple with patchy GLP-1 drug supply in Europe HEALTH TECH Evernorth, the health services arm of Cigna, is planning to make a Stelara biosimilar available for $0 out-of-pocket for select patients PatientPartner raises $7M to help pharma companies match patients with mentors MDLive veterans nab $7.5M Series A for virtual specialty care startup DON’T MISS The Endpoints 11 event is coming: Meet biotech’s hottest new startups

An influential member in Congress is slated to publicly disavow the closely watched Biosecure Act ahead of a planned House vote next week, according to a letter reviewed by Endpoints News. The legislation would force drugmakers to restrict their work with two popular Chinese contractors: WuXi Biologics and its sister company, WuXi AppTec, by 2032. It also names three genomics companies: BGI, MGI and Complete Genomics. James McGovern (D-MA), ranking member of the House Rules Committee, said in the letter that the bill would essentially ban companies without due process. “In short, with no due process for named companies, Congress is relying on questionable data to make important national security decisions. It may be that some of the companies named should be on the list. But others may be punished that should not be,” he wrote in a letter to members of the House that is slated to be sent soon. McGovern has been publicly quiet on the legislation, but this week, he asked some of his colleagues to vote against the bill, according to three sources familiar with the matter. A spokesman for his office confirmed his advocacy against the bill. “Over the last several months, I’ve participated in a number of briefings and conversations related to this bill — in both classified and unclassified settings,” he wrote in the letter. “And all of those have led me to believe that HR 8333, as currently drafted, is a shortsighted way to address the real problem of foreign exploitation of the US biopharmaceutical industry.” It’s unclear if McGovern’s letter will sway the vote on the bill. In McGovern’s district, WuXi Biologics planned to build a $300 million biomedical plant in Worcester, MA, that’s at the center of last-minute lobbying against the legislation. Jon Weaver, CEO of the incubator Massachusetts Biomedical Initiatives, told some members of Congress in emails this week that the Biosecure Act resulted in WuXi Biologics pausing work on the 190,000-square-foot biomanufacturing facility, according to the communications reviewed by Endpoints. It appears to be the first link between the legislation and the stoppage. In May, The Worcester Guardian reported that WuXi Biologics denied the Biosecure Act had caused the company to pause construction on the facility. The company did not respond to requests for comment. Weaver said in the email to lawmakers that WuXi Biologics promised to restart if there’s a government audit process to review companies, rather than “asserting their guilt with no mechanism for justice or reprieve.” “They are confident they will be cleared in such a review and are undertaking a voluntary third-party audit to confirm this,” Weaver wrote. In an interview with Endpoints, Weaver declined to share which legislators he contacted, or their responses. “We’re focused on making this area in Massachusetts a global manufacturing hub,” Weaver said. The legislation has been closely watched in life sciences circles. But few organizations have been willing to publicly express their positions for fear of triggering anti-China hawks who are concerned over national security and, conversely, companies that believe the legislation could slow down scientific progress. If the House passes the legislation, a separate version of the bill would also need to clear the Senate.

A federal judge in Maryland has rejected a request from drugmakers and industry group PhRMA to stop a Maryland law that would lift restrictions on the amount of discounts directed at hospitals serving low-income or uninsured patients. The decision handed down Thursday throws cold water on industry representatives’ request for a preliminary injunction. The case was originally brought by Novartis but has since added AbbVie, AstraZeneca and PhRMA as plaintiffs. The groups argue that Maryland law HB 1056 unlawfully forces the companies to accept an unlimited number of transactions that fall under the umbrella of the federal drug discount program known as 340B. That law allows select hospitals that treat lower-income patients to buy prescription drugs at a discount. The goal is for cash-strapped hospitals to have more financial flexibility, but pharma companies have seen the 340B program balloon in size. In its initial complaint, Novartis argued that Maryland’s law is so vast that it preempts federal law, namely by asking the company to make drugs available that are outside of the scope of 340B and to do so before exclusivity ends. “Absent immediate judicial intervention enjoining H.B. 1056, Novartis will suffer irreparable harm,” lawyers for the company wrote in their May complaint. The contentious case is just the latest in a line of battles over the parameters of the 340B program. The recent Supreme Court ruling on Chevron has added more urgency for lawmakers to find a legislative fix, given the subjectivity of the law in the eyes of drug manufacturers, hospitals and state lawmakers. The Chevron ruling upended precedence that empowered agencies’ interpretations of specific statutes. Senators have been working to do just that, but efforts have been delayed. A bipartisan group of Senators had planned to release a more updated draft in early August but failed to do so. A group of Republican lawmakers on the House side introduced a bill in late May that seeks to rein in how hospitals use the 340B program, including allowing HHS to audit how health systems are using it and creating a definition of patients that are covered under the program.

Cambridge, MA-based startup Vesigen Therapeutics is laying off staff and “evaluating strategic options,” CEO Paulash Mohsen confirmed to Endpoints News via email Friday afternoon. Multiple employees posted to LinkedIn this week about being let go from the company, with one employee saying the company’s “journey” has come to “an early conclusion.” Mohsen said the startup “has not shut down.” “Yes, like many biotechs facing headwinds in the current financing environment, Vesigen has made the difficult decision to reduce our workforce,” Mohsen said. He said the decision came this week and the company is “evaluating strategic options, including with a variety of parties that are interested in delivery.” The company says its patented technology can help to deliver intracellular therapeutics to previously undruggable targets. “Delivery continues to be a significant unmet need for genetic medicines, and we have technology that shows considerable promise in bridging this gap. Our objective continues to be for the technology to succeed and find its way to patients,” he said. He didn’t immediately answer a question about how many employees were impacted. The startup emerged in July 2020 with $28.5 million from Leaps by Bayer, Morningside Ventures and Alexandria Real Estate Ventures, among others, and doesn’t appear to have raised another equity financing since then. At the time of the unveiling, co-founding CEO Robert Millman told Endpoints News the Series A would last the company until 2025. It reeled in a $32 million debt round in early 2023, according to venture data firm PitchBook. The company is attempting to build out a new generation of non-viral delivery technology for applications across gene editing, RNA and protein-based therapies. It is based on work out of Harvard’s Chan School of Public Health, where professor Quan Lu worked on so-called ARMMs, or arrestin domain-containing protein 1 [ARRDC1]-mediated microvesicles. They’re “distinct from exosomes,” Lu wrote in a Nature Communications publication in 2018. The production is more controlled, and the cargos can be recruited into the vesicle during the manufacturing process instead of after. “The barrier to being a drug company right now is not technology or targets,” Millman told Endpoints at the time of the Series A. He departed the company about a year later, and Yumanity veteran Mohsen took over as CEO in 2022. “There’s plenty of targets, there’s plenty of technology, but there’s no way of getting the two of those into a patient.” It is working in four broad therapeutic areas, including ocular diseases, immunology, neurology and blood disorders. In May, the company presented data at the Association for Research in Vision and Ophthalmology and the American Society of Gene & Cell Therapy. It has not yet entered the clinic.

CAMBRIDGE, MA — When looking to the future of the drug industry, the allure of Boston is strong. This week, the Japanese drugmaker Astellas became the latest company to open a research hub in the region, following Eli Lilly, Novo Nordisk and other companies that recently set up similar outposts. “Innovation is our lifeline, and we need to have a footprint here,” Astellas CEO Naoki Okamura told Endpoints News in an interview after a sake barrel-breaking ceremony to celebrate the opening of the company’s new 62,000 square-foot research center in Cambridge, MA. The 400-person center, which will also house some business development and commercial teams, is Astellas’ long-term investment in a future beyond Xtandi, the prostate cancer drug it developed with Pfizer that brought in more than $5 billion in revenue last year. Sales continued to grow in the second quarter, but key patents for the treatment are set to expire over the next three years. In a wide-ranging interview, Okamura discussed the company’s post-Xtandi plans, its M&A strategy, its new focus on protein degraders, its longstanding commitment to cell and gene therapy, and the importance of planting roots in Boston. “This center is going to be a critical key success factor,” he said. In the near-term, Okamura said that Astellas needs to boost sales of five other drugs, including Veozah, a pill for treating hot flashes during menopause; Izervay, an eye injection for a form of vision loss called geographic atrophy; and three cancer drugs named Padcev, Xospata and Vyloy. The latter drug is approved in Japan and currently under review at the FDA, with a decision expected in November. But those five key drugs accounted for just $1.1 billion in sales in 2023, less than 10% of the company’s total revenue. Sales of both Veozah and Izervay have been sluggish. Reaching the company’s profit margin target will require getting some of those drugs approved for additional diseases and running a “leaner organization,” Okamura said. “The strong growth of these five strategic brands is the must,” he said. “But that doesn’t guarantee sustainable growth.” Filling a pipeline The Cambridge research site will help fill the company’s early-stage pipeline. It has a big emphasis on developing protein degraders and other small molecules that act as molecular matchmakers. Astellas’ efforts were spearheaded by its own scientists who were itching to work on the molecules, which promise to tackle many notoriously tough-to-drug proteins. Just a few years after greenlighting the program, Astellas has already made a degrader for KRAS G12D, a mutant protein common in pancreatic, lung and colorectal cancers. It plans to disclose the first glimpse of data from the Phase 1 study at ESMO, a European cancer conference, this month. Tadaaki Taniguchi “This is just a start,” chief medical officer Tadaaki Taniguchi told Endpoints. Astellas has several other protein degraders in the works, including a pan-KRAS degrader and undisclosed programs for cancer and non-cancer targets, he added. But the homegrown molecule may be an outlier in the company’s future pipeline. Astellas got most of its approved drugs, and many of its experimental ones, from other companies, and Okamura expects that trend will continue. “We kind of stopped thinking that internally-developed product is the best product,” Okamura said. “Going forward, there would be very few, if any, compounds that we say that this is our internally-discovered, developed product.” The company has been striking partnerships with smaller biotechs and academic centers, many focused on cell and gene therapies. In January, the company announced a five-year collaboration with Mass General Brigham in Boston, although details about that partnership are sparse. Astellas is also opening its first US-based SakuLab, a rentable lab space and biotech incubator. Several other pharma companies in Boston have similar setups at their research centers, giving them a front-row seat to some of the newest ideas in the industry. “We also need to continue to show our presence, that we are here and are open to collaborate,” Taniguchi said. These efforts are small compared to its $5.9 billion acquisition Iveric Bio and its eye drug Izervay last summer. It was the company’s largest acquisition ever. And while Okamura said Astellas will remain “opportunistic,” another Iveric-sized purchase isn’t happening anytime soon. “We had been watching the compound for more than eight years until we made the final decision to acquire,” Okamura said. “Six billion dollars is somewhat the limit for our financial capacity. So if you ask me, are you going to do a similar size M&A in three years? I don’t think so.” ‘No intention to give up’ The Iveric acquisition also meshed with the company’s longstanding ambitions to create regenerative medicines for vision loss, and it gave Astellas a foothold in the ophthalmology market. Astellas is currently testing a stem cell-derived therapy in people with geographic atrophy. If it works, it could restore eyesight instead of simply slowing vision loss like Izervay. The Phase 1 study is expected to wrap up in January 2026. “Izervay is going to be a front-runner for our follow-on products,” Okamura said. At a time when many pharma companies are abandoning gene therapy programs, especially for rare diseases, Astellas insists that it has no plans to give up on the medicines, even in the face of one of the industry’s most tragic failures. Four boys died after receiving the company’s gene therapy for a rare neuromuscular disease in 2020 and 2021, likely due to liver failure from the high doses of virus used to shuttle the therapeutic gene. But Astellas still wants to find a way to use that gene therapy, and others, more safely. “People tend to look at the four boys’ deaths. And that’s a very unfortunate and tragic situation,” Okamura said. “On the other hand, there are healthier boys who received [the therapy] and survived for three to five years already. So we believe in the power of gene therapy to really make the difference.” Okamura believes Astellas has an obligation to improve the safety of the treatment. It is working with startup Kate Therapeutics on a new version of the therapy that the company hopes is better at targeting muscle cells and avoiding the liver. “We know that the correct transgene to the targeted cell should cure the situation,” Okamura said. “And we have no intention to give up.”