BioSpace

• Another Gene Therapy Death; Biopharma M&A Picks Up; a Vaccine Board of Vaccine Skeptics

  This week, BioSpace is at 50% power as Heather McKenzie and Jef Akst are off attending this year’s BIO Conference in Boston. The half-team discusses this week’s biggest news: the death of another patient who took Sarepta’s Duchenne muscular dystrophy gene therapy Elevidys. The patient was a non-ambulatory teenager who experienced acute liver failure after receiving the gene therapy, which is the same cause of death for an Elevidys patient reported in March. Sarepta announced that it was halting treatments to non-ambulatory patients and on a media call discussed new steps in its therapeutic protocol for preventing further liver injuries.  Elsewhere, mergers and acquisitions are surging across biopharma. Eli Lilly picked up the gene editing company Verve Therapeutics for $1.3 billion, which helped bolster the gene editing space —particularly after Sarepta’s report of the death. Supernus bought Sage Therapeutics for $795 million, five months after Sage rejected a smaller offer from Biogen.  BioNTech also got in on the dealmaking, buying its German rival—scientifically and in the courtroom—CureVac for about $1.25 billion. The deal seemed focused mostly on CureVac’s early-stage cancer immunotherapy pipeline, but analysts were otherwise left scratching their heads on what BioNTech was getting for its money.  Last week on The Weekly the team discussed the sudden dismissal of the CDC’s entire ACIP committee, and this week we have a new slate of members. The eight people replacing the 17 members that were removed last week include allies of HHS Secretary Robert F. Kennedy Jr., many of whom are vaccine skeptics who seem to share his skeptical view of vaccination in general.  

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• RFK Axes CDC Vaccine Advisors ; Metsera’s Weight Loss Win; FDA Supports CGT

  This week the BioSpace team was sent into an after-hours scramble by the news that Health and Human Services Secretary Robert F. Kennedy Jr. had fired the remaining members of the CDC’s Advisory Committee on Immunization Practices. The secretary argued in an op-ed that accompanied the announcement that the “clean sweep” was necessary to “reestablish public confidence in vaccine science.” The move came amid heightening rhetoric from Kennedy about the safety of vaccines and sparked concerns that the Secretary will replace the committee with people who sympathize with his anti-vaccine positions. On the drug development front, Metsera once again made waves with new data for an amylin drug candidate showing weight loss of 8.4% at just 36 days. The drug could be a major competitor for Novo Nordisk’s Cagrisema, which has failed to meet the sky-high expectations of investors despite being the leading amylin candidate in the space. And cell and gene therapy was back in the spotlight last week when the FDA held a round table discussion with industry leaders aimed at eliminating barriers to approval for these complex medicines. This support from the FDA came as Sarepta became the first company to receive a platform technology designation intended to streamline future gene therapy reviews and create predictability for new drug applications created using the same technologies.Over in Biopharm Executive, BioSpace looks at right of first negotiation deals in light of Sanofi’s deal to buy Vigil Neuroscience. How often do these agreements turn into M&A? After some analysis, Jefferies found that a ROFN is not a golden ticket to a buyout.

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• AI's Role in Decoding the FDA's New Regulatory Communications

  In a dynamic regulatory environment, IQVIA’s Michelle Gyzen suggests that AI may be the best and only way to keep with changes that are happening daily–and sometimes hourly.In this discussion Lori and Michelle touch on the governance frameworks for cybersecurity, risk, and how AI transformation and integration is evolving to help regulatory professionals navigate the speed and complexity of global requirements.Host⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠, Head of Insights, BioSpaceGuestMichelle Gyzen, Sr. Director, Strategic Regulatory Solutions; Head of Regulatory Services Innovation & Technology, IQVIA

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• M&A Ticks Up, ASCO Excites and Vaccines Cause More Drama

  The words of the week so far in biopharma are “deals” and “cancer”—or, more specifically, money being invested in cancer and other key therapeutic areas. With the American Society of Clinical Oncology’s annual conference underway in Chicago, Bristol Myers Squibb got in the PD-1/PD-L1xVEGF game, paying potentially more than $11 billion to co-develop BioNTech’s solid tumor bispecific BNT327. Elsewhere, Sanofi nabbed the year’s second-biggest buyout, picking up Blueprint for $9.5 billion, expanding its rare disease portfolio. And Regeneron plunked down up to $2 billion to license a dual GLP-1/GIP receptor agonist from Chinese biopharma Hansoh Pharmaceuticals Group.  Back in Chicago, presentations by AstraZeneca, Gilead and Amgen drew rave reviews from investor analysts, while Pfizer and Arvinas elaborated on mixed data from a PROTAC that showed positive results in only a subsection of breast cancer patients, failing to impress Wall Street. Meanwhile, Bicara’s solid survival stats in head and neck cancer weren’t enough to clear the high bar set by rival Merus. At the meeting, BioSpace’s own Dan Samorodnitsky sat down with Jazz Pharmaceuticals’ CMO Rob Iannone to discuss the company’s recently acquired pediatric glioma drug, and talked AI strategy with AstraZeneca’s head of U.S. oncology for lung cancer Arun Krishna. Dan recaps his ASCO experience here.  Speaking of buzzy therapeutic spaces, there was more action on the vaccines front last week as Health and Human Services Secretary RFK Jr. announced that healthy children and healthy pregnant women would no longer be advised to get vaccinated against COVID-19. However, as of publication, the CDC still recommends a COVID vaccine for healthy children but instead of a universal recommendation advises that the decision should be made between parents and healthcare providers. Against this backdrop, the FDA signed off on Moderna’s next-gen COVID-19 vaccine, mNEXSPIKE, for a limited population in line with its new guidelines. This was a much-needed win for Moderna, which last week had a $760 million-plus government contract for its mRNA-based bird flu vaccine terminated.  Also on the policy front, the Trump administration released its Make America Healthy Again report last week to much scrutiny after reports found studies and references that did not exist.  

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• COVID-19 Vaccine Overhaul, Rocket Grounded, ‘One Tough Hombre’ on MFN

  The name of the biopharma game this season is vaccines—and RFK Jr. wasted no time returning from Memorial Day Weekend before making news on this front, removing the COVID-19 vaccine from the list of recommended immunizations for healthy kids and pregnant women on Tuesday. This follows a rash of recent moves, including a new risk-based strategy for the approval of new COVID vaccines focused on adults over 65 and high-risk individuals six months to 64 years of age and a request that Moderna and partners Pfizer and BioNTech update the myocarditis risk on their vaccines’ labels.This increased vaccine scrutiny by the FDA and Department of Health and Human Services is having a significant impact on biopharma companies, several of whom have received stop-work orders on next-gen COVID vaccines. Meanwhile, Moderna last week pulled the biological license application for its combination COVID-19/flu vaccine, anticipating a request for additional data on flu shot efficacy from the FDA, as mRNA technology continues to be scrutinized after playing the hero during the pandemic. Also last week, the White House released its Make America Healthy Again (MAHA) report, which took aim at vaccines, along with pharma lobbying and GLP-1s.Also on the policy front, a California judge issued an order Thursday that indefinitely stops HHS’ goal of reducing its divisions from 28 to 15 and firing upwards of 10,000 employees, among other reorganization and mass layoff plans. And speaking of government plays that could receive judicial pushback, we received a couple of new updates on the Most Favored Nation (MFN) front: first, President Donald Trump appointed his CMS chief Mehmet Oz as the leader of drug pricing negotiations, calling him “one tough hombre,” and second, HHS provided new guidance for streamlining the process for states to import drugs from Canada.In the obesity realm, Eli Lilly is calling out the Centers for Medicare and Medicaid Services. In an open letter last week, Lilly voiced its displeasure with the agency for a final ruling that left its Zepbound and Novo Nordisk’s Wegovy out of Medicare Advantage and Part D coverage in 2026.And in the R&D realm, we returned from the long weekend to sad news from Rocket Pharmaceuticals, as the company reported that its pivotal Danon disease trial is on hold after the death of a young patient. The death—extremely sad on a human level—is also a setback for the gene therapy space, which had been buoyed earlier this month by the success of a personalized CRISPR treatment received by baby KJ.Finally, BioSpace looks ahead to ASCO#25 where Dan Samorodnitsky will be on the ground in Chicago.

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