In late May, a patient died after receiving Rocket Pharmaceuticals’ investigational gene therapy for Danon disease, spurring the hold. After lowering the dose and changing the regimen of immune modulators patients receive, the company has received FDA clearance for the trial to continue.
The FDA recommends that companies use overall survival as a primary endpoint for clinical trials where feasible. The new guidance follows the surprising return of CBER Head Vinay Prasad, who has previously argued for prioritizing OS.
With its structural changes, CSL expects to generate $500 to $550 million in annualized savings over the next three years.
The CDC no longer recommends COVID-19 vaccines for healthy children and healthy pregnant women, a position that has been opposed by leading medical societies.
A draft copy of an upcoming MAHA report reveals a strategy in lockstep with recent HHS actions such as reviving the Task Force on Safer Childhood Vaccines; Viking Therapeutics reports robust efficacy from mid-stage oral obesity candidate but is tripped up by tolerability concerns; Novo Nordisk wins approval for Wegovy in MASH; and Lilly takes a pricing stand.
The small molecule, vatiquinone, had already flunked a Phase III trial, but the company pushed ahead with an approval bid anyway.
FEATURED STORIES
Keytruda is set to lose exclusivity in 2028, meaning Summit may face competition from cheaper biosimilars. Meanwhile, other branded drugmakers are also seeking to improve on the blockbuster checkpoint inhibitor.
M&A and IPOs got off to a quick start in 2025 only to crash into a wall of policy challenges. Upfront payment for licensing transactions, however, grew as pharmas looked for less-risky deals.
Beam Therapeutics and Verve Therapeutics have each built their lead candidates on a technique billed as a safer alternative to conventional CRISPR. Clinical results have so far been promising.
On the FDA’s docket this month are two expansion bids, one for GSK’s asthma drug Nucala into COPD and another for Merck’s oral cancer drug for a pair of rare tumors.
As the Trump administration—including HHS Secretary Robert F. Kennedy Jr.—plays fast and loose with scientific studies and facts, there may be a more sinister force at play: censorship.
When it comes to vaccination, the COVID-19 pandemic divided American society. President Donald Trump and his new Health and Human Services secretary are bringing down the hammer. What happens when there is no middle ground?
LATEST PODCASTS
In this episode of Denatured, BioSpace’s Head of Insights Lori Ellis, Miguel Forte and Ali Pashazadeh speculate on the impending Trump administration, discuss current challenges faced by CEOs and weigh investment in GLP-1s.
The Novo-Catalent deal now moving ahead highlights unprecedented investment in manufacturing, while also standing out as an exception to the unspoken rule of keeping M&As to less than $5 billion this year.
GSK, Gilead and Arcellx, Vertex and more present new data at the American Society of Hematology annual meeting just as sickle cell therapies Casgevy and Lyfgenia have a new outcomes-based payment model; Eli Lilly and Novo Nordisk pump new funds into manufacturing; and AbbVie makes a Cerevel comeback while uniQure clears a path toward accelerated approval in Huntington’s disease.
Job Trends
Takeda (TSE:4502/NYSE:TAK) today announced data from the Phase 3 ADVANCE-CIDP 3 clinical trial, a long-term extension study evaluating the safety and efficacy of HYQVIA® [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] in patients with chronic inflammatory demyelinating polyneuropathy (CIDP).
Subscribe to Genepool
Subscribe to BioSpace’s flagship publication including top headlines, special editions and life sciences’ most important breaking news
SPECIAL EDITIONS
In this deep dive, BioSpace explores the next big thing in obesity.
BioSpace did a deep dive into biopharma female executives who navigated difficult markets to lead their companies to high-value exits.
BioSpace data show biopharma professionals faced increased competition for fewer employment opportunities during the second quarter of 2025, with increased pressure from further layoffs.
DEALS
-
BioSpace presents 25 noteworthy biopharma startups in ’25; analysts forecast stronger M&A as the J.P. Morgan Healthcare Conference kicks off next week; GLP-1s continue to expand their reach as Novo, Lilly fight against compounders; and a look ahead to five key FDA decisions in Q1. -
Licensing deals have risen in prominence in a restrained market environment. Is it desperation, or an important part of the biotech ecosystem? Experts weigh in. -
Look for renewed investment driven by lower interest rates in the new year, and a continued focus on late-stage assets, oncology and reaping the benefits of AI. -
China is adapting its Life Sciences policy to bolster innovation and data transparency. Big Pharma is taking note. -
The acquisition from Wuxi Biologics, the embattled CDMO named in the BIOSECURE Act, marks another expansion of Merck’s manufacturing operations in Ireland.
WEIGHT LOSS
-
Novo Nordisk’s NovoCare will now provide uninsured or underinsured patients access to Wegovy for just $499 per month—less than half of its list price. -
Pfizer reacts to Donald Trump’s tariff threats on big pharma, another regulatory meeting is canceled under RFK Jr., AbbVie and Eli Lilly strike mid-sized deals in obesity and molecular glues, priority review vouchers set to take a hit and immuno-oncology matures.
-
Merck’s Keytruda holds on to the top spot while AbbVie’s Humira—once the world’s top-selling drug—continues to cede its market share to biosimilar competitors. -
AbbVie is joining the amylin arena, though the pharma is still far behind leaders Novo Nordisk and Eli Lilly. -
Two recent documents—one from the FDA, the other from a commission organized by The Lancet Diabetes & Endocrinology—indicate an evolving mindset toward treating obesity as a chronic disease.
POLICY
-
The eight new committee members replace the 17 Kennedy removed earlier this week. In “repopulating” the committee, the HHS Secretary fulfilled the fears of some analysts, naming scientists who appear to reflect his anti-vaccine views. -
The American Medical Association is also urging an “immediate reversal” of the HHS Secretary’s decision to oust all 17 members of the CDC’s vaccine advisory board. -
Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the floor. At a roundtable last week, FDA leaders promised faster approvals and broad support to the industry. -
Robert F. Kennedy Jr.’s removal of all remaining members of the CDC’s Advisory Committee on Immunization Practices raises questions about the upcoming meeting later this month. Analysts fear the committee could be more sympathetic to the HHS Secretary’s anti-vax viewpoints. -
The reinstatement of the generic drug policy office is the latest reversal of course for Robert F. Kennedy Jr.'s HHS, which also recently rehired FDA staff responsible for making travel arrangements and those involved in user fee program negotiations.
Here are the top biotech companies in California hiring now on BioSpace’s life sciences–focused job board.
To be self-employed in the life sciences, you must have a strong network and background and experience in a relevant area.
While MBAs can reflect positively on job applicants, work experience can often be more valuable, depending on the area.
If management isn’t your strength, there are several upper-level positions that don’t require you to manage others. Here are five high-paying life science jobs that don’t include management.
Ageism, or discrimination against an individual based on their age, is a common barrier many older individuals face in the workplace. Fortunately, there are steps that can be taken to mitigate this discrimination.
Job hopping– frequently changing jobs, typically within a short period of time – gained popularity with the onset of COVID-19 and is still popular now. Here’s what recruiters think about job hopping.
HOTBEDS
REPORTS
In this Employment Outlook report, BioSpace explores current workforce sentiment, job activity trends and the prospective job and hiring outlook for 2025, particularly as it compares to the previous year.
BioSpace’s third report on diversity, equity, inclusion and belonging in life sciences examines dramatic shifts in attitude around diversity initiatives.
CANCER
-
Cell therapy and oncology–focused Carisma Therapeutics started layoffs late last year. Now the company plans to wind down fully. -
While Novartis and Bayer got there first, AstraZeneca, Bristol Myers Squibb and Eli Lilly are all vying to bring their radiopharmaceutical assets to a market projected to be worth over $13 billion by 2033. -
BNT327, a PD L1/VEGF antibody, belongs to a class of next-generation immunotherapies hoping to beat out Keytruda. -
Merck’s new formulation of the mega-blockbuster Keytruda, made in collaboration with Alteogen, could help to keep the drug’s patent cliff at bay. -
The latest data from Johnson and Johnson’s Rybrevant and Lazcluze lung cancer combo was better than standard of care Tagrisso on overall survival. But analysts say the next step is getting a subcutaneous formula approved.
NEUROSCIENCE
-
After failing to hit the primary endpoint in a Phase III trial, Neumora is remixing study parameters in two replicate trials, with data expected in the first half of 2026. -
Despite not differentiating itself from placebo, the Texas-based company said it plans to push pilavapadin into Phase III trials before long. -
Leqembi’s application now moves forward to the European Commission, which will issue a formal verdict for the injection that will apply to all EU member states as well as Norway, Liechtenstein and Iceland. -
Mission Therapeutics is down to its clinical assets MTX652 and MTX325, which work by disabling a key enzyme that interferes with the cell’s normal process of removing faulty or dysfunctional mitochondria. -
The licensing deal follows years of controversy for Cassava, as well as the high-profile late-stage failure of its Alzheimer’s disease drug simufilam.
CELL AND GENE THERAPY
-
Faced with the encroaching threats of patent expirations and generics, biopharma companies in 2024 invested 33% more in licensing deals, on average, than in 2023 with an eye toward enriching their pipelines with novel and potentially more effective therapies.
-
Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio—have products in the pipeline that could improve treatment options for this rare disease. -
CAR T–focused biotech Cargo Therapeutics surprised and disappointed analysts when it announced that it would discontinue a mid-stage trial of its lead program, firi-cel. -
In this episode, presented by the Genscript Biotech Global Forum 2025, BioSpace’s Head of Insights Lori Ellis talks to Tom Whitehead, co-founder of the Emily Whitehead Foundation, about how standard care, cell and gene therapies and their impact on patients. -
Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and more aim to deliver the next wave of progress with near-term data and regulatory milestones.
