FDA
The FDA in September issued two rejections for spinal muscular atrophy therapies—both linked to manufacturing problems—and granted approvals in Barth syndrome and for a subcutaneous version of Merck’s Keytruda that could be key to the blockbuster’s future earnings.
The business separation, expected to be completed by the end of 2026, will result in two new companies, one focused on biopharma operations and the other on royalty management.
The first oral BTK blocker for chronic spontaneous urticaria, Rhapsido offers a more convenient treatment option for patients who still show symptoms after antihistamine treatment.
Novo Nordisk and Heartseed first partnered in 2021 to develop an investigational cell therapy for heart failure.
M&A headlined for a second straight week as Genmab acquired Merus for $8 billion; Pfizer strikes most-favored-nation deal with White House; CDER Director George Tidmarsh caused a stir with a now-deleted LinkedIn post; GSK CEO Emma Walmsley will step down from her role; and uniQure’s gene therapy offers new hope for patients with Huntington’s disease.
As major pharmas pull away from the U.K. and the U.S. risks ceding its lead through a national brain drain, the U.K. must create a new, more robust model for innovation.
Pfizer CEO Albert Bourla directly credited the threat of tariffs with leading to the deal, in which the company will offer drugs on a soon-to-be-launched website called TrumpRx.
FEATURED STORIES
As the political winds shift on a whim and public distrust of the pharma industry reaches fever pitch over drug pricing, executives are being asked to navigate an impassible path.
Generate:Biomedicines’ Nicole Clouse is one of the key legal minds trying to understand who owns what AI creates. The answers are critical to the future of biotech.
If the trend holds, IQVIA expects 2025 deal volume between Chinese and multinational companies to easily eclipse the 100 agreements signed in 2024.
Companies have claimed improvements to yield, batch consistency and output while acknowledging the risks and challenges created by the technology.
The mad rush for safe and effective obesity drugs has winners—including Eli Lilly’s Zepbound and Novo Nordisk’s Wegovy—and losers. Here are five molecules that never made it to the market.
While it’s impossible to make apples-to-apples comparisons of the many obesity candidates with so many differences across clinical trials, we at BioSpace are giving it our best shot.
FROM BIOSPACE INSIGHTS
Not developing potency assays and gaining knowledge about MOAs early in the drug development process not only can break ATMP success but can cause costs and delays that lead to company closures.
UPCOMING EVENTS
LATEST PODCASTS
Robert F. Kennedy Jr. testified in front of largely combative congresspeople on vaccine policy, his MAHA report and more; the mass leadership exodus at the FDA continues as CDER and CBER shed key staff; Kennedy’s revamped CDC vaccine advisors convene for their first meeting; Novo and Lilly present new data at the American Diabetes Association’s annual meeting; and BioSpace recaps BIO2025.
In this episode of Denatured, BioSpace’s Head of Insights Lori Ellis discusses key themes from BIO and DIA, including the funding environment, with Rich Daly, CEO of Catalyst Pharmaceuticals, Peter Ronco, CEO of Emmes Corporation, and Phil Vanek, founder of Redline Bio Advisors.
Another patient has died from acute liver failure after receiving Sarepta’s gene therapy for DMD ; After a quiet start to the year, M&A is back with one deal for a gene editing biotech reinvigorating that sector; and RFK Jr. installs a suite of new vaccine board members who share his skeptical views on vaccines.
Job Trends
Encoded’s layoffs will mostly affect its technology and early-stage research and development functions. The move is expected to keep the biotech operational well into 2026.
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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
  1. BioNTech will get CureVac’s early-stage cancer assets, including its mRNA-based glioblastoma therapy currently in Phase I development. CureVac had previously sued BioNTech for copyright infringement related to mRNA vaccine technology.
  2. Instead of homing in on PSMA—currently the most validated target in prostate cancer—BMS and Philochem will instead collaborate on an early-stage molecule that binds to a novel marker called ACP3.
  3. The deal is Lilly’s second obesity tie-up in a week, after sinking up to $870 million into an agreement with Camurus to develop long-acting versions of molecules against GLP-1 and other incretins.
  4. Looking at licensing deals struck in the past 10 years, Jefferies found that many Big Pharmas do not ultimately follow through with M&A after earning a right of first negotiation. Sanofi, on the other hand, almost always does, as it did with Vigil recently.
  5. Jefferies has predicted more small tuck-in deals to come, as biotechs struggle to access capital despite key clinical milestones on the horizon.
WEIGHT LOSS
  1. H2 2025 catalysts to watch, biopharma implications of President Trump’s tax law, KalVista’s new hereditary angioedema drug that Marty Makary reportedly tried to reject, another lawsuit aimed at Health Secretary Robert F. Kennedy Jr. and a plea from patients with ALS for access to BrainStorm’s NurOwn.
  2. Analysts said the deal with Novo was likely giving Hims “‘credibility’ or increased consumer traffic,” adding that the “litigation risk is back on the table” now that the Danish pharma has stepped away.
  3. After a season of regulatory upheaval, obesity and rare genetic diseases will likely remain major themes for biopharma in 2025, according to Jefferies.
  4. With PN-477, Protagonist is directly going up against Eli Lilly, which is advancing retatrutide, also a triple-G agonist, in a Phase II trial.
  5. Altimmune’s pemvidutide failed to significantly improve fibrosis in MASH patients in a Phase IIb study. The biotech crashed 53% in the aftermath of the readout.
POLICY
  1. The MIT professor of management, who already sits on the CDC’s revamped immunization advisory committee, is a known skeptic of vaccines, particularly mRNA technology.
  2. The White House has denied reports that the government could soon ban COVID-19 vaccines, noting that in the absence of an official announcement, “any discussion about HHS policy should be dismissed as baseless speculation.”
  3. Thousands of employees across the Department of Health and Human Services are set to lose their collective bargaining rights in a move that American Federation of Government Employees national president Everett Kelley called “illegal and immoral.”
  4. There’s still much more to come from the White House on tariffs, but the European Union has now reached a trade agreement with the U.S.
  5. In this episode presented by Cresset, BioSpace’s head of insights Lori Ellis discusses clinical trial fail rates and AI’s potential to reduce preclinical costs with Mutlu Dogruel, VP of AI and Mark Mackey, CSO of Cresset.
CAREER HUB
A BioSpace LinkedIn poll showed that just 19% of respondents believe biopharma professionals need Ph.D.s for scientist roles.
Generative AI could enhance and accelerate the way people work on clinical trials. In this Q&A, a management consultant shares his insights on benefits, risks and more.
While many describe California as having a tough life sciences market, there’s some optimism that employment opportunities will improve soon, according to California Life Sciences President and CEO Mike Guerra.
Looking for a quality control job? Check out these nine companies hiring life sciences professionals like you.
Companies will look at job candidates’ LinkedIn profiles, so make sure yours is strong, from the summary of your expertise and qualifications to testimonials from colleagues.
After more than 20 years at Eli Lilly, Leslie Sam moved into independent consulting. To prepare for the transition, she focused on becoming technically deep and earning industry recognition.
Employed and unemployed biotech and pharma professionals are thinking about job hunting in other fields amidst a challenging labor market.
HOTBEDS
Where are the Best Places to Work in life sciences? BioSpace’s annual Best Places to Work list demonstrates a company’s desirability in the recruitment marketplace - find out who made the list this year.
IN CASE YOU MISSED IT
This latest FDA program aims to provide speedier reviews for generic drugmakers who produce their products in the U.S.
REPORTS
After a tumultuous 2022, life science employers are settling into their hiring goals for 2023. Though they may be hiring at lower volume, the majority of organizations are still actively recruiting.
Economic turbulence has persisted into 2023 and the life science industry is certainly not immune. How are organizations juggling business needs, budgets, recruitment and retention?
Following a tumultuous start to 2023, layoffs have cooled off though recruiting activities have been significantly impacted by the economic environment.
CANCER
  1. In this episode presented by Eclipsebio, BioSpace’s head of insights Lori Ellis continues the discussion on mRNA and srRNA with Andy Geall of Replicate Bioscience and Alliance for mRNA Medicines and Pad Chivukula of Arcturus Therapeutics.
  2. Roche and Genentech were unable to sufficiently demonstrate the benefit of using Columvi in an earlier treatment setting for DLBCL in a U.S. population, according to the FDA.
  3. The panelists flagged safety concerns with Blenrep and GSK’s failure to optimize its dosing regimen for the antibody-drug conjugate in multiple myeloma.
  4. Johnson & Johnson’s $23.7 billion in second-quarter earnings, driven by cancer and neuroscience drugs, exceeded analyst expectations, while CEO Joaquin Duato set a target of $50 billion in oncology sales by 2030.
  5. In advance of this week’s adcomm, the FDA flags ocular toxicities associated with the antibody-drug conjugate, which received accelerated approval in August 2020 but was pulled from the market two years later after a confirmatory trial failed to improve progression-free survival.
NEUROSCIENCE
  1. Sarepta Therapeutics faces serious FDA action after news broke of a third patient death, the FDA gets a new top drug regulator in George Tidmarsh, a handful of new drugs get turned away from the market and pharma companies continue to commit billions to reshoring manufacturing.
  2. Otsuka and Lundbeck’s data are insufficient to establish significant efficacy of Rexulti plus sertraline in PTSD, according to the FDA’s outside experts.
  3. The patient, who was being treated with an investigational gene therapy for limb-girdle muscular dystrophy, died of acute liver failure, the same complication responsible for the deaths of two boys taking Sarepta’s Duchenne muscular dystrophy treatment Elevidys.
  4. FDA reviewers flag “discordant results” in a briefing document published ahead of Friday’s advisory committee meeting for the partners’ application for the antipsychotic in post-traumatic stress disorder.
  5. ALS
    Participants in trials of BrainStorm Cell Therapeutics’ NurOwn filed a Citizens’ Petition with the FDA earlier this month seeking a new review of the stem cell therapy that was rejected in 2022 based on real-world data and 90% survival in an expanded access program.
CELL AND GENE THERAPY
  1. Sarepta Therapeutics’ stock has dropped precipitously as questions swirl around the safety of its gene therapies. Meanwhile, the Duchenne patient community fears losing access to Elevidys while the regulator considers more drastic action.
  2. After initially refusing to suspend Elevidys distribution after two deaths, Sarepta has now given in to the FDA’s request, noting the need to maintain a good working relationship with the regulator.
  3. In light of recent patient deaths, the FDA has also revoked its platform designation for Sarepta’s AAVrh74 technology. The designation, granted last month, was the first of its kind to be announced publicly.
  4. Amid a season of regulatory and scientific advances, experts reveal a culture of data hoarding among cell and gene therapy developers that is reinforcing fragmentation, stalling innovation and delaying access to treatments.
  5. Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta Therapeutics adds a black box warning to the gene therapy for acute liver injury and failure and parts with more than a third of employees.