The Department of Health and Human Services’ mRNA pullback only applies to their use in upper respiratory disease, according to Secretary Robert F. Kennedy Jr.
As Trump has pressured drugmakers to lower the cost of medicines in the U.S., the pharma industry has coalesced behind a message of rebalancing what nations pay to better reflect the innovation and value of drugmaking.
The Boston-based AI/ML startup focuses on endocrine and cardiometabolic diseases and will use that expertise to generate new small molecule obesity medications for Lilly.
After two patients who received the investigational CDC7 blocker died, pushing forward with SGR-2921’s development would be “difficult,” according to Schrödinger, whose stock dropped 17.5% before the opening bell on Thursday.
The death was linked to acute kidney injury in a patient who had a single kidney remaining and a “complex medical history,” according to CytomX.
Vedanta is parting ways with 23 employees, or approximately 20% of its headcount, after Phase II data for microbiome therapy VE202 failed to demonstrate significant response rates in patients with ulcerative colitis.
FEATURED STORIES
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.
From Wall Street to real estate to a “big, ugly” pharma building, Mayo Venture Partner Audrey Greenberg reflects on a career defined by taking a leap at just the right moment.
Some of the most high-profile acquisitions in recent years have involved women-fronted biotechs. BioSpace reviews five of the most notable here.
Seven biotech unicorns are advancing AI-powered drug discovery and development—but must contend with a difficult investing environment where competition is steep and the usual roads to exit are uncertain.
More than thirty years since its 1993 founding, Catherine Owen Adams and Elizabeth Thompson—the R&D combo that has led Acadia since last year—are managing two products on the market and a pipeline estimated to be worth an additional $12 billion in sales.
New data and analyses presented at the American Diabetes Association’s annual meeting highlight the priorities for the next generation of weight loss medicines: muscle preservation, limited side effects and novel targets.
FROM BIOSPACE INSIGHTS
Takeda Oncology US Head of Sales, Annette Montandon, discusses how embodying a patient-first approach is key to the success of colorectal cancer drug FRUZAQLA®.
LATEST PODCASTS
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.
HHS Secretary Robert F. Kennedy Jr. made waves this week, firing the remaining members of the CDC’s Advisory Committee on Immunization Practices; Metsera’s amylin drug produced weight loss of 8.4% at 36 days; and FDA leaders gathered last week to discuss the future of cell and gene therapy, a sector that has been in turmoil since the ousting of CBER Chief Peter Marks.
Job Trends
Known as the home of big-name companies including Johnson & Johnson, New Jersey has become a destination for more and more small and midsize life sciences businesses. Choose New Jersey, Grace Therapeutics and Onyx Equities executives discuss the state’s life sciences scene.
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SPECIAL EDITIONS
In this deep dive, BioSpace examines what’s next for Leqembi, the true cost of anti-amyloid antibodies, and what other Alzheimer’s treatments are coming down the pipeline.
If it feels like there has never been a tougher time to look for work, you’re not alone—and you’re likely not wrong.
In this deep dive, BioSpace examines how small, medium and large companies are using artificial intelligence and machine learning to enhance their drug discovery efforts.
DEALS
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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. -
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. -
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. -
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. -
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
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After consistently failing to meet investor expectations, Novo Nordisk touted a safety profile for CagriSema in line with the GLP1-RA class, while reporting mid-stage data for its GLP1- and amylin-targeting drug amycretin that raised dosing questions. -
Analysts at William Blair say dapiglutide’s 11.6% weight reduction at 28 weeks could still be better, given that Zealand’s study predominantly included men and enrolled patients with lower BMI at baseline. -
In combination with Eli Lilly’s tirzepatide, marketed as Zepbound for obesity, Scholar Rock’s monolonal antibody helped patients lose the same amount of weight as patients on tirzepatide alone while preserving more muscle mass. -
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. -
At 12 weeks, weight loss ranged from 2.6% to 11.3%, compared to a gain of 0.2% in the placebo group. Guggenheim analysts were also impressed by the tolerability profile.
POLICY
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George Tidmarsh has only been at the Center for Drug Evaluation and Research for nine days, but will now add supervision of a second FDA division to his portfolio after Vinay Prasad’s sudden departure. -
As analysts parsed news of Vinay Prasad’s ouster, worries over drug approval delays, cell and gene therapy impacts and more were top of mind. -
Monarez is the first CDC director to be confirmed by the Senate under a new 2023 law and will be the first person without a medical degree to assume leadership of the agency in more than 70 years. -
Under the Inflation Reduction Act, medications with the same active ingredient will be treated as the same drug for price negotiation purposes—even if approved by the FDA under a separate application—disincentivizing companies from investing time and money in gaining approval for new formulations and indications. -
The FDA’s dramatic summer continues to unfold as news broke late Tuesday evening that Vinay Prasad will depart the agency, where he had been the head of the Center for Biologics Evaluation and Research for less than three months.
This webinar will help you discover effective strategies for launching your career in biotechnology. You will gain valuable insights from industry experts and leave with the essential tools you need to thrive in this dynamic field.
A clinical research associate is a professional who oversees clinical trials. Here are our top tips on how to become one.
For biopharma executives who are between roles, navigating the transitionary time can be challenging. However, they can remain visible and valuable so they’re ready to seize their next big opportunity.
In a tough labor market where layoffs continue, some are considering—or even moving—from industry to academia, according to a BioSpace poll. A career coach, scientist and general practitioner turned research coordinator share their thoughts.
Learn how to craft a resume that stands out by exploring 10 common resume mistakes and how to avoid them.
Carlos Doti has moved countries and changed jobs, but one thing hasn’t changed. Making an impact on patient care has always been a priority for the AstraZeneca executive.
HOTBEDS
REPORTS
This labor market report examines Q3 life science job market trends and the recruitment outlook for Q4 and beyond.
BioSpace surveyed life sciences employers to understand attitudes and current trends on AI usage in recruiting. This report explores the benefits of using AI tools in recruitment and provides practical recommendations for HR and talent acquisition professionals to leverage AI effectively. Concurrently, this report highlights the challenges and risks of using these tools without critical thought and intention.
As competition for life sciences talent peaks, BioSpace has undertaken research to determine what employers can do to ensure they don’t lose out on talent they actually want to hire and retain.
CANCER
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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. -
Shanghai-based LaNova Medicines—which has captured the attention of some of the biggest Western pharmas—will be folded into fellow Chinese company Sino Biopharmaceutical in a deal worth up to $951 million. -
Only with the adoption of digital imaging and AI-powered analysis will next-generation precision oncology therapies reach their full potential and ensure no eligible patient is overlooked. -
In this episode presented by Eclipsebio, BioSpace’s head of insights Lori Ellis discusses mRNA and srRNA with Andy Geall of Replicate Bioscience and Alliance for mRNA Medicines, and Pad Chivukula of Arcturus Therapeutics. -
The deal, which involves a $700 million upfront payment, gives AbbVie access to ISB 2001, a clinical-stage first-in-class trispecific antibody currently being tested for certain kinds of multiple myeloma as well as autoimmune indications.
NEUROSCIENCE
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BrainStorm Cell Therapeutics issued a statement Tuesday supporting a Citizens’ Petition submitted to the FDA requesting the approval of its cell therapy NurOwn, whose BLA was withdrawn in 2023. A Phase IIIb trial was scheduled to begin last month. -
After a season of regulatory upheaval, obesity and rare genetic diseases will likely remain major themes for biopharma in 2025, according to Jefferies. -
The high court sides with HHS on HIV PrEP drugs; Health Secretary RFK Jr.’s newly appointed CDC vaccine advisors discuss thimerosal in flu vaccines, skip vote on Moderna’s mRNA-based RSV vaccine; FDA removes CAR T guardrails; AbbVie snaps up Capstan for $1.2B to end first half; and psychedelics take off again with data from Compass and Beckley.
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BPL-003 showed “robust” efficacy data in treatment-resistant depression, according to analysts from Jefferies, who noted that the asset could hit peak market sales of $1 billion. The results clear the way for the asset’s late-stage development and for the completion of a proposed merger with atai Life Sciences. -
The rise of monoclonal antibodies brought back hope for stalling or reversing the devastating neurodegenerative disease. Big Pharma has taken notice with a handful of high-value deals, GlobalData reports.
CELL AND GENE THERAPY
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Changing how biopharmas package their products, how regulators review new drugs and how mutated genes are fixed could make ultrarare disease treatments possible. -
Jefferies analysts called the proxy filing, which is a standard disclosure after a merger agreement, “much more intriguing than normal” given the regulatory turmoil it revealed. -
The FDA is assessing the need for “further regulatory action” on Sarepta’s Duchenne muscular dystrophy gene therapy in the aftermath of two patient deaths, though the regulator has not yet specified what action this could be. -
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.
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Isaralgagene civaparvovec is a “potential best-in-class gene therapy for Fabry disease,” according to analysts at H.C. Wainwright. Sangamo plans to use pivotal Phase I/II data to build an accelerated approval case for the asset.
