AMX0035—approved as Relyvrio in 2022 for amyotrophic lateral sclerosis but voluntarily pulled from the market last year—was unable to distinguish itself from placebo in a mid-to-late-stage trial of progressive supranuclear palsy.
Novartis has bet up to $772 million to gain access to BioArctic’s BrainTransporter platform, which was leveraged in a partnership with Eisai to produce Leqembi.
Regeneron’s cemdisiran, used alone or in combination with its complement inhibitor Veopoz, significantly improved activities of daily living in patients with generalized myasthenia gravis.
The layoffs will affect employees at Pfizer’s Bothell, Washington site, which previously served as the headquarters for Seagen before being acquired by the pharma for $43 billion.
Eli Lilly drops a second Phase III readout for orforglipron; AbbVie committed to the psychedelic therapeutics space with the $1.2 billion acquisition of Gilgamesh’s depression asset; the CDC taps vaccine skeptic Retsef Levi to lead its COVID-19 immunization working group; and the FDA prioritizes overall survival in cancer drug development.
While Truist Securities analysts said the results from the ATTAIN-2 trial leave “room for competition,” they also pointed to a manufacturing advantage that could unlock a “double-digit billion dollar opportunity” for Eli Lilly.
FEATURED STORIES
From innovation in manufacturing to more-flexible regulation and better communication with payers, much needs to happen to make CGTs commercially viable. But it is possible, experts agreed at a recent panel.
The primary focus in scaling up production should first be the adoption of lean manufacturing principles used in virtually every other industry.
After decades of limited progress—owing to the difficulty of treating the disease and resultant market risk—glioblastoma research is entering a new phase spurred by smarter trials, targeted funding and renewed interest from companies like Merck and Jazz Pharmaceuticals.
Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy families have turned to the news for answers. Meanwhile, the FDA insists it remains committed to notifying companies of any regulatory action before sharing information with the media or public.
Against steep odds and well-established paradigms, these four companies have successfully been commercializing their products on their own.
Though nerves abound for funders and founders in the industry, money continues to flow into startups, sometimes in eye-popping numbers. BioSpace rounds up the biggest raises so far this year.
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
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.
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.
In this episode presented by IQVIA, BioSpace’s head of insights Lori Ellis discusses the FDA’s first draft guidance for AI in drug development, published in January 2025, with Archana Hegde, senior director, pv systems and innovations at IQVIA.
Job Trends
Companies are announcing significant investments in U.S. manufacturing in response to looming tariffs. An AstraZeneca executive and Eli Lilly and Novo Nordisk spokespeople discuss potential job and skill-building opportunities and where manufacturing might head in the future.
Subscribe to Genepool
Subscribe to BioSpace’s flagship publication including top headlines, special editions and life sciences’ most important breaking news
SPECIAL EDITIONS
The job response rate has risen year over year, according to BioSpace data, indicating competition for roles posted on our website has increased.
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.
DEALS
-
BioNTech said in 2022 that it faced “threats of a groundless patent infringement suit” from a company that was “unable to bring to market any product to help in the fight against COVID-19.” Now, the mRNA biotech is buying that very company. -
Sanofi paid a more than 300% premium on its acquisition of Vigil Neuroscience, suggesting a fierce battle to seal the deal. Across biopharma, companies are sometimes willing to put it all on the line for the right buyout. Novartis’ recent acquisition of Regulus for $800 million upfront provides a case study. -
The pending deal was rumored overnight after a report from the Financial Times, spurring analysts to speculate that if true, the entire gene editing space would see a boost at the markets. -
Big Pharma executives have not been shy about their desire for deals, but companies have been battling macro headwinds alongside Trump’s policies on drug pricing and tariff threats. -
At a satellite kickoff event to the annual BIO meeting, investment bankers and VCs gave reasons for optimism amid a ‘volatile’ period for the industry.
WEIGHT LOSS
-
After a season of regulatory upheaval, obesity and rare genetic diseases will likely remain major themes for biopharma in 2025, according to Jefferies. -
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. -
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. -
In the race to make the most tolerable obesity drug, there seems to be no clear winner—at least not according to analysts parsing the data presented at the American Diabetes Association annual meeting this week. -
Eli Lilly’s tirzepatide is expected to be worth $62 billion annually by 2030, according to Evaluate. That valuation would be three times larger than what AbbVie’s blockbuster Humira ever achieved.
POLICY
-
Gray Delany’s ouster comes days after Health Secretary Robert F. Kennedy Jr. controversially axed 22 mRNA vaccine contracts under the Biomedical Advanced Research and Development Authority. -
Skysona can now only be used in patients with cerebral adrenoleukodystrophy who have no available treatment alternatives or stem cell donors. -
The recent announcement of RFK Jr.’s termination of mRNA vaccine contracts is the latest effort to undermine this promising technology at the federal level. Pharmaceutical companies and private investors must fill the gap and ensure that research into this critical resource continues. -
After exiting the FDA less than two weeks ago for unclear reasons, Vinay Prasad is once again director of the Center for Biologics Evaluation and Research, HHS confirmed to several outlets Saturday. -
Under PreCheck, the FDA will communicate more frequently with pharmaceutical companies, helping them as they establish or expand manufacturing sites in the U.S.
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.
Managers face a variety of challenges during their careers. Instead of falling into fight or flight, develop resilience to navigate the uncertain moments that pop up from time to time.
HOTBEDS
REPORTS
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.
If people are your greatest asset, it is imperative that your organization maintains a positive employer brand presence and talent pipeline. This report covers how you can build a strong employer brand with prospective employees, current employees - and former employees.
CANCER
-
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. -
The panelists flagged safety concerns with Blenrep and GSK’s failure to optimize its dosing regimen for the antibody-drug conjugate in multiple myeloma. -
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. -
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.
NEUROSCIENCE
-
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. -
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. -
Around 3,500 FDA employees received termination emails; FDA Commissioner Marty Makary suggests lowering industry user fees and tying review times to drug prices; the regulator opens its trove of complete response letters in the name of transparency; and two companies receive rejections for rare disease therapies. -
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. -
Takeda’s oveporexton improved wakefulness, attention and other key narcolepsy endpoints “with a high degree of statistical significance,” according to Jefferies analysts.
CELL AND GENE THERAPY
-
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.
-
Cell and gene therapy leaders say the agency’s decision to remove the Risk Evaluation and Mitigation Strategies that had been attached to approved CAR T cancer therapies reflects “thoughtful consideration of real-world evidence” and “regulatory trust.” -
The pivotal trial for Neurogene’s Rett syndrome gene therapy makes use of baseline controls and a rigorous endpoint that could help ensure a broader label for the drug product, if approved, according to analysts. -
The all-cash buyout, which gives AbbVie access to Capstan Therapeutics’ in vivo edited CAR T therapy for B cell–mediated autoimmune diseases, adds to a growing sense of momentum in M&A, according to BMO Capital Markets. -
Changing how biopharmas package their products, how regulators review new drugs and how mutated genes are fixed could make ultrarare disease treatments possible.
