Dyne is eyeing an accelerated approval filing for DYNE-251 in early 2026 that would pit the asset against Sarepta’s Exondys 51 in a patient population amenable to exon 51 skipping.
Ionis and Ultragenyx are competing to develop oligonucleotide treatments for Angelman syndrome, but will Neuren’s peptide catch up?
The Maryland-based biopharma joins Eli Lilly and Novo Nordisk in trialing a GLP-1 agonist for alcohol- and liver-related conditions.
Pfizer was studying PF-07820435, an orally available agonist of the STING protein, for solid tumors.
Sutro’s stock tumbled nearly 19% after the company announced it will sideline its FRα-targeted antibody-drug conjugate luveltamab tazevibulin, which it was studying for ovarian cancer. The biotech will seek licensing opportunities for the asset.
Pliant follows in the footsteps of Acelyrin, which also enacted a stockholder rights program on Thursday to protect shareholders against Tang Capital’s growing stake in the company.
FEATURED STORIES
Sage Therapeutics discontinued development of its lead candidate dalzanemdor after a third clinical failure, leading analysts to question the biotech’s future profitability.
RFK Jr. as HHS head is perhaps President-elect Donald Trump’s most controversial Cabinet pick now that Matt Gaetz has withdrawn as nominee for Attorney General. With Dr. Oz tapped to lead CMS and maybe Marty Makary at the FDA, it’s going to be quite the show.
A handful of billion-dollar deals in the rare disease space highlights the uptick in Big Pharma’s investment, but it’s still extremely low compared to the money flowing to more common indications.
FROM BIOSPACE INSIGHTS
In a year when eradicated diseases are on the uptick in America, how will American children survive RFK Jr.’s vaccine scrutiny and inconsistency? Two experts call on pharma and regulatory bodies to rebuild trust.
LATEST PODCASTS
In this episode presented by IQVIA, BioSpace’s head of insights Lori Ellis discusses the importance of target product profiles, particularly when navigating funding challenges, with Ian Fisher, head of development analytics.
China continues to be a source of innovation as Pfizer strikes biggest pact yet; HHS provides more info on Trump’s Most Favored Nation executive order; FDA Commissioner Marty Makary and CBER director Vinay Prasad reveal new COVID-19 vaccine strategy following Novavax approval; ODAC underway after chaotic planning; more.
President Donald Trump unwrapped a massive drug pricing policy as CMS prepares for the next round of Medicare drug price negotiations; Vinay Prasad to take the helm at the FDA’s Center for Biologics Evaluation and Research; Bayer cuts 2,000 more employees; Eli Lilly’s Zepbound scores again; and the Galapagos story turns again.
Job Trends
The Merck Foundation, a private charitable organization funded by Merck (NYSE:MRK), known as MSD outside of the United States and Canada, has made an $11 million, six-year (2023 - 2028) commitment to University of New Mexico Health (UNM Health) in support of its new initiative to bring high-quality care to an estimated 11 million people living with cancer in underserved communities throughout India, Indonesia, Malaysia and Vietnam.
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SPECIAL EDITIONS
A new generation of checkpoint inhibitors is emerging, with some showing more promise than others. From recent TIGIT failures to high-potential targets like VEGF, BioSpace explores what’s on the horizon in immuno-oncology.
Peter Marks, the venerable head of the FDA’s Center for Biologics Evaluation and Research, has been forced out. In this special edition of BioPharm Executive, BioSpace takes a deep dive into the instability of the HHS.
Year-over-year BioSpace data show biopharma professionals faced increased competition for fewer employment opportunities during the first quarter of 2025.
DEALS
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Following rumors last week of a potential acquisition by Sanofi, Bristol Myers Squibb on Sunday announced that it paid $4.8 billion to gain access to Mirati Therapeutics’ oncology-focused portfolio. -
Though data became an issue in two separate meetings, the FDA’s Oncologic Drugs Advisory Committee made a potentially precedent-setting decision by voting in favor of US WorldMeds’ neuroblastoma treatment. -
Following a Phase III failure and the departure of its CEO, Mirati Therapeutics is rumored to be engaged in acquisition discussions with French pharma giant Sanofi, according to Bloomberg. -
After finally getting the green light from the Federal Trade Commission last month, Amgen has completed the buyout and expects to provide updated fiscal year 2023 guidance during its third-quarter earnings call. -
The Japan-based pharma is acquiring Orchard Therapeutics for approximately $477.6 million, if all conditions are met. Orchard’s pediatric gene therapy has a PDUFA date set for March 2024.
WEIGHT LOSS
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Oprah Winfrey this week shone the spotlight on these transformative GLP-1 medications. Now, it’s time for Medicare to cover them and expand access to millions of Americans. -
Following its label expansion earlier this month, Medicare on Thursday said it will now cover the use of Novo Nordisk’s Wegovy in patients with overweight or obesity who have preexisting cardiovascular disease. -
A new analysis from the Congressional Budget Office predicts that Novo Nordisk’s semaglutide will likely be subjected to Medicare’s Drug Price Negotiation Program under the Inflation Reduction Act. -
The recent FDA decision will likely mean more Medicare patients gain access to the blockbuster weight loss drug, experts say. Meanwhile, results continue to roll in for GLP-1 agonists for conditions beyond diabetes and obesity. -
Novo Nordisk’s investment of more than half a billion dollars is meant to expand its production capabilities in China and help it meet the global drug demand.
POLICY
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The U.S. Supreme Court on Thursday upheld access to the abortion pill mifepristone, unanimously rejecting a challenge by a group of anti-abortion doctors seeking to undo the FDA’s approval of the drug. -
Based on promising response data from the Phase I/II TRIDENT-1 study, the FDA on Thursday granted Bristol Myers Squibb’s Augtyro accelerated approval for NTRK-positive locally advanced or metastatic solid tumors. -
Shifts in the FDA’s approach present an unprecedented opportunity to solve problems with patient access and trial design. -
The BIOSECURE Act, which seeks to protect U.S. national security from Chinese biotech companies, will not be part of the House of Representatives vote on the 2025 fiscal year National Defense Authorization Act. -
The FDA has a packed calendar this week, with six decisions on the docket, including ones for Merck’s Keytruda, BMS’s Krazati and Sarepta’s Elevidys.
If you’ve never worked from home or had a flexible schedule, how do you portray to a recruiter or hiring manager that you have what it takes to be productive outside of the traditional office?
While you don’t want to be the person that survived mass layoffs only to turn around and ask for a salary bump for reasons you can probably guess, you also don’t want to be severely underpaid or underappreciated.
What factors do you think affect burnout? Do you think some industries are more prone to burnout than others?
Many people stepping into middle age feel that they’re too old to make a career change, but we live in times when an average person changes up to 12 jobs in a lifetime.
If you’ve been furloughed, it’s normal to feel some apprehension, fear and/or doubt about your employment. These are four ways to stay productive if you’ve been furloughed.
Most job applicants don’t know about questions that they should ask at the end of an interview. Here are some not-so-common interview questions that you must remember to ask.
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
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Coherus BioSciences and Junshi Biosciences’ PD-1 inhibitor Loqtorzi significantly boosted progression-free and overall survival in a late-stage study of patients with advanced hepatocellular carcinoma. -
The use of artificial intelligence in the development of cancer vaccines allows for individualized therapy, but the prospect of an ever-changing product poses new challenges for drug developers and regulators. -
AbbVie’s antibody-drug conjugate Elahere, developed by ImmunoGen, elicited a nearly 52% objective response rate in heavily pretreated patients with folate receptor-alpha-positive, platinum-sensitive ovarian cancer. -
BMS presented late-stage results on Tuesday at the ASCO annual meeting which showed the combination of Opdivo and Yervoy lowered the risk of death by 21% in patients with unresectable hepatocellular carcinoma, compared to two kinase inhibitors. -
In addition to missing the mark in overall survival, Gilead reported Thursday a higher number of deaths in the Trodelvy arm of the confirmatory metastatic urothelial cancer study.
NEUROSCIENCE
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AVP-786 was unable to significantly reduce total scores in the Cohen-Mansfield Agitation Inventory versus placebo, Otsuka Pharmaceuticals reported Monday. The company is evaluating the next steps for the drug combination. -
Thanks to a rough launch of its Biogen-partnered Alzheimer’s disease treatment, Eisai will likely miss its target of treating 10,000 patients with Leqembi by the end of March 2024. -
With one disease-modifying therapy already reaching patients and another expected to soon, several biopharma companies anticipate key data for novel assets in the coming 12 months. -
Biotech companies are exploring multiple targets in the fight against neuroinflammation. Experts say these projects could yield the next breakthrough in treating Alzheimer’s disease. -
After showing some unfavorable results for its data, AC Immune on Monday announced it will be getting back its Alzheimer’s disease candidates from Roche’s Genentech.
CELL AND GENE THERAPY
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Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a personalized in vivo CRISPR editing therapy, which substantially eased the symptom burden in an infant. -
Since Elevidys’ accelerated approval in 2023, experts have been clamoring for more data, particularly in older and non-ambulatory children. New results, presented Friday, show mobility improvements in 8- to 9-year-old patients after one year of follow-up. -
Lilly will use Rznomics’ proprietary ribozyme technology to develop RNA editing therapies for congenital hearing loss. -
After warnings that the dragged-out process was putting the cell therapy company at risk of bankruptcy, bluebird bio now has a new deal to offer shareholders. -
It’s another wild twist in the story of Galapagos, a company that has been around for more than 25 years but has yet to get a therapy approved.
