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
Big Pharma had plenty of drama to keep journalists busy this quarter, which painted an accurate portrait of the wild and wonderful world of biopharma.
A tale of two multi-billion schizophrenia deals, AstraZeneca touts strong sales while deflecting questions about an investigation into China exec, the Huntington’s pipeline builds momentum and layoffs continue with Sana Biotechnology and 23andMe.
Suggestions that the U.S. should emulate other countries on drug price controls or patents obscure how our present policies have allowed drug development to flourish.
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
HHS Secretary RFK Jr. removes the COVID-19 vaccine recommendation for healthy kids and pregnant women—the latest in a string of changes to vaccine policies; judge issues an order to halt HHS’ reorganization and mass layoff plans; Rocket Pharmaceuticals’ pivotal Danon disease trial is on hold after a patient death; and President Trump has named Mehmet Oz to spearhead his Most Favored Nation drug pricing policy.
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.
Job Trends
GenScript is excited to announce that Dr. David R. Liu, a prominent figure in the field of genome editing, has been appointed to the company’s scientific advisory board (SAB).
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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 a sweeping cost-cutting plan announced earlier this week, including an 11% workforce reduction, Biogen is acquiring Reata Pharmaceuticals to bolster its neurological and rare disease pipeline. -
The British drugmaker’s rare disease subsidiary Alexion will take on a number of Pfizer’s preclinical gene therapy programs and technologies to advance its genomic medicines pipeline. -
The Federal Trade Commission’s increased scrutiny of mergers has now hit IQVIA, whose proposed acquisition of Propel Media has been challenged by the watchdog agency. -
The Federal Trade Commission released new draft guidelines for assessing mergers, while an Alzheimer’s conference yielded promising data and J&J kicked off Q2 earnings season with a sound beat. -
The proposed regulatory framework provides a tougher stance on mergers, impacting industries such as biopharma where the FTC’s recent lawsuit seeks to block Amgen’s Horizon Therapeutics buy.
WEIGHT LOSS
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The biotech is taking a synergistic approach to obesity with two muscle-preserving antibodies set to enter a Phase II study in mid-2024 in combination with existing incretin-based treatments. -
Driven by soaring demand for its diabetes and weight loss drugs, Novo Nordisk on Wednesday reported better-than-expected full-year 2023 earnings with a 31% sales increase. -
Stringent regulation, manufacturing costs and absence of price control contribute to high GLP-1 agonist costs in the U.S. Patients could go to other countries for cheaper treatment. -
The persistent shortage of genuine glucagon-like peptide 1 receptor agonist products has led to an increase in fake versions, the World Health Organization warned on Monday. -
More than 3,000 people in the U.S. suffered from side effects of Ozempic in 2023, according to exclusive reporting on Wednesday from Reuters citing data from America’s Poison Centers.
POLICY
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Regeneron and Sanofi will have to wait until September 2024 for the FDA to decide whether to expand Dupixent’s label to include uncontrolled chronic obstructive pulmonary disease. -
As evidenced by this week’s buyouts by J&J and Merck, Big Pharma appears to have found a sweet spot favoring smaller deals over megabillion-dollar acquisitions. -
Tris Pharma on Wednesday secured the FDA’s green light for Onyda XR, the first liquid non-stimulant nighttime treatment for attention deficit hyperactivity disorder in pediatric patients. -
In the next two weeks, the FDA is scheduled to decide on four drug applications and hold two highly anticipated advisory committee meetings. -
Experts say approval of Lykos Therapeutics’ MDMA capsules for post-traumatic stress disorder would open the door to further research into psychedelic-assisted therapies.
Establishing rapport with someone you’ve just met who also may hold your future in their hands is not easy. Continue reading to discover how to establish rapport with your interviewer.
The first few minutes of your job interview are critical, as studies show hiring managers form strong impressions to make their hiring decisions based on these early minutes of the interview.
Here are three tips to help you land a management position when you’ve never been a manager.
It has been said that qualifications are more of a “wish list” for employers, who do not expect to find someone who meets all the requirements.
Simply staying organized will help you make sure you never miss a beat. Here are a few small steps that’ll make a big difference.
When using the STAR Method, be sure to discuss the who, what, where, when and how of an experience.
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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The new company, BridgeBio Oncology Therapeutics, is looking to advance two KRAS inhibitors and a blocker of the interaction between the RAS and PI3K pathways. -
Based on strong overall and progression-free survival data in its Phase III confirmatory study, Pfizer and Genmab’s antibody-drug conjugate Tivdak on Monday secured the FDA’s full approval for recurrent or metastatic cervical cancer that had progressed on or after chemotherapy. -
AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu significantly improved progression-free survival in metastatic breast patients with low and ultralow HER2 expression levels who had received at least one line of systemic treatment. -
The FDA on Tuesday approved Day One Biopharmaceuticals’ type II RAF inhibitor Ojemda, which is designed to penetrate the blood-brain barrier, for the treatment of relapsed or refractory pediatric low-grade glioma. -
Bouncing back from a previous rejection, ImmunityBio on Monday secured the FDA’s green light for its IL-15 superagonist Anktiva for non-muscle invasive bladder cancer.
NEUROSCIENCE
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Following the full approval of Leqembi, the CMS suggests expanding its coverage of beta-amyloid PET scans, which could improve the uptake of novel Alzheimer’s disease treatments. -
Part B of the study, cleared to proceed in Canada, remains on hold in the U.S. due to findings from non-clinical chronic toxicology studies. -
Full data for Eli Lilly’s Phase III TRAILBLAZER-ALZ 2 study, presented Monday at the 2023 Alzheimer’s Association International Conference, confirm positive results announced in May. -
Recent data from the Phase III study of donanemab emphasize a correlation between amyloid and tau. Experts say a greater understanding of this link could further Alzheimer’s drug development. -
Following the FDA’s full approval of the Alzheimer’s drug Thursday, the Centers for Medicare and Medicaid Services confirmed broader coverage of Leqembi and released more details on a registry.
CELL AND GENE THERAPY
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While an adverse event reported in Intellia’s gene therapy trial was a “non-concern” for analysts, it follows a handful of patient deaths in other trials for the modality and sent the company’s stock tumbling in pre-market trading. -
Acute systemic infection caused the patient to develop fatal capillary leak syndrome, highlighting the unpredictability of gene therapies and potentially challenging investment in the space, analysts say. -
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.
