News
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.
FEATURED STORIES
Jefferies has predicted more small tuck-in deals to come, as biotechs struggle to access capital despite key clinical milestones on the horizon.
A new report from Pitchbook suggests we’re in for a period of more sustainable investing, with VC firms continuing to create and invest in companies, just more carefully.
The overturning of the FDA’s lab-developed tests rule is just the tip of the iceberg. With the loss of Chevron deference, power has shifted from federal agencies to the courts, with potential implications for everything from the FDA shortage list to CMS drug price negotiations.
Job Trends
Massachusetts’ biopharma jobs increased 2.6% in 2023, according to the MassBio Industry Snapshot. Whether the state’s jobs grow in 2024 remains to be seen based on this year’s layoffs and seemingly slowed hiring based on BioSpace data.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
Vaccine skepticism is at an all-time high in the U.S., and HHS Secretary Robert F. Kennedy Jr. is making some drastic moves in the name of reversing that trend. But misinformation and inconsistencies within the country’s healthcare agencies highlight problems with his approach.
THE LATEST
The star of Monday’s deal is gusacitinib, a small-molecule drug that Formation is developing for chronic hand eczema. Sanofi will explore additional indications for gusacitinib in a Phase I study.
Eli Lilly’s bimagrumab led to weight loss that was due almost entirely to fat reduction when combined with semaglutide, marketed by rival Novo Nordisk as Wegovy. BMO Capital Markets called the data “impressive” while raising concerns about the antibody’s safety profile.
Drug pricing, budget cuts, tariffs and other shifts under the Trump administration undermine the biopharma and healthcare ecosystem.
Speaking at BIO2025, rare disease leaders from Ultragenyx, Amylyx and Yale questioned the need for the new regulatory pathway proposed by FDA Commissioner Marty Makary. They acknowledged, however, that creative thinking is required to enable more treatments for patients with ultrarare diseases.
Jacqueline Corrigan-Curay, who stepped into the role as the agency’s top drug regulator in January, is departing in July, according to an email sent to agency staff.
While Eli Lilly brushed off concerns about gastrointestinal side effects for oral weight loss candidate orforglipron, analysts from William Blair worried that adverse events are not tapering off as expected.
In combination with Roche’s PD-L1 blocker Tecentriq, zanzalintinib bested Bayer’s Stivarga. Exelixis is positioning the drug candidate as a successor to cabozantinib, which is set to lose patent exclusivity in 2030.
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.
Although the company withheld detailed findings from the study of treatment-resistant depression, analysts at Stifel called COMP360’s efficacy “more than good enough” for registrational purposes.
While BMO Capital Markets said that zimislecel is “highly encouraging” for type 1 diabetes, questions regarding its target population and Vertex’s execution hang over the cell therapy’s commercial potential.