Drug Development News

Kaos Capital in a letter to other Capricor Therapeutics shareholders called for “an immediate meeting, board change, and capital-preservation plan.” The biotech recently put all other pipeline work on pause as it awaits an Aug. 22 PDUFA date for embattled cell therapy deramiocel.

The Phase 3 study is evaluating two doses of Novo’s Wegovy pill in people with obesity or overweight in combination with a reduced-calorie diet and increased physical activity.

Scribe Therapeutics’ one-time epigenetic treatment is designed to slash the adherence burden of chronic cholesterol-lowering therapies while sidestepping the irreversibility of gene editing.

The accelerated greenlight for Ultragenyx’s gene therapy for glycogen storage disease has raised analyst expectations for approval of UX111, which the FDA rejected last summer and is currently reviewing for a second time. A decision is expected by mid-September.

Investors had been pessimistic about the probability of success for the personalized cancer vaccine intismeran autogene, so the Phase 3 win was a welcome surprise.

In this episode of Denatured, you’ll hear from Ian McGowan, Chief Medical Officer at Synklino and James Hunter, associate professor of transplantation at Oxford University. We explore transplantation and antiviral innovation and why CMV remains one of the most persistent and consequential challenges facing transplant patients today.

Real progress happens where unmet medical need is greatest and patient impact can be clearly measured. Pediatric rare cancers exemplify these criteria and have therefore generated continued investment, even in a capital-constrained environment.

Melanoma was slower to recur in patients receiving Merck and Moderna's mRNA vaccine with Keytruda, supporting use of the combination in adjuvant treatment. The partners plan to take the data to the FDA.

Touting an “exceptional” gastrointestinal safety profile for its oral obesity drug candidate in a study of healthy volunteers, Enveda plans to test the asset in people stopping GLP-1 treatment.

The discontinuation of the uveitis study adds to Regeneron’s series of clinical setbacks, including the Phase 3 failures of the Dupixent follow-up itepekimab and an investigational combo regimen for melanoma.

With a Phase 3 trial showing a reduction of serious and severe hypoglycemic episodes in patients who have undergone weight loss surgery, Amylyx Pharmaceuticals plans to file for approval this year. If granted market entry, avexitide could hit peak sales of $1.7 billion worldwide, analysts estimate.

EyePoint’s quest to reduce treatment burden for patients with age-related macular degeneration has hit a bump in the road, with a clinical miss driving the biotech’s stock down by 70%.

Ivonescimab notched its third approval in China last week, but the label update has tempered expectations for the Summit Therapeutics/Akeso drug, showing that progression-free survival declines over time.