Drug Development
The FDA’s priority review acceptance of BridgeBio’s BBP-418 is another step toward what William Blair previously dubbed a “diversified commercial portfolio.” It also adds to the rapidly building momentum in muscular dystrophy more broadly.
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Partners Summit Therapeutics and Akeso are expected to steal the show at the American Society of Clinical Oncology’s annual conference with data from their potential Keytruda rival, alongside Revolution Medicine’s groundbreaking pancreatic cancer candidate and other assets that could reshape patient care.
The tragic tale of TIGIT is well known. However, RIPK1, myc, STING and alpha-synuclein have also left a trail of failed clinical trials, canceled partnerships and sunk investments in their wake.
Analysts homed in on Duchenne muscular dystrophy and myotonic dystrophy type 1 assets during first quarter earnings as major players like REGENXBIO and Novartis as well as Dyne, Wave, Solid and Sarepta near the regulatory finish line.
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The investigational kinase inhibitor failed to improve overall survival in the SAPPHIRE study of patients with non-squamous non-small cell lung cancer, causing Mirati to discontinue its development.
The company’s candidate, ANX007, did not significantly slow lesion growth in a Phase II trial but was effective at preserving visual function in patients with a form of dry age-related macular degeneration.
Wave will discontinue development of WVE-004 in C9-associated ALS and frontotemporal dementia after the therapy failed to show any clinical benefit in a mid-stage trial.
Despite a disappointing showing in the Phase III MOVE-FA trial, PTC Therapeutics says it will move forward with regulatory talks for the Friedreich’s ataxia treatment. Meanwhile, the company is discontinuing several early-stage gene therapies and reducing its headcount.
The Swiss pharma has canceled a second mid-stage trial for its investigational schizophrenia drug ralmitaront, as the competition shows promise in the space.
Data from a Phase III trial of apraglutide for a type of short bowel syndrome with intestinal failure is expected by the end of the year. It is also in a Phase II study for acute graft-versus-host disease.
The 61-patient study met the primary endpoint of improved physiologic liver function and secondary endpoints, which included established non-alcoholic steatohepatitis biomarkers, Hepion reported Monday.
Sanofi and Regeneron reported positive results for the anti-inflammatory drug in a chronic obstructive pulmonary disease trial. The companies hope to see approval before a 2024 read-out of a second trial.
More than $5 billion poured into the longevity space in 2022, and experts say the science is now primed to make a real difference in extending human healthspan.
In a 12-2 vote with two abstentions, the advisers said the benefits did not outweigh the risks.