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 Phase III trial met its primary endpoint, a significant reduction of phenylalanine levels in the blood, with no severe adverse events reported, PTC Therapeutics reported Wednesday.
Fenebrutinib in a mid-phase trial reduced brain legions in relapsing forms of multiple sclerosis, the company reported. It is the only reversible BTK inhibitor currently in Phase III MS trials.
Results from Phase IIb VOYAGE study showed Viking’s oral thyroid beta receptor agonist cut liver fat content by up to 51% in confirmed non-alcoholic steatohepatitis patients.
ReCode plans to use its SORT LNP genetic medicines delivery technology to treat CF and PCD patients who don’t respond to current treatments.
The precision neuroscience startup has notched $109 million in seed, Series A and B financing as it looks to further develop a Phase I candidate to treat a rare X-linked neuromuscular disorder.
Veozah, an oral once-daily medication, is the first FDA-approved NK3 receptor antagonist for hot flashes, a common symptom of menopause that can affect health and quality of life.
Preliminary trial results show the GSK five-in-one meningitis vaccine is immunologically effective against the most common strains, as the FDA decision date for Pfizer’s candidate looms.
Nirsevimab lowered hospitalizations due to RSV-related lower respiratory tract disease by 83.21%. Friday’s data drop comes ahead of next week’s advisory committee meeting for Pfizer’s RSV hopeful.
The FDA has four events lined up this week, including a target action date for Krystal Biotech’s gene therapy for a rare skin disease and an adcomm meeting for Pfizer’s RSV vaccine candidate.
The approval of Reata Pharmaceuticals’ Skyclarys for Friedreich’s ataxia highlights progress being made in the treatment of rare mitochondrial diseases.