STAT+: Pharmalittle: We’re reading about another AstraZeneca trial failure, a Novo name change, and more
STAT's Pharmalittle roundup leads with AstraZeneca disclosing that camizestrant, a breast cancer pill, failed a pivotal trial testing it as a first-line therapy in advanced tumors. In combination with another medicine, camizestrant did not outperform standard treatment on a measure of how long patients went before their cancer progressed, a result that could limit the medicine's use. Earlier this month, the drug won accelerated U.S. approval for patients who develop a mutation in their tumors that signals emerging drug resistance, and a successful first-line trial could have opened a much larger market.
The roundup also reports that Scholar Rock received FDA approval for Isembyld, described as the first-ever therapy targeting the loss of muscle in spinal muscular atrophy. The approval is for adults and children 2 years and older who are currently receiving SMA therapies that target SMN2, a key gene for neurons that control movement. The company and clinicians hope this gives patients with the rare neurological disorder a better chance of moving and walking independently.
The item notes the approval as another step forward for spinal muscular atrophy, a rare and progressive disease that destroys nerve cells controlling speaking, breathing, and walking; babies who go untreated usually die by the age of 2. The Pharmalittle headline also references a Novo name change and other news, though the excerpt does not provide details on those items.