Clinical research
New Phase I/II trial results show that one more type 1 diabetes patient achieved insulin independence after treatment with Vertex Pharmaceuticals’ investigational stem cell therapy VX-880.
The Japanese pharma is voluntarily withdrawing its lung cancer drug mobocertinib, marketed as Exkivity, from U.S. and global markets after it missed the mark in a Phase III confirmatory trial.
On Monday, Syndax Pharmaceuticals announced that its menin inhibitor revumenib met the goal in a pivotal leukemia study and stopped the trial early. Their stock price still dropped on the news.
The Swiss pharma will seek accelerated approval for iptacopan in IgA nephropathy next year after the complement blocker demonstrated promising efficacy in the Phase III APPLAUSE-IgAN study.
Shares of the biotech startup jumped as much as 70% on Friday after the company reported promising early-stage results for a four-week trial that included only 24 patients.
An FDA advisory committee this week voted overwhelmingly against BrainStorm Cell Therapeutics’ amyotrophic lateral sclerosis treatment. However, other potential therapies offer hope for ALS patients.
Following disappointing results in a mid-stage social anxiety disorder study, the Australian biotech’s investigational ion channel modulator demonstrated promising effects in patients with post-traumatic stress disorder.
If approved by the FDA, Karuna Therapeutics’ investigational agonist of muscarinic acetylcholine 1 receptors M1 and M4 would provide the first new mechanism of action for schizophrenia patients in decades.
Non-small cell lung cancer patients treated with the drug combination saw a statistically significant and clinically meaningful improvement in progression-free survival compared to AstraZeneca’s osimertinib.
After picking up magrolimab in a $4.9 billion purchase of Forty Seven, a second Phase III study is being discontinued for the anti-CD47 antibody, this time in acute myeloid leukemia.
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