Biotech
Heart failure drug from Tenax Therapeutics fails in pivotal study
Tenax Therapeutics reported negative results this morning from a late-stage clinical trial evaluating an experimental treatment for a type of heart failure associated with high blood pressure in the lungs.
The Tenax drug, an oral formulation of levosimendan, failed to improve exercise capacity or improve overall symptoms compared to a placebo in a Phase 3 study called LEVEL, the company said.
Shares of the biotech company plunged 84% to $2 in early trading.
There are no medicines approved to treat the heart failure condition, called PH-HFpEF. An effective treatment could deliver billions of dollars in sales, which is why investors were closely tracking the Tenax study, even if the outcome was thought to be a crapshoot.
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Biotech
Silence Therapeutics aims at Takeda with new data in rare blood cancer
An experimental medicine from Silence Therapeutics stabilized red blood cell levels in patients with a rare blood cancer in a new trial — a result that could position it to compete with a drug nearing approval from Takeda Pharmaceuticals.
The cancer, polycythemia vera, is marked by runaway production of red blood cells and puts patients at risk of life-threatening blood clots, bleeds, and other cancers.
In the 48-person, Phase 2 trial, 88% of patients who received Silence’s drug, called divesiran, every six weeks or 12 weeks achieved a clinical response, the company said this morning. By contrast, only 19% of patients on placebo did.
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chronic disease
Sionna’s experimental cystic fibrosis pill falls flat in mid-stage trial
Sionna Therapeutics said this morning that its experimental pill for cystic fibrosis did not show any benefit when added onto Vertex Pharmaceuticals’ Trikafta in a Phase 2 trial.
The company will no longer try to advance the drug, called SION-719, as an add-on therapy for CF patients. And it hinted at potential layoffs, saying it will “take actions to preserve capital while evaluating next steps.”
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diagnostics
FDA panel to weigh Grail's Galleri test
Grail’s closely watched Galleri cancer blood test is headed before an FDA advisory committee on Sept. 23, the company announced. This is a key step toward what could become the first FDA-approved multi-cancer early detection test. The company submitted Galleri for approval in January, backed by data from more than 95,000 participants in its PATHFINDER 2 and NHS-Galleri studies.
But the review comes with a significant wrinkle: Grail’s landmark NHS trial failed earlier this year to significantly reduce late-stage cancer diagnoses, though the company reported more encouraging results in a subset of 12 cancers. Grail, which sold more than 61,000 Galleri tests last quarter, continues to expect an FDA decision in the first half of 2027.
rare disease
Shortage of Sanofi medicines for Pompe disease rattles patients
Sanofi is experiencing shortages of two medicines used to treat Pompe disease, and the problem emerged shortly after the FDA warned the company over manufacturing issues at the Irish facility where the drugs are made.
In recent days, the drugmaker has alerted patient groups and physicians in both the U.S. and Europe that supplies of Myozyme, an older standard of care, and Nexviazyme, a newer treatment, are dwindling due to what has been described as a bottleneck in the final manufacturing phase, as well as quality control issues at its Waterford plant, STAT's Ed Silverman reports.
For now, it is not clear when normal supplies will return because Sanofi has indicated it could take months before production has sufficiently resumed and stabilized. But the shortage will continue to reverberate after that occurs, because it will likely take time for normal inventories to be rebuilt.
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