Drugs

Imbria Moving Heart Drug to Phase 3

Imbria Pharmaceuticals says it is planning a Phase 3 study of ninerafaxstat, a novel cardiac mitotrope and partial fatty acid oxidation inhibitor, in patients with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM). The move comes as the company is reporting data from its Phase 2 IMPROVE-HCM clinical trial, which it says showed that ninerafaxstat was well-tolerated, with no safety concerns observed.

It demonstrated that at the 12-week mark the therapy was associated with a statistically significant improvement in a robust functional cardiopulmonary exercise test, a well-established prognostic measure for patients with nHCM and heart failure with preserved ejection fraction, according to the company. “This was associated with a clinically relevant improvement in patient-reported outcomes,” the company says.

“Ninerafaxstat is the first investigational drug to show an improvement in a functional measure assessed by a cardiopulmonary exercise test in nHCM, which, combined with a meaningful improvement in patient symptoms measured by the KCCQ heart failure questionnaire, bodes well for patients suffering from nHCM,” Imbria says.

Hypertrophic cardiomyopathy is the most common inherited cardiac disease, and it is characterized by abnormal thickening of the heart muscle, the company says. “In nHCM, a subset of patients experience a high burden of heart failure symptoms without left ventricular outflow tract obstruction,” it says, adding that this group represents a significant unmet medical need due to no proven pharmacotherapies being available.

Imbria says ninerafaxstat is designed to address cardiac diseases characterized by an imbalance of energy supply and demand in the heart. The partial fatty acid oxidation inhibitor acts to shift the heart's preference from fatty acids towards glucose, and thus improve cardiac function, both at rest and during exercise, the company says.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

Read more

Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome