Imbria Pharmaceuticals, Inc.

Imbria Pharmaceuticals plans to present full data from the phase 2 clinical trial at an upcoming event. Midphase data are arriving thick and fast at Imbria Pharmaceuticals. Days after sharing positive top-line data in one indication, the cardiometabolic biotech has reported a phase 2 trial of the same candidate in a different setting has met its primary endpoint.  Both studies looked at ninerafaxstat, a partial fatty acid oxidation inhibitor that Imbria has identified as a way to enhance cellular energy metabolism and thereby improve outcomes in multiple cardiometabolic diseases. Last week, the biotech chalked up a win in nonobstructive hypertrophic cardiomyopathy, teeing it up to advance into phase 3 in the rare disease indication.  Sunday, Imbria reported its second phase 2 win of the week. The latest top-line success came in a clinical trial that compared ninerafaxstat to placebo in 58 people with chronic stable angina due to obstructive coronary artery disease.   The primary endpoint looked at the incidence and severity of treatment-emergent adverse events from randomization through 10 weeks of follow-up. Imbria cleared the bar for success on the primary goal and shared early evidence of efficacy, linking ninerafaxstat to “clinically and statistically significant improvements in dobutamine stress-induced ischemic left ventricular wall motion abnormalities.”  Imbria added that “this robust anti-ischemic effect was not dependent on changes in myocardial blood flow, consistent with a direct, metabolic mechanism of action of ninerafaxstat targeting the cardiomyocyte.” The biotech also reported a significant increase in myocardial glucose use compared to placebo, a finding that supports the ability of the molecule to induce a switch in substrate use. In a statement, Chief Medical Officer Jai Patel said the results “confirm the anti-ischemic effect of ninerafaxstat and support further development in cardiac ischemic disorders.” “This novel approach may have the potential to achieve optimal relief of angina and improve quality of life of patients when used alone or in combination with existing therapies,” Patel added. Imbria is yet to share numbers to support Patel’s confidence in the program. The biotech plans to present full data from the phase 2 clinical trial at an upcoming event.

Imbria Pharmaceuticals is now eyeing a phase 3 study to further test the efficacy of ninerafaxstat and plans to meet with regulatory authorities soon to discuss the trial design. Imbria Pharmaceuticals’ heart failure medicine met the safety and tolerability goals in a phase 2 trial while also improving results on a cardiopulmonary exercise test, with patients reporting other improvements in their conditions as well. The Boston-based biotech said Tuesday that ninerafaxstat is the first drug to show an improvement in a functional measurement of symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM), an inherited heart disease. In the phase 2 IMPROVE-HCM trial, Imbira was testing the safety and tolerability of the partial fatty acid oxidation inhibitor, which aims to shift the heart’s preference from fatty acid toward glucose. The hope is that this metabolism shift will lead to more efficient energy generation and improve cardiac function. NHCM causes abnormal thickening of the heart muscle leading to complications include a deficiency in cardiac energy and inefficient energy utilization. After 12 weeks of treatment, patients receiving ninerafaxstat had a statistically significant improvement in a functional cardiopulmonary exercise test, which was associated with clinically relevant improvement in patient-reported outcomes, Imbria said. Ninerafaxstat was well tolerated, and there were no safety signals observed. More data from the IMPROVE-HCM trial will be revealed at a future medical meeting. Imbria is now eyeing a phase 3 study to further test the efficacy of ninerafaxstat. The company will meet with regulatory authorities soon to discuss the trial design. With the results suggesting the med can have an impact on cardiac energy deficiency, Imbria thinks ninerafaxstat could have a wider impact in other cardiac diseases including heart failure with preserved ejection fraction (HFpEF), said CEO Anne Prener, M.D., Ph.D. Ninerafaxstat is Imbria’s only pipeline med. The biotech has a three-pronged clinical approach testing the med in nHCM, HFpEF and obstructive coronary arterial disease.

Imbria Pharmaceuticals’ partial fatty acid oxidation inhibitor improved outcomes in Phase II non-obstructive hypertrophic cardiomyopathy patients, with the company noting that the asset’s efficacy could bode well for upcoming results in a separate heart failure trial. Non-obstructive hypertrophic cardiomyopathy (nHCM) affects around one in 500 people, according to the American Heart Association, with a third of cases being non-obstructive. But heart failure with preserved ejection fraction (HFpEF) affects more people, with an estimated 2% of the population . Imbria’s ninerafaxstat is in Phase II development for both conditions. You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.