Tag: non-obstructive

Cytokinetics Teases Upcoming Phase 3 Aficamten Trial in nHCM

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A Phase 3 clinical trial aimed at the non-obstructed HCM population (nHCM) appears to be planned for Cytokinetics’ next-generation myosin inhibitor, aficamten.

Based on positive data from Cohort 4 of the Phase 2 REDWOOD-HCM trial, Cytokinetics is planning to soon launch a Phase 3 clinical trial for non-obstructed hypertrophic cardiomyopathy.  Non-obstructed HCM patients are a group whose only current treatment options are diuretics, beta-blockers, and other drugs used in the traditional heart failure setting.  In extreme cases, heart transplantation may be the only path available to these patients.

According to data presented on Sunday at the American College of Cardiology 72nd Annual Scientific Sessions (held in my beloved hometown of New Orleans, the city with the best and least heart-healthy food in the world), 41 patients with nHCM in the cohort showed improvement to both their NT-proBNP and troponin levels (both are biomarkers of heart wall stress seen in blood tests). And, the drug was generally well tolerated.

Separately, data was presented showing that obstructed HCM (HOCM) patients who took aficamten for a 48 week period showed significant improvement to their left ventricular outflow tract gradients (LVOT) while 88% of patients experienced improvement of at least one NYHA Functional Class.

All 19 HOCM patients enrolled in the trial who fit the eligibility criteria for septal reduction therapy (septal myectomy or septal alcohol ablation) at the start of the trial improved so much that they were no longer eligible after 48 weeks on aficamten.

It would be wonderful to have better options for the treatment HCM, so here’s hoping that aficamten continues down the road to FDA approval.

For more about aficamten, read these prior posts from HCMBeat:

2 Companies Testing Drugs for HCM

HCM Clinical Trials – the Latest News

The Future of HCM Care

Positive Signs from REDWOOD-HCM

Promising Data about Aficamten Presented at Meetings

More on Aficamten & Mavacamten from ACC 2022

Aficamten Updates from Cytokinetics

Aficamten Gets “Breakthrough Drug” Status from FDA

Cytokinetic’s Drug Aficamten & Upcoming HCM Summit – Interview with Dr. Martin Maron

Results Published from REDWOOD-HCM Phase 2 Trial

The Future of HCM Treatment

Tenaya Therapeutics Gets Go-Ahead for HCM Gene Therapy Trial

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Tenaya Therapeutics announced on Monday that they have received FDA clearance to begin a Phase 1 clinical trial of targeted gene therapy for HCM.

Tenaya is developing TN-201, a first in class adeno-associated virus based therapy being developed to treat HCM caused by mutation(s) in the MYBPC3 gene. They anticipate that the trial will begin in the third quarter of 2023. The therapy delivers one fully functional MYBPC3 gene to the patient via injection with a deactivated virus. Tenaya hopes that this therapy will restore normal levels of the MYBPC3 protein, thereby halting disease progression, and even potentially reversing the course of the disease, after just a single treatment.

The TN-201 Phase 1b clinical trial will be a multi-center, open-label study designed to assess the safety of an intravenous infusion of TN-201. They hope to enroll at least 6 symptomatic, non-obstructive HCM patients who carry the MYBPC3 gene and who already have received an automatic implantable cardioverter defibrillator (ICD) as part of their treatment plan to date.

You can read the full  press release here.

Stay tuned to HCMBeat for updates!

Drug Trial for Non-Obstructive HCM

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Yet another company is developing a new drug for hypertrophic cardiomyopathy, and this time, the drug is intended for non-obstructive HCM patients.  The company is Imbria Pharmaceuticals, and this week they announced the randomizing of the first patient in their Phase 2 study of the drug IMB-101 in patients with non-obstructive HCM.  The study, called IMPROVE-HCM, is a Phase 2 study that will look at the safety and tolerability of this drug in non-obstructed HCM patients. IMB-101 is designed to increase the efficiency of the heart’s use of energy which will be measured through cardiopulmonary exercise testing over a 12 week period.

You can read this press release and you can read more about the trial on ClinicalTrials.gov here.

Biventricular Pacemaker For Non-Obstructed HCM Patients?

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A small study of  29 patients conducted recently in the U.K. found that the use of a biventricular pacing  in patients with non-obstructive hypertrophic cardiomyopathy improved symptoms of breathlessness and improved exercise capacity as demonstrated during oxygen consumption testing.

Medications are the only treatments currently available to non-obstructed patients. The authors of this study hypothesized that biventricular pacing could be a viable way to address exercise limitations in non-obstructed patients if medications have been ineffective.

Larger trials may establish biventricular pacing as a viable treatment for non-obstructed patients in the future.

The High Hanging Fruit: Treatment for Non-Obstructive HCM – Commentary by Dr. Sharlene Day

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A recent study conducted in the U.K. evaluated whether the anti-anginal drug trimetazidine would improve symptoms and exercise capacity for those patients with non-obstructive hypertrophic cardiomyopathy. 

Unfortunately, this study which was conducted by Dr. Perry Elliott and his colleagues at University College London, found that trimetatazidine did not improve exercise capacity in these patients. Following the results of this study, trimetazidine will now join ranolazine and spironolactone in the compost heap of drugs which tried and failed to improve HCM symptoms.  While a third drug, perhexiline, was found to improve symptoms for non-obstructive HCM, its limitations, including potentially serious side effects, stand in the way of its common usage.

In a companion editorial to this study entitled “Non-Obstructive Hypertrophic Cardiomyopathy-the High Hanging Fruit,” Dr. Sharlene Day of the University of Michigan’s HCM Center discusses the difficulties seen in drug trials related to non-obstructive HCM.

Continue reading “The High Hanging Fruit: Treatment for Non-Obstructive HCM – Commentary by Dr. Sharlene Day” →