NEW YORK – Tenaya Therapeutics on Thursday said it will broaden eligibility criteria for the Phase Ib/II MyPEAK-1 trial, in which the company is evaluating its investigational gene therapy candidate TN-201 in patients with MYBPC3-associated hypertrophic cardiomyopathy.
An independent data safety and monitoring board endorsed South San Francisco, California-based Tenaya's decision to adjust eligibility criteria in the clinical trial to include patients who do not have an implantable cardioverter defibrillator device and to permit patients with obstructive or nonobstructive forms of hypertrophic cardiomyopathy to enroll.
The board also endorsed the decision to begin treating a cohort of patients with a higher dose of TN-201.
Tenaya has completed dosing the first three patients in the first, low-dose cohort of the open-label, dose-escalating MyPEAK-1 trial, in which patients receive a single intravenous infusion of TN-201, a gene replacement therapy that's designed to deliver a functional copy of the MYBPC3 gene to heart muscle cells and slow or potentially reverse the course of disease. Mutations in MYBPC3 are the most common genetic cause of hypertrophic cardiomyopathy, accounting for up to 30 percent of cases.
TN-201 has a safety and tolerability profile consistent with other adeno-associated virus vector-based gene therapies at this dose, the company said. Tenaya said it will report initial data from that first cohort of patients in December, including safety and tolerability data, analyses of cardiac biopsies, and changes in cardiac biomarkers compared to the baseline.
"The MyPEAK-1 study of TN-201 is primarily intended to establish the safety profile of TN-201 gene therapy, and we are pleased to report that TN-201 has an appropriate tolerability profile at the 3E13 vg/kg dose without unexpected adverse reactions," Tenaya Chief Medical Officer Whit Tingley said in a statement.
Tenaya has implemented several other changes to the clinical trial protocol, including adding a baseline cardiac biopsy and permitting more flexibility in the timing of posttreatment biopsies to characterize expression of the gene therapy over time, as well as increasing the potential number of patients enrolled in the dose-expansion portion of the trial from nine to 24.