SOUTH SAN FRANCISCO - Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a biotechnology firm focusing on heart disease treatments with a market capitalization of $228 million, today shared promising initial results from its MyPEAK-1 clinical trial for TN-201 gene therapy. According to InvestingPro data, the company's stock has shown strong momentum with a 38% return over the past year, despite recent volatility. This therapy is being evaluated for treating MYBPC3-associated hypertrophic cardiomyopathy (HCM), a condition characterized by insufficient levels of the protein MyBP-C that can lead to heart muscle thickening and associated health issues.
The first cohort of patients received a 3E13 vg/kg dose of TN-201 and showed general tolerability and signs of successful gene delivery to heart muscle cells. While the company is currently unprofitable, InvestingPro analysis shows it maintains a strong financial position with more cash than debt on its balance sheet and a healthy current ratio of 5.27, indicating robust short-term liquidity. Over one year, increases in RNA expression and protein levels were noted. Most clinical measures, including circulating biomarkers of cardiac muscle strain and injury, either stabilized or improved from baseline.
The trial's first three patients, all symptomatic adults with severe nonobstructive HCM, were dosed with TN-201 through a one-time intravenous infusion. The therapy was well tolerated, with no cardiac toxicities or other severe adverse effects directly attributed to TN-201. Observed liver enzyme elevations, a known side effect of AAV-based gene therapies, were managed with corticosteroids.
TN-201 demonstrated robust transduction into cardiomyocytes, with measurable transgene RNA expression and a 3% increase in MyBP-C protein levels from weeks 8 to 52 in one patient. This suggests that the therapy is being transcribed and expressed effectively post-delivery.
Clinical measures of HCM showed mostly stable or improved results. Patients 1 and 2 experienced improvements in their New York Heart Association (NYHA) classification, indicating reduced heart failure symptoms.
Tenaya plans to continue monitoring these patients and anticipates additional data from Cohort 1 and the higher dose Cohort 2 in 2025. The company believes these early findings support further investigation into TN-201's potential to address the genetic cause of MYBPC3-associated HCM. With an overall Financial Health score of 'FAIR' from InvestingPro, and two analysts recently revising earnings estimates upward, investors seeking deeper insights can access the comprehensive Pro Research Report, available exclusively to InvestingPro subscribers, covering this and 1,400+ other US stocks.
This information is based on a press release statement from Tenaya Therapeutics, Inc.
In other recent news, Tenaya Therapeutics has made significant strides in its MyPEAK-1 Phase 1b/2 clinical trial for TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy. The Data Safety Monitoring Board has approved the continuation of dosing in the second cohort of the study, following a favorable safety profile observed in Cohort 1. The firm H.C. Wainwright has maintained a Buy rating and a price target of $18.00, while Piper Sandler confirmed its Overweight rating with a steady price target of $40.00.
The company has also initiated a Phase 1b study of its gene therapy, TN-401, aimed at treating arrhythmogenic right ventricular cardiomyopathy. Tenaya anticipates sharing initial clinical data from the RIDGE-1 study in 2025. Furthermore, Tenaya has introduced a new 2024 Inducement Equity Incentive Plan, allowing for the issuance of 1,200,000 shares of common stock.
Meanwhile, Tenaya announced the upcoming departure of Leone Patterson, its Chief Financial and Business Officer, and has begun the search for a new CFO. Analyst firms such as Canaccord Genuity, Leerink Partners, and William Blair have maintained a positive outlook on Tenaya, emphasizing the company's ongoing efforts to advance its gene therapy program.
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