RARE Daily

Tenaya Raises $75 Million to Advance Gene Therapy Programs for Genetic Heart Diseases

November 17, 2022

Tenaya Therapeutics, which is developing potentially curative therapies that address the underlying causes of heart disease, raised $75 million in an underwritten public offering.

The company priced 22.6 million shares of its common stock at $2.60 per share and, to certain investors in lieu of common stock, pre-funded warrants to purchase up to an aggregate of 6.3 million shares of its common stock at a purchase price of $2.599 per each pre-funded warrant.

In addition, Tenaya granted the underwriters a 30-day option to purchase up to an additional 4.3 million shares of its common stock. Before deducting the underwriting discounts and commissions and estimated offering expenses, Tenaya expects to receive total gross proceeds of approximately $75 million, assuming no exercise of the underwriters’ option to purchase additional shares.

In its third quarter 2022 financial report, Tenaya’s management said that prioritization of resources and cash and cash equivalents will be sufficient to support the continued advancement of the company’s product candidates, TN-201, TN-301 and TN-401, two of which target rare genetic causes of heart disease.

“The initiation of our first clinical trial for TN-301 during the third quarter was a major milestone marking our transition to a clinical-stage company. Our unwavering commitment to improve the lives of those affected by heart disease also requires careful management of finite resources and prioritization of portfolio opportunities,” stated Faraz Ali, CEO of Tenaya, in its financial report. “With the extension of our cash runway to mid-2024, we believe we can reach meaningful milestones for our more advanced product candidates, TN-201, TN-301 and TN-401, and still continue to selectively invest in research efforts to advance our deep pipeline of earlier-stage programs.”

The experimental recombinant AAV gene therapy TN-201 is designed to deliver a functional MYBPC3 gene in adults and children with genetic hypertrophic cardiomyopathy (gHCM) due to MYBPC3 gene mutations, estimated to affect more than 115,000 patients in the United States. These mutations can cause the heart walls of affected individuals to become significantly thickened, leading to fibrosis, abnormal heart rhythms, cardiac dysfunction, heart failure (HF), and sudden cardiac death in some adults and children. Based on publicly available information to date, there are currently no approved treatments that address the underlying genetic cause of this disease.

Tenaya says TN-201 has demonstrated significant and durable disease reversal and survival benefit in a relevant murine model after a single dose, as well as tolerability in mice and non-human primates. The program is currently in IND-enabling studies. TN-201 has been granted Orphan Drug designation by the U.S. Food and Drug Administration and the company expects to submit an IND to the FDA by the end of the year.

Tenaya is also developing TN-401, an AAV-based gene therapy designed to deliver a functional PKP2 gene in adults with genetic arrhythmogenic right ventricular cardiomyopathy (gARVC) due to PKP2 gene mutation, which is estimated to affect more than 70,000 patients in the United States. These mutations can cause enlargement of the right ventricle in affected individuals, replacement of heart muscle with fibrotic tissue and fatty deposits, and severely abnormal heart rhythms (arrhythmia) that can make it harder for the heart to function properly and result in sudden cardiac death in some adults and children. There are currently no approved treatments that address the underlying genetic cause of this disease.

Tenaya has demonstrated that TN-401 can prevent and reverse disease progression and survival benefit in a murine model after a single dose. The company is currently initiating IND enabling studies for the program and expects to submit an IND in 2023. It also intends to initiate a global non-interventional study to collect treatment history and seroprevalence to AAV antibodies data among ARVC PKP2 gene mutation carriers.

Photo: Faraz Ali, CEO of Tenaya

Author: Rare Daily Staff

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