Tenaya Therapeutics

• Tenaya Therapeutics has successfully completed a $52.5 million units offering to support the advancement of its innovative cardiac gene therapy programs. • The financing will primarily accelerate the development of treatments targeting genetic heart diseases and heart failure, strengthening the company's position in cardiovascular therapeutics. • This strategic funding round demonstrates continued investor confidence in Tenaya's gene therapy platform and its potential to address significant unmet needs in cardiac medicine.

Tenaya Therapeutics has announced plans to offer units comprising common stock and two series of warrants to strengthen its financial position.

• Tenaya Therapeutics has received an $8 million CLIN2 grant from the California Institute for Regenerative Medicine to support their RIDGE-1 clinical trial of TN-401 gene therapy. • The Phase 1b trial targets PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), a severe genetic heart condition affecting an estimated 70,000 people in the U.S. • Initial data from the RIDGE-1 trial's low-dose cohort is expected in the second half of 2025, with the therapy designed to deliver functional PKP2 genes using AAV9 viral vectors.

Tenaya Therapeutics reported initial data from its MyPEAK-1 trial, evaluating TN-201 gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM).

• Tenaya Therapeutics has dosed the first patient in its Phase Ib RIDGE-1 trial evaluating TN-401, a gene therapy for arrhythmogenic right ventricular cardiomyopathy (ARVC). • TN-401 is an AAV9-based gene therapy designed to address ARVC caused by mutations in the _PKP2_ gene by delivering a functional copy to heart cells. • The RIDGE-1 trial is a dose-escalation study assessing the safety, tolerability, and preliminary efficacy of TN-401 in adults with _PKP2_-associated ARVC. • Initial data from the RIDGE-1 trial is expected in 2025, offering potential insights into a novel approach to treat the underlying genetic cause of ARVC.

• Tenaya Therapeutics has dosed the first patient in the RIDGE-1 Phase 1b trial evaluating TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC). • TN-401 is an AAV9-based gene therapy designed to deliver a functional copy of the PKP2 gene directly to heart cells, addressing the underlying genetic cause of ARVC. • The RIDGE-1 trial is a multicenter study enrolling approximately 15 patients with ARVC across sites in the US, UK, and Europe, with initial data expected next year. • Preclinical data demonstrated TN-401's ability to normalize heart rhythms, reverse disease progression, and extend survival in animal models, supporting its clinical development.

• Tenaya Therapeutics received DSMB clearance to escalate the TN-201 dose to 6x10^13 vg/kg in the MyPeak-1 trial for hypertrophic cardiomyopathy (HCM). • The decision follows positive early safety data from the first cohort (3x10^13 vg/kg) showing no unexpected adverse events related to the gene therapy. • The MyPeak-1 protocol was modified to include baseline biopsies, broaden eligibility, and increase the potential participant number to 24. • Initial findings from the first cohort, including safety, tolerability, biopsy analyses, and biomarker data, are expected in December 2024.

• Tenaya Therapeutics' lead candidate TN-201, an AAV9-based gene therapy targeting MYBPC3, is being developed for treating hypertrophic cardiomyopathy with projected annual revenues of $165 million by 2038. • The novel gene therapy utilizes adeno-associated virus serotype 9 vectors for intravenous delivery of the cardiac myosin-binding protein-C gene, representing a potential breakthrough in cardiac disease treatment. • Despite operating losses of $131.2 million in FY2023, Tenaya continues advancing its diverse cardiovascular pipeline, including treatments for various forms of cardiomyopathy and heart failure.