Tenaya Therapeutics Inc

Previous wording

We are advancing a deep and diverse pipeline of disease-modifying therapies that includes both gene therapies and small molecules discovered internally and developed using our extensive core capabilities to target patients with rare or highly prevalent forms of heart disease. All of our programs are currently being assessed in clinical trials or are in the preclinical stage; we do not have any products approved for sale and have not generated any revenue to date.

Current wording

We are advancing a deep and diverse pipeline of disease-modifying targeted therapies that includes both gene therapies and small molecules discovered internally and developed using our extensive core capabilities to address rare or highly prevalent forms of heart disease. All of our programs are currently being assessed in clinical trials or are in the preclinical stage; we do not have any products approved for sale and have not generated any revenue to date.

Previous wording

TN-201 is our potential first-in-class and best-in-class gene therapy for adults and children with HCM due to MYBPC3 gene mutations. These mutations result in a deficiency of myosin binding protein, which in turn can cause the heart walls of affected individuals to become significantly thickened, leading to fibrosis, abnormal heart rhythms, cardiac dysfunction and heart failure. HCM is a chronic, progressive condition and those diagnosed with the disease often experience significant impairment in overall quality of life and may be at higher risk for serious complications and co-morbidities. TN-201 utilizes a recombinant adeno-associated virus serotype 9 (AAV9) capsid to deliver a working MYBPC3 gene to specific cells of the heart in order to produce cardiac myosin binding protein and thereby slow or even reverse the course of MYBPC3-associated HCM following a single infusion. In October 2023, we began dosing patients in the MyPEAKTM-1 Phase 1b multi-center, open-label clinical trial, designed to assess the safety, tolerability and efficacy of a one-time intravenous infusion of TN-201. MyPEAK-1 is actively enrolling patients at several U.S. clinical sites and initial data from the trial is anticipated in the second half of 2024.

Current wording

TN-201 is our potential first-in-class and best-in-class gene therapy for adults and children with HCM due to MYBPC3 gene mutations. These mutations result in a deficiency of myosin binding protein, which in turn can cause the heart walls of affected individuals to become significantly thickened, leading to fibrosis, abnormal heart rhythms, cardiac dysfunction, heart failure and death. HCM is a chronic, progressive condition and those diagnosed with the disease often experience significant impairment in overall quality of life and may be at higher risk for serious complications and co-morbidities. TN-201 utilizes a recombinant adeno-associated virus serotype 9 (AAV9) capsid and is designed to deliver a working MYBPC3 gene to specific cells of the heart in order to produce cardiac myosin binding protein and thereby potentially slow or even reverse the course of MYBPC3-associated HCM following a single infusion.

Previous wording

To support our development efforts for TN-201 we are conducting two noninterventional studies: a study evaluating seroprevalence to AAV9 antibodies among adults with MYBPC3-associated HCM, and MyClimb, a prospective and retrospective global natural history study focused on pediatric patients with MYBPC3 mutation-associated cardiomyopathy. The seroprevalence study has completed enrollment and data is under analysis. The objective of the natural history study is to characterize the outcomes, burden of illness, risk factors, quality of life, and biomarkers associated with disease progression in pediatric patients with cardiomyopathy due to MYBPC3 gene mutations, as well as treatments and procedures. MyClimb complements existing disease registries focused primarily on adult patient HCM populations and may support and expedite the development of TN-201 in the pediatric patient population. In July 2024, we received rare pediatric disease designation from the FDA for TN-201 in MYBPC3-associated HCM. TN-201 has also been granted orphan drug designation from the FDA, orphan medicinal product designation from the European Commission (EC), and Fast Track Designation from the FDA.

Current wording

To support our development efforts for TN-201 we are conducting two noninterventional studies: a study evaluating seroprevalence to AAV9 antibodies among adults with MYBPC3-associated HCM, and MyClimb, a prospective and retrospective global natural history study focused on pediatric patients with MYBPC3 mutation-associated cardiomyopathy. The seroprevalence study has completed enrollment and data is under analysis. The objective of the MyClimb natural history study is to characterize the outcomes, burden of illness, risk factors, quality of life, and biomarkers associated with disease progression in pediatric patients with cardiomyopathy due to MYBPC3 gene mutations, as well as related treatments and procedures. MyClimb complements existing disease registries focused primarily on adult patient HCM populations and may support and expedite the development of TN-201 in the pediatric patient population. In July 2024, we received rare pediatric disease designation from the FDA for TN-201 in MYBPC3-associated HCM. TN-201 has also been granted orphan drug designation from the FDA, orphan medicinal product designation from the European Commission (EC), and Fast Track Designation from the FDA.

Previous wording

TN-401 is our potential first-in-class and best-in-class AAV9-based gene therapy for the treatment of ARVC due to disease-causing variants in the PKP2 gene. ARVC, also known as arrhythmogenic cardiomyopathy, or ACM, is a chronic, progressive disease characterized by potentially dangerous and frequent ventricular arrhythmias. The disease is associated with significant impairment to patients’ overall quality of life, as well as an elevated risk of sudden cardiac death. PKP2 mutations are the most common genetic cause of ARVC and result in insufficient expression of a protein needed for proper functioning of the desmosomal complex that maintains physical connections and electrical signaling between heart muscle cells.

Current wording

TN-401 is our potential first-in-class and best-in-class AAV9-based gene therapy for the treatment of ARVC due to disease-causing variants in the PKP2 gene. ARVC, also known as arrhythmogenic cardiomyopathy, or ACM, is a chronic, progressive disease characterized by frequent, severe, and potentially life-threatening ventricular arrhythmias. The disease is associated with adverse heart remodeling, fibrosis, cardiac dysfunction, significant impairment to patients’ overall quality of life, as well as an elevated risk of sudden cardiac death. PKP2 mutations are the most common genetic cause of ARVC and result in insufficient expression of a protein needed for proper functioning of the desmosomal complex that maintains physical connections and electrical signaling between heart muscle cells. TN-401 utilizes a recombinant AAV9 capsid and is designed to deliver a working PKP2 gene to specific cells of the heart in order to produce plakophilin protein and thereby potentially slow or even reverse the course of PKP2-associated ARVC following a single infusion.

Previous wording

In October 2023, the FDA provided clearance of our IND application to initiate clinical testing of TN-401. We plan to initiate patient dosing in RIDGETM-1, our Phase 1b multi-center, open-label clinical trial, designed to assess the safety, tolerability and efficacy of a one-time intravenous infusion of TN-401, in the fourth quarter of 2024 and have activated multiple clinical trial sites at leading cardiology centers with ARVC expertise. We intend to expand enrollment of RIDGE-1 outside the U.S. and received approval from the United Kingdom Medicines and Healthcare Products Regulatory Agency, Research Ethics Committee and Health Research Authority for our TN-401 Clinical Trial Authorization in June 2024.

Current wording

In October 2023, the FDA provided clearance of our IND application to initiate clinical testing of TN-401. We plan to initiate patient dosing in RIDGETM-1, our Phase 1b multi-center, open-label clinical trial, designed to assess the safety, tolerability and efficacy of a one-time intravenous infusion of TN-401, in the fourth quarter of 2024 and have activated multiple clinical trial sites at leading cardiology centers with ARVC expertise. We intend to expand enrollment of RIDGE-1 outside the U.S. and received approval from the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA), Research Ethics Committee and Health Research Authority for our TN-401 Clinical Trial Authorization in June 2024.

Previous wording

In support of our development efforts for TN-401, we have initiated a global noninterventional study to collect treatment history and seroprevalence to AAV9 antibodies data among ARVC patients who carry pathogenic or likely pathogenic PKP2 gene mutations.TN-401 has received orphan drug designation from the FDA and orphan medicinal product designation from the EC, as well as Fast Track Designation from the FDA.

Current wording

In support of our development efforts for TN-401, we have initiated a global noninterventional study (RIDGE) to collect natural history and seroprevalence to AAV9 antibodies data among ARVC patients who carry pathogenic or likely pathogenic PKP2 gene mutations. TN-401 has received orphan drug designation from the FDA and orphan medicinal product designation from the EC, as well as Fast Track Designation from the FDA.

Previous wording

In addition to our lead product candidates, we have multiple early-stage programs progressing through preclinical development using various therapeutic approaches, including gene editing, cellular regeneration and gene addition to address other forms of rare and/or prevalent heart disease.

Current wording

In addition to our lead product candidates, we have multiple early-stage programs progressing through preclinical development using various therapeutic approaches, including gene addition, gene editing, gene silencing, and cellular regeneration to address other forms of rare and/or prevalent heart disease.

Previous wording

Our distinct suite of integrated capabilities supports our efforts to discover and develop disease-modifying treatments focused on heart disease. We also continue to invest in complementary new technologies and the optimization of our existing proprietary capabilities, including the use of human-iPSC disease models, machine learning and phenotypic screening, gene editing, capsid engineering and novel promoter constructs to enable the discovery, design, delivery and development of therapeutics that are best suited to a given cardiovascular condition. We have also internalized and integrated both current Good Manufacturing Practices (cGMP) and non-GMP AAV manufacturing capabilities to support our emerging portfolio of gene therapy and cellular regeneration product candidates. Our Genetic Medicines Manufacturing Center, a cGMP facility, is strategically located near our research labs in the San Francisco Bay Area to enable smooth scale-up of production to support our clinical studies and utilizes a modular, scalable design to produce AAV-based gene therapies under current GMP standards. We seek to build on existing proprietary capabilities with the aim of increasing the safety and efficacy of genetic medicines, accelerating early-stage discovery and preclinical optimization and reducing the overall cost of goods by increasing manufacturing productivity.

Current wording

Our distinct suite of integrated capabilities supports our efforts to discover and develop disease-modifying treatments focused on heart disease. We also continue to invest in complementary new technologies and the optimization of our existing proprietary capabilities, including the use of humaniPSC-derived cardiomyocyte disease models, machine learning and phenotypic screening, gene editing, AAV capsid engineering and novel promoter constructs to enable the discovery, design, delivery and development of therapeutics that are best suited to a given cardiovascular condition. We have also internalized and integrated both current Good Manufacturing Practices (cGMP) and non-GMP AAV manufacturing capabilities to support our emerging portfolio of gene therapy and cellular regeneration product candidates. Our Genetic Medicines Manufacturing Center, a cGMP facility, is strategically located near our research labs and Manufacturing Technology Development Center, a non-GMP facility, in the San Francisco Bay Area to enable smooth scale-up of production to support our clinical studies and utilizes a modular, scalable design to produce AAV-based gene therapies under cGMP standards. We seek to build on existing proprietary capabilities with the aim of increasing the safety and efficacy of genetic medicines, accelerating early-stage discovery and preclinical optimization and reducing the overall cost of goods by increasing manufacturing productivity and scalability.

Previous wording

a decrease of $2.4 million in outside services reflecting lower costs for outside lab services as there were no IND-enabling activities in 2024, lower costs for clinical trial activities as we concluded our Phase 1 clinical trial for TN-301 at the end of 2023, and lower costs for preclinical studies as we prioritize to focus on advancing our clinical-stage gene therapy programs; partially offset by

Current wording

a decrease of $1.7 million in outside services reflecting lower costs for outside lab services as there were no IND-enabling activities in 2024.

Previous wording

Interest income primarily consists of interest earned on our cash, cash equivalents and investment balances. The year-over-year decrease of $0.4 million was primarily due to lower cash, cash equivalents and investment balances.

Current wording

Interest income primarily consists of interest earned on our cash, cash equivalents and investment balances. The interest income was $1.1 million and $1.8 million for the three months ended September 30, 2024 and 2023, respectively. The year-over-year decrease of $0.7 million was primarily due to lower cash, cash equivalents and investment balances.

Previous wording

Net loss for the three months ended June 30, 2024, was $29.4 million, compared to a net loss of $33.3 million for the three months ended June 30, 2023.

Current wording

Net loss for the three months ended September 30, 2024, was $25.6 million, compared to a net loss of $29.1 million for the three months ended September 30, 2023.

Previous wording

a decrease of $2.5 million in facility and laboratory costs, reflecting lower facility management fees and lower costs of supplies and materials as there were no IND-enabling activities in 2024; and

Current wording

a decrease of $4.2 million in facility and laboratory costs, reflecting lower costs of supplies and materials and lower facility management fees; and

Previous wording

General and administrative expenses were $8.2 million and $8.6 million for the three months ended June 30, 2024 and 2023, respectively. The year-over-year decrease of $0.5 million, or 5%, was primarily due to a decrease in professional fees.

Current wording

General and administrative expenses were $23.2 million and $24.6 million for the nine months ended September 30, 2024 and 2023, respectively. The year-over-year decrease of $1.3 million, or 5%, was primarily due to decreases in employee-related costs driven by the Workforce Reduction and lower professional fees.

Previous wording

We expect our general and administrative expenses to generally remain flat for the remainder of 2024, as we continue to support our research and development activities and business development opportunities, and incur professional service fees. In addition, we will continue to incur legal, accounting, insurance and other expenses in operating our business as a public company, including costs associated with regulatory and compliance activities.

Current wording

We continue to support our research and development activities and business development opportunities, and incur professional service fees. In addition, we will continue to incur legal, accounting, insurance and other expenses in operating our business as a public company, including costs associated with regulatory and compliance activities. However, we expect our general and administrative expenses to generally remain flat for the remainder of 2024.

Previous wording

Interest income primarily consists of interest earned on our cash, cash equivalents and investment balances. The year-over-year decrease of $1.0 million was primarily due to lower cash, cash equivalents and investment balances.

Current wording

Interest income primarily consists of interest earned on our cash, cash equivalents and investment balances. The interest income was $3.9 million and $5.6 million for the nine months ended September 30, 2024 and 2023, respectively. The year-over-year decrease of $1.7 million was primarily due to lower cash, cash equivalents and investment balances.

Previous wording

Net loss for the six months ended June 30, 2024, was $61.7 million, compared to a net loss of $65.0 million for the six months ended June 30, 2023.

Current wording

Net loss for the nine months ended September 30, 2024, was $87.3 million, compared to a net loss of $94.1 million for the nine months ended September 30, 2023.

Previous wording

Since our inception, we have not generated any revenue and we have incurred significant net losses and negative cash flows from operations. From our inception through June 30, 2024, we have funded our operations primarily from the sale and issuance of our equity securities. As of June 30, 2024, we had cash, cash equivalents and investments in marketable securities of $99.3 million and an accumulated deficit of $464.9 million.

Current wording

Since our inception, we have not generated any revenue and we have incurred significant net losses and negative cash flows from operations. From our inception through September 30, 2024, we have funded our operations primarily from the sale and issuance of our equity securities. As of September 30, 2024, we had cash, cash equivalents and investments in marketable securities of $79.5 million and an accumulated deficit of $490.6 million.

Previous wording

On August 6, 2024, we entered into a Loan Agreement with Silicon Valley Bank (SVB), a division of First-Citizens Bank & Trust Company (the Loan Agreement). Under the Loan Agreement, we have the right to draw down $15.0 million at our discretion, up to an additional $10.0 million upon the satisfaction of certain milestones and up to an additional $20.0 million that may be available at SVB’s discretion, subject to specified conditions.

Current wording

On August 6, 2024, we entered into a Loan Agreement with Silicon Valley Bank (SVB), a division of First-Citizens Bank & Trust Company (the Loan Agreement). As of the filing date of this periodic report, under the Loan Agreement, we have the right to draw down $20.0 million at our discretion, up to an additional $5.0 million upon the satisfaction of certain milestones and up to an additional $20.0 million that may be available at SVB’s discretion, subject to specified conditions.

Previous wording

On February 12, 2024, we completed an underwritten offering of 8,888,890 shares of its common stock at a price of $4.50 per share and, to an investor in lieu of common stock, pre-funded warrants to purchase 2,222,271 shares of its common stock at a price of $4.499 per pre-funded warrant under our registration statement on Form S-3 (File No. 333-266741). The pre-funded warrants can be exercised at any time after issuance for an exercise price of $0.001 per share, subject to certain ownership limitations. We received net proceeds of approximately $46.8 million, after deducting underwriting discounts and commissions of approximately $3.0 million and other offering expenses of approximately $0.2 million.

Current wording

On February 12, 2024, we completed an underwritten offering of 8,888,890 shares of our common stock at a price of $4.50 per share and, to an investor in lieu of common stock, pre-funded warrants to purchase 2,222,271 shares of our common stock at a price of $4.499 per pre-funded warrant under our registration statement on Form S-3 (File No. 333-266741). The pre-funded warrants can be exercised at any time after issuance for an exercise price of $0.001 per share, subject to certain ownership limitations. We received net proceeds of approximately $46.8 million, after deducting underwriting discounts and commissions of approximately $3.0 million and other offering expenses of approximately $0.2 million.

Previous wording

Based on our current operating plan, we believe that our existing cash, cash equivalents and investments in marketable securities will be sufficient to meet our working capital and capital expenditure needs through at least the next twelve months following the date of this Quarterly Report on Form 10-Q.

Current wording

Based on our current operating plan, we believe that our existing cash, cash equivalents and investments in marketable securities, along with the $20.0 million of funds available under its Loan Agreement with SVB, will be sufficient to meet our working capital and capital expenditure needs through at least the next twelve months following the date of this Quarterly Report on Form 10-Q.

Previous wording

In order to complete the development of our product candidates and commercialize our product candidates, if approved, we will require substantial additional funding. Until such time as we can generate significant revenue from sales of our product candidates, if ever, we expect to finance our operations through public or private equity offerings or debt financings or other capital sources, which may include strategic collaborations or other arrangements with third parties, or other sources of financing. We may not be able to raise additional capital on terms acceptable to us or at all. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our stockholders will be or could be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our common stockholders. Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, including restricting our operations and limiting our ability to incur liens, issue additional debt, pay dividends, repurchase our common stock, make certain investments or engage in merger, consolidation, licensing or asset sale transactions. If we raise funds through strategic collaborations, partnerships and other similar arrangements with third parties, we may be required to grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves. If we are unable to raise additional capital on acceptable terms when needed, our business, results of operations, and financial condition would be adversely affected.

Current wording

In order to complete the development of our product candidates and commercialize our product candidates, if approved, we will require substantial additional funding. Until such time as we can generate significant revenue from sales of our product candidates, if ever, we expect to finance our operations through public or private equity offerings, debt financings or other capital sources, which may include strategic collaborations or other arrangements with third parties, or other sources of financing. We may not be able to raise additional capital on terms acceptable to us or at all. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our stockholders will be or could be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our common stockholders. Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, including restricting our operations and limiting our ability to incur liens, issue additional debt, pay dividends, repurchase our common stock, make certain investments or engage in merger, consolidation, licensing or asset sale transactions. If we raise funds through strategic collaborations, partnerships and other similar arrangements with third parties, we may be required to grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves. If we are unable to raise additional capital on acceptable terms when needed, our business, results of operations, and financial condition would be adversely affected.

Previous wording

Net cash used in operating activities for the six months ended June 30, 2024 was $52.3 million, which consisted primarily of a net loss of $61.7 million and a net change in operating assets and liabilities of $5.6 million, partially offset by $14.2 million in non-cash charges. The change in net operating assets and liabilities was primarily due to a decrease in accounts payable and accrued expenses and other current liabilities of $4.9 million and a decrease in operating lease liabilities of $2.2 million, partially offset by a decrease of prepaid expenses and other current assets of $1.1 million. Cash flows from operations are generally impacted by the timing of payments to vendors and vendor payment terms. The non-cash charges primarily consisted of stock-based compensation of $8.8 million and depreciation and amortization of $4.3 million.

Current wording

Net cash used in operating activities for the nine months ended September 30, 2024 was $72.1 million, which consisted primarily of a net loss of $87.3 million and a net change in operating assets and liabilities of $6.9 million, partially offset by $21.0 million in non-cash charges. The change in net operating assets and liabilities was primarily due to a decrease in accounts payable and accrued expenses and other current liabilities of $5.3 million and a decrease in operating lease liabilities of $3.2 million, partially offset by a decrease of prepaid expenses and other current assets of $1.1 million. Cash flows from operations are generally impacted by the timing of payments to vendors and vendor payment terms. The non-cash charges primarily consisted of stock-based compensation of $12.7 million and depreciation and amortization of $6.4 million.

Previous wording

Net cash used in operating activities for the six months ended June 30, 2023 was $56.5 million, which consisted primarily of a net loss of $65.0 million and a net change in operating assets and liabilities of $3.8 million, partially offset by $12.2 million in non-cash charges. The change in net operating assets and liabilities was primarily due to a decrease in operating lease liabilities of $2.0 million and a decrease in accounts payable and accrued expenses and other current liabilities of $1.8 million. Cash flows from operations are generally impacted by the timing of payments to vendors and vendor payment terms. The non-cash charges primarily consisted of stock-based compensation of $7.3 million and depreciation and amortization of $4.3 million.

Current wording

Net cash used in operating activities for the nine months ended September 30, 2023 was $80.1 million, which consisted primarily of a net loss of $94.1 million and a net change in operating assets and liabilities of $5.0 million, partially offset by $19.0 million in non-cash charges. The change in net operating assets and liabilities was primarily due to a decrease in accounts payable and accrued expenses and other current liabilities of $3.3 million and a decrease in operating lease liabilities of $3.1 million, partially offset by a decrease of prepaid expenses and other current assets of $2.0 million. Cash flows from operations are generally impacted by the timing of payments to vendors and vendor payment terms. The non-cash charges primarily consisted of stock-based compensation of $11.4 million and depreciation and amortization of $6.5 million.

Previous wording

Net cash provided by investing activities for the six months ended June 30, 2024 was $17.1 million, which consisted primarily of proceeds from maturities of marketable securities of $52.2 million, partially offset by purchases of marketable securities of $34.5 million.

Current wording

Net cash used in investing activities for the nine months ended September 30, 2024 was $13.0 million, which consisted primarily of purchases of marketable securities of $85.1 million, partially offset by proceeds from maturities of marketable securities of $72.9 million.

Previous wording

Net cash provided by investing activities for the six months ended June 30, 2023 was $33.1 million, which consisted primarily of proceeds from maturities of marketable securities of $75.0 million, partially offset by purchases of marketable securities of $41.0 million.

Current wording

Net cash provided by investing activities for the nine months ended September 30, 2023 was $63.6 million, which consisted primarily of proceeds from maturities of marketable securities of $105.5 million, partially offset by purchases of marketable securities of $41.0 million.

Previous wording

Net cash provided by financing activities for the six months ended June 30, 2024 was $47.3 million, which primarily consisted of net proceeds from follow-on offering of $46.8 million.

Current wording

Net cash provided by financing activities for the nine months ended September 30, 2024 was $47.4 million, which primarily consisted of net proceeds from our February 2024 follow-on offering of $46.8 million.

Previous wording

Net cash provided by financing activities for the six months ended June 30, 2023 was $3.7 million, which primarily consisted of proceeds from at-the-market sales of $3.9 million, partially offset by payments of accrued offering costs.

Current wording

Net cash provided by financing activities for the nine months ended September 30, 2023 was $3.7 million, which primarily consisted of proceeds from at-the-market sales of $3.9 million, partially offset by payments of accrued offering costs.

Previous wording

As of June 30, 2024, there have been no material changes from the contractual obligations and commitments previously disclosed in our Annual Report on Form 10-K for the year ended December 31, 2023.

Current wording

Except as otherwise described in Note 4 to our unaudited condensed financial statements included in this periodic report, there have been no material changes from the contractual obligations and commitments previously disclosed in our Annual Report on Form 10-K for the year ended December 31, 2023.

Previous wording

A summary of our critical accounting policies and estimates is presented in Part II, Item 7 of our Annual Report on Form 10-K for the year ended December 31, 2023. There were no material changes to our critical accounting policies and estimates during the three and six months ended June 30, 2024.

Current wording

A summary of our critical accounting policies and estimates is presented in Part II, Item 7 of our Annual Report on Form 10-K for the year ended December 31, 2023. There were no material changes to our critical accounting policies and estimates during the three and nine months ended September 30, 2024.