Tenaya Therapeutics, Inc.
For the period ended Mar 31, 2025. Filed May 7, 2025.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Mar 31, 2025 · Filed May 7, 2025
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2024-09-30; filed 2024-11-06.
0000950170-24-122359
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-03-31; filed 2025-05-07.
0000950170-25-065741
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 97 previous paragraphs and 94 current paragraphs.
48 unchanged; 31 reworded; 15 added; 15 removed; 3 previous and 0 current paragraphs rearranged.
Tables are not compared: 5 previous and 3 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (15)
Current report
In March 2025, we announced cost containment measures, including a committed plan to reduce our workforce (the 2025 Workforce Reduction), in alignment with our focus on generating data from the ongoing MyPEAKTM-1 and RIDGETM-1 clinical trials of TN-201 and TN-401, respectively. Savings from these measures, combined with proceeds from our follow-on offering completed in March 2025 and cash, cash equivalents and short-term investments, are expected to extend our cash runway past key data junctures for each program and into the second half of 2026.
Current report
In December 2024, we announced early but encouraging initial interim safety and biopsy data from Cohort 1 of MyPEAK-1, and in March 2025, we presented data from Cohort 1 in a Late-Breaker presentation at the 2025 American College of Cardiology Scientific (ACC) Sessions. Interim data presented at ACC included results from serial biopsies taken at two time points for all three patients and assessments of the first two patients at Week 52 and Patient 3 at Week 26. TN-201 has been generally well tolerated at the 3E13 vg/kg dose, and treatment-emergent adverse events (AEs) were primarily mild, manageable and/or reversible. Biopsy data indicated robust transduction as evidenced by the sustained presence of TN-201 DNA in the heart (0.8 to 1.4 vg/dg) at post-dose and week 52 for Patients 1 and 2 and baseline to Week 26 for Patient 3. TN-201 RNA expression was present at post-dose biopsies conducted in the first two patients at Week 8 and increased as much as 13-fold from Week 8 to Week 52 post-dose. MyBP-C protein levels increased from 56 to 59% and from 62 to 64% of normal between Week 8 and Week 52 for Patients 1 and 2, respectively. This increase, combined with the increase observed in TN-201 mRNA expression, suggests that TN-201 gene therapy is successfully being transcribed and expressed after reaching target cells.
Current report
Promising early signals of TN-201’s clinical impact suggesting clinical improvement or stability were observed across multiple parameters measured. Cardiac troponin, a biomarker of myocardial injury, was elevated in Cohort 1 patients at baseline and decreased by more than 60% in two patients, whose levels are now normal or near normal. NT-proBNP, a biomarker of cardiac strain, increased and remained elevated while patients were on immunosuppression, but returned to baseline as immunosuppressive drugs were discontinued. Two of three patients experienced improvements in one or more measures of hypertrophy, or enlargement of the heart, while other assessments remained stable. All Cohort 1 patients with objectively severe disease at baseline and mild-to moderate heart failure symptoms that interfered in activities of daily living experienced improvements in New York Heart Association classification and achieved Class 1, indicating no limitations to physical activities due to symptoms of heart failure.
Current report
The U.S. Food and Drug Administration (FDA) has granted TN-201 Fast Track, Orphan Drug and Rare Pediatric Drug Designations. TN-201 has also received orphan medicinal product designation from the European Commission.
Current report
We dosed the initial patient in RIDGE-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 November 2024. We have activated multiple clinical trial sites at leading cardiology centers in the U.S. with ARVC expertise and intend to expand enrollment of RIDGE-1 to the United Kingdom. We expect to complete enrollment of the first cohort of patients at the 3E13 vg/kg dose in the first half of 2025. An independent safety review will then inform plans for dose escalation to 6E13 vg/kg and/or enrollment of additional patients in the first cohort. Initial clinical data from the first cohort of patients is expected in the second half of 2025. In February 2025, we were awarded a Clinical Grant (Clin2) of $8.0 million from the California Institute for Regenerative Medicine (CIRM), a state of California
Current report
Agency that funds regenerative medicine, stem cell, and gene therapy research. Proceeds from the grant will help fund clinical trial costs for our ongoing Phase 1b RIDGE-1 clinical trial of TN-401 gene therapy.
Current report
In support of our development efforts for TN-401, we initiated RIDGE, a global noninterventional study to collect natural history and seroprevalence to AAV9 antibodies data among ARVC patients who carry pathogenic or likely pathogenic PKP2 gene mutations. Interim data from RIDGE, believed to be the largest natural history study of adults with PKP2-associated ARVC was presented at Heart Rhythm Society’s annual Heart Rhythm meeting in April 2025. Adults with PKP2-associated ARVC experience a high burden of arrhythmias despite treatments with anti-arrhythmic medications, beta blockers and the anti-arrhythmic flecainide, as well as surgical interventions such as ablation and ICD placement. Further, current treatments appeared to do little to halt or prevent progressive structural changes to the heart that occur as a result of PKP2 mutations. A large majority of adults with PKP2-associated ARVC would be eligible to participate in our ongoing Phase 1b RIDGE-1 clinical trial of TN-401 gene therapy based on low levels of antibodies to AAV9.
Current report
Early on in our company history, we invested in differentiated capabilities to enable modality agnostic target identification and validation, anchored in human genetics and the use of human disease models. That proprietary expertise has directly informed the design, optimization and production of our pipeline. To support our initial focus on gene therapy candidates, we internalized expertise in capsid engineering, novel promoter constructs and manufacturing anchored on the use of AAVs as the method of delivery to the heart with the aim of increasing the safety and efficacy of our product candidates and accelerating early-stage discovery and preclinical optimization across modalities.
Current report
For the production of our lead gene therapy candidates, we maintained complete ownership of process development, analytical development, and quality control and have already produced all necessary clinical trial material needed for our ongoing clinical trials at our Good Manufacturing Practice (cGMP)-certified Genetic Medicines Manufacturing Center in Union City, California.
Current report
Research and development expenses were $21.1 million and $25.1 million for the three months ended March 31, 2025 and 2024, respectively. The year-over-year decrease of $4.0 million, or 16%, was primarily due to:
Current report
a decrease of $1.9 million in clinical trial related costs primarily driven by a decrease in clinical support costs, including clinical supply chain services and regulatory consulting fees.
Current report
a decrease of $0.7 million in other research and development costs reflecting lower employee-related costs driven by the 2024 Workforce Reduction.
Current report
CIRM Grant
Current report
In February 2025, we announced we were awarded an $8 million grant from the CIRM to support RIDGE-1. The award is payable to us upon achievement of certain clinical milestones. Additionally, if CIRM determines, in its sole discretion, that we have not complied with the terms and conditions of the grant, CIRM may suspend or permanently cease disbursements. Funds received under this grant may only be used for allowable project costs specifically identified with the CIRM-funded project. Such costs can include, but are not limited to, salary for personnel, itemized supplies, consultants, and itemized clinical study costs. Under the terms of the grant, we will co-fund the research project with CIRM and the amount of our co-funding requirement is predetermined as a part of the award. In April 2025, the Company received $0.6 million from CIRM in connection with the grant.
Current report
On March 5, 2025, we completed an underwritten offering of 75,000,000 units, priced at a public offering price of $0.70 per unit, with each unit consisting of one share of our common stock, a warrant to purchase one share of our common stock at an exercise price of $0.80 per share, which will be immediately exercisable and will expire five years from the date of issuance (a Series A Warrant) and a warrant to purchase one-half of a share of our common stock at an exercise price of $0.70 per share, which will be immediately exercisable and expire on June 30, 2026 (a Series B Warrant), under our registration statement on Form S-3 (File No. 333-266741). We received net proceeds of approximately $48.8 million, after deducting underwriting discounts and commissions of approximately $3.2 million and other offering expenses of approximately $0.5 million.
Removed paragraphs (15)
Previous report
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 report
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 report
Research and development expenses were $20.4 million and $23.1 million for the three months ended September 30, 2024 and 2023, respectively. The year-over-year decrease of $2.7 million, or 12%, was primarily due to:
Previous report
a decrease of $1.8 million in employee-related costs primarily driven by a workforce reduction plan initiated in May 2024 (the Workforce Reduction).
Previous report
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 report
Comparison of the Nine Months Ended September 30, 2024 and 2023:
Previous report
Research and Development Expenses
Previous report
Research and development expenses were $68.1 million and $75.2 million for the nine months ended September 30, 2024 and 2023, respectively. The year-over-year decrease of $7.1 million, or 9%, was primarily due to:
Previous report
a decrease of $3.8 million in outside services reflecting lower outside lab service fees as there were no IND-enabling activities in 2024, partially offset by an increase in clinical trial related costs for TN-201 and TN-401.
Previous report
General and Administrative
Previous report
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 report
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 report
Interest Income
Previous report
Net Loss
Previous report
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.
Reworded paragraphs (31)
Previous wording
Our lead investigational product candidates are TN-201, a gene therapy for myosin binding protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM), TN-401, a gene therapy for plakophilin 2 (PKP2)-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), and TN-301, a small molecule for heart failure with preserved ejection fraction (HFpEF).
Current wording
Our lead investigational product candidates are TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), and TN-301, a small molecule for heart failure with preserved ejection fraction (HFpEF).
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, 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.
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 (MyBP-C), 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 MyBP-C and thereby potentially slow or even reverse the course of MYBPC3-associated HCM following a single infusion.
Previous wording
In October 2023, we began dosing patients in the MyPEAKTM-1 Phase 1b/2 multi-center, open-label clinical trial, designed to assess the safety, tolerability and efficacy of a one-time intravenous infusion of TN-201. We have completed Cohort 1 dosing at the 3E13 vg/kg dose in the MyPEAK-1 trial with no unexpected events or toxicities associated with TN-201 observed. Following review by an independent Data and Safety Monitoring Board (DSMB) of safety data from the first three MyPEAK-1 patients, the DSMB recommended that we proceed with the planned escalation to the 6E13 vg/kg dose, per protocol. MyPEAK-1 is now actively enrolling patients for Cohort 2 at several U.S. clinical sites and initial data from the trial is anticipated in December 2024.
Current wording
MyPEAK-1 is our Phase 1b/2 multi-center, open-label clinical trial, designed to assess the safety, tolerability and efficacy of a one-time intravenous infusion of TN-201. We have completed Cohort 1 dosing at the 3E13 vg/kg dose with no unexpected events or toxicities associated with TN-201 observed. Following review of safety data from the first three MyPEAK-1 patients by an independent data safety monitoring board (DSMB), the DSMB endorsed our plan to escalate to the 6E13 vg/kg dose, per protocol and broaden eligibility criteria. In December, we dosed the first patient in the sentinel phase of Cohort 2 at the 6E13 vg/kg dose and we remain on track to complete Cohort 2 enrollment in the first half of 2025. We plan to present initial Cohort 2 data, along with an update on Cohort 1 patients, in the second half of 2025.
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 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.
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. Initial data indicated that antibodies to AAV9 in the majority of participants were below the eligibility threshold that would allow for participation in MyPEAK-1. The MyClimb natural history study is following patient medical history to characterize the outcomes, burden of illness, risk factors, quality of life, and biomarkers associated with disease progression in pediatric patients. 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. We plan to present data from MyClimb in the second half of 2025.
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 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.
Current wording
TN-401 is our 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 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.
Current wording
TN-401 has received Orphan Drug and Fast Track designation from the FDA and orphan medicinal product designation from the EC
Previous wording
TN-301 is our small molecule inhibitor of histone deacetylase-6 (HDAC6), initially being developed for the potential treatment of HFpEF. HFpEF is characterized by a stiffening of the heart muscle resulting in an inability for the left ventricle to relax and fill with oxygenated blood sufficient to meet the body’s needs. There are several cellular processes thought to underlie the pathophysiology of HFpEF, including increases in fibrosis and inflammation and defects in metabolism. Although HFpEF accounts for approximately half of all heart failures, there are few proven treatment options.
Current wording
TN-301 is our small molecule inhibitor of histone deacetylase-6 (HDAC6), initially being developed for the potential treatment of HFpEF. HDAC6 is a cytoplasmic enzyme known to regulate diverse cellular processes. TN-301’s multi-modal mechanism addresses diverse pathological processes and has shown direct and systemic benefits inin vitro andin vivo studies of HFpEF. HFpEF is characterized by a stiffening of the heart muscle resulting in an inability for the left ventricle to relax and fill with oxygenated blood sufficient to meet the body’s needs. The pathophysiology of HFpEF is complex and is driven by metabolic dysfunction or oxidative stress and inflammation, with diverse conditions serving as risk factors for disease, including obesity, diabetes, aging, kidney disease and hypertension. Although HFpEF accounts for approximately half of all heart failures, there are few proven treatment options.
Previous wording
In October 2023, we shared positive data from our Phase 1 clinical trial of TN-301 in healthy participants at the 2023 Heart Failure Society of America Annual Scientific Meeting. TN-301 was generally well tolerated across the broad range of doses studied. Pharmacokinetic results showed overall dose proportionality with a half-life supportive of once-daily dosing. Increasing doses and exposures with TN-301 correlated with increased pharmacodynamic effects. There were no changes in histone acetylation with TN-301 underscoring the selectivity of TN-301 for HDAC6 and potentially reducing the risk of off target effects. In preclinical studies, selective HDAC6 inhibition has been shown to have comparable efficacy to empagliflozin, a sodium-glucose cotransporter-2 (SGLT2) inhibitor which is approved for the treatment of HFpEF and co-administration of our HDAC6 inhibition with a SGLT2 inhibitor demonstrated additive benefit. Taken together, these data support continued development of TN-301 as a potential treatment for patients with HFpEF. We believe late-stage clinical development of TN-301 is best suited for development by or with a well-resourced partner.
Current wording
We shared positive data from our Phase 1 clinical trial of TN-301 in healthy participants at the 2023 Heart Failure Society of America Annual Scientific Meeting. TN-301 was generally well tolerated across the broad range of doses studied. Pharmacokinetic results showed overall dose proportionality with a half-life supportive of once-daily dosing. Increasing doses and exposures with TN-301 correlated with increased pharmacodynamic effects. There were no changes in histone acetylation with TN-301 underscoring the selectivity of TN-301 for HDAC6 and potentially reducing the risk of off target effects. In comparative studies, selective HDAC6 inhibition as a single agent has been shown to have similar efficacy to empagliflozin, a sodium-glucose cotransporter-2 (SGLT2) inhibitor which is approved for the treatment of HFpEF and co-administration of our HDAC6 inhibition with a SGLT2 inhibitor in a HFpEF mouse model demonstrated additive benefit. Taken together, these data support continued development of TN-301 as a potential treatment for patients with HFpEF and other severe diseases - including those outside of cardiology- in which inflammation, fibrosis and metabolic dysregulation may be implicated, and based on TN-301’s multi-modal mechanism of action, TN-301 may have future potential in a number of related cardiometabolic conditions. We believe late-stage clinical development of TN-301 is best suited for development by or with a well-resourced partner.
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 addition, gene editing, gene silencing, and cellular regeneration 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 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
Research and development activities account for a significant portion of our operating expenses. Research and development expenses relate primarily to discovery and development of our research programs, product candidates and proprietary platform technology, and are recognized as incurred. Internal research and development costs include, among others, employee-related costs (including salaries, benefits and stock-based compensation for employees engaged in research and development functions), laboratory supplies, other non-capital equipment utilized for in-house research, and allocated overhead costs. External research and development expenses include, among others, fees paid to contract research organizations (CROs) to execute preclinical studies and clinical trials on our behalf, consulting fees and fees related to licensing agreements. We do not allocate our costs by research program, product candidate or proprietary platform technology, as a significant amount of research and development expenses represent internal costs, which are deployed across our programs, product candidates, proprietary platform technology, and other activities.
Current wording
Research and development activities account for a significant portion of our operating expenses. Research and development expenses relate primarily to discovery and development of our research programs, product candidates and proprietary platform technology, and are recognized as incurred. Internal research and development costs include, among others, employee-related costs (including salaries, benefits and stock-based compensation for employees engaged in research and development functions), laboratory supplies, other non-capital equipment utilized for in-house research, and allocated overhead costs. External research and development expenses include, among others, fees paid to contract research organizations to execute preclinical studies and clinical trials on our behalf, and consulting fees. We do not allocate our costs by research program, product candidate or proprietary platform technology, as a significant amount of research and development expenses represent internal costs, which are deployed across our programs, product candidates, proprietary platform technology, and other activities.
Previous wording
We expense all research and development costs in the periods in which they are incurred. We enter into various agreements with CROs. Costs of certain research and development activities are recognized based on estimates from a number of factors, including an evaluation of the progress of the activities, as well as input from external service providers.
Current wording
We expense all research and development costs in the periods in which they are incurred. Costs of certain research and development activities are recognized based on estimates from a number of factors, including an evaluation of the progress of the activities, as well as input from external service providers.
Previous 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
Current wording
a decrease of $1.0 million in manufacturing costs reflecting lower employee-related costs driven by our workforce reduction initiated in May 2024 (the 2024 Workforce Reduction), lower costs of supplies and materials and lower facility management fees; and
Previous wording
The process of conducting the necessary research to advance through the clinical stages and ultimately obtain regulatory approval is costly and time-consuming, and the successful development of our product candidates is highly uncertain. As a result, we cannot reasonably estimate or know the nature, timing or estimated costs of the efforts that will be necessary to complete the development of, and obtain regulatory approval for, or when and to what extent we will generate revenue from the commercialization and sale of any of our product candidates. However, we expect our research and development expenses to generally remain flat for the remainder of 2024.
Current wording
The process of conducting the necessary research to advance through the clinical stages and ultimately obtain regulatory approval is costly and time-consuming, and the successful development of our product candidates is highly uncertain. As a result, we cannot reasonably estimate or know the nature, timing or estimated costs of the efforts that will be necessary to complete the development of, and obtain regulatory approval for, or when and to what extent we will generate revenue from the commercialization and sale of any of our product candidates. The level of our research and development expenses over the next twelve months will be subject to operational decisions made following data generated from our MyPEAK-1 and RIDGE-1 clinical trials.
Previous wording
General and administrative expenses were $6.4 million and $7.8 million for the three months ended September 30, 2024 and 2023, respectively. The year-over-year decrease of $1.5 million, or 19%, was primarily due to decreases in employee-related costs driven by the Workforce Reduction and lower professional fees.
Current wording
General and administrative expenses were $6.5 million and $8.7 million for the three months ended March 31, 2025 and 2024, respectively. The year-over-year decrease of $2.2 million, or 26%, was primarily due to decreases in employee-related costs driven by the 2024 Workforce Reduction and lower professional fees.
Previous 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.
Current wording
Interest income primarily consists of interest earned on our cash, cash equivalents and investment balances. The interest income was $0.6 million and $1.5 million for the three months ended March 31, 2025 and 2024, respectively. The year-over-year decrease of $0.8 million was primarily due to lower cash, cash equivalents and investment balances.
Previous 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.
Current wording
Net loss for the three months ended March 31, 2025, was $26.9 million, compared to a net loss of $32.2 million for the three months ended March 31, 2024.
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 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.
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 March 31, 2025, we have funded our operations primarily from the sale and issuance of our equity securities. As of March 31, 2025, we had cash, cash equivalents and investments in marketable securities of $88.2 million and an accumulated deficit of $541.3 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). 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.
Current wording
On August 6, 2024, we entered into a Loan Agreement with Silicon Valley Bank (SVB). As of March 31, 2025, under the Loan Agreement, we have the right to draw down $22.5 million at our discretion, up to an additional $2.5 million upon the satisfaction of a clinical milestone orthe receipt of specified proceeds from equity financings and other qualified funding, 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 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.
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). 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. As of December 31, 2024, all pre-funded warrants have been exercised for an exercise price of $0.001 per share.
Previous wording
On August 10, 2022, we entered into a sales agreement (the Sales Agreement) with Leerink Partners LLC (formerly SVB Securities LLC). Pursuant to the Sales Agreement, we may sell from time to time up to an aggregate of $75.0 million of our common stock through an “at-the-market” (ATM) offering defined in Rule 415 under the Securities Act. The $75.0 million of common stock that may be offered, issued and sold under the Sales Agreement is included in the $300.0 million of securities that may be offered, issued and sold by us under our registration statement on Form S-3 (File No. 333-266741). As of September 30, 2024, we may issue and sell up to approximately $71.0 million in additional shares of our common stock under the ATM.
Current wording
On August 10, 2022, we entered into a sales agreement (the Sales Agreement) with Leerink Partners LLC to establish an “at-the-market” (ATM) offering defined in Rule 415 under the Securities Act. Pursuant to the Sales Agreement, the Company is permitted to offer and sell, from time to time, shares of its common stock having a maximum aggregate offering price of up to $75.0 million. In January 2025, we sold 822,566 shares of our common stock under the ATM offering for net proceeds of $0.9 million, after deducting commissions and offering costs of $0.3 million. As of March 31, 2025, the Company may issue and sell up to approximately $69.8 million of common stock under the ATM offering.
Previous wording
We expect that we will continue to incur operating losses over the foreseeable future. Our operating expenses are expected to remain relatively flat for the next twelve months but may increase in the future, if and as we:
Current wording
We expect that we will continue to incur operating losses over the foreseeable future. Our operating expenses may increase in the future, if and as we:
Previous 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.
Current wording
Based on our current operating plan, we believe that our existing cash, cash equivalents and investments in marketable securities, along with the $22.5 million of funds available under our 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
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.
Current wording
Net cash used in operating activities for the three months ended March 31, 2025 was $23.1 million, which consisted primarily of a net loss of $26.9 million and a net change in operating assets and liabilities of $3.4 million, partially offset by $6.9 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 $2.3 million and a decrease in operating lease liabilities of $0.7 million, an increase of prepaid expenses and other current assets of $0.5 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 $3.7 million and depreciation and amortization of $2.7 million.
Previous 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.
Current wording
Net cash used in operating activities for the three months ended March 31, 2024 was $28.7 million, which consisted primarily of a net loss of $26.9 million and a net change in operating assets and liabilities of $3.6 million, partially offset by $6.9 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 $3.1 million and a decrease in operating lease liabilities of $1.1 million, partially offset by a decrease of prepaid expenses and other current assets of $0.4 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 $4.2 million and depreciation and amortization of $2.2 million.
Previous 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.
Current wording
Net cash provided by investing activities for the three months ended March 31, 2025 was $22.2 million, which consisted primarily of proceeds from maturities of marketable securities of $11.7 million and sales of marketable securities of $11.0 million.
Previous 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.
Current wording
Net cash used in investing activities for the three months ended March 31, 2024 was $4.5 million, which consisted primarily of purchases of marketable securities of $23.6 million, partially offset by proceeds from maturities of marketable securities of $19.7 million.
Previous 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.
Current wording
Net cash provided by financing activities for the three months ended March 31, 2025 was $50.3 million, which primarily consisted of net proceeds from our March 2025 follow-on offering of $49.3 million.
Previous 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.
Current wording
Net cash provided by financing activities for the three months ended March 31, 2024 was $46.8 million, which primarily consisted of net proceeds from the follow-on offering of $46.8 million.
Previous 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.
Current wording
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, 2024.
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 nine months ended September 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, 2024. There were no material changes to our critical accounting policies and estimates during the three months ended March 31, 2025.
Previous wording
See Note 2 to our unaudited interim condensed financial statements for more information about recent accounting pronouncements, the timing of their adoption, and our assessment, to the extent we have made one yet, of their potential impact on our financial condition of results of operations.
Current wording
See Note 2, Summary of Significant Accounting Policies, to our unaudited interim condensed financial statements for more information about recent accounting pronouncements, the timing of their adoption, and our assessment, to the extent we have made one yet, of their potential impact on our financial condition of results of operations.
Rearranged previous paragraphs (3)
Previous report — wording carried elsewhere
The following table summarizes our results of operations for the periods presented:
Previous report — wording carried elsewhere
The following table summarizes our research and development expenses for the periods presented:
Previous report — wording carried elsewhere
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.