Neurogene Inc.

Previous wording

Our first clinical-stage program to utilize the EXACT platform is NGN-401, which is in development for the treatment of Rett syndrome, a disease with a patient population that has a signi ficant unmet need, and that ultimately progresses to substantial neurological and physical impairment and premature death. We have completed dosing in a Phase 1/2 open-label, multi-center clinical trial of NGN-401 gene therapy for the treatment of female patients with classic Rett syndrome that is assessing the safety, tolerability, and efficacy of NGN-401 at a dose of 1E15 vg. Eight pediatric participants in the ages 4-10 years old cohort and two participants in the ages 11 years and older cohort received the 1E15 vg dose. We have begun trial initiation activities for our registrational trial of NGN-401, Embolden TM. Clinical grade NGN-401 is manufactured at our manufacturing facility and was used for dosing in the Phase 1/2 clinical trial and will be used for the Embolden clinical trial. NGN-401 is delivered using a one-time intracerebroventricular (“ICV”) procedure, which we believe is the most suitable route of administration to achieve optimal biodistribution in key regions of the brain and other parts of the nervous system that underlie Rett syndrome pathophysiology.

Current wording

Our first clinical-stage program to utilize the EXACT platform is NGN-401, which is in development for the treatment of Rett syndrome, a disease with a patient population that has a significant unmet need, and that ultimately progresses to substantial neurological and physical impairment and premature death. Our ongoing registrational trial of NGN-401, Embolden TM, is a single-arm, open-label, baseline-controlled trial evaluating the 1E15 vg dose of NGN-401 in 20 females with Rett syndrome. The Embolden trial is designed to evaluate NGN-401 in females ages three and above with potential to support a broad label in a single study and enable an efficient path to market. The first participant was dosed during the fourth quarter of 2025. We completed dosing in a Phase 1/2 open-label, multi-center clinical trial of NGN-401 gene therapy for Rett syndrome, with ten participants receiving the 1E15 vg dose. Clinical grade NGN-401 is manufactured at our manufacturing facility and was used for dosing in the Phase 1/2 clinical trial and is being used for the Embolden trial. NGN-401 is delivered using a one-time intracerebroventricular (“ICV”) procedure, which we believe is the most suitable route of administration to achieve optimal biodistribution in key regions of the brain and other parts of the nervous system that underlie Rett syndrome pathophysiology.

Previous wording

We received clearance of our Investigational New Drug (“IND”) application by the U.S. Food and Drug Administration (“FDA”) in January 2023. The Phase 1/2 clinical trial was also initiated in the United Kingdom (“UK”) and Australia. Participants were dosed in all three regions.

Current wording

We received clearance of our Investigational New Drug (“IND”) application by the U.S. Food and Drug Administration (“FDA”) in January 2023.

Previous wording

In June 2025, we also announced written agreement from the FDA on the key elements of the Embolden registrational trial design. Embolden is a single-arm, open-label, baseline-controlled registrational trial evaluating the 1E15 vg dose of NGN-401 in females ages three years and older with Rett syndrome. The primary endpoint is a responder-based composite endpoint that will assess an improvement in the CGI-I scale with Rett syndrome anchors and the gain of a developmental milestone/skill, compared to the participant’s own baseline. Responders are defined as participants who attain a CGI-I score greater than or equal to 3 (“minimally improved”) and gain any one developmental milestone/skill from a list of 28, as captured through standardized video recordings and independently verified by blinded central raters at the 12-month endpoint. In further maintaining the rigorous design of the Embolden trial, we are electing to dose the last planned participant from the Phase 1/2 trial as part of the registrational Embolden trial and add one more participant to complete the proposed sample size at 20 patients.

Current wording

In June 2025, we first announced written agreement from the FDA on key elements of the NGN-401 Embolden registrational trial design, and we confirmed these elements and the trial design in September 2025. The primary endpoint is a responder-based composite endpoint that will assess an improvement in the Clinical Global Impression–Improvement Scale (“CGI-I”) with Rett syndrome anchors and the gain of a developmental milestone/skill, compared to the participant’s own baseline. Responders are defined as participants who attain a CGI-I score less than or equal to three (“minimally improved”) and gain any one developmental milestone/skill from a list of 28, as captured through standardized video recordings and independently verified by blinded central raters at the 12-month endpoint. A response rate of 35% (or 7 out of 20 patients) is the minimum threshold for success to reject the null hypothesis in the Embolden trial.

Previous wording

In November 2024, we announced positive interim clinical data in participants receiving the 1E15 vg dose (n=4 for efficacy data; n=5 for safety data) in the Phase 1/2 clinical trial with a data cut-off date of October 17, 2024.

Current wording

In November 2025, we announced updated positive interim clinical data from the Phase 1/2 NGN-401 clinical trial in the pediatric cohort (ages 4-10) of participants receiving the 1E15 vg dose (n=8 for efficacy data; n=10 for safety data which includes both pediatric and adolescent/adult participants) with a data cut-off date of October 30, 2025.

Previous wording

The baseline demographics of the first five participants who received the 1E15 vg dose NGN-401 include:

Current wording

The baseline demographics of the eight participants in the pediatric cohort who received the 1E15 vg dose of NGN-401 and were reported on in the efficacy data update include:

Previous wording

In May 2024, we added a cohort evaluating a 3E15 vg dose of NGN-401 (high-dose) to the Phase 1/2 clinical trial. In November 2024, the third participant receiving the 3E15 vg dose died following complications from a rare hyperinflammatory syndrome associated with systemic exposure to high doses of AAV, and we discontinued use of that dose level. Hyperinflammatory syndromes can include hemophagocytic lymphohistiocytosis (“HLH”) and multisystem inflammatory syndrome. Based on research we conducted related to hyperinflammatory syndromes and AAV gene therapy, HLH has only been reported following doses of AAV that are generally in the 1E14 vg/kg range or higher. The 1E15 vg dose level we are moving forward in the trial translates into the E13 vg/kg range, and we are not aware of any case of HLH ever being reported at this dose level.

Current wording

The Phase 1/2 trial previously included a cohort evaluating a 3E15 vg dose of NGN-401. In November 2024, the third participant receiving the 3E15 vg dose died following complications from a rare hyperinflammatory syndrome associated with systemic exposure to high doses of AAV, and we discontinued use of that dose level. Hyperinflammatory syndromes can include HLH and multisystem inflammatory syndrome. Based on research we conducted related to hyperinflammatory syndromes and AAV gene therapy, HLH has only been reported following doses of AAV that are generally in the 1E14 vg/kg range or higher. The 1E15 vg dose level we moved forward in the Phase 1/2 trial and are evaluating in the Embolden registrational trial translates into the E13 vg/kg range, and we are not aware of any case of HLH ever being reported at this dose level.

Previous wording

In December 2024, we updated our Phase 1/2 protocol to remove the 3E15 vg dose from the trial. Despite our research related to hyperinflammatory syndromes and AAV gene therapy showing that HLH has only been reported following doses of systemic AAV that are higher than the 1E15 vg dose level, in an abundance of caution, we have incorporated into our protocol additional monitoring for markers using the HLH diagnostic criteria, such as ferritin, and a treatment algorithm to be used in the event HLH is detected. When administered early, this treatment algorithm has been used successfully to treat cases of HLH both in other AAV gene therapy and other known causes of HLH. There has been no evidence of HLH or hyperinflammatory syndrome in any participant at the 1E15 vg dose level in the NGN-401 clinical trial as of the date of this report.

Current wording

Despite our research related to hyperinflammatory syndromes and AAV gene therapy showing that HLH has only been reported following doses of systemic AAV that are higher than the 1E15 vg dose level, in an abundance of caution, we incorporated into our Phase 1/2 and Embolden protocols additional monitoring for markers using the HLH diagnostic criteria, such as ferritin, and a treatment algorithm to be used in the event HLH is detected. When administered early, this treatment algorithm has been used successfully to treat cases of HLH both in other AAV gene therapy and other known causes of HLH. There has been no evidence of HLH or hyperinflammatory syndrome in any participant at the 1E15 vg dose level in the NGN-401 clinical trial as of the date of this report.

Previous wording

In addition to NGN-401, we were pursuing a conventional gene therapy program for the treatment of CLN5 Batten disease. While we have completed enrollment in a Phase 1/2 clinical trial of NGN-101 in patients with CLN5 Batten disease, in November 2024, we announced that we do not expect to move forward with the NGN-101 CLN5 Batten disease gene therapy program at this time. Given the rarity of the disease, continued investment in the program was predicated on alignment on a streamlined registrational pathway with the FDA. To support a streamlined pathway, we submitted an RMAT application to the FDA. Despite our belief that we met the standard of preliminary clinical evidence required to obtain an RMAT designation, the RMAT application was denied. We are currently evaluating options for the program.

Current wording

In addition to NGN-401, we were pursuing a conventional gene therapy program for the treatment of CLN5 Batten disease. We completed enrollment in a Phase 1/2 clinical trial of NGN-101 in patients with CLN5 Batten disease and in November 2024 announced that we do not expect to move forward with the program at this time. Given the rarity of the disease, continued investment in the program was predicated on alignment on a streamlined registrational pathway with the FDA. To support a streamlined pathway, we submitted an RMAT application to the FDA. Despite our belief that we met the standard of preliminary clinical evidence required to obtain an RMAT designation, the RMAT application was denied. We are currently evaluating options for the program.

Previous wording

Since our inception, we have funded our operations primarily with outside capital (e.g., proceeds from the sale of preferred stock and common stock) and have raised aggregate net proceeds of approximately$521.9 million. However, we have incurred significant recurring losses, including a net loss of$44.7 million and$35.4 million for thesix months ended June 30, 2025 and 2024, respectively. In addition, as of June 30, 2025, we had an accumulated deficit of$307.0 million and cash, cash equivalents and short-term investments totaling$274.5 million. In order to continue our operations, we must achieve profitable operations and/or obtain additional equity or debt financing. Until we achieve profitability, management plans to fund our operations and capital expenditures with cash on hand and the sale and issuance of securities. There can be no assurance that we will be successful in raising additional capital or that such capital, if available, will be on terms that are acceptable to us. If we are unable to raise sufficient additional capital, we may be compelled to consider actions such as reducing the scope of our operations and planned capital expenditures or selling certain assets, including intellectual property assets.

Current wording

Since our inception, we have funded our operations primarily with outside capital (e.g., proceeds from the sale of preferred stock and common stock), we have raised aggregate net proceeds of approximately$552.3 million. However, we have incurred significant recurring losses, including a net loss of$65.6 million and$55.6 million for thenine months ended September 30, 2025 and 2024, respectively. In addition, as of September 30, 2025, we had an accumulated deficit of$327.9 million and cash, cash equivalents and short-term investments totaling$265.4 million. In order to continue our operations, we must achieve profitable operations and/or obtain additional equity or debt financing. Until we achieve profitability, management plans to fund our operations and capital expenditures with cash on hand and the sale and issuance of securities. There can be no assurance that we will be successful in raising additional capital or that such capital, if available, will be on terms that are acceptable to us. If we are unable to raise sufficient additional capital, we may be compelled to consider actions such as reducing the scope of our operations and planned capital expenditures or selling certain assets, including intellectual property assets.

Previous wording

We do not have any products approved for commercial sale and have not generated any commercial revenue from product sales. Our ability to generate product revenue sufficient to achieve and maintain profitability will depend upon the successful development and eventual commercialization of one or more of our product candidates, which we expect, if it ever occurs, will take many years. We expect to spend a significant amount in development and marketing costs prior to such time. We will therefore require substantial additional capital to develop our product candidates and support our continuing operations. We may never succeed in achieving regulatory and marketing approval for our product candidates. We may obtain unexpected results from our preclinical and clinical trials. In November 2024, we announced that we do not expect to move forward with the NGN-101 CLN5 Batten disease gene therapy program at this time. Given the rarity of the disease, continued investment in the program was predicated on alignment on a streamlined registrational pathway with the FDA. To support a streamlined pathway, we submitted an RMAT application to the FDA. Despite our belief that the application met the standard of preliminary clinical evidence required to obtain an RMAT designation, the RMAT application was denied. We arecurrently evaluating options for the program. We may in the future elect to discontinue, delay, or modify additional preclinical and clinical trials of our other product candidates. A change in the outcome of any of these variables with respect to the development of a product candidate could mean a significant change in the costs and timing associated with the development of that product candidate. Accordingly, until such time that we can generate a sufficient amount of revenue from product sales or other sources, if ever, management expects to finance our operations through private or public equity or debt financings, loans or other capital sources, which could include income from collaborations, partnerships or other marketing, distribution, licensing or other strategic arrangements with third parties, or from grants. However, we may be unable to raise additional capital from these sources on favorable terms, or at all, which could have a material adverse effect on our business. Our management cannot provide assurance that we will ever generate positive cash flow from operating activities. Please see the section below entitled Liquidity and Capital Resources for additional details on our substantial capital requirements.

Current wording

We do not have any products approved for commercial sale and have not generated any commercial revenue from product sales. Our ability to generate product revenue sufficient to achieve and maintain profitability will depend upon the successful development and eventual commercialization of one or more of our product candidates, which we expect, if it ever occurs, will take many years. We expect to spend a significant amount in development and marketing costs prior to such time. We will therefore require substantial additional capital to develop our product candidates and support our continuing operations. We may never succeed in achieving regulatory and marketing approval for our product candidates. We may obtain unexpected results from our preclinical and clinical trials. For example, in November 2024 we decided not to move forward with the NGN-101 gene therapy program for CLN5 Batten disease, given the rarity of the disease and the lack of a streamlined registrational pathway with the FDA following denial of our RMAT application for that program. We may in the future elect to discontinue, delay, or modify additional preclinical and clinical trials of our other product candidates. A change in the outcome of any of these variables with respect to the development of a product candidate could mean a significant change in the costs and timing associated with the development of that product candidate. Accordingly, until such time that we can generate a sufficient amount of revenue from product sales or other sources, if ever, management expects to finance our operations through private or public equity or debt financings, loans or other capital sources, which could include income from collaborations, partnerships or other marketing, distribution, licensing or other strategic arrangements with third parties, or from grants. However, we may be unable to raise additional capital from these sources on favorable terms, or at all, which could have a material adverse effect on our business. Our management cannot provide assurance that we will ever generate positive cash flow from operating activities. Please see the section below entitled Liquidity and Capital Resources for additional details on our substantial capital requirements.

Previous wording

Comparison of the Three and Six Months Ended June 30, 2025 and 2024

Current wording

Comparison of the Three and Nine Months Ended September 30, 2025 and 2024

Previous wording

For the three months ended June 30, 2025, we did not generate any revenue, as compared to$0.9 million for thethree months ended June 30, 2024. For the six months ended June 30, 2025, we did not generate any revenue, as compared to$0.9 million for thesix months ended June 30, 2024. We generated licensing revenue from the recognition of upfront payments received under the December 2023 CVR Licensing Agreement and the April 2024 CVR Licensing Agreement. Please see the section below entitled Other Expenses for a discussion on the related CVR liabilities.

Current wording

We did not generate any revenue in the three months ended September 30, 2025 or thethree months ended September 30, 2024. For the nine months ended September 30, 2025, we did not generate any revenue, as compared to$0.9 million for thenine months ended September 30, 2024. We generated licensing revenue from the recognition of upfront payments

Previous wording

Expenses related to the Rett syndrome program increased primarily due to a $0.7 million increase in preclinical costs, $2.4 million increase in clinical trial costs related to the Phase 1/2 and pivotal clinical trial of NGN-401, and a $0.7 million increase in CMC costs. The decrease in expenses related to the Batten disease program was primarily driven by a $1.0 million decrease in clinical trial costs for the Phase 1/2 clinical trial of NGN-101 and $0.2 million decrease in clinical development costs, due to the de-prioritization of the program. The decrease in expenses related to the Early Discovery program was primarily driven by a $1.0 million decrease in preclinical costs.

Current wording

Expenses related to the Rett syndrome program increased primarily due to a $0.2 million increase in preclinical costs, a $1.0 million increase in clinical trial costs related to the Phase 1/2 and pivotal clinical trial of NGN-401, and a $0.6 million increase in CMC costs. The decrease in expenses related to the Batten disease program was primarily driven by a $1.5 million decrease in clinical trial costs for the Phase 1/2 clinical trial of NGN-101 and a $0.2 million decrease in clinical development costs, due to the de-prioritization of the program. The decrease in expenses related to the Early Discovery program was primarily driven by a $0.7 million decrease in preclinical costs.

Previous wording

The increase in unallocated internal expenses was driven primarily by higher salaries, benefits, and stock-based compensation costs due to an increase in research and development headcount, as well as an increase in consumables expense related to CMC.

Current wording

The increase in unallocated internal expenses was driven primarily by higher salaries, benefits, and stock-based compensation costs due to an increase in research and development headcount.

Previous wording

Expenses related to the Rett syndrome program increased primarily due to a $1.9 million increase in preclinical costs, $3.0 million increase in clinical trial costs related to the Phase 1/2 and pivotal clinical trial of NGN-401, and a $1.2 million increase in CMC costs. The decrease in expenses related to the Batten disease program was primarily driven by a $1.3 million decrease in clinical trial costs for the Phase 1/2 clinical trial of NGN-101 and $0.3 million decrease in clinical development costs, due to the de-prioritization of the program. The decrease in expenses related to the Early Discovery program was primarily driven by a $1.1 million decrease in preclinical costs.

Current wording

Expenses related to the Rett syndrome program increased primarily due to a $2.1 million increase in preclinical costs, a $4.0 million increase in clinical trial costs related to the Phase 1/2 and pivotal clinical trial of NGN-401, a $0.6 million increase in clinical development costs, and a $1.8 million increase in CMC costs. The decrease in expenses related to the Batten disease program was primarily driven by a $2.8 million decrease in clinical trial costs for the Phase 1/2 clinical trial of NGN-101 and a $0.5 million decrease in clinical development costs, due to the de-prioritization of the program. The decrease in expenses related to the Early Discovery program was primarily driven by a $1.8 million decrease in preclinical costs.

Previous wording

General and administrative expenses were $6.7 million for thethree months ended June 30, 2025, as compared to$5.3 million for thethree months ended June 30, 2024. The increase was primarily attributable to: (i) an increase of approximately $0.2 million in personnel-related expenses, driven by an increase in headcount to support business operations, (ii) an increase of approximately $0.9 million in stock-based compensation expense, driven by an increase in headcount as well as by an increase of approximately $0.3 million related to PSUs as the first underlying performance condition was deemed probable of achievement and currently considered probable to vest, and (iii) an increase in other costs of approximately $0.3 million related to corporate related expenses and market research costs, as well as to the recovery of approximately $0.1 million related to a business email compromise attack by a third party in the prior period.

Current wording

General and administrative expenses were $6.6 million for thethree months ended September 30, 2025, as compared to$5.9 million for thethree months ended September 30, 2024. The increase was primarily attributable to: (i) an increase of approximately $0.2 million in personnel-related expenses, driven by an increase in headcount to support business operations, (ii) an increase of approximately $0.5 million in stock-based compensation expense, driven by an increase in headcount as well as by an increase of approximately $0.3 million related to PSUs as the first underlying performance condition was deemed probable of achievement and currently considered probable to vest, and (iii) an increase in professional services and consultant fees of approximately $0.1 million driven by increased information technology expenses. These expenses were partially offset by a decrease of approximately $0.2 million in other costs primarily related to corporate expenses and market research costs.

Previous wording

General and administrative expenses were $14.9 million for thesix months ended June 30, 2025, as compared to $10.6 million for thesix months ended June 30, 2024. The increase was primarily attributable to: (i) approximately $0.6 million in personnel-related expenses, driven by an increase in headcount to support business operations, (ii) an increase of $2.9 million in stock-based compensation expense, driven by an increase in headcount as well as by an increase of approximately $1.6 million related to PSUs as the first underlying performance condition was deemed probable of achievement and currently considered probable to vest, and (iii) an increase in other costs of approximately $1.0 million related to corporate related expenses and market research costs, as well as to the recovery of approximately $0.4 million related to a business email compromise attack by a third party in the prior period. These expenses were partially offset by a decrease of approximately $0.3 million in professional services and consulting fees associated with the transition from a private company to a public company in the prior period.

Current wording

General and administrative expenses were $21.4 million for thenine months ended September 30, 2025, as compared to $16.4 million for thenine months ended September 30, 2024. The increase was primarily attributable to: (i) approximately $0.9 million in personnel-related expenses, driven by an increase in headcount to support business operations, (ii) an increase of $3.4 million in stock-based compensation expense, driven by an increase in headcount as well as by an increase of approximately $1.9 million related to PSUs as the first underlying performance condition was deemed probable of achievement and currently considered probable to vest, and (iii) an increase in other costs of approximately $0.8 million related to corporate related expenses and market research costs, as well as to the recovery of approximately $0.4 million related to a business email compromise attack by a third party in the prior period. These expenses were partially offset by a decrease of approximately $0.1 million in professional services and consulting fees associated with the transition from a private company to a public company in the prior period.

Previous wording

Interest income increased by $1.8 million for the six months ended June 30, 2025, as compared to the six months ended June 30, 2024. The increase was primarily due to a significant increase in the amount of our cash, cash equivalents and short-term investments driven by the November 5, 2024 private placement, which was partially offset by a moderate decrease in interest rates.

Current wording

Interest income increased by $2.7 million for the nine months ended September 30, 2025, as compared to the nine months ended September 30, 2024. The increase was primarily due to a significant increase in the amount of our cash, cash equivalents and short-term investments driven by the November 5, 2024 private placement, which was partially offset by a moderate decrease in interest rates.

Previous wording

Other income increased by $1.1 million for the three and six months ended June 30, 2025, as compared to the three and six months ended June 30, 2024, respectively. Both of the increases were primarily attributable to: (i) approximately $0.4 million in Washington state sales tax refunds receivable and (ii) approximately $0.7 million of New York state tax refunds received for the prior period amended returns.

Current wording

Other income increased by $1.1 million for the nine months ended September 30, 2025 as compared to the nine months ended September 30, 2024. The increase was primarily attributable to: (i) approximately $0.4 million in Washington state sales tax refunds and (ii) approximately $0.7 million of New York state tax refunds for the prior period amended returns.

Previous wording

Other expenses decreased by $1.0 million for the six months ended June 30, 2025 as compared to the six months ended June 30, 2024. The decrease was primarily due to (i) approximately $0.8 million of a contingent consideration liability accrual related to the Intellectual Property CVR in connection with the December 2023 Licensing Agreement and the April 2024 Licensing Agreement in the prior period and (ii) approximately $0.3 million of the accrual of a contingent consideration liability related to the Sales Tax CVR for an anticipated sales tax refund from Washington state in the prior period. This was offset by an approximate $0.1 million adjustment to the sales tax refund from Washington state for the six months ended June 30, 2025.

Current wording

Other expenses decreased by $1.0 million for the nine months ended September 30, 2025 as compared to the nine months ended September 30, 2024. The decrease was primarily due to (i) approximately $0.8 million of a contingent consideration liability accrual related to the Intellectual Property CVR in connection with the December 2023 Licensing Agreement and the April 2024 Licensing Agreement in the prior period and (ii) approximately $0.3 million of the accrual of a contingent consideration liability related to the Sales Tax CVR for an anticipated sales tax refund from Washington state in the prior period. This was offset by an approximate $0.1 million adjustment to the sales tax refund from Washington state for the nine months ended September 30, 2025.

Previous wording

As of June 30, 2025, we had cash, cash equivalents and short-term investments totaling$274.5 million. Since inception and through the issuance of these financial statements, we have funded our operations primarily through the sales of convertible preferred stock and common stock for net proceeds of approximately$521.9 million.

Current wording

As of September 30, 2025, we had cash, cash equivalents and short-term investments totaling$265.4 million. Since inception and through the issuance of these financial statements, we have funded our operations primarily through the sales of convertible preferred stock and common stock for net proceeds of approximately$552.3 million, including net proceeds from sale of common stock pursuant to our ATM Sales Agreement.

Previous wording

For the six months ended June 30, 2025, we used $40.2 million of cash in operating activities. Cash used in operating activities reflected our net loss of $44.7 million, a $1.8 million net decrease in our operating assets and liabilities and noncash charges of $6.2 million, which consisted primarily of $7.5 million of stock-based compensation and $1.5 million in depreciation, partially offset by $3.2 million in accretion on the held-to-maturity investments. The primary use of cash was to fund our operations related to the development of our product candidates.

Current wording

For the nine months ended September 30, 2025, we used $59.7 million of cash in operating activities. Cash used in operating activities reflected our net loss of $65.6 million, a $3.1 million net decrease in our operating assets and liabilities and noncash charges of $9.0 million, which consisted primarily of $10.8 million of stock-based compensation and $2.3 million in depreciation, partially offset by $4.0 million in accretion on the held-to-maturity investments. The primary use of cash was to fund our operations related to the development of our product candidates.

Previous wording

For the six months ended June 30, 2024, we used $37.5 million of cash in operating activities. Cash used in operating activities reflected our net loss of $35.4 million, a $7.6 million net decrease in our operating assets and liabilities, and noncash charges of $5.5 million, which consisted primarily of $1.6 million in depreciation and $3.4 million in stock-based compensation. The primary use of cash was to fund our operations related to the development of our product candidates.

Current wording

For the nine months ended September 30, 2024, we used $53.1 million of cash in operating activities. Cash used in operating activities reflected our net loss of $55.6 million, a $5.1 million net decrease in our operating assets and liabilities, and noncash charges of $7.6 million, which consisted primarily of $5.8 million in stock-based compensation, $2.4 million in depreciation and $0.5 million in non-cash operating lease expense, partially offset by $1.5 million in accretion on the held-to-maturity investments. The primary use of cash was to fund our operations related to the development of our product candidates, costs associated with the Reverse Merger (as defined below) and the associated financing (the “Pre-Closing Financing”), and related severance and retention payments to former Neoleukin employees.

Previous wording

For thesix months ended June 30, 2025, net cash flows used in investing activities consisted of purchases of investments of $149.4 million and purchases of property and equipment of $0.9 million, partially offset by proceeds from maturities of short-term investments of $112.7 million.

Current wording

For thenine months ended September 30, 2025, net cash flows used in investing activities consisted of purchases of short-term investments of $226.8 million and purchases of property and equipment of $1.1 million, partially offset by proceeds from maturities of short-term investments of $212.2 million.

Previous wording

For thesix months ended June 30, 2024, net cash flows provided by investing activities consisted of purchases of investments of $42.7 million and purchases of property and equipment of $0.5 million, partially offset by proceeds from maturities of short-term investments of $49.5 million.

Current wording

For thenine months ended September 30, 2024, net cash flows used in investing activities consisted of purchases of short-term investments of $80.2 million and purchases of property and equipment of $0.7 million, partially offset by proceeds from maturities of short-term investments of $58.3 million.

Previous wording

For the six months ended June 30, 2025, net cash flows provided by financing activities were insignificant.

Current wording

For the nine months ended September 30, 2025, net cash flows provided by financing activities primarily consisted of $9.8 million of open market sales of common stock pursuant ATM Sales Agreement.

Previous wording

For the six months ended June 30, 2024, net cash flows used in financing activities consisted of $4.3 million in offering costs paid in connection with the Pre-Closing Financing and $2.9 million in transaction costs related to the Reverse Merger (defined below), partially offset by proceeds of $1.0 million from the exercise of stock options.

Current wording

For the nine months ended September 30, 2024, net cash flows used in financing activities consisted of $4.3 million in offering costs paid in connection with the Pre-Closing Financing and $2.9 million in transaction costs related to the Reverse Merger (defined below), partially offset by proceeds of $1.2 million from the exercise of stock options.

Previous wording

Each contingent value right (“CVR”) distributed pursuant to the CVR Agreement, dated December 18, 2023, by and between us and the Rights Agent (the “CVR Agreement”) contains the contractual right to receive certain net savings, if any, realized by June 30, 2029 in connection with certain legacy lease obligations related to our business prior to the Reverse Merger (the “Lease CVR”). As of June 30, 2025, approximately$1.2 million was recorded as a component of the contingent value rights liability arising from the Lease CVR on our condensed consolidated balance sheet. The commitment relates to Neoleukin’s sublease agreement, effective October 31, 2023, for one of its properties with an unrelated third party for the remainder of the lease term. For more information on the Lease CVR, see Note 9, Commitments and Contingencies—Lease CVR, in the notes to the financial statements included in Part I, Item 1 of this Quarterly Report on Form 10-Q.

Current wording

Each contingent value right (“CVR”) distributed pursuant to the CVR Agreement, dated December 18, 2023, by and between us and the Rights Agent (the “CVR Agreement”) contains the contractual right to receive certain net savings, if any, realized by June 30, 2029 in connection with certain legacy lease obligations related to our business prior to the Reverse Merger (the “Lease CVR”). As of September 30, 2025, approximately$0.7 million was recorded as a component of the contingent value rights liability arising from the Lease CVR on our condensed consolidated balance sheet. The commitment relates to Neoleukin’s sublease agreement, effective October 31, 2023, for one of its properties with an unrelated third party for the remainder of the lease term. For more information on the Lease CVR, see Note 9, Commitments and Contingencies—Lease CVR, in the notes to the financial statements included in Part I, Item 1 of this Quarterly Report on Form 10-Q.

Previous wording

In accordance with the terms of the Sales Tax CVR within the CVR Agreement, we accrued a contingent consideration liability on our condensed consolidated balance sheet. The terms of the CVR Agreement include that CVR holders are eligible to receive certain net proceeds derived from an anticipated sales tax refund from Washington state relating to tax returns filed by Neoleukin prior to Closing. As of June 30, 2025, the liability in the condensed consolidated financial statements arising from the Sales Tax CVR was $0.4 million. For more information on the Sales Tax CVR, see Note 9, Commitments and Contingencies—Sales Tax CVR, in the notes to the financial statements included in Part I, Item 1 of this Quarterly Report on Form 10-Q.

Current wording

In accordance with the terms of the Sales Tax CVR within the CVR Agreement, we accrued a contingent consideration liability on our condensed consolidated balance sheet. The terms of the CVR Agreement include that CVR holders are eligible to receive certain net proceeds derived from an anticipated sales tax refund from Washington state relating to tax returns filed by Neoleukin prior to Closing. In fiscal year 2025, we received proceeds from Washington state for the sales tax refund and remitted the proceeds to the CVR holders. For more information on the Sales Tax CVR, see Note 9, Commitments and Contingencies—Sales Tax CVR, in the notes to the financial statements included in Part I, Item 1 of this Quarterly Report on Form 10-Q.

Previous wording

In August 2024, we entered into a Nonexclusive License Agreement with the Board of Trustees of Leland Stanford Junior University (the “Stanford License Agreement”) to license, on a non-exclusive basis, certain biological materials used in the manufacturing process of our product candidates, including NGN-401. Over the 10-year term of the Stanford License Agreement, we are obligated to pay up to $0.5 million in licensing fees. We may terminate this agreement for convenience upon 30 days’ notice.

Current wording

In August 2024, we entered into a Nonexclusive License Agreement with the Board of Trustees of Leland Stanford Junior University (the “Stanford License Agreement”) to license, on a non-exclusive basis, certain biological materials used in the manufacturing process of our product candidates, including NGN-401. Over the 10-year term of the Stanford License Agreement, we are obligated to pay an annual license maintenance fee. We may terminate this agreement for convenience upon 30 days’ notice.

Previous wording

Our financial statements are prepared in accordance with U.S. GAAP. The preparation of the financial statements and related disclosures requires management to make estimates and judgments that affect the reported amounts of assets, liabilities, costs and expenses, and the disclosure of contingent assets and liabilities in our financial statements. We base our estimates on historical experience, known trends and events and various other factors that management believes are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Management evaluates estimates and assumptions on a periodic basis. Our actual results may differ from these estimates. A summary of our significant accounting policies is presented in Part II, Item 8 of our Annual Report on Form 10-K for the year ended December 31, 2024. There have been no material changes to our significant accounting policies during thesix months ended June 30, 2025.

Current wording

Our financial statements are prepared in accordance with U.S. GAAP. The preparation of the financial statements and related disclosures requires management to make estimates and judgments that affect the reported amounts of assets, liabilities, costs and expenses, and the disclosure of contingent assets and liabilities in our financial statements. We base our estimates on historical experience, known trends and events and various other factors that management believes are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Management evaluates estimates and assumptions on a periodic basis. Our actual results may differ from these estimates. A summary of our significant accounting policies is presented in Part II, Item 8 of our Annual Report on Form 10-K for the year ended December 31, 2024. There have been no material changes to our significant accounting policies during thenine months ended September 30, 2025.