Neurogene Inc.

Previous wording

Our lead clinical-stage program, NGN-401, is in development for the treatment of Rett syndrome, a severe and progressive neurodevelopmental disease with substantial neurological and physical impairments and significant unmet need. NGN-401 is purposefully designed to utilize the EXACT platform and adeno-associated virus (“AAV”) delivery viaintracerebroventricular (“ICV”) administration, and to deliver the full-length MECP2 gene which preserves the complete set of endogenous regulatory elements to restore functional MeCP2 protein. We believe ICV delivery 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. We have completed dosing of 25 participants in our ongoing registrational trial of NGN-401, Embolden TM, a single-arm, open-label, baseline-controlled trial evaluating the 1E15 vg dose of NGN-401 gene therapy in 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. Top-line results are expected in the second half of 2027.

Current wording

Our lead clinical-stage program, NGN-401, is in development for the treatment of Rett syndrome, a severe and progressive neurodevelopmental disease with substantial neurological and physical impairments and significant unmet need. NGN-401 is purposefully designed to utilize the EXACT platform and adeno-associated virus (“AAV”) delivery viaintracerebroventricular (“ICV”) administration, and to deliver the full-length MECP2 gene which preserves the complete set of endogenous regulatory elements to restore functional MeCP2 protein. We believe ICV delivery 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. We have completed dosing of 25 participants in our ongoing registrational trial of NGN-401, Embolden TM, a single-arm, open-label, baseline-controlled trial evaluating the 1E15 vg dose of NGN-401 gene therapy in 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. Topline results are expected in the second half of 2027.

Previous 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 CGI-I with Rett syndrome anchors and the gain of a developmental milestone, 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 from a list of 28, as captured through standardized video recordings and independently verified by blinded central raters at the 12-month endpoint. The primary analysis to support the planned BLA is expected to occur after the first 24 participants have completed 12 months of follow-up. A response rate of 33% is the minimum threshold for success to reject the null hypothesis in the Embolden trial. We expect to report top-line data in the second half of 2027, and therefore we do not plan to share data updates from the trial before that time.

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 CGI-I with Rett syndrome anchors and the gain of a developmental milestone, 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 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 9 of 25 participants (36%) is the minimum threshold for success to reject the null hypothesis in the Embolden trial. We expect to report topline data in the second half of 2027, and therefore we do not plan to share data updates from the trial before that time.

Previous wording

We previously reached alignment with the FDA on our potency assay strategy and chemistry, manufacturing and controls (“CMC”) planning for the program. We initiated our Process Performance Qualification (“PPQ”) campaign in July 2026 and confirmed our commercial manufacturing scale is the same as our current clinical manufacturing scale, removing the need for comparability studies. We expect to complete the PPQ runs by the end of 2026.

Current wording

We previously reached alignment with the FDA on our potency assay strategy and chemistry, manufacturing and controls (“CMC”) planning for the program. We initiated our Process Performance Qualification (“PPQ”) campaign in July 2026 and confirmed our commercial manufacturing scale is the same as our current clinical manufacturing scale, removing the need for drug substance comparability studies. We expect to complete the PPQ runs by the end of 2026.

Previous wording

In June 2024, we announced that NGN-401 was one of four sponsors selected by the Center for Biologics Evaluation and Research at the FDA to participate in the FDA’s Support for clinical Trials Advancing Rare disease Therapeutics (“START”) pilot program based on potential for clinical benefits and clinical development program readiness. As part of the START program, we have opportunities for enhanced communications with the FDA, with the aim to further accelerate the pace of NGN-401’s development. These opportunities are designed to provide frequent advice and regular ad-hoc conversations to address product-specific development issues, including, but not limited to, clinical study design, choice of control group and fine-tuning the choice of patient population.

Current wording

In June 2024, we announced that we were one of four sponsors selected by the Center for Biologics Evaluation and Research at the FDA to participate in the FDA’s Support for clinical Trials Advancing Rare disease Therapeutics (“START”) pilot program based on potential for clinical benefits and clinical development program readiness. As part of the START program, we have opportunities for enhanced communications with the FDA, with the aim to further accelerate the pace of NGN-401’s development. These opportunities are designed to provide frequent advice and regular ad-hoc conversations to address product-specific development issues, including, but not limited to, clinical study design, choice of control group and fine-tuning the choice of patient population.

Previous wording

NGN-401 has also received Orphan Drug designation, Fast Track designation and Rare Pediatric Disease designation from the FDA. NGN-401 also received Orphan designation and Advanced Therapy Medicinal Product designation from the European Medicines Agency (EMA).

Current wording

NGN-401 has also received Orphan Drug designation, Fast Track designation and Rare Pediatric Disease designation from the FDA. NGN-401 also received Orphan designation and Advanced Therapy Medicinal Product designation from the EMA.

Previous wording

Since our inception, we have funded our operations primarily with outside capital (e.g., proceeds from the sale of preferred stock, common stock and pre-funded warrants) and have raised aggregate net proceeds of approximately$692.8 million, including net proceeds of approximately $134.8 million from the July 2, 2026 public offering. See “Liquidity and Capital Resources” for additional information. However, we have incurred significant recurring losses, including a net loss of$65.4 million and$44.7 million for thesix months ended June 30, 2026 and 2025, respectively. In addition, as of June 30, 2026, we had an accumulated deficit of$418.1 million and cash, cash equivalents and short-term investments totaling$225.4 million. Following the completion of our July 2, 2026 public offering, we believe our existing cash, cash equivalents and investments will be sufficient to fund our operations into the first quarter of 2029. We may seek additional capital in the future to advance our pipeline, support our operations or pursue strategic opportunities. 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, common stock and pre-funded warrants) and have raised aggregate net proceeds of approximately$718.7 million, including net proceeds of approximately$134.9 million from the July 2026 public offering and net proceeds of approximately $31.8 million from the Sales Agreement. See “Liquidity and Capital Resources” for additional information. However, we have incurred significant recurring losses, including net losses of$94.4 million and$65.6 million for thenine months ended September 30, 2026 and 2025, respectively. In addition, as of September 30, 2026, we had an accumulated deficit of$447.1 million and cash, cash equivalents and short-term investments totaling$359.7 million. Following the completion of our July 2026 public offering, we believe our existing cash, cash equivalents and investments will be sufficient to fund our operations into the first quarter of 2029. We may seek additional capital in the future to advance our pipeline, support our operations or pursue strategic opportunities. 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

In December 2020, we entered into the Master Collaboration Agreement (“MCA”) with the University Court of the University of Edinburgh (the “University of Edinburgh”), which was amended in November 2023 to extend the term of the MCA to December 2026. This collaboration supports our pipeline development activities, and provides us with the option to in-license product candidates arising from research conducted in Dr. Stuart Cobb’s laboratory. Dr. Cobb serves as our Chief Scientific Officer and is also a Professor at the University of Edinburgh. Under the standard policies of the University of Edinburgh, as a professor inventor, he may be entitled to receive in the future a percentage of certain license-related payments made by us to the University. For more information about the MCA, see “Management’s Discussion & Analysis of Financial Condition and Results of Operations—License and Collaboration Agreements”.

Current wording

In December 2020, we entered into the Master Collaboration Agreement (“MCA”) with the University Court of the University of Edinburgh (the “University of Edinburgh”), which was amended in November 2023 to extend the term of the MCA to December 2026. This collaboration supports our pipeline development activities, and provides us with the option to in-license product candidates arising from research conducted in Dr. Stuart Cobb’s laboratory. Dr. Cobb serves as our Chief Scientific Officer and is also a Professor at the University of Edinburgh. Under the standard policies of the University of Edinburgh, as a professor inventor, he may be entitled to receive in the future a percentage of certain license-related payments made by us to the University. For more information about the MCA, see “Management’s Discussion and Analysis of Financial Condition and Results of Operations—License and Collaboration Agreements”.

Previous wording

Research and development activities are central to our business model. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. We expect our research and development expenses to remain significant over the next several years as we continue patient monitoring activities for our ongoing registrational trial, conduct clinical trials for future product candidates, complete process performance qualification (PPQ) activities, and prepare regulatory filings for our product candidates.

Current wording

Research and development activities are central to our business model. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. We expect our research and development expenses to remain significant over the next several years as we continue patient monitoring activities for our ongoing registrational trial, conduct clinical trials for future product candidates, complete PPQ activities, and prepare regulatory filings for our product candidates.

Previous wording

Comparison of the Three and Six Months Ended June 30, 2026 and 2025

Current wording

Comparison of the Three and Nine Months Ended September 30, 2026 and 2025

Previous wording

Research and development expenses were$25.5 million for thethree months ended June 30, 2026, as compared to$19.4 million for thethree months ended June 30, 2025.

Current wording

Research and development expenses were$23.5 million for thethree months ended September 30, 2026, as compared to$17.2 million for thethree months ended September 30, 2025.

Previous wording

Expenses related to the Rett syndrome program increased primarily due to a $2.9 million increase in clinical trial costs related to the ongoing registrational clinical trial for NGN-401 and a $0.7 million increase in CMC costs primarily related to CDMO support and PPQ runs for NGN-401, partially offset by a $0.6 million decrease in preclinical costs. The decrease in expenses related to the Batten disease program was primarily driven by a $0.3 million decrease in clinical trial costs for the Phase 1/2 clinical trial of NGN-101, due to the de-prioritization of the program.

Current wording

Expenses related to the Rett syndrome program increased primarily due to a $1.3 million increase in clinical trial costs related to the ongoing registrational clinical trial for NGN-401 and a $1.9 million increase in CMC costs primarily related to CDMO support and PPQ runs for NGN-401. The decrease in expenses related to the Batten disease program was primarily driven by a $0.1 million credit received from a CRO for clinical trial costs, due to the de-prioritization of the program. The increase in expenses related to the Early Discovery program was primarily driven by a $0.2 million increase in preclinical costs.

Previous wording

Expenses related to the Rett syndrome program increased primarily due to an $8.0 million increase in clinical trial costs related to the ongoing registrational clinical trial for NGN-401 and a $3.1 million increase in CMC costs primarily related to CDMO support and PPQ manufacturing runs for NGN-401, partially offset by a $1.6 million decrease in preclinical 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, due to the de-prioritization of the program. The decrease in expenses related to the Early Discovery programs was driven by a $0.7 million decrease in preclinical development costs.

Current wording

Expenses related to the Rett syndrome program increased primarily due to a $9.3 million increase in clinical trial costs related to the ongoing registrational clinical trial for NGN-401 and a $5.0 million increase in CMC costs primarily related to CDMO support and PPQ manufacturing runs for NGN-401, partially offset by a $1.5 million decrease in preclinical costs and a $0.2 million decrease in clinical development costs related to NGN-401. The decrease in expenses related to the Batten disease program was primarily driven by a $1.0 million decrease in clinical trial costs and a $0.1 million decrease in CMC costs for the Phase 1/2 clinical trial of NGN-101, due to the de-prioritization of the program. The decrease in expenses related to the Early Discovery programs was driven by a $0.5 million decrease in preclinical development costs.

Previous wording

General and administrative expenses were $11.2 million for thethree months ended June 30, 2026, as compared to$6.7 million for thethree months ended June 30, 2025. The increase was primarily attributable to: (i) a$3.3 million increase in stock-based compensation expense, driven by an increase in headcount as well as by an increase of approximately $2.7 million related to PSU expense as the second underlying performance condition was deemed probable of achievement and currently considered probable to vest, (ii) a$0.6 million increase in personnel-related expenses, reflecting higher headcount to support business operations, (iii) a$0.4 million increase in other expenses, primarily related to higher corporate costs and pre-commercial activities, and (iv) a$0.3 million increase in professional and consultant fees. The increase was partially offset by a$0.1 million decrease in office-related expense.

Current wording

General and administrative expenses were $8.9 million for thethree months ended September 30, 2026, as compared to$6.6 million for thethree months ended September 30, 2025. The increase was primarily attributable to: (i) a$1.2 million increase in stock-based compensation expense, driven by increased headcount and PSU expense recognized after the second underlying performance condition was deemed probable of achievement in the second quarter of 2026, (ii) a$0.9 million increase in personnel-related expenses, reflecting higher headcount to support business operations, and (iii) a$0.2 million increase in professional and consultant fees.

Previous wording

General and administrative expenses were $19.4 million for the six months ended June 30, 2026, as compared to$14.9 millionfor the six months ended June 30, 2025. The increase was primarily attributable to: (i) a$2.7 million increase in stock-based compensation expense, driven by an increase in headcount as well as by an increase of approximately $1.8 million related to PSU expense as the second underlying performance condition was deemed probable of achievement and currently considered probable to vest, (ii) a$0.9 million increase in other expenses, primarily related to higher corporate costs and pre-commercial activities, (iii) a$0.7 million increase in personnel-related expenses, reflecting higher headcount to support business operations and (iv) a$0.4 million increase in professional and consultant fees. These increases were partially offset by a$0.1 million decrease in office-related expense.

Current wording

General and administrative expenses were $28.3 million for the nine months ended September 30, 2026, as compared to$21.4 millionfor the nine months ended September 30, 2025. The increase was primarily attributable to: (i) a$3.9 million increase in stock-based compensation expense, driven by increased headcount and PSU expense after the second underlying performance condition was deemed probable of achievement, (ii) a$0.9 million increase in other expenses, primarily related to higher corporate costs and pre-commercial activities, (iii) a$1.6 million increase in personnel-related expenses, reflecting higher headcount to support business operations and (iv) a$0.6 million increase in professional and consultant fees. These increases were partially offset by a$0.2 million decrease in office-related expense.

Previous wording

Interest income decreased by $1.8 million for the six months ended June 30, 2026, as compared to the six months ended June 30, 2025. The decrease was primarily due to a decrease in short-term investments and lower yields on cash, cash equivalents, and short-term investment balances.

Current wording

Interest income increased by $0.5 million for the three months ended September 30, 2026, as compared to the three months ended September 30, 2025. The increase was primarily due to an increase in the average cash and short-term investment balance related to the July 2026 public offering and the Sales Agreement partially offset by lower yields on cash, cash equivalents, and short-term investment balances.

Previous wording

Interest income decreased by $0.9 million for the three months ended June 30, 2026, as compared to the three months ended June 30, 2025. The decrease was primarily due to a decrease in short-term investments and lower yields on cash, cash equivalents, and short-term investment balances.

Current wording

Interest income decreased by $1.3 million for the nine months ended September 30, 2026, as compared to the nine months ended September 30, 2025. The decrease was primarily due to lower yields on cash, cash equivalents, and short-term investment balances.

Previous wording

Other income decreased $1.1 million for the three and six months ended June 30, 2026 as compared to the three and six months ended June 30, 2025, respectively. The decrease was primarily due to refunds received in 2025 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 decreased by $1.1 million for the nine months ended September 30, 2026, as compared to the nine months ended September 30, 2025. The decrease was primarily due to refunds received in 2025 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.

Previous wording

The change in other expenses was not material for the three and six months ended June 30, 2026 as compared to the three and six months ended June 30, 2025 respectively.

Current wording

The change in other expenses was not material for the three months ended September 30, 2026.

Previous wording

As of June 30, 2026, we had cash, cash equivalents and short-term investments totaling$225.4 million. Since inception, we have funded our operations primarily with outside capital (e.g., proceeds from the sale of preferred stock, common stock and pre-funded warrants) and have raised aggregate net proceeds of approximately$692.8 million, including net proceeds of approximately $134.8 million from the July 2, 2026, public offering described below.

Current wording

As of September 30, 2026, we had cash, cash equivalents and short-term investments totaling$359.7 million. Since inception, we have funded our operations primarily with outside capital (e.g., proceeds from the sale of preferred stock, common stock and pre-funded warrants) and have raised aggregate net proceeds of approximately$718.7 million, including net proceeds of approximately$134.9 million from the July 2026 public offering described below.

Previous wording

In August 2025, we entered into an at-the-market sales agreement (the “Sales Agreement” with Leerink Partners, LLC (“Leerink”) as sales agent, pursuant to which we may offer and sell, from time to time, shares of our common stock with an aggregate price up to $150.0 million through Leerink (the “ATM facility”). As of June 30, 2026,$112.9 million remained available for sale under the Sales Agreement.

Current wording

In August 2025, we entered into an at-the-market sales agreement (the “Sales Agreement”) with Leerink Partners LLC (“Leerink”), as sales agent, pursuant to which we may offer and sell, from time to time, shares of our common stock with an aggregate price up to $150.0 million through Leerink (the “ATM facility”). During the three months ended September 30, 2026, we sold 679,525 shares of our common stock pursuant to the Sales Agreement and received $25.9 million in net proceeds after deducting commissions and other offering expenses. During the nine months ended September 30, 2026, we sold 889,054 shares of our common stock pursuant to the Sales Agreement, and received $31.8 million in net proceeds after deducting commissions and other offering expenses. As of September 30, 2026,$86.2 million remained available for sale under the Sales Agreement.

Previous wording

In July 2026, we issued and sold an aggregate of 4,124,999 shares of our common stock (inclusive of 624,999 shares of common stock pursuant to the exercise in full of the underwriters' option to purchase additional shares) at a public offering price of $30.00 per share and, in lieu of common stock to certain investors, pre-funded warrants to purchase up to 666,666 shares of our common stock at a public offering price of $29.999999 per pre-funded warrant (the "July 2026 Offering"). We received aggregate proceeds of approximately $143.8 million, or approximately$134.8 million after deducting the underwriters’ discount and commissions and estimated offering expenses.

Current wording

In July 2026, we issued and sold an aggregate of 4,124,999 shares of our common stock (inclusive of 624,999 shares of common stock pursuant to the exercise in full of the underwriters’ option to purchase additional shares) at a public offering price of $30.00 per share and, in lieu of common stock to certain investors, pre-funded warrants to purchase up to 666,666 shares of our common stock at a public offering price of $29.999999 per pre-funded warrant (the “July 2026 Offering”). We received net proceeds of approximately$134.9 million, after deducting the underwriters’ discount and commissions and offering expenses.

Previous wording

After giving effect to the July 2026 Offering, we had approximately$360.2 million of cash, cash equivalents and short-term investments. Based on our current operating plan, we believe that our existing cash, cash equivalents and short-term investments should be sufficient to fund our operations into the first quarter of 2029. For more information see “Risk Factors."

Current wording

Based on our current operating plan, we believe that our existing cash, cash equivalents and short-term investments should be sufficient to fund our operations into the first quarter of 2029. For more information, see “Risk Factors.”

Previous wording

For the six months ended June 30, 2026, we used $49.9 million of cash in operating activities. Cash used in operating activities reflected our net loss of $65.4 million, a $3.4 million decrease in our net operating assets and non-cash charges of $12.2 million, which consisted primarily of $11.5 million of stock-based compensation, $1.7 million in depreciation and impairment expense and $0.4 million in non-cash operating lease expense partially offset by $0.7 million in accretion on the held-to-maturity investments and $0.7 million change in the contingent value rights liability. 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, 2026, we used $79.0 million of cash in operating activities. Cash used in operating activities reflected our net loss of $94.4 million, a $0.7 million increase in our net operating assets, partially offset by non-cash charges of $16.2 million, which consisted primarily of $16.6 million of stock-based compensation, $2.5 million in depreciation and impairment expense and $0.6 million in non-cash operating lease expense, partially offset by $2.9 million in accretion on the held-to-maturity investments and $0.7 million change in the contingent value rights liability. 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, 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 increase in our operating assets and liabilities, and non-cash charges of $6.2 million, which consisted primarily of $7.5 million in stock-based compensation, $1.5 million in depreciation and $0.4 million in non-cash operating lease expense, 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 increase in our operating assets and liabilities, and non-cash charges of $9.0 million. The non-cash charges consisted primarily of $10.8 million in stock-based compensation, $2.3 million in depreciation and $0.6 million in non-cash operating lease expense, partially offset by $4.0 million in accretion on the held-to-maturity investments and $0.7 million change in the contingent value rights liability. The primary use of cash was to fund our operations related to the development of our product candidates.

Previous wording

For thesix months ended June 30, 2025, net cash flows used in investing activities consisted of purchases of short-term 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, 2026, net cash flows used in investing activities consisted of purchases of short-term investments of $436.8 million and purchases of property and equipment of $1.0 million partially offset by proceeds from maturities of short-term investments of $324.1 million.

Previous wording

For thesix months ended June 30, 2026, net cash flows provided by investing activities consisted of proceeds from maturities of short-term investments of $249.1 million, partially offset by purchases of short-term investments of $196.2 million and purchases of property and equipment of $0.6 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 the six months ended June 30, 2026, net cash flows provided by financing activities primarily consisted of $5.9 million in net proceeds from open market sales of common stock pursuant to the at-the-market (ATM) sales agreement with Leerink Partners, LLC, $0.2 million from the exercise of stock options and $0.1 million from the proceeds from issuance of common stock under the 2023 Employee Stock Purchase Plan. As of June 30, 2026, the remaining amount authorized for sale under the ATM sales agreement was $112.9 million.

Current wording

For the nine months ended September 30, 2026, net cash flows provided by financing activities primarily consisted of $134.9 million in net proceeds from the July 2026 public offering, $31.9 million in net proceeds from open market sales of common stock pursuant to the Sales Agreement with Leerink Partners LLC, $1.0 million from the exercise of stock options and $0.1 million from the proceeds from issuance of common stock under the 2023 Employee Stock Purchase Plan. As of September 30, 2026, the remaining amount authorized for sale under the Sales Agreement was $86.2 million.

Previous wording

We sub-lease approximately 6,000 square feet of office space for our corporate headquarters in New York, New York. In November 2025, we extended the lease by an additional 19 months at a lower cost per square foot which now expires in January 2028.

Current wording

We lease approximately 6,000 square feet of office space for our corporate headquarters in New York, New York. In November 2025, we extended the lease by an additional 19 months at a lower cost per square foot, and the lease now expires in January 2028.

Previous wording

We lease approximately 6,272 square feet of office space in Seattle, Washington, that was previously used by Neoleukin for additional office and laboratory space for research and development and related uses (the “Eastlake Lease”). The lease expires on September 30, 2026. We also assumed the existing agreement to sublease the Eastlake Lease to an unrelated third party (“Eastlake Sublease”). Pursuant to the terms of the Eastlake Sublease, we are entitled to receive a total of approximately $1.6 million in lease payments. The term of the sublease is through September 30, 2026.

Current wording

Through the end of the third quarter of 2026, we leased approximately 6,272 square feet of office space in Seattle, Washington, that was previously used by Neoleukin for additional office and laboratory space for research and development and related uses (the “Eastlake Lease”). The lease expired on September 30, 2026. We also assumed the existing agreement to sublease the Eastlake Lease to an unrelated third party (“Eastlake Sublease”), which extended through the end of the term of the Eastlake Lease and so also expired on September 30, 2026. Pursuant to the terms of the Eastlake Sublease, we received approximately $1.6 million in lease payments. As of September 30, 2026, the Company had no remaining lease payments receivable under the Eastlake Sublease.

Previous wording

Each contingent value right (“CVR”) distributed pursuant to the CVR Licensing 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 with Neoleukin (the “Lease CVR”). As of June 30, 2026, approximately$0.4 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 Contingent Value Rights 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 with Neoleukin (the “Lease CVR”). As of September 30, 2026, approximately$0.4 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, which expired on September 30, 2026. 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

The December 2023 CVR Licensing Agreement and April 2024 CVR Licensing Agreement collectively account for the total Intellectual Property CVR. As of June 30, 2026, no other development and sales milestones were achieved nor deemed probable of achievement under the December 2023 CVR Licensing Agreement. For more information on the Intellectual Property CVR, see Note 9, Commitments and Contingencies—Intellectual Property CVR, in the notes to the financial statements included in Part I, Item 1 of this Quarterly Report on Form 10-Q.

Current wording

The December 2023 CVR Licensing Agreement and April 2024 CVR Licensing Agreement collectively account for the total Intellectual Property CVR. As of September 30, 2026, no other development and sales milestones were achieved nor deemed probable of achievement under the December 2023 CVR Licensing Agreement or the April 2024 CVR Licensing Agreement. For more information on the Intellectual Property CVR, see Note 9, Commitments and Contingencies—Intellectual Property 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 January 2023, we entered into a Non-Exclusive License Agreement with Sigma-Aldrich Co. LLC, pursuant to which we have a license to certain patents and know-how on a non-exclusive basis related to certain cell lines used in our baculovirus process in exchange for a small annual fee on a product-by-product basis, payable once the first product candidate entered the clinic. In addition, on a product-by-product basis, we are obligated to pay up to $2.5 million in the aggregate for development-related milestones. This agreement remains in force for as long as we continue to possess and use the licensed technology. We may terminate this agreement for convenience upon 60 days’ notice.

Current wording

In January 2023, we entered into a Non-Exclusive License Agreement with Sigma-Aldrich Co. LLC, pursuant to which we have a license to certain patents and know-how on a non-exclusive basis related to certain cell lines used in our baculovirus process in exchange for a small annual fee on a product-by-product basis, payable once the first product candidate entered the clinic. In addition, on a product-by-product basis, we are obligated to pay up to $2.5 million in the aggregate forregulatory-related milestones. This agreement remains in force for as long as we continue to possess and use the licensed technology. We may terminate this agreement for convenience upon 60 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, 2025. There have been no material changes to our significant accounting policies during thesix months ended June 30, 2026.

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, 2025. There have been no material changes to our significant accounting policies during thenine months ended September 30, 2026.