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Camp4 Therapeutics Corp

For the period ended Sep 30, 2026. Filed Nov 5, 2026.

View on SEC.gov

About this filing

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.

Sep 30, 2026 · Filed Nov 5, 2026

CAMP$4.06+9.1%Stored close · as of Nov 6, 2026

What changed in Management’s Discussion and Analysis?

Read the current filing

Previous report: 10-Q

Period ended 2026-06-30; filed 2026-08-13.

0001628280-26-056760

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

Current report: 10-Q

Period ended 2026-09-30; filed 2026-11-05.

0001628280-26-071054

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

The 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 94 previous paragraphs and 94 current paragraphs.

63 unchanged; 26 reworded; 5 added; 4 removed; 1 previous and 0 current paragraphs rearranged.

Tables are not compared: 7 previous and 7 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 (5)

Current report

The ASCEND clinical trial is a Phase 1/2, randomized, double-blind, placebo-controlled, multiple ascending dose (“MAD”) study designed to evaluate the safety, tolerability and pharmacokinetics of CMP-002 and identify an optimal biological dose in participants aged two to less than 18 years with genetically confirmed SYNGAP1-related disorder and a genetically confirmed protein-truncating mutation in SYNGAP1 exons 5–19. Participants will be randomized 3:1 to receive intrathecal administration of CMP-002 or a sham procedure consisting of a pinprick. The clinical trial includes three MAD cohorts with a minimum of eight participants each, followed by an open-label extension study. We expect to dose the first patient in the ASCEND Phase 1/2 clinical trial in the fourth quarter of 2026.

Current report

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

Current report

During the nine months ended September 30, 2025, net cash provided by financing activities was $47.3 million, consisting of net proceeds of $47.5 million from the Initial Closing of the Private Placement. These proceeds were partially offset by financing liability and finance lease principal payments totaling $0.2 million.
Removed paragraphs (4)

Previous report

•finalize preclinical development for CMP-002 and advance into clinical trials;

Previous report

During the six months ended June 30, 2025, net cash used in financing activities was $0.1 million, consisting of principal payments on finance leases.
Reworded paragraphs (26)

Previous wording

We are a clinical-stage biopharmaceutical company pioneering the discovery and development of a new class of RNA-targeting therapeutics with the goal of upregulating gene expression and restoring healthy protein levels to treat a broad range of genetic diseases. Our lead product candidate, CMP-002, has the potential to be the first disease-modifying therapy for the treatment of synaptic Ras GTPase activating protein 1 (“SYNGAP1”)-related disorder, or SYNGAP1, a severe developmental and epileptic encephalopathy (“DEE”) characterized by seizures, developmental delays, and cognitive impairments. SYNGAP1 is caused by haploinsufficiency of the SYNGAP1 gene, where mutation of one functional gene copy results in a reduction in SYNGAP protein levels of up to 50%. While we believe that it remains underdiagnosed, we estimate that there are approximately 21,000 individuals living with SYNGAP1 in the United States and the five largest European markets. There are no approved disease-modifying therapies for SYNGAP1.

Current wording

We are a clinical-stage biopharmaceutical company pioneering the discovery and development of a new class of RNA-targeting therapeutics with the goal of upregulating gene expression and restoring healthy protein levels to treat a broad range of genetic diseases. Our lead product candidate, CMP-002, has the potential to be the first disease-modifying therapy for the treatment of synaptic Ras GTPase activating protein 1 (“SYNGAP1”)-related disorder, or SYNGAP1, a severe developmental and epileptic encephalopathy (“DEE”) characterized by seizures, developmental delays, and cognitive impairments. SYNGAP1 is caused by haploinsufficiency of the SYNGAP1 gene, where mutation of one functional gene copy results in a reduction in SYNGAP protein levels of up to 50%. While we believe that it remains underdiagnosed, we estimate that there are approximately 21,000 individuals living with SYNGAP1 in the United States and the five largest European markets. There are no approved disease-modifying therapies for SYNGAP1.european med

Previous wording

CMP-002 is a novel, intrathecally delivered antisense oligonucleotide (“ASO”) designed to target the SYNGAP1 gene at the transcriptional level to increase gene expression, which may increase SYNGAP protein levels in amounts sufficient to yield therapeutic benefit. In preclinical studies, intracerebroventricular injection of CMP-002 restored SYNGAP protein levels to near normal range in haploinsufficient mice carrying a single copy of the human SYNGAP1 gene after a single dose and rescued motor defects and spatial learning defects following two doses. A single dose of CMP-002 also produced a statistically significant improvement in both seizure threshold and severity of chemically induced tonic-clonic seizures in SYNGAP1 haploinsufficient mice, suggesting the potential for therapeutic benefit across both the neurodevelopmental and seizure phenotypes that characterize SYNGAP1-related disorder. In addition, biweekly intrathecal injections of CMP-002 in cynomolgus monkeys were well tolerated and significantly increased SYNGAP protein levels across multiple brain regions clinically relevant to the disease, with dose-linear increases of CMP-002 in disease-relevant brain regions. We have received clearance from Australia’s Therapeutic Goods Administration and Argentina’s Administración Nacional de Medicamentos, Alimentos y Tecnología Médica to initiate our Phase 1/2 clinical trial of CMP-002 in individuals with SYNGAP1, and we have submitted additional regulatory filings in the European Union and United Kingdom to support broader enrollment across multiple sites. We intend to initiate the Phase 1/2 clinical trial in the fourth quarter of 2026.

Current wording

CMP-002 is a novel, intrathecally delivered antisense oligonucleotide (“ASO”) designed to target the SYNGAP1 gene at the transcriptional level to increase gene expression, which may increase SYNGAP protein levels in amounts sufficient to yield therapeutic benefit. In preclinical studies, intracerebroventricular injection of CMP-002 restored SYNGAP protein levels to near normal range in haploinsufficient mice carrying a single copy of the human SYNGAP1 gene after a single dose and rescued motor defects and spatial learning defects following two doses. A single dose of CMP-002 also produced a statistically significant improvement in both seizure threshold and severity of chemically induced tonic-clonic seizures in SYNGAP1 haploinsufficient mice, suggesting the potential for therapeutic benefit across both the neurodevelopmental and seizure phenotypes that characterize SYNGAP1-related disorder. In addition, biweekly intrathecal injections of CMP-002 in cynomolgus monkeys were well tolerated and significantly increased SYNGAP protein levels across multiple brain regions clinically relevant to the disease, with dose-linear increases of CMP-002 in disease-relevant brain regions. We have received clearance from Australia’s Therapeutic Goods Administration, Argentina’s Administración Nacional de Medicamentos, Alimentos y Tecnología Médica, the United Kingdom’s Medicines and Healthcare Products Regulatory Agency and the regulatory authorities and ethics committees of six European countries to initiate our ASCEND Phase 1/2 clinical trial of CMP-002 in individuals with SYNGAP1.

Previous wording

Our primary therapeutic focus is on diseases of the central nervous system (“CNS”), where there are numerous rare and prevalent haploinsufficient diseases with no approved treatments for which a modest increase in protein expression has the potential to be clinically meaningful. In addition to our SYNGAP1 program, we are advancing discovery programs in other CNS indications. We also intend to leverage strategic discovery partnerships, including our research, collaboration, and license agreement with GlaxoSmithKline, to extend the application of our RAP Platform beyond the CNS and validate our approach to gene upregulation in additional tissues and disease areas.

Current wording

Our primary therapeutic focus is on diseases of the central nervous system (“CNS”), where there are numerous rare and prevalent haploinsufficient diseases with no approved treatments for which a modest increase in protein expression has the potential to be clinically meaningful. In addition to our SYNGAP1 program, we are advancing a preclinical program targeting SHANK3 for Phelan-McDermid syndrome, a neurodevelopmental disorder characterized by autism, developmental delay, intellectual disability, motor impairment and milestone regression, for which there are no approved disease-modifying treatments. We estimate that there are more than 45,000 patients with Phelan-McDermid syndrome in the United States. We expect to designate a development candidate in the SHANK3 program in 2027. We are also evaluating early-stage programs for two additional haploinsufficient diseases of the CNS. Additionally, we intend to leverage strategic discovery partnerships, including our research, collaboration, and license agreement with GlaxoSmithKline Intellectual Property (No.3) Limited ("GSK"), to extend the application of our RAP Platform beyond the CNS and validate our approach to gene upregulation in additional tissues and disease areas.

Previous wording

Since our inception in 2015, we have focused substantially all of our resources primarily on developing our RAP Platform, identifying, developing and progressing our product candidates through preclinical and clinical development, organizing and staffing our company, conducting research and development (“R&D”) activities, establishing and protecting our intellectual property portfolio, and raising capital. To date, we have primarily funded our operations with proceeds from the sale of convertible preferred stock and common stock, including pursuant to an underwritten offering completed in December 2025, a private placement of our common stock and pre-funded warrants (the “Private Placement”), the initial closing of which occurred in September 2025 (the “Initial Closing”), and the second closing of which occurred in August 2026 (the “Second Closing”), and our initial public offering (“IPO”), which closed on October 15, 2024, as well as through revenues from our license and collaboration agreements. Through June 30, 2026, we have received net proceeds of $28.1 million from our December 2025 underwritten offering, $46.7 million from the Initial Closing of the Private Placement, $72.4 million from our IPO and $188.3 million from the sale of our convertible preferred stock prior to our IPO. In addition, through June 30, 2026, we have recognized $24.6 million in research collaboration and license revenue through our development and license agreements. Subsequent to June 30, 2026, we received net proceeds of $46.9 from the Second Closing of the Private Placement. Our ability to generate any product revenue and, in particular, our ability to generate product revenue sufficient to achieve profitability, will depend on the successful development and eventual commercialization of product candidates.

Current wording

Since our inception in 2015, we have focused substantially all of our resources primarily on developing our RAP Platform, identifying, developing and progressing our product candidates through preclinical and clinical development, organizing and staffing our company, conducting research and development (“R&D”) activities, establishing and protecting our intellectual property portfolio, and raising capital. To date, we have primarily funded our operations with proceeds from the sale of convertible preferred stock and common stock, including pursuant to an underwritten offering completed in December 2025, a private placement of our common stock and pre-funded warrants (the “Private Placement”), the initial closing of which occurred in September 2025 (the “Initial Closing”), and the second closing of which occurred in August 2026 (the “Second Closing”), and our initial public offering (“IPO”), which closed on October 15, 2024, as well as through revenues from our license and collaboration agreements. Through September 30, 2026, we have received net proceeds of $28.1 million from our December 2025 underwritten offering, $46.7 million from the Initial Closing of the Private Placement, $46.9 million from the Second Closing of the Private Placement, $72.4 million from our IPO and $188.3 million from the sale of our convertible preferred stock prior to our IPO. In addition, through September 30, 2026, we have recognized $26.5 million in research collaboration and license revenue through our development and license agreements. Our ability to generate any product revenue and, in particular, our ability to generate product revenue sufficient to achieve profitability, will depend on the successful development and eventual commercialization of product candidates.

Previous wording

We have incurred significant operating losses and negative cash flows from operations since our inception. Our net losses were $51.9 million and $25.0 million for the six months ended June 30, 2026 and 2025, respectively. As of June 30, 2026, we had an accumulated deficit of $344.0 million. Substantially all our net losses have resulted from costs incurred in connection with our R&D programs and, to a lesser extent, from general and administrative (“G&A”) costs associated with our operations. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and preclinical studies, our other R&D activities and capital expenditures, and the timing and amount of any milestone or royalty payments due under our existing or future license or collaboration agreements. In addition, we incur additional costs associated with operating as a public company, including significant legal, audit, accounting, regulatory and tax-related services associated with maintaining compliance with exchange listing and requirements of the Securities and Exchange Commission (“SEC”), director and officer liability insurance costs, investor and public relations costs, and other expenses that we did not incur as a private company. If we obtain regulatory approval for our product candidates, we expect to incur significant expenses related to developing our commercialization capability to support product sales, marketing and distribution.

Current wording

We have incurred significant operating losses and negative cash flows from operations since our inception. Our net losses were $80.8 million and $40.1 million for the nine months ended September 30, 2026 and 2025, respectively. As of September 30, 2026, we had an accumulated deficit of $373.0 million. Substantially all our net losses have resulted from costs incurred in connection with our R&D programs and, to a lesser extent, from general and administrative (“G&A”) costs associated with our operations. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and preclinical studies, our other R&D activities and capital expenditures, and the timing and amount of any milestone or royalty payments due under our existing or future license or collaboration agreements. In addition, we incur additional costs associated with operating as a public company, including significant legal, audit, accounting, regulatory and tax-related services associated with maintaining compliance with exchange listing and requirements of the SEC, director and officer liability insurance costs, investor and public relations costs, and other expenses that we did not incur as a private company. If we obtain regulatory approval for our product candidates, we expect to incur significant expenses related to developing our commercialization capability to support product sales, marketing and distribution.

Previous wording

As of June 30, 2026, we had cash and cash equivalents of $86.4 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of June 30, 2026, together with the net proceeds of $46.9 million received from the Second Closing under the Purchase Agreement in August 2026, will be sufficient to fund our operating expenses and capital expenditure requirements through the end of 2028. However, we have based this estimate on assumptions that may prove to be wrong, and we could deplete our capital resources sooner than we currently expect. See the sections titled “—Liquidity and Capital Resources” and “Risk Factors—Risks Related to our Financial Position and Need for Additional Capital” included in our 2025 Form 10-K.

Current wording

As of September 30, 2026, we had cash and cash equivalents of $124.5 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of September 30, 2026 will be sufficient to fund our operating expenses and capital expenditure requirements through the end of 2028. However, we have based this estimate on assumptions that may prove to be wrong, and we could deplete our capital resources sooner than we currently expect. See the sections titled “—Liquidity and Capital Resources” and “Risk Factors—Risks Related to Our Financial Position and Need for Additional Capital” included in our 2025 Form 10-K and in this Quarterly Report.

Previous wording

We recognized $1.8 million in research and collaboration revenue during the three months ended June 30, 2026, compared to $1.5 million during the three months ended June 30, 2025. Research and collaboration revenue recognized in the three months ended June 30, 2026 related to our Research, Collaboration and License Agreement with

Current wording

We recognized $1.9 million in research and collaboration revenue during the three months ended September 30, 2026, compared to $0.8 million during the three months ended September 30, 2025. Research and collaboration revenue recognized in the three months ended September 30, 2026 related to our Research, Collaboration and License Agreement with GSK. Research and collaboration revenue recognized in the three months ended September 30, 2025 included $0.8 million related to our Collaboration and License Agreement with BioMarin.

Previous wording

R&D expenses were $10.8 million for the three months ended June 30, 2026, compared to $10.3 million for the three months ended June 30, 2025. The increase of $0.5 million was primarily driven by higher personnel-related costs of $0.5 million due to increased stock-based compensation expense and an increase of $0.4 million in clinical and preclinical costs as we continue to advance CMP-002, including entering into contractual obligations with a contract research organization for services related to our planned Phase 1/2 clinical trial. These increases were partially offset by decreases of $0.2 million in professional and consulting fees and $0.1 million in facilities-related and overhead expense.

Current wording

R&D expenses were $11.4 million for the three months ended September 30, 2026, compared to $9.9 million for the three months ended September 30, 2025. The increase of $1.6 million was primarily driven by higher clinical and preclinical costs of $1.0 million as we continue to advance CMP-002, including entering into contractual obligations with a contract research organization for services related to our planned ASCEND Phase 1/2 clinical trial and an increase in personnel-related costs of $0.9 million due to increased stock-based compensation expense. These increases were partially offset by a decrease of $0.5 million in facilities-related and overhead expense.

Previous wording

G&A expenses were $4.3 million for the three months ended June 30, 2026, compared to $4.2 million for the three months ended June 30, 2025. The increase of $0.2 million was primarily driven by an increase in other expenses of $0.2 million, primarily related to travel expenses, and an increase of $0.1 million in personnel-related costs due to increased stock-based compensation expense. These increases were partially offset by a decrease of $0.2 million in facilities and overhead expenses resulting from the rent abatement period starting in October 2025 associated with the December 2025 lease modification.

Current wording

G&A expenses were $4.5 million for the three months ended September 30, 2026, compared to $4.6 million for the three months ended September 30, 2025. The decrease of $0.1 million was primarily driven by a decrease in professional and consulting fees of $0.8 million and a decrease of $0.3 million in facilities and overhead expenses resulting from rent abatements provided under the December 2025 amendment to our Cambridge and our Watertown lease. These decreases were partially offset by an increase of $0.7 million in personnel-related costs due to increased stock-based compensation expense and other expenses of $0.3 million primarily related to travel expenses.

Previous wording

Other (expense) income, net for the three months ended June 30, 2026 was a $20.2 million expense, compared to $0.4 million of income for the three months ended June 30, 2025. The change was primarily due to a $20.9 million non-cash expense for the change in fair value of our derivative tranche liability associated with the Second Closing of the Private Placement. The derivative tranche liability was subsequently settled in connection with the Second Closing, which occurred on August 3, 2026. This expense was partially offset by a $0.4 million increase in interest income due to higher average invested cash balances during the three months ended June 30, 2026.

Current wording

Other (expense) income, net for the three months ended September 30, 2026 was a $14.9 million expense, compared to $1.4 million of income for the three months ended September 30, 2025. The change was primarily due to a $14.0 million increase in non-cash expense for the change in fair value of our derivative tranche liability associated with the Second Closing of the Private Placement through its settlement in August 2026. This expense was partially offset by a $0.6 million increase in interest income due to higher average invested cash balances during the three months ended September 30, 2026.

Previous wording

The following table summarizes our results of operations for the six months ended June 30, 2026 and 2025 (in thousands):

Current wording

The following table summarizes our results of operations for the nine months ended September 30, 2026 and 2025 (in thousands):

Previous wording

We recognized $3.1 million in research and collaboration revenue during the six months ended June 30, 2026, compared to $2.4 million during the six months ended June 30, 2025. Research and collaboration revenue recognized in the six months ended June 30, 2026 primarily related to our Research, Collaboration and License Agreement with GSK. Research and collaboration revenue recognized in the six months ended June 30, 2025 included $1.8 million related to our Collaboration and License Agreement with BioMarin and a $0.6 million milestone payment earned under the Fulcrum Agreement.

Current wording

We recognized $5.0 million in research and collaboration revenue during the nine months ended September 30, 2026, compared to $3.2 million during the nine months ended September 30, 2025. Research and collaboration revenue recognized in the nine months ended September 30, 2026 primarily related to our Research, Collaboration and License Agreement with GSK. Research and collaboration revenue recognized in the nine months ended September 30, 2025 included $2.6 million related to our Collaboration and License Agreement with BioMarin and a $0.6 million milestone payment earned under the Fulcrum Agreement.

Previous wording

The following table summarizes our R&D expenses for the six months ended June 30, 2026 and 2025 (in thousands):

Current wording

The following table summarizes our R&D expenses for the nine months ended September 30, 2026 and 2025 (in thousands):

Previous wording

R&D expenses were $21.0 million for the six months ended June 30, 2026, compared to $20.5 million for the six months ended June 30, 2025. The $0.5 million increase was primarily driven by higher personnel-related costs of $0.8 million due to increased stock-based compensation expense and an increase of $0.5 million in clinical and preclinical costs as we continue to advance CMP-002, including entering into contractual obligations with a contract research organization for services related to our planned Phase 1/2 clinical trial. These increases were partially offset by decreases of $0.7 million in professional and consulting fees, primarily due to lower outsourced clinical operations costs following the hiring of internal clinical operations personnel, and a decrease of $0.1 million in facilities-related and overhead expense.

Current wording

R&D expenses were $32.4 million for the nine months ended September 30, 2026, compared to $30.3 million for the nine months ended September 30, 2025. The $2.1 million increase was primarily driven by higher personnel-related costs of $1.7 million due to increased stock-based compensation expense and an increase of $1.6 million in clinical and preclinical costs as we continue to advance CMP-002, including entering into contractual obligations with a contract research organization for services related to our planned ASCEND Phase 1/2 clinical trial. These increases were partially offset by decreases of $0.7 million in professional and consulting fees, primarily due to lower outsourced clinical operations costs following the hiring of internal clinical operations personnel, and a decrease of $0.5 million in facilities-related and overhead expense.

Previous wording

The following table summarizes our G&A expenses for the six months ended June 30, 2026 and 2025 (in thousands):

Current wording

The following table summarizes our G&A expenses for the nine months ended September 30, 2026 and 2025 (in thousands):

Previous wording

G&A expenses were $8.5 million for the six months ended June 30, 2026, compared to $8.0 million for the six months ended June 30, 2025. The increase of $0.6 million was primarily driven by personnel-related costs of $0.5 million due to increased stock-based compensation expense and an increase of $0.3 million in professional and consulting fees. These increases were partially offset by a decrease of $0.4 million in facilities and overhead expenses due to the rent abatement period associated with the December 2025 lease modification starting in October 2025.

Current wording

G&A expenses were $13.0 million for the nine months ended September 30, 2026, compared to $12.6 million for the nine months ended September 30, 2025. The increase of $0.5 million was primarily driven by personnel-related costs of $1.2 million due to increased stock-based compensation expense and an increase of $0.4 million in other expenses primarily related to travel expenses. These increases were partially offset by a decrease of $0.7 million in facilities and overhead expenses due to rent abatements provided under the December 2025 amendment to our Cambridge and our Watertown lease and a decrease of $0.5 million in professional and consulting fees.

Previous wording

Other (expense) income, net for the six months ended June 30, 2026 was a $25.4 million expense, compared to $1.1 million income for the six months ended June 30, 2025. The change was primarily due to a $27.1 million non-cash expense for the change in fair value of our derivative tranche liability associated with the Second Closing of our Private Placement. The derivative tranche liability was subsequently settled in connection with the Second Closing, which occurred on August 3, 2026. This expense was partially offset by a $0.7 million increase in interest income due to higher average invested cash balances during the six months ended June 30, 2026.

Current wording

Other (expense) income, net for the nine months ended September 30, 2026 was a $40.3 million expense, compared to $0.3 million income for the nine months ended September 30, 2025. The change was primarily due to a $41.1 million increase in non-cash expense for the change in fair value of our derivative tranche liability associated with the Second Closing of our Private Placement through its settlement in August 2026. This expense was partially offset by a $1.2 million increase in interest income due to higher average invested cash balances during the nine months ended September 30, 2026.

Previous wording

In connection with the filing of the Shelf Registration Statement, we also entered into a sales agreement (the “Sales Agreement”) with Leerink Partners LLC, as sales agent, pursuant to which we may issue and sell shares of our common stock for a maximum aggregate offering price of up to $100.0 million, which is included in the $300.0 million of securities that may be offered pursuant to the Shelf Registration Statement. Pursuant to the Sales Agreement, we will pay the sales agent a commission rate of up to 3.0% of the gross proceeds from the sale of any shares of our common stock. We are not obligated to make any sales of shares of our common stock under the Sales Agreement. During the three and six months ended June 30, 2026 and the year ended December 31, 2025, we did not issue any shares of our common stock under the Sales Agreement.

Current wording

In connection with the filing of the Shelf Registration Statement, we also entered into a sales agreement (the “Sales Agreement”) with Leerink Partners LLC, as sales agent, pursuant to which we may issue and sell shares of our common stock for a maximum aggregate offering price of up to $100.0 million, which is included in the $300.0 million of securities that may be offered pursuant to the Shelf Registration Statement. Pursuant to the Sales Agreement, we will pay the sales agent a commission rate of up to 3.0% of the gross proceeds from the sale of any shares of our common stock. We are not obligated to make any sales of shares of our common stock under the Sales Agreement. During the three and nine months ended September 30, 2026 and the year ended December 31, 2025, we did not issue any shares of our common stock under the Sales Agreement.

Previous wording

As of June 30, 2026, we had cash and cash equivalents of $86.4 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of June 30, 2026, together with the net proceeds of $46.9 million received from the Second Closing under the Purchase Agreement in August 2026, will be sufficient to fund our operating expenses and capital expenditure requirements through the end of 2028. However, we have based this estimate on assumptions that may prove to be wrong, and we could deplete our capital resources sooner than we currently expect. Our future viability is dependent on our ability to generate cash from our operating activities or to raise additional capital to finance our operations. There is no assurance that we will succeed in obtaining sufficient funding on terms acceptable to us to fund continuing operations, if at all.

Current wording

As of September 30, 2026, we had cash and cash equivalents of $124.5 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of September 30, 2026 will be sufficient to fund our operating expenses and capital expenditure requirements through the end of 2028. However, we have based this estimate on assumptions that may prove to be wrong, and we could deplete our capital resources sooner than we currently expect. Our future viability is dependent on our ability to generate cash from our operating activities or to raise additional capital to finance our operations. There is no assurance that we will succeed in obtaining sufficient funding on terms acceptable to us to fund continuing operations, if at all.

Previous wording

The following table provides information regarding our cash flows for the six months ended June 30, 2026 and 2025 (in thousands):

Current wording

The following table provides information regarding our cash flows for the nine months ended September 30, 2026 and 2025 (in thousands):

Previous wording

During the six months ended June 30, 2026, operating activities used $23.7 million of cash, primarily resulting from our net loss of $51.9 million and the timing of working capital fluctuations of $3.5 million, partially offset by non-cash charges of $31.7 million, including stock-based compensation expense, loss from the change in fair value of our derivative tranche liability, non-cash operating lease expense, and depreciation and amortization.

Current wording

During the nine months ended September 30, 2026, operating activities used $31.8 million of cash, primarily resulting from our net loss of $80.8 million and the timing of working capital fluctuations of $1.4 million, partially offset by non-cash charges of $50.4 million, including stock-based compensation expense, loss from the change in fair value of our derivative tranche liability, non-cash operating lease expense, and depreciation and amortization.

Previous wording

During the six months ended June 30, 2025, operating activities used $24.6 million of cash, primarily resulting from our net loss of $25.0 million and the timing of working capital fluctuations of $3.3 million, partially offset by non-cash charges of $3.7 million, including stock-based compensation expense, non-cash operating lease expense, and depreciation and amortization.

Current wording

During the nine months ended September 30, 2025, operating activities used $35.8 million of cash, primarily resulting from our net loss of $40.1 million and the timing of working capital fluctuations of $3.7 million, partially offset by non-cash charges of $7.9 million, including stock-based compensation expense, non-cash operating lease expense, and depreciation and amortization.

Previous wording

In each of the six months ended June 30, 2026 and 2025, cash used in operations was primarily related to clinical and preclinical efforts, compensation and benefits for our employees, consulting and other professional fees, and rent and overhead for our Cambridge and Boulder leases.

Current wording

In each of the nine months ended September 30, 2026 and 2025, cash used in operations was primarily related to clinical and preclinical efforts, compensation and benefits for our employees, consulting and other professional fees, and rent and overhead for our Cambridge and Boulder leases and, for the nine months ended September 30, 2026, overhead for our Watertown lease.

Previous wording

During the six months ended June 30, 2026 and 2025, net cash used in investing activities was $0.1 million and $0.3 million, respectively, due to purchases of property and equipment.

Current wording

During the nine months ended September 30, 2026 and 2025, net cash used in investing activities was $0.9 million and $0.2 million, respectively, primarily due to purchases of property and equipment.

Previous wording

During the six months ended June 30, 2026, net cash provided by financing activities was $0.1 million, consisting of proceeds from the exercise of stock options and issuance of common stock under the 2024 Employee Stock Purchase Plan.

Current wording

During the nine months ended September 30, 2026, net cash provided by financing activities was $47.1 million, consisting of net proceeds of $46.9 million from the Second Closing of the Private Placement and$0.2 million from the exercise of stock options and issuance of common stock under the 2024 Employee Stock Purchase Plan.

Previous wording

There have been no significant changes to our critical accounting estimates in the preparation of our condensed consolidated financial statements during the six months ended June 30, 2026 compared to those disclosed in our 2025 Form 10-K.

Current wording

There have been no significant changes to our critical accounting estimates in the preparation of our condensed consolidated financial statements during the nine months ended September 30, 2026 compared to those disclosed in our 2025 Form 10-K.
Rearranged previous paragraphs (1)

Previous report — wording carried elsewhere

GlaxoSmithKline Intellectual Property (No. 3) Limited (“GSK”). Research and collaboration revenue recognized in the three months ended June 30, 2025 included $0.9 million related to our Collaboration and License Agreement with BioMarin and a $0.6 million milestone payment earned under the Fulcrum Agreement.