PepGen Inc.
For the period ended Sep 30, 2025. Filed Nov 12, 2025.
View on SEC.govAbout 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, 2025 · Filed Nov 12, 2025
PEPG$2.47+6.5%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-06-30; filed 2025-08-07.
0001193125-25-174979
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-09-30; filed 2025-11-12.
0001193125-25-276001
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 118 previous paragraphs and 113 current paragraphs.
82 unchanged; 27 reworded; 4 added; 8 removed; 1 previous and 0 current paragraphs rearranged.
Tables are not compared: 4 previous and 4 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 (4)
Current report
On September 26, 2025, we issued and sold 31,250,000 shares of common stock shares at a purchase price of $3.20 per share in an underwritten public offering, or the 2025 Offering. Pursuant to the underwriting agreement, we granted underwriters a 30-day option to purchase up to an additional 4,687,500 shares of common stock at a price of $3.20 per share which was exercised in full on September 25, 2025. The 2025 Offering resulted in net proceeds of $107.6 million after deducting underwriters' fees of $6.9 million and estimated offering expenses of $0.5 million.
Current report
We believe that our existing cash, cash equivalents and marketable securities will be sufficient to fund our currently planned operations into the second half of 2027. We do not expect to generate any revenues from product sales unless and until we successfully complete development and obtain regulatory approval for one or more of our product candidates, which will not be for at least the next several years, if ever. If we obtain regulatory approval for any of our product candidates, we expect to incur significant commercialization expenses related to product sales, marketing, manufacturing and distribution. Accordingly, until such time as we can generate significant revenue from sales of our product candidates, if ever, we expect to finance our cash needs through equity offerings, debt financings or other capital sources, including potential collaborations, licenses and other similar arrangements. However, we may be unable to raise additional funds or enter into such other arrangements when needed on favorable terms or at all. Our failure to raise capital or enter into such other arrangements when needed would have a negative impact on our financial condition and could force us to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
Current report
Comparison of the Nine Months Ended September 30, 2025 and September 30, 2024
Current report
On September 26, 2025, we issued and sold 31,250,000 shares of common stock shares at a purchase price of $3.20 per share in the 2025 Offering. Underwriters exercised their option to purchase an additional 4,687,500 shares of common stock at a price of $3.20 per share on September 25, 2025. The 2025 Offering resulted in net proceeds of $107.6 million after deducting underwriters' fees of $6.9 million and estimated offering expenses of $0.5 million.
Removed paragraphs (8)
Previous report
Our clinical development program for PGN-EDO51 included a completed Phase 1 clinical trial in healthy volunteers, or HVs, and two parallel Phase 2 studies of PGN-EDO51 in DMD patients. The first study, CONNECT1-EDO51, or CONNECT1, was an open-label, multiple ascending dose, or MAD, study in boys and young men living with DMD amenable to exon 51 skipping conducted in Canada. The second Phase 2 study of PGN-EDO51, CONNECT2-EDO51, or CONNECT2, was a randomized, double-blind, placebo-controlled MAD study in boys and young men living with DMD amenable to exon 51 skipping. In early 2024, we received authorization from the Medicines and Healthcare products Regulatory Agency, or the MHRA, to initiate CONNECT2 in the United Kingdom, or U.K. In December 2024, we received a clinical hold notice from the U.S. Food and Drug Administration, or FDA, regarding our investigational new drug, or IND, application to initiate the CONNECT2 study in the U.S. In March 2025, we announced our voluntary decision to pause CONNECT2 until we could review results from the 10 mg/kg cohort in the CONNECT1 study.
Previous report
In May 2025, we reported that in the 10 mg/kg cohort (n=4) of the CONNECT1 study, PGN-EDO51 increased exon 51 skipped transcripts to 4.26% (a mean increase of 3.5%); however, total dystrophin only increased to 0.59% of normal levels (a mean increase of 0.36%). The safety profile of PGN-EDO51 continued to be generally favorable and all treatment-related adverse events were mild in nature. No serious adverse events were reported in the study. Since PGN-EDO51 did not achieve target dystrophin levels in CONNECT1 trial, we made the decision to voluntarily discontinue development of our DMD programs.
Previous report
We intend to focus on advancing our development efforts on our PGN-EDODM1 program for the treatment of myotonic dystrophy type 1, or DM1, to address the underlying cause of DM1, that has a high unmet need.
Previous report
PGN-EDODM1
Previous report
Based on our current operating plan, we believe that our existing cash and cash equivalents as of June 30, 2025 will enable us to fund our development activities and other operations into the second quarter of 2026. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. To finance our operations beyond that point we will need to raise additional capital, which cannot be assured. We may be unable to raise additional funds or enter into such other arrangements when needed or on favorable terms, if at all. Any failure to raise capital as and when needed could have a negative impact on our financial condition and on our ability to pursue our business plans and strategies, including our research and development activities. If we are unable to raise capital, we will need to delay, reduce or terminate some or all planned activities to reduce costs.
Previous report
As of August 7, 2025, the issuance date of the interim condensed consolidated financial statements for the three- and six-months ended June 30, 2025 included elsewhere in this Form 10-Q, based on our recurring losses from operations incurred since inception, expectation of continuing operating losses for the foreseeable future and the need to raise additional capital to finance future operations, our management has concluded that there is substantial doubt about our ability to continue as a going concern for a period of one year from the date that such interim condensed consolidated financial statements are issued. See “Note 1 - Nature of Business and Basis of Presentation” to our interim condensed consolidated financial statements in this, and “Liquidity and Capital Resources” below, for additional information on our assessment.
Previous report
Comparison of the Six Months Ended June 30, 2025 and June 30, 2024
Previous report
Based on our current operating plan, we believe that our existing cash and cash equivalents as of June 30, 2025 will enable us to fund our development activities and other operations into the second quarter of 2026. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. To finance our operations beyond that point we will need to raise additional capital, which cannot be assured. We may be unable to raise additional funds or enter into such other arrangements when needed or on favorable terms, if at all. Any failure to raise capital as and when needed could have a negative impact on our financial condition and on our ability to pursue our business plans and strategies, including our research and development activities. If we are unable to raise capital, we will need to delay, reduce or terminate some or all planned activities to reduce costs.
Reworded paragraphs (27)
Previous wording
Our clinical development program for PGN-EDODM1 includes two studies, FREEDOM-DM1, or FREEDOM, a multinational, randomized, double-blind, placebo-controlled Phase 1 single ascending dose, or SAD, study, and FREEDOM2-DM1, or FREEDOM2, a multinational, randomized, double blind, placebo-controlled Phase 2 MAD study. In the FREEDOM study, we are enrolling adult participants with DM1 in multiple geographies including the U.S., U.K. and Canada, to evaluate the safety and tolerability of PGN-EDODM1, as well as oligonucleotide muscle concentrations, splicing correction and functional outcome measures at day 28 and at week 16 following a single dose of PGN-EDODM1. On February 24, 2025, we reported initial data from the 5 mg/kg and 10 mg/kg dose cohorts in this study. PGN-EDODM1 was observed to have a favorable emerging safety profile and robust splicing correction in a dose-dependent manner in the 5 and 10 mg/kg dose cohorts.. Based on the positive splicing correction observed at the 5 and 10 mg/kg dose cohorts and the totality of the blinded safety data to date, we have decided to conclude dose escalation in the FREEDOM trial with the 15 mg/kg cohort. We have completed patient dosing in the 15 mg/kg cohort of the FREEDOM trial and expect to report safety, 28-day splicing and functional benefit data from the 15 mg/kg cohort in early fourth quarter of 2025.
Current wording
Our clinical development program for PGN-EDODM1 includes two studies, FREEDOM-DM1, or FREEDOM, a multinational, randomized, double-blind, placebo-controlled Phase 1 single ascending dose, or SAD, study, and FREEDOM2-DM1, or FREEDOM2, a multinational, randomized, double blind, placebo-controlled Phase 2 MAD study. In the FREEDOM study, we are enrolling adult participants with DM1 in multiple geographies including the U.S., U.K. and Canada, to evaluate the safety and tolerability of PGN-EDODM1, as well as oligonucleotide muscle concentrations, splicing correction and functional outcome measures at day 28 and at week 16 following a single dose of PGN-EDODM1. On September 24, 2025, we reported topline results from the 15 mg/kg cohort in the FREEDOM study of PGN-EDODM1, demonstrating a mean splicing correction of 53.7% following a single 15 mg/kg dose, noting that PGN-EDOM1 was generally well tolerated at this dose, with all drug-related adverse events mild or moderate in severity. Additionally, on February 24, 2025, we reported initial data from the 5 mg/kg and 10 mg/kg dose cohorts in this study where PGN-EDODM1 was observed to have a favorable emerging safety profile and robust splicing correction in a dose-dependent manner in the 5 and 10 mg/kg dose cohorts. Based on the positive splicing correction observed in the FREEDOM trial including the totality of the blinded safety data to date, we have decided to conclude dose escalation in the FREEDOM trial with the 15 mg/kg cohort.
Previous wording
The safety data from the ongoing FREEDOM study has informed the design of FREEDOM2, which is currently open in Canada and U.K. We also plan to open the FREEDOM2 study other geographies, including the U.S., subject to regulatory authorizations. FREEDOM2 is a Phase 2 randomized, double-blind, placebo-controlled MAD study of PGN-EDODM1 in DM1 patients. FREEDOM2 is designed to assess PGN-EDODM1’s safety and tolerability, splicing correction and functional outcome measures in DM1 patients. This study is currently dosing participants in the 5 mg/kg dose cohort and we expect to report data from this cohort in the first quarter of 2026.
Current wording
The safety data from the ongoing FREEDOM study has informed the design of FREEDOM2, which is currently open in Canada and U.K. We also plan to open the FREEDOM2 study other geographies, including the U.S., subject to regulatory authorizations. FREEDOM2 is a Phase 2 randomized, double-blind, placebo-controlled MAD study of PGN-EDODM1 in DM1 patients. FREEDOM2 is designed to assess PGN-EDODM1’s safety and tolerability, splicing correction and functional outcome measures in DM1 patients. All participants in the 5 mg/kg dose cohort have been enrolled and received at least one dose and we expect to report data from this cohort in the first quarter of 2026.
Previous wording
On May 28, 2025, we announced that based on the levels of dystrophin protein measured in the 10 mg/kg cohort of the CONNECT1-EDO51 study investigating our candidate, PGN-EDO51, in Duchenne muscular dystrophy, or DMD, for patients amenable to exon 51 skipping, we have decided to voluntarily discontinue development of PGN-EDO51. Additionally, we intend to wind down all DMD-related research and development activities, including our research efforts relating to exons 44, 45 and 53.
Current wording
We have also initiated research efforts for additional potential investigational drugs addressing other neuromuscular diseases and neurologic disorders. On May 28, 2025, we announced that based on the levels of dystrophin protein measured in the 10 mg/kg cohort of the CONNECT1-EDO51 study investigating our candidate, PGN-EDO51, in Duchenne muscular dystrophy, or DMD, for patients amenable to exon 51 skipping, we have decided to voluntarily discontinue development of PGN-EDO51. Additionally, all DMD-related research and development activities, including our research efforts relating to exons 44, 45 and 53, are being wound down.
Previous wording
Initial Public Offering, ATM Program, Follow-on Offering and Liquidity
Current wording
Initial Public Offering, ATM Program, Follow-on Offerings and Liquidity
Previous wording
On February 5, 2024, we issued and sold 1,000,000 of common stock shares at a purchase price of $10.00 per share under our at-the-market offering program, or ATM program, pursuant to an At-the-Market Equity Offering Sales Agreement, or Sales Agreement, with Stifel, Nicolaus & Company, Incorporated, or Stifel, resulting in net proceeds of $9.9 million. On February 9, 2024, we issued and sold 7,530,000 shares of common stock at a purchase price of $10.635 per share, which was the closing sale price of our common stock on the Nasdaq Global Select Market on February 6, 2024, in an underwritten follow-on offering, or the Follow-on Offering. The Follow-on Offering resulted in net proceeds of $76.4 million after deducting underwriters' fees of $3.7 million. Net proceeds from the ATM program and Follow-on Offering, after deducting underwriters’ fees and costs of the offerings, were $86.3 million.
Current wording
On February 5, 2024, we issued and sold 1,000,000 of common stock shares at a purchase price of $10.00 per share under our at-the-market offering program, or ATM program, pursuant to an At-the-Market Equity Offering Sales Agreement, or Sales Agreement, with Stifel, Nicolaus & Company, Incorporated, or Stifel, resulting in net proceeds of $9.9 million. On February 9, 2024, we issued and sold 7,530,000 shares of common stock at a purchase price of $10.635 per share, which was the closing sale price of our common stock on the Nasdaq Global Select Market on February 6, 2024, in an underwritten follow-on offering, or the 2024 Offering. The 2024 Offering resulted in net proceeds of $76.4 million after deducting underwriters' fees of $3.7 million. Net proceeds from the ATM program and 2024 Offering, after deducting underwriters’ fees and costs of the offerings, were $86.3 million.
Previous wording
Since our inception, we have not generated any revenue from product sales or other sources and have incurred significant operating losses and negative cash flows from our operations. Our primary uses of cash to date have been to fund our research and development activities, business planning, establishing and maintaining our intellectual property portfolio, acquiring and developing product and technology rights, hiring personnel, leasing premises and associated capital expenditures, raising capital, and providing general and administrative support for these operations. To date, we have funded our operations primarily through private placements of our convertible preferred stock and proceeds from our IPO, the ATM program and the Follow-on Offering.
Current wording
Since our inception, we have not generated any revenue from product sales or other sources and have incurred significant operating losses and negative cash flows from our operations. Our primary uses of cash to date have been to fund our research and development activities, business planning, establishing and maintaining our intellectual property portfolio, acquiring and developing product and technology rights, hiring personnel, leasing premises and associated capital expenditures, raising capital, and providing general and administrative support for these operations. To date, we have funded our operations primarily through private placements of our convertible preferred stock and proceeds from our IPO, the ATM program, the 2024 Offering, and the 2025 Offering.
Previous wording
We have incurred operating losses in each year since our inception. Our net losses were $53.3 million and $46.4 million for the six months ended June 30, 2025 and June 30, 2024, respectively. As of June 30, 2025, we had an accumulated deficit of $324.8 million. As of June 30, 2025, we had cash, cash equivalents, and marketable securities of $74.7 million. Notwithstanding our recent decision to cease our research and development efforts in DMD, we expect our expenses and operating losses will continue as we conduct our ongoing preclinical studies and current and planned clinical trials of PGN-EDODM1, continue our research and development activities, utilize third parties to manufacture our product candidates and related raw materials, hire additional personnel, protect our intellectual property and incur additional costs associated with being a public company, including audit, legal, regulatory, and tax-related services associated with maintaining compliance with an exchange listing and SEC requirements, director and officer insurance premiums, and investor relations costs. In addition, we have several development, regulatory and commercial milestone payment obligations under our licensing arrangements. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our preclinical studies, current and planned clinical trials, manufacturing campaigns and our expenditures on other research and development activities.
Current wording
We have incurred operating losses in each year since our inception. Our net losses were $71.3 million and $67.7 million for the nine months ended September 30, 2025 and September 30, 2024, respectively. As of September 30, 2025, we had cash, cash equivalents, and marketable securities of $163.7 million. As of September 30, 2025, we had an accumulated deficit of $342.8 million. Notwithstanding our decision to cease our research and development efforts in DMD, which we announced on May 28, 2025, we expect our expenses and operating losses will continue as we conduct our ongoing preclinical studies and current and planned clinical trials of PGN-EDODM1, continue our research and development activities, utilize third parties to manufacture our product candidates and related raw materials, hire additional personnel, protect our intellectual property and incur additional costs associated with being a public company, including audit, legal, regulatory, and tax-related services associated with maintaining compliance with an exchange listing and SEC requirements, director and officer insurance premiums, and investor relations costs. In addition, we have several development, regulatory and commercial milestone payment obligations under our licensing arrangements. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our preclinical studies, current and planned clinical trials, manufacturing campaigns and our expenditures on other research and development activities.
Previous wording
The following table (in thousands) summarizes our research and development expenses for the three and six months ended June 30, 2025 and June 30, 2024. The direct external development program expenses reflect external costs attributable to our clinical development candidates and preclinical candidates selected for further development. Our internal resources, personnel and infrastructure are not directly tied to any one research or drug discovery program and are deployed across multiple programs. As such, we do not track internal expenses on a program-specific basis.
Current wording
The following table (in thousands) summarizes our research and development expenses for the three and nine months ended September 30, 2025 and September 30, 2024. The direct external development program expenses reflect external costs attributable to our clinical development candidates and preclinical candidates selected for further development. Our internal resources, personnel and infrastructure are not directly tied to any one research or drug discovery program and are deployed across multiple programs. As such, we do not track internal expenses on a program-specific basis.
Previous wording
We continue to conduct our ongoing Phase 1 and Phase 2 clinical trials of our investigational drug candidate, PGN-EDODM1. On May 28, 2025, we announced that we have decided to voluntarily discontinue development of PGN-EDO51 and will be winding down all DMD-related research and development activities. Research and development expenses for our lead program can be variable quarter-over-quarter due to the timing of manufacturing campaigns, which are accounted for under the percentage of completion method. The process of conducting preclinical studies and clinical trials necessary to obtain regulatory approval is costly and time consuming. We may never succeed in achieving marketing approval for our remaining clinical stage product candidate or any new product candidates we develop.
Current wording
We continue to conduct our ongoing Phase 2 clinical trial of our investigational drug candidate, PGN-EDODM1. We have decided to conclude dose escalation in the FREEDOM trial with the 15 mg/kg cohort, based on the positive splicing correction observed and the totality of the blinded safety data to date. As stated above, on May 28, 2025, we announced that we have decided to voluntarily discontinue development of PGN-EDO51 and will be winding down all DMD-related research and development activities. Research and development expenses for our lead program can be variable quarter-over-quarter due to the timing of manufacturing campaigns, which are accounted for under the percentage of completion method. The process of conducting preclinical studies and clinical trials necessary to obtain regulatory approval is costly and time consuming. We may never succeed in achieving marketing approval for our remaining clinical stage product candidate or any new product candidates we develop.
Previous wording
The timelines and costs associated with research and development activities are uncertain and can vary significantly for our product candidate and development program due to the inherently unpredictable nature of preclinical and clinical development. We recently announced our decision to focus our development efforts on PGN-EDODM1, and to cease development of our DMD-related programs, and may make further determinations as to which programs in our research and development pipeline to pursue and how much funding to direct to each program. We will need to raise substantial additional capital in the future.
Current wording
The timelines and costs associated with research and development activities are uncertain and can vary significantly for our product candidate and development program due to the inherently unpredictable nature of preclinical and clinical development. We previously announced our decision to focus our development efforts on PGN-EDODM1, and to cease development of our DMD-related programs, and may make further determinations as to which programs in our research and development pipeline to pursue and how much funding to direct to each program. We will need to raise substantial additional capital in the future.
Previous wording
Research and development expenses decreased by $6.7 million from $25.1 million for the three months ended June 30, 2024, to $18.4 million for the three months ended June 30, 2025. This was attributable to a $6.4 million decrease in manufacturing costs related to the timing of manufacturing campaigns, and a $1.3 million decrease in preclinical costs as our two lead programs had advanced into clinical trials, prior to the discontinuation of our DMD program in late May 2025. These decreases are partially offset by a $0.7 million increase in clinical trial expense driven by charges taken during the current quarter for estimated wind-down costs for the two clinical trials for PGN-EDO51. Additionally, there was a $0.7 million non-cash charge taken during the current year associated with the impairment of unused lab equipment. Personnel-related costs remained flat.
Current wording
Research and development expenses decreased by $4.3 million from $17.7 million for the three months ended September 30, 2024, to $13.4 million for the three months ended September 30, 2025. This was attributable to a $1.4 million decrease in personnel-related costs, including a decrease of $0.5 million in stock-based compensation expense, a $1.2 million decrease in manufacturing costs related to the timing of manufacturing campaigns, and a $1.3 million decrease in preclinical costs as our two lead programs had advanced into clinical trials, prior to the discontinuation of our DMD program in late May 2025. This was additionally driven by a $0.5 million decrease in consulting-related costs and a $0.3 million decrease in facility and office related costs, including depreciation. These decreases are partially offset by a $0.4 million increase in clinical trial expense driven by costs related to our Phase 1 and Phase 2 clinical trials for PGN-EDODM1.
Previous wording
General and administrative expenses increased by $0.2 million from $5.4 million for the three months ended June 30, 2024, to $5.6 million for the three months ended June 30, 2025. The increase was primarily driven by an increase of $0.2 million in consulting and legal expenses and an increase of $0.1 million in facility and office expenses. These increases were partially offset by a decrease in personnel-related costs of $0.1 million primarily due to a decrease in stock-based compensation expense.
Current wording
General and administrative expenses decreased by $0.2 million from $5.4 million for the three months ended September 30, 2024, to $5.2 million for the three months ended September 30, 2025. The decrease was primarily driven by an decrease of $0.6 million in personnel-related costs, including a decrease of $0.4 million in stock-based compensation expense. That decrease is partially offset by a $0.4 million increase in consulting and legal expenses.
Previous wording
Other income (expense), net was income of $0.8 million for the three months ended June 30, 2025 and $2.1 million for the three months ended June 30, 2024. Interest is earned through our cash deposits and U.S. Treasury-backed money market funds.
Current wording
Other income (expense), net was income of $0.6 million for the three months ended September 30, 2025 and $1.8 million for the three months ended September 30, 2024. Interest is earned through our cash deposits and U.S. Treasury-backed money market funds.
Previous wording
The following table summarizes our results of operations for the six months ended June 30, 2025 and June 30, 2024 (in thousands):
Current wording
The following table summarizes our results of operations for the nine months ended September 30, 2025 and September 30, 2024 (in thousands):
Previous wording
Research and development expenses increased by $4.0 million from $39.8 million for the six months ended June 30, 2024, to $43.8 million for the six months ended June 30, 2025. This was attributable to a $1.4 million increase in manufacturing costs related to the timing of manufacturing campaigns, a $2.5 million increase in personnel-related costs, including an increase of $0.5 million in stock-based compensation expense, and a $1.8 million increase in clinical trial costs associated with the advancement of clinical development programs and charges taken during the current quarter for estimated wind-down costs for the two clinical trials for PGN-EDO51. There was also a $0.7 million increase in other research and development expenses related to the non-cash charge taken during the current year associated with the impairment of unused lab equipment and a $0.4 million increase in consulting expense. These increases were partially offset by decreased preclinical costs of $2.6 million during the period as our two lead programs had both advanced into clinical trials, prior to the discontinuation of our DMD program in late May 2025. Additionally, there was a $0.2 million decrease in facility and office related expenses.
Current wording
Research and development expenses decreased by $0.3 million from $57.5 million for the nine months ended September 30, 2024, to $57.2 million for the nine months ended September 30, 2025. This was attributable to a $3.9 million decrease in preclinical costs as our two lead programs had both advanced into clinical trials, prior to the discontinuation of our DMD program in late May 2025. Additionally, there was a $0.6 million decrease in facility and office related expenses, including depreciation, and a $0.2 million decrease in consulting expense. These decreases were partially offset by increased clinical trial costs of $2.3 million associated with the advancement of clinical development programs and charges taken during the second quarter of 2025 for estimated wind-down costs for the two clinical trials for PGN-EDO51. There was also a $1.1 million increase in personnel-related costs, and a $0.2 million increase in manufacturing costs related to the timing of manufacturing campaigns. There was also a $0.7 million increase in other research and development expenses related to the non-cash charge taken during the current year associated with the impairment of unused lab equipment.
Previous wording
General and administrative expenses increased by $1.1 million from $10.4 million for the six months ended June 30, 2024, to $11.5 million for the six months ended June 30, 2025. The increase was primarily driven by an increase of $0.7 million in personnel-related costs, including $0.4 million in stock-based compensation expense. This increase was additionally driven by a $0.3 million increase in consulting and legal expenses.
Current wording
General and administrative expenses increased by $0.8 million from $15.9 million for the nine months ended September 30, 2024, to $16.7 million for the nine months ended September 30, 2025. The increase was primarily driven by an increase in consulting and legal expenses.
Previous wording
Other income (expense), net was income of $2.0 million for the six months ended June 30, 2025 and $3.9 million for the six months ended June 30, 2024. Interest is earned through our cash deposits and U.S. Treasury-backed money market funds.
Current wording
Other income (expense), net was income of $2.6 million for the nine months ended September 30, 2025 and $5.7 million for the nine months ended September 30, 2024. Interest is earned through our cash deposits and U.S. Treasury-backed money market funds.
Previous wording
Income tax expense was nil for the six months ended June 30, 2025 and June 30, 2024.
Current wording
Income tax expense was nil for the nine months ended September 30, 2025 and September 30, 2024.
Previous wording
On February 9, 2024, we issued and sold 7,530,000 shares of common stock in the Follow-on Offering at a purchase price of $10.635 per share, resulting in net proceeds of $76.4 million after deducting underwriters’ fees and offering costs of $3.7 million.
Current wording
On February 9, 2024, we issued and sold 7,530,000 shares of common stock in the 2024 Offering at a purchase price of $10.635 per share, resulting in net proceeds of $76.4 million after deducting underwriters’ fees and offering costs of $3.7 million.
Previous wording
As of June 30, 2025, we had cash, cash equivalents, and marketable securities of $74.7 million. We do not believe that our existing cash, cash equivalents, and marketable securities will be sufficient to fund our currently planned operations for at least 12 months from the date of the filing of this Form 10-Q. Our forecast of the period of time through which our financial resources will be adequate to support our operations is a forward-looking statement that involves risks and uncertainties, and actual results could vary materially. We have based this estimate on assumptions that may prove to be wrong, and we could deplete our capital resources sooner than we expect. Additionally, the process of conducting preclinical studies and testing product candidates in clinical trials is costly, and the timing of progress and expenses in these studies and trials is uncertain.
Current wording
As of September 30, 2025, we had cash, cash equivalents, and marketable securities of $163.7 million. We believe that our existing cash, cash equivalents, and marketable securities will be sufficient to fund our currently planned operations into the second half of 2027. Our forecast of the period of time through which our financial resources will be adequate to support our operations is a forward-looking statement that involves risks and uncertainties, and actual results could vary materially. We have based this estimate on assumptions that may prove to be wrong, and we could deplete our capital resources sooner than we expect. Additionally, the process of conducting preclinical studies and testing product candidates in clinical trials is costly, and the timing of progress and expenses in these studies and trials is uncertain.
Previous wording
The following table sets forth a summary of the net cash flow activity for the six months ended June 30, 2025 and June 30, 2024 (in thousands):
Current wording
The following table sets forth a summary of the net cash flow activity for the nine months ended September 30, 2025 and September 30, 2024 (in thousands):
Previous wording
For the six months ended June 30, 2025, net cash used in operating activities was $46.5 million resulting from our net loss of $53.3 million and changes in our operating assets and liabilities of $1.2 million, partially offset by non-cash adjustments of $7.9 million. The net changes in our operating assets and liabilities were primarily due to a decrease in operating lease liabilities of $1.5 million. The change was further driven by a decrease in accounts payable of $0.9 million, an increase in accrued expenses of $0.5 million and a decrease in prepaids and other current assets of $0.7 million. The non-cash adjustments included $5.8 million of stock-based compensation, $0.7 million of depreciation expense, $1.2 million of amortization of discounts on our marketable securities, and $1.9 million of amortization and interest accretion on our operating lease. Additionally, there was a non-cash adjustment of $0.7 million related to the impairment of unused lab equipment.
Current wording
For the nine months ended September 30, 2025, net cash used in operating activities was $65.8 million resulting from our net loss of $71.3 million and changes in our operating assets and liabilities of $5.8 million, partially offset by non-cash adjustments of $11.3 million. The net changes in our operating assets and liabilities were primarily due to a decrease of $3.5 million in accrued expenses and a decrease of $2.3 million in operating lease liabilities. The non-cash adjustments included $8.2 million of stock-based compensation, $1.1 million of depreciation expense, $1.4 million of amortization of discounts on our marketable securities, and $2.8 million of amortization and interest accretion on our operating lease. Additionally, there was a non-cash adjustment of $0.7 million related to the impairment of unused lab equipment.
Previous wording
For the six months ended June 30, 2024, net cash used in operating activities was $38.9 million resulting from our net loss of $46.4 million, partially offset by changes in our operating assets and liabilities of $1.6 million and by non-cash charges of $5.8 million. The net changes in our operating assets and liabilities were primarily due to an increase in accounts payable of $6.6 million related to manufacturing commitments. The change was further driven by a decrease in accrued expenses of $2.3 million, an increase in prepaids and other current assets of $1.2 million, and a decrease in operating lease liabilities of $1.5 million. The $2.3 million decrease in accrued expenses was primarily due to lower research and development accruals as a result of manufacturing timing, and the payment of our annual employee incentive bonus. The non-cash charges included $4.9 million of stock-based compensation, $0.7 million of depreciation expense, $1.7 million of amortization of discounts on our marketable securities, and $1.9 million of amortization and interest accretion on our operating lease.
Current wording
For the nine months ended September 30, 2024, net cash used in operating activities was $62.9 million resulting from our net loss of $67.7 million, and changes in our operating assets and liabilities of $4.3 million, partially offset by non-cash adjustments of $9.2 million. The net changes in our operating assets and liabilities were primarily due to an increase in accounts payable of $4.9 million related to manufacturing commitments. The change was further driven by a decrease in accrued expenses of $5.8 million, an increase in prepaids and other current assets of $1.2 million, and a decrease in operating lease liabilities of $2.2 million. The $5.8 million decrease in accrued expenses was primarily due to lower research and development accruals as a result of the timing of manufacturing campaigns. The non-cash adjustments included $8.1 million of stock-based compensation, $1.1 million of depreciation expense, $2.9 million of amortization of discounts on our marketable securities, and $2.8 million of amortization and interest accretion on our operating lease.
Previous wording
For the six months ended June 30, 2025, net cash provided by investing activities was $32.1 million resulting from $39.0 million in maturities of marketable securities partially offset by $6.7 million in purchases of marketable securities and $0.2 million in purchases of property and equipment.
Current wording
For the nine months ended September 30, 2025, net cash provided by investing activities was $51.0 million resulting from $58.0 million in maturities of marketable securities partially offset by $6.7 million in purchases of marketable securities and $0.3 million in purchases of property and equipment.
Previous wording
For the six months ended June 30, 2024, net cash used in investing activities was $64.0 million resulting from $94.0 million in purchases of marketable securities partially offset by $30.0 million in maturities and sales of marketable securities.
Current wording
For the nine months ended September 30, 2024, net cash used in investing activities was $64.2 million resulting from $123.9 million in purchases of marketable securities partially offset by $60.0 million in maturities of marketable securities.
Previous wording
For the six months ended June 30, 2025, net cash provided by financing activities was $0.1 million of proceeds from the purchase of shares under employee equity plans.
Current wording
For the nine months ended September 30, 2025, net cash provided by financing activities was $108.2 million resulting from $108.1 million in proceeds from the 2025 Offering and $0.1 million of proceeds from the purchase of shares under employee equity plans, partially offset by $0.1 million in the payment of offering costs.
Previous wording
For the six months ended June 30, 2024, net cash provided by financing activities was $88.3 million resulting from $76.9 million in proceeds from the Follow-on Offering, $9.9 million in proceeds from sales under the Sales Agreement, and $1.9 million from proceeds from the purchase of shares under employee equity plans, partially offset by $0.4 million in the payment of deferred offering costs.
Current wording
For the nine months ended September 30, 2024, net cash provided by financing activities was $88.6 million resulting from $76.9 million in proceeds from the 2024 Offering, $9.9 million in proceeds from sales under the Sales Agreement, and $2.4 million from proceeds from the purchase of shares under employee equity plans, partially offset by $0.5 million in the payment of deferred offering costs.
Rearranged previous paragraphs (1)
Previous report — wording carried elsewhere
We have also initiated research efforts for additional indications, including neuromuscular diseases and neurologic disorders and have several programs in early research.