4D Molecular Therapeutics, Inc.
For the period ended Sep 30, 2025. Filed Nov 10, 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 10, 2025
FDMT$12.25+8.9%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 2025-08-11.
0000950170-25-106244
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-09-30; filed 2025-11-10.
0001193125-25-273343
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 114 previous paragraphs and 113 current paragraphs.
68 unchanged; 39 reworded; 5 added; 5 removed; 2 previous and 1 current paragraphs rearranged.
Tables are not compared: 5 previous and 5 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
In November 2025, we announced positive long-term interim results from the ongoing 4D-150 PRISM Phase 1/2 clinical trial in wet AMD. 4D-150 demonstrated consistent and durable benefit across all three patient cohorts as evidenced by maintenance of visual acuity, control of retinal anatomy and reduction of treatment burden at all time points with 1.5 to 2 years of follow-up. In addition, a consistent dose response was observed between 3E10 vg/eye, the selected Phase 3 dose, and the lower dose of 1E10 vg/eye. The Phase 3 dose achieved clinically meaningful reductions in treatment burden. No new cases of intraocular inflammation were reported during this follow-up period with up to approximately 3.5 years of follow-up.
Current report
In October 2025, we entered into a Collaboration and License Agreement with Otsuka Pharmaceutical Co., Ltd., pursuant to which we granted Otsuka exclusive rights to develop and commercialize 4D-150 for ophthalmological diseases, including wet AMD and DME, in Japan, China, Australia, and other APAC markets. Otsuka has agreed to lead all regulatory and commercialization activities in its licensed territories. We have agreed to continue to lead all Phase 3 clinical activity globally, including within the APAC region. Otsuka has agreed to provide an upfront cash payment of $85 million and certain cost sharing for global development activities. In addition, we are eligible for up to $335.5 million in potential regulatory and commercial milestone payments and tiered double-digit royalties depending on net sales in Otsuka’s licensed territories. We retain full development and commercialization rights for 4D-150 outside the APAC region, including the United States, Latin America, and Europe.
Current report
Restructuring and Other Charges
Current report
a $0.3 million decrease in payroll and personnel expenses primarily due to decreased headcount, offset by one-time severance costs; and
Current report
Other income, net, decreased by $3.0 million, or 41%, from the three months ended September 30, 2024 to the three months ended September 30, 2025. Other income, net, decreased by $5.8 million, or
Removed paragraphs (5)
Previous report
We have incurred significant operating losses and expect that our operating losses will remain relatively stable as we increase expense on Phase 3 studies for 4D-150 in wet AMD offset by reduction in our expenses on early-stage research and development, and maintain, protect and enforce our intellectual property portfolio.
Previous report
Workforce Reduction
Previous report
a $9.5 million increase in payroll and personnel expenses primarily due to increased headcount from an increase in research and development personnel; and
Previous report
General and administrative expenses increased by $3.6 million, or 17%, from the six months ended June 30, 2024 to the six months ended June 30, 2025. The increase was primarily due to:
Previous report
a $2.8 million increase in legal fees and consulting services related to public company compliance costs and an increase in expenses related to information technology systems implementations; and
Reworded paragraphs (39)
Previous wording
In January 2025, we implemented a strategic pipeline prioritization designed to prioritize the development of 4D-150 and 4D-710. Further, we will be seeking strategic alternatives, including potential partnering, for our other clinical stage product candidates: 4D-175 for the treatment of geographic atrophy, 4D-725 for the treatment of alpha-1 antitrypsin deficiency, and 4D-310 for the treatment of Fabry disease cardiomyopathy. In addition, we terminated the development of 4D-110 for the treatment of choroideremia and 4D-125 for the treatment of X-linked retinitis pigmentosa.
Current wording
In January 2025, we implemented a strategic pipeline prioritization designed to prioritize the development of 4D-150 and 4D-710. Further, we will be seeking strategic alternatives, including potential partnering, for our other clinical stage product candidates: 4D-175 for the treatment of geographic atrophy and 4D-725 for the treatment of alpha-1 antitrypsin deficiency. In addition, we terminated the development of 4D-110 for the treatment of choroideremia and 4D-125 for the treatment of X-linked retinitis pigmentosa.
Previous wording
In March 2025, we announced the first patients have been enrolled across multiple sites in the 4FRONT-1, our North American Phase 3 clinical trial evaluating 4D-150 for the treatment of wet AMD. In July 2025, we announced that initial enrollment and site activation for 4FRONT-1 have exceeded initial projections, reflecting continued strong engagement and enthusiasm from investigators and patients. We expect 52-week topline data in the first half of 2027, an acceleration of the timeline from the previous guidance to the second half of 2027, providing more than six months of expected cash runway beyond the expected data readout.
Current wording
In March 2025, we announced the first patients enrolled in 4FRONT-1, our North American Phase 3 clinical trial evaluating 4D-150 for the treatment of wet AMD. In October 2025, we announced that enrollment was exceeding initial projections with more than 200 patients enrolled, reflecting continued strong engagement and enthusiasm from investigators and patients. We expect 52-week topline data in the first half of 2027, an acceleration of the timeline from the previous guidance to the second half of 2027.
Previous wording
Additionally, our second Phase 3 trial of 4D-150 in wet AMD, 4FRONT-2, was initiated in June 2025, ahead of schedule. 4FRONT-2 is a global clinical trial and has an identical design to 4FRONT-1, except for enrolling both treatment naïve and recently diagnosed, treatment-experienced patients. We expect 52-week topline data for 4FRONT-2 in the second half of 2027, consistent with previous guidance.
Current wording
Additionally, 4FRONT-2, our second Phase 3 trial of 4D-150 in wet AMD, was initiated in June 2025, ahead of schedule. 4FRONT-2 is a global clinical trial and has an identical design to 4FRONT-1, except for enrolling both treatment naïve and recently diagnosed, treatment-experienced patients. We expect 52-week topline data for 4FRONT-2 in the second half of 2027.
Previous wording
In July 2025, we presented positive 60-week results from the 4D-150 SPECTRA clinical trial in DME where 4D-150 continued to be well tolerated with no intraocular inflammation observed at any timepoint or dose level. In addition, 4D-150 demonstrated durable and dose-dependent clinical activity with sustained gains in visual acuity and anatomic control between 3E10 vg/eye, the selected Phase 3 dose, and lower doses. The Phase 3 dose achieved clinically meaningful 78% reduction in treatment burden vs. projected on-label aflibercept 2mg Q8W. The FDA and EMA are aligned on a proposed single Phase 3 clinical trial being acceptable for regulatory submissions for 4D-150 in DME.
Current wording
In July 2025, we presented positive 60-week results from the 4D-150 SPECTRA clinical trial in DME where 4D-150 continued to be well tolerated with no intraocular inflammation observed at any timepoint or dose level. In addition, 4D-150 demonstrated durable and dose-dependent clinical activity with sustained gains in visual acuity and anatomic control between 3E10 vg/eye, the selected Phase 3 dose, and lower doses. The Phase 3 dose achieved clinically meaningful 78% reduction in treatment burden vs. projected on-label aflibercept 2mg Q8W. The FDA and EMA are aligned on a proposed single Phase 3 clinical trial being acceptable for possible future licensure for 4D-150 in DME.
Previous wording
Our second priority product candidate is 4D-710, which we believe is the first known genetic medicine to demonstrate successful delivery and expression of the CFTR transgene in the lungs of people with cystic fibrosis (CF). We believe these results will translate into durable clinical improvements in people with CF, including improved lung function and quality of life. We expect to provide an interim data update from the ongoing AEROW Phase 1 clinical trial and a program update in the fourth quarter of 2025.
Current wording
Our second priority product candidate is 4D-710, which we believe is the first known genetic medicine to demonstrate successful delivery and expression of the CFTR transgene in the lungs of people with cystic fibrosis (CF). We believe these results will translate into durable clinical improvements in people with CF, including improved lung function and quality of life. In October 2025, we announced a funding agreement with the Cystic Fibrosis Foundation ("CFF") to provide up to $11 million in additional funding, including $7.5 million in an initial tranche, which was completed in October 2025. The proceeds of this funding agreement enabled the start of the Phase 2 stage of the AEROW clinical trial, redosing, and Phase 3 readiness activities. We expect to provide an interim data update from the ongoing Phase 1 stage of the AEROW clinical trial and a program update by year end 2025.
Previous wording
In February 2024, we completed an underwritten public offering (the “2024 Offering” and, together with the 2023 Offering and the 2021 Offering (as defined below), the “Follow-on Offerings”) in which 6,586,015 shares of our common stock were sold at an offering price of $29.50 per share, as well as pre-funded warrants to purchase 3,583,476 shares of our common stock at an offering price of $29.4999 per underlying share pursuant to our effective registration statement on Form S-3. The net proceeds from the 2024 Offering were $281.2 million, after deducting underwriting discounts and commissions and other offering expenses. We also granted the underwriters the option to purchase up to 1,525,423 additional shares of common stock in connection with the offering.
Current wording
In November 2025, we completed an underwritten offering (the “2025 Offering”) in which 8,385,809 shares of our common stock were sold at an offering price of $10.51 per share, as well as pre-funded warrants to purchase 1,128,949 shares of our common stock at an offering price of $10.5099 per underlying share pursuant to an effective Registration Statement on Form S-3. The net proceeds from the 2025 Offering were approximately $93.3 million, after deducting the underwriting discounts and commissions and other estimated offering expenses.
Previous wording
Our net losses were $54.7 million and $35.0 million for the three months ended June 30, 2025 and 2024, respectively, and $102.6 million and $67.4 million for the six months ended June 30, 2025 and 2024, respectively. As of June 30, 2025, we had an accumulated deficit of $678.8 million. We do not expect positive cash flows from operations in the foreseeable future. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and our expenditures on other research and development activities.
Current wording
We have incurred significant operating losses. Our net losses were $56.9 million and $43.8 million for the three months ended September 30, 2025 and 2024, respectively, and $159.5 million and $111.2 million for the nine months ended September 30, 2025 and 2024, respectively. As of September 30, 2025, we had an accumulated deficit of $735.7 million. We do not expect positive cash flows from operations in the foreseeable future. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and our expenditures on other research and development activities.
Previous wording
We will require substantial additional funding to support our continuing operations and further the development of our product candidates. Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through the sale of equity, debt financings, or other capital sources, which could include income from collaborations, strategic partnerships, or other strategic arrangements, for the foreseeable future. Adequate funding may not be available when needed or on terms acceptable to us, or at all. Our ability to raise additional funds may be adversely impacted by potential worsening global economic conditions and the recent disruptions to, and volatility in, the credit and financial markets in the United States and worldwide resulting from conflicts in the Middle East, the lingering impact of the COVID-19 pandemic, the war in Ukraine, rising interest rates, tariffs, inflation and otherwise. If we fail to obtain necessary capital when needed on acceptable terms, or at all, it could force us to delay, limit, reduce or terminate our product development programs, commercialization efforts or other operations. Insufficient liquidity may also require us to relinquish rights to product candidates at an earlier stage of development or on less favorable terms than we would otherwise choose. We cannot assure you that we will ever be profitable or generate positive cash flow from operating activities.
Current wording
We will require substantial additional funding to support our continuing operations and further the development of our product candidates. Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through the sale of equity, debt financings, or other capital sources, which could include income from collaborations, strategic partnerships, or other strategic arrangements, for the foreseeable future. Adequate funding may not be available when needed or on terms acceptable to us, or at all. Our ability to raise additional funds may be adversely impacted by potential worsening global economic conditions and the recent disruptions to, and volatility in, the credit and financial markets in the United States and worldwide resulting from conflicts in the Middle East, the lingering impact of the COVID-19 pandemic, the war in Ukraine, rising interest rates, tariffs, inflation, government shutdowns and otherwise. If we fail to obtain necessary capital when needed on acceptable terms, or at all, it could force us to delay, limit, reduce or terminate our product development programs, commercialization efforts or other operations. Insufficient liquidity may also require us to relinquish rights to product candidates at an earlier stage of development or on less favorable terms than we would otherwise choose. We cannot assure you that we will ever be profitable or generate positive cash flow from operating activities.
Previous wording
On July 2, 2025, we announced a workforce reduction of approximately 25% of current and planned roles in July 2025, primarily in the areas supporting early-stage research and development and support functions. In connection with the workforce reduction, we estimate that we will pay cash expenses of approximately $3 million, including severance, benefits, and related termination costs, which will be paid primarily in the third quarter of 2025. The workforce reduction is expected to provide annual cash compensation cost savings of approximately $15 million and offsets additional expenses expected based on the accelerated timelines for the 4FRONT clinical trials and BLA preparation. As of June 30, 2025, we had cash, cash equivalents and marketable securities of $417.0 million, which we believe is sufficient to support planned expenses to deliver 52-week topline data from 4FRONT-1 and 4FRONT-2 Phase 3 clinical trials and Biologics License Application (“BLA”) preparation for 4D-150 in wet AMD, continue Phase 1/2 and pre-Phase 3 planning activities for 4D-150 in diabetic macular edema and continue ongoing Phase 1/2 development of 4D-710 in cystic fibrosis.
Current wording
On July 2, 2025, we announced a workforce reduction of approximately 25% of current and planned roles, primarily in the areas supporting early-stage research and development and support functions to implement a strategic pipeline prioritization to focus on the development of 4D-150 and 4D-710. In connection with the work force reduction, the Company incurred total expense of $3.2 million and paid cash expenses of approximately $3.0 million including severance, benefits and related termination costs in the third quarter of 2025. There are no future payments in connection with the workforce reduction. The workforce reduction is expected to provide annual cash compensation cost savings of approximately $15 million and offsets additional expenses expected based on the accelerated timelines for the 4FRONT clinical trials and BLA filing.
Previous wording
Our research and development expenses primarily consist of costs incurred for the discovery and preclinical and clinical development of our product candidates. These expenses include salaries and personnel-related costs, including stock-based compensation of our scientific personnel performing research and development activities; laboratory supplies; research materials; fees paid to CROs to execute preclinical studies and clinical trials; fees paid to CMOs to manufacture materials for preclinical studies and clinical trials; fees related to obtaining technology licenses; consulting costs; costs related to seeking regulatory approval of our product candidates; and allocated facility-related costs, information technology costs, depreciation expense, and other overhead.
Current wording
Our research and development expenses primarily consist of costs incurred for the discovery and preclinical and clinical development of our product candidates. These expenses include salaries and personnel-related costs, including stock-based compensation of our clinical, medical, chemistry, manufacturing and controls and scientific personnel performing research and development activities; laboratory supplies; research materials; fees paid to CROs to execute preclinical studies and clinical trials; fees paid to CMOs to manufacture materials for preclinical studies and clinical trials; fees related to obtaining technology licenses; consulting costs; costs related to seeking regulatory approval of our product candidates; and allocated facility-related costs, information technology costs, depreciation expense, and other overhead.
Previous wording
At this time, we cannot reasonably estimate or know the nature, timing or estimated costs of the efforts that will be necessary to complete the development of, and obtain regulatory approval for, any of our product candidates. However, we expect our overall research and development expenses to remain relatively stable in the near term as we leverage prior investments and streamline operations to offset for 4D-150 Phase 3 studies. The process of conducting the necessary clinical research to obtain regulatory approval is costly and time-consuming, and the successful development of our product candidates is highly uncertain. See the section titled “Risk Factors” for additional risks regarding regulatory development and approval.
Current wording
At this time, we cannot reasonably estimate or know the nature, timing or estimated costs of the efforts that will be necessary to complete the development of, and obtain regulatory approval for, any of our product candidates. However, we expect our overall research and development expenses to increase in the near term primarily for 4D-150 Phase 3 studies in wet AMD and DME. The process of conducting the necessary clinical research to obtain regulatory approval is costly and time-consuming, and the successful development of our product candidates is highly uncertain. See the section titled “Risk Factors” for additional risks regarding regulatory development and approval.
Previous wording
We expect our general and administrative expenses to remain relatively stable or potentially decline in the near term as a result of operational streamlining while maintaining patents for our product candidates, consulting, legal and accounting services associated with maintaining compliance with stock exchange listing and requirements of the SEC, investor relations costs, director and officer insurance premiums, and other costs associated with being a public company.
Current wording
We expect our general and administrative expenses to remain relatively stable in the near term as a result of operational streamlining while maintaining patents for our product candidates, consulting, legal and accounting services associated with maintaining compliance with stock exchange listing and requirements of the SEC, investor relations costs, director and officer insurance premiums, and other costs associated with being a public company.
Previous wording
Comparison of the Three and Six Months Ended June 30, 2025 and 2024
Current wording
Comparison of the Three and Nine Months Ended September 30, 2025 and 2024
Previous wording
Revenue increased by an immaterial amount from the three months ended June 30, 2024 to the three months ended June 30, 2025, and decreased by an immaterial amount from the six months ended June 30, 2024 to the six months ended June 30, 2025. Revenue for both periods was from CFF.
Current wording
Revenue increased by $0.1 million from the three months ended September 30, 2024 to the three months ended September 30, 2025, and increased by $0.1 million from the nine months ended September 30, 2024 to the nine months ended September 30, 2025. Revenue for both periods was from CFF.
Previous wording
Research and development expenses increased by $16.1 million, or 51%, from the three months ended June 30, 2024 to the three months ended June 30, 2025. The increase was primarily due to the following:
Current wording
Research and development expenses increased by $10.9 million, or 28%, from the three months ended September 30, 2024 to the three months ended September 30, 2025. The increase was primarily due to the following:
Previous wording
a $9.3 million increase in clinical trial costs mainly due to increased clinical trial activity for our product candidates, primarily 4D-150;
Current wording
a $9.6 million increase in clinical trial costs mainly due to increased clinical trial activity for our product candidates, primarily 4D-150;
Previous wording
a $2.1 million increase in facilities and other research and development expenses primarily due to higher rent, software and information technology expenses.
Current wording
a $1.6 million increase in facilities and other research and development expenses primarily due to higher rent and increased clinical trial activity for our product candidates.
Previous wording
Research and development expenses increased by $28.9 million, or 48%, from the six months ended June 30, 2024 to the six months ended June 30, 2025. The increase was primarily due to the following:
Current wording
Research and development expenses increased by $39.9 million, or 41%, from the nine months ended September 30, 2024 to the nine months ended September 30, 2025. The increase was primarily due to the following:
Previous wording
a $13.8 million increase in clinical trial costs mainly due to increased clinical trial activity for our product candidates, primarily 4D-150;
Current wording
a $23.2 million increase in clinical trial costs mainly due to increased clinical trial activity for our product candidates, primarily 4D-150;
Previous wording
a $4.7 million increase in payroll and personnel expenses primarily due to increased headcount of research and development personnel; and
Current wording
a $9.3 million increase in payroll and personnel expenses primarily due to increased headcount of research and development personnel and one-time severance costs; and
Previous wording
a $5.6 million increase in facilities and other research and development expenses primarily due to higher rent, consulting services, software and information technology expenses.
Current wording
a $7.4 million increase in facilities and other research and development expenses primarily due to higher rent and increased clinical trial activity for our product candidates.
Previous wording
an $0.8 million increase in payroll and personnel expenses primarily due to increased headcount of general and administrative personnel.
Current wording
General and administrative expenses decreased by $0.8 million, or 6%, from the three months ended September 30, 2024 to the three months ended September 30, 2025. The decrease was primarily due to decreased headcount of general and administrative personnel.
Previous wording
General and administrative expenses increased by $0.9 million, or 9%, from the three months ended June 30, 2024 to the three months ended June 30, 2025. The increase was primarily due to an increase in legal fees and consulting services related to public company compliance costs and increase in expenses related to information technology systems implementations.
Current wording
General and administrative expenses increased by $2.7 million, or 8%, from the nine months ended September 30, 2024 to the nine months ended September 30, 2025. The increase was primarily due to an increase in legal fees and consulting services.
Previous wording
Other income, net, decreased by $2.7 million, or 36%, from the three months ended June 30, 2024 to the three months ended June 30, 2025. It decreased by $2.8 million, or 21%, from the six months ended June 30, 2024 to the six months ended June 30, 2025. The decrease is attributable to lower balances of cash equivalents and marketable securities.
Current wording
28%, from the nine months ended September 30, 2024 to the nine months ended September 30, 2025. The decrease is attributable to lower balances of cash equivalents and marketable securities.
Previous wording
In February 2024, we completed the 2024 Offering in which 6,586,015 shares of our common stock were sold at an offering price of $29.50 per share, as well as pre-funded warrants to purchase 3,583,476 shares of our common stock at an offering price of $29.4999 per underlying share. The net proceeds from the 2024 Offering were $281.2 million, after deducting underwriting discounts and commissions and other offering expenses. We also granted the underwriters the option to purchase up to 1,525,423 additional shares of common stock in connection with the offering.
Current wording
In February 2024, we completed the 2024 Offering in which 6,586,015 shares of our common stock were sold at an offering price of $29.50 per share, as well as pre-funded warrants to purchase 3,583,476 shares of our common stock at an offering price of $29.4999 per underlying share. The net proceeds from the 2024 Offering were $281.2 million, after deducting underwriting discounts and commissions and other offering expenses. We also granted the underwriters the option to purchase up to 1,525,423 additional shares of common stock in connection with the offering. In March 2024, the underwriters exercised their option and purchased 1,259,299 additional shares of common stock resulting in net proceeds of $34.9 million, after deducting underwriting discounts and commissions.
Previous wording
In June 2024, we entered into a Sales Agreement (the “Leerink Sales Agreement”) with Leerink Partners LLC (“Leerink”) as sales agent to sell shares of our common stock, from time to time, with aggregate gross sales proceeds of up to $250.0 million pursuant to a Registration Statement on Form S-3 that we filed with the SEC in February 2024 as an “at-the-market” offering under the Securities Act. For the six months ended June 30, 2025, no shares were sold pursuant to the Leerink Sales Agreement.
Current wording
In June 2024, we entered into a Sales Agreement (the “Leerink Sales Agreement”) with Leerink Partners LLC (“Leerink”) as sales agent to sell shares of our common stock, from time to time, with aggregate gross sales proceeds of up to $250.0 million pursuant to a Registration Statement on Form S-3 that we filed with the SEC in February 2024 as an “at-the-market” offering under the Securities Act. For the nine months ended September 30, 2025, no shares were sold pursuant to the Leerink Sales Agreement.
Previous wording
As of June 30, 2025, we had cash, cash equivalents and marketable securities of $417.0 million.
Current wording
As of September 30, 2025, we had cash, cash equivalents and marketable securities of $372.2 million.
Previous wording
We have experienced recurring net losses and had an accumulated deficit of $678.8 million as of June 30, 2025. Our transition to profitability is dependent upon the successful development, approval and commercialization of our product candidates and those of our collaboration partners and achieving a level of revenue adequate to support our cost structure. We expect to continue to incur losses for the foreseeable future.
Current wording
We have experienced recurring net losses and had an accumulated deficit of $735.7 million as of September 30, 2025. Our transition to profitability is dependent upon the successful development, approval and commercialization of our product candidates and those of our collaboration partners and achieving a level of revenue adequate to support our cost structure. We expect to continue to incur losses for the foreseeable future.
Previous wording
We expect that our research and development and general and administrative expenses will continue to increase for the foreseeable future. Additionally, we expect our capital expenditures will increase significantly in the future for costs associated with building commercial manufacturing capacity. As a result, we will need significant additional capital to fund our operations, which we may obtain through one or more equity offerings, debt financings or other third-party funding, including potential strategic alliances and licensing or collaboration arrangements.
Current wording
We expect that our overall research and development and general and administrative expenses will increase. As a result, we will need significant additional capital to fund our operations, which we may obtain through one or more equity offerings, debt financings or other third-party funding, the Otsuka Collaboration and Licensing Agreement, and additional potential strategic alliances and licensing or collaboration arrangements.
Previous wording
expanding our manufacturing facilities and working with our contract manufacturers to scale up the manufacturing processes for our product candidates;
Current wording
working with our contract manufacturers to scale up the manufacturing processes for our product candidates;
Previous wording
We do not have any committed external sources of funds. Accordingly, we will be required to obtain further funding through public or private equity offerings, debt financings, collaborations and licensing arrangements or other sources to complete the clinical development for the product candidates for treatment of wet AMD, DME, GA, cystic fibrosis lung disease, alpha-1 antitrypsin deficiency lung disease, Fabry disease cardiomyopathy or any other indication we may pursue. If we raise additional funds by issuing equity securities, our stockholders may experience dilution. Any future debt financing into which we enter would result in fixed payment obligations and may involve agreements that include grants of security interests on our assets and restrictive covenants that limit our ability to take specific actions, such as incurring additional debt, making capital expenditures, granting liens over our assets, redeeming stock or declaring dividends, that could adversely impact our ability to conduct our business. Any debt financing or additional equity that we raise may contain terms that could adversely affect our common stockholders. Further, additional funds may not be available when we need them, on terms that are acceptable to us, or at all. Our ability to raise additional capital may be adversely impacted by potential worsening global economic conditions and the recent disruptions to and volatility in the credit and financial markets in the United States and worldwide resulting from the war in Ukraine, conflicts in the Middle East, any expansion of these conflicts, rising interest rates and inflation, natural disasters and pandemics.
Current wording
We have limited committed external sources of funds. Accordingly, we will be required to obtain further funding through public or private equity offerings, debt financings, collaborations and licensing arrangements or other sources to complete the clinical development for the product candidates for treatment of wet AMD, DME, GA, cystic fibrosis lung disease, alpha-1 antitrypsin deficiency lung disease or any other indication we may pursue. If we raise additional funds by issuing equity securities, our stockholders may experience dilution. Any future debt financing into which we enter would result in fixed payment obligations and may involve agreements that include grants of security interests on our assets and restrictive covenants that limit our ability to take specific actions, such as incurring additional debt, making capital expenditures, granting liens over our assets, redeeming stock or declaring dividends, that could adversely impact our ability to conduct our business. Any debt financing or additional equity that we raise may contain terms that could adversely affect our common stockholders. Further, additional funds may not be available when we need them, on terms that are acceptable to us, or at all. Our ability to raise additional capital may be adversely impacted by potential worsening global economic conditions and the recent disruptions to and volatility in the credit and financial markets in the United States and worldwide resulting from the war in Ukraine, conflicts in the Middle East, any expansion of these conflicts, rising interest rates and inflation, natural disasters and pandemics.
Previous wording
If we are unable to obtain additional funding, we expect to delay, reduce or eliminate some or all of our research and development programs, product portfolio expansion or investment in internal manufacturing capabilities, which could adversely affect our business. If we raise additional funds through collaborations or marketing, distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to future revenue streams or product candidates or grant licenses on terms that may not be favorable to us.
Current wording
If we are unable to obtain additional funding, we expect to delay, reduce or eliminate some or all of our research and development programs, product portfolio expansion or investment in manufacturing capabilities, which could adversely affect our business. If we raise additional funds through collaborations or marketing, distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to future revenue streams or product candidates or grant licenses on terms that may not be favorable to us.
Previous wording
Net cash used in operating activities was $59.3 million for the six months ended June 30, 2024. This was primarily due to the net loss of $67.4 million, adjusted for noncash charges of $12.2 million and net changes in operating assets and liabilities of $4.2 million. Noncash charges included $12.0 million of stock-based compensation expense, $2.4 million of depreciation and amortization and $0.9 million for the amortization of operating lease right-of-use assets, offset by $3.1 million net amortization/accretion of premium/discount on marketable securities. Net changes in operating assets and liabilities included a $1.0 million decrease in operating lease liabilities, a $0.1 million decrease in accounts payables, a $2.3 million increase in prepaid expenses and other current assets and a $1.6 million increase in other assets, offset by a $0.8 million increase in accrued and other liabilities and a $0.1 million increase in deferred revenue.
Current wording
Net cash used in operating activities was $137.6 million for the nine months ended September 30, 2025. This was primarily due to the net loss of $159.5 million, adjusted for noncash charges of $18.5 million and net changes in operating assets and liabilities of $3.4 million. Noncash charges included $16.5 million of stock-based compensation expense, $3.5 million of depreciation and amortization and $2.2 million for the amortization of operating lease right-of-use assets, offset by $3.6 million net accretion of discount on marketable securities and $0.1 million change in fair value of derivative liability. Net changes in operating assets and liabilities included increases of $5.8 million in accrued and other liabilities and $2.4 million in accounts payable and a $2.7 million decrease in prepaid expenses and other current assets, offset by decreases of $2.3 million in operating lease liabilities and $0.1 million in deferred revenue and a $5.1 million increase in other assets.
Previous wording
Net cash used in operating activities was $91.1 million for the six months ended June 30, 2025. This was primarily due to the net loss of $102.6 million, adjusted for noncash charges of $13.1 million and net changes in operating assets and liabilities of $1.6 million. Noncash charges included $11.9 million of stock-based compensation expense, $2.2 million of depreciation and amortization and $1.4 million for the amortization of operating lease right-of-use assets, offset by $2.4 million net accretion of discount on marketable securities. Net changes in operating assets and liabilities included a $1.5 million decrease in operating lease liabilities and a $5.1 million increase in other assets, offset by a $4.3 million increase in accounts payable and a $0.7 million increase in accrued and other liabilities.
Current wording
Net cash used in operating activities was $88.7 million for the nine months ended September 30, 2024. This was primarily due to the net loss of $111.2 million, adjusted for noncash charges of $18.2 million and net changes in operating assets and liabilities of $4.3 million. Noncash charges included $18.5 million of stock-based compensation expense, $3.6 million of depreciation and amortization and $1.4 million for the amortization of operating lease right-of-use assets, offset by $5.3 million net accretion of discount on marketable securities. Net changes in operating assets and liabilities included an increase of $8.9 million in accrued and other liabilities which were offset by decreases of $1.4 million in operating lease liabilities and $0.6 million in accounts payable and increases of $2.2 million in other assets and $0.5 million in prepaid expenses and other current assets.
Previous wording
Net cash provided by investing activities was $18.1 million for the six months ended June 30, 2025. This was due to $268.3 million in maturities of marketable securities, offset by purchases of marketable securities of $249.5 million and purchases of property and equipment of $0.7 million.
Current wording
Net cash provided by investing activities was $35.9 million for the nine months ended September 30, 2025. This was due to $301.2 million in maturities of marketable securities, offset by purchases of marketable securities of $264.7 million and purchases of property and equipment of $0.6 million.
Previous wording
Net cash used in investing activities was $336.8 million for the six months ended June 30, 2024. This was due to purchases of marketable securities of $374.1 million and purchases of property and equipment of $1.0 million, offset by $38.3 million in maturities of marketable securities.
Current wording
Net cash used in investing activities was $309.5 million for the nine months ended September 30, 2024. This was due to purchases of marketable securities of $409.9 million and purchases of property and equipment of $2.8 million, offset by $103.2 million in maturities of marketable securities.
Previous wording
Net cash provided by financing activities was $0.9 million for the six months ended June 30, 2025 which was due to proceeds from the issuance of common stock from ESPP purchases of $0.9 million.
Current wording
Net cash provided by financing activities was $1.0 million for the nine months ended September 30, 2025 which was due to proceeds from the issuance of common stock from ESPP purchases of $0.9 million and proceeds from the issuance of common stock from the exercise of stock options and warrants of $0.1 million.
Previous wording
Net cash provided by financing activities was $336.1 million for the six months ended June 30, 2024. This was primarily due to proceeds from the issuance of common stock upon public offering, net of issuance costs, of $316.1 million, proceeds from the issuance of common stock from the 2022 ATM Offering Program of $15.3 million, proceeds from the issuance of common stock from the exercise of stock options and warrants of $4.0 million and proceeds from the issuance of common stock from ESPP purchases of $0.7 million.
Current wording
Net cash provided by financing activities was $336.6 million for the nine months ended September 30, 2024. This was primarily due to proceeds from the issuance of common stock upon public offering, net of issuance costs, of $316.1 million, proceeds from the issuance of common stock from the 2022 ATM Offering Program of $15.3 million, proceeds from the issuance of common stock from the exercise of stock options and warrants of $4.5 million and proceeds from the issuance of common stock from purchases from the Company’s 2020 Employee Stock Purchase Plan (“ESPP”) of $0.7 million.
Previous wording
During the three and six months ended June 30, 2025, there were no changes to our critical accounting policies and significant judgments and estimates as disclosed in our Annual Report on Form 10-K for the year ended December 31, 2024.
Current wording
During the three and nine months ended September 30, 2025, there were no changes to our critical accounting policies and significant judgments and estimates as disclosed in our Annual Report on Form 10-K for the year ended December 31, 2024.
Rearranged current paragraphs (1)
Current report — wording carried from elsewhere
receiving milestone, royalty or other payments under any current or future collaboration or license agreements;
Rearranged previous paragraphs (2)
Previous report — wording carried elsewhere
In March 2024, the underwriters exercised their option to purchase 1,259,299 additional shares of common stock resulting in net proceeds of $34.9 million, after deducting underwriting discounts and commissions.
Previous report — wording carried elsewhere
In March 2024, the underwriters exercised their option and purchased 1,259,299 additional shares of common stock resulting in net proceeds of $34.9 million, after deducting underwriting discounts and commissions.