11/06/2025 | Press release | Distributed by Public on 11/06/2025 07:35
MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our unaudited condensed consolidated financial statements and related notes appearing at the beginning of this Quarterly Report on Form 10-Q and our audited consolidated financial statements and related notes appearing at the end of our Annual Report on Form 10-K for the year ended December 31, 2024. Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report on Form 10-Q, including information with respect to our plans and strategy for our business, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including those factors set forth in the "Risk Factors" section of this Quarterly Report on Form 10-Q, our actual results could differ materially from the results described in, or implied by, the forward-looking statements contained in the following discussion and analysis.
Overview
We are a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases. We have assembled a portfolio of clinical and preclinical product candidates that aim to modify fundamental biological pathways associated with the formation and function of red blood cells, specifically heme biosynthesis and iron homeostasis. Our current pipeline includes bitopertin for the treatment of erythropoietic porphyrias, or EPs, including erythropoietic protoporphyria, or EPP, and X-linked protoporphyria, or XLP, and Diamond-Blackfan Anemia, or DBA; DISC-0974 for the treatment of anemia of myelofibrosis, or MF, anemia of inflammatory bowel disease, or IBD, and anemia of chronic kidney disease, or CKD; and DISC-3405 for the treatment of polycythemia vera, or PV, sickle cell disease, or SCD, and other hematologic disorders. In addition, our preclinical programs include DISC-0998 for the treatment of anemia associated with inflammatory diseases. Our approach to product candidate development leverages well-understood molecular mechanisms that have been validated in humans. We believe that each of our product candidates, if approved, has the potential to improve the lives of patients suffering from hematologic diseases.
Bitopertin is the lead product candidate in our heme biosynthesis modulation portfolio. Bitopertin was previously evaluated by F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc., or collectively, Roche, in a comprehensive clinical program in over 4,000 individuals in other indications which demonstrated the activity of bitopertin as a glycine transporter 1, or GlyT1, inhibitor and its effect on heme biosynthesis. We are initially developing bitopertin for the treatment of EPs, including EPP and XLP, which are part of a group of severe diseases, known as porphyrias, caused by defects in the heme biosynthesis pathway that cause an accumulation of toxic metabolites referred to as porphyrins, resulting in skin hypersensitivity to sunlight and some types of artificial light. In June and December 2023, we presented interim data from BEACON, a Phase 2 open-label, parallel-dose clinical trial of bitopertin in EPP and XLP patients conducted at sites in Australia. In April 2024, we presented topline data from AURORA, a Phase 2, randomized, double-blind, placebo-controlled clinical trial of bitopertin in EPP patients conducted at sites in the United States. Additional analyses of the BEACON and AURORA trials were presented in June 2024 and in December 2024. In both trials, bitopertin significantly reduced the toxic metabolite, protoporphyrin IX, or PPIX, and was associated with improvements in measures of time spent in sunlight and quality of life, demonstrating a clear association between PPIX reduction and clinical endpoints. In addition, bitopertin was generally well-tolerated. All participants who complete AURORA or BEACON are eligible to participate in HELIOS, an ongoing open-label, long-term extension study of bitopertin in EPP and XLP. In June 2025, we presented interim data from HELIOS, in which longer term treatment of bitopertin was associated with sustained reductions in PPIX, as well as improvements in quality of life, and improved liver biomarkers. In our end-of-Phase 2 meeting, the U.S. Food & Drug Administration, or the FDA, agreed with the potential for reduction of PPIX to serve as a surrogate endpoint to support a potential accelerated approval of bitopertin in EPP and XLP. In our Type C meeting with the FDA in December 2024, we aligned with the FDA on the design of APOLLO, a randomized, double-blind, placebo-controlled, clinical trial of bitopertin in EPP and XLP patients that in the setting of an accelerated approval would serve as a post-marketing confirmatory clinical trial. APOLLO is also intended to serve as a registrational trial with respect to any potential future marketing applications for bitopertin in EPP and XLP in jurisdictions outside the United States. We initiated the APOLLO trial in May 2025, and on September 29, 2025 we submitted an NDA for accelerated approval of bitopertin in EPP and XLP in the United States based on our existing data. In October 2025, we were awarded a Commissioner's National Priority Voucher, or CNPV, from the FDA for bitopertin in EPP and XLP. The CNPV program, announced in June 2025, is designed to accelerate the development and review of certain drugs aligned with US national health priorities by offering the opportunity to reduce drug application review times to one to two months from NDA acceptance. The voucher entitles the recipient to certain benefits, including enhanced communications and rolling review to allow for shortened review time. We plan to accelerate activities to support a potential US approval and launch of bitopertin for EPP and XLP in late 2025 or early 2026 based on the accelerated review timeline associated with receipt of the CNPV. We have also entered into a collaborative research and development agreement with the National Institutes of Health, or NIH, to conduct a clinical trial of bitopertin in DBA, which began in July 2023. We are planning additional trials of bitopertin in other indications.
DISC-0974 is the lead product candidate in our iron homeostasis portfolio and was licensed from AbbVie Deutschland GmbH & Co. KG, or AbbVie. DISC-0974 is designed to suppress hepcidin production and increase serum iron levels. We completed a Phase 1 clinical trial in healthy volunteers in the U.S. in June 2022 with results showing an acceptable tolerability profile and evidence of target engagement, iron mobilization and augmented erythropoiesis. We initiated a Phase 1b/2 clinical trial in June 2022 in patients with anemia of MF, and initiated a separate Phase 1b/2 clinical trial in February 2023 in patients with non-dialysis dependent CKD
and anemia. We presented interim data from both of these trials in December 2023 as well as additional interim data for anemia of MF in June 2024 and for non-dialysis dependent CKD and anemia in October 2024. This additional interim data included safety data and changes in hepcidin, iron, and hemoglobin levels for additional patients, as well as longer follow-up. In December 2024, we presented additional analyses of the Phase 1b study in anemia of MF, and in June 2025 we presented longer term data from the continuation phase of this study. We initiated RALLY-MF, the open-label Phase 2 portion of this clinical trial, in December 2024 and we expect to report initial data from this Phase 2 trial at the American Society of Hematology Annual Meeting in December 2025. Topline data from this trial is expected in 2026, which, if positive, is expected to support discussions with regulatory agencies on the potential regulatory path for DISC-0974 in anemia of MF. In October 2025, we announced that our recently completed Phase 1b, double-blind, placebo-controlled study of DISC-0974 in non-dialysis dependent CKD and anemia demonstrated engagement of mechanism with variable effects on hemoglobin. In this trial, DISC-0974 was generally well-tolerated and associated with substantial decreases in hepcidin, increases in iron, and improvements in markers of erythropoiesis. Meaningful hemoglobin increases observed in only a subset of patients were in part driven by those with higher baseline erythropoietin levels. We expect to report full data from this trial at the American Society of Nephrology (ASN) Kidney Week in November 2025, and we are assessing options for our DISC-0974 non-dialysis dependent CKD and anemia program based on full analysis of this data. We are also planning additional trials of DISC-0974 in other anemias of inflammation. For example, we plan to initiate a Phase 2 clinical trial of DISC-0974 in patients with IBD and anemia in the first quarter of 2026, and we are planning exploratory studies in additional patient populations with anemia of chronic disease. We are also developing a preclinical anti-hemojuvelin, or HJV, monoclonal antibody, DISC-0998, which also targets hepcidin suppression and was licensed from AbbVie. DISC-0998 is designed to increase serum iron levels and has an extended serum half-life as compared to DISC-0974. We believe this profile may be desirable in certain subsets of patients with anemia associated with inflammatory diseases.
Lastly, we are developing DISC-3405, a monoclonal antibody against Transmembrane Serine Protease 6, or TMPRSS6, that we licensed from Mabwell Therapeutics, Inc., or Mabwell. DISC-3405 is part of our iron homeostasis portfolio and is designed to induce hepcidin production and reduce serum iron levels. We initiated a Phase 1 clinical trial of DISC-3405 in healthy adult volunteers in October 2023 and presented interim data from the single-ascending dose, or SAD, portion of this clinical trial in June 2024. We presented data from the multiple ascending dose, or MAD, portion of this Phase 1 healthy volunteer study of DISC-3405 in December 2024, and presented updated data from both the SAD and MAD portions of this clinical trial in June 2025. We are developing DISC-3405 for the treatment of PV, SCD, and other hematologic disorders. We initiated a Phase 2 clinical trial of DISC-3405 in PV in the first half of 2025, with initial data expected in 2026. We also initiated a Phase 1b clinical trial of DISC-3405 in patients with SCD in October 2025, with initial data expected in 2026, and plan to explore the role of therapeutic iron restriction in other indications.
As of September 30, 2025, we had cash, cash equivalents and marketable securities of $615.9 million. In October 2025, we completed an underwritten public offering of shares of our common stock and pre-funded warrants for net proceeds of approximately $210.9 million, after deducting estimated offering expenses payable by us. We believe that our cash, cash equivalents and marketable securities, including the net proceeds of our October 2025 underwritten public offering, will be sufficient to fund our current operating and capital expenditure plans and our debt service obligations into 2029, without taking into account any potential net cash inflows from bitopertin or any other marketed product, if approved during such period. We have based these estimates on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. See "Liquidity and Capital Resources"for additional details.
Financial Operations Overview
Revenue
We have not generated any revenue since our inception and do not expect to generate any revenue from the sale of products in the near future, if at all. If our development efforts are successful and result in commercialization of one or more product candidates or if we enter into collaboration or license agreements with third parties, we may generate revenue in the future from product sales, payments from such collaboration or license agreements or a combination thereof.
Operating Expenses
Research and Development Expenses
Research and development expenses consist primarily of costs incurred in connection with the research and development of our product candidates. These expenses include:
We expense research and development costs as incurred. Costs incurred for external development activities are recognized based on an evaluation of the progress to completion of specific tasks using information provided to us by our vendors. Payments for these activities are based on the terms of the individual agreements, which may differ from the pattern of costs incurred, and may be reflected in our condensed consolidated financial statements as prepaid or accrued expenses. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are recorded as prepaid expenses and expensed as the related goods are delivered or the services are performed or when it is no longer expected that the goods will be delivered or the services rendered.
We typically use our employee and infrastructure resources across product candidates and development programs. We track external development costs by product candidate or development program, but we do not allocate personnel costs or other internal costs to specific product candidates or development programs.
We expect that our research and development expenses will increase substantially as we advance our programs into and through clinical development. At this time, we cannot accurately estimate or know the nature, timing and costs of the efforts that will be necessary to complete the preclinical and clinical development of any product candidates we may develop. A change in the outcome of any number of variables with respect to product candidates we may develop could significantly change the costs and timing associated with the development of that product candidate. We may never succeed in obtaining regulatory approval for any product candidates we may develop. The successful development of any product candidate is highly uncertain. This is due to the numerous risks and uncertainties associated with product development, including the following:
Selling, General and Administrative Expenses
Selling, general and administrative expenses consist primarily of salaries and related costs for personnel in executive, finance, commercial, corporate and business development, and administrative functions. Selling, general and administrative expenses also include legal fees relating to patent and corporate matters, including noncapitalizable transaction costs; professional fees for accounting, auditing, tax compliance and administrative consulting services; investor and public relations expenses; commercial planning and market research; director and officer insurance costs and other insurance costs; and facility related expenses including maintenance and allocated expenses for rent and other operating costs.
We anticipate that our selling, general and administrative expenses will increase substantially in the future as we increase our headcount to support our continued research and development and potential commercialization activities, including establishing a sales, marketing and distribution infrastructure to commercialize any product candidates for which we may obtain marketing approval.
Other Income (Expense), Net
Interest Income
Interest income primarily consists of interest earned on cash equivalents, consisting of money market funds, U.S. treasury securities and certificates of deposit, as well as marketable securities, consisting of U.S. treasury securities, U.S. government agency securities and corporate debt securities.
Interest Expense
Interest expense primarily consists of amortization of debt issuance costs and discount and interest expense under the Hercules Loan Agreement.
Results of Operations
Comparison of the Three and Nine Months Ended September 30, 2025 and 2024
The following table summarizes our results of operations for the three and nine months ended September 30, 2025 and 2024 (in thousands):
|
Three months ended September 30, |
Nine months ended September 30, |
|||||||||||||||||||||||
|
2025 |
2024 |
Change |
2025 |
2024 |
Change |
|||||||||||||||||||
|
Operating expenses: |
||||||||||||||||||||||||
|
Research and development |
$ |
50,334 |
$ |
24,685 |
$ |
25,649 |
$ |
124,416 |
$ |
71,874 |
$ |
52,542 |
||||||||||||
|
Selling, general and administrative |
17,362 |
8,171 |
9,191 |
44,636 |
23,296 |
21,340 |
||||||||||||||||||
|
Total operating expenses |
67,696 |
32,856 |
34,840 |
169,052 |
95,170 |
73,882 |
||||||||||||||||||
|
Loss from operations |
(67,696 |
) |
(32,856 |
) |
(34,840 |
) |
(169,052 |
) |
(95,170 |
) |
(73,882 |
) |
||||||||||||
|
Other income (expense), net: |
||||||||||||||||||||||||
|
Interest income |
6,412 |
6,362 |
50 |
20,349 |
15,454 |
4,895 |
||||||||||||||||||
|
Interest expense |
(917 |
) |
- |
(917 |
) |
(2,724 |
) |
- |
(2,724 |
) |
||||||||||||||
|
Other income (expense) |
(18 |
) |
9 |
(27 |
) |
47 |
(5 |
) |
52 |
|||||||||||||||
|
Total other income (expense), net |
5,477 |
6,371 |
(894 |
) |
17,672 |
15,449 |
2,223 |
|||||||||||||||||
|
Loss before income taxes |
(62,219 |
) |
(26,485 |
) |
(35,734 |
) |
(151,380 |
) |
(79,721 |
) |
(71,659 |
) |
||||||||||||
|
Income tax expense |
(102 |
) |
(114 |
) |
12 |
(273 |
) |
(179 |
) |
(94 |
) |
|||||||||||||
|
Net loss |
$ |
(62,321 |
) |
$ |
(26,599 |
) |
$ |
(35,722 |
) |
$ |
(151,653 |
) |
$ |
(79,900 |
) |
$ |
(71,753 |
) |
||||||
Research and Development Expenses
The following table summarizes our research and development expenses for the three and nine months ended September 30, 2025 and 2024 (in thousands):
|
Three months ended September 30, |
Nine months ended September 30, |
|||||||||||||||||||||||
|
2025 |
2024 |
Change |
2025 |
2024 |
Change |
|||||||||||||||||||
|
Bitopertin |
$ |
11,833 |
$ |
7,261 |
$ |
4,572 |
$ |
43,954 |
$ |
20,742 |
$ |
23,212 |
||||||||||||
|
DISC-0974 |
$ |
7,369 |
$ |
5,671 |
1,698 |
$ |
18,105 |
$ |
12,731 |
5,374 |
||||||||||||||
|
DISC-3405 |
$ |
11,910 |
$ |
3,254 |
8,656 |
$ |
15,486 |
$ |
11,823 |
3,663 |
||||||||||||||
|
Other research programs and expenses |
$ |
5,386 |
$ |
1,705 |
3,681 |
$ |
13,491 |
$ |
7,062 |
6,429 |
||||||||||||||
|
Personnel-related (including equity-based compensation) |
$ |
13,836 |
$ |
6,794 |
7,042 |
$ |
33,380 |
$ |
19,516 |
13,864 |
||||||||||||||
|
Total research and development expenses |
$ |
50,334 |
$ |
24,685 |
$ |
25,649 |
$ |
124,416 |
$ |
71,874 |
$ |
52,542 |
||||||||||||
Research and development expenses were $50.3 million for the three months ended September 30, 2025, compared to $24.7 million for the three months ended September 30, 2024. The increase of $25.6 million was primarily due to an increase of $8.7 million in DISC-3405 development expenses driven by a $10.0 million milestone payment triggered by the first administration to a patient in a Phase 2 clinical trial. Personnel-related costs increased $7.0 million related to higher research and development headcount, including an increase of $2.7 million in stock-based compensation driven by awards granted under our equity compensation plans. There was also a $4.6 million increase in bitopertin development expense reflecting ongoing costs associated with both the APOLLO
trial and the HELIOS long-term extension trial. Other research programs and expenses increased $3.7 million related to expanded research initiatives and ongoing program support. Additionally, DISC-0974 development expenses increased $1.7 million primarily due to clinical trial advancement and drug manufacturing activities.
Research and development expenses were $124.4 million for the nine months ended September 30, 2025, compared to $71.9 million for the nine months ended September 30, 2024. The increase of $52.5 million was primarily due to an increase of $23.2 million in bitopertin development expense consisting of a $10.0 million milestone payment triggered by the initiation of our APOLLO trial and a $13.2 million increase in ongoing costs associated with both the APOLLO study and the HELIOS long-term extension trial. Personnel-related costs increased $13.9 million related to higher research and development headcount, including an increase of $6.3 million in stock-based compensation driven by awards granted under our equity compensation plans. Other research programs and expenses increased $6.4 million related to expanded research initiatives and ongoing program support. Additionally, DISC-0974 development expenses increased $5.4 million primarily due to clinical trial advancement and drug manufacturing activities. DISC-3405 development expenses increased $3.7 million driven by a $10.0 million milestone payment triggered by the first administration to a patient in a Phase 2 clinical trial, partially offset by a decrease of $6.3 million related to manufacturing costs incurred in the nine months ended September 30, 2024 that did not recur in the nine months ended September 30, 2025.
Selling, General and Administrative Expenses
The following table summarizes our selling, general and administrative expenses for the three and nine months ended September 30, 2025 and 2024 (in thousands):
|
Three months ended September 30, |
Nine months ended September 30, |
|||||||||||||||||||||||
|
2025 |
2024 |
Change |
2025 |
2024 |
Change |
|||||||||||||||||||
|
Personnel-related (including equity-based compensation) |
$ |
10,587 |
$ |
4,970 |
$ |
5,617 |
$ |
25,715 |
$ |
14,506 |
$ |
11,209 |
||||||||||||
|
Legal, consulting and professional fees |
5,432 |
2,423 |
3,009 |
14,866 |
6,177 |
8,689 |
||||||||||||||||||
|
Other expenses |
1,343 |
778 |
565 |
4,055 |
2,613 |
1,442 |
||||||||||||||||||
|
Total selling, general and administrative expenses |
$ |
17,362 |
$ |
8,171 |
$ |
9,191 |
$ |
44,636 |
$ |
23,296 |
$ |
21,340 |
||||||||||||
Selling, general and administrative expenses were $17.4 million for the three months ended September 30, 2025, compared to $8.2 million for the three months ended September 30, 2024. The increase of $9.2 million was due to an increase of $5.6 million in personnel-related costs due to higher selling, general and administrative headcount, including an increase of $2.4 million in stock-based compensation driven by awards granted under our equity compensation plans. There was also a $3.0 million increase in legal, consulting, and professional fees primarily related to developing our commercialization capabilities.
Selling, general and administrative expenses were $44.6 million for the nine months ended September 30, 2025, compared to $23.3 million for the nine months ended September 30, 2024. The increase of $21.3 million was due to an increase of $11.2 million in personnel-related costs due to higher selling, general and administrative headcount, including an increase of $5.5 million in stock-based compensation driven by awards granted under our equity compensation plans. There was also a $8.7 million increase in legal, consulting, and professional fees primarily related to developing our commercialization capabilities.
Other Income (Expense), Net
Other income (expense), net was $5.5 million for the three months ended September 30, 2025, compared to $6.4 million for the three months ended September 30, 2024. This decrease of $0.9 million was driven by an increase in interest expense associated with our term loan.
Other income (expense), net was $17.7 million for the nine months ended September 30, 2025, compared to $15.4 million for the nine months ended September 30, 2024. This increase of $2.3 million was primarily due to higher interest income driven by larger cash, cash equivalents and marketable securities balances, partially offset by an increase in interest expense associated with our term loan.
Income tax expense was $0.1 million and $0.3 million for the three and nine months ended September 30, 2025, respectively, compared to $0.1 million and $0.2 million for the three and nine months ended September 30, 2024, respectively. This expense primarily relates to state income tax resulting from an increase in interest income.
Liquidity and Capital Resources
Sources of Liquidity
Since our inception, we have not generated any revenue from product sales and have incurred significant operating losses. We expect to continue to incur significant expenses and operating losses in the foreseeable future as we advance the clinical development of our product candidates. We expect that our research and development and selling, general and administrative costs will continue
to increase significantly, including in connection with conducting clinical trials and manufacturing for our product candidates to support potential commercialization and providing selling, general and administrative support for our operations, including the costs associated with operating as a public company. As a result, we may need additional capital to fund our operations, which we may obtain from additional equity or debt financings, collaborations, licensing arrangements or other sources. See "Risk Factors" included within Item 1A of this Quarterly Report on Form 10-Q for additional risks associated with our substantial capital requirements.
To date, we have funded our operations primarily with proceeds from the sale of our convertible preferred stock and common stock, the proceeds from the merger with Gemini, proceeds from various private and public sales of our equity securities and proceeds from borrowings under the Hercules Loan Agreement. Through September 30, 2025, we have received net proceeds of $144.5 million from sales of our Series Seed, Series A and Series B convertible preferred stock, $89.5 million from the merger with Gemini, $730.8 million from various private and public sales of our equity securities, and $27.6 million from borrowings under the Hercules Loan Agreement. As of September 30, 2025, we had cash, cash equivalents and marketable securities of $615.9 million.
In October 2025, we completed an underwritten public offering of shares of our common stock and pre-funded warrants for net proceeds of approximately $210.9 million, after deducting estimated offering expenses payable by us.
We have incurred significant operating losses since inception and, as of September 30, 2025, had an accumulated deficit of $449.7 million. In addition, we expect to continue to incur significant and increasing expenses and operating losses for the foreseeable future. We believe that our cash, cash equivalents and marketable securities, including the net proceeds of our October 2025 underwritten public offering, will be sufficient to fund our current operating and capital expenditure plans and our debt service obligations into 2029 without taking into account any potential net cash inflows from bitopertin or any other marketed product, if approved during such period. We have based these estimates on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. We may also pursue additional cash resources through public or private equity offerings, collaborations or additional draws under the Hercules Loan Agreement.
Cash Flows
The following table provides information regarding our cash flows for each period presented (in thousands):
|
Nine Months Ended |
||||||||
|
2025 |
2024 |
|||||||
|
Net cash (used in) provided by: |
||||||||
|
Operating activities |
$ |
(137,093 |
) |
$ |
(66,353 |
) |
||
|
Investing activities |
(171,406 |
) |
(290,906 |
) |
||||
|
Financing activities |
256,731 |
189,517 |
||||||
|
Net decrease in cash, cash equivalents and restricted cash |
$ |
(51,768 |
) |
$ |
(167,742 |
) |
||
Operating Activities
Our cash flows from operating activities are greatly influenced by our use of cash for operating expenses and working capital requirements to support our business. We have historically experienced negative cash flows from operating activities as we invested in developing our portfolio, drug discovery efforts, conducting clinical trials and manufacturing, and related infrastructure. The cash used in operating activities resulted primarily from our net losses adjusted for non-cash charges and changes in components of operating assets and liabilities, which are primarily the result of increased expenses and timing of vendor payments.
During the nine months ended September 30, 2025, net cash used in operating activities of $137.1 million was primarily due to our net loss of $151.7 million and the change in operating assets and liabilities of $4.9 million. These were partially offset by non-cash expenses of $19.4 million which included $24.3 million in stock-based compensation, reduced by $6.9 million from amortization and accretion of investment securities.
During the nine months ended September 30, 2024, net cash used in operating activities of $66.4 million was primarily due to our net loss of $79.9 million, which includes interest income of $15.5 million; partially offset by non-cash expenses of $9.8 million primarily related to stock-based compensation expense of $12.5 million offset by amortization/accretion of investment securities of $3.1 million and changes in operating assets and liabilities of $3.7 million, including a reduction in accounts payable for the payment of a milestone payment of $5.0 million to Mabwell.
Investing Activities
During the nine months ended September 30, 2025, net cash used in investing activities was primarily due to purchases of marketable securities of $491.3 million, partially offset by maturities of marketable securities of $320.8 million.
During the nine months ended September 30, 2024, net cash used in investing activities was primarily due to purchases of marketable securities of $313.9 million, partially offset by maturities of marketable securities of $23.0 million.
Financing Activities
During the nine months ended September 30, 2025, net cash provided by financing activities of $256.7 million consisted primarily of aggregate net proceeds of $243.4 million and $9.8 million from our underwritten offering in January 2025 and sales of shares of common stock in Cantor ATM offerings, respectively.
During the nine months ended September 30, 2024, net cash provided by financing activities of $189.5 million consisted primarily of cash net proceeds of $172.5 million from the underwritten offering and aggregate net proceeds of $14.8 million from at-the-market offerings.
Future Funding Requirements
We expect our expenses to increase substantially in connection with our ongoing activities, particularly as we advance our product candidates into and through clinical development and continue to operate as a public company. Our funding requirements and the timing and amount of our operating expenditures will depend largely on:
Identifying potential product candidates and conducting preclinical studies and clinical trials is a time consuming, expensive and uncertain process that takes years to complete, and we may never generate the necessary data or results required to obtain marketing approval and achieve product sales. In addition, our product candidates, if approved, may not achieve commercial success. Our commercial revenues, if any, will be derived from sales of products that may take longer than we anticipate to become commercially available, if they become commercially available at all. Accordingly, we may need to obtain substantial additional funds to achieve our business objectives.
Until such time, if ever, as we can generate substantial revenue from product sales, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, and marketing, distribution or licensing arrangements with third parties. However, we may be unable to raise additional funds or enter into such other arrangements when needed on favorable terms or at all. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our existing stockholders may be materially diluted, and the terms of such securities could include liquidation or other preferences that adversely affect the rights of our common stockholders. Debt financing and preferred equity financing, if available, may involve agreements that include restrictive covenants that limit our ability to take specified actions, such as incurring additional debt, making capital expenditures or declaring dividends. If we raise funds through collaborations, strategic alliances or marketing, distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings or other arrangements when needed, we may be required to delay, reduce or eliminate 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.
Contractual Obligations and Other Commitments
The following table summarizes our contractual obligations as of September 30, 2025 and the effects that such obligations are expected to have on our liquidity and cash flows in future periods (in thousands):
|
Payments Due by Period |
||||||||||||||||||||
|
Total |
Less Than |
1 to 3 |
3 to 5 |
More Than |
||||||||||||||||
|
Operating lease commitments(1) |
$ |
3,337 |
$ |
800 |
$ |
1,425 |
$ |
1,112 |
$ |
- |
||||||||||
|
Total |
$ |
3,337 |
$ |
800 |
$ |
1,425 |
$ |
1,112 |
$ |
- |
||||||||||
(1) Amounts reflect contractual rent payments due for our leased and subleased office spaces in Watertown, Massachusetts as of September 30, 2025 and include contractual payments due under our forward-starting lease which is expected to commence in December 2026. The term date for our existing lease is December 31, 2029 and the term date for our existing sublease is November 30, 2026.
We enter into contracts in the normal course of business with CROs, CDMOs and other third parties for preclinical studies, clinical trials and manufacturing services. These contracts typically do not contain minimum purchase commitments and are generally cancelable by us upon written notice. Payments due upon cancellation consist of payments for services provided or expenses incurred, including noncancelable obligations of our service providers, up to the date of cancellation and, in the case of certain arrangements with CROs and CDMOs, may include non-cancelable fees. These payments are not included in the table above as the amount and timing of such payments are not fixed and estimable.
We have also entered into license agreements under which we are obligated to make specified milestone and royalty payments. We have not included future payments under these agreements in the table of contractual obligations above since the payment obligations under these agreements are contingent upon future events such as regulatory milestones or generating product sales. We are unable to estimate the timing or likelihood of achieving these milestones or generating future product sales. For additional information about our license agreements and amounts that could become payable in the future under such agreements, see our condensed consolidated financial statements.
Critical Accounting Policies and Estimates
Our management's discussion and analysis of our financial condition and results of operations is based on our condensed consolidated financial statements, which have been prepared in accordance with U.S. generally accepted accounting principles, or GAAP. The preparation of our condensed consolidated financial statements and related disclosures requires us to make estimates and judgments that affect the reported amounts of assets, liabilities, and costs and expenses. We base our estimates on historical experience, known trends and events and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. We evaluate our estimates and assumptions on an ongoing basis. Our actual results may differ from these estimates under different assumptions or conditions. See our Annual Report on Form 10-K for the year ended December 31, 2024 for more information about critical accounting policies as well as a description of our other significant accounting policies.
There have been no material changes to our critical accounting estimates from those described in Part II, Item 7. "Management's Discussion and Analysis of Financial Condition and Results of Operations" included in our Annual Report on Form 10-K for the year ended December 31, 2024.
Recently Issued and Adopted Accounting Pronouncements
A description of recently issued and certain recently adopted accounting pronouncements that have or may potentially impact our financial position and results of operations is included in Note 2 - Summary of Significant Accounting Policiesto our consolidated financial statements in our Annual Report on Form 10-K for the year ended December 31, 2024.