11/05/2026 | Press release | Distributed by Public on 11/05/2026 07:41
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, 2025. 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; selcodebart (DISC-0974) for the treatment of anemia of myelofibrosis, or MF, and anemia of inflammatory bowel disease, or IBD; 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.
Heme Biosynthesis: Bitopertin
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 October 2026, we announced positive topline results from APOLLO, a randomized, double-blind, pivotal Phase 3 trial evaluating the safety and efficacy of bitopertin versus placebo in adults and adolescents aged 12 or older with EPP or XLP. Bitopertin achieved statistical significance on both co-primary endpoints versus placebo, with reduction in whole-blood metal-free protoporphyrin IX, or PPIX, from baseline (-41.4% vs. placebo, p<0.0001) and increase in average monthly total time in light without pain during month 6 (+12.2 hours vs. placebo, p=0.0010). Bitopertin also achieved statistical significance on all secondary endpoints versus placebo, including reduction in incidence rate of phototoxic reactions over the 6-month treatment period (p<0.0001), increase in cumulative total time in light without pain over the 6-month treatment period (p<0.0001), and increase in change from baseline in average daily sunlight exposure time to prodrome (p=0.0077, adults only). In addition, bitopertin demonstrated additional quality of life benefits versus placebo, including improved Patient Global Impression of Change, or PGIC, score (p<0.0001) and Patient Global Impression of Severity, or PGIS, score (p<0.0001). Bitopertin was generally well-tolerated. Consistent with prior studies, dizziness was the most commonly reported treatment emergent adverse event, or TEAE, in the bitopertin group (50%) and was mild or moderate and generally transient. There were no TEAEs above Grade 3.
We have also completed two Phase 2 clinical trials of bitopertin: BEACON, an open-label, parallel-dose clinical trial in EPP and XLP patients conducted at sites in Australia, and AURORA, a randomized, double-blind, placebo-controlled clinical trial in EPP patients conducted at sites in the United States. In both trials, bitopertin significantly reduced PPIX and was associated with improvements in measures of time spent in sunlight and quality of life. In addition, bitopertin was generally well-tolerated. All participants in AURORA and BEACON were eligible to participate in HELIOS, an ongoing open-label, long-term extension study of bitopertin in EPP and XLP patients. Interim data from HELIOS, presented in June 2025 and June 2026, demonstrated that longer term treatment of bitopertin was associated with sustained reductions in PPIX, as well as improvements in quality of life and liver biomarkers.
In September 2025 we submitted a New Drug Application, or NDA, for accelerated approval of bitopertin in EPP and XLP in the United States based on our Phase 2 data. The U.S. Food and Drug Administration, or the FDA, accepted our NDA for review in November 2025 and in February 2026 issued a complete response letter, or CRL. The FDA concluded that the AURORA and BEACON trials did not show evidence of association between percent change in PPIX and sunlight exposure-based endpoints as measured in the trials, resulting in uncertainty regarding whether bitopertin's effect on PPIX is reasonably likely to predict clinical benefit, despite the strong mechanistic and biological plausibility supporting the use of the PPIX biomarker in protoporphyria. In our Type A meeting during the second quarter of 2026, the FDA indicated that results of the Phase 3 APOLLO trial, if successful,
could serve as the basis for a response to the CRL and could potentially support a traditional approval. With the positive topline results from APOLLO, we plan to engage with the FDA and resubmit an NDA by the end of 2026, with an FDA decision anticipated by mid-2027.
Our APOLLO clinical trial is also intended to serve as a registrational trial with respect to any potential future marketing applications for bitopertin in EPP and XLP outside the United States. We continue to evaluate potential additional trials of bitopertin in other indications.
Iron Homeostasis: Selcodebart (DISC-0974) and DISC-3405
Selcodebart is the lead product candidate in our iron homeostasis portfolio and was licensed from AbbVie Deutschland GmbH & Co. KG, or AbbVie. Selcodebart is designed to suppress hepcidin production and increase serum iron levels. We completed a Phase 1 clinical trial in healthy volunteers in the United States 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, with results from the Phase 1b portion of this clinical trial showing substantial and sustained reductions in hepcidin levels and increases in iron alongside strong hematologic responses across patient types and an acceptable tolerability profile. We initiated RALLY-MF, the open-label Phase 2 portion of this clinical trial, in December 2024 and reported initial data in December 2025 and interim data in June 2026, with results demonstrating meaningful overall anemia responses across all patient subgroups, regardless of baseline transfusion status and independent of concomitant Janus Kinase, or JAK, inhibitor therapy use. We expect to share additional data and hold an end-of-Phase 2 meeting with the FDA for selcodebart in anemia of MF by the end of 2026. We anticipate initiating a Phase 3 clinical program for selcodebart in anemia of MF in the first half of 2027, subject to the outcome of our end-of-Phase 2 meeting with the FDA. We also initiated RALLY-IBD, a Phase 2 clinical trial of selcodebart in patients with IBD and anemia in the first quarter of 2026, with initial data anticipated in 2027, 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 selcodebart. We believe this profile may be desirable in certain subsets of patients with anemia associated with inflammatory diseases.
In addition, 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, with results showing an acceptable tolerability profile and evidence of target engagement, including dose-related increases in serum hepcidin and corresponding reductions in serum iron across all dose levels. We are developing DISC-3405 for the treatment of PV, SCD, and other hematologic disorders. We initiated RESTORE-PV, an open-label Phase 2 clinical trial of DISC-3405 in patients with PV, in June 2025, and reported initial efficacy data from Cohort A of this trial in September 2026, with results demonstrating that treatment with DISC-3405 increased hepcidin and lowered serum iron, translating to reduction in phlebotomy events, controlled hematocrit, and improved symptom burden in patients with PV. In this initial data, DISC-3405 was generally well-tolerated in participants in Cohort A and Cohort B, with adverse events that were consistent with underlying disease and a low rate of injection site reactions, which were mild and self-limited. We expect to report additional data from our RESTORE-PV clinical trial by the end of 2026 and topline data in 2027. If positive, and subject to engagement with the FDA, we anticipate initiating a Phase 3 clinical program for DISC-3405 in PV in 2027. We also initiated a Phase 1b, open-label clinical trial of DISC-3405 in patients with SCD in October 2025, with initial data expected in the fourth quarter of 2026, and plan to explore the role of therapeutic iron restriction in other indications.
Funding
As of September 30, 2026, we had cash, cash equivalents and marketable securities of $682.8 million. In October 2026, we completed an underwritten public offering of shares of our common stock for net proceeds of approximately $485.9 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 2026 underwritten public offering, will be sufficient to fund our current operating and capital expenditure plans and our debt service obligations into 2030, 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 or costs of the efforts that will be necessary to complete their preclinical and clinical development. Changes in the outcome of any number of variables with respect to these programs could significantly affect the costs and timing associated with their development. We may never succeed in obtaining regulatory approval for any product candidates that have not yet been approved. The successful development of any product candidate is highly uncertain due to the numerous risks and uncertainties inherent in 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 the 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 and corporate debt securities, 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, 2026 and 2025
The following table summarizes our results of operations for the three and nine months ended September 30, 2026 and 2025 (in thousands):
|
Three months ended |
Nine Months Ended |
|||||||||||||||||||||||
|
2026 |
2025 |
Change |
2026 |
2025 |
Change |
|||||||||||||||||||
|
Operating expenses: |
||||||||||||||||||||||||
|
Research and development |
$ |
44,080 |
$ |
50,334 |
$ |
(6,254 |
) |
$ |
136,917 |
$ |
124,416 |
$ |
12,501 |
|||||||||||
|
Selling, general and administrative |
20,081 |
17,362 |
2,719 |
61,843 |
44,636 |
17,207 |
||||||||||||||||||
|
Total operating expenses |
64,161 |
67,696 |
(3,535 |
) |
198,760 |
169,052 |
29,708 |
|||||||||||||||||
|
Loss from operations |
(64,161 |
) |
(67,696 |
) |
3,535 |
(198,760 |
) |
(169,052 |
) |
(29,708 |
) |
|||||||||||||
|
Other income (expense), net: |
||||||||||||||||||||||||
|
Interest income |
6,454 |
6,412 |
42 |
19,879 |
20,349 |
(470 |
) |
|||||||||||||||||
|
Interest expense |
(1,714 |
) |
(917 |
) |
(797 |
) |
(3,466 |
) |
(2,724 |
) |
(742 |
) |
||||||||||||
|
Other (expense) income |
(5 |
) |
(18 |
) |
13 |
5 |
47 |
(42 |
) |
|||||||||||||||
|
Total other income (expense), net |
4,735 |
5,477 |
(742 |
) |
16,418 |
17,672 |
(1,254 |
) |
||||||||||||||||
|
Loss before income taxes |
(59,426 |
) |
(62,219 |
) |
2,793 |
(182,342 |
) |
(151,380 |
) |
(30,962 |
) |
|||||||||||||
|
Income tax expense |
(109 |
) |
(102 |
) |
(7 |
) |
(229 |
) |
(273 |
) |
44 |
|||||||||||||
|
Net loss |
$ |
(59,535 |
) |
$ |
(62,321 |
) |
$ |
2,786 |
$ |
(182,571 |
) |
$ |
(151,653 |
) |
$ |
(30,918 |
) |
|||||||
Research and Development Expenses
The following table summarizes our research and development expenses for the three and nine months ended September 30, 2026 and 2025 (in thousands):
|
Three months ended |
Nine Months Ended |
|||||||||||||||||||||||
|
2026 |
2025 |
Change |
2026 |
2025 |
Change |
|||||||||||||||||||
|
Bitopertin |
$ |
6,992 |
$ |
11,833 |
$ |
(4,841 |
) |
$ |
24,998 |
$ |
43,954 |
$ |
(18,956 |
) |
||||||||||
|
Selcodebart (DISC-0974) |
8,422 |
7,369 |
1,053 |
28,885 |
18,105 |
10,780 |
||||||||||||||||||
|
DISC-3405 |
4,880 |
11,910 |
(7,030 |
) |
19,027 |
15,486 |
3,541 |
|||||||||||||||||
|
Other research programs and expenses |
6,185 |
5,386 |
799 |
13,792 |
13,491 |
301 |
||||||||||||||||||
|
Personnel-related (including equity-based compensation) |
17,601 |
13,836 |
3,765 |
50,215 |
33,380 |
16,835 |
||||||||||||||||||
|
Total research and development expenses |
$ |
44,080 |
$ |
50,334 |
$ |
(6,254 |
) |
$ |
136,917 |
$ |
124,416 |
$ |
12,501 |
|||||||||||
Research and development expenses were $44.1 million for the three months ended September 30, 2026, compared to $50.3 million for the three months ended September 30, 2025. The decrease of $6.3 million was driven by a $7.0 million reduction in expenses related to DISC-3405, primarily related to a $10.0 million milestone payment incurred during the comparative period. There was also a decrease of $4.8 million in bitopertin development expenses, primarily related to drug manufacturing activities in the comparative period that did not recur in the current period. These decreases were partially offset by an increase in personnel related costs of $3.8 million related to higher research and development headcount, including an increase of $2.9 million in stock-based compensation driven by awards granted under our equity compensation plans. Selcodebart development expenses increased $1.1 million, primarily related to drug manufacturing activities. Other research programs and expenses also increased $0.8 million.
Research and development expenses were $136.9 million for the nine months ended September 30, 2026, compared to $124.4 million for the nine months ended September 30, 2025. The increase of $12.5 million was primarily due to an increase of $16.8 million in personnel-related costs related to higher research and development headcount, including an increase of $8.8 million in stock-based compensation driven by awards granted under our equity compensation plans. Selcodebart development expenses increased $10.8 million primarily related to clinical trial advancement and drug manufacturing activities. DISC-3405 development expenses increased $3.5 million primarily driven by increased clinical trial and drug manufacturing activities of $8.5 million which were partially offset by lower milestone payments of $5.0 million. Bitopertin development expenses decreased $19.0 million primarily driven by a $10.0 million milestone payment and drug manufacturing activities in the comparative period that did not recur in the current period.
Selling, General and Administrative Expenses
The following table summarizes our selling, general and administrative expenses for the three and nine months ended September 30, 2026 and 2025 (in thousands):
|
Three months ended |
Nine Months Ended |
|||||||||||||||||||||||
|
2026 |
2025 |
Change |
2026 |
2025 |
Change |
|||||||||||||||||||
|
Personnel-related (including equity-based compensation) |
$ |
12,903 |
$ |
10,587 |
$ |
2,316 |
$ |
40,618 |
$ |
25,715 |
$ |
14,903 |
||||||||||||
|
Legal, consulting and professional fees |
5,796 |
5,432 |
364 |
16,091 |
14,866 |
1,225 |
||||||||||||||||||
|
Other expenses |
1,382 |
1,343 |
39 |
5,134 |
4,055 |
1,079 |
||||||||||||||||||
|
Total selling, general and administrative expenses |
$ |
20,081 |
$ |
17,362 |
$ |
2,719 |
$ |
61,843 |
$ |
44,636 |
$ |
17,207 |
||||||||||||
Selling, general and administrative expenses were $20.1 million for the three months ended September 30, 2026, compared to $17.4 million for the three months ended September 30, 2025. The increase of $2.7 million was primarily due to an increase of $2.3 million in personnel-related costs due to increased selling, general and administrative headcount, including an increase of $2.2 million in stock-based compensation driven by awards granted under our equity compensation plans.
Selling, general and administrative expenses were $61.8 million for the nine months ended September 30, 2026, compared to $44.6 million for the nine months ended September 30, 2025. The increase of $17.2 million was primarily due to an increase of $14.9 million in personnel-related costs due to increased selling, general and administrative headcount, including an increase of $7.4 million in stock-based compensation driven by awards granted under our equity compensation plans and an increase of $1.6 million related to an organizational restructuring. There were also increases of $1.1 million and $1.2 million in other expenses and legal, consulting, and professional fees, respectively, primarily related to developing our commercialization capabilities.
Other Income (Expense), Net
Other income (expense), net was $4.7 million and $16.4 million for the three and nine months ended September 30, 2026, respectively, compared to $5.5 million and $17.7 million for the three and nine months ended September 30, 2025, respectively. Other income (expense), net consists primarily of interest income from our cash, cash equivalents and marketable securities, partially offset by interest expense associated with our term loan.
Income tax expense was $0.1 million and $0.2 million for the three and nine months ended September 30, 2026, respectively, compared to $0.1 million and $0.3 million for the three and nine months ended September 30, 2025, respectively. This expense primarily relates to state income tax resulting from 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 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, 2026, 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, $941.8 million from various private and public sales of our equity securities, and $57.6 million from net borrowings under the Hercules Loan Agreement. As of September 30, 2026, we had cash, cash equivalents and marketable securities of $682.8 million.
In October 2026, we completed an underwritten public offering of shares of our common stock for net proceeds of approximately $485.9 million, after deducting estimated offering expenses payable by us.
We have incurred significant operating losses since inception and, as of September 30, 2026, had an accumulated deficit of $692.8 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 2026 underwritten public offering, will be sufficient to fund our current operating and capital expenditure plans and our debt service obligations into 2030, 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 debt financing.
Cash Flows
The following table provides information regarding our cash flows for each period presented (in thousands):
|
Nine Months Ended |
||||||||
|
2026 |
2025 |
|||||||
|
Net cash (used in) provided by: |
||||||||
|
Operating activities |
$ |
(146,639 |
) |
$ |
(137,093 |
) |
||
|
Investing activities |
105,267 |
(171,406 |
) |
|||||
|
Financing activities |
40,210 |
256,731 |
||||||
|
Net decrease in cash, cash equivalents and restricted cash |
$ |
(1,162 |
) |
$ |
(51,768 |
) |
||
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, 2026, net cash used in operating activities of $146.6 million was primarily due to our net loss of $182.6 million. This was partially offset by non-cash expenses of $40.2 million, which included $40.5 million in stock-based compensation, and the change in operating assets and liabilities of $4.2 million.
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.
Investing Activities
During the nine months ended September 30, 2026, net cash provided by investing activities was primarily due to maturities of marketable securities of $410.2 million, partially offset by purchases of marketable securities of $305.0 million.
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.
Financing Activities
During the nine months ended September 30, 2026, net cash provided by financing activities of $40.2 million consisted primarily of net proceeds from the issuance of long-term debt of $30.0 million and proceeds from stock option exercises of $9.1 million.
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 pursuant to our Controlled Equity Offering Sales Agreement, or the Cantor ATM Agreement, with Cantor Fitzgerald & Co., or Cantor, respectively.
Future Funding Requirements
We expect our expenses to continue to increase in connection with our ongoing activities, particularly as we continue the research and development of, initiate and complete clinical trials of, and seek regulatory approval for, our product candidates. In addition, depending on the status of regulatory approval, or 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. We may also need to raise additional funds sooner if we choose to pursue additional indications and/or geographies for our current or future product candidates or otherwise expand more rapidly than presently anticipated. Furthermore, we expect to continue to incur costs associated
with operating as a public company. Our future capital requirements will depend on and could increase significantly as a result of many factors, including:
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 product revenue, 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, 2026 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) |
$ |
2,505 |
$ |
534 |
$ |
1,745 |
$ |
226 |
$ |
- |
||||||||||
|
Total |
$ |
2,505 |
$ |
534 |
$ |
1,745 |
$ |
226 |
$ |
- |
||||||||||
(1) Amounts reflect contractual rent payments due for our leased and subleased office spaces in Watertown, Massachusetts as of September 30, 2026 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, 2025 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, 2025.
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 Policies to our consolidated financial statements in our Annual Report on Form 10-K for the year ended December 31, 2025.