Disc Medicine, Inc.
For the period ended Mar 31, 2025. Filed May 7, 2025.
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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.
Mar 31, 2025 · Filed May 7, 2025
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2024-09-30; filed 2024-11-12.
0000950170-24-124650
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-03-31; filed 2025-05-07.
0000950170-25-065069
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 105 previous paragraphs and 98 current paragraphs.
59 unchanged; 28 reworded; 11 added; 15 removed; 3 previous and 0 current paragraphs rearranged.
Tables are not compared: 8 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 (11)
Current report
In January 2025 we completed an underwritten offering of shares of our common stock and pre-funded warrants for net proceeds of $243.4 million, after deducting offering expenses. As of March 31, 2025, we had cash, cash equivalents and marketable securities of $694.7 million. We believe that our cash, cash equivalents and marketable securities will be sufficient to fund our current operating and capital expenditure plans and our debt service obligations into 2028, 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.
Current report
Research and Development Expenses
Current report
Selling, General and Administrative Expenses
Current report
Other Income (Expense), Net
Current report
Interest Expense
Current report
Interest expense primarily consists of amortization of debt issuance costs and discount and interest expense under the Hercules Loan Agreement.
Current report
Research and Development Expenses
Current report
Research and development expenses were $27.8 million for the three months ended March 31, 2025, compared to $23.7 million for the three months ended March 31, 2024. The increase of $4.1 million was primarily due to increases of $2.4 million and $2.1 million in DISC-0974 and bitopertin development expense, respectively, related to advancing the clinical trials and drug manufacturing activity. In addition, personnel-related costs increased $2.5 million related to higher research and development headcount, including an increase of $1.4 million in stock-based compensation driven by awards granted under our equity compensation plans. These increases were partially offset by a decrease of $3.0 million in DISC-3405 development expense related to manufacturing costs incurred in the three months ended March 31, 2024 that did not recur in the three months ended March 31, 2025.
Current report
Selling, General and Administrative Expenses
Current report
The following table summarizes our selling, general and administrative expenses for the three months ended March 31, 2025 and 2024 (in thousands):
Current report
Other Income (Expense), Net
Removed paragraphs (15)
Previous report
Change in Fair Value of Contingent Value Right ("CVR") Liability
Previous report
In December 2022, we entered into the Contingent Value Rights Agreement (the “CVR Agreement”), described in more detail in Note 1 to our consolidated financial statements in our Annual Report on Form 10-K for the year ended December 31, 2023 and Note 3 to our unaudited condensed consolidated financial statements, which provided a CVR to pre-merger common stockholders of Gemini Therapeutics, Inc. (“Gemini”) to receive common stock from the Company upon its receipt of certain proceeds, calculated in accordance with the CVR Agreement, resulting from a disposition of Gemini’s pre-merger assets within one year after the closing of the merger. The disposition period ended December 29, 2023 and no dispositions of Gemini’s pre-merger assets were made during the disposition period. Therefore, there were no CVR payments made to the holders and there will not be any future CVR payments to the holders. Prior to December 29, 2023, the CVR liability was measured at fair value as of each reporting date and the change in the fair value for the period, if any, was recorded in the condensed consolidated statements of operations and comprehensive loss in the change in fair value of contingent value right liability. The change in fair value of the CVR liability was $1.5 million for the nine months ended September 30, 2023.
Previous report
Research and development expenses were $24.7 million for the three months ended September 30, 2024, compared to $14.4 million for the three months ended September 30, 2023. The increase of $10.3 million in research and development expenses was primarily due to a $3.6 million, $2.2 million, and $1.4 million increase in development expense for DISC-0974, bitopertin, and DISC-3405, respectively, each related to increased clinical study and drug manufacturing activity. Personnel-related costs also increased $2.9 million related to higher research and development headcount, including an increase of $1.2 million in stock-based compensation driven by the Company's annual grant to employees in January 2024.
Previous report
The following table summarizes our general and administrative expenses for the three months ended September 30, 2024 and 2023 (in thousands):
Previous report
Income Tax Expense
Previous report
Comparison of the Nine Months Ended September 30, 2024 and 2023
Previous report
Research and development expenses were $71.9 million for the nine months ended September 30, 2024, compared to $46.7 million for the nine months ended September 30, 2023. The increase of $25.2 million in research and development expenses was primarily due to a $9.0 million increase in personnel-related costs related to higher research and development headcount, including an increase of $3.7 million in stock-based compensation driven by the Company's annual grant to employees in January 2024 and a $9.0 million and $6.0 million increase in development expense related to increased clinical study and drug manufacturing activity for bitopertin and DISC-0974, respectively; partially offset by a $0.6 million decrease related to the DISC-3405 program which incurred $10.0 million in an upfront license fee in the nine months ended September 30, 2023, offset by the increase in development expense in the nine months ended September 30, 2024 related to increased clinical study and drug manufacturing activity.
Previous report
The following table summarizes our general and administrative expenses for the nine months ended September 30, 2024 and 2023 (in thousands):
Previous report
General and administrative expenses were $23.3 million for the nine months ended September 30, 2024, compared to $14.7 million for the nine months ended September 30, 2023. The increase of $8.6 million in general and administrative expenses was primarily due to an increase of $7.7 million in personnel-related costs due to higher general and administrative headcount, including an increase of $5.1 million in stock-based compensation driven by the Company's annual grant to employees in January 2024.
Previous report
Other income (expense), net was $15.4 million for the nine months ended September 30, 2024, compared to other income (expense), net of $8.6 million for the nine months ended September 30, 2023. The change of $6.8 million in other income (expense), net was primarily due to an increase in interest income based on larger cash, cash equivalents, and marketable securities balances and a decrease in expense of $1.5 million due to the disposition period of the CVR liability ending in December 2023.
Previous report
Income Tax Expense
Previous report
In addition, in November 2024, we and certain of our subsidiaries, collectively referred to as the Borrower, entered into a Loan and Security Agreement, or the Hercules Loan Agreement, with the lenders party thereto and Hercules Capital, Inc., as administrative agent and collateral agent, providing for up to $200.0 million of senior secured term loans available to Borrower in multiple tranches, including an initial $30.0 million advance funded on November 6, 2024. The Hercules Loan Agreement is described in more detail in Note 17 to our unaudited condensed consolidated financial statements contained in Part I, Item I of this Quarterly Report on Form 10-Q.
Previous report
Emerging Growth Company and Smaller Reporting Company Status
Previous report
We are an “emerging growth company,” as defined in the Jumpstart Our Business Startups Act of 2012, or the JOBS Act, and we may take advantage of certain exemptions from various reporting requirements that are applicable to other public companies that are not emerging growth companies. We may take advantage of these exemptions until we are no longer an emerging growth company under Section 107 of the JOBS Act, which provides that an emerging growth company can take advantage of the extended transition period afforded by the JOBS Act for the implementation of new or revised accounting standards. We have elected to avail ourselves of the extended transition period and, therefore, while we are an emerging growth company, we will not be subject to new or revised accounting standards at the same time that they become applicable to other public companies that are not emerging growth companies, unless we choose to early adopt a new or revised accounting standard. We will remain an emerging growth company until the earlier of (1) the last day of the fiscal year (a) following the fifth anniversary of the closing of our initial public offering which occurred in August 2020, (b) in which we have total annual gross revenue of at least $1.235 billion, or (c) in which we are deemed to be a large accelerated filer, which means the market value of our common stock that is held by non-affiliates exceeds $700 million as of the prior June 30th, and (2) the date on which we have issued more than $1.0 billion in non-convertible debt during the prior three-year period.
Previous report
Additionally, we are a “smaller reporting company” as defined in Item 10(f)(1) of Regulation S-K. Smaller reporting companies may take advantage of certain reduced disclosure obligations, including, among other things, providing only two years of audited financial statements.
Reworded paragraphs (28)
Previous wording
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, and anemia of chronic kidney disease, or CKD; and DISC-3405 (formerly MWTX-003) for the treatment of polycythemia vera, or PV, and other hematologic disorders. In addition, our preclinical programs also 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.
Current wording
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, and anemia of chronic kidney disease, or CKD; and DISC-3405 for the treatment of polycythemia vera, or PV, and other hematologic disorders. In addition, our preclinical programs also 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.
Previous wording
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. 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. 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. Additional analyses of the BEACON and AURORA trials were presented in June 2024 and we plan to share further analyses in December 2024. All participants in AURORA and BEACON are eligible to participate in HELIOS, an ongoing open-label, long-term extension study of bitopertin in EPP and XLP. We are also planning APOLLO, a randomized, double-blind, placebo-controlled clinical trial of bitopertin in EPP and XLP patients. As a result of our end-of-Phase 2 meeting with the U.S. Food & Drug Administration, or the FDA, we agreed with the FDA on all proposed attributes of the APOLLO trial, which we plan to initiate by mid-2025. In addition, the FDA agreed with the potential for reduction of protoporphyrin IX, or PPIX, to serve as a surrogate endpoint to support a potential accelerated approval. Under the FDA's Accelerated Approval Program, we would have the potential to submit a New Drug Application, or NDA, for bitopertin in EPP and XLP based on our existing data and, subject to further discussion with the FDA, the APOLLO trial would serve as a confirmatory trial. We will be meeting with the FDA to finalize the details of APOLLO and plan to provide an update in the first quarter of 2025 on this discussion as well as timing for a potential NDA filing under an accelerated pathway. We have also entered into a collaborative research and development agreement with the National Institutes of Health, or NIH, to conduct an NIH-sponsored clinical trial of bitopertin in DBA. The FDA authorized the clinical trial to proceed and the trial began in July 2023. We are planning additional trials of bitopertin in other indications.
Current wording
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 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. Under the FDA’s Accelerated Approval Program, we would have the potential to submit a New Drug Application, or NDA, for bitopertin in EPP and XLP based on our existing data, and we would be required to conduct a post-marketing confirmatory clinical trial. 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. We have now initiated the APOLLO trial and anticipate submitting an NDA for accelerated approval of bitopertin in EPP and XLP in the second half of 2025. 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 U.S. We have also entered into a collaborative research and development agreement with the National Ins…
Excerpt shortened here; the source contains the full paragraph.
Previous wording
DISC-0974 is the lead product candidate in our iron homeostasis portfolio and was in-licensed from AbbVie Deutschland GmbH & Co. KG, or AbbVie. DISC-0974 is designed to suppress hepcidin production and increase serum iron levels. We submitted an Investigational New Drug application, or IND, to the FDA for DISC-0974 in June 2021, received clearance in July 2021, and participants 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 non-dialysis dependent CKD and anemia in October 2024, which additional interim data included safety and changes in hepcidin, iron, and hemoglobin levels for additional patients, as well as longer follow-up. We anticipate presenting additional analyses of the Phase 1b study in anemia of MF in December 2024. In addition, we are developing a preclinical anti-hemojuvelin, or HJV, monoclonal antibody, DISC-0998, which also targets hepcidin suppression and was in-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.
Current wording
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 initiated the open-label Phase 2 portion of this clinical trial. We expect to report initial data from this Phase 2 trial in the second half of 2025. We also expect to report data from the multiple-ascending dose, or MAD, portion of the Phase 1b trial in patients with non-dialysis dependent CKD and anemia in the second half of 2025. We are also planning additional trials of DISC-0974 in other anemias of inflammation. In addition, we are 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.
Previous wording
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. An IND for DISC-3405 was cleared by the FDA, and a Phase 1 clinical trial in healthy adult volunteers was initiated in October 2023. Interim data was presented from the single-ascending dose ("SAD") portion of the Phase 1 clinical trial of DISC-3405 in healthy volunteers in June 2024. We anticipate presenting data from the multiple-ascending dose ("MAD") portion of the Phase 1 healthy volunteer study of DISC-3405 in December 2024. We expect to develop DISC-3405 for the treatment of PV and other hematologic disorders.
Current wording
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 MAD portion of this Phase 1 healthy volunteer study of DISC-3405 in December 2024. We expect to develop DISC-3405 for the treatment of PV and other hematologic disorders and plan to initiate a Phase 2 clinical trial of DISC-3405 in the first half of 2025.
Previous wording
the ability to raise additional funds necessary to complete clinical development of and commercialize our product candidates;
Current wording
our ability to raise additional funds to the extent necessary to complete clinical development of and commercialize our product candidates;
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establishing agreements with third-party manufacturers for supply of product candidate components for our clinical trials;
Current wording
our ability to establish agreements with third-party manufacturers for supply of product candidate components for our clinical trials;
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commercializing product candidates, if and when approved, whether alone or in collaboration with others; and
Current wording
our ability to commercialize product candidates, if and when approved, whether alone or in collaboration with others; and
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obtaining and maintaining third-party insurance coverage and adequate reimbursement for any approved products.
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our ability to obtain and maintain third-party insurance coverage and adequate reimbursement for any approved products.
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General and administrative expenses consist primarily of salaries and related costs for personnel in executive, finance, commercial, corporate and business development, and administrative functions. 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; director and officer insurance costs and other insurance costs; and facility related expenses including maintenance and allocated expenses for rent and other operating costs.
Current wording
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.
Previous wording
We anticipate that our 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 of our product candidates.
Current wording
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.
Previous wording
Interest income primarily consists of interest earned on cash equivalents, consisting of money market funds, U.S. treasury securities and certificates of deposit, and marketable securities, consisting of U.S. treasury securities and U.S. government agency securities.
Current wording
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 and U.S. government agency securities.
Previous wording
General and administrative expenses were $8.2 million for the three months ended September 30, 2024, compared to $4.5 million for the three months ended September 30, 2023. The increase of $3.6 million in general and administrative expenses was primarily due to a $2.8 million increase in personnel-related costs due to higher general and administrative headcount, including an increase of $1.8 million in stock-based compensation driven by the Company's annual grant to employees in January 2024.
Current wording
Selling, general and administrative expenses were $12.2 million for the three months ended March 31, 2025, compared to $7.8 million for the three months ended March 31, 2024. The increase of $4.4 million was due to an increase of $2.0 million in personnel-related costs due to higher selling, general and administrative headcount, including an increase of $0.9 million in stock-based compensation driven by awards granted under our equity compensation plans. There was also a $2.1 million increase in legal, consulting, and professional fees primarily related to developing our commercialization capabilities.
Previous wording
Other income (expense), net was $6.4 million for the three months ended September 30, 2024, compared to other income (expense), net of $4.9 million for the three months ended September 30, 2023. The change of $1.5 million in other income (expense), net was primarily due to an increase in interest income based on larger cash, cash equivalents, and marketable securities balances.
Current wording
Other income (expense), net was $6.0 million for the three months ended March 31, 2025, compared to $4.5 million for the three months ended March 31, 2024. The increase of $1.5 million was primarily due to an increase in interest income based on increases in our cash, cash equivalents and marketable securities balances.
Previous wording
Income tax expense was $0.1 million for the three months ended September 30, 2024, compared to less than $0.1 million for the three months ended September 30, 2023. This expense relates to state income tax resulting from interest income.
Current wording
Income tax expense was $0.1 million for the three months ended March 31, 2025, compared to less than $0.1 million for the three months ended March 31, 2024. This expense relates to state income tax resulting from an increase in interest income.
Previous wording
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 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 general and administrative support for our operations, including the costs associated with operating as a public company. As a result, we will 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” for additional risks associated with our substantial capital requirements.
Current wording
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.
Previous wording
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 at-the-market offerings, proceeds from a registered direct offering, proceeds from a follow-on public offering, proceeds from an underwritten offering, and proceeds from borrowing under the Hercules Loan Agreement described below. Through September 30, 2024, 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, $53.5 million from sales of common stock in a pre-closing private financing, $62.4 million from sales of common stock and pre-funded warrants in a registered direct offering, $147.9 million from sales of common stock and pre-funded warrants in a public follow-on offering, $172.5 million from sales of common stock in an underwritten offering and $41.3 million from at-the-market offerings. As of September 30, 2024, we had cash, cash equivalents, and marketable securities of $487.4 million.
Current wording
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 March 31, 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, $721.0 million from various private and public sales of our equity securities, and $27.6 million from borrowings under the Hercules Loan Agreement. As of March 31, 2025, we had cash, cash equivalents and marketable securities of $694.7 million.
Previous wording
We have incurred significant operating losses since inception and, as of September 30, 2024, had an accumulated deficit of $268.5 million. In addition, we expect to continue to incur significant and increasing expenses and operating losses for the foreseeable future. We believe that our current cash resources, including our borrowing of $30.0 million under the Hercules Loan Agreement as of November 6, 2024, will enable us to fund our current planned operating expense and capital expenditure requirements well into 2027. We may also pursue additional cash resources through public or private equity offerings, collaborations or additional debt financings.
Current wording
We have incurred significant operating losses since inception and, as of March 31, 2025, had an accumulated deficit of $332.1 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 will be sufficient to fund our current operating and capital expenditure plans and our debt service obligations into 2028 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 financings.
Previous wording
During the nine months ended September 30, 2023, net cash used in operating activities of $55.7 million was primarily due to our net loss of $52.9 million and a decrease in operating liabilities of $8.5 million primarily related to the payment of merger-related costs which were included in accounts payable at December 31, 2022, partially offset by non-cash expenses of $5.4 million primarily related to stock-based compensation expense of $3.6 million and the change in fair value of the contingent value right liability of $1.5 million.
Current wording
During the three months ended March 31, 2025, net cash used in operating activities of $41.4 million was primarily due to our net loss of $34.1 million and the change in operating assets and liabilities of $11.5 million. These were partially offset by non-cash expenses of $4.2 million which included $6.4 million in stock-based compensation, reduced by $2.5 million from amortization and accretion of investment securities.
Previous wording
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.
Current wording
During the three months ended March 31, 2024, net cash used in operating activities of $33.6 million was primarily due to our net loss of $26.9 million, which includes interest income of $4.5 million; and changes in operating assets and liabilities of $10.9 million, including a milestone payment of $5.0 million to Mabwell; offset by non-cash expenses of $4.2 million primarily related to stock-based compensation expense of $4.1 million.
Previous wording
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.
Current wording
During the three months ended March 31, 2025, net cash used in investing activities was primarily due to purchases of marketable securities of $347.3 million, partially offset by maturities of marketable securities of $75.5 million.
Previous wording
During the nine months ended September 30, 2023, net cash used in investing activities was due to purchases of property and equipment.
Current wording
During the three months ended March 31, 2024, net cash used in investing activities was due to purchases of property and equipment.
Previous wording
During the nine months ended September 30, 2023, net cash provided by financing activities of $231.6 million consisted primarily of aggregate net proceeds of $147.9 million from sales of common stock and pre-funded warrants in a public follow-on offering, aggregate net proceeds of $62.4 million from sales of common stock and pre-funded warrants in a registered direct offering and aggregate net proceeds of $19.5 million from at-the-market offerings.
Current wording
During the three months ended March 31, 2025, net cash provided by financing activities of $244.3 million consisted primarily of aggregate net proceeds of $243.4 million from our underwritten offering in January 2025.
Previous wording
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.
Current wording
During the three months ended March 31, 2024, net cash provided by financing activities of $15.9 million consisted primarily of aggregate net proceeds of $14.8 million from the at-the-market offerings.
Previous wording
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 operate as a public company. Our funding requirements and the timing and amount of our operating expenditures will depend largely on:
Current wording
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:
Previous wording
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 we do not expect to be commercially available for many years, if ever. Accordingly, we will need to obtain substantial additional funds to achieve our business objectives.
Current wording
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.
Previous wording
(2) Table excludes the impact of tenant improvement allowance reimbursements of $0.8 million which are expected to be received in less than one year. Of this total, $0.5 million relates to our existing leased office space and $0.3 million relates to our forward-starting lease.
Current wording
(2) Table excludes the impact of tenant improvement allowance reimbursements of $0.2 million which are expected to be received in less than one year. Of this total, $0.1 million relates to our existing leased office space and $0.1 million relates to our forward-starting lease.
Previous wording
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, costs and expenses, and the disclosure of contingent assets and liabilities in our condensed consolidated financial statements. 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, 2023 for more information about critical accounting policies as well as a description of our other significant accounting policies.
Current wording
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.
Previous wording
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 to our consolidated financial statements in our Annual Report on Form 10-K for the year ended December 31, 2023.
Current wording
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, 2024.
Rearranged previous paragraphs (3)
Previous report — wording carried elsewhere
The following table summarizes our results of operations for the nine months ended September 30, 2024 and 2023 (in thousands):
Previous report — wording carried elsewhere
The following table summarizes our research and development expenses for the nine months ended September 30, 2024 and 2023 (in thousands):
Previous report — wording carried elsewhere
Income tax expense was $0.2 million for the nine months ended September 30, 2024, compared to $0.1 million for the nine months ended September 30, 2023. This expense relates to state income tax resulting from interest income.