IRulya Therapeutics Inc.
For the period ended Mar 31, 2025. Filed May 13, 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 13, 2025
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2024-09-30; filed 2024-11-13.
0001662774-24-000011
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-03-31; filed 2025-05-13.
0001662774-25-000032
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 148 previous paragraphs and 92 current paragraphs.
34 unchanged; 33 reworded; 21 added; 73 removed; 8 previous and 4 current paragraphs rearranged.
Tables are not compared: 5 previous and 3 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 (21)
Current report
We intend to focus our development expertise and financial resources toward advancing a Phase 3 NEAT clinical trial, which is an international multicenter, randomized, double-blind, placebo-controlled study to evaluate the neurological effects of eDSP on patients with A-T. As of May 13, 2025, 63 participants have been enrolled in the Phase 3 NEAT clinical trial of eDSP, including 56 participants in the six to nine year-old primary analysis population and seven participants aged 10 years or older. 30 participants have entered the OLE study.
Current report
Absent additional funding to extend our cash runway beyond the first quarter of 2026, we are contemplating an early conclusion of enrollment for our pivotal Phase 3 NEAT clinical trial by the end of June 2025 to align the reporting of topline results with the our existing cash runway in early 2026. Several factors contribute to this potential shift in strategy, including slower than anticipated enrollment, uncertain macroeconomic environment, challenging academic site environment, and an operationally well-executed study with limited withdrawals and low rates of missing data and procedural deviations. The combination of these factors leads us to now consider concluding enrollment prior to reaching the pre-specified target of 86 patients in the primary analysis population. The potential to conclude enrollment at the end of June 2025 is expected to provide an approximate 80% power to determine a statistically significant difference in the primary endpoint. Additionally, this timeline would allow the company to report topline results by early 2026 while maintaining a positive cash balance. If additional funding is secured in the near term, we intend to continue enrollment as previously planned targeting 86 patients with A-T ages six to nine years old in the primary analysis population. Assuming positive results from the NEAT study, we plan to submit applications for approval in the U.S. and Europe in the second half of 2026.
Current report
Our strategic focus is to apply our resources and capital toward the advancement of our proprietary AIDE technology platform and Phase 3 lead asset, eDSP, targeted to treat A-T.
Current report
Interest Income
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Other Expense, net
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Critical Accounting Estimates
Current report
For a description of our significant accounting policies, see Note 2 to our consolidated financial statements.
Current report
Of our policies, the following are considered critical to an understanding of our consolidated financial statements as they require the application of subjective and complex judgment, involving critical accounting estimates and assumptions impacting our consolidated financial statements:
Current report
•Research and Development Expenses
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•Identifiable Intangible Assets
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•Contingent Consideration
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•Long-term Debt
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For a discussion about the critical accounting estimates and assumptions impacting our consolidated financial statements, see the Critical Accounting Estimates section within MD&A of our Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on March 24, 2025.
Current report
For the three months ended March 31, 2025, we recorded a fair value adjustment for contingent consideration which resulted in a $1.9 million increase primarily due to the passage of time. We owe no further payments to EryDel shareholders for development-related milestones.
Current report
Interest Income
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Other Expense, net
Current report
We evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about our ability to continue as a going concern within one year after the date that the unaudited condensed consolidated financial statements are issued. We have incurred losses and generated negative operating cash flows since our inception and anticipate that we will continue to incur losses for at least the next several years. The transition to profitability is dependent upon the successful development, approval and commercialization of our product candidate, and the achievement of a level of revenues adequate to support our cost structure. As of March 31, 2025, we had cash, cash equivalents and short-term investments of $31.6 million. Based on our current operating plan, we believe that our cash and cash equivalents balance as of March 31, 2025 will not be sufficient to fund operations and capital expenditures for the twelve months following the filing of this Quarterly Report on Form 10-Q, and we will need to obtain additional funding. We intend to obtain additional funding through available financing sources which may include additional public offerings of common stock, private financing of debt or equity, and/or the pursuit of strategic partnerships, licensing arrangements or collaborations. Management’s belief with respect to our ability to fund operations is based on estimates that are subject to risks and uncertainties. If actual results are different from management’s estimates, we may need to seek additional funding sooner than would otherwise be expected. There can be no assurance that we will be able to obtain additional funding on acceptable terms, if at all. If we are unable to obtain sufficient funding, we may be required to delay development efforts, limit activities and reduce research and development costs, which could adversely affect our business prospects. Because of the uncertainty in securing additional funding and the insufficient amount of cash and cash equivalent resources as of March 31, 2025, we concluded that substantial doubt exists with respect to our ability to continue as a going concern within one year after the date that these unaudited condensed consolidated financial statements are issued.
Current report
Financing
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Equity Financing
Current report
On December 18, 2024, the Company entered into a Controlled Equity Offering SM Sales Agreement, with Cantor Fitzgerald & Co. and H.C. Wainwright & Co., LLC, or the Agents, relating to the sale of shares of our common stock, par value $0.001 per share. In accordance with the terms of this agreement, the Company may offer and sell up to $21.9 million of shares of common stock.
Current report
During the three months ended March 31, 2025, we did not sell any shares of common stock pursuant to the Sales Agreement. During April and through May 12, 2025, the Company utilized its ATM program to raise net proceeds of approximately $2.9 million by issuing 2,766,549 shares of common stock.
Removed paragraphs (73)
Previous report
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations.
Previous report
As of November 13, 2024, 32 patients have been enrolled in the Phase 3 NEAT clinical trial of EryDex in A-T. We expect to report topline results from this trial in the fourth quarter of 2025, with a potential NDA submission in 2026, assuming positive study results.
Previous report
During the third quarter of 2024, and in accordance with the EryDel Purchase Agreement, we paid the former EryDel shareholders a cash milestone payment of $5 million related to the initiation of the Phase 3 clinical trial of EryDex in A-T. Additionally, on October 20, 2024, in accordance with the EryDel Purchase Agreement, no indemnification claim against the EryDel Shareholders had been made by the Company and all 725,037 Indemnity Holdback Shares were released to the former EryDel shareholders. No additional shares are required to be issued in connection with the EryDel Acquisition.
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Proprietary AIDE Technology Platform
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Our proprietary AIDE technology platform is a novel drug/device combination that uses an automated process designed to encapsulate a drug into the patient’s own red blood cells. The AIDE technology drug/device combination consists of a specialized automated equipment including the RCL, a sterile single-use consumable treatment kit called EryKit, Syringe Kit, drug, and process solutions. Red blood cells have several characteristics that make them a potentially ideal vehicle for drug delivery, including potentially better tolerability, enhanced tissue distribution, reduced immunogenicity, and prolongation of circulating half-life. Our AIDE technology is designed to harness these benefits to allow for the chronic administration of drugs that have limitations due to toxicity, poor biodistribution, suboptimal pharmacokinetics, or immune response. In this way, our AIDE technology may confer benefits over conventional therapies and if so, AIDE has the potential to be applied to a broad range of small or large molecule drugs and biologics. The use of autologous blood may minimize safety risks associated with the use of donor blood and may reduce the potential immunogenic risks associated donor cells and engineered cell therapies.
Previous report
The RCL is a proprietary CE marked non-invasive device that allows blood processing at the point of care. With a user-friendly touch screen interface, the RCL automates the AIDE process technology by handling the blood, drug, and processing solutions with the use of the proprietary EryKit. Our EryKit is a CE marked medical device that provides the essential single-use components for loading the patient’s red blood cells with DSP by using the RCL. The RCL, EryKit, and Syringe Kit have been CE marked in accordance with the MDR. Process solutions (Hypotonic Solution 1&2 and Hypertonic Solution PIGPA) are proprietary sterile solutions, CE marked according to the MDD, aimed to create suitable osmotic condition during the process to allow the drug diffusion into red blood cells and the subsequent restoring of physiological condition.
Previous report
The automated AIDE process and treatment is designed to be completed at the point-of-care and includes a series of steps which take approximately two hours from start to finish. This process includes:
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•Collection of patient’s blood using our proprietary Syringe Kit.
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•Processing the patient's collected blood in the RCL using our proprietary EryKit.
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•Autologous red blood cells in the RCL are swollen and their pores are “opened” in two steps using two sequential hypotonic process solutions.
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•Drug is added to the RCL and enters into the opened red blood cells.
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•Physiological osmotic conditions are then restored by adding a hypertonic solution that “reseals” the red blood cells.
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•Drug that is not encapsulated during the process is removed by extensive washing with an injectable saline solution.
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•Upon completion of the AIDE process, the drug encapsulated red blood cells are infused into the patient.
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Potential Benefits of Red Blood Cell Encapsulated Drug Delivery
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Many efficacious drugs have limited therapeutic potential because of dose limiting toxicity, while other drugs may have efficacy ceilings due to inferior biodistribution, pharmacokinetics and pharmacodynamics. Our proprietary AIDE technology uses an automated process designed to encapsulate a drug into the patient’s own red blood cells to deliver a therapy in a potentially more effective and safer way. Autologous red blood cells have several characteristics that make them an ideal vehicle for drug delivery:
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•Potential for improved biodistribution as encapsulated drug in autologous red blood cells is designed to enable the slow release of the drug from the red blood cells traversing through the body, various tissue beds, and many capillaries for desired therapeutic effect.
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•Potential for altered pharmacokinetics and pharmacodynamics, including long circulating half-life, and altered or improved tissue distribution. The altered pharmacokinetics and pharmacodynamics of the encapsulated drug delivery enabled by autologous red blood cells may significantly increase the desired therapeutic effect and/or improve the safety profile of the therapy.
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•Potential for improved biocompatibility through the use of autologous red blood cells to, thereby avoiding issues with donor compatibility.
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•Potential for the encapsulation of small or large molecules, peptides, and proteins inside of autologous red blood cells to limit biodegradability, thereby altering the encapsulated drug’s metabolism and degradation.
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•Potential for significantly decreasing adverse effects of treatments.
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New Indications and Program Expansion Potential
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The flexibility of the AIDE platform has the potential to provide benefits over conventional therapies and if so, could be applied to a broad range of drugs ranging from small to large molecules, as well as biologics. Potential benefits include better tolerability, enhanced tissue distribution, reduced immunogenicity, and prolongation of circulating half-life which we believe could be an advantage for treating patients.
Previous report
We selected Duchenne muscular dystrophy (DMD) as our second development program. We consider DMD an ideal indication for EryDex as corticosteroids are the standard of care for this rare disease, but its utility is limited by significant chronic toxicity due to adrenal suppression. As a result, corticosteroid treatment in patients with DMD is commonly interrupted during adolescence due to interference with sexual maturation and delayed puberty. As a next step, we intend to focus on generating a Phase 2 clinical trial designed to evaluate EryDex for the potential treatment of patients with DMD, including those with corticosteroid intolerance who represent the majority of the DMD population. We plan to initiate a DMD Phase 2 study in 2025, which we plan to conduct utilizing capital efficient study approaches.
Previous report
We also completed an initial assessment of other potential indications for EryDex where chronic corticosteroid treatment is – or has the potential to become – a standard of care if there were not corticosteroid-related safety concerns. This evaluation process spanned across ataxia, neuromuscular, hematology, cancer, and autoimmune disease indications with a focus on rare diseases. The prioritized list of other potential rare disease targets beyond A-T and DMD includes: 1) autoimmune hepatitis, 2) dermatomyositis, 3) pemphigus vulgaris, 4) Hashimoto's encephalopathy, 5) Becker muscular dystrophy, 6) pediatric lupus, 7) juvenile idiopathic arthritis, 8) myasthenia gravis, 9) limb-girdle muscular dystrophy, 10) chronic inflammatory demyelinating polyradiculoneuropathy, and 11) pulmonary sarcoidosis. To support our drug development pipeline expansion in a capital efficient structure, we would prioritize an investigator initiated clinical trial approach to evaluate EryDex for other potential rare disease indications. In addition, we intend to evaluate other potential applications of the AIDE technology platform using drugs and biologics targeted at rare and debilitating diseases to further expand our drug development pipeline.
Previous report
Our AIDE technology platform reflects more than 20 years of innovation and approximately $100 million of investment, which has resulted in innovation that creates high barriers to competitive entry. The RCL, EryKit, Syringe Kit, and process solutions are proprietary products and CE marked in the EU, in accordance with the MDR and MDD.
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Limitations of Chronic Corticosteroid Administration
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DSP is a corticosteroid well known for its anti-inflammatory properties as well as its dose-limiting toxicity due to adrenal suppression. Our AIDE technology is designed to encapsulate DSP in a patient’s own red blood cells and to alter the biodistribution, pharmacokinetics, and pharmacodynamics of the DSP allowing for both effective and safe treatment.
Previous report
The optimal efficacy of corticosteroids is the result of two pharmacokinetic characteristics: 1) an initial bolus to achieve a high Cmax that results in high levels of corticosteroid receptor occupation; and 2) sufficient sustained tissue concentrations that allow for continued receptor site occupancy over time.
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In order for a conventional corticosteroid to achieve these characteristics, the drug must be dosed frequently, typically daily. Long-term daily dosing regimens sufficient to provide efficacy, lead to significant and debilitating long-term adverse effects associated with HPA axis suppression. Corticosteroid therapy without significant long-term safety liabilities would represent a major advancement in the treatment of many chronic diseases where corticosteroids are already known to be beneficial.
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Phase 3 Lead Asset – EryDex for the Potential Treatment of A-T
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Our top strategic priority is to complete the Phase 3 clinical trial of EryDex, called NEAT (Neurologic Effects of EryDex on Subjects with A-T; NCT06193200 / IEDAT-04-2022), to evaluate its safety and efficacy for the potential treatment of A-T. We also plan to continue investigating additional indications for EryDex. These priorities include the following activities and corporate milestones:
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•Initiate our open label extension study in 2024.
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•Complete enrollment in our Phase 3 NEAT clinical trial in the first half of 2025.
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•Report our Phase 3 NEAT clinical trial top-line results in the fourth quarter of 2025.
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•Prepare for a U.S. NDA submission in 2026, provided we obtain positive NEAT clinical trial results.
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•Pursue potential strategic partnerships to out-license ex-U.S. regional territories to extend operational runway to support potential NDA approval of EryDex, as well as further advance other potential indications.
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Pivotal Phase 3 NEAT Clinical Trial of EryDex in Patients with A-T
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We dosed the first patient with A-T in our Phase 3 NEAT clinical trial in the second quarter of 2024. As of November 13, 2024, we have enrolled 32 patients. NEAT is an international, multi-center, randomized, double-blind, placebo-controlled study to evaluate the neurological effects of EryDex in patients with A-T. To date, we have enrolled seven patients with A-T in the NEAT clinical trial across clinical sites in the U.S., U.K., and European Union and expect NEAT site activation and patient screenings to accelerate over the next quarter. We plan to enroll approximately 86 patients with A-T ages six to nine years old randomized (1:1) between EryDex or placebo, and approximately 20 patients with A-T ages 10 years or older. Participants who complete the full treatment period, complete the study assessments, and provide informed consent will be eligible to transition to an open label extension program after trial completion. The primary efficacy endpoint for the NEAT clinical trial will be measured by the change from baseline to last visit completion in RmICARS.
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Regulatory Interactions
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Reworded paragraphs (33)
Previous wording
The following discussion and analysis of our financial condition and results of operations should be read in conjunction with (i) our unaudited condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and (ii) our audited consolidated financial statements and related notes and the section titled "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, 2023 ("Annual Report on Form 10-K") filed with the Securities and Exchange Commission (the “SEC”), on April 1, 2024. Unless the context requires otherwise, references in this Quarterly Report on Form 10-Q to the “Company,” “Quince,” “we,” “us” and “our” refer to Quince Therapeutics, Inc. and "our legacy assets" refer to atuzaginstat (COR388), COR588, COR852, and COR803, collectively.
Current wording
You should read the following discussion and analysis of our financial condition and results of operations together with our unaudited condensed consolidated financial statements and related notes, included in “Part I. Item 1. Financial Statements” of this Quarterly Report on Form 10-Q. This discussion contains forward-looking statements that involve risk and uncertainties, such as statements of our plans, objectives, expectations, and intentions, that are based on the beliefs of our management. Our actual results could differ materially from those discussed in these forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those discussed in the “Risk Factors” section of this Quarterly Report on Form 10-Q and our Annual Report on Form 10-K. Unless the context requires otherwise, references in this Quarterly Report on Form 10-Q to the “Company,” “Quince,” “we,” “us,” and “our” refer to Quince Therapeutics, Inc. and its consolidated subsidiaries.
Previous wording
We are a late-stage biotechnology company dedicated to unlocking the power of a patient’s own biology for the treatment of rare diseases.
Current wording
We are a late-stage biotechnology company dedicated to unlocking the power of a patient’s own biology for the treatment of rare diseases. Our proprietary AIDE technology is an innovative drug/device combination platform that uses an automated process to encapsulate a drug into a patient’s own red blood cells. Red blood cells have several characteristics that make them an excellent vehicle for drug delivery potential including better safety and tolerability, enhanced tissue distribution, reduced immunogenicity, and prolongation of circulating half-life. Our AIDE technology is designed to harness many of these benefits to allow for new and improved therapeutic options for patients living with high unmet medical needs. eDSP is the first product in development that leverages our AIDE technology and is composed of DSP encapsulated in autologous red blood cells targeted to treat a rare pediatric neurodegenerative disease, A-T. DSP is a corticosteroid well-described for its anti-inflammatory properties, but is also coupled with serious adverse events, including adrenal suppression. eDSP is designed to maintain the well-described efficacy of DSP while reducing or eliminating the significant adverse events that accompany chronic corticosteroid treatment. The altered biodistribution, pharmacokinetics, and pharmacodynamics of eDSP enabled by autologous red blood cells may, therefore, improve the safety profile, and maintain or increase the desired therapeutic effect of DSP.
Previous wording
We also completed a patient sizing project with third-party analysis from IQVIA Medical Claims (Dx), PharmetricsPlus (P+), and IQVIA Analytics, which confirmed that there are approximately 4,600 diagnosed patients with A-T in the U.S. Currently, there are no approved treatments for A-T and the global market, based on our internal estimates and assumptions, represents a more than $1 billion peak commercial opportunity. We believe this could allow EryDex to demonstrate the clinical benefit and commercial potential of our AIDE technology.
Current wording
Currently, there are no approved treatments for A-T and the global market, based on our internal estimates and assumptions, represents a more than $1 billion peak commercial opportunity. We believe this makes eDSP an ideal lead asset to demonstrate the clinical and commercial potential of our AIDE technology.
Previous wording
With the acquisition of EryDel, we expect our research and development expenses to remain in the current levels as we continue our Phase 3 NEAT clinical trial and potentially expand into new indications.
Current wording
We expect our research and development expenses to remain in the current levels as we continue our Phase 3 NEAT clinical trial and expand into new indications, including Phase 2 study in DMD.
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We record fair value adjustment for contingent consideration primarily due to the passage of time related to the contingent consideration earnout resulting from the EryDel Acquisition. Changes in the fair value of the contingent consideration obligations may result from changes in probability assumptions with respect to the likelihood of achieving the various contingent payment obligations.
Current wording
We record fair value adjustment for contingent consideration primarily due to the expected timing of achieving various milestones, and the passage of time related to the contingent consideration earnout resulting from the EryDel Acquisition. Changes in the fair value of the contingent consideration obligations may result from changes in probability assumptions with respect to the likelihood of achieving the various contingent payment obligations.
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Interest and other income, net consists primarily of interest earned on our short-term and long-term investments portfolio.
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Interest income consists primarily of interest earned on our short-term and long-term investments portfolio.
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Research and development expenses were $4.9 million for the three months ended September 30, 2024, compared to $1.4 million for the three months ended September 30, 2023, an increase of $3.5 million.
Current wording
Research and development expenses were $8.1 million for the three months ended March 31, 2025, compared to $3.7 million for the three months ended March 31, 2024, an increase of $4.4 million.
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The costs for EryDex development increased by $8.9 million compared to the same period from the prior year due to the start-up and ramping up costs related to our Phase 3 NEAT clinical trial. This increase was primarily due to clinical trial costs of $8.1 million and $0.8 million in manufacturing costs.
Current wording
The costs for eDSP development increased by $3.4 million compared to the same period from the prior year due to the ramping up and continuation costs related to our Phase 3 NEAT clinical trial and OLE. This increase was primarily due to clinical trial costs of $6.2 million and $0.1 million in manufacturing costs.
Previous wording
Our personnel related costs decreased by $0.1 million during the nine months ended September 30, 2024 as compared to the nine months ended September 30, 2023, mainly as a result of a $0.6 million decrease in allocated stock-based compensation costs, offset by a $0.5 million increase in personnel related expenses.
Current wording
Our personnel related costs increased by $0.9 million during the three months ended March 31, 2025 as compared to the three months ended March 31, 2024, mainly as a result of incurring a $0.4 million severance costs and $0.3 million in allocated stock-based compensation costs.
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Facilities and other research and development expenses increased by $31.0 thousand for the three months ended September 30, 2024, as compared to the three months ended September 30, 2023 primarily due to an increase in rent and facilities expenses.
Current wording
Facilities and other research and development expenses decreased by $39 thousand for the three months ended March 31, 2025, as compared to the three months ended March 31, 2024 primarily due to a decrease in storage and facilities expenses.
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General and administrative expenses decreased by $1.03 million to $3.63 million for the three months ended September 30, 2024, from $4.66 million for the three months ended September 30, 2023. The decrease in general and administrative expenses is primarily due to a decrease of $0.9 million in consulting and professional fees, $0.5 million decrease in legal fees, and $0.1 million decrease in corporate insurance. These decreases were partially offset by an increase of $0.1 million in personnel related expenses, $0.1 million increase in audit and tax fees, $0.1 million increase in commercial and new product planning expenses, and $0.1 million increase in other professional and administrative expenses year over year.
Current wording
General and administrative expenses decreased by $0.2 million for the three months ended March 31, 2025 as compared to the three months ended March 31, 2024. The change in general and administrative expenses is primarily due to a decrease of $0.1 million in consulting and professional expenses and $0.2 million in other professional and administrative expenses. These decreases were partially offset by an increase of $0.1 million in personnel-related expenses.
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For the three months ended September 30, 2024, we recorded a fair value adjustment for long-term debt which resulted in a $0.4 million charge primarily due to the passage of time and the interest accrued for the loan with the EIB.
Current wording
For the three months ended March 31, 2025, we recorded a fair value adjustment for long-term debt which resulted in a $0.4 million charge primarily due to the passage of time and the interest accrued and paid for the loan with the EIB.
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Interest income decreased by $276.0 thousand for the three months ended September 30, 2024, as compared to the three months ended September 30, 2023. The change was due to a decreased yields on our investment portfolio which and decreased average balances.
Current wording
Interest income decreased by $0.5 million for the three months ended March 31, 2025, as compared to the three months ended March 31, 2024. The change was due to decreased yields on our investment portfolio and decreased average balances.
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Other income (expense), net increased by $0.4 million for the three months ended September 30, 2024 primarily due to $0.3 million unrealized gains resulting from changes in foreign exchange rates.
Current wording
Other expense, net decreased by $0.3 million for the three months ended March 31, 2025 primarily due to the effects resulting from changes in foreign exchange rates.
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We recorded tax expense of $0.1 million for the nine months ended September 30, 2024 and tax benefit of $0.2 million for the nine months ended September 30, 2023. The change is primarily related to foreign withholding tax, interest on uncertain tax position liability and net of tax benefit from decrease in EryDel's deferred tax liabilities.
Current wording
We recorded tax expense of $47 thousand for the three months ended March 31, 2025 and $31 thousand for the three months ended March 31, 2024. Tax expense is primarily driven by foreign withholding tax, interest on uncertain tax position liability and net of tax benefit from decrease in EryDel's deferred tax liabilities.
Previous wording
To date, we have financed our operations primarily through the issuance and sale of convertible promissory notes and redeemable convertible preferred stock and common stock. From inception through September 30, 2024, we received net proceeds of approximately $303.9 million from the issuance of redeemable convertible preferred stock, convertible promissory notes and common stock.
Current wording
We have not generated any revenue and we have never been profitable. To date, we have financed our operations primarily through the issuance and sale of convertible promissory notes and redeemable convertible preferred stock and common stock. From inception through March 31, 2025, we received net proceeds of approximately $304.2 million from the issuance of redeemable convertible preferred stock, convertible promissory notes and common stock.
Previous wording
To date, we have not generated any revenue and we have never been profitable. We have incurred net losses since the commencement of our operations. As of September 30, 2024, we had an accumulated deficit of $364.0 million. We incurred a net loss of $44.4 million in the nine months ended September 30, 2024. We do not expect to generate product revenue unless and until we obtain marketing approval for and commercialize a drug candidate, and we cannot assure you that we will ever generate significant revenue or profits.
Current wording
We have incurred net losses since the commencement of our operations. As of March 31, 2025, we had an accumulated deficit of $391.5 million. We incurred a net loss of $15.0 million in the three months ended March 31, 2025. We do not expect to generate product revenue unless and until we obtain marketing approval for and commercialize a drug candidate, and we cannot assure you that we will ever generate significant revenue or profits.
Previous wording
As of September 30, 2024 and December 31, 2023, we had cash, cash equivalents and short-term investments of $47.8 million and $75.1 million, respectively. Based on our current business plans, we believe that our existing capital resources will be sufficient to fund our projected operating requirements for at least the next twelve months from the date of the issuance of the accompanying condensed consolidated financial statements. Our cash, cash equivalents, and marketable debt securities are held in a variety of deposit accounts, interest-bearing accounts, U.S government securities, debt securities in government-sponsored entities, and money market funds. Cash in excess of immediate requirements is invested with a view toward liquidity and capital preservation, and we seek to minimize the potential effects of concentration and credit risk. Our cash equivalents and short-term investments are held in money market funds, certificate of deposits, and government agency obligations.
Current wording
Our cash, cash equivalents, and marketable debt securities are held in a variety of deposit accounts, interest-bearing accounts, U.S government securities, debt securities in government-sponsored entities, and money market funds. Cash in excess of immediate requirements is invested with a view toward liquidity and capital preservation, and we seek to minimize the potential effects of concentration and credit risk. Our cash equivalents and short-term investments are held in money market funds, certificate of deposits, and government agency obligations.
Previous wording
We believe that our existing cash, cash equivalents and investments will be sufficient to fund our planned operations into the first quarter of 2026, but does not include any costs or cash expenditures associated with initiating additional programs. We have based this estimate on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we expect.
Current wording
While we currently believe that our existing cash, cash equivalents and investments will be sufficient to fund our planned operations through Phase 3 NEAT topline results expected in early 2026, that assumption does not include any costs or cash expenditures associated with initiating additional programs.
Previous wording
Our primary use of cash is to fund operating expenses, which consist primarily of research and development expenditures related to EryDex, the initiation and continuation of the Phase 3 NEAT clinical trial, and general and administrative expenditures. Cash used to fund operating expenses is impacted by the timing of when we pay these expenses, as reflected in the change in our outstanding accounts payable and accrued expenses.
Current wording
Our primary use of cash is to fund operating expenses, which consist primarily of research and development expenditures related to eDSP, the initiation and continuation of the Phase 3 NEAT clinical trial and OLE, and general and administrative expenditures. Cash used to fund operating expenses is impacted by the timing of when we pay these expenses, as reflected in the change in our outstanding accounts payable and accrued expenses.
Previous wording
We may continue to require additional capital to develop our drug candidates and fund operations for the foreseeable future. We may seek to raise capital through private or public equity or debt financings, collaborative or other arrangements with other companies, or through other sources of financing. Adequate additional funding may not be available to us on acceptable terms or at all. Our failure to raise capital as and when needed could have a negative impact on our financial condition and our ability to pursue our business strategies. Following our acquisition of EryDel, we anticipate that we will need to raise substantial additional capital, the requirements of which will depend on many factors, including:
Current wording
We expect that we will require additional capital to develop our drug candidates and fund operations for the foreseeable future. We may seek to raise capital through private or public equity or debt financings, collaborative or other arrangements with other companies, or through other sources of financing. Adequate additional funding may not be available to us on acceptable terms or at all. Our failure to raise capital as and when needed could have a negative impact on our financial condition and our ability to pursue our business strategies. We anticipate that we will need to raise substantial additional capital, the requirements of which will depend on many factors, including:
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•payment of future milestones to EryDel shareholders and payments to the EIB in respect of obligations under the EIB Facility;
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•payment of future milestones to EryDel shareholders and payments to the EIB in respect of obligations under the EIB Loan;
Previous wording
If we raise additional funds by issuing equity securities, our stockholders will experience dilution. Any future debt financing into which we enter may impose upon us additional covenants that restrict our operations, including limitations on our ability to incur liens or additional debt, pay dividends, repurchase our common stock, make certain investments and engage in certain merger, consolidation or asset sale transactions. Any debt financing or additional equity that we raise may contain terms that are not favorable to us or our stockholders. If we are unable to raise additional funds when needed, we may be required to delay, reduce, or terminate some or all of our development programs and clinical trials. We may also be required to sell or license to other rights to our drug candidates in certain territories or indications that we would prefer to develop and commercialize ourselves.
Current wording
If we raise additional funds by issuing equity securities, our stockholders will experience dilution. Any future debt financing into which we enter may impose upon us additional covenants that restrict our operations, including limitations on our ability to incur liens or additional debt, pay dividends, repurchase our common stock, make certain investments and engage in certain merger, consolidation or asset sale transactions. Any debt financing or additional equity that we raise may contain terms that are not favorable to us or our stockholders. If we are unable to raise additional funds when needed, we may be required to delay, reduce, or terminate some or all of our development programs and clinical trials. We may also be required to sell or license to others rights to our drug candidates in certain territories or indications that we would prefer to develop and commercialize ourselves.
Previous wording
In connection with the acquisition of EryDel on October 20, 2023, we assumed an unsecured line of credit between EryDel and the European Investment Bank (the "EIB Loan"). The EIB Loan was amended and restated as of the acquisition date. The EIB Loan provides for maximum borrowings of 30.0 million euro through four tranches; tranche A, 3.0 million euro; tranche B, 7.0 million euro; tranche C, 10.0 million euro; and tranche D, 10.0 million euro. Each tranche is subject to conditions precedent related to our business and capitalization. As of September 30, 2024, only tranches A and B have been drawn. All amounts due under tranche A and B are payable on their maturity date of August 2026. Tranche C and D are payable in equal installments of principal together with all amounts outstanding under the tranches on the repayment dates specified in the relevant Disbursement Offer. The first Repayment Date of tranche C shall fall not earlier than twelve months from the Disbursement Date of such tranche. The last Repayment Date of tranche C and tranche D shall fall not later than 5 years from the Disbursement Date of tranche C and tranche D, respectively. The EIB Loan bears interest at fixed rates for each tranche and is payable on the maturity date for each Tranche. The fixed rates range from 7.0% to 9.0% per annum. As of September 30, 2024, principal of 10.0 million euros ($11.2 million) was outstanding on the EIB Loan and it is recorded as Long-term debt on the condensed consolidated balance sheet at fair value with imputed interest of 9.0% included.
Current wording
In connection with the acquisition of EryDel on October 20, 2023, we guaranteed the EIB Loan. The EIB Loan was amended and restated as of the acquisition date. The EIB Loan provides for maximum borrowings of 30.0 million euro through four tranches; tranche A, 3.0 million euro; tranche B, 7.0 million euro; tranche C, 10.0 million euro; and tranche D, 10.0 million euro. Each tranche is subject to conditions precedent related to our business and capitalization. As of March 31, 2025, only tranches A and B have been drawn. All amounts due under tranche A and B are payable on their maturity date of August 2026. Tranche C and D are payable in equal installments of principal together with all amounts outstanding under the tranches on the repayment dates specified in the relevant disbursement offer. The first repayment date of tranche C shall fall not earlier than twelve months from the disbursement date of such tranche. The last repayment date of tranche C and tranche D shall fall not later than 5 years from the disbursement date of tranche C and tranche D, respectively. The EIB Loan bears interest at fixed rates for each tranche and both principal and interest are payable on the maturity date for each tranche (with the exception of 2% cash interest which shall accrue and be payable quarterly during fiscal year 2025 pursuant to the terms of the Amendment, which shall correspondingly reduce the deferred interest rate accruing during such period). The fixed rates range from 7.0% to 9.0% per annum. As of March 31, 2025, principal of 10.0 million euros ($10.8 million) was outstanding on the EIB Loan and it is recorded as Long-term debt on the consolidated balance sheet at fair value with imputed interest of 9.0% included.
Previous wording
We may voluntarily prepay, in whole or in part with a prepayment premium. In the event of an occurrence of an event of default, a change in control and certain other prepayment events, as specified in the Debt Agreement, we will be required to prepay the outstanding EIB Debt together with an additional remuneration buyout fee, as specified in the Debt Agreement. The Debt Agreement includes a provision for additional remuneration to be paid in addition to interest. The amount of additional remuneration to be paid is equal to 2.5% of revenue up to 125.0 million euros, plus 1.85% of revenue between 125.0 and 250.0 million euros, plus 1.0% of revenue in excess of 250.0 million euros, multiplied by a varying percentage based on how many tranches have been drawn. The varying percentage is equal to 30.0% in the event tranche A has been drawn, 50.0% in the event tranche A and B have been drawn, 80.0% in the event tranche A, B and C have been drawn, and 100.0% in the event all four tranches have been drawn. The additional remuneration is payable for seven years, during the period January 1, 2026, through December 31, 2032. In the event of an occurrence of an event of default or prepayment, we may be required to pay an additional remuneration buyout fee.
Current wording
We may voluntarily prepay, in whole or in part with a prepayment premium. In the event of an occurrence of an event of default, a change in control and certain other prepayment events, as specified in the Debt Agreement, we will be required to prepay the outstanding EIB Loan together with an additional remuneration buyout fee, as specified in the Debt Agreement. The Debt Agreement includes a provision for additional remuneration to be paid in addition to interest. The amount of additional remuneration to be paid is equal to 2.5% of revenue up to 125.0 million euros, plus 1.85% of revenue between 125.0 and 250.0 million euros, plus 1.0% of revenue in excess of 250.0 million euros, multiplied by a varying percentage based on how many tranches have been drawn. The varying percentage is equal to 30.0% in the event tranche A has been drawn, 50.0% in the event tranche A and B have been drawn, 80.0% in the event tranche A, B and C have been drawn, and 100.0% in the event all four tranches have been drawn. The additional remuneration is payable for seven years, during the period January 1, 2026, through December 31, 2032. In the event of an occurrence of an event of default or prepayment, we may be required to pay an additional remuneration buyout fee.
Previous wording
In November 2024, the Company entered into an amendment (the “Amendment”) of the Debt Agreement with EIB which waives the Minimum Cash Covenant from January 1, 2025 and up to the earlier of December 31, 2025, or the date the Minimum Cash Covenant is restored. Under the terms of the Amendment, the Company agreed to amendments requiring monthly reporting of cash balances and additional limitations on certain permitted acquisitions. Additionally, during the waiver period, the Company agreed to convert 2% of the total 9% deferred interest on Tranches A and B to be payable quarterly, with the first payment due on March 31, 2025, and a one-time fee of 20,000 euros in connection with the Amendment.
Current wording
The Debt Agreement requires us to maintain a minimum cash balance of 14.65 million euros ($15.8 million) until the outstanding obligations under the Debt Agreement, together with accrued interest and all other amounts accrued or outstanding under the agreement, is repaid in full (the “Minimum Cash Covenant”). In November 2024, we entered into an amendment (the “Amendment”) of the Debt Agreement with EIB which waives the Minimum Cash Covenant from January 1, 2025, and up to the earlier of December 31, 2025, or the date the Minimum Cash Covenant is restored (such period, the “Waiver Period”). Under the terms of the Amendment, we agreed to amendments requiring monthly reporting of cash balances and additional limitations on certain permitted acquisitions. Additionally, solely during the waiver period, 2% cash interest on the outstanding principal amounts of tranches A and B are payable on March 31, 2025, June 30, 2025, September 30, 2025 and December 31, 2025, reducing the total deferred interest on tranches A and B to 7% for the duration of the Waiver Period, and a one-time fee of 20,000 euros in connection with the Amendment. As of March 31, 2025, we have been in compliance with all covenants under the Debt Agreement.
Previous wording
Cash used in operating activities was $12.2 million for the nine months ended September 30, 2023. Cash used in operating activities was primarily due to our net loss of $22.5 million for the period, adjusted for $8.6 million of non-cash items, including $4.1 million in stock-based compensation and $5.9 million in impairment loss due to the write down of the IPR&D asset, as well as a net increase in our current assets of $2.4 million, offset by a net decrease in accounts payable, accrued expenses and other current liabilities of $0.7 million.
Current wording
Net cash used in operating activities was $9.6 million for the three months ended March 31, 2025. Cash used in operating activities was primarily due to our net loss of $15.0 million for the period, adjusted for $3.4 million of non-cash items, including $1.9 million change in the fair value of contingent consideration liabilities, $1.4 million in stock-based compensation, $0.4 million change in the fair value of the EIB Loan and a net decrease in our operating assets of $1.1 million, offset by a net increase in our accounts payable, and accrued expenses and other current liabilities of $0.9 million.
Previous wording
Cash used in operating activities was $24.4 million for the nine months ended September 30, 2024. Cash used in operating activities was primarily due to our net loss of $44.4 million for the period, adjusted for $22.4 million of non-cash items, including $17.1 million goodwill impairment charge, $2.1 million change in the fair value of contingent consideration liabilities, $3.6 million in stock-based compensation, and a net increase in our operating assets of $2.5 million, offset by a net increase in our accounts payable, and accrued expenses and other current liabilities of $0.1 million.
Current wording
Net cash used in operating activities was $8.4 million for the three months ended March 31, 2024. Cash used in operating activities was primarily due to our net loss of $11.1 million for the period, adjusted for $3.6 million of non-cash items, including $2.5 million change in the fair value of contingent consideration liabilities, $1.3 million in stock-based compensation, and a net decrease in accounts payable, accrued expenses and other current liabilities of $0.3 million, partially offset by increases in our current assets of $0.5 million.
Previous wording
Cash provided by investing activities was $11.0 million for the nine months ended September 30, 2024, primarily related to the maturities of short-term investments of $100.7 million, and the purchase of investments of $89.6 million.
Current wording
Cash provided by investing activities was $10.9 million for the three months ended March 31, 2025, primarily related to the maturities of short-term investments of $13.0 million, and the purchase of investments of $2.0 million.
Previous wording
Cash used by investing activities was $11.1 million in the nine months ended September 30, 2023, primarily related to the purchase of investments of $95.1 million, and maturities of investments of $84.5 million.
Current wording
Cash provided by investing activities was $8.9 million in the three months ended March 31, 2024, primarily related to the maturities of short-term investments of $48.2 million, and the purchase of investments of $39.3 million.
Previous wording
Cash used in financing activities was $4.8 million for the nine months ended September 30, 2024, which consisted of a cash milestone payment of $5 million, partially offset by proceeds of $0.2 million from the exercise of stock options in the period.
Current wording
Cash used in financing activities was $0.1 million for the three months ended March 31, 2025, which consisted of net proceeds from the exercise of stock options in the period.
Previous wording
Cash provided by financing activities was $0.1 million in the nine months ended September 30, 2023, which consisted of net proceeds from the exercise of stock options in the period.
Current wording
Cash provided by financing activities was $0.2 million in the three months ended March 31, 2024, which consisted of net proceeds from the exercise of stock options in the period.
Previous wording
We enter into contracts in the normal course of business with third party contract organizations for clinical trials, non-clinical studies and testing, manufacturing, and other services and products for operating purposes. The amount and timing of the payments under these contracts varies based upon the timing of the services. We have recorded accrued expense of approximately $1.5 million in our condensed consolidated balance sheet for expenditures incurred by these vendors as of September 30, 2024. We have approximately $27.5 million in cancellable future operating expense commitments based on existing contracts as of September 30, 2024. These obligations will be satisfied in the normal course of business, but generally no longer than 12 months. As of September 30, 2024, the fair value of the EIB loan is $14.9 million and it is recorded as long-term debt on the condensed consolidated balance sheet at fair value. As of September 30, 2024, the fair value of long-term contingent consideration on our books for the earnout related to the EryDel Acquisition is $54.8 million, refer to Note 3 to the condensed consolidated financial statements for further details.
Current wording
We enter into contracts in the normal course of business with third party contract organizations for clinical trials, non-clinical studies and testing, manufacturing, and other services and products for operating purposes. The amount and timing of the payments under these contracts varies based upon the timing of the services. We have recorded accrued expense of approximately $5.3 million in our condensed consolidated balance sheet for expenditures incurred by these vendors as of March 31, 2025. We have approximately $22.8 million in cancellable future operating expense commitments based on existing contracts as of March 31, 2025. These obligations will be satisfied in the normal course of business, but generally no longer than 12 months. As of March 31, 2025, the fair value of the EIB Loan is $15.3 million and it is recorded as long-term debt on the condensed consolidated balance sheet at fair value. As of March 31, 2025, the fair value of long-term contingent consideration on our books for the earnout related to the EryDel Acquisition is $58.6 million, refer to Note 3 to the condensed consolidated financial statements for further details.
Rearranged current paragraphs (4)
Current report — wording carried from elsewhere
Fair Value Adjustment for Contingent Consideration
Current report — wording carried from elsewhere
Fair Value Adjustment for Long-term Debt
Current report — wording carried from elsewhere
Fair Value Adjustment for Contingent Consideration
Current report — wording carried from elsewhere
Fair Value Adjustment for Long-term Debt
Rearranged previous paragraphs (8)
Previous report — wording carried elsewhere
Our proprietary AIDE technology platform is an innovative drug/device combination platform that uses an automated process to encapsulate a drug into a patient’s own red blood cells. Red blood cells have several characteristics that make them a potentially ideal vehicle for drug delivery, including potentially better tolerability, enhanced tissue distribution, reduced immunogenicity, and prolongation of circulating half-life. Our AIDE technology is designed to harness many of these benefits to allow for new and improved therapeutic options for patients living with high unmet medical needs. The AIDE technology platform may confer benefits over conventional therapies and if so, AIDE has the potential to be applied to a broad range of small or large molecule drugs and biologics.
Previous report — wording carried elsewhere
EryDex is the first product in development that leverages our AIDE technology and is composed of DSP encapsulated in autologous red blood cells targeted to treat a rare pediatric neurodegenerative disease called A-T. DSP is a corticosteroid well known for its anti-inflammatory properties, as well as its dose-limiting toxicity due to adrenal suppression. EryDex is designed to alter the pharmacokinetics and biodistribution of the DSP allowing for effective and safe treatment.
Previous report — wording carried elsewhere
The costs for EryDex development increased by $3.2 million in the three months ended September 30, 2024 compared to the same period from the prior year due to the initiation of the Phase 3 NEAT clinical trial. This increase was primarily due to clinical trial costs of $2.9 million and $0.3 million manufacturing costs.
Previous report — wording carried elsewhere
We recorded tax expense of $13.0 thousand for the three months ended September 30, 2024, primarily related to foreign withholding tax, interest on uncertain tax position liability and net of tax benefit from decrease in EryDel's deferred tax liabilities.
Previous report — wording carried elsewhere
The following sets forth our results of operations for the nine months ended September 30, 2024 and 2023 (in thousands, except for percentages):
Previous report — wording carried elsewhere
Research and Development Expenses(in thousands, except for percentages):
Previous report — wording carried elsewhere
Interest income decreased by $0.1 million for the nine months ended September 30, 2024, as compared to the nine months ended September 30, 2023. The change was due to decreased yields on our investment portfolio and decreased average balances.
Previous report — wording carried elsewhere
As of September 30, 2024, we have been in compliance with all covenants under the Debt Agreement.