Inhibikase Therapeutics, Inc.
For the period ended Mar 31, 2025. Filed May 14, 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 14, 2025
IKT$1.50+4.2%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2024-09-30; filed 2024-11-14.
0000950170-24-126766
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-03-31; filed 2025-05-14.
0000950170-25-071635
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 141 previous paragraphs and 104 current paragraphs.
58 unchanged; 21 reworded; 25 added; 60 removed; 2 previous and 0 current paragraphs rearranged.
Tables are not compared: 4 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 (25)
Current report
You should read the following discussion and analysis of our financial condition and results of operations together with our condensed consolidated financial statements and the related notes appearing elsewhere in this Quarterly Report on Form 10-Q (this “Quarterly Report”) and our audited financial statements and related notes thereto for the year ended December 31, 2024 included in our Annual Report on Form 10-K for the year ended December 31, 2024, which was filed with the Securities and Exchange Commission (the “SEC”) on March 27, 2025 (the “Annual Report”). Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report, including information with respect to our plans and strategy for our business, includes forward-looking statements that involve risks, uncertainties, and assumptions. Factors that might cause future results to differ materially from those projected in the forward-looking statements include, but are not limited to, those set forth in our Annual Report and in other filings with the SEC.
Current report
We are a clinical-stage pharmaceutical company developing therapeutics to modify the course of cardiopulmonary diseases namely, Pulmonary Arterial Hypertension (“PAH”), that arise from aberrant signaling through the Abelson Tyrosine Kinase, and type III receptor tyrosine kinases including platelet derived growth factor receptors and c-KIT. Our lead product candidate is IKT-001, a prodrug of imatinib mesylate, for PAH which is an orphan indication. We have completed non-human primate safety studies and a bioequivalence clinical trial in healthy volunteers to determine the doses of IKT-001 that are equivalent to imatinib mesylate and the results are being utilized to set the doses for a forthcoming Phase 2b study for PAH that we expect to initiate in the second half of 2025.
Current report
IKT-001 and PAH
Current report
IKT-001 emerged from our medicinal chemistry program that aimed to develop improvements to drugs that inhibit Abelson Tyrosine Kinase and type III receptor tyrosine kinases. IKT-001, a prodrug of imatinib mesylate, was designed to improve areas of the molecule that might play a role in the gastrointestinal (“GI”) side effects commonly observed with oral imatinib mesylate, the current standard of care. A three-part dose finding/dose equivalence study in 66 healthy volunteers (known as ‘the 501 trial’) was completed with IKT-001 in 2023. The study was designed to evaluate the 96-hour single-dose pharmacokinetics of imatinib delivered as IKT-001 and determine the dose relationship between IKT-001 and imatinib mesylate. Based on this study it was determined that bioequivalence was established with a 300 mg dose of IKT-001 to a dose of 230 mg of imatinib mesylate while a 500 mg dose of IKT-001 was established as bioequivalent to a dose of 383 mg of imatinib mesylate. These doses are adequate to cover the target systemically and were similar to the doses of imatinib mesylate used in the Phase 3 IMPRES trial in PAH.
Current report
PAH is a rare disease of the pulmonary microvasculature found in 15 to 50 persons per million within the United States and Europe. The global PAH market size was valued at $7.66 billion in 2023 and is estimated to grow at a compound annual growth rate of 5.4% between 2024 to 2030. Most of the treatments that constitute the standard of care (e.g. ERAs, PDE5s, prostacyclins) primarily act as vasodilators. In 2024, sotatercept was approved for the treatment of PAH on top of standard-of-care (“SOC”). Sotatercept is recombinant fusion protein that acts as a trap for transforming growth factor-beta superfamily ligands, including activin A and bone morphogenetic protein 9. These ligands may play a role in the development and progression of PAH by promoting cell proliferation and fibrosis.
Current report
The success of sotatercept has created renewed enthusiasm around the anti-proliferative pathways in PAH. As previously mentioned, imatinib inhibits Abelson Tyrosine Kinase and type III receptor tyrosine kinases and through these pathways inhibits platelet-derived growth factor receptor which is involved in cell proliferation and angiogenesis as well as stem cell factor receptor which targets mast cells and other hematopoietic progenitors. Through these targets imatinib may inhibit vascular smooth muscle cell proliferation and fibrosis. This pathway may provide an alternate pathway for disease modification in PAH.
Current report
The first reports of the use of imatinib in PAH were published in 2005 and 2006. A Phase 2, randomized control trial, was subsequently conducted showing clinical benefit of imatinib in PAH. In 2013, the outcome of a Phase 3 trial (IMPRES) evaluating imatinib mesylate as a treatment for PAH was reported, demonstrating that imatinib may improve key parameters associated with PAH. In this study, imatinib improved exercise capacity and hemodynamics in patients with advanced PAH but approval was precluded because of the bleeding risk associated with concomitant anti-coagulant therapy and the high discontinuation rate in the imatinib group.
Current report
More recently, a contemporary study published in the American Journal of Respiratory and Critical Care Medicine in an open label, single arm, four (4) center study with seventeen (17) patients confirmed that imatinib 100-400mg was well tolerated (with zero discontinuations for tolerance or side effects) in WHO Group 1 PAH population, and demonstrated a treatment effect on total pulmonary resistance (“TPR”) that compares favorably with other novel therapies for PAH, including sotatercept and seralutinib. Acknowledging the limitations of cross study comparisons and the small size of the study, we believe this recent study supports our thesis that systemic exposure of imatinib can be well tolerated and provide strong efficacy to patients suffering from PAH.
Current report
We currently have commercialization rights to IKT-001 and patent protection in the United States until 2033 for IKT-001 with upcoming patent application filings potentially extending patent protection for certain methods of treatment using IKT-001 until 2045.
Current report
Research and Development
Current report
Selling, General and Administrative
Current report
Research and Development
Current report
Selling, General and Administrative
Current report
Selling, general and administrative expenses increased by $3,218,210 or 158.4% to $5,249,291 from $2,031,081 in the prior comparable period. The $3.2 million increase was primarily driven by an increase of $1.0 million in legal, consulting and compliance related fees, an increase of $1.5 million in stock-based compensation and an increase of $0.7 million in personnel related costs including severance costs for the Company's former Chief Executive Officer.
Current report
Change in Fair Value Contingent Consideration
Current report
Change in fair value contingent consideration increased by $1,164,864 or 100% from $0 in the prior comparable period. The increase is a result of the change in fair value of the contingent consideration related to the CorHepta transaction from the transaction date on February 21, 2025 to March 31, 2025.
Current report
Interest income increased by $786,546 or 592.6% to $919,271 from $132,725 in the prior comparable period. The increase was driven by interest earned on our cash, cash equivalents and marketable securities.
Current report
At March 31, 2025, we had cash, cash equivalents and marketable securities of $93,180,291.
Current report
Cash Provided by (Used in) Investing Activities
Current report
Contingent Consideration Liabilities
Current report
We evaluate acquisitions of assets and other similar transactions to assess whether or not the transaction should be accounted for as a business combination or asset acquisition by first applying a screen test to determine if substantially all of the fair value of the gross assets acquired is concentrated in a single identifiable asset or group of similar identifiable assets. If the screen is met, the transaction is accounted for as an asset acquisition. Significant judgment is required in the application of the screen test to determine whether an acquisition is a business combination or an acquisition of assets.
Current report
On February 21, 2025, we entered into an Agreement and Plan of Merger and Reorganization (“Merger Agreement”) with Project IKT Merger Sub, Inc., a Delaware corporation and our wholly-owned subsidiary (“Merger Sub”) and CorHepta Pharmaceuticals, Inc. a Delaware corporation (“CorHepta”). We determined that the transaction represented an asset acquisition as defined by ASC 805 as substantially all of the value was attributed to a single intangible asset, in-process research and development (“IPR&D”).
Current report
The fair value was determined based on our share price at closing. We agreed to issue 4,979,101 shares of our common stock to the shareholders of CorHepta, of which (i) 829,849 shares were fully vested on the acquisition date, (ii) 2,489,030 shares represented contingent consideration and will vest no later than February 21, 2026, subject to the achievement of certain milestones, and (iii) 1,660,222 shares represented post-merger compensation expense, subject to both service- and performance-based vesting conditions.
Current report
As of the acquisition date, the achievement of one of the contingent consideration milestones was deemed probable, and the fair value of the related shares was included in the purchase price of the acquisition. We remeasure the initial contingent consideration recognized at acquisition to fair value at each reporting date and recorded a change in fair value of $1,164,864 for the period ended March 31, 2025, which is included within operating expenses. We will recognize a contingent consideration liability and corresponding expense for the remaining contingent consideration shares in future periods when it is probable that a liability has been incurred and the amount of that liability can be reasonably estimated.
Current report
The IPR&D had not reached technological feasibility and had no alternative future use at the acquisition date, and therefore, the acquired IPR&D asset of $7,357,294 was written-off as research and development expense in our condensed consolidated statement of operations and comprehensive loss immediately following the acquisition in accordance with ASC 730.
Removed paragraphs (60)
Previous report
Forward-Looking Statements
Previous report
This Quarterly Report on Form 10-Q (“Report”) (including but not limited to this Item 2, “Management’s Discussion and Analysis of Financial Condition and Results of Operations”) contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), that are intended to qualify for the “safe harbor” created by those sections. In addition, we may make forward-looking statements in other documents filed with or furnished to the SEC, and our management and other representatives may make forward-looking statements orally or in writing to analysts, investors, representatives of the media and others. 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 the related notes to those statements included elsewhere in this Report. This discussion and analysis and other parts of this Report contain forward-looking statements based upon current beliefs, plans and expectations related to future events and our future financial performance that involve risks, uncertainties and assumptions, such as statements regarding our intentions, plans, objectives, expectations, forecasts and projections. Our actual results and the timing of selected events could differ materially from those anticipated in these forward-looking statements as a result of several factors.
Previous report
All statements included or incorporated by reference in this Report, other than statements or characterizations of historical fact, are forward-looking statements. Forward-looking statements can generally be identified by the fact that they do not relate strictly to historical or current facts and include, but are not limited to, statements using terminology such as “can”, “may”, “could”, “should”, “assume”, “forecasts”, “believe”, “designed to”, “will”, “expect”, “plan”, “anticipate”, “estimate”, “potential”, “position”, “predicts”, “strategy”, “guidance”, “intend”, “seek”, “budget”, “project” or “continue”, or the negative thereof or other comparable terminology regarding beliefs, plans, expectations or intentions regarding the future. You should read statements that contain these words carefully because they:
Previous report
discuss our future expectations;
Previous report
contain projections of our clinical trials, future results of operations or of our financial condition; and
Previous report
state other “forward-looking” information.
Previous report
We believe it is important to communicate our expectations. However, forward-looking statements are based on our current expectations, assumptions, estimates, approximations and projections about our business and our industry and management’s beliefs, all of which are subject to change. Forward-looking statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties and other factors. Accordingly, our actual results and the timing of certain events may differ materially and adversely from those expressed or implied in such forward-looking statements due to a variety of factors and risks, including, but not limited to, those set forth in this Item 2, “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and in our unaudited condensed consolidated financial statements and notes thereto included in this Report, those set forth from time to time in our other filings with the SEC, including our Annual Report on Form 10-K for the fiscal year ended December 31, 2023, and the following factors and risks:
Previous report
We are a clinical-stage drug development company with limited resources, a limited operating history and have no products approved for commercial sale, which may make it difficult to evaluate our current business and predict our future success and viability;
Previous report
If we are unable to successfully raise additional capital, particularly with respect to our neurodegenerative programs, our future clinical trials and product development could be limited and our long-term viability may be threatened;
Previous report
While the U.S. Food and Drug Administration, or FDA, lifted the clinical holds with respect to the risvodetinib (IkT-148009) programs relating to Parkinson’s disease and Multiple System Atrophy, or MSA, we may be subject to further clinical holds by the FDA in the future;
Previous report
IkT-001Pro may not improve the side effect profile compared to imatinib mesylate or may not be effective as an add-on treatment for PAH;
Previous report
Drug development is a highly uncertain undertaking and involves a substantial degree of risk. We have never generated any revenue from product sales, we may never generate any revenue from product sales, and we may fail to generate further revenue from grants or contracts or to be profitable;
Previous report
The wars between Russia and Ukraine and between Israel and Hamas could materially adversely affect our business, results of operations, and financial condition;
Previous report
Our results of operations have been adversely affected and, in the future, could be materially adversely impacted by the COVID-19 virus;
Previous report
Adverse developments affecting financial institutions, companies in the financial services industry or the financial services industry generally, including those we do business with, could adversely affect our operations and liquidity;
Previous report
We have incurred significant net losses since our inception and anticipate that we will continue to incur net losses for the foreseeable future;
Previous report
Due to the significant resources required for the development of our programs, and depending on our ability to access capital, we must prioritize development of certain product candidates;
Previous report
We currently contract with various research institutions to perform the research and development activities needed to develop our products, and if we ever choose to or need to find alternative research institutions, we may not be able to do so at all or, if we are able to do so, it may be costly and may cause significant delays in the development and commercialization of our products;
Previous report
Positive results from early preclinical or clinical studies of our product candidates are not necessarily predictive of the results of later preclinical studies and any current and future clinical trials of our product candidates;
Previous report
We have no history of completing clinical trials for novel drug substances or commercializing pharmaceutical products, which may make it difficult to evaluate the prospects for our future viability;
Previous report
Our clinical trials may reveal significant adverse events, toxicities or other side effects not seen in our preclinical studies and may result in a safety profile that could inhibit regulatory approval or market acceptance of any of our product candidates;
Previous report
We have concentrated much of our research and development efforts to date on the treatment of neurodegenerative diseases, a field that has seen limited success in drug development, although we now intend to expend considerable resources on PAH;
Previous report
We may encounter substantial delays in our current and planned clinical trials, or may not be able to conduct or complete our clinical trials on the timelines we expect, if at all;
Previous report
Our current and planned clinical trials may fail to demonstrate substantial evidence of the safety and efficacy of our product candidates, which would prevent, delay or limit the scope of regulatory approval and commercialization;
Previous report
Clinical development is a lengthy and expensive process with an uncertain outcome, and failure can occur at any stage of clinical development;
Previous report
The manufacture of our product candidates is complex and difficulties may be encountered in production;
Previous report
If, in the future, we are unable to establish sales and marketing capabilities or enter into agreements with third parties to sell and market any product candidates we may develop, we may not be successful in commercializing those product candidates if and when they are approved;
Previous report
Even if any product candidates we develop receive marketing approval, they may fail to achieve the degree of market acceptance by physicians, patients, healthcare payors, and others in the medical community necessary for commercial success;
Previous report
Even if we are able to commercialize any product candidates, such products may become subject to unfavorable pricing regulations, third-party reimbursement practices, or healthcare reform initiatives, which would harm our business;
Previous report
The regulatory approval processes of the FDA, European Medicines Agency, and comparable foreign regulatory authorities are lengthy, time consuming, and inherently unpredictable. Regulatory authorities have substantial discretion in the approval process and may refuse to accept an application, may disagree with our regulatory strategy or proposed pathway for approval or may decide that our data are insufficient for approval and require additional preclinical, clinical or other studies;
Previous report
We expect to depend in whole or in part on collaborations with third parties for the research, development and commercialization of any product candidates we may develop;
Previous report
We contract with third parties for the manufacture of materials for our research programs, preclinical studies and current clinical trials and expect to continue to do so for any future clinical trials and for commercialization of any product candidates that we may develop;
Previous report
We depend on a small number of third-party suppliers for key raw materials used in the manufacturing processes for our product candidates, and the loss of these third-party suppliers or their inability to supply us with adequate raw materials could harm our business; and
Previous report
If we are unable to obtain and maintain patent protection for any product candidates we develop, our competitors could develop and commercialize products or technology similar or identical to ours, and our ability to successfully commercialize any product candidates we may develop, and our technology may be adversely affected.
Previous report
Any or all of our forward-looking statements may turn out to be wrong. They may be affected by inaccurate assumptions that we might make or by known or unknown risks and uncertainties. Actual outcomes and results may differ materially from what is expressed or implied in our forward-looking statements.
Previous report
All forward-looking statements included in this Report are made as of the date hereof, in each case based on information available to us as of the date hereof, and we assume no obligations to update any forward-looking statement or risk factor, unless we are required to do so by law. If we do update one or more forward-looking statements, no inference should be drawn that we will make updates with respect to other forward-looking statements or that we will make any further updates to those forward-looking statements at any future time.
Previous report
Forward-looking statements may include our plans and objectives for future operations, including plans and objectives relating to our product candidates and our future economic performance, projections, business strategy and timing and likelihood of success. Assumptions relating to the forward-looking statements included in this Report involve judgments with respect to, among other things, future economic, competitive and market conditions, future business decisions, and the time and money required to successfully complete development and commercialization of our product candidates, all of which are difficult or impossible to predict accurately and many of which are beyond our control.
Previous report
Any of the assumptions underlying the forward-looking statements contained in this Report could prove inaccurate and, therefore, we cannot assure you that any of the results or events contemplated in any of such forward-looking statements will be realized. Based on the significant uncertainties inherent in these forward-looking statements, the inclusion of any such statement should not be regarded as a representation or as a guarantee by us that our objectives or plans will be achieved, and we caution you against relying on any of the forward-looking statements contained herein.
Previous report
IkT-001 Pro and PAH. IkT-001Pro emerged from the Company’s medicinal chemistry program that aimed to improve on-dosing side effects for drugs that inhibit the Abelson Tyrosine Kinase family. IkT-001Pro, a prodrug of the anticancer agent imatinib mesylate, was designed to mask areas of the molecule that might play a role in the on-dosing GI and other side effects commonly observed with oral imatinib mesylate. A three-part dose finding/dose equivalence study in 66 healthy volunteers (known as ‘the 501 trial’) was completed with IkT-001Pro in 2023. The study was designed to evaluate the 96-hour single-dose pharmacokinetics of imatinib delivered as IkT-001Pro and determine the dose of IkT-001Pro that can deliver the equivalent of either 400 mg or 600 mg imatinib mesylate. Bioequivalence to 400 mg imatinib mesylate was established to our satisfaction for a 600 mg dose of IkT-001Pro. We further evaluated 600 mg imatinib mesylate and believe that a dose between 800 mg and 900 mg of IkT-001Pro is the preferred dose of IkT-001Pro to deliver a dose of imatinib equivalent to 600 mg imatinib mesylate. On January 19, 2024, members of the Company along with its medical consultants met with the FDA Hematological Malignancy Review Team (the “Review Team”) in a Pre-New Drug Application, or pre-NDA, meeting to discuss our bioequivalence studies of IkT-001Pro and its path to approval. All questions were addressed and summarized in official meeting minutes issued by the FDA on February 12, 2024. During the meeting, we inquired whether additional clinical studies would be needed to seek approval and discussed manufacturing and quality control requirements for approval. The Review Team acknowledged that the 505(b)(2) pathway appeared to be the appropriate pathway for approval of IkT-001Pro and indicated that, pending formal review of our clinical data, clinical studies completed to date indicate that
Previous report
Risvodetinib and neurodegenerative diseases. Risvodetinib emerged from the Company’s RAMPTM medical chemistry program and was shown to be a selective inhibitor of the non-receptor Abelson Tyrosine Kinases. In a series of pre-clinical studies, including therapeutic dosing animal models of inherited and sporadic Parkinson’s disease, risvodetinib was shown to be an effective, disease-modifying therapeutic. These studies established that risvodetinib is capable of protecting neurons from degradation, restoring lost motor and non-motor function, suppressing neuroinflammation and clearing the pathology arising from alpha-synuclein aggregate deposition in animal models of human disease. These studies prompted clinical development of risvodetinib in patients in untreated Parkinson’s disease. The Phase 2 ‘201 Trial’ was a twelve-week study of three doses of risvodetinib in participants who have untreated Parkinson’s disease and was placebo controlled with 1:1:1:1 randomization. The primary endpoints of this trial were safety and tolerability and a secondary endpoint included a hierarchy of 15 endpoints whose purpose was to evaluate treatment benefit in the brain and GI tract. The 201 Trial was completed on October 6, 2024 and trial outcomes are anticipated to be made public in the fourth quarter of 2024.
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Reworded paragraphs (21)
Previous wording
We are a clinical-stage pharmaceutical company developing protein kinase inhibitor therapeutics to modify the course of cardiopulmonary and neurodegenerative diseases and other diseases that arise from aberrant signaling through the Abelson Tyrosine Kinases. The Company’s multi-therapeutic pipeline has developed IkT-001Pro, a prodrug of the anticancer agent imatinib mesylate, for Pulmonary Arterial Hypertension (PAH). In 2023, the Company completed a bioequivalence clinical trial in healthy volunteers to determine the dose of IkT-001Pro that is equivalent to either 400 mg or 600 mg imatinib mesylate and the results are being utilized to set the doses in a Phase 2b trial to determine if IkT-001Pro could be a disease-modifying treatment for PAH. The Company has also developed risvodetinib (also known as IkT-148009), a selective inhibitor of the non-receptor Abelson Tyrosine Kinases that targets the treatment of Parkinson’s disease inside and outside the brain. In 2021, the Company commenced clinical development of risvodetinib. In 2023, the Company initiated the Phase 2 201 trial for risvodetinib (IkT-148009) as a treatment for Parkinson’s disease and completed that trial on October 6, 2024. We expect results from this trial to be reported in the fourth quarter of 2024.
Current wording
We were previously pursuing risvodetinib (also known as IkT-148009), a selective inhibitor of the non-receptor Abelson Tyrosine Kinases that targets the treatment of Parkinson’s disease, but we have discontinued this program and subsequent to March 31, 2025 have outlicensed risvodetinib.
Previous wording
600 mg and 800 mg IkT-001Pro provide similar exposures to 400 mg and 600 mg imatinib mesylate, respectively. The Review Team also discussed the possible difference between IkT-001Pro and imatinib mesylate absorption in the gut and recommended that we evaluate whether IkT-001Pro and imatinib mesylate behave differently with respect to certain gut transporters that regulate absorption. This evaluation was completed and determined that IkT-001Pro and imatinib mesylate have similar behavior toward the transporters P-glycoprotein (PGP) and the Breast Cancer Resistance Protein BCRP. Finally, a number of recommendations were discussed to prevent the potential mix-up between IkT-001Pro and imatinib mesylate either at the pharmacy or by patients for two drugs delivering the same active ingredient. The Company discussed alternate dosage forms for IkT-001Pro relative to imatinib mesylate as the primary mitigation strategy and will provide a justification of the dosage forms chosen and why they are unlikely to cause medication errors if/when the Company submits an NDA for approval of IkT-001Pro in these cancer indications.
Current wording
On January 19, 2024, we met with the Food and Drug Administration (“FDA”) Hematological Malignancy Review Team (“Review Team”) in a Pre-New Drug Application (“pre-NDA”), meeting to discuss our bioequivalence studies of IKT-001 and its path to approval. All questions were addressed and summarized in official meeting minutes the issued by the FDA on February 12, 2024. During the meeting, we inquired whether additional clinical studies would be needed to seek approval and discussed manufacturing and quality control requirements for approval. The Review Team acknowledged that the 505(b)(2) pathway appeared to be the appropriate pathway for approval of IKT-001. The Review Team also discussed the possible difference between IKT-001 and imatinib mesylate absorption in the gut and recommended that we evaluate whether IKT-001 and imatinib mesylate behave differently with respect to certain gut transporters that regulate absorption. This evaluation was completed and determined that IKT-001 and imatinib mesylate have similar behavior toward the transporters P-glycoprotein (“PGP”) and the Breast Cancer Resistance Protein (“BCRP”). Finally, a number of recommendations were discussed to prevent the potential mix-up between IKT-001 and imatinib mesylate either at the pharmacy or by patients for two drugs delivering the same active ingredient. The Company discussed alternate dosage forms for IKT-001 relative to imatinib mesylate as the primary mitigation strategy and will provide a justification of the dosage forms chosen and why they are unlikely to cause medication errors if/when the Company submits a New Drug Application (“NDA”) for approval of IKT-001 in these cancer indications.
Previous wording
In 2013, the outcome of a Phase 3 trial evaluating imatinib mesylate as a treatment for PAH was reported, demonstrating that imatinib can modify the course of disease in PAH. Co-administration of medications with harmful drug-drug interactions precluded the approval of imatinib as add-on therapy in PAH despite the success of the Phase 3 trial. PAH is a rare disease of the pulmonary microvasculature, with about 30,000 cases in the U.S. that principally afflicts women between the ages of 30 and 60. The global PAH market size was valued at $7.66 billion in 2023 and is estimated to grow at a compound annual growth rate (CAGR) of 5.4% between 2024 to 2030. As the Company considered revisiting the use of imatinib in PAH, the Company recognized that changes in standard-of-care for these patients may have alleviated much of the safety risk previously observed for imatinib in PAH patients. This analysis prompted the Company to file a pre-IND (PIND) meeting request to discuss the application of IkT-001Pro as a potential disease-modifying treatment for PAH. To evaluate this further, members of the Company met with the FDA Division of Cardiology and Nephrology in a PIND meeting to discuss the Company’s plan to utilize IkT-001Pro at 300 mg or 500 mg in a Phase 2b efficacy, safety and tolerability study in PAH. At the meeting, the FDA confirmed that IkT-001Pro would be viewed as a New Molecular Entity (NME) and that the appropriate path for approval remained to be the 505(b)(2) statute. This opens up the possibility of IkT-001Pro being granted NME status and patent exclusivity on approval. The FDA requested at the PIND meeting that we conduct a comparative cell-culture based study of the hERG ion channel, a standard cardiovascular safety test performed for any NME for which a new IND is to be opened. Neither IkT-001Pro nor imatinib mesylate were found to be inhibitors of hERG. Following completion of this study, the IND was filed with the FDA on August 9, 2024 and cleared to initiate a Phase 2b trial on September 9, 2024. On October 21, 2024, the Company closed a private placement with an initial investment of approximately $110 million, before deducting placement fees and offering expenses, to support this program. If the warrants are exercised for cash, the aggregate financing may be up to $275 million. The Company intends to use the net proceeds from the private placement to finance the initiation of a Phase 2b trial in PAH and for general corporate purposes. The Company has also applied for Orphan Drug Designation for delivery of imatinib by IkT-001Pro for PAH.
Current wording
As we considered revisiting the use of imatinib in PAH, we recognized that changes in SOC for these patients may have alleviated much of the safety risk previously observed for imatinib in PAH patients. This analysis prompted us to file a pre-IND (“PIND”) meeting request to discuss the application of IKT-001 as a potential disease-modifying treatment for PAH. To evaluate this further, members of the Company met with the FDA Division of Cardiology and Nephrology in a PIND meeting to discuss our plan to utilize IKT-001 in a Phase 2b efficacy, safety and tolerability study in PAH. At the meeting, the FDA confirmed that IKT-001 would be viewed as a New Molecular Entity (“NME”) and that the appropriate path for approval remained to be the 505(b)(2) statute. This opens up the possibility of IKT-001 being granted NME status and market exclusivity on approval. The FDA requested at the PIND meeting that we conduct a comparative cell-culture based study of the human Ether-a-go-go-related Gene (“hERG”) ion channel, a standard cardiovascular safety test performed for any NME for which a new Investigative New Drug Application (“IND”) is to be opened. Neither IKT-001 nor imatinib mesylate were found to be inhibitors of hERG. Following completion of this study, the IND was filed with the FDA on August 9, 2024 and we were cleared to initiate a Phase 2b trial on September 9, 2024. On October 21, 2024, we closed a private placement with gross proceeds of approximately $110 million, before deducting placement fees and offering expenses, to support this program. If the warrants issued in such offering are exercised for cash, the total gross proceeds from the financing may be up to $275 million. We intend to use the net proceeds from the private placement to finance the initiation of a Phase 2b trial in PAH and for general corporate purposes. We have had discussions with the FDA regarding Orphan Drug Designation (“ODD”) for delivery of imatinib by IKT-001 for PAH and plan to apply for ODD once the required pre-clinical studies are complete.
Previous wording
external research and development expenses, including expenses incurred under arrangements with third parties, such as CROs, preclinical testing organizations, clinical testing organizations, CMOs, academic and non-profit institutions and consultants;
Current wording
external research and development expenses, including expenses incurred under arrangements with third parties, such as contract research organizations (“CROs”), preclinical testing organizations, clinical testing organizations, contract manufacturing organizations (“CMOs”), academic and non-profit institutions and consultants;
Previous wording
Research and development expenses increased by $964,322 or 29.9% to $4,189,873 from $3,225,551 in the prior comparable period. The $1.0 million increase in research and development expenses was due to an increase of $0.8 million in PAH expenses and a net increase of $0.2 million in other research and development expenses.
Current wording
Research and development expenses increased by $7,762,300 or 282.1% to $10,513,579 from $2,751,279 in the prior comparable period. The $7.7 million increase in research and development expenses was due to an increase of $9.3 million in PAH expenses which included a one-time (non-cash) expense charge for the acquired IPR&D related to the CorHepta acquisition cost of $7.4 million as at the acquisition date of February 21, 2025, a decrease of $2.2 million in the discontinued risvodetinib (IkT-148009) program and a net increase of $0.6 million in other research and development expenses.
Previous wording
From our inception up until our December 2020 IPO, we funded our operations primarily through private, state and federal contracts and grants. In October 2024, the Company raised approximately $110 million in gross proceeds from its October 2024 Offering.
Current wording
From our inception up until our December 2020 initial public offering, we funded our operations primarily through private, state and federal contracts and grants. In October 2024, we raised approximately $99.6 million in net proceeds from its October 2024 Offering.
Previous wording
The Company has incurred recurring losses and at September 30, 2024 had an accumulated deficit of $82,288,034.
Current wording
We have incurred recurring losses and at March 31, 2025 had an accumulated deficit of $108,099,346.
Previous wording
Since our inception, we have incurred significant losses and negative cash flows from operations. We have an accumulated deficit of $82,288,034 at September 30, 2024. We expect to incur substantial additional losses in the future as we conduct and expand our research and development activities.
Current wording
Since our inception, we have incurred significant losses and negative cash flows from operations. We have an accumulated deficit of $108,099,346 at March 31, 2025. We expect to incur substantial additional losses in the future as we conduct and expand our research and development activities.
Previous wording
We believe that our existing cash, cash equivalents and marketable securities as of September 30, 2024 and the gross proceeds of approximately $110 million the Company raised in October 2024, will enable us to fund our operating requirements for at least the next twelve months. However, we have based these estimates on assumptions that may prove to be wrong, and we could deplete our working capital sooner than planned.
Current wording
We believe that our existing cash, cash equivalents and marketable securities as of March 31, 2025, will enable us to fund our operating requirements for at least the next twelve months following the date of this Quarterly Report. However, we have based these estimates on assumptions that may prove to be wrong, and we could deplete our working capital sooner than planned.
Previous wording
our efforts to enhance operational, financial and information management systems and hire additional personnel, including personnel to support development of our product candidates; and
Current wording
our efforts to enhance operational, financial and information management systems and hire additional personnel, including personnel to support development of our product candidates;
Previous wording
the costs and ongoing investments to in-license and/or acquire additional technologies.
Current wording
the costs and ongoing investments to in-license and/or acquire additional technologies; and
Previous wording
possible delays or interruptions to preclinical studies, clinical trials, our receipt of services from our third-party service providers on whom we rely;
Current wording
possible delays or interruptions to preclinical studies, clinical trials, our receipt of services from our third-party service providers on whom we rely, or our supply chain due to epidemics or pandemics.
Previous wording
Net cash flows used in operating activities for the nine months ended September 30, 2023, totaled $14,642,612, and consisted primarily of a net loss of $14.9 million adjusted for non-cash stock compensation of $0.4 million, depreciation and lease expense of $0.2 million, decrease in prepaid expenses and other assets of $0.2 million, increase in prepaid research and development of $0.8 million, decrease in accounts payable of $0.4 million, and a decrease in accrued expenses and other current liabilities of $0.5 million.
Current wording
Net cash flows used in operating activities for the three months ended March 31, 2025, totaled $4,103,530, and consisted primarily of a net loss of $13.7 million adjusted for non-cash stock compensation of $2.0 million, a write-off of in-process research and development of $7.4 million, a decrease in the fair value of contingent consideration of $1.2 million, an increase in accounts payable of $0.7 million, and an increase in accrued expenses and other current liabilities of $0.8 million.
Previous wording
Net cash flows used in operating activities for the nine months ended September 30, 2024, totaled $13,802,492, and consisted primarily of a net loss of $15.4 million adjusted for non-cash stock compensation of $0.2 million, a decrease in prepaid research and development of $0.1 million, an increase in accounts payable of $1.3 million, and a decrease in accrued expenses and other current liabilities of $0.1 million.
Current wording
Net cash flows used in operating activities for the three months ended March 31, 2024, totaled $3,867,018, and consisted primarily of a net loss of $4.6 million adjusted for non-cash stock compensation of $0.1 million, an increase in accounts payable of $0.5 million, and an increase in accrued expenses and other current liabilities of $0.2 million.
Previous wording
Net cash flows provided by investing activities for the nine months ended September 30, 2023, totaled $13,772,261, of which $20.6 million was used for the purchase of marketable securities investments and $34.4 million was provided by maturity of marketable securities.
Current wording
Net cash flows provided by investing activities for the three months ended March 31, 2025, totaled $21,054,342, of which $21.5 million was provided by maturity of marketable securities and $0.4 million related to acquired in-process research and development associated with the CorHepta acquisition discussed above.
Previous wording
Net cash flows provided by investing activities for the nine months ended September 30, 2024, totaled $1,757,524, of which $10.3 million was used for the purchase of marketable securities investments and $12.1 million was provided by maturity of marketable securities.
Current wording
Net cash flows provided by investing activities for the three months ended March 31, 2024, totaled $3,311,814, of which $8.4 million was used for the purchase of marketable securities investments and $5.1 million was provided by maturity of marketable securities.
Previous wording
Net cash flows provided by financing activities for the nine months ended September 30, 2024, totaled $3,793,209, which consisted of $3.8 million of net proceeds from issuance of common stock and pre-funded warrants in connection with our May 2024 Offering and our ATM Offering.
Current wording
Net cash flows provided by financing activities for the three months ended March 31, 2025, totaled $0.
Previous wording
Net cash flows provided by financing activities for the nine months ended September 30, 2023, totaled $8,543,037, which consisted of net proceeds from issuance of common stock and pre-funded warrants in connection with our January 2023 Offering.
Current wording
Net cash flows provided by financing activities for the three months ended March 31, 2024, totaled $366,999, which consisted of primarily of net proceeds from issuance of common stock in connection with our ATM offering.
Previous wording
On April 18, 2022, the Company entered into an operating lease agreement through September 30, 2025 for its office space in Lexington, Massachusetts. The Lexington lease contains escalating payments during the lease period. Upon execution of this lease agreement, the Company prepaid one month of rent, applied to the first month's rent, and a security deposit, which will be held in escrow and credited at the termination of the lease. Our total lease obligation is $153,288, consisting of minimum annual rental obligations of $38,322 for fiscal year 2024 and $114,966 for fiscal year 2025.
Current wording
On April 18, 2022, we entered into an operating lease agreement through September 30, 2025 for our office space in Lexington, Massachusetts. The Lexington lease contains escalating payments during the lease period. Upon execution of this lease agreement, we prepaid one month of rent, applied to the first month's rent, and a security deposit, which will be held in escrow and credited at the termination of the lease. Our total lease obligation is $76,644, consisting of minimum annual rental obligations for fiscal year 2025.
Previous wording
This discussion and analysis of our financial condition and results of operations is based on our unaudited condensed consolidated financial statements, which have been prepared in accordance with generally accepted accounting principles in the United States, or US GAAP. The preparation of these unaudited condensed consolidated financial statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the unaudited condensed consolidated financial statements, as well as the reported expenses incurred during the reporting periods. Our estimates are based on our historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. While our significant accounting policies are described in more detail in the notes to our unaudited condensed consolidated financial statements included elsewhere in this Report, we believe that the following accounting policies are critical to understanding our historical and future performance, as these policies relate to the more significant areas involving management’s judgments and estimates.
Current wording
This discussion and analysis of our financial condition and results of operations is based on our unaudited condensed consolidated financial statements, which have been prepared in accordance with generally accepted accounting principles in the United States. The preparation of these unaudited condensed consolidated financial statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the unaudited condensed consolidated financial statements, as well as the reported expenses incurred during the reporting periods. Our estimates are based on our historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. While our significant accounting policies are described in more detail in the notes to our unaudited condensed consolidated financial statements included elsewhere in this Quarterly Report, we believe that the following accounting policies are critical to understanding our historical and future performance, as these policies relate to the more significant areas involving management’s judgments and estimates.
Previous wording
As part of the process of preparing financial statements, we are required to estimate and accrue expenses. A portion of our research and development expenses is comprised of external costs, which we track on a program-specific basis. We record the estimated expenses of research and development activities conducted by third-party service providers as they are incurred and provided within research and development expense in the condensed consolidated statements of operations and comprehensive loss. These services include the conduct of clinical studies, preclinical studies and consulting services. These costs are a significant component of our research and development expenses.
Current wording
As part of the process of preparing financial statements, we are required to estimate and accrue expenses. A portion of our research and development expenses is comprised of external costs, which we track on a program-specific basis. We record the estimated expenses of research and development activities conducted by third-party service providers as they are incurred and provided within research and development expense in the condensed consolidated statements of operations and comprehensive loss. These services include the conduct of clinical studies, preclinical studies and consulting services. These costs are a significant component of our research and development expenses. Typically, upfront payments and milestone payments made for the licensing of technology are expensed as research and development in the period in which they are incurred, except for payments relating to intellectual property rights with future alternative use which will be expensed when the intellectual property is in use. Non-refundable advance payments for goods or services to be received in the future for use in research and development activities are recorded as prepaid expenses. The prepaid amounts are expensed as the related goods are delivered or the services are performed.
Rearranged previous paragraphs (2)
Previous report — wording carried elsewhere
Comparison of the Nine Months Ended September 30, 2024 and 2023
Previous report — wording carried elsewhere
The following table sets forth the significant components of our results of operations: