Imatinib mesylate

-- Transformative Financing worth up to $275 million from Top-Tier Institutional Healthcare Investors -- -- Advancement of IkT-001Pro into a Late-Stage Clinical Trial Program in Pulmonary Arterial Hypertension -- --201 Trial Results to be reported in 4Q2024 -- BOSTON and ATLANTA, Nov. 14, 2024 (GLOBE NEWSWIRE) -- Inhibikase Therapeutics, Inc. (Nasdaq: IKT) (“Inhibikase” or “Company”), a clinical-stage pharmaceutical company developing protein kinase inhibitor therapeutics to modify the course of Cardiopulmonary and Neurodegenerative disease through Abelson Tyrosine Kinase inhibition, today reported financial results for the third quarter ended September 30, 2024 and highlighted recent developments. “The financing we concluded in October was a transformational moment for Inhibikase, and positioned us to advance IkT-001Pro into a late-stage clinical trial in Pulmonary Arterial Hypertension (PAH),” said Dr. Milton Werner, President and Chief Executive of Inhibikase. “This investment, with top tier dedicated healthcare investment funds, recognized the potential of IkT-001Pro to improve the lives of patients afflicted with PAH. We believe IkT-001Pro, a prodrug of imatinib, has the potential to ameliorate the safety and tolerability profile that precluded approval of imatinib for the treatment of PAH more than 10 years ago. This investment further validates the Company’s innovative approach to the development of kinase inhibitor therapeutics in the non-oncology setting.” Recent Developments and Upcoming Milestones: Closed up to $275 million financing in October 2024 in support of advancing IkT-001Pro into a late-stage clinical trial in PAH Private placement of approximately $110 million from the issuance and sale of shares of the Company’s common stock and accompanying warrants with potential aggregate financing of up to approximately $275 million upon the full cash exercise of the warrants issued in the private placement, before deducting placement fees and offering expenses. The financing will fund execution of the Phase 2b ‘702’ trial in Pulmonary Arterial Hypertension (PAH) and general corporate purposes. Added three highly accomplished leaders in biopharmaceutical development and a Partner of Soleus Capital to the Board of Directors coincident with the financing: Roberto Bellini, Managing Partner of BSQUARED Capital and former Chief Executive Officer of BELLUS Health Inc.Amit Munshi, Chief Executive Officer of Orna Therapeutics and former CEO of Arena Pharmaceuticals,Arvind Kush, Chief Financial Officer and Chief Business Officer of Candid Therapeutics and former CFO of RayzeBioDavid Canner, Partner at Soleus CapitalIn addition to joining the Board of Directors, Messers. Bellini, Munshi and Kush each participated in the financing; Mr. Bellini has been appointed Independent Chairperson of the Inhibikase Board. Advancement of IkT-001Pro as a therapy in Pulmonary Arterial Hypertension: The Company received its Study May Proceed letter on September 9, 2024.The active ingredient in IkT-001Pro, imatinib, has previously been shown to be disease-modifying for PAH. The Company believes that IkT-001Pro could have a more favorable safety and tolerability profile compared to imatinib for this indication.Following the Company’s pre-NDA meeting with the FDA in January 2024, Inhibikase enhanced its manufacturing process development efforts for IkT-001Pro to support late-stage clinical development. Ongoing activities include development of new dosage forms, a more efficient production process and a high throughput tableting process that will lead to dosage forms for 001Pro tablets that are differentiated from generic imatinib mesylate in alignment with FDA feedback. The last patient and last visit in the Phase 2 201 Trial evaluating risvodetinib (also known as IkT-148009) in untreated Parkinson’s disease occurred in October, 2024; topline data is expected in the fourth quarter of 2024. The trial randomized 126 patients total, 120 of which completed the 12 week double blind dosing period. Third Quarter Financial Results Net Loss: Net loss for the quarter ended September 30, 2024, was $5.8 million, or $0.65 per share, compared to a net loss of $4.6 million, or $0.75 per share in the quarter ended September 30, 2023. The net loss per share does not give effect to the shares issued in the October 2024 financing. R&D Expenses: Research and development expenses were $4.2 million for the quarter ended September 30, 2024 compared to $3.23 million in the quarter ended September 30, 2023. SG&A Expenses: Selling, general and administrative expenses for the quarter ended September 30, 2024 were $1.6 million compared to $1.62 million for the quarter ended September 30, 2023. Cash Position: Cash, cash equivalents and marketable securities were $3.2 million as of September 30, 2024, which does not include the gross proceeds of approximately $110 million from the October 2024 Offering. About Inhibikase (www.inhibikase.com) Inhibikase Therapeutics, Inc. (Nasdaq: IKT) is a clinical-stage pharmaceutical company developing Abelson Tyrosine Kinase inhibitor therapeutics for Cardiopulmonary and Neurodegenerative disease. Inhibikase’s cardiopulmonary disease portfolio is led by IkT-001Pro, a prodrug of imatinib mesylate, for Pulmonary Arterial Hypertension that will deliver imatinib in a form that the Company believes will provide a better patient experience with fewer on-dosing side-effects. Inhibikase's neurodegenerative disease portfolio is led by risvodetinib, a selective c-Abl inhibitor to treat Parkinson’s and Parkinson’s-related disease. Inhibikase is headquartered in Atlanta, Georgia with offices in Lexington, Massachusetts. Social Media Disclaimer Investors and others should note that the Company announces material financial information to investors using its investor relations website, press releases, SEC filings and public conference calls and webcasts. The Company intends to also use X, Facebook, LinkedIn and YouTube as a means of disclosing information about the Company, its services and other matters and for complying with its disclosure obligations under Regulation FD. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking terminology such as "believes," "expects," "may," "will," "should," "anticipates," "plans," or similar expressions or the negative of these terms and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based on Inhibikase's current expectations and assumptions. Such statements are subject to certain risks and uncertainties, which could cause Inhibikase's actual results to differ materially from those anticipated by the forward-looking statements. Important factors that could cause actual results to differ materially from those in the forward-looking statements include our ability to commence and execute a Phase 2b ‘702’ trial to evaluate IkT-001Pro as a treatment for Pulmonary Arterial Hypertension (PAH), whether our top line data from our Phase 2 201 Trial evaluating risvodetinib in untreated Parkinson’s disease will show a statistically significant clinical benefit to trial participants, as well as such other factors that are included in our periodic reports on Form 10-K and Form 10-Q that we file with the U.S. Securities and Exchange Commission. Any forward-looking statement in this release speaks only as of the date of this release. Inhibikase undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. Contacts: Company Contact:Milton H. Werner, PhDPresident & CEO678-392-3419info@inhibikase.com Investor Relations:Michael MoyerLifeSci Advisorsmmoyer@lifesciadvisors.com Inhibikase Therapeutics, Inc.Condensed Consolidated Balance Sheets(Unaudited) September 30,2024 December 31,2023 (unaudited) (Note 2)Assets ​Current assets: ​Cash and cash equivalents $913,420 $9,165,179 Marketable securities 2,330,226 4,086,873 Prepaid research and development 112,225 219,817 Deferred offering costs 553,318 — Prepaid expenses and other current assets 280,914 739,179 Total current assets 4,190,103 14,211,048 Equipment and improvements, net 53,667 73,372 Right-of-use asset 133,105 222,227 Total assets $4,376,875 $14,506,647 Liabilities and stockholders’ equity ​Current liabilities: ​Accounts payable $2,529,220 $646,767 Lease obligation, current 145,210 150,095 Accrued expenses and other current liabilities 2,161,374 2,259,955 Insurance premium financing payable 71,662 381,784 Total current liabilities 4,907,466 3,438,601 Lease obligation, net of current portion — 90,124 Total liabilities 4,907,466 3,528,725 Commitments and contingencies (see Note 13) ​Stockholders’ equity: ​ ​Preferred stock, $0.001 par value; 10,000,000 shares authorized; 0 shares issued and outstanding at September 30, 2024 and December 31, 2023 — — Common stock, $0.001 par value; 100,000,000 shares authorized; 7,464,070 and 6,186,280 shares issued and outstanding at September 30, 2024 and December 31, 2023 7,464 6,186 Additional paid-in capital 81,748,225 77,871,584 Accumulated other comprehensive (loss) income 1,754 877 Accumulated deficit (82,288,034) (66,900,725)Total stockholders' equity (530,591) 10,977,922 Total liabilities and stockholders’ (deficit) equity $4,376,875 $14,506,647 Inhibikase Therapeutics, Inc.Condensed Consolidated Statements of Operations and Comprehensive Loss(Unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Revenue: Grant revenue$— $79,569 $— $260,500 Total revenue — 79,569 — 260,500 Costs and expenses: Research and development 4,189,873 3,225,551 10,016,982 10,615,368 Selling, general and administrative 1,637,603 1,622,894 5,643,386 5,331,358 Total costs and expenses 5,827,476 4,848,445 15,660,368 15,946,726 Loss from operations (5,827,476) (4,768,876) (15,660,368) (15,686,226)Interest income (expense) 49,410 173,677 273,059 835,283 Net loss (5,778,066) (4,595,199) (15,387,309) (14,850,943)Other comprehensive loss, net of tax Unrealized gains (loss) on marketable securities 2,778 1,571 877 (104,861)Comprehensive Loss$(5,775,288) $(4,593,628) $(15,386,432) $(14,955,804)Net loss per share – basic and diluted$(0.65) $(0.75) $(2.03) $(2.48)Weighted-average number of common shares – basic and diluted 8,882,570 6,162,671 7,592,103 5,977,841

PLYMOUTH, Mass.--( BUSINESS WIRE )--IDRx, Inc., a clinical-stage biopharmaceutical company dedicated to developing and commercializing precision therapeutics for the treatment of cancers with well-characterized biology and significant unmet medical need, today announced updated clinical data from StrateGIST 1, the company’s ongoing Phase 1/1b trial of IDRX-42 in patients with advanced gastrointestinal stromal tumors (GIST). These data continue to demonstrate promising anti-tumor activity of IDRX-42 in patients with advanced GIST and will be highlighted today in an oral presentation at the Connective Tissue Oncology Society (CTOS) 2024 Annual Meeting, being held November 13-16, in San Diego, CA. IDRX-42 is an investigational oral, small molecule, tyrosine kinase inhibitor (TKI) designed to selectively target the most prevalent KIT mutations that either drive the growth, proliferation, and survival of tumor cells (activating mutations in exons 9 and 11), or confer resistance to currently available therapies (resistance mutations in exons 13 and 17). “ In patients living with GIST, the therapeutic potential of KIT targeted TKIs has been limited by drug resistance caused by an inability to target the spectrum of the most prevalent KIT mutations, and poor tolerability,” said Suzanne George, M.D., Chief, Division of Sarcoma at the Dana-Farber Cancer Institute. “ These updated data from StrateGIST 1 are highly encouraging with respect to the clinical activity observed in later-line patients with a variety of KIT activating and resistance mutations. I am excited by the potential of IDRX-42, with its differentiated clinical profile demonstrated to date, to help meet this critical need for GIST patients.” “ We are particularly excited about the emerging durability data from StrateGIST 1. These data, together with the continued strengthening of response rate and tolerability data, underscore the potential for IDRX-42 to improve outcomes for GIST patients and support our plans to move IDRX-42 into late-phase development,” said Tim Clackson, Ph.D., Chief Executive Officer of IDRx. “ With FDA alignment now in place, we plan to conduct StrateGIST 3, a randomized, Phase 3 registrational trial which will evaluate the efficacy and safety of IDRX-42 as compared to sunitinib in second-line GIST patients. We remain steadfast in our commitment to transform the lives of patients with GIST, including in early-line settings, where the standards of care have remained unchanged for almost 20 years and a significant unmet need remains.” Presentation Highlights As of the data cut-off date of September 30, 2024, 89 patients have been treated with IDRX-42 in the Phase 1 portion of the ongoing StrateGIST 1 trial across dose cohorts ranging from 120 mg once daily (QD) to 1200 mg total daily dose (600 mg twice daily (BID)). All 89 patients had KIT -mutant GIST. 87 patients were evaluable for efficacy as of the data cut-off date. The two non-evaluable patients were recently enrolled and remain on treatment, awaiting their first post-baseline tumor assessment. While a maximum tolerated dose was not reached, dose escalation in the Phase 1 portion of the trial has been completed, and the Phase 1b dose confirmation portion of the trial has been initiated utilizing a dose of 300 mg in tablet formulation, which has comparable steady-state plasma exposure to a dose of 400 mg in capsule formulation. Patients were heavily pretreated overall, with a median of four prior lines of therapy. 17% (15/89) patients had received one prior line of therapy (IDRX-42 evaluated as second-line). 11% (10/89) patients had received two prior lines of therapy (IDRX-42 evaluated as third-line). 72% (64/89) patients had received three or more prior lines of therapy (IDRX-42 evaluated as ≥ fourth-line). 28% (25/89) received three or more prior lines of therapy that did not include ripretinib. As of the data cut-off date, 63% (56/89) of patients remained on study treatment, and the median duration of treatment was 6.6 months. The ORR by modified RECIST v1.1 in the total efficacy evaluable population was 29% (25/87) treated across all lines of therapy, including one complete response (CR) and 24 partial responses (PRs). Two of the PRs were awaiting confirmation at the time of the data cut. At the 400 mg capsule/300 mg tablet QD recommended Phase 1b dose (RP1bD), the ORR was 26% (10/38) in the total efficacy evaluable population. The ORR in second-line patients was 53% (8/15) including one CR and 7 PRs. At the RP1bD or below, the ORR was 50% (7/14) across second-line patients. In second-line patients (n=15), the 6-month PFS was 85%, and the median was not yet reached. The median PFS in third- or later-line patients both at the RP1bD (n=25) and across all doses (n=72) was 9.2 months. The median PFS in third-line patients overall (n=10) was 12.9 months. The median PFS in fourth- or later-line patients who had not previously received ripretinib (n=25) was 9.2 months, and at the RP1bD (n=10) was 11 months. IDRX-42 was generally well-tolerated and treatment-related adverse events (TRAEs) were mainly low grade at the RP1bD. The most frequently reported TRAEs (≥20%) were gastrointestinal symptoms (diarrhea, nausea, decreased appetite, vomiting, dysgeusia) and fatigue. An 8% dose reduction rate due to TRAEs was observed at RP1bD, with no TRAEs leading to dose discontinuation. The Phase 1b portion of StrateGIST 1 commenced enrollment in May 2024 and includes a first-, second-, and two later-line cohorts. The second- and two later-line cohorts are nearly fully enrolled. IDRx has aligned with the FDA on fundamental elements of the trial design, dose, and statistical analysis for StrateGIST 3, a global, randomized, Phase 3 registrational trial which will evaluate IDRX-42 as a second-line therapy in patients diagnosed with metastatic and/or unresectable GIST with disease progression on, or intolerance to, imatinib, the current first-line standard of care for GIST patients. Presentation Details Title: The Novel KIT inhibitor IDRX-42 Shows Promising Activity in Second- and Later-Line Gastrointestinal Stromal Tumors (GIST): Results from a Phase 1 Trial (StrateGIST 1) Presenter: Suzanne George, M.D. Abstract Number: P 10 Presentation Type: Oral Abstract Session Time: Thursday, November 14, 10:30 a.m. – 11:30 a.m. PST Location: Harbor Ballroom, Manchester Grand Hyatt, San Diego, CA About GIST Gastrointestinal stromal tumor (GIST) is the most common subtype of soft tissue sarcoma occurring in the gastrointestinal tract. Between 4,000 to 6,000 people are diagnosed with GIST in the United States each year. Approximately 80% of all GIST cases are driven by mutations in the KIT gene. An estimated 90% of patients who receive imatinib, the current standard of care for GIST, will experience disease progression related to the emergence of KIT resistance mutations. In unresectable or metastatic GIST, clinical benefits from existing treatments can vary by mutation type. Despite the widespread availability of tyrosine kinase inhibitors approved for GIST, first-line and second-line standards of care have remained unchanged for almost 20 years and significant unmet need remains. About the StrateGIST 1 Study StrateGIST 1 is an ongoing, open-label, first-in-human Phase 1/1b study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary anti-tumor activity of IDRX-42 in patients with metastatic and/or surgically unresectable GIST after progression on imatinib and other approved drugs. The study is currently enrolling patients with documented pathogenic mutation in KIT or any platelet-derived growth factor receptor alpha (PDGFRA) mutation (other than PDGFRA exon 18) at sites in the U.S., United Kingdom, Belgium, the Netherlands, France, Germany, Italy, Spain, South Korea, and China. The Phase 1b portion commenced enrollment in May 2024 and includes four exploratory cohorts based on defined lines of prior tyrosine kinase inhibitor therapy, including first-, second-, third- and later-line therapies. About IDRX-42 IDRX-42 is an investigational oral, small molecule KIT tyrosine kinase inhibitor in clinical development as a monotherapy for the treatment of gastrointestinal stromal tumors (GIST). IDRX-42 is designed to selectively target the most prevalent forms of KIT mutations that either drive the initial growth, proliferation, and survival of tumor cells, or confer resistance to currently available therapies. In pre-clinical studies, IDRX-42 demonstrated superior anti-tumor activity as compared to imatinib, the current first-line of therapy, in tumors associated with both exon 9 and 11 KIT mutations. IDRX-42 was also shown in pre-clinical studies to have strong inhibition activity against the most prevalent KIT resistance mutations in exons 13 and 17, suggesting that IDRX-42 could prevent or substantially delay the emergence of such mutations. In xenograft models expressing secondary resistance mutations in KIT exon 13 or 17, IDRX-42 treatment resulted in potent and dose-dependent antitumor activity superior to the second-line standard of care agent, sunitinib. IDRX-42 is currently being evaluated in StrateGIST 1, a first-in-human Phase 1/1b trial. IDRX-42 has been granted Fast Track and Orphan Drug designations by the U.S. Food and Drug Administration for the treatment of GIST after disease progression on imatinib. About IDRx IDRx is a clinical-stage biopharmaceutical company dedicated to developing and commercializing precision therapeutics for the treatment of cancers with well-characterized biology and significant unmet medical need. IDRx aims to address the limitations of today’s precision cancer medicines with highly potent and selective targeted therapies to stop key tumor escape mechanisms and prolong patients’ response to therapy. IDRx’s lead program is IDRX-42, which is designed to selectively target the most prevalent forms of KIT mutations and has the potential to improve outcomes for gastrointestinal stromal tumor (GIST) patients as a first- or second-line therapy, where the standards of care have remained unchanged for almost 20 years and significant unmet need remains. Founded in 2021, IDRx’s leadership team has extensive experience in drug development and collectively has been involved in the discovery, development, and commercialization of more than 10 approved medicines. To learn more, please visit www.idrx.com and follow us on LinkedIn .