Announcement • Apr 01
Inhibitor Therapeutics Inc Provides Clinical Formulation and Ip Update on Itraconazole Program for Gorlin Syndrome Inhibitor Therapeutics, Inc. provided an update on its ongoing pharmacokinetic clinical program supporting the treatment of surgically eligible basal cell carcinomas in Basal Cell Carcinoma Nevus Syndrome, also known as Gorlin Syndrome, and announced plans to file a new global patent application covering its proprietary itraconazole formulation. Inhibitor's comparative PK, safety and tolerability study is being conducted in healthy adult subjects under fasting conditions in Malaysia and is designed as a three-way crossover study against a reference listed drug. The study is being executed in collaboration with Avior Bio, Inc. The Company’s collaboration with Avior Bio, spanning formulation development through clinical execution, has proceeded in line with planned timelines and budget and is intended to support the Company's regulatory strategy under the FDA's 505(b)(2) pathway. The study is currently progressing through its crossover dosing periods, with Period 2 scheduled for April 10, 2026, and Period 3 scheduled for April 24, 2026. Following the final PK sampling in Period 3, plasma samples are expected to be shipped for bioanalysis on or about May 1, 2026. Bioanalytical work is expected to require approximately two weeks, followed by approximately two additional weeks of statistical analysis. The Company expects to receive top-line comparative PK and relative bioavailability results in advance of the full Clinical Study Report, which will follow completion of the final study documentation. Based on the Company's current development plan, Inhibitor believes this PK study is expected to represent the final clinical study required prior to submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration for its BCCNS program. A key differentiating feature of the Company's program is its proprietary amorphous formulation approach. In its conventional crystalline form, itraconazole is known to present significant solubility limitations, which can contribute to variability in absorption and systemic exposure. Inhibitor's formulation strategy is designed to address those limitations by incorporating itraconazole in an amorphous morphology, which is intended to enhance solubility and dissolution behavior by reducing particle agglomeration and improving dispersion. This approach may support improved intestinal absorption and more consistent, predictable pharmacokinetic performance. The Company's Investigational Medicinal Product Dossier (IMPD) characterizes the underlying challenges associated with itraconazole and supports the scientific rationale for the formulation strategy. While the bulk drug substance is described as crystalline and practically insoluble in water, the formulated product candidate evaluated in the dossier was shown by X-ray diffraction to be amorphous, without detectable crystalline peaks, and was developed with the intent of suppressing crystallization and improving intestinal solubility and absorption. In parallel with clinical advancement, Inhibitor is preparing a new patent application directed to the composition of its proprietary formulation. The Company expects to seek global patent protection for the formulation and, if granted, the patent would be owned by Inhibitor Therapeutics without any ongoing royalty obligations. The Company believes this planned filing has the potential to materially strengthen its intellectual property estate by expanding protection around the formulation itself, complementing its existing portfolio related to the use of itraconazole in oncology indications. If granted, the Company believes such patent protection could enhance the strategic value of the BCCNS program as it advances toward potential NDA submission, commercialization and broader partnering discussions. Gorlin Syndrome is a rare hereditary disorder characterized by the development of multiple basal cell carcinomas over a patient's lifetime. Inhibitor is developing itraconazole for surgically eligible BCCs in BCCNS as a differentiated therapeutic approach intended to address a significant unmet need for patients who often face repeated surgical procedures and associated morbidity. Announcement • Aug 22
Inhibitor Therapeutics, Inc. Provides Update on its Clinical Development Plan Inhibitor Therapeutics, Inc. provided an update on development efforts for its itraconazole formulation for the treatment of Basal Cell Carcinoma Nevus Syndrome, otherwise known as Gorlin Syndrome. Inhibitor has allied with The Gorlin Syndrome Alliance and sought supportive guidance from the accomplished and highly-experienced members of its Scientific Advisory Board in efforts to pursue an expeditious path to acceptance by the U.S. Food and Drug Administration (FDA). Inhibitor has submitted a Pre-Investigational New Drug application (PIND) which has been reviewed by the FDA's Division of Dermatology and Dentistry and will have input from the Division of Oncology 3 where necessary. INTI has received preliminary comments from the FDA with guidance in preparation for an Investigation New Drug Application (IND) Inhibitor has engaged Avior Bio for the development of a new proprietary/patentable formulation of itraconazole that will be bridged to the formulation used within Inhibitor's completed clinical study and to the generic formulation of itraconazole via a pharmacokinetic (PK) study. Itraconazole has a well understood safety profile and, based upon the data developed in the completed clinical study, demonstrated clinically meaningful efficacy in the treatment of Basal Cell Carcinomas in patients with Gorlin Syndrome. Of the 477 targeted tumors studied throughout the clinical study, it was reported that 399 had a reduction of any size, 64 had no change and 14 increased in size. A clinically meaningful reduction of size of 30% or greater was found in 275 (57.7%) of the 477 tumors; 130 (27.3%) of the targeted tumors resolved completely. Inhibitor's plan for continued development of its itraconazole therapeutic include i) completing the new proprietary formulation inclusive of patent creation; ii) conducting/compiling results of the bridging PK study; and iii) preparing and submitting its IND. Inhibitor anticipates that it will be prepared to submit this IND to the FDA for their review and guidance by the end of First Quarter 2025. Announcement • Dec 20
Inhibitor Therapeutics, Inc. Announces the Use of Itraconazole to Treat Basal Cell Carcinoma Nevus Syndrome At the forefront of the Inhibitor Therapeutics, Inc. announced that itraconazole to treat basal cell carcinomas in Basal Cell Carcinoma Nevus Syndrome (BCCNS) a Gorlin Syndrome. This rare hereditary cancer syndrome is characterized by patients with hundreds to thousands of basal cell carcinomas. BCCNS is an orphan disease with approximately 11,000 patients in the USA. Inhibitor believes that itraconazole has clinically meaningful effects on the BCCs, which led to recruit a team of five experienced Mohs surgeons to form a Scientific Advisory Board (SAB) to critically review the outcomes of Inhibitor's phase 2b trial in BCCNS. They are academically affiliated, well-respected leaders in the practice of treating basal cell carcinomas and other skin cancers The purpose of the SAB is to assess the clinical benefits of adding itraconazole to the therapeutic options for the basal cell carcinomas in these patients. The initial focus of the SAB is a review of the final clinical outcomes of INTI's phase 2b study of itraconazole in BCCNS. Due to a recently resolved dispute with the former supply partner, the trial results are now available but have not been publicly released, nor shared with the trial investigators, or filed with the FDA for guidance. The Phase 2b SCORING Trial completed during 2018 assessed 477 pre-existing surgically eligible target lesions across 38 patients. Unlike virtually all other disseminated cancers that have a single primary with metastatic lesions, the standard measure of response is RECIST (Response Criteria in Solid Tumors) which are size criteria based upon an analysis of response by patient. Patients with BCCNS are predominantly concerned with the high number of surgical interventions required during their lifetime and the disfigurement that results from these procedures, particularly on areas of the body such as the face that are exposed to the public. Therefore, a therapy without significant toxicity that can reduce the number of lesions that require a scar producing intervention will fulfill an unmet need for these patients. The review of the Phase 2b Clinical Study Report by Scientific Advisory Board will focus on whether the SCORING trial is sufficiently clinically meaningful to warrant filing itraconazole for a New Drug Application ("NDA") as the first and only drug to be approved to treat BCCNS. The Gorlin Syndrome Alliance (a non-for-profit independent patient advocacy organization) (GSA) conducted an EL-PFDD (ex externally led patient focused drug development) meeting on October 8, 2021. This was conducted to help advance the GSA's mission to thoughtfully support, comprehensively educate, and aggressively seek the best treatments and a cure for those affected by Gorlin Syndrome. She received her undergraduate degree from Cornell University and her medical degree from Penn State University College of Medicine. She is a Mohs micrographic surgeon with more than 30 years' experience in Mohs Surgery and skin cancer management. She also has performed numerous clinical trials related to skin cancer. Dr. Billingsley is a past president of the American College of Mohs Surgery. She is affiliated with the Gorlin Syndrome Alliance and is a member of their Medical and Scientific Advisory Committee. Additionally, Dr. Christensen focuses on complex skin cancer issues such as field characterization, preventative strategies in high-risk patients and management of advanced or aggressive skin cancer. Dr. Christensen has published extensively on skin cancer pathogenesis and treatment and has experience in clinical trials for organizations such as the American Academy of Dermatology and the American College of Mohs surgery, and currently serves as the Treasurer for the International Transplant Skin Cancer Collaborative. 
