Inhibitor Therapeutics, Inc.

Overview

We are a pharmaceutical development company focused on the development and potential commercialization of therapeutics based on already approved active pharmaceutical ingredients, with our primary current focus on basal cell carcinoma nevus syndrome (“BCCNS”), also known as Gorlin Syndrome. Our business strategy is centered on repurposing well-characterized drug substances and, where appropriate, supporting those programs with differentiated formulation, regulatory and intellectual property strategies intended to create a more efficient development path and enhance commercial value.

Our lead program is based on itraconazole, an FDA-approved antifungal agent, for the chronic management of basal cell carcinomas in patients with BCCNS. We believe BCCNS represents a compelling initial indication because it is a serious, lifelong condition with significant recurrent procedural burden, limited durable treatment options, and a specialist-managed patient population. At present, our business model is primarily oriented toward advancing this program through regulatory development and potential strategic transactions, including licensing, partnership or divestment.

Recent Program Developments

In February 2026, with support from Frameshift Management, Inc. (“Frameshift”), we submitted a formal meeting request and briefing materials to the U.S. Food and Drug Administration (“FDA”) regarding our proposed development pathway for itraconazole in BCCNS. The FDA has since informed us that the request has been granted as a Type C meeting, with written responses expected in May 2026. We intend to request that the FDA provide a videoconference in lieu of written responses. The meeting request asks the Agency to address, among other topics, the appropriateness of per-lesion response as a primary efficacy endpoint in BCCNS, the sufficiency of the existing HP2001 efficacy and safety dataset, and the potential use of expedited programs and a 505(b)(2) new drug application pathway.

We have also engaged Avior Bio, Inc. (“Avior”) to develop a proprietary micronized/amorphous itraconazole formulation intended to support our BCCNS program. Formulation development has been completed, and we are pursuing pharmacokinetic work designed to compare the Avior formulation to previously studied itraconazole formulations used in clinical settings. We believe this formulation strategy may support a differentiated chemistry, manufacturing and controls package, may improve bioavailability and plasma consistency, and may enhance the economic durability of the program if paired with new patent protection and orphan-drug exclusivity.

As part of our development activities, we also maintain relationships with patient advocacy groups and incorporate insights from these interactions into our development strategy.

Selected Program Metrics

Disease Background and Unmet Need

BCCNS is a rare hereditary cancer predisposition syndrome characterized by the continuous development of basal cell carcinomas over a patient’s lifetime. The syndrome has been described as affecting approximately 1 in 30,000 to 1 in 31,000 individuals (Bree & Shah, 2011; National Organization for Rare Disorders), and our current internal estimates assume a U.S. patient population of approximately 11,000 based on these prevalence rates and U.S. Census data. Patients often begin developing tumors at a relatively young age, and the disease course is chronic rather than episodic (Kimonis et al., 1997).

The burden of BCCNS is not limited to the number of tumors. In the Gorlin Syndrome Alliance Voice of the Patient report submitted to the U.S. Food and Drug Administration, patients described having dozens to more than one thousand basal cell carcinomas removed over the course of their lives, with some individuals undergoing a substantial number of surgical interventions (Gorlin Syndrome Alliance, 2018). Patients and caregivers describe the disease as physically painful, psychologically burdensome and progressively disfiguring. We believe these characteristics distinguish BCCNS from more typical presentations of basal cell carcinoma and support the view of the disease as a chronic condition requiring ongoing management.

Current Treatment Paradigm

Patients with BCCNS are typically managed by dermatologists and frequently require treatment by Mohs micrographic surgeons as part of ongoing care. As a result, we believe that Mohs surgeons represent a concentrated and identifiable group of specialists involved in the management of this patient population. We further believe that this group consists of approximately 1,000 to 1,500 specialists in the United States, and that this concentrated prescriber base may be relevant in the context of potential future commercialization or strategic transactions.

Management of basal cell carcinomas in patients with BCCNS remains predominantly procedural. Standard care generally consists of repeated Mohs micrographic surgery, surgical excision, curettage, electrodessication and other lesion-directed dermatologic interventions as tumors arise (National Comprehensive Cancer Network; American Academy of Dermatology). These approaches are effective for removing individual lesions but do not address the underlying predisposition to continued tumor development.

In selected cases, systemic therapies such as Hedgehog pathway inhibitors may be used, particularly in patients with locally advanced or inoperable disease. Approved agents including vismodegib and sonidegib have demonstrated clinical activity in basal cell carcinoma; however, their use may be limited by tolerability considerations and discontinuation rates in some patients (Sekulic et al., 2012; Migden et al., 2015). As a result, many patients with BCCNS continue to rely primarily on repeated procedural interventions over time.

We believe the current treatment paradigm reflects a reactive, lesion-by-lesion approach to disease management, which may result in a cumulative procedural burden for patients given the chronic and recurring nature of BCCNS.

Illustrative Monthly Procedural Cost Ranges in BCCNS

Our Program and Clinical Experience with Itraconazole

Our lead clinical dataset comes from Study HP2001, a Phase IIb open-label study of itraconazole in patients with BCCNS. According to our February 2026 FDA meeting materials, the study enrolled 38 patients with 477 surgically eligible target basal cell carcinomas at baseline. In those materials, we reported a per-lesion objective response rate of 57.7% and a patient-level disease control rate of 97.4%.

In a separate scientific and strategic review prepared for the program, we further summarized lesion-level outcomes as follows: 27.3% of lesions resolved completely, an additional 30.4% achieved partial response, 39.4% remained stable or modestly reduced, and only 2.9% exhibited measurable growth during therapy. That review also reported a median duration of tumor reduction of 505 days. We believe these data support the concept of itraconazole as a chronic tumor-control therapy for a disease defined by repeated emergence of independent lesions.

Our FDA briefing materials also emphasize that BCCNS presents a unique endpoint challenge because patients develop numerous independent primary tumors rather than a single clonally related mass. For that reason, we have sought Agency feedback on whether per-lesion analysis is an appropriate primary efficacy framework for this indication and whether the existing HP2001 dataset, together with the known safety profile of itraconazole, may support an NDA strategy.

Scientific Rationale: Anti-Angiogenic Activity

Our scientific positioning for itraconazole in BCCNS is centered on anti-angiogenesis. Itraconazole has been reported to interfere with vascular endothelial growth factor receptor 2 (“VEGFR2”) trafficking and glycosylation, thereby reducing VEGF-driven signaling and neovascularization (Chong et al., 2007; Aftab et al., 2011). Published preclinical studies cited in our scientific review indicate that itraconazole can reduce endothelial migration, sprouting and microvessel density, effects that are directly relevant to tumor vascular support (Chong et al., 2007).

Our materials also describe a complementary endothelial mechanism involving VDAC1, AMP-activated protein kinase and mTORC1. In this framework, itraconazole disrupts endothelial bioenergetics, activates AMPK and suppresses mTORC1, which can reduce endothelial proliferation and angiogenesis (Aftab et al., 2011). We believe this anti-angiogenic profile is particularly relevant in BCCNS because patients develop continual waves of new lesions over time, and vascular suppression offers a disease-control rationale not limited to a single lesion or treatment episode.

A further advantage for a cutaneous oncology program is skin exposure. Published pharmacokinetic work summarized in our scientific review indicates that itraconazole achieves sebum concentrations approximately 5 to 10 times higher than plasma, with sustained deposition in the stratum corneum after treatment (Abuhelwa et al., 2015). We believe this skin-distribution profile supports the biological plausibility of chronic oral itraconazole as a systemic therapy for a disease manifested primarily in the skin.

Formulation Strategy, Avior Relationship and Economic Implications

We engaged Avior to develop what we refer to internally as Micro-ITRA, a proprietary micronized/amorphous itraconazole formulation intended for long-term administration in BCCNS. We retain all rights to this formulation and any associated intellectual property arising from its development. Our scientific materials describe this formulation as designed to improve dissolution, bioavailability and plasma consistency. We believe these characteristics may be important both clinically and commercially: clinically, because they may support more predictable systemic exposure over chronic dosing; commercially, because a differentiated formulation may provide a basis for patent protection and help distinguish our program from commodity itraconazole products. We believe this formulation represents a core proprietary component of our development strategy and is intended to serve as the basis for our regulatory, intellectual property and commercial positioning.

We intend to pursue patent protection covering the Avior formulation and related methods of use. If obtained, this protection could operate alongside existing and potential regulatory exclusivities to strengthen the durability of the asset. From an economic standpoint, additional formulation-based protection may reduce substitution risk, support lifecycle management, extend the period over which we may negotiate commercial value with partners and enhance the overall strategic attractiveness of the program in a licensing or divestment process. We believe that ownership of a differentiated formulation may be important in supporting exclusivity, reducing generic substitution risk and enhancing the potential value of the program in any future strategic transaction.

We also believe the formulation work may fit well with a 505(b)(2) strategy. FDA guidance explains that section 505(b)(2) can be appropriate for applications that rely in part on investigations not conducted by or for the applicant and for which the applicant does not have a right of reference, including published literature or prior Agency findings of safety and effectiveness for an approved drug. Subject to FDA feedback, we believe that pairing existing itraconazole clinical and safety knowledge with a differentiated formulation and appropriate bridging data could create an efficient regulatory and economic pathway for the program.

Regulatory Strategy and Potential Exclusivity

Our current regulatory focus is on obtaining FDA alignment regarding endpoint selection, data sufficiency and filing strategy. The FDA’s formal meeting guidance states that Type C meetings are used to discuss development and review issues other than those assigned to Type A or Type B meetings. Through the Type C interaction granted for our program, we are seeking guidance on whether the HP2001 dataset can support a registration strategy, whether per-lesion response is an acceptable efficacy framework in BCCNS and whether our program may be appropriate for expedited programs.

We are also evaluating potential eligibility for Fast Track designation, Breakthrough Therapy designation, Accelerated Approval and Priority Review. FDA describes these programs as tools intended to expedite development or review for therapies that address serious conditions and unmet medical need. We already hold orphan-drug designation for itraconazole in BCCNS. FDA states that orphan designation provides the potential for seven years of market exclusivity after approval, separate from patent rights, for the approved rare-disease use if statutory and factual requirements are satisfied at approval.

Taken together, we believe our regulatory strategy is important not only for development efficiency but also for asset value. A successful 505(b)(2) path, if available, may reduce development complexity relative to a full development program. Orphan exclusivity, if realized upon approval, may provide a protected commercial window. Additional formulation patent protection, if granted, could extend the practical economic life of the asset beyond regulatory exclusivity.

Market Opportunity and Economic Framework

Our internal economic report frames BCCNS as a chronic specialty dermatology opportunity rather than a conventional acute-treatment market. Using an estimated U.S. BCCNS population of approximately 11,000 patients (Bree & Shah, 2011; National Organization for Rare Disorders), one-third market penetration would correspond to roughly 3,700 treated patients annually. At an illustrative therapy price of $4,000 per month, annual gross revenue at that penetration level would be approximately $178 million; at $5,000 per month, annual gross revenue would be approximately $222 million.

We view those price levels as grounded in the structural economics of current care (Guy et al., 2015; American Academy of Dermatology). Our economic work emphasizes that itraconazole is intended to shift care from episodic, procedure-driven intervention toward more predictable chronic disease management, not to eliminate surgery entirely. In that framework, the commercial value proposition is tied to reducing procedure frequency and intensity over time, improving predictability for patients and payors, and offering a fixed-price systemic approach in a population defined by lifelong recurrent procedural burden.

Although any commercial outcome will depend on regulatory approval, physician adoption, reimbursement, competition and other factors, we believe the BCCNS program has attributes that are attractive from an economic standpoint: a defined rare-disease population, a concentrated prescriber base, significant unmet need, relatively straightforward oral administration, and the potential for layered exclusivity if orphan protection and formulation patents are realized.

Illustrative Economic Snapshot

Intellectual Property

On December 12, 2023, we entered into an Exclusive License Agreement with Johns Hopkins University pursuant to which we obtained exclusive worldwide rights to U.S. Patent No. 8,980,930, entitled “New Angiogenesis Inhibitors.” We believe this licensed asset supports the development of itraconazole as an oncology therapeutic and remains an important foundational component of our intellectual property estate.

In addition to this licensed patent, our current intellectual property efforts are focused on the Avior formulation and related methods of treatment in BCCNS. We intend to continue evaluating additional patent opportunities that may strengthen the program’s defensibility and commercial life. We also rely on trade secrets, know-how and development data as part of our overall strategy to protect program value.

Commercial and Strategic Alternatives

We do not currently market any FDA-approved products and have not established a commercial sales infrastructure. At this stage, our primary commercial strategy is to advance the BCCNS program through regulatory development toward potential approval and to evaluate strategic transactions that may maximize the value of the program. These may include regional or global licensing, co-development arrangements, strategic partnerships or divestment of the program or related assets.

We are actively evaluating potential strategic transactions and may engage with pharmaceutical or biotechnology companies that have development, regulatory and commercial capabilities in dermatology or oncology. Any such transaction may include upfront payments, development and regulatory milestones, royalties on future net sales or other forms of consideration, although there can be no assurance that any agreement will be completed on acceptable terms, or at all.

We believe that pursuing strategic transactions may allow us to leverage external development, regulatory and commercial capabilities while reducing capital requirements and execution risk. In addition, we believe that advancing the program through key regulatory milestones, including potential FDA feedback and, if applicable, approval, may enhance its attractiveness as a potential licensing or divestment opportunity. We may seek to retain economic participation in any such transaction through milestone payments, royalties or other structured consideration.

Competition

The BCCNS treatment landscape includes repeated procedural intervention, non-pharmacologic management strategies such as rigorous sun protection, and systemic treatment approaches that may be used in selected settings. Competition may also arise from companies pursuing therapies for basal cell carcinoma or rare dermatology populations more broadly.

Our principal competitive challenge is not solely another drug candidate, but the entrenched procedural standard of care. We believe our program is differentiated by its chronic disease-control positioning, oral route of administration, existing body of itraconazole safety knowledge, anti-angiogenic rationale, and the possibility of supporting differentiated access and exclusivity through formulation and regulatory strategy.

Government Regulation

Pharmaceutical products are subject to extensive regulation in the United States and other jurisdictions. In the United States, the FDA regulates, among other things, preclinical and clinical development, manufacturing, labeling, approval, marketing, promotion and post-approval reporting. Before a drug may be marketed in the United States, the sponsor generally must complete applicable development work, submit an NDA and obtain FDA approval.

Our program is expected to be regulated as a drug product. Depending on FDA feedback, our future regulatory path may involve a 505(b)(2) NDA strategy, chemistry, manufacturing and controls work related to our proprietary formulation, inspection of manufacturing facilities and, if applicable, post-approval commitments or pharmacovigilance requirements.

Human Capital Resources

As of the date of this Report, we have two full-time employees and six part-time employees. The full-time employees include our Executive Chairman who is involved in our clinical development program history and status, as well as vetting additional opportunities and operations, as well as our Vice President of Operations. The part-time employees include our Interim CFO, as well as administrative, legal and accounting functions. None of our employees are covered by collective bargaining agreements. From time to time, we also employ independent contractors to support our clinical development and administrative functions. We consider the number of our employees, their compensation, and their functions to be appropriate for the current status of our business, and we also consider relations with each of our employees to be good. Each of our employees has entered into confidentiality, intellectual property assignment and non-competition agreements with us.

Corporate History

We were founded under the name “Commonwealth Biotechnologies, Inc.” in Virginia in 1992, and completed an initial public offering in October 1997 (we refer to our company prior to our emergence from bankruptcy as CBI). CBI previously provided, on a contract basis, specialized life sciences services to the pharmaceutical and biotechnology sector. On January 20, 2011, CBI filed a voluntary petition for bankruptcy. We recommenced our business operations in August 2013 as a Delaware corporation following the emergence of CBI from its voluntary bankruptcy.

After approximately five years of extended litigation between a minority shareholder and the former majority shareholder of the Company, the matter was finalized through mediation in 2022. As part of the settlement agreement, the former majority shareholder surrendered all equity securities of the Company for cancellation. Certain defendants in the litigation were directors and officers of the Company at the time, and as a result of the legal settlement, resigned from these positions. New officers and directors were then elected and the Company continued under new management.

Available Information

Our Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K, and amendments to reports filed pursuant to Sections 13(a) and 15(d) of the Exchange Act are filed with the SEC. Such reports and other information that we file with the SEC are available free of charge on our website at http://www.inhibitortx.com when such reports are available on the SEC website. The SEC maintains an Internet site that contains reports, proxy and information statements and other information regarding issuers like us that file electronically with the SEC at http://www.sec.gov. The contents of these websites are not incorporated into this filing. Further, the foregoing references to the URLs for these websites are intended to be inactive textual references only.