Company Overview

Company Description

We are an innovative, clinical-stage biopharmaceutical company focused on the treatment of debilitating diseases through the acquisition, development, and commercialization of novel drug candidates with compelling mechanisms of action, regulatory pathways, and commercial opportunities.

Our lead product candidate, LX-101, is a novel, clinical-stage, next generation, targeted therapy directed to the insulin-like growth factor-1 (“IGF-1”) receptor (“IGF-1R”) with a differentiated payload-bearing mechanism of action. The IGF-1/IGF-1R pathway has been implicated in a host of malignancies and autoimmune diseases and we believe represents a scientifically and medically rational target with a multi-billion-dollar market opportunity. The IGF-1R target has been commercially validated, as Tepezza® (teprotumumab), an IGF-1R-directed, naked, non-payload-bearing, monoclonal antibody developed by one of our competitors, generated $1.97 billion in revenue in 2022. This was Tepezza’s third year on the market, and is approved in just one indication, an autoimmune condition known as thyroid eye disease (“TED”).

LX-101, formerly named 765IGF-MTX, a drug candidate for which Lirum has acquired a license, has been clinically evaluated by the licensor in Phase 1a trials of patients with advanced, pretreated cancer, where it was found to be well-tolerated and demonstrated single agent activity. We are developing LX-101 in certain oncology and autoimmune indications, including TED, and we are seeking to establish LX-101 as the standard of care for treating these diseases.

Our Approach

Our goal is to build a leading biopharmaceutical company. We intend to leverage our experience and expertise in drug development and commercialization to create a pipeline of differentiated therapies for debilitating diseases with high unmet medical need. We plan to advance our agents into and through regulatory pathways for indications with attractive market opportunities and commercialize our products.

Our lead product candidate, LX-101, is a novel, clinical stage, next generation, precision-engineered targeted therapy directed to the IGF-1R. The IGF-1/IGF-1R pathway has been implicated in a host of diseases. Moreover, IGF-1R has been commercially validated, and we believe represents a scientifically and medically rational target with a multi-billion-dollar market opportunity.

LX-101 consists of a proprietary, precision-engineered IGF-1 variant coupled to a cytotoxic payload, methotrexate. Methotrexate is a cytotoxic inhibitor of DNA synthesis, repair, and cellular replication and has been used to treat patients with certain cancers and autoimmune diseases, including TED, making it, we believe, a rational payload to employ in these disease settings.

LX-101 has been clinically evaluated by the licensor in Phase 1a trials of patients with advanced, pretreated cancer, where it was found to be well-tolerated and demonstrated single agent activity. Nineteen patients were enrolled with advanced, pretreated solid or hematologic tumors that expressed some degree of IGF-1R (i.e., IGF-1R low-high). We are encouraged that there were patients who experienced disease control, including responses, at the tested doses. This includes a partial response (“PR”) in a patient with basal cell carcinoma that metastasized to the lung who was treated at the highest tested dose. There was also activity and disease control at lower doses, including the following: one bone marrow complete response (“BMCR”) in a patient with advanced myelodysplastic syndrome (“MDS”) and four stable diseases (“SD”), including one with a pathologic complete response, in patients with colon cancer, MDS, endometrial cancer, and Hodgkin’s lymphoma, respectively. Neither a dose limiting toxicity (“DLT”) nor a maximum tolerated dose (“MTD”) were reached and, accordingly, we plan to further dose escalate and optimize the administration schedule.

We believe that leveraging LX-101’s novel and differentiated features, along with the well-documented involvement of IGF-1R in a multitude of diseases, will support our efforts to maximize the therapeutic and commercial potential of LX-101. As a next step, we plan to build upon the Phase 1a clinical experience and advance LX-101 through additional clinical trials in select oncology and TED indications. Positive data in these settings, we believe, will be both value-creating as well as informative for our navigation of LX-101 through potentially expedited development pathways, although we cannot be assured that LX-101 or any future products will qualify, to attractive market opportunities. We also intend to expand our portfolio via acquisitions of additional drug candidates that meet our criteria for successful drug development and commercialization. With this approach, we believe that we can create a positive impact on patients while creating shareholder value.

We were incorporated in November 2021 under the laws of the State of Delaware under the name Lirum Therapeutics, Inc. Our principal executive offices are located at 590 Madison Ave, 21st Floor, New York, NY 10022, and our telephone number is (646) 964-1402. Our website address is www.lirumtx.com.