Lirum Therapeutics Announces FDA “Study May Proceed” Letter Enabling Inclusion of LX-101 in the RAPID Platform Clinical Trial for Ewing Sarcoma and Desmoplastic Small Round Cell Tumor
New York, NY, Oct. 20, 2025 (GLOBE NEWSWIRE) -- Lirum Therapeutics, Inc. (“Lirum”), an innovative clinical-stage biopharmaceutical company focused on the treatment of debilitating diseases, announced today that the U.S. Food and Drug Administration (FDA) has issued a “Study May Proceed” letter enabling LX-101 to be evaluated within their platform trial in adolescent and young adult patients diagnosed with relapsed/refractory Ewing Sarcoma (ES) or desmoplastic small round cell tumor (DSRCT), two aggressive orphan cancers with high unmet medical need.
The RAPID (Rapidly Assess Promising Innovative Drugs), is a patient-centric, multicenter, open-label platform trial led by Joseph Ludwig, M.D., professor at The University of Texas MD Anderson Cancer Center. It is designed to evaluate promising therapies for adolescent and young adult patients, similar to other highly successful platform trials, RAPID employs a master protocol that enables the study of both single-agent and novel drug combinations in patients with relapsed/refractory ES and DSRCT.
Recently, at the 2025 American Association for Cancer Research (AACR) Annual Meeting, preclinical data highlighting LX-101’s broad activity across pediatric cancers with well-established ties to the IGF-1R pathway was presented. The data demonstrated LX-101’s potent activity against ES in both in vivo and in vitro models, and against DSRCT in in vitro models. Notably, these cancers have strong ties to IGF-1R, including DNA-level gene fusions (i.e., EWSR1-FLI1 in Ewing’s and EWSR1-WT1 in DSRCT) that affect IGF-1R signaling, providing a strong scientific and clinical rationale for LX-101 in these indications.
About Lirum Therapeutics, Inc.
Lirum is 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. Lirum’s lead candidate, LX-101, is a novel clinical-stage targeted therapy directed to the insulin-like growth factor 1 receptor (IGF-1R) with a differentiated mechanism of action. LX-101 is currently being manufactured for clinical use with plans to re-enter human trials in novel oncology and autoimmune indications, including thyroid eye disease (TED). For more information on Lirum, please visit www.lirumtx.com.
Forward Looking Statements
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Company Contact:
Matt Hoberman
Investor Relations
IR@lirumtx.com
Phone: (646) 389-6015
Lirum Therapeutics, Inc.
1270 Avenue of the Americas, 7th Floor
New York, NY 10020
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