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Kintara’s VAL-083 Gets Orphan Drug Status for Rare Childhood Cancer

Kintara’s VAL-083 Gets Orphan Drug Status for Rare Childhood Cancer

The FDA has granted Orphan Drug status to Kintara Therapeutics’ VAL-083 candidate for the treatment of diffuse intrinsic pontine glioma, a rare, very aggressive and hard to treat childhood brainstem tumor.

VAL-083 is a chemotherapeutic that has shown potential effects against several cancers, including in the central nervous system, ovaries, lung, bladder, head and neck.

The drug is being advanced into a phase 2/3 clinical trial supported by the company’s internal research and National Cancer Institute-sponsored clinical studies.

Orphan Drug status is given to investigational drugs or biological products to prevent, diagnose or treat a rare disease or condition. Sponsors benefit from tax credits for qualified clinical trials, exemption from user fees and the potential for seven years of market exclusivity.


Located in San Diego, California, Kintara is dedicated to the development of novel cancer therapies for patients with unmet medical needs. Kintara is developing two late-stage, Phase 3-ready therapeutics for clear unmet medical needs with reduced risk development programs. The two programs are VAL-083 for glioblastoma (GBM) and REM-001 Therapy for CMBC.

VAL-083 is a 'first-in-class', small-molecule chemotherapeutic with a novel mechanism of action that has demonstrated clinical activity against a range of cancers, including central nervous system, ovarian and other solid tumors (e.g., NSCLC, bladder cancer, head and neck) in U.S. clinical trials sponsored by the National Cancer Institute (NCI). Based on Kintara's internal research programs and these prior NCI-sponsored clinical studies, Kintara is currently advancing VAL-083 in the Global Coalition for Adaptive Research registrational Phase 2/3 clinical trial titled Glioblastoma Adaptive Global Innovative Learning Environment (GBM AGILE) Study to support the development and commercialization of VAL-083 in GBM.

Kintara also has a proprietary, late-stage photodynamic therapy platform that holds promise as a localized cutaneous, or visceral, tumor treatment as well as in other potential indications. REM-001 Therapy, which consists of the laser light source, the light delivery device, and the REM-001 drug product, has been previously studied in four Phase 2/3 clinical trials in patients with CMBC who had previously received chemotherapy and/or failed radiation therapy. In CMBC, REM-001 has a clinical efficacy to date of 80% complete responses of CMBC evaluable lesions and an existing robust safety database of approximately 1,100 patients across multiple indications. Kintara has paused the REM-001 CMBC program to conserve cash resources.

December 19, 2022



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