Initial Patients Treated in Phase 2 Trial of STAT3 Inhibitor with Radiation for Glioblastoma

HOUSTON, Sept. 9, 2024. Moleculin Biotech, Inc., a Phase 3 clinical-stage pharmaceutical company, has commenced patient enrollment and treatment in a Phase 2 study of WP1066 combined with radiation therapy for glioblastoma. This research is under Northwestern University's Investigative New Drug application, referencing Moleculin’s own IND, which was FDA-approved in April 2022. The trial is sponsored by the NIH and BrainUp, a nonprofit organization focused on brain cancer awareness.

The Phase 2, open-label, multi-arm trial, identified as NU 21C06, investigates the combination of WP1066 with radiation therapy for newly diagnosed IDH wild-type, MGMT-unmethylated glioblastoma patients. The primary focus of this study is to measure progression-free survival, while secondary goals include analyzing the tumor microenvironment.

Dr. Priya Kumthekar, Associate Professor at Northwestern University and Co-Investigator of the study, highlighted the importance of starting patient treatment in addressing the unmet needs for glioblastoma therapies. She expressed optimism based on preclinical data showing that combining WP1066 with radiation therapy has enhanced survival rates and induced anti-tumor immune responses.

Dr. Amy Heimberger, Co-Investigator and Vice Chair for Research at Northwestern University’s Department of Neurological Surgery, echoed this sentiment. She noted the rapid recruitment pace for the trial, with four participants currently active and an anticipated completion of the safety lead-in group of six subjects shortly. If these initial treatments are well-tolerated, the study will continue to enroll more patients.

WP1066, Moleculin’s leading Immune/Transcription Modulator, aims to stimulate the immune system against tumors by inhibiting the faulty activity of regulatory T cells and key oncogenic transcription factors such as p-STAT3, c-Myc, and HIF-1α. These transcription factors are critical due to their involvement in cancer cell survival, proliferation, angiogenesis, metastasis, and inflammation.

Walter Klemp, Moleculin’s Chairman and CEO, emphasized the vital role of the NU 21C06 study in advancing STAT3-targeting technologies. STAT3 has been a challenging target in cancer therapy, but the collaborative efforts between Moleculin and Northwestern University, backed by the NIH and BrainUp, hold promise. Klemp believes that WP1066 could demonstrate significant anti-tumor activity across various hard-to-treat cancers.

Glioblastoma, originating in the brain, has an average annual incidence rate of 3.19 per 100,000 people in the United States. It is the most aggressive primary brain tumor, with a median survival of merely 15 months. Glioblastoma constitutes 54% of all gliomas and 16% of all primary brain tumors. Despite advancements in other cancer treatments, the survival rate for glioblastoma has remained stagnant over the past three decades.

Moleculin has received Orphan Drug Designation for WP1066 for brain tumors and Rare Pediatric Disease designation for three other pediatric indications.

About Moleculin Biotech, Inc.
Moleculin Biotech, Inc. is a Phase 3 clinical-stage pharmaceutical company developing therapies for hard-to-treat tumors and viruses. The company's lead program, Annamycin, is a next-generation anthracycline designed to overcome multidrug resistance and avoid the cardiotoxicity associated with current anthracyclines. Annamycin is being developed for treating relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases.

Moleculin is launching the MIRACLE (Moleculin R/R AML AnnAraC Clinical Evaluation) Trial (MB-108), a pivotal Phase 3 trial evaluating Annamycin with cytarabine (AnnAraC) for relapsed or refractory AML. Following a successful Phase 1B/2 study (MB-106), with guidance from the FDA, Moleculin believes it is closer to potential approval for Annamycin for AML.

Additionally, Moleculin is advancing WP1066, which inhibits p-STAT3 and other oncogenic transcription factors while boosting immune response, targeting brain tumors, pancreatic cancers, and others. The company is also working on WP1122, an antimetabolite for treating viruses and certain cancers.

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