Moleculin Starts NIH-Funded Phase 2 Trial of STAT3 Inhibitor for Glioblastoma Treatment

Moleculin Biotech, Inc., a clinical-stage pharmaceutical company focused on developing treatments for challenging tumors and viruses, has announced a significant milestone. The company has commenced an Investigator-Initiated Phase 2 study at Northwestern University to evaluate the combination of WP1066 and radiation therapy in treating adult glioblastoma patients. This trial, designated NU 21C06, is conducted under Northwestern University's Investigative New Drug (IND) application, which references Moleculin's IND approved by the U.S. Food and Drug Administration (FDA) in April 2022. Funding for this important research comes from the National Institutes of Health (NIH) and BrainUp®, a non-profit organization committed to increasing awareness of brain cancer.

Dr. Priya Kumthekar, Associate Professor and Co-Investigator for the study, emphasized the pressing need for effective glioblastoma treatments. "Based on the data available, combining WP1066 with radiation therapy could provide a new treatment option for patients battling this aggressive cancer," she remarked. "We are excited to begin this critical study with Moleculin."

WP1066 is Moleculin's leading Immune/Transcription Modulator. This drug is designed to enhance the immune response against tumors by interfering with regulatory T cells' errant activity and inhibiting essential oncogenic transcription factors such as p-STAT3, c-Myc, and HIF-1α. Targeting these factors is vital due to their involvement in cancer cell survival, proliferation, angiogenesis, invasion, metastasis, and the inflammation associated with tumors.

The NU 21C06 trial is a Phase 2, open-label, multi-arm study focusing on newly diagnosed IDH wild-type, MGMT-unmethylated glioblastoma patients. The primary objective is to assess progression-free survival, with secondary outcomes examining the tumor microenvironment.

Dr. Amy Heimberger from Northwestern University highlighted the promising preclinical results of WP1066. "WP1066 has shown significant anti-tumor activity in various tumor cell lines and improved survival in multiple animal models. Combining WP1066 with radiation in glioma models produced both therapeutic responses and notable changes in immune surveillance within the gliomas, which we hope to replicate in human subjects," she explained.

Walter Klemp, Chairman and CEO of Moleculin, added that the company is also exploring WP1066 for pediatric brain tumors and other indications. "We are committed to its continued development and evaluation across additional indications," Klemp stated.

Glioblastoma is a prevalent brain tumor with an annual incidence rate of 3.19 per 100,000 people in the United States. This tumor is notoriously aggressive, with a median survival rate of just 15 months. Despite significant advancements in other cancer treatments, the survival rate for glioblastoma has remained relatively unchanged over the past three decades. Glioblastoma accounts for 54% of all gliomas and 16% of all primary brain tumors, underscoring the urgent need for new treatment options.

Moleculin has received Orphan Drug Designation for WP1066 for brain tumors and Rare Pediatric Disease designation for three pediatric indications. This underscores the drug's potential importance in treating these challenging cancers.

Moleculin Biotech, Inc., is actively developing a range of drug candidates, including Annamycin, a next-generation anthracycline for relapsed or refractory acute myeloid leukemia and soft tissue sarcoma lung metastases, and WP1220 for the topical treatment of cutaneous T-cell lymphoma. The company also focuses on antimetabolites like WP1122, with potential applications in treating both viral infections and cancers.

The commencement of the NU 21C06 Phase 2 study marks a pivotal step in Moleculin's efforts to develop innovative cancer treatments, particularly for glioblastoma, one of the most challenging brain tumors.

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