FDA Grants Rare Pediatric Disease Designation to Treatment for Ependymoma

Ependymoma is a rare type of tumor that develops in the brain or spinal cord and can occur at any age, but is most common in young children.

The FDA has awarded a Rare Pediatric Disease Designation (RPD) to WP1066, a p-STAT3 inhibitor that treats patients with ependymoma, a rare type of tumor that develops in the brain or spinal cord.

Ependymoma forms in the ependymal cells of the brain and spinal cord, which line the passageways through which cerebrospinal fluid feeds brain flows. The disease can occur at any age, but it is most common in young children.

WP1066, which appears to directly trigger the death of tumor cells and prompt the immune system to attack tumors, was previously granted 3 RPD indications in diffuse intrinsic pontine glioma, medulloblastoma and atypical teratoid rhabdoid tumors.

“The FDA’s recognition of the high prevalence and unmet needs in the ependymoma treatment landscape, especially in pediatric patients, is an important milestone as we continue to develop and expand the WP1066 development program,” said Walter Klemp, Chairman and CEO from Moleculin. , in a press release. “We currently have orphan drug designation for WP1066 for the treatment of brain tumors, as well as RPD designation for 3 other pediatric indications, and believe that ependymoma is another important rare indication.”

The new drug is an immune / transcription modulator that can directly inhibit certain important oncogenic transcription factors, including the activated form of STAT3 known as p-STAT3, which is considered a major regulator of tumor activity. The drug can also inhibit several other signaling proteins associated with tumor development and stimulates a natural immune response and memory to combat tumor progression.

WP1066 has demonstrated anti-tumor activity in a wide variety of tumor cell lines, indicating a survival advantage in several animal models. Furthermore, WP1066 can enhance the anti-tumor effects of the immune system and its response to tumor activity. Research also suggests that the agent may have a double-acting mechanism that allows the treatment of a range of resistant tumors and can be combined in new combinations for unresponsive tumors.

Subject to FDA approval of WP1066 for ependymoma, Moleculin Biotech, the drug developer, would be eligible for a voucher that could be redeemed to receive priority review for a subsequent marketing application for another product candidate or sold or transferred .

“We continue to be encouraged by the data that WP1066 has shown to date and believe it has the potential to be an effective therapy for pediatric patients with ependymoma,” Klemp said in the publication.

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