FDA Grants Rare Pediatric Disease Designation to WP1066 for Patients With Ependymoma


The FDA has granted a rare pediatric disease designation to the novel p-STAT3 inhibitor WP1066 for the treatment of patients with the rare brain and spinal cord malignancy, ependymoma.

The FDA has granted a rare pediatric disease (RPD) designation to the novel p-STAT3 inhibitor WP1066 for the treatment of patients with the rare brain and spinal cord malignancy, ependymoma, according to a press release from the developer, Moleculin Biotech, Inc.

"The FDA's recognition of the high prevalence and unmet needs in the treatment landscape for ependymoma, especially in pediatric patients is a significant milestone as we continue to advance and expand the WP1066 development program. 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 represents another important rare indication. We continue to be encouraged by the data WP1066 has demonstrated to date and believe it has the potential to be an effective therapy for pediatric patients with ependymoma," said Walter Klemp, the chairman and chief executive officer of Moleculin Biotech, Inc, in the press release.

WP1066 is currently under active investigation in 2 phase 1 studies as treatment of pediatric patients with malignant brain tumors like malignant glioma, glioblastoma, and medulloblastoma. In one of the phase 1 study exploring the agent a treatment of progressive medulloblastoma, malignant glioma or any other recurrent/progressive malignant brain tumor (NCT04334863), an estimated 36 patients will be enrolled.The co-primary end points of the study include finding the maximum tolerated dose of the agent as well as safety and tolerability. The secondary end points include pharmacokinetics, along with change in overall response rate, change up immunological response, time to response or progression, change in progression-free survival, and change in overall survival up to 2 months after being dosed with WP1066.

Patients eligible for enrollment include those aged 3 to 25 years old who have histologically confirmed disease who have previously undergone standard-of-care treatment and recovered from acute treatment-related toxicities. Patients are required to have a Karnofsky or Lansky Performance Scale score greater than 60% as well as normal organ and marrow function. Any patients who experience seizures must be stable before entering the study.

Exclusion from the study is based on the receipt of prior anti-cancer therapy, or biologic or investigational agent or other therapies too close to the time of dosing in the study. Some patients were also excluded if they have a history of allergic reactions attributed to compounds of similar to WP1066, an uncontrolled intercurrent illness, human immunodeficiency virus, uncontrolled seizures, and cardiac irregularity.

Based on this RPD, WP1066 may later be granted a rare pediatric disease priority review for a marketing application as part of the FDA’s Safety and Innovation Act, which was curated to streamline the development of novel therapies for rare pediatric diseases.


Moleculin awarded new rare pediatric disease Designation from U.S. FDA for WP1066 for the treatment of ependymoma. News release. April 14, 2021. Accessed April 14, 2021. https://bit.ly/3tjHSni

Related Videos