Adastra Pharmaceuticals Announces FDA and EMA Orphan Drug Designation Has Been Granted for Zotiraciclib in the Treatment of Glioma

On December 4, 2019 Adastra Pharmaceuticals, Inc., a biopharmaceutical company focused on the development of first-in-class therapeutics for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted Orphan Drug Designation to zotiraciclib for the treatment of glioma (Press release, Adastra Pharmaceuticals, DEC 4, 2019, View Source [SID1234551941]). Zotiraciclib is currently the subject of two separate Phase 1b clinical trials in glioblastoma (GBM). Adastra expects to report results from the two studies during 2020.

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The first Phase 1b clinical trial, sponsored by the National Cancer Institute (NCI), is a multi-arm, dose-finding study examining the combination of zotiraciclib and temozolomide (TMZ) for the treatment of recurrent malignant gliomas. Having recently enrolled the Phase 1 portion, the NCI and Adastra expect to report top-line data early in 2020, expanding upon results previously reported at the 2019 ASCO (Free ASCO Whitepaper) Annual meeting and also via e-Poster at the 2019 Society of Neuro Oncology.

The second Phase 1b trial of zotiraciclib is being conducted by the European Organisation for Research and Treatment of Cancer (EORTC) and is designed as a three-parallel cohort, open-labeled, non-randomized, multicenter study in elderly patients with IDH1R132H-non mutant and MGMT promoter-unmethylated anaplastic astrocytoma or glioblastoma. Group A consists of First-Line patients with unmethylated disease and is designed to determine the application of zotiraciclib in combination with radiation. Group B consists of First-Line patients with methylated disease and is designed to determine zotiraciclib use in combination with TMZ. Group C consists of patients with recurrent GBM and is single agent zotiraciclib intended to measure progression free survival at six months.

"We are very pleased to have been granted Orphan Drug Designation by the FDA and EMA for zotiraciclib in the treatment of glioma. These designations are important in the continued development of zotiraciclib and the ongoing clinical trials in the U.S. at the NCI and throughout Europe with the EORTC," said Scott Megaffin, Chief Executive Officer of Adastra. "Though rare, glioblastoma is one of the most devasting and difficult-to-treat cancers, urgently necessitating the development of new treatments with unique mechanisms of action. Data from the ongoing clinical trials of zotiraciclib have been encouraging and are suggestive of a therapy that is safe and capable of eliciting clinical benefit in high grade gliomas. We greatly look forward to reporting top-line data from the NCI Phase 1b study in early 2020, as well as completing enrollment in two of the EORTC study cohorts later in the year."

About Orphan Drug Designation
The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. and not more than five in 10,000 in the E.U. Orphan Drug Designation provides important incentives, including marketing exclusivity upon approval and tax credits for qualified clinical testing, as well as fee exemptions and reductions.