Oncoceutics Awarded FDA Orphan Grant For ONC201 Multiple Myeloma Trial

Philadelphia, PA (August 16, 2016) – Oncoceutics announced that it has been awarded an orphan grant from the U.S. Food and Drug Administration (FDA) to evaluate its lead molecule, ONC201, a selective antagonist of DRD2 that belongs to the superfamily of G protein-coupled receptors (GPCRs), in a multiple myeloma clinical trial.

The award totals $1.7 million in funding to support a Phase II program with ONC201 in relapsed/refractory multiple myeloma. Wolfgang Oster, MD PhD, and CEO of Oncoceutics, is the Principal Investigator for this clinical research program.

ONC201 has shown excellent anti-cancer activity in several challenging preclinical models of refractory multiple myeloma. In clinical trials, ONC201 has exhibited exceptional safety, a therapeutic pharmacokinetic (PK) profile, induction of pharmacodynamic (PD) markers, and early efficacy signals in a number of different cancer types, including Non-Hodgkin’s Lymphoma.

As previously reported in companion research articles published in the journal Science Signaling, ONC201 uses unique triggers to activate the integrated stress response (ISR), which upregulates a host of proteins that induce tumor cell death and inhibit the synthesis of proteins that are key for cancer growth. Multiple myeloma cells are particularly sensitive to ISR activation, which is also a key mechanism of action of proteasome inhibitors that are approved for the treatment of multiple myeloma.

“We are very pleased with the FDA’s support for our program in multiple myeloma, a hematologic malignancy that shows high responsiveness to ONC201 in numerous challenging preclinical models,” said Lee Schalop, MD, Chief Business Officer and co-founder of Oncoceutics. “This grant, along with other competitive grants and private-public partnerships that have been awarded to our ongoing programs with leading institutions, validates the significant opportunity that exists in our new chemical class of imipridones to address unmet needs in oncology.”

“While significant progress has been made in the field of multiple myeloma and the treatments have meaningfully improved, patients with resistant disease need additional options,” said Bart Barlogie, MD, Director of Research of the Myeloma Program of the Tisch Cancer Institute of the Icahn School of Medicine at Mount Sinai. “It is gratifying to see introductions of new classes of compounds, such as ONC201 that represents the first clinical stage drug of the imipridone family that offer disruptive approaches in how to tackle this challenging disease.”

About Oncoceutics

Oncoceutics, Inc. is a clinical-stage drug discovery and development company with a novel class of selective G protein-coupled receptor antagonists for oncology. The first lead compound to result from this program is ONC201, an orally active DRD2 small molecule antagonist that is well-tolerated and effective against advanced cancers. The company recently completed a successful Phase I study in solid tumors and has begun additional Phase I/II and Phase II clinical programs in both solid and hematological malignancies. Oncoceutics and collaborative groups have received more than $7 million in grants over the last two years, including grants from the National Cancer Institute, the U.S. Food and Drug Administration, the Pennsylvania Department of Health, and The Musella Foundation. In addition, outside interest in the company’s portfolio has resulted in several R&D alliance agreements between Oncoceutics and leading comprehensive cancer centers, including The University of Texas MD Anderson Cancer Center and the Fox Chase Cancer Center. The company has established a robust intellectual property position, including several issued patents.

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