EXCLUSIVE: Pediatric Patients Could Benefit Most From New Oncoceutics Cancer Drug, Says Exec

Published: Jul 02, 2015

EXCLUSIVE: Pediatric Patients Could Benefit Most From New Oncoceutics Cancer Drug, Says Exec
July 2, 2015
By Riley McDermid, BioSpace.com Breaking News Sr. Editor

The novelty and safety of Oncoceutics’ new cancer drug ONC201 is particularly promising for the treatment of pediatric patients, and may be an option when other therapies are no longer effective, a company executive told BioSpace Thursday.

This week Oncoceutics, Inc. announced the publication of novel findings in the journal Cell Cycle that showed ONC201 is an effective anti-cancer agent against pediatric non-Hodgkin’s lymphoma (NHL) when used as a monoagent. It was also promising in combination with other chemotherapeutic agents.

Joshua E. Allen, vice president of Research & Development at Oncoceutics, Inc., told BioSpace that the anti-cancer activity of ONC201 has been demonstrated in several preclinical models, including refractory solid tumors and a transgenic lymphoma model.

“In this study, the authors report that ONC201 causes cell death in pediatric as well as adult NHL cell lines. Molecular studies identified a biomarker that correlates with response and could be used for further clinical studies,” he said.

A clinical study that will follow-up these preclinical findings will begin soon at The University of Texas MD Anderson Cancer Center under Michael Wang, MD, Professor, Department of Lymphoma/Myeloma, and a leading specialist in NHL. BioSpace talked with Allen about why ONC201 appears to be working so well and what could be on the horizon for the rest of 2015.

BIOSPACE: In “layman’s terms”, how does ONC201 therapy work, and why does that make it effective?

ONC201 is an oral, small-molecule drug that acts against numerous types of cancer tumors by sparing healthy cells and targeting the most potent tumor suppressor pathways. ONC201 acts against a wide range of tumor types and overcomes complex mechanisms used by cancer cells to develop therapeutic resistance, including cancer stem cells. ONC201 works by specifically engaging signaling pathways in tumor cells that are individually recognized for their ability to shrink tumors in the clinic, and it does this by a unique trigger with a selectivity that spares normal cells.

BIOSPACE: How is this new data compelling or different?

This new data points out two specific types of non-Hodgkin’s lymphoma where the combination of strong efficacy and excellent safety profiles of ONC201 could be of particularly high clinical value. The results suggest that mantle cell lymphoma and Burkitt’s lymphoma cells that are resistant to standard-of-care therapies are highly responsive to ONC201 as a single agent. This lays the groundwork for future clinical studies and reinforces the motivation for the phase I/II non-Hodgkin’s lymphoma trial with ONC201 at MD Anderson Cancer Center. The new data also suggests that if ONC201 needs to be combined in the clinic with other therapies in hematological malignancies, cytarabine is a good candidate based on synergistic antitumor efficacy.

BIOSPACE: Off the record, what are the competitors in this space?

There are many recent market introductions and drugs in development in the field of oncology, but there are no direct competitors in this exact space; it’s a first-in-class molecule. ONC201 is especially valuable as a therapy for frail or young patients, who doctors seek to spare from debilitating toxicities and the risk of secondary cancers. ONC201, a therapy less toxic and mutagenic than most existing therapies, could be very attractive for such patients.

BIOSPACE: What’s your timeframe for seeking regulatory approval? Are you looking for approval for multiple indications?

The company is targeting several unmet medical need indications, including refractory lymphomas, leukemias, and glioblastoma multiforme, which may allow an expedited regulatory approval.

BIOSPACE: What stage of trials are you in for ONC201?

Phase I in solid tumors with single agent ONC201 is in completion and Phase II trials are being conducted in the second half of 2015. Phase I and II studies in hematological malignancies will likely run until the end of 2015. Phase III programs directed towards an FDA approval could be conducted in 2017 and 2018, depending on the tumor type and specific indication that is chosen for the first market introduction.

BIOSPACE: Does this new data change that?

These new results highlight two types of lymphoma as potential lead indications for ONC201: one type predominantly inflicts a pediatric population and the other type commonly inflicts frail patients, a characteristic which is further exacerbated by toxicities from standard treatments. In conjunction with the clear unmet medical need, the excellent safety of ONC201 favorably skews the benefit-to-risk ratio in these tumor types. Therefore, the clinical development to regulatory approval may be rapidly achieved in these indications for ONC201.

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