Petra Pharma Presents Preclinical PIP4K2 Inhibitor Data as a Potential Treatment for Hematologic Malignancies at the 2018 American Society of Hematology Annual Meeting

Data Provide Supporting Evidence for the Company's Identified Clinical Development Candidates

NEW YORK, NY, USA I December 1, 2018 I Petra Pharma Corporation, a drug development company focused on novel therapies that modulate phosphoinositide (PI) signaling pathways, today presented preclinical data in an oral presentation at the American Society of Hematology (ASH) 2018 Annual Meeting. The data presented show that potent and selective inhibitors of PIP4K2 are effective at inhibiting growth of a variety of hematologic cancers – including leukemia- and lymphoma-derived lines.

These data were presented in an oral presentation entitled "Development of Inhibitors of PIP4K2 as a Treatment for Patients with Hematologic Malignancies" during the "Chemical Biology and Experimental Therapeutics: New Targeted Therapies and Drug Development" session.

Recent evidence indicates autophagy – a process that delivers cellular components to lysosomes for degradation – promotes tumorigenesis and tumor cell survival in solid tumors and hematologic malignancies. PIP4K2 is a family of lipid kinases that are critically involved in autophagy by aiding in the fusion of autophagosomes (vesicles containing cellular material slated for degradation) with lysosomes.

Lewis Cantley, Ph.D., Scientific Co-Founder of Petra Pharma, added, "Paired with current understanding of PIP4K2, our data suggest the PIP4K2 family of enzymes could serve as the foundation for potential treatments of hematologic malignancies."

"We've explored the structure activity relationship of PIP4K2 inhibitors using fragment- and structure-based drug discovery and have found highly potent and selective inhibitors of PIP4K2 with exceptional drug-like properties," said David McElligott, Ph.D., Chief Scientific Officer of Petra Pharma and the study's lead investigator. "We are encouraged by the results from this most recent study and look forward to continuing our investigation of PIP4K2 inhibitors."

The research demonstrated PIP4K2 inhibitors induced rapid regression of an acute myeloid leukemia tumor (MOLM-16) in a mouse xenograft model. These studies showed dose-dependent tumor growth control with sustained regression of tumor volume with once-daily oral dosing of a prototype molecule. The stable mouse body weight over the course of the study suggests the molecule is well-tolerated with this dosing protocol. Meanwhile, a preliminary toxicity study in rats indicates a clean safety profile with no identifiable issues.

"Petra is committed to advancing the promise of phosphoinositide signaling pathways to generate important and novel therapies for unaddressed medical needs," said Brian O'Callaghan, President and Chief Executive Officer of Petra Pharma. "The results of this study stand on the shoulders of more than 30 years of research by our scientific co-founders and support our belief that the PIP4K2 enzyme family could be the key to identifying impactful new medicines for the treatment of hematologic cancers. We are excited to premiere these data at this year's ASH Annual Meeting."

About Petra Pharma Corporation 
Petra is a pharmaceutical company dedicated to discovering and developing novel therapies that modulate phosphoinositide (PI) signaling pathways for the treatment of cancer and other serious diseases. PI signaling pathways play a central role in a variety of important cellular processes, including cell division, growth, trafficking and signaling, and impacting human health. Petra was founded in 2016 to advance the cutting-edge research of Dr. Lewis Cantley from Weill Cornell Medical Center and Dr. Nathanael Gray from Harvard Medical School and Dana-Farber Cancer Institute. Petra Pharma's office and lab headquarters is located at the Alexandria Center for Life Science, New York City's first and only premier life science campus. For more information, visit www.petrapharmacorp.com.

SOURCE: Petra Pharma

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