PLYMOUTH — ProNAi Therapeutics Inc., a cancer drug development company, said last week that it had concluded its initial human clinical trial of PNT2258, a new type of nucleic acid drug.
It is presenting the results of the Phase I study of PNT2258, an anti-Bcl-2 cancer drug, at the annual EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics, a conference hosted by the European Organization for Research and Treatment of Cancer, the National Cancer Institute and the American Association for Cancer Research.
The company also expanded its management team with the recent appointments of board member and venture capital investor Mina Sooch as president and CEO, and Richard Messmann, M.D., as Chief Medical Officer.
“The encouraging clinical results from our 22 patient study, conducted at START in Texas, showed we can achieve exposure levels in humans that were many times the level needed to achieve anti-tumor effect in animals without significant liver or blood platelet toxicity,” Messmann said. “We also demonstrated statistically significant, dose-dependent, and specific knockdown of the Bcl-2 gene with suppression of protein levels up to 60 percent. Patients received the drug for extended periods of time without having material side effects seen with other anti-Bcl-2 drugs or nucleic acid cancer drugs in development. These results indicate that we can administer the drug either as a single agent or potentially in combination with other anti-cancer drugs without adding patient side effects.”
Added Sooch: “PNT2258 represents the first of many targeted drugs in ProNAi’s portfolio that utilize its emerging DNA interference platform called DNAi. We look forward to rapidly expanding PNT2258’s clinical development to confirm efficacy in patients with Bcl-2-driven tumors, such as diffuse large B-cell lymphoma, follicular lymphoma and chronic lymphocytic leukemia. We also anticipate eventual development into other solid tumors such as cancers of the lung, head and neck, and sarcomas. ProNAi continues to pursue both venture capital funding and partnerships with pharmaceutical companies to advance these promising potential therapies for cancer. ProNAi is proud to be a pioneer in nucleic acid therapeutics by overcoming the drug delivery challenges of RNA and DNA based therapies as it has demonstrated safe and effective systemic delivery beyond the liver in humans.”
Prior to joining ProNAi, Sooch, who has more than 20 years of experience in pharmaceuticals, healthcare, and mergers and acquisitions after receiving her MBA from Harvard University, helped co-found Apjohn Ventures Fund and several biotech start-ups. Messmann, who has over 20 years extensive clinical experience as a medical oncologist and drug-development physician, recently served as Vice President of Medical Affairs for Endocyte, where he led the development of vintafolide for ovarian cancer through successful late stage clinical trials. The executive management team is rounded out by Wendi Rodrigueza, Chief Scientific Officer (formerly Novartis, Curagen, and Esperion), and ProNAi scientific advisors, led by Bruce Zetter of Harvard Medical School.
PNT2258 is a 24-mer, single stranded, chemically unmodified (i.e., native) DNA oligonucleotide sequence called PNT100 encapsulated in a specialized anionic and pH “tunable” liposome. PNT2258, which is delivered via a two-hour infusion, has a long half-life of several days allowing it to infiltrate cancer cells, release into the nucleus, and initiate apoptosis (cell death) by silencing the Bcl-2 target gene.
ProNAi Therapeutics is a venture backed biotechnology company based in Plymouth and Kalamazoo. The novel DNA interference drug platform is proprietary to ProNAi. Unlike many RNA products (antisense, RNAi, microRNA, etc) that work in the cytoplasm of a cell, DNAi drugs are designed to target sequences that reside in the non-coding regions of the genome and block transcription in the nucleus, thereby silencing the gene of interest at the source. DNAi therapeutics have the potential to treat cancer and other genetic diseases with an innovative and targeted approach.
More at www.pronai.com.