PLYMOUTH (WWJ) — ProNai Therapeutics Inc. reported promising new results Sunday from an ongoing Phase II study of its lead drug candidate at the 55th Annual Meeting of the American Society for Hematology in New Orleans, La.

ProNai’s drug has a new mechanism of action, targeting cancer cell genes called BCL2, and thereby inhibiting cancer cell proliferation, drivining cancer cell death, and minimizing effects on nearby healthy tissues.

The drug, called PNT2258, is being tested in patients with relapsed or refractory non-Hodgkins lymphoma, a disease in which the body’s white blood cells turn into cancer cells.

“PNT2258 is a promising novel approach to treating lymphoma, and cancer therapy in general,” said the study’s principal investigator, Ayad Al-Katib, M.D., director of the Van Elslander Cancer Center at St. John Providence Hospital and Medical Center. “This is exciting and offers hope to our patients whose tumors have failed to respond to other therapies. It was a pleasure to share the results with so many of my colleagues at ASH.”.

The data presented included the following:
* PNT2258, a first-in-class BCL2 targeted drug, exhibits single agent anti-tumor activity in patients with recurrent or refractory NHL.
* 82 percent of patients had tumor shrinkage when receiving single-agent therapy with PNT2258. To date, overall response rate in patients with follicular lymphoma is 40 percent and in patients with diffuse large B-cell lymphoma (DLBCL) is 50 percent.
* Several patients have elected to receive additional maintenance therapy after their planned 6 treatment cycles.
* PNT2258 is safe at a dose of 120 mg/m2 IV administered for 2-3 hours on days 1-5 of a 21-day schedule. No tumor lysis syndrome or major organ toxicities were observed. No occurrences of elevated liver enzymes, hyperkalemia, hyperphosphatemia, hypocalcemia, renal failure/dysfunction, or infections were noted. Additionally, no Grade 4 toxicities.
* PNT2258 drug exposures levels exceeded by at least four-fold that required for anti-tumor activity in xenograft studies of human tumors, consistent with the Phase I study.
* Preliminary pharmacodynamic markers demonstrated on-target BCL2 activity, including lymphocyte and platelet effects.
* With the promising results in DLBCL and FL patients, additional PNT2258 single-agent and combination studies are planned.

“Patient with symptomatic disease experienced significant improvement in quality of life as their disease responded to treatment,” said study investigator Wael Harb, M.D., of Horizon Oncology Research in Lafayette, Ind. “Infusions were very well tolerated without any significant immediate or cumulative toxicities. The complete responses and durability of responses observed to date suggest that PNT2258 may become a valuable new therapy for NHL patients who have very limited treatment options.”

Added ProNai CEO Mina Sooch: “These results reinforce our enthusiasm for PNT2258 and its potential to treat hematologic tumors. In particular, the data presented at ASH are the first to show systemic, clinical activity by a DNAi drug. We look forward to expanding the current trial and initiating new studies in NHL patients in 2014. In addition, our first peer-reviewed publication has just been accepted this December in Cancer Chemotherapy and Pharmacology titled: A Phase 1 study of the BCL2 targeted deoxyribonucleic acid inhibitor PNT2258 in patients with advanced solid tumors. 2013 was a milestone year for ProNAi as we clinically validated our DNAi drug delivery platform and advanced a new BCL2 targeted drug candidate with potential synergies to current and many new lymphoma therapies.”

ProNai, founded in 2004, has a proprietary and differentiated DNA interference technology. Beyond PNT2258, the company has a broad pipeline of DNAi leads for over 30 cancer and non-cancer targets, including CMYC and KRAS. ProNAi’s business strategy is to establish multiple partnerships across its portfolio of DNAi drug candidates.

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