ProNAi Reports Significant Activity and Ongoing Durable Responses in Relapsed or Refractory Non-Hodgkin’s Lymphoma with PNT2258
Updated results from pilot Phase II trial of the BCL2 targeting DNAi® therapeutic reported at ASH 2014 –
VANCOUVER, BC and PLYMOUTH, MI, Dec. 5, 2014 /CNW/ – ProNAi Therapeutics Inc., a private hematology/oncology company dedicated to developing and commercializing a new class of therapeutics based on its proprietary DNAi® platform, today reported that a number of patients with relapsed or refractory Non-Hodgkin’s Lymphoma treated with its first DNAi-based therapeutic, PNT2258, achieved meaningful therapeutic outcomes and continue to exhibit durable clinical responses.
The interim results from the ongoing pilot Phase II trial of PNT2258, a DNAi-based therapeutic targeting BCL2, are being reported in a poster presentation at the 56th Annual Meeting of the American Society of Hematology (ASH), being held in San Francisco. The investigators for the study concluded that:
“These results demonstrate that PNT2258 is an obviously active, well-tolerated therapeutic that warrants advanced clinical development,” said Dr. Nick Glover, President and CEO of ProNAi Therapeutics. “As such, in the coming months we will be initiating robustly designed studies of PNT2258 as a single agent in patients with refractory or relapsed DLBCL and in Richter’s transformed DLBCL, areas of high unmet medical need in which PNT2258 appears to be particularly effective. Given PNT2258’s favorable safety profile and the prospect for a BCL2-targeted agent to augment complementary mechanistic approaches, combination clinical studies with other targeted agents are also being planned.”
The primary objective of the pilot Phase II open label efficacy study (PNT2258-02; NCT01733238) was to determine anti-tumor activity and collect safety data on recurrent or treatment-refractory NHL patients receiving single-agent PNT2258.
Of the 13 patients treated, four were diagnosed with DLBCL; five patients were diagnosed with FL, two with mantle cell lymphoma (MCL) and two with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). The median age of the patients was 63, and they had received an average of two prior systemic treatments. Nearly half (46%) of the subjects were refractory to prior treatment.
Patients received 120 mg/m2 of PNT2258 intravenously on days 1-5 of a 21-day cycle for 6-8 cycles, and then continued with 100 mg/m2 on days 1-2 of a 28-day cycle, until disease progression.
A poster of these data will be presented at the 56th Annual Meeting of the American Society of Hematology, being held in San Francisco. The poster (Abstract #1716) is entitled: “The BCL2 Targeted Deoxyribonucleic Acid Inhibitor (DNAi) PNT2258 Is Active in Patients with Relapsed or Refractory Non-Hodgkin’s Lymphoma”. The poster will be presented from 5:30 – 7:30 pm PT on Saturday, December 6th, 2015 at the Moscone Center, West Building, Level 1.
About PNT2258 and DNAi
PNT2258 is the first clinical stage DNAi-based therapeutic, and is designed to exquisitely target the regulatory region of the BCL2 gene, modulating transcription and effecting BCL2 function. PNT2258 is a proprietary liposomal formulation of PNT100, a 24-base phosphodiester DNA sequence. The liposomes are specialized anionic and pH “tunable” nanoparticles composed of four lipids. After intravenous injection, PNT2258 achieves systemic distribution, where PNT100 enters into nuclei to target the non-coding, non-transcribed regulatory region of the BCL2 gene upstream of the promoter start site resulting in an anti-proliferative and apoptotic effect.
ProNAi’s proprietary DNAi® (DNA interference) therapeutic platform delivers rationally-designed DNA oligonucleotides into cells in order to target sequences residing within 5′-non-coding regions of the genome. These regulatory regions are susceptible to DNAi hybridization during transcription, cell cycling and gene up-regulation. The specific hybridization of the DNAi oligonucleotides with the targeted complementary strand of genomic DNA modulates transcription, affecting the function of the target gene.
About ProNAi Therapeutics
ProNAi Therapeutics is a private company dedicated to developing and commercializing a new class of therapies based on its proprietary DNAi®platform for patients with cancer and hematological diseases. ProNAi’s lead DNAi drug PNT2258 is specifically designed to treat cancers that overexpress BCL2. To date, 35 patients have been treated with PNT2258 in a Phase I and a pilot Phase II trial, with compelling efficacy and safety results reported. In May 2014, ProNAi raised approximately $60M from leading healthcare investors. For more information, please visit www.pronai.com.
SOURCE ProNAi Therapeutics Inc.
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