ProNAi Therapeutics, Inc. Announces First DNAi(R) Mechanism of Action Studies for PNT2258 Oncology Drug Candidate

Pioneer in DNAi(R)-Based Drug Development Examines Role of BCL2 Gene in Triggering Apoptosis of Malignant Cells

KALAMAZOO, Mich.--(BUSINESS WIRE)--ProNAi Therapeutics, Inc., a biopharmaceutical company pioneering a new class of nucleic-acid drugs based on DNA interference (DNAi®), today announced its first mechanism of action studies for its PNT2258 oncology drug candidate, centering on the BCL2 gene's role in triggering cell death in cancer cells. BCL2 plays a central role in dysregulation of apoptosis in malignant cells, which presents an opportunity for molecular targeted drug discovery. PNT2258 targets a DNA sequence upstream of BCL2 promoters within a region of genomic instability, and ProNAi's preclinical research is currently examining methods for safe and effective delivery of oligonucleotides to regulate BCL2 in difficult-to-treat cancers. Chemotherapy resistance of Non-Hodgkin's Lymphoma and many other tumors are linked to overexpression of BCL2.

To date in preclinical studies in mice, PNT2258 has demonstrated antitumor activity in Non-Hodgkin's Lymphoma, prostate cancer and melanoma xenografts. IND-enabling CMC studies are currently underway, and ProNAi anticipates filing for IND status for PNT2258 in late 2007.

Dr. Richard D. Gill, President and CEO of ProNAi, said, "We are very excited by the positive results of the first mechanism of action studies for our DNAi®-based oncology therapeutic candidate. This discovery highlights the continued progress of PNT2258 toward IND status - while keeping pace with other nucleic acid-based approaches in the quest for safe, effective delivery of therapeutic oligonucleotides."

Robert Forgey, Co-Founder and Chief Operating Officer of ProNAi, said, "Using a DNAi®-based strategy to target BCL2 is emerging as a very promising avenue to potentially address certain difficult-to-treat cancers. To date, all alternative approaches to regulating BCL2 in man lack proofs-of-concept, so we are actively pursuing this intriguing possibility, as we continue to advance our preclinical studies."

DNAi® is a novel approach to targeting genomic DNA using sequence-specific therapeutic agents, employing single strands of DNA to target and treat genes responsible for complex genetic diseases, such as cancer.

By acting at the DNA level, where only one or two copies of the gene exist per cell, treatment can be targeted more efficiently by DNAi® drugs. With fewer targets, the activity of a DNAi® drug is expected to last longer at lower doses, and reduce some of the toxicity issues prevalent with other marketed therapies. Additionally, DNA-related therapies are potentially more cost effective to produce.

About ProNAi Therapeutics, Inc.

ProNAi Therapeutics, Inc. is a biopharmaceutical drug development company pioneering a new class of nucleic-acid drugs based on DNA interference (DNAi®), which employs single strands of DNA to target and treat non-transcribed regions of genomes responsible for complex genetic diseases. ProNAi is currently developing multiple DNAi®-based drug candidates with the potential to treat multiple cancers, including non-Hodgkin's lymphoma, prostate, melanoma, breast, and colon cancers. The company's lead drug candidate, PNT2258, which has demonstrated in vivo efficacy in a variety of human tumor xenograft models, is currently in preclinical development. ProNAi is also exploring the potential of DNAi®-based therapies for indications such as diabetes, Alzheimer's and inflammatory disease.

ProNAi is based in Kalamazoo, Michigan. For more information, please visit:

Contact: Aviator PR Mario Fante, 617-763-9887

Source: ProNAi Therapeutics, Inc.

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