ProNAi Announces Progress on Its Novel Cancer-Fighting Drug

Mina Sooch, Detroit Xconomist and president and CEO of ProNAi Therapeutics, says 2013 was a landmark year for the biotech startup based in Plymouth, MI. ProNAi, which was spun out of technology discovered at Wayne State University and the Karmanos Cancer Institute in Detroit, aims to “quiet” disease using novel drugs that target cancer and other complex genetic diseases at the level of underlying DNA.

Last month, ProNAi presented its findings from an ongoing Phase 2 clinical trial of its drug, called PNT 2258, at the annual American Society of Hematology (ASH) meeting in New Orleans. The drug is made up of DNA snippets encapsulated in lipid nanoparticles to help protect the drug on its journey to the desired biological target inside cells. ProNAi’s compound targets the cancer gene BCL2 and turns it off, so to speak.

At ASH, ProNAi presented data from a study of patients whose disease had worsened after prior therapy. The study showed 3 out of 11 patients (27 percent) had experienced significant tumor shrinkage, which was the kind of encouraging early sign researchers want to see before advancing a new drug into larger, controlled trials. Besides the three responders, other patients in the study responded well enough to treatment that they were able to return to work or go on vacations, Sooch says. Only two patients in the study failed to respond to the drug, she added.

Sooch says not only has PNT 2258 shown to be effective and shrinking tumors and keeping them that way, but it was also found to be safe in the small study. Unlike most other cancer drugs, it’s non-toxic, doesn’t cause hair loss or weight loss, and doesn’t cause anemia, Sooch says.

Sooch and the ProNAi team—there are less than 10 employees —say they are excited about the data and plan to expand the Phase 2 study to enroll more patients in 2014. The company is also mulling over whether to pursue another financing round, a strategic partnership with a big pharmaceutical company, or an IPO.

Sooch says there’s only one other company developing drugs to target BCL2, but that company, North Chicago, IL-based AbbVie (NYSE: ABBV), is concentrating on different blood cancers such as multiple myeloma and leukemia. In 2013, ProNAi raised more than $11 million from investors to continue its clinical studies.

ProNAi also plans to continue to cultivate a pipeline of future drugs for development. ProNAi’s proprietary platform uses “DNA interference” technology, which is supposed to work by taking snippets of DNA that zero in on specific disease-causing stretches of DNA, and stop the production of diseased proteins. The fatty ball of lipids acts to protect the DNAi molecule from enzymes in the bloodstream that could degrade the drug before it reaches the target. The technology, Sooch says, can be applied to any gene of interest —usually cancer-causing genes that don’t respond to existing therapies.

“We usually pick the bad ones,” Sooch adds. “Right now, we have 30 candidates in the pipeline, and we’re moving two to three forward. We’re excited to build more products using our platform, and we expect to have another clinical trial started by the end of 2015.”

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