ProNAi Therapeutics Inc (NASDAQ:DNAI) just reported that it is set to deliver interim results from an ongoing phase II in its lead oncology candidate at the 52nd Annual Meeting of the American Society of Clinical Oncology (ASCO). The event is scheduled to take place on the 6th of next month, and if market interest in the release date announcement is anything to go by, it could have a considerable impact on the company’s near term valuation. With this in mind, here’s a look at what’s set to be presented, and what to keep an eye on as indicative of bullish or bearish response.
So, the drug. A wave of oncology drugs has hit the development space over the last half decade, and many are simply iterations of one another in a particular family of molecule. ProNAi’s candidate, however, is a little different. It’s called PNT2258, and is what the company refers to as a DNAi. DNAi is another word for a single stranded DAN oligonucleotide (think of this as a type of building block of DNA) surrounded by a lipid outer coating. A team at Wayne State University figured out that these oligonucleotides can interact with certain oncogenes (which are the mutated genes that cause cancer) and affect their behavior. ProNAi picked up the tech from Wayne State, and developed PNT228, which specifically targets an oncogene called BCL2. BCL2 is an oncogene that impacts apoptosis. Apoptosis is the process through which cells induce their own death. In cancer cells, apoptosis isn’t as efficient as it should be, and cells that should die, don’t. Through the introduction of PNT2258, ProNAi is hoping it can reverse the the apoptosis inhibition that BCL2 induces, and in turn kill cancer cells. Specifically, and as under investigation in the trial in question, relapsed or refractory diffuse large B-cell lymphoma cells.
So what are we looking for in the upcoming trial data?
The trial in question is called Wolverine DLBCL, and it is set up to investigate both efficacy and safety concurrently. Each patient will receive the drug intravenously, 120mg/m2, on the first five days of a 21-day cycle. The administration period lasts for six months total. The primary endpoint is overall response rate, and while the measurement point for this overall response is not explicitly clear, in all likelihood the ORR in this instance refers to tumor size. So, we are looking for a marked reduction in tumor size (in B cell lymphoma, these tumors are generally in the lymph nodes) as an indication that the drug is effective in treating this indication. A host of secondary endpoints are in play, including time to response, which just means how long did it take for the tumor in question to start reducing in size, and overall survival. OS ranges from 20-50% in this sort of cancer across a five-year period. The measurement will initially address a 24-month period, but we should get some indication of it comparable efficacy (versus current SOC) even at this early stage.
What’s the upside on approval for the drug? If ProNAi can carry PNT2258 through to commercialization, it will gain access to a multi billion-dollar market. A recent analysis suggested that treatment market is set to rise slowly in value from $4.38 billion in 2014 to reach $5.45 billion by 2024. The company can’t expect to get full penetration, of course, but anywhere in the low to mid teens would still offer up a considerable premium on its current market capitalization, which came in at al little over $150 million at last count.
With this in mind, if the data comes out as indicative of the drug’s efficacy, we can expect some immediate upside in ProNAi. It’s the company’s lead candidate, and the technology that underpins its MOA is the underlying technology behind much of its wider clinical and preclinical pipeline. As such, the downside could be just as severe as the upside could be rewarding, if the data suggests the technology doesn’t work in a clinical setting. Risky, but one to keep an eye on if phase II interim comes out positive. The poster will be released at the company’s website premarket on June 6, and the presentation will take place the same day.