Ra Pharmaceuticals Plummets on Phase II Initial Clinical Trial Data, What You Need to Know

Ra Pharmaceuticals Inc (NASDAQ: RARX) shares ended down just over 20% today. The stock’s plummet was primarily attributed to its announcement of initial data from its Phase II clinical program for the evaluation of RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria. However, just because the stock reported unfavorable initial data, it does not necessarily mean that the company can’t catch a bounce.

Check out RARX on the daily chart below.

Ra Pharmaceuticals

Source: TradingView

Let’s take take a look at the data release, as well as Ra Pharmaceuticals’ drug pipeline.

Ra Pharmaceuticals Initial Data from Phase II Clinical Trial

The Phase II clinical trial program was aimed at the evaluation of the safety, tolerability, PK/PD and preliminary efficacy of RA101495 in patients with PNH. According to the company, there were only three eculizumab-naive patients enrolled, two of which completed seven weeks of follow-up and the data from these patients were included in the company’s initial data set.

Some key highlights from the Phase II Clinical Trial of RA101495 in PNH initial data included:

  • No patients exhibited any safety or tolerability concerns, and no injection site reactions.
  • There were rapid declines in lactate dehydrogenase (LDH), with a mean LDH at week seven of 1.6X upper limit of normal.
  • There was near-complete inhibition of hemolytic activity
  • Achieved 100% compliance with once daily, subcutaneous self-administration.

However, you should take note that only three patients were enrolled in this trial.

One of the main reasons the stock plummeted was attributed to one patient reporting the recurrence of PNH symptoms and had an elevated LDH level. However, the episode was short-lived and was resolved quickly with LDH levels reaching 1.3X ULN by the seventh week with the resolution of the hemolytic symptoms.

According to Ra Pharmaceuticals President and Chief Executive Officer (CEO) Doug Treco, PhD, “We are pleased by the initial results from this study, our first in PNH patients, whose primary purpose is to gain important safety, preliminary efficacy, PK and PD data in the target patient population…These data will help inform dose selection for pivotal trials and will help refine our clinical development strategy. We look forward to giving you an update on our progress around year-end.”

That in mind, investors and traders would have to wait until late 2017 to receive more data surrounding this treatment.

Ra Pharmaceuticals Pipeline

Now, Ra Pharmaceuticals still has some drugs in its pipeline, and there are multiple indications for RA101495, even if the clinical trial data for PNH misses its primary and secondary endpoints, the treatment could still potentially be used for other conditions. Here’s a look at Ra Pharmaceuticals’ pipeline.

Ra Pharmaceuticals

Source: Ra Pharmaceuticals

The Bottom Line

The company does not seem to be a one-trick pony, and even if one drug fails, there are still some other treatments that could potentially receive U.S. Food and Drug Administration (FDA) approval. However, keep in mind that would be years down the road. The stock was down over 20% on the day, and this could potentially serve as a mean reversion opportunity for those who have a high risk tolerance.


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