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Rxi Pharmaceuticals (NASDAQ:RXII)

On Tuesday April 19, 2016 Rxi Pharmaceuticals announced that it would present pre-clinical data at a medical conference, known as 2016 ARVO Conference. ARVO stands for Association for Research in Vision and Ophthalmology. The pre-clinical data to be presented will be the company’s drug in corneal scarring. In order to prevent injury to the eye, doctors prescribe steroid eye drops or antibiotics. This is done in an attempt to curb corneal scarring from occurring.

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The Press Release sheds light on the Poster that will be presented:

On Sunday, May 1, 2016, new data from ongoing research utilizing the Company’s proprietary RNAi platform in the area of therapeutic topical delivery to the eye, will be presented in the 3:15 – 5:00 p.m. PDT poster session: Corneal Wound Repair and Healing. These data may be viewed on Poster 1280 – D0228 entitled, “sd-rxRNA®: Self-Delivering RNAi Compounds Show Potential for Corneal Indications Following Topical Application.”

There are some current treatments for corneal scarring but they are very invasive, and sometimes don’t even help the problem in question. For example, some treatment options are laser surgery to reduce scarring, and corneal transplant surgery. Rxi is creating a topical gel that will be used to heal corneal scars, and thus save patients from further damage. In addition applying a topical gel is less intrusive than the other methods mentioned above. The reason why Rxi can turn its RNAi technology, sd-rxRNA, into liquid form is because it is soluble. It can be converted into liquid or topical form, and that is a great advantage for creating a corneal scar drug.

Sarepta Therapeutics (NASDAQ:SRPT)

On Thursday April 21, 2016 shares of Sarepta Therapeutics tumbled by 44% after the FDA briefing documents posted a negative perception about the efficacy of Eteplirsen. The drug, Eteplirsen, had been in clinical trials to treat patients with a rare muscle-wasting disease known as Duchenne Muscular Dystrophy  or DMD. The FDA was not to keen on the data! Matter of fact, they were quick to state that they can’t approve a drug just because it’s an unmet medial need. It has to show some statistical evidence of clinical efficacy, and that statement was made clear by the FDA.

Although FDA is prepared to be flexible with respect to a devastating illness with no treatment options, we cannot approve drugs for which substantial evidence of effectiveness has not been established”

The question of the efficacy was just part of the problem, because an even bigger problems still loomed. One problem was that the trial was done only using 12 patients. The problem with this, is that a trial can’t obtain the proper statistical analysis with such a small patient population. I think that Sarepta would have been better off recruiting at least 35 to 40 patients, only then would the FDA feel more comfortable with the trial design. The final problem is that the trial that was done, had no control — placebo — in place. In order to see if the Eteplirsen was working in DMD patients, the company should have had a placebo arm to be able to compare the data. Instead, Sarepta chose the shortcut way and compared their drug with historical control.

UPDATE: FDA advisory panel voted 4/25/16:

Panel tone was overall negative, and the final vote outcome for approval was 3 “yes” 7 “no” and 3 “abstain” This means that the panel was not satisfied with the efficacy or trial design of the Eteplirsen trial. The main concern cited during the panel discussion, which was cited by a majority of the panel members, was that there was no placebo, and everything was compared to historical control. Historical control is bad because it doesn’t identify data that is current. Which is why the panel members were really frustrated with this detail.

Is there any hope left for Sarepta’s Etelplirsen approval in DMD? There is a small chance left, but with this overall negative adcom panel vote I would say there’s probably a 5% chance of approval. Only if the FDA disregards the advisory panel’s recommendation will Eteplirsen be approved. The FDA has a few times in the past done this, but it is very rare and I don’t expect it to happen in this instance. The final FDA review on whether to approve Eteplirsen or not is set to take place on May 26.

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Disclosure: Long Rxi Pharmaceuticals (RXII)

[Image Courtesy of Wikipedia]

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