On Monday September 14, 2015 shares of Raptor Pharmaceuticals ended the day down 38% after the company announced that it had failed a phase 2b liver study in children with NASH — non-alcoholic steatohepatitis. The phase 2b trial enrolled up to 169 children with NASH between the ages of eight and seventeen. The company randomized these patients into many different types of dosing groups.
Patients were randomized to either take 600 mg, 750 mg, 900 mg of Raptor’s drug RP-103 or a placebo compound instead. The compound RP-103 was created to target the underlying factors of metabolic diseases such as NASH. The phase 2b trial treating these patients with NASH is known as the “Cynch” Trial. Another name for NASH is NAFLD — which stands for non-alcoholic Fatty liver disease. The primary endpoint of the trial was to determine if RP-103 could produce a two point decrease in the NAFLD score — score used to monitor NASH — and no worsening of the fibrosis of the patients livers’ during the trial.
As noted above the stock crashed after the news because the company did not see a two point decrease in the NAFLD score and the drug wasn’t able to inhibit worsening of the fibrosis of these patients’ livers. With these results on hand Raptor Pharmaceuticals has chosen to completely discontinue this program entirely, which means it will definitely not be seeing a comeback any time in the future. The company has stated that a further detailed analysis of these results will be displayed at the upcoming American Association for the Study of Liver Diseases — AASLD — meeting. This meeting is being held November 13th through the 17th. The only positive thing about RP-103 is that it is being studied for other indications, which may or may not yield successful future clinical results.
Aerie Pharmaceuticals (NASDAQ:AERI)
On Wednesday September 16, 2015 shares of Aeri Pharmaceuticals were up 75% in after-hours trade after the company announced positive phase 3 results for its Rhopressa trial. Rhopressa is a once daily triple action eye drop drug intended to relieve intraocular pressure in patients with either glaucoma or ocular hypertension. One thing to note is that there are a total of three different Rhopressa trials that are running in tangent with one another.
The results displayed on September 16th come from the phase 3 trial known as the “Rocket 2” Study. The Rhopressa drug was dosed as both a once daily and twice daily type of drug. The good news was that the drug was able to meet on the primary endpoint in both the once daily and twice daily dosing regimen. The primary endpoint was for Rhopressa to demonstrate non-inferiority compared to timilol — a placebo drug for these patients — twice daily regimen.
It would have been enough for the drug to achieve its primary endpoint but it also was able to show intraocular pressure lowering through the entire 90-day efficacy period. There was one adverse event seen in the trial of eye redness but that was only seen in the once daily dosing arm of the study. In addition this adverse event is not a serious one therefore it doesn’t take away from the efficacy of the drug to help treat these patients with glaucoma.
It is great to see that investors received a much needed redemption after the previous results which ended in a huge disaster for the share price of Aeri and its shareholders. That is because back in April 23, 2015 shares of Aeri Pharmaceuticals tumbled as much as 64% in one day after announcing that Rhopressa had not met the primary endpoint compared to the placebo timilol. Investors now should keep an eye on the 12-month safety data for Rhopressa which is set to be released in late 2015 or early 2016.
Now that Aeri Pharmaceuticals has these positive results from the Rocket 2 study, and the previous positive Rocket 1 Study results the company expects to file an NDA for Rhopressa in the glaucoma indication by mid 2016.
Can-Fite Biopharma (NASDAQ:CANF)
On Thursday September 17, 2015 shares of Can-Fite Biopharma soared 92% after the company reported that it was given the FDA Fast Track Status for its liver cancer drug. The company’s liver cancer drug is known as CF-102 and is currently in a phase 2 clinical trial targeting Child Pugh Class B Cirrhosis. This CF-102 drug is being tested against a placebo to determine if it can achieve greater efficacy.
The patients in this trial are very ill, because these patients had already failed with a previous treatment that uses standard of care known as Nexavar. The current phase 2 trial uses 25 mg of CF-102 compared to a placebo compound. The reason why Can-Fite chose the 25 mg dosing of this drug is because in a previous phase 1/2 trial it was established to be the most efficacious dose.
Fast-Track Status is given to biotechnology companies to be able to bring drugs that treat unmet medial needs to market at a quicker pace. When a biotechnology company receives this indication there are many benefits that help move the drug along quicker through the approval process. For instance a biotechnology company gets additional communication from the FDA which is a huge advantage. Not only that but it gives the company the ability to file the New Drug Application — NDA — on a rolling basis.
What that means that the company can submit the NDA early and then update the results as they come in. This avoids having to wait until all clinical results are in and then having to wait a year before the FDA can review the drug. As you can see this provides quicker access for any biotechnology company that receives this FDA Fast Track Status.
[Image Courtesy of Hy-Lok]