Ampio Pharmaceuticals (NASDAQ:AMPE)
On April 20, 2015 Shares of Ampio Pharmaceuticals fell about 66% after the company reported that it had failed to meet the primary endpoint of a phase 3 clinical trial, known as the STRIDE trial. This trial recruited 320 patients with Osteoarthritis and patients were randomized into two different groups. One group took 4ml of inra-articular injections of Ampion while the other group took injections of a placebo instead. Osteoarthritis is a joint disorder that occurs because of wear and tear and aging. A lot of analysis was done and Ampio determined that the trial failure was due in part because of a huge increase of efficacy in the placebo group which is typically unheard of.
For example the placebo drug saw an improvement of efficacy from 12% to 60% which pretty much mixed the overall data up in a bad way. This is because in other similar osteoarthritis trials saline — placeo — only saw a range of efficacy between 30% to 35%.Although this looks bad there might be a saving grace for Ampio because it might be able to use phase 3 data from the other two trials it is running known as “SPRING” and “STRUT” to file for a Biologics License Application — BLA.
Ampio now states that is has enough cash to last at least through 2016, which indicates that the company is still in good shape cash position-wise. On the other hand the FDA may require additional Osteoarthritis trials for Ampion, but if that is the case the company will have the cash to meet those needs anyways. If trials aren’t required then the FDA may just accept the two other phase 3 trials as being sufficient enough for a BLA approval. There are no current cures for osteoarthritis and these patients experience pain daily with no good treatment options. Ampio believes that its Injection of Ampion will help alleviate the symptoms of these patients and improve their daily lives.
Alnylam Pharmaceuticals (NASDAQ:ALNY)
On April 21, 2015 shares of Alnylam Pharmaceuticals were up about 2% after the company reported positive results in their phase 2 open-label extension study, for a disease known as TTR-Amyloidosis. The subcategory for this disease is known as Familial Amyloidotic Polyneuropathy — FAP, which is a devastating disease. FAP is a serious disease in which patients have both the mutant and wild type TTR genes that deposit in the body. These excessive proteins are deposited in the peripheral nerves and heart which may eventually lead to death.
Alnylam is using the drug Patisiran which has continued to show safety every time and this time was no different. The final results showed that patients taking Patisiran showed a mean decrease of 2.5 in modified Neuropathy Impairment Score (mNIS+7) at 12 months. This compares to historical control who saw a mean increase of 13 to 18 points in mNIS+7 at the same time period. In addition to that effect these patients saw a gene knockdown of 80% throughout the trial of the TTR gene. This may prove useful because this gene knockdown of the TTR gene can possibly halt neuropathy progression in these patients.
This phase 2 open-label extension study was run just to test for safety of the Patisiran drug in this patient population. Despite that this drug had showed great efficacy in this trial and Alnylam can move on with the potential to have the first RNAi approved drug. The validation of RNAi therapeutics will come from Alnylam’s other trial in phase 3 known as “APOLLO” which is an FAP trial enrolling global patients from all over the world. This phase 3 trial validation along with additional 18-month open-label extension study results coming sometime this year in 2015 will finally validate RNAi therapeutics as a success. The only thing left will be to put Patisiran up for FDA approval and possibly make RNAi history.
Aerie Pharmaceuticals (NASDAQ:AERI)
On April 23, 2015 shares of Aerie Pharmaceuticals tanked 70% after the company reported that it had failed to meet the primary endpoint of a phase 3 clinical trial, known as Rocket 1. The company had developed a drug known as Rhopressa to lower intraocular pressure in patients with glaucoma or hypertension. Rhopressa was given to patients as a once-daily, triple action eye drop to help these patients with their eye pain.The Rocket Trial recruited up to 370 patients in total that were randomized into two different groups.
One group took a twice daily timilol — beta blocker drug typically given to glaucoma patients — and the other group took once daily eye drop of Rhopressa. The primary endpoint of of the trial was not met as Rhopressa did no better than the placebo — timilol. The primary endpoint for the study was looking for Rhopressa to show non-inferiority of intraocular pressure as compared to timilol. Think things can’t get worse with this trial? Well they did because in addition for Rhopressa missing on the primary endpoint, Rhopressa arm also saw a decline of efficacy of 20% on 36 out of 182 patients.
The question now is can the company redeem itself after this trial failure? Well the good news here compared to some other biotechnology stocks is that there is another phase 3 trial to fall back on. This other phase 3 trial is known as Rocket 2 conveniently and it is also testing Rhopressa for patients with intraocular pressure of glaucoma. Investors may have to wait out a few months for the next phase 3 trial readout of results, because these results are expected sometime in Q3 2015. This trial will be crucial for the future of the company and the future of Rhopressa in treating patients with glaucoma and ocular hypertension. If the company is not successful it may have to go back and evaluate the next course of action. Whether or not the company runs a new phase 3 trial will depend upon the data seen in Rocket 2 so investors should keep an eye on this until then.