Flim-flam alert: Ampio Pharmaceuticals (AMPE) wants us to believe that patient enrollment in its ongoing study of Optina in diabetic macular edema is being cut short because doctors say the drug is working.
Really? The hip waders I normally wear for protection from biotech bullshit aren't tall enough to wade through Ampio's current press release. I need a full haz-mat suit. Let's unpack the truth about what Ampio is really up to with Optina. Carefully.
1. The Optina study was supposed to complete enrollment of 450 diabetic macular edema patients in the first quarter. That's what Ampio told investors in its 10-K filing on February. At that time, the study had enrolled "over 300" patients and top-line results were expected in the third quarter. Enrollment in the Optina study reached 346 patients, as of Ampio's first-quarter 2014 10-Q filing on May 7.
Today, Ampio says enrollment in the Optina study is being stopped at "over 355 patients" and top-line results will be ready in the fourth quarter. Ampio's Chief Medical Officer Vaughan Clift:
Since DME is a very serious and progressive condition that often leads to blindness, the company believes it is in the patient's best interest to analyze the data as soon as possible to confirm any observed benefits. By reducing the patient sample size, we can expect data by Q4, 2014.
Vaughan, I'm confused. Not only has Ampio missed its own enrollment guidance, but with fewer patients actually participating in the Optina study, the analysis and release of top-line results will take longer?
2. Ampio's explanation for chopping patient enrollment is even stranger. Here's Vaughan, again:
There have been anecdotal reports from several principal investigators that Optina may be providing benefit to patients. The decision to reduce the sample size of the trial took into consideration requests from these site investigators, asking to extend the open label enrollment portion (where all patients receive active drug dose) of the trial beyond the 12 weeks allowed for in the protocol. These clinicians believed that their patients were improving while on the active drug and subsequently regressed quickly once they completed the open label phase. The enrollment in the open label extension phase was much higher than expected.
The idea that study investigators would call up Ampio and say, "Stop the study early because some of our patients are doing better!" has to be one of the most ridiculous excuses I've ever heard. Last October, Ampio claimed independent data monitors performed an interim analysis of the Optina study. These monitors determined that the study should continue to completion. If Optina was, in fact, providing extra-ordinary benefit to DME patients, then the study should be stopped for efficacy, per an analysis by independent data monitors. That hasn't happened.
3. Extending the open-label portion of the Optina study has no bearing at all on patient enrollment. As designed, DME patients are randomized to receive treatment for 12 weeks with one of two doses of Optima or a placebo. After 12 weeks, the Optina patients are taken off drug and followed for four more weeks, after which they can starting taking Optina again as part of the open-label extension. Likewise, patients randomized to placebo can also "cross over" to Optina treatment in the extension study.
If investigators called the company and asked to extend the open-label extension study to 24 weeks (to pick a number) instead of 12 weeks, or even shorten the four-week "washout" period between the regular study and the extension, why would this compel Ampio to enroll fewer patients and take longer to analyze the study results? It wouldn't, of course, but Ampio is betting that investors aren't smart enough to figure out the bamboozle.
4. Last point. The Optina study is supposed to be double blinded, meaning patients and doctors don't know who is receiving Optina or placebo. If that's true, how are investigators determining that patients are benefiting from Optina?
Interested in understanding more about how Ampio misleads investors about Optina? Last October, I wrote a story on Ampio hiding the need to conduct additional Optina studies. And of course, Ampio's claim that 12-week studies of DME drugs are enough for FDA approval doesn't stand up to regulatory precedents.