Seeking Alpha's Brian Wilson believes Ampio Pharmaceuticals (AMPE) is on the fast track towards a 2014 FDA approval filing for the diabetic macular edema drug Optina.
He's wrong. Ampio's recent SEC filings paint a far more uncertain picture about Optina.
This drug [Optina] is being developed under a 505(b)(2) pathway, meaning that it can be FDA approved after completion of its current clinical trial. This is possible because of a FDA rule that would let Ampio use clinical trial data collected from other sources in its NDA, meaning that the company can utilize data supporting the use of danazol for other indications. The 450 patient Phase III trial is nearing completion, and should produce data in 2014 that will be usable in a submission for FDA approval...
In its most 10-Q for the September quarter, filed on Nov. 8, Ampio strikes all mention of Optina's development under the FDA's 505(b)(2) pathway.
The disappearance of the 505(b)(2) language is strange because in previous quarterly filings with the SEC going back to the middle of 2012, Ampio was quite adamant about reaching agreement with FDA on an accelerated clinical development path for Optina. Optina is a low dosage of the steroid danazol, which has been in use for more than 30 years.
For instance, this is how Ampio described Optina in its 10-Q for the March 2013 quarter:
The FDA granted Optina 505(b)(2) status in July, 2012, and we commenced enrollment in the clinical trial in February 2013. Drugs designated under this pathway can be approved on a single trial.
You won't find those two sentences in Ampio's most recent 10-Q.
Here's why. In a prospectus filed on Sept. 26 tied to Ampio's most recent financing, the company slipped in new Optina disclosures:
While we believe the data from a single clinical trial would support a NDA filing, we will assess the need for an additional trial in conjunction with the FDA upon the successful outcome of the trial in support of a 505(b)(2) NDA. The FDA has previously indicated that a Phase III trial may be necessary following the current trial. [Emphasis mine.]
The "current trial" has enrolled 250 of a planned 450 patients with diabetic macular edema, comparing Optina to a placebo over 12 weeks of treatment. Results are expected in the first quarter of next year.
As I've previously reported, the FDA has never approved a drug for diabetic macular edema based on a 12-week clinical trial. Roche (ROG - Get Report), Regeneron Pharmaceuticals (REGN - Get Report) and Alimera Sciences (ALIM - Get Report) all ran one-year and two-year studies of their respective diabetic macular edema drugs. There's little reason to believe FDA will lower approval standards for Ampio, which means the ongoing study won't be enough.
Hence Ampio's new disclosure about the need for another Optina clinical trial -- hidden in an SEC filing where Ampio promoters like Seeking Alpha's Wilson don't bother to look.
-- Reported by Adam Feuerstein in Boston.
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