Ampio hasn't adequately explained the rationale for FDA approving Optina for the treatment of DME based on a single, 12-week study when every other company developing drugs for the same indication conduct two-year studies.
The company claims Optina has so-called "505(b)2 status" because its active ingredient danazol is already an FDA-approved steroid. "A portion of 505(b)2 drugs are approved based on a single clinical trial," Ampio states.
True, but once again, Ampio only tells investors part of the story. A 505(b)2 approval application allows a company to use some efficacy and safety data from clinical trials that it did not conduct. But a 505(b)2 filing doesn't relieve a company of proving the efficacy and safety of a drug for a new indication. Danazol has been around for a long time but it's not approved for DME and FDA won't consider the drug for approval without sufficient clinical data from new studies -- 505(b)2 or not.
Moreover, no oral drug has ever been approved for the treatment of DME (Lucentis, Eylea and Iluvien are all injections or implants.) That sets an even higher regulatory hurdle for Ampio relative to its competitors. A single 12-week study is not going to cut it.August is the estimated completion date of the Optina study, according to Ampio's ClinicalTrials.gov listing. The study began enrolling patients in late February and all patients must undergo at least 12 weeks of treatment. If you do the math, the timetable requires Ampio to enroll 450 DME patients in three months. If the last patient is enrolled by the end of May, Ampio could have 12 weeks of treatment for all patients by the end of August. Impossible. Consider this: Regeneron, working with partner Bayer, enrolled its U.S-based DME phase III trial of 402 patients in nine months. Last June, I explained how Ampio had spun data from a previously conducted clinical trial to make Optina look more effective than it truly was in DME patients. Ampio called this previous study a success but the facts show the study actually failed. True to form, Ampio is spinning again, this time with the design of a pivotal clinical trial that will be laughed out of the FDA if by some miracle it ever gets there in the first place. -- Reported by Adam Feuerstein in Boston. Follow @AdamFeuerstein