Ampio's Eye Drug Trial Design Won't Pass FDA Muster

GREENWOOD VILLAGE, Colo. ( TheStreet) -- Ampio Pharmaceuticals' ( AMPE) "phase III" clinical trial of the low-dose steroid Optina in diabetic macular edema finally showed on the registry maintained by the U.S. Food and Drug Administration, one month after the company announced treatment of the first patient.

Why the delay? Ampio blames the grinding gears of government for the late registration. The more likely reason, in my book, is that Ampio waited as long as possible before disclosing the glaring design flaws and unrealistic expectations for the Optina study.

Ampio is telling investors that positive results from the Optina study could lead to an approval filing for the drug as a new treatment for diabetic macular edema. That's total nonsense. The efficacy and safety data to be collected from this study falls far short of what FDA would require for approval. At best, Ampio is conducting a phase II study of Optina, to call it a phase III study or a pivotal, registration-quality study is totally misleading.

Diabetic macular edema is a swelling in the central portion of the retina caused by damaged or leaky blood vessels. If untreated, DME can lead to vision loss and blindness. The Ampio study intends to enroll 450 DME patients and randomize them to treatment with Optina (a low-dose reformulation of the generic oral steroid danazol) or a placebo for 12 weeks. The study's primary efficacy endpoint is improvement in vision, measured by the Best Corrected Visual Acuity (BCVA) test.

The FDA has never approved a DME drug based on efficacy from a 12-week clinical trial.

Roche's ( RHHBY) eye drug Lucentis was approved as a treatment for DME in August 2012 based on two phase III studies that measured vision improvement using the BCVA test over two years. The studies included an additional year of patient follow up.

Regeneron Pharmaceuticals ( REGN) is seeking to expand the approval of its eye drug Eylea to allow for the treatment of DME patients. Phase III studies currently underway also measure vision improvement for two years.

Alimera Sciences ( ALIM) also ran two-year studies for its DME drug Iluvien, which like Optina, is a steroid.

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