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 ClinicalTrials.gov 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.
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