GREENWOOD VILLAGE, Col. ( TheStreet) -- Ampio Pharmaceuticals ( AMPE) announced Monday "positive clinical trial results" from a study of a low-dose generic steroid in diabetic macular edema that looks much more like a failure. The study in question enrolled 32 patients with diabetic macular edema (DME), or swelling of the central portion of the retina caused by damaged and leaky blood vessels. DME can occur in patients with diabetes, and if untreated, can lead to blindness. Ampio treated the 32 DME patients with one of three oral doses of Optina, a low-dose reformulation of the generic steroid danazol, or a placebo. Ampio claims low doses of danazol can reverse inflammation thereby making blood vessels less leaky. To test this theory, the Optina study's primary endpoint was the reduction of macular edema after four and 12 weeks of treatment, assessed using a device that measures the thickness of the retina. The Optina study failed. No statistically significant difference in retinal thickness was measured between patients treated with any three of the Optina doses compared to a placebo. The only glimmer of optimism came from the lowest dose of Optina showing a trend towards reduced retinal thickness over placebo; that result, however, was not statistically significant, Ampio said. The highest dose of Optina tested in the study didn't produce a benefit for DME patients, according to an Ampio statement issued in March. On Monday, Ampio said nothing about the performance of mid- and high-dose Optina dose groups, but Vaughan Clift, Ampio's chief regulatory officer, told me in a phone call Monday that no significant benefit in either the middle or high-dose Optina patients was observed in the study. Yet Clift strongly disagreed with any suggestion that the Optina study was a failure. He insisted that not only was the study an absolute success, but the data show Optina to be "the most exciting development in diabetic eye care in the last 30 years… We can stop blindness." Really? True, if you believe in Ampio's spin, er… I mean… interpretation of data reportedly demonstrating Optina can reduce retinal thickening as long as the dose is aligned correctly with the body mass index (BMI) of the patient.
Lower doses of Optina, Clift explained, only work in low-BMI patients. If you use a low dose of Optina in an obese patient, the drug doesn't work. You'd think higher doses of Optina would work regardless of whether the patient had a low or high BMI. If Optina acted like most drugs, a higher dose would lead to greater benefit (up to a point). That's called a positive dose response. But not true with Optina, according to Clift's interpretation of the study results. Higher doses of Optina only work in patients with a high BMI. If you give a higher dose of Optina to a skinny patient, the drug stops working. If BMI is so crucial to the efficacy of Optina, why hasn't Ampio ever mentioned this correlation before? Previous disclosures about the Optina study don't talk about efficacy being dependent on patients' respective BMIs, including the press release issued by Ampio last March. Clift claims Ampio kept all this BMI stuff top secret because of in-progress patent filings. If the company had made public the fact that Optina's efficacy is linked to patient weight, he adds, Big Pharma companies like Pfizer would have swooped in to steal the intellectual property supporting Optina.
Remember, Optina is just low doses of the cheap, generic steroid danazole. Before speaking with Clift, Ampio's spokesperson Rick Giles told me that the company discovered the Optina-BMI link only after the clinical trial was underway and the data were being analyzed. Clift said Giles was mistaken. "There is nothing mysterious in this effect," says Clift. "There is no conspiracy, we're not trying to lie, cheat or steal." But it surely sounds as if Ampio is engaging in one of the most egregious examples of retrospective data-mining I've ever come across. If Optina was truly a cure for DME-related blindness, patients' vision should improve. Ampio measured the visual acuity of patients in the Optina trial but Clift refused to disclose the results. By comparison, Lucentis, marketed by Novartis ( NVS) and Roche ( RHHBY) as a therapy for DME and other related eye diseases, actually helps patients see better. Regeneron Pharmaceuticals ( REGN) hopes to show the same with its recently approved drug Eylea. As incredulous as Ampio's Optina spin job appears to be, the company's stock is up 27 cents, or 8%, to $3.47 in late Monday trading. Smart investors know stock prices can be deceiving in the short run, yet there's little in Monday's Optina data to justify having much long-term confidence in the drug's future. --Written by Adam Feuerstein in Boston. >To contact the writer of this article, click here: Adam Feuerstein. >To follow the writer on Twitter, go to http://twitter.com/adamfeuerstein. >To submit a news tip, send an email to: firstname.lastname@example.org. Follow TheStreet on Twitter and become a fan on Facebook.