Ocuphire still focused on FDA approval despite diabetic eye drug missing phase 2 endpoint

Ocuphire Pharma’s diabetic retinopathy (DR) candidate missed the primary endpoint of a phase 2 trial, sending the company’s shares down over 25%. But the biotech has suggested that the drug’s effect on both eyes could still offer a path to FDA approval.

The ZETA-1 study evaluated daily 600 mg doses of the oral drug, dubbed APX3330, or placebo in 103 patients across the U.S., who had at least one eye with moderately severe to severe non-proliferative DR or mild proliferative DR. The trial failed to show that the candidate induced a two-step improvement in a diabetic retinopathy severity score in the affected eye, which was the primary endpoint.

Searching for positives in its post-market release yesterday, the company went hard on the fact that APX3330 hit the secondary endpoint of preventing clinically meaningful progression of DR after 24 weeks of treatment, which Ocuphire said was a “potential phase 3 registration endpoint.” The candidate also demonstrated favorable safety and tolerability, Ocuphire pointed out.

“Although we did not meet the primary endpoint, we are pleased that the ZETA-1 results on key pre-specified endpoints demonstrated positive outcomes with a favorable systemic and ocular safety profile that support our plans to move forward to an end-of-phase 2 meeting with the FDA,” Ocuphire CEO Mina Sooch said in the release.

It wasn’t enough to convince investors, however, who sent Ocuphire’s stock down almost 27% in pre-market trading this morning to $2.75 per share from a Wednesday closing price of $3.76.

Still, the company can afford to be relatively relaxed about its plans thanks to a $35 million upfront payment in November from FamyGen Life Sciences to license Ocuphire’s late-stage Nyxol eye drops for mydriasis, presbyopia and night vision disturbances. The “financial strength” derived from the deal—which includes the prospect of additional milestone and royalty payments—means the company has “considerable flexibility to design and initiate the pivotal stage of the APX3330 program,” Sooch added.

Diabetic patients with non-proliferative retinopathy currently have limited treatment options to prevent progression of retinopathy and loss of vision, the Cleveland Clinic Foundation’s Professor of Ophthalmology Peter Kaiser, M.D., said in the release. “In diabetic patients, APX3330 has demonstrated a favorable safety profile and has the advantage of being an oral agent treating both eyes at once,” Kaiser explained.