Acquiring the rights and intellectual property from two German university scientists, Enzo Biochem Inc. has expanded its immune regulation portfolio to include a candidate to treat uveitis, an eye disease that can lead to blindness.
Based in Farmingdale, N.Y., Enzo is developing a gene medicine for HIV-1 infection and immune regulation medicines for hepatitis B, hepatitis C and Crohn's disease. The newly acquired candidate for uveitis also is designed as an immune regulator.
"The thinking is this will lead to a long-term maintenance of this dangerous immune condition for those subjects that have it and are progressing," said Dean Engelhardt, Enzo's executive vice president.
Posterior uveitis, the chronic inflammation of a part of the eye known as the uvea, is believed to result from an immune reaction against some of the antigens in the eye, specifically the S-antigen protein and the interphotoreceptor retinoid-binding protein.
Two scientists, Stephan Thurau and Gerhild Wildner, from the eye clinic of Ludwig Maximilians University in Munich, Germany, discovered that 14-amino-acid long peptide, as it is called, promoted recovery when fed to rats with experimental allergic uveitis. Following favorable preclinical studies, the researchers conducted a small Phase I trial in Germany that showed positive effects, including a decrease in inflammation and an improvement in visual acuity.
"They had eight patients, which they treated orally with this particular 14-amino-acid long peptide, which is not from the antigen itself but from [the protein] HLA-B27, interestingly enough," Engelhardt said. "When they fed this, the inflammation decreased. And most unusually, for five of those eight patients, inflammation was decreased even a year later, so it led to a very durable response."
Enzo intends to continue with clinical development; it is putting together a protocol to start a double-blind, randomized Phase II trial as soon as possible in Germany. The candidate has orphan drug status in Europe. Development plans for the U.S. market are not yet disclosed.
Engelhardt declined to say whether the agreement for the compound includes milestone payments or royalties to the two scientists, Thurau and Wildner, but he did say they will be a part of any success that results from the technology.
"They will be compensated," he told BioWorld Today. "They've done the pioneering work, they made the discovery, and they're the inventors on the patents."
About 38,000 people are diagnosed with uveitis in the U.S. each year. Engelhardt said it affects 17 people out of 100,000 in both the U.S. and Europe. There are some options for treatment that might help in preserving sight, but the disease can make patients more at risk for developing cataracts, glaucoma or retinal detachment.
"It can be managed with prednisone, but it's a course of medication that can have reasonably unpleasant side effects," Engelhardt said.
If the 14-amino-acid long peptide works, it should cut down on systemic reactions and provide patients with a safer treatment option.
Behind Enzo's four current clinical programs in the fields of HIV, hepatitis B and C, and Crohn's disease, the company is studying preclinical candidates for ulcerative colitis, hepatocellular carcinoma, and graft-vs.-host disease. Its research is looking at several candidate compounds that could produce new mineral and organic bone, providing new therapies for osteoporosis and fractures. In addition to its therapeutic pipeline, Enzo sells its labeling and detection products for gene sequencing and genetic analysis to the life sciences market. The company was founded in August 1976.
Concurrent with news of the uveitis drug, Enzo released its third-quarter results Thursday. For the nine months ended April 30, Enzo reported total revenues of $32.5 million, and a net income of $5 million. The company posted cash, cash equivalents and investments of more than $80 million.
Enzo's stock (NYSE:ENZ) dropped 61 cents Friday to close at $14.12.