The growth factor BetaKine has shown a statistically significantability to heal macular holes in the retina, both in terms ofrepairing tissue damage and restoring lost vision.
Preliminary data from the Phase II clinical trial on BetaKine,which is being developed by Celtrix Pharmaceuticals Inc.(NASDAQ:CTRX), were presented Tuesday at the annual meetingof the Association for Research in Vision and Ophthalmology(ARVO) in Sarasota, Fla. They demonstrated that BetaKine wasable to repair macular holes in the retinas of more than 90percent of treated patients and that it restored substantialvision in 79 percent of the individuals treated with the higherdose in the study.
"The way the trial was designed, the endpoint is very clear,"explained Sandra McNamara, Celtrix's chief financial officer.That the data are statistically significant is reflected by the pvalue of 0.001, she told BioWorld.
The placebo-controlled trial included 90 patients with macularhole damage, divided into three arms. They received either aplacebo or one of two doses (1,330 nanograms or 660nanograms) of BetaKine (transforming growth factor beta-2,which is known to be a potent peptide) applied around theedges of the macular hole during a surgical procedure knownas vitrectomy, which has been used for the past few years as away of "limiting the problems that arise with macular holes,"which damage fine, central vision, according to McNamara.
According to retinal surgeon William Smiddy of the BascomPalmer Eye Institute, University of Miami School of Medicine,who co-managed the clinical trial, "After three months ofhealing, the macular hole had closed in 96 percent of the eyesreceiving the higher dose of BetaKine and 93 percent of theeyes receiving the lower dose. Of those receiving the placebo,the macular holes had closed in 55 percent of the eyes." Andone year after treatment, "a significant majority of patients hadimproved vision," Smiddy added.
Celtrix of Santa Clara, Calif., is planning to initiate Phase IIItrials this year on BetaKine for treating macular holes, on whichit received orphan drug status from FDA in January. McNamaratold BioWorld that there are currently an estimated 10,000cases in the U.S., and that "the market is small enough thatwe're not anticipating competition."
But with safety data on more than 500 patients, Celtrix is"aggressively exploring other opportunities for using BetaKineto treat a variety of vision-limiting or potentially blindingconditions," said Bruce Pharriss, chief executive officer.
Celtrix's stock (NASDAQ:CTRX) jumped $1.50 on Monday inanticipation of these Phase II results, to close at $9.50, butclosed Tuesday at $8.75 a share, off 75 cents.
-- Jennifer Van Brunt Senior Editor
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