已经公布了初步的实验结果，效果非常好。
QLT ANNOUNCES POSITIVE INTERIM RESULTS FROM PHASE Ib STUDY OF ITS ORAL SYNTHETIC RETINOID COMPOUND IN LEBER CONGENITAL AMAUROSIS
FOR IMMEDIATE RELEASE APRIL 20, 2010
VANCOUVER, CANADA—QLT Inc. (NASDAQ: QLTI; TSX: QLT) (“QLT” or the “Company”) today announced interim results from the first 3 subjects enrolled in a Phase 1b clinical proof-of-concept study of QLT091001 in the treatment of Leber congenital amaurosis (LCA), an inherited progressive retinal degenerative disease that leads to retinal dysfunction and significant visual impairment beginning at birth. QLT091001 is an orally administered synthetic retinoid replacement for 11-cis­-retinal, which is a key biochemical component of visual function.
The Phase 1b trial is a short-term, open-label, single-center study to evaluate the safety profile and effects on retinal function in 8 pediatric subjects (aged 5 to 14 years) diagnosed with LCA due to inherited deficiency of retinal pigment epithelium protein 65 (RPE65) or lecithin:retinol acyltransferase (LRAT). Based on the positive results from the first 2 pediatric patients, a protocol exception was granted to also treat an adult patient. Subjects receive daily oral doses of QLT091001 for 7 days at the Montreal Children's Hospital at the McGill University Health Centre, Montreal, Canada under the supervision of the trial's principal investigator, Robert K. Koenekoop, M.D., Ph.D. Patients were monitored to ensure overall safety. Efficacy assessments included several visual function parameters including best-corrected visual acuity and visual field testing.
Interim Results
Three subjects aged 10, 12, and 38 years, all of whom have a genetic mutation in LRAT, have been enrolled and treated to date. After 7 days of treatment with QLT091001, all of the subjects experienced clinically relevant improvements in one or more visual function parameters, including best-corrected visual acuity, Goldmann visual field, and/or retinal sensitivity as measured by full-field sensitivity threshold testing. Subjects have also reported meaningful improvements in their visual performance related to tasks of daily living. The onset of visual changes was rapid and there was progressive improvement beyond the 7 days of treatment, with some effects persisting for up to 4 months after treatment was completed. Improvements were most pronounced in the youngest subject, but clinically relevant changes were also noted in the one adult subject treated to date. The study treatment has been well-tolerated, with mild to moderate adverse events observed including transient headache and photophobia, and an increase in triglyceride levels. The study is ongoing and will enroll additional subjects, including those who have LCA due to mutations in RPE65. Because of the prolonged treatment effects, the study will also continue to gather longer-term follow-up data on these subjects. Completion of the current trial is expected before year end.
The results from the first 3 subjects will be introduced and discussed by Dr. Koenekoop in a previously scheduled mini-symposium entitled, "An Overview of Retinal Dystrophies: from Gene Discoveries to New Therapies" at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting in Fort Lauderdale, Florida on May 3, 2010 at 3:45 p.m. (Eastern Time).
“These preliminary results are very exciting, are better than expected, and provide a measure of hope that a treatment might be developed for this devastating disease. We are intent on continuing the trial and undertaking further research into the safety and efficacy of this compound,” said Dr. Koenekoop, Director of the McGill Ocular Genetics Laboratory and Chief of Pediatric Ophthalmology at Montreal Children's Hospital. “We look forward to sharing these data with the ophthalmology community beginning at ARVO in early May.”
“We are very excited about the positive outcomes for these patients and are eager to complete patient enrollment,” said Bob Butchofsky, President and Chief Executive Officer of QLT.
While these early results are promising, the safety and efficacy of QLT091001 remains to be fully evaluated through additional preclinical and clinical testing. QLT091001 cannot be made available to patients with LCA outside of regulated clinical trials, such as the current study. |
