Cephalon Inc. said Tuesday a second Phase III Europeanstudy of Myotrophin for amyotrophic lateral sclerosis(ALS) confirmed findings from an earlier NorthAmerican trial showing the drug slowed progression ofthe fatal disease and lessened its severity.
Data from the 183-patient study at eight medical centersin six European countries revealed those receivingMyotrophin, which is recombinant human insulin-likegrowth factor, experienced 22 percent less deteriorationthan those taking a placebo.
The statistically significant findings, Cephalon said,confirmed results of a Phase III North American trial,reported in June 1995, demonstrating the drug slowedprogression of ALS, or Lou Gehrig's disease, and reducedfunctional severity of the disorder. As in the NorthAmerican trials, the drug was judged safe and well-tolerated in Europe.
Jason Rubin, spokesman for the West Chester, Pa.-basedCephalon, said an 18-month analysis of patients from thefirst Phase III study also revealed a six-month "survivalbenefit" for those receiving Myotrophin compared withpatients in the placebo group. The 18-month analysisincluded nine months of follow-up after the clinical trial.
"We've not yet done a comparable survival analysis ofthe European data," Rubin said.
The consistency of Myotrophin's performance in the twotrials, he added, should "enable us to take a very strongcase to the FDA."
Cephalon, which developed Myotrophin in a 50-50collaboration with Emeryville, Calif.-based Chiron Corp.,expects to file a new drug application with the FDA in thefirst quarter of 1996. Filing for clearance in Europe isexpected to follow later the same year.
Cephalon's stock (NASDAQ:CEPH) closed Tuesday at$30, up 25 cents. Chiron (NASDAQ:CHIR) ended theday at $91, a $3 increase.
Matthew Geller, an analyst with Oppenheimer & Co. inNew York, said the data from the European Myotrophintrials are complex.
"So far, it looks successful for approval," he said. "At thispoint the results look promising, but to fully understandthe clinical implications, further analysis is needed."
Geller also noted Cephalon's report on prolongingsurvival in the first Phase III study involved analysis afterthe clinical trial ended. "It has not been provedrigorously," he added.
In the nine-month European trials, 124 patients receivedMyotrophin and 59 received placebo. Of the 23 patientswho died during the studies, more were in theMyotrophin group than in the placebo group.
But Rubin observed, "There was no statisticallysignificant difference in the deaths that occurred in theplacebo and treatment groups."
Currently no effective treatment is marketed for LouGehrig's disease. The degenerative neuromusculardisease destroys motor neurons, leading to loss of musclecontrol and death from respiratory failure three to fiveyears after onset of the disorder. An estimated 70,000people worldwide have ALS and half are in the U.S.
Rhone-Poulenc Rorer Inc., of Collegeville, Pa., has theonly ALS drug candidate under FDA review in the U.S.In September 1995, the FDA's Peripheral and CentralNervous System Drugs Advisory Committeerecommended approval of Rilutek, which is a smallmolecule compound. Rorer also has filed for marketclearance in Europe.
Rilutek demonstrated statistical significance in extendingpatients' lives three months, but was not effective inslowing muscle function deterioration.
Neither Myotrophin nor Rilutek are considered cures forALS. But Cephalon has said Myotrophin, indemonstrating it can slow progression of the disease, isthe first treatment to alter the course of the disorder. n
-- Charles Craig
(c) 1997 American Health Consultants. All rights reserved.