Regeneron Pharmaceuticals Inc. reported Thursday at theannual meeting of the American Academy of Neurology that itspotential treatment for amyotrophic lateral sclerosis,recombinant human ciliary neurotrophic factor (CNTF), waswell-tolerated in a Phase I safety trial.
Analyst Franklin Berger of Josephthal, Lyon & Ross attendedthe meeting and said the results seemed "very positive."
The Tarrytown, N.Y., company (NASDAQ:REGN) has completed aPhase II trial and is in a Phase III trial now with rhCNTF inmore than 30 centers in the U.S. and Canada.
Results of the Phase I trial in 57 ALS patients were presentedby Benjamin Brooks of the University of Wisconsin, who saidthe substance, which stimulates repair of damaged nerve cells,"is the first drug of its kind to be tested in a humanneurodegenerative disease."
Doses similar to those used in the Phase III study producedfew side effects and created biologically active plasmaconcentrations after being injected under the skin.
Regeneron collaborators Hiroshi Mitsumoto and Ken Ikeda ofthe Cleveland Clinic Foundation studied the compound inWobbler mice, which suffer from an inherited motor neurondegenerative disease similar to human ALS.
Misumoto said this was the first potential ALS therapy he hastested in Wobbler mice that reduced loss of motor nerve cellsand decreased muscle atrophy, although he cautioned that suchresults do not guarantee success in ALS patients.
ALS, or Lou Gehrig's disease, is a degenerative condition that isalways fatal within five to seven years in adults. It has no cure.
Other companies addressing ALS are Cephalon Inc. of WestChester, Pa., which is developing a recombinant form of insulin-like growth factor and Synergen Inc. of Boulder, Colo., which iscollaborating with Syntex Corp. on CNTF. -- Nancy Garcia
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