A closely watched experimental drug to treat Lou Gehrig’s disease failed to work in a late-stage clinical trial, the drug’s developer, Biogen Idec, said Thursday. The company said it would discontinue further work on the drug.
Biogen said that the drug was not effective in either slowing the loss of muscular function or prolonging the lives of people with the disease, formally known as amyotrophic lateral sclerosis, or A.L.S.
There were also no signs that the drug, dexpramipexole, worked in any subgroup of patients, Biogen said.
“As a physician who has treated people with A.L.S., I hoped with all my heart for a different outcome,'’ Dr. Douglas Kerr, director of neurodegeneration clinical research at Biogen, said in a statement.
A.L.S., which attacks the nerves that control muscles, causes gradual paralysis and typically results in death within a few years of diagnosis. There are about 30,000 Americans with the disease.
There is only one drug approved to treat it, Rilutek, made by Sanofi, which doctors say has only modest effectiveness. Many other drugs have failed in clinical trials, in part because scientists do not understand the cause of A.L.S. and therefore do not know how to treat it.
While expectations had not been that high that dexpramipexole would succeed in its phase 3 trial, they were higher than for many previous A.L.S. drugs, given what some doctors viewed as strong results in a smaller, phase 2 trial.
“I’m more excited about this compound than any compound I’ve ever tested in A.L.S.,'’ Dr. Robert Miller, director of A.L.S. research at the California Pacific Medical Center in San Francisco, said in April on a call with investors hosted by Deutsche Bank. Patients getting the highest dose had an almost a 50 percent slower decline in muscular function than those receiving a placebo.
Biogen had licensed dexpramipexole from Knopp Biosciences, a privately held company in Pittsburgh. Biogen’s trial included 943 patients in 11 countries. The main measurement of success was a composite that took into account both deaths from the disease and the decline of functionality. Patients in the trial were allowed to take Rilutek.
Dr. Jeffrey D. Rothstein, director of the Brain Science Institute at Johns Hopkins University School of Medicine, said the results were disappointing but not surprising.
“We really, really need a new drug,'’ Dr. Rothstein, who was not involved in the phase 3 trial, said by e-mail. He said he thought the phase 2 results for dexpramipexole had been only “marginal,'’ so he was not surprised the latest trial did not succeed.
The reaction on Wall Street was somewhat muted because of the modest expectations for the trial to succeed. Biogen’s shares were down about 4 percent.
“We (and most of the Street) had characterized this trial as a high risk trial,'’ Mark Schoenebaum, an analyst at ISI Group, wrote in a research note Thursday.
Other companies are also developing drugs to treat A.L.S. Neuraltus Pharmaceuticals, a privately held company in Palo Alto, Calif., is preparing to enter the final stage of clinical trials for NP001.
The company announced in late October that its phase 2 trial, which involved 136 patients, failed to show a statistically significant benefit compared with a placebo. But the company said that 27 percent of patients getting the high dose of NP001 had no progression of their disease for six months, two and a half times as many as in the placebo group.
Cytokinetics, of South San Francisco, Calif., is in midstage testing of a compound, tirasemtiv, which might make muscles respond more forcefully to nerve signals.
Biogen Gives Up on Drug for Lou Gehrig’s Disease
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Biogen Gives Up on Drug for Lou Gehrig’s Disease
