Biogen Idec sued over deal tied to ALS drug

ALS research firm wants samples back; compound’s future development at stake

When biotechnology firm Biogen Idec Inc. halted development of an experimental drug to treat Lou Gehrig’s disease in January, it disappointed tens of thousands of patients who suffer from the devastating neurological disorder formally called ALS.

Now the company that licensed the drug compound to the Weston biotech three years ago is alleging in a lawsuit that Biogen Idec is breaching its agreement and dealing in bad faith by refusing to return biosamples from its late-stage clinical trial.

Knopp Neurosciences Inc., a nine-year-old Pittsburgh company formed to discover treatments for ALS, said it is hoping to use the blood samples from 943 patients who took part in the Biogen Idec-sponsored trial to identify patients who might still benefit from the drug compound, known as dexpramipexole. Knopp is pushing forward with its own work on dexpramipexole, and also wants Biogen Idec to return extensive regulatory data generated by the failed trial.


“We want the opportunity to continue developing this drug for ALS,” said Tom Petzinger, cofounder of Knopp. “For us to do that, we need our regulatory records back. And we also need the biosamples so we can better understand who responds to this drug.”

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Biogen Idec said that even though the drug compound proved ineffective, it plans to use the blood samples for research into other therapies. The company contends it has no contractual requirement to return the samples to Knopp.

“We don’t have an obligation under the licensing deal to transfer that data,” said Daniel McIntyre, senior vice president for public affairs at Biogen Idec. “We’d need specific permission from patients before we could transfer the data, and we don’t have that permission.”

The stakes are high in the lawsuit, which is scheduled to be heard the week of June 17 in US District Court in Boston. The biosamples “are very precious to the ALS research community for understanding the disease,” said Dr. Merit Cudkowicz, chief of neurology at Massachusetts General Hospital. Cudkowicz, who was lead investigator for the Biogen Idec trial, did not take a position on the dispute.

The lawsuit has caught the attention of ALS patients and their supporters, who are hoping that, whatever the outcome, biopharmaceutical scientists can capitalize on the research done by both Knopp and Biogen Idec.


Amyotrophic lateral sclerosis, which affects about 3,000 Americans, prevents nerve cells from sending messages to muscles, leading to muscle weakening, twitching, and, eventually, paralysis. Most patients die within three to five years after being diagnosed.

The only ALS medicine approved by the Food and Drug Administration is Riluzole, which can slow symptoms, but other drugs are in development.

“There are very exciting therapies in the pipeline right now which are showing progress in the treatment of ALS,” said Lynn Aaronson, executive director of the Massachusetts chapter of the ALS Association, a Norwood-based nonprofit. “There’s still hope, and there’s still a lot of room for development of ALS drugs.”

Knopp lodged its initial lawsuit against Biogen Idec on Feb 25, charging the Massachusetts company wasn’t complying with a commitment in the licensing agreement to return “all materials,” including the blood samples.

In an amended complaint, filed last Friday, Knopp also alleged Biogen Idec is withholding the file on dexpramipexole’s development, including a range of data and the “safety database” from the late-stage clinical trial named Empower.


“Failure to preserve the Empower biosamples will deprive ALS sufferers of potential life-altering treatments,” the Knopp suit maintained. It also asserted that, “Holding the [data] is the prerequisite to Knopp being legally permitted to conduct any post-Empower [dexpramipexole] clinical research or even approach the FDA or other regulatory authorities about such research.”

Knopp, founded in 2004, was named for Walter Knopp, an ALS patient who gave a gift to the University of Pittsburgh to research ALS treatments. Company founders licensed technology from the University of Virginia, where researchers discovered the utility of dexpramipexole in treating ALS.

After running a midstage clinical trial that established “proof of concept” for the compound, Knopp turned it over to Biogen Idec in a 2010 licensing deal that gave the Pittsburgh company up to $345 million in upfront cash, equity, milestone payments, and royalties.

After Biogen Idec disclosed that the late-stage trial had failed and that it was ending the program, Knopp conducted its own post-trial analysis that suggested some patients could benefit from the compound.

Biogen Idec defends its own trial assessment. “While we were deeply disappointed in the results, we’re confident in the validity of our data,” McIntyre said. “We think it would be unfair to offer patients false hopes based on the Empower results.”

He also said, “We have no intention of leaving these biosamples on a shelf. We have an active ALS research program under way.”

Cudkowicz, the lead investigator, said the Biogen Idec trial was “well done” and its results conclusive. She said Knopp’s analysis could have some validity, but also could represent a statistical anomaly.

“It’s hard to know if they found something or they didn’t,” Cudkowicz said. “I’m not ready to throw that [analysis] out yet, but I’m not ready to say we should do another study on dexpramipexole.”

Robert Weisman can be reached at Follow him on Twitter @GlobeRobW.