Biogen Idec Inc. chief executive George Scangos was skeptical when he learned company researchers were pursuing a new treatment for Lou Gehrig’s disease. No one knows the cause of the illness that killed the famous baseball player more than 70 years ago, and no medicine for it has been shown to slow its advance for long.
Scangos changed his mind after he took charge of the Weston-based company two years ago and reviewed data about dexpramipexole. The compound, which may slow disease progression, is now in the final stages of clinical trials required for US Food and Drug Administration approval, with results expected this year.
“I don’t know any disease that’s in more need of therapy than ALS,” said Scangos, whose company is the world’s largest maker of drugs for multiple sclerosis, in a telephone interview. “It’s certainly risky, but the data speaks for itself. So we made a calculated bet.”
ALS, or amyotrophic lateral sclerosis, affects about 30,000 people at any time in the US, causing nerve damage that leads muscles to shut down progressively over three to five years until most patients die from respiratory failure. Lou Gehrig, who played for the New York Yankees, retired from baseball in 1939 when he was diagnosed with the disease, and died two years later, bringing wider awareness to ALS yet hardly any treatments.
There’s one drug on the market, Paris-based Sanofi’s Rilutek, and it provides only a modest benefit in reducing ALS’s progression. Rilutek has US sales of about $50 million, according to Eric Schmidt, an analyst with Cowen & Co. in New York.
The market for dexpramipexole, if it’s approved, may top $1 billion a year, Schmidt estimates. The company paid $80 million in cash and stock for a licensing deal in August 2010 with closely held Knopp Biosciences, which first developed the drug, and will pay an additional $265 million if certain regulatory and sales goals are met.
The compound looked promising enough in earlier trials by Knopp to attract the attention of Al Sandrock, Biogen’s head of neurology research and a former physician who worked with ALS patients. It’s designed to improve functioning of the mitochondria, the energy producers in cells, and to provide protection to neurons under stress.
Phase 2 trials showed that after 12 weeks, people on the highest dose of dexpramipexole had about a 35 percent slowing of ALS progression compared with patients taking a placebo, said Merit Cudkowicz, principal investigator of the phase 2 and phase 3 trials and director of the ALS Multidisciplinary Clinic at Massachusetts General Hospital in Boston.
Patients given higher doses of the medicine got better results, Cudkowicz said in a telephone interview. “It was a nice dose-dependent effect. We didn’t expect that at 12 weeks.”
Biogen’s phase 3 trial, generally the last stage before review by regulators, enrolled its first patient in March 2011, and the study was filled within four to five months. That’s half the time a trial this size, involving about 900 patients, would normally take, Cudkowicz said. It was the fastest study enrollment in Biogen’s history.
This doesn’t guarantee the treatment will work, researchers and others caution. There have been as many as 20 consecutive failed studies of experimental medicines for ALS, Cowen’s Schmidt wrote in a February research note, citing physician consultants.
In 2010, data published in the journal Neurology showed that lithium -- thought to be helpful for ALS patients after a small Italian study -- didn’t provide any benefit. In 2003, a study published in the Annals of Neurology showed that creatine, an over-the-counter supplement taken by many for the disease, doesn’t help. Cephalon Inc., the biotechnology company purchased last year by Teva Pharmaceutical Industries Ltd. for $6.2 billion, struggled for years in the 1990s to get a drug called Myotrophin approved before giving up as trial results failed to persuade regulators.
For some patients, Biogen’s medicine is their best hope.
“I’d be waiting at the pharmacy for it as soon as it’s FDA- approved,” said Laura Tuttle, a registered nurse who was diagnosed with ALS in 2009, too long ago to be eligible for Biogen’s phase 3 study.
Her first hint of the disease came in April 2009, when she was walking through an airport and noticed her left foot “wasn’t cooperating.” She wasn’t diagnosed though until the end of that year, after she’d started tripping and falling. Having worked with ALS patients, she’d already guessed she had the disease. Tuttle said she finally told her physicians, “’What’s it going to take for us to call it what it is?’ It was almost as if they were afraid to tell me.”
Tuttle now gets around in a power wheelchair and occupies only the first floor of her house outside Boston. Her husband is her primary caregiver. She has participated in studies for two experimental treatments for ALS, including ceftriaxone, an antibiotic in phase 3 trials. It had to be administered intravenously twice a day, and infusions took about two hours every morning and evening, she said.
Biogen’s dexpramipexole is a pill, another reason patients are so excited by it. The last study turned up no major safety concerns.
Some patients want to know why a medicine that’s shown safety and some efficacy in earlier trials isn’t being made available immediately, said Lucie Bruijn, chief scientist at the ALS Association. She cautions that although dexpramipexole is promising, “this is nowhere near a cure.” Still, patients who’ve seen Biogen’s success with multiple sclerosis, a disease of the central nervous system that can cause limb numbness, hope the company can accomplish the same in ALS, she said.
Biogen applied for FDA approval earlier this year for BG- 12, its first pill for MS, which may draw as much as $3 billion in annual revenue if approved, according to estimates from Cowen’s Schmidt and other analysts. The company also makes Avonex, an injectible treatment for MS that had revenue of $2.7 billion last year, and Tysabri, an intravenous therapy that sold $1.1 billion.
The data from Biogen’s phase 3 trial in ALS are expected in the second half of this year, and the medicine is on a fast- track path with the FDA.
“This is far from a long shot,” CEO Scangos told investors at JPMorgan Chase & Co.’s annual health-care conference in January. “We are genuinely hopeful that we will be able to provide meaningful therapy for the thousands, tens of thousands, of ALS patients who right now have very little to help them.”