Biogen Idec Inc. shares soared to a record high after its experimental drug for Alzheimer’s slowed progression of the disease in a small study. The result offers a glimmer of hope after a string of failures by competitors who have tackled the ailment.
In an early-stage trial of 166 patients, Biogen’s drug BIIB037 reversed build-up of beta amyloid in the brain and also reduced cognitive decline, with higher doses and longer treatment resulting in more improvements. Beta amyloid, a protein fragment that creates plaque in the brain, was observed with PET scans. Cognition was measured with tests used by neurologists, including one the Food and Drug Administration may use to assess the drug for approval.
“Either result alone would have been exciting, but to see them both in the same study makes it even more exciting,” said Alfred Sandrock, Biogen’s chief medical officer. The results are positive enough that Biogen is skipping midstage testing and moving directly into final-stage trials that will begin later this year, Sandrock said.
The company is pushing rapidly ahead into an area where other drugmakers have tried and fell short in final-stage trials. The medications solanezumab, from Eli Lilly & Co., and bapineuzumab, developed by Pfizer Inc., Johnson & Johnson and Elan Corp., both failed to show a significant effect on the disease in trials. Lilly is currently retesting solanezumab in earlier-stage patients, while bapineuzumab has been scrapped.
In less than five years, Biogen’s market capitalization has grown from about $15 billion to almost $110 billion, making the company one of biotechnology’s biggest. Its biggest holders are institutional investors such as Fidelity Investments, Vanguard Group Inc., BlackRock Inc. and T Rowe Price Group Inc.
Biogen’s shares have gained 28 percent this year through Thursday’s close, and rose 7.5 percent to $466.03 at 11:40 a.m. in New York, after earlier reaching an intraday all-time high of $480.18.
With no drug to cure Alzheimer’s or lessen its advance available for sale, a working treatment offers a huge opportunity for drugmakers like Biogen. Since Biogen reported preliminary findings from the BIIB037 trial in December, the stock had gained 41 percent.
Investors have awaited the full trial results to gauge whether they back up the initial optimism over the drug’s chances. Chief Executive Officer George Scangos told investors at the Cowen Group Health Care Conference in early March that management was “quite excited” about the data and that late-stage trials should start later this year.
“It’s a major advance in confirming amyloid beta is the right target,” said Colin Masters, a laureate professor at the University of Melbourne who has studied Alzheimer’s disease for more than 30 years. He spoke by phone from Nice, France, where he’s attending the scientific meeting where the results were presented.
The trial may have avoided pitfalls that tripped up other drugmakers because it focused on patients in the early stage of Alzheimer’s disease, and made sure they had beta amyloid in the brain to verify they had Alzheimer’s, Sandrock said.
After a year at the highest dose of treatment, the PET scans showed that plaque was reduced to a level that was “very close to the upper limit of normal,” he said. At the lowest dose, the reduction wasn’t statistically significant.
On cognition, based on what’s called the Clinical Dementia Rating, patients in the placebo group worsened by an average 2.04 points after one year, compared with 1.7 points among patients who took the lowest dose and 0.59 points in the highest dose.
“We are excited about it,” said Keith Fargo, director of scientific programs and outreach at the Alzheimer’s Association, a Chicago-based non-profit advocacy group. “We have to do the big phase 3 study to see if it’s actually going to work. There have been a lot of promising phase 1 and phase 2 trials that didn’t work out.”
The drug had side effects, notably brain swelling known as amyloid-related imaging abnormalities, or ARIA, that can cause headaches. Among patients carrying the ApoE4 gene, which is strongly linked with Alzheimer’s, 55 percent of patients taking the trial’s highest dose of 10 milligrams reported ARIA, compared with 5 percent among those in the 1 mg dose group. That resulted in 35 percent of the ApoE4 carriers on the highest dose discontinuing treatment.
“The field is still learning about ARIA, but it appears to occur more frequently with these anti-amyloid therapeutics,” said James Hendrix, director of Global Science Initiatives at the Alzheimer’s Association. “This is an important adverse event that has to be monitored as they move into Phase 3.”
Patients carrying the ApoE4 gene may be candidates for lower doses of the drug, said Asthika Goonewardene, an analyst for Bloomberg Intelligence in New York. Biogen is still awaiting 54-week data from a 6 mg dose group.
Alzheimer’s is the most common form of dementia, affecting more than 5 million Americans, according to the Alzheimer’s Association. The disease, which is progressive, causes symptoms including memory loss, confusion and behavior changes. There is no approved cure for Alzheimer’s.
Biogen is best known for its multiple sclerosis drug Tecfidera, which brought in $2.9 billion in sales last year. The company bought BIIB037, or aducanumab, from Neurimmune Therapeutics in 2007.
“You can never be certain in this industry,” Sandrock said. “But we feel confident enough in the data that we’ve seen that it’s a good investment to make to move forward aggressively into phase 3.”