March 24, 2015
By Riley McDermid, BioSpace.com Breaking News Sr. Editor
Investors and analysts that were delighted with last week’s data from Biogen Idec that showed the company’s experimental Alzheimer’s drug BIIB037 reduces amyloid plaque in the brain have hit a new roadblock, as new research shows the disease may be more effectively treated if a separate part of the brain is targeted.
In fact, that research, from a recent study by the Mayo Clinic published Tuesday in the journal Brain, showed that dysfunctional tau protein is the primary reason for cognitive decline and memory loss seen in Alzheimer’s. That’s bad news for Biogen, which only targets amyloid plaque with BIIB037.
“Amyloid has a relationship with cognitive decline, but if you’re looking at both of them together, tau is the bad guy,” Melissa Murray, a neuroscientist at the Mayo Clinic, told Bloomberg News.
The study released Tuesday measured 3,600 brains from patients who died at different stages of dementia, with Alzheimer’s confirmed around 1,400. But when researchers stacked up the amount of amyloid and tau as the disease progressed, they found that it was level of tau that caused the fastest deterioration.
Dean Hartley, director of science initiatives at the Alzheimer’s Association, told Bloomberg there might still be room for both treatments.
“Depending on where you are in the disease, you may need certain types of treatments,” Hartley said. “If you’re later in the disease, maybe tau is extremely important, but if we don’t want any of the clinical symptoms to develop, then maybe we want to target amyloid.”
Biogen‘s news Friday was closely watched because it gave new details from an initial announcement in December, when Biogen announced a statistically significant reduction in amyloid levels and improved cognition compared to a placebo.
Now, Mark Schoenebaum, a biotech analyst and medical doctor who leads the biotech team at ISI Evercore, said in a note to investors Friday that the drug is already setting itself up as a competitor for Eli Lilly and Company ’s solanezumab. “The doctor’s overall impression of the data was extremely positive, with caveats,” wrote Schoenebaum in a note.
“In our doctor’s opinion, the data presented today put BIIB037 ‘in a completely different league’ than Lilly‘s solanezumab,” said Schoenebaum. “He said that most investigators were very positive about the data, and some were ecstatic. For our doctor, it was critical there was a dose response curve on MMSE and CDR-SB in a relatively short time frame.”
BIIB037 binds to amyloid plaques in the brain as a way to slow the progress of Alzheimer’s disease (AD). Biogen‘s head of research and development, Doug Williams, said at the time it had achieved those endpoints and more, reducing amyloid levels in “both a dose- and time-dependent fashion.”
Williams said that BIIB037 improved cognition in patients with early signs of the disease 54 weeks after starting treatment, in a Phase 1 trial involving 200 patients. He added that Biogen will present full results of the trial at a medical meeting next year.
Because of those encouraging results at such an early stage of testing, the company will be leap-frogging BIIB037 into Phase III trials, said Biogen. It did not give a date for when the trials would start but analysts have projected the likely start will be mid-2015.
Wall Street analysts presented with the data at Deutsche Bank‘s BioFEST conference in Boston In Dec. 6 were literally speechless at the impact of the results in a field known for chronic failures.
“You’ve got me tongue-twisted with the Alzheimer’s data,” Deutsche Bank analyst Robyn Karnauskas told Williams during the presentation, reported CNBC.
The overwhelmingly encouraging results were a surprise to biotech watchers, because several large companies have tried and failed to develop similar Alzheimer’s therapies in recent year, including Johnson & Johnson , Eli Lilly and Company (LLY) and Pfizer Inc. .
The size of the market for patients with AD varies in estimates, but Schoenebaum has predicted pricing to weigh in at around $25,000 per course.
“There are 600,000 mild AD patients in the U.S.,” he wrote. “At a net price of around $20,000 (below TNFs), 65 percent penetration would yield a potential sales opportunity of around $8 billion in the U.S. alone. The ex-U.S. patient opportunity is similar, but price will probably be lower.”
That continues a consistent trend of anti-amyloid studies focusing on the mild patient subset, said Joshua Schimmer, a biotech analyst at Piper Jaffray, on Dec. 7.
“It is plausible to believe that early intervention on amyloid is a requisite for success, so while many companies and investors have given up hope on this indication, Biogen seems to have a viable shot on goal left for this massive commercial opportunity,” wrote Schimmer in a note to investors.
“Investors finally have an attractive pipeline program to focus on to help alleviate pressure from ongoing MS drug launches, patent and PML considerations for Tecfidera, and uncertainty on the anti-Lingo program,” said Schimmer.
The news is also good for Biogen’s market reputation, which has suffered from a Street perception that it’s drug pipeline has been fickle and inconsistent.
“We believe this will finally bring stability to Biogens multiple while the bottom line continues to grow at 25 percent or more per year. As such, Biogen moves up on our large cap conviction list as our second favorite pick,” wrote Schimmer.
“We believe that as investors pick over multiple datasets for crenezumab and solenezumab that show a benefit in the mild patient subsets, consider that the bapineuzumab studies included patients without amyloid accumulation and reflect on the BIiB037 data,” said Schimmer, “they will see an attractive risk reward profile that skews largely to the upside considering the multi-billion dollar potential Alzheimer’s opportunity. We look forward to a formal data presentation, complete with press release.”
BioSpace Temperature Poll
U.K.-based GlaxoSmithKline filed a WARN letter in late February with the state of Pennsylvania indicating another 150 people would be laid off in its commercial and research and development group near Philadelphia. BioSpace wants to know if you think Pharm Country will do what it takes to keep biotech jobs in the area?
