A $63 billion biotech is returning to the scene of its worst failure in search of new treatments for a deadly muscle disorder (BIIB)

Biogen facility Cambridge, Massachusetts biotech

An ALS trial failure five years ago was a costly and disappointing setback for Biogen.
But today Biogen is becoming a major player again. It just bought a potential drug in a $90 million deal, and has four treatments in development.
The company's head of neuromuscular disorders said there's reason to believe this time could be more successful — and it could have bigger implications for the company's pipeline.

Five years ago, a drug for a rare and deadly disease failed to live up to its promise in a crucial and costly late-stage clinical trial.

The drug’s developer, the biopharmaceutical company Biogen, abandoned the project, which then-CEO George Scangos would later call “the single-most negative trial I’ve ever seen,” according to a MedCityNews report. The cost was an estimated $75 million to $100 million for the trial alone, the Wall Street Journal reported at the time.

It was a devastating development for a disease known as amyotrophic lateral sclerosis, or ALS, which has no cure or effective treatments. An estimated 14,000 to 15,000 Americans have ALS, which refers to a group of rare neurological diseases that cause progressive loss of muscle control, affecting patients’ ability to walk, eat and breathe.

The drug development space has been littered with defeat after defeat for companies tackling ALS. The few drugs on the market now don't work particularly well, and can command high price tags, with one new option costing roughly $150,000 a year.

But today, Biogen is investing again in treating ALS, including through a recent licensing deal valued at more than $90 million for the ALS drug BIIB067.

A big bet on treating ALS

The neuroscience-focused drugmaker believes in taking on these types of risky diseases in new ways, Chris Henderson, its head of neuromuscular and movement disorders, told Business Insider. A succesful treatment for ALS would likely be a blockbuster treatment, generating big profits for Biogen. Success in ALS could also have positive benefits for Biogen's wider pipeline of potential treatments, he said.

“We’re probably the single company with the biggest interest in ALS currently,” Henderson said.

In total, the company is testing four drugs to treat ALS in clinical trials, the most of any company. Biogen also has led the highest number of clinical trials for ALS in the industry, according to a recent report from Datamonitor Healthcare.

At least 90% of ALS cases are considered “sporadic,” meaning there is no clear cause. The remaining 5% to 10% are connected to genetic risk, according to the National Institutes of Health.

Biogen's new drug, BIIB067, targets ALS cases tied to a particular genetic mutation, in the SOD1 gene, which Biogen says accounts for roughly 2% of ALS cases overall.

Growing interest

Henderson, who also serves as chief advisor to the foundation Target ALS, said that he has seen corporate interest in ALS drug development surge of late. Just five companies came to the group’s first meeting, he said, compared with 65 at the most recent one; he cited Genentech and the buzzy biotech Denali as other leaders in the area.

There are approximately 26 companies involved in US ALS drug development, according to the Datamonitor Healthcare report, with 28 ALS drugs in clinical trials.

Biogen’s bulked-up ALS pipeline is particularly notable given the company’s bumpy history with ALS — and Henderson said that there’s reason to believe this effort could be more successful.

The biotech decided to license BIIB067 based on an analysis of an early-stage study, which found that those on the highest dose of the drug showed a statistically-significant lowering of SOD1 protein levels in cerebrospinal fluid. There was also evidence in the form of a “numerical trend” suggesting the drug could slow functional decline, according to a Biogen release.

The population of individuals with ALS being targeted in this trial is notably small, but Henderson said that its results could eventually translate more widely, with success representing the “first big crack in the glass ceiling of ALS.”

“One reason for thinking the chance of success is much higher here is that we are going after the single genetic cause of disease in these patients,” with a drug that specifically focuses on the disease’s trigger, he said. “We want to gradually go from these rare forms into the whole disease, which would be just an amazing achievement if we can get there.”

There could also be major implications for Biogen's wider pipeline of treatments. BIIB067 is an antisense oligonucleotide (ASO), just like Spinraza, the spinal muscular atrophy therapy that has quickly become one of Biogen’s most important products.

A $1 billion partnership

The category of medicine has been a focus of a decade-long, $1-billion partnership between Biogen and the drugmaker Ionis.

But Spinraza was tested only on children, which has made use in adults somewhat controversial. If BIIB067, which is being tested in adults, is successful, that could bolster the approach, and lead to big returns on the huge investment Biogen made in it.

BIIB067 is next headed into what is the equivalent of a late-stage trial, to see how well it works in individuals with the disease, and Biogen isn’t making a timeline public.

Biogen is also partnering with Ionis on another ALS therapy, BIIB078, and bought a number of products, including an ALS drug candidate, from Karyopharm in an up to $217 million deal early this year.

There’s also a muscle-strengthening agent that Biogen bought earlier this year, which could have potential in ALS patients as well as those with spinal muscular atrophy.

Some patients have taken Spinraza and "they're doing well, but not perfectly," Henderson said. "If we can add to that muscle strength...we can then come to a more complete treatment."

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