As Biogen awaits an FDA decision on its controversial Alzheimer's disease hopeful aducanumab, the company is setting the stage for an entry into the hot gene therapy field.
Biogen has drawn up plans for a new gene therapy manufacturing facility at its Research Triangle Park campus in North Carolina. The 175,000-square-foot facility will be designed to scale up the manufacturing of the products that are in Biogen's growing gene therapy pipeline. Biogen expects the facility to be up and running by 2023.
The company will invest some $200 million in the new site, with plans to add about 90 new jobs to its current Research Triangle Park workforce of 1,900. The company currently has 20 clinical programs across its North Carolina operations and performs commercial manufacturing at Research Triangle Park for drugs such as Tysabri, Avonex and Plegridy, a Biogen spokesperson said via email.
The expansion comes as Biogen searches for revenue sources to pick up the slack from declining sales of its spinal muscular atrophy blockbuster Spinraza and multiple sclerosis drug Tecfidera. The company is expecting the FDA to rule on aducanumab in June, but it will most definitely need more blockbuster prospects.
Biogen does have one late-stage gene therapy in its pipeline, BIIB111 (timrepigene emparvovec) to treat the inherited retinal disease choroideremia. It also boasts a mid-stage ophthalmology candidate in BIIB112, designed to treat X-linked retinitis pigmentosa. But its recent investments in the field indicate much bigger ambitions.
In March 2019, Biogen laid out $877 million to acquire Nightstar Therapeutics, which is developing gene therapy treatments for choroideremia and X-linked retinitis pigmentosa. Prior to that, the company dove into gene therapies to treat eye diseases like choroideremia and XLRP when it paid $124 million to partner with Applied Genetics Technologies.
There have been some bumps along the way, to be sure. Biogen ultimately pulled out of the Applied Genentics deal after a clinical setback in 2018. And in October, it quietly pulled the plug on spinal muscular atrophy gene therapy hopeful BIIB089, which had been placed on an IND hold due to toxicity concerns.
But Biogen has continued to invest in the gene therapy field. In July, for example, the company teamed up with Harvard's Massachusetts Eye and Ear to develop a gene therapy for certain patients with blinding diseases such as retinitis pigmentosa. The company has also entered new pacts with Atalanta and ViGeneron to fuel its gene therapy ambitions, Biogen's spokesperson said.
A win in the hot gene therapy field could significantly boost Biogen's top line, as its other business areas start to sputter. Fourth-quarter Spinraza sales fell 34% in the U.S., landing at $160 million, despite a 13% increase in overseas sales. The drug generated $2.05 billion in 2020, down slightly from the previous year.
Meanwhile, generic competition caused sales of Biogen's multiple sclerosis blockbuster Tecfidera to fall 13% to $3.84 billion last year. All told, the company recorded $13.45 billion in sales for the year, down 6% from 2019.
Biogen is currently all-in on aducanumab, which it sees as a “catalyst” for near-term growth, CFO Michael McDonnell said on an analyst call in February. But the company has high hopes for the rest of its pipeline, too, with four phase 3 readouts and four phase 2 readouts expected later this year.
Editor's note: This story has been updated with additional comments from Biogen.