- Biogen won't be taking on a heavy manufacturing lift with its proposed acquisition of Nightstar Therapeutics, a potential upside for the buyer given the often arduous process involved in producing gene therapies.
- Nightstar is developing gene therapies for severe retinal diseases based on its adeno-associated virus technology. "A relatively low viral load is needed to treat the targeted retinal tissue in the eye, resulting in more facile manufacturing at the required scale," Biogen's head of R&D Michael Ehlers said on a Monday investor call discussing the deal.
- Ehlers noted too that Nightstar outsources its Good Manufacturing Practice needs — including those related to testing, storage and distribution — to a global network of qualified partners. "The combination of their networks ... together with our clinical development, commercialization capabilities will readily be able to manufacture and supply patients in the market as needed," he said.
Biogen has its sights set on gene therapies for inherited retinal diseases. After backing away from a collaboration with Florida-based AGTC, the big biotech now plans on dropping roughly $800 million to snag Nightstar.
The target company offers two mid- to late-stage candidates as well as an AAV platform that Biogen intends to keep focused solely on ophthalmology. What Nightstar won't bring, however, is in-house manufacturing capabilities.
But that may not be such a bad thing. Leerink analyst Joseph Schwartz highlighted how gene therapies for the eye use less material and a flat dose compared to those that use sequential dosing and target parts of the body with more tissue.
Biogen "may have been strategic to acquire the technical and clinical expertise within [Nightstar] without paying too much premium" for its own manufacturing capabilities, Schwartz wrote in a March 4 note.
Aside from costs, gene therapy manufacturing also comes with a bevy of logistical and regulatory challenges.
Jeff Marrazzo, CEO of Spark Therapeutics, told BioPharma Dive in January that the vast majority of the roughly 60,000-page Biologics Licensing Application submitted for its now-marketed product Luxturna (voretigene neparvovec) was about CMC — short for chemistry, manufacturing and controls.
"We developed 41 different assays to test and release Luxturna," Marrazzo said. Luxturna, indicated for a certain type of rare eye disorder, secured the first ever U.S. approval for a gene therapy targeting an inherited disease.
Marrazzo noted that, despite a positive collaboration with the Food and Drug Administration, there are still aspects of gene therapy development that need to be more fully ironed out between drug manufacturers and regulators, including CMC.
Still, Biogen does benefit from having Spark establish some of the groundwork for an approval pathway for inherited retinal disease gene therapies.
Biogen estimates that Nightstar's two most advanced therapies could see a market launch in the early 2020s.
Biogen leadership on the March 4 call wouldn't say how much money the company plans to spend on ophthalmology R&D, though an update on spending will come alongside second quarter earnings results. That also happens to be around the time Biogen and Nightstar anticipate their deal to close.
Nightstar recorded $31 million in operating expenses, which included $22 million worth of R&D costs, for the first nine months of 2018.