Biogen to acquire gene therapy company for $800m

Biotechnology company Biogen has announced it will acquire clinical-stage gene therapy company, Nightstar Therapeutics for approximately $800 million.

The acquisition is intended to help Biogen move into the gene therapy space for retinal diseases. The deal will give Biogen access to Nightstar’s portfolio of treatments. This includes its lead asset NSR-REP1, which is designed to treat choroideremia (CHM), a rare, degenerative, X-linked inherited retinal disorder, which leads to blindness and has no approved treatments.

NSR-REP1 is comprised of an adeno-associated virus (AAV) vector which can enable the REP-1 protein to restore membrane trafficking and potentially, slow, stop or reverse the decline in vision.

The treatment is currently being evaluated in a Phase 3 Star trial with data expected sometime in 2020. Previous data show that NSR-REP1 demonstrated a potential slowing of decline in visual acuity as well as improved vision in some patients.

“Ophthalmology is an emerging growth area for Biogen, and we are excited about the opportunity to work with the talented employees at Nightstar to advance potentially transformative gene therapy programs for rare retinal diseases,” said Michel Vounatsos, Biogen’s chief executive officer. “With this proposed acquisition, we are continuing to bolster our pipeline and further execute on our strategy to develop and expand a multi-franchise neuroscience pipeline across complementary modalities. Nightstar would accelerate our entry into ophthalmology by contributing two mid- to late-stage gene therapy assets, with the potential to create long-term shareholder value.”

Nightstar’s pre-clinical pipeline also includes a treatment for Stargardt disease and potential programmes targeting Best disease and other genetic forms of retinitis pigmentosa.

The acquisition is expected to be completed by the middle of 2019.