Biogen has signed another deal to help build its gene therapy presence, this time paying around $800m for UK-based player Nightstar Therapeutics. The London-based company is focused on developing adeno-associated virus (AAV) treatments for inherited retinal disorders (IRDs), one of the ‘low hanging fruits’ in gene therapy as delivery to the eye is relatively straightforward. Biogen gets one late-stage programme with the Nightstar acquisition, namely its NSR-REP1 candidate for X-linked IRD choroideremia (CHM), plus another therapy for X-linked retinitis pigmentosa (XLRP) in phase 1/2. Both CHM and XLRP are sight-robbing conditions that affect males and have no approved treatments, and Biogen says Nightstar’s candidates are potentially first-in-class therapies, despite the high level of interest in gene therapy for IRDs. Following after is a preclinical programme in Stargardt disease caused by mutations in the ABCA4 gene, which can cause blindness in both men and women in their twenties. Biogen is offering $25.50 per share in Nightstar, which is a premium of around two-thirds on its Nasdaq closing price of $15.16 ahead of the weekend. The US biopharma company says it will fund the acquisition with cash on hand. Nightstar has a phase 3 trial ongoing of NSR-REP1, which uses an AAV vector to deliver the REP1 gene during a surgical procedure, and reported encouraging phase 1/2 results with the therapy in the journal Nature Medicine last year. Its phase 1/2 trial in XLRP involves a similar AAV-based therapy that in this case delivers the RPGR gene deficient in this disorder. Last September, Nightstar presented preliminary proof-of-concept data from the trial including dose-related improvements in macula sensitivity. For Biogen, the deal bolsters its position in gene therapy – a category it has been steadily building since around 2014 when it formed an alliance with Sangamo – and also “accelerates our entry into ophthalmology,” according to the company’s CEO Michel Vounatsos (pictured below). Back in 2016, one of Biogen’s biggest plays in gene therapy was a $2bn deal with the University of Pennsylvania for access to gene transfer and gene-editing methods, which came after smaller deals including the licensing of an AAV vector platform from Regenxbio and an investment in Solid Bioscience’s Duchenne Muscular dystrophy programme. A year earlier, it paid $124m upfront and pledged up to $1.1bn in milestones for rights to two of AGTC’s gene-based ophthalmic therapies, but walked away from that agreement late in 2018 after disappointing results in an X-linked retinoschisis (XLRS) trial. The two partners had been working together on an XLRP candidate but AGTC is now progressing that independently. Meanwhile, Biogen’s move follows other recent gene therapy acquisitions in the biopharma sector. That includes Roche’s $4.3bn deal for Spark Therapeutics – which already sells a gene therapy for IRD called Luxturna – and Novartis’ takeover of AveXis for $8.7bn last year to lay claim to a gene therapy for spinal muscular atrophy (SMA) that could threaten Biogen’s own Spinraza (nusinersen) drug treatment. And just last week, Sarepta agreed a $165m takeover deal for Myonexus.
US biotech Biogen has bolstered its gene therapy portfolio by acquiring UK-based Nightstar Therapeutics for around $800m in a cash deal. Biogen will gain Nightstar's adeno-associated virus treatments for inherited retinal disorders (IRD). Nightstar has developed candidates for the blindness-inducing X-linked IRDs choroideremia and retinitis pigmentosa, both of which are potential first-in-class therapies. The acquisition is expected to close later this year.