Biogen finalises acquisition of gene therapy company Nightstar Therapeutics

Biogen announced Friday that it has completed its $800-million purchase of Nightstar Therapeutics, which focuses on adeno-associated virus (AAV) treatments for inherited retinal disorders. The acquisition gives Biogen access to Nightstar's gene-therapy candidate NSR-REP1 for the treatment of choroideremia, as well as its experimental NSR-RPGR therapy for X-linked retinitis pigmentosa.

Biogen CEO Michel Vounatsos said the transaction "further bolsters our pipeline and is an important step forward toward our goal of a multi-franchise portfolio across complementary modalities." He added "we look forward to working now as one Biogen team with the goal of bringing breakthrough therapies to patients to slow or halt blindness across a range of inherited retinal diseases."

Nightstar initiated the Phase III STAR trial of NSR-REP1 in patients with choroideremia last year. At the time, the company cited data from 32 patients treated with NSR-REP1 across four Phase I/II studies showing that over 90 percent of treated participants had maintained or improved their visual acuity over a one-year follow-up. NSR-REP1 is designated a regenerative medicine advanced therapy by the FDA for this indication.

Nightstar has also unveiled preliminary data from the Phase I/II XIRIUS study illustrating that NSR-RGPR improved visual function as measured by microperimetry in patients with X-linked retinitis pigmentosa. The company later announced plans to initiate a Phase II/III expansion trial of the therapy.

Meanwhile, Biogen recently terminated its collaboration with Applied Genetic Technologies after data from a Phase I/II trial of rAAV2tYF-CB-hRS1, an investigational AAV-based gene therapy for X-linked retinoschisis due to mutations in the RS1 gene, showed no signs of clinical activity at six months.

Other recent gene therapy-focused acquisitions include Roche's deal to purchase Spark Therapeutics for around $4.3 billion, gaining the latter's investigational haemophilia A treatment SPK-8011. More recently, Vertex entered into an agreement to acquire Exonics Therapeutics for as much as about $1 billion, as well as a separate accord expanding its partnership with CRISPR Therapeutics.

For related analysis, see ViewPoints: If you can't build it, buy it – Biogen looks elsewhere to augment gene therapy effort. See also ViewPoints: ASGCT 19 Preview- what to watch in gene and cell therapy.  

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