Biogen targets world-leading gene therapies in AU$1.14 billion acquisition of Nightstar

Under terms of the proposed takeover, US based Biogen will pay US$25.50 (AU$36.21) in cash for each NST share, representing a total transaction value of approximately US$800 million (AU$1.14 billion).If the deal were to proceed, UK-based NTS – a gene therapy spin-out company established by the University of Oxford and Syncona – will add two separate gene therapies for inherited retinal disease to Biohen’s portfolio.According to Biogen, the clinical programs for choroideria (CHM) and X-linked retinitis pigmentosa (XLRP) are potentially first-in-class, mid to late-stage clinical assets, while NTS possesses other preclinical programs.Biogen CEO Mr Michel Vounatsos said the acquisition would expand the therapy portfolio of the company, which largely focuses on neurological and neurodegenerative diseases.“Ophthalmology is an erging growth area for Biogen, and we are excited about the opportunity to work with the talented ployees at Nightstar to advance potentially transformative gene therapy programs for rare retinal diseases,” he said.“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 complentary modalities. NST’s lead asset is NSR-REP1 gene therapy for the treatment of CHM. Data from the Phase 1/2 trial of the clinical program donstrated potentially meaningful slowing of decline in visual acuity as compared to natural history, as well as signs of improved visual acuity in some patients.NST’s second clinical program is NSR-RPGR for the treatment of XLRP, which is a rare inherited retinal disease that primarily affecting males, and has no approved treatments. Phase 1/2 data from the dose escalation portion of the XIRIUS trial donstrated an increase in central retinal sensitivity.NST’s preclinical pipeline also includes therapies for Stargardt disease and potential programs targeting best vitelliform macular dystrophy and other genetic forms of retinitis pigmentosa.Biogen expects to complete the acquisition by mid-2019.