Company: Nightstar Therapeutics Limited
Symbol: NITE
Description: They are a leading clinical-stage gene therapy company focused on developing and commercializing novel one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness.
Shares: 5.36 million ADSs
Range: $13.00-$15.00
Trade Date: 9/28
Underwriter(s): Jefferies, Leerink Partners, BMO Capital Markets
Co-Manager(s): Wedbush PacGrow, Chardan
Business: Leveraging their expertise in ophthalmology, gene therapy and drug development, they are developing a pipeline of proprietary product candidates that are designed to substantially modify or halt the progression of inherited retinal diseases for which there are no currently approved treatments. Their lead product candidate, NSR-REP1, for the treatment of choroideremia, or CHM, is entering Phase 3 clinical development in the first half of 2018 and represents the most clinically advanced product candidate for this indication worldwide. In data from 32 patients treated with NSR-REP1 across four open-label clinical trials, over 90% of treated patients maintained their visual acuity over a one-year follow-up period. In some cases, they also observed substantial improvements in visual acuity. They are also conducting a Phase 1/2 clinical trial with theirsecond product candidate, NSR-RPGR, for the treatment of X-linked retinitis pigmentosa, or XLRP. Their third product candidate, NSR-BEST1, is in preclinical development for the treatment of Best vitelliform macular dystrophy, or Best disease.
Competition and Sector Performance: Healthcare Related IPOs as of 9.21.17pm
Insider Buying: Certain of their existing stockholders have indicated an interest in purchasing up to an aggregate of approximately $25 million of shares of their common stock.
Collaborations & License Agreement:
Oxford University Innovation Limited – In November 2013, they entered into a license agreement with Oxford. Under the 2013 Oxford Agreement, they acquired an exclusive worldwide license, with the right to grant sublicenses, to develop, research, make, import, use, sell, lease, license or otherwise commercially exploit products, including NSR-REP1, covered by the patents and know-how relating to vectors for use in gene therapy for CHM licensed under the 2013 Oxford Agreement for any and all uses.
Additionally, in November 2015, they entered into five separate license agreements with Oxford, under which they acquired exclusive worldwide licenses, with the right to grant sublicenses, to develop, research, make, import, use, sell, lease, license or otherwise commercially exploit products, including NSR-RPGR, NSR-BEST1 and three other preclinical gene therapy product candidates, covered by the licensed patents and know-how relating to gene therapy for any and all uses.
Oxford BioMedica (UK) Limited — In December 2013, they entered into a license agreement with Oxford BioMedica (UK) Limited, or BioMedica. Under the BioMedica Agreement, they acquired a non-exclusive license in the United States to manufacture, use, supply, sell, offer to sell, store, develop, research and import certain AAV products for the treatment of CHM, with the right to grant sublicenses to development partners.
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