Biogen to acquire UK gene therapy company Nightstar Therapeutics for $800m

Biogen acquisition of Nightstar Therapeutics : US biotech company Biogen has signed a deal of $800 million to acquire Nightstar Therapeutics, a British clinical-stage gene therapy company, as per the latest pharma acquisition news.

Nightstar Therapeutics primarily focuses on the developing adeno-associated virus (AAV) treatments for inherited retinal disorders.

Biogen acquisition of Nightstar Therapeutics

According to the deal terms, Biogen offers to pay $25.50 in cash for each share of the gene therapy company.

NSR-REP1 is the lead candidate of Nightstar Therapeutics and is presently being evaluated in the phase 3 STAR trial as a treatment of Choroideremia (CHM) – a rare degenerative and X-linked inherited retinal disease, which results in blindness and presently has no approved treatments. The findings of the late-stage trial are slated to be disclosed in H2 2020.

Biogen acquisition of Nightstar Therapeutics
Biogen acquisition of Nightstar Therapeutics. Image courtesy of 02
Svilen Milev
Svilen Milev/

Choroideremia results from the loss of function in the CHM gene that encodes the Rab escort protein-1 (REP-1) and the inherited retinal disease primarily affects males.

NSR-REP1 consists of an AAV vector, which is administered through a subretinal injection, that provides functioning CHM gene and thus the expression of the REP-1 protein, which is important for restoring photoreceptor function and eventually in reversing the decline in eyesight.

Phase 1/2 findings from the dose escalation part of the XIRIUS trial for NSR-RPGR showed a surge in central retinal sensitivity. On the other hand, a phase 2/3 dose expansion portion of the XIRIUS trial is currently in progress.

The preclinical pipeline of Nightstar Therapeutics also has NSR-ABCA4, which is being developed to address Stargardt disease, along with potential programs targeting Best vitelliform macular dystrophy (Best disease) and other genetic types of retinitis pigmentosa.

Michel Vounatsos – Biogen CEO, commenting on Biogen acquisition of Nightstar Therapeutics, said: “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.

“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.”

Biogen acquisition of Nightstar Therapeutics is anticipated to be wrapped up in the middle of this year, subject to regulatory approvals and meeting of other conditions.

Commenting on Biogen acquisition of Nightstar Therapeutics, David Fellows – CEO of Nightstar Therapeutics, said: “Our agreement with Biogen will give us the platform and resources to expand our mission to maintain and restore sight in patients with inherited retinal diseases. This transaction accelerates treatment to patients through Nightstar’s key retinal gene therapy programs that modify or halt progression of blindness.

“Together, with Biogen’s expertise in rare diseases, worldwide reach and extensive resources, we will dramatically improve the lives of patients around the world who currently have no treatment options. We are proud of what Nightstar has accomplished, and we thank our team for their tireless work to improve the lives of our patients and their families.”

Related posts