Nightstar acquired by Biogen for $800m

By Maggie Lynch contact

- Last updated on GMT

(Image: Getty/gradyreese)
(Image: Getty/gradyreese)

Related tags: Gene therapy, Gene therapy candidate, acquistion, Biogen, Drug delivery, Roche, Novartis

Biogen agrees Nightstar acquisition for $800m to take on its pipeline of AAV treatments for retinal disorders, diversifyingy its pipeline in the rare disease market.

Per the agreement, Biogen will pay the clinical stage, gene therapy company $25.50 for each share – amounting to a total payment of $800m (€706.8m). The acquisition is predicted to be complete by mid-2019.

Nightstar Therapeutics is focused on the development of gene therapy products for retinal disorders and ophthalmology.

Biogen said in a statement that, with the field of ophthalmology growing, it is using this acquisition as an opportunity to expand its stake in the sector and grow its pipeline for retinal treatments​.

“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,”​ Michel Vounatsos, Biogen CEO, said in a statement.

“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,” ​he added.

This proposed deal comes after larger pharma companies have shown an increased appetite for investing in the gene therapy sector of late, as Roche acquired the gene therapy company​, Spark Therapeutics, and. Novartis purchased AveXis​.

Acquired assets

Per the agreement, Biogen will acquire Nightstar’s asset, NSR-REP1, for the treatment of choroideremia (CHM), a rare, degenerative, inherited retinal disorder that is currently without treatment options.

NSR-REP1, an adeno-associated virus (AAV) based treatment is administered by subretinal injection, providing a functioning CHM gene to restore the expression of the REP-1 protein. The lack of a REP-1 protein prevents proper protein trafficking in the eye – leading to abnormal and impaired elimination of waste products from photoreceptors causing the loss of night vision, and eventually complete blindness.

The asset can potentially reverse or slow the decline in vision caused by CHM. The drug candidate is being evaluated in an ongoing Phase III trial, where it was able to slow the decline of visual acuity compared to the natural history of the disease.

Nightstar has another ongoing clinical program that Biogen will acquire – its candidate, NSR-RPGR, for the treatment of pigmentosa, another inherited retinal disease, caused by mutations in the retinitis pigmentosa GTPase regulator gene, resulting in a lack of active protein transport in photoreceptors.

A Phase I/II trial of NSR-RPGR demonstrated an increase in central retinal sensitivity and potential reverse of the decline in vision. A Phase II/III dose expansion of the trial is ongoing. 

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