UPDATE : Monday, June 1, 2020
Biogen seeks gene therapy pipeline for rare eye disease
  • By Kim Yun-mi
  • Published 2019.03.06 14:41
  • Updated 2019.03.06 14:41
  • comments 0

The biotech industry is paying attention to whether Biogen’s recent acquisition of Nightstar Therapeutics will make up for the failed collaboration with other biotech firms for gene therapies to treat rare eye diseases.

On Monday, Biogen announced that it would buy the London-based gene therapy firm Nightstar for $877 million.

Nightstar’s research focuses on adeno-associated virus (AAV) in inherited retinal disorders. The company’s NSR-REP1 is the lead candidate product for the treatment of choroideremia (CHM), a rare retinal disorder.

CHM primarily affects males. The rare disease causes a gradual loss of vision due to the degeneration of the photoreceptors in the pigmented layer of the retina.

Nightstar is conducting a phase-3 study on NSR-REP1 and expecting results to come out in the second half of 2020.

The company is also developing NSR-RPGRNST, a candidate treatment for X-linked retinitis pigmentosa, in a phase-2/3 trial.

According to sources, Biogen will pay $25.5 per share of Nightstar, which is 82 percent higher than the price of Nightstar’s offering price but 14 percent lower than the Nightstar’s peak price six months ago.

The 82 percent premium was not very impressive, and Nightstar did not even come within the top 10 list of takeout of biotechs for the past five years, sources said.

Roche acquired Spark on Feb. 25 for $4.8 billion, which was 398 percent above the Spark’s IPO price. Roche’s takeover put Spark on the seventh in the ranking of premium to IPO price in the past five years.

Announcing the buyout, Biogen declared that ophthalmology was a new growth area for the biotech firm. The company said it would continue to enhance pipelines in neuroscience.

“Nightstar would accelerate our entry into ophthalmology by contributing two mid- to late-stage gene therapy assets,” Biogen CEO Michel Vounatsos said.

It was not the first time that Biogen tried to expand gene therapy pipelines for the treatment of rare eye diseases.

The company collaborated with AGTC in ophthalmology in 2015, and with Regenxbio in 2016, to develop AAV vectors for the treatment of X-linked retinitis pigmentosa. However, the tie-up with AGTC ended in December after a clinical trial failure.


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