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Biogen Acquires Gene Therapy Company in $800M Deal

Eye exam

(U.S. Air Force Medical Service)

4 Mar. 2019. A company developing treatments for inherited eye diseases that replace malfunctioning with healthy genes is being acquired by biotechnology company Biogen. The deal for purchasing Nightstar Therapeutics in London by Biogen in Cambridge, Massachusetts is valued at $800 million.

Nightstar Therapeutics is a 5 year-old enterprise that creates therapies to treat disorders in the retina, light-sensitive tissue that lines the back of the eye, caused by genetic mutations. The company licenses research on gene therapy by Oxford ophthalmologist Robert MacLaren, the company’s scientific founder. Nightstar’s gene therapies are delivered with adeno-associated viruses, benign and naturally occurring microbes that can infect cells, but do not integrate with the cell’s genome or cause disease, other than at most mild reactions in humans.

The company’s lead product, code-named NSR-REP1, is designed to treat choroideremia, an inherited progressive disease of the retina, affecting mainly males, for which there is now no cure. Choroideremia usually begins as impaired vision at night, followed by narrowing of the field of vision and loss of visual acuity, or ability to see details. The worsening vision is a result of a breakdown of photoreceptor cells in the retina and nearby blood vessels, followed eventually by full blindness. The Choroideremia Research Foundation estimates the disease affects 1 in 50,000 individuals.

NSR-REP1 is administered to patients as one-time surgical injections into the retina, and is now in a late-stage clinical trial. An early- and mid-stage trial led by MacLaren and reported in October 2018, injected NSR-REP1 into one eye of participants with choroideremia and compared visual acuity in the treated compared to untreated eye. Results show after 2 years, more gains in visual acuity compared to the untreated eye. The therapy was largely safe for patients, although 2 participants suffered complications.

Nightstar’s second therapy candidate in a clinical trial, code-named NSR-RPGR, is a treatment for retinitis pigmentosa, also an inherited disease of the retina. This form of the disorder, known as X-linked retinitis pigmentosa, is caused largely by a recessive mutation in the retinitis pigmentosa GTPase regulator or RPGR gene. The mutation also causes a breakdown in photoreceptor cells leading to blind spots, night blindness, and tunnel vision, progressing to total blindness. As reported by Science & Enterprise in March 2017, the clinical trial is recruiting 24 men with X-linked retinitis pigmentosa at ophthalmology clinics in Oxford and Manchester.

Biogen specializes in neurological disorders, but is also developing gene therapies for inherited eye diseases, including X-linked retinitis pigmentosa. Michel Vounatsos, Biogen’s CEO says in a company statement, “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.”

The agreement calls for Biogen to acquire all Nightstar shares for cash at $25.20, a 66 percent premium over Friday’s closing price of $15.16. A Nightstar statement calculates the acquisition value at $877 million, but Biogen says transaction expenses and anticipated cash at closing will reduce that value somewhat to $800 million. The acquisition is expected to close by mid-2019, to comply with investment laws in the U.K.

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