Biogen Expands Into Gene Therapy for Eye Disease
04 Mars 2019 - 1:12PM
Dow Jones News
By Denise Roland
Biogen Inc. has agreed to buy British gene-therapy company
Nightstar Therapeutics PLC, the latest deal by a big drugmaker in
this cutting-edge area of medicine.
The all-cash deal, which values Nightstar at around $877
million, hands Biogen a gene therapy for choroideremia, a rare,
inherited eye disease that leads to blindness. The treatment is
undergoing late-stage clinical trials. Nightstar, which was founded
in 2013 based on research at the University of Oxford, also has
several earlier-stage gene therapies in its pipeline, all aimed at
rare inherited eye disorders.
Biogen Chief Executive Michel Vounatsos said the deal would
accelerate the company's entry into the "emerging growth area" of
eye diseases. It also marks a quick return into gene therapy after
Biogen ended an earlier collaboration in the area following a
failed clinical trial. That partnership, with Applied Genetic
Technologies Corporation, also centered on the development of gene
therapies for inherited eye disorders. It ended after AGTC said its
therapy wasn't effective.
Gene therapies are eye-catching for their apparent ability to
cure otherwise devastating illnesses with a single treatment. They
typically target diseases caused by a single faulty gene and work
by introducing the working version of that gene into the body using
an inactive virus.
This area of medicine is now gaining momentum after a cautious
start. The first gene therapies were tested in humans as early as
1990 but suffered a setback when, around 10 years later, several of
those patients died. Dozens of gene therapies are now undergoing
clinical trials, and large drugmakers are starting to make big bets
on such treatments.
Last week, Roche Holding AG agreed to pay $4.8 billion to
acquire Spark Therapeutics Inc., whose first product, targeting
another rare eye disorder, has been on sale in the U.S. for about a
year.
Novartis AG last year snapped up AveXis Inc. for $8.7 billion.
AveXis's leading product, for a fatal infant muscle-wasting disease
called spinal muscular atrophy, is expected to get the nod from
U.S. regulators later this year.
Still, questions remain about gene-therapies' commercial
potential. They tend to target conditions with small numbers of
patients. The first gene therapy to launch in Europe, which
targeted a rare eye disorder, was pulled from the market after low
demand. And while many appear to cure previously intractable
diseases, their novelty means there isn't yet conclusive evidence
that those effects don't wane over time.
Another complication: gene therapies typically come with big
price tags. Insurers aren't set up to pay high upfront costs for
medical therapies, even if that expenditure is lower than the total
sum they would pay for a less effective long-term treatment.
Spark's therapy, called Luxturna, costs $850,000 to treat both
eyes
With more gene therapies poised to enter the market in the next
few years, drug companies and insurers are starting to explore new
payment models, such as paying in installments.
Write to Denise Roland at Denise.Roland@wsj.com
(END) Dow Jones Newswires
March 04, 2019 06:57 ET (11:57 GMT)
Copyright (c) 2019 Dow Jones & Company, Inc.
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