GlaxoSmithKline files for gene therapy OK as a case of the jitters sets in

GSK CEO Andrew Witty

Close to 5 years after GlaxoSmithKline ($GSK) signed on to collaborate with the San Raffaele Telethon Institute for Gene Therapy in Italy, the partners have stepped up with a European application to start marketing a gene therapy for extraordinarily rare cases of immune deficiency triggered by ADA-SCID. And its delivery into regulatory hands comes as the gene therapy field has been recoiling from some notable setbacks that have begun to cloud what has been a bullish sector in biotech.

The drug is the rarely mentioned GSK2696273, a gene therapy which uses a viral vector to insert working copies of the ADA gene into stem cells extracted from the bone marrow of patients. The cells are then reintroduced to the patient, who can expect to start making the gene on their own, repairing their immune system.

The Europeans will be reviewing an application built on data from 18 patients, with the first treatment dating back 13 years. All are alive, though three had to have follow-up enzyme replacement therapy or a bone marrow transplant, which is what most of these patients rely on today.

An approval for GlaxoSmithKline in Europe would have wide implications. With so few patients, the center in Italy may well become a Mecca for patients around the world, including the U.S.

"This is an important landmark for clinicians, researchers and all the staff at TIGET who have been working side by side with GSK towards approval of this gene therapy," says Alessandro Aiuti, the clinical research coordinator at TIGET. "In the past years we have witnessed how a single infusion of gene modified stem cells has changed the lives of these children and their families. If authorized, we will be ready to offer gene therapy at our center to ADA-SCID patients in need from Europe and other countries."

For GlaxoSmithKline, the application marks a rare bright spot in what has been a bleak period for the pharma giant. Struggling to restore some luster to its revenue numbers, analysts have become increasingly testy about CEO Andrew Witty's slowly evolving game plan for a turnaround. The move also highlights the growing interest in gene therapy even as investors are getting rattled by evidence of the risk of failure and lingering questions about how these therapies--expected to cost in the $1 million range--will be paid for.

Just yesterday Spark Therapeutics ($ONCE) had to beat off an assault on its share price after another, unrelated gene therapy designed to help the blind see by restoring functional copies of the RPE65 gene--what their lead therapy is supposed to do--began to fail within a few years. Spark's share price ended down 6% for the day, even after its CEO asserted that their gene therapy was of a distinctly different design. And just a few days ago the share price at Celladon ($CLDN) was eviscerated by the news that its gene therapy for heart disease had failed a crucial study, raising doubts about their whole approach.

Even if others in the field can complete small, successful registration studies of their own, these setbacks are raising fears that payers will be expected to cover a huge upfront cost for a drug that may have no long-term track record in the clinic. And without evidence of a durable cure, the pressure will grow on finding new ways to pay for these treatments over the long term.

Investors and big partners have been turning to gene therapies in growing numbers as biotechs like bluebird bio ($BLUE) under Nick Leschly have raised hopes that the field has finally arrived, ready to deliver new therapies that are safe and effective. That newfound faith inspired a move by Biogen ($BIIB) to get into the field, starting its own unit, while Bristol-Myers Squibb ($BMY) struck a $1 billion deal with uniQure ($QURE) on a cardiovascular program just a few weeks ago.

Faith is a fragile commodity in biotech, much easier to break than it is to build. And this is one arena in drug R&D where the fate of all the players seems more closely connected than the norm.

- here's the release

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