Avalanche eye therapy
meets safety goal; vision improvement modest
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[June 16, 2015]
By Ransdell Pierson and Bill Berkrot
(Reuters) - Avalanche Biotechnologies Inc's
experimental gene therapy for a leading cause of blindness met its
primary safety goal in a small mid-stage trial, while staving off vision
loss.
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The product, called AVA-101, was well tolerated and was not
associated with any serious adverse events in the 12-month study
involving patients with the age-related "wet" form of macular
degeneration, the company said.
Shares of Avalanche initially jumped more than 25 percent in after
hours trading. However, the modest vision improvement seen with
AVA-101 in the study may have been disappointing to investors, and
the stock changed course, falling 35 percent.
The medicine is injected into the lower retina, instead of into the
vitreous fluid, the mode of delivery used for existing treatments
such as Roche Holding AG's Lucentis and Regeneron Inc's Eylea.
Like those treatments, the Avalanche product is meant to stem the
proliferation of blood vessels in the eye that leak and damage
vision.
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All subjects in the 32-patient study received prior treatment with
Lucentis (ranibizumab). Those who then received the Avalanche drug
on average gained 2.2 letters on an eye chart, while those who did
not saw their vision decrease by 9.3 letters.
While the 11.5-letter difference was deemed to be significant,
patients in earlier studies of Eylea and Lucentis tended to
experience greater vision improvement.
A larger percentage of patients who received AVA-101 maintained
stable vision, while more patients in the control group required
rescue therapy with Lucentis, the company said.
The treatments work by blocking a protein called VEGF. Avalanche's
AVA-101 inserts a gene that creates an anti-VEGF protein and keeps
producing it, while Lucentis and Eylea are injections of an anti-VEGF
protein itself. AVA-101 need only be given once, unlike Lucentis and
Eylea, which require frequent injections.
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Avalanche and Regeneron last year forged an agreement under which
Regeneron has a time-limited right of first negotiation to certain
rights to AVA-101.
"Now that we have Phase II data, we will start the process of
delivering that data to Regeneron," Thomas Chalberg, Avalanche's
chief executive, said in an interview.
Chalberg said his company has not yet decided whether to partner the
drug. "But we think if we do partner, Regeneron would be a great
one."
Avalanche shares fell to $25.20 in extended trading from a Nasdaq
close at $38.88.
(Reporting by Ransdell Pierson and Bill Berkrot; Editing by Steve
Orlofsky and Diane Craft)
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