Drug Development, Gene Therapy

In a PhIII showdown with Spark, uniQure switches out its lead gene therapy for hemophilia B

Late last year, the struggling gene therapy biotech uniQure $QURE did some painful cost cutting, dropping programs, axing staff and tailoring the pipeline to concentrate on a hemophilia B program for AMT-060, which they said could be hustled through a pivotal program and into the market, racing with a rival therapy from industry leader Spark Therapeutics.

Steven Pipe

This morning, though, uniQure executed a quick switch on its lead effort. AMT-060, which had been designated as a breakthrough drug at the FDA, has been replaced by AMT-061. And the execs at uniQure say that both the FDA and the EMA have essentially decided to keep this new drug in their VIP drug program and on the fast track to a registration trial.

Investors loved it. UniQure’s stock shot up more than 60% after the news hit.

The way uniQure makes this sound, researchers tweaked their original therapy to come up with something better. In their statement, the biotech says that AMT-060 and AMT-061 are identical except for “two nucleotide substitutions in the coding sequence for FIX.”

The gene variant, referred to as FIX-Padua, expresses a protein with a single amino acid substitution that has been reported in multiple preclinical and nonclinical studies to provide an approximate 8- to 9-fold increase in FIX activity compared to the wild-type FIX protein. All other critical quality attributes of AMT-061 are expected to be comparable to those of AMT-060, as AMT-061 utilizes the same AAV5 capsid and proprietary insect cell-based manufacturing platform.

They also want to style this new AMT-061 as a much, much better therapy.

Steven Pipe, professor of pediatrics and pathology and pediatric medical director of the hemophilia and coagulation disorders program at the University of Michigan, had this to say:

“Based on the data generated to date, AMT-061 may be the first gene therapy to provide durable, curative benefits to nearly all patients with hemophilia B, without the complications associated with capsid-related immune responses.  I very much look forward to serving as an investigator in this exciting Phase III program.”

Once a leader in gene therapy, uniQure had to drop Glybera — approved in Europe but never pitched in the US — after it was reportedly used just one time. Now Spark appears to be in the lead after an FDA panel endorsed its gene therapy for a rare eye disease. And one-time player Dimension Therapeutics was forced out of hemophilia B by weak results, with Ultragenyx picking up the rest of the pipeline in a buyout.

The race goes on.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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