Deals, Setbacks

Biogen shrugs off $1B-plus gene therapy pact after PhI/II implosion — AGTC shares crater

More than 3 years after Biogen $BIIB fronted their $1 billion-plus pact with Florida-based biotech AGTC with $124 million in cash to build their new gene therapy unit, the Boston-based biotech is bailing on the pact involving rare eye diseases.

Sue Washer

Applied Genetic Technologies Corporation says their Phase I/II trial for rAAV2tYF-CB-hRS1, designed to cure X-linked retinoschisis due to mutations in the RS1 gene, was a flop. There were no signs of clinical activity at 6 months.

Once Biogen wraps up a few obligations by March 8, the execs will wipe their hands clean of the alliance and AGTC is dropping the program in the wake of the implosion.

Shares of the smaller company $AGTC plunged 44% Thursday morning.

The focus at AGTC will now shift to ongoing Phase I/II trials in achromatopsia and X-linked retinitis pigmentosa.

CEO Sue Washer had this to say about the deal termination:

AGTC will regain full rights to the XLRP Phase 1/2 clinical program, which has completed its dose escalation phase ahead of our previous guidance and is moving directly to an expansion phase in 2019. AGTC’s two ongoing clinical programs in ACHM are proceeding as previously disclosed and the company is expecting data readouts from all three trials in 2019. We will also regain control of the preclinical programs in adrenoleukodystrophy (ALD) and two ophthalmic programs.


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Biotech Investment Analyst
SV Health Investors Boston, MA
Director, Program Management
Contrafect Corporation New York, NY
Director, Translational Sciences
Cadent Therapeutics Cambridge, MA

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