viewApplied Genetic Technologies Corp

Applied Genetic’s gene therapy fails to show effect in XLRS study

Lack of progress prompted Biogen Inc to terminate its collaboration agreement with Applied Genetic in the XLRS study and three other discovery programs

A woman undergoing an eye examination
The Florida biotech uses a proprietary gene therapy platform to develop genetic therapies for patients with rare diseases and its initial focus is in ophthalmology

Shares in Applied Genetic Technologies Corp (NASDAQ:AGTC) tumbled Thursday in premarket trade after a Phase 1/2 clinical trial assessing gene therapy rAAV2tYF-CB-hRS1 in patients with an inherited retinal disorder called X-linked retinoschisis (XLRS) showed underwhelming results.  

The company said that six-month data from the XLRS study supported “general safety and tolerability” of Applied Genetic’s gene delivery platform, but “did not demonstrate signs of clinical activity.”

READ: AGTC granted FDA orphan drug designation for gene therapy to treat achromatopsia

Shares in the Alachua, Florida-based biotech plunged 39.8% to $3.95 before the opening bell.

In more bad news, lack of progress prompted Biogen Inc’s (NASDAQ:BIIB) to terminate its collaboration agreement with Applied Genetic Technologies in the XLRS study, the X-linked retinitis pigmentosa (XLRP) programs and three other discovery programs.

The company said it will complete patient monitoring activities on the XLRS program according to the clinical protocol but will not further develop the product. However, it plans to advance the XLRP program, and will “determine next steps” for the three discovery programs over the next several months.

“The data from the XLRS trial support the general safety and tolerability of our gene delivery platform and further reinforce our confidence in our ongoing Phase 1/2 trials in achromatopsia (ACHM) and XLRP,” said Applied Genetic Technologies CEO Sue Washer in a statement.

The Florida clinical-stage biotechnology company uses a proprietary gene therapy platform to develop genetic therapies for patients suffering from rare diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trials in X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP) and achromatopsia, a hereditary visual disorder which is characterized by the absence of color vision and decreased vision.


Contact Uttara Choudhury at [email protected]

Follow her on Twitter@UttaraProactive 

Quick facts: Applied Genetic Technologies Corp

Price: 5.22 USD

Market: NASDAQ
Market Cap: $134.5 m

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