The failure of a gene therapy trial has led to the termination of a three-year-old partnership between one of the largest biotechnology companies and the therapy’s developer.
Gainesville, Florida-based Applied Genetic Technologies Corporation, also known as AGTC, said Wednesday that topline interim data from its Phase I/II study of rAAV2tYF-CB-hRS1 in X-linked retinoschisis, or XLRS, resulting from RS1 gene mutations did not show the therapy to have clinical activity despite being safe and well-tolerated. Consequently, Biogen is terminating the partnership between the two companies, which was announced in July 2015.
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AGTC’s stock, which closed at $6.56 Wednesday, was down 38 percent on the Nasdaq when markets opened Thursday and was down to $3.56 in afternoon trading. Shares of other companies developing viral vector-based gene therapies – including Spark Therapeutics, bluebird bio, BioMarin and Sangamo Therapeutics – also fell Thursday, trading at prices that were down 1 to more than 5 percent from Wednesday. Biogen’s shares were down less than 1 percent.
The partnership involved two AGTC therapies and included a $124 million upfront payment, plus potential milestones and royalties that would have exceeded $1 billion. At the time the partnership was announced, the XLRS therapy was in clinical development – its clinical trial had initiated in May 2015 – while another, for X-linked retinitis pigmentosa, or XLRP, was in preclinical development. The XLRP therapy, rAAV2tYF-GRK1-RPGR, is in an ongoing Phase I/II study enrolling 15 patients that started in April, according to ClinicalTrials.gov.
With Biogen’s termination of the agreement, control of both programs will revert back to AGTC, the company said. “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 I/II trials in achromatopsia and XLRP,” AGTC CEO Sue Washer said in a statement.
According to its pipeline page, the AGTC’s ACHM programs are both in Phase I/II development. Meanwhile, it has another program, for optogenetics, in preparation for clinical development, and another for age-related macular degeneration in preclinical proof of concept. The company is developing gene therapies for rare diseases using adeno-associated viral vectors.
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Meanwhile, Biogen’s pipeline includes Phase III programs in stroke, multiple sclerosis and Alzheimer’s disease.
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