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Applied Genetic Technologies (AGTC) 1st patient of 2nd cohort in P1/2 clinical XLRP study

July 23, 2018 10:24am

… Which evaluates the safety and efficacy of an AAV-based gene therapy while earning $10 M enrollment milestone

AGTC jumped +$0.30 or +7.41% to $4.35 – a better showing, bumpy share pricing and about time news

2019 is still a lengthy road for data … as enrollments and clinical expertise has been sorely lacking.

Applied Genetic Technologies Corporation (AGTC has enrolled the first patient of the second cohort in the P1/2 clinical trial evaluating the safety and efficacy of an investigational AAV-based gene therapy for the treatment of X-linked retinitis pigmentosa (XLRP). Under the terms of its collaboration with Biogen (BIIB), AGTC will receive a milestone payment of $10 million.

At least a milestone under its collaboration with Biogen (BIIB) – not “chump change” but, won’t complete the dose escalation portion of the trial until Q1/19.

The P1/2 trial is an open-label, dose escalation study designed to assess the safety and efficacy of subretinal administration of the AAV-based gene therapy in patients diagnosed with XLRP caused by mutations in the RPGR gene. The primary focus of the study will be to assess the safety of the vector and subretinal delivery procedure through analysis of focal (ocular) and systemic treatment-emergent adverse events. Potential efficacy will be measured by evaluation of changes in retinal structure, visual function and quality of life.

In addition to the XLRP study, AGTC has ongoing clinical trials in X-linked retinoschisis (XLRS), and achromatopsia (ACHM). Earlier this year, AGTC completed its target enrollment of 27 patients in the P1/2 clinical trial for its XLRS product candidate with the primary endpoint of this clinical trial is safety; this trial will measure biologic activity by assessing changes in visual function, retinal structure and quality of life. Topline six-month data across both safety and biologic activity endpoints could be available by the end of 2018 with the primary analysis of the full twelve-month active trial data six months later.

2019 is still a lengthy road for data … as enrollments and clinical expertise has been sorely lacking.

35 companies, 1 interpreter!

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