Applied Genetic Technologies Corporation (AGTC) Treats First Patient of Second Cohort in Phase 1/2 Clinical XLRP Study


AGTC Treats First Patient of Second Cohort in Phase 1/2 Clinical XLRP Study; Earns $10 Million Enrollment Milestone

Applied Genetic Technologies Corporation (AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that it has enrolled the first patient of the second cohort in the company’s Phase 1/2 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.

“We are pleased to announce this important milestone under our collaboration with Biogen and remain on track to complete the dose escalation portion of the trial in the first quarter of 2019,” said Sue Washer, President and CEO of AGTC.

The Phase 1/2 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.

Ms. Washer continued, “In addition to achieving this milestone for our XLRP study, we are improving execution through enhanced clinical operations, clinical outreach and patient advocacy efforts to support enrollment of our three additional clinical trials. We remain grateful to our team, the investigators and the patients who have volunteered for these studies for their commitment to accelerating the development of gene therapies for inherited retinal diseases.”

In addition to the XLRP study, the company has ongoing clinical trials in X-linked retinoschisis (XLRS), and achromatopsia (ACHM).

XLRS Phase 1/2 Clinical Trial
Earlier this year, AGTC completed its target enrollment of 27 patients in the Phase 1/2 clinical trial for its XLRS product candidate as part of the company’s collaboration with Biogen. The primary endpoint of this clinical trial is safety, and available data thus far have shown that the XLRS product candidate is generally safe and well tolerated.  In addition to safety, this trial will measure biologic activity by assessing changes in visual function, retinal structure and quality of life.  The company expects to provide topline six-month data across both safety and biologic activity endpoints by the end of 2018 with the primary analysis of the full twelve-month active trial data six months later.

ACHM Phase 1/2 Clinical Trials
The company is presently enrolling patients in two parallel Phase 1/2 clinical trials of its product candidates for achromatopsia (ACHM) caused by mutations in the two most common ACHM genes, CNGB3 and CNGA3. In the ACHM CNGB3 trial, AGTC has completed enrollment of seven patients. The company expects to complete the dose escalation portion of the CNGB3 trial in the first quarter of 2019.  The ACHM CNGA3 trial has enrolled and dosed two patients.

For more information on AGTC and its pipeline of AAV-based gene therapy candidates in rare disease, please visit or  contact [email protected]

About X-linked Retinitis Pigmentosa (XLRP)

XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life.  AGTC was granted U.S. Food and Drug (FDA) orphan drug designation in 2017, as well as European Commission orphan medicinal product designation in 2016, for its gene therapy product candidate to treat XLRP caused by mutations in the RPGR gene.

About Achromatopsia (ACHM)
Achromatopsia is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by achromatopsia, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind.


Please enter your comment!
Please enter your name here