Clinical Trials

people in lab with woman in the foreground looking through microscope

VISTA Trial

We are now pre-screening for a phase 2/3 clinical trial for X-Linked Retinitis Pigmentosa. Learn more about the clinical trial or how to qualify for no-cost genetic testing and/or prescreening.

Clinical Trials

Name
VISTA
Asset
AGTC-501
Disease
X-Linked Retinitis Pigmentosa (XLRP)
Phase
2/3

VISTA is a planned randomized, controlled, masked, multi-center pivotal study evaluating the efficacy, safety, and tolerability of 2 doses of AGTC-501 compared to an untreated control group.

With the VISTA study, Beacon will use the data generated from the VISTA trial, in combination with data from the Phase 1/2 HORIZON and Phase 2 SKYLINE studies, including all available long-term data, to support a BLA in the US and a MAA in Europe for the treatment of patients with XLRP.

Name
DAWN
Asset
AGTC-501
Disease
X-Linked Retinitis Pigmentosa (XLRP)
Phase
2

DAWN is a non-randomized, open-label study of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV-vector based gene therapy targeting RPGR protein. The purpose of Dawn is to assess 2 different doses of the study drug for efficacy, safety, and tolerability in a participant’s eye that was not previously treated (fellow eye) for XLRP.

Clinical Trials – Enrollment Completed

Name
SKYLINE
Asset
AGTC-501
Disease
X-Linked Retinitis Pigmentosa (XLRP)
Phase
2

The HORIZON protocol was expanded to add a randomized, masked, multi-center, Phase 2 trial (SKYLINE) comparing two doses of AGTC-501.

SKYLINE is an ongoing clinical trial which has completed enrollment of 14 male participants who received gene therapy in one eye to further assess safety and potential efficacy of AGTC-501 gene therapy. AGTC- 501 has demonstrated positive and clinically important and significant improvement in retinal sensitivity and a clear differentiation between dose groups were demonstrated at month 12. An efficacious dose is evident. AGTC-501 is generally safe and well tolerated.

The benefit: risk profile is favorable and supports continued clinical development for the treatment of patients with XLRP.

Name
HORIZON
Asset
AGTC-501
Disease
X-Linked Retinitis Pigmentosa (XLRP)
Phase
1/2

HORIZON in an ongoing Phase 1/2 clinical trial which has completed enrollment of 29 male participants and all participants are in long-term follow-up.

Participants received a single dose of AGTC-501 in one eye to obtain initial data about safety and potential efficacy of AGTC-501 gene therapy. The 12-month post treatment data results demonstrate AGTC-501 is generally safe and well tolerated.

Name
CLARITY
Asset
AGTC-401
AGTC-402
Disease
Achromatopsia
Phase
1/2

Achromatopsia (ACHM) is an inherited condition associated with extremely poor visual acuity, caused by mutations in one of several genes, with the two most common being mutations in either the CNGB3 or CNGA3 genes.

AGTC completed enrollment for the Phase 1/2 gene therapy clinical trials for individuals with a mutation in either CNGA3 or CNGB3 genes. Beacon Therapeutics is not continuing the development of either achromatopsia product candidates at this time. We will continue to follow these patients for the full course of the trial, which includes four years of follow-up.

For further information please contact us at info@beacontx.com