Allergan and Editas Medicine Initiate the Brilliance Phase 1/2 Clinical Trial of AGN-151587 (EDIT-101) for the Treatment of LCA10

Expect first patient dosing in second half of 2019

DUBLIN, Ireland and CAMBRIDGE, MA, USA I July 25, 2019 I Allergan plc (NYSE: AGN), a leading global pharmaceutical company, and Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced the Brilliance Phase 1/2 clinical trial of AGN-151587 (EDIT-101) is open for patient enrollment. AGN-151587 is an experimental medicine under development for the treatment of Leber congenital amaurosis 10 (LCA10), an inherited form of blindness caused by mutations in the CEP290 gene. The clinical trial will be the world’s first in vivo study of a CRISPR-based genome editing medicine, where the editing takes place inside the human body.

“We are very proud of our continued commitment to developing innovative treatments for unmet needs in eye care. Beginning patient enrollment in the AGN-151587 clinical trial with our partners at Editas is an important step toward our goal of developing a game-changing, transformative, CRISPR-based medicine for people with LCA10,” said David Nicholson, Ph.D., Chief Research and Development Officer, Allergan.

The Brilliance clinical trial is a Phase 1/2 study to evaluate AGN-151587 for the treatment of LCA10. The study will assess safety, tolerability, and efficacy in approximately 18 patients. Up to five cohorts across three dose levels will be enrolled in this open label clinical trial in multiple centers. Both adult and pediatric patients (3 – 17 years old) with a range of vision will be evaluated. Patients will receive a single dose of AGN-151587 administered via subretinal injection in one eye following vitrectomy. Additional details are available on (NCT#03872479).

“Now that enrollment is underway, we are one step closer to delivering a transformative medicine to LCA10 patients,” said Charles Albright, Ph.D., Chief Scientific Officer, Editas Medicine. “The team at Editas looks forward to continuing to collaborate with our partners at Allergan, patient advocacy organizations, and the inherited retinal diseases community as we develop this and other durable experimental medicines for patients with devastating ocular diseases.”

Sites in the U.S. are currently enrolling patients for the trial, including Massachusetts Eye and Ear, an international center for treatment and research and a teaching hospital of Harvard Medical School.

“Today marks an important day for the inherited retinal disease community, and specifically those affected by LCA10. We are very excited to have another potentially life changing medicine enter the clinic and join Allergan and Editas in celebrating this milestone,” said Ben Yerxa, Ph.D., CEO, Foundation Fighting Blindness.

About AGN-151587 (EDIT-101)
AGN-151587 (EDIT-101) is a CRISPR-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10). AGN-151587 is administered via a subretinal injection to reach and deliver the gene editing treatment directly to photoreceptor cells.

About Leber Congenital Amaurosis
Leber congenital amaurosis, or LCA, is a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes. It is the most common cause of inherited childhood blindness, with an incidence of two to three per 100,000 live births worldwide. Symptoms of LCA appear within the first years of life, resulting in significant vision loss and potentially blindness. The most common form of the disease, LCA10, is a monogenic disorder caused by mutations in the CEP290 gene and is the cause of disease in approximately 20‑30 percent of all LCA patients.

About the Editas Medicine-Allergan Alliance
In March 2017, Editas Medicine and Allergan Pharmaceuticals International Limited (Allergan) entered a strategic alliance and option agreement under which Allergan received exclusive access and the option to license up to five of Editas Medicine’s genome editing programs for ocular diseases, including AGN-151587 (EDIT-101). Under the terms of the agreement, Allergan is responsible for development and commercialization of optioned products, subject to Editas Medicine’s option to co-develop and share equally in the profits and losses of two optioned products in the United States. In August 2018, Allergan exercised its option to develop and commercialize AGN-151587 globally for the treatment of LCA10. Additionally, Editas Medicine exercised its option to co-develop and share equally in the profits and losses from AGN-151587 in the United States. Editas Medicine is also eligible to receive development and commercial milestones, as well as royalty payments on a per-program basis. The agreement covers a range of first-in-class ocular programs targeting serious, vision-threatening diseases based on Editas Medicine’s unparalleled CRISPR genome editing platform, including CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a). 

About Allergan plc
Allergan plc (NYSE: AGN), headquartered in Dublin, Ireland, is a global pharmaceutical leader focused on developing, manufacturing and commercializing branded pharmaceutical, device, biologic, surgical and regenerative medicine products for patients around the world. Allergan markets a portfolio of leading brands and best-in-class products primarily focused on four key therapeutic areas including medical aesthetics, eye care, central nervous system and gastroenterology. As part of its approach to delivering innovation for better patient care, Allergan has built one of the broadest pharmaceutical and device research and development pipelines in the industry.

With colleagues and commercial operations located in approximately 100 countries, Allergan is committed to working with physicians, healthcare providers and patients to deliver innovative and meaningful treatments that help people around the world live longer, healthier lives every day.

For more information, visit Allergan's website at

About Editas Medicine
As a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit  

SOURCE: Editas Medicine

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