MeiraGTx Announces AAV-CNGB3 Granted Fast Track Designation by U.S. FDA for Treatment of Achromatopsia

August 20, 2018

LONDON and NEW YORK, Aug. 20, 2018 (GLOBE NEWSWIRE) -- MeiraGTx Holdings Plc (NASDAQ:MGTX), a vertically integrated, clinical stage gene therapy company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its AAV-CNGB3 gene therapy product candidate for the treatment of achromatopsia (ACHM) caused by mutations in the CNGB3 gene.

ACHM is an inherited retinal disease that severely limits a person’s sight by preventing cone photoreceptors in the eye from functioning. Individuals with ACHM are often legally blind from birth, have extreme sensitivity to light, and experience involuntary eye movements. AAV-CNGB3 is an investigational gene therapy treatment designed to restore cone function, delivered to the cone receptors at the back of the eye via subretinal injection.

“We are thrilled by this designation and the continued recognition by the FDA of AAV-CNGB3’s potential to become a much-needed treatment option for ACHM patients suffering from this debilitating disease,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “Fast Track designation is not just a milestone for the company, but also for the ACHM community. With the ability to communicate closely and often with the FDA, we are hopeful that we can expedite our commitment to bring a safe and effective therapy to patients in need.”

AAV-CNGB3 has been granted orphan drug designation (ODD) by the FDA and orphan medicinal product designation by the European Medicines Agency (EMA), as well as rare pediatric disease designation by the FDA and PRIME designation by the EMA, for the treatment of ACHM caused by mutations in the CNGB3 gene. MeiraGTx is currently conducting a Phase 1/2 clinical trial of AAV-CNGB3 in both adult and pediatric patients, with 14 patients treated to date. Eleven adult patients have been treated in three dose escalation cohorts and three pediatric patients have now been treated in the extension phase of the study. The company anticipates completing dosing of up to eight pediatric patients in the second half of 2018.

The FDA’s Fast Track process is designed to expedite the development and review of drugs used to treat serious conditions and fill an unmet medical need. Fast Track designation enables the company to have early and frequent communication with the FDA throughout the drug development and review process, with the potential for faster drug approval and patient access. Today’s announcement marks the second Fast Track designation granted to MeiraGTx in 2018. In April 2018, the company was also granted Fast Track designation for AAV-RPGR for the treatment of another debilitating ocular disorder, X-linked retinitis pigmentosa (XLRP) due to defects in the RPGR gene.

About Achromatopsia
Achromatopsia is an inherited retinal disorder that specifically prevents cone photoreceptors from functioning. ACHM is characterized by severely reduced visual acuity of 20/200 or worse, disabling light sensitivity (photoaversion) and involuntary back and forth eye movements (nystagmus). ACHM occurs in approximately one in 30,000 people in the United States, with 92 percent of cases caused by mutations in CNGB3 and CNGA3 genesCurrently, there are no effective treatments for this disease.

About MeiraGTx
MeiraGTx (NASDAQ:MGTX) is a vertically integrated, clinical stage gene therapy company with four ongoing clinical programs and a broad pipeline of preclinical and research programs. MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team, MeiraGTx has taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTx’s initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, severe forms of xerostomia and neurodegenerative diseases. Though initially focusing on the eye, salivary gland and central nervous system, MeiraGTx intends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases.

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Kelly Boothe (415) 946-1076

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Source: MeiraGTx