MeiraGTx Announces Acquisition of Vector Neurosciences

MeiraGTx Holdings Plc recently announced it has acquired Vector Neurosciences Inc. in an all-stock transaction. As a result of the acquisition, which was signed and closed October 5, 2018, MeiraGTx has expanded its portfolio of clinical-stage product candidates to include adeno-associated virus encoding glutamic acid decarboxylase (AAV-GAD), a gene therapy product candidate ready for continued Phase 2 clinical development for Parkinson’s disease. A prior Phase 2 clinical trial of AAV-GAD was completed and was the first successful randomized, double-blind, sham-controlled trial of its kind for a gene therapy product candidate targeting a brain disorder.

“This strategic acquisition gives us an exciting mid-stage product candidate with promising, sham-controlled clinical data and expands our portfolio of potential therapies for neurodegenerative diseases,” said Alexandria Forbes, PhD, President and Chief Executive Officer of MeiraGTx. “We are excited to continue moving AAV-GAD through clinical development and look forward to potentially offering a novel therapy to patients with Parkinson’s disease.”

“With demonstrated expertise in gene therapy clinical development and manufacturing, we are very pleased to work with the talented team at MeiraGTx to continue the development of this novel gene therapy product candidate. AAV-GAD has the potential to transform the treatment of Parkinson’s disease patients, providing hope for patients with limited treatment options,” said Michael Kaplitt, MD, PhD, Co-founder of Vector.

 In a blinded Phase 2 clinical trial of AAV-GAD in patients with medically refractory Parkinson’s disease, 45 patients were randomized 1:1 to receive either AAV-GAD gene therapy delivered by injection into the subthalamic nucleus (STN) on both sides of the brain or bilateral sham surgery. Subjects were followed for one year and all results remained blinded until the final treated patient reached the 6-month primary endpoint. The trial met the pre-specified, per-protocol primary endpoint, with a significant improvement in the off-medication motor section of the Unified Parkinson’s Disease Rating Scale (UPDRS) part 3 compared to baseline. There was also a significant difference in the degree of improvement compared with patients in the sham arm. Other endpoints also showed significant improvements in AAV-GAD treated patients compared to patients in the sham arm.

AAV-GAD is an investigational gene therapy medicine designed to deliver the glutamic acid decarboxylase (GAD) gene to the subthalamic nucleus in order to increase production of GABA, the primary inhibitory neurotransmitter in the human brain. GAD is the rate-limiting enzyme in the synthesis of GABA, therefore it is believed that increasing subthalamic nucleus GAD expression through gene therapy will result in normalization of motor circuits and improve symptoms in Parkinson’s disease patients without affecting other brain regions that can be responsible for complications of existing therapies. AAV-GAD has received Fast Track designation from the United Stated Food and Drug Administration.

 Affecting nearly one million Americans and 10 million worldwide, Parkinson’s disease is the second-most common neurodegenerative disease after Alzheimer’s disease and is the 14th-leading cause of death in the United States. It is associated with a progressive loss of motor control (e.g., shaking or tremor at rest and lack of facial expression), as well as non-motor symptoms (e.g., depression and anxiety). There is no cure for Parkinson’s disease and 60,000 new cases are diagnosed each year in the United States alone.

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. For more information, visit