Gene Therapy Company MeiraGTx Buys Vector Neurosciences for Its Phase II Parkinson's Product
Published: Oct 09, 2018 By Mark Terry
In a previous Phase II clinical trial of AAV-GAD in patients with medically refractory Parkinson’s disease, patients who received the gene therapy hit the trial’s primary endpoint, which was a significant improvement in the off-medication motor section of the Unified Parkinson’s Disease Rating Scale (UPDRS) part 3 compared to baseline.
In the trial, 45 patients were randomized one-to-one 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. Sham surgery is a minor “placebo” surgery that mimics the gene therapy procedure. There was a significant difference in improvement in the patients receiving the STN injections over the sham surgery.
“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, president and chief executive officer of MeiraGTx, in a statement. “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.”
AAV-GAD is a gene therapy that delivers the glutamic acid decarboxylase (GAD) gene to the subthalamic nucleus to increase production of GABA, the inhibitory neurotransmitter in the human brain. GAD is a rate-limiting enzyme in GABA synthesis, so the theory is that increasing subthalamic nucleus GAD expression via gene therapy will cause normalization of motor circuits and improve Parkinson’s symptoms without affecting other parts of the brain.
The U.S. Food and Drug Administration (FDA) has designated AAV-GAD Fast Track status.
In late August, the FDA granted MeiraGTx’s AAV-CNGA3 rare pediatric disease designation. AAV-CNGA3 is a gene therapy for patients with achromatopsia (ACHM) caused by mutations in the CNGA3 gene. ACHM is an inherited retinal disease that prevents cone photoreceptors in the eye from working properly. Patients with ACHM are often legally blind at birth, are extremely sensitive to light, and have involuntary eye movements.
The FDA also granted AAV-CNGA3 orphan drug designation, and the European Medicines Agency (EMA)’s Committee for Orphan Medicinal Products recommended orphan medicinal product designation as well.
“We are very pleased that the FDA and EMA continue to recognize the important research MeiraGTx is doing for people living with rare inherited disorders such as ACHM,” said Forbes in a statement. “Our focus remains on patient benefit and developing products that offer a cure to people living with serious diseases. Working closely with expert clinicians and the FDA, we look forward to bringing impactful therapies to those in need.”
No financial details were released over the acquisition of Vector Neurosciences.
“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,” said Michael Kaplitt, co-founder of Vector, in a statement. “AAV-GAD has the potential to transform the treatment of Parkinson’s disease patients providing hope for patients with limited treatment options.”