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This Day That Year: MeiraGTx Holdings

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Shares of MeiraGTx Holdings plc (MGTX), which touched a 52-week low of $7.98 on July 25, 2018, have since gained more than 150 percent.

It was on this day last year, i.e. on June 8, 2018, that MeiraGTx went public on the Nasdaq Global Select Market by setting a public offering at $15 per share.

MeiraGTx Holdings is a clinical-stage gene therapy company, with five ongoing clinical programs in the ocular and neurodegenerative disease space.

The lead product candidate is AAV-GAD gene therapy for advanced Parkinson's disease. AAV-GAD came under MeiraGTx' fold when it acquired Vector Neurosciences Inc. last year.

AAV-GAD is designed to deliver the glutamic acid decarboxylase (GAD) gene to the subthalamic nucleus in order to increase the production of gamma-aminobutyric acid (GABA), the primary inhibitory neurotransmitter in the human brain. In Parkinson's disease patients, the amount of GABA in the brain is reduced, which leads to symptoms like tremors, leg motor restlessness, and levodopa-induced dyskinesia.

AAV-GAD successfully completed a phase II trial when it was under the aegis of Vector Neurosciences.

MeiraGTx expects to meet with the FDA in mid-2019 to define the clinical pathway to support regulatory approval of AAV-GAD in Parkinson's disease. The Company anticipates providing a regulatory and clinical development update in the second half of 2019 following interactions with the regulators.

The other clinical programs in the pipeline are:

-- AAV-CNGB3 for achromatopsia caused by mutations in the CNGB3 gene. This product is under a phase I/II trial in adult and pediatric achromatopsia patients. The trial has completed enrollment - with a total of 23 patients having been treated.

Achromatopsia is a condition characterized by partial or total absence of color vision. (Source: NIH).

-- AAV-RPGR for X-Linked Retinitis Pigmentosa, a rare, inherited condition that causes blindness in men. This investigational gene therapy is under a phase I/II trial, and is currently enrolling patients in the U.S. and UK.

MeiraGTx is partnered with Johnson & Johnson's Janssen Pharmaceutical for the development of gene therapy candidates AAV-CNGB3 and AAV-RPGR.

-- AAV-RPE65 for the treatment of RPE65-deficiency, a rare, genetic disorder that causes impaired vision from birth and results in the degeneration of the entire retina over time.

The 6-month data from a phase I/II study of AAV-RPE65 for RPE65-deficiency reported last month demonstrated statistically significant improvement in vision-guided mobility and visual function in treated eyes compared to untreated eyes.

MeiraGTx intends to meet with regulatory authorities in the second half of 2019 to define the development pathway for regulatory approval of AAV-RPE65.

-- AAV-AQP1 for Grade 2/3 radiation-induced Xerostomia, also known as dry mouth, in patients who have had radiotherapy of the oral cavity and neck region. This product is under a phase I dose-finding study. An additional multi-center phase I/II trial of AAV-AQP1 is expected to be initiated in 2019.

Since inception, the Company has incurred significant operating losses, and as of March 31, 2019, had an accumulated deficit of $166.3 million.

Net loss for the three months ended March 31, 2019, was $18.0 million or $0.62 per share compared to a net loss of $16.4 million or $1.91 per share in the year-ago quarter.

MeiraGTx is a clinical-stage company and has no marketed products. Therefore, no revenue has been generated from product sales to date.

The Company ended March 31, 2019, with cash and cash equivalents of approximately $227 million, which is expected to be sufficient to fund its expenses into 2022.

MGTX has thus far hit a low of $7.98 and a high of $24.76. The stock closed Friday's (June 7) trading at $20.99, up 1.84%.

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