AAV2hAQP1 gene therapy(National Institutes of Health)

IPO letter in the billboard screen jxfzsy/Getty Images/iStockphoto MeiraGTx has filed with the U.S. Securities and Exchange Commission (SEC) its intentions to launch an initial public offering (IPO). New York City-based MeiraGTx has filed with the U.S. Securities and Exchange Commission (SEC) its intentions to launch an initial public offering (IPO) to raise $86 million. The company expects to use the money raised to advance several of its gene therapy products into and through clinical trials. The company expects ordinary shares will trade on the Nasdaq Global Market under the “MGTX” ticker symbol. Underwriters will be Bofa Merrill Lynch, Barclays, Evercore ISI and Chardan. According to the filing, the company currently has nine product candidates in preclinical to Phase I/II clinical trials for a variety of disorders, including achromatopsia (CNGB3 and CNGA3), X-linked RP (RPGR), RPE65-Deficiency (RPE65) and others. Its first area of clinical focus is eye diseases, with three ongoing clinical programs and a fourth expected to launch a Phase I/II trial in 2019. Both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted orphan drug designation to each product candidate in its ongoing clinical programs, including those to treat CNGB3, RPGR and RPG65, as well as for their product candidate to treat AIPL1. The FDA also granted rare pediatric disease designation for its programs to treat mutations in RPE65 and CNGB3 and Fast Track designation for its XLRP program caused by mutations in RPGR. The EMA gave it PRIME designations for its clinical program to treat mutations in CNGB3. MeiraGTx spun out of Kadmon Holdings in 2015. It also partnered with UCL Institute of Ophthalmology, a gene therapy spinout of Athena Vision . It is led by Alexandria Forbes, formerly of Kadmon. On April 23, MeiraGTx announced the FDA had granted Fast Track designation for AAV-RPGR to treat X-linked retinitis pigmentosa (XLRP) caused by defects in the retinitis pigmentosa GTPase regulator (RPGR) gene. “XLRP is a devastating condition that causes rapid progression to blindness and currently has no approved treatment options,” Forbes, president and chief executive officer of MeiraGTx, said in a statement. “This Fast Track designation is an important milestone for both patients living with XLRP and MeiraGTx, allowing our team to communicate closely and often with the FDA as we work to bring a much-needed therapy to patients. Marking the third regulatory milestone our ocular program has received this year, we look forward to continuing this momentum in our commitment to develop innovative gene therapy treatments for patients.” According to the S-1 filing with the SEC, the company’s second area of focus is xerostomia, a chronic and debilitating disorder of the salivary glands. It can be caused by several different attacks on the salivary glands, including radiation therapy for head and neck cancer and some autoimmune diseases. The company stated, “A Phase I clinical trial of our gene therapy product candidate, aAV-AQP1, is ongoing in patients who have survived cancer free for five or more years following treatment for head and neck cancer and are suffering from grade 2 or 3 radiation induced late xerostomia, or RIX….We also intend to initiate a Phase I/II clinical trial of AAV-AQP1 for the treatment of patients with chronic xerostomia caused by Sjogren’s syndrome, an autoimmune disease affecting more than two million people in the United States.”