As for the IPO, it generated $75 mln in gross proceeds. The deal was led by BofA Merrill Lynch, Barclays, and Evercore ISI. The stock is set to open for trading later this morning on the Nasdaq.
MGTX is a clinical stage gene therapy company with four ongoing clinical programs and a broad pipeline of preclinical and research programs. It has core capabilities in viral vector design and optimization, gene therapy manufacturing, and a potentially transformative gene regulation technology. The company has taken a portfolio approach by licensing, acquiring, and developing various technologies.
Its initial focus is on three distinct areas: inherited retinal diseases (IRDs), severe forms of xerostomia (dry mouth), and neurodegenerative diseases. Eye diseases are its first area of clinical focus and the aim is to provide treatments that will halt vision loss in patients. It currently has three ongoing clinical programs in IRDs with an additional program expected to initiate a Phase 1/2 clinical trial in 2019. The focus of these programs includes achromatopsia related to mutations in CNGB3, X-linked retinitis pigmentosa related to mutations in RPGR, and inherited retinal dystrophy caused by mutations in RPE65. It also has a product candidate that was made for compassionate use under a specials license in the UK to treat patients with Leber congenital amaurosis 4, or LCA4, caused by mutations in AIPL1.
MeiragTx's second area of focus is xerostomia (dry mouth), a chronic and debilitating disorder of the salivary glands in which saliva production is impaired. A Phase 1 trial of its gene therapy product candidate, AAV-AQP1, is ongoing in patients who have survived cancer and are suffering from grade 2 or 3 radiation induced late xerostomia, or RIX. MeiragTx also intends to initiate a Phase 1/2 trial of AAV-AQP1 which treats xerostomia caused by Sjogren's syndrome, an autoimmune disease affecting more than two mln people in the US. The FDA has granted orphan drug designation to AAV-AQP1.
Neurodegenerative diseases are MeiragTx's third area of focus. Its first target indication is ALS and the company expects to file an IND and initiate a clinical trial of its first product candidate in 2019. MeiragTx believes its approach to treating ALS patients is different because, rather than targeting a specific genetic defect that defines a small subset of ALS patients, MeiragTx aims to target the underlying cell biology driving motor neuron death in ALS, potentially enabling the company to treat a broader patient population that includes both sporadic and inherited forms of the disease.
On a final note, like most clinical stage gene therapy companies, MeiragTX does not really have any financials yet. It began operations in 2015 through the acquisition of Athena Vision and certain assets from Kadmon Corp. It has never been profitable and does not expect to be profitable in the foreseeable future. Typically, it takes about 6-10 years to develop a new drug from the time it enters Phase 1 clinical trials to when it is approved for treating patients, but in many cases it may take longer.