Gene therapy startup MeiraGTx files for $86M IPO

Nasdaq
MeiraGTx hopes to start a pivotal trial of achromatopsia gene therapy AAV-CNGB3 next year. (Nasdaq)

MeiraGTx has filed for an $86 million IPO to advance a handful of gene therapies into and through clinical trials. The transatlantic biotech is initially targeting inherited retinal diseases with therapies designed to halt vision loss.

MeiraGTx was created in 2015 and quickly landed assets, staff and work space from Kadmon and a partnership with UCL gene therapy spinout Athena Vision. Under the leadership of Alexandria Forbes, Ph.D., formerly of Kadmon, MeiraGTx then acquired the rest of Athena and spent more than $80 million to establish the technology platform and manufacturing infrastructure needed to realize its plan to become a vertically-integrated gene therapy specialist. 

Perceptive Advisors helped to bankroll the activity, giving it a 16% stake in the startup. With work on a 29,000-square-foot manufacturing facility in London now complete and four gene therapies in the clinic, MeiraGTx is seeking money from public investors to finance the next stage of its strategy. 

Your Daily Newsletter — Free

Enjoying this story? Subscribe to FierceBiotech!

Biopharma is a fast-growing world where big ideas come along every day. Our subscribers rely on FierceBiotech as their must-read source for the latest news, analysis and data in the world of biotech and pharma R&D. To read on the go, sign up today to get biotech news and updates delivered right to your inbox!

The focus in the months and years to come is clinical development of the assets, many of which have their roots in the work Robin Ali, Ph.D., performed at the UCL Institute of Ophthalmology. That work provided preclinical data suggesting targeting CNGB3 may restore cone function and ultimately led to a pipeline of clinical-phase ophthalmology candidates. 

MeiraGTx’s CNGB3 candidate, AAV-CNGB3, is one of the clinical assets. Researchers in the U.K. have given the therapy to 10 achromatopsia patients. The trial is yet to observe improvements in visual function but has generated evidence of decreased photoaversion, a disabling symptom of achromatopsia that makes it uncomfortable for sufferers to be in normally-lit rooms. Data are due to in 2019. If all goes to plan, MeiraGTx hopes to start a pivotal trial later that year.

In parallel, MeiraGTx is running phase 1/2 trials of two other ophthalmology assets and one salivary gland therapy. Like the AAV-CNGB3 study, some of the trials have shown early hints of efficacy but nothing more.

MeiraGTx has hit some potholes in getting to this point. The start of the trial of RPE65-deficiency ophthalmology candidate AAV-RPE65 was held up by a partial clinical hold related to an aspect of its manufacturing process. MeiraGTx got the hold lifted in October and filed an IND for CNGB3 candidate AAV-CNGB3 later that month, only for that submission to run into difficulties, too. 

The FDA put a clinical hold on the AAV-CNGB3 IND after spotting a disparity between the targeted titer and readout from PCR analysis. MeiraGTx submitted its second response earlier this month, pinning the blame on a PCR assay artifact, but for now the hold remains in place. The U.K. clinical trial is proceeding while the IND is on hold.