A Biotech Unveils Its New Gene Control Technology

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When she helped start

MeiraGTx Holdings

in 2015, CEO Zandy Forbes wanted to find a way to precisely control the output of genes implanted in people to fix genetic disorders. In an analyst meeting Wednesday, MeiraGTx unveiled that technology.

The small-cap developer of gene therapies presented data showing its ability to insert genes in lab animals that generate antibodies against cancer or Alzheimer-associated proteins, or crucial hormones like insulin. But the company’s modified genes only become active when MeiraGTx (ticker: MGTX) administers an otherwise harmless triggering drug. Meira believes its gene regulation technology can allow simple genetic remedies for diabetes and obesity, or put antibody drugs inside the blood-brain barrier. It could also lower the sky-high pricing of gene therapies.

“We’ve created an entire system for regulating genes using small molecules,” Forbes told her audience. “Not just on and off, but specific, granular regulation based on the dose of orally given small molecules.”

The gene therapies marketed by

Roche Holding

(RHHBY) or

Novartis

(NVS)—and under development by dozens of others, including

Pfizer

(PFE) and

Sarepta Therapeutics

(SRPT)—use hollowed-out viruses to add artificial genes to the cells of patients with disorders caused by flaws in their natural genes, such as muscular dystrophy or hemophilia. Once inside, the transferred genes start cranking out the protein that patients had lacked. Regulating the output of these transferred genes has been hard.

MeiraGTx says its gene regulation technology involves something called a “riboswitch”—extra genetic instructions that it attaches to the therapeutic gene before treatment. Those instructions effectively keep the gene in an “off” position after it’s implanted. A patient could then turn the gene on and off—and control its output over a wide range—with a drug delivered as pills, eye drops or injection.

“We can potentially activate insulin with a pill to exactly the right does when you need it,” Forbes told her Wednesday audience.

One-time gene therapies have been priced over $1 million. Forbes said that Meira’s gene regulation platform would allow for a more modest charge for the initial procedure, then ongoing charges for the cheap-to-produce pills.

As Barron’s reported in October, MeiraGTx has long hinted of a solution to the gene regulation challenge. Meira stock leapt after our report, from around $13 to more than $23, before settling back to the high teens. In Thursday trading, the shares were off 2% to $20.50, which values the company around $900 million.

Like many corporate research programs, MeiraGTx has kept its gene regulation work under wraps while surrounding it with patent protection. That means that its claims for the technology haven’t yet been exposed to the scrutiny and reproducibility that helps validate published scientific research.

The MeiraGTx riboswitch technology is intriguing, wrote RBC Capital Markets analyst Luca Issi in a Thursday note. “However, the program has yet to enter the clinic,” he cautioned, “so it will take time to play into the stock unless a transformational deal is executed ahead of clinical data.”

CEO Forbes told Wednesday’s listeners that MeiraGTx will start clinical trials of its first small-molecule regulatory drug next year, then test its riboswitch system in several gene therapies that Meira already has in clinical trials for a kind of inherited blindness—partnered with 

Johnson & Johnson

(JNJ)—and for a terrible dry-mouth side effect of radiation treatment.

For the large clinical trials that would be needed in diabetes, obesity, cancer or Alzheimer’s, Forbes says MeiraGTx is in ongoing discussions with potential partners. “When I said the opportunities are boundless,” she said, “that really is the case.”

RBC’s Issi rates the small-cap stock an Outperform, with a $27 price target—citing MeiraGTx’s current clinical trials of its own gene therapies, and a valuation that he terms “undemanding.”

Write to Bill Alpert at [email protected]