FDA Places Clinical Hold on Translate Bio’s mRNA Drug Study

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Translate Bio’s plans to test a messenger RNA (mRNA) drug in patients who have a rare metabolic disorder have been halted by the FDA.

Lexington, MA-based Translate Bio (NASDAQ: [[ticker:TBIO]]) said Tuesday that the regulator verbally notified it of “additional clinical and nonclinical questions” about the company’s submission. Until those questions are resolved, the FDA has placed a clinical hold on Translate Bio’s planned Phase 1/2 study.

Translate Bio’s stock price dipped more than 9.3 percent to $5.01 per share following the company’s announcement.

Translate Bio researches synthetic mRNA drugs, which are intended to coax the body into producing therapeutic proteins. The company’s drug MRT5201 was developed to treat an inherited disorder in which patients don’t produce enough ornithine transcarbamylase (OTC), an enzyme needed to break down and remove nitrogen from the body. OTC deficiency leads to a buildup in the blood of ammonia, a neurotoxin. Treatment includes dietary restrictions intended to limit the production of ammonia in the body, as well as drugs that stimulate nitrogen removal. In some cases, the condition is addressed with a liver transplant. But that option comes with the risks of complications and requires patients to take immuno-suppressive drugs for life.

The intravenous Translate Bio drug was developed to deliver mRNA to the liver, conveying the genetic information for producing deficient OTC enzyme. If it works, the company says liver cells will produce OTC. The company has another mRNA drug in its pipeline: MRT5005 is in a Phase 1/2 study in cystic fibrosis. Translate, which was founded as RaNA Therapeutics, acquired both mRNA drugs from Shire in 2017.

Translate Bio’s application to start a clinical trial testing MRT5201 was submitted in December. The company offered no details about the questions that the FDA had about its drug or clinical trial plans. But Translate Bio said it expects to receive formal written communication from the FDA with more information, and it will work with the regulator to address the questions.

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Author: Frank Vinluan

Xconomy Editor Frank Vinluan is a business journalist with experience covering technology and life sciences. Based in Raleigh, he was a staff writer at the Triangle Business Journal covering technology, biotechnology and energy before joining MedCityNews.com as North Carolina bureau chief. Prior to moving to North Carolina’s Research Triangle in 2007 he held business reporting positions at The Des Moines Register and The Seattle Times.