Magenta Therapeutics Wins RMAT from FDA for Cell Therapy Drug


MGTA-456 will target inherited metabolic disorders, which are conditions that are the result of genetic defects.

Magenta Therapeutics (NASDAQ:MGTA) received a boost on Wednesday (September 4) from the US Food and Drug Administration (FDA) for its cell therapy, MGTA-456.

In a press release, the company said that the regulatory agency has given its therapy a Regenerative Medicine Advance Therapy (RMAT) designation to treat a range of inherited metabolic disorders.

According to the FDA, RMAT designation is awarded to cell therapies that can treat or cure life-threatening diseases. In order to receive this designation, a drug must show in preliminary clinical trials that it can also potentially treat unmet needs for its targeted disease.

Magenta’s MGTA-456 will target inherited metabolic disorders, which are conditions that are the result of genetic defects. A Phase 2 clinical trial of MGTA-456 is currently underway in patients aged six months and older who have Hurler syndrome, cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy or globoid cell leukodystrophy.

The primary goal of MGTA-456 is to make sure these diseases don’t progress by providing a large quantity of stem cells that can reproduce a patient’s immune system.

“This RMAT designation was based on the encouraging clinical data we have presented thus far, and it is an important milestone that recognizes the transformative, life-saving potential of MGTA-456 for children suffering from inherited metabolic disorders,” said John Davis, chief medical officer at Magenta.

Davis added that the company will continue working with the FDA to further MGTA-456 in the Phase 2 study and will move it into pivotal studies sometime next year.

Metabolic disorders make it so that the body is unable to process fats, proteins, sugars and nucleic acids. A report from Research and Markets estimates that the overarching metabolic disorder treatment market will reach roughly US$89 billion by 2025, increasing at a 7.56 percent compound annual growth rate between now and then.

Fueling that growth will be a rise in conditions such as diabetes, hypercholesterolemia and obesity. By 2040, it’s projected that one out of every 11 people will have one of these conditions. Grand View Research indicates that the US leads in the chronic obesity epidemic, although developing countries are beginning to see a higher number of metabolic diseases, including diabetes.

According to the report, more than 5 percent of the global population has diabetes and requires constant medication.

Magenta’s Phase 2 study of MGTA-456 has shown that enzyme levels in patients with Hurler syndrome normalize at the 42 day mark after treatment, while toxic metabolites decrease at the six month post-treatment mark.

Similarly, cALD patients had a reduction of brain inflammation 28 days after treatment and stable neurological scores six months post treatment.

In total, five patients were studied and all reached the FDA-approved primary endpoint. Data from the Phase 2 trial is expected sometime before the end of this year.

Wednesday’s announcement from Magenta follows the release of its Q2 financial results in early August. In addition to its MGTA-456 trial, the company began a Phase 1 study in April of MGTA-145 in autoimmune diseases, such as genetic conditions and blood disorders.

Magenta expects it will have data from this study sometime in the second half of this year; should the results go as planned, the company intends to move the candidate into a Phase 2 study in patients with multiple myeloma and non-Hodgkin’s lymphoma in 2020.

The company’s financial position in cash and its equivalents stood at US$172.3 million at the end of the quarter when compared to US$142.6 million at the end of last year. Thanks to the completion of a public offering of its common stock — which raised US$60.3 million in May — the company anticipates it will have enough cash on hand well into the back half of 2021.

Shares of Magenta bumped up 2.05 percent on Wednesday to close at US$9.97. Year-to-date, the company’s share price is up roughly 63 percent.

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Securities Disclosure: I, Jocelyn Aspa, hold no direct investment interest in any company mentioned in this article.

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