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Emmaus who? A biotech you never heard of just won an historic drug OK for sickle cell disease

After pursuing an R&D odyssey that stretched out more than 15 years, little Emmaus Life Sciences in Torrance, CA has just won an FDA OK for Endari, the first new drug approved for sickle cell disease in close to 20 years.

Operated by Yutaka Niihara, Emmaus gained the backing of a large majority of outside experts brought in by the agency to review the drug. “Until now, only one other drug was approved for patients living with this serious, debilitating condition,” noted the FDA’s Richard Pazdur in marking the OK.

While the pivotal data from the trial — which was started in 2010 — was marred by a high number of dropouts from both the drug and placebo arms of the study, Emmaus significantly cut the number of days patients with this drug spent in the hospital, as well as the rate of potentially lethal cases of acute chest syndrome.

I queried a spokesman for the company to see if they had a commercial organization or plans to sell the drug themselves and got this statement in return:

Endari is a significant milestone for the sickle cell patient community. It is the first advancement in treatment of this devastating disease in nearly 20 years and the first-ever treatment option for children. We expect to begin marketing Endari in the fourth quarter of 2017 and have plans to price Endari at a very reasonable amount to ensure patient access. We will apply for European market approval in late 2017 and we’re working with various groups and regulatory entities to provide access to Endari in the Middle East and Africa.

Late Friday the company added this: “Currently we are anticipating that the list price range will be approximately $11,000 to $18,000 per year depending on dosing.”

The drug, though, is not in the least bit new. It’s better known as L-glutamine, an old, cheap over-the-counter drug which the company sells as NutreStore for short bowel syndrome as well as a nutritional supplement. The biotech reported to the SEC that it netted only $107,000 in sales revenue on it for the first quarter after accumulating a total deficit of $113 million. In SEC filings, the company has noted that its own internal financial reporting process suffers from material weaknesses.

In one of many odd facts I’ve collected about this company, new FDA chief Scott Gottlieb was on the board at Emmaus in the second half of 2015, one of many board postings he took over the past decade. Niihara’s bio also cites a role as a professor at the David Geffen School of Medicine at UCLA. A spokesperson for the school tells me he’s a volunteer clinical professor at the school of medicine who works at Harbor-UCLA Medical Center.

At one point Niihara had struck a deal to merge into a troubled Generex $GNBT, which recently completed a 1-for-1000 reverse stock split to resolve its penny stock status. In early January, Niihara took the post of executive chairman at the company while it was still being traded on the pink sheets. But according to documents filed with the SEC, Generex never was able to recapitalize in order to complete the $225 million acquisition deal. Generex today has a market cap of only $3 million.

Endari’s OK marks the 24th new drug approval at the FDA for the year, which is already past the mark posted for all of a lackluster 2016.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
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Atlas Venture Cambridge, MA

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