EMMA: Emmaus Sickle Cell Treatment Expands Reach

By Brad Sorensen, CFA

OTC:EMMA

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Emmaus Life Sciences EMMA recently announced the approval of Endari, the company's signature drug for sickle cell disease, by the United Arab Emirates Ministry of Health for the treatment of sickle cell disease. There are approximately 600 people with sickle cell disease in the UAE according to the company—but around 225,000 in the Middle East North Africa (MENA) region.

Emmaus also has applications in with Kuwait and Saudi Arabia for approval of Endari. We view this as a positive development and a good indicator of further growth potential in the region.

Emmaus also announced recently that Endari has been accepted for an e-poster at the Annual Meeting of the British Society for Haematology to be held April 3-5, 2022. We view this development as another positive development in the ongoing effort to expand the market for Endari.

Sickle cell disease is a genetic mutation that causes an individual's blood cell to distort into a C, or sickle, shape. This mutation reduces the blood cells' ability to transport oxygen throughout the body. Another problem arising from sickle cell disease is that the sickled blood cells tend to break down rapidly and becoming very "sticky", resulting in patients with sickle cell disease having a propensity to have blood cells that clump together. This clumping causes the blood cells to become stuck and cause damage within blood vessels.

As a result of this clumping and damage to the blood vessels, sufferers of sickle cell can have reduced blood flow to distal organs, which leads to physical symptoms such as incapacitating pain, tissue and organ damage and even early death. Other symptoms of sickle cell disease can include painful swelling of the feet and hands, fatigue from anemia due to the reduced blood cell count, and a potential yellowing of the skin or whites of the eyes.

Sickle cell disease impacts approximately 100,000 people in the US and an estimated 20-25 million people worldwide, a disease for which there is no cure and few effective treatments. And it appears to impact the black community in an outsize fashion with the CDC reporting that about 1 in 13 black babies born are born with the sickle cell trait. This results in 1 out of every 365 black babies being born with sickle cell disease, while only 1 in every 16,300 Hispanic-American births are born with sickle cell.

As mentioned, there are no official cures for sickle cell disease, although there have been reported cases, by the CDC, of stem cell transplants being used to "cure" sickle cell. This potential cure is an exciting new potential option but will likely not be appropriate for many sickle cell victims. The risks that come along with stem cell transplants, according to the CDC, including life-threatening illness or death and it can be quite difficult to find a matching donor. For most sickle cell sufferers, being able to manage and control symptoms would likely be a much preferable, and more readily available, option. And that's what Endari, the therapy for treatment of sickle cell disease created by Emmaus, can provide.

Endari has been proven to reduce complications of sickle cell disease in adults and children 5 years and older and gained the orphan drug designation from the FDA, which is reserved for those drugs and therapies targeted at conditions affecting fewer than 200,000 Americans. This designation provides the company that receives it with the possibility of receiving tax credits for qualified clinical trials, an exemption from user fees, and, most importantly for Emmaus at this point, 7 years of market exclusivity after approval of the drug or therapy. The effectiveness of Endari in the fight against sickle cell disease is demonstrated by the results from recent clinical studies, which included 230 patients with sickle cell anemia or sickle thalassemia. Importantly, prior to the study, all 230 patients had at least two painful crises within 12 months of entering the trial.

We believe in the prospects of Emmaus to expand the use rate of Endari but acknowledge that there are many uncertainties surrounding the growth rates and approvals for the expansion of the uses of Endari. But we believe there are solid prospects and good odds for success and believe EMMA is worth a look at the current levels for investors with a higher risk tolerance.

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