Emmaus Life Sciences Inc Tackles Debilitating Sickle Cell Disease, Considering Other Cures

The company achieved net income status two years after entering its commercial phase and aspires to continued growth and expansion in the years to come, to be profitable and to be able to pay dividends.

“Our aim was not to develop a pharmaceutical company, but simply to offer something practical, safe and effective,” says Dr Yutaka Niihara, explaining how he and his colleague Willis Lee formed Emmaus Life Sciences Inc (OTCQX : EMMA) and developed Endari (L-glutamine), a treatment for debilitating sickle cell anemia.

“Fortunately, we have found something based on our basic scientific research,” added Niihara, president and CEO of Emmaus alongside Lee, president and CEO of the company.

Niihara had originally planned to become an oncologist when he entered the fellowship program at Harbor-UCLA Medical Center in 1989. That changed when, as part of his training, he saw the devastating impact of sickle cell disease on patients. hematology patients – young people with pain who he says it is significantly worse than that caused by cancer, with hardly any treatment at the time other than narcotics.

The genetic disease is the result of a genetic mutation and is more likely in areas where malaria is common, or in ethnic groups that have migrated from areas such as Africa, India and the Middle East. While those who have only one sickle cell gene are resistant to malaria, complications arise in those who have two or more genes.

Stiff and deformed red blood cells cause obstruction of small blood vessels in patients. Niihara says it’s like having mini strokes or heart attacks all over the body, causing severe pain. It can also lead to decreased immunity in people with the disease, making them more susceptible to infectious diseases as well as strokes, heart attacks, kidney, liver and lung failure.

“It really is a horrible disease,” he says. “In this country, although we have some of the best medical care in the world, life expectancy is around 40 years. If you go to developing countries, it’s only in your twenties at best.

Until 1997, no drugs were available to alleviate the effects of the disease. Then, the United States Food & Drug Administration (FDA) granted regular approval to hydroxyurea, which increases fetal hemoglobin and decreases the number of attacks. However, the long-term effects of the drug are not yet known.

Numbers game

It’s a numbers game, according to Niihara, and the fact that it is seen as a more prevalent disease in developing countries makes the search for drugs to treat the disease less lucrative for the pharmaceutical industry.

Until the mid-2000s, Niihara notes, many large pharmaceutical companies were not interested in the fight against so-called orphan diseases. Globally, he says, there are around 20 to 25 million people with sickle cell disease, but in the United States there are only around 100,000 patients and even fewer in Europe.

“For the efforts you put into developing drugs, they didn’t think they would get a return,” Niihara adds. “With sickle cell disease, unfortunately, we don’t have strong advocates or activists. This could be due to the socio-economic status due to the ethnic origin of those affected; I’m not really sure.

While Niihara and his colleagues were recognized by the National Institutes of Health (NIH) and the FDA as very promising developers of sickle cell drugs, the big pharmaceutical companies were not interested in supporting them, which led him to to form Emmaus, Lee joining society in its embryonic state by mobilizing the necessary capital from “family and friends”.

Orphan drug designation

The FDA’s orphan drug designation for sickle cell disease – with equivalent status in many other developed countries – has made the development of drugs to fight the disease more lucrative because of the economic benefits that come with the designation.

“And once you’ve developed it, you can get by charging huge sums of money because you’re protected,” Niihara notes. “Insurance companies are almost obliged to cover this type of drug even if it is very expensive.

After a 20-year hiatus with no new drug candidates having been proposed since hydroxyurea, in 2017 the FDA approved Endari.

Emmaus, now a commercial-stage biopharmaceutical company, began marketing Endari in 2018, rapidly building up a regular customer base of around 500 patients, which grew to around 800 by the end of 2019.

However, growth has leveled off at around 1,000 patients in 2020 and 2021 due to the coronavirus (COVID-19) outbreak. With around 20,000 to 25,000 requiring frequent medical attention, he believes the company could reach around 5,000 patients in the United States as COVID-19 restrictions relax.

“We are implementing some strategic plans to try to achieve this in 2022,” he adds.

Global expansion plans

With a limited number of sickle cell patients in the United States, Niihara reveals that Emmaus is also growing in the Middle East, where the disease is much more prevalent. It has already acquired a temporary license in Bahrain and is working on an early access basis with other countries including the United Arab Emirates, Saudi Arabia, Oman, Kuwait and Qatar.

“I hope that we will have full marketing approval in these countries very soon,” he said. “We also have a presence in Europe on the basis of early access, particularly in France and the UK. ”

Other applications for Endari

While Endari may have a limited customer base in the United States for patients targeting sickle cell anemia, Niihara says the drug can also reverse diverticulosis, a bowel disease that is common in older patients in the United States and in other developed countries, with around 40% of those over 60 likely to develop it. Of those, he says about 10-20% will develop a more serious condition known as diverticulitis.

“Often they require surgery or hospitalization with antibiotics to reduce inflammation,” he says. “We have never had anything to reverse this diverticulosis, which is the cause of diverticulitis, and we have good evidence that Endari can reverse it.”

The first phase of a pilot trial showed 100% disease reversal in one patient in six months and 50% reversal in a second patient.

“So we are repeating the pilot trial at another institution in Texas and if we can confirm the previous study on five patients, we plan to embark on double-blind phase 3 randomized trials,” he said. .

Endari, or L-glutamine, is known to help many different conditions, including gastritis, Niihara says. However, as it cannot obtain exclusivity on a cure for gastritis, this is not financially feasible as Emmaus is the first company to investigate Endari as a cure for diverticulosis.

“We’ve been able to get the patent almost all over the world, so we’re really focusing on that,” he says.

A third-party facility is also using Endari for research on burn patients to see if it speeds healing, he adds.

More drugs in preparation

Endari aside, Niihara says Emmaus is also working on another compound for the treatment of cancers.

In October 2021, the company signed an agreement with South Korean company Kainos (LSE: KNOS) Medicines, granting it an exclusive license to patent rights, know-how and other intellectual properties relating to the new inhibitor IRAK4. by Kainos (LSE: KNOS), referred to as KM10544, for the treatment of cancers, including leukemia, lymphoma and solid tumors.

“We were fortunate enough to acquire the product and we did our own preclinical work,” he says. “The results were more dramatic than what they shared with us, so we went ahead and licensed the product and we’re developing it. So far, we have observed significant efficacy in our laboratory against certain difficult-to-treat hematologic malignancies. ”

Dividends also in preparation

Unlike many of his peers, Niihara says that Emmaus has already been able to generate net income within two years of its commercial phase because it invests in drug development on a tight budget. With its planned expansion, he says the business is likely to double or even triple the size of the business in the near term.

“We have around 1,000 patients in treatment, but we think the US and the Middle East combined will quickly get us to 5,000 patients and I don’t think we’re going to significantly increase our spending,” he said. -he declares.

While Emmaus is already showing positive net income, Niihara says there is significant profit potential as it expands its customer base and continues to develop its pipeline. With this, the company wants to start paying dividends in the near future to reward its shareholders.

“We have an exciting pipeline,” he concludes. “For example, there has never been a treatment that can actually reverse diverticulosis and the market is 600 times larger than that for sickle cell anemia. We want to show investors that we are walking the path to these achievements. “

Contact the author at stephen.gunnion@proactiveinvestors.com

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