Emmaus Life Sciences Profile
Emmaus Life Sciences, Inc. operates as a commercial-stage biopharmaceutical company. The company engages in the discovery, development, marketing, and sale of treatments and therapies, primarily for rare and orphan diseases.
The company's lead product, Endari (prescription grade L-glutamine oral powder) is approved by the U.S. Food and Drug Administration (FDA) to reduce the acute complications of sickle cell disease (SCD) in adult and pediatric patients five years of age and older. Endari has received Orphan Drug designation from the FDA and Orphan Medicinal designation from the European Commission, which designations affords marketing exclusivity for Endari for a seven-year period expiring in July 2024.
Endari is marketed and sold in the U.S. by the company's internal commercial sales team. Endari is reimbursable by the Centers for Medicare and Medicaid Services, and every state provides coverage for Endari for outpatient prescriptions to all eligible Medicaid enrollees within their state Medicaid programs. Endari is also reimbursable by many commercial payors. The company has agreements in place with the nation's leading distributors, as well as physician group purchasing organizations and pharmacy benefits managers, making Endari available at selected retail and specialty pharmacies nationwide.
The FDA has acknowledged that the clinical benefit of Endari was observed irrespective of hydroxyurea use, which supports the use of Endari as a monotherapy or in combination with hydroxyurea as safe and effective treatment options for patients with SCD. Endari's safety profile was similar to the placebo and Endari was well-tolerated in pediatric and adult patients alike.
Oncology Project
In October 2021, the company entered into a License Agreement with Kainos Medicine, Inc., a South Korean corporation (Kainos), under which Kainos has granted the company an exclusive license in the territory, including the U.S., the U.K. and the European Union (EU) to patent rights, know-how and other intellectual property relating to Kainos's novel IRAK4 inhibitor, referred to as KM10544, for the treatment of cancers, including leukemia, lymphoma, and solid tumor cancers. Based upon the positive pre-clinical results, the company intends to conduct further testing in vivo to evaluate KM10544's toxicity and efficacy against targeted cancers, including acute myeloid leukemia and Waldenstrom Macroglobulinemia (WM).
In 2021, the company licensed a small molecule (KM10544) targeting IRAK4 signaling pathway to treat leukemia and lymphomas. Leukemia is a cancer of blood-forming tissue causing high variation of its manifestation and therefore requiring many different treatment options.
The company is conducting pre-clinical studies to assess KM10544’s efficacy in two cancer cell lines, acute myeloid leukemia and Waldenstrom macroglobulinemia. In in vitro studies, KM10544 suppressed the proliferation and induced apoptosis (cell death) in both cancer cell lines with minimal toxic effects in healthy human cell lines, including human dermal fibroblasts and human adipose stromal cells. The company plans to undertake further in vivo testing to evaluate its toxicity (maximal dose and side effects) and to determine how KM-10544 cans affect (tumor elimination, delay of growth) against acute myeloid leukemia and WM.
Chondrocyte Cell Sheet Technology
The company has developed chondrocyte and osteoblast 'cell sheets' using human mesenchymal stem cells and is conducting pre-clinical studies to assess the potential of the cell sheets to articular cartilage injury, osteoarthritis and other cartilage-related conditions and bone diseases, such as osteoarthritis, nonunion, and Paget's disease. The company's cell sheets offer several potential advantages over existing treatment options, including reduced chemical toxins and xeno-products needed during cell sheet generation, easier and more convenient cell coverage of the injured tissue (transplantation on the damaged area), and allogeneic (i.e., use of stem cells from one individual in another individual) transplantation.
Cultured Autologous Oral Mucosal Epithelial Cell Sheets (CAOMECS)
The lead CAOMECS program is for the treatment of corneal diseases.
Device Measuring Cell Sheets Transparency
The company also has developed a device for measuring the maturity of biological cell cultures for harvesting of cell sheets, as well as the number and transparency of cells present in one or more cell sheets of the biological cell cultures. This semi-automatic device is a potentially essential tool for quality control in the growing field of cell sheet translational medicine. The potential application of this device includes assessment of the transparency of donor's cornea before transplantation. The company has filed a patent application in the U.S. for this technology and is in the process of improving the device. The company may seek a potential partner to develop or commercialize the device.
Summary of Pipelines Products
The development of the company's potential anti-cancer treatments, cell sheet technologies, including chondrocyte cell sheets for treating bone related conditions and CAOMECS for treating corneal and other diseases are in the early stages.
Highlights
In December 2022, the company received an Endari Marketing Authorization from the Pharmaceutical and Herbal Medicines Registration and Control Administration (Drug and Foods Control) of the Kuwait Ministry of Health.
In November 2022, the company received an Endari Marketing Authorization from the Registration Committee for Pharmaceutical Companies and their products of the Qatar Ministry of Public Health.
In April 2022, the company announced the launch of innovative full-service Telehealth Solution with its strategic partners, including Asembia LLC and UpScript IP Holdings, LLC.
In March 2022, the company received approval for marketing of Endari to treat SCD from the United Arab Emirates (U.A.E.) Ministry of Health.
Commercialization and Distribution
The United States
The company's in-house commercial team includes marketing, market access, patient support, and distribution support personnel. The company has a Commercial Patient Assistance Program (C- PAP) to provide financial assistance to eligible patients who are unable to afford their monthly co-payments for Endari. The company also maintains the Endari Patient Support Program to provide eligible patients access to Endari where appropriate.
The company's sales and marketing efforts focus on the following groups: pediatric and adult hematologists who treat SCD patients with sickle cell disease, Community Based Organizations (CBOs), government payors, insurance companies, and pharmacy benefit managers.
The company has contracted with AmerisourceBergen Specialty Group (ASD Healthcare LLC and US Bioservices Corporation), AmerisourceBergen Corporation companies, McKesson Plasma and Biologics LLC, a McKesson Corporation company, Cardinal Health 108, LLC, a Cardinal Health Inc. company, CVS Caremark, L.L.C., and a CVS Health Corporation company to distribute Endari to selected pharmacies and hospitals. AmerisourceBergen Corporation, McKesson Corporation, Cardinal Health, Inc, and Caremark are the four largest specialty distributors of prescription drugs in the U.S.
The company's two largest distributors, ASD Healthcare LLC and McKesson Plasma and Biologics LLC, each accounted for more than 25% of net revenue for the year ended December 31, 2022. On a combined basis, these distributors accounted for approximately 79% of net revenue in 2022.
Outside the United States
In May 2019, the company announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use, or (CHMP), adopted a negative opinion regarding its application for marketing authorization, or MAA, for the company's prescription grade L-glutamine oral powder, to be known as Xyndari in the European Union, or EU based upon the CHMP's position that its main clinical study did not conclusively support Xyndari's efficacy. In light of the CHMP's opinion, the company withdrew its MAA. The company is considering whether to pursue possible alternative regulatory pathways in an effort to afford SCD patients in EU countries access Xyndari.
Post-Brexit, the company assessed its overall submission package to determine strategies for scientific advice from the United Kingdom, or U.K., Medicines and Healthcare Products Regulatory Agency in preparation for a possible submission of a MAA for Xyndari. The company expects to provide an update of its assessment in the second half of 2023.
The company has entered into exclusive distribution agreements with strategic partners to register, commercialize and distribute Endari in the Gulf Cooperation Council countries (GCC) and other countries throughout the Middle East and North Africa (MENA) region in collaboration with its branch office in Dubai.
The company is party to an exclusive early access agreement pursuant to which its strategic partner distributes Endari on an early access basis in France, the Netherlands, and the United Kingdom (U.K.).
Diverticulosis
The company is party to a distributor agreement with Telcon RF Pharmaceutical, Inc. (Telcon) pursuant to which the company granted Telcon exclusive rights to its prescription grade L-glutamine (PGLG) oral powder for the treatment of diverticulosis in South Korea, Japan, and China. The agreement contemplates that Telcon will be responsible at its expense for obtaining marketing authorization assuming FDA approval is obtained and for all other commercial activities in the territories.
Suppliers
There are limited suppliers of PGLG, and the company obtains substantially all its PGLG, directly or indirectly, from Ajinomoto Health and Nutrition North America, Inc. (Ajinomoto), a subsidiary of Ajinomoto North American Holdings, Inc.
In June 2017, the company entered into an API supply agreement with Telcon RF Pharmaceutical, Inc. (Telcon), a South Korea-based company.
Research and Development
For the year ended December 31, 2022, the company's research and development expenses included $1.7 million.
Patents, Proprietary Rights and Know-How
The company has Orphan Drug market exclusivity for the treatment of sickle cell anemia with Endari in the United States through July 7, 2024 and, if approved in the EU, for ten years from the approval date. It may seek to pursue improvements and reformulations of Endari to preserve its intellectual property rights in Endari following the expiration of its Orphan Drug designation.
Patents
The company has issued patents related to compositions, including PGLG and methods involving the administration of PGLG for the treatment of diverticulosis in the United States, Europe, Japan, Australia, India, Mexico, China, Indonesia, Korea, and Russia. Associated patent applications are pending in the United States, the EU, Brazil, Korea, and Russia.
Patents directed to compositions for decreasing HbA1C levels in individuals who are shown to have average blood sugar levels in the diabetic range have issued in Japan, Indonesia, and the Philippines. Associated applications are pending in the United States, Europe, Brazil, India, China, the Philippines, and Japan.
The company has issued patents directed to the treatment of hypertriglyceridemia in Japan and the Philippines. A corresponding European patent application has been granted and is the subject of an Opposition proceeding. Associated applications are pending in the United States, Brazil, India, China, and the Philippines.
A patent application directed to the treatment of sickle cell using a multi-component composition is pending in the United States and Europe. An international application directed to the same invention has been filed under the Patent Cooperation Treaty.
The company has a pending international (PCT) patent application directed to the treatment of cancers with KM10544, alone or in combination with a Bruton Tyrosine Kinase (BTK) inhibitor or a Poly (ADP-ribose) polymerase (PARP) inhibitor. The cancers being treated may have a MyD88 mutation (e.g., the L265P missense mutation) and can include hematologic cancers, such as Waldenstrom Macroglobulemia (WM), Acute Myeloid Leukemia (AML), and diffuse large B-cell lymphoma (DLBCL), and solid tumors such as rectal cancer, pancreatic cancer, and small cell lung cancer. The application is expected to enter into national stages by June 2024 and has a projected expiration date in 2042.
The company has a pending PCT patent application directed to devices and methods for measuring the thickness of cell sheets. The technology is important for the preparation and use of stem cell-derived cells sheets, such as corneas and cell sheets. This non-invasive approach can determine the time progression of cell sheet growth, the maturity of the cell sheet, and the number of cells per cell sheet before harvesting and transplantation. The application is expected to enter into national stages by July 2023 and has a projected expiration date in 2042.
The company has a pending PCT patent application directed to the preparation and use of cell sheets. The prepared cell sheets can be used for the treatment of cartilage-related conditions such as cartilage injury and osteoarthritis, and bone diseases, such as nonunion bone diseases and Paget’s disease. The cell sheets can be formed of a multilayer of cells grown and harvested in an intact cell sheet that can be transplanted as a patch on the injured area. The application is expected to enter into national stages by April 2024 and has a projected expiration date in 2042.
Trademarks
The company holds the U.S. trademark registrations for 'Emmaus Medical' and 'Endari' and a trademark registration for 'Xyndari' (as Endari will be marketed if approved) in the EU.
Government Regulation
In addition to obtaining FDA approval for each product candidate before the company can market it as a product, the manufacturing establishment from which it obtains it must be registered and is subject to periodic FDA post approval inspections to ensure continued compliance with current Good Manufacturing Practices (cGMP) requirements. All facilities and manufacturing processes used by third parties to produce the company's product candidates for clinical use in the United States and its products for commercialization must be in compliance with cGMP requirements and are subject to periodic regulatory inspections.
History
The company was founded in 2000. It was formerly known as Emmaus Holdings, Inc. and changed its name to Emmaus Life Sciences, Inc. in 2011.
