The world's first oral tablet of edaravone is about to hit the market.
On December 23, Aucontal Biotech submitted an application for the listing of AUKONTALS (edaravone tablets) to the US FDA for the treatment of amyotrophic lateral sclerosis (ALS). The drug was granted orphan drug status by the FDA in 2021.
Aucontal Biotech said that edaravone tablets not only take the lead in dosage form innovation, but also have great clinical potential in solving the multiple treatment challenges faced by ALS patients. It is expected to become the first orphan drug listed in the United States by a domestic pharmaceutical company in the CNS field, tapping into the rare disease ALS market.

▍ALS "difficult to cross"
ALS is a fatal neurodegenerative disease that gradually causes patients to lose their voluntary muscle control function by destroying motor neurons in the brain and spinal cord. The incidence of the disease is relatively low, but it has a very serious impact on the quality of life of patients.
At present, with the intensification of global aging, the number of ALS patients will continue to grow. Along with this, there is an increasing demand for effective treatment options.

According to Stratistics MRC's forecast data, the global ALS treatment drug market will be worth US$860 million in 2024 and will reach US$2.31 billion in 2030, with a compound annual growth rate of 12.6%. Despite this, the lack of convenient, safe and affordable treatment options on the market, especially the lack of oral drugs, has limited the widespread treatment of the disease and the improvement of patients' quality of life.
After Edaravone was launched in the United States in 2017, it provided patients with a new option, but the patient compliance of both its injection and the later approved oral suspension was poor. Aozhong's oral tablets significantly improved patient compliance and also showed obvious advantages in drug stability and cost of use.

▍Achievement of the name of "first"
Edaravone is currently the only neuroprotectant with evidence-based medicine. It has been shown to inhibit lipid peroxidation and vascular endothelial damage, and improve brain edema, tissue damage, and delay neuronal death. In this case, why is the development progress of tablets slower than other dosage forms? The relevant person in charge of Aukontal explained that edaravone has poor water solubility and is also a P-glycoprotein (Pgp) substrate, with extremely low oral bioavailability (3-5%), so no oral preparation has been launched in more than 20 years. Through its unique solid dispersion technology, Aukontal has increased the oral bioavailability by dozens of times. The formula patent has been authorized in China and the United States, and the European patent is about to be authorized. As the world's first oral tablet of edaravone, AUKONTALS has obvious differentiated advantages. Compared with existing injections and oral suspensions, edaravone oral tablets have significantly improved the convenience of medication and patient compliance. In addition, the room temperature storage conditions of the tablets greatly improve the patient's medication experience and reduce the burden on patients and their families.

▍Accelerate the march into CNS to create a Pipeline-in-a-pill blockbuster
From the timeline, ALS will be the starting point for Auzone Biotech to move towards commercialization, and AUKONTALS is expected to be approved by the FDA for listing in 2025. In addition to ALS, AUKONTALS's R&D team is planning to actively explore its clinical development and application in other fields based on the results of its team's many years of basic research, such as:
• For the treatment of AIS indications, it is expected to submit a key registration clinical trial application to the FDA in January 2025 and submit a listing application in 2026.
• For the treatment of Alzheimer's disease (AD), it is expected to start an international multicenter Phase II clinical trial in early 2025.
• For autism spectrum disorder (ASD), edaravone tablets have been approved by the CDE for Phase II clinical trials. AUKONTALS is one of the few clinical trial drugs for the core symptoms of ASD, and is expected to bring new hope to the majority of ASD children and families.

"In the next few years, Aukontal will continue to focus on the research and development of innovative drugs for central nervous system diseases. On the one hand, it will focus on realizing the potential of AUKONTALS as a pipeline-in-a-pill, while actively developing and establishing new CNS product pipelines, striving to become a biotech with international competitiveness and influence in the CNS field." The company's head said. Although the rare disease market is limited by factors such as the type of disease, the road to commercialization is not as mature as other popular tracks such as tumors, but Aukontal does not seem to intend to fight alone.

In July 2024, Aukontal reached a cooperation with Huadong Medicine to exclusively license the development, registration, production and commercialization rights of Edaravone Tablets (TTYP01 Tablets) for the treatment of AIS in mainland China, Hong Kong, Macau and Taiwan to the latter. According to the terms of the agreement, Aukontal will receive a down payment of 100 million yuan, a total of development, registration and sales milestone payments of up to 1.185 billion yuan, and a double-digit net sales commission fee.

Regarding the overseas rights it has retained, Aukontal said it is actively exploring cooperative development with other pharmaceutical companies and other options. By cooperating with the world's leading research institutions and pharmaceutical companies, Aukontal hopes to accelerate the clinical progress of new drugs and leverage the resources and market channels of its partners to quickly achieve global promotion of its products in order to maximize the value of its products. As the world's first oral tablet of edaravone, the success of AUKONTALS will enable Aukontal to achieve the important milestone of commercialization by bringing new options to ALS patients. Industry insiders generally believe that the launch of this innovative product is not only expected to fill many gaps in the field of ALS treatment, but is also likely to boost the enthusiasm and progress of neurological drug research and development.

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