Auzong Bio Submits NDA for Global First Oral Edaravone Tablet TTYP01 (AUKONTALS) to FDA for ALS Treatment; East China Medicine Holds Exclusive China Rights

On December 25, Auzone Biologics announced that it had formally submitted a New Drug Application (NDA) to the U.S. FDA for its investigational drug candidate TTYP01 edaravone tablets (brand name: AUKONTALS), indicated for the treatment of amyotrophic lateral sclerosis (ALS). Auzone Biologics anticipates that the application will receive FDA approval for marketing within 2025.

As the world’s only oral edaravone tablet, TTYP01 offers improved patient compliance and convenience, covering both the acute and recovery phases of stroke, and is expected to deliver greater clinical benefits to a broad population of stroke patients. Furthermore, TTYP01 is simultaneously expanding its indications to include autism spectrum disorder (ASD) in children, amyotrophic lateral sclerosis (ALS), Alzheimer’s disease (AD), and acute high-altitude sickness.

In July this year, Huadong Medicine obtained exclusive rights to develop, register, manufacture, and commercialize TTYP01 in China, with an upfront payment of RMB 100 million and milestone payments for development, registration, and sales capped at RMB 1.185 billion.

According to Auzone Biological, TTYP01 is an orally administered edaravone tablet independently developed by the company after seven years of overcoming technical challenges. Its active ingredient, edaravone, is an antioxidant neuroprotective agent that inhibits oxidative damage to brain cells, vascular endothelial cells, and nerve cells by scavenging free radicals and suppressing lipid peroxidation.

Through the development of solid dispersion technology and the integration of key excipients, Suzhou Auzone Biological Technology Co., Ltd. has successfully addressed challenges associated with edaravone, including poor water solubility, P-glycoprotein (Pgp)-mediated efflux, susceptibility of the active pharmaceutical ingredient (API) to oxidation, and inadequate stability. This innovation has increased oral bioavailability by dozens of times, significantly enhancing drug accessibility, convenience, and patient compliance, thereby holding promise for the treatment of acute and chronic conditions related to oxidative stress in the neurological field.

Currently, TTYP01 has completed Phase I clinical trials in China and Australia, respectively. TargetingAcute Ischemic Stroke (AIS)The indication has completed Phase III clinical trials in China, and a New Drug Application (NDA) is expected to be submitted to the NMPA in the near term. Meanwhile, Auzone Biologics plans to submit an application for pivotal registration clinical trials for AIS to the FDA in January 2025, with the U.S. marketing application expected to be filed by June 2026;Amyotrophic Lateral Sclerosis (ALS)The indication has been granted FDA Orphan Drug designation. The FDA has approved the completion of bioequivalence (BE) studies for this product, allowing it to be submitted for marketing approval; the U.S. New Drug Application (NDA) has currently been submitted; regardingAutism Spectrum Disorder (ASD) in ChildrenThe indication has received NMPA approval to conduct clinical trials and is currently in the subject enrollment phase; targetingAlzheimer's Disease (AD)Indications are expected to initiate an international, multicenter Phase II clinical trial in the first half of 2025. TargetingAcute Mountain Sickness (AMS)Preclinical studies for the indication have been completed, and an Investigational New Drug (IND) application will be submitted shortly; further development will continue for other oxidative stress-related diseases.

The results of the Phase III clinical trial of TTYP01 in China showed that the proportion of subjects with a modified Rankin Scale (mRS) score of 0–1 on Day 90 was significantly higher in the TTYP01 group than in the placebo group, with statistical significance, thereby meeting the primary endpoint; furthermore, the safety profile was favorable.

According to the announcement, compared with a 14-day in-hospital regimen of edaravone injection, TTYP01 can reduce the consumption of medical resources and is suitable for long-term self-administration by patients. A 28-day treatment course achieves free radical scavenging, thereby reducing tissue damage and cerebral edema, improving neurological function, and alleviating patient symptoms. Existing studies have shown that extending the duration of treatment helps prevent and manage post-stroke cognitive impairment and motor dysfunction.

According to Citeline’s analysis, the drug is projected to reach peak annual sales of $300 million in the U.S. market following its launch.

In 2019, Tanabe Mitsubishi’s edaravone injection was approved in China for the indication of “inhibiting the progression of functional impairment caused by amyotrophic lateral sclerosis (ALS)” and was included in the first batch of the National Key Monitoring List for Rational Drug Use.

In 2022, edaravone injection was included in the list of the seventh batch of China’s National Centralized Drug Procurement. As there are no intellectual property disputes regarding edaravone in China, numerous domestic companies have participated in its development. Pharmaceutical companies that have passed or are deemed to have passed the consistency evaluation include Simcere Pharmaceutical, Jilin Boda, Yangtze River Pharmaceutical Group, and Qilu Pharmaceutical, among others. According to the Insight database, edaravone has achieved substantial sales, ranking among the top four chemical drugs for the nervous system at public medical institutions in China, with sales peaking at RMB 5.33 billion in 2016. In recent years, with the implementation of new policies such as medical insurance cost containment and key monitoring lists across various regions, the sales growth of edaravone has begun to slow down, even experiencing negative growth in 2018; nevertheless, its sales revenue remained as high as RMB 5.026 billion.

According to Simcere Pharmaceutical’s financial report, revenue in the neurology segment reached approximately RMB 2.267 billion in 2023, representing a year-on-year increase of 41.0%. The flagship product, Xianbixin (Edaravone and Dexborneol Concentrated Solution for Injection), achieved domestic sales of RMB 1.846 billion. Edaravone and Dexborneol Concentrated Solution for Injection is one of the improved new drugs containing edaravone currently marketed in China. It was jointly developed by Nanjing Ningdan and Simcere Pharmaceutical and received approval in 2020 for improving neurological symptoms, activities of daily living, and functional impairment caused by acute ischemic stroke. Clinical data have demonstrated that it can significantly reduce brain nerve injury induced by acute ischemic stroke (AIS) and extend the existing therapeutic time window from 24 hours to 48 hours.

Another approved improved new drug of edaravone was approved in 2022, jointly developed by Nanjing Baixinyu Pharmaceutical Co., Ltd. and Beijing Tiantan Hospital, with independent intellectual property rights, and is the world's first sublingual medication for ALS to be marketed.