After failing to attain reimbursement for its Amyotrophic Lateral Sclerosis (ALS) treatment NEURONATA-R® in Korea, Corestem has its sights set on the US market and an Investigational New Drug (IND) approval from the FDA.
Dr Kyung Suk Kim, Corestem’s CEO, recently spoke to PharmaBoardroom to give an update on the company’s progress in recent years and its pivot towards international markets.
She begins, “On our journey to develop NEURONATA®, we have reached a number of key milestones. In 2014, NEURONATA-R® became the first commercially licensed stem cell therapy for ALS, receiving conditional approval. Also, we finally received designated orphan drug status from the FDA in 2018 and from the European Medicines Agency (EMA) in 2019.”
We plan to first enter countries which are highly active in their policy considerations of innovative treatments for patients with rare diseases, such as the US.
“Since the conditional approval in 2014, NEURONATA-R® has been treating more than 300 patients and has demonstrated its safety and efficacy by the Phase II results. In particular, the number of foreign ALS patients travelling to Korea to receive treatment is on the rise, as the world’s first ALS treatment approval and the Phase II report has attracted the attention of the academic side.”
However, it has not been all plain sailing. Dr Kim notes that, “In early 2019, we tried to get reimbursement for NEURONATA-R® under the Korean national health insurance system (NHIS). We tried to negotiate at the production cost level to benefit patients as much as possible but were not covered by insurance. This outcome led to the realization that achieving reimbursement for NEURONATA-R® in Korea was unlikely. Following subsequent negotiations with the Health Insurance Review & Assessment Service (HIRA), we changed our strategy, instead prioritising the global market.”
Dr Kim continues, “In order to enter the global market, the North American market was essential, and the level of data we had in the United States was really important. Therefore, we met the US Food and Drug Administration (FDA) in September 2019 for a preliminary Investigational New Drug (IND) meeting, briefly discussing the clinical design and protocol. Subsequently, we made our IND application for Phase III clinical trials. We have since received several deficiencies from the US FDA and are in the process of responding. We remain confident that we can resolve these issues and finally receive our IND approval.”
ALS is a disease where motor nerve cells, which move the skeletal muscles of the body, deteriorate and eventually die. This gradually results in the paralysis of skeletal muscles, progressing across the entire body. In the latter stages of the disease, a patient cannot swallow or breathe properly, relying on the aid of gastrostomy and a respirator. The prevalence rate is 4-6 in 100,000 per year, which has increased with an ageing population.
Because of the nature of NEURONATA-R®, which works as an autologous bone marrow mesenchymal stem cell therapy, the treatment has a shelf life of just 48 hours, causing issues in terms of distribution outside of Korea. For this reason, Dr Kim underlines the importance of finding reliable manufacturing and distribution partners as the firm continues its internationalisation push.
She states, “Corestem is starting from a position as a bio venture company, now becoming more innovative and forward-thinking. We plan to first enter countries which are highly active in their policy considerations of innovative treatments for patients with rare diseases, such as the US. However, it is true that considering the size of the company, it is hard to penetrate one market alone. Thus, we have been looking for partners to license-out our technology after receiving IND approval. Fortunately, some of the major players within the top 50 pharma companies have already declared an interest in collaborating. They are awaiting the outcome of our current clinical trials before finalising any licensing-out agreement.”
Dr Kim adds, “In the future, we also have ambitions to form partnerships in Europe. We are open to collaborations with different partners in different regions, rather than a comprehensive global deal.”
“Since the treatment only lasts up to 48 hours after manufacturing, shipping or flying the product from Korea, as is routine with a chemical treatment, is unrealistic. Consequently, the potentials of manufacturing NEURONATA-R® in accordance with current Good Manufacturing Practice (cGMP) is the most critical point in discussions with potential partners. Furthermore, It is important to clarify, prior to any agreement, which party will control the facility and how it can be guaranteed that the facility will be able to logistically reach the whole of the USA according to GDP (Good Distribution Practice) in effect.”
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