Rakuten Medical is a biotechnology company working in the space of photoimmunotherapy for the treatment of cancer.
What is the relationship between Rakuten Medical and Rakuten Group?
Rakuten Medical is an independent biotech company headquartered in San Diego, founded in 2010 as Aspyrian Therapeutics, Inc. Our connection with the Rakuten Group began in 2013, not through the company itself but through the investments of Mickey Mikitani, the founder, CEO and Chairman of Rakuten Group, Inc. He was drawn to our photoimmunotherapy platform, which we licensed from the US National Cancer Institute (NCI) that year. The technology, invented by Dr. Kobayashi, a chief scientist at NCI, caught Mikitani’s interest while searching for innovative cancer treatments for his father. Despite its early stage, Mikitani personally invested in our company, showing faith in the potential of photoimmunotherapy. Later, Rakuten Group also invested, and since 2019, we have operated under the name Rakuten Medical.
Can you explain the concept and uniqueness of photoimmunotherapy?
Photoimmunotherapy (PIT) is an innovative treatment that combines drug conjugates with light-illumination devices. The drug component involves a cell-targeting moiety such as an antibody to deliver a photoactivatable dye (IR700) to targeted cells, such as cancer cells, which is then activated by specific light illumination (690nm) to destroy the cell membrane. Unlike traditional chemotherapy, which uses toxins, PIT physically destroys cancer cells, leading to acute necrotic cell death. This rapid action not only targets cancer cells directly but also potentially boosts the patient's immune response by exposing the contents of destroyed cancer cells to the immune system. We believe this dual effect – direct cell killing and immune system enhancement – is unique to PIT and was developed from technology invented at the NCI.
Why focus on head and neck cancer for your leading product, ASP-1929? Are there plans to expand your pipeline?
Our technology is a platform technology, meaning we believe it can be adapted to target various cancers by switching the cell-targeting moiety.
We chose head and neck cancer for our initial focus due to the significant medical need and our access to the anti-EGFR antibody, cetuximab, for commercialization. Head and neck cancer, especially in its local regional and distal forms, presents a substantial unmet medical need. This strategic choice demonstrates our technology's potential in addressing significant medical challenges while laying the groundwork for future applications across different cancer types.
Why was ASP-1929 approved in Japan first? When shall we expect approval in the U.S.?
The approval of ASP-1929 in Japan was based on Phase 1 and 2 data generated in the United States, combined with a smaller study in Japan, satisfying the requirements of the PMDA (Pharmaceuticals and Medical Devices Agency). ASP-1929 has received Fast Track designation from the U.S. FDA, indicating our intent to enter the U.S. commercial market. Japan was chosen for initial approval due to its specific unmet medical needs for head and neck cancer and its system allowing early approval from Phase 1 and 2 data. This strategy also enabled us to demonstrate the technology's effectiveness and scalability in a regulated environment, paving the way for planned entry into larger markets like the United States. We are currently progressing through a global Phase 3 study, including the U.S., with the aim for market entry in the coming years.
You have recently closed your Series E funding. What comes next for you? Do you foresee an IPO anytime soon?
As the CEO, exploring all financing options is essential, especially as we progress through the late stage of development and begin generating commercial revenue in Japan. An IPO remains a possibility as we need significant capital to advance our programs. The choice between going public and staying private will be determined by what offers the best opportunity at the time. If going public presents clear benefits, we will consider it as a viable option.
Do you envision forming partnerships with bigger pharmaceutical companies given the uniqueness of your technology?
We are very open to partnerships that can advance our technology. Our collaboration with Hikma in the MENA region exemplifies our approach to partnerships, especially in markets where we lack direct access. Our goal is to fully realize the potential of our technology. Hence, we are seeking partners that share our vision for the technology's success.
If we speak again in three years, what progress do you hope to share about Rakuten Medical?
In three years, I aim to report significant advancements in our clinical studies, reflecting our mission to bring our technology to patients awaiting new treatments. Maintaining our company's spirit and direct connection to patient needs, healthcare providers, and families is crucial. We focus on the mission to conquer cancer, driven by the desire to make a meaningful impact on patients and the broader medical community. Success with our technology could encourage other companies to innovate, contributing to a diverse array of technologies to combat cancer. Our ambition is to inspire the industry through our achievements, fostering an environment where success breeds further innovation.
