BOSTON, Mass. -- By Lee Han-soo/Korea Biomedical Review correspondent -- Takeda Pharmaceuticals, the first Asian-based company to secure a spot among the 10 largest global pharma companies by sales, has redefined its research and development (R&D) strategy for groundbreaking discoveries and cutting-edge therapies, the company’s head of R&D said.

Korea Biomedical Review met with Andy Plump, President of R&D at Takeda, to talk about the company's achievements and its goals ahead in Boston, Mass., on June 6.

Plump joined Takeda when Christophe Weber assumed the role of CEO at Takeda in 2015.

"Since then, the company has embarked on a mission to revamp its R&D strategy,” he said.

Recognizing the need for change, the company made critical decisions to redefine its approach. Originally focused on the Japanese market and small molecules, the company shifted its strategy to concentrate on transformative therapies in three core areas: oncology, gastrointestinal (GI), and neuroscience.

The company also adopted a modality-agnostic approach, embracing different treatment modalities to address unmet medical needs.

The decision led to the complete overhaul of Takeda's research organization, adopting an externally facing model and establishing partnerships with over 200 biotech companies worldwide.

Such partnerships also include those with Korean companies, such as Bioinsight and LegoChem Biosciences.

Takeda's expansion into rare diseases after the acquisition of Shire in 2019 and its growing interest in data, digital, and technology (DD&T) further solidified its position as a tech-driven biopharmaceutical company.

According to Plump, Takeda's success extends beyond its global biopharmaceutical status, as it maintains strong ties to its Japanese roots and core values.

While Takeda has more employees in Massachusetts than in Japan and generates three times the revenue in the U.S., the company manages to uphold its international diversity and honor its heritage.

Strong pipeline addressing global unmet medical needs

Regarding Takeda's R&D pipeline, Plump revealed that the company's dedication to rebuilding has led to a focus on high-impact and transformative therapies.

"Takeda's emerging pipeline comprises approximately 40 new molecular entities, with 10 in late-stage development," he said.

Plump mentioned some of the promising candidates in the development phase.

Among the upcoming medicines in Takeda's pipeline is TAK-755, an enzyme replacement therapy targeting congenital thrombotic thrombocytopenic purpura, a rare genetic disease.

He highlighted the remarkable potential of this therapy to save lives, with significant differences observed in clinical trials compared to existing plasma exchange therapy.

Additionally, Takeda recently acquired the program TAK-279, a promising molecule that has the potential to become a best-in-class treatment for psoriasis and various inflammatory indications, he said.

Takeda acquired the drug from Nimbus Therapeutics in December of last year for $4 billion.

"The drug is currently in phase 2 trials, and we believe very strongly that the candidate will become a best-in-class treatment for psoriasis, which is the lead indication we are developing," he said. "What's really exciting is that TAK-279 has the potential to extend across five to maybe even 20 different inflammatory indications."

The inflammatory and immunologic disease space has become increasingly competitive in recent years, with Takeda joining esteemed players such as Janssen, AbbVie, Merck, Pfizer, Lilly, and BMS.

Plump attributed the market's appeal to the increasing understanding of the human immune system and its modulation. This knowledge proves advantageous for reducing immune responses in inflammatory diseases and enhancing them for cancer treatment.

He acknowledged the challenges associated with assessing the market value and commercial viability of drug candidates during the early stages of development.

"Takeda employs standard methodologies to understand the product profile, competitiveness against existing treatments, market size, and pricing," he said. "However, uncertainties such as safety events, efficacy, and disease prevalence often influence market uptake and competitiveness, necessitating ongoing adjustment."

Another challenge is the prevalence of the disease.

"We start developing a medicine after we believe we have understood the prevalence of disease," he said. "However, the number of patients that show up for clinical trials is much more than we predicted."

Data, digital, and technology to streamline Takeda's R&D process

As a result, Takeda has taken significant strides in leveraging data, digital, and technology (DD&T) in its R&D strategy.

"Artificial intelligence (AI) plays a crucial role in optimizing molecule design and development, with successful collaborations resulting in the creation of small molecules through in silico approaches," Plump said. "The recent acquisition of TAK-279, generated using algorithms in silico, exemplifies Takeda's commitment to DD&T-driven innovation."

Moreover, the company employs AI extensively in clinical trial site selection, protocol development, and decentralized clinical trials, aiming to enhance efficiency, diversity, and patient engagement, he added.

Notably, Plump stressed that choosing a site for a clinical trial is one of the biggest limiting factors and the largest cost driver.

"This becomes a problem when the selected clinical sites don't end up enrolling patients that we need, as it ends up slowing the study down and costing a fortune," he said. "Therefore, we use data and technology to identify where patients are and what a protocol should look like."

The company also conducts decentralized clinical trials as a solution. While 40 percent of the company's trials have elements of decentralization as of now, by the end of next year 100 percent of Takeda's relevant trials we'll have elements of decentralization, he added.

Plump stressed that when Takeda completes such a process, there could be instances where a patients participating in the trial will only need actually to go into the clinical site once a year, and everything else can be done remotely using data and digital.

"Takeda Pharmaceuticals continues to lead the way in transformative innovation, fueling hope for patients worldwide," he said. "With a robust R&D pipeline, a commitment to patient-centricity, and a focus on data, digital, and technology, Takeda is poised to redefine the future of biopharmaceutical advancements and drive positive change in the lives of patients."

Korea should focus on its own strength when building biocluster

During the interview, Plump shared insights on building a successful biocluster.

When asked about suggestions for the Korean government, which expressed interest in developing a biocluster akin to Boston's, Plump advised against replication.

Instead, he recommended leveraging Korea's unique strengths while studying successful clusters worldwide.

Plump cited Singapore as an example, highlighting their transition from aspiring to innovative biotech cluster to excelling in manufacturing.

He emphasized Korea's abundant talent pool in life sciences, technology, data, and digital technology, emphasizing the importance of integrating these strengths into health sciences to remain ahead of future trends in biocluster development.