Precision Medicine and its Impact on the Global Therapeutic Landscape

Frost & Sullivan’s Senior Research Analyst, Aarti Chitale looks at the sizeable impact that precision medicine, especially regenerative medicine, is already having on the global therapeutic landscape, and how the Asia-Pacific region is positioning itself at the forefront of this trend.

Providing the right treatment/ right drug at the right time to the right patient is the key premise of Precision Medicine. As the name suggests it is a field of science which promises individualized and precise diagnosis and treatment in a targeted manner, therefore carrying the potential of curing some of the most challenging diseases. Up until the last decade, not many people knew about personalized medicine. However, incredible advances in genomics, such as whole-genome sequencing using cutting edge technologies like Next-generation sequencing, have turned the tables around. These improved abilities, where the treatment is designed around the patient, are altering the very paradigm of drug therapies.

The global cell and gene therapy market is estimated at a value of over $3 billion in 2018 and is expected to surpass $18 billion by 2025, according to Frost & Sullivan estimates

With the advent of precision medicine, companies the world over are developing ground-breaking curative therapies, which promise a root cause analysis to reach the origin of a condition and thereby cure it. Regenerative medicine is one such field of precision medicine, which is witnessing this transformation in real-time, catering to some of the most critical unmet medical needs including certain cancers, rare and orphan diseases and pediatric/in-born genetically induced defects.

Although there is an equivalent focus on small molecule therapies which are expected to continue to dominate the majority of the pharmaceutical landscape, there is a growing demand for these novel therapies. Post-2000s, the industry has witnessed a major shift from traditional medicine to biologic therapies resulting in a surge of research projects. At present, there are more than 300 cell and gene therapies in clinical development, targeting over 100 diseases.

The global cell and gene therapy market is estimated at a value of over $3 billion in 2018 and is expected to surpass $18 billion by 2025, according to Frost & Sullivan estimates. A robust growth rate of over 30% is a result of aggressive research and development activities, primarily in the areas of stem cell therapies, CAR-T Cell therapies and gene therapies. Novartis and Gilead Sciences were the first companies to launch products (Kymriah and Yescarta, respectively) in the United States in 2017 followed by approvals in Europe, China and Japan in 2018.

Bolstering Regenerative Medicine in Asia Pacific by Acquiring Advanced Infrastructure and Technological Expertise

Over the last few years, there has been a shift from western countries towards APAC for drug development and manufacturing activities. China, Japan, South Korea and India are emerging as the key centres for cell and gene therapy research. China is emerging as one of the fastest-moving nations in this context by adopting the latest manufacturing technologies supporting “Small Volume High Value Manufacturing”. The country has well established single use facilities acquired from industry majors such as GE Healthcare. Several biopharma companies in China are already using GE’s KuBio™ Manufacturing facility and plan to acquire several more Flexfactory™ platforms for cell therapy development.

Another example of GE’s investment in the region includes their collaboration with Singapore’s Agency for Science, Technology and Research (A*STAR) to support research and development in advanced medical technologies that will deliver sustainable healthcare solutions for patients in Asia. Many other major OEM’s such as Merck Sereno and Siemens Healthineers have been investing in the region, supporting humungous manufacturing automations. In March 2018, Merck was named the “Best Bioprocessing Supplier for Single-use Systems in Asia” at the Asia-Pacific Bioprocessing Excellence Awards.

Alongside automations, companies based in the region are attracting better investments in the form of private equity and joint ventures. One of the most recent examples is the $85 million series B funding received by Suzhou, China-based immune cell and gene therapy startup, Gracell Biotechnologies. The investment was led by Singapore based government investment firm Temasek Holdings. The funding amount is to be used for clinical development of their modified immune cells called T-cells, which have already completed the pre-clinical phase.

All these activities together are propelling investments in the Asia Pacific region, making it one of the most profitable markets for cell and gene therapy development.

Re-Defining Regulatory Policies to Match the Changing Market Needs

Taking into account the rising interest in the region, local regulatory bodies are making necessary amendments in their policies. For example, the Japanese Government implemented the Pharmaceutical and Medical Devices Act (PMD) and Safety for Regenerative Medicine Act (SRM) in 2014, which allows companies from the Western region to invest into Japan for regenerative medicine research. Furthermore, the country has also put into place an expedited regulatory approval process for these therapies which significantly reduces the approval timelines from three years to one year, while allowing for conditional approvals, especially in case of non-availability of therapies.

The Chinese FDA now accepts foreign clinical trial data for the drug approval process in China. Also, Taiwan’s National Cheng Kung University (NCKU) in March 2013, announced the setup of a new Center for Pharmaceutical Regulatory Science, which is intended to ‘promote the administration and education of pharmaceutical regulation in Taiwan.’ These activities are further attracting investors to conduct research in the region.

Adoption of Collaborative Drug Development Approach Supporting Small to Mid-Segment Participants

The regenerative medicine landscape is unique in itself with a maximum number of small to mid-segment players possessing specific therapeutic expertise, especially across the APAC region. These players, alongside existing market leaders, are largely responsible for propelling the research activities. Although pipelines are surging, these players lack the required funding or facilities to continue their efforts, therefore encouraging them to adopt collaborative models for drug development. Some of the most prominent models are risk-sharing and fast to market model (outsourcing) which not only reduce the costs but also decrease the associated risks. Geographic expansion is another key driver propelling collaborations in the industry.

Most Recent collaborations

In early 2019, Takeda announced multiple cell therapy collaborations to speed-up the discovery of next-generation cancer immunotherapies. Organizations including Memorial Sloan Kettering Cancer Center (MSK) alongside industry players such as Crescendo Biologics and Noile-Immune Biotech Inc. will now work with Takeda to develop novel therapies for cancer treatment.
Merck’s collaboration with a Chinese Biotech company GenScript, for plasmid and viral vector manufacturing is an example for gene therapy partnership which aims at accelerating the industrialization and commercialization of cell and gene therapy in China.
Novartis collaborated with CBMG in 2018, for the local production of their cutting edge CAR-T Cell therapy Kymriah, potentially expanding further into the South East Asia region. Moreover, CBMG is also developing its own range of cell therapies including their proprietary CAR-T cell therapy, CD-19, which is in early stage development.

Some of the key players with upcoming novel therapies in the region include –

Cynata Therapeutics, Carlton, Australia: Cynata has developed a unique Cymerus^TM technology platform, for commercially scalable and cost-effective production of MSCs from induced Pluripotent Stem Cells (iPSCs). Their lead product CYP-001 with an Orphan Drug Designation from the FDA to treat GvHD, is considered as a promising alternative to existing therapies by reducing the overdependence on steroids.
Medipost, South Korea: Medipost is amongst the earliest players in stem cell therapy segment, not just in the APAC region but globally. It holds a competitive edge with its product Cartistem, which is the world’s first allogeneic stem cell drug approved for commercial use by the Ministry of Food & Drug Safety in Korea. Also, their cord blood bank CELLTREE is the leading umbilical cord blood (UCB) storage bank as well as the country’s largest HSC transplant program provider. A strong pipeline comprising of Pneumostem (Granted orphan drug designation in the US and the EU) and Neurostem (Early stage development for Alzheimer’s disease) and a collaborative business approach supports Medipost in targeting both domestic and global markets.
JCR Pharmaceuticals, Tokyo, Japan: Founded in 1975, JCR is one of the leading pharma companies in Japan and aims to strengthen its position in the regenerative medicine by building on the success of TEMCELL from Mesoblast. Their recent co-development and licensing agreement with Teijin for JTR-161/JR-161 will provide JCR the opportunity to manufacture and supply the commercial products in Japan.

From being a generic and biosimilar oriented market to emerging as a hub of cell and gene therapy research, Asia Pacific has come a long way across the global pharma landscape. With several ground-breaking manufacturing technologies gaining traction, local players are adopting a collaborative business model to gain a competitive edge, thus supporting the rise of APAC as a primary location to support the thriving regenerative medicine landscape.

The Road Ahead

The recent success of multiple cell and gene therapies is supporting the industry transition towards personalized medicine and value based care there by propelling the adoption of unique business models to withstand the growing demand. As a result, a plethora of opportunities are set to open up for market players in the APAC region to be the ultimate destination for cost efficient and faster clinical research. Moreover, the success of these therapies offers promising opportunities for previously untreated diseases areas. Hence, cell and gene therapy truly holds the potential to be the ultimate medical panacea.