How a Japanese Biotech Company Is Innovating Drug Development Technologies – SPONSOR CONTENT FROM THE GOVERNMENT OF JAPAN – Harvard Business Review

Medical researchers have leveraged technology to create major breakthroughs in the past few decades, accelerating the understanding of diseases, and their causes and treatment.

Our accumulating knowledge also has accelerated the ability to translate science into practical therapies, but there are still many challenges: while researchers seek the right drug compounds that can target and deliver treatment for specific diseases, traditional drug innovation models can be slow and come with high costs.

Japans rising biotech company, PeptiDream, is tackling these issues, deploying a unique proprietary drug development technology and an innovative business model that will further research on and development and manufacture of peptides to deliver new medical therapies.

We really want to be a drug discovery engine, says CEO Patrick Reid.

Until recently, most advances in drug delivery have focused on small-molecule and large-molecule drugs, also known as antibodies. But now macrocyclic peptides are emerging as an important new avenue.

How are peptides different? Both small- and large- molecule drugs come with advantages and limitations. The small molecule drugs are chemically synthesized in a lab and taken as a pill or capsule, so the active ingredient is easily absorbed into the bloodstream.Because they are small, molecules can penetrate cell membranes, making these drugs highly effective. But they can be unstable and they break down in the body, creating unwanted side effects. Formulating these drugs to take on specific new targets also can be slow and expensive.

Protein-based therapeutics (large-molecule drugs) aremade by using living cells. They typically are not pills, but instead must be injected or infused. These large- molecule therapies, unlike the smaller-molecule drugs, cannot penetrate cells. But these drugs are easier to design for specific targets typically a cell-surface receptor on the outside of the cell. However, these therapies cannot reach all required targets, and they can stay in the body too long causing side effects.

Enter peptides, compounds that consist of amino acids linked together and can be synthesized in the lab. Pioneering research by Suga Hiroaki, PeptiDream co-founder and professor at the University of Tokyo, established a way to ensure that a new kind of peptide compound can remain stable in the body and find a range of therapeutic targets with high specificity. They can also be broken down by and cleared from the body with greater specificity, making them an important new development in pharmaceuticals.

And these macrocyclic peptides can be combined, using a much larger set of amino acids than occur in nature giving researchers the ability to experiment with many more combinations. PeptiDreams Peptide Discovery Platform System (PDPS) is a proprietary technology that allows drug researchers to make trillions of peptide libraries. Reid describes PeptiDream as platform company that enables his researchers and others to make the process of discovering hits the starting point for developing drugs more efficient.

We are not simply developing a single drug and trying to bring that all the way to approval; we are championing and developing an entirely new class of molecules, Reid says.

The platform has created an unusual set of collaborations for PeptiDream, whose drug discovery partnerships have included Merck, Bayer, Genentech and Novartis. This collaborative network has accelerated peptides development, Reid says, creating a large wave of compounds that should move into the clinic in the next few years.

Our network of partners has allowed PeptiDream to function as company ten to twenty times its actual size, he says. With more than 100 discovery programs in parallel across a wide range of diseases, targets and administration routes, we are expanding the knowledge, understanding and appreciation of these molecules in therapeutics and diagnostics and more.

It also was crucial that PeptiDream, as a startup, was able to focus on developing the platform for peptide drug discovery, something large pharmaceuticals had not done because of the cost and the long, uncertain time horizon. Japan embraced PeptiDream, initially as a largely bootstrapped company, and then when it went public in 2013, Reid says.

In the U.S. and Europe, we probably would have been pressured to borrow funds in order to grow faster, he says. Many companies in the U.S. with internal pipelines fail due to time and pressure constraints. They burn a lot of money very quickly.

PeptiDream is now a $7 billion company and is also a founding investor in a contract manufacturing company, PeptiStar. Collaboration with other companies is crucial, says PeptiStar CEO Kameyama Yutaka, as new ecosystems for research, manufacturing and supply of peptide drugs are developed. In fact, PeptiDream, together with other co-founding investors Shionogi and Sekisui Chemical, has attracted additional ten investors as active R&D collaborators.

In order to accelerate the practical application and market creation of peptide therapeutics as next-generation drugs beyond biopharmaceuticals, the Japanese government also supports PeptiStar, providing 9 billion-yen (about $83 million) grant as part of the governments program Cyclic Innovation for Clinical Empowerment, under the National Healthcare Policy. The money will allow PeptiStar, established in 2017, to become a leader in both scientific and business process innovation, says Kameyama.

The current capacity of peptide manufacturing is limited, and it could be a big bottleneck of peptide medicine developments, he says. This quick fundraising will accelerate the development and commercialization of our ability to prepare the peptide compounds. And the support they have given us will also encourage many other partners in the important development of peptides.

Peptides have not been around very long, and as with any new technology, there is room for improvement, including costs, Kameyama says. We want to make production cheaper and higher quality, and collaboration is a competitive advantage. If a company established its own manufacturing facility, it would take time and money. But with a joint venture like ours, the cost and the sharing of technology and knowledge are very different.

Both Reid and Kameyama credit the Japanese research and business ecosystem with their success. Professor Sugas breakthrough work is just one spinoff of innovation coming from Japanese universities, where a pool of highly skilled research workers has developed.

Japans challenge to create peptide drug market continues. To learn more click here.

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Note: All Japanese names in this article are given in the traditional Japanese order, with surname first.

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How a Japanese Biotech Company Is Innovating Drug Development Technologies - SPONSOR CONTENT FROM THE GOVERNMENT OF JAPAN - Harvard Business Review