This is an exclusive interview with Dr. Paul Wotton, who joined Cynata Therapeutics Board of Directors in June, 2016. He was previously President and CEO of Ocata Therapeutics, Inc. (NASDAQ: OCAT) joining the company in July 2014 and managing it through an all-cash take-over by Astellas Pharma valued at $379 million. Prior to Ocata, Dr. Wotton had served as President and CEO of Antares Pharma Inc. (NASDAQ: ATRS), since October, 2008. Prior to joining Antares, Dr. Wotton was the CEO of Topigen Pharmaceuticals and prior to Topigen, he was the Global Head of Business Development of SkyePharma PLC.
Dr. Wotton is also a member of the board of Vericel Corporation, an American company developing autologous cellular therapies and also past Chairman of the Emerging Companies Advisory Board of BIOTEC Canada. Dr. Wotton received his Ph.D. in pharmaceutical sciences from the University of Nottingham and an MBA from Kingston Business School.
Enjoy Dr. Wotton’s insights in his interview below.
Interview with Dr. Paul Wotton, Board Member for Cynata Therapeutics and Previous CEO of Ocata Therapeutics
Cade Hildreth: What is your personal/professional background?
Dr. Paul Wotton: I originally trained as a pharmacist, undertaking my degree at University College in London. After completing my Ph.D. at Nottingham, I went straight into the industry with my first position in the development labs at Merck’s operations in the UK (known as Merck, Sharp and Dohme). It was there, and subsequently at Abbott, also in the UK, that I developed a particular interest in the corporate side, moving quickly to a series of positions in corporate/business development with companies in Europe and in the USA.
In 2004 I moved to Canada to take up the position of President and CEO at Topigen and then in 2008 I moved to the US to take up the role of COO and then eventually President and CEO at Antares Pharma.
My most recent position was President and CEO of Ocata Therapeutics, a cell therapy company, where in early 2016 I oversaw the successful acquisition of that company by Astellas Pharma in a $379 million all cash transaction.
Cade Hildreth: What role did you play at Ocata and in the recent sale of the company to Astellas Pharma?
Dr. Paul Wotton: As President and CEO I was directly involved in the original interaction with Astellas and the subsequent negotiation and execution of the sale.
Cade Hildreth: What piqued your interest about becoming involved with Cynata Therapeutics?
Dr. Paul Wotton: I see Cynata’s Cymerus™ technology as providing a new generation approach to the therapeutic use of stem cells, in this case mesenchymal stem cells (MSCs). It is clear from the great interest in MSCs, as evidenced by the numerous clinical trials of MSC therapeutics now underway, that successful commercialisation of these therapies will demand a robust, consistent and industrialisable manufacturing process.
I see Cynata’s technology as being a critical success factor for success in this space as a result.
Cade Hildreth: How does Cynata’s Cymerus technology overcome the complexities and challenges typically associated with manufacturing stem cells at a commercial scale?
Dr. Paul Wotton: The challenges with current methods that derive MSCs from primary sources such as bone marrow, adipose or other tissues are two-fold. First there is the requirement for multiple donors, along with the attendant issues of the source material being accordingly different. The second is the mandatory requirement of massive in culture expansion of the purified donor-derived starting material.
MSCs are scarce in tissues and the required expansion from individual donations to manufacture the millions of doses required for economically important diseases represents a significant challenge. There is an emerging literature highlighting the potential negative impact of such manufacturing processes on the quality and potency of the cells.
The advent of iPSC technologies, particularly now where non-integrative reprogramming is feasible, has opened up an enormous opportunity for medical applications using these technologies. In the case of Cynata’s Cymerus technology, which uses iPSCs to generate MSCs, the iPSC is derived from a single, one-time donor, meaning the cells are all genetically identical, and the expansion required for commercial manufacture is effected at the level of the iPSC.
Differentiation of the expanded iPSC pool is the final step in the industrial process. iPSCs have been shown by many investigators to be essentially capable of infinite self-renewal, so expansion at this level and subsequent differentiation is the perfect solution to current challenges of commercial manufacture of MSC therapeutics.
Cade Hildreth: What strategic advantage(s) does Cynata have within the stem cell market?
Dr. Paul Wotton: Cynata’s technology comes from one of the world’s leading and most productive centers of stem cell research, the University of Wisconsin – Madison. Moreover, as the company is developing a platform technology it has multiple shots on goal.
Finally, because a substantial amount of research and clinical development has already been undertaken for MSCs, Cynata is able to leapfrog that existing body of clinical data with a much lower risk manufacturing proposition, rather than discovery per se.
Cade Hildreth: The RIKEN Center for Developmental Biology (CDB) recently announced it will be resuming its retinal iPSC study in humans using allogeneic cells, with the first patients being treated as soon as next year.
As Cynata is positioned to launch the world’s first allogeneic iPSC clinical trial this year using iPSC-derived MSCs for graft-versus-host disease (GvHD), what impact does this announcement have on Cynata?
Dr. Paul Wotton: Japan is playing a leading role in the development of this new generation of therapeutic products and companies like Astellas and Fujifilm are leading the way. Allogeneic cells are the only way, in my opinion, to make cell therapy viable as a commercial proposition for the treatment of the population at large.
Cynata sees Japan as a very progressive and dynamic market for its technologies and is working actively to develop partnerships in that country. The data driven progress of the Riken program highlights the viability of using iPSCs to derive therapeutic cells.
Cade Hildreth: What is the global significance of Cynata’s upcoming clinical trial for GvHD?
Dr. Paul Wotton: This trial will be the initial first-in-man study for Cynata’s Cymerus MSCs. It is also on track to be the first time an allogeneic, iPSC-derived product has been used in humans. As such, it is significant not just for the Company, but also for the iPSC movement worldwide.
GvHD can be a particularly devastating disease and MSCs have been shown in several clinical studies to offer a potential treatment for the condition. Should Cynata’s product show positive data in the clinical trial, noting that safety is the primary endpoint, it will provide important evidence that our technology can progress to address key unmet medical needs.
Cade Hildreth: In your opinion, what is the market potential for mesenchymal stem cells (MSCs) relative to other stem cell types?
Dr. Paul Wotton: MSCs have been shown to have profound immunomodulatory properties. This characteristic, together with the fact that they are potentially immune-privileged, has led to the investigation of their clinical utility in a wide range of disease targets and modes of delivery.
Other types of stem cell tend to be relevant to a specific disease and moreover, if engraftment is required for their continued function, then matching the stem cell with the donor, and/or use of immunosuppressant drugs, will be required. Potential avoidance of the need for immune suppression is a major factor for allogeneic cell therapy products to be used widely.
Cade Hildreth: From your experience within the regenerative medicine sector, what trends have you observed that may benefit Cynata?
Dr. Paul Wotton: The increased interest by large pharmaceutical companies in cell therapy coupled with the forward thinking approach of regulatory bodies worldwide are trends worth noting. There is no doubt that the existing processes for producing therapeutic MSCs have allowed medical science to pave a path to eventual widespread clinical use of MSCs in a large range of disease targets.
The availability of cost effective manufacturing technology was a considerable unmet need in this segment and Cynata’s technology platform provides a new, robust, economical and practical stem cell production method that lends itself to commercial production.
Cade Hildreth: What is your opinion of Cynata’s partnering strategy for generating company value (upfront payments, milestones, and royalties)?
Dr. Paul Wotton: Cynata has a clearly stated goal to partner its technologies and has begun to execute on that strategy. Because it is a platform, the Cymerus technology offers the ability to enable multiple partners in multiple geographies to access the technology for their own particular disease target or therapeutic category. As with many enabling technologies I see the potential for numerous partnerships in this area.
Cade Hildreth: How does Cynata’s cost-savings advantages impact the types of industry partners Cynata can attract?
Dr. Paul Wotton: Reduced to its basic elements, only three things matter for the commercial success of a therapeutic product: it must be safe, it must be effective (especially vis-à-vis competitors), and it must be able to be manufactured consistently and economically. Without the latter, a product is not a product at all.
Experienced pharmaceutical and big biotech companies clearly recognise this and as MSCs prove themselves to be safe and effective medicines then I expect we will see a race to access enabling manufacturing technologies. Cynata is perfectly placed with its Cymerus technology to take advantage of that.
Cade Hildreth: What types of future partnerships, collaborations, or investments interest Cynata?
Dr. Paul Wotton: Cynata is firmly focused on its business strategy of partnering and as such is actively seeking and dealing with partners, both potential and current. At the same time, the Company has also been active in ensuring that its MSCs are being investigated for utility by leading scientists worldwide, just as the current program with Harvard/MGH is intended to do. Having its cells used in this way, and becoming the subject of publications, is an important validation of the utility and relevance of the MSCs produced using the Cymerus technology.
Cade Hildreth: Thank you for the honor of doing this interview and for sharing your insights on Cynata’s unique Cymerus technology for manufacturing iPSC-derived MSCs.
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