ASX-listed Cynata Therapeutics, a Melbourne-based, clinical-stage biotech company specialising in cell therapeutics, has produced trial results confirming the initial success of its Cymerus mesenchymal stem cells, or “MSCs” in restoring cardiac function following a heart attack. Foreshadowing what could be a remarkable breakthrough, a study conducted at the Westmead Institute of Medical Research in Sydney demonstrated the treatments ability to enhance the recovery of blood supply to the damaged heart through the generation of new blood vessels.
Cynata said the positive efficacy data support its previous claims regarding the superior therapeutic effects of Cymerus MSC treatment when compared to both conventional bone marrow-derived MSC treatment and placebo.
The new data demonstrated the Cymerus MSC treatment enhanced the recovery of blood supply to the damaged heart in two key areas developing twice the number of new blood capillaries.
Cynata will now continue to work with relevant parties to determine the next steps for this program and the broader clinical development of the treatment.
With the benefit of this further data we are now able to better understand the beneficial effects of our Cymerus MSCs in this model of heart attack. It is particularly notable that the latest results are consistent with the greater benefit demonstrated by Cymerus MSCs over BM-MSCs in the earlier studies of heart function. Given the scalability and consistency advantages of the Cymerus MSC manufacturing process over conventional processes, the potential therapeutic advantages shown here are particularly interesting as a putative treatment for post heart-attack cardiac damage.
These latest findings only add to the already impressive list of treatments the Dr Ross Macdonald-led Cynata has built up since its inception back in 2011. The company has already demonstrated the utility of its Cymerus MSC technology in preclinical models of asthma, diabetic wounds, sepsis, acute respiratory distress syndrome, “ARDS” cytokine release syndrome and pulmonary fibrosis.
Equally, Cynata’s lead product candidate, CYP-001, has met all necessary clinical endpoints and demonstrated positive safety and efficacy data for the treatment of steroid-resistant acute graft-versus-host disease, or “GvHD” in a Phase 1 trial. The company now plans to advance its Cymerus MSCs into Phase 2 trials for severe complications arising from COVID-19, acute GvHD and a Phase 3 trial in osteoarthritis.
GvHD is a debilitating disease which can occur after a bone marrow transplant operation when the donor’s immune cells attack the recipient of the transplant. Cynata said corticosteroid therapy is typically effective in 50 per cent of patients, but mortality rates can be as high as 90 per cent if the steroid treatment proves non-effective.
The Phase 1 clinical trial of CYP-001 on 15 patients with steroid-resistant, acute GvHD showed an improvement in the severity of the condition in 13 out of the 15 recipients by day 100, giving an overall response rate of 87 per cent.
The Australian-based, disruptive regenerative medicine products company has teamed up with Fujifilm Corporation of Japan to help develop and commercialise a number of Cynata’s products. In 2019, it granted Fujifilm an exclusive, worldwide license to commercialise CYP-001 for the prevention and treatment of GvHD in humans.
Dr Macdonald said in an interview last week that the deal with Fujifilm could lead to the global marketing of CYP-001 with a potential future financial reward of up to $100 million heading Cynata’s way.
Cynata said Fujifilm would be required to pay a US$3m upfront licensing fee, followed by milestone payments of up to $60m which could ultimately lead to ongoing, double-digit royalty payments.
According to Cynata, MSCs have huge therapeutic potential for numerous unmet medical needs which have already been demonstrated and widely documented. But Cynata’s unique, trademarked Cymerus technology addresses many of the complexities and challenges of manufacturing these stem cells at a commercial scale.
The trademark ‘Cymerus’ refers to the patented process of generating cell-based products from intermediate cells, known as mesenchymoangioblasts, or “MCAs” which in turn are derived from induced pluripotent stem cells, or “iPSCs” This technology was originally developed at the University of Wisconsin-Madison, or “UWM” a world-renowned leader in stem cell research.
Core patents underpinning Cynata’s Cymerus technology are owned by the Wisconsin Alumni Research Foundation which has automatic rights to all intellectual property arising from the efforts in ‘next-gen’ stem cell research at UWM.
Cynata was granted an exclusive worldwide license to the relevant patents and has also been granted a license to a broad portfolio of patents held by Cellular Dynamics International which is now a Fujifilm company. It has also filed patent applications on inventions it has made in developing the Cymerus technology.
MSCs have huge therapeutic potential for numerous unmet medical needs but have traditionally been difficult to gather from humans, despite being found throughout the body.
By using the unique manufacturing platform, called Cymerus, which enables the company to economically produce therapeutic MSCs at scale with consistent quality, Cynata said it was able to overcome the inherent challenges in first-generation methods of manufacturing MSCs for therapeutic use.
This in turn means it can manufacture robust and consistent MSCs in an economically viable process and all under Good Manufacturing Practice requirements.
First-generation methods of MSC production relied on the isolation of MSCs from tissue donations, for example, bone marrow or fat. This approach was associated with a number of important limitations, including dependence on donors and the variability between them, the relative scarcity of MSCs in adult tissue and the low proliferative capacity of adult stem cells.
Cynata also said the loss of potency and reduced efficacy was associated with extensive MSC culture expansion, undertaken in an effort to maximise the number of MSCs from each donation.
Conversely, since iPSCs can proliferate indefinitely, and MCAs themselves can expand into extremely large quantities of MSCs, Cynata expects to be able to manufacture all of the cells that it will ever need from a single Master Cell Bank of iPSCs – derived from a single donor.
Cynata was originally founded in October 2011 by two of the inventors of the Cymerus technology; Professor Igor Slukvin and Dr Maksym (Maxim) Vodyanik in collaboration with Australian technology entrepreneur, Dr Ian Dixon.
In November 2013, Cynata was acquired by ASX-listed company, EcoQuest Limited, which subsequently changed its name to Cynata Therapeutics Limited. The Cynata founders all still hold shares in the company and Professor Slukvin and Dr Vodyanik remain closely involved with the company’s product development activities.
Cynata said the Cymerus technology has several characteristics which make it ideal for the commercial development of cell-based therapeutics. Most critically, the Cymerus manufacturing process ensures the cells for therapeutic use can be produced in virtually limitless quantities from a single donor.
This means that Cynata will not be required to routinely seek out fresh donors to meet its manufacturing demands. This has the potential to create a new standard in the emergent arena of regenerative medicine and stem cell therapeutics and provides Cynata with both a unique differentiator and an important competitive position.
Management said the main advantages of the Cymerus Technology were versatility, manufacturing scalability, cost point, clinical predictability, and safety of its MSC product range.
The results of recent pre-clinical and clinical studies into the regenerative qualities of its therapeutic MSC products look to be lining up in support, echoing the opening line from the 1970s’ TV show, The Six Million Dollar Man: “We have the technology, we can rebuild him.”
With a shareholding in Cynata at just a tad under 7 per cent, Fujifilm must also think so.
NB: This article is for general financial markets news purposes only and is not to be taken as an endorsement of, or advertisement for any individual product, medicine, or drug.
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