What is Cynata’s relationship with FUJIFILM?
In September 2021 Cynata entered into a strategic partnership agreement with FUJIFILM Corporation of Japan. This agreement included core terms for Fujifilm to provide clinical and commercial manufacturing services for Cynata’s Cymerus™ MSC products. Fujifilm remains a shareholder in Cynata.
Is Cynata engaging with other potential partners?
Yes, Cynata maintains an active business development program aimed at attracting commercial partners across multiple indications and geographies. The company's completed clinical trials and ongoing pipeline, including Phase 2 and Phase 3 studies, provide important validation of the Cymerus technology and create opportunities for partnerships. Commercial discussions typically involve multiple stages, including confidential disclosure agreements, due diligence, and term sheets before definitive agreements are reached. Cynata continues to engage with potential partners following positive clinical results, particularly from its recently completed diabetic foot ulcer trial.
What is the regulatory pathway in countries such as the USA?
Each country has its own regulatory process for governing the conduct of clinical trials and the granting of marketing authorisations for medical products. In selecting the jurisdictions in which Cynata has pursued clinical development it has given careful consideration to the quality of the relevant regulatory jurisdiction, the time and costs to satisfy the regulatory requirements, the ultimate pathway to market and the value thereof, the availability of patients and the particular standards of care of the relevant disease. The USA is the most commercially attractive market for pharmaceutical products and Cynata has engaged with the relevant regulatory agency, the US Food and Drug Administration (FDA) and in 2022 successfully obtained clearance of an Investigational New Drug (IND) in respect of our Phase 2 clinical trial in acute graft-versus-host disease (aGvHD).
Clearance of Cynata's IND application confirms the FDA is satisfied with both the clinical and preclinical data as well as the manufacturing and quality control data on our product that we submitted in support of this application.
This was transformative for Cynata as it provides a gateway in the USA to potential further clinical targets and is a critical validation step for Cynata’s ongoing commercial partnering activities.
What is Cynata’s Cymerus™ technology?
The trademark Cymerus™ refers to the patented process of generating cell-based products from intermediate cells, known as mesenchymoangioblasts (MCAs), which in turn are derived from induced pluripotent stem cells (iPSCs). This technology was originally developed at the University of Wisconsin-Madison, WI, USA.
At present, Cynata is focused on the production of mesenchymal stem cell (MSC)-based products using the Cymerus technology.
Why are iPSCs important for regenerative medicine?
iPSCs (induced pluripotent stem cells) represent a breakthrough in regenerative medicine because they offer similar capabilities to embryonic stem cells (ESCs) without the ethical concerns...
This discovery is revolutionary because it eliminates the need for constantly finding new donors for cell therapies. Instead, a single donor can provide cells that can be used to create unlimited therapeutic cells. This makes iPSCs ideal building blocks for developing new cell-based treatments, which is why many researchers and companies worldwide are now using them to create innovative therapies.
Are there different ways of producing iPSCs? What method was used to produce Cynata’s iPSCs?
Yes, there are a number of different ways of producing iPSCs. It is important to be aware of the distinction between different reprogramming methods, as studies conducted on iPSCs produced by first generation methods are generally not relevant to iPSCs produced using more recent technology...
Cynata’s iPSC bank was manufactured by Cellular Dynamics International (now called Fujifilm CDI), using a non-integrating episomal reprogramming method based on that originally developed at UWM. Additionally, Cynata’s iPSCs were derived from a fully consented donor, in compliance with the FDA’s GMP requirements.
Are iPSCs safe for human use?
It is important to understand that iPSCs themselves are not administered to patients. Instead, the iPSCs are used as a starting material to produce other types of cells, such as MSCs in Cynata’s case...
All completed trials have met their safety endpoints: the Phase 1 GvHD trial (completed 2018) showed no treatment-related serious adverse events, and the recent Phase 1 diabetic foot ulcer trial (completed 2024) demonstrated that CYP-006TK was safe and well-tolerated with no suspected serious adverse reactions...
What is the difference between Cynata’s technology and first generation methods of MSC production?
First generation methods rely on the isolation of MSCs from donated tissue (for example bone marrow, fat or placenta), followed by “culture expansion”...
Cynata’s Cymerus™ technology uses a completely different approach, which does not involve the isolation of MSCs from tissue donations...
What are the key competitive advantages of Cynata’s Cymerus technology compared with conventional processes for manufacturing MSC-based therapeutic products?
Cynata’s proprietary Cymerus technology provides multiple clear advantages over conventional methods...
| Conventional Processes | Cymerus™ Process | Significance for Cynata |
|---|---|---|
| Continuous supply of new donors required | One donor, one time | Lower cost; simplified logistics; highly consistent product |
| Comparability testing required every time a new donation is used | Not applicable | Lower cost, minimal risk (MSC product from different donors must be proven to be the same: highly risky given every donor is different) |
| Several thousand doses per donation | Effectively limitless | Lower cost; simplified logistics |
| High MSC expansion (25–40 population doublings) | Low MSC expansion (10 population doublings) | Ensures Cynata MSC product is consistently highly potent: MSCs lose potency when highly expanded |
| Variable cellular “age” | Low – iPSC-derived MSCs are more primitive | Ensures Cynata MSC product is consistently highly potent |
| Medium to high risk of contamination with off-target cell types | Negligible | Lower risk of adverse reaction in patients; regulatory benefits |
How substitutable are different stem cell products; is it possible for one stem cell product to be substituted for another?
Unlike traditional medicines where generic versions can directly replace brand-name drugs, stem cell products cannot simply be substituted for each other. This is because stem cell therapies are far more complex than conventional medicines.
Each stem cell product has unique characteristics based on:
- - The type of cells used
- - How they're manufactured
- - Their specific properties and behaviour
Current regulations for generic drugs don't apply to cell therapies due to this complexity. So while two stem cell products might target the same condition, they can't be considered interchangeable in the way that generic drugs can replace brand-name drugs. Each stem cell therapy must be evaluated on its own merits for safety and effectiveness.
How many doses of MSCs can be produced from a tissue donation?
Traditional MSC production methods can generate tens of thousands of doses from a single donation. While adequate for clinical trials, successful commercial products could require millions of doses per year to treat conditions like stroke, heart attack, and diabetes. This would mean needing hundreds of new donors annually.
Cynata's Cymerus™ technology overcomes this limitation by producing an effectively limitless supply of MSCs from a single donation, without excessive culture expansion that can reduce cell effectiveness.
Why is the ability to manufacture an effectively limitless supply of MSCs from the same starting material important?
There are multiple problems associated with relying on a continuous supply of new donations as starting material:
- Significant logistical challenges and costs associated with collecting tissue donations
- Difficult to find sufficient numbers of suitable donors
- Screening and testing donors is time consuming and expensive
Changing the starting material is likely to change the characteristics of the end product:
- - MSC quantity and quality varies between donations
- - Requires comparability testing – costly and complex
- - Risk of failure in demonstrating comparability, leading to regulatory issues
In contrast, the Cymerus process avoids these challenges, enabling lower cost, batch-to-batch consistency and commercial scalability.
Blood transfusions and organ transplants rely on a continuous supply of donors. If that works for blood and organs, why is it not ideal for MSCs?
While donor-based systems work for blood and organs, they have challenges:
- - Blood shortages and transplant waiting lists
- - Very high costs (e.g., over $620 million annually for Australia's blood service)
MSCs, unlike blood and organs, are regulated like drugs – requiring strict consistency. This makes donor-dependent MSC production impractical. Cynata’s Cymerus™ technology solves this by enabling donor-independent manufacturing of MSCs.
Why is it important to manufacture a consistent MSC product?
Consistency is a fundamental requirement for all commercial therapeutic products. If commercial batches are not the same as those used in clinical trials, there is a risk the treatment won’t work as expected – or at all. Regulatory authorities therefore place great emphasis on batch-to-batch consistency.
Does Cynata use embryonic stem cells?
No. Cynata uses induced pluripotent stem cells (iPSCs), which are derived from adult donor cells.
There have been reports of stem cell companies allegedly exploiting “loop-holes” to supply unregulated and/or unproven medical treatments. How is this different to what Cynata does?
The regulatory exemptions are for autologous treatments – using a patient’s own cells – and are small-scale. Cynata produces allogeneic (off-the-shelf) therapies, treating unrelated patients at scale, which must pass regulatory approvals like drugs.
Cynata’s products are subject to oversight by major regulatory bodies (FDA, EMA, TGA), and undergo rigorous clinical trials to demonstrate safety, efficacy, and manufacturing quality.
Has Cynata conducted any clinical trials with its Cymerus MSCs?
Yes. Completed trials:
- - Phase 1 in aGvHD (2018): 87% overall response by Day 100, 60% 2-year survival
- - Phase 1 in diabetic foot ulcers (2024): 83.6% wound reduction at 24 weeks vs 47.8% in controls
Ongoing trials:
- - Phase 2 in aGvHD (completion mid-2025)
- - Phase 3 in osteoarthritis (results expected 2026)
- - Phase 1/2 in kidney transplantation (Cohort 1 results in Q2 2025)
All trials show Cymerus MSCs are safe and well tolerated with no serious adverse events.