Melbourne, Australia; 15 November 2017: Australian stem cell and regenerative medicine company, Cynata Therapeutics Limited (ASX: CYP), is pleased to release a new investor presentation to be presented at a series of upcoming institutional investor meetings.
Paul Wotton (Cynata Chairman) and Ross Macdonald (Cynata CEO) will be meeting with a series of new and existing institutional investors prior to the upcoming AGM on November 17.
Cynata Therapeutics provides investors with excellent exposure to a rapidly growing regenerative medicine and stem cell sector via its patented CymerusTM technology, a platform able to manufacture mesenchymal stem cells (MSCs) at a commercial scale. The new investor presentation highlights Cynata Therapeutics’ compelling investment case and provides information about the Company’s progress.
Operational progress
- 8 participants now enrolled in world first clinical trial of CYP-001 for the treatment of steroidresistant
graft-versus-host disease (GvHD) - Enrolment of the 8th and final patient in Cohort A represents the half-way point of the trial
- and an independent safety and monitoring review will be triggered 28 days after this participant
receives the infusion of CYP-001
- 8 participants now enrolled in world first clinical trial of CYP-001 for the treatment of steroidresistant
Investment highlights
- Unique platform to efficiently mass-produce mesenchymal stem cells (MSCs), a highly promising type of therapeutic stem cell
- Large, active and growing market, with over 650 trials investigating the efficacy of MSCs in treating diseases including osteoarthritis, stroke and cardiovascular disease
- Scalable business model intended to target a broad range of disease target areas over time, and monetise these through licensing & partnerships
- Cynata’s initial target area is GvHD, intended to prove the quality of the MSC’s produced by its patented Cymerus platform
- Monetisation of the business model has already commenced, with license option and strategic alliance transaction entered into with Fujifilm, Cynata’s largest shareholder with 9%
About Cynata Therapeutics (ASX: CYP)
Cynata Therapeutics Limited (ASX: CYP) is an Australian clinical stage stem cell and regenerative medicine company that is developing a therapeutic stem cell platform technology, Cymerus™, originating from the University of Wisconsin-Madison, a world leader in stem cell research. The proprietary Cymerus™ technology addresses a critical shortcoming in existing methods of production of mesenchymal stem cells (MSCs) for therapeutic use, which is the ability to achieve economic manufacture at commercial scale.
Cymerus™ utilises induced pluripotent stem cells (iPSCs) to produce a particular type of MSC precursor, called a mesenchymoangioblast (MCA). The Cymerus™ platform provides a source of MSCs that is independent of donor limitations and provides an “off-theshelf” stem cell platform for therapeutic product use, with a pharmaceutical product business model and economies of scale. This has the potential to create a new standard in the emergent arena of stem cell therapeutics and provides both a unique differentiator and an important competitive position.
About the Phase 1 clinical trial (Protocol Number: CYP-GvHD-P1-01)
The trial is entitled “An Open-Label Phase 1 Study to Investigate the Safety and Efficacy of CYP-001 for the Treatment of Adults With Steroid-Resistant Acute Graft Versus Host Disease”. Participants must be adults who have undergone an allogeneic haematopoietic stem cell transplant (HSCT) to treat a haematological (blood) disorder and subsequently been diagnosed with steroid-resistant Grade II-IV GvHD. The first eight participants will be enrolled in Cohort A and receive two infusions of CYP-001 at a dose of 1 million cells per kilogram of body weight (cells/kg), up to a maximum dose of 100 million cells. There will be one week between the two CYP-001 infusions in each participant. The next eight participants will be enrolled into Cohort B and receive two infusions of CYP 001 at a dose of 2 million cells/kg, up to a maximum dose of 200 million cells.
The primary objective of the trial is to assess safety and tolerability, while the secondary objective is to evaluate the efficacy of two infusions of CYP-001 in adults with steroid-resistant GvHD. The primary evaluation period will conclude 100 days after the first dose in each participant. Efficacy will be assessed on the basis of response to treatment (as determined by change in GvHD Grade) and overall survival at 28 and 100 days after the administration of the first dose. After the completion of the primary evaluation period, participants will enter a longer term non-interventional follow-up period, which will continue for up to two years after the initial dose.
