MSCs Demonstrate Anti-Cancer Effects in Pre-Clinical Studies

Cynata Therapeutics Limited Level 3, 62 Lygon Street, Carlton, Victoria 3053, Australia PO Box 7165, Hawthorn North, Victoria 3122 T: + 613 9824 5254 F: + 613 9822 7735 E: [email protected] ABN - 98 104 037 372 ASX ANNOUNCEMENT 18 October 2018 Engineered Cymerus™ MSCs Demonstrate Anti-Cancer Effects in Preclinical Studies Positive results in pre-clinical models support the use of the Cymerus platform to target various cancers Melbourne, Australia; 18 October 2018: Cynata Therap eutics Limited (ASX: CYP), a clinical stage Biotechnology company speciali sing in cell therapeutics is pleased to announce results from its preclinical program using the proprietary Cymerus platform technology to develop genetically engineered cells, derived from mesenchymal stem cells (MSCs), to treat cancer. Key Highlights • Genetically engineered cells were successfully produced using the Cymerus platform to express diagnostic and therapeutic anti-cancer agents. • Engineered Cymerus cells can be effectively tracked by real time imaging in vivo in mouse tumour models • Engineered Cymerus cells showed highly p romising therapeutic benefits in different mouse tumour models. The studies were conducted under the direction of Professor Khalid Shah, Vice Chair of Research at Brigham and Women's Hospital in Boston, Massachusetts, and Director of the Center for Stem Ce ll Therapeutics and Imaging at Harvard Medical School. Professor Shah commented, “Our results validate the use of this cell -based approach, as it provides a continuous pool of therapeutic protein around the tumour cells, circumventing the protein short hal f-lives. Overall, the study outcomes support the use of engineered Cymerus MSCs for cell therapy to target various malignancies.” Dr Kilian Kelly, Cynata’s Vice President, Product Development, said, “These highly encouraging results suggest that the Cymerus platform can be exploited to produce targeted anti-cancer therapies, which may have significant advantages over conventional cancer treatments, from both a safety and efficacy perspective. Importantly, this engineering technique could also facilitate the creation of novel engineered therapies for areas of high unmet need in other therapeutic areas”. Study Design The first stage of the program aimed to evaluate the feasibility of using a lentiviral engineering process to cause Cymerus MSCs to express transgenes (genes from other cells) , and to investigate the persistence of engineered cells in mouse models. Cymerus MSCs were also engineered to express a protein that is known to exert anti -cancer effects. The effects of these engineered Cymerus MSCs on the viability of both human melanoma (skin cancer) and human glioblastoma (brain cancer) cells were evaluated in in vitro test systems and the therapeutic efficacy of the engineered Cymerus MSCs was also tested in a well-defined mouse model of glioblastoma. Cynata Therapeutics Limited Level 3, 62 Lygon Street, Carlton, Victoria 3053, Australia PO Box 7165, Hawthorn North, Victoria 3122 T: + 613 9824 5254 F: + 613 9822 7735 E: [email protected] ABN - 98 104 037 372 Study Outcomes The preliminary studies confirmed that: • The Cymerus platform can be successfully engineered to express transgenes in a stable manner. • Engineered Cymerus MSCs persist in vivo for a sufficient length of time to facilitate a statistically significant therapeutic effect in a preclinical model of Glioblastoma. In the second stage of the program, e ngineered Cymerus MSCs were shown to cause a significant reduction in the viability of human glioblastoma cells when compared to either the control (p<0.005) or to direct administration of the therapeutic protein (p<0.05), and a significant reduction in the viability of human melanoma cells c ompared to the control (p<0.005). Furthermore, in an in vivo mouse model of glioblastoma, tumour progression was slower in mice that received the engineered Cymerus MSCs compared to those receiving the control. Following this positive outcome Cynata intends to continue to pursue this promising new approach to cancer treatment as part of its pipeline strategy. Ends CONTACTS: Dr Ross Macdonald, CEO, Cynata Therapeutics, +61 (0)412 119343, [email protected] Rosa Smith, Australia Media Contact, +61 (0) 475 305 047, [email protected] Annie Starr, U.S. Media Contact, +1 973.768.2170 , [email protected] About Cynata Therapeutics (ASX: CYP) Cynata Therapeutics Limited (ASX: CYP) is an Australian clinical -stage stem cell and regenerative medicine company focused on the development of therapies based on Cymerus™, a proprietary therapeutic stem cell platform technology. Cymerus overcomes the challenges of other production methods by using induced pluripotent stem cells (iPSCs) and a precursor cell known as mesenchymoangioblast (MCA) to achieve economic manufacture of cell therapy products, including mesenchymal stem cells (MSCs), at commercial scale and without the limitation of multiple donors. Cynata’s lead product candidate CYP -001 met all clinical endpoints and demonstrated positive safety and efficacy data for the treatment of steroid -resistant acute graft -versus-host disease (GvHD) in a Phase 1 trial. Cynata plans to advance its Cymerus™ MSCs into Phase 2 trials for GvHD and critical limb ischemi a. In addition, Cynata has demonstrated utility of its Cymerus MSC technology in preclinical models of asthma, critical limb ischemia, diabetic wounds, heart attack and cytokine release syndrome, a life -threatening condition stemming from cancer immunotherapy.

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