Early Safety and Efficacy Data in Cynata’s Phase 1 Trial of CYP-001 in GvHD

Encouraging Early Safety and Efficacy Data in Cynata’s Phase 1 Trial of CYP-001 in GvHD; DSMB Recommendation to Initiate Enrolment of Second Patient Cohort

Australian stem cell and regenerative medicine company, Cynata Therapeutics Limited (ASX: CYP) announced that the independent Data Safety Monitoring Board (DSMB) has recommended that Cynata’s clinical trial of its lead Cymerus™ mesenchymal stem cell (MSC) product CYP-001 should progress to the next stage as planned.

Key Highlights:

• All eight participants in Cohort A (lower dose cohort) have demonstrated at least a Partial

Response (defined as an improvement in the severity of GvHD by at least one grade compared

to baseline)

• No treatment-related serious adverse events or safety concerns have been identified

• DSMB recommendation to progress clinical trial to second cohort (Cohort B)

• Patient enrolment in Cohort B (higher dose cohort) now open at seven trial sites in the U.K.

and Australia

Cynata’s clinical trial, which is the first clinical trial in which patients have been treated with an allogeneic, induced pluripotent stem cell (iPSC)-derived therapeutic MSC product, consists of a planned total of 16 patients with steroid-resistant acute graft-versus-host disease (GvHD). The recommendation to progress to the next stage (Cohort B) followed an independent review by the DSMB of the eight participants in Cohort A. Recruitment for Cohort A commenced in May 2017, and there are currently seven trial sites active and ready to enrol participants into Cohort B.

Steroid-resistant GvHD patients today have a dismal prognosis, where mortality rates are very high. At this time, seven of the eight participants in Cohort A are alive. One participant died after developing pneumonia, which is a common finding in recipients of bone marrow transplants and similar procedures.1 This death was not considered to be treatment-related. Participants enrolled in Cohort A of the dose-escalation trial received a dose of CYP-001 that was anticipated to be at the lower end of the effective dose range (one million cells per kilogram of bodyweight, up to a maximum of 100 million cells per infusion). In Cohort B, a further eight participants will receive two infusions of CYP-001 at a dose of two million cells per kilogram of bodyweight, up to a maximum of 200 million cells per infusion.

Dr Ross Macdonald, CEO of Cynata Therapeutics, said, “We are thrilled to report this encouraging early review of the Phase 1 trial of CYP-001, which marks the first time that patients have been treated with an allogeneic, induced pluripotent stem cell-derived therapeutic MSC product. The improvement in GvHD grade observed in 100% of these gravely ill people is very promising, especially given the low dose administered in Cohort A. The positive DSMB recommendation is an important milestone that enables us to begin enrolment in Cohort B, and advance toward our goal of completing the trial later this year. A successful outcome will support the application of CYP-001 in many medically and commercially significant targets where therapeutic MSCs have shown promising results.”

Next Steps:

Patient enrolment into Cohort B is now open at seven active sites across the U.K. and Australia. Cynata looks forward to providing further updates to the market as the study progresses.

Ref: Cynata PR

Science Validation & The Rising Sun

Strategy, survival, direction, competition and growth opportunities are but a few fundamental elements in the maze of everyday life all professionals must navigate - no more so than those on the leading edge of managing scientific innovation.

Being at the forefront of change often elicits backdraft currents and polarization of entrenched positions. This is natural and can be seen as a positive reinforcement of the high threshold one must strive to in order to achieve acceptance. A sort of quality assay if you will - one which is analogous to peer review in a business setting.

A lot has been written on the news of the Ocata/Astellas deal regarding the value calculation and little on the actual merits of Validation for Ocata’s science, the step forward in its development plan and the broader implications for the stem cell sector.

This writer has covered Ocata for a long time and can attest to the finer details of the saga that was its Survival and Vindication. A road which has seen its fair share of episodic highlights - perhaps too many.

One could speak at length and talk of the dedicated character of those involved with the science and the constant pressure within & on the company to prove itself in a demanding uncompromising field. One piece termed the company a “lightning rod” - the story has all the hallmarks.

The end of an era? Yes, perhaps it is in many ways. I prefer to see it as the close of the 2nd act in a 3 act structure, where the rain is falling and mingling with the tears.

Lost independence isn’t easy to come to grips with. However, moving on from being a small volatile publicly traded company, with all the influences that entails, to being housed within a protective and nurturing parental structure is a very positive outcome - for the programs and patients in need. 

It signifies so much with regard to the science and efforts to help define the standards of a new treatment methodology in medicine. 

The deal is a solid affirmation from established pharma that the stem cell therapeutic sector is worth banking on. This comes on the heels of other momentum building developments in the space and perhaps is indicative of a growth driven consolidation phase.

There are many worthwhile questions surrounding the announcement and events leading up to the decision to sell that remain, even after recent company disclosures, and one would look to those involved to address them for the record. Lock stock comes to mind?

There is a saying which is often spoken in Catalonia - “the sun always rises” and aims to reinforce and embrace the positive.

Cheers