Special Report: While it’s not overly common, graft-versus-host disease (GvHD) — particularly the acute, steroid-resistant variety — is one of the most medically challenging diseases on the planet.

But one Australian company just got a step closer to an effective treatment.

GvHD is a medical complication following the receipt of transplanted bone marrow tissue from a person with different genetics, regularly occurring in patients who’ve had cancer chemotherapy or radiotherapy and require a bone marrow transplant.

Acute, steroid-resistant GvHD, which Cynata Therapeutics (ASX:CYP) is looking to treat with its stem cell product CYP-001, has mortality rates as high as 80-90 per cent.

An effective treatment would be a significant breakthrough.

Cynata thinks it might be the company to deliver that, following the release of further positive interim data.

The latest study was split into two Cohorts, A and B, with Cohort B receiving a higher dose of CYP-001 than that of Cohort A.

The Preliminary Evaluation Period for both Cohorts consisted of two milestones: progress after the 28-day mark, and the 100-day mark.

Cynata received positive feedback after both the 28-day and 100-day marks for Cohort A, and has further progressed with the receipt of encouraging data at the 28-day mark for Cohort B.

The Day 100 time point will be reached for Cohort B around the end of August this year.

Four out of the seven patients in Cohort B saw their GvHD signs and symptoms completely resolved, while a further two showed an improvement in the severity of GvHD by at least one grade compared to baseline.

The higher dose in Cohort B — the seven patients were given two infusions of CYP-001 at a dose of two million cells/kg, up to a maximum dose of 200 million cells, double that of Cohort A — resulted in significantly faster results.

By Day 28, only one patient in Cohort A had their signs and symptoms resolved.

The highly promising data supported rapid acceleration into further trials, said Cynata’s CEO Dr Ross Macdonald.

“These results are highly encouraging, and together with the data from Cohort A, support the advancement of CYP-001 into a Phase 2 trial in GvHD.”

(Clinical trials are generally divided into three phases. Phase 1 focuses on safety, Phase 2 tests for effectiveness and Phase 3 examines whether the new drug is an improvement on existing treatment.)

Perhaps the best part of the news is that the established safety profile of CYP-001 and compelling data in pre-clinical models of a range of diseases mean trials in other indications could potentially bypass further Phase I trials.

“There is a high unmet need for a consistent and scalable source of high-quality MSCs,” Dr Macdonald said.

“We are evaluating our options strategically, but potential other indications include heart attack, asthma, brain cancer and critical limb ischemia.”


This special report is brought to you by Cynata Therapeutics.

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