Alive and Kicking: Watch this space on controversy-free stem-cell therapy trials, says Cynata

Cynata Therapeutics flags three trial results in the short term for three different conditions, including diabetic leg ulcers
Prescient Therapeutics prepares for a blood cancer trial and potential fast-track approval
Pacific Edge chairman agrees to stay on as the diagnostics company sorts out US reimbursement

Alive and Kicking is renowned biotech journo Tim Boreham’s new daily wrap covering morning movers and shakers of note in the ASX Healthcare sector, Monday through Thursday.

Stem-cell therapy developer Cynata Therapeutics (ASX:CYP) has told investors to expect the results of no fewer than three major trials between late 2024 and early 2026.

“We have a huge amount of upcoming news flow from our clinical programs,” CEO Dr Kilian Kelly said in a webinar this morning.

Cynata’s Cymerus platform is all about harvesting induced pluripotent stem cells (iPSCs), from which mesenchymal stem cells (MSCs) are derived.

MSCs are adult stem cells which can be isolated from human and animal sources and can produce more than one kind of specialist cell. While MSCs show potential to treat a range of illnesses, they require material from multiple donors – and that’s problematic.

The key advantage of iPSCs is that they are generated from a donation from a single donor, resulting in product consistency, scalability and potency. They can be derived from anywhere in the body – typically skin and blood – and grown in limitless quantities in the lab.

“They have similar properties to embryonic stem cells, but because they are derived from adult donors and not embryos we avoid all the ethical controversies that have gone along with [using] embryonic stem cells,” Dr Kelly said.

“We think these are an ideal starter material for the scalable production of cellular therapies.”

First off the rank is phase I data from a diabetic foot ulcer trial, with results in late 2024 or early 2025. The treatment incorporates the stem cells with a wound dressing.

In late 2025 the company expect to release interim results from its phase II trial for graft versus host disease (GvHD), the body’s rejection of material after a bone marrow transplant.

The company has also completed enrolment for a phase III osteoarthritis trial and expects to show its wares in late 2026.

Currently, the most widely used stem cell therapy is hematopoietic stem-cell transplantation, to treat conditions leukemia and lymphoma.

With a $34 million market cap, Cynata is considered the ‘mini me’ version of leading stem cell exponent Mesoblast (ASX:MSB). The latter is worth $1.1 billion but has suffered myriad approval setbacks. That said, it is girding for imminent FDA approval of a GvHD treatment.

Cynata shares this morning were unchanged at 19 cents.

Prescient Therapeutics aims for trial to tackle rare blood cancer

This morning was ‘show and tell’ time for cell-therapy developers on another front, with cancer drug developer Prescient Therapeutics (ASX:PTX) outlining a phase II trial which it hopes to kick off this year (pending US Food and Drug Administration approval of the study design).

This one targets cutaneous T-cell lymphomas, or CTCLs, a rare type of white blood -cell cancer.

Pitched at expedited FDA approval, the trial will follow a 25-patient phase 1b study that showed a superior overall response rate – the proportion of patients who have a partial or complete response to therapy – over the standard-of-care treatments.

If and when the drug is approved, Prescient hopes to leverage the results for a separate study for peripheral t-cell lymphoma (PTCL), a more common blood cancer but one for which existing treatments are available.

While CTCL is an ‘orphan’ condition, Prescient still estimates the addressable market at a chunky UD$748 million by 2032.

By the way, PTX-100 works by inhibiting three key pathways to tumour growth.

While it’s a legacy program for Prescient – the company has since expanded to ‘sexier’’ Car-T therapies – this morning’s prezzo makes clear it shouldn’t be forgotten.

Prescient shares were up 9.5% to 4.6 cents

Hanging around to lend a hand

It’s always nice to wanted, as Pacific Edge (ASX:PEB) chairman Chris Gallaher can attest.

The board of the oncology diagnostics outfit has asked Gallaher to defer his retirement, planned for the end of the year, to “provide stability and confidence to Pacific Edge and its stakeholders amid the continued uncertainty over the Medicare coverage status for the company’s Cxbladder tests.”

The Medicare in question is the public US body, not our beloved Medicare. In July Novitas, the Medicare administrator responsible for Pacific Edge’s US lab, confirmed that it had been granted an extension “beyond the normal statutory timeline” to determine the reimbursement status of the tests.

The Dunedin-based Pacific Edge provides its Cxbladder tests globally via its certified labs in New Zealand and the US. The assays are used for diagnosing bladder cancer and patients with hematuria, which is blood in the urine (a.k.a not a good thing).

Pacific Edge reported revenue of NZ$23.9 million for the year to March 2024, up 22% and a loss of NZ$29.5 million. The revenue was derived mainly from the US, where the tests currently are reimbursed but Gallaher says “the finalisation [of the determination] remains the biggest factor impacting the company’s near-term prospects.”

Gallaher and independent director Mark Green were to have stepped down at the company’s AGM in Auckland on September 24, but now only Green will bid adieu.

Pacific Edge shares were unchanged at 4.5 cents