Faron Pharmaceuticals Limited (AIM:FARN) has announced initial positive results from phase II of its BEXMAB trial of its cancer treatment Bexmarilimab, indicating significant progress in treating myelodysplastic syndrome (MDS) patients who have previously failed hypomethylating agent (HMA) therapy.
This comes after earlier study results showed promising outcomes.
In the phase II trial, the treatment group expanded from eight to 14 patients with refractory or relapsed forms of this blood cancer with limited treatment options.
This demonstrated an impressive objective response rate (ORR) of 79%. The objective response rate measures the percentage of patients whose cancer shrinks or disappears after treatment.
These patients experienced a median overall survival (mOS) of approximately 13 months, compared to the historical expectation of 5-6 months for similar patients under standard care.
The phase I trial had already set a positive precedent, with an overall response rate of 87.5% (7/8) MDS patients who had failed previous HMA therapy.
These patients were treated with a combination of bexmarilimab and chemotherapy called azacitidine.
The treatment has been well tolerated, showing no dose-limiting toxicity. The true remission rate, where the disease is no longer detectable, stands at 64%.
The detailed results for these patients are as follows: one complete response (CR), seven marrow complete remissions (mCR), one partial response (PR), two haematological improvements, two cases of stable disease (SD), and one case of progressive disease (PD). Notably, two patients have moved on to receive bone marrow transplants, a potentially curative treatment rarely achieved in this patient population due to the difficulty in achieving remission.
Historically, similar patient groups treated with existing alternatives have shown a 0-20% response rate, without the deep and durable remissions observed in this trial. The median overall survival for Phase 1 patients with adequate follow-up is currently estimated at 13.4 months, though this figure may still evolve.
Bexmarilimab is an experimental drug targeting the CLEVER-1 receptor on myeloid cells, reprogramming them to activate the body's anti-tumour immune response. Azacitidine is a chemotherapy drug commonly used to treat MDS.
CEO Dr Juho Jalkanen said: "This is a significant milestone for Faron. Many of us have recognised the grave need for new treatment options in r/r MDS.
"With these first results from the phase II continuing the positive results already seen in phase I, we are committed to rapidly advancing Bexmarilimab to market, because patients are waiting for treatment options like this."