Australian cell therapy biotech Mesoblast has released results this week showing its allogenic cell therapy, designed to target pain in those with degenerative disc disease, provides pain relief for at least three years.
The last year has been mixed for the company, which saw shares crash by 21% in December when Novartis pulled out of an agreement to develop remestemcel-L –one of Mesoblast’s cell therapy treatments– to treat acute respiratory distress syndrome (ARDS) due to COVID-19.
This was due to a late-stage trial failure, so was not totally unexpected, but amounts to a loss of up to US$1.2B for the company.
Remestemcel-L is still in late-stage development to combat graft versus host disease in both children and adults, a dangerous side effect that often occurs in response to procedures such as bone marrow transplantation to treat blood cancer.
The current results, released at the Biotech Showcase event in the U.S., are from the first Phase III trial of the company’s allogenic ‘off-the-shelf’ cell therapy rexlemestrocel-L in 404 patients with chronic low back pain due to degenerative disc disease.
This study showed that the treatment resulted in consistent pain relief for up to 36 months. Patients who had experienced chronic lower back pain for less than six years (68 months) benefitted more from the therapy than those with longer periods of chronic pain. The researchers think this is likely because the therapy is most effective when there is still active inflammation and irreversible fibrosis of the intervertebral disc has not yet occurred.
The team also noted that despite being told to continue with the medications they were taking at the beginning of the study, 28% of those given rexlemestrocel-L who were taking opioids at the beginning of the study had stopped taking them at 36 months compared with 8% of saline treated controls.
Although this is a Phase III study, more data is needed before this treatment can be approved by regulatory authorities. A second Phase III trial is planned, the results from which will guide potential approvals in both the E.U. and U.S.A. Rexlemestrocel-L is also in earlier stage development for treatment of advanced chronic heart failure.
Following a review of the data from the first Phase III study of rexlemestrocel-L for treatment of chronic lower back pain at the end of 2021, the FDA’s Office of Tissues and Advanced Therapies has agreed a primary endpoint with Mesoblast for the new study. This will be pain reduction at 12 months, with functional improvement and reduction in opioid use as secondary endpoints for a new Phase III study. At least 20% of trial participants will be from the EU to help support an approval by the EMA, should the findings be positive.
While cell therapies are still very new and experimental, the lower back pain market is large and if the company achieves market approval of rexlemestrocel-L for this indication it could be very profitable. It remains to be seen if they can overcome previous disappointments and take this therapy to market.