Results support progression of clinicals to P3
Mesoblast's (ASX: MSB) (USOTC: MBLTY) P2 clinical trial for lumbar spinal fusion had successfully met its safety and efficacy endpoints.
The results suggest that Mesoblast's NeoFuse product comprising allogeneic Mesenchymal Precursor Cells (MPCs) is as effective for interbody lumbar fusion as the gold standard, bone autograft, without the need for a 2nd surgical procedure and its attendant morbidity risks. These results support the progression of clinical development of NeoFuse to a P3 trial in interbody lumbar fusion.
24 patients were enrolled and randomized over 5 sites in the US with 8 patients in each treatment arm - bone autograft standard of care (Control), 25 million MPCs (25M), and 75 million MPCs (75M). Patients underwent the surgical procedure, 1 or 2 level fusions using a posterior approach to the spine, and were evaluated for safety and efficacy. The median follow-up times for the 3 treatment groups were 23.9, 20.7, and 22.9 months for the bone autograft, 25 M, and 75 M groups, respectively.
MPCs were well tolerated with no cell-related serious adverse events and no ectopic bone formation at all. Notably, MPC treated groups had 30-43% lower mean estimated blood loss during surgery compared to the autograft treatment group (p less than 0.05 for the 25 M group). At 12 months, fusion was achieved in 85.7% of patients in the 25 M treatment group compared to 62.5% in the 75 M and 75% in the control patient groups. Overall, patients from all 3 treatment groups had a clinically significant and comparable decrease in low back and leg pain, assessed on the Visual Analogue Scale and functional improvement, assessed by the Oswestry Disability Index questionnaire.
The Bottom Line: MSB's cells may provide a significant and valuable option to achieve fusion in patients with severe spinal disc degeneration and these results warrant progressing to a P3 study. Key take-away points are:
- P2 trial results using MSB's allogeneic, or "off-the-shelf", Mesenchymal Precursor Cells (MPCs) demonstrated rates of fusion success that were comparable to the gold standard bone autograft;
- MPCs were well tolerated with no cell-related serious adverse events and no evidence of any ectopic bone formation;
- At 12 months, fusion was achieved in 85.7% and 62.5% of patients in the low and high MPC treatment groups compared to 75% of patients who received bone autograft;
- Significant improvements in low back pain scores and total disability index were seen in both MPC groups and were comparable to those receiving bone autograft;
- Those receiving MPCs had 30-43% less blood loss during the surgical procedure than those receiving bone autograft (p less than 0.05);
- These results indicate that lumbar vertebral fusion using allogeneic MPCs is as effective as bone autograft; and finally,
- Eliminating the need for an autograft bone harvest will avoid the risks of this procedure, including pain, infection and blood loss.
Other than autograft, there is no approved product for posterior spinal fusion, a surgical approach which accounts for 62% of all lumbar fusion procedures. There is a critical unmet need for new technologies to achieve fusion that are safe, effective, and that eliminate the need for bone autograft.
MSBLTY closed at $28.09 on 1/10/13 and is a BUY with a 10 - 15% upside on results!
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