ProTrans demonstrates effect at 2 years after repeated treatments
NextCell Pharma AB ("NextCell") today announces that the ProTrans-Repeat trial met its primary safety endpoint. There were no severe adverse events recorded during the 12-month follow-up period after a second dose of ProTrans™ (ProTrans). Furthermore, a strong tendency of sustained efficacy was observed in the three patients receiving high dose ProTrans.
- Primary safety endpoint met
- Tendency of sustained efficacy observed for high dose patients
- 2-year preservation of insulin production significantly higher for patients treated with high dose as compared to low dose
ProTrans-Repeat started in May 2019 and is a continuation of ProTrans-1, with the purpose of obtaining data on repeated treatments. 3 patients per dosage group were treated with low, medium and high dose, respectively (total 9 patients). All patients included in ProTrans-1 were later included in ProTrans-Repeat, directly after completing or up to 7 months after completion of ProTrans-1. Patients were given the same dose again, this time with ProTrans from another manufacturing batch, and followed for 12 months.
"The observed sustained insulin production for now two years in the three patients treated with high dose ProTrans is remarkable and there is definitely a need for larger trials with this therapy”, says professor Per-Ola Carlsson, Uppsala University and Principal Investigator.
The primary endpoint in this study is safety parameters including adverse events and hypoglycaemia, allergic reactions, ophthalmologic examination, ECG, vital signs, and laboratory assessments at day 372. ProTrans treatment appears safe even with repeated treatments without any reported severe adverse events. No safety issues were observed for any of the three evaluated doses.
| Average loss of C-peptide | Historical | ||||
| (% delta AUC C-peptide) | controls* | Controls | Low | Medium | High |
| 12-24 months after diagnosis | -30% | -28% | -9% | -4% | |
| 24-36 months after diagnosis | -45% | -28% | -23% | -40% | -4% |
| 12-36 months after diagnosis | -60% | -49% | -44% | -6% | |
| Average loss of C-peptide | Historical | ||||
| (abs delta AUC C-peptide) | controls* | Controls | Low | Medium | High |
| 12-24 months after diagnosis | -0.17 | -0.28 | -0.05 | -0.03 | |
| 24-36 months after diagnosis | -0.17 | -0.22 | -0.18 | -0.24 | -0.03 |
| 12-36 months after diagnosis | -0.34 | -0.50 | -0.27 | -0.06 | |
| *Historical controls have been extrapolated from fig. 3 in Diabetes 61:2066–2073, 2012, by Greenbaum et al. | |||||
The secondary endpoint is delta-change of C-peptide Area Under the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at day 372 (after completion of ProTrans-1) following ProTrans infusion when compared to test performed before start of treatment when compared to control patients. The percentage change in insulin production in the control group varied greatly between individuals, the mean loss was -28% (SD +/- 35%) after 12 months. The low medium and high dose treated patients had a mean loss of -23% (SD +/- 16%), -40% SD (+/- 22%) and -4% (SD +/- 19%) respectively. Statistical significance was not achieved since the number of patients in each group were too few and the variation among control patients. The numeric effect of high dose treatment is encouraging.
The studies were designed for safety, however, we see a clear dose dependent efficacy trend also after 24 months. The 2-year preservation of insulin production is statistically significantly higher for patients treated with high dose as compared to low dose in absolute units (p<0.05) and was close to achieving significance assessed as percentage of preserved insulin production (p=0.059). Two out of three patients treated with high dose had an increase in endogenous insulin production after 2 years.
“There is a tendency on dose dependent effect of ProTrans with sustained excellent safety. Trendlines of insulin production are consistent and even low dose appears to have some effect. The trials have only been dimensioned for safety. To be able to show effect is a great sign of strength even if we have to cautious about interpreting data from these few subjects”, says Mathias Svahn CEO.
The two trials, ProTrans-1 and ProTrans-Repeat have provided long-term data for more than 24 months, the first patients have soon been monitored for 36 months. This is very valuable in the development of ProTrans and hopefully data for up to 60 months can be generated. High dose ProTrans was the only dose used in ProTrans-2 and later planned for ProTrans-3.
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Mathias Svahn, CEO
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About NextCell Pharma AB
NextCell is a Phase II cell therapy company with the lead candidate ProTrans™, for the treatment of type-1 diabetes. Focus is to take ProTrans™ to market approval via a phase III study. Furthermore, NextCell operates Cellaviva, Scandinavia's largest stem cell bank for family-saving of stem cells from umbilical cord blood and umbilical cord tissue with permission from IVO.
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