Regenxbio announces expansion of RGX-121 clinical program
Regenxbio Inc. (NASDAQ:RGNX) announced the expansion of its Mucopolysaccharidosis Type II clinical program. The company stated that the expansion will allow it to gain additional understanding of the neurodegenerative aspects of the disease. The expansion will also let Regenxbio in a broader patients population.
The Phase I/II study seeks to examine a single intracisternal administration of RGX-121 in patients under the age of 5 years. All these patients have severe MPS II. The company has already dosed six patients across two dose levels. The main aim of the study is to assess the safety and tolerability of RGX-121 and to evaluate the impact of the drug candidate on biomarkers of I2S enzyme activity, neurocognitive development and other clinical measures.
Regenxbio intends to start expanded enrollment of patients in Cohort 2 immediately. The decision has been taken based on the basis of recommendations made by the Independent Data Monitoring Committee and MPS II treating physicians. Steve Pakola, M.D., Chief Medical Officer of Regenxbio said, “Regenxbio is committed to advancing potential gene therapy treatment options for MPS II, as there remains a significant unmet medical need to address the neurological manifestations and prevent or stabilize cognitive decline for patients.”
As of September 16, 2020, RGX-121 was found to be well-tolerated in all six patients. No drug-related serious adverse events were reported. The company now plans to dose up to six additional patients with a second dose level of 6.5x1010 genome copies per gram (GC/g) of brain mass. Regenxbio expects to provide further updates from the study by the end of 2020.
Regenxbio also reported that the FDA has cleared its Investigational New Drug Application. The company plans to start the second Phase I/II trial of the drug candidate for treating pediatric patients with severe MPS II
Thanks for reading. At the Total Pharma Tracker, we do more than follow biotech news. Using our IOMachine, our team of analysts work to be ahead of the curve.
That means that when the catalyst comes that will make or break a stock, we’ve positioned ourselves for success. And we share that positioning and all the analysis behind it with our members.
