Epidermolysis bullosa (EB) is a rare condition affecting approximately 500,000 individuals globally. Patients in the United States and Europe account for 50,000 of these individuals.
The hereditary condition is caused by a gene mutation in the proteins which ensure the integrity of the skin. As a result of this mutation, patients suffer extreme skin fragility which leads to blistering and pain in the skin and the mucous membranes.
Unfortunately, there has yet to be a treatment approved for this condition besides supportive therapies. Patients may take antibiotics and use sterile dressings, but nothing has been formulated to combat the skin fragility itself.
However, a new potential therapy is being examined by RegeneRx Pharmaceuticals and there has already been positive results. The treatment aims to support the healing of the wounds on the skin as well as protect the cells, their migration, and the integrity of the tissue. These multiple methods of action should help to provide multiple benefits to patients, none of which have been possible before.
RegeneRx Pharmaceuticals has just announced that the very first patient enrolled in their Phase 2 trial for EB has had a positive response to the therapy. With the treatment, they experienced complete wound healing.
This single-blinded trial is examining RGN-137 as a new, topical treatment for EB. It’s single-blinded nature means that which wounds are treated with the therapy and which wounds are treated with placebo are not hidden from the sponsors. However, the patients themselves are blinded.
In this study, only two wounds are treated on each patient- one with the therapy (which is a dermal gel) and one with placebo. In the first patient who was treated, complete wound healing was revealed in the wound treated with RGN-137. The wound treated with placebo did not reveal healing.
This trial is taking place at 5 different hospitals which specialize in EB.
It is being sponsored by Lenus Therapeutics as part of a joint venture. The goal is to develop RGN-137 in the United States, Japan, Korea, Australia, Canada, and Europe. It will be developed as an indication for dermal wound healing.
If this Phase 2 trial continues to produce positive results (which we all hope it will), it will serve as the basis of a Phase 3 investigation. This trial will be designed using the Phase 2 trial results as a model.
Stay tuned to learn more about this investigation and its continued results.
You can read more about this Phase 2 trial and RGN-137 here.