March 29, 2021
Medsenic announced today the positive results of its Phase II clinical study “GMED-16-001” with its lead product Arscimed®, an intravenous formulation of arsenic trioxide, in patients with Chronic Graft Versus Host Disease (GvHD).
“These excellent results are a significant step forward for the management of patients with cGvHD, a rare, complex and extremely debilitating autoimmune disease affecting over 40,000 people worldwide and for which there is no satisfactory treatment. The study data validate the therapeutic potential of our product Arscimed®, as a new selective immunosuppressive/anti-inflammatory drug for cGvHD. We look forward to confirming its efficacy in a Phase III study as well as its significant impact on improving the quality of life of GvHD patients,” said Prof. François Rieger, President, and co-founder of Medsenic.
The primary endpoint of this prospective Phase II multicentre, non-randomised study was the improvement of treatment response, i.e., complete or partial disease remission 6 months after GvHD diagnosis, with Arscimed® in combination with prednisone with or without cyclosporine.
Over a 4-week period, 21 patients with moderate to severe chronic GvHD received as first-line treatment daily intravenous infusions of Medsenic’s arsenic trioxide drug. The first improvements were observed 6 weeks after the first infusion. The reduction of the initial dose of 1 mg prednisone/kg/day (standard care with or without cyclosporine) can be started at this stage, with a view to discontinue corticosteroids once complete remission has been achieved. This ability to eliminate the use of corticosteroids is a major advantage of arsenic trioxide treatment.
After the 6-month follow-up, the study’s primary endpoint was met in 15 of 21 patients, with a 75% clinical efficacy rate (95% exact CI: [50.9%; 91.3%]). This very encouraging result was confirmed by a sustained response at 12 months post-treatment in all patients.
The active pharmaceutical ingredient (API) of Arscimed® is arsenic trioxide. Medsenic has harnessed its expertise to develop and manufacture a formulation of arsenic trioxide for IV injection. Arsenic trioxide belongs to a new class of drugs able to radically modify the autoimmune cascade and normalize the immune system without causing nonspecific immunosuppression.
The main action of arsenic trioxide is the activation of a strong oxidative stress-induced pathway in activated immune cells, and the elimination of certain subtypes of pathogenic immune cells eliciting autoimmune reactions. It also suppresses abnormal biological processes associated with immune disorders, such as the excessive production of proinflammatory cytokine.
cGvHD – Chronic Graft versus Host Disease – is a complex autoimmune reaction that develops following bone marrow transplants or, more precisely, allogeneic hematopoietic stem cells, with a frequency of 30-60 % of the treated patients. It affects each year approximately 16,000 people in the European Union and 20,000 in the United States and Canada, which places it under the classification of Orphan Disease.
After grafting, the donor immunocompetent cells often trigger an immune response against the recipient – called the “Host”. They will recognize the recipient’s own antigens as foreign and will seek to destroy them. The donor’s T cells thus attack the host’s tissues and organs. This phenomenon can even be observed between donor and host who are very close immunologically. This disease remains a major obstacle to therapeutic transplants in hemato-oncology… A so-called acute GvHD occurs in the weeks following transplantation. After a certain period of time, the reaction changes in nature and displays autoimmune disease characteristics. It becomes chronic, with a continuous aggravation, often uncontrolled by conventional immunosuppressive treatments, and has a poor prognosis, making cGvHD potentially fatal. This is why there is an urgent need for new therapeutic approaches.