Hybrigenics is one step closer to enter the American market. The FDA granted Orphan Drug designation to the company’s Acute Myeloid Leukemia (AML) Treatment. Currently in preclinical stage, the therapy showed efficacy in combination with two AML-drugs that are already approved.
AML is the second most frequent form of leukemia and accounts for about 30% of all leukemic patients. It is characterized by a fast-increasing overproduction of immature white blood cells, called myeloblasts. These cells rapidly crowd the bone marrow, soon preventing it from making normal blood cells. They can also spill out into the blood stream and circulate around the body. Due to their immaturity, they are unable to function properly.
Current treatments include the two hypo-methylating anticancer drugs azacytidine or decitabine. Hybrigenics’ lead candidate is inecalcitol, a Vitamin D inhibitor that is currently tested in combination with those two. Preclinical trials showed that the combination improved efficacy to limit leukemia progression than the two approved drugs agents alone. Based on these, the FDA granted Orphan drug designation to the therapy.
This is very good news for Hybrigenics, a French company that is publicly listed in Paris and has a market cap of €58M. The designation will facilitate the next steps by reducing costs and enable a faster access to the patients. Moreover, it opens up the possibility to enter a much bigger market. In the United States, AML has recently become the most frequent leukemia with 36% of all newly diagnosed cases. In the end of last year, Hybrigenics also opened a subsidary in Boston/Cambridge, a first approach to the US after it survived 18 years in France. With this new designation, the company can easily conduct a part of the coming clinical studies there, as planned.