12-07-2012: Cytheris SA announced that the European Commission has granted an orphan designation for Cytheris' CYT017, glycosylated recombinant human interleukin-7 (glycosylated r-h-IL7), for the treatment of Progressive Multifocal Leukoencephalopathy (PML).
Additionally, Cytheris obtained scientific advice for a phase IIb study protocol with CYT107 in HIV-related PML. Cytheris has reached an agreement with the EMA on the key study endpoints. Cytheris will start this phase IIb study, intended to be pivotal, in early 2013.
"This orphan designation strongly supports the development of CYT107 as the treatment for PML. The disease has a mortality rate of around 40 to 50 per cent at one year whilst survivors often present severe neurological complications and disabilities," said Therese Croughs, chief medical officer at Cytheris. "We already gathered clinical data supporting the efficacy and safety of CYT107 in PML through several compassionate use treatments. We are committed to conduct our pivotal clinical study as rapidly as possible."
CYT107 is also in development in HIV Immune Non-Responders (HIV-INR) virologically controlled by anti-retroviral treatment. Fifteen to twenty five per cent of HIV patients are lymphopenic despite optimal treatment (HAART) leading to a significant increase in risk of death and serious complications. CYT107 also holds great promise in other lymphopenic conditions such as idiopathic lymphopenia or cancer associated lymphopenia.