Novimmune SA announces that patients diagnosed with hemophagocytic lymphohistiocytosis (HLH), and who are participating in the company's ongoing clinical trial of its NI-0501 monoclonal antibody, will now be able to be treated with the drug without having to first fail conventional treatments.
The company recently filed a protocol amendment with the US Food and Drug Administration as well as European regulatory authorities in order to allow first-line treatment in the study. Based on available preliminary data from HLH patients treated with NI-0501 after having failed conventional therapies, regulatory authorities and the Ethical Committees having already completed the review process allowed the proposed amendment to proceed with no change.
The amended protocol has since been implemented at the HLH Center of Excellence of the Cincinnati Children's Hospital Medical Center, at Texas Children's Hospital in Houston, Texas, and at Alfred I. DuPont Hospital for Children in Wilmington, Del., and some participating hospitals in Europe. It is expected that all designated study sites will be authorized to implement the amended study protocol within the next several weeks.
"I am extremely pleased that, from now on, critically ill children at any stage of the disease, will have the opportunity to receive a targeted therapy which, because of its mechanism of action, does not cause the toxicities associated with the use of conventional HLH therapies" said Cristina de Min, MD, Chief Medical Officer at Novimmune.
The study is designed to evaluate the potential of NI-0501 as a targeted treatment for the disease with the objective of obtaining marketing authorization. NI-0501 is the first treatment specifically developed for HLH, a disease for which no drugs are currently approved. The study is currently underway at four investigational sites in the US and 14 sites in five European countries.
NI-0501 is a fully human monoclonal antibody that neutralizes interferon gamma, a cell-signaling protein believed to play a pivotal role in the development of HLH. In the ongoing Phase II study, NI-0501 was initially administered to patients showing unsatisfactory response or intolerance to conventional therapies including chemotherapy, immune-suppressive medicines, and immune-depleting drugs. The approved amendment to the protocol allows NI-0501 to be administered to patients as a first-line treatment before any other intervention.
HLH is a rare and severe disorder characterized by abnormal activation of the human immune system. The resulting inflammation rapidly attacks various parts of the body leading to multi-organ failure. The disease is fatal within a few weeks if left untreated and has a 40% to 50% mortality rate with current available treatments. HLH primarily affects infants and young children, but is becoming increasingly recognized in adults as the worldwide medical community becomes more familiar with its symptoms.
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