Date: 2015-12-01
Type of information: Presentation of results at a congress
phase: 2
Announcement: presentation of results at the 2015 Annual Meeting of the American Society of Hematology in Orlando, Florida on Tuesday, December 8.
Company: NovImmune (Switzerland)
Product: NI-0501
Action
mechanism: monoclonal antibody. 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.
Disease: hemophagocytic lymphohistiocytosis (HLH)
Therapeutic area: Rare diseases - Immunological diseases
Country: Austria, Germany, Italy, Spain, Czech Republic, USA
Trial
details: International, multicenter, long-term, follow-up study that will enroll HLH patients who have received NI-0501 in a previous clinical trial, in the context of the clinical development program for NI-0501. 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.(NCT02069899)
Latest
news: * On December 1, 2015, Novimmune announced that the abstract on Hemophagocytic Lymphohistiocytosis (HLH) will be presented in the Late-Breaking Abstracts oral session at the 2015 Annual Meeting of the American Society of Hematology in Orlando, Florida on Tuesday, December 8. This abstract reports preliminary results from Novimmune’s pilot Phase 2 study in children with primary HLH treated with NI-0501, a fully human monoclonal antibody that targets interferon-gamma and is the first treatment developed specifically for HLH. Oral abstract presentation on HLH is as follows: A Novel Targeted Approach to the Treatment of Hemophagocytic Lymphohistiocytosis (HLH) with an Anti-Interferon Gamma (IFN-gamma) Monoclonal Antibody (mAb), NI-0501: First Results from a Pilot Phase 2 Study in Children with Primary HLH. Authors: Michael Jordan, MD, Franco Locatelli, Prof, MD, PhD, Carl Allen, MD, PhD, Fabrizio De Benedetti, MD, PhD, Alexei A. Grom, MD, Maria Ballabio, MD, Walter Giovanni Ferlin, PhD, NI-0501-04 Study Group and Cristina De Min, MD (Abstract Number: 87376) * On March 16, 2015, Novimmune announced 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 FDA 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. 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.
