The FINANCIAL — Novartis announced new data from two Phase II trials of ACZ885, in patients with one of two rare syndromes characterized by periodic fevers. Key findings include reductions in disease attack frequency, maintenance of symptom relief, normalization of blood markers of inflammation, and quality of life improvements.
Both rare orphan diseases, Familial Mediterranean Fever (FMF) and tumor necrosis factor (TNF) receptor-associated periodic syndrome (TRAPS), are serious, inherited autoinflammatory diseases characterized by recurrent (periodic) fever attacks, rash, arthritis, and severe symptoms that can lead to fatal complications.
"During attacks, patients suffering with these periodic fever syndromes can be incapacitated with pain and fever," said Dr Ahmet Gül, lead FMF study investigator and Professor of Rheumatology at the Istanbul Faculty of Medicine, in Istanbul, Turkey. "Although rare, these syndromes have a serious impact on the patients and families by limiting their ability to participate in daily life during an attack, so it is critically important to research new treatment options that can neutralize the underlying inflammation, and help patients to live a normal life."
The results of the two ACZ885 studies, which met their primary endpoints, are being presented at the 2012 Annual Scientific Meeting of the American College of Rheumatology (ACR) in Washington D.C., US, which takes place on 9-14 November. ACZ885 inhibits interleukin-1 beta (IL-1 beta), which plays a key role in several debilitating autoinflammatory diseases, including rare periodic fever syndromes.
"The role of IL-1 beta as a key player in a number of serious autoinflammatory conditions is becoming ever clearer", said John Hohneker, Head of Global Development for Integrated Hospital Care for the Pharmaceuticals Division of Novartis. "We are dedicated to realizing the potential of ACZ885, wherever alternative treatment options fail to appropriately help patients and whenever a strong scientific rationale comes into play."
In the Phase II FMF study, 100% of patients (nine out of nine) achieved at least a 50% reduction in the frequency of disease attacks during three months of ACZ885 treatment. All nine patients in the trial had previously experienced at least one attack per month over three months before receiving ACZ885, while using standard-of-care medication. As Novartis AG reported, during the three-month ACZ885 treatment period, eight of the nine patients were attack-free, while blood markers of inflammation (C-reactive protein, CRP, and serum amyloid A, SAA) normalized by Day 8 after ACZ885 dosing and remained low throughout the study. The overall response to treatment was reported to be 'very good' by physicians in all cases, and by the patients in seven cases.
New data from the Phase II study in TRAPS showed that after rapid clinical remission and normalization of CRP and/or SAA, which was maintained with continued ACZ885 treatment, the median time to relapse after ACZ885 withdrawal was three months. Patients also regained their previous response upon re-dosing with ACZ885[2]. Prior to study entry, patients experienced a mean of 10 attacks per year. When assessed during the initial four-month ACZ885 treatment period, patients experienced improvements in both physical and mental measures of quality of life.
Adverse events (AEs) observed in the studies were similar to those already seen for ACZ885's approved indication in Cryopyrin-Associated Periodic Syndromes (CAPS). Infections, mostly upper respiratory tract infections (URIs) were the most commonly reported category of AE in both the FMF and TRAPS studies. No serious adverse events (SAEs) were reported in the FMF study; while in the TRAPS study, two SAEs were reported: a URI that lasted two days and a severe disease attack with chest pain.
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