International Thalassaemia Day is marked on May 8 every year and is observed by the Thalassaemia International Federation (TIF). This International Thalassaemia Day, recognizes and celebrates the power of knowledge and brings the global haemoglobin disorders community together, by raising awareness, sharing knowledge, and bringing thalassaemia to the attention of as many people as possible worldwide. “Be Aware. Share. Care: Working with the global community as one to improve thalassaemia knowledge”, is the theme of 2022 day and is an open call to action to all supporters to promote awareness about thalassaemia and its global impact and share essential information and knowledge to support the best possible health, social and other care of patients with this disease. The theme seeks to inspire every individual to contribute, at the personal level, to the fight against thalassaemia and serves as a powerful reminder that everyone has a substantial role to play and a responsibility to act. For more info, see here.

Imara's IMR-687, marketed under the brand name Tovinontrine, is an oral disease-modifying treatment for sickle cell disease (SCD) and β-thalassaemia. IMR-687 was developed to selectively block phosphodiesterase 9 (PDE9) in red blood cells, which in turn increases cyclic GMP levels, as a means to reactivate the production of foetal haemoglobin (HbF), a well-established modifier of disease severity. Findings from the interim analyses in the Phase IIb studies forces Imara to discontinue both the Ardent and Forte trials, as well as the further development of tovinontrine in SCD and beta-thalassaemia. While the treatment was generally well-tolerated, no meaningful benefit was achieved in the rate of vaso-occlusive crises (SCD), transfusion burden or improvement in most disease-related biomarkers (β-thalassaemia). The company is grateful to the patients, investigators and their teams for their participation in these trials and to the extended Imara team for their role and dedication in generating the comprehensive interim results. Additional information: press release.

A recent study published in the Journal of Clinical Medicine demonstrated that Hemopexin which is a naturally-occurring protein safely reduced the signs of sickle cell disease (SCD) crises in cell and animal models. Hemopexin scavenges free heme in the bloodstream and is found at significantly lower levels in SCD patients. In an SCD mouse model, treatment with hemopexin decreased vascular inflammation and blockages associated with vaso-occlusive crises. Researchers wrote that ‘hemopexin is a promising new candidate to treat acute vaso-occlusive crises in people living with SCD and provides a basis for clinical development for this indication’. These findings support an ongoing Phase 1 multicenter clinical trial (NCT04285827), sponsored by the therapy’s developer CSL Behring, to evaluate the safety, tolerability, and pharmacokinetics of hemopexin (CSL889) in about 32 SCD adults, ages from 18 to 60. More information: Original publication.

The Global Globin Network (GGN), an initiative of the Human Variome Project (HVP), addresses the global health problems imposed by haemoglobinopathies, with focus on the integration of advanced diagnostic techniques in health systems and the systematic collection and sharing of variation and epidemiological data in internationally recognized databases. By pooling knowledge globally, GGN allows data-driven implementation of disease management and prevention programmes. In an article published in the March 31 issue of the Journal of Personalised Medicine, the GGN proposes a universally applicable system for evaluating and grouping countries based on qualitative indicators according to the quality of care, treatment, and prevention strategies for haemoglobinopathies. This approach serves both as a baseline for evaluating progress over time and for identifying areas of local priority that could benefit from knowledge and skill transfer under the proposed improvement scheme. All curated data is based on expert opinion and is accessible through the ITHANET Portal (https://www.ithanet.eu/). Country groupings into four categories A through D are based, respectively, on A) availability of well-established services with a national system for prevention and control, B) partial availability of services as part of a fragmented national control programme, C) availability of services but not as part of a sustainable national control programme, and D) scarcity of services or lack thereof. The classification of countries into groups based on similar needs and priorities for knowledge sharing and collaboration seeks to narrow the gap between countries, towards improved health outcomes for patients nationally within GGN partner countries and internationally. The paper is available here.

 

 

The U.S. Food and Drug Administration (FDA) has recently granted Orphan Drug Designation to Naproxcinod, a CINOD (Cyclooxygenase-Inhibiting Nitric Oxide-Donating) anti-inflammatory candidate, for the treatment of sickle cell disease (SCD) in the United States (US). The drug is developed by Nicox SA and exclusively licensed to Fera Pharmaceuticals in the US. Naproxcinod is an anti-inflammatory treatment that works by releasing nitric oxide and at the same time suppressing the activity of the enzyme cyclooxygenase, which is responsible for producing pro-inflammatory molecules. The treatment is designed to reduce pain and inflammation, and improve blood flow.
Nicox already completed a broad clinical program for naproxcinod in osteoarthritis, including three phase 3 studies with over 2,700 patients. Fera has conducted pre-clinical work on naproxcinod in models of SCD with promising findings furthering the development of the treatment for vaso-occlusive crises (VOCs), a frequent complication of SCD. More info: Fera press release, Sickle Cell Disease News

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