TIF is excited to be participating in the 26th EHA Annual Congress, held this year virtually on 09 – 17 June 2021.
Ms Lily Cannon, TIF Operations Manager, attended a satellite symposium entitled ‘Pyruvate Kinase Activation: Update on Clinical Trial Results across Hemolytic Anaemias’ discussed by Dr EJ van Beers (Netherlands), Dr Hanny Al-Samkari (USA), and Dr Kevin Kuo (Canada).
The results of the clinical trials of Mitapivat in pyruvate kinase deficiency, thalassaemia, and sickle cell disease were presented, demonstrate across all disease areas that the activation of pyruvate kinase with Mitapivat represents a potential therapeutic mechanism that may improve clinical outcomes in each of the aforementioned conditions.
More specifically, Mitapivat has shown:
For Pyruvate Kinase Deficiency: 2 Phase 3 clinical studies (one with patients who are not regularly transfused and one for patients who are regularly transfused) showed a sustained increase in haemoglobin (40% and 37% respectively), thus reducing their transfusion burden. 22% of regularly transfused patients becoming transfusion – free, and half of those achieving normal haemoglobin levels.
For Thalassaemia: A phase 2 clinical study with non-transfusion dependent α- and β-thalassaemia showed a mean haemoglobin increase of 1.3g/dl sustained over 12 week period. These positive results will drive forward to two phase 3 trials, to be initiated later in 2021, one for NTDT patients and one for TDT patients.
For Sickle Cell Disease: A proof-of-concept phase 1 study in sickle cell disease has been completed, demonstrating safety and tolerability in patients as well as changes in haemoglobin (mean increase of 1.3g/dl) and haemolytic markers. The trial essentially proved that Mitapivat is potentially applicable to improve the clinical outcomes for sickle cell disease patients, and as such as phase 2/3 trial will be initiated later this year.
The esteemed panel discussed the potential therapeutic value of Mitapivat across the three haemolytic anaemias, remarking that the patient-reported outcome tools developed for the studies demonstrated that even a modest improvement in haemoglobin levels (>1g/dl) went a long way to improve quality of life markers. Furthermore, looking to the future the panel indicated that there are numerous contributing factors to the achievement of transfusion-independence in transfusion-dependent thalassaemia patients, speculating that genotype and those who start transfusions later in life will more likely achieve transfusion independence.
Concluding the panel indicated the promising results of all the clinical studies, across disease areas will continue to be evaluated.
Dr Androulla Eleftheriou, TIF Executive Director, and Ms Lily Cannon have attended the following satellite symposia on 15 June 2021:
- MDS AND Β-THALASSEMIA: UPDATES IN ANEMIA TREATMENTS discussed by Prof. Vip Viprakasit (Thailand), Prof. Thomas Coates (USA) and Prof. Ali Taher (Lebanon).
- GETTING TO THE ROOT OF SICKLE CELL DISEASE – PATHOPHYSIOLOGY MEETS CLINICAL OUTCOMES discussed by Prof. Baba Inusa (UK) and Prof. Elliot Vichinsky (USA).
- GENE THERAPY FOR HEMOGLOBINOPATHIES: PROGRESS AND CLINICAL PERSPECTIVES discussed by Prof. Franco Locatelli (Italy), Prof. Alexis Thompson (USA) and Dr. Julie Kanter (USA)
MDS AND Β-THALASSEMIA: UPDATES IN ANEMIA TREATMENTS
The wider benefits of reducing transfusions and iron overload in β-thalassaemia patients were discussed by Prof. Vip Viprakasit (Thailand), Prof. Thomas Coates (USA), and Prof. Ali Taher (Lebanon). Prof. Taher presented the latest results of the BEYOND clinical trials that studied the safety and efficacy of Luspatercept in NTDT patients; 77.1% of participants achieved an increase in haemoglobin and 89.6% of these remained transfusion-free for at least 24 weeks.
Prof. Coates highlighted the unique challenges presented by NTDT in terms not only of diagnosis but of the initiation of transfusion (especially in patients that have higher haemoglobin levels) to promote growth and development. Human population movements in the last decade have seen a surge of NTDT patients in Northern Europe and the US, mandating international cooperation with medical specialists in high prevalence countries imperative, concluded Prof. Coates. The esteemed panel agreed that Luspatercept will be a game-changer for NTDT patients, allowing patients to forego transfusions and their complications.
GENE THERAPY FOR HAEMOGLOBINOPATHIES: PROGRESS AND CLINICAL PERSPECTIVES
An overview of the current approaches to gene therapy (gene addition and gene editing) for thalassaemia and sickle cell disease were discussed by Prof. Franco Locatelli, Italy, Prof. Alexis Thompson, USA, and Dr. Julie Kanter, USA. The experts commented on the burden of disease for patients indicating blood transfusion and pain to be the largest elements of burden for thalassaemia and sickle cell disease patients respectively. The elimination of either of these would hugely impact the quality of life of patients and alleviate the stress of daily life.
The eligibility criteria for patients, including upper / lower age limits continues to be a discussion topic for experts who indicated that such therapies may be more applicable to younger populations with good organ function, low iron load, and without severe complications. Nonetheless, the data from both authorised the gene therapy product and gene editing studies still in clinical trials are still being obtained in regards to safety and efficacy, with promising results.
GETTING TO THE ROOT OF SICKLE CELL DISEASE: PATHOPHYSIOLOGY MEETS CLINICAL OUTCOMES
An in-depth analysis of the clinical manifestations in relation to the underlying disease mechanisms of sickle cell disease were discussed by Prof. Baba Inusa (UK) and Prof. Elliot Vichinsky (USA).
The expert panel, highlighting the severe complications of sickle cell disease and their management, provided an all-round perspective of the effective management of the disease, noting the impact on the quality of life of patients. The panellists remained inspired by the plethora of new investigative clinical trials for the provision of more options for sickle cell disease patients both in terms of clinical management and curative approaches.
IRON CHELATION IN 2021
Dr Androulla Eleftheriou has attended an education session delivered by Prof. John Porter focusing on Iron Chelation in 2021 on 15 June 2021.
EHA-PATIENT JOINT SYMPOSIUM: REDUCING BUREAUCRACY IN CLINICAL TRIALS
ΤΙF has attended the 3rd session of the EHA-Patient Joint Symposium for 12 June 2021.
The Symposium entitled ´´Reducing Bureaucracy in Clinical Trials´´ highlighted the need for quality studies in a less bureaucratic regulatory environment. EHA together with patient representatives, including Mr George Constantinou on behalf of TIF (an expert patient and TIF Board Member), lead an initiative in collaboration with the European Medicines Agency (EMA) to identify pragmatic solutions.
A relevant Position Paper has been issued by this multi-stakeholder consortium.
EDUCATIONAL AND SCIENTIFIC WORKING GROUPS SESSIONS
Dr Androulla Eleftheriou, and Dr Michael Angastiniotis, TIF Medical Advisor, have attended education and scientific working group sessions on 11 June 2021.
- Prof. Antonio Piga on the potential of hepcidin as a therapeutic agent in haemoglobinopathies, Prof. John Porter on Iron Chelation in 2021
- Disease global morbidity and mortality
- Dr Lucia de Franceschi on novel fetal haemoglobin inducer drugs
EHA-PATIENT JOINT SYMPOSIUM: LESSONS FROM COVID-19
TIF has also attended the 2nd session of the EHA-Patient Joint Symposium for 11 June 2021.
The panel, including representatives of the European Medicines Agency (EMA), healthcare professionals and patients was chaired by Mr George Constantinou, an expert patient, leader of TIF’s Patient Advocacy Group and member of TIF’s Board of Directors.
The Symposium entitled ‘Lessons from the COVID-19 pandemic’ highlighted that the lives of chronic haematology patients have not changed; however the patient pathway has been radically altered in favour of telemedicine (e.g. telephone consultations). This has impacted patients psychologically as have social distancing measures, heightening isolation and marginalisation emotions.
The pandemic is a multi-faceted challenge for healthcare systems, that are in urgent need of reinforcement in order to improve readiness for future viral pandemics; the foundations are already being laid in the European Union with the revision of key legislations and the broadening of the mandate of the EMA to allow for more flexibility and adaptability in the regulation of new medicinal products. These mechanisms will hopefully continue to be implemented in non-pandemic products, thus getting them to patients faster.
Mr Constantinou highlighted the important role that patient organisations, such as TIF, have played throughout the course of the pandemic in the provision of quality, evidence-based information about therapies and vaccines which could not have been done without the continuous collaboration of treating physicians and professional societies such as the EHA.
EHA-PATIENT JOINT SYMPOSIUM: FAIR PRICING OF ORPHAN MEDICINES
Dr Androulla Eleftheriou , TIF Executive Director, and Ms Lily Cannon, TIF Operations Manager, have attended the 2nd session of the EHA-Patient Joint Symposium for 9 June 2021.
The Symposium entitled ‘Fair pricing of orphan medicines‘ highlighted the importance of achieving a fair for all pricing for lifesaving innovative products and therapies, including the recently authorised gene and cell-based therapies.
The eminent discussion panel included patient representatives, health economists, payers, and healthcare professionals who jointly agreed that such therapies are radically changing medical care. Ηowever, methods to increase accessibility whilst recognizing the disease burden of rare haematological diseases and simultaneously safeguarding the sustainability of healthcare systems in fairly providing medicines for all patients, are urgently needed.
The engagement of patients and real-world data via patient registries is more important than ever before to inform the decisions of health authorities, as noted also in TIF’s Global Thalassaemia Review 2021.