VIFOR-PHARMA
Vifor Pharma today announced that the first patient has been enrolled in a double-blind, randomized phase-IIa clinical trial evaluating the safety, efficacy and tolerability of vamifeport in adult patients with sickle cell disease. Vamifeport, developed by Vifor Pharma, is a novel oral ferroportin inhibitor investigated for treatment of diseases characterized by ineffective production of red blood cells and iron overload, including SCD and beta-thalassemia. It has been granted orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of SCD.
Dr. Klaus Henning Jensen, Chief Medical Officer of Vifor Pharma Group commented, “Starting the phase-IIa trial is an important milestone in the clinical development of vamifeport for sickle cell disease, a debilitating orphan condition for which there are very limited treatment options. Patients worldwide continue to suffer from consequences of the disease which dramatically impacts their quality of life and life expectancy. We look forward to continuing development of our potential new treatment for these patients”.
SCD is a group of inherited red blood cell disorders and is a genetic condition present at birth. In this disease, red blood cells carry abnormal haemoglobin, which makes them prone to rupture, causing adhesion of sickle cells and inflammatory cells to the blood vessels. This ultimately leads to an obstruction of blood flow and organ damage. There are an estimated 150,000 patients in Europe and the US combined living with sickle cell disease.
About the SCD-202 trial
The phase-IIa study is a randomized, double-blind, placebo-controlled, parallel group trial aiming to recruit 25 patients at 14 clinical sites in the United States, the United Kingdom, Lebanon and Greece. The primary objective of the trial is to assess the change in markers of hemolysis (the rupturing of red blood cells) upon treatment with vamifeport. Patients will undergo initial treatment for 4 weeks, followed by an additional 4-week period with either maintenance of the dose level or a dose step-up. The study also includes a 4-week follow-up period after treatment. End of enrolment is expected mid-2022 with topline results anticipated at the end of 2022.
Further information about the study, including eligibility requirements, is available at www.clinicaltrials.gov (NTC04817670).
About Vifor Pharma Group
Vifor Pharma Group is a global pharmaceuticals company. It aims to become the global leader in iron deficiency, nephrology and cardio-renal therapies. The company is a partner of choice for pharmaceuticals and innovative patient-focused solutions. Vifor Pharma Group strives to help patients around the world with severe and chronic diseases lead better, healthier lives. The company develops, manufactures and markets pharmaceutical products for precision patient care. Vifor Pharma Group holds a leading position in all its core business activities and consists of the following companies: Vifor Pharma and Vifor Fresenius Medical Care Renal Pharma (a joint company with Fresenius Medical Care). Vifor Pharma Group is headquartered in Switzerland, and listed on the Swiss Stock Exchange (SIX Swiss Exchange, VIFN, ISIN: CH0364749348).
For more information, please visit viforpharma.com .
About vamifeport
Vamifeport is an oral inhibitor of ferroportin, an enzyme essential for the body’s transport of iron and plays a key role in regulating iron uptake and distribution in the body. Vamifeport binds to ferroportin and blocks it to prevent excessive iron release into the blood. As iron is needed for the formation of haemoglobin, this relative decrease in iron availability is expected to reduce the concentration of abnormal haemoglobin in red blood cells and prevent ensuing unfavorable events. Vamifeport-mediated ferroportin inhibition may lead to less haemolysis, decrease of inflammation and better blood flow with consequent improvement of both the symptoms and clinical outcomes of SCD.
Vamifeport is currently also in phase II development for beta-thalassemia, an inherited rare blood disorder that reduces the production of functional haemoglobin in red blood cells, which can lead to a lack of oxygen in many parts of the body and potentially cause anaemia.
View source version on businesswire.com: https://www.businesswire.com/news/home/20211210005374/en/
Link:
About Business Wire
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
Sonrotoclax Data at ASH 2025 Confirm Foundational Potential Across B-cell Malignancies8.12.2025 00:00:00 CET | Press release
Novel BCL2 inhibitor sonrotoclax monotherapy demonstrates deep and durable clinical responses in R/R MCL and R/R CLLSonrotoclax in combination with BRUKINSA demonstrated rapid MRD negativity in treatment-naive CLL, regardless of high-risk features BeOne Medicines Ltd. (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, today announced new data on sonrotoclax, a next-generation investigational BCL2 inhibitor, demonstrating meaningful clinical benefit as monotherapy and in combination across B-cell malignancies. These data were featured at the 67th American Society of Hematology (ASH) Annual Meeting & Exposition in Orlando, Florida. The five presentations highlight durable responses in heavily pretreated patients with relapsed/refractory (R/R) mantle cell lymphoma (MCL) and additional studies showing deep, rapid, and sustained undetectable minimal residual disease (uMRD) rates with sonrotoclax-based combinations in patients with treatment-naive chronic lymphocytic leukemi
Andersen Consulting tilføjer samarbejdsfirmaet Codezilla7.12.2025 21:19:00 CET | Pressemeddelelse
Andersen Consulting styrker sine kompetencer inden for digital transformation gennem en samarbejdsaftale med Codezilla, et firma med hovedsæde i Rumænien, der udvikler specialtilpasset software. Codezilla specialiserer sig i at udvikle skræddersyede softwareprodukter, der løser forretningsmæssige udfordringer gennem en tværfaglig tilgang, som kombinerer softwareudvikling med dybdegående marketingekspertise. Med over 30 år på markedet fungerer firmaet som implementerings- og digital konsulentressource for reklamebureauer, samtidig med at de arbejder med en bred kundebase, herunder virksomheder inden for sundhedssektoren og medicinsk udstyr. Codezillas interne teams strækker sig over teknik, design og strategi og leverer omnichannel-løsninger til både regionale og globale kunder. "Vi tror på, at fantastisk software er resultatet af solid ingeniørkunst og disciplineret eksekvering," udtaler Sebastian Doroftei, administrerende direktør for Codezilla. "Vores samarbejde med Andersen Consulti
Hemato-Oncology Trials: AOP Health Presents New Results at Top Congress ASH7.12.2025 17:00:00 CET | Press release
AOP Health continues to advance its clinical research program in myeloproliferative neoplasms, a special group of rare blood cancers. The company, specialized in rare diseases, presented the results of two scientific investigations at the 67th American Society of Hematology Association (ASH) Annual Meeting 2025 held in Orlando, FL, USA. The results provide new insights in treatment strategies. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20251207587915/en/ Dr. Martin Steinhart, CEO AOP Health; Photo credit: AOP Health/Studio Koekart ROP-ET and BESREMI PASS One of the clinical studies, ROP-ET, examined the use of ropeginterferon alfa-2b in people with essential thrombocythemia (ET), a disease in which the body produces too many platelets. The trial, a prospective, multicenter, single-arm phase III study, investigated the safety and efficacy of ropeginterferon alfa-2b in ET patients unable to receive available cytoreductive th
CoMotion GLOBAL 2025 Launches in Riyadh: Global Mobility Leaders Unite in Saudi Capital to Chart Urban Future7.12.2025 13:00:00 CET | Press release
Summit debuts Mayors in Motion initiative and CoMotion Urban Visionary Distinction as Riyadh showcases its rise as global mobility testbed Riyadh is rapidly becoming one of the world's most ambitious urban mobility laboratories, where next-generation technologies move from blueprint to real-world deployment on city streets at unprecedented scale. CoMotion GLOBAL 2025, the world's most influential gathering of urban mobility leaders, opens today in Riyadh for a three-day summit bringing together innovators from Africa, Asia, Europe, the Americas, and the Middle East. Running December 7-9, the event will explore how electrification, autonomy, AI-enabled transport, and giga-project urbanism are reshaping cities worldwide. The summit will spotlight everything from high-performance EVs and breakthrough autonomous fleets to emerging-market transport solutions and new mobility models, demonstrating how the Kingdom is opening new pathways for global mobility leadership. Strategic Partnerships
Deciphera Announces Oral Presentation of Positive Topline Results from Phase 2a Study of Sapablursen in Polycythemia Vera at the 67th American Society of Hematology (ASH) Annual Meeting6.12.2025 15:30:00 CET | Press release
Results from Phase 2a IMPRSSION study demonstrate sapablursen significantly reduced phlebotomy rate, controlled hematocrit and increased serum hepcidin Sapablursen was generally safe and well tolerated Results support further development of sapablursen in a Phase 3 study Deciphera Pharmaceuticals, a member of Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; “Ono”), today announced the oral presentation of positive results from the Phase 2a IMPRSSION study of sapablursen in patients with polycythemia vera (PV) at the 67th American Society of Hematology (ASH) Annual Meeting, taking place December 6-9, 2025, in Orlando, FL. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20251206361611/en/ The results were presented by Ionis Pharmaceuticals, who discovered and developed sapablursen and conducted the IMPRSSION study. In March 2025, Ionis and Ono entered into a license agreement in which On
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.
Visit our pressroom