A Phase 2a, Randomized, Open-Label Study to Evaluate Multiple Dosing Regimens of Subcutaneous ALXN1820 in Adult Patients with Sickle Cell Disease

Dai, Y.; Hill, Kelley; Harris, Anne; Shen, Tong; Yuan, Chao‐Xing; Gasteyger, Christoph

doi:10.1182/blood-2022-166694

Cited by 3 publications

(2 citation statements)

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“…This makes the complement system a potential therapeutic target for SCD. ALXN1820 is a humanized, bispecific, VHH (variable heavy domain) antibody that simultaneously binds human albumin and properdin [83]. The antibody selectively inhibits CAP (complement alternate pathway) activation and has an extended circulatory half-life due to its binding to albumin.…”

Section: Alxn1820mentioning

confidence: 99%

“…The antibody selectively inhibits CAP (complement alternate pathway) activation and has an extended circulatory half-life due to its binding to albumin. Mouse models have demonstrated that properdin inhibition results in significantly reduced VOCs and hemolysis, making ALXN1820 a promising new therapy [83]. The PHOENIX trial (NCT05565092) is an ongoing phase 2a trial investigating the safety and efficacy of ALXN1820 in adults with SCD.…”

Section: Alxn1820mentioning

confidence: 99%

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Current and Future Therapeutics for Treating Patients with Sickle Cell Disease

Barak,

Hu,

Matthews

et al. 2024

Cells

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Sickle cell disease (SCD) is the most common genetic blood disorder in the United States, with over 100,000 people suffering from this debilitating disease. SCD is caused by abnormal hemoglobin (Hb) variants that interfere with normal red blood cell (RBC) function. Research on SCD has led to the development and approval of several new SCD therapies in recent years. The recent FDA-approved novel gene therapies are potentially curative, giving patients an additional option besides a hematopoietic bone marrow transplant. Despite the promise of existing therapies, questions remain regarding their long-term pharmacological effects on adults and children. These questions, along with the exorbitant cost of the new gene therapies, justify additional research into more effective therapeutic options. Continual research in this field focuses on not only developing cheaper, more effective cures/treatments but also investigating the physiological effects of the current therapies on SCD patients, particularly on the brain and kidneys. In this article, we undertake a comprehensive review of ongoing clinical trials with completion dates in 2024 or later. Our exploration provides insights into the landscape of current therapeutics and emerging novel therapies designed to combat and potentially eradicate SCD, including the latest FDA-approved gene therapies.

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Section: Alxn1820mentioning

confidence: 99%

Section: Alxn1820mentioning

confidence: 99%