La Jolla Pharmaceutical Company has announced plans to discontinue Study LJ401-BT01 due to mixed clinical results. LJPC-401 is a synthetic human hepcidin for the treatment of conditions characterized by iron overload. Study LJ401-BT01 (NCT03381833) is designed to evaluate the safety and efficacy of LJPC-401 as a treatment for iron overload in beta thalassemia patients who, despite chelation therapy, have cardiac iron levels above normal. A recent interim analysis involving half of the enrolled population suggested lack of efficacy, with patients on the treatment and control arms exhibiting similar changes in cardiac iron levels, the primary endpoint, as well as key secondary endpoints. For more information, read La Jolla News here.

Oxbryta™ is an oral medication for the treatment of sickle cell disease (SCD) by directly inhibiting sickle haemoglobin polymerisation. As a condition for having received accelerated approval from the FDA, Oxbryta™ will be assessed in a post-approval confirmatory study, called HOPE-KIDS 2, which is set to launch by the end of the year. The trial will test Oxbryta™ tablets at a daily dose of 1500 mg, its approved dose, in pediatric patients (age 2 to 15) with SCD, and its primary goal will be to demonstrate that treatment with Oxbryta™ can reduce the risk of stroke in SCD children at 24 weeks of treatment. The trial will use transcranial doppler (TCD) to measure flow velocity. The trial will run at 50 sites across the U.S., Europe, and Africa. For more info see here.

Luspatercept-aamt, marketed under the brand name REBLOZYL® (Celgene and Acceleron Pharma Inc.), has received a Priority Review designation from the U.S. Food and Drug Administration (FDA) for the treatment of anaemia in beta-thalassemia (see FDA press release here). This is exciting news for transfusion-dependent beta-thalassaemia patients as REBLOZYL®, for the first time, will help decrease the number of blood transfusions and consequent iron-induced dysfunction by decreasing the iron-loading burden. The approval for REBLOZYL® is based on the results of the BELIEVE trial, which showed that 21.4% patients treated with REBLOZYL® achieved at least 33% reduction in transfusions compared to 4.5% of patients who received a placebo. The transfusion reduction meant that the patient needed fewer transfusions over 12 consecutive weeks while taking REBLOZYL®. Common side effects of the treatment included headache, bone pain, arthralgia (joint pain), fatigue, cough, abdominal pain, diarrhea and dizziness. REBLOZYL® also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The FDA granted approval of REBLOZYL® to Celegene Corporation. See Celgene press release here.

Voxelotor, marketed under the brand name Oxbryta™ (Global Blood Therapeutics Inc., GBT), has received Accelerated Approval from the U.S. Food and Drug Administration (FDA) for the treatment of adults and pediatric patients aged 12 years and older with sickle cell disease (SCD). Oxbryta™ is a once-daily pill that works by directly inhibiting sickle haemoglobin (Hb) polymerization, the main cause of SCD. The drug’s approval came three months earlier than expected and is based on results from the randomized, global Phase 3 HOPE trial (NCT03036813). Trial findings were published in The New England Journal of Medicine, showing clinically meaningful and statistically significant improvements in Hb levels, accompanied by reductions in red blood cell destruction (hemolysis). Briefly, 51.1% of patients receiving Oxbryta™ for 24 weeks at 1500 mg daily, its now approved dose, achieved a greater than 1 g/dL increase in Hb compared with 6.5% receiving placebo (n=274, P<0.001). Common side effects for patients taking Oxbryta™ were headache, diarrhea, abdominal pain, nausea, fatigue, rash and pyrexia (fever). In spite FDA Fast Tract designation, further clinical trials are required to verify and describe the drug's clinical benefit. Oxbryta™ also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. For more information, read GBT press release here and FDA press release here

The Cooley’s Anemia Foundation is accepting applications for medical research grants and fellowships in areas related to thalassaemia. The awards are in 3 categories:

1. Support for Ongoing Clinical Research in Thalassaemia (Deadline: December 20, 2019 for a letter of intent and February 3, 2020 for invited full applications).

2. Clinical Trials in Thalassaemia Cell and Gene Therapy Deadline: December 20, 2019 for a letter of intent and February 3, 2020 for invited full applications).

3. Research Fellowships  (Deadline: February 3, 2020).

For a description of each award category and further information on the criteria for each, please click here.

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