Red blood cell maturation agent Rotexip significantly improves anemia and quality of life in patients with transfusion-independent β-thalassemia

Bristol-Myers Squibb (NYSE: BMY) and Acceleron Pharmaceuticals (NASDAQ: XLRN) jointly announced today the first data of the Phase II clinical study BEYOND
.

"The chronic anemia and iron overload experienced by patients with transfusion-independent β-thalassemia may lead to a series of clinical complications
.

Rotexip is the world's first and currently only red blood cell maturation agent for the treatment of related anemias such as β-thalassemia and low-risk myelodysplastic syndrome.
It has been approved in the European Union, the United States and Canada
.

“In the BEYOND study, Rottsipp significantly improved the patient’s condition and we are very encouraged
.

"The research results presented at the EHA conference further emphasize the multiple benefits of Rotexip in the treatment of anemia and the realization of blood transfusion
.

About BEYOND study results

BEYOND is a phase II, randomized, double-blind, placebo-controlled multicenter study, which aims to explore the efficacy and safety of rotexip versus placebo in the treatment of adult transfusion-independent β-thalassemia
.

       In this study, 145 patients were randomized to receive rotexip1 1 mg/kg (adjustable to 1.
25 mg/kg) or placebo at a 2:1 ratio, subcutaneously injected once every 3 weeks, treatment for ≥48 weeks
.

       In the case of no red blood cell transfusion, from the 13th week to the 24th week in a continuous 12-week interval, 74 (77.
1%) of the 96 patients treated with rotexip reached the primary endpoint of the study, that is, the average Hb increased by ≥1.
0 g/dL from baseline, and 0 (0%) of 49 patients in the placebo group (P<0.
0001)
.

       The most common adverse events that occurred during treatment at any level in ≥5% of patients were bone pain (36.
5% in the rotexip group vs 6.
1% in the placebo group), headache (30.
2% vs 20.
4%) and arthralgia ( 29.
2% vs 14.
3%)
.
In patients treated with rotexcept, there were no reports of malignant tumors or thrombotic events
.

       About beta thalassemia

       Beta-thalassemia is an inherited blood disease caused by hemoglobin gene defects, and it is one of the most common autosomal recessive genetic diseases
.
According to statistics, the total annual incidence of symptomatic individuals worldwide is 1/100,000, and that of the European Union is 1/10,000
.
This disease is related to ineffective red blood cell production.
Ineffective red blood cell production can cause the production of unhealthy red blood cells and a decrease in the number of red blood cells, which often leads to severe anemia
.
In this case, patients are usually weak and may cause other complications and other serious health problems
.
At present, there are limited treatments for β-thalassemia-related anemia.
The main treatment includes frequent red blood cell transfusion, but it may cause iron overload, which may lead to serious complications such as organ damage
.
5 Transfusion-independent thalassemia refers to thalassemia that does not require a life-long regular transfusion of red blood cells to survive.
Such patients usually only need occasional or frequent blood transfusions within a certain period of time
.

       About Rottsip

       Rotexip is the world's first red blood cell maturation agent, which has been proven to promote late red blood cell maturation in animal models
.
As one of the global strategic cooperation projects, Bristol-Myers Squibb and Acceleron are jointly developing Rotsipp
.
Currently, Rotexip has been approved for treatment in the United States:

       • Adult β-thalassemia patients who require regular red blood cell transfusion

       •Erythropoiesis stimulant therapy fails and requires the infusion of 2 or more red blood cells within 8 weeks of very low to moderate risk myelodysplastic syndrome with ring sideroblasts (MDS-RS) or myeloproliferation Anemia associated with abnormal/myeloproliferative tumors with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T) in adult patients

       Rotexip cannot be used as a substitute for red blood cell transfusion for patients who need immediate correction of anemia
.

       references:

       1.
REBLOZYL Summary of Product Characteristics.
Accessed May 2021.

       2.
REBLOZYL US Prescribing Information.
Accessed May 2021.

       3.
REBLOZYL Canada Product Monograph.
Accessed May 2021.

       4.
ClinicalTrials.
gov.
A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia (BEYOND).
Available at: https:// ?term=BEYOND&cond=Beta-Thalassemia&rank=2.
Accessed May 2021.

       5.
Galanello R, Origa R.
Beta thalassemia.
Orphanet Journal of Rare Diseases.
2010;5(11).
Available at: https://ojrd.
biomedcentral.
com/articles/10.
1186/1750-1172-5-11.
Accessed May 2021.

       6.
Musallam, KM, Rivella, S.
, Vichinsky, E.
, & Rachmilewitz, EA (2013).
Non-transfusion-dependent thalassemias.
Haematologica, 98(6), 833–844.
https://doi.
org/ 10.
3324/haematol.
2012.
066845.
Accessed May 2021.
 

       Note: Rotsipp has not yet been approved in mainland China