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 purpose of this study is to evaluate the efficacy and safety of the world's first red blood cell maturation agent rotexip combined with best supportive therapy for the treatment of adult non-transfusion dependent (NTD) β-thalassemia

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

Therefore, they urgently need treatment options

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
.

For eligible patients, Rotexip has become an important treatment option

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

” Mr.

"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
.

In addition, Rotexip has also shown its potential role in the treatment of non-transfusion dependent diseases.

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
.

Eligible patients are patients with β-thalassemia or hemoglobin (Hb) E complex β-thalassemia, ≥18 years of age, and received red blood cell transfusion ≤5 units within 24 weeks before randomization, with an average baseline Hb People whose value is less than or equal to 10.

       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
.

The two groups of patients continued to receive the best supportive treatment, including red blood cell transfusion and iron removal therapy

       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)
.

Of the 55 patients with an average baseline Hb<8.

       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