Global Blood introduces two Sanofi drugs for sickle cell disease

Global Blood introduces two Sanofi drugs for sickle cell disease

Global Blood introduces two Sanofi drugs for sickle cell disease.  This includes an upfront cost of $2.25 million and potential development, regulatory and commercial milestone payments.

On March 16, 2021, EST, according to foreign media reports, following the successful launch of the sickle cell disease drug Oxbryta on the market, the global blood therapy company Global Blood Therapeutics (GBT) is planning to introduce two early stages of Sanofi Project to consolidate its R&D pipeline. According to the documents, Sanofi will receive up to US$353 million in fees in exchange for the global use rights of the two assets, including US$2.25 million in upfront costs and potential development, regulatory and commercial milestone payments.

It is reported that these two early projects are from the Bioverativ division of Sanofi. Bioverativ was acquired by Sanofi for US$11.6 billion in early 2018. At that time, Bioverativ inherited Biogen’s two mature hemophilia products, Eloctate and Alprolix, and several rare blood disease pipelines were in preclinical research. , The indications cover many areas such as sickle cell anemia, hemophilia, and thalassemia.

Specific to the sickle cell drug target of this transaction, its working principle is also different from that of GBT’s flagship product Oxbryta. In general, Oxbryta’s mechanism of action is to prevent hemoglobin s from polymerizing or sickening. One Sanofi project uses a different anti-sickle mechanism from Oxbryta, and another project uses a new method to Reduce inflammation and oxidative stress.

“These mechanisms may be complementary to the Oxbryta mechanism, implying the possibility of this combination.” GBT said in a statement.

Sickle cell disease (SCD) is a serious, progressive, and debilitating genetic disease. Mutations in the β-globin gene lead to abnormal sickle hemoglobin (HbS) production. HbS makes red blood cells sick and Fragile, leading to chronic hemolytic anemia, vascular disease, elusive and painful vascular occlusion crisis. Simply put, hemoglobin polymerization is the fundamental cause of sickling and destruction of SCD red blood cells, and Oxbryta is also the first therapeutic drug approved to directly inhibit sickle hemoglobin polymerization.

In November 2019, Oxbryta received accelerated FDA approval for the treatment of hemolytic anemia in children and adults with SCD ≥12 years of age. Prior to this, the FDA has successively granted Oxbryta the breakthrough drug designation, fast-track status, orphan drug designation, and rare pediatric disease designation for the treatment of SCD.

In Europe, GBT announced on February 23, 2021 that the European Medicines Agency (EMA) has accepted Oxbryta’s marketing authorization application (MAA) and has initiated a standard review process. The application seeks EMA to grant Oxbryta full approval, which is also used to treat hemolytic anemia in sickle cell disease (SCD) patients 12 years and older.

HOPE is a randomized, double-blind, placebo-controlled phase 3 study, conducted in 60 research institutions in 22 countries around the world, and evaluated two dose levels of Oxbryta (1500mg and 900mg, once a day orally administered) and placebo The efficacy and safety of the treatment of SCD.

The study enrolled 274 SCD patients aged 12 years and older. The patients were randomly divided into groups at a ratio of 1:1:1. The results showed that the study reached the primary endpoint: at week 24 of treatment, Oxbryta was significantly more significant than placebo Increased hemoglobin levels and decreased hemolysis biomarkers. These results are consistent with the inhibition of HbS polymerization, Oxbryta shows the potential to improve disease.

It is also worth mentioning that as a condition of accelerated approval by the US FDA, GBT will continue to investigate Oxbryta in the HOPE-KIDS 2 study, which is a post-approval confirmatory study that uses transcranial Doppler (TCD) blood flow. Speed ​​to assess Oxbryta’s ability to reduce the risk of stroke in children aged 2-15.

At present, as the first drug to treat the root cause of SCD disease, the industry is still optimistic about Oxbryta’s commercial prospects. The pharmaceutical market research organization EvaluatePharma previously released a report predicting that Oxbryta will become the world’s best-selling SCD drug. Will reach 1.98 billion US dollars. Of course, some people in the industry believe that the commercialization of Oxbryta is not as good as expected, and the first year sales of last year may not be satisfactory.

Going back to the deal with Sanofi, in fact, this is not the first time GBT has made a move.

In August 2018, GBT announced that it had reached an exclusive global license agreement for inclacumab with Roche. GBT plans to use this drug to treat the vascular occlusion crisis in SCD patients. Roche will receive an advance payment of US$2 million from GBT and may receive up to Approximately US$125 million in development and marketization milestone payments; in December 2019, GBT announced a collaboration with Syros Pharmaceuticals to develop new therapies for sickle cell and β-thalassemia based on the Syros gene control platform.

(source:internet, reference only)

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