Novo Nordisk Submits IND Application in China for Etavopivat, a PKR Agonist Acquired for $1.1 Billion

On December 25, the official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration announced that Novo Nordisk had submitted a clinical trial application for the new drug Etavopivat (Class 1) and the application has been accepted.

Source of the image: Screenshot from the CDE official website

According to publicly available data, Etavopivat is a selective pyruvate kinase-R (PKR) agonist that can activate the natural PKR within red blood cells, thereby reducing the content of the anaerobic glycolysis product 2,3-DPG in red blood cells. This enables the cells to carry more oxygen, increase ATP production, and reduce hemolysis and the formation of sickle cells. It is currently being developed for the treatment of sickle cell anemia (SCD).

Develop the domestic market

As an investigational, once-daily selective PKR agonist, Etavopivat can reduce the content of anaerobic glycolysis product 2,3-DPG in red blood cells by activating the natural PKR within them. This enables the cells to carry more oxygen, increase adenosine triphosphate (ATP) production, and reduce hemolysis and the formation of sickle cells, thereby improving red blood cell function as well as deformability, membrane repair capability, red blood cell health, and lifespan.

In the previous Phase 1 clinical trial conducted overseas, Etavopivat has demonstrated good tolerability and shown potential to improve red blood cell health, increase hemoglobin levels, reduce recurrent vaso-occlusive crisis (VOC) symptoms in patients, and enhance their quality of life.

Etavopivat is currently being evaluated in the global Hibiscus Phase 2/3 clinical trial for its efficacy and safety in patients with SCD. Additionally, another Phase 2 clinical trial, Gladiolus, is underway to assess the drug's effects in transfusion-dependent SCD patients, as well as those with another inherited hemoglobin disorder known as thalassemia.

In February 2020, the drug was granted Fast Track designation, Rare Pediatric Disease designation, and Orphan Drug designation by the U.S. FDA, as well as Orphan Drug designation by the European Commission (EC).

The clinical trial application of Etavopivat in China may further expand the domestic market and provide another drug option for SCD patients.

Acquired for $1.1 billion

Strengthening the Rare Disease Product Portfolio

Etavopivat was originally the lead pipeline of Forma Therapeutics. On September 1, 2022, Novo Nordisk and Forma Therapeutics announced that they had reached a definitive agreement under which Novo Nordisk would acquire the latter for a total price of $1.1 billion, or $20 per share, representing a premium of up to 92%. Since then, Novo Nordisk has brought Etavopivat into its portfolio.

In addition to Etavopivat, Forma also has another rare disease pipeline drug, Olutasidenib. Olutasidenib is an oral IDH1 enzyme inhibitor that can cross the blood-brain barrier and is being developed for the treatment of glioblastoma, relapsed/refractory acute myeloid leukemia, myelodysplastic syndromes, and other IDH1-mutant solid tumors.

Ludovic Helfgott, Executive Vice President of Novo Nordisk and Head of Rare Diseases, stated, "For over 40 years, Novo Nordisk has been committed to researching and providing transformative medicines for patients with rare and serious diseases worldwide. By incorporating Forma's differentiated approach to address unmet patient needs, we have taken another step forward in enhancing our company’s pipeline for sickle cell disease. We are ambitious in creating a first-class portfolio of single-agent and combination therapies to address both the complications and the root causes of sickle cell disease."

Source: https://mp.weixin.qq.com/s/EJQBne0y48H_r-alCwywvQ

Editor: Pea Shooter

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