Novo Halts Phase 2 of Forma-Acquired Myelodysplastic Drug

Novo Nordisk has recently discontinued a Phase 2 clinical trial assessing etavopivat for treating anemia in adults with various forms of myelodysplastic syndromes (MDS). This decision follows an update to the U.S. federal clinical trials database. The trial was terminated after a futility assessment indicated a low probability of achieving meaningful patient benefits.

Myelodysplastic syndromes are conditions where red blood cells fail to mature and function properly, often leading to leukemia. Despite this setback, Novo Nordisk continues to test etavopivat in mid- and late-stage trials for other conditions like sickle cell disease and thalassemia. A spokesperson for Novo Nordisk emphasized that the decision to halt the trial was difficult but necessary, reflecting the low likelihood of success in treating MDS.

The Danish pharmaceutical company did not prominently feature MDS in its acquisition announcement of Forma Therapeutics in late 2022. However, treating MDS was one of the potential applications for etavopivat that Novo Nordisk was exploring. The company remains optimistic about the drug's potential for sickle cell disease and thalassemia, asserting their confidence in its promise and their commitment to its continued development.

In January, Novo Nordisk highlighted the potential of etavopivat for MDS in its annual report. Nonetheless, the company has been realigning its pipeline since acquiring Forma Therapeutics. For instance, Novo has sold a preclinical cancer candidate to Debiopharm and a Phase 1 prostate cancer drug to Pathos AI.

Other pharmaceutical companies are also working on treatments for MDS-associated anemia. Agios Pharmaceuticals is developing a pyruvate kinase (PK) activator named tebapivat. Agios’ more advanced PK activator, mitapivat, is already approved for treating adult PK deficiency and is marketed under the name Pyrukynd. Additionally, Bristol Myers Squibb has developed Reblozyl, which is also indicated for MDS-related anemia.

Despite the termination of the Fortitude trial, Novo Nordisk is committed to exploring etavopivat's potential for other conditions. This development does not diminish their belief in the drug's ability to significantly impact patients' lives. As the company continues to navigate the complexities of drug development, it remains focused on delivering innovative treatments for sickle cell disease and thalassemia.