Novo Nordisk Acquires Etavopivat Maker Forma Therapeutics for $1.1B

Forma is sponsoring two studies on the safety, effectiveness of etavopivat for sickle cell patients

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Forma Therapeutics, which is currently developing an experimental treatment for sickle cell disease (SCD) called etavopivat, is being acquired by Novo Nordisk, the two companies announced.

“Novo Nordisk has worked for more than 40 years to develop and deliver transformative medicines to patients around the world with rare and devastating diseases. By adding Forma’s differentiated approach to address unmet needs for patients, we are taking a step forward in enhancing our sickle cell disease pipeline,” Ludovic Helfgott, executive vice president and head of Rare Disease at Novo Nordisk, said in a press release.

“Today’s announcement is an exciting milestone that accelerates Forma’s purpose to transform the lives of patients with sickle cell disease and other serious hematological diseases,” Frank Lee, president and CEO of Forma, said.

Under the agreement, Novo Nordisk will purchase all outstanding shares of Forma’s common stock for $20 per share, for a total value of roughly $1.1 billion. The agreement has been approved by Forma’s board of directors; it will officially close following customary conditions.

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“We have an ambition to build a leading portfolio with standalone and combination treatments to tackle the complications and underlying causes of sickle cell disease,” Helfgott said.

SCD is caused by mutations that affect hemoglobin, the protein that red blood cells use to carry oxygen through the bloodstream. When not bound to oxygen, the mutated protein forms clumps inside red blood cells, deforming them into a sickle-like shape that gives SCD its name.

Etavopivat, formerly known as FT-4202, is designed to lower the levels of 2,3 diphosphoglycerate (2,3 DPG), a metabolic intermediate that can prevent oxygen from binding to hemoglobin by boosting the activity of an enzyme called pyruvate kinase-R (PKR) that normally uses 2,3 DPG as a substrate.

By reducing 2,3 DPG levels, the experimental oral therapy would increase hemoglobin’s affinity to oxygen, ultimately reducing red blood cell sickling.

Data from a previous Phase 1 trial (NCT03815695) involving SCD patients and healthy volunteers demonstrated etavopivat was generally well tolerated and able to improve measures of red blood cell health.

Forma is sponsoring a Phase 2/3 trial called HIBISCUS (NCT04624659), which is comparing two doses of etavopivat (200 or 400 mg/day) against a placebo in people with SCD, ages 12–65. The study’s main goals are to evaluate the effects of treatment on hemoglobin after six months, and on rates of painful vaso-occlusive crises (VOCs) after a year.

HIBISCUS aims to enroll about 344 participants. Enrollment is underway at more than 50 sites in the U.S., Canada, U.K., France, Germany, Italy, and Spain.

Forma is also running a Phase 2 study called GLADIOLUS (NCT04987489), which is assessing the safety and clinical activity of etavopivat (400 mg/day) in people with SCD requiring chronic red blood cell transfusions. The goal is to test whether the therapy can reduce the need for transfusions over 12 weeks.

GLADIOLUS is expected to enroll 60 participants, ages 12–65. Recruitment is ongoing at Children’s Hospital Los Angeles in California. In addition to SCD, the study is also open to people with another hemoglobin-related disorder called thalassemia.