Merck, AstraZeneca announce Phase 3 success for rare cancer drug

Merck and AstraZeneca are aiming to extend the use of their MEK inhibitor Koselugo to adult patients with neurofibromatosis type 1, following positive outcomes from a Phase 3 study. On Tuesday, the companies revealed that Koselugo achieved a “statistically significant and clinically meaningful” enhancement in response rate compared to placebo in adults, fulfilling the primary goal of the KOMET study. This crucial trial involved 145 adults suffering from symptomatic and inoperable plexiform neurofibromas.

AstraZeneca’s rare disease division, Alexion, plans to present the findings to regulatory authorities and share them at an upcoming medical conference, as per the press release. Koselugo had previously received approval in 2020 for specific pediatric patients aged 2 and older affected by this genetic disorder, which leads to benign tumors developing along nerves.

In the case of neurofibromatosis type 1, the enzymes MEK1 and MEK2 are overly active, leading to the formation of noncancerous tumors known as plexiform neurofibromas around the nerves. Koselugo works by inhibiting these enzymes, aiming to decelerate the growth of these tumors.

AstraZeneca obtained the rights to Koselugo (selumetinib) from Array BioPharma in 2003. However, the drug did not succeed in several cancer trials. In 2017, the UK pharmaceutical company included Koselugo in an $8.4 billion collaboration agreement with Merck, which also encompassed the drug Lynparza.