AstraZeneca: Koselugo Phase III Trial Meets Primary Goal In Adults With Neurofibromatosis Type 1

(RTTNews) - British drug maker AstraZeneca Plc (AZN.L, AZN) announced Tuesday positive data from global KOMET Phase III trial, in which Koselugo (selumetinib) showed statistically significant and clinically meaningful objective response rate versus placebo in adults with neurofibromatosis type 1 or NF1.

Alexion, AstraZeneca Rare Disease plans to share these data with regulatory authorities and present at a forthcoming medical meeting.

AstraZeneca and Merck & Co., Inc. are jointly developing and commercialising Koselugo globally.

NF1 is a rare, progressive genetic condition affecting an estimated 1.7 million individuals worldwide, majority adults. In 30-50% of patients, tumours develop on the nerve sheaths and may cause debilitating symptoms. There are no approved treatments for adults, leaving many to experience disfigurement, dysfunction, persistent pain or endure multiple surgeries.

In the largest, global randomised double-blind placebo-controlled multicentre Phase III trial in adults with NF1, Koselugo, an oral, selective MEK inhibitor, met its primary endpoint.

The company noted that results demonstrated reduction in tumour volume, building on established safety and efficacy profile of Koselugo in children and supporting expanded use in adults.

The safety profile of Koselugo in this study was consistent with that observed in clinical trials among children and adolescents. No new safety signals were identified.

Marc Dunoyer, Chief Executive Officer, Alexion, AstraZeneca Rare Disease, said, "These promising results demonstrate that Koselugo, the first and only approved targeted therapy for certain children with NF1 PN, now has the potential to benefit adult patients for whom there are no approved targeted therapies. As the largest and only global placebo-controlled Phase III trial in adults with NF1 PN, KOMET reinforces our leadership in advancing potential treatment options for people living with this debilitating disease."

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