SpringWorks eyes US filing for MEK inhibitor after positive PhIIb data in rare disease

Spring­Works Ther­a­peu­tics has un­veiled pos­i­tive da­ta from a mid-stage tri­al of its drug can­di­date for neu­rofi­bro­mato­sis type 1 (NF1) as­so­ci­at­ed plex­i­form neu­rofi­bro­mas (PN), paving the way for a US fil­ing and po­ten­tial pri­or­i­ty re­view next year.

The Phase IIb ReNeu tri­al stud­ied mir­dame­tinib in 114 sub­jects aged two years and old­er with in­op­er­a­ble NF1-as­so­ci­at­ed PNs and re­sult­ing sig­nif­i­cant mor­bid­i­ty. The tri­al’s pri­ma­ry end­point was ob­jec­tive re­sponse rate, which was de­fined as a min­i­mum 20% re­duc­tion in tar­get tu­mor vol­ume. Topline da­ta showed that 52% of chil­dren and 41% of adults achieved this mea­sure.

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