Big, historic day in Brussels! Today CTF Europe is kicking off a groundbreaking new clinical trial initiative designed to move more NF treatments to patients - faster. We're launching the EU-PEARL NF1 platform basket trial, which will test multiple therapies in parallel across different NF1 manifestations - and bring new therapies into the trial more quickly, instead of waiting years for separate, one-off trials to open one drug at a time.

This is a historic milestone: Europe's first platform basket trial for a rare disease. And the blueprint we've designed with our partners has implications far beyond NF - more than 7,000 rare diseases could benefit from this kind of trial model.

NF1 isn't one-size-fits-all - and this trial is built for that reality. It will study multiple NF1 manifestations within one coordinated trial structure, led by:

• Plexiform neurofibromas (PN): Jaishri Blakeley + Ignacio Blanco
• Cutaneous neurofibromas (cNF): Pierre Wolkenstein + Laura Fertitta
• Low-grade gliomas / optic pathway gliomas (LGG/OPG): Darren Hargrave + Astrid Marie Sehested
• Biostatistics: Guenter Heiman

This European launch builds on the master protocol and trial strategy CTF developed through EU-PEARL - EU Patient- cEntric clinicAl tRial pLatforms(a European public-private partnership supported by Innovative Health Initiative (IHI)/ IMI ) - and on what we've learned through INTUITT-NF2 in the U.S., where this same trial model is accelerating therapies for NF2-related schwannomatosis. CTF identified brigatinib and brought it into INTUITT - and the trial is showing tumor shrinkage in NF2 patients.

A special note of gratitude: CTF Europe presented Guenter Heiman with our Cloud Carrier Award for his incredibly strong support in the development of this NF1 platform basket trial.

Thank you to EFPIA - European Federation of Pharmaceutical Industries and Associationsfor hosting today's convening in Brussels.

What's next?The first patient recruitment later this year, watch ctf.org and our social channels for those updates.