MTX-101
A once-daily sublingual tablet for chronic migraine patients who don't respond to, or can't access, current CGRP therapies.
What sets it apart
A different mechanism
MTX-101 targets a different receptor from today's migraine preventives and modulates the effects of both CGRP and PACAP, two peptides thought to drive migraine. Approved oral CGRP drugs such as Nurtec®, Qulipta® and Ubrelvy® act only on the CGRP receptor.
A tablet, not an injection
Taken once a day under the tongue and stored at room temperature. Patients who stop CGRP antibodies, Botox® or infusions because of the route would have an oral option outside CGRP.
A known safety heritage
MTX-101 is repurposed from an approved ophthalmic medicine, so its systemic safety profile is already characterized. Tolerability as a daily sublingual tablet will be assessed in Phase 2b.
A shorter regulatory route
Because the active ingredient is already approved, MTX-101 may qualify for the 505(b)(2) pathway, which can rely in part on existing safety data.
Evidence to date
Full datasets are available to qualified parties under a confidentiality agreement.
Laboratory studies
In human artery tissue, MTX-101 reversed vasodilation induced by both CGRP and PACAP.
Early clinical studies
Early studies showed signals in both migraine prevention and acute relief.
Phase 2a pilot, completed 2025
A randomized, double-blind, placebo-controlled cross-over study of sublingual MTX-101 in acute migraine. NCT06728553 on ClinicalTrials.gov
Development path
The FDA has agreed to the Phase 2b design. A partner in place at Phase 2b would fund the remaining program.
- CompleteEarly clinical studies and Phase 2a pilot
- NextPhase 2b in chronic migraine
- ThenTwo Phase 3 trials
- Target 2033US launch, with expansion to episodic migraine
Intellectual property
- Method of use
- Issued US patent
- Sublingual formulation
- Patent application pending
See the data behind MTX-101
Clinical results, mechanism detail and FDA correspondence are available under a confidentiality agreement.