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 preventives such as Nurtec and Qulipta 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 adults with episodic 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.