For the first time, there are HHT therapies in late-stage trials.
There is still no treatment approved specifically for hereditary hemorrhagic telangiectasia (HHT). That is changing. Several programs are now moving through clinical trials, and Cure HHT is a named partner on the ones furthest along.
Drug development
Where HHT therapies stand today
Between 2018 and 2022, three potential HHT therapies were in development, and not one of them had reached a trial in people. Today there are 15 in the pipeline, and several are in trials that people living with HHT can join.
Each bar below is one therapy. The further the bar travels, the further that program has moved toward a treatment a doctor could prescribe.
2018 to 2022 · three therapies in development
Today · 15 therapies in development
Therapies are unnamed because several sponsors have not yet made their programs public. Source: Cure HHT 2025 Impact Report.
No therapy has been approved specifically for HHT yet. Approval is the last column on the chart, and reaching it depends on people taking part in the trials that come before it.
Clinical Trial Network
Partners at the forefront of drug development
A company deciding where to run a study needs sites that already see HHT patients, already apply the diagnostic criteria the same way, and can begin enrolling without a year of setup. The Cure HHT Clinical Trial Network is that set of centers, built and maintained so that a sponsor with a promising molecule has somewhere to take it.
These companies have HHT programs running in the network today.
Why this is happening now
A pipeline does not appear on its own
Rare diseases do not attract drug developers by default. Companies need to see a defined patient population, trial-ready centers, and a community that will take part. Cure HHT has spent years building exactly that.
The Clinical Trial Network gives sponsors a set of centers prepared to run HHT studies. The registries give researchers a picture of the population. And the relationships came first: the Vaderis partnership began in 2021, well before there was a Phase 3 study to announce.
Milestones along the way
2021
Cure HHT partnership with Vaderis begins, an early step into industry-sponsored HHT research
Oct 2024
PATH trial results published in the New England Journal of Medicine
Nov 2024
FDA grants Fast Track designation to the Vaderis allosteric AKT-inhibitor VAD044 for HHT
Jul 2026
First patient dosed in the DIAMOND trial
Aug 2026
Global Phase 3 HEROIC study initiated