The FTD research landscape is on the verge of major developments. For the first time, a Phase 3 trial has been completed for a potentially disease-modifying treatment for genetic FTD, with results expected in the coming months, and a number of other trials are in the pipeline. As the data readout for this study approaches, we know that many families will have questions for their healthcare providers.

Approximately 40% of FTD cases are currently thought to be familial or have genetic underpinnings. Significant drug development progress is being made for genetic FTD, which will have learnings for sporadic FTD. The AFTD website has more information on FTD genetics and gene therapy.

Many current trials tackle the progranulin hypothesis – the idea that for people with FTD caused by GRN variants, restoring progranulin levels will reduce FTD pathophysiology and symptoms. While there are other major genes which contribute to FTD, GRN is the genetic target furthest along in trials. That is why we are focusing on GRN research here. There are also trials on other genes and aberrant proteins, which are being tested in people with related diseases such as ALS and Alzheimer’s, and which may also hold promise for FTD.

After a Phase 3 trial concludes, scientists review the results to see how the drug performed relative to the overall trial’s “endpoints,” or the criteria chosen before the trial to evaluate the treatment. Endpoints may include whether the drug resulted in biological changes or improved participants’ quality of life. Scientists also determine if the drug caused any adverse health effects and their severity.

After a trial has concluded and the results have been fully analyzed, they are often published and posted on ClinicalTrials.gov, a website maintained by the U.S. Department of Health & Human Services. The biopharmaceutical company that conducted the trial will also typically issue a press release containing its final results; AFTD will share updates as appropriate.

At the end of a clinical trial, the scientists may find that the drug did not significantly impact the trial’s endpoints in the intended direction. But even when a trial does not achieve its goals, it still provides a great deal of information, such as what works and does not work to treat FTD.

Results may also be inconclusive – neither clearly positive nor negative – and therefore require further study and/or additional input from the community.

But the results could bring good news for families facing FTD – actual positive or promising outcomes. In that case, the company that conducted the trial can formally ask the U.S. Food and Drug Administration (FDA) for approval by submitting a New Drug Application or a Biologics License Application. The FDA then evaluates all the data on the safety and therapeutic benefit of the drug or treatment before deciding whether to award its approval.

If approved, the FDA determines the drug’s label, which includes information such as how to use the drug, who it is for, and the stage of disease it is approved to treat. In many cases,  the FDA will ask the sponsor to re-review it before it gives approval, which may require additional studies and/or data-gathering. The FDA’s website contains more information on the drug review and approval process.

People impacted by FTD are a critical piece of the scientific puzzle. Whether or not a family is eligible or interested in participating in a clinical trial, there are a number of other ways they can help the field move forward towards a treatment. Here are more ways to participate in the science of FTD and more on the role of the community in drug development.

For the most up-to-date information on FTD trials, consider suggesting that patients and families enroll in the FTD Disorders Registry, which can keep them informed about new and emerging research opportunities. You can also refer them to AFTD’s HelpLine (info@theaftd.org, 866-507-7222) or clinicaltrials.gov.

Below is a list of status updates as of August 2025 from six active genetic FTD trials. (AFTD is an informational resource and does not specifically encourage or discourage patient participation in any specific clinical trial.)

Sponsor: Alector (with GSK)
Study: INFRONT-3 (Phase 3)
Drug: AL001 (latozinemab) – anti-sortilin monoclonal antibody; blocks sortilin-mediated granulin degradation
Delivery route: Intravenous
Update: Phase 3 enrollment is complete and results are expected by the end of the 2025 calendar year. Phase 2 updates were previously provided, and the FDA granted AL001 Breakthrough Therapy Designation in 2024.
Clinicaltrials.gov: NCT04374136

Sponsor: AviadoBio
Study: ASPIRE-FTD (Phase 1/2)
Drug: AVB-101 – GRN AAV9 gene therapy, increases progranulin levels by delivering a healthy GRN gene into the brain
Delivery route: Intrathalamic injection
Update: Dosing is completed of a second cohort in Phase1/2 and dosing of a third cohort is intended in Q3 2025, with the expectation of sharing early biomarker data in 2026.
Clinicaltrials.gov: NCT06064890

Sponsor: Denali Therapeutics & Takeda Pharmaceuticals
Study: Phase 1/2
Drug: TAK-594/DNL593 – recombinant progranulin combined with transport vehicle technology, increases progranulin levels by delivering the progranulin protein across the blood brain barrier
Delivery route: Intravenous
Update: Phase 1 part A data dosing of health volunteers was previously reported and part B dosing of people with GRN-related FTD is ongoing
Clinicaltrials.gov: NCT05262023

Sponsor: Passage Bio
Study: upliFT-D (Phase 1/2)
Drug: PBFT02 – GRN AAV1 gene therapy, increases progranulin levels by delivering a healthy GRN gene into the brain
Delivery route: Intracisternal injection
Update: Interim data has been announced from Dose 1 and interim data from Dose 2 is expected in the second half of 2025. Plans to soon include participants with FTD-C9orf72.
Clinicaltrials.gov: NCT04747431

Sponsor: Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly 
Study: PROCLAIM (Phase 1/2)
Drug: PR006 – GRN AAV9 gene therapy, increases progranulin levels by delivering a healthy GRN gene into the brain
Delivery route: Intracisternal injection
Update: Previously received Orphan Drug Designation (US FDA and European Commission) and US Fast Track Designation, interim results for phase1/2 were published in 2024 and recruitment is ongoing.
Clinicaltrials.gov: NCT04408625

Sponsor: Vesper Biotechnology
Study: SORT-IN-2 (phase 1/2)
Drug: VES001 – small molecule, inhibits sortilin-mediated granulin degradation
Delivery route: Oral
Update: Phase 1 data in healthy volunteers was previously reported and Phase 1b/2a has initiated in asymptomatic patients with GRN-related FTD, which enrolled six people with expected results in the second half of 2026.
Clinicaltrials.gov: NCT06705192