In August, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to Alector for the continued development of AL001, a human recombinant monoclonal antibody designed for the treatment of FTD caused by mutations in the progranulin gene.

In order to better understand FTD-related challenges, the California-based biotechnology company reached out to AFTD during the development phase of AL001, which is “an immuno-neurology drug designed to repair the immune system to elicit a therapeutic benefit,” according to Arnon Rosenthal, Ph.D., Alector’s chief executive officer.

The FDA grants orphan drug designation to therapies designed to aid in the diagnosis, prevention and/or treatment of rare diseases, which they define as those affecting fewer than 200,000 people in the U.S. Receiving an orphan drug designation qualifies the company for a range of incentives intended to support continued development of the drug, including tax credits and exemption from regulatory fees. AL001 is one of a number of drugs currently vying for approval to be the first FDA-approved therapeutic option for persons diagnosed with FTD.

“The main focus [of AL001] is people with progranulin gene mutations — a rare subgroup of people with FTD,” said Nadine Tatton, Ph.D., AFTD’s scientific director. “The level of progranulin in the blood of a mutation carrier is about half the level you would see in a healthy individual. Alector has developed a therapy that can increase the blood levels of progranulin, an antibody that binds to its target and converts the person to producing normal progranulin levels.”

Earlier this year, Dr. Tatton visited Alector’s headquarters in San Francisco, along with AFTD program director Sharon S. Denny, M.A. They were joined by research scientists working on AL001, persons affected by FTD and their care partners, and former AFTD Board Chair Jary Larsen, Ph.D.

“Alector is in the process of moving its therapies from basic science and drug development into the clinical setting,” said Denny. “They reached out to us because they want to learn more about the impact of the disease as it plays out in peoples’ lives.”

Alector has initiated a phase 1 clinical trial of AL001 to test the safety and tolerability of the therapy. The trial currently seeks volunteers with a progranulin gene mutation who have been diagnosed with FTD, as well as progranulin gene mutation carriers who are asymptomatic and healthy volunteers. More information about the clinical trial can be found on the Featured Studies section of AFTD’s website.

“Our meeting with Alector reflects increasing awareness that the person diagnosed is an important part of the clinical trial landscape,” Denny said. “What a person diagnosed considers an important outcome can be very different from the opinions of a researcher. AFTD can act as a bridge between the FTD community and the scientific world.”