AML treatment ICT01 granted FDA’s orphan drug status
Developer calls designation 'a catalyst' for immune-modulating therapy
ICT01, an immune-modulating therapy being developed by Imcheck Therapeutics for acute myeloid leukemia (AML), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA) as a potential treatment for this aggressive type of blood cancer.
The FDA awards this designation to investigational treatments for diseases that affect fewer than 200,000 Americans. Its goal is to offer extra incentives for companies investing in treatments for rare diseases. Such status confers perks that include tax breaks, fee waivers, and a guarantee of seven years of market exclusivity if the therapy is ultimately approved.
“Orphan drug designation is a catalyst,” Pierre d’Epenoux, CEO of Imcheck, said in a company press release. “It validates our regulatory strategy, de-risks and supports clinical development acceleration, and sends a strong signal about the unique potential of ICT01 to transform AML treatment as well as other solid tumor indications.”
Stephan Braun, MD, PhD, chief medical officer of Imcheck, said that winning orphan drug designation “is a significant recognition of ICT01’s innovative therapeutic potential to meet the urgent unmet medical needs of AML patients.”
An antibody-based therapy, ICT01 is designed to boost the activity of a specialized subtype of immune cells called gamma9delta2 T-cells. These cells normally act to patrol the body, looking for cells that have essentially gone rogue — such as those cells that are infected with viruses or cells that have become cancerous. By increasing the activity of these cells, ICT01 aims to activate the immune system to more effectively identify and eliminate cancer cells.
Phase 1/2 trial now testing ICT01 as treatment for AML, other cancers
A Phase 1/2 clinical trial dubbed EVICTION (NCT04243499) is currently testing ICT01 in AML and many other types of cancer. Earlier this year, Imcheck presented new data from AML patients in that trial who were treated with ICT01 in combination with the chemotherapy drugs azacitidine and venetoclax (Aza-Ven). The study specifically enrolled newly diagnosed AML patients who were not eligible for intensive chemotherapy.
Those results showed that, of 39 evaluable patients, 74% had complete remission — meaning they had no detectable cancer cells and their blood cell counts had returned to normal. Most of the patients who weren’t in complete remission had no detectable cancer cells, but still had abnormal blood cell counts.
This important regulatory milestone reinforces our confidence that ICT01 will become the first immunotherapy for AML patients and supports our goal of rapidly advancing ICT01 into pivotal studies based on the unprecedented results observed in the clinic to date.
Data also indicated that ICT01 was activating the immune system as intended, and safety findings were generally consistent with the known safety profile of the Aza-Ven chemotherapy regimen.
“This important regulatory milestone reinforces our confidence that ICT01 will become the first immunotherapy for AML patients and supports our goal of rapidly advancing ICT01 into pivotal studies based on the unprecedented results observed in the clinic to date,” Braun said.
The EVICTION trial is slated for completion this year.
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