The trial expects to enroll its first patient in the first half of 2023.
The FDA has cleared Cellevolve Bio’s investigational new drug application (IND) and granted orphan drug designation to CE-VST01-JC, a T cell therapy targeting human polyomavirus 2, commonly known as the John Cunningham or JC virus, for treating progressive multifocal leukoencephalopathy (PML).1
"We are thrilled to have received IND clearance to begin our phase 2 clinical study," Derrell Porter, MD, founder and chief executive officer, Cellevolve, said in a statement.1 "This is a significant milestone for both Cellevolve and the PML community: ASCEND-JC will be the largest ever cell therapy study conducted in PML, and this is Cellevolve's first IND approval and a powerful proof point for our business model of partnering with innovators around the world to bring cell therapies through late stage development and into commercialization."
CE-VST01-JC will be evaluated in the randomized, double-blind phase 2 ASCEND-JC study (NCT05541549) initiating soon. The study has a target enrollment of 60 participants across the US, Canada, and the EU.Cellevolve plans to enroll the first participant in the first half of 2023. Participants will receive 1 × 108 CE-VST01-JC cells or placebo via intravenous infusion. The study is primarily evaluating time to disease progression, as measured by modified Rankin Score.
PML is a demyelinating neurological disease caused by JC virus in immunocompromised patients, in which it is usually progressive and often fatal.An estimated 4,000 throughout the US and Europe develop PML annually. There are no approved treatment options for PML. CE-CST01-JC is an allogeneic JC-specific T-cell therapy that targets JC virus to treat PML.
Cellevolve has collaborated with multiple organizations to aid development of CE-VST01-JC.2 The company announced a collaboration with Australia-based QIMR Berghofer Medical Research Institute and its cell therapy manufacturing facility Q-Gen Cell Therapeutics in December 2021. QIMR agreed to manufacture a clinical supply for early-stage development, and Professor Rajiv Khanna, PhD, agreed to research JC and BK virus specific T-cells and allogeneic cell therapies.
“Rapidly progressing illnesses such as PML require safe, efficacious and speedy treatments since the longer patients wait, the more debilitating the outcome thus promptly halting disease progression is critical,” Porter said in a statement at that time.2 “We are honored to partner with leading VST researcher Professor Khanna and his team at QIMR Berghofer to advance a novel, bioengineered, JCV-specific off-the-shelf T-cell therapy to the clinic in this first randomized cell therapy clinical trial.”
At the same time, Cellevolve also announced a partnership with Be the Match for cellular products as well as with Cryoport Systems for packaging, storage, labelling, distribution and logistics support for clinical trials in the US and EU.
“There is a huge unmet medical need for a novel, FDA-approved, PML treatment based on the gravity of this disease and the overall patient burden,” David B. Clifford, MD, Melba and Forest Seay Professor of Clinical Neuropharmacology in Neurology, Washington University School of Medicine, added to the statement.2 “Additionally, off-the-shelf cell therapies by design will enable faster delivery and therapeutic access, which is integral to addressing this progressive disease for patients all over the world.”