Cellevolve Bio Receives FDA Clearance of IND Application and Orphan Drug Designation for CE-VST01-JC for the Treatment of Progressive Multifocal Leukoencephalopathy (PML)

• CE-VST01-JC is an allogeneic, off-the-shelf, T cell therapy targeting the JC virus, the cause of PML, a rare, rapidly progressing, often fatal illness for which there are no approved treatment options.
• ASCEND-JC is a Phase 2, placebo-controlled study designed to assess the safety and efficacy of CE-VST-01-JC. First patient enrolled anticipated in the first half of 2023.

SAN FRANCISCO, Oct. 25, 2022 /PRNewswire/ -- Cellevolve Bio, Inc. ("the Company" or "Cellevolve"), a clinical stage development and commercialization platform company focused on cell therapies, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for CE-VST01-JC, a JC Virus Specific T Cell Therapy (JC-VST) that is being studied for the treatment of progressive multifocal leukoencephalopathy (PML). CE-VST01-JC also received FDA orphan drug designation.

"We are thrilled to have received IND clearance to begin our Phase 2 clinical study," said Derrell Porter, M.D., Founder and CEO, Cellevolve. "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."

PML is a demyelinating disease of the central nervous system (CNS) caused by JCV and occurs in immunocompromised patients. PML is rapidly progressive, debilitating, and often fatal. There are no FDA or EMA approved treatments for this condition, which affects ~ 4,000 individuals in the US and Europe annually. ^1,2

The safety and efficacy of CE-VST01-JC will be evaluated in ASCEND-JC, a global, multi-center, randomized, double-blind, placebo-controlled Phase 2 study that will enroll up to 60 patients across the US, Canada, and EU. The first patient enrolled is anticipated in the first half of 2023.

1. NORD (2021). Progressive Multifocal Leukoencephalopathy. Retrieved from https://rarediseases.org/rare-diseases/progressive-multifocal-leukoencephalopathy/. Accessed on September 10, 2022.
2. Cortese I, Reich DS, Nath A. Progressive multifocal leukoencephalopathy and the spectrum of JC virus- related disease. Nat Rev Neurol. 2021a;17(1):37-51. Doi:10.1038/s41582-020-00427- y.

Cellevolve's lead product, CE-VST01-JC, is in clinical development as an allogeneic, off-the-shelf, JC-specific T-cell therapy (JC-VST) targeting JC polyoma virus (JCV) in patients that develop progressive multifocal leukoencephalopathy. CE-VST01-JC will be evaluated in ASCEND-JC, a global, multi-center, randomized, double-blind, placebo-controlled, Phase 2 trial in patients with PML due to JCV. The study is designed to evaluate whether CE-VST01-JC will slow and ultimately halt neurological progression in patients with PML compared with placebo as evaluated by a modified Rankin Score (mRS).

Cellevolve Bio is a development and commercialization company focused on cell therapies. The Company's mission is to partner with innovators to translate early stage clinical therapies into commercially available treatments for neglected diseases, including pediatric and rare diseases, which can have a major impact if they are advanced into clinical development and beyond. Founded by leaders with extensive experience developing and launching therapeutics, Cellevolve understands that the development and commercialization of cell therapies requires unique and specialized capabilities. The Company is addressing this need with it's partnering, development approach and integrated Cellution™ platform that translates breakthrough science into powerful new therapies for patients.

For more information, please visit www.cellevolve.com.

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SOURCE Cellevolve