Cellevolve Bio Submits Investigational New Drug (IND) Application for Global Phase 2, Placebo-controlled Study of CE-VST01-JC for the Treatment of Progressive Multifocal Leukoencephalopathy (PML)

• CE-VST-01-JC is a novel, allogeneic, off-the-shelf, T cell therapy that targets the JC Virus, the virus that causes progressive multifocal leukoencephalopathy (PML)
• ASCEND-JC is a randomized, double-blind, placebo-controlled, phase 2 study with sites planned across US, Europe, and Canada
• Licensed and developed in collaboration with QIMR Berghofer Medical Research Institute's Professor Rajiv Khanna; NIH's Irene Cortese, M.D. will be the Lead Primary Investigator for the study

SAN FRANCISCO, Sept. 20, 2022 /PRNewswire/ -- Cellevolve Bio, Inc. ("the Company" or "Cellevolve"), a development and commercialization platform company focused on cell therapies, today announced the submission of an investigational new drug (IND) application to the US Food and Drug Administration (FDA) for CE-VST01-JC, it's most advanced clinical stage program. This experimental therapeutic is a JC Virus Specific T Cell Therapy (JC-VST) that has been engineered to specifically target the JC polyoma virus (JCV) and it's immunogenic antigens. The IND supports a proposed indication for the treatment of progressive multifocal leukoencephalopathy (PML) secondary to JCV.

ASCEND-JC is a global, randomized, double-blind, placebo-controlled, phase 2 study that will enroll up to 60 patients with PML. This study will evaluate the ability of CE-VST01-JC to slow or halt the progression of PML as measured by modified Rankin Score (mRS). The first patient to enroll is anticipated in the first half of 2023.

"This significant regulatory milestone is a very important step in our journey to bring an effective treatment to PML patients and their families, who are in dire need of new treatment options. I am excited about the promise and potential of CE-VST01-JC and proud of my Cellevolve colleagues and collaborators who have worked hard to get us to this point. " said Derrell Porter, M.D., Founder and CEO, Cellevolve. "I am honored to work hand-in-hand with Rajiv Khanna, Ph.D. and Irene Cortese, M.D., to start ASCEND-JC, our first clinical trial."

PML is a demyelinating disease of the central nervous system (CNS) caused by JCV and occurs in immunocompromised patients.  JCV is a common virus, found in up to 85% of the general adult population.¹ Several diseases, conditions, and therapeutics weaken the immune system and increase the risk of developing PML. Risk factors include hematological malignancies, HIV, autoimmune diseases (e.g. multiple sclerosis, sarcoidosis, primary immune deficiencies), bone marrow and solid organ transplants, as well as their treatments.² Once developed, 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

Cellevolve and the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH), are collaborating on ASCEND-JC. Dr. Irene Cortese will serve as our lead primary investigator and NIH will serve as a hub site for the execution of this clinical trial and will partner on further investigations on laboratory specimens. "Today is an important first step toward identifying treatments that can hopefully become widely accessible to PML patients, as we prepare to initiate the first large scale cell therapy clinical trial to treat PML," said Dr. Cortese.

This IND submission represents an important first step in the execution of ASCEND-JC. Importantly, this is one of many future milestones in our mission to accelerate the transformation of innovations into treatments for neglected diseases. Additional progress for CE-VST01-JC, ASCEND-JC and Cellevolve will be the subject of future announcements.

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, randomized, double-blind, placebo-controlled, phase 2 trial in patients with PML caused 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 platform company focused on cell therapies. The Company's mission is to accelerate the revolution in cell therapy by translating innovation into commercially available treatments for neglected diseases, including pediatric and rare diseases. Founded by leaders with extensive experience developing and launching therapeutics, Cellevolve understands that cell therapy innovations require unique and specialized capabilities to advance them to approval and beyond. Many of these potential therapies can have a major impact on neglected diseases if they are advanced into clinical development and commercialization. The Company is addressing this need with it's partnering approach and integrated Cellution™ clinical development and commercialization platform that translates breakthrough science from the world's leading innovators into powerful new therapies for patients.

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

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SOURCE Cellevolve