NEW YORK, March 30, 2023—ProJenX, Inc., a clinical-stage biotechnology company developing novel, brain-penetrant therapies targeting biologically-defined pathways for the treatment of amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, today announced the formation of its Scientific Advisory Board (SAB). The SAB, which includes internationally renowned experts in stem cell biology, ALS disease modeling and drug discovery, and clinical development across neurodegeneration, will provide strategic guidance for the development of ProJenX’s lead candidate prosetin—a brain-penetrant, orally available, MAP4K inhibitor—and additional pipeline expansion activities.

The inaugural members are Leonard van den Berg, MD, PhD (UMC Utrecht), Claire Henchcliffe, MD, DPhil (UC Irvine), Joe Lewcock, PhD (Denali Therapeutics), Lee Rubin, PhD (Harvard University), and Neil Shneider, MD, PhD (Columbia University).

Dr. Rubin, Professor of Stem Cell and Regenerative Biology at Harvard University and Co-Director of the Neuroscience Program at the Harvard Stem Cell Institute, said, “My own laboratory’s longstanding interest in patient-derived models of ALS and in the identification of neuroprotective compounds pointed us, much like ProJenX, to MAP4Ks as key regulators of motor neuron degeneration. I am excited by the research behind prosetin and look forward to working with ProJenX to elucidate and intervene in the key cellular pathways involved in ALS and neurodegeneration.”

“To meaningfully alter the course of disease in ALS, we must advance better-validated therapeutic candidates,” said Dr. Shneider, Director of the Eleanor and Lou Gehrig ALS Center and Claire Tow Associate Professor of Neurology at Columbia University Irving Medical Center. “The long-term collaboration between Columbia University researchers and Project ALS that led to prosetin is an example of the rational, scientifically rigorous approach required for clinical success, and I am eager to work with the ProJenX team to move prosetin forward in ALS.”

Erin Fleming, Co-Founder and Vice President of Research & Development at ProJenX, said, “We are honored to convene a world-class Scientific Advisory Board, who represent peerless experience and knowledge across ALS drug development, data-driven clinical trial design, and the MAP kinase pathway in neurodegeneration. With our advisors’ leadership, ProJenX is poised to translate decades of scientific discovery to people with ALS—beginning with prosetin.”

Biographies of ProJenX SAB members can be viewed below.

About ProJenX

ProJenX is a clinical-stage biotechnology company developing novel, brain-penetrant, targeted therapies to address neurodegenerative diseases, with an initial focus on ALS. ProJenX was created out of a long-term research collaboration between Project ALS and researchers at Columbia University to rapidly develop and commercialize its lead therapy candidate, prosetin, for people living with ALS. At the heart of ProJenX’s approach is an innovative, patient-specific, cell-based drug discovery platform that can be leveraged for research and drug development for ALS and other debilitating brain diseases. For more information, visit projenx.com.

About Prosetin

Prosetin is a potent, oral, brain-penetrant, mitogen-activated protein kinase (MAP4K) inhibitor targeting endoplasmic reticulum (ER) stress. ER stress is a common feature across sporadic and familial forms of ALS, and MAP4Ks emerged as the critical regulators of ER stress-mediated motor neuron loss in a patient-specific, cell-based discovery platform developed by researchers at Columbia University. ProJenX is currently conducting a three-part Phase 1 clinical trial, PRO-101, investigating prosetin in healthy individuals and people living with ALS. Prosetin is an investigational new drug and has not been approved by the FDA.

Media Contact:

Madeline Stabinski or David Schull
Russo Partners
(858) 717-2310
[email protected]
[email protected]

 

ProJenX Scientific Advisory Board Biographies

Leonard van den Berg, MD, PhD

Leonard H. van den Berg is a professor of neurology who holds a chair in experimental neurology of motor neuron diseases at the University Medical Center Utrecht in the Netherlands. He also is director of the center’s Laboratory for Neuromuscular Disease, founder and director of the Netherlands ALS (amyotrophic lateral sclerosis) Center, chairman of the Neuromuscular Centre the Netherlands, and chairman of the European Network to Cure ALS (ENCALS), a network of the European ALS Centres.

Prof. Leonard van den Berg did a fellowship at the Neurological Institute at Columbia University in New York and obtained his PhD degree in 1995 in Utrecht. He has been professor of Experimental Neurology since 2005 and leads a research group focused on translational research into ALS and other diseases of motor neurons. His research has been focused on the search for effective treatment for patients with motor neuron diseases and motor neuropathies by delineating the biological and molecular pathways that initiate and/or drive motor neuron degeneration.

Claire Henchcliffe, MD, DPhil

Claire Henchcliffe is the Chair and Stanley van den Noort Professor of Neurology, University of California, Irvine since 2020. Prior to this, she spent 17 years at Weill Cornell Medical College (WCMC)/New York Presbyterian Hospital, New York City, becoming Professor of Neurology and Neuroscience, and Vice Chair for Clinical Research in Neurology. Her undergraduate and graduate training was completed at the University of Oxford, University of Cambridge, UK, and the University of California at Berkeley, followed by neurology residency training and movement disorder fellowship at the College of Physicians and Surgeons of Columbia University in New York City. Her clinical and research focus is on Parkinson’s disease and related neurodegenerative disorders, including working to develop new Parkinson’s disease therapeutics such as stem cell-based and gene therapy interventions.

Joe Lewcock, PhD

Joe Lewcock serves as CSO and Head of Discovery at Denali Therapeutics, a biotechnology company focused on novel therapeutic strategies for neurodegenerative diseases including Alzheimer’s, Parkinson’s, ALS, and genetically defined rare disorders. In this role, he serves as a member of the Denali Management team and coordinates the preclinical stage portfolio, which has resulted in the progression of 8+ molecules ranging from small molecules to biotherapeutics advancing to clinical trials. His group defines therapeutic strategies based on genetically defined pathways that contribute to neurodegeneration including Lysosomal Function, Glial Biology, and Cellular Homeostasis. As delivery to the brain represents a major challenge, Joe’s discovery team has developed Denali’s proprietary Transport Vehicle (TV) platform to shuttle biologics across the Blood-Brain Barrier. This highly modular technology enables effective brain delivery and broad biodistribution of a range of cargos including enzymes, antibodies, and oligonucleotides.

Prior to joining Denali in early 2016, Joe spent 9 years at Genentech, where he helped to build the neuroscience research team and portfolio. In his role as Director of the Department of Neuroscience, he was responsible for generating the disease area strategy and management of the neuroscience portfolio, which included both large and small molecule programs. During his time at Genentech, Joe also served as a project team leader where he led a program for treatment of ALS from target discovery to IND filing and led a discovery lab focused on identification of new therapeutic drug targets for ALS, Alzheimer’s Disease, and other neurodegenerative diseases. He received his BS from University of California, San Diego, a Ph.D. from Johns Hopkins University School of Medicine, and did a Postdoctoral fellowship at the Salk Institute.

Lee Rubin, PhD

Lee Rubin is Professor of Stem Cell and Regenerative Biology at Harvard University and Co-Director of the Neuroscience Program at the Harvard Stem Cell Institute. His work focuses on neurodegenerative and neuromuscular disorders and has a strong translational focus. Currently, his efforts are focused on producing muscle stem cells to treat muscular and neuromuscular disorders and, importantly, on discovering therapeutics capable of reversing the degenerative changes associated with brain aging.

Dr. Rubin received his PhD in Neuroscience from the Rockefeller University and had postdoctoral training, also in Neuroscience, at Harvard Medical School and Stanford University School of Medicine.

Neil Shneider, MD, PhD

Neil Shneider serves as the Claire Tow Associate Professor of Motor Neuron Disorders and the Director of the Eleanor and Lou Gehrig ALS Center at Columbia University. He is an investigator in the Center for Motor Neuron Biology and Disease where his lab focuses on the study of models and mechanisms of ALS and the discovery and development of novel therapeutics for ALS and related disorders. Dr. Shneider worked with Ionis Pharmaceuticals to develop ION363 (Jacifusen), an anti-sense oligonucleotide (ASO) for ALS patients with rare mutations in the FUsed in Sarcoma (FUS) gene. Dr. Shneider is a graduate of Harvard College and earned his M.D. and Ph.D. degrees at the Columbia University College of Physicians and Surgeons.

In partnership with n-Lorem Foundation and Columbia University, Dr. Shneider founded Silence ALS, an initiative to develop ASOs for ALS patients with nano-rare, pathogenic mutations in ALS genes. Dr. Shneider was co-chair of the Translating Fundamental Research into Potential ALS Therapies Working Group for the NIH ALS Strategic Planning Workshop.