ProJenX has deep roots in the ALS community. Named for Jenifer Estess, who founded the nonprofit Project ALS with her family and friends after receiving an ALS diagnosis at 35, the development of our ALS clinical candidate prosetin has been guided by the ALS community since day 1. ProJenX formed a Community Advisory Board at inception, which has allowed us to fully integrate ALS lived expertise into decision-making ranging from regulatory hurdles to early phase clinical trial design. At the 2024 Northeast ALS Consortium annual meeting, we are pleased to co-present on this experience with members of our CAB.
CAB members Gretchen Roy and Michele Stellato share their backgrounds and experience with the ProJenX CAB below.
Gretchen Roy, ALS caregiver:
Michele Stellato, living with ALS:
Click below for a downloadable version of the presentation:
Clinical Outcomes of ALSFRS-R, Slow Vital Capacity, and Plasma Neurofilament Light to be Evaluated in Placebo Digital Twins of Study Participants Dosed with Prosetin
NEW YORK, Sept. 26, 2024 /PRNewswire/ — ProJenX, Inc., a clinical-stage biotechnology company developing novel therapies targeting biologically defined pathways for the treatment of amyotrophic lateral sclerosis (ALS) and other debilitating brain diseases, today announced its partnership with Unlearn to augment PRO-101, its Phase 1 clinical trial of prosetin—a brain-penetrant, MAP4 kinase (MAP4K) inhibitor—with Unlearn’s advanced generative artificial intelligence (AI) technology.
Unlearn, a pioneering technology company innovating machine learning to revolutionize medical research, will implement its ALS-Digital Twin Generator (ALS-DTG) to produce digital twins of PRO-101 clinical trial participants living with ALS to evaluate the clinical outcomes of ALSFRS-R, Slow Vital Capacity, and plasma neurofilament light over a 52-week open-label extension. Digital twins will act as placebos of participants dosed with prosetin.
Unlearn’s ALS-DTG is an advanced machine-learning generative model trained on patient-level data to generate probabilistic forecasts of future health outcomes, known as digital twins, for individual clinical trial participants. At the start of a trial, baseline data are collected from all participants. The ALS-DTG uses these data to forecast longitudinal clinical placebo outcomes, biomarkers, and labs—regardless of the participant’s randomization assignment. Digital twins help address key challenges in clinical development and are delivered through Unlearn’s Platform.
“ProJenX and Unlearn’s collaboration aims to extract valuable insights that will help us expedite clinical trials and more efficiently evaluate prosetin in individuals living with ALS,” said Stan Abel, president and CEO at ProJenX. “Digital twins provide the opportunity for us to compare participants with ALS who are dosed with prosetin to their digitally generated placebo versions of themselves based on their baseline measurements. In PRO-101, this collaboration will enhance our interpretation of the effects of prosetin on ALS patients to support a more efficient and data-driven clinical development program.”
“With hundreds of thousands of people affected by ALS, the need to accelerate research that can transform treatment for this debilitating and paralyzing disease has never been more pressing,” said Steve Herne, CEO of Unlearn. “ProJenX is making significant strides in this area, and we are confident that our collaboration will continue to support their efforts to develop impactful therapies that improve the lives of patients everywhere.”
About PRO-101
PRO-101 is a hybrid phase 1 clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in healthy volunteers and patients with ALS. Parts 1a and 1b, which have been completed, consisted of a randomized, double-blind, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, and pharmacokinetics of single ascending and multiple doses of prosetin in healthy volunteers. Parts 1c and 1d will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in ALS patients. For more information about PRO-101, contact [email protected].
About ProJenX
ProJenX is a clinical-stage biotechnology company developing novel, brain-penetrant, targeted therapies to address untreatable brain 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 Unlearn
Unlearn is a pioneering AI company with a product-driven focus, dedicated to transforming medicine by eliminating trial and error. Our Platform harnesses the power of digital twins—advanced computational models of patients—to empower pharmaceutical and biotech companies to overcome critical challenges in clinical development.
For more information, please visit www.unlearn.ai or follow @UnlearnAI on Twitter/X and @unlearn-ai on LinkedIn.
ProJenX Announces Formation of Clinical Advisory BoardNEW YORK, NY, August 27, 2024—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 Clinical Advisory Board (ClAB). The ClAB, which includes internationally renowned experts in clinical trial strategy and design for ALS and related neurodegenerative diseases, will provide strategic guidance for the clinical development of ProJenX’s lead therapeutic candidate, prosetin—a novel, brain-penetrant, MAP4 kinase (MAP4K) inhibitor—and additional pipeline programs.
Prosetin is being evaluated in Study PRO-101, a hybrid Phase 1 clinical trial designed to evaluate its safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers and participants with ALS. Parts A and B of the study, which assessed single and multiple ascending doses of prosetin in healthy volunteers, are complete, and positive safety, tolerability, and pharmacokinetic data from 48 healthy volunteers supports continued exploration of prosetin in ALS participants.
The inaugural ClAB Chair is Angela Genge, MD, FRCP(C), eMBA (McGill University), who is joined by members Jinsy Andrews, MD, MSc (Columbia University), Leonard van den Berg, MD, PhD (UMC Utrecht), and Merit Cudkowicz, MD, MSc (Massachusetts General Hospital).
Dr. Genge, Director of the ALS Global Centre of Excellence at the Montreal Neurological Institute and Professor of Neurology at McGill University, said, “Improved clinical outcomes for ALS patients will only be achieved through stewarding novel, scientifically rational investigational therapies supported by compelling and thorough preclinical data into the ALS clinical trial pipeline. Prosetin has been developed specifically for ALS and demonstrates robust neuroprotection in diverse ALS patient-derived stem cell models of the disease, and I am excited to guide Study PRO-101 as Chair of the ProJenX Clinical Advisory Board, as well as Principal Investigator of one of the trial study sites.”
Erin Fleming, Co-Founder and Chief Operating Officer at ProJenX, said, “As we initiate the first-ever clinical study of prosetin in people living with ALS, we are thrilled to convene a world-class Clinical Advisory Board, which will bring unrivaled experience and knowledge across ALS drug development and data-driven clinical trial design to the prosetin development program. With our advisors’ leadership, ProJenX is poised to translate decades of scientific discovery to people with ALS.”
ProJenX’s Clinical Advisory Board members are:
Angela Genge, MD, FRCP(C)
Chair, ProJenX Clinical Advisory Board
McGill University
Dr. Angela Genge is the director of the Amyotrophic Lateral Sclerosis (ALS) clinic at The Neuro at Montreal Neurological Institute-Hospital and the executive director of The ALS Center of Excellence. She earned her MD at the Memorial University of Newfoundland, completed a fellowship in neuromuscular diseases at The Neuro, and obtained her Canadian and American certifications in internal medicine and neurology. Since her appointment as director of the CRU from 2004-2024, she established it as one of the largest neurological clinical research centers in Canada, home to a first-of-its-kind Phase 1 Unit dedicated to neurological diseases. Recognized as an international leader in clinical trial design and an expert in rare neurological conditions. Dr. Genge has led trials involving ALS, the dementias, myopathies, neuropathies, myasthenia gravis, and pain. She has extensive experience in drug development, including early phase, post-approval real world evidence (RWE), and regulatory and medical affairs. Dr. Genge has served as a consultant for biotechs in rare disease and on numerous advisory boards and data and safety monitoring boards. Her work and dedication have been acknowledged with multiple awards, including the 2023 Wings over Wall Street Award, 2018 Forbes Norris Award from the International Alliance for ALS/MND Associations and the Governor General Diamond Jubilee Award.
Jinsy Andrews, MD, MSc
Columbia University
Jinsy A. Andrews, MD, MSc, FAAN is an Associate Professor of Neurology, in the Division of Neuromuscular Medicine and serves as the Director of Neuromuscular Clinical Trials. She currently oversees neuromuscular clinical trials and cares for patients with neuromuscular disease, primarily with Amyotrophic Lateral Sclerosis (ALS). Dr. Andrews has extensive experience in all phases of human clinical trials and drug development in both the academic and industry settings. Dr. Andrews is the elected co-chair of the Northeastern ALS (NEALS) Consortium, which is a network of over 100 ALS clinical research centers internationally. She is also elected to the National Board of Trustees of the ALS Association and is a Fellow of the American Academy of Neurology (FAAN). Dr. Andrews has also received the Diamond Award for ALS clinical research from Wings Over Wall Street and the Muscular Dystrophy Association.
Dr. Andrews received her BS from Union College, M.Sc. in Biostatistics (Patient-Oriented Research) from Columbia University’s Mailman School of Public Health and M.D. from Albany Medical College. She completed her residency training in Neurology at the University of Connecticut and served as the Chief Neurology Resident in her final year. Dr. Andrews completed fellowship training in Neuromuscular Disease/ALS and Clinical Neurophysiology at Columbia University. She is board certified in Neurology, Neuromuscular Disease, and Electrodiagnostic Medicine.
Leonard van den Berg, MD, PhD
University Medical Center Utrecht
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.
Leonard H. van den Berg is also director of the center’s Laboratory for Neuromuscular Disease, director of the Netherlands ALS Center, and chairman of the European Network to Cure ALS (ENCALS), a network of the European ALS Centres.
After receiving his medical degree from the University of Groningen in the Netherlands, Prof. van den Berg completed a fellowship in neuroimmunology at the Neurological Institute at Columbia University in New York. He then completed his PhD degree at the University Medical Center Utrecht in a program that combined a residency in neurology with scientific research.
As a practicing neurologist specializing in neuromuscular diseases and neuroimmunology, Prof. van den Berg’s research pursuits lie in immune-mediated or degenerative diseases of motor neurons. A major emphasis of his research has been in ALS and other motor neuron disorders, and he has been a principle investigator on numerous drug trials concerning treatment options for the disease.
Merit Cudkowicz, MD
Massachusetts General Hospital
Dr. Merit Cudkowicz is the Chair of Neurology and Director or the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School in Boston. Dr. Cudkowicz is one of the founders and former co-directors of the Northeast ALS Consortium (NEALS), a group of over 150 clinical sites in the United States, Canada, Europe and the Middle East dedicated to performing collaborative academic-led clinical trials and research studies in ALS. She helped bring forward the FDA approved treatment Qalsody for people with SOD1 ALS. She is leading the first Platform Trial initiative in ALS and is also the Principal Investigator of the Clinical Coordination Center for the National Institute of Neurological Disorders and Stroke’s Neurology Network of Excellence in Clinical Trials (NeuroNEXT). Dr. Cudkowicz mentors neurologists at MGH and globally in careers in experimental therapeutics.
ProJenX Announces Receipt of $1M Hoffman ALS Clinical Trial Award from the ALS AssociationFunding will support the first study of prosetin in people living with ALS
NEW YORK, June 20, 2024 – 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 debilitating brain diseases, today announced the receipt of a $1M Hoffman ALS Clinical Trials Award from the ALS Association. The grant will help fund the development of prosetin, a novel, brain-penetrant, MAP4 kinase (MAP4K) inhibitor, in the Part 1c portion of the PRO-101 clinical trial of prosetin in participants living with ALS.
PRO-101 evaluates the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin. ProJenX has completed the Part 1a and Part 1b portions of PRO-101 in healthy volunteers. Part 1c of the PRO-101 clinical trial is expected to initiate enrollment in Q3 2024.
“We are honored that the ALS Association recognizes the promise of prosetin and has selected ProJenX for a Hoffman Clinical Trial Award,” said Erin Fleming, Co-Founder and Chief Operating Officer at ProJenX. “Their support enables the rapid initiation of the first clinical study of prosetin in individuals living with ALS, which is a significant milestone for prosetin, ProJenX, and the broader ALS community that has supported this program since inception.”
“The ALS Association’s Hoffman Clinical Trial Awards Program supports early-stage clinical trials of new treatments that hold promise for those living with ALS,” said Kuldip Dave, Senior Vice President of Research at the ALS Association. “By funding Part 1c of the PRO-101 trial, we are pleased to support the first dosing of prosetin in people living with ALS. By funding programs at this critical stage, we hope to accelerate the development of therapeutic candidates that can help make ALS a livable disease.”
“Decades of collaborative preclinical research funded by Project ALS at Columbia University pinpointed core mechanisms of ALS and resulted in the development of prosetin to counter ALS pathology across multiple forms of the disease,” said Jinsy Andrews, MD, MSc, clinical advisor to ProJenX and Director of Neuromuscular Clinical Trials at the Columbia University Irving Medical Center. “I am excited to work with ProJenX to initiate Part 1c of the PRO-101 clinical trial, which will evaluate safety across multiple doses of prosetin and also explore biomarkers indicative of prosetin’s potential impact on ALS disease progression.”
About PRO-101
PRO-101 is a three-part phase 1 clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in healthy volunteers and patients with ALS. Parts 1a and 1b, which have been completed, consisted of a randomized, double-blind, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, and pharmacokinetics of single ascending and multiple doses of prosetin in healthy volunteers. Based on favorable safety, tolerability, and pharmacokinetic data from this portion of the study, Part 1c will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in ALS patients. Following the Part 1c, a long-term Open Label Extension (OLE) of 12 or more months will occur, allowing observation of the long-term behavior of prosetin in ALS patients, measured via biomarkers of disease progression, neurodegeneration, and neuroinflammation in plasma. For more information about PRO-101, contact [email protected].
About the ALS Association
The ALS Association is the largest philanthropic funder of ALS research in the world. The Association funds global research collaborations, assists people with ALS and their families through its nationwide network of care and certified clinical care centers, and advocates for better public policies for people with ALS. The ALS Association is working to make ALS a livable disease while urgently searching for new treatments and a cure. For more information about The ALS Association, visit our website at als.org.
About ProJenX
ProJenX is a clinical-stage biotechnology company developing novel, brain-penetrant, targeted therapies to address untreatable brain 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.
Media Contact:
David Schull
Russo Partners
(858) 717-2310
[email protected]
NEW YORK, NY, March 28, 2024 – ProJenX, a clinical-stage biotechnology company developing novel, brain-penetrant therapies targeting biologically-defined pathways for the treatment of amyotrophic lateral sclerosis (ALS) and other debilitating brain diseases, today announced that the United States Food and Drug Administration (FDA) has removed a partial clinical hold on Study PRO-101, a hybrid Phase 1 clinical trial evaluating prosetin—a first-in-class MAP4 kinase (MAP4K) inhibitor—in healthy volunteers and participants with ALS. The healthy volunteer portions of the trial have been completed, and the company is initiating Part 1c of PRO-101, designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in participants with ALS.
Stan Abel, President and Chief Executive Officer at ProJenX, commented, “Recent clinical trial results in ALS have further demonstrated the urgent need for meaningful therapies that can alter the course of this devastating disease. Our Columbia University and Project ALS co-founders have invested over two decades in scientific research that shows a critical role for MAP4K inhibition on ALS motor neuron survival, and we are thrilled to be advancing prosetin to people living with ALS in the coming months.”
“Following recent Clinical Trial Application authorizations in Canada and Europe, we are very pleased that FDA has lifted this partial clinical hold, which previously limited prosetin dose levels in Study PRO-101 in the United States,” added Erin Fleming, Co-Founder and Chief Operating Officer at ProJenX. “The FDA’s decision allows us to fully pursue a global strategy to bring this promising investigational treatment to people with ALS and other neurodegenerative diseases.”
Prosetin is a selective, oral, brain-penetrant, MAP4K inhibitor developed by ProJenX co- founders at Columbia University for the treatment of ALS. Following the discovery that MAP4K inhibition confers potent motor neuron protection across multiple patient stem cell-derived models of ALS, prosetin was optimized for potency against MAP4Ks, efficacy in motor neuron rescue, and preferential distribution to the CNS.
“There is an immense unmet need for safe and effective treatment options for ALS, and prosetin is an exciting investigational therapy option in the current clinical trial landscape,” said Jinsy Andrews, MD, MSc, Associate Professor of Neurology and Director of Neuromuscular Clinical Trials at Columbia University. “I am encouraged by prosetin’s compelling preclinical efficacy data and by its consistent safety and tolerability profile in healthy volunteers and chronic toxicology studies, and look forward to working with the ProJenX team to bring Study PRO-101 to people living with ALS in the United States.”
About PRO-101
PRO-101 is a three-part Phase 1 clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in healthy volunteers and patients with ALS. Parts 1a and 1b, which have been completed, consisted of a randomized, double-blind, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, and pharmacokinetics of single ascending and multiple doses of prosetin in healthy volunteers. Based on favorable safety, tolerability, and pharmacokinetic data from this portion of the study, Part 1c will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in ALS patients. For more information about PRO-101, contact [email protected].
About ProJenX
ProJenX is a clinical-stage biotechnology company developing novel, brain-penetrant, targeted therapies to address untreatable brain 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.
Media Contact:
Margot Shanahan
(917) 423-6476
[email protected]
NEW YORK, NY, February 14, 2024 – ProJenX, a clinical-stage biotechnology company developing novel, brain-penetrant therapies targeting biologically-defined pathways for the treatment of amyotrophic lateral sclerosis (ALS) and other debilitating brain diseases, today announced that it has received authorization in the European Union (EU) for study PRO-101, a hybrid Phase 1 clinical trial evaluating prosetin—a first-in-class MAP4K inhibitor—in healthy volunteers and participants with ALS. Following recent Health Canada authorization of this study, the European authorization will expand the global footprint of Part 1c of PRO-101, designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in participants with ALS.
“This EU clinical trial authorization will allow us to expand the first study of prosetin for people living with ALS to European participants,” said Erin Fleming, Co-Founder and Chief Operating Officer at ProJenX. “We are committed to initiating ALS patient enrollment as quickly as possible.”
Prosetin is a selective, oral, brain-penetrant, MAP4K inhibitor developed by ProJenX co-founders at Columbia University for the treatment of ALS. Following the discovery that MAP4K inhibition confers potent motor neuron protection across multiple patient stem cell-derived models of ALS, prosetin was optimized for potency against MAP4Ks, efficacy in motor neuron rescue, and preferential distribution to the CNS.
“ALS is a devastating disease, and there is an immense unmet need for therapeutic options to slow or halt its rapid progression,” said Leonard H. van den Berg, MD, PhD, Professor of Neurology at UMC Utrecht, Chairman of the Treatment Research Initiative to Cure ALS (TRICALS), and PRO-101 study investigator. “MAP4K inhibition has been shown to protect motor neurons from endoplasmic reticulum stress and other well-established pathological mechanisms of ALS, and we are excited to partner with ProJenX on study PRO-101, the first clinical trial to explore the potential benefits of MAP4K inhibition for ALS patients.”
About PRO-101
PRO-101 is a three-part Phase 1 clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in healthy volunteers and patients with ALS. Parts 1a and 1b, which have been completed, consisted of a randomized, double-blind, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, and pharmacokinetics of single ascending and multiple doses of prosetin in healthy volunteers. Based on favorable safety, tolerability, and pharmacokinetic data from this portion of the study, Part 1c will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in ALS patients. For more information about PRO-101, contact [email protected].
About ProJenX
ProJenX is a clinical-stage biotechnology company developing novel, brain-penetrant, targeted therapies to address untreatable brain 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.
Media Contact:
Margot Shanahan
(917) 423-6476
[email protected]
NEW YORK, NY, November 29, 2023 – ProJenX, a clinical-stage biotechnology company developing novel, brain-penetrant therapies targeting biologically-defined pathways for the treatment of amyotrophic lateral sclerosis (ALS) and other debilitating brain diseases, today announced authorization of its clinical trial application (CTA) by Health Canada for study PRO-101, a global, hybrid Phase 1 clinical trial evaluating prosetin—a first-in-class MAP4K inhibitor—in healthy volunteers and participants with ALS. The Health Canada CTA authorization supports initiation of Part 1c of PRO-101, designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in participants with ALS.
“Today’s announcement is an important milestone for the prosetin program and our ProJenX community,” said Erin Fleming, Co-Founder and Chief Operating Officer at ProJenX. “Following Health Canada’s CTA authorization, we will be able for the first time to evaluate prosetin in people living with ALS. We are encouraged by the safety and tolerability profile that has emerged from completed healthy volunteer studies of prosetin, and we are laser-focused on initiating ALS patient enrollment in Canada as quickly as possible.”
Prosetin is a selective, oral, brain-penetrant, MAP4K inhibitor developed by ProJenX co-founders at Columbia University for the treatment of ALS. Following the discovery that MAP4K inhibition confers potent motor neuron protection across multiple patient stem cell-derived models of ALS, prosetin was optimized for potency against MAP4Ks, efficacy in motor neuron rescue, and preferential distribution to the CNS.
“Despite scientific advances, ALS remains a fatal, devastating neurodegenerative disease that demands more meaningful treatment options,” said Angela Genge, MD, FRCP(C), eMBA, Associate Professor, Department of Neurology and Neurosurgery at McGill University, Director of the ALS Centre of Excellence for Research and Patient Care at the Montreal Neurological Institute (The Neuro), and PRO-101 study investigator. “ProJenX has generated compelling preclinical data showing that prosetin may protect motor neurons against well-established pathological mechanisms in ALS, and I look forward to working with their team on the first-ever study evaluating its activity in ALS patients.”
About PRO-101
PRO-101 is a three-part Phase 1 clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in healthy volunteers and patients with ALS. Parts 1a and 1b, which have been completed, consisted of a randomized, double-blind, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, and pharmacokinetics of single ascending and multiple doses of prosetin in healthy volunteers. Based on favorable safety, tolerability, and pharmacokinetic data from this portion of the study, Part 1c will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in ALS patients. For more information about PRO-101, contact [email protected].
About ProJenX
ProJenX is a clinical-stage biotechnology company developing novel, brain-penetrant, targeted therapies to address untreatable brain 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.
Media Contact:
Margot Shanahan
(917) 423-6476
[email protected]
NEW YORK, Nov 2, 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 debilitating brain diseases, today announced the initial closing of a $15 million Series A financing. Led by Medical Excellence Capital, which also led the company’s seed financing round and created ProJenX in collaboration with Project ALS and researchers at Columbia University, the Series A will fund the continued development of ProJenX’s lead compound, prosetin, for the treatment of ALS.
In addition, ProJenX announced the appointment of Rick Hartz to its board of directors. Rick is currently Senior Vice President, Global Pharma & Human Health Business Development at Merck & Co., Inc. Rick has more than 30 years of commercial, marketing, and business development leadership experience across multiple therapeutic areas. At Merck, he leads a global commercial team responsible for cardiovascular, respiratory, metabolic, infectious disease, hospital/specialty, immunology, and neuroscience. Previous positions at Merck include SVP for Global Oncology Partnerships, SVP US Managed Markets and Chief Marketing Officer, US Market. He earned a bachelor’s degree in economics from Dickinson College and an MBA from The Wharton School of the University of Pennsylvania.
“We are focused on accelerating the development of prosetin to meet the critical need for new treatments for people living with ALS,” said Stan Abel, the company’s president and CEO. “We are encouraged by the therapeutic window emerging from our phase 1 trial and ongoing nonclinical studies and are looking forward to advancing prosetin to people living with ALS in early 2024. We are also thrilled to welcome Rick to our board and know that he will add tremendous value given his extensive global commercial and business development experience at Merck.”
“I am excited to join the ProJenX Board and contribute to its mission to advance promising therapies for ALS and other neurodegenerative diseases,” added Mr. Hartz. “With an orphan drug designation, prosetin is poised to advance toward a global pivotal trial next year and I look forward to working with my fellow board members and the ProJenX team to help guide the growth of the company.”
Prosetin is a first-in-class, novel, selective, oral, brain-penetrant, mitogen-activated protein kinase kinase kinase kinase (MAP4K) inhibitor. MAP4Ks were discovered as critical regulators of neuronal survival in a patient-specific, cell-based discovery platform developed by ProJenX co-founders at Columbia University, and prosetin was optimized for potency against MAP4Ks, efficacy in motor neuron rescue, and preferential distribution to the CNS. Prosetin is an investigational new drug and has not been approved by the FDA.
About ProJenX
ProJenX is a clinical-stage biotechnology company developing novel, brain-penetrant, targeted therapies to address untreatable brain 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.
Media Contact:
David Schull
Russo Partners
(858) 717-2310
[email protected]
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.