CHICAGO, Dec. 12, 2023 (GLOBE NEWSWIRE) — Vanqua Bio today announced that its development candidate VQ-101, a best-in-class brain-penetrant small molecule allosteric activator of glucocerebrosidase (GCase), will enter clinical development in the first quarter of 2024, with an initial indication in Parkinson’s disease (PD). To support the company’s clinical evolution, Vanqua Bio also announced the appointment of three executives to leadership roles: Jesse Cedarbaum, MD and Omer Siddiqui have joined the company as Chief Medical Officer and Chief Development Officer, respectively, and Rand Sutherland, MD, MPH has joined the Board of Directors.
“The advancement of VQ-101 and expansion of the leadership team significantly furthers Vanqua’s mission of helping to usher in a new era of hope for patients living with neurodegenerative disorders,” said Jim Sullivan, PhD, Chief Executive Officer of Vanqua Bio. “Jesse, Omer, and Rand have extensive expertise in rare diseases and neurodegenerative disorders, and bring first-hand knowledge of what it takes to translate cutting-edge science into breakthrough therapies that can transform patient care and outcomes. Their insights will be critical to the success and growth of Vanqua Bio as we expand our pipeline and advance VQ-101 into clinical trials.”
Vanqua is initially developing VQ-101 for the treatment of Parkinson’s disease with mutations in GBA1 (GBA-PD), the gene that encodes GCase. Mutations in GBA1 are the most common genetic risk factor for PD, representing approximately 10% of patients with PD worldwide. Mutations in the GBA1 gene result in a decrease in the activity of GCase. Reductions in the activity of GCase disrupt the function of lysosomes, the recycling centers of the cells, enabling toxic forms of proteins, including alpha synuclein, to accumulate and harm neurons.
In preclinical studies in patient-derived neuronal cells, VQ-101 robustly activates wild-type and mutant forms of GCase, reduces lipid substrates, and blocks the accumulation of pathogenic forms of alpha synuclein. In ex vivo studies in whole blood, VQ-101 potently activates GCase in multiple species including human healthy volunteers and patients with GBA-PD. In vivo, VQ-101 demonstrated enhancement of GCase activity in four animal species, and significant CNS exposure following once-daily dosing. VQ-101 demonstrated a very promising profile in preclinical GLP safety and tolerability studies and is projected to be administered once daily in humans. Vanqua expects to initiate clinical trials in the first quarter of 2024, in which the company will seek to demonstrate safety and target engagement in healthy volunteers and GBA-PD patients.
Dr. Cedarbaum is an experienced neurologist and clinical trialist with expertise in Movement Disorders. He has spent the majority of his career leading teams in the areas of neurodegenerative, neuropsychiatric, and retinal disorders at Regeneron Pharmaceuticals, Elan Corporation, Cytokinetics, Bristol-Myers Squibb and Biogen. His research activities have encompassed efforts to develop novel clinical outcome measures and biomarkers, including digital health technologies for clinical trials.
As Chief Development Officer, Mr. Siddiqui will drive the progression of Vanqua Bio’s programs through clinical development. Previously, he served as head of clinical development operations at Alector, a biotech company working on leveraging the immune system to discover cures for neurodegenerative diseases. In that role, he led the strategy and execution of trials for the portfolio of drugs in the clinic across multiple neurological indications.
Rand Sutherland, MD, MPH, is a physician-scientist focused on developing novel treatments for patients with rare and difficult-to-treat conditions, across multiple therapeutic areas. Previously, he served as CEO of Seeker Biologics and President of Translate Bio. Dr. Sutherland has served in various research, development, and medical affairs roles where he and his colleagues designed and executed innovative clinical development programs for novel therapies in lysosomal storage diseases, rare neurological conditions, and rare blood disorders.
About Vanqua Bio
Founded in 2019 and headquartered in Chicago, Vanqua Bio is a biopharmaceutical company dedicated to discovering and developing next-generation medicines that have the potential to transform the lives of patients with neurodegenerative diseases. We are supported by world-class investors, including OrbiMed, Omega Funds, Surveyor Capital (a Citadel company), Avoro Ventures, Casdin Capital, Pontifax, Eli Lilly and Company, Logos Capital, and Osage University Partners. Our technology platform utilizes human genetics and patient-derived neuronal cells to identify, validate, and clinically translate novel disease pathways associated with lysosomal dysfunction or aberrant activation of the innate immune system. Initially, we are targeting glucocerebrosidase (GCase) as a potential treatment for Parkinson’s disease (PD). Our lead program, VQ-101, is expected to enter clinical development in the first quarter of 2024. Additional programs address overactivation of the innate immune system in central and peripheral neurodegenerative disorders, including Alzheimer’s disease. For more information, go to www.vanquabio.com.