BPGbio Presents Novel Drug Target for Huntington's Disease and AI-Discovered Biomarkers for Alzheimer's Disease at Society for Neuroscience 2024

• BPG-0812, a novel ubiquitin-proteasome UBE2K target and UBE2K unloading inhibitor, shows potential to reduce toxic protein aggregates in Huntington's disease

• Protein biomarkers for Alzheimer's disease, identified in collaboration with Harvard Medical School, reveal changes in the liver and immune system that are causally linked to cognitive decline

• The results highlight the capabilities of the NAi Interrogative Biology platform in novel drug discovery and biomarker identification in even some of the most challenging neurological diseases

BOSTON, October 2, 2024–(BUSINESS WIRE)–BPGbio, Inc., a leading clinical-stage biology and AI biopharmaceutical company focused on mitochondrial biology and protein homeostasis, will present the results of two transformative studies at the annual meeting of the Society for Neuroscience, Neuroscience 2024. The conference will take place October 5-9, 2024 in Chicago, Illinois. Both studies utilized BPGbio's proprietary NAi Interrogative Biology® platform, which leverages a hypothesis-free, biology-focused approach to drug and diagnostic discovery.

In the first study, titled “Disruption of Mutant, PolyQ Expanded Huntingtin Aggregation by Modulation of UBE2K, a Novel Ubiquitin-Proteasome Drug Target,” researchers showed that reduction of the E2 ubiquitin-conjugating enzyme UBE2K/HIP2 leads to a loss of mutant Htt aggregates that cause Huntington's disease. The researchers also showed that treatment with the UBE2K discharge inhibitor BPG-0812, a small molecule drug candidate, slowed the discharge of ubiquitin from UBE2K. The results of this study suggest that modulation of UBE2K activity can effectively disrupt the aggregation of mutant Htt, providing a promising new avenue for therapeutic interventions in Huntington's disease.

The second largest study, titled “A systems biology approach to biomarker discovery in Alzheimer's disease using multi-omics and Bayesian artificial intelligence,” identified several novel liver and immune system protein biomarkers associated with cognitive decline. In particular, a biomarker can predict the time to cognitive decline in patients with Alzheimer's disease. These biomarkers were discovered through an analysis of plasma and buffy coat samples from participants in the Massachusetts Alzheimer Disease Research Center's longitudinal study in collaboration with Harvard Medical School. The results, from individuals with varying degrees of cognitive impairment, highlight how liver and immune system dysfunction contribute to cognitive decline and offer significant potential for diagnosing and monitoring the progression of Alzheimer's disease.

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“Rare and age-related neurodegenerative diseases such as Huntington's disease and Alzheimer's disease pose a significant health and economic burden to society in the coming decades. These results highlight the power of our NAi Interrogative Biology platform in discovering disease-modifying therapeutic targets and “This could one day change the lives of people with neurodegenerative diseases,” said Niven R. Narain, Ph.D., president and CEO of BPGbio. “Our research reveals new pathways and possibilities for diagnosing Alzheimer's disease and treating Huntington's disease, where patient groups urgently need new solutions.

Eric Nestler, MD Ph.D., Nash Family Professor of Neuroscience, director of the Friedman Brain Institute and dean of academic and research affairs at the Icahn School of Medicine at Mount Sinai, as well as a member of the scientific advisory board of BPGbio and past president of the Society for Neuroscience, commented: “The ability to uncover novel drug targets and biomarkers using advanced AI-driven platforms such as BPGbio's NAi Interrogative Biology platform is critical to the field of neuroscience. These findings not only deepen our understanding of complex diseases such as Alzheimer's disease and Huntington's disease, but also pave the way for more precise therapeutic interventions that are critical to addressing the profound unmet needs of these vulnerable populations .”

Details about the poster presentation:

Title: A systems biology approach to biomarker discovery in Alzheimer's disease using multi-omics and Bayesian artificial intelligence
Date and time: Monday, October 7, 2024, 1:00 p.m. – 2:00 p.m. (CDT).
Location: MCP Hall A
Moderator: Michael Kiebish, Ph.D.
Program number: PSTR207.21

Title: Disruption of mutant polyQ-expanded huntingtin aggregation through modulation of UBE2K, a novel ubiquitin-proteasome drug target
Date and time: Wednesday, October 9, 2024, 8:00 a.m. – 9:00 a.m. CDT
Location: MCP Hall A
Moderator: Gali Maor, Ph.D.
Program number: PSTR394.09

About BPGbio

BPGbio is a leading biology and AI-driven clinical development biopharmaceutical company focused on mitochondrial biology and protein homeostasis. The company has an extensive pipeline of AI-developed therapeutics in oncology, rare diseases and neurology, including several in late-stage clinical trials. BPGbio's novel approach is based on NAi, its proprietary Interrogative Biology Platform, which is protected by over 400 U.S. and international patents; one of the world's largest clinically annotated non-governmental biobanks of longitudinal samples; and exclusive access to the world's most powerful supercomputer. With these tools, BPGbio is redefining how patient biology can be modeled using tailored Bayesian AI designed specifically to solve grand biological challenges. Headquartered in Greater Boston, the company is at the forefront of a new era in medicine, combining biology, multimodal data and AI to transform the way we understand, diagnose and treat disease. For more information, visit www.bpgbio.com.

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