Coenzyme Q10 Deficiency

Primary CoQ10 deficiency is a rare mitochondrial disorder that impacts fewer than one in 100,000 individuals. Credit: StudioMolekuul via Shutterstock. BPGbio has received a Rare Pediatric Disease Designation from the US Food and Drug Administration (FDA) for BPM31510IV to treat primary coenzyme Q10 deficiency, an ultra-rare mitochondrial disorder. Primary CoQ10 deficiency affects fewer than one in 100,000 people. It hampers the body’s ability to produce CoQ10, a molecule crucial for cellular energy production and biomembrane stabilisation. The deficiency can lead to severe developmental delays, weakness of muscle, seizures, and potentially fatal organ damage, with symptoms often appearing in early childhood. Oral CoQ10 supplements failed to reach therapeutic levels in severely affected organs. BPGbio’s BPM31510IV has shown potential in addressing this gap, leveraging the company’s NAi Interrogative Biology platform, which combines patient biology with AI-driven analysis. See Also: Potential for peptides to replace antibodies in radiopharma, says WuXi AppTec exec BPGbio’s mitochondrial disorder drug gets paediatric disease status It is not only a lead candidate for solid tumours such as glioblastoma multiforme and pancreatic cancer but also shows promise for various rare diseases. BPGbio president and CEO Niven Narain said: “BPM31510 has been developed in topical, IV and oral formulations and is a prime example of the power of our biology-first approach to drug discovery and development, illustrating the powerful predictive capabilities of our NAi Interrogative Biology platform, and the benefit of validating AI-derived predictions through wet lab experiments and in vivo modelling. “By using hypothesis-free causal AI to analyse vast amounts of biological data, we were able to uncover and validate new potential applications BPM31510, including its potential to address mitochondrial diseases like primary CoQ10 deficiency.” BPGbio’s investigational treatment has also received orphan drug designation for glioblastoma multiforme, pancreatic cancer, and epidermolysis bullosa, in addition to the rare pediatric disease designation for primary CoQ10 deficiency.

BOSTON, MA, USA I August 26, 2024 I BPGbio, Inc ., a leading biology-first, AI-powered, clinical stage biopharma focused on mitochondrial biology and protein homeostasis, today announced that Vivek K. Vishnudas, Ph.D. , Chief Technology Officer and R&D Site Head at BPGbio, will present on the company’s Protein Homeostasis program at the 15th Annual Drug Innovation Forum 2024 . Dr. Vishnudas’ presentation, entitled “A Novel and Differentiated Approach to Targeted Protein Degradation – Leveraging the Ubiquitin Conjugating Enzyme (E2) Family, ” will be part of the opening sessions on September 4 in Berlin, Germany. The topic of Targeted Protein Degradation (TPD) has gained momentum in recent years as a strategy for modulating difficult-to-target proteins or protein targets that do not offer beneficial clinical outcomes from traditional small molecule inhibition. While conventional degraders harness E3 proteins to achieve degradation, recent advancements have demonstrated that E2 proteins—once thought to be challenging to target—can be potent effectors of TPD, opening new avenues for drug discovery. BPGbio’s TPD program focuses on targeting E2s, paving the way for the design of novel bifunctional degraders and monovalent glues. “I am honored to present BPGbio’s first-in-class E2 program to fellow researchers and industry peers,” said Dr. Vishnudas. “We have a unique approach to drug design and we look forward to meeting with potential partners in the biopharma industry who want to join us in bringing our novel therapeutics to patients in need.” In addition to utilizing E2s, the BPGbio’s protein homeostasis program features a proprietary library of more than 1,000 Ro3 fragments discovered by BPGbio that are potential ligands and seed compounds to a variety of E2 targets, proprietary ternary structures, a computational tool kit for E2 ligand design, and assays for rapidly attaining selectivity and specificity. The BPGbio Protein Homeostasis program also includes E2-based molecular glue lead compounds for Huntingtin homeostasis. The presentation will cover the following key topics: BPGbio’s therapeutic pipeline also includes drug candidates for glioblastoma (orphan drug), pancreatic cancer (orphan drug), primary CoQ10 deficiency , epidermolysis bullosa (EB, orphan drug), squamous cell carcinoma (SCC, orphan drug), sarcopenia , solid and liquid tumors, Huntington’s disease (orphan drug) and Parkinson’s disease. About BPGbio BPGbio is a leading biology-first AI-powered clinical stage biopharma focused on mitochondrial biology and protein homeostasis. The company has a deep pipeline of AI-developed therapeutics spanning oncology, rare disease and neurology, including several in late-stage clinical trials. BPGbio’s novel approach is underpinned by NAi , its proprietary Interrogative Biology Platform, protected by over 400 US and international patents; one of the world’s largest clinically annotated non-governmental biobanks with longitudinal samples; and exclusive access to the most powerful supercomputer in the world. With these tools, BPGbio is redefining how patient biology can be modeled using bespoke Bayesian AI specifically designed for solving large-scale biology challenges. Headquartered in greater Boston, the company is at the forefront of a new era in medicine, combining biology, multi-modal data, and AI to transform the way we understand, diagnose, and treat disease. For more information, visit www.bpgbio.com . SOURCE: BPGbio