BIORCHESTRA Announces Significant Progress in Studies of BMD-001 Drug Program

Encouraging non-human primate (NHP) findings support the progression of IV-formulated BMD-001 ahead of formal clinical studies for Alzheimer's disease,Amyotrophic Lateral Sclerosis,and Parkinson's disease

Further validation of Brain Targeting RNAi Nanomedicine (BTRiNTM),including target knockdown efficiency data across the diverse brain regions,accelerating pharmacokinetic/pharmacodynamic (PK/PD) studies and the pending release of microRNA scope data ahead of an Investigational New Drug (IND) submission

Continued invention and scientific insights in neurodegeneration biology fuel a broad early-stage pipeline of therapeutic candidates

CAMBRIDGE,Mass.and DAEJEON,South Korea,April 24,2023 -- BIORCHESTRA,a biotherapeutics companyfocused on rare and degenerative diseases within the Central Nervous System (CNS),today announced significant progress in its lead drug program – BMD-001. Leveraging a proprietary,IV-formulated brain-targeting RNAi Nanomedicine (BTRiNTM) platform that combines targeted cell delivery capabilities and proprietary RNA chemistries,BMD-001 has achieved significant milestones,including:

Broad biodistribution across the brain,demonstrating disease target knockdown and related functional outcomes; and

Downstream engagement of epigenetic disease hallmarks,including amyloid β plaques and neurofibrillary tangles.

BIORCHESTRA's Scientific Founder,Chairman,and Chief Executive Officer,Dr.Branden Ryu,commented,"We are encouraged by these NHP findings,revealing that our IV formulation achieves consistent,sustained broad brain biodistribution and remarkable target knockdown across multiple measures. Today's results underpin the company's accelerated development ofBMD-001 within our neurodegenerative disease franchise focused on Alzheimer's disease,and Parkinson's disease."

President & Chief Medical Officer,Dr. Louis St. L. O'Dea,"Beyond achieving broad biodistribution in the brain,demonstrating significant target knockdown and related functional outcomes,our IV-formulated nanomedicine platform uniquely targets upstream epigenetic hallmarks associated with core downstream neurodegenerative disease pathologies. These include amyloid β plaques,neurofibrillary tangles,sustained activation of brain-resident macrophages and other immune cells that exacerbate amyloid and tau pathology." Dr. O'Dea added,"These results are notable given the challenges that have been encountered in providing meaningful therapeutic solutions for the rising number of patients,and their families,affected by neurodegenerative diseases." Dr. O'Dea concluded,"Based on my experience in RNA therapeutics at Moderna and Akcea/Ionis,I see these results as very promising ahead of early meetings with international regulatory agencies to discuss and gain agreement on our development strategies."

About Brain Targeting Ribonucleic Acid Interference (RNAi) Nanomedicine (BTRiNTM)

BTRiNTM targets an amino acid receptor in the blood-brain barrier shared by neurons,astrocytes,and microglia. In addition to significant functional outcomes in a non-human primate study of Alzheimer's disease,BTRiNTM has been leveraged with ASO and siRNA to validate the effective knockdown of a target implicated in Amyotrophic Lateral Sclerosis and Parkinson's disease.  Ongoing company discovery and development research will leverage BTRiNTM for other underserved diseases,such as Glioblastoma multiforme,Lewy body dementia,Spinocerebellar ataxia type three,Frontotemporal dementia,Progressive supranuclear palsy. For more information,visit: www.biorchestra.com/science/our-scientific-approach

AboutBMD-001

Under development for efficient and effective IV administration targeting specific micro-RNA associated with neuroinflammation,mitochondrial dysfunction,neurodegeneration,and amyloid protein deposition in Alzheimer's disease,and Parkinson's disease. BMD-001 has successfully demonstrated the suppression of specific microRNA across rodent and non-human primate models,normalizing homeostatic processes,reducing amyloid and tau protein deposition,reducing neuroinflammation,and improving cognitive function. 

About BIORCHESTRA

BIORCHESTRA is a biotherapeutics company focused on rare and neurodegenerative diseases within the Central Nervous System by leveraging its proprietary Brain Targeting RNAi Nanomedicine (BTRiNTM) platform. BTRiNTM combines targeted cell delivery capabilities and proprietary RNA chemistries to develop first and best-in-class therapeutics. The company has extensive research and development and GMP manufacturing capabilities in Daejeon, South Korea,and U.S. headquarters in Cambridge,Massachusetts,U.S.A. For more information,visit:  www.biorchestra.com and follow us on Twitter @biorchestra and LinkedIn.

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