Protected: Pipeline - SciNeuro Pharmaceuticals

SNP614

Leucine-rich repeat kinase 2(LRRK2) is a multifunctional protein with GTPase & kinase activities. LRRK2 gene mutations are associated with increased activities of LRRK2 protein, conferring elevated risk for Parkinson’s disease (PD). SNP614 is a chemically modified antisense oligonucleotide (ASO) that efficiently downregulates LRRK2 mRNA thereby lowering its levels and activities linked to PD development and progression. ASO treatment enables a disease-modifying therapy and is superior to current standard of care (SoC) symptomatic approaches. The SNP614 program is being developed through a funding partnership with The Michael J Fox Foundation – a global leader in Parkinson’s Disease research (Also see the SciNeuro-MJFF news) and the LRRK2 Investigative Therapeutics Exchange (LITE) Program.

SNP203

Progranulin (PGRN) is a secreted glycoprotein growth factor encoded by the GRN gene that plays a critical role in regulating lysosomal function, neuronal health and survival, and inflammation. Loss-of-function mutations in GRN cause frontotemporal dementia (GRN-FTD) and are associated with Alzheimer’s disease (AD). Extracellular PGRN levels are regulated by its cell surface receptor, Sortilin 1 (SORT1), through the endocytic degradation pathway. SNP203 is a best-in-class anti-SORT1 antibody that restores extracellular PGRN levels by blocking the PGRN-SORT1 interaction and reducing PGRN endocytic clearance and thus increasing extra-cellular PGRN levels. Compared to competitor molecules, SNP203 demonstrates superior potency, improved pharmacokinetics (PK), lower dosing requirements, and a more patient-friendly delivery method. (Also see SNP837)

SNP234

Amyloid plaques are a hallmark of Alzheimer’s disease and other neurodegenerative disorders. A major component of these plaques, aggregated beta-amyloid proteins, are widely considered central to pathogenesis. SNP234 is a next-generation anti-amyloid antibody with exceptional potency and selectivity for toxic aggregated species over non-pathogenic monomeric forms. By removing pathogenic aggregates and preventing their further deposition, SNP234 holds promise as a disease-modifying therapeutic that may slow the progression of amyloid-related neurodegeneration and cognitive decline. (Also see SNP837)

SNP201

Alpha-synuclein is a major component of Lewy bodies, a pathological hallmark of synucleinopathathies, including Parkinson’s disease (PD), dementia with Lewy bodies (DLB), and multiple system atrophy (MSA). SNP201 is a high affinity alpha-synuclein antibody, binding preferentially to aggregated forms of alpha-synuclein known to be pathogenic. Superior binding affinity and epitope recognition enable a favorable target engagement to achieve better clinical efficacy. (Also see SNP837)

SNP318

Lipoprotein-Associated Phospholipase A2 (Lp-PLA2) is an enzyme that hydrolyzes oxidized phospholipids typically on low-density lipoprotein (LDL) to yield lysophosphatidylcholine (lysoPC), a potent proinflammatory factor in both CNS and periphery. LysoPC induces vascular inflammation, which leads to damages in blood vessels in the brain including blood-brain barrier (BBB) and blood retinal-barrier (BRB). SNP318 is a CNS-penetrant inhibitor of Lp-PLA2 with the potential to repair BBB and BRB, thereby restoring vascular health to achieve clinical benefit in neurodegenerative diseases including Alzheimer’s disease and macular diseases, where vasculature integrity is central to disease progression and management.

SNP837

The blood-brain barrier (BBB) is a key structure that protects the brain by selectively allowing molecule and nutrient exchange between the brain and peripheral circulation. This barrier system prevents typical drug molecules from entering the brain, hampering their therapeutic effect. SciNeuro has developed a shuttle platform to enable therapeutic molecules to be transported into the brain. The modular shuttle, when combined with a cargo (e.g., antibody), effectively delivers the therapeutic moiety to the CNS. The elevated CNS concentration and broad biodistribution to the deep brain regions, enhances target engagement and thereby therapeutic potential.

SciNeuro’s BBB shuttle platform demonstrates best in class characteristics, in both efficiency and pharmacokinetics. In addition, it is designed to be modularly applicable across a variety of therapeutic modalities.

SNP622

Apolipoprotein E (APOE) is essential for the transport of lipids throughout the body. APOE4 is one of three alleles (APOE2, APOE3, and APOE4) which represents the significant genetic risk factor for Alzheimer’s disease. APOE4 exerts proinflammatory and disease-accelerating effects that dominate over the neuro-protective effects of APOE. Reduction of brain APOE4 therefore represents a therapeutic approach to slow disease progression. SNP622 is a potent anti-sense oligonucleotide (ASO) that reduces APOE expression in relevant human brain cells with superior features relative to a benchmark ASO, particularly in vivo efficacy.

SNP210

Cytoplasmic TDP-43 (transactive response DNA binding protein of 43 kDa) forms toxic aggregates featuring C-terminal phosphorylation and correlating with neurodegeneration. Reduction of extracellular TDP-43 protects neurons from TDP-43 induced pathology and death. SNP210 is a human autoantibody isolated from patient with high affinity of disease-variant of TDP-43. Targeted disease indications include amyotrophic lateral sclerosis (ALS) and certain forms of frontotemporal dementia (FTD).