Seelos Therapeutics announced the initiation of a preclinical study of SLS-007 in Parkinson's disease (PD) delivered through an adeno associated viral (AAV) vector targeting the non-amyloid component core (NACore) of alpha synuclein (α-synuclein).

Seelos has initiated an in vivo preclinical study of SLS-007 in rodents to assess the ability of two specific novel peptides, S62 and S71, delivered via AAV1/2 viral vector, to protect dopaminergic function in the preformed α-synuclein fibril (PFF) rodent model of PD. Production of AAV1/2 vectors encoding each of the two novel peptides incorporating hemagglutinin (HA) tags has already been completed.

This preclinical study is designed to establish the in vivo pharmacokinetic and pharmacodynamic profiles and target engagement parameters of SLS-007. Top-line data is currently expected in late 2020/early 2021.

"In in vitro models, halting or slowing the aggregation of alpha synuclein dramatically slowed the formation of Lewy Bodies which are the hallmarks of the pathogenesis of Parkinson's," said Raj Mehra, Ph.D., Chairman and CEO of Seelos. "This program should complement SLS-004 in which we also recently began studies."

SLS-007 is a family of rationally designed peptidic inhibitors which target the NACore of α-synuclein to inhibit protein aggregation in patients with PD. This central segment of α-synuclein termed NACore that is comprised of residues 68-78, has a critical role in the aggregation and cytotoxicity of α-synuclein. Overexpression of α-synuclein leads to the formation of α-synuclein aggregates which comprise Lewy bodies and neurites which are the hallmarks of the pathogenesis of PD. Recent in vitro and cell culture research have shown that SLS-007 has the potential to stop the propagation and seeding of α-synuclein aggregates.