The Seelos Path

Addressing unmet needs in neurological diseases and disorders and in rare diseases

Advancing multiple late-stage therapeutic candidates for CNS disorders

Developing therapeutics with proven mechanisms of action

Identifying opportunities in large markets

Acute Suicidal Ideation and Behavior (ASIB) in Major Depressive Disorder (MDD) and Post-Traumatic Stress Disorder (PTSD) 

  • High area of unmet need
  • No currently approved therapy
  • Fast-acting intranasal administration
  • J&J expects multibillion-dollar opportunity in the underlying depression indication

ALS (or Lou Gehrig’s disease)

  • Mutations in the C9orf72, SOD1, FUS, and TARDBP genes can cause familial ALS and contribute to the development of sporadic ALS
  • In-vivo studies of trehalose in ALS:
    • Increases the clearance of TDP-43
    • Decreases SOD1 and SQSM1/p62 aggregates and monomers
    • Delays the progression of the disease
    • Preserves ventral horn motor neurons
    • Increases muscle fiber size

Sanfilippo Syndrome

  • Pediatric orphan disease also known as MPS disorder
  • Prolonged enhancement of Lysosomal Pathway
  • Promising activity in other orphan indications,
    e.g. OPMD and SCA3

Parkinson’s Disease (PD)

  • Broad spectrum usage possible in both early and late stage PD patients
  • SLS-004: Gene therapy program targeting the regulation of the SNCA gene, which encodes alpha-synuclein (α-synuclein)
  • SLS-006: Unique L-Dopa sparing efficacy
  • SLS-007: Peptide-based approach targeting the NACore
  • ~10 million patients worldwide

Rare pediatric disease / Asthma / Other potential targets

PD2 Antagonists:

  • Rare pediatric disease
  • Asthma
  • Unknown indication

Seelos Criteria for Drug Development

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  1. Assets must serve a large unmet medical need, orphan population or a unique approach to existing treatments
  2. Assets must possess extensive scientific rationale and/or existing human data
  3. Seelos will look for clinically ready assets or assets where minimal pre-clinical work will be needed
  4. The clinical development pathway should make economic sense and be appropriate for a company our size

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