Trehalose by Seelos Therapeutics for Oculopharyngeal Muscular Dystrophy: Likelihood of Approval

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Trehalose overview

Trehalose is under development for the treatment of oculopharyngeal muscular dystrophy (a type of congenital myopathy), Huntington’s disease (HD), friedreich ataxia, Amyotrophic lateral sclerosis, Sanfilippo syndrome and undisclosed liver disease. The drug candidate is administered through intravenous infusion. Trehalose is a naturally occurring sugar containing two D-glucose units in an alpha,alpha-1,1 linkage. Trehalose reduces the polyA binding protein nuclear 1 (PABPN1) aggregates seen in oculopharyngeal muscular dystrophy.

It was also under development for the treatment of spino bulbar cerebellar ataxia (SBMA)/Kennedy’s disease, Alzheimer’s disease and Machado-Joseph disease/spinocerebellar ataxia 3 (SCA3).

Seelos Therapeutics overview

Seelos Therapeutics (Seelos) is a clinical-stage biopharmaceutical company that focuses on developing novel technologies and therapeutics for the treatment of central nervous system (CNS) and other rare disorders. Its lead programs include SLS-002, SLS-005, and pipeline products include SLS-004, SLS-007, SLS-008, SLS-010 and SLS-012. Seelos pipeline products find application in treating various multiple CNS disorders including parkinson’s disease, sanflippo syndrome, suicidality post-traumatic stress disorders, major depressive disorders and other diseases such as atopic dermatitis, asthma, and pediatric esophagitis. It operates along with its subsidiaries. Seelos is headquartered in New York, the US.

For a complete picture of Trehalose’s drug-specific PTSR and LoA scores, buy the report here.