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Pipeline

Advancing a Differentiated Pipeline of Potentially First-to-Market and Best-in-Class Programs

Pipeline and Anticipated Milestones

Drug Candidate Mechanism of Action Indication
Preclinical
Phase 1
Phase 2
Phase 3
 Anticipated Milestones
Tralesinidase Alfa (TA-ERT) Enzyme Replacement MPS IIIB
(Sanfilippo Syndrome
Type B)
BLA Filing
Fourth Quarter 2026
SPR202 CRH Antagonist
mAb 		Congenital Adrenal
Hyperplasia
(CAH)

References:
CRH = Corticotropin-releasing hormone
mAb = monoclonal antibody

Tralesinidase Alfa Enzyme Replacement

Transformative Therapy for MPS IIIB

(Sanfilippo Syndrome Type B)

Program Overview

Development Stage

BLA submission anticipated in Q4 2026, targeting accelerated approval.

Condition

Mucopolysaccharidosis Type IIIB (MPS IIIB), or Sanfilippo Syndrome Type B.

Prevalence

~0.5 to 4 per 100,000 live births1 2 3 4

Current Standard of Care

No approved treatment options.

Disease Impact

A terminal neurodegenerative genetic disorder caused by a
buildup of toxic sugars called glycosaminoglycans (GAGs) in the central nervous system (CNS).

Treatment Modality

Potential first-to-market enzyme replacement therapy (ERT) for MPS IIIB.

Program Designations

Breakthrough, Rare Pediatric Disease, Fast, Track, and Orphan Drug designations.

Unmet Medical Need

  • MPS IIIB is a progressive and fatal disease with no approved therapies.
  • Patients face severe neurodegeneration, developmental regression, and multi-system decline.
  • Current management is limited to supportive care only.

References:

  1. Rouse, Courtney J.1; Jensen, Victoria N.1; Heldermon, Coy D.2,*. Mucopolysaccharidosis type IIIB: a current review and exploration of the AAV therapy landscape. Neural Regeneration Research 19(2):p 355-359, February 2024. [est. incidence of 1/200,000 births * ~3.6 MM Us Census estimate of ages 0-1 in 2024].
  2. Chien, et al. Newborn Screening for Morquio disease and other lysosomal storage disease: results from the 8-plex assay from 70,000 newborns, Orphanet Journal of Rare Diseases (2020) 15:38
  3. Blackswan Analysis ISOPR Poster, 2019.
  4. Chien, et al. Newborn Screening for Morquio disease and other lysosomal storage disease: results from the 8-plex assay from 70,000 newborns, Orphanet Journal of Rare Diseases (2020) 15:38