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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
NEUROLOGY
Tralesinidase Alfa (TA-ERT) Enzyme Replacement1 MPS IIIB
(Sanfilippo Syndrome
Type B)1
BLA Filing
Fourth Quarter 2026
Tildacerfont/ Cortibon* CRF1 receptor antagonist small modecule2 Major Depressive
Disorder
(MDD)3
Phase 2 Data
1H 2026

References:
*Tildacerfont and Cortibon, a Companion Diagnostic, developed in partnership with HMNC Brain Health
CRF1= Corticotropin-releasing Factor Type-1

  1. Muschol et al., A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B. J Clinical Investigations 2022.
  2. Sarafoglou et al., Tildacerfont in Adults With Classic Congenital Adrenal Hyperplasia: Results from Two Phase 2 Studies. J Clinical Endocrinology and Metabolism.
  3. HMNC BRAIN HEALTH, a disruptive clinical stage biopharma company pioneering precision psychiatry.

Tralesinidase Alfa Enzyme Replacement

Transformative Therapy for MPS IIIB

(Sanfilippo Syndrome Type B)

Program Overview

Development Stage

BLA submission anticipated in 1H 2026, targeting accelerated approval.

Condition

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

Prevalence

~1 in 200,000 newborns.¹

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

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. Huang et al., Disease modeling for Mucopolysaccharidosis type IIIB using patient derived induced pluripotent stem cells. Experimental Cell Research 202.

Tildacerfont and Cortibon

Advancing Precision Treatment for Major Depressive Disorder (MDD)

Program Overview

Development Stage

Phase 2 ongoing; Phase 2 data anticipated in 1H 2026.

Condition

Major Depressive Disorder (MDD)

Prevalence

~300 million people globally1 (~4% of the world’s population2 and ~9% of US adults3) and is on the rise, representing a major unmet need in psychiatry.

Current Standard of Care

Primarily SSRIs and SNRIs, with ~15-30% of MDD patients showing treatment resistance4 and ~30-50% of patients failing first-line therapies.5

Disease Impact

A serious and potentially chronic disorder which may lead to emotional, behavioral, and health complications.

Mechanism of Action

CRF1 receptor antagonist targeting stress-related pathways in depression.

Cortibon Diagnostic

Designed to identify patients more likely to respond to CRF1 receptor antagonism by utilizing genetic markers.

Unmet Medical Need

  • Treatment-resistant depression affects millions globally, with limited therapeutic options.
  • Existing antidepressants often fail to address underlying stress-related pathways.
  • Tildacerfont and Cortibon may offer a novel approach, addressing a critical gap in the therapeutic landscape.
References:
  1. Nature: Major depressive disorder: hypothesis, mechanism, prevention and treatment – Feb 2024.
  2. Based on 2025 global population 8 billion people worldwide.
  3. JAMA: Management of Depression in Adults – June 2024.
  4. Sousa et al., Treatment-resistant depression and major depression with suicide risk—The cost of illness and burden of disease. Frontiers Public Health 2022.
  5. Depression Treatment after Unsatisfactory Response to SSRIs when used as First-line Therapy –Agency for Health Care Program 2010.