Therapeutic Spectrum

Full Strategic Pipeline

ACA Therapeutics is a clinical-stage biopharmaceutical company leveraging its proprietary Upstream–Midstream–Downstream (UMD) Framework to systematically address disease biology across multiple pathways. Our platform is designed to efficiently generate and prioritize therapeutic programs across oncology, neurology, metabolic, and rare diseases. Our pipeline reflects a prioritized development strategy, with a lead clinical program and a broad portfolio of indications supported by translational research and prepared for efficient advancement toward IND submission.

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I. Lead Clinical Programs

  • ACA-001: Pancreatic Cancer — Phase I IND Cleared (Trial Ready)
  • ACA-002: Type 2 Diabetes (T2DM) & Beta-Cell Restoration — Phase I IND Submission Planned
  • ACA-003: Benign Prostatic Hyperplasia (BPH) with Chronic Non-bacterial Prostatitis (CNP) —Phase I IND Submission Planned
  • ACA-004: Autism Spectrum Disorder (ASD) — Targeting Immune-Brain Axis Dysregulation —Phase I IND Submission Planned
  • ACA-005: Alzheimer’s Disease (AD) — Restoring Cognitive Pathways — Phase I IND Submission Planned

II. Core Pipeline Programs (IND-Ready Potential)

Oncology & Rare Cancers

  • ACA-006: Neuroendocrine Tumors (NEC)
  • ACA-007: Colorectal Cancer
  • ACA-008: Prostate Cancer
  • ACA-009: Gynecologic & Fallopian Tube Cancer (FTC)
  • ACA-010: Squamous Cell Carcinoma
  • ACA-011: Renal Cell Carcinoma
  • ACA-012: Bladder Cancer
  • ACA-013: Gastric Cancer / GIST
  • ACA-014: Brain Tumors & Glioma
  • ACA-015: Melanoma
  • ACA-016: Universal Lymphoma Subsets
  • ACA-017: Myelodysplastic Syndrome (MDS)
  • ACA-018: Acute Myeloid Leukemia (AML)
  • ACA-019: Appendiceal Cancer
  • ACA-020: Cholangiocarcinoma (iCCA)
  • ACA-021: Chronic Lymphocytic Leukemia (CLL)
  • ACA-022: Ewing Sarcoma
  • ACA-023: Follicular Lymphoma
  • ACA-024: Hepatocellular Carcinoma (HCC / FL-HCC)
  • ACA-025: Hodgkin Lymphoma
  • ACA-026: Multiple Myeloma
  • ACA-027: Small Intestine Cancer (SIC)
  • ACA-028: Breast Cancer (Triple Negative / General)
  • ACA-029: Immune-Driven NF-κB/AKT Dysregulation Subtype Disorders
  • ACA-030: Lung Cancer (NSCLC/SCLC)

Neurology & Neuropsychiatry

  • ACA-031: ADHD (attention deficit hyperactivity disorder) — Systemic Neuromodulation
  • ACA-032: Myasthenia Gravis (MG) & Thymoma — Neuromuscular Junction Restoration
  • ACA-033: Parkinson’s Disease (PD) — Resolving Neuroinflammation

Ophthalmology & Regenerative Care

  • ACA-034: Macular Degeneration — Retinal Micro-environment Restoration
  • ACA-035: Optic Nerve Regeneration & Advanced Fundus Diseases

Systemic Immune & Metabolic Pillar

  • ACA-036: Hyperlipidemia / Dyslipidemia — Upstream Metabolic Reset for Lipid Homeostasis & Cardiovascular Protection
  • ACA-037: Obesity — Upstream Metabolic Reset for Weight Homeostasis & Metabolic Comorbidity Resolution
  • ACA-038: Atopic Dermatitis
  • ACA-039: Restless Leg Syndrome (RLS)
  • ACA-040: Polycythemia Vera
  • ACA-041: Salivary Gland Adenocarcinoma
  • ACA-042: Soft-Tissue Sarcomas
  • ACA-043: Psoriasis
  • ACA-044: Vocal Cord Leukoplakia
  • ACA-045: Lyme Disease (Chronic/Refractory)
  • ACA-046: Osteoarthritis (Chronic Joint Inflammation)
  • ACA-047: Chronic Kidney Disease (CKD)
  • ACA-048: Graves’ Disease (Autoimmune Hyperthyroidism)
  • ACA-049: Hashimoto’s Thyroiditis (Autoimmune Hypothyroidism)

III. Rare Diseases / Orphan Indications

Platform leverages upstream reset to enable low-dose intermittent downstream therapy, reducing toxicity and cost. Many carry Orphan Drug Initiative potential.

  • [ACAR – 001]: Prader-Willi Syndrome
  • [ACAR – 002]: Leber Hereditary Optic Neuropathy (LHON)
  • [ACAR – 003]: Non-Dystrophic Myotonia
  • [ACAR – 004]: Pediatric Glioma
  • [ACAR – 005]: Prurigo Nodularis
  • [ACAR – 006]: Rett Syndrome
  • [ACAR – 007]: Duchenne Muscular Dystrophy (DMD)

Platform Advantage

The UMD Framework enables a systems-level approach by targeting upstream drivers, midstream signaling pathways, and downstream disease manifestations. This integrated strategy supports the efficient generation of multiple therapeutic programs and allows flexible prioritization for clinical development and partnership opportunities.

By restoring upstream immune‑metabolic balance, our platform is designed to complement existing therapies, enabling lower doses, reduced treatment burden, and improved long‑term outcomes.

This upstream‑reset approach reduces the biological pressure that drives chronic, lifelong treatment dependence. By stabilizing immune, metabolic, and neuro‑inflammatory networks at their source, the platform has the potential to transform many current “forever medications” into time‑limited, lower‑intensity regimens—enhancing safety, lowering cost, and expanding therapeutic possibilities across rare and complex diseases.

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