Phase 1: Clinical Trials

Phase 1: Clinical Trials

Details:

Objective and Scope:

  • The primary goal of Phase 1 trials is to assess VE3’s safety, tolerability, and pharmacokinetics in humans.
  • Secondary objectives include identifying the maximum tolerated dose (MTD), exploring initial signs of efficacy, and establishing a dose-escalation strategy for subsequent trials.

Patient Selection and Enrollment:

  • Enroll approximately 20–40 patients diagnosed with advanced cancers expressing high levels of ALDH1A3, such as breast, lung, and colorectal cancers.
  • Use biomarker-driven diagnostics to identify patients most likely to respond to VE3 therapy, ensuring precision and relevance.
  • Prioritize individuals for whom standard-of-care therapies have failed, offering a novel treatment option.

Trial Design:

  1. Dose Escalation (3+3 Design):
    • Start with a low dose of VE3 and incrementally increase the dose in cohorts of 3–6 patients until the MTD or dose-limiting toxicity (DLT) is observed.
  2. Safety and Tolerability Assessment:
    • Monitor patients for adverse events, both short-term (e.g., nausea, fatigue) and long-term (e.g., organ toxicity).
    • Perform regular blood tests, imaging, and clinical evaluations to ensure patient safety.
  3. Pharmacokinetics (PK) and Pharmacodynamics (PD):
    • Measure drug absorption, distribution, metabolism, and excretion to determine the optimal dosing schedule.
    • Analyze the effect of VE3 on ALDH1A3 activity and tumor biology to confirm its mechanism of action.
  4. Exploratory Efficacy Assessment:
    • Track tumor response through RECIST (Response Evaluation Criteria in Solid Tumors) to evaluate partial or complete tumor shrinkage.
    • Document progression-free survival (PFS) and overall survival (OS) trends as early efficacy indicators.

Achievements:

  1. Safety Profile Established:
    • Demonstrated that VE3 is well-tolerated at therapeutic doses, with minimal off-target effects.
  2. Optimal Dose Identified:
    • Defined a safe and effective dose for Phase 2 trials, ensuring patient safety while maximizing therapeutic benefit.
  3. Mechanism Validation in Humans:
    • Provided clinical evidence that VE3 effectively inhibits ALDH1A3, reducing tumor progression and therapy resistance.
  4. Combination Therapy Feasibility:
    • Explored VE3’s potential to synergize with existing standard-of-care therapies, broadening its clinical application.

Strategic Impact:

  1. Regulatory Advancement:
    • Generated critical data to support Phase 2 trial approvals and regulatory filings in multiple jurisdictions.
  2. Investor Confidence:
    • Demonstrated Theranib’s ability to transition successfully from preclinical to clinical development, attracting further investment.
  3. Patient-Centric Innovation:
    • Delivered a promising new therapeutic option for patients with high unmet needs, reinforcing Theranib’s commitment to transforming cancer care.