Q1 2024 Earnings Summary
- Robust Alzheimer's Phase II trial design: The trial leverages enrichment criteria to select patients with biomarkers of neuroinflammation, enabling a shorter, 6‑month study that is statistically well-powered to detect cognitive benefits while minimizing placebo exposure.
- Potential regulatory acceleration: Executives indicated that if the extraordinary Phase II results are delivered—with patients on XPro showing stability compared to a declining placebo group—the FDA could become more supportive, potentially fast-tracking the drug’s pathway.
- Scalable, safe, and versatile NK cell platform: The INKmune program's outpatient, cytokine-free NK cell activation approach has demonstrated a strong safety profile (e.g., no fever or inflammatory reactions) and efficient manufacturing & logistics, which supports expansion into multiple solid tumor indications.
- Regulatory Uncertainty: The company’s reliance on a 6-month Phase II trial to generate data, while the FDA may require larger and potentially longer trials, creates a risk of regulatory hurdles and additional costly trials if agencies do not accept the abbreviated design.
- Enrollment and Patient Population Concerns: There is uncertainty in the trial’s patient mix, with potential variability in the proportion of patients with mild cognitive impairment versus mild Alzheimer’s. This unclear enrollment strategy and reliance on enrichment criteria might hinder the statistical power and interpretation of results.
- Complications with Open-Label Extension Data: The issues surrounding the open-label extension study—including enrollment closure and challenges in analyzing long-term safety and efficacy due to variable prior treatments—could cloud the overall clinical picture and impact investor confidence.
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FDA Approval
Q: Could flat cognition spur accelerated approval?
A: Management indicated that if the trial shows an extraordinary difference between stable cognition in the XPro group versus rapid decline in the placebo group, the FDA might consider accelerated approval. However, they are cautious and are not betting heavily on this outcome. -
Enrollment Mix
Q: What is the mix of enrolled patient types?
A: Management expects the trial to have around a 50-50 mix of mild AD and MCI patients, balancing initial enrollment of mild AD with later inclusion of MCI, which should support robust statistical outcomes. -
TRD Timeline
Q: When will the TRD trial start?
A: The TRD trial’s advancement depends on available resources, with plans to begin once the FDA IND is accepted and the first patient is enrolled later this year, as Alzheimer’s remains the primary focus. -
Regulatory Jurisdictions
Q: Will non-US regulators follow the FDA?
A: Management noted that while the EMA tends to follow the FDA, agencies like the MHRA may take a more independent approach. Each jurisdiction works through its own considerations. -
Placebo Decline
Q: How long do placebo effects last with cholinesterase inhibitors?
A: Patients on acetylcholinesterase inhibitors typically plateau after 3–6 months, ensuring that any cognitive decline is more marked after that period, helping to distinguish the treatment effect. -
Open-Label Extension
Q: Is dosing ongoing in the open-label extension?
A: Some patients continue dosing under the open-label extension, though the data analysis is complicated by varied prior treatments. The approach is driven by practical drug supply and cost management considerations. -
INKmune Safety
Q: Why does INKmune avoid causing fever?
A: INKmune works by activating NK cells without stimulating T cells, which prevents the release of inflammatory cytokines and thereby avoids fever and related side effects. -
Renal Cancer Potential
Q: Can INKmune be effective in renal cancer too?
A: Management sees promise in renal cancer due to its high NK cell infiltration, analogous to their prostate cancer trial, which supports the rationale for targeting multiple solid tumors. -
Trial Duration & Biomarkers
Q: Is a 6‐month trial sufficient with biomarkers?
A: The design leverages biomarkers to enrich the patient population, allowing a 6-month trial to yield a robust and statistically significant readout, similar to recent anti-amyloid studies.