BIOAGE LABS, INC. STIFEL 2026 HEALTHCARE CONFERENCE - BIOAGE LABS INC

Explicit Guidance:

• No financial guidance (revenue, earnings, margins, capex, cash flow, dividends, or buybacks) provided.
• Cash Balance: Company reported a cash balance of just over $350 million in the last quarter, sufficient to fund all current activities through proof-of-concept for indicated programs.

Business-Segment Outlook:

• Ophthalmology (Primary Focus): Shifted strategic priority to ophthalmology for the lead program BGE-102 (NLRP3 inhibitor), specifically targeting Diabetic Macular Edema (DME) and Geographic Atrophy (GA).
• QUEL-DME Trial: Ongoing Phase 2 trial targeting visual acuity improvement in center-involved DME patients (monotherapy vs. baseline; combination with VEGF vs. VEGF alone).
  • Readout: Expected end of next year (approx. FY2025).
  • Patient Segments: Targeting treatment-naive patients with compromised vision and patients refractory to VEGF therapy; aiming to reduce injection burden for the "watch and wait" cohort (approx. 40% of the market).
  • Goal: Demonstrate benefit in visual acuity and potentially reduce treatment frequency by addressing underlying inflammation (IL-1beta, pyroptosis) alongside VEGF.
• Geographic Atrophy (GA): Identified as a high-priority indication following successful preclinical models showing prevention of lipofuscin accumulation; plans to explore after establishing pharmacodynamics in the eye via the DME trial.
• Cardiometabolic (NLRP3): The QUEL-CV trial (dose-ranging for peripheral inflammation/metabolic markers, liver MRI) is reading out at the end of this year (FY2024).
  • Strategic Shift: Atherosclerotic cardiovascular disease (ASCVD) is no longer a priority; the program is on pause pending a "decisive win" in the upcoming Artemis trial (acute MI setting) to reverse current skepticism regarding upstream NLRP3 efficacy in CVD.
  • Other Cardiometabolic Indications: Potential exploration of MASH, insulin resistance, and kidney disease based on rich data from the QUEL-CV trial.
• APJ Program:
  • IND Submission: Targeting end of this year (FY2024) for next-generation oral and parenteral programs.
  • Clinical Readout: Expected to debut in the clinic shortly after IND (beginning of next year) with proof-of-concept data.
  • Value Prop: Preclinical data suggests APJ agonism combined with incretins can double weight loss and preserve muscle mass/synthesis in aging.

Market and Competitive Outlook:

• Ophthalmology Demand: Anticipates significant market share capture by addressing the "huge disconnect" in patient willingness to undergo frequent intravitreal injections; potential to treat bilateral disease simultaneously with an oral agent.
• Competitive Landscape: Differentiates via a novel binding site on NLRP3 (blocking active and inactive forms) versus competitors (e.g., Pfizer's MCC950, other inflammasome programs) targeting the ATPase pocket.
• Mechanism Comparison: Views NLRP3 as a superior upstream target compared to IL-6 inhibitors (e.g., Roche/Regeneron) and direct IL-1beta inhibitors, given the capture of IL-18 and pyroptosis in the retinal microvasculature.
• Pricing/Reimbursement: Implicitly targeting value proposition around reduced treatment burden and improved adherence for patients avoiding needles.

Macro / Industry Outlook:

• Regulatory Path: Plans to announce an additional indication for NLRP3 (BGE-102) once plans are "fully solidified," though specific timing or indication name is not disclosed.
• IP Strategy: Intellectual property (IP) around the unique binding site covers specific structures and broadly covers ligands interacting with this site through 2045.
• Clinical Development: Management remains confident in current timelines despite the pause on ASCVD, citing preparatory CMC work done for potential Phase 3 enablement if the Artemis trial results provide a positive signal.

Strategic Initiatives:

• Portfolio Rebalancing: Shifting focus from cardiovascular to ophthalmology and APJ programs to leverage best-in-class tissue distribution (retina/CSF) of BGE-102.
• IND Submission: Targeting end-of-year IND for next-gen APJ programs to facilitate clinical debut in early 2025.
• Evidence Generation: Leveraging data from the QUEL-CV trial (end of FY2024) to define dosing and downstream marker relationships for future indications.
• Partnership/Out-licensing Potential: Not explicitly stated, but the "beachhead" strategy in ophthalmology suggests a path to broader commercialization or collaboration following successful DME readout.

Risks and Headwinds to Outlook:

• Artemis Trial Results: A negative or non-decisive outcome in the Artemis acute MI trial could confirm skepticism regarding NLRP3 efficacy in cardiovascular settings, reinforcing the shift away from ASCVD.
• Clinical Trial Failure: Risk of failure to demonstrate visual acuity improvement in the QUEL-DME trial, which would stall the primary ophthalmology strategy.
• Regulatory Uncertainty: Timing of the additional NLRP3 indication announcement depends on internal solidification of plans, with no guaranteed timeline.
• Competition: Rapidly evolving landscape in GLP-1/APJ space and ophthalmology; competitors in complement inhibition (e.g., Flamazon) showing early promise in GA.
• Data Interpretation: Management expresses skepticism that the Zeus trial failure was due to patient selection ("too sick"), suggesting a fundamental biological hurdle for upstream inflammation in CVD that may not be easily overcome.

Management Tone on the Future:

• Confidence: High confidence in the unique biological profile of BGE-102 (complete target inhibition, tissue distribution) and the "best-in-class" potential in ophthalmology.
• Pragmatism: Willing to pivot capital allocation away from cardiovascular indications if clinical data (Zeus, Artemis) does not support it, despite significant preparatory work already invested.
• Optimistic but Guarded: Enthusiastic about APJ and ophthalmology programs but cautious regarding the CVD narrative, explicitly stating a need for a "decisive win" to reverse current stance.
• Financial Stability: Expresses confidence in the balance sheet ($350M cash) to fund the current pipeline through key proof-of-concept milestones without immediate dilution.

Miscellaneous:

• Target Biology: Emphasized that NLRP3 drives sterile inflammation (metabolic stress, nutrient excess) distinct from pathogen-driven inflammation.
• Biomarkers: CRP used as a primary PD marker in QUEL-CV; visual acuity as primary endpoint in QUEL-DME; MRI liver imaging for metabolic profiling.
• Safety: Phase 1 data described "placebo-like tolerability" and excellent safety profile in obese/inflamed cohorts.