STIFEL 2026 HEALTHCARE CONFERENCE - KORSANA BIOSCIENCES INC

Financial Performance:

• Reported $456 million in cash on the balance sheet as of September end; raised over $500 million since inception two years ago.
• Current cash runway extends into 2029, covering development through key clinical catalysts.
• No revenue or earnings data reported as the company is pre-commercial and in early clinical development.

Guidance and Future Outlook:

• Anticipates first healthy volunteer data (Phase 1) by mid-2025; interim proof-of-concept data in early Alzheimer's patients expected by late 2025 or Q1 2026.
• Targeted Phase 3 trial design is "enabling," aiming to de-risk the program through integrated single ascending dose (SAD), multiple ascending dose (MAD), and expansion cohorts.
• Long-term goal includes potential expansion into pre-symptomatic populations where earlier plaque clearance may delay symptom onset.
• Company intends to explore combination therapies involving amyloid clearance and additional targets in 5–10 years.

Business Segments and Product Lines:

• Lead Asset (KRS-A028): A disease-modifying antibody targeting pyroglutamate-modified forms of amyloid (pGlu-Abeta) using a transferrin receptor shuttle mechanism.
• Differentiation Strategy: Designed to preserve phagocytosis for plaque clearance while minimizing Complement-Dependent Cytotoxicity (CDC) and Antibody-Dependent Cellular Cytotoxicity (ADCC) to reduce anemia risk.
• Dosing Profile: Aims for subcutaneous administration at 150 mg once monthly (1 mL volume), matching Trorimimab's brain exposure but potentially offering superior potency.
• Pipeline Status: Discovery programs exist for additional targets but are undisclosed to maintain competitive focus on the lead asset.

Market and Competitive Landscape:

• Competitors: Roche's Trorimimab (current benchmark for speed/safety), Eli Lilly's Remternetug (also pGlu-Abeta selective), and Biogen's Aducanumab/Leqembi.
• Market Opportunity: Alzheimer's disease is viewed as an inflection point; approved therapies are already generating over $1 billion annually with a vast untreated population.
• Competitive Edge: Corsana positions KRS-A028 in a "sweet spot" between full effector function and effector-null molecules, balancing deep plaque clearance with improved safety (low ARIA, reduced anemia) and convenience (sub-Q).
• Market Trends: Shift toward earlier intervention (pre-symptomatic) where safety and tolerability will be critical for market adoption.

Risks and Challenges:

• ARIA Risks: While shuttling is expected to reduce Amyloid-Related Imaging Abnormalities (ARIA) to single-digit percentages, safety is still being validated in human trials.
• Anemia: Transferrin receptor targeting poses a historical risk of anemia (reticulocyte depletion); management is attempting to mitigate this via engineered antibody Fc regions.
• Trial Complexity: Success relies on the Phase 1-2 study providing sufficient Phase 3-enabling data; failure to demonstrate dose-response or safety could delay development.
• Pre-symptomatic Data: While promising, efficacy in pre-symptomatic patients relies on extrapolation from symptomatic data and time-to-event endpoints.

Management Commentary and Tone:

• Tone: Confident and focused; management emphasizes the company is "well-capitalized" and "focused" on a single major unmet need.
• Strategic View: CEO Jonathan Violin describes the current landscape as an opportunity to build a "best-in-class" molecule by optimizing existing shuttle technology rather than starting from scratch.
• Confidence: High confidence in preclinical modeling predicting 150 mg sub-Q dosing, leveraging 30 years of accumulated knowledge on transferrin targeting.

Analyst Questions and Answers:

• Mechanism of Action: Clarified that pGlu-Abeta is the optimal target due to higher plaque formation stickiness and superior clearance rates seen with Dinaranumab and Remternetug.
• Safety Profile: Management explained that Trorimimab's ARIA is linked to high peripheral doses causing vessel crowding; KRS-A028's shuttle allows lower peripheral exposure, theoretically decoupling plaque clearance from ARIA.
• Pre-symptomatic Market: Acknowledged this as a key future opportunity but noted that safety, tolerability, and convenience will be even more critical in this population than in symptomatic patients.
• Platform Utility: Stated that while other shuttle programs exist, KRS-A028's specific design to balance effector function provides a unique value proposition.

Other Key Points:

• Company Structure: Recently went public via reverse merger in September; based on a clean balance sheet with no immediate debt concerns.
• Clinical Design: The Phase 1-2 study is structured to be "Phase 3 enabling," integrating SAD, MAD, and expansion cohorts to accelerate the path to registration.
• Therapeutic Philosophy: Believes amyloid clearance will remain the backbone of Alzheimer's therapy, but combination approaches may be necessary for optimal long-term treatment.
• Geography: No specific geographic markets were highlighted, but the focus is on a global unmet need driven by aging demographics.