KURA ONCOLOGY VIRTUAL INVESTOR EVENT - KURA ONCOLOGY INC

Explicit Guidance:

• No formal financial guidance (revenue, earnings, margins, capex) provided.
• Clinical Development Timeline: Aiming to initiate First-in-Human (FIH) studies in Healthy Volunteers in the "next year" (FY2026).
• Data Disclosure: Anticipates potential data disclosure in late 2028 to early 2029.
• Financial Milestone: Secured $50 million in financing led by BDF Partners to support KO7246 through IND-enabling development, initial clinical proof-of-concept, and the advancement of a second menin inhibitor candidate.

Business-Segment Outlook:

• Corporate Structure: Caspian Therapeutics established as a dedicated entity to develop menin inhibitors for diabetes; Cura Oncology retains ~50% ownership and contributes IP/infrastructure via intercompany services.
• Pipeline Focus: Primary focus on KO7246 (oral menin inhibitor) for Type 1 and Type 2 diabetes to restore endogenous insulin-producing capacity.
• Second Candidate: Financing will support the advancement of a second menin inhibitor candidate beyond KO7246.
• Target Population: Patient selection will prioritize individuals with residual beta cell capacity (measured via C-peptide), targeting the "long tail" of Type 1 diabetes and stable Type 2 patients.

Market and Competitive Outlook:

• Mechanism Differentiation: KO7246 demonstrates ~3x greater potency than balamenib (ICOBAMEN/BMF219) in vitro and superior selectivity for beta cells without stimulating non-beta cell proliferation (unlike harmine/DERP1A inhibitors).
• Competitive Positioning: Designed to be complementary to GLP-1 receptor agonists rather than a direct competitor; leverages potential synergies where KO7246 expands beta cell mass while GLP-1s improve cell function.
• Unmet Need: Addresses a gap for therapies capable of restoring endogenous beta cell function and physiologic insulin response, a capability not currently available in the standard of care.
• Dosing Strategy: Intent to determine optimal dosing paradigm (continuous vs. intermittent) based on FIH exposure-response data; potential for a large therapeutic window similar to established menin inhibitors in oncology.

Macro / Industry Outlook:

• Safety Profile: Preclinical and clinical experience with menin inhibitors in oncology (AML) suggests a clean safety profile suitable for chronic metabolic disease use, distinct from class peers reporting liver toxicity (potentially due to non-Menin targets).
• Regulatory Path: Reliance on C-peptide levels as a primary biomarker for proof-of-concept; regulatory strategy involves demonstrating preservation or enhancement of endogenous insulin production compared to baseline.

Strategic Initiatives:

• Clinical Entry: Advancing KO7246 through IND-enabling studies (toxicology, exposure, manufacturing) to support initial clinical program.
• Team Building: Caspian leadership (led by COO Rob Spencer) is establishing dedicated resources for diabetes development while leveraging Cura's existing infrastructure.
• Combination Potential: Exploring potential future combinations with agents that modulate autoimmunity for Type 1 diabetes patients to protect newly generated beta cells.
• Future Indications: Exploring other chronic metabolic diseases and additional beta-cell specific applications beyond T1D and T2D, though specific indications remain undisclosed.

Risks and Headwinds to Outlook:

• Biology Thresholds: Treatment efficacy depends on a threshold of remaining beta cells; patients with insufficient baseline mass may not respond (observed in STZ rat models as "non-responders").
• Variable Response Rates: Anticipated variability in response timing among patients due to differences in starting beta cell mass; potential for a binary response (full recovery vs. no response) in early stages.
• Autoimmunity Challenges: In Type 1 diabetes, newly generated beta cells remain vulnerable to the underlying autoimmune destruction, potentially requiring combination therapies to sustain efficacy.
• Unknowns in Human Translation: Preclinical rodent models show rapid beta cell proliferation (e.g., 100% glucose control in 8 weeks); human translation speed and magnitude remain uncertain.
• Data Privacy: Specific experimental designs for patient subtypes to demonstrate unique efficacy are currently proprietary and not disclosed.

Management Tone on the Future:

• Confidence Level: High confidence in the scientific hypothesis (beta cell regeneration) supported by consistent preclinical data across mouse, rat, and human islet models.
• Caution: Explicitly states it is "too early" to define the dosing paradigm (continuous vs. intermittent) and emphasizes that safety is the primary constraint.
• Differentiation: Management asserts KO7246 has a distinct, cleaner pharmacologic profile compared to competitors (specifically highlighting the lack of CDK9 inhibition seen in ICOVAMENIB).
• Strategic Focus: Emphasizes a "dedicated investment" structure to ensure the diabetes program receives specialized leadership and resources separate from Cura's oncology focus.

Miscellaneous:

• Partnerships: Financing included participation from Eli Lilly & Company and the T1D Fund.
• Historical Context: Cura's oncology menin inhibitor experience (e.g., Ziftomenib) informs the safety design of KO7246, specifically removing off-target interactions (SIP system, HERG) and conducting full GLP-grade genotoxicity testing suitable for chronic use.
• Comparison to Competitors: KO7246 outperformed ICOVAMENIB in blocking the Menin-MLL interaction and showed efficacy in MENIN-dependent models where ICOVAMENIB failed.