GUGGENHEIM 3RD ANNUAL HEALTHCARE INNOVATION CONFERENCE - LISATA THERAPEUTICS INC

Explicit Guidance:

• No explicit quantitative financial guidance (revenue, earnings, margins, or capex) provided for any future period.
• Capital runway: The $225 million private placement funds operations through the middle of 2028.
• Catalyst timing:
  • End of next year (2027): Data from Phase 2b (SHGTG) to support dose selection for the half-life extended candidate; interim Phase 2 Acromegaly data; full Phase 2 Acromegaly proof-of-concept data.
  • 2027 (Start): Initiation of two Phase 3 programs (SHGTG and Acromegaly).

Business-Segment Outlook:

• SHGTG (MARS001):
  • Expected efficacy in target patients (triglycerides >500 mg/dL) to remain in the 68% reduction range (observed 60–80% in study subsets) based on current trends, comparable to APOC3 class.
  • Phase 2b is a "pure SHGTG study" with the primary goal of confirming the scaled efficacy profile and supporting Phase 3 dosing.
  • Future expansion opportunities include mixed hypertriglyceridemia, remnant cholesterol, and ASCVD indications via partnership.
  • Next-generation pipeline: An siRNA adipose-directed version of the ANGPTL4 antibody is in development, potentially partnered for broader indications.
• Acromegaly (Next-gen GH receptor antagonist):
  • Targeting the ~20,000 uncontrolled patients (2/3 of the 30,000 US patient pool) who do not respond to current Somatostatin Receptor Ligands (SRLs).
  • Goal to match or exceed the efficacy of Pfizer's Pegvisomant (>80% control in trials) with a biweekly subcutaneous auto-injector dosing regimen.
  • Phase 2 study design includes 10-week endpoints, bracketing dose levels, and assessment in both monotherapy and add-on to SRLs.
  • Market opportunity: Potential to address double the addressable patient count of the current SRL class ($1 billion+ U.S. market projection), contingent on increased diagnosis rates.

Market and Competitive Outlook:

• Leveraging Market Building: Anticipates benefiting from market education and patient identification efforts by competitors Ionis (Lekvio) and Arrowhead (expected next year), similar to Lekvio's projected $5 billion trajectory.
• Differentiation Strategy: Positioned to capture the majority of new starts among diabetic patients, who are currently the primary population for SHGTG but represent an unmet need due to glycemic worsening risks with APOC3 inhibitors.
• Physician Demand: Early market research indicates strong physician preference for the NGPTL4 mechanism over APOC3 for diabetic patients due to the lack of dysglycemia and liver fat signals.
• Competition:
  • APOC3 class: Acknowledged as "groundbreaking" but limited by class effects on glucose and liver fat.
  • Acromegaly: Differentiates from existing SRLs (low response rate ~33%) and Pegvisomant (daily dosing, LFTs, injection site reactions).

Macro / Industry Outlook:

• Diabetes Prevalence: Management notes that 60–70% of severe hypertriglyceridemia patients have established diabetes or are pre-diabetic, driving the demand for non-glycemic-worsening therapies.
• Regulatory/Commercial: Anticipates broad coverage for the eventual label; no specific macroeconomic or FX headwinds mentioned.
• Trends: Expectations for increased diagnosis and awareness in the acromegaly space (currently ~30k US patients, often underdiagnosed) driven by competitors like Vertex and Krenetics.

Strategic Initiatives:

• Pipeline Progression: Transitioning from Phase 1/2a validation to Phase 2b (SHGTG) and Phase 2 (Acromegaly) in 2027, with immediate follow-on Phase 3 initiation for both indications.
• Product Optimization: Developing a half-life extended version of the NGPTL4 antibody for Phase 3 to optimize dosing frequency.
• Capital Efficiency: Focusing on SHGTG as the primary near-term driver while maintaining a "laser-focused" approach despite being a small company, leveraging the $225M to de-risk two distinct asset lines.
• Partnerships: Open to partnering for next-generation siRNA programs or expansion into ASCVD indications where internal resources are insufficient.

Risks and Headwinds to Outlook:

• Data Variance: Phase 2b SHGTG results are based on small sample sizes (dozen patients with TG >500 mg/dL) in the current dataset; confidence relies on the strong correlation trend rather than statistical power.
• Timeline Compression: Management acknowledges the volume of catalysts (Phase 2 data, Phase 3 starts for two assets) in a short period, noting "it's a lot to do in a relatively short period."
• Competition: Reliance on competitors' success in market building; potential for Arrowhead or Ionis to capture significant share or dictate pricing dynamics.
• Development Uncertainty: Phase 2 Phase 3 transition risks inherent to first-in-class modalities; dosing frequency and safety in Phase 3 remain to be fully proven.

Management Tone on the Future:

• High Confidence: Describes the reverse merger position as "fortunate" with two molecules having "initial clinical validation."
• Optimism: Characterizes 2027 as a "big year" with "huge catalysts."
• Strategic Caution: Hedges confidence in the 70% efficacy claim by noting the small current numbers, but expresses strong belief in the mechanistic trend (scaled efficacy with baseline TG).
• Market Positioning: Views the current landscape as an opportunity to enter a "large group of patients" with a "differentiated profile" rather than competing head-on on the same metrics as established APOC3 drugs.

Miscellaneous:

• Ticker Status: Reference to the active ticker being "Lissata" at the time of the conference, transitioning to "Mireya Therapeutics."
• Mechanism Insight: NGPTL4's unique mechanism directs triglycerides to adipose tissue rather than the liver, hypothesized to avoid the ectopic fat deposition (liver fat, dysglycemia) seen with APOC3 inhibitors.
• Market Size Estimates: U.S. SHGTG market projected at over $10 million; U.S. Acromegaly SRL market projected at $1 billion.