Lexicon Pharmaceuticals (LXRX) investor relations material

Strategic focus and recent milestones

• Five key areas of focus include regulatory progress, clinical trials, partnerships, and new data generation.

• Zynquista resubmission for type 1 diabetes is progressing, with new safety data from an ongoing trial and FDA engagement on DKA event rates.

• SONATA phase III trial in HCM is enrolling, with data expected late next year or early 2027.

• LX9851, a preclinical obesity asset, was licensed to Novo Nordisk for over $1 billion in potential payments.

• INPEFA heart failure drug partnered with Viatris, with approvals and filings expanding internationally.

Zynquista regulatory pathway and clinical data

• FDA recognizes the need for glycemic control beyond insulin in type 1 diabetes and is open to non-traditional data sources for DKA risk.

• Efficacy for lowering A1C, reducing blood pressure, and severe hypo events is accepted; focus is on new DKA exposure data.

• Data from a large Danish study (Steno) is being used to address DKA concerns, saving significant time and cost.

• Agreement reached with FDA on exposure requirements and DKA event rate bands for resubmission.

• Approval could mark the first non-insulin agent for glycemic control in type 1 diabetes, addressing a major unmet need.

SONATA HCM program and broader implications

• SONATA is the pivotal registration trial for HCM, powered for a clinically meaningful KCCQ benefit.

• Complementary studies will inform combination therapy approaches, especially for non-obstructive HCM.

• Sotagliflozin's dual SGLT1/2 mechanism may offer unique benefits in HCM, including improved energetics and reduced diastolic dysfunction.

• Anti-stroke and anti-MI effects demonstrated in large studies are particularly relevant for HCM patients at risk of AFib.