LB Pharmaceuticals (LBRX) investor relations material

Key milestones and recent achievements

• Successfully completed IPO in a challenging market, securing around $300 million in net proceeds, providing capital through Q2 2028 for key clinical programs.

• Initiated preparations for phase III schizophrenia trial, targeting Q1 2026 start, with all sites and drug supply being readied post-IPO.

• Increased phase III sample size from 400 to 460 to boost statistical power from 80% to 85%, with no impact on timelines or site numbers.

• Top-line data for phase III schizophrenia trial expected in the first half of 2027.

• Phase II and III trials share similar design, sites, and geography, with phase III extending duration to six weeks and reducing arms to manage placebo rates.

Clinical development strategy and regulatory pathway

• Phase III trial powered for a PANSS delta of six, with assumptions aligned to prior schizophrenia trials and more conservative than phase II.

• FDA confirmed phase II trial was registrational in quality, enabling a streamlined path to approval with one successful phase III trial.

• Open-label safety study planned to accrue 1,500 patient exposures, with NDA-enabling studies running in parallel for rapid submission post-phase III.

• Blinded standard deviation assessment may be conducted during phase III, with details to be disclosed as the trial progresses.

Product differentiation and market positioning

• LB-102, a methylated derivative of amisulpride, offers improved brain penetration, once-daily dosing, and potentially better tolerability.

• Demonstrated competitive efficacy and a best-in-class safety profile, with very low rates of EPS compared to leading antipsychotics.

• Phase II data showed significant benefits in cognition and negative symptoms, supporting further exploration in these domains.

• Amisulpride never approved in the U.S. due to IP and regulatory hurdles, despite global use and physician interest.