LB Pharmaceuticals (LBRX) 3rd Annual Guggenheim Healthcare Innovation Conference summary

Strategy and milestones

• Vision: build a fully integrated neuromedicines business; LB-102 is the lead asset, supported by decades of clinical and real-world validation for amisulpride outside the U.S.

• NOVA 1 showed statistically significant results across doses; FDA discussions indicate a potential registration path requiring one additional Phase III study.

• NOVA 2 schizophrenia data expected H1 2027; bipolar depression Phase II data expected Q1 2028; planned MDD Phase II data expected H1 2029.

• Cash runway extends beyond Q2 2029 and covers the clinical milestones, plus development of a long-acting injectable and exploration of negative symptoms and Alzheimer’s disease.

LB-102 profile and differentiation

• Methylation of amisulpride improved lipophilicity and brain penetration; 50 mg LB-102 corresponds to ~400 mg amisulpride at ~70% receptor occupancy and enables once-daily dosing.

• NOVA 1 showed competitive efficacy and a potentially differentiated tolerability profile, including low extrapyramidal symptoms (EPS), negligible sedation and few GI effects.

• EPS rates in NOVA 1 were <1% at 50 mg and 5.6% at 100 mg, versus 3.7% for placebo; preclinical findings suggest selective activity in the associative rather than motor striatum.

• No titration, once-daily dosing, no food effect and few drug interactions are intended to support adherence; negative symptoms and cognition could provide additional benefit.

Phase III and regulatory plans

• NOVA 2 is designed similarly to NOVA 1, with three arms instead of four; placebo controls include experienced sites, central raters, two vendors to screen professional patients and real-time study monitoring.

• Success criteria emphasize a statistically robust result, competitive efficacy and a safety profile consistent with NOVA 1; two doses offer physicians flexibility to start low and escalate if needed.

• NOVA 2 readout is the primary gate for a pre-NDA meeting planned for H2 2027; discussions will align FDA on submission scope and timing, including long-term safety data.

• Composition patents extend into the early 2040s, including patent term extension.