Lipella Pharmaceuticals (LIPO) investor relations material

Corporate strategy and technology

• Proprietary multi-lamellar liposomal technology enables targeted drug delivery to mucosal tissues, minimizing systemic exposure and enhancing safety and efficacy.

• Focus on serious conditions lacking adequate treatments, leveraging the 505(b)(2) regulatory pathway for efficient clinical development.

• Asset pipeline includes two phase two assets, one IND-approved asset, and one preclinical asset, all using liposomal formulations.

• Lead asset, LP-10 oral rinse (LP-310), targets oral lichen planus (OLP), a T-cell-mediated autoimmune disorder with no approved therapies.

Clinical trial results and safety

• Phase two multi-center dose escalation study involved 27 adults with moderate to severe OLP, using LP-10 oral rinse twice daily for four weeks.

• No serious adverse events or discontinuations; most adverse events were mild to moderate, with minimal systemic absorption of tacrolimus.

• 76% of subjects had undetectable blood levels of tacrolimus; no cases of hypertension, renal impairment, or allergic reactions.

• Over 1,500 doses were well tolerated, with no intractable pain or significant side effects.

Efficacy and patient outcomes

• Statistically significant improvements in clinician-assessed and patient-reported outcomes, including pain, sensitivity, and symptom severity, observed as early as week one and sustained through week six.

• All participants reported some improvement, with most noting moderate to much improvement by week four.

• Three patients who failed conventional steroid rinses improved and received expanded use access from the FDA.