Assembly Biosciences (ASMB) investor relations material

Company overview and strategic focus

• Focused on HSV-2 (recurrent genital herpes), hepatitis delta, and recently expanded into cholestatic liver diseases (PBC, PSC).

• Lead HSV-2 compounds (5366 and 1179) are helicase primase inhibitors targeting high recurrence cases.

• Long-term partnership with Gilead, which has opt-in rights and recently exercised them for the HSV-2 program.

• Pipeline includes small molecule antivirals and liver disease therapies, leveraging expertise from prior antiviral work.

• Expansion into liver diseases driven by mechanistic overlap and clinical opportunity.

Recent data and clinical validation

• Four phase I clinical trials completed last year, including HBV and hep delta programs.

• HSV-2 compounds showed over 95% reduction in viral shedding and over 90% lesion reduction in phase I-B.

• Gilead opted into the HSV-2 program early, before phase I-B completion, based on strong data.

• Hep delta 6250 demonstrated target engagement and safety in phase I-A, supporting advancement to phase II.

• HBV compound being partnered out to focus on cure strategies.

Competitive positioning and differentiation

• HSV-2 compounds demonstrated higher potency and efficacy compared to existing helicase-primase inhibitors and Valtrex.

• Once-weekly or monthly oral dosing planned, improving convenience over current therapies.

• 6250 is a small molecule NTCP inhibitor, aiming to match or exceed efficacy of bulevirtide with oral dosing.

• 6250 showed strong PK, safety, and pharmacodynamic markers in phase I-A, with no pruritus observed.

• Differentiation in cholestatic liver disease by targeting NTCP, offering potential additive or standalone benefits over PPARs and IBAT inhibitors.