Regulus Therapeutics (RGLS) investor relations material

Executive summary

• Entered into a definitive merger agreement with Novartis AG for $7.00 per share in cash plus a $7.00 CVR, for total equity value up to $1.7 billion, pending regulatory and shareholder approval, expected to close in H2 2025.

• Farabursen (RGLS8429) for ADPKD advanced through Phase 1b with positive topline results across all cohorts, showing mechanistic dose response and halted kidney volume growth, with constructive FDA feedback on an accelerated approval pathway.

• On track to initiate a pivotal Phase 3 trial for farabursen in Q3 2025, following FDA alignment on trial design and endpoints.

• Cash, cash equivalents, and short-term investments totaled $65.4 million as of March 31, 2025, expected to fund operations through Q1 2026.

• Net loss increased to $9.6 million for Q1 2025, up from $8.5 million in Q1 2024, driven by higher R&D and G&A expenses.

Financial highlights

• Q1 2025 research and development expenses were $6.8 million, up from $6.0 million in Q1 2024, mainly due to clinical advancement of the ADPKD program and increased stock-based compensation.

• General and administrative expenses rose to $3.7 million, a 33% increase year-over-year, primarily from higher stock-based compensation.

• Interest and other income was $0.9 million, up from $0.4 million in Q1 2024, reflecting higher interest income.

• Net cash used in operating activities was $10.6 million for Q1 2025, compared to $8.7 million in Q1 2024.

• No revenue from product sales; accumulated deficit reached $569.2 million as of March 31, 2025.

Outlook and guidance

• Existing cash resources expected to fund operations through Q1 2026; additional capital will be required if the Novartis merger is not completed.

• If the merger fails, the company may need to scale back operations, seek additional financing, or consider strategic alternatives.

• Phase 3 pivotal trial for farabursen in ADPKD planned to start in Q3 2025, with a 12-month htTKV endpoint for Accelerated Approval and a 24-month eGFR endpoint for Full Approval.

• Farabursen is positioned for a pivotal Phase 3 trial, with potential for accelerated approval based on FDA feedback.