Regulus Therapeutics Inc. (RGLS)·Q1 2025 Earnings Summary

Executive Summary

• Regulus reported Q1 2025 net loss of $9.6M (EPS -$0.15), driven by R&D of $6.8M and G&A of $3.7M; cash, cash equivalents and short-term investments were $65.4M, with runway into early 2026 .
• Company advanced farabursen (RGLS8429) with full Cohort 4 topline data showing mechanistic biomarker response and mean halting of htTKV growth over 4 months; Phase 3 pivotal trial initiation targeted for Q3 2025 with FDA alignment on accelerated approval endpoint (12‑month htTKV) and full approval endpoint (24‑month eGFR) .
• Regulus entered a definitive agreement to be acquired by Novartis for $7.00 per share upfront plus a $7.00 CVR tied to regulatory approval of farabursen (total potential equity value ≈$1.7B); closing expected in H2 2025, subject to conditions .
• Wall Street consensus (S&P Global) for Q1 2025 was unavailable via our tool; therefore, estimate comparisons to SPGI consensus cannot be provided. Values retrieved from S&P Global were unavailable due to CIQ mapping limitations.

What Went Well and What Went Wrong

What Went Well

• Positive Cohort 4 efficacy signals: biomarker response (PC1/PC2) similar to prior cohort and mean htTKV growth halted over 4 months; exploratory pool across high-dose cohorts showed htTKV reduction versus large historical placebo group (p=0.0056) .
• FDA alignment on Phase 3 pivotal design including accelerated approval path via 12‑month htTKV and full approval via 24‑month eGFR; single active dose and 2:1 randomization confirmed .
• Strategic outcome: signed merger agreement with Novartis, potentially providing scale and global capabilities to bring farabursen to ADPKD patients; boards of both companies unanimously approved .
  • “We’ve made important progress... results suggesting that kidney volume growth rate was halted after only a relatively short treatment period. We look forward to... Phase 3... in the third quarter of this year.” — CEO Jay Hagan .
  • “Results give us confidence that 300 mg provides optimal target exposure... repeated demonstration... growth of the kidney... is halted...” — President/Head of R&D Preston Klassen, M.D. .

What Went Wrong

• Net loss increased year-over-year: Q1 2025 net loss $9.6M vs $8.5M in Q1 2024, reflecting higher R&D and G&A to advance programs .
• Sequential cash burn: cash decreased to $65.4M at 3/31/2025 from $75.8M at 12/31/2024, though runway still extends into early 2026 .
• Transaction and regulatory uncertainty: tender offer and merger subject to customary conditions and regulatory approvals; CVR milestone risk; potential operational disruption highlighted in forward-looking disclosures .

Financial Results

Key P&L, EPS, and Cash Metrics (Oldest → Newest)

Notes:

• Revenue not disclosed in press releases; company reports operating expenses and net loss. No product revenue indicated in Q1 materials .

Clinical KPIs (Cohort 4 and High-Dose Pool)

Guidance Changes

Earnings Call Themes & Trends

Note: No Q1 2025 earnings call transcript available in our document repository; themes from press releases and 8‑K filings .

Management Commentary

• CEO Jay Hagan: “We’ve made important progress... evidence of a mechanistic dose response... results suggesting that kidney volume growth rate was halted... heading towards initiation of the pivotal Phase 3 trial in the third quarter of this year.”
• President/Head of R&D Preston Klassen, M.D.: “Results... indicate maximal anti‑miR‑17 activity... repeated demonstration... kidney growth is halted... 300 mg... appropriate dose to take forward into Phase 3... htTKV effects... clinically meaningful...” .
• Novartis CMO Shreeram Aradhye: Farabursen “represents a potential first‑in‑class medicine... enhanced efficacy, tolerability and safety versus standard of care... aim to bring a better treatment option to patients” .

Q&A Highlights

• No Q1 2025 earnings call transcript found; Q&A themes and any guidance clarifications not available in our source set [Search attempt returned none].

Estimates Context

• S&P Global/Capital IQ consensus for Q1 2025 EPS and revenue was unavailable due to SPGI mapping limitations for RGLS; as a result, we cannot provide SPGI-based comparisons. Values retrieved from S&P Global were unavailable due to CIQ mapping limitations.

Key Takeaways for Investors

• Cohort 4 data strengthens the mechanistic and exploratory efficacy case for farabursen, with significant biomarker improvements and halted htTKV growth over a short period; supports dose selection and de-risks Phase 3 endpoints .
• FDA alignment on accelerated approval (12‑month htTKV) and full approval (24‑month eGFR) reduces regulatory uncertainty around Phase 3 design and increases program visibility into H2 2025‑2026 milestones .
• Cash runway into early 2026 provides operational flexibility through Phase 3 initiation; sequential cash usage is consistent with clinical advancement pace .
• The Novartis transaction adds a strategic path to commercialization, subject to closing conditions and CVR milestone achievement; potential upside via CVR contingent on regulatory approval of farabursen .
• Near-term catalysts: Phase 3 trial launch in Q3 2025 and ongoing regulatory interactions; potential tender offer and merger progress updates in H2 2025 .
• Risk factors remain: tender offer completion, regulatory approvals, CVR achievement, and typical development risks per forward-looking statements; monitor for any negative data updates or timeline shifts .
• With no product revenue and ongoing R&D investment, valuation hinges on clinical/regulatory milestones and the merger timeline; updates on Phase 3 enrollment and interim analyses will be stock drivers .