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Regulus Therapeutics Inc. (RGLS)·Q4 2024 Earnings Summary

Executive Summary

  • Q4 2024 delivered continued clinical execution and regulatory progress: net loss was $12.8M (–$0.20 per share) on total operating expenses of $13.7M; year-end cash, cash equivalents and short-term investments were $75.8M, with runway into early 2026 .
  • Positive interim data from Cohort 4 (14 of 26 subjects) of farabursen (RGLS8429) showed biomarker (PC1/PC2) increases with mechanistic dose response and data suggesting a reduction in htTKV growth rate after 3 months; complete safety from all 26 subjects showed the drug was well-tolerated .
  • The company reached alignment with FDA on key Phase 3 single-pivotal trial components, including a 12-month htTKV interim endpoint for potential Accelerated Approval and a 24-month eGFR endpoint for potential Full Approval—an important de-risking milestone .
  • Near-term catalysts: topline Cohort 4 data from all 26 subjects “in the coming weeks,” and advancing toward the Phase 3 design agreed with FDA; these are likely stock drivers for a development-stage biotech without revenue .
  • Street consensus from S&P Global was unavailable in our system for RGLS this quarter; therefore, we cannot perform a beat/miss analysis versus estimates (we will monitor for mapping updates) [GetEstimates attempt – no values returned].

What Went Well and What Went Wrong

What Went Well

  • Positive Cohort 4 interim readout: “we continued to see an encouraging safety and tolerability profile and evidence of a mechanistic dose response through increases in polycystin biomarker levels as well as results suggesting a reduction on kidney volume (htTKV) growth rate” (CEO) .
  • Regulatory de-risking: alignment with FDA on a single pivotal Phase 3 design with a 12-month htTKV interim for potential Accelerated Approval and a 24-month eGFR endpoint for potential Full Approval .
  • Solid liquidity for execution: $75.8M in cash, cash equivalents and ST investments at 12/31/24; runway into early 2026, enabling continued development through key milestones .

What Went Wrong

  • Operating expenses rose materially year over year (R&D $9.7M vs $5.8M; G&A $4.1M vs $2.5M), widening the net loss to $12.8M vs $8.1M in Q4 2023 .
  • Sequentially, while OpEx moderated vs Q3 (Q4 total OpEx $13.7M vs Q3 $15.2M), the company remains loss-making with no reported revenue lines, keeping dependence on external capital a risk factor .
  • Share count is substantially higher YoY (65.5M vs 20.2M weighted average), reflecting dilution typical of development-stage biotech financing cycles and pressuring per-share metrics .

Financial Results

P&L and Operating Metrics (USD $000s)

Metric (USD $000s unless noted)Q2 2024Q3 2024Q4 2024
R&D Expense8,309 11,347 9,672
G&A Expense3,951 3,863 4,074
Total Operating Expenses12,260 15,210 13,746
Other Income, net1,225 1,148 955
Net Loss(11,036) (14,062) (12,791)
Diluted EPS ($)(0.17) (0.21) (0.20)
Weighted Avg Shares64,465,185 65,471,132 65,499,573

Cash and Investments

MetricQ2 2024Q3 2024Q4 2024
Cash, Cash Equivalents & ST Investments ($MM)$95.9 $87.3 $75.8

Q4 YoY Comparison (USD $000s unless noted)

MetricQ4 2024Q4 2023
R&D Expense9,672 5,762
G&A Expense4,074 2,538
Total Operating Expenses13,746 8,300
Other Income, net955 239
Net Loss(12,791) (8,061)
Diluted EPS ($)(0.20) (0.40)
Weighted Avg Shares65,499,573 20,222,111

Notes: Company materials present operating lines; there is no reported product/collaboration revenue in these press releases/8-K summaries .

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Cash RunwayMulti-yearRunway into H1 2026 Runway into early 2026 Maintained (minor wording change)
Cohort 4 Topline (farabursen)Near-termEarly 2025 topline from a substantial number of patients Topline from all 26 subjects “in the coming weeks” Refined/accelerated clarity
Phase 3 Path (farabursen)2025+On track for End-of-Phase 1 meeting by YE 2024 FDA alignment reached on single pivotal design; 12-mo htTKV interim for potential AA; 24-mo eGFR for Full Approval; 2:1 randomization; EOW dosing Achieved regulatory alignment

Earnings Call Themes & Trends

Note: A Q4 2024 earnings call transcript was not available in our source; themes below reflect disclosures across Q2–Q4 company materials.

TopicPrevious Mentions (Q-2 and Q-1)Current Period (Q4 2024)Trend
R&D Execution (farabursen)Advanced to Cohort 4; Cohort 3 showed stronger PC1/PC2 response and 70% of patients with htTKV reductions; no safety concerns Cohort 4 interim (14/26) confirmed mechanistic biomarker response; suggested reduced htTKV growth; safety well-tolerated across all 26 Improving clinical evidence
Regulatory PathwayPlan/request for End-of-Phase 1 meeting in Q4 2024; path toward potential accelerated approval discussed Alignment achieved with FDA on single pivotal Phase 3, 12-mo htTKV interim AA and 24-mo eGFR for full approval Clarity increased
Biomarkers & ImagingDose-responsive PC1/PC2; exploratory htTKV reductions at 2–3 mg/kg Continued PC1/PC2 increases; signal on htTKV growth reduction after 3 months at 300 mg fixed dose Consistent to improving
Safety ProfileWell-tolerated; no safety concerns in Cohort 3 Well-tolerated across all 26 subjects in Cohort 4 Consistently favorable
Liquidity/RunwayCash runway into H1 2026 $75.8M at 12/31/24; runway into early 2026 Stable runway

Management Commentary

  • “Following the rapid progress in 2024 advancing farabursen for ADPKD, we recently announced interim topline data from 14 subjects in the fourth cohort… encouraging safety and tolerability… mechanistic dose response… results suggesting a reduction on kidney volume (htTKV) growth rate” — Jay Hagan, CEO .
  • “We have reached alignment with FDA on the key components of the design of a Phase 3 single pivotal trial. The proposed pivotal trial includes an interim analysis to enable Accelerated Approval based on a 12-month htTKV endpoint…” — Jay Hagan, CEO .

Q&A Highlights

  • A Q4 2024 earnings call transcript was not available in the document set; no Q&A highlights or clarifications could be sourced from a call transcript for this period [ListDocuments showed no Q4 2024 transcript; none retrieved].

Estimates Context

  • S&P Global consensus estimates for revenue and EPS were unavailable in our system for RGLS this quarter; as a result, we cannot compare reported results to Street expectations and cannot identify beats/misses vs consensus. We will monitor for availability in future periods.

KPIs (Clinical and Operating)

KPIQ2 2024Q3 2024Q4 2024
Cohort 4 enrollment (farabursen)Initiated; 300 mg fixed dose EOW for 3 months Completed enrollment of 26 patients 26 patients dosed; interim analysis of 14 patients
Biomarker response (PC1/PC2)Cohort 3: significant PC1/PC2 increases vs placebo; dose-responsive Continued evidence of dose response Continued PC1/PC2 increases; mechanistic dose response
Imaging (htTKV)70% with reductions post 3 mg/kg in Cohort 3; exploratory Exploratory reductions highlighted at ASN poster Suggested reduction in htTKV growth rate after 3 months
SafetyWell-tolerated; no safety concerns (Cohort 3) —Well-tolerated across all 26 in Cohort 4
Cash runwayInto H1 2026 Into H1 2026 Into early 2026

Key Takeaways for Investors

  • Regulatory clarity is a major de-risking event: FDA alignment on a single pivotal Phase 3 with a 12-month htTKV interim for potential Accelerated Approval materially sharpens the development path and potential timelines .
  • Clinical signals are tracking in the right direction: Cohort 4 interim data reinforced dose-responsive biomarker improvements and suggested htTKV growth rate reduction, with a favorable safety profile across all 26 subjects—key for a chronic kidney indication .
  • Near-term catalyst density remains high: topline from all 26 Cohort 4 subjects “in the coming weeks,” plus movement toward the agreed Phase 3 design could be stock-moving events .
  • Liquidity supports execution through milestones: $75.8M cash at year-end and runway into early 2026 provide capacity to progress into pivotal-stage activities; watch for future financing needs as Phase 3 begins .
  • Expense growth reflects pipeline momentum but keeps losses elevated: YoY OpEx increases widened the net loss; this is typical for development-stage biotechs but underscores funding dependence in absence of revenue .
  • No Street estimate comparison this quarter: S&P Global consensus data were unavailable in our system; maintain focus on clinical/regulatory catalysts as the primary drivers pending future estimates availability.

Sources: Q4 2024 8-K/press release and exhibit (financials and program updates) ; Q3 2024 8-K/press release ; Q2 2024 8-K/press release ; press release on Cohort 4 enrollment completion .