Executive Summary

Q2 2024 was defined by clinical progress in ADPKD: cohort 3 of RGLS8429 showed statistically significant increases in urinary PC1/PC2 versus placebo and 70% of subjects had reductions in htTKV after three months, with no safety concerns .
Operating spend ramped as programs advanced: Total OpEx rose to $12,260k from $8,826k in Q1 and $7,315k in Q2 2023; net loss increased to $11,036k ($0.17 per share) versus $8,469k ($0.29) in Q1 and $7,013k ($0.37) in Q2 2023 .
Liquidity remains adequate for execution: cash, cash equivalents, and investments were $95,929k at 6/30/24, with runway guided into H1 2026; company also announced addition to Russell 3000/2000 indexes, which can improve liquidity and visibility .
Near-term catalysts: request End-of-Phase 1 meeting with FDA in Q4 2024 and provide topline data for cohort 4 (300 mg fixed dose) in early 2025 .

What Went Well and What Went Wrong

What Went Well

RGLS8429 delivered meaningful signals: “Data from cohort three showed continued improvement on both polycystin and height adjusted total kidney volume (htTKV)…with greater mechanistic activity” and more patients showing reductions in kidney volume .
Clinical KPIs strengthened: statistically significant percent change from baseline in PC1 and PC2 at 3 mg/kg vs placebo and 70% of patients with htTKV reductions; safety was clean with no concerns .
Corporate positioning improved: addition to Russell 3000/2000 indexes and promotion of Rekha Garg, M.D., M.S., to Chief Medical Officer enhances institutional visibility and clinical leadership bench .

What Went Wrong

Higher OpEx driven by program advancement widened losses: Q2 R&D $8,309k (vs $6,040k in Q1; $4,976k in Q2’23) and G&A $3,951k (vs $2,786k in Q1; $2,339k in Q2’23) increased net loss to $11,036k .
Cash declined quarter-over-quarter as spend increased: cash/investments fell to $95,929k from $107,725k in Q1 (though runway remains into H1 2026) .
No revenue and limited ability to compare to Wall Street estimates: company is pre-revenue; S&P Global consensus mapping for RGLS was unavailable, limiting beat/miss analysis for EPS/revenue .

Financial Results

Income Statement (Quarterly comparison)

Metric (USD Thousands unless noted)	Q2 2023	Q4 2023	Q1 2024	Q2 2024
Research and Development ($USD Thousands)	4,976	5,762	6,040	8,309
General and Administrative ($USD Thousands)	2,339	2,538	2,786	3,951
Total Operating Expenses ($USD Thousands)	7,315	8,300	8,826	12,260
Other Income, net ($USD Thousands)	303	239	357	1,225
Net Loss ($USD Thousands)	7,013	8,061	8,469	11,036
Net Loss per Share ($USD)	$0.37	$0.40	$0.29	$0.17
Weighted Avg Shares (Units)	19,101,969	20,222,111	28,746,802	64,465,185

Notes: Revenue not reported; margins not applicable given pre-revenue status .

Balance Sheet (Quarter-end)

Metric (USD Thousands)	Q4 2023	Q1 2024	Q2 2024
Cash, Cash Equivalents & Investments	23,767	107,725	95,929
Total Assets	30,750	114,138	104,119
Term Loan, less debt issuance costs	1,334	540	—
Stockholders’ Equity	21,187	107,588	98,181

Clinical KPIs (Phase 1b MAD)

KPI	Cohort 2	Cohort 3	Notes
Subjects randomized (n)	14 (3:1 RGLS8429:placebo)	16 (3:1 RGLS8429:placebo)	Double-blind, QOW x 3 months
Dose	2 mg/kg	3 mg/kg	Fixed-dose cohort 4: 300 mg QOW x 3 months
Safety	Well tolerated; no concerns	Well tolerated; no concerns
Urinary PC1/PC2	Greater biological activity vs 1 mg/kg/placebo	Statistically significant increase vs placebo	PC1/PC2 inverse correlate with disease severity
htTKV	Reductions in patients with highest PC1/PC2 increases	70% of subjects showed reductions after 3 months	Exploratory MRI suggests reduction at 3 mg/kg

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
Cash Runway	Company	Into H1 2026	Into H1 2026	Maintained
End-of-Phase 1 FDA Meeting	Q4 2024	Accelerated approval pathway discussed with FDA; Type D meeting held (single pivotal Phase 2 with TKV endpoint; post-approval Phase 3 eGFR)	On track to request EOP1 meeting with FDA in Q4 2024; “meet with FDA near the end of this year”	Increased specificity
Cohort 3 Topline Timing	Mid-2024	Expected mid-2024	Achieved June 24, 2024	Achieved
Cohort 4 Status/Topline	2024–2025	Screening planned Q2 2024; sample size up to 30	Enrollment ongoing; topline data early 2025	Updated timeline
Russell Index Inclusion	2024	Expected addition announced June 4	Addition announced in Q2 release	Achieved

Earnings Call Themes & Trends

Topic	Previous Mentions (Q4 2023 & Q1 2024)	Current Period (Q2 2024)	Trend
R&D execution	Positive cohort 2 topline; completed cohort 3 enrollment; initiated cohort 4	Statistically significant PC1/PC2 at 3 mg/kg; 70% htTKV reductions; clean safety; cohort 4 enrolling	Strengthening
Regulatory/legal	FDA Type D meeting confirmed accelerated approval pathway framework	On track for EOP1 FDA meeting in Q4 2024	Progressing
Financing & liquidity	Closed oversubscribed $100M private placement; runway into H1 2026	Runway maintained; Russell 3000/2000 addition	Improved visibility/liquidity
Leadership	—	Promotion of Rekha Garg, M.D., to CMO	Strengthened
Clinical KPIs	PC1/PC2 increases correlated with htTKV reductions in highest responders	Broader consistency at 3 mg/kg; 70% htTKV reduction rate	Stronger signal

Management Commentary

“Data from cohort three showed continued improvement on both polycystin and height adjusted total kidney volume (htTKV)…with greater mechanistic activity…This growing data set leaves us excited about the potential of RGLS8429…We remain on track to meet with FDA near the end of this year.” — Jay Hagan, CEO .
“We are pleased to see the consistent mechanistic response and reduction in htTKV at the 3 mg/kg dose…These data support our ongoing and final 300 mg fixed dose cohort…basis for a potentially pivotal Phase 2/3 study under an Accelerated Approval regulatory pathway.” — Preston Klassen, M.D., President & Head of R&D .

Q&A Highlights

No Q2 2024 earnings call transcript was available in the document catalog; however, the company hosted a conference call on June 24, 2024 to discuss cohort 3 topline results .
Clarifications from press releases: cohort 4 topline expected in early 2025; EOP1 meeting planned in Q4 2024 .

Estimates Context

Wall Street consensus (S&P Global) for EPS, revenue, target price, and recommendation was unavailable due to missing CIQ mapping for RGLS in the S&P Global connector; as a result, beat/miss analysis versus estimates cannot be determined at this time. Values retrieved from S&P Global were unavailable.

Key Takeaways for Investors

Clinical momentum is building: statistically significant biomarker changes and 70% htTKV reductions at 3 mg/kg, with clean safety, strengthen probability of success in forthcoming fixed-dose cohort 4 and potential pivotal Phase 2/3 design under accelerated approval framework .
Execution runway looks adequate: $95,929k cash/investments at Q2-end and runway guided into H1 2026 support key milestones (EOP1 FDA meeting in Q4 2024, cohort 4 topline early 2025) without near-term financing needs .
Increased visibility and potential liquidity from Russell index inclusion may broaden the shareholder base and facilitate capital access if needed .
Near-term trading setup: watch for FDA EOP1 meeting request/acceptance and any feedback on pivotal trial endpoints (TKV and eGFR) as potential stock-moving events .
Expense trajectory: OpEx growth reflects deliberate investment in R&D; monitor quarterly burn and cash trends as cohort 4 scales (up to 30 patients) .
Regulatory path clarity: prior FDA dialogue supports accelerated approval with a single pivotal trial on TKV growth; post-approval eGFR requirement should be factored into timelines and probabilities .
Estimate benchmarking gap: with S&P Global consensus unavailable, rely on operational milestones and cash runway progression for near-term assessment; update beat/miss analytics when mapping is restored.

Sources: Q2 2024 8-K and press release (financials and program updates) ; Cohort 3 topline press release and 8-K ; Q1 2024 8-K/press release ; Q4 2023 8-K ; Russell index inclusion .

Regulus Therapeutics Inc. (RGLS)·Q2 2024 Earnings Summary