Executive Summary

Q3 2024 was a pivotal setup quarter focused on Phase 3 MIRACLE trial readiness; Moleculin reiterated first dosing in Q1 2025 and later amended the protocol to allow an earlier unblinding at 45 subjects in H2 2025, accelerating visibility into primary efficacy and safety, a potential stock catalyst .
Financially, net loss widened to $(10.6)M with EPS of $(2.85), driven by higher R&D (annamycin production and sponsored research) and warrant-related non-cash/transaction items; cash ended at $9.4M with runway into Q1 2025 .
Clinical KPIs continued to improve: median durability of CRc exceeded 8 months; overall median survival now over 7 months; 4 of 9 responders have been bridged to transplant—key de-risking indicators for Phase 3 .
Management emphasized derisked efficacy vs HiDAC and adaptive design/early unblinding under Project Optimus; tone was confident and execution-focused, with global site engagement to accelerate recruitment .
Consensus estimates from S&P Global were unavailable for EPS/Revenue, limiting quantitative “beat/miss” comparisons; investor focus should center on trial milestones and financing cadence rather than near-term P&L metrics. Values retrieved from S&P Global unavailable.

What Went Well and What Went Wrong

What Went Well
- “Median durability [of CRc] continuing to climb – and is now in excess of 8 months,” reinforcing durability and quality of responses in MB-106 and supporting Phase 3 design .
- FDA-endorsed adaptive Phase 3 design with early unblinding reduces traditional efficacy risk and improves visibility for investors and partners; management views AnnAraC efficacy as “almost 3x greater than HiDAC alone” in prior datasets, materially derisking the endpoint .
- Protocol amendment to unblind preliminary primary efficacy and safety at 45 subjects in H2 2025—“potentially less than 12 months away from definitive unblinded data”—a clear timeline catalyst .
What Went Wrong
- Net loss and EPS worsened QoQ, reflecting R&D scaling and warrant-related costs: Q3 net loss $(10.6)M vs $(4.3)M in Q2, with transaction costs and loss on issuance tied to warrants and a loss from change in fair value .
- Cash declined to $9.4M with runway only into Q1 2025, increasing near-term financing sensitivity despite the $5.5M upfront financing and potential $11M via milestone-linked warrants .
- S&P Global consensus estimates were unavailable, limiting visibility into Street expectations; investors must rely on clinical timelines and financing disclosures rather than beat/miss framing. Values retrieved from S&P Global unavailable.

Financial Results

Quarterly Financials (oldest → newest)

Metric	Q1 2024	Q2 2024	Q3 2024
Revenues ($USD Thousands)	$0	$0	$0
Net Loss ($USD Thousands)	$(4,970)	$(4,319)	$(10,592)
Diluted EPS ($)	$(2.02)	$(1.70)	$(2.85)
R&D Expense ($USD Thousands)	$4,252	$4,090	$4,932
G&A + D&A ($USD Thousands)	$2,425	$2,095	$2,203
Total Operating Expenses ($USD Thousands)	$6,677	$6,185	$7,135
Loss/(Gain) from Warrant FV ($USD Thousands)	$1,455 gain	$1,696 gain	$(1,728) loss
Transaction Costs Allocated to Warrant Liabilities ($USD Thousands)	-	-	$(993)
Loss on Issuance of Warrant Liabilities ($USD Thousands)	-	-	$(847)
Interest Income, net ($USD Thousands)	$241	$159	$102
Cash & Equivalents (Period End, $USD Thousands)	$16,824	$10,845	$9,405
Weighted Avg Shares	2,466,174	2,543,244	3,714,278

YoY Comparison (Q3)

Metric	Q3 2023	Q3 2024
R&D Expense ($USD Thousands)	$3,280	$4,932
G&A + D&A ($USD Thousands)	$2,667	$2,203
Total Operating Expenses ($USD Thousands)	$5,947	$7,135
Net Loss ($USD Thousands)	$(5,609)	$(10,592)
Diluted EPS ($)	$(2.82)	$(2.85)

KPIs (Clinical Efficacy Indicators)

KPI	Q1 2024	Q2 2024	Q3 2024
CR Rate (2nd-line, %)	50% (5/10)	43% (2nd/3rd line subset)	N/A
CRc Rate (2nd-line, %)	60% (6/10)	50% (2nd/3rd line subset)	N/A
CRc Rate (1st + 2nd-line combined, %)	62%	N/A	N/A
Median Durability of CRc (months)	4.9 and climbing	~7 and climbing	>8 and climbing
Overall Median Survival (months)	N/A	N/A	>7
Responders Bridged to BMT (count)	N/A	N/A	4 of 9

Notes: Q2 rates are reported for 2nd/3rd-line evaluable subset; Q3 emphasizes durability/OS rather than updated CR/CRc rates as per disclosures .

Segment breakdown: Not applicable (pre-revenue) .

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
First subject treated in MIRACLE	Q1 2025	Q1 2025	Q1 2025	Maintained
Interim unblinding (dose selection)	Mid 2026	Unblind n=75 (Part A)	Early unblinding at n=45 in H2 2025; additional unblinding n=30–45 in 2026	Accelerated visibility
Part B sample size	2026–2028	~240 total (often described as 120/arm)	~244 total (statistical “cost” of added unblinding)	Increased
Recruitment update checkpoint	Q4 2025	n=40	n=45	Increased
Cash runway	Through Q1 2025	Into Q4 2024 cash; with planned $12M raise into Q2 2025	Into Q1 2025; plus $5.5M upfront financing and up to $11.0M via milestone-linked warrants	Improved vs Q2 cash-only

No revenue/OpEx/tax guidance provided; primary guidance centers on clinical milestones and financing disclosures .

Earnings Call Themes & Trends

Topic	Previous Mentions (Q1 & Q2)	Current Period (Q3)	Trend
Adaptive design / Project Optimus	Planning for pivotal adaptive Phase 3 post EOP meeting	Emphasis on early unblinding to reduce risk and enhance visibility	Increasing transparency
Efficacy vs HiDAC	Strong CR/CRc rates reported (Q1/Q2)	Management frames AnnAraC efficacy ~3x HiDAC; endpoint derisking	Confidence rising
Recruitment & global sites	Contracting to begin in 2H’24	~60 sites interested; leadership visiting high-potential sites globally	Execution focus
Early data visibility	Traditional unblinding at n=75 (prior)	Protocol amendment enables unblinding at n=45 in H2’25	Accelerated
SPA (Special Protocol Assessment)	Not discussed earlier	FDA advised against SPA to avoid delays; flexible protocol beneficial	Pragmatic approach
Financing strategy	Intent to raise $12M (S-1)	$5.5M upfront financing plus milestone-linked warrants; runway into Q1’25	Near-term runway secured
STS program	Ongoing Phase 1B/2; preclinical data positive	Likely partner pathway for pivotal; focus internal resources on MIRACLE	Partner-oriented
PK / Dose optimization	N/A	No correlation in PK between 190 and 230 mg/m²; supports dose selection	Dose flexibility

Management Commentary

“Median durability continuing to climb – and is now in excess of 8 months,” reinforcing durability and clinical quality .
“Our ability to unblind early because of this adaptive design means we won’t have to wonder whether our Phase III trial is tracking with endpoint expectations,” highlighting visibility and risk reduction .
“The amended protocol enables us to share definitive data earlier…potentially less than 12 months away from definitive unblinded data that could be a strong indicator of our likelihood of approval,” framing near-term catalysts .
“Annamycin…was designed to be 100% non-cardiotoxic…avoids cross-resistance…patent protected through 2040,” positioning competitive differentiation .

Q&A Highlights

OS update: Overall median survival now over 7 months, roughly ~2x literature expectations for this population; aligns with improving durability .
Trial sizing and unblinding: Clarification that 240 in Part B refers to 120 per arm; Part A subject count refined for statistical optimization; added early unblinding at 45 subjects .
R&D expense: Q3 included a “slug” of annamycin production and sponsored MD Anderson research, contributing to higher R&D .
Phase 3 cost: Run-rate refined by geography and site mix; indicative cost cadence discussed with sensitivity to region-specific per-patient costs .
SPA decision: FDA advised not to pursue SPA to avoid delays and maintain flexibility; alignment with FDA reduces need for SPA .
Partnering: STS pivotal likely via partner/external funding; internal focus on MIRACLE .
Share count/warrants: Discussion of fully diluted impacts as warrants move in-the-money .

Estimates Context

Wall Street consensus (S&P Global) for Q3 2024 EPS/Revenue was unavailable, preventing formal beat/miss analysis. Values retrieved from S&P Global unavailable.
Given pre-revenue status and R&D scaling, investor emphasis should be on cash runway, financing cadence, and MIRACLE trial milestones rather than near-term P&L comparisons .

Key Takeaways for Investors

Protocol amendment enabling early unblinding at 45 subjects in H2 2025 is a major visibility catalyst that can accelerate partner discussions and partially de-risk financing; monitor IRB approval and site activation pace .
Clinical durability and OS trends continue to improve, with multiple responders bridged to transplant—key efficacy quality indicators that support AnnAraC’s differentiation vs HiDAC .
Near-term financing sensitivity persists: cash into Q1 2025; track warrant exercises, potential equity raises, and geographic site mix (U.S. vs. Eastern Europe/Western Asia) given per-patient cost differentials .
Expect trial recruitment updates Q4 2025 (n=45) and preliminary unblinded efficacy/safety readouts thereafter—trading setups likely around these milestones .
STS program likely advances via partnering—optional upside with reduced internal capital burden; monitor 2025 MB-107 final data readout .
Non-cash warrant liabilities and transaction costs can add P&L volatility; interpret EPS trends alongside operating drivers (R&D production and sponsored research) .
Management’s tone and engagement (global site visits, KOL support) remain confident and execution-driven—focus on recruitment velocity and dose optimization outcomes under Project Optimus .

Moleculin Biotech, Inc. (MBRX)·Q3 2024 Earnings Summary