Executive Summary

Q4 2024 results were typical of a pre-revenue biotech: no revenue, markedly lower quarterly net loss vs Q3 as warrant fair-value changes and lower OpEx benefited EPS; cash fell to $4.3M at year-end, with February financings adding $9.3M gross and extending runway into Q3 2025 .
Clinical execution advanced: first country approval in Europe, positive FDA guidance enabling ~10% reduction in Part B size, and an amended protocol to unblind primary efficacy at 45 subjects in H2 2025 .
Management reiterated MIRACLE Phase 3 startup timing (first subject targeted in Q1 2025) and highlighted multiple interim unblindings that could be catalysts in H2 2025 and H1 2026 .
Consensus for Q4 EPS was -$1.28; actual diluted EPS was approximately -$0.37, a significant beat driven by non-operating items and lower OpEx; revenue remained $0 as expected for development-stage biotech (Values retrieved from S&P Global)* [Q4 EPS est: GetEstimates].
Near-term stock drivers: site activation pace, first patient dosing, H2’25 unblinding (n=45) and STS MB-107 readout by end of April 2025 .

What Went Well and What Went Wrong

What Went Well

Positive FDA guidance on the IND amendment allowed a ~10% reduction in Part B size, accelerating timelines and modestly lowering trial scope .
First country approval (Ukraine) to begin recruiting for MIRACLE; multiple subjects being screened and additional ethics approvals (Georgia, Egypt) in progress .
Financing momentum: $5.8M warrant exercises and $3.5M registered direct, plus management’s 8-K disclosure of $9.3M gross proceeds in February, extending runway into Q3 2025 .
Quote: “Unlike most pivotal Phase 3 trials, we will have multiple unblinding of data… roughly thirty days after the forty fifth subject is treated, we will be able to see how the drug is performing” — Walter Klemp .

What Went Wrong

Cash balance declined to $4.3M at year-end, reflecting sustained OpEx; company still requires significant additional financing to fully conduct trials as described .
Management disclosed MIRACLE cost could reach $60–70M if fully executed; burn expected to rise from ~$5M/qtr in 2025 to $7–8M/qtr in 2026 as CMC ramps for NDA .
U.S. enrollment likely to lag other geographies given longer IRB/contracting cycles, potentially front-loading recruitment to Ukraine/Middle East/EU before U.S. sites are active .

Financial Results

Quarterly comparison vs prior periods and consensus

Metric	Q2 2024	Q3 2024	Q4 2024
Revenue ($USD Millions)	$0.00	$0.00	$0.00*
Net Income ($USD Millions)	$(4.32)	$(10.59)	$(1.88)*
Diluted EPS ($USD)	$(1.70)	$(2.85)	$(0.37)*
Total Operating Expenses ($USD Millions)	$6.19	$7.14	$6.64*
Cash and Cash Equivalents ($USD Millions)	$10.85	$9.41	$4.28

Q4 2024 vs Consensus (Wall Street/S&P Global): EPS consensus -$1.28 vs actual -$0.37 → bold beat; revenue consensus $0 → actual $0 (Values retrieved from S&P Global)* [Q4 estimates: GetEstimates].

Full-year comparison (FY 2024 vs FY 2023)

Metric	FY 2023	FY 2024
R&D Expense ($USD Millions)	$19.49	$17.73
G&A + D&A ($USD Millions)	$10.14	$8.91
Net Loss ($USD Millions)	$(29.77)	$(21.76)
Diluted EPS ($USD)	$(15.07)	$(6.32)
Cash and Cash Equivalents at Year-End ($USD Millions)	$23.55	$4.28

Notes: Moleculin is pre-revenue; margins are not meaningful with $0 revenue. Q4 improvements vs Q3 reflect lower OpEx and favorable warrant liability fair-value changes .

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
First subject treated (MIRACLE)	Q1 2025	Q1 2025	Q1 2025	Maintained
Early unblinding (n=45)	H2 2025	Q4 2025 (n≈40)	H2 2025 (n=45)	Raised/Accelerated (earlier and larger cohort)
Part A conclusion	H1 2026	Mid 2026 (n≈75)	H1 2026 (n≈75–90)	Clarified (range)
Part B size	Post-Part A	~240 (120/arm)	~220 (FDA-guided ~10% reduction)	Lowered (~10%)
STS MB-107 data readout	H1 2025	Early 2025	1H 2025 (final data readout by end of April)	Narrowed (specific timing)
Cash runway	FY 2025	Into Q1 2025	Into Q3 2025 (after ~$9.3M Feb financing)	Extended

Earnings Call Themes & Trends

Topic	Previous Mentions (Q2 2024, Q3 2024)	Current Period (Q4 2024)	Trend
Trial design and early visibility	EOP1B/2 positive; plan for adaptive Phase 3; initial n (75) then dose selection; visibility discussed	Multiple interim unblindings (n=45 in H2’25; n≈75–90 in H1’26); rationale for dose arms (230 vs 190 mg/m²) under Project Optimus	Increased clarity; accelerated early data window
Regulatory engagement (FDA)	FDA aligned on doses and adaptive design	FDA guidance enabled ~10% reduction in Part B; collaboration emphasized	Constructive; process optimization
Enrollment geography	Global: EU/ME first, U.S. slower due to IRB/hospital contracts	U.S. likely last to enroll; multiple sites screened; ~25 sites selected, ~45 in process	Execution ramping globally
Cost/burn and funding	Cash to Q4’24/Q1’25; financing plans	Burn ~$5M/qtr in 2025, rising to $7–8M/qtr in 2026; trial could cost $60–70M; recent $9.3M gross proceeds	Higher spend ahead; active financing
Efficacy narrative (AML)	Phase 1B/2 CRc 41–60% in 2L/3L cohorts; durability mDOR ~7–8 months	Durability climbing; OS ~11 months; MRD negativity 78%; venetoclax resistance overcome; no cardiotoxicity observed	Strengthening dataset; KOL validation

Management Commentary

“Unlike most pivotal Phase 3 trials, we will have multiple unblinding of data… roughly thirty days after the forty fifth subject is treated, we will be able to see how the drug is performing.” — Walter Klemp .
“Our Phase II data just keep getting better. Overall survival keeps climbing at eleven months and durability has reached nine months… and we continue to see a complete absence of drug related cardiotoxicity.” — Walter Klemp .
“Combined cash balance of approximately $13,000,000 takes us into the third quarter of twenty twenty five… OpEx reduced ~$3,000,000 in 2024 vs 2023.” — Jonathan Foster .
“FDA encouraged comparing AnnAraC vs HiDAC; prior large R/R AML studies reported ~17.5% CR on HiDAC, versus ~50% CR we observed.” — John Paul Waymack .

Q&A Highlights

Dose selection and interim unblinding: Management will select the optimal dose (230 vs 190 mg/m²) based on efficacy, safety, PK; if superiority is clear at n=45, 190 mg/m² arm may cease to accelerate Part A completion .
Trial cost and burn: Phase 3 cost could reach $60–70M; burn ~$5M/qtr in 2025, rising to $7–8M/qtr in 2026 as CMC for NDA scales .
Strategy on STS pivotal: Strong OS in late-line STS drew interest from leading European sarcoma centers; company prefers partner or IIT approach .
Special Protocol Assessment (SPA): FDA advised not to pursue SPA to avoid delays; current alignment allows flexibility without tying protocol hands .
U.S. enrollment cadence: U.S. likely lags due to IRB/contracting timing; initial recruitment expected from Ukraine, Egypt, Georgia, then EU, then U.S. .

Estimates Context

Q4 2024: EPS consensus -$1.28 vs actual diluted EPS approximately -$0.37 → bold beat; revenue consensus $0 vs actual $0 (Values retrieved from S&P Global)* [GetEstimates Q4].
FY 2024: Consensus primary EPS -$7.60 vs company-reported diluted EPS -$6.32, implying potential upward revisions on lower-than-expected GAAP loss; EBITDA actual ~$(26.52)M (Values retrieved from S&P Global)* [GetEstimates FY].
Target price consensus ~$6.67; recommendation text unavailable (Values retrieved from S&P Global)* [GetEstimates FY].

Key Takeaways for Investors

Interim unblinding at n=45 in H2 2025 is the core catalyst; multiple data looks provide unusually early visibility for a pivotal oncology trial .
The significant Q4 EPS beat vs consensus was driven by lower OpEx and non-operating items (warrant liability gains); do not extrapolate to operating profitability; focus on cash runway and financing cadence (Values retrieved from S&P Global)*.
Regulatory and protocol optimizations (FDA guidance, Part B reduction) modestly de-risk timelines and capital needs; execution hinges on rapid global site activation and recruitment .
Efficacy narrative remains compelling: Phase 2 CR ~50% in R/R AML, MRD negativity 78%, rising durability and OS, non-cardiotoxic profile—key for clinician adoption upon approval .
Funding is a swing factor: February raises ($9.3M gross) extend runway into Q3 2025; expect continued capital markets activity ahead of data to fund MIRACLE and CMC .
Near-term trading: Watch for press on first patient dosed, incremental site approvals, and MB-107 STS readout by end of April; data visibility in H2’25 could reset risk perception .
Medium-term thesis: If interim efficacy meaningfully exceeds HiDAC (~17–18% CR), pathway to accelerated approval could shorten; partner interest likely tied to H2’25/H1’26 data .

Footnote: *Values retrieved from S&P Global.

Moleculin Biotech, Inc. (MBRX)·Q4 2024 Earnings Summary