Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat tumors and viruses [3] - The company's lead program, Annamycin, is a next-generation anthracycline designed to avoid multidrug resistance and cardiotoxicity, currently in development for relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases [3] Clinical Trials - The company has initiated the MIRACLE Trial (MB-108), a pivotal Phase 3 trial evaluating Annamycin in combination with cytarabine for treating relapsed or refractory AML [4] - Following a successful Phase 1B/2 study (MB-106) and FDA input, the company believes it has de-risked the development pathway for Annamycin's potential approval for AML [4] Additional Drug Development - Moleculin is also developing WP1066, an Immune/Transcription Modulator targeting brain tumors and pancreatic cancers by inhibiting oncogenic transcription factors and stimulating immune response [5] - The company is working on a portfolio of antimetabolites, including WP1122, aimed at treating pathogenic viruses and certain cancer indications [5] Upcoming Events - Moleculin will present at the Webull Financial Corporate Connect Webinar Series: Biotech/MedTech on August 20, 2025, at 2:00 PM ET, with CEO Walter Klemp as the presenter [1]

[Q2 2025 Financial Results and Corporate Highlights](index=1&type=section&id=Moleculin%20Reports%20Second%20Quarter%202025%20Financial%20Results%20and%20Highlights) The company highlights significant Q2 2025 progress in its pivotal MIRACLE trial and positive Annamycin data [Overview](index=1&type=section&id=Overview) Moleculin advanced its pivotal Phase 2B/3 MIRACLE trial for AML and reported positive data for Annamycin in soft tissue sarcoma - The company is actively recruiting for its Phase 2B/3 MIRACLE trial, with sites now open in the US and EU[1](index=1&type=chunk) - Moleculin expects to have **over 20 additional clinical sites** in the EU and US recruiting for the MIRACLE trial by the end of Q3 2025[1](index=1&type=chunk)[5](index=5&type=chunk) - The initial pivotal data readout from Part A of the MIRACLE trial is targeted for **before the end of 2025**[1](index=1&type=chunk)[2](index=2&type=chunk) - The company reported **compelling topline data** from its Phase 1B/2 trial of Annamycin for soft tissue sarcoma lung metastases[2](index=2&type=chunk) [Clinical Development Update](index=1&type=section&id=Clinical%20Development%20Update) The company details advancements in its key clinical programs, including Annamycin for AML and STS, and WP1066 for brain tumors [Annamycin Program](index=1&type=section&id=Annamycin%20Program) The Annamycin program is advancing with its pivotal MIRACLE trial, positive STS data, and strengthened intellectual property - Annamycin has received **Fast Track and Orphan Drug Designations** from the FDA for AML, and Orphan Drug Designation for soft tissue sarcoma[12](index=12&type=chunk) - The company bolstered its Annamycin intellectual property with a notice of intent to grant a **new European patent**[5](index=5&type=chunk) - Engaged industry veteran Adriano Treve to explore **strategic partnerships** related to Annamycin[5](index=5&type=chunk) [Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) - MIRACLE Trial](index=1&type=section&id=Relapsed%20or%20Refractory%20(R%2FR)%20Acute%20Myeloid%20Leukemia%20(AML)%20-%20MIRACLE%20Trial) The global pivotal MIRACLE trial for R/R AML is expanding sites, with key data readouts expected before year-end 2025 - The MIRACLE trial is a global, pivotal Phase 2B/3 study evaluating Annamycin in combination with Cytarabine (AnnAraC) for R/R AML[3](index=3&type=chunk)[4](index=4&type=chunk) - Trial Progress: Four sites are actively screening subjects in Ukraine, Georgia, Spain, and the US, with **over 20 additional sites expected by the end of Q3 2025**[5](index=5&type=chunk) - The FDA provided **positive feedback** on a pediatric study plan for Annamycin in children with R/R AML, with the trial set to start in 2027[5](index=5&type=chunk) Key MIRACLE Trial Milestones | Milestone | Expected Timeline | | :--- | :--- | | First patients treated in US and EU | Q3 2025 | | Data readout (n=45) unblinded review | Q4 2025 | | Interim data (n=~75-90) & Optimum Dose set | 1H 2026 | | Begin enrollment of 3rd line subjects | 2027 | | Begin Rolling NDA Submission for R/R AML | 2028 | [Soft Tissue Sarcoma (STS) Lung Metastases](index=3&type=section&id=Soft%20Tissue%20Sarcoma%20(STS)%20Lung%20Metastases) Positive topline results from the Phase 1B/2 trial showed a favorable median overall survival for Annamycin in STS lung metastases - The Phase 1B/2 trial (MB-107) demonstrated a **median overall survival (OS) of 13.5 months** for subjects who were on a median 7th line of therapy[11](index=11&type=chunk) - This OS of 13.5 months **compares favorably** to the 8-12 months for standard of care treatments in advanced STS as a 2nd line therapy[11](index=11&type=chunk) - The company expects to identify the next phase of development or a pivotal investigator-initiated trial program in **Q4 2025**[12](index=12&type=chunk) [WP1066 & Brain Tumors](index=3&type=section&id=WP1066%20%26%20Brain%20Tumors) The WP1066 program is advancing through an investigator-initiated trial for glioblastoma and a preclinical collaboration for IV formulations - An externally funded Phase 1B/2 trial of oral WP1066 in combination with radiation for glioblastoma (GBM) is **actively recruiting** at Northwestern University[13](index=13&type=chunk) - A preclinical study agreement is in place with Emory University to evaluate various WP1066 IV formulations, with results expected in **H2 2025**[13](index=13&type=chunk) [Q2 2025 Financial Results](index=3&type=section&id=Summary%20of%20Financial%20Results%20for%20the%20Second%20Quarter%202025) The company reports its second-quarter financial performance, including operating expenses, cash position, and consolidated statements [Financial Performance Summary](index=3&type=section&id=Financial%20Performance%20Summary) The company reported lower R&D expenses and ended Q2 2025 with $7.6 million in cash, sufficient to fund operations into Q4 2025 Q2 2025 Operating Expenses (in millions) | Expense Category | Q2 2025 | Q2 2024 | Change | | :--- | :--- | :--- | :--- | | Research & Development | $3.6 | $4.1 | -$0.5 | | General & Administrative | $2.1 | $2.1 | $0.0 | - The company held **$7.6 million in cash and cash equivalents** as of June 30, 2025[15](index=15&type=chunk) - Current cash reserves are expected to fund planned operations into the **fourth quarter of 2025**[15](index=15&type=chunk) [Condensed Consolidated Financial Statements](index=6&type=section&id=Condensed%20Consolidated%20Financial%20Statements) Unaudited statements show increased liabilities and a net loss of $7.6 million for the quarter ending June 30, 2025 Condensed Consolidated Balance Sheet (in thousands) | Account | June 30, 2025 | Dec 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $7,557 | $4,278 | | Total current assets | $9,076 | $5,194 | | Total assets | $21,593 | $16,925 | | Total current liabilities | $7,913 | $5,359 | | Total liabilities | $28,758 | $10,946 | | Total stockholders' equity (deficit) | $(7,165) | $5,979 | Condensed Consolidated Statement of Operations (in thousands, except per share data) | Account | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | Research and development | $3,600 | $4,090 | | General and administrative | $2,120 | $2,095 | | Loss from operations | $(5,720) | $(6,185) | | Net loss | $(7,640) | $(4,319) | | Net loss per common share | $(0.49) | $(1.70) | [Corporate Information](index=3&type=section&id=About%20Moleculin%20Biotech%2C%20Inc.) An overview of the company's focus as a clinical-stage entity developing treatments for hard-to-treat cancers and viruses [Company Overview](index=3&type=section&id=Company%20Overview) Moleculin is a Phase 3 company developing treatments for difficult cancers, led by its next-generation anthracycline, Annamycin - Moleculin is a **Phase 3 clinical-stage company** with a pipeline targeting difficult cancers and viruses[16](index=16&type=chunk) - Lead program Annamycin is a next-generation anthracycline designed to **avoid cardiotoxicity and multidrug resistance**[16](index=16&type=chunk) - Other pipeline assets include **WP1066**, an Immune/Transcription Modulator for brain tumors, and **WP1122**, an antimetabolite for viruses and cancers[19](index=19&type=chunk)

Core Insights - Moleculin Biotech, Inc. is making significant progress in its Phase 2B/3 MIRACLE trial for Annamycin, targeting relapsed or refractory acute myeloid leukemia (AML) with an expected data readout before the end of 2025 [1][2][21] Clinical Development Updates - The MIRACLE trial is expanding with over 20 additional clinical sites expected to begin recruitment by the end of Q3 2025, including locations in the US, Europe, and the Middle East [1][7] - The trial utilizes an adaptive design, with the first 75 to 90 subjects randomized to receive different doses of Annamycin combined with high-dose cytarabine [6][10] - Preliminary efficacy data is expected to be unblinded for the first 45 subjects treated before the end of 2025, with a second unblinding anticipated in the first half of 2026 [8] Financial Results - For Q2 2025, the company reported a research and development expense of $3.6 million, down from $4.1 million in Q2 2024, primarily due to reduced clinical trial activity [18] - General and administrative expenses remained stable at $2.1 million for both Q2 2025 and Q2 2024 [18] - As of June 30, 2025, the company had cash and cash equivalents of $7.6 million, which is expected to fund operations into Q4 2025 [19] Annamycin Development Milestones - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory AML and soft tissue sarcoma [15] - Positive topline results from the U.S. Phase 1B/2 trial for Annamycin in treating soft tissue sarcoma lung metastases showed a median overall survival of 13.5 months, compared to 8-12 months for standard treatments [13] Intellectual Property and Strategic Partnerships - The company has bolstered its intellectual property portfolio with a notice of intent to grant a new European patent for Annamycin [7] - An industry veteran has been engaged to explore strategic partnerships related to Annamycin [7]

[PART I – FINANCIAL INFORMATION](index=3&type=section&id=PART%20I%20%E2%80%93%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements (Unaudited)](index=3&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) The unaudited financial statements for June 30, 2025, show a **$7.2 million stockholders' deficit** and a **$14.1 million net loss**, primarily due to increased warrant liabilities, raising significant going concern doubts with **$7.6 million cash** on hand [Condensed Consolidated Balance Sheets](index=3&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of June 30, 2025, total assets rose to **$21.6 million**, but total liabilities surged to **$28.8 million** due to a **$20.6 million warrant liability**, resulting in a **$7.2 million stockholders' deficit** from a prior $6.0 million equity Condensed Consolidated Balance Sheet Highlights (in thousands) | Metric | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $7,557 | $4,278 | | Total current assets | $9,076 | $5,194 | | Total assets | $21,593 | $16,925 | | **Liabilities & Stockholders' Equity** | | | | Total current liabilities | $7,913 | $5,359 | | Warrant liability | $20,553 | $5,229 | | Total liabilities | $28,758 | $10,946 | | Total stockholders' equity (deficit) | $(7,165) | $5,979 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=4&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) The company reported **no revenue** and a widened **net loss of $14.1 million** for the six months ended June 30, 2025, primarily due to an **$18.2 million loss on warrant issuances**, despite a partially offsetting **$18.7 million gain** from fair value changes and decreased R&D expenses Statement of Operations Highlights (in thousands, except per share data) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | :--- | :--- | | Revenues | $0 | $0 | $0 | $0 | | Research and development | $3,600 | $4,090 | $7,036 | $8,342 | | General and administrative | $2,091 | $2,064 | $4,568 | $4,457 | | Loss from operations | $(5,720) | $(6,185) | $(11,663) | $(12,862) | | Gain from change in fair value of warrant liability | $9,609 | $1,696 | $18,663 | $3,151 | | Loss on issuance of warrant liabilities | $(10,352) | $0 | $(18,150) | $0 | | Net loss | $(7,640) | $(4,319) | $(14,076) | $(9,289) | | Net loss per share - basic and diluted | $(0.49) | $(1.70) | $(1.13) | $(3.71) | [Condensed Consolidated Statements of Cash Flows](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) For the six months ended June 30, 2025, **net cash used in operations decreased to $10.1 million**, while **financing activities provided $13.4 million**, leading to a **$3.3 million net increase in cash**, with the period ending at **$7.6 million** Cash Flow Summary (in thousands) | Metric | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(10,146) | $(12,666) | | Net cash used in investing activities | $0 | $(13) | | Net cash provided by (used in) financing activities | $13,422 | $(25) | | Net increase (decrease) in cash and cash equivalents | $3,279 | $(12,705) | | Cash and cash equivalents, end of period | $7,557 | $10,845 | [Notes to Condensed Consolidated Financial Statements](index=7&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) The notes detail the company's late-stage pharmaceutical focus on Annamycin's Phase 3 trial, disclose **Nasdaq non-compliance** and a **going concern issue** due to insufficient cash, and highlight the significant impact of **2025 public offerings and warrant issuances** on financials - The company is a late-stage pharmaceutical development company conducting a pivotal Phase 3 trial for Annamycin for the treatment of relapsed/refractory (R/R) acute myeloid leukemia (AML)[19](index=19&type=chunk) - The company received notifications from Nasdaq for non-compliance with the minimum stockholders' equity rule (**$2.5 million**) on May 23, 2025, and the minimum bid price rule (**$1.00**) on June 27, 2025[23](index=23&type=chunk)[24](index=24&type=chunk) - Management has substantial doubt about the company's ability to continue as a going concern, as its cash on hand of **$7.6 million** as of June 30, 2025, is not sufficient to fund planned operations for at least one year[33](index=33&type=chunk) - In June 2025, the company closed a public offering raising gross proceeds of **$5.9 million**, with the full proceeds recorded as warrant liabilities, resulting in a **$10.4 million loss on issuance**[53](index=53&type=chunk)[54](index=54&type=chunk) - In February 2025, the company raised total gross proceeds of **$9.3 million** through two transactions: a securities purchase agreement and a warrant exercise inducement offer[56](index=56&type=chunk)[57](index=57&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=20&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management's discussion highlights the pivotal Phase 3 MIRACLE trial for Annamycin, with interim data expected by year-end 2025, alongside a **Q2 2025 net loss of $7.6 million** and critical liquidity concerns requiring **$10 million in additional financing** to extend operations into Q1 2026 [Our Business and Recent Developments](index=22&type=section&id=Our%20Business%20and%20Recent%20Developments) The company, a late-stage pharmaceutical firm, is focused on its pivotal Phase 3 MIRACLE trial for Annamycin in R/R AML, with interim data expected by year-end 2025, supported by trial expansion, EMA approval, positive FDA feedback, and new patent grants - The company is conducting a pivotal Phase 3 trial (MIRACLE) for Annamycin in R/R AML, with an interim data unblinding expected by the end of 2025[88](index=88&type=chunk) - As of July 25, 2025, over **35 sites** have been selected for the MIRACLE trial, with four actively recruiting and **eight subjects treated**[102](index=102&type=chunk) - The European Medicines Agency (EMA) approved the MIRACLE trial application for all **nine submitted EU countries** in May 2025[107](index=107&type=chunk) - The FDA has agreed to a single pediatric approval study for Annamycin in combination with Cytarabine for pediatric patients with R/R AML[109](index=109&type=chunk) - The company received a Notice of Intent to Grant for a European patent and was granted two additional U.S. patents for Annamycin, with terms extending until **2040**[117](index=117&type=chunk)[121](index=121&type=chunk) [Results of Operations](index=28&type=section&id=Results%20of%20Operations) For Q2 2025, R&D expenses decreased to **$3.6 million**, while a **$9.6 million non-cash gain** from warrant fair value changes was offset by a **$10.4 million loss on warrant issuance**, contributing to a widened net loss for the six-month period Comparison of Operating Expenses (in thousands) | Expense Category | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | :--- | :--- | | Research and development | $3,600 | $4,090 | $7,036 | $8,342 | | General and administrative | $2,091 | $2,064 | $4,568 | $4,457 | - The decrease in R&D expense for both the three and six-month periods is mainly related to a reduction in clinical trials activity[127](index=127&type=chunk)[131](index=131&type=chunk) - For Q2 2025, the company recorded a **$9.6 million gain** from the change in fair value of warrant liability, compared to a **$1.7 million gain** in Q2 2024, principally due to a decline in the company's share price[128](index=128&type=chunk) - The June 2025 offering resulted in a **$10.4 million loss on issuance** of warrant liabilities and **$1.2 million** in expensed transaction costs allocated to these warrants[129](index=129&type=chunk) [Liquidity and Capital Resources](index=30&type=section&id=Liquidity%20and%20Capital%20Resources) As of June 30, 2025, the company held **$7.6 million in cash**, with **$10.1 million used in operations** and **$13.4 million provided by financing**, but requires an additional **$10 million** to fund operations into Q1 2026, raising significant going concern doubts Sources and Uses of Cash (in thousands) | Metric | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(10,146) | $(12,666) | | Net cash provided by (used in) financing activities | $13,422 | $(25) | | Net increase (decrease) in cash and cash equivalents | $3,279 | $(12,705) | - The company believes its cash on hand as of June 30, 2025, is sufficient to fund planned operations only into the **fourth quarter of 2025**[145](index=145&type=chunk) - The company must seek additional funding of approximately **$10 million** to support the MIRACLE trial and operations into Q1 2026[145](index=145&type=chunk) - In July 2025, the company entered into an At-The-Market (ATM) agreement to sell up to **$6.5 million** of its common stock[140](index=140&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=34&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section is omitted as the company qualifies as a smaller reporting company, rendering the disclosure not applicable - This disclosure is not applicable as the company is a smaller reporting company[149](index=149&type=chunk) [Controls and Procedures](index=34&type=section&id=Item%204.%20Controls%20and%20Procedures) Management, including the CEO and CFO, concluded that disclosure controls and procedures were **effective as of June 30, 2025**, with no material changes to internal control over financial reporting during the quarter - The CEO and CFO evaluated disclosure controls and procedures and determined they were effective as of June 30, 2025[150](index=150&type=chunk) - No changes in internal control over financial reporting occurred during the three months ended June 30, 2025, that materially affected, or are reasonably likely to materially affect, internal controls[151](index=151&type=chunk) [PART II – OTHER INFORMATION](index=34&type=section&id=PART%20II%20%E2%80%93%20OTHER%20INFORMATION) [Legal Proceedings](index=34&type=section&id=Item%201.%20Legal%20Proceedings) The company reported no legal proceedings during the period - None[153](index=153&type=chunk) [Risk Factors](index=34&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant risks from **Nasdaq non-compliance** with minimum stockholders' equity and bid price rules, with potential delisting and further dilution from Series E warrants impacting liquidity and capital raising efforts - The company is not in compliance with Nasdaq's continued listing requirements, specifically the Equity Rule (minimum **$2.5 million stockholders' equity**) and the Bid Price Rule (minimum **$1.00 per share**)[155](index=155&type=chunk)[156](index=156&type=chunk)[160](index=160&type=chunk) - If the company is unable to regain compliance, its common stock will be delisted from Nasdaq, which could materially harm its financial condition and stock liquidity[155](index=155&type=chunk)[165](index=165&type=chunk) - The accounting for Series E warrants as a liability may hinder the company's ability to meet the stockholders' equity requirement[158](index=158&type=chunk) - Future financing or a reverse stock split could trigger anti-dilution provisions in the Series E warrants, potentially reducing their exercise price and significantly increasing the number of shares issuable upon exercise, causing further dilution to shareholders[167](index=167&type=chunk)[168](index=168&type=chunk)[171](index=171&type=chunk) [Unregistered sales of Equity Securities and Uses of Proceeds](index=36&type=section&id=Item%202.%20Unregistered%20sales%20of%20Equity%20Securities%20and%20Uses%20of%20Proceeds) The company reported no unregistered sales of equity securities during the period - None[172](index=172&type=chunk) [Other Information](index=37&type=section&id=Item%205.%20Other%20Information) No directors or executive officers adopted or terminated Rule 10b5-1 or non-Rule 10b5-1 trading arrangements during the reporting period - No directors or executive officers adopted or terminated a Rule 10b5-1 trading arrangement or other non-Rule 10b5-1 trading arrangement during the quarter[175](index=175&type=chunk) [Exhibits](index=38&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including warrant forms, an At The Market Offering Agreement, and officer certifications

Core Insights - Moleculin Biotech, Inc. announced promising preclinical data for its lead drug candidate, Annamycin, showing significant efficacy against various liver cancers, including hepatocellular carcinoma, colorectal liver metastases, and pancreatic ductal adenocarcinoma liver metastases [1][4][5] - Annamycin is currently in late-stage clinical development for the treatment of acute myeloid leukemia (AML) in combination with cytarabine, with preliminary data expected in the second half of 2025 [1][7] Company Overview - Moleculin Biotech, Inc. is a Phase 3 clinical stage pharmaceutical company focused on developing therapeutic candidates for hard-to-treat tumors and viruses, with Annamycin being a next-generation anthracycline designed to avoid multidrug resistance and cardiotoxicity [6][8] - The company is also developing WP1066, an immune/transcription modulator targeting brain tumors and other cancers, along with a portfolio of antimetabolites for potential treatment of pathogenic viruses and certain cancer indications [8] Preclinical Findings - Annamycin demonstrated targeted accumulation in organs, particularly the liver, spleen, lungs, and pancreas, leading to higher concentrations compared to doxorubicin, which is critical for treating liver-localized tumors [5] - In orthotopic hepatocellular carcinoma models, Annamycin showed excellent anti-tumor activity, significantly reducing tumor progression and improving survival rates [5] - The drug also exhibited significant efficacy in inhibiting metastatic growth in colorectal liver metastasis models and showed promising results in managing advanced pancreatic cancer with liver involvement [5] Safety Profile - Annamycin has a favorable safety profile, showing low or no cardiotoxicity, which is a significant advantage over traditional anthracyclines like doxorubicin, often limited by dose-dependent cardiac side effects [5]

Core Viewpoint - Moleculin Biotech, Inc. has received a Notice of Intent to Grant for a European patent that will enhance its market position for Annamycin, a potential first non-cardiotoxic anthracycline, aimed at treating hard-to-treat tumors and viruses [2][3][4] Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat tumors and viruses, with its lead program being Annamycin [5] - Annamycin is designed to avoid multidrug resistance and lacks the cardiotoxicity associated with current anthracyclines, currently in development for relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma lung metastases [5] Patent and Development - The new European patent will cover methods for creating a preliposomal Annamycin lyophilizate with improved stability and high purity, with a base patent term extending until 2040 [3][4] - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for relapsed or refractory AML and STS lung metastases, as well as Orphan Drug Designation from the EMA [4] Clinical Trials - The company has initiated the MIRACLE Trial (MB-108), a pivotal Phase 3 trial evaluating Annamycin in combination with cytarabine for relapsed or refractory AML, following a successful Phase 1B/2 study [6] - The development pathway for Annamycin is considered substantially de-risked with input from the FDA [6] Additional Developments - Moleculin is also developing WP1066, an immune/transcription modulator targeting various cancers, and a portfolio of antimetabolites including WP1122 for potential treatment of pathogenic viruses and certain cancer indications [7]

Core Insights - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat tumors and viruses [1][3] - The company's lead program, Annamycin, is a next-generation anthracycline designed to avoid multidrug resistance and cardiotoxicity, currently in development for relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases [3][4] - The MIRACLE Trial (MB-108) is a pivotal Phase 3 trial evaluating Annamycin in combination with cytarabine for the treatment of relapsed or refractory AML, with a successful Phase 1B/2 study providing confidence in the development pathway [4] Company Overview - Moleculin Biotech, Inc. is advancing a pipeline of therapeutic candidates, including WP1066, an immune/transcription modulator targeting brain tumors and pancreatic cancers, and a portfolio of antimetabolites like WP1122 for treating pathogenic viruses and certain cancers [5] Recent Developments - Walter Klemp, the company's Founder, President, CEO, and Chairman, participated in the Virtual Investor "What's Your Story" Summer Spotlight On-Demand Conference, sharing insights into his dedication to the company and its programs [2]

Core Viewpoint - Moleculin Biotech, Inc. has appointed Adriano Treve as a Strategic Advisor for partnerships, leveraging his extensive experience in global healthcare to enhance the company's strategic positioning and partnership opportunities as it advances its clinical trials [1][3][5]. Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat cancers and viral infections [1]. - The company's lead program, Annamycin, is designed to avoid multidrug resistance and cardiotoxicity associated with current anthracyclines, targeting relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma [10][11]. Key Developments - The Phase 2B/3 MIRACLE trial for Annamycin in combination with cytarabine is ongoing, with the first unblinding of data for 45 patients expected in the second half of 2025 and a second unblinding anticipated in the first half of 2026 [4][8]. - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for treating relapsed or refractory AML, as well as similar designations from the EMA [9]. Strategic Partnerships - Mr. Treve's appointment is expected to enhance Moleculin's ability to secure partnerships that could optimize the value of Annamycin, which has the potential to significantly impact the global oncology drug market [5][6]. - The company is experiencing increased interest from potential strategic partners as it approaches key milestones in its clinical trials [6]. Clinical Trial Details - The MIRACLE trial is a pivotal, adaptive design Phase 3 study evaluating Annamycin in combination with cytarabine for adult patients with relapsed or refractory AML, conducted across multiple sites in the US, Europe, and the Middle East [11]. - The trial follows a successful Phase 1B/2 study, which has de-risked the development pathway towards potential approval for Annamycin [11].

Core Points - The Regulation Agency for Medical and Pharmaceutical Activities (RAMPA) in Georgia has approved the Clinical Trial Application (CTA) for the pivotal Phase 2B/3 MIRACLE trial of Annamycin in combination with cytarabine for treating adult patients with relapsed or refractory acute myeloid leukemia (AML) [2][3] - The European Medicines Agency (EMA) has also granted approval for the trial, enhancing its profile and recruitment efforts [3][5] - Enrollment in Part A of the MIRACLE trial has reached seven subjects treated, with one additional subject in screening, and the company plans to expand to over 30 clinical sites by the end of the year [3][4] Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat cancers and viral infections, with Annamycin as its lead program [11][12] - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for treating relapsed or refractory AML, as well as similar designations from the EMA [8][11] - The MIRACLE trial is designed to combine data from the Phase 2B and Phase 3 portions to measure the primary efficacy endpoint, utilizing an adaptive design [3][12] Trial Details - The MIRACLE trial will randomize the first 75 to 90 subjects to receive high-dose cytarabine combined with either placebo or two different doses of Annamycin [3][6] - The first unblinding of preliminary efficacy data is expected in the second half of 2025, with a second unblinding anticipated in the first half of 2026 [4][6] - Approximately 220 additional subjects will be randomized in Part B of the trial to receive either high-dose cytarabine plus placebo or the optimum dose of Annamycin [6] Recruitment and Collaboration - The company expects to add 16 additional clinical sites in Europe and the US by the end of August, building to over 30 sites for Part A of the trial [3][4] - The recruitment success is attributed to the capabilities of clinical sites, particularly the ARENSIA Exploratory Medicine's research clinic in Kyiv, Ukraine [3][9]

Core Viewpoint - Moleculin Biotech, Inc. has announced a public offering of 16,080,000 shares of common stock and Series E warrants, priced at $0.37 per share, aiming to raise approximately $5.9 million to advance its drug development pipeline [1][2]. Group 1: Offering Details - The public offering includes 16,080,000 shares of common stock and Series E warrants to purchase up to 48,240,000 shares, with a combined offering price of $0.37 per share [1]. - The offering is expected to close on or about June 23, 2025, subject to customary closing conditions [2]. - Gross proceeds from the offering are anticipated to be approximately $5.9 million before deducting fees and expenses [2]. Group 2: Use of Proceeds - The net proceeds from the offering will be used to advance the development of Annamycin and two other drug portfolios through clinical development [2]. - Funds will also support preclinical studies and research sponsorship, as well as working capital needs [2]. Group 3: Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing therapies for hard-to-treat tumors and viruses, with its lead program Annamycin targeting relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma [6][7]. - The company is conducting the MIRACLE trial, a pivotal Phase 3 study evaluating Annamycin in combination with cytarabine for AML treatment [7]. - Additionally, Moleculin is developing WP1066, an immune/transcription modulator for various cancers, and a portfolio of antimetabolites including WP1122 for treating pathogenic viruses [8].