What Moleculin does

Moleculin Biotech, Inc. (NASDAQ: MBRX) is a Phase 3 clinical-stage pharmaceutical company headquartered in Houston, TX (5300 Memorial Drive, Suite 950), developing a pipeline of small-molecule drug candidates for hard-to-treat tumors and viruses. The company's lead asset is Annamycin, a next-generation liposomal anthracycline engineered to eliminate the cardiotoxicity and multidrug resistance limitations of conventional anthracyclines such as doxorubicin. Annamycin has demonstrated no clinically significant cardiotoxicity across 90 patients in five completed clinical trials, even at cumulative doses exceeding conventional anthracycline limits, with independent cardiac safety review by Cleveland Clinic confirming stable mean ejection fraction. Additional pipeline assets include WP1066 (a first-in-class STAT3 immune/transcription modulator for pediatric brain tumors and adult glioblastoma, supported by 50+ peer-reviewed publications), WP1122 (a 2-deoxy-D-glucose prodrug with improved blood-brain barrier penetration for glioblastoma), and WP1220 (a topical analog for cutaneous T-cell lymphoma).

The company's pivotal MIRACLE Phase 2B/3 trial is evaluating Annamycin in combination with cytarabine (AnnAraC) in relapsed/refractory AML across nine countries (US, Spain, Italy, Poland, Czechia, Romania, Ukraine, Lithuania, and Georgia), with enrollment of the 45th subject triggering interim data unblinding expected mid-2026. The company holds FDA Fast Track, FDA Orphan Drug, EMA Orphan Drug, and FDA Rare Pediatric Disease designations across its pipeline, plus a global patent estate jointly owned with The University of Texas System extending through June 2040 across 12 jurisdictions.

Moleculin is pre-revenue with no commercialized products and reported a net loss of $33.7M for FY2025. The business model targets potential FDA/EMA approval and commercialization likely through partnership with established pharmaceutical companies rather than independent launch. Capital is sourced from public equity offerings and warrant exercises advised by Roth Capital Partners, including approximately $8.3M raised in February 2026 and $6.5M in December 2025, with $10.3M in cash and equivalents as of March 31, 2026 and a market capitalization of approximately $13.36M.

Name

Moleculin

Legal name

Moleculin Biotech, Inc.

Website

https://moleculin.com

Company type

Public

Founded year

2015

Operating status

Operating

Headcount range

11–50 employees

Short description

Moleculin Biotech is a Phase 3 clinical-stage pharmaceutical company developing non-cardiotoxic anthracycline and STAT3 inhibitor drug candidates for relapsed/refractory AML, soft tissue sarcoma lung metastases, and brain tumors. The company is pre-revenue and relies on equity and warrant-based financing to advance its pipeline.

Ownership category

akta.pro rank

Product category

Clinical-stage oncology pharmaceuticals

NAICS

Pharmaceutical and Medicine Manufacturing (3254)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Rare Oncology & Hematologic Malignancy Therapies (HLAIAIAC)

akta.pro secondary industry

Immunology & Inflammation Pharmaceuticals (HLAIAAAD)

Keywords

• Clinical-stage oncology drugs
• Anthracycline drug development
• Acute myeloid leukemia therapy
• Soft tissue sarcoma treatment
• STAT3 inhibitor therapeutics

Headquarters

HQ city

Houston

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel

Revenue model

1. Pharmaceutical Product Revenue: Moleculin is a Phase 3 clinical stage pharmaceutical company with no current revenue. The company is focused on advancing Annamycin through clinical trials toward potential FDA approval and commercialization for the treatment of relapsed/refractory AML and soft tissue sarcoma lung metastases.

Go-to-market motion2 records

Distribution channels1 record

Marketing channels5 records

Core offering

Moleculin Biotech is a Phase 3 clinical-stage pharmaceutical company developing a pipeline of proprietary drug candidates targeting hard-to-treat tumors and viruses. Its lead candidate, Annamycin, is a next-generation, non-cardiotoxic liposomal anthracycline being advanced in a pivotal Phase 2B/3 trial (MIRACLE) for relapsed/refractory acute myeloid leukemia (AML) and in Phase 1B/2 for soft tissue sarcoma lung metastases. Additional candidates include WP1066 (STAT3 inhibitor), WP1122 (glycosylation inhibitor), and WP1220 (topical p-STAT3 inhibitor for cutaneous T-cell lymphoma).

Product overview

Moleculin Biotech is a Phase 3 clinical stage pharmaceutical company advancing a pipeline of therapeutic drug candidates addressing hard-to-treat tumors and viruses. The company's portfolio includes: Annamycin, a next-generation non-cardiotoxic anthracycline in pivotal Phase 3 trials for AML and Phase 1B/2 for soft tissue sarcoma lung metastases; WP1066, an immune/transcription modulator targeting p-STAT3 for brain tumors and pancreatic cancer; WP1122, a metabolism/glycosylation inhibitor for glioblastoma and pancreatic cancer; and WP1220, a topical treatment for cutaneous T-cell lymphoma. The lead program Annamycin is designed to avoid cardiotoxicity and multidrug resistance mechanisms that limit conventional anthracyclines.

Differentiator

Problem solved

Functional benefit

Brands

• Annamycin: Lead product candidate - next-generation non-cardiotoxic anthracycline for treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma lung metastases
• WP1066
• WP1122
• WP1220
• AnnAraC
• MIRACLE Trial
• CEO Corner

Products and services

• Annamycin A next-generation, non-cardiotoxic liposomal anthracycline drug candidate designed to treat relapsed/refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases. Demonstrates lack of cardiotoxicity across 90 patients in completed trials and avoids multidrug resistance mechanisms.
• WP1066 A first-in-class immune/transcription modulator targeting p-STAT3 and other oncogenic transcription factors while stimulating natural immune responses. In development for brain tumors (including pediatric DIPG), pancreatic cancer, and other oncology indications. Subject of over 50 peer-reviewed publications.
• WP1122 A prodrug of 2-deoxy-D-glucose (2-DG) acting as a metabolism/glycosylation inhibitor with improved cellular uptake, extended drug half-life, and enhanced blood-brain barrier penetration. In development for glioblastoma (with FDA Orphan Drug Designation) and pancreatic cancer.
• WP1220

Quantifiable outcome

• 40% composite complete remission rate in first 30 MIRACLE trial subjects vs 17-18% historical cytarabine benchmarks
• +3 more outcomes

Segments3 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature5 records

Partnerships

Seven partnerships are on record, tiered minor and core.

• Aflac Cancer and Blood Disorders Center (Emory University)minorStrategic or Co-development Partner · 17 December 2025Conducted positive Phase 1 clinical trial of WP1066 for treatment of pediatric recurrent malignant brain tumors in 10 children
• CIC biomaGUNE (Spain)minorStrategic or Co-development Partner · 8 December 2025Research and material transfer agreement to evaluate Annamycin for treating glioblastoma multiforme, with preclinical research being conducted in mouse models under direction of Principal Investigator
• UNC-Chapel Hill (William C. Zamboni)minorStrategic or Co-development Partner · 12 November 2025Investigator-initiated preclinical research agreement evaluating Annamycin for pancreatic cancer treatment, comparing liposomal Annamycin against Doxil and Free-doxorubicin in PDAC GEMM models
• Atlantic HealthminorStrategic or Co-development Partner · 24 October 2025Collaboration for Phase 1B/2 clinical trial evaluating Annamycin for third-line treatment of advanced pancreatic cancer, expected to cost approximately $1 million from 2026 to 2030
• The University of Texas SystemcoreStrategic or Co-development PartnerJoint ownership of Annamycin intellectual property including patents for liposomal formulation and reconstitution methods, extending through June 2040 globally
• Cleveland CliniccoreStrategic or Co-development PartnerIndependent cardiac safety review of Annamycin pooled data from 90 patients across five clinical trials, confirming no clinically significant cardiotoxicity
• Northwestern UniversityminorStrategic or Co-development PartnerInvestigator-initiated Phase 2 study evaluating WP1066 in combination with radiation therapy for adult glioblastoma under Northwestern's IND application (NU 21C06)

Scale indicators8 records

Expansion highlights6 records

Direct peers

• Jazz Pharmaceuticals: Jazz markets Vyxeos, an approved liposomal daunorubicin + cytarabine combination for AML — the closest direct competitor to Moleculin's AnnAraC program. Both companies target relapsed/refractory AML with liposomal anthracycline + cytarabine approaches, making Jazz the most directly comparable commercial-stage peer.
• Aprea Therapeutics: Aprea is a clinical-stage oncology company focused on hematologic malignancies including AML. Comparable to Moleculin in size, stage, and indication focus, with a similarly underexplored market cap and reliance on pivotal data readouts.
• Actinium Pharmaceuticals: Actinium is a clinical-stage company developing targeted radiotherapies for hematologic cancers, including AML. Comparable in size, listing (NYSE American), and focus on relapsed/refractory AML — a directly overlapping clinical and commercial thesis.
• Kura Oncology: Kura is a clinical-stage precision oncology company with programs in hematologic malignancies and solid tumors. Comparable in size, pipeline depth, and strategy of leveraging orphan/rare disease designations in oncology.
• GlycoMimetics: GlycoMimetics is a clinical-stage company developing therapies for hematologic diseases, including AML. Comparable as a small-cap clinical-stage oncology peer focused on underserved blood cancer indications.
• Syros Pharmaceuticals: Syros is a clinical-stage biopharmaceutical company developing small-molecule therapies for hematologic malignancies. Comparable in stage, size, and target indication (hematologic cancers with high unmet need).

Broad incumbents

• Bristol-Myers Squibb: BMS markets AML therapies including Onureg (oral azacitidine) and has established a major AML franchise. Relevant as a potential strategic acquirer/partner for late-stage AML assets like Annamycin.
• AbbVie: AbbVie is a major oncology player and acquired CEO Klemp's prior company Soliton. Represents a plausible strategic acquirer profile for late-stage oncology assets like Annamycin.
• Daiichi Sankyo: Daiichi Sankyo is the historical pioneer of anthracycline drug development (doxorubicin/daunorubicin originator). Relevant as a benchmark for anthracycline technology ownership and a potential strategic acquirer of next-generation liposomal anthracyclines.

Emerging players

• CNS Pharmaceuticals: CNS Pharma is a clinical-stage oncology company where Moleculin's CSO Donald Picker also serves as part-time CSO. Comparable as a small-cap clinical-stage oncology peer, though CNS focuses on brain tumors (GBM) rather than AML.

Market position

Strengths3 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/moleculinbio
• Facebookfacebook.com/moleculin
• LinkedInlinkedin.com/company/moleculin-llc

Revenue estimate

Valuation estimate

Number of profiles

Profiles5 records

Funding overview

Funding rounds14 records

Investors1 record

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A1 record

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Moleculin