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Repurposing a Potent MDM2 Inhibitor for Leukemia Treatment
Application
VERU-111, also known as sabizabulin, is a potent microtubule inhibitor and, notably, an MDM2 inhibitor. This repurposed compound demonstrates remarkable efficacy in the treatment of Acute Myeloid Leukemia (AML).
Key Benefits
Unique dual-action functionality as a microtubule and MDM2 inhibitor.
Successful inhibition of MDM2, an oncogene...
Published: 2/13/2025
Contributor(s): Deborah DeRyckere, Muxiang Zhou, Lu Bing Gu, Douglas Graham, Tao Liu
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ONC201 for the Treatment of Vascular Aneurysms
Application
A repurposed therapeutic for the preventative and treatment of aneurysms.
Key Benefits
A repurposed cancer drug that targets cells known to be damaged during the progression of an aneurysm.
The drug has been tested within an animal model of aortic aneurysms, where it prevented the severe complications of the disease and protected the...
Published: 3/28/2025
Contributor(s): Alejandra Maria San Martin
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Therapeutic to Treat Age-Dependent Idiopathic Pulmonary Fibrosis
Application
Therapeutic for treating age-associated lung fibrosis.
Key Benefits
Tecfidera (dimethyl fumarate - DMF) is an FDA approved therapy to treat multiple sclerosis via oral administration.
A novel formulation for inhaled delivery demonstrated efficacy for a new indication: Idiopathic Pulmonary Fibrosis (IPF), where it promotes the reversal...
Published: 2/13/2025
Contributor(s): Louise Hecker, Kosuke Kato, Priyadarshini Muralidharan, Heidi Mansour
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Combination of Tetrahydrobiopterin and Arginine/ ± Arginase Inhibitors for the Treatment of COVID-19
Application
A novel drug combination to address acute arginine/BH4 dysregulation that develops with COVID-19 and MIS-C infection.
Key Benefits
Potential to improve outcomes of those infected with COVID-19 with few side effects.
Repurposing opportunity with the combination of two approved drugs (tetrahydrobiopterin and arginine).
Addresses...
Published: 3/28/2025
Contributor(s): Claudia Morris, Lou Ann Brown
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Small Molecule Inhibitors for the Treatment of COVID-19 Infection
Application
Small molecule therapy to treat patients infected with COVID-19 virus.
Key Benefits
Current FDA-approved therapeutic can be repurposed to fight COVID-19 infection.
Treatment option for infected COVID-19 patients.
Market Summary
Coronavirus (COVID-19) is an infectious disease that causes severe acute respiratory syndrome coronavirus...
Published: 2/13/2025
Contributor(s): Jack Arbiser, Martijn van Hemert, Melissa Thaler
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Repurposed Treatment for Cocaine Use Disorder
Application
Repurposed drug for transcriptomic-driven treatment of cocaine use disorder.
Key Benefits
FDA-approved drug.
Outperformed current targets undergoing clinical trials for CUD at the mRNA level.
Market Summary
Cocaine abuse is associated with substantial morbidity and mortality, and there are no approved medications for the treatment...
Published: 3/28/2025
Contributor(s): Rohan H.C. Palmer, Spencer Huggett
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Drug Combination to Treat Lung Cancer Metastasis
Application
Combination product co-targeting PDH and GLUT1 to inhibit lung cancer metastasis.
Key Benefits
Combination of drugs exhibits greater synergism in cancer cell lines (H1299, 4T1, H1792).
Has potential to prevent metastasis in a variety of cancers.
Decreases both cell viability and invasive area.
Repurposed drugs.
Market Summary
Metastatic...
Published: 7/30/2024
Contributor(s): Malathy (mala) Shanmugam, Adam Marcus
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HDAC Inhibitors for Managing Vascular Conditions and Diabetic Peripheral Neuropathies
Application
Treatment of diabetic neuropathy using epigenetic modulators, histone deacetylase inhibitors (HDACis), to ameliorate or prevent vascular conditions and neuropathic pains by nerve damages.
Key Benefits
Reverses or protects against diabetes-induced nerve damages by direct injection of HDACi.
Relatively low regulatory risk because many...
Published: 2/13/2025
Contributor(s): Young-Sup Yoon, Ji Woong (Jay) Han, Hyunsuk Shim
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Therapeutic Compounds Inhibiting DUSP5 and DUSP6 for Cardiomyocyte Proliferation and Cardiac Regeneration
Application
Therapeutics using siRNAs and small molecules to treat congenital heart defect and myocardial injuries.
Key Benefits
Reactivates cardiomyocyte (CM) cell replication, allowing regeneration of heart muscle after cardiac injuries.
Therapeutic compounds may be administered for transient regeneration/repair of heart muscle after cardiac...
Published: 2/13/2025
Contributor(s): Ahsan Husain, Nawazish Naqvi
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Targeted Anti-Cancer Nanoparticle Therapy
Application
A drug-carrying nanoparticle that selectively targets cancer cells.
Key Benefits
Incorporates an already proven anti-cancer therapeutic.
Increases deliverability.
Targets cancer cells.
Market Summary
Tris DBA palladium has been shown to have anti-cancer activity against melanoma and pancreatic cancer. However, Tris DBA has not...
Published: 4/3/2025
Contributor(s): Jack Arbiser, Lily Yang
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