Overview & Pipeline

Raptor is building a balanced, robust pipeline of drug candidates that may expand the reach and benefit of existing therapeutics. Raptor’s product portfolio includes both candidates from our proprietary drug targeting platforms and in-licensed and acquired product candidates.

Underlying all of our programs is what we believe to be a strong IP position that we actively manage and expand. Raptor's management team has extensive experience building successful companies and developing therapeutics from concept to market.

Investigational Product Pipeline (Potential Clinical Indications)

Scroll your mouse over the chart to link to relevant Raptor product details.

DR Cysteamine - Cystinosis DR Cysteamine - NASH DR Cysteamine - Huntingtons Convivia - ALDH2 Deficiency Convivia - ALDH2 Deficiency Tezampanel - Migraine Tezampanel - Migraine Tezampanel - Anti-Platelet NGX426 NGX426 Heptide - Hepatocellular Carcinoma/Hepatitis C Heptide - Hepatocellular Carcinoma/Hepatitis C Neurotrans - Neurodegenerative Diseases Neurotrans - Neurodegenerative Diseases WntTide - Breast Cancer WntTide - Breast Cancer

Our clinical-stage product pipeline includes three active development programs, and three product candidates for which we are seeking partners. Additionally, our preclinical pipeline includes one product candidate and three development programs based upon our proprietary
drug-targeting platforms, one of which is currently partnered with Roche.

Clinical Development Programs

Our three clinical development programs include an existing therapeutic that we are reformulating for potential improvement and application in new disease indications. These clinical development programs include the following:

DR Cysteamine is Raptor's proprietary delayed-release formulation of cysteamine bitartrate.

Raptor is seeking business development partners for three clinical-stage product candidates:

  • Convivia™ for the potential management of acetaldehyde toxicity due to ALDH2 Deficiency, an inherited metabolic disorder
  • Tezampanel and NGX426, non-opioids for the potential treatment of:

Convivia™ addresses ALDH2 deficiency. The ALDH2 enzyme is a component of the primary metabolic pathway for ethanol and other alcohols. Consumption of alcohol by individuals with ALDH2 deficiency leads to an accumulation of acetaldehyde, a known carcinogen, in blood, saliva, liver and other tissues. This condition is commonly known as alcohol intolerance or Asian Flush.

Tezampanel and NGX426, the oral prodrug of tezampanel, are first-in-class compounds that may represent novel treatments for both pain and non-pain indications. Tezampanel and NGX426 are ionotropic glutamate receptor antagonists that target the AMPA and kainate sub-type receptors. Published data show that during a migraine, increased levels of glutamate activate AMPA and kainate receptors, result in the transmission of pain and, in many patients, the development of central sensitization.

Preclinical Programs

Raptor's preclinical platforms consist of highly targeted therapeutics for the potential treatment of multiple indications, including liver diseases, neurodegenerative diseases and breast cancer.

Raptor's Receptor-Associated Protein (RAP) Platform

  • HepTide™ for the potential treatment of primary liver cancer and hepatitis C
  • NeuroTrans™ to potentially deliver therapeutics across the blood-brain barrier

Raptor's Mesoderm Development Protein (Mesd) Platform

  • WntTide™ for the potential treatment of breast cancer

The science behind these proprietary platforms leverages the binding capabilities of certain receptor chaperone proteins, including the tissue-specific binding of receptor-associated protein (RAP) and mesoderm development (Mesd). Raptor's scientists bioengineer variants of these proteins with specific tissue selectivity. These protein variants may then effectively transport drugs to the targeted tissue.

Raptor is also examining its glutamate receptor antagonists, tezampanel and NGX426, for the treatment of thrombotic disorder. Research conducted at Johns Hopkins University (JHU) by Craig Morrell, D.V.M., Ph.D., and Charles Lowenstein, M.D. demonstrated the importance of glutamate release in promoting platelet activation and thrombosis. Research shows that AMPA/Kainate receptor antagonists including tezampanel (NGX424) retard thrombus development in vitro and in vivo. This research identifies these receptors on as a new antithrombotic target with a different mechanism of action than Plavix®, aspirin or anti-IIb-IIIa biologics.