Overview & Pipeline

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

Raptor's clinical-stage product pipeline includes three active development programs based on the Company's proprietary delayed-release formulation of cysteamine bitartrate (RP103 capsules and RP104 tablets), as well as the Company's ConviviaTM (oral 4-methylpyrazole) program which the Company licensed to Uni Pharma Ltd., Taiwan, and tezampanel. Raptor's preclinical pipeline includes two development programs based upon the Company's proprietary drug-targeting platform, and one in-licensed preclinical product candidate..

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 Tezampanel - Anti-Platelet Heptide - Hepatocellular Carcinoma/Hepatitis C Neurotrans - Neurodegenerative Diseases WntTide - Breast Cancer

Underlying all of Raptor's programs is a strong portfolio of intellectual property that Raptor actively manages and expands. Raptor's management team has extensive experience building successful companies and developing therapeutics from concept to market.

Clinical Development Pipeline

Raptor has three active clinical development programs based on its proprietary delayed-release formulation of cysteamine bitartrate, an existing therapeutic that Raptor has reformulated for potential improvement and application in new disease indications. These clinical development programs include:

RP103 is Raptor's proprietary delayed-release microsphere formulation of cysteamine bitartrate in capsules. Immediate release cysteamine bitartrate capsules are currently marketed for the treatment of cystinosis. RP103 may require less frequent dosing and reduce gastro-intestinal side effects compared to the current standard of care.

RP104 is Raptor's proprietary delayed-release formulation of cysteamine bitartrate in tablets.

Raptor's other clinical development programs include:

  • Convivia™(oral 4-methylpyrazole) for the potential management of acetaldehyde toxicity due to ALDH2 deficiency, an inherited metabolic disorder (Phase 2, licensed to Uni Pharma Ltd., Taiwan)
  • Oral tezampanel, a non-opioid glutamate receptor antagonist as a potential anti-platelet agent

Convivia™ is a Phase 2 product candidate that 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 is a first-in-class compound that may represent a novel treatment for both pain and non-pain indications. Tezampanel is an ionotropic glutamate receptor antagonist.

Six Phase 2 trials with Tezampanel have shown analgesic activity.  The FDA has previously agreed that Tezampanel may move into Phase 3 for acute migraine.

Raptor is examining tezampanel 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 retard thrombus development in vitro and in vivo. This research identifies these receptors as a new antithrombotic target with a different mechanism of action than Plavix®, aspirin or anti-IIb-IIIa biologics.

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 other liver diseases
  • NeuroTrans™ to potentially deliver therapeutics across the blood-brain barrier

Raptor's Mesoderm Development Protein (Mesd) Product Candidate

  • 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.