Prospect Profile: Raptor Pharmaceutical Corp.


Prospect Profile highlights a newly funded bio/pharmaceutical company. This includes important insights into the featured company’s product pipeline, manufacturing and business relationships, and likely sourcing opportunities.

Raptor Pharmaceutical Corp. is a U.S. public pharmaceutical company that develops treatments for metabolic disorders. As reported in the July 3, 2014 issue of the PharmSource Lead Sheet, Raptor raised $70 million in the completion of an IPO.

Corporate Highlights

  • Raptor Pharmaceutical Corp. was incorporated in Delaware in May 2006.
  • Corporate Headquarters: Novato, California – 21,330 square feet of office and laboratory space.
  • As of December 31, 2013, Raptor had 70 employees.

Sourcing Opportunities

  • Raptor plans to use the funds for expansion of the company’s manufacturing capacity, expansion of commercial operations in Europe, advancement of the company’s development programs and general corporate purposes, including working capital.
  • The company has no internal manufacturing capabilities. During 2014 and throughout most of 2015, Raptor expects to continue to rely on suppliers for API and third-party manufacturers for the conversion to finished drug product.
  • Raptor relies on CROs, academic institutions, major medical institutions, or contract manufacturing organizations to conduct, supervise or monitor some or all aspects of clinical trials involving product candidates.
  • The company relies on third parties for pharmaceutical services and distribution of PROCYSBI in the US and the EU.

Business Relationships

  • In June, 2009 Raptor entered into a collaboration and licensing agreement with Roche to evaluate therapeutic delivery across the blood-brain barrier (BBB), starting with Raptor’s NeuroTrans BBB transporter platform. Under the terms of the agreement, Roche will fund studies of select molecules attached to NeuroTrans, a derivative of the receptor-associated protein. The agreement provides Roche with an exclusive worldwide license to NeuroTrans for use in the delivery of diagnostic and therapeutic molecules across the BBB. Raptor received an initial upfront fee for the collaboration to cover its portion of the initial studies, along with development milestone payments and royalties.


Product Candidate Indication Dosage Form Status Next Anticipated Step
Cysteamine dioxygenase Diseases for which cysteamine is a therapeutic TBA Preclinical TBA
HepTide Hepatocellular carcinoma TBA Preclinical TBA
Convivia ALDH2 deficiency (Ethanol intolerance) Oral Phase II TBA
RP103 Leigh syndrome Delayed-release Capsule Phase II IND submitted for Phase IIb in Dec. 2013. Interim results expected by the end of 2014.
RP103 Non-alcoholic fatty liver disease in children Delayed-release Capsule Phase IIb Phase IIb results expected in H1 2015
RP103 Huntington’s disease Delayed-release Capsule Phase II/III Plans to apply for orphan drug designation in the EU
PROCYSBI Nephropathic cystinosis Delayed-release Capsule Marketed N/A



(In $ thousands) 2013 2012
Revenues 16,872
R&D Expenditures 29,177 8,963
General & Administrative Expenditures 37,948 8,971
Total Operating Expenses 68,778 17,934
Capital Expenditures 1,603 55


Contact Information

Raptor Pharmaceutical Corp. Key Officers
5 Hamilton Landing, Suite 160 Christopher M. Starr, PhD, CEO
Novato, CA 94949, USA Georgia Erbez, CFO
Phone: 415-408-6200 Todd C. Zankel PhD, CSO
Fax: 415-382-8002 Patrice P. Rioux, MD, PhD, CMO
Web: Mary Jo Bagger, Senior Director, Clinical Operations

Ryan is a Senior Research Analyst at PharmSource. He holds a BS degree in biology, with a minor in chemistry and a BA degree in Spanish, from the University of North Carolina at Chapel Hill. Prior to joining PharmSource, Ryan worked as a Research Technician in the Neuroscience Center of the UNC Chapel Hill School of Medicine, and has been a named author on research publications.

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