Year: 2013-14

Company: Raptor

Liaison(s): Kai Brown, Ben Cadieux

Raptor Pharmaceuticals Inc. is a publicly traded biopharmaceutical company that develops and commercializes therapeutic drugs for rare inherited diseases. The company was founded in 2005 by former executives from Biomarin Pharmaceuticals Inc., and they have been actively pursuing the development of multiple drugs in several disease indications. Their current lead product is a small molecule therapeutic known as Procysbi® (delayed release cysteamine bitartrate), which was approved for the treatment of nephropathic cystinosis in April 2013. Raptor is currently seeking to expand the utility of this active ingredient, RP103, into other potential indications such as for Huntington ’s disease (HD). In February of 2014, Raptor reported the completion of an 18-month Phase 2/3 clinical trial conducted by the Centre Hospitalier Universitaire d’Angers (CHU) in France, which indicated that the therapeutic administration of RP103 attenuated the total motor decline of HD patients. The current Standard Of Care (SOC) for HD management aims to treat chorea, one aspect of the motor decline experienced by HD patients. Given that there are currently no drugs that slow the disease progression, the CHU trial results indicate a promising potential for this first-in-class drug. In order to streamline the development and potential commercialization of RP103 for treatment of HD, the team focused on identifying signatures of HD diagnosis, disease progression and treatment outcomes. The project was divided into three phases. In Phase I, the team conducted an extensive literature review in order to establish the landscape of HD. This was accomplished by curating published data on HD pathogenesis, patient Quality Of Life (QOL), clinical trials, current SOC and alternative therapies. Additionally, this data was used as a basis of information for the next project phase. Phase II focused on designing questions for patient and Health Care Provider (HCP) surveys, which were conducted by a contracted market research firm. These surveys helped identify patient referral pathways, HD diagnosis protocols and unmet clinical needs through consistencies and discordances among primary and secondary research. In the final phase, the team identified key opinion leaders (KOLs) using quantitative weighting measures, and began to address the clinical need for HD patient outcomes research by utilizing a proprietary insurance claims dataset. It is expected the output from this TMP will give Raptor insights into signature issues associated with HD treatment and economics, and support the marketing strategies for RP103.