Therapeutic Pipeline
The table below outlines RXi’s current therapeutic pipeline. The programs include a combination of internal development and collaborative efforts.
RXi’s Therapeutic Strategy
RXi’s therapeutic platform has the potential to be broadly applicable to multiple therapeutic areas. The company’s core focus leverages RXi’s unique technologies and competencies to advance potential candidates in select therapeutic areas with attractive market potential. RXi will also explore additional indications of strategic interest to the company which will be advanced internally and with partners through preclinical studies. As RXi executes on its core focus and strategic interest areas, additional areas may be pursued opportunistically.
Core Focus: RXi will focus its internal therapeutic development efforts in two main areas, anti-scarring and retinal disorders. The company is prepared to advance product candidates from these core areas into clinical development and through proof-of-concept. RXi would also consider partnering these product candidates either during or after this development process.
Dermatology: Anti-ScarringAnti-scarring is an attractive therapeutic indication with clear development precedent and limited competition for effective therapies. The company estimates that the US potential market for skin scarring is up to $4 billion with approximately 42 million skin-scarring surgical procedures annually. RXi’s sd-rxRNA™ (self-delivering rxRNA™) compounds have shown robust delivery and effective target silencing in skin using local administration. The company intends to select an anti-scarring development candidate in 2010 and file an IND in 2011. RXi’s success with RNAi therapeutics in anti-scarring may provide additional opportunities in other dermatology applications as well as in anti-fibrotic indications including pulmonary fibrosis, liver fibrosis, acute spinal cord injury, ocular scarring and restenosis, which together encompass a potential market size of more than $16 billion.
Ophthalmology: Retinal DisordersThere are multiple retinal diseases with large unmet medical need that are not addressed with currently available therapies. These diseases include wet and dry age-related macular degeneration, diabetic retinopathy and diabetic macular edema, which together affect approximately 18 million people in the US, and which have an estimated market potential of up to $20 billion. RXi has shown data demonstrating unprecedented delivery and effective target silencing in the retina with sd-rxRNA compounds. By applying its unique technology with existing and novel targets, and potentially multiple targets, the company believes it has the potential to develop next generation treatments for retinal disorders. Further, the company believes that there is opportunity to potentially improve on existing therapies, extend the time required between doses and utilize new modes of administration for delivery to the eye. The company intends to select a retinal disorder development candidate in 2011.
Strategic Interest: RXi will explore additional indications and proceed through preclinical development in areas that are of strategic interest to the company, partners or collaborators.
Neurology: Spinal CordRXi is exploring indications accessible by spinal cord delivery of sd-rxRNA. Direct dosing to the spinal cord could be used for severe central nervous system or spinal cord diseases in both orphan and non-orphan indications, both of which have multibillion dollar markets. RXi plans to leverage early proof-of-concept studies and collaborations to advance programs in this area. The company intends to advance potential candidates through preclinical studies and seek partners to help support further development.
Oncology: Liver Metastases and Hepatocellular carcinomaIn the area of oncology, RXi will be concentrating initially on liver metastases and hepatocellular carcinoma using systemic delivery of sd-rxRNA or rxRNA™ compounds in combination with delivery vehicles. These two disease areas of high unmet need have a US market potential of up to $3.2 billion and $800 million respectively. Given the emerging emphasis on multi-targeted therapies for cancer, RXi has the ability to develop RNAi compounds against multiple gene targets to generate effective combination treatments. The unique features of sd-rxRNA may offer a new alternative to treating cancers and could lead to attractive product candidates to further advance in conjunction with partners.
Opportunistic Pursuit: RXi will continue to evaluate multiple opportunities and is prepared to engage in active therapeutic development in cases where a program is funded by a partner or where RXi attains promising initial results. These opportunities include a variety of therapeutic areas, such as Hepatic disease (HCV infection), respiratory disease (asthma, COPD), additional oncological indications and other areas.
