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siRNA for the treatment of Osteoarthritis

Product Description
R001/2 is a novel drug candidate for the treatment of early and late stage osteoarthritis. R001/2 is an intra-articular (IA), disease modifying osteoarthritis drug (DMOAD) that blocks cartilage degradation and inflammation through the inhibition of two key genes using a proprietary formulation of small interfering RNA. The drug was designed to have a long retention time in the joints of animals and people, thus limiting treatment to 4-6 injections per year.

Indication and Market
By 2030 an estimated 25 million adults (9.3%) in the USA will report arthritis-attributable activity limitations. These estimates may be conservative as they do not account for the current trends in obesity, which may contribute to future cases of osteoarthritis (source: Center for Disease Control). Pharmaceutical sales are expected to increase from $7.0B in 2012 to $14.0B in 2022. The greatest unmet need in OA is for disease-modifying agents, particularly if associated with pain-modulating effect. Preliminary analysis of the market opportunity for a DMOAD with clinically relevant impact on disease progression, an acceptable safety profile, and a market entry in the 2014 timeframe points toward an incremental global revenue forecast of $1 to 2 billion at peak penetration.

Product Rationale
Joint pathology in osteoarthritis is characterized by degradation of articular cartilage, along with bone remodeling (subchondral bone sclerosis and osteophyte formation), resulting in impaired joint function. Current therapies alleviate the mild to moderate pain and inflammation associated with OA, but do not protect the cartilage from further damage and have not demonstrated an effect on disease progression. Therefore, therapeutics that prevent or slow the alteration of joint structure and function will address a major unmet medical need. The primary cause of OA is inflammation and cartilage loss mediated by proteolytic degradation of aggrecan and type II collagen, key components of the cartilage extracellular matrix. R001/2 effectively blocks both the inflammation and cartilage loss associated with OA.

Preclinical Development
Intra-articular injection of R001/2 demonstrated both prophylactic and therapeutic efficacy (>80% suppression of inflammation and cartilage erosion) in the rat collagen induced model of OA at 2, 4 and 6 weeks. R001/2 blocks all components of the disease including fibrillation, fibrosis and cartilage erosion making it an attractive DMOAD. Administration of the drug does not cause local irritation or toxicity in rats after IA administration at efficacious doses. A proprietary formulation using polyarginine peptides has been optimized to keep the product in the knee cavity for more than 1 month allowing for infrequent IA dosing in OA patients.

Intellectual Property
GIBH has filed intellectual property rights for composition of matter, production and medical use of R001/2 for the treatment of arthritic diseases and other joint maladies associated with inflammation and matrix destruction.

R001/2 is a combination of two synthetic oligonucleotides in a formulation suitable for delivery into the knee and hip joints of patients.

Primary: Osteoarthritis
Secondary: Rheumatoid arthritis

Syringe, containing R001/2 in solution for optimal, intra-articular injection, directly into the joints of patients.

Development Status
Lead optimization: Preclinical proof-of-concept in an industry-standard model of arthritis established. Candidate selection of the drug is scheduled for early 2014 and IND filing is to be initiated in 2014.

Commercialization Strategy
Commercial partners are being sought for development and marketing in China, the EU and North America.


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