Targeted Drug Therapeutics An overview of our technologies and their potential 505(b)(2) applications Sino-American Regulatory Consulting Alliance Yongtian Ni (On behave of our partner) December 4, 2011

An overview of our technologies and their potential 505(b)(2) applications Our Mission and Experience  Develop innovative therapies for millions of patients not meeting their treatment goals. *Near-term disease targets: metabolic diseases (e.g., diabetes, diabetic complications, cardiovascular, dyslipidemia), alcoholism, osteoporosis, infectiousdiseases, dementia related disorders. * Long-term: Develop new therapies for cancer, vascular diseases, and arthritis.  Scientific Focus Areas: * Solve difficult drug targets * Solve formulation issues (small molecules, peptides, proteins) * Solve patient compliance and safety issues * Develop next generation drug products (with improved efficacy and safety) * Develop new drug delivery platforms for different disease targets  Addressable global market: Billions  Team experience: Supported 18 NDAs/ANDAs, 5 INDs, 6 drug candidates at preclinical/preformulation stage that resulted in 14 commercial drug products.  Global research collaborations in United States, Europe, and Asia SARCA

Unmet Medical Needs That We Aim to Fulfill We target patient population that are not meeting treatment goals. Our approach:  Improve safety-efficacy profile (e.g., combination drugs providing synergistic effects)  Simplify dosing by providing fixed dose combo-formulation  Improve convenience by provide sustained release formulations  (i.e., fewer injections (via QD, BIW, QW dosing), small needle (31G))  Reduce treatment cost (i.e., increase drug potency, minimize packaging) SARCA

Solving A Common Problem with Most Peptides/Proteins Most peptide/protein drugs have very short in vivo half-life (minutes to hours) and high concentration fluctuations in blood. Thus, multiple daily doses (e.g., BID,TID, or QID) are often necessary to balance efficacy and toxicity. We address the problem by developing controlled release and long-acting formulations using stealth technologies that are capable of providing sustained delivery of drug(s) while maintaining it’s blood concentration within the therapeutic limits. Our aim is to minimize side effects associated with spikes and need for frequent dosing. This is particularly important when treating chronic conditions. SARCA

Our (nano) Drug Delivery Platform SARCA

Unique Drug Delivery Platform (Improved Safety-Efficacy Profile and Cost-Effectiveness) SARCA  Good product appearance * Translucent solution * Uniform particle distribution (40 nm, monodisperse,thermodynamically stable)  Safe formulations * GRAS excipients with good safety records  Convenient administration * Reusable or disposable pen device * Flexible dosing * 31gauge needle * No needle clogging issue  Long product shelf-life * Delivery system has 2+ year stability  Cost-effective manufacturing * Efficient manufacturing process (hours vs days required for polymer microsphere) * Scalable process

Later Stage Drug Product Candidates SARCA

Early Stage Drug Product Candidates SARCA

Summary SARCA We has a full range of R&D capabilities:  Designed prospective drug products with superior potency, safety, pharmaceutical properties (several hundreds candidates in our drug library)  Solved a broad range of pharmacology and pharmaceutical sciences issues  (e.g., solubility, safety, combo-drug compatibility, patient compliance)  Developed sustained release formulations (daily to weekly dosing options)  Developed innovative combination drug regimes  Developed efficient manufacturing process (large-scale batch produced in hours)  Demonstrated proof-of-concepts in animal subjects  Integrated NCE discovery-drug delivery approach, providing effective product life-cycle management (patent/market exclusivity, formulation patent exclusivity)