Routes of
administration Oral Biological half-life 24-36 hours PubChem CID 44137686 | ATC code none CAS Number 1165910-22-4 Formula C14H12F6N2O | |
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Legal status US: Investigational New Drug IUPAC ID 4-((R)-2-((R)-2,2,2-trifluoro-1-hydroxyethyl)pyrrolidin-1-yl)-2-trifluoroMethyl)benzonitrile | ||
LGD-4033 (VK5211, Ligandrol) is an investigational selective androgen receptor modulator (SARM) for treatment of conditions such as muscle wasting and osteoporosis, discovered by Ligand Pharmaceuticals and currently under development by Viking Therapeutics.
Contents
Commercial Environment
Viking Therapeutics does not currently manufacture any pharmaceutical products, but is developing several products to treat metabolic and endocrine disorders. Thus, the company relies upon investments from stockholders and business partners. The company completed its Initial Public Offering in the 2nd quarter of Fiscal Year 2015, generating $22.3M in net proceeds. Viking Therapeutics assumed the largest expense in 2014 from cited Research and Development costs ($21.2M), which may be related to purchase of required equipment and facilities to develop the drug candidates licensed by Ligand Pharmaceuticals in that year, including VK5211.
Financial Risks of Development
Viking Therapeutics is yet to bring a drug to market. If one or several of their drug candidates does not pass clinical trials before one is successfully brought to market, then a significant amount of stockholders would likely sell their stocks, seriously reducing revenue and capability to continue development of VK5211. Additionally, as noted in the Form 10-Q submitted on 10 November 2016, Viking currently relies heavily upon licensed technologies by Ligand Pharmaceutics. If Viking Therapeutics were to lose this license, then its operating capability would be severely impacted.
Other Commercial Aspects
Ligand Pharmaceutics has disclosed that its royalty fee incurred upon Viking Therapeutics for production and sale of VK5211 would be 7.25-9.25% of the product revenue.
Another promising drug candidate, VK2809, is expected to drive a major shift in the market cap of Viking Therapeutics and its share of the market for TRβ agonists, which is currently dominated by Madrigal Pharmaceuticals. This would improve the company portfolio as a whole, and would allow for greater capitalization upon the potential success of VK5211.
Intellectual Property
Ligand Pharmaceuticals has patents filed for the manufacture and therapeutic use of certain compounds occupying several stated chemical structures, which are intended for use as selective androgen receptor modulators. The patent is filed under the following designations: US8519158 B2, US8865918, US9359285, US20070254875, US20140005186, US20150099720, and WO2005090282A1. The patents will expire on March 12, 2025. These patents effectively protect any future capitalization upon VK5211 in the market by Viking and Ligand through their licensing agreement.
Regulatory Information
VK5211 is currently an Investigational New Drug.
Pre-Clinical
Oral administration of the drug to cynomolgus monkeys at daily doses varying from 0 to 75 mg/kg over 13 weeks demonstrated significant body weight gain in both males and females. After 48 days, the 75 mg/kg dose testing was halted due to toxicity concerns, but this did not negatively impact development as the dose is significantly higher than those being utilized in the Phase 2 clinical trial.
Clinical Trials
In a Phase 1 clinical trial of 76 adult male humans in which the dose size was varied, a dose-dependent increase in lean body mass was observed with no significant adverse events over 21 days.
The Phase 2 clinical trial, initiated on 3 November 2016, consists of 120 patients recovering from hip fracture surgery. The randomized study participants will receive either a placebo or varying dose sizes of VK5211 over a period of 12 weeks, with improved lean body mass as the primary endpoint. Other endpoints include satisfactory results in terms of quality of life, safety, and pharmacokinetics.