RXi Pharmaceuticals Corporation (OTCQB: RXII) is a biotechnology company focused on discovering, developing and commercializing therapies leveraging its breakthrough RNAi delivery platforms. After spinning off from Galena Biopharma Inc. (NASDAQ: GALE) in April of 2012, the company has made significant strides developing its proprietary delivery platforms and has acquired Opko Health Inc.’s RNAi assets (NASDAQ: OPKO) as of March 2013.
In this article, we’ll take a look at RNA and its potential therapeutic potential, analyze how RXi Pharmaceuticals’ RNAi-based platforms and compounds improve upon the current therapeutics, and then take a look at why investors might be interested.
What Exactly Is RNA?
Ribonucleic acid (“RNA”) is a macromolecule that performs multiple vital roles in the coding, decoding, regulation and expression of genes. When cells need to produce proteins, they activate the portion of DNA that codes for a given protein, which produces multiple copies of that piece of DNA in the form of messenger RNA, or mRNA. These mRNA are then used to translate the genetic code into protein using the cells’ manufacturing ribosomes.
Aside from acting as a DNA photocopier for protein manufacturing, RNA can act as an enzyme (“ribozyme”) to speed up chemical reactions. The macromolecule also plays an important role in regulating cell processes, while defects in RNA have been implicated in a number of clinically important diseases, including heart disease, some cancers, stroke and others. As a result, RNA has come to the forefront of clinical research over the past few years.
RNA Interface and Its Applications
A particularly interesting area of RNA research is RNA interference (“RNAi”), which is a biological process where RNA molecules inhibit gene expression by destroying mRNA. These processes can play an important role in not only defending cells against viruses and other parasitic nucleotide sequences, but also in directing gene expression on a more generic level. In diseases where certain genes are overexpressed, for instance, RNAi may be able to help regulate them.
By 2002, RNAi was selected as the “Breakthrough of the Year” by the journal Science, bringing the new process into the limelight. And in 2006, the Nobel Prize in Medicine was awarded to the co-discoverers of RNAi, including Dr. Craig Mello, a co-founder of RXi Pharmaceuticals. The novel approach to drug development processes enables scientists to potentially highly selectively treat any one of the thousands of human genes, including those untouchable using other modalities.
Breakthrough Platform Technology
The problem with RNAi therapeutics has traditionally been delivering the compounds to the right areas in the body. While encapsulating RNA in a lipid-based particle has improved circulation time and cellular uptake, the process remains far from efficient at achieving the desired response rate in a reasonable amount of time. Apart from increasing side effect profiles of the treatments, these additional delivery vehicles also add uncertainty to the compound’s overall efficacy depending on various conditions.
RXi Pharmaceuticals has taken an entirely different approach by building drug-like properties in to the RNAi compound itself. These “self-delivering”, proprietary, and novel compounds have been developed under the trade name sd-rxRNA®. The company’s suite of such compounds can be used to treat a variety of acute and chronic diseases, using both local and systemic administration, providing a significant competitive advantage in the field.
Robust Therapeutic Pipeline
RXi Pharmaceuticals has developed a robust therapeutic pipeline led by RXI-109, a self-delivering RNAi compound (sd-rxRNA®) being developed for the reduction of dermal scarring in planned surgeries. In two Phase I clinical trials, the compound was well tolerated with no serious local or systemic side effects observed, and the company expects to report full top-line results during the second quarter of this year, with Phase II clinical trials following afterwards.
Currently, there are no FDA-approved drugs to prevent scar formation, meaning a therapeutic of this type could have great benefit for trauma and surgical patients, as a treatment during the surgical revision of existing unsatisfactory scars, and in the treatment, removal and inhibition of keloids – scars that extend beyond the original skin injury. With approximately 42 million surgical procedures per year, the market could be worth up to $4 billion annually in the U.S.
The company also has a number of other earlier stage therapeutic candidates in development, but remains largely focused on developing its RXI-109 before investing significant time and capital into them. These assets encompass those purchased in March of 2013 from OPKO Health, including 12 patent families with claims relating to important biological targets believed to play a role in diseases of the eye, cancer, immune disorders and inflammation.
Potential Investment Opportunity
RNA plays an important role in gene expression, making it an important clinical target for therapeutics companies. With its robust clinical pipeline and extensive experience with RNAi, RXi Pharmaceuticals represents a unique play on the space. The company’s lead candidate has significant promise targeting a large $4 billion per year market with no FDA-approved treatments, while its innovative platform yield significant long-term potential in many areas.
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