RXi Pharmaceuticals Presents New Preclinical RNAi Data At The Keystone Mechanism And Biology Of Silencing Conference

RXi Pharmaceuticals Corporation (Nasdaq: RXII), a recognized leader in RNA interference (RNAi) based therapeutic discovery and development, today announced that new preclinical data using proprietary self-delivering RNAi (sd-rxRNA™) compounds, including RXI-109, will be presented at the Keystone Symposia’s Mechanism and Biology of Silencing conference being held March 20-25, 2011 in Monterey, California.
“RXi has made significant scientific progress and the data to be presented at this conference will highlight important advancements in our technology, platform and therapeutic programs. We have developed a novel class of self-delivering RNAi compounds with improved drug-like properties that may enable the development of therapeutics for a wide range of indications, including our lead development candidate, RXI-109, for the reduction of dermal scarring,“ said Anastasia Khvorova, Ph.D., RXi’s Chief Scientific Officer.

RXi Pharmaceuticals Obtains Direct Ownership of Previously Licensed RNAi Delivery Intellectual Property

RXi Pharmaceuticals Corporation (Nasdaq: RXII), a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi), today announced that it has recently acquired direct ownership of technology for which it had previously exercised its option to exclusively license from Advirna LLC. The novel acquired technologies potentially enable in vivo delivery of RNAi therapeutics. Terms of the agreement were not disclosed.

Tod Woolf, Ph.D., President and Chief Executive Officer of RXi, stated, “RXi’s self-delivering rxRNA™ (sd-rxRNA™) technology platform, which is based on a combination of this technology acquired from Advirna and RXi internally developed technology, has shown extremely promising results in pre-clinical studies. This prompted us to acquire direct ownership of the technology instead of merely licensing it. By combining this newly acquired technology into RXi’s own portfolio and gaining full control of the patent prosecution, we enhance the potential of obtaining broader claims that would further strengthen RXi’s intellectual property position.”

The broad clinical potential of Advirna’s technology in combination with RXi’s rxRNA™ platform, which includes sd-rxRNA’s, may enable RXi to develop a robust and sustainable RNAi therapeutic platform with significant competitive advantages in efficacy, toxicity, ease of administration and manufacturing costs when compared to traditional RNAi compounds. The unique capabilities of this technology are generating substantial interest in the scientific and business community.

RXi Pharmaceuticals to Webcast Presentation at the Rodman & Renshaw Annual Global Investment Conference

RXi Pharmaceuticals Corporation (Nasdaq: RXII), a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi), today announced that the Company`s President and Chief Executive Officer, Tod Woolf, Ph.D., will present a corporate overview at the Rodman & Renshaw Global Investment Conference on Friday, September 11, 2009 at 11:15am ET, at the New York Palace Hotel in New York City.

Dr. Woolf will also be participating in a panel discussion on RNAi Therapeutics, taking place on Thursday, September 10, 2009 at 8:25am ET.

A live webcast of the presentation will be available on the "Investors" section of the Company's website, www.rxipharma.com. A replay of the presentation will be available for 30 days.

RXi Pharmaceuticals CEO to Present a Session Keynote at IBC’s Oligonucleotide Therapeutics Conference

WORCESTER, Mass.--RXi Pharmaceuticals Corporation (Nasdaq: RXII), a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi), today announced that the Company’s President and CEO, Tod Woolf, Ph.D., will deliver a session keynote presentation at IBC’s Oligonucleotide Therapeutics - From Concept to Creation Conference, which is part of Drug Discovery and Development Week, taking place August 3-6, 2009 in Boston, MA.

RNA interference (RNAi) technology has been heralded as a major breakthrough in science with the potential to block the expression of disease-causing genes. Dr. Woolf’s keynote “Thirty Years of Targeting RNA: A Retrospective” will explore the great variety of therapeutic oligonucleotide properties and how these characteristics provided insight on designing compounds with “self-delivery” properties, or delivery to cells and tissues without the use of a separate delivery vehicle. His presentation will begin at 9:10 am on Monday, August 3, 2009.

Dr. Woolf will also participate in a CEO panel discussion on RNA therapeutics that brings together leaders in the RNAi field to discuss the current and potential states of development, as well as how companies are capitalizing on the promise of this new class of drugs. The session will be held from 11:50 to 12:30 on Tuesday August 4, 2009. Furthermore, Dr. Woolf will serve as a panelist in a roundtable discussion on the intellectual property landscape in RNAi therapeutics at 4:15 on August 3, 2009.

In addition to the Keynote address and the CEO panel discussion by Dr. Woolf, the conference also includes a presentation by RXi’s Vice President of Technology Development, Dmitry Samarsky, Ph.D., on the “Development of Novel RNAi Therapeutic Compounds and In Vivo Delivery Approaches,” as well as a presentation by one of RXi’s collaborators, Gary Ostroff, Ph.D., using a technology platform licensed by RXi, the Glucan Encapsulated RNAi Particle (GeRP) delivery system.

IBC’s Drug Discovery and Development Week, to be held at the World Trade Center in Boston, MA, expects more than 3,000 pharmaceutical, biotechnology, government and academic researchers, executives, and technology providers to participate. The five targeted scientific conferences at this event focus on practical strategies and forward-looking approaches to help accelerate small molecules, antibody therapeutics and oligonucleotide therapeutics from early discovery to the clinic.

RXi Pharmaceuticals Acquires Exclusive License to Proprietary, Potentially Paradigm-Changing RNAi Delivery Technology from Advirna LLC

WORCESTER, Mass.--RXi Pharmaceuticals Corporation (Nasdaq: RXII), a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi), today announced that it has exercised its option to an exclusive worldwide license to novel technologies from Advirna LLC to enable in vivo delivery of RNAi therapeutics. Terms of the agreement were not disclosed.

The RNAi delivery technologies licensed from Advirna encompass four distinct approaches, with the primary focus on “self-delivery” of RNAi therapeutics; that is, RNAi delivery without the use of a separate delivery vehicle. In evaluating the Advirna technology, RXi observed that this technology delivers RNAi molecules to cells spontaneously upon direct administration, both in vitro and in vivo, without requiring a delivery vehicle or transfection formulation. RXi has reproduced this result in many cells, and has seen activity even with cells that are difficult to transfect.

Tod Woolf, Ph.D., President and Chief Executive Officer of RXi, stated, “The ability to create self-delivering RNAi therapeutics promises to provide significant competitive advantages in efficacy, toxicity, ease of administration and manufacturing costs. This unique capability is creating a great deal of interest in the scientific and business community and we believe this exciting technology could simplify clinical development of RNAi therapeutics and could position RXi to take the lead in the area of RNAi therapeutics.“

The delivery technology licensed from Advirna has been integrated into RXi’s rxRNA™ platform to create self-delivering rxRNAs (sd-rxRNAs) with the following potential advantages:

Spontaneous cellular uptake: delivery of RNAi therapeutics without the added expense of liposomes, complexes, nanoparticles or other delivery vehicles Patient-friendly administration routes: subcutaneous administration to treat patients without the need of medical professional supervision Bio-distribution to targeted tissues: distribution of rxRNAs to certain tissues while reducing renal clearance Reduced side effect profiles: reduced side effects that can be associated with formulations that contain an added delivery vehicle RXi is integrating its comprehensive delivery platform, including the new technologies licensed from Advirna, with its proprietary rxRNA™ compounds to develop a sustainable pipeline of products for unmet medical needs. RXi has a strong IP position in both RNAi compounds and delivery approaches and is using its scientific expertise in these areas to identify lead therapeutic candidates for inflammatory diseases, such as rheumatoid arthritis, diabetes, and Crohn’s disease, as well as other indications.

RXi Pharmaceuticals Presented Breakthrough RNAi Delivery Technologies at the TIDES® 2009 Conference

WORCESTER, Mass.-RXi Pharmaceuticals Corporation (Nasdaq: RXII), a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi), today announced that the Company’s Chief Scientific Officer, Anastasia Khvorova, Ph.D., presented new pre-clinical data on novel RNAi therapeutic approaches at IBC’s TIDES Oligonucleotide and Peptide® Technology and Product Development Conference in Las Vegas, Nevada. This conference is one of the longest-running, most prestigious conferences on oligonucleotide-based therapeutics, a field of drug development that includes RNAi. The conference was not webcasted.

Regarded as a revolutionary discovery in biology, RNA interference (RNAi) is a naturally occurring mechanism whereby short, double-stranded RNA molecules interfere with the expression of genes in living cells. RXi’s world-leading scientists have made significant advancements in this technology and Dr. Khvorova presented new forms of potential RNAi drugs with improved therapeutic properties.

Tod Woolf, Ph.D., President and CEO of RXi Pharmaceuticals commented: “Our research team is making significant progress, and I am very impressed with the amount of robust scientific data generated by our laboratory. The breakthrough technologies presented yesterday by Dr. Khvorova have the potential to address multiple multi-billion dollar therapeutic markets, may lead to improved therapeutics for many currently untreatable diseases and will help RXi realize its goal of becoming the technology leaders in the RNAi therapeutics space.”

RXi presented data on the following therapeutically advanced RNAi compounds and delivery technologies:

Glucan Encapsulated RNAi Particles (GeRPS) – The GeRP technology delivers RNAi compounds directly to macrophages. RXi has been building on the work published in an April 30th Nature article reporting positive pre-clinical data demonstrating that administration of RNAi therapeutics effectively reduces systemic inflammatory response. RXi has brought the GeRP technology in-house, established manufacturing in its own laboratory and has advanced the technology to the level necessary for further pre-clinical development.

Self-delivering RNA (sdRNA) – The proprietary self-delivering-rxRNA™ technology promotes spontaneous cellular uptake of the RNAi therapeutic, which is similar to the uptake of traditional small molecule drugs. Without the use of an additional delivery vehicle, this RNAi delivery technology potentially results in greater efficacy along with lower costs and side effects attributed to a delivery formulation. RXi believes that self-delivering rxRNA™ should support direct application or subcutaneous administration, opening the door for many clinical areas of RNAi therapeutic development.

Advancements to rxRNA™ compounds – Improvements have been made to rxRNA™ compounds allowing them to work in concert with the new delivery technologies. Efficient cellular uptake is critical for an effective therapeutic and advancements have been made to minimize rxRNA™ compounds to improve delivery. These minimized compounds also reduce the immune response and are easier and less costly to manufacture. These advancements are proprietary to RXi and encompass novel and unconventional chemistries when compared to the current siRNA molecules.

The combination of these new advanced technologies may enable RXi to discover and develop drugs for multiple indications and to build a sustainable pipeline of products for unmet medical needs.

RXi Pharmaceuticals Announces Publication in Nature of Positive Pre-clinical Data Demonstrating that Oral Administration of RNAi Therapeutics Effectiv

RXi Pharmaceuticals Announces Publication in Nature of Positive Pre-clinical Data Demonstrating that Oral Administration of RNAi Therapeutics Effectively Reduces Systemic Inflammatory Response

WORCESTER, Mass.-RXi Pharmaceuticals Corporation (Nasdaq: RXII), a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi), today announced the publication of an article in the journal Nature that presents new pre-clinical data demonstrating that low dose (20 μg/kg) oral administration of RNAi therapeutics resulted in a significant reduction of a systemic inflammatory response in an established mouse model of inflammation.

This important work on oral delivery of RNAi compounds was published by Michael Czech, Ph.D., co-founder of, and Scientific Advisor to, RXi Pharmaceuticals and Professor and Chair of Molecular Medicine and Professor of Biochemistry and Molecular Pharmacology, University of Massachusetts Medical School (UMMS) (Aouadi, et al, Nature, Vol. 458 (7242), pp. 1180-1184). RXi Pharmaceuticals has the exclusive worldwide rights to certain patent applications claiming aspects of the technology behind the oral delivery of RNAi therapeutics upon which this work was based under a license agreement entered with UMMS in October 2008.

Commenting on today’s news, Tod Woolf, Ph.D., President and Chief Executive Officer of RXi Pharmaceuticals, noted, “Oral administration of RNAi therapeutics using this particular technology is a major scientific breakthrough that was not previously thought practical. As a result of the publication of Dr. Czech’s data in Nature, one of the premier scientific journals, it now appears that this method may constitute a significant advance in RNAi delivery. This new orally available, low dose RNAi therapy may be able to control many severe inflammatory diseases without the need for more invasive types of administration such as injection, which is the current standard of care for approved non-RNAi therapeutics. We believe that oral RNAi therapeutics, if successfully developed, could address the multi-billion dollar markets of rheumatoid arthritis, Crohn’s disease, atherosclerosis, inflammatory bowel disease and diabetes. As a result, this new technology provides many potential scientific and commercial opportunities for RXi Pharmaceuticals, and we are focusing our resources on this exciting therapeutic area.”

In the published work, which was funded by UMMS and the National Institutes of Health, oral delivery of micron sized RNAi encapsulation vehicles made of beta 1,3 D glucan (called “GeRPs”) target macrophages in the gut that then migrate to tissues throughout the body. Gene silencing of the cytokine TNF-alpha or the protein kinase MAP4K4 that controls TNF-alpha expression was observed in isolated immune cells of treated mice, as compared to controls, after oral administration of the respective GeRP-encapsulated RNAi compound. Silencing of the MAP4K4 target gene mRNA level was observed in macrophages isolated from spleen, liver and lung tissues. Additional experiments featured in the publication showed that the gene silencing with this technology and delivery of unmodified siRNAs lasted about eight days following the termination of oral administration.

The data presented in Nature shows that the GeRPs efficiently target immune cells and that efficient gene silencing can be observed specifically in macrophages. In addition, the work revealed that MAP4K4 is a novel mediator of cytokine expression, providing an opportunity to use this target to attenuate the inflammatory response in human diseases.

Michael Czech, Ph.D., noted, “Macrophages are key cells which orchestrate immune responses in man. Our collaboration with Gary Ostroff, Ph.D., Professor of Molecular Medicine, and my colleague here at UMMS, has led to encouraging results that show oral delivery of RNAi compounds is feasible for reducing inflammatory gene expression in these cell types. We are very excited about this technology and the potential to move it forward in collaboration with RXi Pharmaceuticals.”

Earlier this year, a collaboration between RXi Pharmaceuticals and the UMMS research team received funding from a Massachusetts Life Sciences Center Cooperative Research Grant to further develop this technology. The three-year grant of up to $250,000 per year, which is part of the state’s $1 billion, 10-year life-science initiative, will help fund the use of RXi Pharmaceutical’s rxRNA™ compounds to advance the progress of oral administration of RNAi therapeutics.

RXi Pharmaceuticals has an agreement with UMMS whereby UMMS has granted RXi an exclusive license to certain patent applications to this technology and co-exclusive options to other underlying technology. UMMS and inventors of this intellectual property may receive developmental milestones and/or royalty payments pursuant to the agreement.

RXi Pharmaceuticals Appoints Richard Chin, M.D. to Board of Directors

WORCESTER, Mass.-RXi Pharmaceuticals Corporation (Nasdaq: RXII), a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi), today announced that Richard Chin, M.D. has been appointed to the Company’s Board of Directors, effective as of April 23, 2009.

Dr. Chin is a physician with extensive industry experience in biologic and small molecule drug development. He is currently the Chief Executive Officer of Institute for OneWorld Health, a nonprofit pharmaceutical company developing drugs for neglected diseases in developing countries. Dr. Chin joined OneWorld Health from OXiGENE (Nasdaq: OXGN), a company developing vascular targeting drugs for oncology and ophthalmology, where he served as President and Chief Executive Officer. Previously, Dr. Chin served as Senior Vice President and Head of Global Development for Elan Corporation. Dr. Chin has also held various clinical and scientific roles for Genentech, Inc. including Head of Clinical Research for the Biotherapeutics Unit, at one point overseeing the clinical development of approximately half of the drugs at Genentech, and began his career in industry at Procter & Gamble Pharmaceuticals, where he served as Associate Medical Director.

Dr. Chin has a strong track record in the drug development industry, and he was responsible for initiating over 40 new drug programs (IND’s), and bringing 8 new drugs to market. He is an authority on clinical trials, and has authored a textbook, Principles and Practice of Clinical Trial Medicine.

He received a B.A. in Biology, magna cum laude, from Harvard University and law degree with honors from Oxford University in England under a Rhodes Scholarship. Dr. Chin holds a Medical Degree from Harvard Medical School and is licensed to practice medicine in California. He previously served on the Adjunct Clinical Faculty of Stanford University School of Medicine and currently serves on the Board of Directors of Genmedica, located in Barcelona, Spain.

Sanford Hillsberg, Chairman of RXi’s Board of Directors noted, “Dr. Chin is an outstanding addition to our Board. Dr. Chin has a wide range of experience in the drug development industry and will provide a wealth of knowledge and insight as RXi Pharmaceuticals moves into developing its RNAi therapies. He brings experience from some of the world’s leading drug development companies, including Genentech, Procter and Gamble Pharmaceuticals and Elan Pharmaceuticals and he has direct experience with getting new therapeutics to the IND stage and through the FDA. Dr. Chin will provide experienced support and advice as RXi moves into the next stage of its drug development.”

“I look forward to working with the entire RXi team and help them realize the considerable potential of their RNAi therapeutic product candidates. They have a promising future,” stated Richard Chin, M.D. incoming director. “I am honored to be part of this exciting, growing company that is working to create some truly innovative new therapeutics.”

“We are fortunate to attract such a talented and seasoned biopharmaceutical expert to our Board of Directors,” said Tod Woolf, Ph.D., President and Chief Executive Officer of RXi Pharmaceuticals. “We are delighted to welcome Richard to our board and I look forward to working with him. “

The Board has determined that Dr. Chin qualifies as an independent director under the categorical independence standards set forth in the Company’s Corporate Governance Guidelines, as well as applicable Securities and Exchange Commission and Nasdaq requirements. Dr. Chin’s appointment represents an addition to the RXi Pharmaceuticals Board of Directors, bringing the total number of board members to seven, five of whom are independent directors.