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RXi Says Initial Rx Focus Will Be Anti-Scarring, Retinal Disorders


By Doug Macron

RXi Pharmaceuticals last week unveiled its therapeutic strategy, naming programs in dermal and ocular disorders as its key focuses, and achieving a corporate goal that the firm had failed to achieve under previous management.

These core areas, RXi President and CEO Noah Beerman said during a presentation at the Jefferies 2010 Global Life Sciences conference, "represent opportunities where RXi has the ability to take products into the clinic and through proof of concept."

Both of these programs will center around RXi's so-called self-delivering rxRNAs, which are designed to reach target tissues and organs without a delivery vehicle, and he added that the firm expects its dermatology effort to yield an investigational new drug application by the end of next year.

Meanwhile, RXi has also selected indications of strategic interest that "represent opportunities for us, alone and with partners, to take products through preclinical development," as well as "opportunistic areas [that] represent attractive opportunities, but ones that we have to de-emphasize until we have additional data on the core areas," he said.

Indications of strategic interest involve the spinal cord and liver cancer, while opportunistic areas include liver and respiratory diseases, fibrotic conditions, and various cancers.

Since it was spun out of CytRx in 2007, RXi has disclosed an interest in a variety of indications at different times, ranging from Alzheimer's disease and amyotrophic lateral sclerosis to metabolic conditions such as obesity and type II diabetes.

Under previous CEO Tod Woolf, the company had planned to select its first therapeutic candidate by the end of 2009 (GSN 6/25/2009). RXi missed this goal, and around the end of that year Woolf stepped down.

Upon taking the helm of RXi last November, Beerman made the establishment of a focused pipeline a key goal for the RNAi drug shop (GSN 11/19/2009), and in announcing the company's drug-development plans last week he achieved a goal for which industry watchers had long been waiting.

RXi's most advanced program is in dermal anti-scarring, and Beerman said during last week's conference that the company has a "number of candidates under evaluation." A lead candidate is expected to be chosen this year, with an IND filed before the end of 2011.

"Anti-scarring represents a tremendous opportunity … [with a] very high unmet need in this space [since there are] no prescription drugs available," with applications for a treatment ranging from cosmetic surgery to burn care and wound healing, he noted.

At the same time, RXi sees "a clear development precedent" for its program in a biologic called Juvista, which is under phase III development by Renovo in collaboration with Shire.

"We have a roadmap on what to do, [although] we may choose to do things similarly or differently," Beerman said. "We also know there is significant partnership potential as we move forward," which is key for the company since it has said that it will secure at least one industry partnership in 2010.

On deck for RXi in ocular disease are programs in retinal disorders, primarily wet and dry age-related macular degeneration, diabetic macular edema, and diabetic retinopathy, Beerman said.

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Approved treatments for these indications exist, but RXi said its candidate exploits well-validated targets and has ahead of it a well-marked path to commercialization.

Beerman also said he expects RXi's drug to have an edge over the competition given the "unprecedented delivery" to the back of the eye and "very effective target gene silencing" using intravitreally injected sd-rxRNAs.

Perhaps more important is the potential for RXi's compounds to offer "improved dosing flexibility or dosing modalities," he said.

For example, the gold-standard of care in wet AMD is Genentech's Lucentis, which is administered via intravitreal injection every two to four weeks. Beerman speculated that an RXi compound for the disease could potentially be dosed every six weeks.

"This is an area where we do have a competitive environment, but where we do have a truly next-generation product" to respond to it in the sd-rxRNA molecules, he said. It is also "an area where truly innovative therapies could have very significant market opportunities, and we expect to have selected a development candidate next year."

In terms of areas of strategic interest, which fall behind the core programs, RXi is focused on spinal cord injury, which "provides the potential for an early proof of concept [with] well-established targets for a number of [other] diseases," such as ALS, spinocerebellar ataxia, and chronic pain, according to Beerman.

Currently, RXi has data demonstrating direct delivery of sd-rxRNAs to spinal cord cells through intrathecal delivery in collaboration with Robert Brown of the University of Massachusetts Medical School.

In oncology, the company is keeping its eye on liver metastases and hepatocellular carcinoma, which would mark RXi's initial foray into systemic delivery.

"RNAi has certainly shown an increasing opportunity in oncology, and we believe the [sd-rxRNAs] in particular will have excellent uptake and penetration based on additional therapeutic development we're working on," Beerman noted.

Lastly, RXi broke out indications of opportunistic interest that the company is "excited about, but we will not invest in further unless it's through a partnership or if it leverages off of data from some of the other work," he said at the Jefferies event.

For example, targets in dermal anti-scarring are also related to a number of fibrotic indications, such as liver and pulmonary fibrosis, while the company's work in liver cancer could lay the groundwork for a program in hepatitis C, he explained.