Skip to main content
Premium Trial:

Request an Annual Quote

RXi Selects Candidate in Anti-Scarring Program as Promised, But Will Likely Miss Partnership Goal

Premium

By Doug Macron

RXi Pharmaceuticals last week announced that it had selected a lead candidate from its anti-scarring program as the first compound it will move into human testing, meeting a previously stated corporate goal for 2010.

And while company's other goal for the year — the establishment of an industry partnership — remains an overall objective, RXi President and CEO Noah Beerman indicated that it may not happen until 2011.

RXi said that it has chosen RXI-109, which targets an undisclosed gene associated with fibrosis, as the focus of its first clinical drug-development effort. An investigational new drug application for the agent is expected to be filed before the end of 2011, with an initial indication of reduction of surgical scarring.

“Dermal anti-scarring is an excellent match for RXi’s … therapeutic platform,” CSO Anastasia Khvorova said in a statement. “The disease biology is well understood, the targets are well validated, and intradermal injection is an acceptable administration route."

Notably, RXI-109 employs RXi's so-called self-delivering RNAi technology, which is designed to reach target tissues and organs without a delivery vehicle. In January, the company reported in vitro and in vivo data showing that the molecules are efficiently taken up by a variety of cells, and are effective in vivo when administered either systemically or locally (GSN 1/21/2010).

About six months later, the company announced that its anti-scarring and retinal disease programs, both of which are based on the self-delivering approach, had become its primary areas of focus (GSN 6/17/2010).

With "a clear development precedent" set for the anti-scarring program by Renovo and Shire's phase III scar treatment Juvista, Beerman said at the time that the dermatology effort would yield an IND in 2011, although a lead candidate had yet to be chosen.

With the selection of RXI-109, the firm has hit "a significant milestone … [that] demonstrates the company’s commitment to achieving our corporate goals and executing on our strategy," he said in a statement released last week.

Indeed, settling on a compound to move into the clinic has long been an important objective for the company, albeit an elusive one. Since it was first spun out of CytRx in 2007, RXi has expressed interest in a variety of disease areas including amyotrophic lateral sclerosis and type II diabetes.

Under its previous CEO, the company was aiming to select a lead candidate by the end of 2009 (GSN 6/25/2009), but failed to do so. When Beerman took the company's reins in late 2009, he made defining RXi's pipeline a key goal (GSN 11/19/2009), but it wasn't until this summer when he unveiled dermal and ocular conditions as the firm's initial focus that he committed to any kind of timeline.

In September, Beerman presented proof-of-concept in vivo data on both programs (GSN 9/16/2010). In studies on rat skin, injections of self-delivering RNAi agents knocked down as much as 78 percent of the dermal mRNA target, and the silencing of the target gene lasted for at least seven days after the last injection. Meanwhile, in mouse eye models, self-delivering molecules demonstrated uptake and localization to the mouse retina after 24 hours.

A lead candidate from the retinal disease program is expected to be chosen next year, he said at the time.

But even though it remains on track with its planned drug-development efforts, it appears that RXi will fall short on its promise of closing at least one corporate partnership before the end of 2010.

In April, Beerman announced that in addition to selecting a lead drug candidate in 2010, RXi was "confident" that RXi would secure at least one industry alliance — it's first — before year end (GSN 4/1/2010).

"The corporate partnership is an important piece of all RNAi companies' … strategies, and certainly we're no different," he said at the time. "We continue to be very aggressive in all of our efforts and … continue to have active discussions with a number of different potential partners on a variety of different opportunities."

However, in a statement announcing the selection of RXI-109 last week, Beerman said RXi is "focused on completing a corporate partnership," but that it only anticipates being able to do so "in the coming months," even though there are less than two weeks left in 2010.

If RXi doesn't manage to close a partnership before year end as promised, it wouldn't be the first time it missed such guidance. Most recently, the company's former CFO Stephen DiPalma said that a partnership would be consummated before the end of 2008.

Officials from RXi did not return a request for comment.


Have topics you'd like to see covered in Gene Silencing News? Contact the editor
at dmacron [at] genomeweb [.] com

The Scan

Push Toward Approval

The Wall Street Journal reports the US Food and Drug Administration is under pressure to grant full approval to SARS-CoV-2 vaccines.

Deer Exposure

About 40 percent of deer in a handful of US states carry antibodies to SARS-CoV-2, according to Nature News.

Millions But Not Enough

NPR reports the US is set to send 110 million SARS-CoV-2 vaccine doses abroad, but that billions are needed.

PNAS Papers on CRISPR-Edited Cancer Models, Multiple Sclerosis Neuroinflammation, Parasitic Wasps

In PNAS this week: gene-editing approach for developing cancer models, role of extracellular proteins in multiple sclerosis, and more.