RXi Pharmaceuticals  Corp. is at a crossroads.
The small Worcester-based company, potentially on the cutting edge of one of biology’s most promising frontiers, struggled through the recession as many early-stage public biotech companies did: with little cash and wavering investor confidence.
But in the past few months, the company has narrowed its therapeutic focus to eye and skin diseases, won a $600,000 grant from the National Institutes of Health, signed a deal with a global corporate partner and sought to shore up its share price by diversifying its investor portfolio.
RXi has, until now, conducted early-stage preclinical work in RNA interference. RNAi prevents genes from producing disease-causing proteins by binding to and destroying targeted messenger RNA molecules to silence the bad gene. Researchers believe shutting off these genes could treat a variety of diseases.
The company announced in June it would narrow its focus, initially, to skin and eye conditions, because the company feels most confident about the delivery methods for the technology in those areas.
CEO Noah Beerman said the company planned to file two Investigational New Drug applications — the first step in the U.S. regulatory process — by 2012. The company is pursuing a drug candidate to treat scarring and estimates that the potential market in the U.S. is up to $4 billion with approximately 42 million skin-scarring surgical procedures annually.
In ophthalmology, the company plans to pursue several eye diseases that, when combined, affect approximately 18 million people in the United States and have an estimated market potential of up to $20 billion, Beerman said.
“Like a lot of new trends in biotech, initially, there is a lot of excitement. Then people get working on it and realize it’s 20 years to get somewhere,” said Steve Yoo, vice president at Boston boutique life sciences bank Leerink Swann.
Yoo said that the small number of human trials and lack of long-term safety studies in the field are beginning to weigh on investors.
RXi (Nasdaq: RXII) is working to blunt against potential weariness in the market. The company announced a partnership with Netherlands-based Royal Philips Electronics, whose health-care division has a strong presence in ultrasound technology, to pursue image-guided drug delivery as a potential way to administer RNAi therapies. Financial terms were not disclosed.
This type of narrow partnership is likely to become more common in the RNAi field, analysts say, as big corporate partners become more wary of broad technology platform deals with no specific focus.
RXi has a challenge outside of advancing its technology and attracting partners, and that is to diversify its portfolio of investors. The company spun off from Los Angeles-based CytRx Corp. in 2007, which had owned almost half of the company until the last several months.
“There has been selling pressure on the stock, not because of the fundamentals of the company, but because CytRx has sold 2 million out of its 5 million shares,” Beerman said.
He added that both sides are working to reduce CytRx share of the company and diversify its investor mix, in a methodical way. To that end, last month, investor Kevin Tang and his associated companies bought 952,369 shares of RXi, 5.18 percent, according to regulatory documents.
RXi employs about 30 people and plans to hire about a half-dozen more. The company had a net loss of $3.9 million in the first quarter of 2010, down from a loss of $4.2 million for the corresponding period last year. The company had cash and cash equivalents of $14.8 million on March 31, up from $14.8 million on Dec. 31, 2009.
RXi’s stock closed at $2.31 on Tuesday, up a penny from the previous close of $2.30. The company’s stock has traded between $1.51 and $8.99 over the past year.