Last week, Vertex Pharmaceuticals ($VRTX) got FDA approval for Kalydeco, the first cystic fibrosis drug to address the disease's cause, rather than just its symptoms. The price tag: $294,000. But as observers pointed out at the time, that's not as expensive as it gets. Today's drugs for debilitating and rare diseases command 6 figures almost as a matter of course. Medical Billing & Coding's blog reminds us just how much--and shows where Kalydeco ranks. Story

Shares of NeurogesX, already badly beaten up over the past year, tanked after regulators raised questions about the efficacy of its pain patch for HIV-related pain. Its stock ($NGSX) dropped to as low as 75 cents a share and then recovered somewhat by midmorning as analysts sifted through the agency's remarks, which are intended to help shape an expert panel review coming up Thursday.

Wedbush analyst Gregory Wade tells Bloomberg that he was none too surprised to see the critical remarks. And even though he doesn't believe that the in-house regulatory review of the application will prevent a vote in its favor, any approval still wouldn't do much to improve the company's prospects.

About 1 in 3 HIV patients suffers from HIV-related neuropathy, notes NeurogesX. But one of the regulators doesn't believe that they'd be well served by Qutenza.

"It would not be in the best interest of these patients for us to approve a product for which substantial evidence of efficacy has not been demonstrated, or one for which the benefits do not clearly outweigh the risks," said Bob Rappaport, director of the agency's division of anesthesia, analgesia and addiction products, according to the Bloomberg report.

- here's the article from Bloomberg

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After burning through more than $122 million, the late-stage osteoporosis drug developer Radius Health is filing to go public in the hope of raising up to $86 million. Radius is betting that it can whet the appetite of investors with the prospects for its pivotal trial for BA058. But over the past three years investors have been quick to pass on such offerings, put off by the heavy risk of failure that dogs every drug program.

Radius Health in-licensed BA058 from Ipsen and has been studying the treatment's ability to build bones in osteoporosis patients. As FierceBiotech noted in December, when the Cambridge, MA-based biotech drew down another tranche of its last $91 million venture round, Radius believes that it can significantly improve on current treatments, which are designed to slow bone loss and reduce pain in patients. Radius is also partnered with Eisai on a midstage treatment for hot flashes without the side effects associated with hormone therapy.

Radius, which is helmed by former Genzyme CFO Michael Wyzga, plans to list on Nasdaq under the RDUS symbol. The biotech did not list the number of shares it plans to offer or a price range.

Biotech IPOs have generally received rough handling on Wall Street. With the help of its backers, Christoph Westphal pulled off a successful initial offering for Verastem ($VSTM) recently. But Cempra's experience last week is more common. The company had to cut its share price in half and boost the number of shares sold to raise $50 million. Merrimack, a Boston biotech with five programs, recently pulled its IPO, citing market conditions.

A successful IPO here would be a coup for Radius' investors: MPM Capital, BB Biotech Ventures, MPM Bio IV NVS Strategic Fund, The Wellcome Trust, HealthCare Ventures and Scottish Widows Investment Partnership.

- here's the S-1 form on file at the SEC
- read the story from the Boston Business Journal

Special Report: The 10 biotech IPOs of 2011

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Shares of Orexigen Therapeutics ($OREX) shot up 18% yesterday after the biotech laid out a special protocol assessment pact with the FDA, detailing the design of a big new safety study of its weight drug Contrave. But investors are going to have to wait quite awhile before they get a better idea of the developer's chances at a comeback.

Orexigen made headlines when it raced Vivus ($VVUS) and Arena ($ARNA) to the FDA with an application for a weight drug, vying for the lead in a race for the first new obesity drug approval in more than a decade. But the biotech was handed a major defeat when regulators concluded that they needed hard evidence that a treatment designed to be used by a mass audience didn't have any hidden cardio risks. Orexigen then lined up $87 million in new funding for the study, vowing to complete the quest.

In the SPA, Orexigen and the FDA agreed that investigators will recruit 10,000 patients and then see how long it takes to hit 87 major adverse cardio events in the patient population. Once they hit that stage--which the developer estimates will be in less than two years--Orexigen will come up with an interim analysis and see if it can win an approval with the data.

"A few months ago, we received detailed written correspondence from the FDA's Director of the Office of New Drugs that identified a clear and feasible path forward for this important potential obesity therapy," says CEO Michael Narachi. "We believe the rapid progress we have since made with the FDA's Division of Metabolic and Endocrinologic Products on the detailed protocol and plans for analysis is further indication of the alignment we have reached within the FDA on the requirements for resubmission of the Contrave NDA."

- here's the press release
- read the Reuters story
- get the report from the San Diego Business Journal

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Merck says that vorapaxar, its troubled blood thinner once considered the jewel in Schering-Plough's crown when the pharma giant bought it for $41 billion, hit its primary efficacy endpoint in a second major late-stage study. But Merck ($MRK) investigators also noted that while the drug--which proved too dangerous to continue the first study to its conclusion--looked positive on the top line, patients taking it also suffered from a significant increase in bleeding.

Vorapaxar has caused a lot of trouble for Merck, which once hoped that it had a blockbuster treatment on its hands when the deal for Schering-Plough went through. A year ago the company shut down the first big study after investigators tracked a dangerous increase in the risk of bleeding.

In this second study, TRA-2P, researchers were testing the treatment using a composite score for cardiovascular death, heart attack and stroke, paying particular attention to whether it would prevent clots. The data will be reviewed in March.

"In developing vorapaxar, Merck and our scientific collaborators set a very high bar--would the addition of vorapaxar to standard of care provide incremental benefit in preventing clots?" said Peter S. Kim, president, Merck Research Laboratories. "We are pleased that TRA-2P met its primary endpoint, and we look forward to discussing the results with the scientific community."

So where does it go from here? For Merck's part the pharma company says it will ponder all the data and then consider its options, including a potential regulatory filing. With one monumental pratfall and mixed results for the second study, plenty of questions beg to be answered first.

- here's the press release

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