HCV News Extravaganza
Sep08

HCV News Extravaganza

Apparently everybody in the hepatitis C race was busy over the holiday weekend, as Tuesday brought a flood of news from the sector. There was good news, bad news, and an acquisition. Last things first: the acquisition. Bristol-Myers Squibb announced it will fork over $885 million for Zymogenetics, its partner in the development of pegylated interferon-lambda, in Phase II trials to treat hepatitis C (HCV). If you’ll recall, last year BMS paid $85 million upfront and a $20 million licensing fee for access to the drug. Under that arrangement, the Seattle-based biotech would have scored up to $430 million in milestones if the therapy actually made it to patients. Given Zymogenetics product pipeline and its one marketed product, Recothrom, the $885 million price tag doesn’t sound so outlandish. Interferon-lambda uses the same cell-signaling pathway as interferon-alfa, one of the two cornerstones of current HCV therapy. But as we wrote earlier this year, because interferon-lambda has fewer functions in the body than interferon-alfa, it is expected to be as effective with milder side effects. Onto the bad news: Idenix Pharmaceuticals said FDA put a clinical hold on two of its hepatitis C drugs, IDX184 and IDX320, due to liver toxicities in a small trial testing the safety of giving both drugs to healthy people. The company’s stock took a beating on the news, with shares falling by 47% to close at $3.18 yesterday. The question now is which of the molecules is causing the elevated liver enzymes. Leerink Swann analyst Howard Liang commented on the issue in a note to investors this morning: “The lack of association between the liver toxicity signals and IDX184 exposure and more extensive safety data on IDX184 would suggest us to that IDX320 is more likely the culprit than IDX184, which is the more important asset in our view." And the good news (part 1): Vertex Pharmaceuticals released more positive Phase III data for telaprevir, its much-anticipated protease inhibitor for HCV. The drug candidate was tested in some of the toughest patients—those who didn’t respond to or had only a partial response to the standard of care (pegylated interferon and ribavirin) or whose disease relapsed after standard of care. Vertex said 65% of those HCV patients were “cured” when adding telaprevir to the treatment regimen, compared to 17% in the control arm, which was given just the standard of care. Take a look at the company’s press release for more details on each segment of patients, but the relapsers had the most success with treatment, with a smaller portion (31%) of the folks that didn’t respond at all to interferon and ribavirin seeing complete suppression of...

Read More
Vertex Unveils Positive Telaprevir Data
May25

Vertex Unveils Positive Telaprevir Data

Ending months of anticipation, Vertex Pharmaceuticals unveiled the first set of data from a Phase III trial of telaprevir, a protease inhibitor for the treatment of hepatitis C. The company is expected to submit for regulatory approval later this year, and launch the drug in 2011. Vertex said that 75% of genotype-1 patients—viewed as the toughest to treat--who received 12 weeks of telaprevir treatment on top of the current standard of care (48 weeks of pegylated interferon and ribavirin) were cured of the infection. Only 44% of patients in the control arm, which received only the standard of care, were cured after 48 weeks. Leerink Swann analyst Seamus Fernandez told investors the results set an "impressively high bar" for treatment in HCV. Importantly, adding the drug to the standard of care will lessen the total treatment time for many HCV patients. In addition to not being very effective, many people can’t tolerate the harsh side effects associated with interferon and ribavirin. Physicians liken the 48-week regimen to living with a nasty flu for a year. In hopes of halving the number of weeks on interferon and ribavirin, Vertex conducted what is called a “response-guided trial.” If the virus was sufficiently quelled after four weeks with the telaprevir addition, patients went on to receive just 24 weeks total of therapy. The company said “the majority” of patients received just 24 weeks of treatment. That 24-week figure is a critical one. Merck is hot on Vertex’s heels with its own protease inhibitor, boceprevir. Both companies are expected to launch their drugs next year, and with similarly mild safety issues, analysts say the drug that can shut down the disease the quickest will win. Merck is also conducting a response-guided study and at its R&D day said a retrospective look at its Phase II data suggests patients can be successfully treated in 24 weeks with boceprevir. BMO Capital Markets analyst Jason Zhang was dead on with his estimates for the drug. As we wrote earlier this month: Zhang expected the Phase III data to show a sustained viral response (the equivalent of a cure) of 75% of patients receiving telaprevir. His guess for telaprevir’s biggest competitor, Merck’s boceprevir? 74% sustained viral response. We’ll have to wait and see how close he comes on that figure, as Merck has been vague about when its Phase III results will be released. The big pharma firm has only said that it expects to present the data at an upcoming conference. My guess? We will likely be waiting for the American Association of Liver Disease’s annual shindig, also known as “The Liver Meeting,” in October. For...

Read More
Post-BIO News Roundup
May07

Post-BIO News Roundup

Been focusing on Chicago and this year's BIO extravaganza all week? Here's a sampling of news you might have missed. Pirfenidone rejected In a surprisingly twist, FDA refused to approve InterMune’s lung treatment pirfenidone, despite a positive recommendation from its advisory committee. The agency wants another lengthy trial to better demonstrate pirfenidone is effective at treating idiopathic pulmonary fibrosis, a debilitating and ultimately fatal lung disease for which there are no approved treatments in the U.S. or Europe. InterMune’s stock fell over 75% on the news. Check out this piece in Forbes' health care blog on whether FDA is these days less likely to listen to its advisory panels. Birth Control Pill Exalted The mainstream media celebrated as "The Pill" turned 50. Technically, they're celebrating the 50th anniversary of its approval by FDA. C&EN covered the chemistry story of the pill in "The Top Pharmaceuticals That Changed The World" special issue, back in 2005. Don't miss the classy 1950s era photo of Carl Djerassi. Resveratrol Trial Halted A GlaxoSmithKline clinical trial studying a reformulated version of resveratrol was suspended on April 22 due to safety concerns, but company officials say the complications may or may not be related to the drug. The big question remains-what does this all mean for the effort to make drugs out of resveratrol, the trace component of red wine that's been touted as a cancer fighter and a fountain of youth in a bottle? Resveratrol research has been in the spotlight a lot lately, not just for good reasons. GSK got the reformulated resveratrol (also called SRT-501) when it acquired biotech company Sirtris in 2008, to the tune of $720 million. Sirtris based its business around evidence that resveratrol turned on enzymes called sirtuins. The belief was that this activity could underlie some of resveratrol's beneficial effects. Sirtris developed other drug candidates based on this idea that the company says look nothing like resveratrol, and some of those entitites are in clinical trials as well. But the sirtuin connection has been called into question on multiple occasions. And now, with news that some patients in the resveratrol clinical trial developed cast nephropathy, a condition that can lead to kidney failure, the Wall Street Journal Health Blog is wondering aloud whether resveratrol's 15 minutes of pharmaceutical fame are coming to an end. GlaxoSmithKline officials say they are studying the data further, and that they stopped the trial "out of an abundance of caution", according to the Wall Street Journal. The trial was conducted in patients with multiple myeloma, and apparently, cast nephropathy is common in myeloma patients. Commenters at "In the Pipeline" (which, by the...

Read More
The Race For the Next Big Thing in HCV
May03

The Race For the Next Big Thing in HCV

All spring, biotech watchers have been anxiously awaiting Phase III data for two new drugs to treat Hepatitis C, Vertex Pharmaceutical’s telaprevir and Merck’s boceprevir. Both drugs are expected to be approved next year, ushering in a new era in the treatment of HCV. This week’s cover story takes a look beyond that first wave of new drugs for HCV to assess the pipeline of second-generation compounds. After all, improving cure rates by adding a direct-acting antiviral like telaprevir or boceprevir to the current standard of care (PEG-interferon/ribavirn) will be great, but creating a cocktail of small molecules that work on their own would be even better. As the article notes, everybody wants to be the Gilead Sciences of the HCV market. Gilead has cornered the HIV market with a pill that combines three antivirals in one, and is hoping to unroll a four-in-one pill soon. Only there's a catch: unlike in HIV, where there is a steady stream of new infections each year, the rate of new infections in HCV has slowed considerably. As such, there will be a flood of patients seeking treatment--and ideally be cured of the disease--over the next decade, after which industry observers expect the patient pool to shrink. Industry observers expect to see more licensing and M&A activity in the HCV world as companies with antivirals in the late stages of development seek partners with compounds with complementary activity to their own drugs. "Larger companies cannot afford to wait five to six years for clinical development," says Decision Resources analyst Alexandra Makarova. "Its not even a choice of saving money—either you are late for the bus or not. You have to partner with someobody developing drugs in phase II or late phase I." Some deals have already been made, enabling the first studies of combinations of small molecules in the absence of interferon and ribavirin: -Roche, which has a vested interested in maintaining its leading position in the HCV market, partnered with Pharmasset in 2004 for PSI-6130, a nucleoside polymerase inhibitor that the companies later turned into the prodrug R-7128. Two years later, it snagged InterMune’s ITMN-191, for $60 million upfront and up to $470 million in milestones. The companies will split sales of ITMN-191 down the middle. Roche has already conducted a small clinical trial in New Zealand testing the efficacy of using a combination of R-7128 and ITMN-191 together. -Gilead has had mixed luck in its deal-making: the company entered into a HCV development with Achillion Pharmaceuticals in 2004, but later killed development of GS-9132 after it had unwanted side effects. In 2008, Gilead ended a four-year HCV collaboration with Genelabs....

Read More