Chris C. asks, "Do you have any indication of when the FDA is going to meet regarding Mela Sciences' (MELA) MelaFind application? I thought the date was early January but see no indication of such. Any insight or information you have is much appreciated." The last we heard from Mela was in November when an FDA advisory panel voted (surprisingly) to endorse the approval of the company's MELAFind skin cancer-detection device. The positive panel vote came over the strong objections of the FDA, which voiced strong objections to MELAFind's approvability. Advisory panel votes aren't binding on the FDA, and the agency doesn't have a deadline by which it needs to make an approval decision, so it's impossible to predict when that FDA decision may come. Mela has not had much to say publicly since November but I do hear CEO Joe Gulfo has been meeting privately with investors, saying he expects FDA approval and talking up MELAFind launch plans. Whether or not Mela has met with the FDA since the November panel isn't clear, although the company hasn't made any announcement about an FDA meeting.
Steve M. asks, "I was wondering if you could give us an update on Labopharm (DDSS) and their new
In a December Mailbag column about BioVest ( BVTI.PK) in which I expressed skepticism about the company's lymphoma vaccine BioVaxID, I wrote: "Investors and potential partners were clearly skeptical about the BiovaxID data, and rightly so because the results likely over-stage the vaccine's efficacy. The study, as designed, uses an unconventional accounting of patient responses, essentially not starting the disease-progression clock until six months after patients are treated initially with a standard regime of drugs used to put the cancer into remission." This prompted an email response from Biovest spokesperson Doug Calder: "I'm not quite sure I understand the point you are trying to make as the data reported as the 2009 ASCO Plenary Session measured disease-free survival for vaccinated patients (BiovaxID or control) from the time of randomization. And the patients had to complete chemotherapy and achieve a complete remission in order to be randomized. As the mechanism of action for active immunotherapy is to prime the immune system, it only makes sense to eliminate the immune-compromised state of the patient prior to vaccination, thus the trial design. If you are suggesting that the DFS clock starts six months following randomization, you are entirely incorrect. "Or perhaps you are questioning why patients were not vaccinated when randomized? The reason for this was related to an approximate 6-12 month delay to allow patients to have an immune recovery period (post-chemotherapy), again consistent with a vaccination approach. This delay is immaterial in measuring clinical benefit for the reason I mentioned before being that the results were measured from time of randomization for both cohorts." I'm sticking with my bearish thesis: The FDA will not approve BiovaxID based because the phase III study was flawed and produced biased results that skewed the true efficacy of the vaccine. I'm not the only person who believes this to be true. So did Dr. Ron Levy, the ASCO 2009 plenary session discussant who reviewed the phase III BiovaxID data. I encourage anyone conducting due diligence on Biovest to watch the 15-minute video of Levy's presentation that can be found on the ASCO web site. The more relevant reason Biovest chose not to randomize patients at the beginning of the study is because manufacturing the BiovaxID vaccine can take as long as six months. That means there wasn't any vaccine for patients to receive when they entered the study. By requiring patients to complete six months of chemotherapy first, then achieve and maintain a complete remission, Biovest eliminates a wide swath of lymphoma patients who don't respond to initial treatment. In other words, Biovest makes believe that some really sick patients don't exist when it comes to measuring the efficacy of BiovaxID. Said Levy, in his ASCO presentation: "The patients who made it to this
Mike emails, "Looking at February's calendar I noticed Cell Therapeutics (CTIC) is back for another run at the FDA. Just by skimming the headlines it looks like they managed to obtain improved numbers based on their latest study. What are your thoughts? Have you followed this since the rejection at all? Will you somehow be involved to give the play-by-play as you did at the panel meeting? That was great, not for Cell Therapeutics but for investors hanging on to the edge of their seats." Skimming headlines about Cell Therapeutics' lymphoma drug pixantrone isn't enough because the new data presented in December weren't really any different from data previously seen. Cell Therapeutics obviously disagrees, but I still view pixantrone as a me-too anthracycline which still lacks demonstrable proof of less cardiac toxicity. That's also the basic view of the FDA, which is why the agency rejected the drug last year. Moving on, Cell Therapeutics is appealing that FDA rejection with a meeting and decision expected in the first quarter, according to Cell Therapeutics spokesman Dan Eramian. If you're looking for a near-term trading catalyst for Cell Therapeutics, this is it, although the odds of FDA doing a 180-degree turn and approving pixantrone on appeal are very, very small. At the same time, Cell Therapeutics is preparing to conduct a new phase III study of pixantrone (dubbed PIX306) in patients with aggressive non-Hodgkin's lymphoma. Eramian says Cell Therapeutics is still negotiating with the FDA to reach agreement on a Special Protocol Assessment for the PIX306 study, but the company hopes to enroll the first patient in the current quarter. It's worth noting that Cell Therapeutics is already well past its initial timeline for the follow-on pixantrone study and SPA agreement. Last July, executives told investors to expect an SPA in place and first patient enrolled by November 2010, with the goal of having the study completed by the end of 2011. Well, it's January 2011 and the study hasn't even started, which suggests that the new pixantrone study results won't be ready until well into 2012.
I write about drugs and drug stocks but rarely do I write about, or hear from, patients. That's why the following email from Steve G. regarding Vertex Pharmaceuticals ( VRTX) and its hepatitis C drug telaprevir was a welcome change of pace: "Great coverage on Vertex vs. Merck as it relates to the horse race for Hepatitis C treatment. I personally hope that Vertex comes out the winner in market share. My interest is not from a financial gain perspective, but from that of being a successful clinical trial patient in the PROVE 3 clinical trial. I had previously failed the standard-of-care treatment with interferon and ribavirin back in 2003. I entered the clinical trial in May 2007 and later found out that I was actually viral free after only 2 weeks on triple treatment, which included telaprevir. I continued the full 12 weeks of triple treatment and then was given a placebo for the last 12 weeks with the interferon and ribavirin (total 24 weeks). The treatment was no cakewalk, but the telaprevir did not exacerbate any symptoms that I didn't already experience with the original standard-of-care treatment. I have maintained an SVR for over 3.5 years. I would call that a cure! I am one happy camper and feel quite blessed! Anyway, I thought I would toss in my obviously biased point of view. Keep up the great writing!" That's great news, Steve. Thanks for sharing. --Written by Adam Feuerstein in Boston. >To contact the writer of this article, click here: Adam Feuerstein. >To follow the writer on Twitter, go to http://twitter.com/adamfeuerstein. >To submit a news tip, send an email to: email@example.com.