- The "good" complete response letter is one in which FDA tells Orexigen that Contrave is approvable without the need for new pre-approval clinical studies or new efficacy and safety data. Full approval of Contrave would be granted later in 2011 after the two sides reach agreement on a post-approval trial to assess cardiovascular safety and nailing down a risk management plan. A "good" complete response letter pushes Orexigen's stock price higher.
- The "bad" complete response letter is one in which FDA tells Orexigen that pre-approval clinical trials and/or efficacy and safety data are required before Contrave can be cleared for marketing. A "bad" complete response letter sends Orexigen's stock lower. How low depends on how much and what type of data FDA requires for approval. If the FDA goes against the wishes of the advisory panel and asks for a pre-approval cardiovascular outcomes study, Orexigen is cooked extra crispy. If the data request can be fulfilled without a new clinical trial and still leaves open the possibility of a 2011 Contrave approval, the stock may only fall a bit or even stay relatively flat.
Jim Johnson emails, " Cel-Sci ( CVM) announced that it started the Phase III clinical trial for Multikine. Is it me or is Cel-Sci the expert in taking old information and/or already-announced information, publishing it to the wires and reeling in new bag holders on small price-per-share jumps? Is this company for real or is this just another PR tactic?" You're being too harsh on Cel-Sci! I think it's great that the company has taken investors on an around-the-world tour as it issues press releases on each and every country allowing the Multikine phase III study to proceed. Personally, I've used the Mulitkine press releases to help my kids learn geography: Russia, Ukraine, Taiwan, Hungary, Mississipi (a state, not a country, I know), India. It takes a village to raise a child but a mini-U.N. to conduct a Multikine phase III study. The important thing missing from all these Cel-Sci press releases is an announcement of the first patient enrolled in the Multikine study. Where are the actual patients eager for treatment with Multikine? Cel-Sci plans to enroll 880 patients in the phase III study -- an ambitious enrollment goal, for a drug with a paucity of phase II data. The Multikine data that do exist shouldn't inspire much confidence at all in the drug's efficacy. I wrote about the multiple problems and shortcomings of the Multikine phase II study last year, in two parts. If you're interested in following what will likely be the very slow progress of the Multikine phase III study, I'd watch for the number of U.S. hospitals open to enrollment. Right now, that list stands at one -- North Mississippi Health Services of Tupelo, Miss., according to ClinicalTrials.gov. A diminutive roster of U.S. enrolling sites is a red flag -- indicative of underwhelming interest in Multikine amongst U.S. doctors. Cel-Sci plans to seek approval of the drug here and the FDA will want to see a significant amount of data from U.S. patients.
Sticking with the obesity drug theme, Mark M. emails a question regarding Vivus ( VVUS): "I appreciate your balanced and unemotional reporting of Qnexa and other weight-loss drugs as they attempt to gain FDA approval. Other than your articles, there seems to be a significant amount of bias in most of the reporting on weight-loss drug developments, maybe a reflection of positions held. In the case of Qnexa, I do not really understand how the FDA can question the safety of low-dose topiramate when they have already approved its use in other higher dose applications. Perhaps you can address this issue in a future article." It's a great question. The thing to keep in mind is that the risk-benefit of any drug depends largely on the disease being treated and the patient population who will take the drug. Let's start with an easy example. Cancer drugs, generally, cause significant side effects. That makes sense because a lot of chemotherapy drugs, for instance, are basically poison designed to kill tumor cells. Healthy cells are often collateral damage, too. Doctors, patients and the FDA are okay with the high toxicity of cancer drugs because they're used to treat patients with cancer, a fatal disease that is usually (not always) worse than whatever toxicity or side effects the treatment may cause. If chemotherapy drugs were used to treat a less serious and non-fatal disease like psoriasis, the risk-benefit trade off would be unacceptable to everyone. Psoriasis can be a nasty disease, but patients aren't going to die from it. Moreover, psoriasis is a chronic disease, meaning patients require long-term treatment. If they stop taking whatever drug they're using to keep the psoriasis at bay, the disease will return. Again, chemo drugs would never be acceptable to treat psoriasis even if the efficacy was fantastic because the cumulative toxicity of long-term exposure would likely kill patients.
Mike J. asks, "Follow you regularly, I appreciate your work. Did the Ariad Pharmaceuticals ( ARIA) move surprise you? I was thinking ponatinib would be more the future driver of this stock." Ariad shares 34% to $7.32 intraday on Jan. 28 following the release of positive results from the phase III study of ridaforolimus ('rida) in sarcoma. The stock has since settled back to around $6.76 as I write this column Thursday. Aria was a $2 stock in early 2010. Mike is correct in that a lot of investors are looking past 'rida and focusing more on ponatinib. 'Rida's clinical development and most of the drug's economics are controlled by Merck ( MRK). The Mtor inhibitor class of cancer drugs to which 'rida belongs is competitive and the 'rida data in sarcoma -- while meeting the primary endpoint with statistical significance -- are not without concerns.
Dan O. emails, "Hello Adam, I am a long-time member of RealMoney Silver and without doubt your insights are my most treasured reading. I have long followed Avanir Pharmaceuticals ( AVNR) and would appreciate any updates you might share. One would suspect that something is askew at Avanir headquarters? Why would a stock with such promise act so sickly when it looks to have such potential." Avanir is supposed to launch Neudexta in February as a treatment for pseudobulbar affect (PBA). Until the launch occurs and script data starts flowing, investors will probably be sitting on the sidelines. That's a fairly well-worn script in biotech investing these days. Drug gets approved, stock pops, then sells off on concerns about commercialization. Stock recovers and does well if drug sells well (Alexion Pharmaceuticals) or remains underwater if drug performs poorly (Vanda Pharmaceuticals.)
Pat. W. asks, "Adam, I have been following Discovery Labs ( DSCO) for many years and I thinking it's time to invest in Surfaxin getting a favorable review. My concern is buying before a possible reverse stock split and/or continued dilution of the stock, but I like your optimistic view of Surfaxin. What price per share range would you give Discovery if Surfaxin gets approval? Thanks." I was feeling more snarky than usual Thursday morning, so in response to Discovery's announcement of yet another conference call scheduled to explain the 4,672nd delay in the Surfaxin resubmission process, I tweeted this: "Memo to $DSCO CEO Tom Amick: STFU! No conf calls. No PRs. Not until you get Surfaxin approved. No one cares until then -- 2015? 2020?" And I mean it. Amick really need to shut his mouth and just get the job done. Enough explanations, enough excuses. Talk to investors after Surfaxin is approved. Until then, his words are useless. As for investing in Discovery, haven't you been hurt enough already? Revisit the stock when Surfaxin is approved, if ever. --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.