Can someone please walk me through what just happened with this stock? I'm not sure how to feel about the sudden drop? Are the documents bad? Good? Nobody really knows? Is this normal before ODAC to have the stock drop this far? In the past I have seen other companies increase before events of this nature. So, what's going on here? Manipulation? I just can't get a good read from any of the posts that are currently on the MB.
Can a long please answer these questions for me? Or shorts that have any clue whatsoever? I honestly don't need to hear about AF or any of the other jackasses that are playing games, I just need to hear an educated response.
Watching my account slowly dwindle, but still holding out for a great year with TLON.
Here are the key issues:
1) Are there any treatments approved for this stage of ALL? No, the FDA documents state this openly. There are options, such as increasing the dosage of previously used chemo, but these patients have already failed/refractory to previously used chemo, so this is a fall back strategy but not an approved/established strategy. So this meets the requirement for Accelerated Approval. Talon's clinical presenter tomorrow will need to emphasize this fact from the clinical perspective.
2) Is the treatment providing a meaningful clinical benefit? The benefit is complete response in the range of 15-20% (depending on the definition used), and 17% of patients receiving Marqibo went on to received a potentially curative stem cell transplant. The benefit from Marqibo was either direct (complete response) or indirect (disease stabilization that allowed further treatment and then stem cell transplant). The Talon Clinical Presenter tomorrow will need to emphasize this positive clinical benefit.
3) Is Marqibo safe? Marqibo benefits from 30 years of vincristine use with known side effects and effective management strategies of these side effects. The FDA agrees that Marqibo does not result in side effects different from or worse than vincristine, although the FDA notes that Marqibo also doesn't appear to have any better safety profile than traditional vincristine.
4) Has Talon demonstrated an acceptable risk/benefit? The most recently approved treatment for 1st Failure in ALL, Nelarabine, had a complete response rate of 23% (in healthier, 1st failure, patients that the Marqibo trials, 2nd/3rd failure) and had a similar percent of patients going on to stem cell transplant while at the same time was a brand new medicine with new side effects. Marqibo achieved similar clinical outcomes as Nelarabine in the same type of Ph. 2, single arm, single agent clinical trial design. Nelarabine was approved under the Accelerated Review Subpart H program at the FDA, and so will Marqibo.
Now, why the price drop. FDA briefing documents are very clinically focused and emphasize any possible safety issue. The FDA decided that the complete response rate was not 20% as stated by Talon, and was instead either 17% or 15% depending on how they defined response in one particular patient. The FDA also questioned whether stem cell transplant bridging benefited directly and indirectly from Marqibo. My answer is above. The FDA indicated that safety is an issue for both vincristine and similarly for Marqibo but that the safety issues are no different for Maribo. Talon's job tomorrow is to drive home the clinical benefit related to stem cell transplant bridging, complete response, the known safety profile of vincristine and the fact that no other treatments or standard treatment pathways exist for this stage of ALL.
Nice recap. Obviously, these patients have little hope - the question is whether the cost (in terms of dollars, patient time and side effects) and risks (will the treatment actually lead to some patients dying earlier?) of treating this patient group is worth the 15%-20% that may actually derive some benefit? It wasn't really clear what that benefit was - the response is not that durable (the cr's relapse within a couple of months, anyway) and the ones that got stem cell treatments didn't seem to survive very long, anyway?
It is very simple actually. The FDA very rarely approves a drug based on small single arm trials (maybe a half dozen cases in the past decade) and when it does it is typically based on spectacular data (ala SGEN). HANA/TLON's self-reported data were marginal, and the FDA review shows an even lower confirmed response rate with a much higher rate of serious AEs than the company reported. This makes it almost a certainty that the FDA will require data from the HALLMARQ trial before approval, and this trial will require tens, perhaps hundreds, of millions to complete. No one, not Warburg or Deerfield, is dumb enough to finance a trial this size based on such marginal data, which means Marqibo is essentially dead money. The only question is whether TLON has any liquidation value, but given the size of their debt load, this is unlikely. Therefore, post rejection on Wed, the stock should be in the 5-10 cent range and everyone one on this board will be screaming for the lawyers. That is the TLON story in 500 words or less.
If you care to cut your losses and try to recoup, may I suggest imuc.ob? I feel this is the best potential 10 bagger in the biotech space at the moment.
That's a bit of an overstatement - is the data marginal, yes, but is the data better than historical data for the few alternatives that these relapsed/refractory patients have, yes. This trial was a crapshoot by HANA to get the quickest approval possible for Marquibo, because they didn't have the funds to support a larger, controlled pivotal trial. If they get rejected, does that mean Marquibo has no future, no. The pk/pd data on this drug clearly points to an improvement over standard vincristine - i.e. it can be delivered in greater doses, stays in the body longer, and has a similar safety profile as standard vincristine, which is still widely used in the front-line treatment of many blood cancers. So, there is a future for this drug (as well as TLON's other drug, Medadione), regardless of the pending decision. What that means in terms of dilution and potential share price over the next few years, who knows, but it ain't good.