Really good results for phase 2! Makes me more confident for Phase 3 BPH, I just need to keep on reminding myself to be patient. Not the easiest thing to do.
Anyone know if Paul has ever entertained the idea? There's no news of manufacturing readiness which makes me speculate a partnership. It would have made sense for Recordati to put an offer in before Phase III results. I expect several companies to bid for NYMX if results are good.
Get the product to market first for MI indication. Discuss with the FDA about the SPRING trial and combine the STEP safety for a single injection indication. If approved for MI, the followup for single injection will be next on AMPE's timeline. The N may be less as a followup request from the FDA which is what everyone would want. It is not ideal but this is an okay route to take with less dilution for shareholders.
Overall, decent results though statistical analysis couldn't be performed on the ampion group. Safety looks good even with the temperature excursion. Without the temperature excursion this would have been a sure thing...now hopefully a perfectly executed MI study. I want to know what the FDA thinks - we may hear more on this in the coming weeks when their reviewers provide informal responses. Stil, I'm comforted by the fact that several ex-FDA reviewers are AMPE's consultants. They'll provide AMPE with a framework to position the BLA successfully. The buying continues...
CEO said 6-8 months and VP clinical said Q3. My predictions are after hours, next week or second week of september. Past that the only certainty is before November conference.
I need to add as well. My greatest worry WAS that the biological would not be stable at low temperatures. This however is not the case. The sole reason why efficacy may decrease is because the biological adheres to glass at low temperatures and require external energy to bring back in to solution to be injected. This is very common for biologicals. If AMPION maintains its structure at every temperature -20 to 30 then we have a dosing problem. To a statistician, this now becomes a dose-temperature dependent data set. It can be isolated. Had the biological broken down across the 70%, the entire set would have been thrown out because there's no way to predict the behaviour of each individual point. Now well all wait for the results of each subset.
So AMPE ran STEP at 30% to protocol, 70% to a range (CSO needs to run some in house tests to determine concentration of AMPION between T1-T2). If appropriately separated, AMPE will have 3 results 1) 30% at T15, 70% at T1-T2, and 3) Combined at T1-T2 and T15. Any of 1-3 if better than saline is still in the eyes of the FDA compelling data. The MI study will need to be pivotal so that they can have a single injection (STEP+SPRING) and multiple indication (MI) indication. I think this is a strong buy because the science is sound, some of the clinical results are showing that it is good and the market is overreacting. We can go on and on about incompentence but this was not management...this was the contracting company.
Why's everyone so anxious? Look to some of my older posts. All you need to know is 70% of the data is compromised so 30% of data (roughly 125 points) in STEP is usable as agreed by the FDA. In a couple of weeks when AMPE is done separating the good from bad you will get results (may not be statistically significant) but enough to be combined with SPRING. I'm curious to see if a lower dose/so the whole compromised study combined is better than saline injections. If that is true...well AMPE will be up to $10+. AMPE will then add the MI study to this and file for their BLA hence it will be Q1 or Q2 2015. Why Jeff and Citi decreased PT? Because risk has increased...if STEP is not statistically significant the investment thesis for Ampion cannot be confirmed until MI studies are complete. Some companies make their MI study pivotal instead of single injection...this is what has happened to AMPE just not by choice. The contracting company will owe AMPE quite a bit of money, so weep for them. Some of that cash will come back and be used for MI study. I highly doubt the CEO will let them go on doing their business.
ri66, my hopes are the AMPE will release the subset 25-30% that is unaffected by the temperature which will substantiate SPRING. Based on all trial results so far there will be low risk of this not happening but statistical significance will be affected. Regardless, the MI will then be used with Spring so the BLA will proceed. I work in Regulatory so I can tell you this is a procedural mishap that gives a very rare buying opportunity. Most often it is the drug that fails and results/statistical significance is affected. AMPE however has enough cash to finish MI studies and may show healing. I still think there are big pharmas looking at AMPE and weighing the benefits of purchasing this company right now - it is undervalued. There's growing evidence that HA injections do not benefit patients so there is a frantic search for the next OA standard care. Ampion looks like it will be that treatment with solid science and trial results so far. If MI studies are good it will be breakthrough.
This is a market overreaction... at 25% you still have 125 good study points. This explains why the FDA are willing to accept the STEP study (minor indication). In 2-3 weeks we will see that subset with similar SPRING results. The MI (major indication for biologic) will need to be executed to perfect statistical significance for the FDA to accept the BLA. It would be wise for management to include the STEP subset and MI study with SPRING instead of risking the approval with just MI and SPRING. While Jefferies has lowered the PT to $5 I think it will shortly return to $8-12. AMPE were extremely lucky in that they had already started MI studies. If there was ever a time for a pharma acquisition it would be now!
the MI study is better than STEP... I'm happy with the merger, buyout, licensing that's coming:
Does Ampio have enough money to maintain operations through this year and all of next year while completing the planned clinical trials and filing for regulatory approval for the two lead drugs?
Yes, as long as we do not directly commercialize either of our two lead drugs, AmpionTM or OptinaTM, which is not our intention.
Since our August 21st release reporting technical problems with the STEP study and delayed release of the analysis of Study data, we have received many calls from interested parties for a more comprehensive explanation of the issues and asked to address a number of incorrect rumors that have surfaced. Ampio management has done its best to answer every call but has realized that the fairest way to proceed is to present all shareholders with the questions and our answers in this written format. While the events of the past few days have certainly been frustrating for shareholders and Ampio management, there is no change in management's excitement about the success of Ampio. As always, Ampio has alternate plans to overcome unexpected events and meet our corporate goals. We will always act responsibly to protect our company and its shareholders. With all the positive activities taking place at Ampio, that require the full attention of our executive management, it is time to focus our attention to the tasks at hand, move forward and continue our work. The company's goal remains to file the BLA in Q1 2015 and it will be supported by the SPRING study results in combination with either the STEP study or MI study or a combination of all three.
Bill, I'm a regulatory expert and I can tell you that anyone can extend their conversation time with the FDA. AMPE could have asked for an additional 15 days to review. This PR release was not well written and requires a CC to clear things up. Did the FDA review data? What type of data? In what way is the FDA willing to accept the STEP results? Why would AMPE need to contact regulatory advisors to determine if their BLA filing is enough? Why didn't AMPE separate data unless that actually are being honest and that upon discovery of breaking T protocol they called the FDA and informed investors. The CSO having worked with ampion would know the yield and resulting efficacy. He would have told the lawyers his expectations if they did not see the results. Our poor CEO is obligated to tell investors right away and so we are stuck in this murky mess.
I had said earlier in the day that this cyclic group can survive a freeze/thaw situation. I'm not entirely sure about the amino groups but the FDA is willing to accept this study as supporting data for the BLA which is a big win. AMPE therefore must have presented stability data and basically said that their efficacy might be affected because of lower active yield. Can they isolate sites or patients that were affected by the temperature difference and compare both situations? We'll hear more when they release topline and explain with and without partitioning. This is unfortunate but at the same time AMPE is extremely lucky in that they have multi-injection studies running that are yielding 86% improvement. I would have preferred it if AMPE had talked to the FDA, ran their analysis and separated data and then presented topline. But no...they took the amateur route and fed incomplete news to investors causing a 25% drop.
There's a lot of diketopiperazine compounds out there that are stored below 0 C. My slight worry is for the amino acid side chains which may be affected...but to what extent? The more I read into the chemistry and tease out stability I feel comfortable with the imminent STEP results. True, protocol was not followed during transport but it was injected at greater than 15 C. AMPE can do some quick tests to determine the yield of ampion after several freeze/thaws. I wonder if they disclosed this to the FDA which convinced them to accept STEP as still supporting the BLA. If anything, this gives more weight to the multiple injection studies and less reliance on STEP which might work in their favour.
The company would not know yet if this has affected their study. Dissecting their wording I read that low temperature may affect efficacy and that is where the market will react. At less 0 C what happens to the drug? When the drug is returned to 15 C, what's the remaining active yield? Is it 60-90? If so, you can trend the results and build off of STEP to see what predicted would be. Not a total disaster considering we know what the multiple injections studies reductions are. Physicians will only be looking to the multiple injections for their patients. AMPE already stated that the FDA is willing to examine the STEP study (I suspect they looked at stability data) and were still fine with the trial. I expect STEP to be slightly lower than SPRING results but with the caveat of temperature affecting the % of actives given to patients.
less 0 C what happens to the drug? When the drug is returned to 15 C, what's the remaining active yield? Is it 60-90? If so, you can trend the results and build off of STEP to see what predicted would be. Not a total disaster considering we know what the multiple injections studies reductions are. Physicians will only be looking to the multiple injections for their patients. AMPE already stated that the FDA is willing to examine the STEP study (I suspect they looked at stability data) and were still fine with the trial. I expect STEP to be slightly lower than SPRING results but with the caveat of temperature affecting the % of actives given to patients.
The company would not know yet if this has affected their study. Dissecting their wording I read that low temperature may affect efficacy and that is where the market will react. At
Sentiment: Strong Buy
I look at it from an acquirer's point of view. Would I put an offer in before or after STEP? The STEP results will solidify if this is going to be first line minus regeneration results. The company has manufacturing ready so it's a matter of acquiring this company before it starts working on other applicaton sites. Then there is optina, which has absolutely destroyed my investment thesis for other DMA companies. We're looking at a pill that has equivalent if not better results than implants. These two products are moving very quickly through regulatory pathways which is exciting!