The STEP study of Ampion™ had a technical problem that is being evaluated for deviation from protocol in temperature excursions in over 70% of the drug supply for this study. This excursion in temperature down to freezing is likely to have affected the level of active ingredients in the injected dose of AmpionTM. One of the active ingredients of Ampion™, aspartyl- alanyl diketopiperazine (DA-DKP) is extremely stable at low and high temperatures, however if the glass vial containing the drug is frozen or is at freezing temperatures, some of the DA-DKP will come out of solution and together with other compounds present in Ampion™ will adhere/crystallize on the inner glass surface of the vial. Thawing the vial to room temperature, without vigorous agitation for a prolonged period of time, will not bring these active ingredients back into solution. As this level of agitation is unlikely to have occurred at the clinical site, it is anticipated that if vials reached those low temperatures, a lower than expected level of DA-DKP than required by the Study Protocol would have been administered in the trial.
Sentiment: Strong Buy
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.
A discussion with the FDA informing them of the protocol deviation was very positive and the FDA will be evaluating the available data from the trial when presented to them to decide what can be salvaged and how
In parallel with the ending of the STEP study, the company began a placebo-controlled, double-blinded multiple injections trial of AmpionTM for treatment of OA of the knee that is expanding to 14 clinical sites. This trial was launched in order to expand the Indications for Use (IFU) to multiple injections rather than a single injection. Approximately 300 patients with moderate to severe OA will be enrolled and treated as soon as possible. The study end point will be at 20 weeks from the first injection. The primary end point is WOMAC A (pain) and secondary end point will be WOMAC C (function). The open label portion of this trial enrolled 7 patients who demonstrated remarkable results, both in pain reduction and improvement in function of the knee at 4 and 6 weeks after initial treatment. We anticipate analyzing data of the open label portion of the study, including MRI's and synovial fluid, at 12 weeks, which will occur in about a month. Forty (40) additional patients will follow the same protocol as the open label portion of the multiple injections study and enrollment is nearly complete.
Sentiment: Strong Buy