If not the FDA then who convinced Thera to go for General rather than HIV NASH? And why?
SPCEO1 wrote: Below are the paragraphs realted to NASH in the ATM prospectus. Someone may have already posted this, but in case not, here it is again. Qwerty will feel good about the part that says the FDA really did want them to do a phase II trial first. The company has indicated that the FDA will seemingly hold their phase III trial results to a somewhat higher standard since agreed to wave the phase II requirement. The language in the risk section of any prospectus is meant to present the worst case scenario, so read the below with that in mind but there are some interesting tidbits in it. You can see why finding a partner on attractive terms may be a challenge. Accordingly, I have to think that has been the early experience and TH is now rebooting in hope of boosting the share price via the KOL meeting so they can pursue NASH on their own via new money raised with the ATM. Alternatively, perhaps the KOL event is meant to encourage potential partners as well. If they are able to drag there NASH effort over the finish line by either finalizing a nice partnership or going it alone after a fund raising, then it will be an amazing achievement. It is amazing they got it this far but they have to "stick the landing" to make it all worthwhile. In light of all this, what we will learn at the KOL meeting takes on added significance. If that event is a flop, it is possible TH might just have to give up on NASH. I sure hope they are working overtime to make sure investors who can make a difference to the stock are at the KOL event. While most of us are here for cancer now, if they can pull this off and give us some value for NASH to, it would be stunning. I believe they can do that but they ahve to execute on selling to investors like they never have before.
Here is the language from the prospectus with some highlights in red from me:
Risks Related to the Phase 3 Clinical Trial Evaluating Tesamorelin for the Treatment of NASH in the General Population
The conduct of the Phase 3 clinical trial evaluating tesamorelin for the treatment of NASH in the general population will be costly and the Corporation has decided to secure additional resources, including finding a partner, prior to initiating such clinical trial, all of which will result in a postponement of the initiation of such trial. If tAlthough the Corporation has begun the search for a potential partner, there can be no assurance that a partner will be found or that a partnership agreement will be entered into on terms satisfactory to the Corporation. If a partner is not found, the Corporation will need to look for alternatives to secure additional resources but there can be no guarantee that the Corporation will secure such resources in an amount sufficient to initiate its Phase 3 clinical trial. Moreover, the Corporation has no meaningful Phase 2 clinical data evaluating tesamorelin for the treatment of NASH in the general population and any results obtained from the conduct of one Phase 3 clinical trial will have to show substantial evidence that tesamorelin is safe and effective for the treatment of NASH in the general population. Finally, the Corporation’s decision to design its Phase 3 clinical trial to meet the FDA’s primary endpoint may prevent the Corporation from seeking approval of tesamorelin for the treatment of NASH in the general population from the EMA since the primary endpoint for this agency is different from that of the FDA.If the Corporation is unable to secure additional resources to initiate its Phase 3 clinical trial, the conduct of such trial could be cancelled. Moreover, if the Corporation is unable to meet the endpoints of its Phase 3 clinical trial or does not receive approval for tesamorelin for the treatment of NASH in the general population, its potential long-term revenues, growth and prospects will be materially adversely affected.
The Corporation has recently held discussions with the FDA and the EMA to finalize its Phase 3 clinical trial design. As a result of such meetings, the trial design will result in higher costs than what the Corporation had previously estimated. The Corporation has decided to postpone the initiation of its Phase 3 clinical trial evaluating tesamorelin for the treatment of NASH in the general population until it can secure additional resources to execute its program and has initiated a search to find a partner for that purpose.
There can be no guarantee that the Corporation will be able to initiate its Phase 3 clinical trial evaluating tesamorelin for the treatment of NASH if it is unable to secure substantial additional resources, either from this Offering, a partnership or other means that it could resort to. In addition, the Corporation may not be able to find a partner to help with securing additional resources. Even if the Corporation finds a partner, the terms and conditions pursuant to which such partner may be interested in assisting the Corporation may not be suitable to the Corporation or may be unfavorable. Under such circumstances, the Corporation may decide to forego the search of a partner and turn to alternative sources of financing. If the Corporation is unable to secure additional resources, it may further postpone the initiation of its Phase 3 clinical trial until it can secure additional resources or may cancel its Phase 3 clinical trial evaluating tesamorelin for the treatment of NASH in the general population. If the Corporation is unable to, or does not proceed with, the development of tesamorelin for the treatment of NASH in the general population, it could have a material adverse effect on its potential long-term revenues, growth and prospects.
Even if the Corporation secures additional resources to initiate its Phase 3 clinical trial, there can be no guarantee that the FDA will approve tesamorelin for the treatment of NASH in the general population since the FDA recommended the Corporation to conduct a Phase 2 clinical trial to generate data resulting from the use of tesamorelin in patients suffering from NASH and since the Corporation must meet the primary endpoints set forth by the FDA in its guidelines. Given the lack of Phase 2 data resulting from the use of tesamorelin in patients suffering from NASH, the data from the Phase 3 clinical trial will have to demonstrate substantial evidence of the safety and effectiveness of tesamorelin for the treatment of NASH in the general population. In addition, even if the Corporation meets the primary endpoints of the clinical trial through the conduct of one Phase 3 clinical trial, the FDA could require the Corporation to conduct an additional study.
The Corporation has decided to design its Phase 3 clinical trial based on the FDA guidelines requiring it to demonstrate “NASH resolution and no worsening of fibrosis” as primary endpoints. This trial design does not follow the current EMA guidelines which require a sponsor to demonstrate both (i) NASH resolution and no worsening of fibrosis and (ii) improvement of fibrosis by one stage without worsening of NASH as primary endpoints. Therefore, even if the Corporation meets the primary endpoints for FDA purposes, the EMA may not approve tesamorelin for the treatment of NASH in this territory since the trial was not designed to demonstrate both endpoints.
If the Corporation is unable to obtain approval of tesamorelin for the treatment of NASH in the United States, this would have material adverse effects on its revenues, financial results and long-term growth and prospects. In addition, even if the FDA approves tesamorelin for the treatment of NASH, the lack of an approval in Europe will limit the Corporation’s ability to maximize its revenue growth potential, therefore potentially hampering its long-term growth and prospects.