Take this for whatever it is worth, and it may not be worth much.
It is reported that GALT is due out with its topline NASH (Nonalcoholic Steatohepatitis) CX trial results in December this year. The company has hinted at the possibility of a Breakthrough Therapy designation if they meet their regulatory endpoints on Hepatic Venous Pressure Gradient (HVPG) and Fibrosis. There seems to be a feeling that there may be a pathway toward provisional approval as they outlined their plans to break the patient groups up into subsets for a follow on phase 3 trial. My understanding is that a few points of improvement in HVPG may result in provisional approval.
Dr Traber said “the study had a rigorous FDA agreed design that measures many parameters in patients with NASH cirrhosis who have not had serious complication and are not yet candidates for a liver transplant.” This FDA agreement may be highlighted to show a lot went into the design of this trial to get an agreement on acceptable endpoints. If you read into this statement a little deeper you can see the FDA may essentially be saying get us the HVPG data and if it meets these endpoints we are going to give you provisional marketing approval with follow-up data.
The trial was designed for 156 and in a way was oversubscribed. Having 152 patients left in the trial at this late stage is encouraging since if you assume the current dropout rate continues then only 5 more patients would be expected to drop out over remaining 5 months of the trial ending in August. The reason this point is important goes to the power of the study which should be approximately 95%. It is difficult for patients to undergo biopsies but for a good reason -- like the maybe the patients feel it is worth the effort. It was previously assumed that there would may be a 25% drop out rate.
There is a hint -- a mere hint--that maybe the end points of the Galt study are being met for possible provisional approval.
Further, the Methacetin breath test employed by Galt in its trial is being evaluated as a diagnostic that measures the level of liver damage. Positive data on this less invasive breath test could be a blessing for all drug companies in the NASH space if it also reduces the need for a liver biopsy. For GALT, if provisional approval is given in December it could potentially mean having to do away with HVPG which is a costly and arduous test in its Phase III trials and allow for the quick recruitment of patients in phase III.
Please do your own due diligence on all of this, and do not rely on what I have said here. My comments are pure speculation based on my understanding of what I have read, which may not be accurate. Furthermore ,I/we can not predict the future of success of the GALT clinical trial no matter what. GALT is a dangerously speculative investment. If the results of this clinical trial fail, those invested in GALT may lose their entire investment.
