Recently, Genfit (OTCPK:GNFTF) announced that it had received positive results treating patients with primary biliary cholangitis (PBC). These positive results were obtained using the company's NASH drug elafibranor. Positive results from the study were obtained after patients were treated over a 12-week period. Even though this was a small study, the drug clearly proved to work in this patient population. It believes that it has enough data to move on to the next stage of clinical development.
Phase 2 Data
The phase 2 study recruited patients both in the U.S. and in Europe. It was a double-blind, randomized, placebo-controlled study that treated patients for a total of 12-weeks. The main goal was to treat patients who didn't respond to ursodeoxycholic acid (UDCA) with elafibranor. What is UDCA? In essence, it is the go-to treatment for patients with primary biliary cholangitis (PBC). Patients in the study either took 80 mg, 120 mg once daily of elafibranor or placebo. The primary endpoint was looking to see a change at week-12 in serum alkaline phosphatase (ALP) from baseline. This primary endpoint was easily met. Matter of fact both does of elafibranor achieved a significant decrease in mean ALP. The 80 mg dose saw a -48% decrease, while the 120 mg dose saw a -41% decrease, and placebo saw an increase of +3%. The mean decrease of ALP observed against placebo for the 80 mg dose of elafibranor was -52% (p<0.001). Even the secondary endpoint which looked at the responder analysis of patients treated elafibranor compared to placebo was met. It was shown that both the 80 mg and 120 mg of elafibranor, achieved a significant response rate on the composite endpoint of 67% and 79% respectively compared to placebo with only 6.7%. With this data on hand, Genfit can move on the next stage of clinical development. How does the decrease in ALP relate to PBC? That's because patients with PBC have obtained damage on the bile ducts of the liver. The cause of the damaged bile ducts is not entirely known, but typically autoimmune disease is the likely cause. Bile can't get out of the liver and starts to damage the liver. Those who have a damaged liver have higher than normal levels of ALP in the bloodstream. As you can see above, both doses of elafibranor significantly decreased mean ALP levels for these patients. That is indicative that both doses of the drug were able to reverse the damage done to livers.
As of September 30, 2018 Genfit had cash and cash equivalents of around $250 million. This compares to the same time period last year when it only had $129.4 million. This biotech has a big pipeline, because it is exploring elafibranor in multiple liver diseases. For instance it has several studies (three) using elafibranor for NASH fibrosis. In addition, it is using elafibranor for PBC as well noted above. It has even branched out to other clinical products in the pipeline like TGFTX4 for an undisclosed target of fibrosis/cirrhosis. Then it is using a similar product like TGFTX1 for autoimmune diseases like psoriasis. All these studies take a lot of cash to run. That's why Genfit recently announced it plans to offer shares in a public offering in the United States. The terms and pricing of the ADS shares being offered have not yet been announced. Thus far, a draft registration statement on form F-1 was sent to the U.S. Securities and Exchange commission.
The positive results obtained in the phase 2 study treating patients with PBC, shows that elafibranor is able to significantly decrease mean ALP levels in this patient population. As noted above, a mean decrease in ALP levels means the liver is starting to improve. With the results on hand, Genfit will be able to possibly move on to a phase 3 study. The risk is that the results produced in this phase 2 study, may not be reproduced again in a larger study. However, I believe that Genfit has a versatile NASH drug. I think it will be okay with its pipeline in the long-term.
This article is published by Terry Chrisomalis, who runs the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace. If you like what you read here and would like to subscribe to, I'm currently offering a two-week free trial period for subscribers to take advantage of. My service offers deep dive analysis of many pharmaceutical companies.The Biotech Analysis Central SA marketplace is $49 per month, but for those who sign up for the yearly plan will be able to take advantage of a 33.50% discount price of $399 per year.
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