Galectin Therapeutics Inc (NASDAQ:GALT) is crash-landing 35% to the market floor once investors caught word of the biotech firm’s Phase 2b NASH-CX study results that failed to achieve statistical significance for its primary endpoint. Therefore, even if GALT’s proprietary compound GR-MD-02 pointed to a positive trend in the total group of patients both with and without esophageal varices, the issue lies with the difference- one that never offered statistically significant improvement.
What happened? Worthy of note, Galectin found increasing variability in hepatic venous pressure gradient (HVPG) measurements for nonalcoholic steatohepatitis (NASH) patients with esophageal varices, which led to the failure of the primary endpoint. The trial was split, with half the patients enrolled in the clinical study without esophageal varices.
However, some statistically significant and clinically meaningful impacts were noticed in NASH cirrhosis patients without esophageal varices when treated with Galectin’s drug. The secondary endpoint part of the trial fared far better, with a liver biopsy assessment taken of the total pool of 161 patients indicating GALT achieved statistical significance in the improvement of hepatocyte ballooning. In NASH, this is crucial, as a pathology of non-alcoholic fatty liver disease points to this kind of degeneration, a type of liver parenchymal, or hepatocyte death. Cells dying in this way “balloon” in size. In the analysis taken of Galectin’s Phase 2b study, a statistically significant lessening in the development of new esophageal varices was observed in patients without varices at baseline.
The biotech firm performed a rigorous assessment of the response to therapy by evaluating the percent of patients who had a reduction of HVPG from baseline (Responder Analysis). Responders were defined as having reductions of HVPG from baseline that have been shown to be clinically significant, an absolute reduction of ≥ 2 mmHg of HVPG from baseline or a ≥ 20 percent reduction of HVPG from baseline. Based on reduction in absolute HVPG, patients without varices who received a 2 mg/kg dosage of GR-MD-02 showed a statistically significant greater percentage of responders than those without varices in the placebo group (44 percent versus 15 percent, p=0.02). The same statistically significant results were seen when responders were analyzed based on a ≥ 20 percent reduction from baseline HVPG (40 percent versus 15 percent, p=0.03).
“There is no current therapy for patients with NASH cirrhosis — and a therapy such as GR-MD-02 that could improve portal hypertension and potentially prevent the development of esophageal varices in NASH cirrhosis and subsequent complications — would be clinically valuable,” said Stephen A. Harrison, M.D., one of lead investigators of the NASH-CX trial, medical director of Pinnacle Clinical Research in San Antonio, Texas, and visiting professor of medicine at the University of Oxford, United Kingdom. “An indication of NASH cirrhosis without varices would be clinically meaningful to physicians, because it is standard of care for all patients with cirrhosis to have an upper endoscopy to assess for the presence of esophageal varices.”
“We believe this is the first large, randomized clinical trial of any drug to demonstrate a clinically meaningful improvement in portal hypertension or liver biopsy in patients with NASH cirrhosis without varices,” said Dr. Peter G. Traber, M.D., CEO and CMO of Galectin Therapeutics. “Furthermore, we believe that the results stratify a large and easily identifiable group of patients. The results also suggest several potential registration endpoints that may be employed in a phase 3 program, including absolute or percent changes in HVPG, the percentage of patients who respond with a clinically relevant reduction in HVPG (Responder Analysis), and the development of esophageal varices, which may be considered a clinical outcome by regulatory agencies. Additionally, we are gratified to note that the drug was also well tolerated, and no safety concerns were detected.
“We would like to express our gratitude to the NASH patients who participated in this trial and to their physicians. Their unwavering commitment, over the year-long course of therapy, allowed this trial to be completed in a timely manner, and their dedication to helping others find a treatment for NASH is most laudable.”
On the ratings front, Galectin stock has been the subject of a number of recent research reports. In a report issued on November 28, H.C. Wainwright analyst Ed Arce maintained a Buy rating on GALT, with a price target of $6.00, which implies an upside of 329% from current levels. On November 14, Roth Capital’s Yasmeen Rahimi reiterated a Buy rating on the stock and has a price target of $10.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Ed Arce and Yasmeen Rahimi have a yearly average return of 20.3% and 7.2% respectively. Arce has a success rate of 46% and is ranked #274 out of 4727 analysts, while Rahimi has a success rate of 50% and is ranked #2420.