Primary bile bile teritis (PBC) best-in-class, breakthrough drug! Clinical success of phase III of the PPAR xenon agitant seladelpar III!

!--ewebeditor:page title"--August 06, 2020 // -- CymaBay Therapeutics is a clinical biopharmaceutical company dedicated to developing innovative treatments for liver diseases and chronic diseases with highly unmet medical needs.
, the company recently released positive topline results for the evaluation of the ENHANCE study for the treatment of primary bile bile bile teritis (PBC) III.
seladelpar is a powerful, selective peroxidase proliferative activator (PPAR) agitant that has been shown to be anti-bile siltation and anti-inflammatory in clinical studies treating PBC.
data from the ENHANCE study confirm that seladelpar is fast, effective, safe and well-tolerated, with rapid and significantly reduced serum alkaline phosphatase levels (ALP), significant anti-inflammatory effects, and rapid and significant reduction of itching symptoms.
these results support Seladelpar's potential as the best-in-class, breakthrough treatment for PBC.
, CEO and President of Cyma Bay Therapeutics, said, "We are very excited about the safety and effectiveness of Seladelpar in PBC patients.
Although the ENHANCE study was terminated early before the end of the 52-week treatment period, patient topline data at 12 and 26 weeks showed that seladelpar had strong anti-bile siltation, anti-inflammatory and anti-itching activity.
These results confirm the results we observed in the Phase II Open Label study and help to increase our confidence that when we restart the PBC PHASE III study, seladelpar will be used as a new treatment to address the critical needs of patients who are not being met.
would like to thank all the patients and researchers who participated in the ENHANCE study and continue to support our efforts.
"PBC" (Photo Source: Genfit.com) ENHANCE is a randomized, double-blind, placebo-controlled global study of 265 PBC patients who did not respond well to bear deoxycholic acid (UDCA) (at least 12 months after treatment, serum alkaline phosphatase levels (ALP) were 1.67 x normal upper limit (ULN)) or had poor tolerance to UBC.
the study, these PBC patients were randomly assigned to receive a placebo, seladelpar 5 mg, seladelpar 10 mg, once a day orally. The main result indicator of
's study was the proportion of respondents, defined as patients with ALP levels of 1.67 x ULN after 52 weeks of treatment and a decrease of more than 15% from the baseline, with normal total biliary erythrin levels.
because the study was terminated early and the number of patients reached the 52-week point was small, the main outcome indicator was modified to a three-month point in time before the database was locked, reaching that point in time in 167 of the 265 patients.
additional key analysis compared the normalization rate of ALP with the itching burden assessed by the Numerical Assessment Scale (NRS);
baseline examination, the average ALP levels in the placebo, 5mg and 10mg groups were 293, 290, 291 IU/L, respectively.
itching NRS (0-10) score of 4 points based on baseline examination, about 30% of patients had moderate to severe itching.
baseline characteristics are balanced between the three groups and represent the group of high-risk PBC patients.
results showed that seladelpar met the main composite outcome indicators and was highly statistically significant: after 3 months of treatment, 78.2% of patients in the 10mg group (n-55) and 57.1% in the 5mg group (n-56), while the placebo group (n-56) had only 12.5% (p?lt;0.0001).
In patients treated with seladelpar, rapid, dose-dependent decreases in ALP were observed as early as one month, with an average decrease of 38%, 30, and 2%, respectively, in the 10mg (n-78), 5mg (n-78) and placebo groups (n-78).
the anti-bile siltation effect of seladelpar was further confirmed, with 27.3% of patients in the 10mg group normalizing ALP levels at 3 months, while the placebo group was 0% (p.lt;0.0001).
these endpoints showed similar patterns over a six-month period, but the number of patients who reached this point in time in the study was smaller.
in patients with NRS 4, Seladelpar also showed significant, dose-dependent itching reduction after only 3 months of treatment compared to placebo.
itching NRS in the 10mg group was 3.2 points lower than the baseline, while the placebo group was reduced by an average of 1.6 points.
Seladelpar also showed strong anti-inflammatory activity during the three months of treatment, with a 17 percent reduction in the average glupropanyl transaminase (ALT) in the 10mg group, compared with 3 percent in the placebo group.
effects of the treatment of seladelpar on gamma-glutamine transferase (GGT) were also significant, with a 36% reduction in the 10mg group and a 7% reduction in the placebo group.
total biliobin remained stable in all 3 groups.
studies, seladelpar has good safety and tolerance.
similar to adverse events in the Seladelpar and placebo groups.
no increase in ALT at level 3 was observed. Dr. Maryn Mayo, M.D., Professor, Southwest Medical Center, Massachusetts Institute of Technology,
seladeldelpar Molecular Structure (Photo: medchemexpress.cn), commented: "Itching is a disturbing symptom of PBC, which is experienced by up to 70% of patients.
for patients with moderate to severe itching, the negative impact on quality of life was significant.
in this control data set, Seladelpar's role in reducing itching provided clinical benefits to a large number of patients, which has not been proven in other good control global studies conducted so far in PBC patients.
there is currently no proven treatment for bile siltation itching, seladelpar has the potential to be a breakthrough treatment option for PBC patients.
"!-- The results of this study are exciting and optimistic for !-- PBC patients," said Professor Gideon Hirschfield, M.D., of the University of Toronto.
data from this study show that seladelpar is safe and tolerable, and its efficacy suggests that seladelpar has the potential to be the best-in-class treatment option for PBC patients.
in the medical field, Seladelpar's experience has reached a fairly high level.
I am confident that researchers and patients will continue to support CymaBay's future efforts to make this breakthrough treatment available to PBC patients living everywhere.
"primary bile bile bile teritis (PBS) is a serious life-threatening autoimmune liver disease.
is characterized by impaired bile flow (bile siltation) and the accumulation of toxic bile acid.
accompanied by inflammation and destruction of the bile tube in the liver, can develop into fibrosis, cirrhosis and liver failure.
clinical symptoms of PBC include fatigue and itching, which can be very disabled in some patients.
PBC is primarily a female disease: 1 in 1,000 women over the age of 40 has PBC.
seladelpar is a powerful, selective, oral PPAR anthicate that is being developed to treat liver disease PBC and non-alcoholic fatty hepatitis (NASH).
for PBC, Seladelpar has been eligible for orphan medicine from the FDA and the EUROPEAN EMA.
addition, Seladelpar Therapeutics PBC has been granted FDA Breakthrough Drug Qualification (BTD) and EMA Priority Drug Qualification (PRIME).
() Original source: CymaBay Announces Positive Topline Results from ENHANCE for Seladelpar in Patients with Primary Biliary Cholangitis !--/ewebeditor:page.