CymaBay announced positive interim results from its ongoing low-dose Phase 2 study of seladelpar in patients with primary biliary cholangitis (PBC), a life-threatening and life-limiting chronic cholestatic liver disease. In the first part of the study, patients at high risk of disease progression, with an inadequate response to ursodeoxycholic acid (UDCA), as characterized by a persistent elevation in alkaline phosphatase (AP), or who were intolerant to UDCA, received either 5 mg or 10 mg of seladelpar once-daily. A planned interim analysis of the first 24 patients enrolled in these two dose groups demonstrated after 12 weeks of treatment a significant AP reduction from baseline of 39% and 45% for the 5 mg and 10 mg groups, respectively. On seladelpar, 45% of patients in the 5 mg and 82% of patients in the 10 mg dose groups had AP values < 1.67 times the upper limit of normal (ULN). AP is an established surrogate marker of disease progression in PBC, and reaching a level of < 1.67 x ULN is a key component in the composite endpoint used for regulatory approval.
Alongside substantial reductions in AP, patients in both dose groups experienced decreases in other liver markers of cholestasis including gamma glutamyl transferase and total bilirubin. Seladelpar also improved metabolic and inflammatory markers with patients experiencing decreases in low-density lipoprotein-C and high sensitivity C-reactive protein.
There were no serious adverse events and no safety transaminase signal was observed at either dose. Instead, mean transaminase levels decreased over the course of treatment, further supporting seladelpar’s anti-inflammatory activity. Consistent with prior studies, there was no signal for drug-induced pruritus.
After sharing preliminary results from the study, the FDA has agreed to allow continuation of seladelpar treatment beyond six months for the 5 mg and 10 mg doses.
“The data emerging from this study are impressive and support our hypothesis that lower doses of seladelpar than previously studied retain strong efficacy without raising a concern with transaminase elevations. We also see that seladelpar activity is not associated with drug-induced itch, an important benefit for patients with PBC. If these results are maintained over longer periods, we think that seladelpar could offer patients significant advantages over existing treatments,” said Professor Gideon Hirschfield, Centre for Liver Research, University of Birmingham, UK.
Dr. Pol Boudes, Chief Medical Officer of CymaBay added, “We’d like to thank the investigators and their staff as well as the patients and their families for their tremendous support. These interim results support the potent anti-cholestatic and anti-inflammatory effects of seladelpar. We are particularly excited about the FDA’s decision to allow dosing of seladelpar beyond six months enabling us to turn our attention towards planning the Phase 3 program.”
“The clinical and regulatory progress to date represent meaningful advancement in the development of seladelpar for patients with PBC,” said Sujal Shah, Interim President and CEO of CymaBay. “We are very encouraged by these results and the potential for seladelpar to improve treatment alternatives in PBC and other chronic liver diseases.”
Read the full results of the “Seladelpar Interim Data Phase 2 Low Dose Study in PBC” by
clicking here
On Tuesday, September 12, 2017 a delegation from the Canadian PBC Society met with elected MPPs and Ministry of Health staff at Queen’s Park Legislature in Toronto in our first PBC Awareness Day. Meeting in small groups, MPP’s asked lots of questions and PBC members related personal stories and concerns. With a full day of meetings and a lovely lunch in the legislative dining room we are hopeful that our voices have been heard and are needs will be addressed. We plan to be back next year! See more photos in our photo gallery. 
On Tuesday, September 12, 2017 the Canadian PBC Society launched A PBC Patient Charter, with input from more than 100 PBC patients and their families. Read the complete charter by 
On September 6, 2017 in honour of International PBC Day, a group from the USA and Canada met over lunch in Niagara Falls, NY. This was our 2nd annual cross-border lunch. Next year our lunch will be back on the Canadian side of the Falls. We hope you will plan to join us as we share stories and get to know each other. It is always good to talk to people about our rare disease to see what we have in common and understand our differences. 
After waiting 20 years, this month PBC patients have started on their new treatment option, Ocaliva. For patients who do not respond to ursodeoxycholic acid this has been tremendous news. In six months we will report back to let you know how these patients are doing. 
It is with great sadness that we announce the passing of Erica Nirenberg. Many of you will remember Erica for her kindness and diligence in arranging the room for monthly support group meetings. “She knitted and crocheted for many charitable organizations and kept Dr. Heathcote’s feet warm for many years with handmade slippers!. A truly sweet, generous woman.”
On June 4, The Canadian PBC Society had a tremendous day at our annual fundraising event ‘A Day at the Races’. We had a great turn out as our members and their friends and families joined us to celebrate and discuss all the accomplishments in the PBC community this year. We ate great food, watched amazing races, enjoyed a wonderful silent auction, and all in good company. We would like to thank all of our donors and sponsors, with whom without this event would not be made possible. The Canadian PBC society is grateful for all our supporters and look forward to ‘A Day at the Races’ next year! 
annual ‘A Day at the Races’ fundraiser on the website on our PBC PHOTO GALLERY which I hope you are able to locate at the top of this page under the blue tabs or just click