Update from CymaBay on Seladelpar study

[PBCCarol]

Important Seladelpar Update – From PBCers Organization and Canadian PBC Society

Clinical trials for PBC patients put on hold

Seladelpar, a drug developed for PBC patients, has shown positive results in Phase 2 studies- normalizing ALP levels, while not associated with itching. In November with FDA breakthrough therapy designation for PBC, CymaBay announced an extended Phase 3 study. Previously, a 52 week study of Seladelpar was undertaken for NASH(Non-alcoholic steatohepatitis) patients.

Today CymaBay announced that it will be putting on hold its PBC clinical trials, whilst halting other trials. It is important to note that as notified publicly by Cymabay, there have not been any concerns raised in the PBC trial, but the company is acting out of an abundance of caution, and with patient safety foremost.

The company cited atypical findings observed in the study of NASH patients; while blood work (biochemical) improved, biopsies showed abnormalities in liver cells (histology). CymaBay has initiated a series of investigative actions to better understand these findings with the focus on ensuring patient safety and care, and will be updating all stakeholders regularly.

We feel it’s important to make sure you are aware of this issue. If you are currently enrolled in a Seladelpar study and have any questions or concerns, you should talk to your doctor.

List of Seladelpar clinical trial sites:

USA ​CANADA

AZ: Chandler, Phoenix, Tucson​ AB: Calgary

CA: Los Angeles, Stanford, UC Davis, San Francisco​ ON: Toronto

CO: Denver​ QC: Montreal

CT: New Haven​ MB: Winnipeg

FL: Boca Raton, Lakewood Ranch, Miami, Tampa

GA: Atlanta

IL: Chicago

IN: Indianapolis

KS: Kansas City

LA: Tulane

MA: Boston

MI: Novi

MN: Maplewood, Minneapolis

MS: Jackson, Tupelo

MO: Kansas City

NY: NYC, Rochester

NC: Asheville, Duke

OH: Cleveland

PA: Bethlehem, Hershey, Pittsburgh

TN: Germantown, Knoxville, Nashville

TX: Dallas, Ft Worth, Houston, San Antonio

UT: Murray

VA: Newport News, Richmond

WA: Seattle

[Ktltel]

Thank you for the information PBCCarol.

This is so disappointing. I know some were taking Seladelpar and had been having some success for a while. I had negative results taking OCA and had hoped that Seladelpar was my next step. Unfortunately not.

Is there anything else in the pipeline that is helping ones?

Stella❤

[PBCCarol in reply to Ktltel]

We can’t rule out that this may be resolved and Seladelpar studies may resume after more investigation. There are a few other things in the pipeline - after the holiday I will try to post info on the current studies. You can also go to the research page on pbcers.org to see some info on them and on signing up for our patient registry

[Ktltel in reply to PBCCarol]

Thank you.❤

[CollMc]

I am so disappointed I am part of the study half way there was on week 28. They put it on hold and said not to take any more😢if your like me and can’t tolerate Ursodiol it’s very disappointing. Hopefully they will be able to continue but for now I have nothing. All the headlines were about the stock

plunge how about care and concern we have so few options Need to find a cure or at least a drug .

[PBCCarol in reply to CollMc]

I can only imagine how disappointed you feel - this treatment was helping so many people the were participating in the study. Of course the media will cover the stock plunge but I can assure you that those in the company do have care and concern for the patients. They needed to put the study on hold while they investigate whether the results that appeared in the NASH study can be found in the PBC study also. They are looking out for the patients even though it may not seem it right now. Thank you for your part in helping with this study for a new treatment. Only we can do that!

Carol