On February 28, 2019 CymaBay Therapeutics, Inc. (NASDAQ: CBAY) a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported financial results and a corporate update for the quarter and year ended December 31, 2018 (Press release, CymaBay Therapeutics, FEB 28, 2019, View Source [SID1234533857]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The initiation of ENHANCE, our global, Phase 3 registration study of seladelpar in primary biliary cholangitis (PBC) at the end of 2018 was a major milestone for CymaBay," said Sujal Shah, President and CEO of CymaBay. "ENHANCE was initiated on the strength of what we believe to be a very compelling and differentiated profile in Phase 2, which highlighted the potential for seladelpar to offer patients with PBC improved efficacy and better tolerability than existing second line treatment. Seladelpar has now been granted Breakthrough Therapy Designation for PBC by the FDA and PRIority MEdicine status by the EMA. In parallel, we are advancing clinical development of seladelpar in nonalcoholic steatohepatitis (NASH) and were excited earlier this month to announce the completion of enrollment in our Phase 2b study, one quarter ahead of schedule. Seladelpar is the only highly selective and potent PPARδ agonist in development for liver disease, and we believe it may be particularly well suited to treat NASH. The ability of seladelpar to lower bile acids, cholesterol and lipotoxic lipids, coupled with anti-inflammatory and anti-fibrotic actions may position it as a foundational therapy for NASH."

Recent Business Highlights

In February 2019, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for seladelpar for the treatment of early stage PBC in combination with ursodeoxycholic acid (UDCA) in adult patients with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA.
In February 2019, enrollment was completed in a Phase 2b dose-ranging, paired liver biopsy study of seladelpar for the treatment of nonalcoholic steatohepatitis (NASH).
A total of 181 patients enrolled with elevated liver fat and biopsy-confirmed NASH.
Topline data on the primary efficacy outcome, the change from baseline in liver fat content at 12 weeks as measured by magnetic resonance imaging using the proton density fat fraction method (MRI-PDFF), are expected 2Q 2019.
Fourth Quarter 2018 Highlights

Initiated ENHANCE, a global, Phase 3 registration study of seladelpar for the treatment of primary biliary cholangitis (PBC).
ENHANCE is being conducted in more than 150 centers in over 20 countries. The study is intended to establish the efficacy and safety of seladelpar for the treatment of PBC to support the submission of a global registration dossier with health authorities to obtain approval.
The study is expected to be fully enrolled by the end of 2019 with the 52-week treatment period targeted to be completed by the end of 2020.
Positive data from an ongoing Phase 2 study of seladelpar in PBC were featured at late-breaking presentations during The Liver Meeting 2018 hosted by the American Association for the Study of Liver Diseases.
Sustained anti-cholestatic and anti-inflammatory effects observed with no worsening of pruritus through 52 weeks.
Results highlight the potential for seladelpar to offer patients an efficacious and safe second line treatment option.
Results suggest that seladelpar is not associated with drug-induced pruritus and may support the hypothesis that seladelpar decreases pruritus in PBC patients.
Held $178.7 million in cash, cash equivalents and marketable securities at December 31, 2018. Existing cash is expected to fund the current operating plan into 2021.
Fourth Quarter Ended December 31, 2018 Financial Results

There was no collaboration revenue in the fourth quarter of 2018 as compared to $5.2 million of revenue in the same period of 2017. Fourth quarter 2017 revenue earned was primarily due to the achievement of a $5.0 million milestone under a collaboration agreement with Kowa Pharmaceuticals America, Inc.
Research and development expenses were $16.4 million in the fourth quarter of 2018 as compared to $6.7 million in the same period of 2017. The increase was primarily driven by increases in seladelpar-related clinical trial expenses from the expansion and extension of our PBC Phase 2 clinical study, start-up activities related to our ENHANCE PBC Phase 3 clinical study, the ongoing enrollment of our NASH Phase 2b clinical study, and the execution of other NDA-enabling studies.
General and administrative expenses were $4.2 million in the fourth quarter of 2018 as compared to $2.9 million in the same period of 2017. The increase was driven primarily by employee compensation expense as we hired additional personnel to support our expanding operations.
Net loss was $19.4 million, or ($0.32) per diluted share in the fourth quarter of 2018, as compared to $5.0 million, or ($0.11) per diluted share in the same period of 2017. Net loss was higher primarily due to increased research and development expenses and decreased revenues.
Year Ended December 31, 2018 Financial Results

There was no collaboration revenue for the year ended December 31, 2018 as compared to revenue of $10.0 million in the prior year. Collaboration revenue was recognized in 2017 upon the delivery of our performance obligation under a collaboration agreement with Kowa Pharmaceuticals America, Inc.
Research and development expenses were $58.1 million in the year ended December 31, 2018 as compared to $18.9 million in the same period of 2017. The increase was primarily due to increased R&D project costs related to the expansion and extension of our PBC Phase 2 clinical study, start-up activities related to our ENHANCE PBC Phase 3 clinical study, enrollment in our NASH Phase 2b clinical study, and the execution of other NDA-enabling studies. R&D project costs also increased due to manufacturing of seladelpar to support ongoing and planned clinical trials and other development activities. Internal R&D costs also increased, primarily due to higher employee compensation related expenses as additional clinical, scientific and regulatory personnel were hired to support expanding clinical development activities.
General and administrative expenses were $14.4 million in the year ended December 31, 2018, as compared to $12.4 million in the same period of 2017. The increase was primarily due to higher compensation and consulting expenses, partially offset by a decrease in severance.
Net loss was $72.5 million, or ($1.26) per diluted share in the year ended December 31, 2018, as compared to $27.6 million, or ($0.79) per diluted share in the same period of 2017.
Conference Call Details
CymaBay management will host a conference call today at 4:30 p.m. ET to discuss fourth quarter 2018 financial results and provide a business update. To access the live conference call, please dial 877-407-0784 from the U.S. and Canada, or 201-689-8560 internationally, Conference ID# 13687140. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source