CymaBay Therapeutics is sprinting to the finish line with a breakthrough treatment for a serious liver disease. And it's pushing CBAY stock to a record high on a triple-digit run this year.
After wrestling with setbacks that pushed the biotech to dollar-stock status, CymaBay is emerging as an important player in the battle against a debilitating illness. In June, CymaBay cemented its lead in the primary biliary cholangitis drugs market. Known simply as PBC, the chronic liver disease slowly destroys the bile ducts, leading to fatigue and itching.
CymaBay is drawing attention for seeking to address a critical problem in treating the disease. First treatments typically don't resolve all issues for many patients. The only approved second drug for PBC patients worsens the itching associated with the disease, a condition that CymaBay Chief Executive Sujal Shah describes as "menacing."
"Itching in PBC is almost, you could consider internal or systemic," he told Investor's Business Daily. "Your mind tells you your arm, elbow or leg is itching. You itch and you get no relief. So, this symptom decreases quality of life very significantly for people living with PBC."
But CymaBay could change that next year. And CBAY stock investors are closely watching.
CBAY Stock: 2024 Could Be A Game-Changer
CymaBay has had a really good year.
In November, the company's drug, called seladelpar, emerged as the "second-line winner" in PBC treatment, Leerink Partners analyst Thomas Smith said in a recent report.
Typically, patients with PBC receive a generic drug called ursodeoxycholic acid. It can help prevent or delay liver damage in most people, but it's less likely to help with itching and fatigue, according to the Mayo Clinic. After a year, patients can add another drug — known as a second-line treatment — called Ocaliva. But Ocaliva can make the itching associated with PBC worse.
Shah hopes seladelpar will be considered the more effective second-line treatment, competing directly with Ocaliva. Italy's Alfasigma now owns Ocaliva following its takeover of Intercept Pharmaceuticals. Seladelpar uses a different mechanism from Ocaliva to reduce the body's synthesis of bile acid.
In a study called Response, nearly 62% of patients who received a combination of seladelpar and the first-line drug showed an improvement in levels of an enzyme called alkaline phosphatase and bilirubin, a waste product. Increases in both can signal liver dysfunction.
Notably, a quarter of seladelpar achieved normal alkaline phosphatase levels.
PBC Treatment Improves Itching
In a bullish sign for CBAY stock, patients who received seladelpar for six months reported a 3.2-point lower level of itching on a 10-point scale. In comparison, placebo recipients had a 1.7-point improvement. Before treatment, these patients rated their itching symptoms as at least a 4 on a scale of zero to 10. Patients continued reporting improvements in itching through a year of treatment.
"We believe these results further strengthen seladelpar's clinical data package, showing further improvements on itch and impact on sleep," Leerink analyst Thomas Smith said in a report.
Meanwhile, rivals Genfit and Ipsen said their experimental PBC treatment led to a non-statistically significant improvement in itching symptoms. CBAY stock jumped 3.5% the day Genfit and Ipsen reported these results on Nov. 13. Genfit shares eked out a fractional gain.
CymaBay CEO Shah notes itching in PBC isn't well understood. Seladelpar lowers the body's synthesis of bile acid and reduces both inflammation and a cytokine called IL-31. The cytokine is also tied to itching in patients with PBC.
"There are a number of different elements that make it unique in terms of reducing itch," he said.
Looming Approval For Seladelpar?
On Dec. 15, CymaBay asked the Food and Drug Administration to approve seladelpar on top of the first-line treatment for PBC, for patients who don't respond to ursodeoxycholic acid or can't tolerate it. CBAY stock rose almost 2% that day.
CymaBay is seeking a speedier priority review, which could mean gaining approval by mid-August. Leerink's Smith said he expects the FDA to ultimately sign off on seladelpar and for the drug to lead the second-line pack.
"We also continue to see seladelpar's profile — with a balance of competitive biochemical response, a unique (itch) improvement benefit, and potential edge on safety/tolerability — to drive preference over competing agents and cement seladelpar as the treatment of choice for second-line PBC," he said in a recent report.
Smith reiterated his outperform rating on CBAY stock.
CBAY Stock Already Leads The Pack
CymaBay stock currently has a best-possible Relative Strength Rating of 99. This means shares rank in the leading 1% of all stocks when it comes to 12-month performance, according to IBD Digital.
On a year-to-date basis, as of the close on Dec. 27, CBAY stock has soared 290%. Shares climbed out of dollar-stock status for the last time on June 30 and have continued rising since, MarketSmith.com shows.
CBAY stock's longer-term performance has also been impressive. Shares hit a two-year low at 1.67 in May 2022. On Dec. 28, CymaBay stock touched a record high at 25.33. That's a 1,417% gain over 19 months. The finish line in PBC treatment is now within view, CEO Shah says.
After that, CymaBay will focus on patients with less severe PBC who still have heightened levels of alkaline phosphatase and bilirubin, but not high enough to require a second-line drug like seladelpar or Ocaliva. The high levels of those substances leave patients at risk of worsening liver damage.
"We are very focused on the goal line, which remains to get seladelpar approved and into the hands of people with PBC," Shah said. "We have a lot of discipline around that focus."
Follow Allison Gatlin on X, the platform formerly known as Twitter, at @IBD_AGatlin.
