NEWTON, Mass. - Karyopharm Therapeutics Inc. (NASDAQ:KPTI), a pharmaceutical company focused on cancer treatment, has announced changes to the Phase 3 SENTRY trial for myelofibrosis following guidance from the U.S. Food and Drug Administration (FDA). The trial will now measure the absolute total symptom score (Abs-TSS) over a 24-week period as one of its co-primary endpoints, replacing the previous measure of a 50% improvement in total symptom score (TSS50).
The SENTRY trial, which is evaluating the efficacy of selinexor in combination with ruxolitinib in myelofibrosis patients, will retain spleen volume response rate ≥ 35% (SVR35) as a co-primary endpoint. Phase 1 data showed promising improvements in both Abs-TSS and SVR35, with an approximate doubling of SVR35 to nearly 80% compared to historical data on JAK inhibitor monotherapy and a meaningful average improvement of 18.5 points in Abs-TSS at week 24.
In response to these findings and to enhance the statistical power of the study, Karyopharm is proactively increasing the total sample size to approximately 350 patients. The expected top-line data read-out remains scheduled for the second half of 2025.
Dr. Raajit Rampal, a leading researcher in the field, expressed confidence in the trial data, noting the significant unmet need in myelofibrosis treatment and the meaningful improvements observed with the selinexor and ruxolitinib combination.
Abs-TSS is considered a more accurate assessment of symptom improvement and is supported by key investigators and patient advocacy organizations for its relevance to patients' experiences.
Karyopharm will discuss the SENTRY trial further in a conference call today featuring Drs. Raajit Rampal and John Mascarenhas.
This article is based on a press release statement from Karyopharm Therapeutics Inc.
In other recent news, Karyopharm Therapeutics Inc. faces the risk of delisting from the Nasdaq Global Select Market due to its stock price not meeting the minimum bid price for over a month. Despite this, the pharmaceutical company has a 180-day period until March 17, 2025, to regain compliance. Karyopharm is also witnessing a leadership change with the resignation of Michael Mason, the Executive Vice President, Chief Financial Officer, and Treasurer, effective November 5, 2024.
Two distinct firms, H.C. Wainwright and RBC Capital, have revised Karyopharm's financial outlook. H.C. Wainwright has adjusted the company's price target to $7.00 from $8.00, maintaining a Buy rating and slightly improving the full-year 2024 earnings per share (EPS) estimate. RBC Capital, on the other hand, has maintained an Outperform stock rating, highlighting the potential of the drug selinexor and projecting U.S. sales potential of $400-500 million.
These recent developments indicate a period of significant change for Karyopharm Therapeutics, with efforts underway to regain stock price compliance and find a successor for the CFO position.
InvestingPro Insights
As Karyopharm Therapeutics Inc. (NASDAQ:KPTI) advances its Phase 3 SENTRY trial for myelofibrosis, investors should consider some key financial metrics and insights from InvestingPro. Despite the company's focus on developing promising cancer treatments, it faces some financial challenges.
According to InvestingPro data, Karyopharm's market capitalization stands at $125.93 million, reflecting its current position in the biotech sector. The company's revenue for the last twelve months as of Q2 2024 was $145.67 million, with a notable gross profit margin of 89.19%. This impressive margin, highlighted as an InvestingPro Tip, suggests efficient management of production costs relative to revenue.
However, investors should be aware that Karyopharm is currently not profitable, with an operating income of -$130.95 million over the same period. An InvestingPro Tip warns that the company is quickly burning through cash, which is a critical consideration for a biotech firm investing heavily in clinical trials like SENTRY.
On a positive note, another InvestingPro Tip indicates that Karyopharm's liquid assets exceed its short-term obligations, providing some financial flexibility as it pursues its research and development goals.
For those interested in a deeper analysis, InvestingPro offers 5 additional tips that could provide valuable insights into Karyopharm's financial health and market position. These additional tips could be particularly useful for investors looking to understand the company's prospects as it progresses with the SENTRY trial and other pipeline projects.
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