Corcept (CORT) falls short in ALS while ‘unanticipated observation’ sent Keros (KROS) shares into freefall
Corcept Therapeutics Incorporated (NASDAQ: CORT) said after the markets closed on December 11 that its phase II trial evaluating its cortisol modulator dazucorilant in patients with amyotrophic lateral sclerosis (ALS) did not meet its primary endpoint. The primary endpoint was the change from baseline in the ALS Functional Rating Scale-Revised.
Though dazucorilant did not demonstrate any quality-of-life improvements to the ALS patients, Corcept was able to cite a very slim silver lining in that there were zero deaths in the 24-week study among patients receiving 300 mg dazucorilant. In contrast, five deaths occurred within the placebo arm. Another cohort in the study was administered 150 mg dazucorilant.
Corcept derives most of its revenue from Korlym, a drug to treat hypercortisolism in patients suffering from Cushing’s syndrome. The company’s science centers on modulating the effects of cortisol. Corcept expected dazucorilant could regulate cortisol in ALS patients and help slow neurodegeneration and reduce neuroinflammation. The company said patients who completed the study at the 300 mg dazucorilant dose could participate in an open-label, long-term extension study.
Because of expanded use of Korlym, Corcept’s stock climbed impressively this year. We issued a report forecasting a positive outcome for the phase II ALS trial in May 2024. At the time, Corcept’s stock traded at $27.04 per share.
Though the ALS trial results pushed Corcept shares down slightly on December 12, 2024, the company’s stock closed at $56.33 per share, reflecting an approximate 108% increase from the time of the May report. We are discontinuing coverage and exiting part of its position in CORT.
Just more than a week after Keros Therapeutics, Inc. (NASDAQ:KROS) licensed worldwide rights to its cancer drug elritercept for a $200 million upfront payment from Takeda (TSE:4502/NYSE:TAK), Keros disclosed on December 12 it stopped administering the two highest doses of its pulmonary arterial hypertension drug cibotercept in the phase II Tropos trial due to safety concerns. Following the elritercept deal, cibotercept was the lead asset of Keros. Investors pummeled Keros shares on the news, sending the company’s stock down ~70%.
Keros held back administering the two highest doses because of reports of excess fluid buildup in the pericardial sac around the heart. CEO Jasbir Seehra said on a conference call the fluid buildup was an unanticipated observation in the trial.
Cibotercept, transforming growth factor-beta (TGF-β) drug, works to treat PAH in a similar manner as Merck & Co., Inc.’s (NYSE:MRK) Winrevair (sotatercept). Sotatercept was approved in early 2024 to treat class I and class II PAH patients. Merck recently disclosed strong phase III data to support sotatercept as a treatment for class III and class IV (more severe) PAH patients.
In data reports from earlier cibotercept trials, Keros did not appear to disclose significant safety concerns regarding the drug. The company said it would continue dosing the 1.5 mg/kg cohort of the Tropos trial while halting the 3.0 mg/kg and 4.5 mg/kg dosing. Keros also said it would do more work to understand the fluid buildup which was not apparent in previous preclinical and healthy volunteer studies.
We issued a report forecasting a positive outcome for the Tropos trial in December 2024. Keros guided it planned to release the Tropos data in the second quarter of 2025.
Disclosures: Discontinuing coverage and exiting parts of position in CORT and KROS.