Corcept Therapeutics is on a roll the last month because it has been able to establish two key events from its pipeline. For starters, it was able to report that it had met the primary endpoint of its pivotal phase 3 ROSELLA study for the treatment of patients with platinum-resistant ovarian cancer (PROC). This was a crucial study because the goal was to see if its cortisol-modulating drug, relacorilant, plus nab-paclitaxel would be able to beat nab-paclitaxel alone.
The primary endpoint was looking for progression-free survival [PFS] between the two dosing groups. This efficacy measure was met with statistical significance, in that the patients who took relacorilant plus nab-paclitaxel achieved a 30% reduction in the risk of disease progression compared to those who were only given nab-paclitaxel. It is important to note, though, that the PFS primary endpoint measure was that of assessment by a blinded independent central review [BICR].
Despite this data being reported, there are a few catalysts to look forward to soon enough. With the primary endpoint being met, the company is now in line to be able to file a New Drug Application to the FDA for relacorilant for the treatment of patients with platinum-resistant ovarian cancer for starters. Plus, the ability to file the Marketing Authorization Application to the European Medicines Agency [EMA] for this drug to treat this specific group of ovarian cancer patients as well.
Besides the ability for the company to achieve success, there is something far more important to consider here, which is that after front-line nab-paclitaxel [Abraxane], there is no recourse for these patients as the options are limited. This is where relacorilant comes into play, in that it could possibly be established as a new standard of care treatment option for platinum-resistant disease. The reason why is because the cortisol modulation that the drug provides is effective, but at the same time it provides a less burdensome treatment option. In essence, it is safer for patients to take, as opposed to the patients being given nab-paclitaxel.
The ability for the company to do well with targeting these ovarian cancer patients is based on its science in using cortisol modulation to regulate cortisol in the body, which is important in generating the stress response of a patient. It also plays a critical role in metabolism as well. For example, high levels of cortisol in metabolic dysfunction-associated steatohepatitis [MASH] patients lead to buildup of fat accumulation and inflammation. It is believed that reducing cortisol could reduce the buildup of fat around the liver and thus lead to reduced fibrosis.
The drug being developed to target patients with MASH is miricorilant and this is being tested in a phase 2 study to target these patients. The bottom line here is that cortisol affects multiple aspects of the body, and in essence, the development of cortisol drugs can be applied to a wide variety of disorders. Cortisol in the body can have an effect on the central nervous system [CNS], immune system, cardiovascular system, and bone.
Speaking of the ability of cortisol drugs developed by this company to target other disorders, the company several weeks ago announced that the FDA had accepted its New Drug Application of relacorilant for the treatment of patients with endogenous hypercortisolism [known as Cushing’s Syndrome as well]. Not only was the NDA of this drug to treat these patients accepted by the FDA, but the agency set a Prescription Drug User Fee Act [PDUFA] date of December 30th of 2025. This means that the U.S. agency could end up possibly approving the drug either on or before that date. This leads to another milestone to keep an eye on as this company continues to build upon its cortisol modulation drug development program.
Cushing’s syndrome is a devastating disorder and results when the body produces too much of the hormone cortisol. This is an important hormone, as it is responsible for several items in a person’s body, such as regulating blood pressure, breaking down fat substances, and suppressing inflammation, just to name a few. The thing is that this disorder just doesn’t happen by chance alone; it is caused by either a tumor that forms resulting in elevated levels of this hormone, or taking glucocorticoid medicine. Such glucocorticoids are given to patients with autoimmune disorders to suppress massive inflammation observed in them. While it helps in this regard, it can end up resulting in elevated levels of cortisol.
Obviously, if the tumor is the thing causing excess cortisol levels, then surgery to remove it might be an option alongside chemotherapy. In some cases, some therapies like ketoconazole can slow down the production of cortisol, allowing patients to achieve some relief. The hope is that this company ends up achieving U.S. marketing approval of relacorilant for these patients to give them a possible alternative treatment option.