So yes, the LITESPARK-015 clinical trial is a phase 2 clinical trial for patients with advanced and progressive pheochromocytomas and paragangliomas. This is based on the fact that a substantial number of those patients have abnormalities in several genes that regulate the energy production of the cell and that participate in the Krebs cycle, such as, for instance, the succinate dehydrogenase...
So yes, the LITESPARK-015 clinical trial is a phase 2 clinical trial for patients with advanced and progressive pheochromocytomas and paragangliomas. This is based on the fact that a substantial number of those patients have abnormalities in several genes that regulate the energy production of the cell and that participate in the Krebs cycle, such as, for instance, the succinate dehydrogenase. In addition, there are other molecular abnormalities that involve the hypoxia pathway, such as the protein of HIF-alpha, that may be also the target for belzutifan medical therapy. So with that in mind, a phase two clinical trial with belzutifan was developed for patients with metastatic, not curable, progressive pheochromocytomas and paragangliomas. The primary endpoint is the objective response rate. Secondary endpoints included the disease control rate, the duration of response, the progression-free survival, safety, and blood pressure control. We also had evaluation of quality of life. And in the clinical trial, we included 72 patients. 72 patients who had pheochromocytomas and paragangliomas that were not curable. All of them exhibit evidence of disease progression over a period of time no longer than 12 months. And as you may know, pheochromocytomas and paragangliomas frequently exhibit an excessive secretion of catecholamines. And yes, many of those patients had hypertension because of that, and they had adequate control of blood pressure before they entered into the clinical trial. In addition, patients required to have an acceptable performance status. All patients have histopathological confirmation of the diagnosis of pheochromocytoma and paraganglioma, and all of them had clinical and radiographic confirmation of evidence of disease progression that was not curable in any way. Now, regarding the primary endpoint of objective response rate, the objective response rate was 26%. In total, 19 patients out of 72 had a confirmed partial response. And the clinical benefit rate was 85%. Of interest in many patients who have a stable disease, whenever they had hypertension due to an excessive secretion of catecholamines. Many of them exhibit improvement in blood pressure, up to the point that 32% of patients were able to discontinue antihypertensive medications by at least 50% when compared with baseline for a period of time that was not shorter than six months. The duration of response was another very interesting aspect related to the clinical trial. The majority of patients exhibited a prolonged response to therapy. In fact, the median duration of response in the clinical trial was 20.4 months. And another very important aspect here is the progression-free survival, which was 22.3 months with an overall survival that was not reached in the median numbers. So the results were actually quite impressive in that regard. And something that to me is even more interesting is the preservation and improvement of quality of life that we noticed in the majority of patients who were treated with belzutifan in the clinical trial. In reality, more than 70% of patients exceeded improvement in global health status, as well as the performance of physical functioning that they had. And when you look at the individual symptom scales, majority of symptom scales were negative. That means that the majority of symptoms improved while they were responding to belzutifan in the clinical trial. What kind of symptoms? For instance, pain, the lack of appetite, the constipation, which could be quite overwhelming and related to the catecholamine excess, improving in the clinical trial substantially. And when you look at the safety profile of the medication, it’s quite similar to whatever we have seen in other clinical trials. We didn’t see really hypertensive crisis associated with belzutifan. What we mainly noticed was anemia, which happened in 88% of patients. There were no grade 5 adverse events. The number of grade 4 adverse events was only 3%, and 43% of patients had a grade 3 adverse event profile. So in general, the medication demonstrated to be active for patients with pheochromocytomas and paragangliomas. It improved symptoms related to pheochromocytomas, meaning hypertension, which is an important aspect of the disease. The quality of life of patients improves, and the safety profile is the one that has been previously described, making this medication actually a quite acceptable treatment for patients with pheochromocytomas and paragangliomas. And as you may know, currently, we don’t have any medications really approved for the treatment of pheochromocytomas and paragangliomas. And as you may know, currently we don’t have any medications really approved for the treatment of pheochromocytomas and paragangliomas around the world. And because of the duration of response that was noted in the clinical trial, because of the improvement in quality of life, and because of how impressive the primary endpoint was, yes, the medication was granted with an FDA approval on May 14 of this year. So this is currently the only medication FDA approved for the treatment of metastatic or not resectable pheochromocytomas and paragangliomas that are progressive in the United States.
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