Drugs, positive results for innovative therapy against pulmonary hypertension

In the treatment of pulmonary arterial hypertension (PAH) (WHO group 1), the results of the phase 3 Stellar study are positive, which evaluated the safety and efficacy of sotatercept, potentially the first of a new class of drugs in clinical development phase, in addition to standard therapy. The trial reached the primary efficacy endpoint, demonstrating a statistically and clinically significant improvement in the ability to perform physical exercise after 24 weeks of treatment, assessed with the 6Mwd test that measures the distance traveled by patients in 6 minutes of walking. This was announced in a well-known MSD, a pharmaceutical company known as Merck in the United States and Canada. Pulmonary arterial hypertension – recalls the company – is a rare, progressive, high-mortality blood vessel disease characterized by constriction of small pulmonary arteries and high blood pressure in the pulmonary circulation. Currently, an estimated 40,000 people in the United States and 30,000 in the European Union are affected by the condition, which for many patients progresses rapidly despite the current standard of care. This condition causes significant strain on the heart, resulting in limitation of physical activity, heart failure and shortened life expectancy. The 5-year mortality rate for patients with Pah is about 43%. “The results observed in the Stellar study – says Dean Y. Li, president of Merck Research Laboratories – show the potential of sotatercept to transform patient treatment. with Pah. In the study sotatercept, added to the currently approved standard therapy, showed a significant primary efficacy outcome in improving walking distance in 6 minutes from baseline to 24 weeks. including a benefit over time to the clinical worsening of patients – he adds – are particularly relevant. We are moving rapidly, at a regulatory level, to make this experimental therapy available to patients “. In the study, 8 of the 9 secondary efficacy objectives – reports Msd again. – achieved statistically significant results, including the percentage of participants who achieved a improvement of the multicomponent parameter defined as improvement of the distance covered in 6 minutes (6Mwd) and of the level of cardiac function (value of the N-terminal pro-B natriuretic peptide, NT-proBNP); improvement or maintenance of WHO functional class II and time to death or clinical deterioration (Ttcw). The score of the Pah-Sympact questionnaire *, which assessed the cognitive / emotional impact – the last secondary outcome evaluated – did not reach statistical significance. The general safety profile was in line with what was observed in phase 2. The results of the study will be presented at an upcoming scientific congress. ActRIIA / Smad2 / 3) and antiproliferatives (mediated by BMPRII / Smad1 / 5/8) associated with remodeling of the pulmonary artery wall and right ventricle. It is a potentially first-in-class experimental activin receptor fusion protein of type IIA-Fc (ActRIIAFc) under development for the treatment of adult patients with pulmonary arterial hypertension (WHO group 1). The drug has received the designation of Breakthrough Therapy by the American Food and Drug Administration (Fda) and that of Priority Medicines by the EMA (European Medicines Agency) for the treatment of Pah, conditions that speed up its approval. MSD holds the exclusive rights to sotatercept in the area of ​​pulmonary hypertension through the acquisition of Acceleron Pharma Inc. The drug is the subject of a commercial agreement with Bristol Myers Squibb.