Macitentan

Two-thirds of patients saw a sustained reduction in their pulmonary arterial pressure. Cereno Scientific’s hypertension drug was shown to improve the risk score of 43% of patients in a phase 2 study.Of the 21 participants with pulmonary arterial hypertension (PAH), nine demonstrated an improved risk score—a measurement of the risk of death from the condition—after 12 weeks of treatment with Cereno’s histone deacetylase (HDAC) inhibitor, dubbed CS1.A further six patients reported a stable risk score, meaning 71% of patients saw their score either improve or hold steady, the Swedish biotech noted in a post-market release Friday.Two-thirds of patients saw a sustained reduction in their pulmonary arterial pressure, while a third reported an improved functional class, a term for symptoms and physical capacity.Cereno’s shares were trading up 25% on the Stockholm stock exchange at 8.72 Swedish krona on Monday afternoon local time compared to a Friday closing price of 7 krona.The trial was also a success on its primary endpoint of proving the safety and tolerability of CS1, with no treatment-related serious adverse events. No changes in liver lab values or clinically significant drug-related bleedings or decreases in platelet counts were recorded, Cereno said. The company is now looking at moving CS1 into a pivotal trial and said it will use Friday’s “encouraging results” as the basis for opening talks with regulators.“We are excited to take the next steps in our journey to make a meaningful impact for patients with PAH,” Cereno’s Head of R&D Rahul Agrawal said in the release.“The CS1-003 trial demonstrated compelling signs of clinical efficacy already over a 12-week treatment period and we expect to see additional positive impact of CS1 with longer use of our drug in patients with PAH,” Agrawal added.HDACs help regulate gene expression. The FDA has already improved four HDAC inhibitors to treat blood cancers, including Merck & Co.’s Zolinza. Cereno is hoping CS1 will have success against PAH by “targeting the root cause of the disease, aiming to reverse the pathological vascular remodeling of the small lung arteries.”PAH is a rare, rapidly progressing disease in which the blood vessels narrow in the lungs, increasing blood pressure and leading to heart failure. Johnson & Johnson dominates the PAH market with its prostacyclin receptor agonist Uptravi and the endothelin receptor agonist Opsumit.In March, Merck & Co. muscled in on the action with the approval of the activin signaling inhibitor Winrevair, which the pharma has touted as acting directly on the underlying problem that causes the disease.Despite the increasingly crowded space, Cereno claimed in its release that “existing treatment alternatives are insufficient and do not address the root cause of the disease.”

The European Commission has approved Johnson and Johnson’s Yuvanci for pulmonary arterial hypertension, offering the first single-pill treatment option to European patients. Credit: andreswd via Getty Images. The European Commission (EC) has approved Johnson and Johnson’s Yuvanci (macitentan + tadalafil) for the treatment of pulmonary arterial hypertension (PAH). The approval marks Yuvanci as the only single-tablet combination therapy available for patients with PAH. PAH is a rare, progressive disorder in which patients have high blood pressure in the pulmonary arteries for no apparent reason. Current treatment for PAH involves first-line dual combination therapy with macitentan and tadalafil for patients without cardiopulmonary comorbidities. This currently consists of a multi-pill regimen. “This can deeply impact their [PAH patients’] daily lives as they might struggle to maintain adherence to their treatment regimen,” said Dr. Tamara Werner-Kiechle, the Europe, Middle East, and Africa (EMEA) therapeutic area lead for neuroscience and cardiopulmonary drugs at Johnson & Johnson Innovative Medicine in a 30 September press release. Johnson and Johnson has developed the Yuvanci as a substitution therapy for the long-term treatment of PAH in adults of the World Health Organization’s (WHO) Functional Class (FC) II to III. This includes patients with PAH that causes a slight limitation in physical activity, yet they are still comfortable at rest, and “patients with marked limitations to physical activity”, as per WHO. Patients will need to have already been treated with a two-pill combination of macitentan and tadalafil to be eligible for Yuvanci. Johnson and Johnson currently markets macitenan monotherapy under the brand name Opsumit, whilst Eli Lilly sells tadalafil as Cialis for PAH. The approval is based on data from the Phase III A DUE study (NCT03904693) , which also led to a positive recommendation for Yuvanci’s approval by the European Medicines Agency’s (EMA) Committee for Human Medicinal Products in July. The pharmaceutical giant presented data from the Phase III A DUE clinical study at the American College of Cardiology’s 72nd Annual Scientific Session. In the double-blind study, Yuvanci-treated patients showed significant improvements in the co-primary endpoints of pulmonary vascular resistance (PVR) and pulmonary haemodynamic improvement. Patients who took the combination therapy demonstrated a 44% decrease in PVR compared to a 22% decrease in the Adcirca monotherapy group. See Also: Almac Group North American Headquarters Expansion, Pennsylvania, USA LOTTE BIOLOGICS’ East Syracuse Pharmaceutical Manufacturing Facility, US Opsumit is an endothelin receptor antagonist that treats PAH by inhibiting pathways related to vasoconstriction, fibrosis and proliferation. Meanwhile, Cialis is a phosphodiesterase type-5 inhibitor that treats the disorder by increasing cGMP, leading to a relaxation of the pulmonary vascular smooth muscle cell and vasodilation of the pulmonary vascular bed. Johnson and Johnson submitted a new drug application to the US Food and Drug Administration (FDA) for Yuvanci in May 2023, receiving an approval for its submission in March 2024.