The European Medicines Agency (EMA) is still in the process of reviewing odronextamab for its effectiveness in treating Relapsed or Refractory Diffuse Large B-cell lymphoma (R/R DLBCL) and Relapsed or Refractory Follicular Lymphoma (R/R FL). Within the European Union, odronextamab has received special status as an Orphan Drug for DLBCL and FL. However, its potential use for R/R DLBCL and R/R FL is presently being studied in clinical trials and has not yet been approved by any regulatory body.
Novo Nordisk Bolsters Cardiovascular Disease Portfolio with Cardior Pharmaceuticals Acquisition
Novo Nordisk and Cardior Pharmaceuticals have revealed that Novo Nordisk will be acquiring Cardior for a sum of up to 1.025 billion Euros. This includes an initial payment as well as potential additional payments contingent upon the accomplishment of specific milestones in development and commercialization. Cardior stands at the forefront of pioneering treatments that focus on RNA to prevent, repair, and reverse heart diseases. Through targeting unique non-coding RNAs, the company aims to tackle the fundamental reasons behind cardiac dysfunctions, with the ultimate goal of creating enduring benefits for patients.
The deal encompasses Cardior’s primary drug candidate CDR132L, presently undergoing phase II clinical trials for heart failure treatment. This acquisition marks a significant advancement in Novo Nordisk’s plan to enter the cardiovascular disease sector. Novo Nordisk is dedicated to creating a purposeful and influential collection of treatments, utilizing both internal research and external collaborations, to tackle the substantial gaps that remain in addressing cardiovascular disease, the leading cause of mortality worldwide.
Martin Holst Lange, Novo Nordisk’s executive vice president for Development, stated that by incorporating Cardior into Novo Nordisk, they aim to fortify their lineup of projects in cardiovascular disease. Novo Nordisk already has ongoing programs in all stages of clinical development in this area. Lange emphasized their admiration for Cardior’s scientific endeavors, particularly highlighting CDR132L. This treatment boasts a unique mechanism of action and the potential to be a pioneering therapy intended to slow down or partially reverse the progression of heart failure in individuals.
Novo Nordisk intends to start another phase II study focusing on CDR132L in individuals with chronic heart failure and cardiac hypertrophy, a condition leading to thickened and rigid heart muscle walls, which hampers the heart’s pumping function. This move will not influence Novo Nordisk’s earlier projected operating profit for 2024 or its current share buy-back program. The funding for this acquisition by Novo Nordisk will come from its existing financial reserves.
CHMP Greenlights Novartis Fabhalta – First-Ever Oral Monotherapy for Adult PNH Patients
Novartis has reported that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a favorable recommendation for the approval of Fabhalta (iptacopan). This recommendation is for its use in treating hemolytic anemia in adults with paroxysmal nocturnal hemoglobinuria (PNH).
According to Antonio Risitano, M.D., Ph.D., President of the International PNH Interest Group and Head of the Hematology and Hematopoietic Transplant Unit at the AORN San Giuseppe Moscati in Avellino, Italy, Fabhalta has the potential to significantly impact patient care by alleviating the challenges faced by individuals with PNH. Backed by substantial evidence and a proven track record of safety, Fabhalta may revolutionize treatment approaches. Clinical trials have shown that oral iptacopan offers superior improvement in hemoglobin levels without necessitating red blood cell transfusions, surpassing the performance of anti-C5 therapies. This could lead to the normalization of hemoglobin levels for most patients, marking a potentially groundbreaking advancement for those managing this persistent blood disorder.
The CHMP’s positive decision was influenced by strong data from two Phase III trials: APPLY-PNH, which involved patients with persistent anemia despite prior anti-C5 therapy, comparing those who switched to Fabhalta with those who continued anti-C5 treatment; and APPOINT-PNH, which focused on patients new to complement inhibitors. In APPLY-PNH, after 24 weeks, 82.3% of Fabhalta patients previously on anti-C5 treatment achieved a sustained increase in Hb levels of ≥2 g/dL without needing transfusions, compared to only 2.0% of those on anti-C5 (a significant difference of 80.2%, P<0.0001). In APPOINT-PNH, 92.2% of patients new to complement inhibitors, and using Fabhalta achieved this same outcome. APPLY-PNH also revealed a notable transfusion avoidance rate of 94.8% for Fabhalta patients previously on anti-C5, compared to 25.9% for those continuing anti-C5 treatment (a significant difference of 68.9%, P<0.0001). Both studies demonstrated Fabhalta’s effectiveness in controlling intravascular hemolysis (IVH), indicated by maintaining mean lactate dehydrogenase (LDH) levels below 1.5 times the upper limit of normal. APPLY-PNH patients also reported feeling less fatigued, as shown by improvements in Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-F) scores. Across both APPLY-PNH and APPOINT-PNH, the safety profile of iptacopan remained consistent.
After the CHMP suggests approving Fabhalta for adult PNH patients with hemolytic anemia, the European Commission (EC) will make a conclusive judgment in around two months.
Idorsia’s TRYVIO Gets FDA Nod, First-of-Its-Kind Endothelin Receptor Antagonist for High Blood Pressure When Other Antihypertensives Fall Short
Idorsia Ltd has revealed that the FDA granted approval for TRYVIO (aprocitentan) to address hypertension in conjunction with additional antihypertensive medications, aimed at decreasing blood pressure in adult patients whose current drug regimens are not effectively managing their condition. Lowering blood pressure diminishes the likelihood of fatal and non-fatal cardiovascular incidents, notably strokes and heart attacks. The suggested dose for TRYVIO is 12.5 mg taken orally once per day, with or without food.
“Presently, there exist millions of Americans whose blood pressure remains inadequately managed despite available treatments. This poses a significant public health concern, contributing to a heightened occurrence of cardiovascular and cerebrovascular incidents. To combat this challenge, Idorsia has developed aprocitentan, an endothelin receptor antagonist designed for these particular patients. Idorsia undertook an extensive clinical initiative involving patients who continued to experience hypertension despite being on at least three medications at their optimal doses, and at times even four, five, or six antihypertensives. I am immensely proud of the Idorsia team and delighted that physicians will soon have a fresh therapeutic avenue for patients grappling with uncontrolled blood pressure.”
Jean-Paul Clozel, MD and Chief Executive Officer of Idorsia
TRYVIO (aprocitentan) is a type of endothelin receptor antagonist that works by blocking the interaction between endothelin (ET)-1 and the ETA and ETB receptors. The actions of ET-1 closely resemble the mechanisms behind hypertension, and ET-1 plays a significant role in stimulating the production of aldosterone. Before the approval of TRYVIO, no systemic medications for lowering blood pressure specifically targeted the ET pathway. Instead, approved antihypertensive treatments focused on methods such as controlling salt and water balance (diuretics), countering the renin-angiotensin-aldosterone system (RAAS), reducing the entry of extracellular calcium into cells (calcium channel blockers), decreasing sympathetic activity (beta blockers, central alpha-agonist agents), or inducing non-selective vasodilation.
TRYVIO underwent assessment as a standalone treatment in a Phase II investigation involving individuals with hypertension. Additionally, it was examined as an additional therapy in a Phase III trial named PRECISION among patients with confirmed resistant hypertension. During PRECISION, aprocitentan was not only well received but also demonstrated superiority over a placebo in reducing blood pressure by week 4, maintaining this effect through week 40.
AbbVie Grows Its Presence in Inflammatory and Autoimmune Diseases with Landos Biopharma Acquisition
AbbVie Inc. and Landos Biopharma, Inc. have revealed a firm arrangement in which AbbVie is set to purchase Landos, a clinical-stage biopharmaceutical enterprise concentrating on pioneering oral treatments for individuals with autoimmune illnesses. At the forefront of Landos’ portfolio is NX-13, a groundbreaking oral NLRX1 agonist (a part of the NOD-like receptor family) with a dual-action mechanism that is both anti-inflammatory and aids in the restoration of epithelial tissue.
“We are acquiring Landos with the goal of advancing the clinical development of NX-13, an innovative, first-of-its-kind oral medication that holds promise for improving the lives of individuals with ulcerative colitis and Crohn’s disease,” stated Dr. Roopal Thakkar, Senior Vice President and Chief Medical Officer of Global Therapeutics at AbbVie.
“This announcement underscores the exceptional capabilities of Landos’ skilled team and their dedication to our shared mission of developing oral therapies to address unmet medical needs,” remarked Gregory Oakes, President and CEO of Landos. “NX-13, with its unique bimodal mechanism of action, could offer a fresh approach to treating ulcerative colitis and Crohn’s disease. With AbbVie’s expertise in therapeutic areas and global development, we believe they are well-positioned to propel NX-13 forward.”
NLRX1 plays a role in controlling the balance between immune function and metabolism, as well as in regulating inflammation. Its activation affects various aspects of how inflammatory bowel disease (IBD) develops. The NEXUS clinical trial, a randomized Phase II study of NX-13 for ulcerative colitis (UC), is presently accepting participants in the United States and Europe (NCT05785715). AbbVie has agreed to acquire Landos for $20.42 per share in cash upon completion, totaling approximately $137.5 million, along with an additional non-tradable contingent value right per share worth up to $11.14, around $75 million in total. This contingent value right is based on achieving a clinical development milestone. The transaction is anticipated to be finalized in the second quarter of 2024, pending customary closing requirements and approval from Landos’ shareholders.
