Immunoglobulin deposition disease

J&J’s Darzalex-VRd can now be given to newly diagnosed MM patients eligible for ASCT. Credit: Michael Vi/Shutterstock The European label for Johnson and Johnson Innovation Medicine’s (J&J) Darzalex (daratumumab) has been expanded to include patients with newly diagnosed multiple myeloma (MM) eligible for autologous stem cell transplant (ASCT). The label expansion for the anti-CD38 antibody was supported by data from the ongoing Phase III PERSEUS trial that was submitted to to the European Medicines Agency in March 2024. Darzalex initially received approval as a monotherapy for MM from the EC in May 2016. Along with MM, the antibody is marketed for treating primary system amyloidosis, and is in clinical studies for a range of oncological, immunological, and metabolic disorders. Projections from GlobalData estimate the drug will generate $11.5bn in revenue this year, set to increase to an annual $20bn in revenue by 2030. GlobalData is the parent company of Pharmaceutical Technology . See Also: TreeFrog and University of Pittsburgh probe TLS role in immuno-oncology Be Bio raises $82m to advance haemophilia B gene therapy to Phase I/II trial Co-formulated with Halozyme’s Enhanze drug delivery technology, Darzalex is the only subcutaneous, CD38-targeting MM treatment approved. The PERSEUS trial (NCT03710603) found that a Darzalex-VRd quadruple regimen resulted in a 58% reduced risk of disease progression or death compared to bortezomib, lenalidomide, and dexamethasone alone. Among 709 randomised MM patients, Darzalex therapy led to improved minimal residual disease (MRD) negativity (75.2% vs 47.5%), complete response (87.9% vs 70.1%), and sustained MRD negativity for ≥12 months (64.8% vs 29.7%). The quadruple regimen of Darzalex with bortezomib, lenalidomide, and dexamethasone received US Food and Drug Administration (FDA) approval for the same indication in July 2024, following FDA approvals to treat MM for Darzalex with bortezomib, thalidomide and dexamethasone (VDd) in 2019 and as a monotherapy in 2015. In the UK, Darzalex monotherapy was approved to treat MM by the National Institute for Health and Care Excellence (NICE) in January 2018. NICE later published guidance recommending Darzalex VDd for MM in April 2023.

Khondrion appoints Jasper Levink as Chief Financial and Business Officer NIJMEGEN, the Netherlands – 11 September 2024: Khondrion, a clinical stage biopharmaceutical company discovering and developing therapies targeting primary mitochondrial diseases, today announces the appointment of Jasper Levink MSc, as Chief Financial and Business Officer, effective immediately. Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: “We are delighted to welcome Jasper to the Khondrion Leadership Team as our new Chief Financial and Business Officer. His financial and business development experience will be extremely important as we continue to execute our strategy, progressing our late stage candidate, sonlicromanol, alongside the development of our pipeline and expansion of our clinical capabilities.” Jasper Levink, Chief Financial and Business Officer at Khondrion, added: “I am excited to be joining Khondrion at a pivotal time in its development, helping to drive the Company’s mission of bringing clinically meaningful medicines to patients living with devastating mitochondrial diseases. After the successful completion of four clinical trials of sonlicromanol and with preparations underway for registrational phase 3 studies, I am eager to bring my financial, operations and business development experience to the accomplished team and to work with medical and patient communities to leverage the transformational potential of sonlicromanol for people living with primary mitochondrial diseases.” Mr. Levink has over a decade of experience in business development and finance in biotech. Earlier roles include Partner and Managing Director at ttopstart, a boutique consulting company dedicated to business development and finance in biotech, and CBO at LenioBio, a German biotech pioneering the first eukaryotic cell-free protein expression platform. Recently he has taken up the role of CEO at SixtyFour Therapeutics, an early-stage biotech company developing therapies against autoimmune diseases. He will combine that role with his contributions to Khondrion. – Ends – Contacts: Khondrion BVProf. Dr. Jan Smeitink, CEOE-mail: info@khondrion.com Tel: +31-24-7635000www.khondrion.com ICR Consilium David Daley, Kris Lam Email: khondrion@icrhealthcare.com About KhondrionKhondrion is a clinical stage biopharmaceutical company developing therapies for patients with m.3243A>G primary mitochondrial diseases. The company’s lead asset, sonlicromanol, is a brain- penetrant, reductive and oxidative distress modulator with anti-inflammatory properties. One of the most advanced disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol has been tested in four clinical trials in patients with m.3243A>G primary mitochondrial disease, as well as in the first wave of a 6-month Phase 2 study in children with genetically-confirmed primary mitochondrial diseases and who suffer from motor symptoms. The compound has been granted orphan drug designations for the treatment of MELAS, Leigh disease and patients with maternally inherited diabetes and deafness (MIDD) in Europe, and for all inherited mitochondrial respiratory chain disorders in the US. It has also been granted a Rare Pediatric Disease designation in the US for the treatment of MELAS. Sonlicromanol and other compounds from Khondrion’s proprietary library have the potential to be developed for a wide range of diseases and conditions with the aim of benefiting patients whose daily lives are severely impacted by mitochondrial impairment. For more information visit www.khondrion.com. About mitochondrial disease Mitochondrial disease occurs when mitochondria, found within all cells of the human body and responsible for producing the energy necessary for cells to function, are defective. This can result in a wide range of serious and debilitating illnesses occuring shortly after birth or later in life. Signs and symptoms of these can include: cognitive problems, learning disabilities, blindness, deafness, heart failure, diabetes, fatigue, intolerance to exercise, muscle weakness and gait problems, and stunted growth. Orphan diseases of the oxidative phosphorylation system like Leigh disease, MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders, MIDD (maternally inherited diabetes and deafness), LHON (Leber's hereditary optic neuropathy) and other respiratory chain/ oxidative phosphorylation disorders, are all examples of mitochondrial disease.