CD38

The detailed study results confirmed interim findings, showing stable kidney function and sustained treatment effect more than 18 months after the last dose of felzartamab Felzartamab, an investigational anti-CD38 monoclonal antibody, is a potential first-in-class therapeutic candidate for a range of rare immune-mediated indications with planning underway for Phase 3 development IgA Nephropathy (IgAN) is a leading cause of chronic kidney disease with up to 40% of IgAN patients progressing to end stage kidney disease about 20 years after diagnosis Oct. 26, 2024 -- Biogen Inc. (Nasdaq: BIIB) – today presented complete results from the Phase 2 IGNAZ study evaluating felzartamab, an investigational anti-CD38 monoclonal antibody, in people living with IgA nephropathy (IgAN). The results showed substantial reductions in proteinuria, stabilization of kidney function, and sustained treatment effect more than 18 months after the last dose of felzartamab. The complete results were shared during an oral presentation at Kidney Week 2024, the American Society of Nephrology’s annual meeting, in San Diego, California. "The complete results of the IGNAZ Study reaffirm our interim findings, showing a reduction in proteinuria, stabilization of kidney function, and sustained treatment effect more than 18 months after the last dose of felzartamab,” said Jonathan Barratt, MD, PhD, FRCP, Mayer Professor of Renal Medicine at the University of Leicester. “This is promising news for patients and supports the potential of felzartamab to be a meaningful treatment option for people living with IgA nephropathy, a leading cause of chronic kidney disease.” The Phase 2 IGNAZ study (n=54) explored the efficacy and safety of felzartamab in patients with IgAN and high risk of progressive kidney dysfunction. With respect to efficacy, patients receiving a nine-dose regimen of felzartamab over a six-month treatment period experienced substantial reductions in proteinuria levels as assessed by the urinary protein:creatinine ratio (UPCR) and stabilization of kidney function, as measured by the estimated glomerular filtration rate (eGFR), through 24 months. Notably, patients maintained a mean reduction of approximately 50% in the UPCR through month 24, which was more than 18 months after the last dose was administered. These results suggest that felzartamab may have the potential to preserve kidney function and be administered on treatment cycles instead of continuous dosing. Further analysis revealed that felzartamab administration resulted in selective and durable reductions in IgA antibody levels, while IgG and IgM levels recovered to baseline 3 months off-treatment. This selective reduction may offer maintenance of significant immune functions essential for infection protection. Overall, administration of felzartamab was generally well tolerated with a safety profile consistent with prior studies. “We are encouraged by the overall results of the IGNAZ study, especially given the significant unmet medical need for additional treatments to address high-risk IgA nephropathy,” said Uptal Patel, M.D., Head of Development, HI-Bio at Biogen. “We are grateful to all the participants, investigators and study staff who contributed to this study, whose findings will help us continue to evaluate felzartamab’s role in preserving kidney function as we plan for Phase 3.” Felzartamab is an investigational therapeutic human monoclonal antibody directed against CD38, a protein expressed on mature plasma cells. Felzartamab is a potential first-in-class therapeutic candidate with promise as a pipeline-in-a-product across a range of immune-mediated diseases. Felzartamab has been shown in clinical studies to selectively deplete CD38+ plasma cells, which may allow applications that ultimately improve clinical outcomes in a broad range of diseases driven by pathogenic antibodies. Felzartamab was originally developed by MorphoSys AG for multiple myeloma. Human Immunology Biosciences (HI-Bio) exclusively licensed the rights to develop and commercialize felzartamab across all indications in all countries and territories excluding China (including Macau and Hong Kong and Taiwan). Biogen acquired HI-Bio in July 2024. Felzartamab is an investigational therapeutic candidate that has not yet been approved by any regulatory authority and its safety and effectiveness have not been established. Immunoglobulin A nephropathy (IgAN) is the most common primary glomerulonephritis worldwide. It is a leading cause of chronic kidney disease with up to 40% of IgAN patients progressing to end stage kidney disease about 20 years after diagnosis. IgAN accounts for about 40% of all native-kidney biopsies in Japan, 25% in Europe, 12% in the United States, but less than 5% in central Africa.1 Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients’ lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth. References: Rajasekaran et al. (2021) IgA nephropathy: An interesting autoimmune kidney disease. Available at https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5198292/. Hastings et al (2018) Clinical Research, Life Expectancy for Patients From the Southeastern United States With IgA Nephropathy. Available at https://www.kireports.org/article/S2468-0249(17)30362-5/fulltext The content above comes from the network. if any infringement, please contact us to modify.

Johnson & Johnson (J&J) has announced that a subcutaneous (SC) formulation of Darzalex (daratumumab) has been approved by the European Commission (EC) as part of a combination treatment for newly diagnosed multiple myeloma (NDMM). The drug has been authorised for use in combination with bortezomib, lenalidomide and dexamethasone (VRd) to treat NDMM patients who are eligible for an autologous stem cell transplant (ASCT). The EC’s decision was supported by positive results from the late-stage PERSEUS study, which randomised more than 700 ASCT-eligible NDMM patients to receive either Darzalex SC plus VRd as an induction and consolidation therapy, followed by Darzalex SC and lenalidomide maintenance therapy, or VRd during induction and consolidation, followed by lenalidomide maintenance. The Darzalex-based regimen was shown to reduce the risk of disease progression or death by 58% compared to VRd after a median follow-up of 47.5 months, and resulted in deeper responses. More than 35,000 people were diagnosed with multiple myeloma (MM), an incurable blood cancer that affects a type of white blood cell called plasma cells, in the EU in 2022. First approved by the EC for MM in 2016, Darzalex is a monoclonal antibody that has been designed to attach to the protein CD38, which is found in high amounts on abnormal white blood cells in multiple myeloma. By attaching to CD38 on these cells, the drug activates the immune system to kill the abnormal white blood cells. Edmond Chan, senior director, EMEA therapeutic area lead haematology, Innovative Medicine, J&J, said: “The EC’s approval of this [Darzalex] quadruplet regimen marks a pivotal step forward in the treatment of NDMM. “By incorporating [Darzalex] SC into this regimen we are further optimising frontline therapy for patients, building on our aim to transform outcomes, and establish new standards of care for eligible patients from induction through to maintenance.” The authorisation comes less than a month after the EC approved J&J’s Yuvanci (single tablet combination therapy of macitentan and tadalafi) as a long-term treatment for pulmonary arterial hypertension. The regulator also recently approved the company’s Rybrevant (amivantamab) as part of a combination treatment for a new subset of advanced lung cancer patients.

SARASOTA, Fla. and MIAMI, Oct. 24, 2024 (GLOBE NEWSWIRE) -- NAYA Biosciences (“NAYA”) (NASDAQ: NAYA), a life science portfolio company dedicated to bringing breakthrough treatments to patients in oncology, autoimmune diseases, and fertility, today announced an update regarding its clinic trial plans. The Company received regulatory approval from the Israeli Ministry of Health in July 2024 and subsequent institutional review board clearance to initiate patient enrollment in up to 7 academic centers for its clinical trial evaluating the safety and efficacy of NY-303, its GPC3-targeting NK Engager bispecific antibody, as a monotherapy for the treatment of hepatocellular carcinoma in patients not responding to first-line immunotherapy. “With the completion of the recent merger, we can now begin our focus on planning for the initiation of clinical trials with our lead GPC3-targeting NK engager bispecific antibody, which is a significant milestone for NAYA Biosciences,” commented NAYA Biosciences President and NAYA Therapeutics CEO Dr. Daniel Teper. “We are passionate about bringing new options to patients not responding to first line immunotherapy and expect that positive Phase I safety and preliminary efficacy data will support accelerated clinical development in HCC as well as exploration in additional solid and pediatric tumors.” The Phase 1 part of the trial will consist of a dose-escalation where patients responding to treatment will continue weekly administration as long as no disease progression is observed. Key endpoints include safety, pharmacokinetics, activity markers, preliminary clinical efficacy, and time-to-progression. NAYA is planning to start patient recruitment in early 2025 at leading medical centers in Israel including Hadassah Hospital, Sheba Medical Center, and Sourasky Medical Center. The Phase 2a part of the trial is anticipated to be expanded to the United States and Europe subject to additional regulatory approvals and will evaluate NY-303 at two dose levels selected in Phase I. Key efficacy endpoints will include objective response rate and progression-free survival. “Hepatocellular carcinoma is a leading cause of cancer death worldwide with a rapidly increasing global trend,” commented NAYA Therapeutics Chief Medical Officer Dan Chiche, MD. “Currently, less than 30% of patients treated with current standard of care respond to immunotherapy (confirmed objective response) and mean progression-free survival is less than 7 months. NY-303’s dual targeting of NK cells and GPC3 is a unique, novel mechanism of action supported by scientific data showing potential to unlock non-response to checkpoint inhibitors.” About HCC Hepatocellular carcinoma (HCC) is the most common form of primary liver cancer, often developing in individuals with chronic liver conditions like hepatitis B or C, cirrhosis, and Metabolic Dysfunction-Associated SteatoHepatitis (MASH). HCC is a global health challenge, with over 900,000 new cases annually, making it the sixth most common cancer worldwide. The incidence of HCC in the United States has been steadily rising. Survival rates for HCC are often low due to late detection. The five-year survival rate for liver cancer in the United States is around 20%, but this varies widely depending on the stage at diagnosis. For early-stage HCC, where surgical resection or liver transplantation is possible, survival rates can improve dramatically, while advanced stages have far poorer outcomes. Globally, survival rates are similarly low, with wide variation based on healthcare access and screening programs. Recent advances in immunotherapy such as immune checkpoint inhibitors are offering new hope for patients with advanced HCC but new treatment approaches are still critical for improving survival and quality of life for patients. About NY-303 NY-303 is a first-in-class therapeutic candidate that leverages the engagement and activation of natural killer (NK) cells using a bispecific antibody targeting both GPC3, an oncofetal protein uniquely expressed on liver cancer cells, and NKp46, a cell surface receptor on natural killer (NK) cells that plays a central role in NK cell activation and the elimination of target cells. The unique mechanism enables NY-303 to turn the tumor form a “cold” into a ‘hot’ stage making liver cancer cells susceptible against immunotherapy to target and destroy cancer cells. This innovative approach is especially promising for patients with liver cancer, such as hepatocellular carcinoma (HCC), who face limited treatment options and poor survival rates but also for other GPC3-expressing tumors like lung squamous cell carcinoma, ovarian cancer, and pediatric cancer. Currently, liver cancer therapies include surgery, liver transplantation, targeted drugs, and immunotherapies like checkpoint inhibitors, but the prognosis remains grim, especially for advanced-stage disease. With a global five-year survival rate of around 20%, and even lower in more advanced cases, there is a critical need for more effective therapies. NY-303 represents a new opportunity for liver cancer patients by harnessing the body’s immune system to specifically attack tumor cells and engage NK-cells. Unlike traditional treatments, this therapy is designed to offer potent anti-cancer effects with a potentially safer profile, reducing the side effects often associated with chemotherapy and other conventional treatments and overcoming non-responding tumors against immunotherapies. For patients with HCC, NY-303 could significantly improve outcomes by offering a more precise, targeted approach to eradicating cancer, which is critical given the high recurrence rates and challenges in treating advanced liver cancer. As part of the next generation of immunotherapies, NY-303 may redefine treatment possibilities and offer renewed hope to those facing this difficult diagnosis. About NAYA BiosciencesNAYA Biosciences (NASDAQ: NAYA) is a life science portfolio company dedicated to bringing breakthrough treatments to patients in oncology, autoimmune diseases, and fertility. Our hub & spoke model harnesses the shared resources of a parent company and agility of lean strategic franchises, enabling efficient acquisition, development, and partnering of assets and allowing for optimized return on investment by combining scalable, profitable commercial revenues with the upside of innovative clinical-stage therapeutics. NAYA’s expanding portfolio of assets currently includes NY-303, a GPC3 and NKp46 targeting bispecific antibody for the treatment of hepatocellular carcinoma (HCC) with a unique mode of action targeting non-responders to the current immunotherapy standard of care (approximately 70% of the current treatable market) cleared to enroll patients in a Phase 1/2a monotherapy trial in early 2025, NY-338, a CD38 and NKp46 targeting bispecific antibody for the treatment of multiple myeloma and autoimmune diseases with a differentiated safety and efficacy profile, and INVOcell®, an FDA-approved fertility device which has demonstrated comparable success rates to traditional In Vitro Fertilization (IVF). Safe Harbor StatementThis release includes forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The Company invokes the protections of the Private Securities Litigation Reform Act of 1995. All statements regarding our expected future financial position, results of operations, cash flows, financing plans, business strategies, products and services, competitive positions, growth opportunities, plans and objectives of management for future operations, as well as statements that include words such as "anticipate," "if," "believe," "plan," "estimate," "expect," "intend," "may," "could," "should," "will," and other similar expressions are forward-looking statements. All forward-looking statements involve risks, uncertainties, and contingencies, many of which are beyond our control, which may cause actual results, performance, or achievements to differ materially from anticipated results, performance, or achievements. Factors that may cause actual results to differ materially from those in the forward-looking statements include those set forth in our filings at www.sec.gov. We are under no obligation to (and expressly disclaim any such obligation to) update or alter our forward-looking statements, whether as a result of new information, future events or otherwise. NAYA Investor & Media ContactAnna Baran-DjokovicSVP, Investor Relations+1-305-615-9162anna@nayabiosciences.com