Refractory sprue

The process for Canadians to access new medicines is complex and involves several different federal, provincial, and territorial agencies. As Roche Canada, we stand ready to work collaboratively to bring Columvi to Canadian patients who need it, as soon as possible. Positive recommendations from CADTH and INESSS are important steps towards public access to Columvi® (glofitamab for injection); provincial and territorial governments will make the final decision on public reimbursement.1,2 MISSISSAUGA, ON, March 7, 2024 /CNW/ - Hoffmann-La Roche Limited (Roche Canada) is pleased to announce that on March 5, 2024, the Institut National d'Excellence en Santé et en Services Sociaux (INESSS) recommended Columvi® (glofitamab for injection) for public reimbursement. This recommendation follows the recent positive recommendation from the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) issued on February 21, 2024. Reaching these important milestones is the result of close collaboration with both CADTH and INESSS and further emphasizes the need to move quickly through the subsequent steps to public reimbursement. Roche Canada now looks forward to working together with the pan-Canadian Pharmaceutical Alliance (pCPA), as well as the provincial and territorial governments, to achieve sustainable, equitable access for Canadian patients. Columvi is the only fixed treatment duration bispecific antibody authorized in Canada to treat diffuse large B-cell lymphoma.3,4,5 It received the first global marketing authorization when it was authorized by Health Canada almost one year ago on March 24, 2023. Since then, it has been authorized and publicly reimbursed in five other countries, and our aim is to ensure swift access to Columvi in Canada, as well. This recommendation shows that both CADTH and INESSS recognize the clinical evidence and benefit shown by Columvi for use in Canadians living with relapsed or refractory DLBCL. In Canada, it is estimated that in 2023, 4,000 Canadians were diagnosed with diffuse large B-cell lymphoma.6,7,8 "It is exciting to see our continued progress towards public access to glofitamab, a targeted bispecific antibody treatment for Canadians living with relapsed or refractory DLBCL," said Dr. John Kuruvilla, Hematologist in the Division of Medical Oncology and Hematology, Princess Margaret Cancer Centre. "This treatment is an important option to have available for patients that have received multiple prior treatments." "I am pleased to see INESSS and CADTH issue positive recommendations for glofitamab." said Dr. Nathalie Johnson, Hematologist at the Jewish General Hospital and Associate Professor at McGill University. "It is a fixed-duration treatment that could benefit patients with relapsed or refractory DLBCL in Quebec and the rest of Canada." "The positive recommendations for this therapy are wonderful news for relapsed or refractory diffuse large B-cell lymphoma patients as we are one step closer to having public access to this treatment option." said Antonella Rizza, CEO, Lymphoma Canada. Columvi is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, DLBCL arising from follicular lymphoma (trFL), or primary mediastinal B-cell lymphoma (PMBCL), who have received two or more lines of systemic therapy and are ineligible to receive or cannot receive CAR-T cell therapy or have previously received CAR-T cell therapy.9 Full details on the recommendation, including conditions for reimbursement, are available on the CADTH and INESSS websites. About Columvi (glofitamab for injection)9 COLUMVI (glofitamab for injection) is a bispecific monoclonal antibody that binds bivalently (with high avidity) to CD20 expressed on the surface of B cells and monovalently to CD3 in the T-cell receptor complex expressed on the surface of T cells. By simultaneous binding to CD20 on the B cell and CD3 on the T cell, COLUMVI mediates the formation of an immunological synapse with subsequent potent T-cell activation and proliferation, secretion of cytokines, and release of cytolytic proteins that results in the lysis of CD20-expressing B cells. COLUMVI comes as a concentrate for solution for infusion, with each vial containing either 2.5 mg (in 2.5 mL) or 10 mg (in 10 mL) of COLUMVI. Each mL contains 1 mg of COLUMVI. About Diffuse Large B-cell Lymphoma (DLBCL) Lymphoma is the name for a group of blood cancers that develop in your lymphatic system.10 Lymphomas occur in two main types: Hodgkin lymphoma and non-Hodgkin lymphoma. In particular, diffuse large B-cell lymphoma (DLBCL) is an aggressive (fast-growing) blood cancer and the most common form of non-Hodgkin lymphoma (NHL).11 While it is generally responsive to treatment in the frontline, as many as 40% of people will relapse or have refractory disease, where options may not offer optimal outcomes for patients and managing the disease can become complex.12 About Roche Canada At Roche Canada, patients and science are at the heart of everything we do. Our passion for science and our commitment to relentlessly pursuing the impossible for patients have made us one of the world's leading pharmaceutical, in-vitro diagnostics, and diabetes care management companies. With our combined strength in diagnostics and pharmaceuticals, we're driving personalized healthcare (PHC) forward, while ensuring we deliver meaningful benefits for patients and a sustainable healthcare system. Because we're committed to making quality healthcare accessible to everyone. And we're adding our expertise in new areas, such as artificial intelligence, real world data collection and analysis and collaborating with many different sectors and industries. Having the courage to reinvent ourselves and question the status quo is what patients and the healthcare system expect from Roche - and our commitment is as strong today as it was on the first day of our Canadian journey in 1931. Today, Roche Canada employs more than 1,800 people across the country through its Pharmaceuticals division in Mississauga, Ontario as well as its Diagnostics and Diabetes Care divisions in Laval, Quebec. For more information, please visit or follow Roche Canada on LinkedIn, or on X / Twitter @RocheCanada. SOURCE Roche Canada

A new research paper offers the most thorough look yet at a study that sparked the drug industrys recent experimentation with cell therapy for autoimmune diseases.The paper, which was published in The New England Journal of Medicine on Wednesday, comes from a team of scientists at the Friedrich Alexander University, Erlangen-Nurnberg in Germany. They detail how 15 people with severe autoimmune conditions experienced either disease remission or a sharp reduction in symptoms after an infusion of a kind of cell therapy known as CAR-T.The patients, who had tough-to-treat forms of lupus, inflammatory myositis or systemic sclerosis, have since been followed for a median of 15 months. None have needed medications they typically used to manage their conditions, and all treatment-related side effects were judged by investigators to be mild or moderate.The results were presented at a medical meeting in December, but the NEJM paper gives fuller detail of the study findings.The findings are remarkable, wrote John Isaacs, a professor of clinical rheumatology at Newcastle University, in an editorial also published Wednesday. Isaacs wasnt involved in the study.The paper adds to researchsuggestingCAR-T therapy, a method of engineering immune cells to fight cancer, might be applied to autoimmune disease. The idea is that CAR-T treatments, rather than destroying tumors, could eliminate the antibody-producing B cells that have malfunctioned in many autoimmune disorders.Though the study in Germany is small and lacks a placebo control, its results have already influenced a wide range of drugmakers. Since 2022, biotechnology and pharmaceutical firms have begun about a dozen clinical trials testing cell therapies in lupus. At least six others have received Food and Drug Administration clearance to begin testing.Drugmakers are also evaluating CAR-T therapies in other autoimmune conditions like myasthenia gravis and multiple sclerosis.Theres a huge potential for patients to really benefit, said Samit Hirawat, chief medical officer of Bristol Myers Squibb, one of the companies developing a CAR-T treatment for autoimmune disease, in a recent interview.The new NEJM results highlight the approachs promise, as well as the questions developers will have to answer in testing.The study suggests CAR-T may induce a reset of patients autoimmune disease. In the months following treatment, participants B cell networks were reconstituted, and the number of destructive autoantibodies associated with inflammatory disease diminished. Participants have gone up to two years after their B cells were replenished without signs of a relapse. Other markers of disease have also disappeared.Even though it is premature to judge whether these patients are indeed cured from their autoimmune disease CAR T cells at least appear to be able to achieve sustained disease- and drug-free remission, the study authors wrote.However, due to the trials small size and scope, its unclear how long the benefits from treatment will last, whether people might need to be retreated, or if any long-term health issues arise.When used in cancer, for example, CAR-T has in rare cases been associated with secondary malignancies.Published research has also linked depleted B cell counts to poor health outcomes from COVID-19, Isaacs noted. Multiple study participants contracted COVID-19 after therapy in the autoimmune disease study.The reduction but not elimination of some protective and vaccine-related antibodies raise the question of whether people will need new shots, the authors wrote.Additionally, developers must figure out the best chemotherapy conditioning regimen to prepare patients for treatment. Investigators in the German study used a pair of drugs that are typically administered before CAR-T therapies. But they noted how the field will need to determine whether and what doses are needed in an autoimmune disease setting, where the expectations for a drugs safety profile are different than with deadly cancers.If those problems can be solved and extended follow-up reinforces the current data, the benefit-to-risk ratio is likely to prove acceptable to both physician and patient, at least in certain cases of refractory disease, Isaacs wrote.Nonetheless, he cautioned, such therapy is in its early days, and there remains much to observe and understand. '

First patient dosed with ExoFlo™ in open-label trial AUSTIN, Texas--(BUSINESS WIRE)-- Direct Biologics LLC, a late-stage biotechnology company leveraging its regenerative medicine platform using extracellular vesicles (EVs) derived from bone marrow mesenchymal stem cells (MSCs) to address multiple disease indications, announced it has dosed the first patient in its Phase 1 clinical trial evaluating ExoFlo™ for the treatment of patients with medically refractory Crohn’s disease. “We are excited to announce the initiation of our Crohn’s disease clinical trial as we expand our ExoFlo clinical development program into inflammatory bowel disease,” said Amy Lightner, M.D., Chief Medical Officer of Direct Biologics. “Crohn’s disease is a debilitating lifelong condition that affects every aspect of daily life. With this new trial of ExoFlo, we are seeking to further illustrate the potential anti-inflammatory, immunomodulatory and regenerative properties of ExoFlo, each of which we believe to be crucial in the treatment of Crohn’s disease.” The open-label Phase 1 clinical trial (NCT05130983) is designed to evaluate the safety and efficacy of ExoFlo for the treatment of medically refractory Crohn’s disease. The trial is expected to enroll 10 patients between the ages of 18 and 75 who have been diagnosed with medically refractory moderate-to-severe Crohn’s disease of at least six months duration and have failed at least one monoclonal antibody therapy. The trial will randomize the 10 patients to two treatment arms. One arm will receive intravenous ExoFlo monthly, and the other arm will receive intravenous ExoFlo every other month. The primary objective for this trial is to ensure safety in this patient population by recording any adverse or serious adverse events. The secondary objective is to evaluate the efficacy of intravenous ExoFlo in inducing clinical response, clinical and endoscopic remission, and improving disease-specific, health-related quality of life. The trial will measure changes in laboratory values including C-reactive protein and fecal calprotectin, validated clinical scores, validated endoscopic scores, and validated patient survey scores, among others. About Crohn’s Disease Crohn’s disease is an idiopathic chronic inflammatory disease of the intestinal tract, which continues to increase in incidence for unknown reasons, resulting in a significant burden to the healthcare system. Crohn’s disease is characterized by transmural inflammation of the intestinal wall which can result in inflammatory, fibrostenosing or penetrating disease of the intestinal tract. This results in patients experiencing chronic abdominal pain, flares resulting in hospitalization or change in medications, and risk of surgical intervention. Despite the ever-growing armamentarium of immunosuppressive medications available, up to 70% of patients still undergo an intestinal resection for medically refractory disease. This current treatment paradigm presents significant potential side effects from approved medications, morbidity with frequent hospitalizations, risk with recurrent intestinal operations, and significantly impaired quality of life in thousands of patients. About Direct Biologics Direct Biologics is a late-stage biotechnology company leveraging a regenerative medicine platform which uses extracellular vesicles (EVs) secreted from mesenchymal stem cells to address multiple disease indications. Direct Biologics’ mission is to be the global leader in regenerative medicine through discovery, innovation, advancement of science, and treatment of patients in a safe and effective manner. Our therapeutic product candidate, ExoFlo, is designed to be a scalable, reproducible, and effective next-generation biologic that leverages our proprietary EV platform technology designed to reduce inflammation, modulate the immune system, and restore tissue through cellular regeneration. Direct Biologics is currently conducting the global Phase 3 EXTINGUISH clinical trial of ExoFlo for the treatment of hospitalized adults with Acute Respiratory Distress Syndrome (ARDS) due to COVID-19. In addition, the Company has initiated Phase 1 clinical trials with ExoFlo for the treatment of ulcerative colitis and Crohn’s disease, and expanded access trials in solid abdominal organ transplantation, and severe COVID-19 ARDS patients. Direct Biologics intends to pursue additional clinical applications including all-cause ARDS, perianal fistulizing Crohn’s disease, and necrotizing enterocolitis. Headquartered in Austin, Texas, Direct Biologics also has an R&D facility at the Center for Novel Therapeutics on the campus of University of California at San Diego, and operations and order-fulfillment center in San Antonio, Texas. For more information, please visit and follow us on Twitter @directbiologics.