Dafsolimab setaritox/Grisnilimab setaritox

Italfarmaco Group, based in Milan, Italy, is making headway against Duchenne muscular dystrophy (DMD). The company announced positive results from the Phase III EPIDYS study assessing Givinostat, a histone deacetylase (HDAC) inhibitor that received a Rare Pediatric Disease Designation from the U.S. Food and Drug Administration. The study met its primary endpoint, a positive change from baseline in the time to climb four stairs at 18 months compared to placebo. The results demonstrated a slower decline in performing this functional task in the Givinostat-treated group. Additionally, the company noted that key secondary endpoints were also met, consistent with the primary endpoint. Givinostat continued to demonstrate a tolerability pro line with previous studies. In all, the study results show that the addition of Givinostat to steroid treatment leads to clinical benefits for DMD patients. Italfarmaco intends to submit the data to the FDA as well as European regulators for potential approval. Additionally, the company will present the results of the Phase III study for publication in a peer-reviewed journal. “These topline Phase III results in DMD add to the growing dataset we have collected over the last years which have shown positive outcomes with our investigational therapeutic candidate, Givinostat, when taken together with steroids in patients with DMD, providing new hope for boys with DMD, their families and the medical community,” Paolo Bettica, chief medical officer of Italfarmaco Group said in a statement. “There is a tremendous unmet medical need for additional drugs to treat this debilitating rare disease and with these positive results, we intend to meet with regulatory agencies to share these findings and discuss a path forward to submit the complete dataset in a marketing application for potential approval.” Elsewhere around the Globe: Clover Biopharmaceuticals: Based in Shanghai, Clover announced new positive clinical data in individuals who have received a third dose of SCB-2019 (CpG 1018/Alum) as a homologous booster against the Omicron variant of COVID-19. The third dose demonstrated a significant, 19-fold increase in neutralizing antibody levels against the Omicron BA.2 variant compared to pre-booster levels among baseline seronegative participants. Immutep Limited: Australia’s Immutep announced new data from second-line non-small cell lung cancer patients in the Phase II TACTI-002 trial will be presented at the IASLC 2022 World Conference on Lung Cancer in August. EnGeneIC: Also based in Australia, EnGeneIC, is conducting studies of its COVID-19 vaccine that it says provides immunity against all known variants. Trials have shown the novel vaccine works by stimulating an entirely different immune pathway from other vaccines, producing “high affinity” antibodies that neutralize all COVID-19 variants, EnGeneIC said. Thirty-two healthy participants received two doses three weeks apart. Of those, 27 have passed the 28-day safety assessment with no side effects. Critically, they all have high-affinity antibodies capable of neutralizing all COVID-19 mutants, including Omicron. Noema Pharma: Switzerland-based Noema’s Investigational New Drug Application for a Phase IIb study of the company’s PDE10A inhibitor gemlapodect has been greenlit by the U.S FDA. The company will evaluate the drug candidate in Childhood Onset Fluency Disorder, also known as stuttering. The Phase IIb study is expected to read out at the end of 2023. Treos Bio Limited: Based in London, Treos Bio announced the dosing of its first patient in the Phase II OBERTO 301 study of PolyPEPI1018, the company’s lead product candidate. In the trial, PolyPEPI1018 is being combined with atezolizumab, Roche’s PD-L1 inhibitor, for the treatment of patients with late-stage microsatellite stable metastatic colorectal cancer. Ixaka Ltd.: Also based in London, Ixaka Ltd. and Germany’s Minaris Regenerative Medicine GmbH have signed an agreement that enables technology transfer and clinical and commercial manufacture of REX-001, Ixaka’s lead autologous multi-cell therapy. REX-001 is being developed for the treatment of chronic limb-threatening ischemia in patients with diabetes. The scope of the manufacturing agreement includes the scale-up and technology transfer with the goal of supplying the Phase II clinical trials and supporting commercial supplies in the EU and later in the United States. Xenikos B.V.: Located in The Netherlands, Xenikos enrolled the first patient in its pivotal Phase III trial, comparing T-Guard with ruxolitinib for the treatment of patients with Grade III or IV steroid-refractory acute graft-versus-host disease following allogeneic hematopoietic stem cell transplant. Xenikos expects the data from this study to support the submission of a Biologics License Application in the U.S. based on Day 28 data from the first 150 patients. Cyxone: Sweden’s Cyxone filed a submission to the National Institute of Pharmacy and Nutrition, the Hungarian Medical Product Agency, for approval for a Phase IIb study assessing drug candidate rabeximod as a potential treatment for rheumatoid arthritis. Applications will follow this first submission to relevant regulatory authorities in other European countries, the company said. Transgene: Based in France, Transgene and Sweden’s BioInvent International AB announced positive safety and progress in the ongoing Phase I/IIa study assessing BT-001 in patients with solid tumors, including melanoma. The initial data generated in Phase I part A demonstrated that BT-001 alone is well tolerated, with first signs of anti-tumor activity in a hard-to-treat population. BT-001 is based on Transgene’s oncolytic vector and is encoding BioInvent’s proprietary anti‑CTLA‑4 antibody. Confo Therapeutics: Belgium’s Confo Therapeutics has been awarded a €1.7 million grant from Flanders Innovation & Entrepreneurship that will support its discovery programs for novel GPCR candidates with its proprietary ConfoBody technology. MRM Health: Also based in Belgium, MRM Health announced it received regulatory approval from the Federal Agency for Medicines and Health Products in Belgium to initiate a Phase II trial with the novel next-generation optimized consortium therapy, MH002, in patients with Pouchitis, inflammation of the surgically constructed pouch following colectomy. SciSparc Ltd.: Israel-based SciSparc announced that it completed the development of its top-tier drug candidate SCI-110, which will be used in its upcoming Phase IIb study. "Our singular objective in developing SCI-110 is to develop a safe and effective treatment for this devastating, unmet medical need as soon as possible; and this milestone brings us one step closer," SciSparc CEO Oz Adler said.

T-Guard®, under development by Xenikos B.V., is being studied in comparison to ruxolitinib in patients with Grade III or IV SR-aGHVD CARY, N.C., June 28, 2022 /PRNewswire/ -- Veloxis Pharmaceuticals, an Ashai Kasei company, today announced dosing of the first patient by its partner, Xenikos, in a global pivotal Phase 3 clinical study [NCT04934670] designed to evaluate T-Guard® versus ruxolitinib for the treatment of patients with Grade III or IV steroid-refractory acute graft-versus-host disease (SR-aGVHD) following allogeneic hematopoietic stem cell transplant (allo-HSCT). T-Guard is currently being developed by Xenikos B.V., a privately-held biotechnology company that develops innovative immunotherapies for treating patients with severe immune disease and post-transplant rejection. In September 2021, Xenikos secured €40 million in convertible debt consisting of two equal tranches of €20 million, led by Veloxis Pharmaceuticals with participation from existing investors, Medicxi, RA Capital Management, Oost NL and Sanquinnovate. In connection with the financing, Veloxis has obtained two sequential call options to acquire the company. This study comparing T-Guard to ruxolitinib, if successful, can help bring a new treatment option to patients. Tweet this "Patients who experience SR-aGVHD have limited treatment options and sadly, their condition is often fatal" said Ulf Meier-Kriesche, chief scientific officer of Veloxis. "This Phase 3 study comparing T-Guard, with its novel mechanism of action, to ruxolitinib, if successful, can help bring a new treatment option to patients who urgently need it." "Veloxis is looking forward to the potential addition of T-Guard to its pipeline as we develop a broad portfolio of novel drugs to meet the needs of different groups of transplant patients," says Mark Hensley, CEO of Veloxis. Xenikos reached agreement with the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) on the design of this global, pivotal, randomized Phase 3 study, which is planned to enroll 246 patients and has been designed to test for superiority of T-Guard compared to ruxolitinib for the treatment of Grade III or IV SR-aGVHD. The study will be conducted at 75 transplant centers across the U.S. and Europe and will be executed in collaboration with the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). Patients will be randomized 1:1 to receive either T-Guard or ruxolitinib. Participants in the T-Guard arm will receive a one-week course of treatment with T-Guard as a four-hour infusion every other day. Each dose consists of 4mg/m2 body surface area (BSA). Participants in the ruxolitinib arm will receive 10mg of ruxolitinib twice daily for a minimum of 56 days. The primary endpoint of the study is complete response (CR) rate at Day 28, which is an important surrogate for long-term survival in patients with SR-aGVHD. Key secondary objectives include overall survival at Days 60, 90, 180 and 365, duration of complete response (CR), time to CR, overall response rate at Days 14, 28 and 56, GVHD-free survival, incidence of infections and safety. There will be a safety run-in phase at the beginning of the study whereby the Data and Safety Monitoring Board (DSMB) will evaluate the first 24 patients. Xenikos expects to report results from the safety run-in phase during the first half of 2023. The trial will also include an interim analysis for futility after 46 patients become evaluable for the primary endpoint. A second interim analysis will be performed once 150 participants have reached Day 28. Xenikos expects the data from this study to support the submission of a Biologics License Application (BLA) in the U.S. based on Day 28 data from the first 150 patients. Data from the full 246 patients is expected to support the submission of a Marketing Authorization Application (MAA) in the EU. Acute Graft-Versus-Host Disease Following allogeneic stem cell transplantation, most patients have a high risk of developing graft-versus-host disease (GVHD). With GVHD, the donor's immune cells attack the patient's cells. Acute GVHD occurs early after transplantation and can be relatively mild or quite severe, even life-threatening, if not treated. Although GVHD can often be treated successfully with steroids, few options are available if the disease progresses or becomes resistant to steroid treatment, and the long-term survival of patients with steroid-refractory acute GVHD (SR-aGVHD) is less than 20%, highlighting the urgent need for effective new therapies. T-Guard®: Helping Reset the Body's Immune System T-Guard is designed to reset the body's immune system in life-threatening T cell‒mediated conditions, potentially including prevention of transplant-related rejection, treatment of acute solid-organ rejection, and severe autoimmune disease. T-Guard consists of a unique combination of toxin-conjugated monoclonal antibodies that target CD3 and CD7 molecules on immune cells. Preclinical and early clinical testing have shown that T-Guard can specifically identify and eliminate mature T cells and NK cells with tolerable treatment-related side effects. T-Guard's action is believed to be short-lived, which may alter a patient's vulnerability to opportunistic infections compared to currently available therapies. T-Guard has been previously studied in a Phase 1/2 study in comparison to institutional historical controls in patients being treated second-line for SR-aGVHD following hematopoietic stem cell transplantation (HSCT). To learn more about that study, please find it here (link). T-Guard has been granted Orphan Drug Designation in both the EU and the U.S. About the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) The BMT CTN conducts rigorous multi-institutional clinical trials of high scientific merit, focused on improving survival for patients undergoing hematopoietic cell transplantation and/or receiving other cellular therapies. The BMT CTN has completed accrual to more than 50 Phase II and III trials at more than 100 transplant centers and enrolled over 16,000 study participants. BMT CTN is funded by the National Heart, Lung, and Blood Institute and the National Cancer Institute, both parts of the National Institutes of Health (NIH) and is a collaborative effort of 20 Core Transplant Centers/Consortia, the Center for International Blood and Marrow Transplant Research (CIBMTR), the National Marrow Donor Program (NMDP)/Be The Match and the Emmes Company, LLC, a clinical research organization. CIBMTR is a research collaboration between the NMDP/Be The Match and the Medical College of Wisconsin. The BMT CTN 2002 study is being led by Drs. Mehdi Hamadani, Protocol Officer, Scientific Director of CIBMTR, Medical College of Wisconsin; John Levine, Protocol Co-chair, Director of BMT Clinical Research, Mt. Sinai School of Medicine; Gérard Socié, Protocol Co-chair, Head of Hematology-Transplantation, Hôpital Saint-Louis; and Gabrielle Meyers, Protocol Co-chair, Associate Professor of Medicine, Oregon Health and Science University. More information about the BMT CTN can be found at bmtctn.net. About Xenikos Xenikos develops innovative immunotherapies based on conjugated antibodies. This novel therapeutic approach helps reset the immune system in patients who have a severe immune disease or have developed post-transplantation rejection. A randomized Phase 3 registration trial evaluating the company's flagship product, T-Guard® for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) is underway in the U.S. and Europe. About Veloxis Pharmaceuticals Veloxis Pharmaceuticals, Inc, an Asahi Kasei company, is a fully integrated specialty pharmaceutical company committed to improving the lives of transplant patients. Headquartered in Cary, North Carolina, USA, Veloxis is focused on the global development and commercialization of medications utilized by transplant patients and by patients with serious related diseases. For further information, please visit . About Asahi Kasei The Asahi Kasei Group contributes to life and living for people around the world. Since its foundation in 1922 with ammonia and cellulose fiber business, Asahi Kasei has consistently grown through the proactive transformation of its business portfolio to meet the evolving needs of every age. With more than 40,000 employees around the world, the company contributes to sustainable society by providing solutions to the world's challenges through its three business sectors of Material, Homes, and Healthcare. Its healthcare operations include devices and systems for acute critical care, dialysis, therapeutic apheresis, transfusion, and manufacture of biotherapeutics, as well as pharmaceuticals and diagnostic reagents. For further information, please visit . SOURCE Veloxis Pharmaceuticals