Malignant ascites

The European Medicines Agency’s human medicines committee again decided not to recommend the renewal of the conditional marketing authorization for PTC Therapeutics’ Duchenne muscular dystrophy drug Translarna. The treatment has gone through multiple reviews by the committee. Friday’s decision is the fourth time the EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended against the drug. The CHMP first decided against Translarna in September 2023, then confirmed that decision in January 2024, following a re-examination requested by PTC Therapeutics. In June, the committee reviewed the drug after the European Commission asked the committee to consider additional data — but the committee’s decision remained the same. PTC Therapeutics asked the committee again to reconsider in July, which led to this fourth decision. Since its initial authorization in 2014, PTC Therapeutics has carried out two more studies in an attempt to show the drug’s effectiveness. The first study failed to confirm the efficacy of the treatment, though the data suggested that a subgroup of patients — those with a progressive decline in their ability to walk — might see more efficacy. A second study did not reach statistical significance in that subgroup of patients. The drug will remain available while the EMA reviews CHMP’s recommendation. “CHMP again based its decision on the results of the primary analysis subpopulation of Study 041 instead of the totality of evidence for Translarna,” PTC Therapeutics CEO Matthew Klein said in a statement . Leerink Partners wrote Friday that they expect this to be “the final negative opinion,” adding that although PTC Therapeutics resubmitted an NDA to the FDA in late July, there have been no more updates in the US, and they “remain skeptical on the likelihood of US approval.” In addition to the Translarna decision, here’s a look at the medicines that CHMP recommended ahead of a potential thumbs-up from the European Commission:

“Bispecific Antibodies Sales Surpassed USD 8 Billion In 2023 With More Than 50% YOY Growth” Bispecific antibodies represent a cutting edge class of therapeutic agents at the forefront of revolutionizing modern medicine. These molecules, engineered to bind simultaneously to two different targets, offer a unique and potent approach to treating a variety of diseases, particularly cancer. With their ability to harness the body's immune system to precisely target tumor cells while sparing healthy tissue, bispecific antibodies have garnered significant attention in both clinical and commercial markets across the globe. Clinically, bispecific antibodies clench immense promise as a cornerstone of precision medicine, offering targeted therapy tailored to individual patients' molecular pro By engaging immune effector cells, such as T cells or natural killer (NK) cells, bispecific antibodies orchestrate precise and potent immune responses against cancer cells, leading to tumor regression and improved patient outcomes. The versatility of bispecific antibodies allows for the development of therapies targeting various tumor antigens and immune cell populations, spanning a broad range of cancers and other ailments. Download Research: Besides that, the commercial market for bispecific antibodies is experiencing rapid growth, driven by the increasing demand for innovative and effective therapies across diverse therapeutic areas. The approval and commercial success of several bispecific antibodies, such as blinatumomab for acute lymphoblastic leukemia (ALL) and catumaxomab for malignant ascites, have validated the therapeutic potential of this class of molecules and spurred further investment and development. Pharmaceutical companies, biotechnology firms, and academic institutions are actively pursuing bispecific antibody programs, capitalizing on advancements in antibody engineering technology and strategic collaborations to accelerate the translation of promising candidates into clinical market. Several key factors driving the clinical and commercial success of bispecific antibodies include rise in cancer incidences, augmenting research and development, involvements of various stakeholders from the pharma and biotech company along with fostering research collaborations and investments in the realm. For instance, in December 2023, a research collaboration and license agreement are signed by Elpiscience and Astellas for the novel bispecific macrophage engager, ES019, and other programs. For these two programs, both the companies will work together to conduct early-stage research. In addition to cancer malignancies, other disorders like autoimmune diseases, infectious diseases, and neurological disorders are also being researched in the sphere. Their adaptable mode of action and capacity to interact with various targets render them appealing options for tackling unfulfilled medical requirements in various therapeutic domains. For instance, in a study conducted by researchers at The University of Texas MD Anderson Cancer Center, in 2023, developed fully human bispecific antibodies that bind to two different epitopes of the receptor binding domain (RBD) of the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) spike protein and neutralize a wide range of variants including the Omicron variant. One of the key prime movers which aid to expand the market of bispecific antibodies is the bestow from regulatory agencies and augmenting approval to novel and innovative bispecific antibodies. For instance, in August 2023, European Commission (EC) has approved TALVEY (talquetamab) for use as a monotherapy in treating adult patients with relapsed and refractory multiple myeloma (RRMM). Talquetamab is a bispecific antibody that engages T cells specifically by binding to CD3, which is present on the surface of T cells, and G protein-coupled receptor class C group 5 member D (GPRC5D), a novel target that is expressed on the surface of hard keratinized tissues and multiple myeloma cells. Looking ahead, the commercial market for bispecific antibodies holds immense potential for growth and expansion. Continued research, clinical development, and regulatory approvals will be instrumental in unlocking the full therapeutic potential of bispecific antibodies and addressing unmet medical needs across diverse patient populations. With their transformative impact on patient care and the potential to reshape the treatment landscape, bispecific antibodies are poised to play a pivotal role in advancing the future of medicine and improving outcomes for patients worldwide.

“Currently More Than 180 Oncolytic Virus Immunotherapies Are In Clinical Trials” Conventionally, the management of cancer was predominantly restricted to surgery, radiation therapy, in addition to chemotherapy. Even though these therapies have shown significant abolition of primary tumors, their allied systemic toxicities and the high incidences of relapse are the foremost limitations. The escalation in prevalence of cancer and the curbs of conventional therapies have propelled the demand of novel targeted therapies which can overcome the limitations and have enhanced specificity and target ability towards the disease. One such approach is using the virus as a therapeutic approach to deliver cancer fighting genes to the tumor cell and thereby inducing an immuno modularity response that will aid to cause death in cancerous cell. This led the development of oncolytic virus therapy as a promising treatment to cure rising cancer malignancies. The oncolytic virus therapy clenches long history which can be backpedalled the late 1950s to 1970s epoch in which the multiple clinical trials and studies were ongoing with the wild type viruses in order to understand a novel approach to treat tumor cells. Moreover, oncolytic viral therapy provides enormous advantage such as higher response towards cancer cells owing to the presence of impaired antiviral defense in addition to better delivery of therapeutic payloads to tumor cells as compared to the conventional treatment which aims to reduce or delays the symptoms of cancer. Download Oncolytic Virus Immunotherapies Clinical Trials Landscape Research: The research and development picked up its pace ever since the oncolytic virus therapy called Oncorine was approved by the National Medical Products Administration (NMPA) to treat Head and neck cancer in the year 2005. However, the drug was only accessible in China owing to the strict rules and regulations. Nevertheless, the approval of Oncorine has provided surplus thrust to the research and development across the globe which led to the approval by FDA for the first viral therapy, T- VEC (Imlygic) to treat melanoma. One of the primary impetuses for the growth of oncolytic virus therapy domain was due to the rising ascendancy of these therapies in the clinical studies. Preliminary data from the clinical trial studies demonstrates that more than 150 clinical researches are currently ongoing for the treatment of innumerable seldom and common diseases such as Alzheimer disease, herpes simplex 1, advanced biliary tract cancer, chronic hepatitis b, refractory malignant ascites as well as hepatocellular carcinoma. The clinical pipeline for the domain of viral therapy is mounting at an incredible gait. For instance, a modified human recombinant type 5 adenovirus with genetic modifications H101 is a novel treatment regimen used to provide treatment for patients suffering from advanced biliary tract cancer. The study is lately in the phase I clinical stage which is sponsored and collaborated by Fudan University. To further enhanced in addition to improve the efficacy, novel viral oncolytic therapy are used in combination with other drugs with the purpose to provide improved and advanced treatment regimen to the patients suffering from cancer. For instance, a novel Telomerase-specific Type 5 Adenovirus OBP-301 viral therapy in conjugation with Carboplatin, Paclitaxel as well as radiation therapy is used to cure adenocarcinoma. The clinical studies in lately in the phase I clinical stage which is sponsored and collaborated by NRG Oncology and National Cancer Institute (NCI). Researches believed that the oncolytic virus therapy clench potential as well as long lasting promising therapeutic effects. As a consequence, multiple pharmaceutical companies along with centers such as City of Hope Comprehensive Cancer Center, New York Medical College, Children's Hospital of Pennsylvania, Mayo Clinic in Arizona, Peking University First Hospital as well as Beijing Tiantan Hospital, Capital Medical University are working recklessly for the purpose to develop advanced oncolytic virus therapy that controls the prognosis of cancer diseases along with other indications such as advanced bladder carcinoma, pancreatic cancer, solid tumor and gastrointestinal cancer. All these examples mentioned above demonstrate that the clinical and translational market for oncolytic virus therapy is growing at an absurd pace.