10 potential heavy-duty therapies are fresh out of which patients will they benefit?

Photo Source: Reference s2,VX-445, Tezacaftor and IvacaftorIndications: Cystic Fibrosis: Vertex PharmaceuticalsVertex has selected the company's in-research therapy VX-445, with approved therapies Tezacaftor and Ivacaftor forming a 3-in-association therapy for the treatment of cystic fibrosisCystic fibrosis is a genetic disease caused by a mutation in the genes of the coding cystic fibrosis trans membrane conductive regulator (cystic fibrosis trans conduction regulator, CFTR), resulting in a loss of CFTR protein or functional defectsVX-445 is a new generation of CFTR protein rectors that restore the function of CFTR proteins that carry F508del mutations, thereby improving respiratory function in CF patientsIt has reached the end of Phase 3 clinical trials with a combination of combination therapies, Tezacaftor and IvacaftorThis combination therapy may treat 90% of patients with cystic fibrosisThe company plans to file a new drug application (NDA) with the U.SFDA in the third quarter of this yearUpadacitinibIndications: Rheumatoid arthritis: AbbVieAbbVie developed upadacitinib as a second-generation JAK inhibitor that is highly selective for JAK1 subtypesJAK kinase family inhibitors have shown excellent efficacy in the treatment of inflammatory and autoimmune diseases such as rheumatoid arthritis and Crohn's disease's application for a new drug for upadacitinib to treat rheumatoid arthritis was approved by the FDA in February Recently released clinical trial results show that the treatment can relieve patients' symptoms in the long term and be more effective than existing common therapies Trastuzumab deruxtecan (DS-8201) indications: HER2-plus metastatic breast cancer, gastric cancer : Daiichi Sankyo, AstraZeneca trastuzumab dercante (DS-8201) is an innovative target for HER2 It uses the first three-phase development of innovative antibody coupling technology, so that targeted HER2 antibodies can connect more cytotoxins, thereby enhancing the effectiveness of anti-tumor This ADC achieved a 93.7 percent disease control rate in clinical trials treating PATIENTs with HER2-plus metastatic breast cancer first three plans to submit regulatory applications for advanced or metastatic breast cancer later this year, while the two companies are conducting other clinical trials to explore the aDC's efficacy in treating other cancers that express HER2 Liso-Cel (full name Lisocabagene maraleucel, also known as JCAR017) indications: Recurrent incurable (R/R) chronic lymphatic leukemia (CLL), small lymphocytic lymphocytic lymphocytic (SLL) company: Celgene liso-cel, developed by Junos, a new-based company, is a target TOCD19CAR-T therapy At the just-concluded annual meeting of the American Society of Clinical Oncology (ASCO), New Base released the latest results of a Phase 1/2 clinical trial of the therapy, which achieved 82% ORR and 46% full remission (CR) in R/R patients who had received a variety of pre-treatments, including ibrutinib notably, 75 percent of the 20 patients who received the micro-residual lesions (MRD) test had negative blood MRD levels and 65 percent of patients with negative bone marrow MRD levels Zolgensma (onasemnogene abeparvovac-xioi) indications: Spinal Muscular Dystrophy (SMA) : Novartis This innovative gene therapy has recently been approved by the FDA to treat Type 1 SMA patients It is the first gene therapy approved by the FDA to treat SMA the gene therapy, originally developed by AveXis, introduces SMN1 gms that express normal motor neuron survival protein (SMN) into the patient through the AAV9 virus vector, allowing the patient's cells to express the SMN protein for a long time to achieve a "cure" effect it's worth noting that clinical trials have shown that this gene therapy not only saves the lives of patients with the most severe type 1 SMA, but also improves the motor ability of patients with milder type 2 and other types of SMA Tirzepatide (LY3298176) Indications: Type 2 Diabetes, Non-Alcoholic Fatty Hepatitis (NASH) Company: Eli Lilly and Company Lilly developed tirepatide (LY3298716) is a peptide drug with gastric inhibition peptide (GIP) receptors and glucagon-like-1 (GLP-1) receptor The new drug, which is being developed, improves pancreatic beta cell function and increases insulin sensitivity in patients clinical trial data presented at the 79th American Diabetes Association (ADA) Conference showed that tirzepatide can lead to significant reductions in patients' glycated hemoglobin (A1C) levels and weight Furthermore, tirzepatide can also improve nash-related biomarker levels in the treatment of people with type 2 diabetes Picture Source: Resources Sacituzumab Govitecan Indications: Solid Tumor Company: Immunomedics Sacituzumab Govitecan is an innovative ADC It connects monoclonal antibodies that target TROP-2 antigens with cytotoxic drugs called SN-38 TROP-2 is a receptor that is overexpressed on the surface of many human tumor cells and has limited levels of expression in normal tissues SN-38 is an active metabolite of an anti-cancer chemotherapy drug The treatment has been approved by the FDA for breakthrough treatment for patients with three-negative breast cancer (TNBC) At the same time, the FDA granted it fast-track eligibility to treat patients with TNBC, NSCLC and small cell lung cancer (SCLC) Ozanimod Indications: Multiple Sclerosis (MS) Research and Development: Celgene Ozanimod is a new oral selective pyridoxol 1-phosphate (S1P) receptor regulator with selective high affinity with s1P1 and S1P5 receptor subtypes In lymphatic tissue, ozanimod promotes the presence of lymphocytes, thereby reducing the number of lymphocytes to the central nervous system, which in this way relieves symptoms of multiple sclerosis in two Phase 3 clinical trials, ozanimod was able to reduce the annual recurrence rate in MS patients The new drug's application has been accepted by the FDA and the EUROPEAN Union EMA and is expected to be responded to in the first half of 2020 Brolucizumab Indications: Wet Age-Associated Macular Degeneration (Wet AMD) : Novartis Brolucizumab is a humanized single-stranded antibody variable region fragment that binds to all VEGF-A proteins with high affinity to inhibit their function and activation of VEGF receptors Wet AMD patients suffer damage to the retinal structure due to abnormal vascular hyperplasia under the macular plaque and the oozing of fluid from these blood vessels By inhibiting the VEGF signaling pathway, brolucizumab can inhibit the growth of new vascular lesions, remove retinal water, and improve the patient's vision in two Phase 3 clinical trials, brolucizumab was less effective in improving patients' vision than standard therapies, and better than standard therapies in improving retinal water Novartis submitted a new drug application to the FDA in April using priority review vouchers, and if approved, it is expected to launch the innovative therapy by the end of the year Voxelotor Indications: Sickle Cell Anemia (SCD) : Global Therapeutic Bloods (GBT) Voxelotor is an oral small molecule drug It can help hemoglobin to better bind to oxygen, inhibit the polysaccosis of sickle hemoglobin, thus preventing the occurrence of the process of sickle deformation of blood cells, and thus improve hemolytic anemia This innovative therapy has been approved by the FDA as a breakthrough therapy and approved by the FDA to apply for new drugs using the Accelerated Approval Channel The results of Phase 3 clinical trials recently published show that voxelotor can significantly improve hemoglobin levels in SCD patients GBT plans to complete voxelotor's rolling new drug application later this year References: 1 , Innovation and Unmet Need Drive Prescription Drug Sales to $1.18tr by 2024: New Report From Evaluate Ltd Retrieved June 17, 2019, from , EvaluatePharma World Preview 2019, Outlook to 2024 Retrieved June 17, 2019, from Hoyd June 18, 2019, from 4, Celgene Corporation Announces Initial Phase 1/2 Liso-cel Data in Patients with Relapsed/Refractory CLL, Leing These High with-Risk Disease, Treatlyed with Ibrutinib, at ASH 2018 Hoyd June 18, 2019, from 5 Retrieved June 18, 2019, from Retrieved June 18, 2019, from