20 innovative therapies worth paying attention to in 2020 (part 1)

There is still one week to go 2020 is coming In today's article, the content team of Wuxi apptec will share with readers 20 innovative therapies worthy of attention in 2020 based on public materials Today's article will introduce 10 potential heavy therapies that are expected to be approved by FDA in 2020 Incisiran: the first RNAi treatment company for mass diseases: Novartis / the medicines company indication: reduce LDL-C, developed jointly by the medicines company and Alnylam, the RNAi treatment incisiran is expected to become the first RNAi treatment for mass diseases In clisiran reduces PCSK9 protein expression by targeting PCSK9 protein mRNA Compared with PCSK9 inhibitors, it has the advantage that patients need only two injections a year to help control LDL-C levels Even with the best statins, there are still many patients with LDL-C levels that are not within the recommended range Inclsiran is expected to provide a simple and innovative treatment option for these patients Novartis' US $9.7 billion acquisition of the medicines company also demonstrates its recognition of the potential of the therapy as a blockbuster drug Bempedoic acid: the "first in class" treatment company to reduce bad cholesterol: indication of esperion: low density lipoprotein cholesterol level is too high The bempedoic acid developed by esperion is an oral ATP citrate lyase (ACL) inhibitor It can reduce the biosynthesis of cholesterol and increase the level of LDL-C receptor, which leads to more LDL-C being removed from blood circulation and lower the level of LDL-C in blood It can be used as a single drug therapy or in combination with Pfizer's zeetimibe to help those patients who have not reached the LDL-C level after receiving the best statins The new drug application for the innovative treatment is expected to receive a response from the FDA early next year ▲ mechanism of action of bempedoic acid / ezetimibe (picture source: esperion company website) risdiplam: oral therapy company to change the course of SMA disease: Roche indication: spinal muscular atrophy (SMA) This year is a breakthrough year for SMA patients The introduction of zolgensma gene therapy from Novartis provides them with a potential cure Although the long-term efficacy of zolgensma still needs to be further observed and evaluated, this treatment allows SMA patients to see the hope of "one treatment, lifelong benefit" Next year, SMA patients are expected to receive an oral SMA therapy Risdiplam developed by Roche company improves the level of SMN protein in patients by influencing the mRNA splicing process of SMN2 gene encoding SMN protein It can not only save the life of children with type 1 SMA who have the most severe symptoms, but also improve their motor ability when they are treated with type 2 and type 3 SMA who have less symptoms Although oral therapy does not have the same "once and for all" potential as gene therapy, it is a more familiar dosage form for doctors and patients, and may have more extensive accessibility Risdiplam's new drug application has obtained the priority review qualification granted by the US FDA Valoctocogene roxaparvovec: the first gene therapy company to treat hemophilia A A: biomarin indications: severe adult hemophilia A for severe hemophilia A, even if the treatment of conventional coagulation factor VIII (factor VIII) 2-3 times a week, still can not completely prevent the occurrence of bleeding events Valoctocogene roxaparvovec uses AAV5 virus vector to deliver the transgenic expression factor VIII to patients The results of clinical trials showed that the average annual bleeding rate and factor VIII use rate of severe hemophilia patients decreased by 96% in the three years after one treatment Biomarin has filed an application for listing in the European Union, and yesterday submitted an application for approval (BLA) for the gene therapy to the US FDA Obecholate: the first innovative treatment company for nonalcoholic steatohepatitis: intercept indications: nonalcoholic steatohepatitis (NASH) 2019 is a wake-up year for NASH treatment Many single or combination therapies for NASH have been frustrated in clinical trials, which reminds us that Nash is still a very complex disease Among the setbacks, the success of ocept's obacholic acid (OCA) in phase 3 clinical trials is particularly prominent The new drug application of the fxragonist has obtained the FDA's priority review qualification and is expected to become the first approved treatment for NASH next year ▲ OCA produces efficacy by activating FXR (photo source: reference [3]) sacituzumab govitecan: rejuvenation of antibody coupled drugs: immunomedics indications: a major breakthrough in the field of antibody coupled drugs (ADC) for triple negative breast cancer this year Three new drugs have been approved by FDA, including polatuzumab of Roche's Genentech company Vedotin piiq), padcev (enfortumab vedotin ejfv) jointly developed by Seattle Genics and Asteris, and enhertu (FAM trastuzumab deruxtecan nxki) jointly developed by AstraZeneca, the first three republics Sacituzumab govitecan, developed by immunomedics, is an ADC that connects the antibody targeted at Trop-2 with SN-38, the active metabolite of irinotecan Trop-2 is an over expressed cell surface receptor in a variety of human tumors In April this year, Everest medicine, invested and established by Kangqiao capital, entered into a cooperation with immunomedics to obtain the exclusive R & D rights of this ADC in Greater China, South Korea and some Southeast Asian countries and regions, with a total cooperation amount of up to $835 million Immunomedics has submitted sacituzumab govitecan's BLA to FDA for three line treatment of three negative breast cancer patients Filgotinib: looking for a safe and effective JAK inhibitor company: Gilead scientific indications: JAK signaling pathway plays an important role in cancer, autoimmune diseases and inflammatory diseases, and also provides multiple important targets for the development of new drugs This year, two second-generation JAK inhibitors, rinvoq (uPA acitinib), from abbvie, and inrebic (fedratinib), from celgene, were approved Because JAK / STAT signaling pathway mediates the signal transmission of many cytokines, toxic and side effects are concerned by the industry, especially in the treatment of non fatal inflammatory diseases (such as rheumatoid arthritis, psoriasis), with good safety and tolerance is more prominent Potential thrombotic risk leading to patient death is an important reason to limit the application of JAK inhibitors that have been approved ▲ the JAK signaling pathway and the in-process therapy targeting this pathway (photo source: Nature Reviews Drug Discovery) the Jak1 inhibitor filgotinib jointly developed by Gilead science and Galapagos did not show a clear correlation with the risk of thrombus or the elevation of platelet level in safety clinical trials The company has recently submitted a new drug application for the innovative treatment to the FDA FDA's evaluation of the safety of this Jak1 inhibitor will be the focus of the industry Roxadustat: anaemia therapy company targeting norwin signaling pathway: indication of fabergen: renal anemia developed by fibrogen, Astellas and AstraZeneca is an oral small molecule hypoxia inducible factor prolinyl hydroxylase inhibitor (hif-phi) for the treatment of renal anemia The physiological function of HIF can not only improve the expression level of erythropoietin (EPO), but also promote the expression of erythropoietin receptor, iron absorption and circulation protein Its targeted "oxygen sensing pathway" just won the Nobel Prize in physiology or medicine in October this year This innovative therapy has been approved in China for the treatment of dialysis dependent and non dependent renal anemia patients Fabergin has recently submitted a new drug application to the US FDA Ozanimod: multiple sclerosis treatment company with twists and turns: Bristol Myers Squibb / new base indication: multiple sclerosis ozanimod is an innovative treatment for multiple sclerosis by Bristol Myers Squibb It is an oral specific sphingosine-1-phosphate (S1P) receptor modulator with selective high affinity for S1P1 and S1P5 receptor subtypes In the lymphoid tissue, ozanimod can promote the resident of lymphocytes, thus reducing the number of lymphocytes to the central nervous system, which can alleviate the symptoms of multiple sclerosis The NDA for the innovative treatment developed by celgene was rejected by the FDA, but in June, both the FDA and the EU EMA accepted its listing application This kind of drug with twists and turns in the R & D journey is only one last step away from benefiting patients Aducanumab: the new Alzheimer's disease drug company: Biogen indications: the application of aducanumab, a β - amyloid antibody developed by the early Alzheimer's disease company and Eisai company in 2020, and its review process will become the focus of the industry Not only because no new drug has been approved in the field of Alzheimer's disease (AD) since 2003, aducanumab may become the first innovative therapy to change the process of ad disease; moreover, the review process and results of this new drug will become one of the wind vane to evaluate FDA's attitude towards the review of innovative therapy after the new director, Dr Stephen Hahn In the past two years, the FDA, led by former director Dr Scott Gottlieb, has significantly accelerated the review of innovative therapies In 2019, the approval of multiple innovative therapies is 3-5 months ahead of schedule However, whether FDA's evaluation of the effectiveness of innovative therapies is rigorous has become a controversial topic in the industry Two key phase 3 trials of aducanumab did not give consistent results How to balance the urgent needs of patients and maintain strict standards to evaluate the effectiveness of treatment will be a problem for FDA Whether the treatment will be approved or not may face great uncertainty In addition to the above innovative therapies, there will be a number of car-t therapies that are expected to be approved in 2020, including LISO cel targeting CD19 antigen, IDE cel targeting BCMA antigen, and kte-x19 targeting CD19 antigen by Gilead science Tomorrow, we will take stock of the ongoing clinical therapies that may not be approved by FDA in the next year Please pay attention to them Reference: [1] Schwartz et al., 2017) JAK occupation a s a thermal strategy for immune and informational diseases Nature reviews drug discovery, https://doi.org/10.1038/nrd.2017.201 [2] vantage 2020 preview – what's in store for the biopharma sector? 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