The U.S. Food and Drug Administration today approved Ofev (nintedanib) capsules to slow the rate of decline in pulmonary function in adults with interstitial lung disease associated with systemic sclerosis or scleroderma, called SSc-ILD. It is the first FDA-approved treatment for this rare lung condition.
"Patients suffering from scleroderma need effective therapies, and the FDA supports the efforts of drug companies that are designing and conducting the clinical trials necessary to bring treatment options to scleroderma patients," said Nikolay Nikolov, M.D., associate director for Rheumatology of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA's Center for Drug Evaluation and Research. "Nintedanib is now a treatment option to slow the rate of decline in pulmonary function in patients who have interstitial lung disease from scleroderma."
Scleroderma is a rare disease that causes tissue throughout the body, including the lungs and other organs, to thicken and scar. Interstitial lung disease or ILD is a condition affecting the interstitium, which is part of the lung's structure, and is one of the most common disease manifestations of sclerderma. SSc-ILD is a progressive lung disease in which lung function declines over time, and it can be debilitating and life-threatening. ILD is the leading cause of death among people with scleroderma, typically resulting from a loss of pulmonary function that occurs when the lungs cannot supply enough oxygen to the heart. Approximately 100,000 individuals in the United States have scleroderma, and approximately half of scleroderma patients have SSc-ILD.
The effectiveness of Ofev to treat SSc-ILD was studied in a randomized, double-blind, placebo-controlled trial of 576 patients ages 20-79 with the disease. Patients received treatment for 52 weeks, with some patients treated up to 100 weeks. The primary test for efficacy measured the forced vital capacity, or FVC, which is a measure of lung function, defined as the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible. Those who took Ofev had less lung function decline compared to those on placebo.
The overall safety profile observed in the Ofev treatment group was consistent with the known safety profile of the therapy. The most frequent serious adverse event reported in patients treated with Ofev was pneumonia (2.8% Ofev vs. 0.3% placebo). Adverse reactions leading to permanent dose reductions were reported in 34% of Ofev-treated patients compared to 4% of placebo-treated patients. Diarrhea was the most frequent adverse reaction that led to permanent dose reduction in patients treated with Ofev.
The prescribing information for Ofev includes warnings for patients with moderate or severe hepatic (liver) impairment, those with elevated liver enzymes and drug-induced liver injury and patients with gastrointestinal disorders. Ofev may also cause embryo-fetal toxicity that can result in fetal harm, arterial thromboembolic events (blood clots), bleeding and gastrointestinal perforation. P-gp and CYP3A4 inhibitors may increase nintedanib exposure, and patients taking these inhibitors should be closely monitored for tolerability of Ofev. Common side effects noted with Ofev include diarrhea, nausea, abdominal pain, vomiting, liver enzyme elevation, decreased appetite, headache, weight loss and hypertension (high blood pressure).
Ofev was originally approved in 2014 for adult patients with idiopathic pulmonary fibrosis (IPF), which is another interstitial lung condition.
Ofev received Priority Review designation, under which the FDA's goal is to take action on an application within six months where the agency determines that the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing or preventing a serious condition. Ofev also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
The FDA granted the approval of Ofev to treat SSc-ILD to Boehringer Ingelheim Pharmaceuticals Inc.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
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Gotham Therapeutics Establishes Advanced Oncology Profiling Cascade with ProQinase to Progress its Portfolio of Epitranscriptomic Drug CandidatesWEDNESDAY, OCTOBER 30, 2019Gotham Therapeutics, a biotechnology company developing a novel drug class targeting RNA-modifying proteins, and ProQinase, an early stage drug-discovery company, recently acquired by Malvern/PA-based Reaction Biology Corp., today announced that they have established an array of advanced biochemical and cellular assays to characterize epitranscriptomic-directed compounds.“Establishing tailored target engagement, cell biology, phenotypic, and in-vivo assays to evaluate compounds originating from our three most advanced epitranscriptomic programs in parallel is a crucial next step to develop our broad pipeline towards the clinic,” said Dr. Gerhard Müller, Chief Scientific Officer of Gotham Therapeutics. “By pursuing the full range of the reader-writer-eraser continuum, we are able to focus on targets with the most compelling links to disease and advance those programs in tandem.”“Combining our high proficiency in providing customized solutions as early stage drug-discovery services, including tailor-made biochemical and cell-based assays, with Gotham’s expertise in epitranscriptomics, we were able to establish in a very short time a set of customized enzymatic and cellular assays for compounds addressing three different mRNA-modifying targets,” said Dr. Sebastian Dempe, Chief Executive Officer of ProQinase. “We look forward to supporting Gotham as the company advances its programs into lead optimization and to continued work with the Gotham team assisting the company to advance its broad pipeline in a time-effective manner.”In addition to establishing a tailor-made profiling cascade for compounds from its three most advanced programs, Gotham has also made progress in strengthening its drug discovery engine and developing its candidate base. Gotham previously completed the gene-to-lead phase for its drug discovery project targeting the METTL3/METTL14 complex and established a robust discovery process as a platform for future projects. The company has also generated a library of high-quality compounds tailor-made for accelerated hit generation and hit-to-lead expansion against large parts of the epitranscriptomic target space. This library includes a collection of over 2,000 analogues covering over 80 distinct chemotypes that will be used to further accelerate Gotham’s drug discovery efforts as it expands its pipeline.The original link:https://www.pharmafocusasia.com/news/gotham-therapeutics-establishes-advanced-oncology-profiling-cascade-with-proqinase-to-progress-its-portfolio-of-epitranscriptomic-drug-candidates2019 Asia-pacific pharma IP Leader Summit: http://en.zenseegroup.com/p/510934/will be held in Beijing on November 14-15, and will attract more than 500 industry experts from domestic and foreign pharmaceutical companies, biotechnology companies, governments, associations, law firms, intellectual property agents and other companies to attend.Official registration and consultation channels:Contact：AnnPhone: 021-65650305Email：Marketing@zenseegroup.comhttp://en.zenseegroup.com/p/510934
Gilead and Glympse Bio Announce Strategic Collaboration for Use of Biomarker Technology in NASH Clinical DevelopmentTUESDAY, OCTOBER 29, 2019Gilead Sciences Inc. and Glympse Bio, Inc., today announced that the companies have entered into a strategic collaboration in nonalcoholic steatohepatitis (NASH) clinical development. Glympse Bio’s proprietary synthetic biomarkers – bioengineered to identify stage and progression of disease as well as early detection of treatment response – will be used to determine clinical trial participants’ stage of disease at initial screening and to determine responses to study treatment in Gilead’s NASH clinical program.“We are excited about the opportunity to partner with Glympse Bio to help inform our NASH development program,” said Mani Subramanian, MD, PhD Senior Vice President, Liver Diseases, Gilead Sciences. “By utilizing this innovative technology, we hope to better characterize this complex disease and improve our understanding of how our compounds impact disease progression.”Glympse Bio’s proprietary technology, Glympse Inside™, combines synthetic biomarkers with machine learning approaches to identify the stage and monitor progression of important, complex diseases such as cancer, fibrosis, inflammation, and infections, in real time.“We are very excited about partnering with Gilead, a leader in drug development, to help drive earlier and more favorable outcomes for patients,” said Caroline J Loew, President and CEO, Glympse Bio. “Gilead’s commitment to developing innovative medicines in areas of high unmet medical need aligns with our mission of transforming disease detection and measuring treatment response, all with the goal of helping improve the lives of patients.”information source:pharma focus AsiaThe original link:https:https:https://www.pharmafocusasia.com/news/gilead-and-glympse-bio-announce-strategic-collaboration-for-use-of-biomarker-technology-in-nash-clinical-development2019 Asia-pacific pharma IP Leader Summit:http://en.zenseegroup.com/p/510934/ will be held in Beijing on November 14-15, and will attract more than 500 industry experts from domestic and foreign pharmaceutical companies, biotechnology companies, governments, associations, law firms, intellectual property agents and other companies to attend.Official registration and consultation channels:Contact：AnnPhone: 021-65650305Email：Marketing@zenseegroup.comhttp://en.zenseegroup.com/p/510934
HistoIndex Announces Global Partnerships To Expand AI-Based Digital Pathology PlatformMONDAY, OCTOBER 28, 2019HistoIndex announces the expansion of its AI-based digital pathology platform towards large-scale NASH preclinical programs for pharmaceutical and biotech companies to achieve quantifiable and reproducible data on therapeutic responses in NASH animal models, and to aid CROs as well as research agencies in validating new models mimicking the NASH disease.Published preclinical studies have demonstrated the use of HistoIndex's fully quantifiable Second Harmonic Generation (SHG) technology as a highly accurate stain-free method that can monitor the efficacy of various therapeutic agents, by quantifying more than 450 NASH-associated parameters in fibrosis, inflammation, ballooning and steatosis. The information of these NASH-associated parameters gathered from the entire liver tissue, provides comprehensive insights on the mechanism of action of the therapeutic agent. As a drug discovery tool, HistoIndex's SHG-enabled digital pathology platform will allow pharmaceutical and biotech companies to select promising lead candidates for further optimization and make informed decisions in the management of their NASH drug development pipeline.In addition to ongoing NASH clinical trials, HistoIndex is currently involved in multiple preclinical studies, most of which are conducted by major pharmaceutical and biotech companies, medical universities, CROs and research agencies. Notably, HistoIndex has recently entered into a collaboration with the A*STAR's Genome Institute of Singapore (GIS) to validate their in vivo and in vitro NASH models based on Asian-centric clinical data. Says Professor Patrick Tan, Executive Director of the Genome Institute of Singapore (GIS), "At GIS, we conduct our preclinical studies on fatty liver involving data from animal models based on patient-derived transcriptomic data. This has a high translational potential as it helps us to pinpoint new therapeutic targets and their validation. Therefore, using a fully quantitative, reliable and objective pathological evaluation such as HistoIndex's AI-based digital pathology platform is essential to the success of our efforts in therapeutic target discovery."HistoIndex is also a partner with globally-renowned CRO, WuXi Apptec, in advancing their NASH preclinical programs with advanced R&D and smart imaging analysis capabilities. Published study data will be available for discussions during networking opportunities throughout AASLD's The Liver Meeting® in November 2019. "We are very excited to extend our AI-based SHG platform to preclinical studies to help companies with drug discovery programs select promising lead candidates for further optimization and subsequent clinical development," says Dr Poon Thong Yuen, Chief Executive Officer of HistoIndex. "Our SHG image analysis platform has already been used to analyze the efficacy of a series of promising drug candidates for NASH within various animal models commonly-used by the industry, and we believe these preclinical partnerships will help drive the adoption of our platform significantly."information source:pharma focus AsiaThe original link:https:https://www.pharmafocusasia.com/news/histoindex-announces-global-partnerships-to-expand-ai-based-digital-pathology-platform2019 Asia-pacific pharma IP Leader Summit: http://en.zenseegroup.com/p/510934/will be held in Beijing on November 14-15, and will attract more than 500 industry experts from domestic and foreign pharmaceutical companies, biotechnology companies, governments, associations, law firms, intellectual property agents and other companies to attend.Official registration and consultation channels:Contact：AnnPhone: 021-65650305Email：Marketing@zenseegroup.comhttp://en.zenseegroup.com/p/510934
AVROBIO Receives Orphan-Drug Designation from the U.S. FDA for AVR‑RD‑02 for the Treatment of Gaucher DiseaseFRIDAY, OCTOBER 25, 2019AVROBIO, Inc., today announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for the Company’s investigational gene therapy, AVR-RD-02, for the treatment of Gaucher disease. AVR-RD-02 consists of the patient’s own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease. The Company is actively recruiting in Canada for its Phase 1/2 clinical trial of AVR-RD-02, which seeks to evaluate the safety and efficacy of the therapy in patients with Type 1 Gaucher disease.“Under the existing standard of care, patients with Gaucher disease are bound to a lifelong infusion schedule of enzyme replacement therapies, and still experience painful and progressive symptoms such as debilitating musculoskeletal pain and fatigue,” said Birgitte Volck, MD, PhD, President of Research and Development at AVROBIO. “Orphan-drug designation recognizes the unmet need of populations with rare diseases like Gaucher where AVROBIO strives to transform lives by addressing the underlying cause of the disease with a single dose of gene therapy.”Orphan-drug designation is granted by the FDA to drugs and biologics which are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan-drug designation provides certain incentives, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers.information source:pharma focus AsiaThe original link:https:https://www.pharmafocusasia.com/news/avrobio-receives-orphan-drug-designation-from-the-us-fda-for-avrrd02-for-the-treatment-of-gaucher-disease2019 Asia-pacific pharma IP Leader Summit: http://en.zenseegroup.com/p/510934/will be held in Beijing on November 14-15, and will attract more than 500 industry experts from domestic and foreign pharmaceutical companies, biotechnology companies, governments, associations, law firms, intellectual property agents and other companies to attend.Official registration and consultation channels:Contact：AnnPhone: 021-65650305Email：Marketing@zenseegroup.comhttp://en.zenseegroup.com/p/510934
FRIDAY, OCTOBER 25, 2019AZTherapies, Inc., a biopharmaceutical company developing therapeutics to extend brain health, today announced the acquisition of Smith Therapeutics, a private biopharmaceutical company with a shared goal of targeting neuroinflammation to treat neurodegenerative disease. Smith Therapeutics’ Founder and Chief Executive Officer Philip Ashton-Rickardt, Ph.D., has joined the senior leadership team at AZTherapies as Senior Vice President of Immunology. Financial terms of the acquisition were not disclosed.Smith Therapeutics’ proprietary research platform focuses on the use of modified T cells to restore a healthy balance of inflammatory and regulatory cells in the brain. To date, Smith has successfully engineered immunosuppressive T regulatory (Treg) cells with Chimeric Antigen Receptors (CARs) targeting brain glial cells. Previous research has demonstrated the ability of Tregs to dampen microglial activity and reduce neuroinflammation in models of neurodegeneration, suggesting their potential utility in the treatment of diseases including Progressive Supranuclear Palsy (PSP), Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, Amyotrophic Lateral Sclerosis (ALS), and others.“This acquisition represents a meaningful step forward for us as we continue to strengthen our leadership position in the development of therapies targeting neuroinflammation to stop or slow the progression of neurodegenerative diseases,” said David R. Elmaleh, Ph.D., Founder, CEO, and Chairman of AZTherapies. “We are excited to be working with Philip as we add this cutting-edge technology to our portfolio of innovative programs. An esteemed immunologist and inventor of the technology, Philip brings unparalleled expertise to the company and we look forward to advancing this CAR-Treg program further into IND-enabling studies and into clinical development as rapidly as possible.”Dr. Ashton-Rickardt commented on the acquisition and his appointment: “Our shared rationale of targeting neuroinflammation as the root cause of neurodegenerative disease makes this acquisition a great strategic fit for us. With AZTherapies’ expertise in drug development and clinical trial execution, we believe that together, we are well positioned to advance our CAR-Treg technology and fundamentally change neurodegenerative disease progression.”Prior to launching Smith Therapeutics in 2017, Dr. Ashton-Rickardt was Chair in Immunology at Imperial College London, Visiting Professor, Brigham and Women’s Hospital, Harvard Medical School, and Associate Professor in the Department of Pathology at the University of Chicago. His work has been recognized by his peers through the award of tenure from The University of Chicago and by his fellow citizens as a recipient of the Early Career Award for Scientists and Engineers from President Bill Clinton. He has published more than 65 peer-reviewed papers in more than 30 academic journals (including Cell, Science, Immunity, and Nature Immunology), has served as an editor for several academic journals, and has been a member of grant review boards globally. Dr. Ashton-Rickardt received a B.Sc. in Biochemistry from the University of London, King’s College with honors, a Ph.D. in Molecular Biology from the University of Edinburgh, and completed post-doctoral work at the University of Edinburgh and the Massachusetts Institute of Technology in Molecular Biology and Molecular Immunology, respectively.information source:pharma focus AsiaThe original link:https:https://www.pharmafocusasia.com/news/aztherapies-strengthens-neuroinflammation-targeted-pipeline-through-acquisition-of-smith-therapeutics2019 Asia-pacific pharma IP Leader Summit: http://en.zenseegroup.com/p/510934/will be held in Beijing on November 14-15, and will attract more than 500 industry experts from domestic and foreign pharmaceutical companies, biotechnology companies, governments, associations, law firms, intellectual property agents and other companies to attend.Official registration and consultation channels:Contact：AnnPhone: 021-65650305Email：Marketing@zenseegroup.comhttp://en.zenseegroup.com/p/510934