Rheumatoid arthritis drug diminishes Zika birth defects in mice

medicalxpress | May 06, 2019

In experiments with pregnant mice infected with the Zika virus, Johns Hopkins Medicine researchers report they have successfully used a long-standing immunosuppressive drug to diminish the rate of fetal deaths and birth defects in the mice's offspring. The U.S. Food and Drug Administration-approved medicine, anakinra, once commonly used to treat rheumatoid arthritis and other autoimmune diseases in newborns and adults, has largely been replaced by more effective drugs. However, in the Zika-infected mouse experiments, the drug appears to interfere with inflammation in the pregnant animals' placentas, the researchers say. There also is evidence the drug directly reduces inflammation in fetal brains. A report on the findings was published in the April issue of the Journal of Clinical Investigation Insights.

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BUSINESS INSIGHTS

Qualigen Therapeutics Acquires Majority Stake in Infectious Disease Diagnostics Technology Company NanoSynex

Qualigen Therapeutics, Inc. | June 03, 2022

Qualigen Therapeutics, Inc. a diversified life sciences company focused on developing treatments for adult and pediatric cancers with potential for Orphan Drug Designation, while also commercializing diagnostics, today announces it has completed the acquisition of a majority stake in NanoSynex Ltd., an Israeli-based developer of next generation diagnostics technology. NanoSynex’s award winning technology is an Antimicrobial Susceptibility Testing (AST) platform that aims to provide clinical laboratories worldwide with a rapid, accurate and personalized test for bacterial infections, especially difficult-to-treat drug resistant strains, with the goal of quickly matching the correct antibiotics at the correct concentration to treat a patient’s specific infection. Antibiotic misuse and overuse have given significant rise to more frequent and more deadly antibiotic resistant bacteria, such as Methicillin-resistant Staphylococcus aureus (MRSA). Multi-drug resistant pathogens such as these have been declared a substantial threat to U.S. public health and national security by the Institute of Medicine and a federal Interagency Task Force on Antimicrobial Resistance. According to some estimates, the global infectious disease diagnostics market is expected to reach ~$39.8 Billion by 2026 from ~$28.1 Billion in 2021. “Living through two and a half years of COVID-19 has taught us the critical role diagnostics plays in combating infectious disease, and the need to be prepared for the next wave. NanoSynex’s technology shows great promise in rapidly and accurately matching antibiotics to the target bacteria, and in doing so, quickly equipping healthcare providers with the data needed to treat their patients with the right antibiotic at the right dose at the right time. We strongly believe in the potential of this platform, and our ability to leverage our long-standing diagnostics development, regulatory and commercial expertise to help bring the NanoSynex diagnostics technology to market sooner.” Michael Poirier, Qualigen's Chairman and CEO “We are excited about beginning a new journey with Qualigen and joining forces in a common mission to develop innovative diagnostics. This partnership takes the NanoSynex AST platform to the next level," added NanoSynex Co-Founder and Chief Executive Officer, Diane Abensur Bessin. NanoSynex is led by a dynamic team of healthcare subject matter experts, microbiologists, and engineers, including its co-founders – Diane Abensur Bessin and Michelle Heymann, who were selected for Forbes’ 2020 Tech 30 Under 30 list. Qualigen’s diagnostics team, led by Shishir Sinha, Senior VP and Chief Operating Officer, will work closely alongside the Israeli-based NanoSynex team on technology development, regulatory path, and commercial preparedness. Qualigen also envisions potential synergies with its proprietary FastPack® diagnostics platform to further strengthen the Company’s diagnostics business which has seen a post-COVID-19 resurgence. Qualigen’s purchase of the controlling interest in NanoSynex was primarily accomplished via a stock-for-stock acquisition with a controlling shareholder of NanoSynex. Qualigen also provided an investment in NanoSynex at closing and will provide future milestone-based funding leading to the commercialization of this technology. NanoSynex’s Board will initially be comprised of two current NanoSynex directors – NanoSynex co-founders - and two Qualigen appointees – Mr. Poirier and Mr. Sinha. About Qualigen Therapeutics, Inc. Qualigen Therapeutics, Inc. is a diversified life sciences company focused on developing treatments for adult and pediatric cancer, as well as maintaining and expanding its core FDA-cleared FastPack® System, which has been used successfully in diagnostics for over 20 years. Our investigational QN-302 compound is a small molecule selective transcription inhibitor with strong binding affinity to G4s prevalent in cancer cells; such binding could, by stabilizing the G4s against “unwinding,” help inhibit cancer cell proliferation. Our investigational QN-247 compound inhibits nucleolin, a key multi-functional regulatory protein that is overexpressed in cancer cells; QN-247 may thereby be able to inhibit the cells’ proliferation. QN-247 has shown promise in preclinical studies for the treatment of acute myeloid leukemia (AML). The investigational compounds within Qualigen’s RAS-F family of RAS oncogene protein-protein interaction inhibitor small molecules are believed to inhibit or block the binding of mutated RAS genes’ proteins to their effector proteins, thereby leaving the proteins from the mutated RAS unable to cause further harm. In theory, such mechanism of action may be effective in the treatment of about one quarter of all cancers, including certain forms of pancreatic, colorectal, and lung cancers. In addition to its oncology drug pipeline, Qualigen has an established diagnostics business which manufactures and distributes proprietary and highly accurate rapid blood testing systems to physician offices and small hospitals for the management of prostate cancer and other diseases and health conditions. About NanoSynex NanoSynex is a MedTech company that aims at providing new solutions to improve testing quality, patient outcomes, and reduce healthcare costs by speeding up diagnostic processes. NanoSynex is focused on the development and commercialization of a rapid innovative Antimicrobial Susceptibility Test (AST). The technology is based on a purely phenotypic approach and uses a microfluidic disposable test card platform and method that optimizes bacterial growth. This disruptive development was born from exciting research discoveries at the lab of Professor Shulamit Levenberg, former Dean of the Technion Institute of Technology – Biomedical Engineering Faculty.

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SoniVie receives IDE approval from FDA for its Pilot study to treat Hypertension with its Renal Artery Denervation TIVUS™ technology

SoniVie | June 20, 2022

SoniVie, an Israeli company developing a novel proprietary Therapeutic Intra-Vascular Ultrasound System to treat a variety of hypertensive disorders, announced that on May 5th 2022 the U.S. Food and Drug Administration granted IDE approval for its "REDUCED1" Pilot study to treat Resistant Hypertension Patients with Renal Artery Denervation using TIVUS™, its innovative Ultra-Sound Ablation System. Resistant hypertension is defined as blood pressure higher than 140/90 mmHg despite use of three antihypertensive medications of different classes at the best tolerated doses, one of which must be a diuretic. Millions of people world-wide suffer from resistant hypertension which substantially increases the risk of heart attack, stroke and kidney failure. "We are very pleased that FDA has approved the REDUCED1 study. Sites initiation has started, and many clinical teams have responded very favourably about participating to the study. There is a significant number of patients that may benefit from our technology and we are genuinely happy for this important step towards the introduction of TIVUS™ in the US. There is a lack of effective therapeutic solutions for patients suffering from resistant hypertension, and physicians are looking forward to a safe, effective and easy to use device treatment," Christian Spaulding, CMO, SoniVie Ltd "This is a significant US Regulatory milestone for SoniVie, starting the feasibility study using the Ultra-Sound ablation platform in the US for the Renal Denervation indication. This is a major step and priority in the company's history," says Tomaso Zambelli, CEO, SoniVie LTD. The REDUCED1 study will further expand the Company's clinical experience based on two clinical trials in Renal Denervation performed using the earlier generation of the TIVUS™. Renal Denervation with TIVUS™ is a minimally invasive procedure that uses high-frequency non-focused Ultra-Sound energy to ablate nerves in the renal artery. This causes a reduction in the nerve activity, which may decrease blood pressure. This procedure is designed for patients who suffer from resistant hypertension. About SoniVie SoniVie is a medical device company developing the TIVUS™, the only Ultra-Sound Denervation platform with active development programs in three therapeutic areas: pulmonary artery denervation for pulmonary hypertension, renal artery denervation for resistant hypertension, and lung denervation for chronic obstructive pulmonary disease with chronic bronchitis. These diseases affect millions of patients in the United States and Europe.

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BUSINESS INSIGHTS

Innocoll Announces Publication of Positive Results from Pivotal Clinical Trial of POSIMIR in Arthroscopic Subacromial Decompression

Innocoll Pharmaceuticals | July 07, 2022

Innocoll Pharmaceuticals Limited, a commercial-stage biotechnology company and portfolio business of Gurnet Point Capital, announced the publication of the results of Study BU-002-IM evaluating the safety and efficacy of POSIMIR® for infiltration use in 107 patients undergoing arthroscopic subacromial decompression. The peer-reviewed paper, entitled “SABER-Bupivacaine Reduces Postoperative Pain and Opioid Consumption After Arthroscopic Subacromial Decompression: A Randomized, Placebo-Controlled Trial,” was published online on May 17, 2022 in the Journal of the American Academy of Orthopedic Surgeons Global Research & Reviews. According to the report, POSIMIR 5 mL administered into the subacromial space at the end of arthroscopic subacromial decompression surgery in this double-blind, randomized, controlled, multicenter study, reduced mean postoperative pain on movement over 72 hours by 1.3 points on a 0-10 scale compared with vehicle control, a relative reduction of 20%. Simultaneous improvements in postoperative opioid use during the same 72-hour period provided evidence that the observed pain reduction was clinically meaningful. The median time to first request for opioid rescue medication was 12.4 hours among patients treated with POSIMIR compared with 1.2 hours in patients who received vehicle control. The median total opioid consumption in intravenous (IV) morphine milligram equivalents over 72 hours was 4.0 mg in the POSIMIR group vs 12.0 mg in the vehicle control group. The proportion of patients who were opioid free at 72 hours was 40% in the POSIMIR group vs 16% in the vehicle control group. As reported in the paper, the most common treatment-emergent adverse events were headache, nausea, musculoskeletal pain, cardiac disorders, skin and subcutaneous tissue disorders, injury and procedural complications, general disorders and administration site conditions and respiratory, thoracic, and mediastinal disorders. Of 2 serious adverse events reported among patients in these treatment groups, only 1 occurred during the immediate 2-week postsurgical follow-up period, and none was considered related to treatment. There were no TEAEs leading to study discontinuation. “These very compelling results, demonstrating up to three days of local analgesia after a single intra-operative injection, make POSIMIR an important new tool in treating postoperative pain from arthroscopic subacromial decompression,” said Sten Rasmussen, MD PhD, Professor and Head of Department of Clinical Medicine, Aalborg University, Denmark, the senior author and a principal investigator in the POSIMIR study. “We are excited to publish the positive results of this pivotal clinical trial of POSIMIR, the second approved product in our portfolio of nonopioid, extended-duration, locally-acting analgesics for postsurgical pain control. As the only company with two non-opioid extended release bupivacaine products on the market, we look forward to bringing POSIMIR to the orthopedic community,” Louis Pascarella, Innocoll President and Chief Executive Officer About POSIMIR POSIMIR for infiltration use contains more bupivacaine in a single dose than any other approved, sustained-release bupivacaine product. It employs a proprietary extended-release technology that enables the continuous release of bupivacaine, a non-opioid local anesthetic, directly to the surgical site for 3 days after administration. POSIMIR was approved in February, 2021 by the US Food and Drug Administration. POSIMIR is indicated in adults for administration into the subacromial space under direct arthroscopic visualization to produce post-surgical analgesia for up to 72 hours following arthroscopic subacromial decompression. Use in any other surgical procedure is investigational. POSIMIR is a registered trademark of Innocoll Pharmaceuticals Limited. About Subacromial Decompression Shoulder Surgery Subacromial decompression is a type of shoulder surgery used to treat impingement syndrome, a common repetitive-use injury that causes pain when the arm is raised over the head. The procedure is performed arthroscopically, meaning that several small incisions are made in the skin and muscle of the shoulder through which a camera (arthroscope) and surgical instruments are inserted during surgery. Arthroscopic subacromial decompression is generally performed as outpatient surgery, and most patients go home within a few hours of surgery. The most intense pain typically occurs during the first 3 days and is often managed with a combination of nerve block and oral opioids. There are over 600,000 surgeries involving arthroscopic subacromial decompression performed each year in the U.S. About Innocoll Pharmaceuticals Limited Innocoll Pharmaceuticals Limited is a global biotech pharmaceutical company headquartered in Athlone, Ireland and is a subsidiary of Innocoll Biotherapeutics Holding Limited. The Innocoll group of companies is focused on the development and commercialization of pharmaceutical technologies to meet some of today’s most important healthcare challenges. About Gurnet Point Capital Gurnet Point Capital is a unique healthcare investment platform within the B-Flexion Group and led by a team with deep expertise in an industry for which they share a passion, both as investors and senior executives. GPC invests long-term capital and supports entrepreneurs in building a new generation of companies that deliver outsized returns through active ownership. Based in Cambridge, MA, its remit encompasses life sciences and health care focused businesses, with a particular emphasis on businesses that have high growth potential in the product development and commercialization stages of their evolution. With its strategy of driving best in class operational transformation for these businesses, to create social impact while generating significant economic value, Gurnet is able to deliver differentiated results for its investors and partners.

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BUSINESS INSIGHTS

Foundation Medicine Announces Strategic Collaboration with Arvinas

Foundation Medicine | June 06, 2022

Foundation Medicine, Inc., a pioneer in molecular profiling for cancer, announced a collaboration with Arvinas, Inc., to develop FoundationOne®Liquid CDx as a companion diagnostic for use with Arvinas’ bavdegalutamide an investigational novel PROTAC® protein degrader targeting the androgen receptor (AR). Arvinas’ bavdegalutamide is being developed for the potential treatment of men with metastatic castration resistant prostate cancer who have progressed on existing therapies. Arvinas is a clinical-stage biotechnology company and a pioneer in the rapidly growing field of targeted protein degradation. Arvinas’ proprietary PROTAC® targeted protein degraders, or proteolysis-targeting chimeras, work by harnessing the body’s natural protein disposal system to selectively and efficiently degrade and remove disease-causing proteins. AR activity is a key driver of prostate cancer, which makes the ability to regulate AR signaling an important factor in controlling disease progression. “We look forward to collaborating with Foundation Medicine to develop a companion diagnostic aimed at improving patient access. Foundation Medicine’s deep understanding of cancer genomics, scalable solutions, and regulatory expertise makes them an ideal partner for us as we develop bavdegalutamide as a potential new therapy for men with prostate cancer.” Ron Peck, M.D., chief medical officer at Arvinas Foundation Medicine’s portfolio of FDA-approved comprehensive genomic profiling tests offer physicians both blood- and tissue-based testing options for detecting genomic alterations that help guide personalized treatment decisions. As companion diagnostics, FoundationOne®CDx and FoundationOne®Liquid CDx allow oncologists to identify patients who may be appropriate for FDA-approved targeted therapies. “We are proud to serve as an end-to-end partner for Arvinas as they pioneer this new approach to treat cancer,” said Sanket Agrawal, chief biopharma business officer, Foundation Medicine. “Bringing our capabilities to this emerging area of biotechnology sets us on an exciting path to deepen our collective understanding of cancer biology and deliver more novel treatment options to patients now and in the future.” Foundation Medicine is an essential partner for biopharma organizations navigating the complexity of cancer care and research. This latest collaboration adds to its more than 65 global biopharma and biotechnology partnerships aimed at getting targeted cancer treatments to patients faster. About FoundationOne®CDx FoundationOne CDx is a next-generation sequencing based in vitro diagnostic device for detection of substitutions, insertion and deletion alterations (indels), and copy number alterations (CNAs) in 324 genes and select gene rearrangements, as well as genomic signatures including microsatellite instability (MSI) and tumor mutational burden (TMB) using DNA isolated from formalin-fixed, paraffin-embedded (FFPE) tumor tissue specimens. FoundationOne CDx is for prescription use only and is intended as a companion diagnostic to identify patients who may benefit from treatment with certain targeted therapies in accordance with their approved therapeutic product labeling. Additionally, FoundationOne CDx is intended to provide tumor mutation profiling to be used by qualified health care professionals in accordance with professional guidelines in oncology for patients with solid malignant neoplasms. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Some patients may require a biopsy. About FoundationOne®Liquid CDx FoundationOne Liquid CDx is a qualitative next generation sequencing based in vitro diagnostic test for prescription use only that uses targeted high throughput hybridization-based capture technology to analyze 324 genes utilizing circulating cell-free DNA (cfDNA) isolated from plasma derived from anti-coagulated peripheral whole blood of advanced cancer patients. The test is FDA-approved to report short variants in over 300 genes and is a companion diagnostic to identify patients who may benefit from treatment with specific therapies (listed in Table 1 of the Intended Use) in accordance with the approved therapeutic product labeling. Additional genomic findings may be reported and are not prescriptive or conclusive for labeled use of any specific therapeutic product. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Patients who are negative for companion diagnostic mutations should be reflexed to tumor tissue testing and genomic alteration status confirmed using an FDA-approved tumor tissue test, if feasible. About Foundation Medicine Foundation Medicine is a pioneer in molecular profiling for cancer, working to shape the future of clinical care and research. We collaborate with a broad range of partners across the cancer community and strive to set the standard for quality, scientific excellence, and regulatory leadership. Our deep understanding of cancer biology helps physicians make informed treatment decisions for their patients and empowers researchers to develop new medicines. Every day, we are driven to help our partners find answers and take action, enabling more people around the world to benefit from precision cancer care.

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