Insmed Incorporated announced that the U.S. Patent and Trademark Office has issued patent No. 10,251,900 for certain uses of ARIKAYCE. The claims of the patent relate in part to methods for treating Mycobacterium avium complex lung disease via administration of ARIKAYCE to patients previously unresponsive to MAC therapy. This is the 10th patent issued by the USPTO for ARIKAYCE in MAC lung disease and the second with an expiry date of May 15, 2035. ARIKAYCE was granted accelerated approval by the U.S. Food and Drug Administration on September 28, 2018, for the treatment of MAC lung disease as part of a combination antibacterial drug regimen for adult patients who have limited or no alternative treatment options. “We are very pleased to receive another patent for ARIKAYCE that reinforces our exclusivity in the U.S. to 2035 for methods that align closely with our approved label, further strengthening our global patent portfolio,” said Will Lewis, Chairman and Chief Executive Officer of Insmed. “In addition, as we advance toward regulatory filings for ARIKAYCE in Europe and Japan, we are continuing to pursue intellectual property protection in these and other major markets worldwide.”
https://thefly.com/landingPageNews.php?id=2890489
Tuesday, April 9, 2019
Chimerix beefs up management team
Michael A. Sherman, former CEO of Endocyte, appointed as CEO of Chimerix, effective immediately.
Michael T. Andriole, former CFO of Endocyte, appointed to the newly created position of Chief Business Officer.
INmune Bio looks to help treat Alzheimer’s disease and cancer
Therapy candidate INB03, meant to inactivate a kind immunosuppressive cell called myeloid-derived suppressor cells (MDSCs) while boosting anti-tumor immune cells, may help overcome resistance to immunotherapies, including immune checkpoint inhibitors, INmune Bio’s co-founder Raymond J. Tesi, MD, said at a conference.
Tesi, CEO of INmune Bio, presented the potentialities of the company’s new therapy at the Cambridge Healthtech Institute’s 4th Annual Immuno-Oncology Summit Europe recently in London.
His presentation was titled “Targeting Myeloid-Derived Suppressor Cells to Overcome Resistance to Checkpoint Inhibitors.”
MDSCs appear in advanced forms of cancer and are at the heart of cancer resistance to the body’s immune system. MDSCs move to where tumors are and release cell signaling proteins called cytokines to form an immunosuppressive shield that protects tumors from immune system attacks.
The shield also neutralizes treatments that rely on anti-cancer immune responses, such as immunotherapy with checkpoint inhibitors. In fact, the higher the number of MDSCs in a patient’s blood, the more severe the cancer is predicted to be and the more likely it is to be resistant to immunotherapy.
“Experts agree that decreasing the number of MDSC may improve the response to immunotherapy such as checkpoint inhibitors and may improve the ability of the immune system to fight the cancer,” INmune Bio states on its website.
INB03 was designed to inactivate MDSCs and destroy the protective shield that prevents immune cells from coming in and fighting it. The treatment is expected to strengthen patients’ immunity to cancer and give immunotherapies a better chance at being effective.
Specifically, INB03 is a second-generation inhibitor of tumor necrosis factor (TNF). It targets only cell-free TNF without affecting other types of TNF that are attached to the membrane of cells.
Because of this selectivity, the therapy does not suppress the immune system, a known side effect of currently approved, first-generation TNF inhibitors, such as those used for autoimmune disorders including rheumatoid arthritis.
The new compound acts in three steps. First, it blocks the proliferation and function of MDSCs, breaking the protecting shield of immunosuppressive signals produced by those cells.
This promotes the infiltration of innate immune cells (part of the body’s first-line of defenae), specifically natural killer (NK) cells and dendritic cells, which recognize and attack the tumor and also cross-talk with other immune cell types to reinforce subsequent immune responses.
From this cross-talk, more cytokines are released that call in cytotoxic T-cells, immune cells able to directly attack and kill the tumor. In animal studies, treatment with INB03 resulted in smaller and fewer tumors, extending survival.
A video explaining INB03’s mode of action is available here.
The potential therapy is now being tested in patients with advanced, metastatic, solid tumors in a Phase 1 trial (ACTRN12618000675224). The study is intended to address if the treatment is safe and well-tolerated and collect initial data about its efficacy. Its target population are patients with increased biomarkers of inflammation and a high number of MDSCs in their blood.
Three different doses of the treatment will be tested, all given by under-the-skin (subcutaneous) injections once per week for nine months or until disease progression.
Primary outcome measures will be the occurrence of adverse events, the therapy’s pharmacokinetics (absorption, distribution, metabolism, and elimination) of single and multiple doses, and change in high sensitivity C-reactive protein serum levels, a biomarker of inflammation.
Participants may opt to continue on INB03 at any time if their doctor believes the therapy is beneficial in any way.
An interim analysis of the results will be conducted after three months of treatment, and an optimal dose for a subsequent Phase 2 trial will be determined.
“I am excited to have the opportunity to present and exchange ideas with some of the most influential members of the immuno-oncology community,” Tesi said in a press release. “We are committed to developing treatments that target the patient’s innate immune system and facilitating an understanding that chronic inflammation is a root cause and agitator of cancer and many other diseases. By exchanging ideas with top researchers, we can help further push developments that lead to effective patient responses at the bedside.”
Unilever’s Schmidt’s Naturals to launch hemp-oil deodorants in September
Anglo-Dutch consumer goods giant Unilever PLC will be taking its first step into the U.S. hemp market in September, when its subsidiary brand Schmidt’s Naturals launches a line of hemp-oil deodorants that will be available at certain retailers.
That line will be followed later in the fall by a separate range of CBD products, according to Schmidt’s Naturals CEO Michael Cammarata. That’s assuming that regulations for CBD, a non-intoxicating ingredient in the cannabis plant, have been clarified by then, he told MarketWatch.
“We are working with our supply chain to ensure that we comply with FDA and state rules,” Cammarata said.
CBD was expected to be fully legalized along with hemp as part of the December Farm Bill, but instead it was moved under the purview of the U.S. Food and Drug Administration from the Drug Enforcement Administration. That’s because the FDA views it as a drug.
CBD is an ingredient in the only cannabis-based drug that has won FDA approval as a treatment for severe forms of childhood epilepsy. The FDA has promised to hold talks on regulating the substance this month and outgoing commissioner Scott Gottlieb said he would seek pathways for approval. But until those have been created, the FDA is not allowing companies to add CBD to food, beverages or cosmetics, forcing many companies to put their plans on hold.
Amgen says FDA approves EVENITY for treatment of osteoporosis
Amgen and UCB announced that the U.S. Food and Drug Administration has approved EVENITY for the treatment of osteoporosis in postmenopausal women at high risk for fracture. EVENITY is the first and only bone builder with a unique dual effect that both increases bone formation and to a lesser extent reduces bone resorption to rapidly reduce the risk of fracture. A full course of EVENITY therapy is 12 monthly doses administered by a healthcare provider. Since osteoporosis is a chronic disease, continued therapy with an anti-resorptive agent should be considered once EVENITY therapy is completed. “One in two women will experience a fracture due to osteoporosis in her lifetime. These fractures can be devastating, with many leading to hospital stays and life-altering consequences. The FDA approval of EVENITY represents an important therapeutic development for patients who need a medicine that can rapidly increase bone mineral density and help reduce the risk of future fractures within 12 months,” said David M. Reese, M.D., executive vice president of Research and Development at Amgen. “Postmenopausal osteoporosis is a significant women’s health issue that far too often gets overlooked. As a leader in bone health with more than 20 years of osteoporosis research experience, Amgen is as committed as ever to combatting this disease to help women at high risk for fracture reduce their risk of a first and subsequent fracture.”
Centene to invest $100M in Alzheimer’s, diabetes research at Washington U
St. Louis-based Centene Corp. announced it will fund up to $100 million in research at Washington University School of Medicine.
The funding will be aimed at research in Alzheimer’s disease, breast cancer, diabetes and obesity at Washington University, also based in St. Louis. Those particular diseases were targeted because they are common, debilitating and often deadly diseases that affect individuals worldwide, officials said.
Under the agreement, Centene’s funding would be dispersed over 10 years to the School of Medicine’s Personalized Medicine Initiative. Innovations that come from the initiative will be commercialized through a joint venture between the school and Centene.
Officials said the agreement would allow Centene to leverage the university’s research and biomedical capabilities in CRISPR as well as its talent in researching the microbiome, immunomodulatory therapies, cancer genomics, neurodegeneration, cellular reprogramming, chemical biology and informatics.
It’s the latest in a spending spree by Centene, which announced last month it would purchase rival insurer WellCare Health Plans in a deal valued at $17.3 billion. It will create one of the largest sponsors of government plans across the three main markets—Medicare, Medicaid and the Affordable Care Act exchanges—following the WellCare merger.
Last fall, it was announced Centene backed pharmacy benefit manager RxAdvance in a $50 million funding round.
“We share the goal of helping to improve the health of our communities through research, education and customized treatment for people suffering from chronic illnesses,” said Michael Neidorff, chairman and CEO for Centene, in a statement. “We believe personalized medicine is the path to ensure patients get the targeted health care they need to fight disease.”
The funds are also expected to support more than a dozen centers at the School of Medicine.
“We will be bringing together world-class resources and intellectual horsepower from every basic and clinical scientific discipline to urgently accelerate the timeline for developing therapies that are more precisely targeted, with aspirations to do so in the next five to seven years,” said David Perlmutter, M.D., executive vice chancellor for medical affairs at the School of Medicine.
5 drugs in development Optum says payers, employers should be watching
Amid rising drug costs, employers and payers should be keeping a closer eye on products in the pipeline as they design formularies.
Specifically, there are certain emerging therapies that could lead to significant costs down the road, according to Optum, which recently released its list of five drugs to watch in 2019.
Those drugs are:
- Golodirsen, a treatment for Duchenne muscular dystrophy
- Siponimod, which aims to delay the progression of multiple sclerosis
- Zynquista, or sotagliflozin, a new oral insulin adjunct medication for people with Type 1 diabetes
- Risankizumab, an injectable medication for plaque psoriasis
- Ultomiris, a longer-lasting medication for paroxysmal nocturnal hemoglobinuria, a rare blood disorder
David Calabrese, OptumRx vice president and chief pharmacy officer, told FierceHealthcare that drugs like these, particularly those for rare diseases, have been consistent cost drivers. He said it’s crucial for insurers to be watching these products early, so those with limited value can be determined sooner.
“I think it’s just exercising a heightened level of due diligence,” he said. “Historically, organizations like ours would wait until the product was FDA approved, then dive into the data. We’ve taken a much more proactive approach over the course of the last half decade or so.”
Optum projects spending several years out in its analysis. For example, sales of Zynquista and other sotagliflozin products could peak at $1.3 billion by 2024, according to the report. That drug did hit a snag with the Food and Drug Administration, however, which declined to approve it in March, likely delaying a 2019 launch.
Golodirsen is in development by Sarepta Therapeutics, which also currently offers a similar product in that class, Exondys-51, priced at $300,000 per year—so Optum expects Golodirsen to be similarly costly.
Aside from greater awareness, what can payers do to better manage the costs of expensive new therapies? Alongside monitoring emerging therapies and treatments for rare conditions earlier, Optum has developed clear guidelines for physicians, so they know when it’s appropriate to provide these pricey drugs to patients, Calabrese said.
It does this using a digital tool called PreCheck MyScript, which notifies doctors within the electronic health record of their options for certain conditions, what a patient’s out-of-pocket costs would be and if a prior authorization is required. Physicians then have the option to apply immediately within in the system for prior authorization if they choose that drug, Calabrese said.
“They’d know immediately whether that product would be approved or not for that given patient at that given time,” Calabrese said.
In addition, Optum recommends that payers offer a robust list of biosimilars to ensure that patients have access to lower-cost alternatives to pricey brand drugs. While this is a challenge, as the biosimilar pipeline is limited at present, Calabrese said one of the key functions of its specialty pharmacy is to make patients and doctors more aware of these alternatives.
It’s also key to closely monitor drugs like those on Optum’s list for effectiveness and any potential negative side effects after offering coverage, so that they’re included in formularies where it makes the most sense, he said.
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