PRNewswire | August 04, 2023
Ionis Pharmaceuticals, Inc. announced that it has entered a collaboration and license agreement with Novartis for the discovery, development and commercialization of a novel medicine for patients with lipoprotein(a), or Lp(a)-driven cardiovascular disease (CVD). This builds on the companies' existing collaboration focused on the development and commercialization of pelacarsen, which Novartis is currently evaluating in a Phase 3 cardiovascular outcome study. The next generation compound will be a potential follow-on to pelacarsen.
"We are pleased to expand our productive collaboration with Novartis aimed at delivering transformative therapies to patients with elevated Lp(a) who are at high risk of cardiovascular events," said Brett P. Monia, Ph.D., Ionis' chief executive officer. "This collaboration is designed to leverage Ionis' advancing RNA-targeting platform technologies to deliver a novel Lp(a)-targeting therapy that we expect will provide industry-leading efficacy and dosing frequency."
Ionis will receive a $60 million upfront payment from Novartis and is eligible to earn development, regulatory and commercial milestone payments and tiered royalties. Novartis will be solely responsible for the development, manufacturing and potential commercialization of the next generation Lp(a) therapy.
The agreement is subject to the satisfaction of requirements of the Hart-Scott-Rodino Antitrust Improvements Act of 1976.
Lp(a) is a lipoprotein particle assembled in the liver that consists of a low-density lipoprotein cholesterol (LDL-C)-like particle and apolipoprotein(a). Lp(a) levels in the blood can vary greatly between individuals primarily due to genetic variations and do not correlate with LDL-C levels. Even patients with LDL-C lowered to target levels (<70 mg/dL) remain at high-risk of cardiovascular events if they have high levels of Lp(a). Elevated Lp(a) is a genetically determined condition that is not responsive to lifestyle changes, therefore patients are unable to adequately control their Lp(a) levels through improved diet or increased physical activity. Elevated Lp(a) is recognized as an independent, genetic cause of coronary artery disease, heart attack, stroke, peripheral arterial disease and aortic stenosis. Currently, there is no effective drug therapy to specifically and robustly lower elevated levels of Lp(a).
Pelacarsen was discovered by Ionis and licensed to Novartis in 2019 for exclusive worldwide development, manufacturing and commercialization. It is an investigational antisense medicine designed to reduce apolipoprotein(a) production in the liver to offer a direct approach for reducing circulating Lp(a). It is estimated that more than eight million people worldwide have elevated Lp(a) and CVD.
Pelacarsen is currently being evaluated in Lp(a) HORIZON (NCT04023552), a global, multicenter, double-blind, placebo-controlled pivotal Phase 3 study conducted by Novartis. The study completed enrollment with 8,323 participants. Data are expected in 2025.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been a leader in RNA-targeted therapy, pioneering new markets and changing standards of care. Ionis currently has four marketed medicines and a promising late-stage pipeline highlighted by cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision to become the leader in genetic medicine, utilizing a multi-platform approach to discover, develop and deliver life-transforming therapies.
Businesswire | August 18, 2023
Koneksa, a healthcare technology company pioneering evidence-based digital biomarkers, today announced the first collaboration in the Koneksa Academic Partnership Program for Parkinson’s Disease (KAPP-PD) to incorporate its Neuroscience Toolkit into research programs.
The first collaboration of the KAPP-PD program, with Oregon Health & Science University (OHSU), enables the inclusion of app-and actigraphy-based digital measures and electronic patient reported outcome (ePRO) instruments from Koneksa’s Neuroscience Toolkit into a study comparing short-acting versus long-acting levodopa in Parkinson’s disease. Koneksa’s Neuroscience Toolkit implements a variety of digital assessments that can be combined to collect data for measures of interest.
The OHSU team, led by Dr. Delaram Safarpour, MD, MSCE, FAAN, is investigating changes in the neurophysiological correlates of PD with carbidopa-levodopa immediate release compared to long-acting levodopa (Rytary), as well as changes in gait performance at intervals of 30 minutes for a total of 4 hours during each visit. This research will provide strong support for clinical profiles of short- versus long- acting levodopa in PD corresponding to objective physiological measures.
“We are collaborating with Koneksa to enhance our study’s objectives: We’ll be including smartphone- and actigraphy-based digital measures and electronic patient reported outcome (ePRO) instruments into our study,” said Martina Mancini, Ph.D., co-investigator of the study, director of the Balance Disorders Laboratory, and Associate Professor of Neurology, OHSU School of Medicine. “Our lab uses new technologies to better understand and measure motor impairments, with the goal of learning how to better help patients with these conditions. Novel, objective metrics, like those made possible with Koneksa’s technology and toolkit, allow us to expand upon what we’ve been able to do in the clinic. In particular, digital at-home measures of daily physical activity, gait and mobility, motor functionality, sleep, and ePRO measures of quality of life all have the potential to uniquely complement OHSU’s in-clinic assessments, offering new and real-world-setting patient insights to support the study’s objectives.”
“Koneksa is proud to extend our contribution to critical neuroscience research through KAPP-PD, and we’re delighted to get the program underway with our first collaboration,” said John Wagner, MD, Ph.D., Chief Medical Officer, Koneksa. “These measures will support OHSU in both in-clinic and at-home patient monitoring of critical parameters of interest for this study. We’re committed to helping to get new and better treatments to patients faster, and that’s a vision that OHSU’s Balance Disorders Laboratory shares.”
About the Koneksa Academic Partnership Program – Parkinson’s Disease
The Koneksa Academic Partnership Program – Parkinson’s Disease (KAPP-PD) is a structured collaboration between Koneksa and select academic partners that enables researchers to incorporate Koneksa’s Neuroscience Toolkit into research programs focusing on Parkinson’s disease. Researchers gain access to an array of devices that capture key measures objectively and sensitively, as well as training materials for site coordinators and study subjects, a cloud-based platform for real-time monitoring, and industry-leading analytics and patented algorithms.
Koneksa is a healthcare technology company pioneering evidence-based digital biomarkers to accelerate clinical research and guide decision making in drug development and market strategy. Our evidence-based biomarker solutions enable efficient clinical trial designs to help innovative therapies reach patients faster. Koneksa aims to revolutionize treatment effect detection in clinical research and improve patient outcomes.
prnewswire | August 29, 2023
Halozyme Therapeutics, Inc. announced the approval of Roche's Tecentriq® SC (atezolizumab), using Halozyme's ENHANZE® drug delivery technology, by the Medicines and Healthcare Products Regulatory Agency (MHRA) in Great Britain. Tecentriq® subcutaneous (SC) is co-formulated with Halozyme's proprietary recombinant human hyaluronidase enzyme, rHuPH20, allowing for subcutaneous delivery, which takes approximately seven minutes, compared with 30-60 minutes for intravenous (IV) infusion. Tecentriq® SC will be available to patients in Great Britain for certain types of lung, bladder, breast, and liver cancer.7
Multiple oncology studies suggest that most cancer patients prefer to receive their treatments via subcutaneous delivery, instead of intravenous infusion because of reduced discomfort, ease of administration, and shorter duration of treatment.2,4,11
"We are delighted that Roche has received approval of Tecentriq SC in Great Britain. Subcutaneous delivery of Tecentriq provides cancer patients a faster and more flexible treatment option and may alleviate pressure on healthcare system resources," said Dr. Helen Torley, president and chief executive officer of Halozyme. "We look forward to additional regulatory approvals of our partner products utilizing ENHANZE."
Tecentriq® SC is the first subcutaneous anti-PD-(L)1 cancer immunotherapy available to patients in Great Britain and is Roche's fourth subcutaneous cancer therapy using Halozyme's ENHANZE® drug delivery technology.8-10 The approval is based on clinical data from the Phase IB/III IMscin001 study, which showed comparable levels of Tecentriq® in the blood when administered subcutaneously, and a safety and efficacy profile consistent with the IV formulation.12 While the IMscin001 trial was conducted within the hospital setting, the SC formulation may be suitable for out-of-hospital administration by a healthcare professional.
The U.S. Food and Drug Administration (FDA) is currently evaluating Tecentriq® SC for regulatory approval.
Halozyme is a biopharmaceutical company bringing disruptive solutions to significantly improve patient experiences and outcomes for emerging and established therapies. As the innovators of the ENHANZE® technology with the proprietary enzyme rHuPH20, Halozyme's commercially-validated solution is used to facilitate the delivery of injected drugs and fluids in order to reduce the treatment burden to patients. Having touched more than 700,000 patient lives in post-marketing use in six commercialized products across more than 100 global markets, Halozyme has licensed its ENHANZE® technology to leading pharmaceutical and biotechnology companies including Roche, Takeda, Pfizer, AbbVie, Eli Lilly, Bristol-Myers Squibb, Alexion, argenx, Horizon Therapeutics, ViiV Healthcare and Chugai Pharmaceutical.
Halozyme also develops, manufactures and commercializes, for itself or with partners, drug-device combination products using its advanced auto-injector technology that are designed to provide commercial or functional advantages such as improved convenience and tolerability, and enhanced patient comfort and adherence. The Company has a commercial portfolio of proprietary products including XYOSTED®, TLANDO® and NOCDURNA® and partnered commercial products and ongoing product development programs with several pharmaceutical companies including Teva Pharmaceuticals and Idorsia Pharmaceuticals.