Huntington drug successfully lowers levels of disease-causing protein

medicalxpress | May 06, 2019

An international clinical trial has found that a new drug for Huntington disease is safe, and that treatment with the drug successfully lowers levels of the abnormal protein that causes the debilitating disease in patients. In a study published today in the New England Journal of Medicine, researchers from UBC and their colleagues have demonstrated for the first time that the drug, IONIS-HTTRX (now known as RO7234292) successfully lowered levels of the mutant huntingtin protein—the toxic protein that causes Huntington disease—in the central nervous system of patients. "This is a tremendously exciting and promising result for patients and families affected by this devastating genetic brain disorder," said Dr. Blair Leavitt, neurologist and director of research at the Centre for Huntington Disease at UBC. "For the first time, we have evidence that a treatment can not only decrease levels of the toxic disease-causing protein in patients, but that it is also safe and very well tolerated."

Spotlight

Dr. Ray Jupp, Sanofi; Barbara Ryan, Deutsche Bank; Renee Cohen, Shire; and Ken Kaitin, Tufts Center for the Study of Drug Development, Tufts University, sound off on why they are a part of Pharma Faceoff, a pharmaceutical industry talk show featuring top industry experts.

Spotlight

Dr. Ray Jupp, Sanofi; Barbara Ryan, Deutsche Bank; Renee Cohen, Shire; and Ken Kaitin, Tufts Center for the Study of Drug Development, Tufts University, sound off on why they are a part of Pharma Faceoff, a pharmaceutical industry talk show featuring top industry experts.

Related News

RESEARCH

Nanoform and Celanese Explore Ways to Improve Drug Delivery

Nanoform, Celanese | May 26, 2021

Nanoform Finland Plc, an innovative nanoparticle medicine enabling company, and Celanese Corporation, a global specialty materials company, announced today plans to investigate potential synergies in the field of nanoparticle-enabled drug delivery. The objective of this research is to determine the utility of combining Nanoform's nanoparticle platform technologies with Celanese's VitalDose® EVA copolymer delivery technology for drug-eluting implants. The objectives are to enable the development of next-generation drug delivery systems that can handle an increased drug load and have better-sustained release properties. Nanoform and Celanese intend to collaborate on formulation development, using each company's formulation expertise. "We are excited to announce our collaboration with Celanese. One of Nanoform's primary goals is to allow next-generation drug therapies through the use of our nanoparticles. Given the inherent difficulty in loading an adequate amount of drug into many medical devices, we see a huge opportunity in employing our nanoparticles to overcome this fundamental challenge. We are excited about the potential to use the combination of these two technologies to provide new therapies to patients "said Nanoform CEO Edward Hggström. "Celanese has a good track record of developing drug-eluting implants for women's health, ophthalmology, and central nervous system disorders by integrating our VitalDose® EVA copolymer delivery technology with proteins, peptides, and small molecules. Exploring Nanoform's nanoparticle technologies offers up new possibilities for enhancing formulation performance with existing products and enabling future products. We are excited to collaborate with Nanoform and hope that our efforts will result in innovations that will benefit patients "Laura Brand, Celanese Vice President of Medical & Pharmaceuticals, said.

Read More

PHARMA TECH

KSP Specialty Pharmacy now offers access to non-oncology drugs and launches new website featuring pharmacy patient portal

Karmanos Cancer Institute | October 05, 2021

The Barbara Ann Karmanos Cancer Institute and KSP Specialty Pharmacy (KSP) are pleased to announce the launch of new services and a new website: www.ksppharmacy.org. The new site features provider resources and information for payers and pharmaceutical companies. Most notably, the site includes an online portal where patients can refill their prescriptions. This tool also allows patients to see their list of medications, get reminders and interact with their pharmacist. A portal for patient education and tips is forthcoming. Additionally, KSP has begun offering non-oncology specialty drugs to serve providers and patients throughout the McLaren Health Care network. KSP is a specialty pharmacy within the McLaren Health Care Network, serving the needs of patients with complex disease states and high-cost therapeutic treatments. KSP began as an extension of the Barbara Ann Karmanos Cancer Institute in 2019 and has since grown to assist with specialty medication needs of patients throughout the McLaren system in Michigan, Ohio and Indiana. Additionally, the Karmanos ambulatory pharmacy, which provides non-specialty medication, has launched a portal with similar functionality. Those undergoing cancer treatment at Karmanos can now access their prescription information and request refills on the Karmanos pharmacy web page. We are continually striving to provide our patients with tools and convenience to help them understand their treatment. The new website and app will provide the outstanding service we offer over the phone and in person on a new, convenient platform, - Stephen Smith, RPh, MS, FASHP, Chief Pharmacy Officer. KSP provides patient care and customer service through a panel of expert clinical pharmacy specialists that can assist in drug education and management of not only a patient's specialty prescriptions, but their entire prescription portfolio. The pharmacy team has direct access to the patient's medical records and providers to ensure the time to delivery of the first dose is swift. In fact, the average rate to the first dose at KSP during fiscal year 2021 is 3.92 days, while the industry standard is more than ten days. A primary goal is to ensure that our in-house social worker and account resolution team find unique programs to allow patients access to high-cost medications without being financially burdened. This allows patients to stay adherent and compliant with their prescribed therapies and start a path to faster healing and recovery. Specialty medications are used to treat complex health conditions that include hepatitis C, multiple sclerosis, cancer, rheumatoid arthritis, organ transplant, migraines and others. KSP Specialty Pharmacy's expanded services include the ability to dispense medications for patients within the McLaren Health System who face these disease states. These medications may need to be administered by a health care professional, self-injected or taken by mouth, which may require clinical monitoring. Specialty medications often require special handling, delivery and storage requirements. KSP is accredited by the Utilization Review Accreditation Commission (URAC) and the Accreditation Commission for Health Care (ACHC). URAC designation was earned in 2020 and allows for the provision of agents in other specialized areas, such as HIV, gastroenterology, neurology and many others. About the Barbara Ann Karmanos Cancer Institute Karmanos Cancer Institute is a leader in transformative cancer care, research and education through courage, commitment and compassion. Our vision is a world free of cancer. As part of McLaren Health Care, Karmanos is the largest provider of cancer care and research in the state of Michigan. For more than 75 years, our administrative and research headquarters, along with our premier specialty cancer hospital, have been located in downtown Detroit. Our 15 network sites throughout the state deliver market-leading cancer care and clinical trials conveniently to the communities where many of our patients live. Karmanos is recognized by the National Cancer Institute as one of the best cancer centers in the nation. Our academic partnership with the Wayne State University School of Medicine provides the framework for cancer research and education – defining new standards of care and improving survivorship.

Read More

PHARMA TECH

Xeris Pharmaceuticals Enters Collaboration Agreement With Merck

Xeris | October 25, 2021

Xeris Biopharma Holdingsa biopharmaceutical company developing and commercializing unique therapies for patient populations in endocrinology, neurology, and gastroenterology, today announced a collaboration agreement with Merck , with an option to license Xeris “We are quite excited to be working closely with Merck on this opportunity using our novel platform technology, XeriJect The XeriJect platform has broad applicability in improving the delivery of large molecules, such as monoclonal antibodies. This is another in a series of collaborations Xeris has undertaken with top 10 pharma companies evaluating the XeriJect technology.” Paul R. Edick, Chairman and CEO of Xeris Biopharma Mr. Edick continued, "These collaborations are important to validating our technology. We recognize they take time to complete and thus, they are not immediately critical to our near-term core business. However, should Merck exercise the negotiated license agreement, they could be very valuable to Xeris. It is the continued growth of Gvoke® and Keveyis®, the potential for the approval and launch of Recorlev, a pipeline supported by our novel technologies, our strong cash balance, and an expected $50 million in synergies from the Strongbridge acquisition that puts Xeris in a solid financial position.” About Xeris Biopharma Holdings Xeris (Nasdaq: XERS) is a biopharmaceutical company developing and commercializing unique therapies for patient populations in endocrinology, neurology, and gastroenterology. Xeris has two commercially available products, Gvoke®, a ready-to-use liquid glucagon for the treatment of severe hypoglycemia and Keveyis®, the first and only FDA-approved therapy for primary periodic paralysis. Xeris also has a robust pipeline of development programs to extend the current marketed products into important new indications and uses and bring new products forward using its proprietary formulation technology platforms, XeriSol™ and XeriJect™, supporting long-term product development and commercial success. Forward-Looking Statements Any statements in this press release about future expectations, plans and prospects for Xeris Biopharma Holdings, Inc. including statements regarding the market and therapeutic potential of its products and product candidates, expectations regarding clinical data or results from planned clinical trials, estimates and projections about the potential benefits of the Strongbridge Biopharma acquisition, estimates and expectations regarding potential collaborations, the timing or likelihood of regulatory approval and commercialization of its product candidates, the timing or likelihood of expansion into additional markets, the timing or likelihood of identifying potential development and commercialization partnerships, the potential utility of its formulation platforms and other statements containing the words “will,” “would,” “continue,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those indicated in the forward-looking statements. Such risks and uncertainties include, but are not limited to, failure to realize the expected benefits of the acquisition, failure to promptly and effectively integrate Strongbridge’s businesses, general economic and business conditions that affect the combined companies following the consummation of the acquisition, the impact of the COVID-19 pandemic on the combined businesses following the consummation of the transaction, changes in global, political, economic, business, competitive, market and regulatory forces, future exchange and interest rates, changes in tax laws, regulations, rates and policies, future business acquisitions or disposals and competitive developments. These forward-looking statements are based on numerous assumptions and assessments made in light of Xeris’ experience and perception of historical trends, current conditions, business strategies, operating environment, future developments, and other factors it believes appropriate. By their nature, forward-looking statements involve known and unknown risks and uncertainties because they relate to events and depend on circumstances that will occur in the future. The factors described in the context of such forward-looking statements in this communication could cause Xeris’ plans with respect to Strongbridge, Xeris’ plans with respect to XeriJect, Xeris’ actual results, performance or achievements, industry results and developments to differ materially from those expressed in or implied by such forward-looking statements. Although it is believed that the expectations reflected in such forward-looking statements are reasonable, no assurance can be given that such expectations will prove to have been correct and persons reading this communication are therefore cautioned not to place undue reliance on these forward-looking statements which speak only as at the date of this communication. Additional information about economic, competitive, governmental, technological, and other factors that may affect Strongbridge is set forth in Item 1A, “Risk Factors,” in Strongbridge’s 2020 Annual Report on Form 10-K, which has been filed with the SEC, the contents of which are not incorporated by reference into, nor do they form part of, this communication. Any forward-looking statements in this communication are based upon information available to Xeris, as of the date of this communication and, while believed to be true when made, may ultimately prove to be incorrect. Subject to any obligations under applicable law, Xeris does not undertake any obligation to update any forward-looking statement whether as a result of new information, future developments or otherwise, or to conform any forward-looking statement to actual results, future events, or to changes in expectations. All subsequent written and oral forward-looking statements attributable to Xeris or any person acting on behalf of any of them are expressly qualified in their entirety by this paragraph.

Read More