Novartis, shadowed by data scandal, renews case for Zolgensma

BioPharma Dive | September 19, 2019

Infants likely to develop a severe form of spinal muscular atrophy, but who were not yet symptomatic, could sit and stand following treatment with Novartis' gene therapy Zolgensma, according to new study results disclosed by the Swiss drugmaker. The data, presented Thursday at the European Paediatric Neurology Society's annual meeting in Athens, Greece, are an update from an initial glimpse the company gave in May. Since then, more babies with the inherited neuromuscular disease achieved motor milestones like sitting independently for more than 30 seconds or standing with assistance. Typically, infants diagnosed with severe SMA are never able to sit, and most die by age two. Five patients in the trial, called SPR1NT, achieved the maximum score on a University of Pennsylvania scale used to gauge motor abilities, an improvement from the three who did by the May update. Two others have reached normal levels.

Spotlight

RJ Lewis Founder & CEO eHealthcare Solutions explains why it's important for pharma to take risks and conduct A/B testing to find and implement better digital solutions.

Spotlight

RJ Lewis Founder & CEO eHealthcare Solutions explains why it's important for pharma to take risks and conduct A/B testing to find and implement better digital solutions.

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PHARMA TECH

Quantum Leap and SignalPath to Integrate Clinical Trial Software Infrastructure Across I-SPY Platform Trials

Quantum Leap, SignalPath | February 24, 2021

Quantum Leap Healthcare Cooperative (Quantum Leap) and SignalPath reported a partnership today that will further enable the I-SPY platform trials with vigorous, streamlined technology infrastructure to drive the straightforwardness and efficiency of trial execution. SignalPath will integrate existing work process instruments being utilized to control the I-SPY trials into SignalPath's research operations and financial platform. In view of the public health emergency surrounding the COVID-19 pandemic, this advanced technology infrastructure will initially be conveyed for I-SPY COVID-19 with subsequent expansion to I-SPY2. “As we continue to grow our platform trials with a focus on I-SPY COVID-19, and the growing complexity of processing drug candidates through our platform trials, we need sophisticated infrastructure to ensure we can maintain our efficiency and agility. SignalPath provides the missing link for a comprehensive, tech-enabled trial technology platform to unlock the full potential of adaptive trial designs. This further empowers us to address urgent health needs that can only be solved through exceptional, technology-enabled clinical research,” observed James Palazzolo, CEO of Quantum Health. The I-SPY platform has had incredible accomplishment in working with leading academic medical centers around the United States. SignalPath is helping to extend that footprint across the a-list community health systems on the SignalPath platform. The objective is to continue to draw in community health systems with hearty research programs in cutting edge research to help their patients. “We founded SignalPath to re-imagine clinical trial execution through technology,” stated Brad Hirsch, MD, CEO and Co-Founder, SignalPath. “The Quantum Leap team has already accomplished an amazing amount in the fight against breast cancer and is now applying their full force to drive insights into COVID-19. It is an incredible opportunity for us to collaboratively support trial execution through our powerful technology infrastructure to meaningfully reduce the complexity for both the Quantum Leap team and the sites involved in the I-SPY trials. Everyone’s goal is the same - to bring life-saving medicines to patients that need them most.” About SignalPath SignalPath is the premier clinical trial technology partner for research sites and sponsors, focused on improving the ease and efficiency of trial execution and management. Supporting over 5,000 trials across more than 300 sites and thousands of active users, SignalPath is the industry standard that addresses key pain points in the conduct of trials. About Quantum Leap Healthcare Collaborative Quantum Leap Healthcare Collaborative is a 501c(3) charitable organization established in 2005 as a collaboration between medical researchers at University of California, San Francisco and Silicon Valley entrepreneurs. Our mission is to integrate care and research, and to foster high-impact trials with embedded clinical processes and systems technology and improved data management, greater access to clinical trial matching, and greater benefit to patients, providers, and researchers. Our goal is to improve and save lives. Quantum Leap provides operational, financial, and regulatory oversight to I-SPY. About the I-SPY TRIALs The I-SPY TRIAL (Investigation of Serial studies to Predict Your Therapeutic Response with Imaging And moLecular analysis) was designed to rapidly screen promising experimental treatments and identify those most effective in specific patient subgroups based on molecular characteristics (biomarker signatures). The trial is a unique collaborative effort by a consortium that includes the Food and Drug Administration (FDA), industry, patient advocates, philanthropic sponsors, and clinicians from 16 major U.S. cancer research centers. Under the terms of the collaboration agreement, Quantum Leap Healthcare Collaborative is the trial sponsor and manages all study operations.

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PRACTICE MANAGEMENT

Aprecia Announces New Leadership Team

Aprecia Pharmaceuticals | October 21, 2021

Kyle Smith has been named President and Chief Operating Officer of Aprecia Pharmaceuticals, LLC, effective immediately. Mr. Smith is a 10-year veteran with Aprecia and has served as Vice President of Operations for the past 3 years. As President and Chief Operating Officer, Mr. Smith will be responsible for leading day-to-day business activities. Aprecia has further strengthened its leadership team by promoting Patrick Staudt to Vice President of Operations, effective immediately. A 14-year veteran of Aprecia, Mr. Staudt has led the East Windsor, NJ manufacturing operations for more than 10 years and has demonstrated the talent and experience to lead expected, expansive growth through multiple technology platforms. Mr. Smith and Mr. Staudt have been cornerstones of Aprecia's history of technology and manufacturing innovation. They have been key contributors in establishing the company as the leader in the development and manufacturing of pharmaceutical products through 3D-Printing. Their experiences with Aprecia have helped craft the company culture and 3DP pharmaceutical manufacturing innovation, and their contributions have created a momentum which continues to accelerate through important partnerships and novel products. "As Aprecia's technology platforms expand and industry partnerships advance, Aprecia requires additional talent and resources to meet these growing demands, We are fortunate to have these seasoned technology experts and corporate leaders to promote and guide the organizational growth well into the future." Chris Gilmore, Aprecia's Chief Executive Officer Mr. Kyle Smith holds a Master of Business Administration degree from Miami University, and a Bachelor of Science in Chemical Engineering from Georgia Institute of Technology. Mr. Patrick Staudt holds a Bachelor of Science degree in Biomedical/Medical Engineering from Drexel University. About Aprecia Founded in 2004, Aprecia received the first and only FDA approved three-dimensionally-printed (3DP) pharmaceutical product approval in 2015. Aprecia uses its ZipDose® Technology to create rapidly disintegrating oral dosage forms that are easy to take and easy to administer. Without compression during the 3DP manufacturing process, engineered and coated particles such as taste masking and modified release are possible on large dose products (over a 1000mg). Aprecia directly owns a patent estate for novel 3DP machines and pharmaceutical 3DP applications. It licenses its exclusive technology platform to pharmaceutical partners as a means to extend product lines, improve patient reach and experience, and address FDA requirements as a pediatric delivery form.

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BUSINESS INSIGHTS

InDex Pharmaceuticals strengthens the clinical development organization in preparation of the start of the phase III study CONCLUDE with cobitolimod

InDex Pharmaceuticals | August 23, 2021

InDex Pharmaceuticals Holding AB today announced that two new employees have been appointed in the clinical development organisation in preparation of the start of the phase III study CONCLUDE with cobitolimod. Anders Bröijersén is joining InDex as Senior Medical Director Clinical Operations and Johan Levin as Project Manager Clinical Operations. Anders Bröijersén is a medical doctor and PhD with 15 years of experience in Clinical Development, Medical Affairs and Drug Safety at leading pharmaceutical companies such as Boehringer Ingelheim and MSD. Anders joins InDex from Sobi where he currently serves as Medical Director, Global Pharmacovigilance & Patient Safety. Johan Levin is a trained nurse and has more than 20 years of experience in Clinical Development at leading pharmaceutical companies and clinical research organizations (CROs) such as MSD and Covance. He currently works as senior Clinical Research Associate (CRA) at PRA Health Sciences. "I am pleased that InDex has been able to attract two highly qualified new employees and welcome Anders and Johan to the team," said Peter Zerhouni, CEO of InDex Pharmaceuticals. "We are expanding the organisation in preparation of the start of our global phase III study CONCLUDE with cobitolimod to be able to be active towards the study sites in collaboration with our CRO. This was a successful model for patient recruitment in our previous study CONDUCT." The phase III study CONCLUDE with cobitolimod will include 440 patients and be conducted at several hundred clinics in over 30 countries. It is a randomised, double-blind, placebo-controlled, clinical study to evaluate cobitolimod as a novel treatment for patients with moderate to severe left-sided ulcerative colitis. Cobitolimod Cobitolimod is a first-in-class Toll-like receptor 9 (TLR9) agonist that can provide an anti-inflammatory effect locally in the large intestine, which may induce mucosal healing and relief of the clinical symptoms in ulcerative colitis. Cobitolimod met the primary endpoint in the phase IIb study CONDUCT and demonstrated an outstanding combination of efficacy and safety. The results have been published in the reputable medical journal, The Lancet Gastroenterology & Hepatology. Data from four previous completed placebo-controlled clinical trials support the efficacy and safety demonstrated in the CONDUCT study. InDex Pharmaceuticals InDex is a pharmaceutical development company focusing on immunological diseases where there is a high unmet medical need for new treatment options. The company's lead asset is the drug candidate cobitolimod, which is in late stage clinical development for the treatment of moderate to severe ulcerative colitis – a debilitating, chronic inflammation of the large intestine. InDex has also developed a platform of patent protected discovery stage substances, so called DNA based ImmunoModulatory Sequences (DIMS), with the potential to be used in the treatment of various immunological diseases.

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