GlobeNewswire by notified

Novartis Provides Update on AVXS-101 Intrathecal Clinical Development Program

Share
  • Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (IT) formulation in older patients with SMA to further support registration


Basel, September23, 2020Novartis Gene Therapies recently received feedback from the US Food and Drug Administration (FDA) following their review of data from the STRONG study of the intrathecal (IT) formulation of AVXS-101 in older patients with spinal muscular atrophy (SMA). The FDA has acknowledged the potential of AVXS-101 IT in this patient population and recommends a pivotal confirmatory study to supplement the existing STRONG data and further support the regulatory submission for AVXS-101 IT.

This guidance provides clarity on the path to registration for AVXS-101 IT. Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. This request for a study is unrelated to the partial clinical hold on AVXS-101 IT, and the new study will not be initiated in the US until the hold has been lifted by the FDA. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment. This does not impact marketed Zolgensma®(onasemnogene abeparvovec) and the company continues to advance its regulatory filings and intravenous clinical studies.

Novartis Gene Therapies reaffirms its commitment to the SMA community and to pursuing solutions for patients with all types of SMA, including older children and adults. All patients deserve a gene therapy designed to address the genetic root cause of their disease with a single dose.

Zolgensma is approved in the US, Japan and, most recently, Brazil, for patients with SMA under the age of two. Zolgensma also continues to have a strong launch in Europe where it is approved for babies and young children with a clinical diagnosis of SMA Type 1 or SMA with up to three copies of the SMN2 gene, with dosing guidance provided up to 21 kg. More than 600 patients have benefited from Zolgensma, including through clinical trials, commercially and through the managed access program. This number is expected to continue to grow as this transformative gene therapy is approved in additional markets and as the company pursues additional studies to fully explore the impact of Zolgensma across a broad population of patients with SMA.

The company’s commitment to SMA extends beyond gene therapy to branaplam (LMI070), an oral, once-weekly RNA splicing modulator also currently under development, to expand the treatment options for SMA patients. 

About Zolgensma® (onasemnogeneabeparvovec)
Zolgensma® is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion. Zolgensma was approved by the US Food and Drug Administration in May 2019 and represents the first approved therapeutic in the company’s proprietary platform to treat rare, monogenic diseases using gene therapy. In addition to the US, Zolgensma is approved in Japan, Europe and Brazil. More than 600 patients have been treated with Zolgensma, including clinical trials, commercially and through the managed access program. Novartis Gene Therapies is pursuing registration in close to three dozen countries with regulatory decisions anticipated in Switzerland, Canada, Israel, Australia, and South Korea in late-2020 or early 2021.

About Spinal Muscular Atrophy
SMA is the leading genetic cause of infant death. If left untreated, SMA Type 1 leads to death or the need for permanent ventilation by the age of two in more than 90% of cases.
SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. It is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression. This is especially critical in SMA Type 1, where motor neuron degeneration starts before birth and escalates quickly. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities. More than 30% of patients with SMA Type 2 will die by age 25.

About Novartis Gene Therapies
Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases. Utilizing cutting-edge technology, we are turning promising gene therapies into proven treatments, beginning with our transformative gene therapy for spinal muscular atrophy (SMA). This therapy is now approved in the US, Japan, Europe and Brazil, and additional registrations are being pursued in close to three dozen countries, with regulatory decisions anticipated in Switzerland, Canada, Israel, Australia, Argentina and South Korea in late 2020 or early 2021. Our robust AAV-based pipeline is advancing treatments for Rett syndrome; a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene; and Friedreich’s ataxia. We are powered by the world’s largest gene therapy manufacturing footprint of more than one million square feet, enabling us to bring these therapies to patients around the world at quality and scale.

Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “to initiate,” “to evaluate,” “to further support,” “potential,” “recommends,” “to supplement,” “being evaluated,” “will,” “to advance,” “could,” “anticipated,” “remains,” “continues,” “to advance,” “reaffirms,” “commitment,” “to pursuing,” “to address,” “explore,” “investigational,” “launch,” “under development,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for AVXS-101 IT, Zolgensma and branaplam, or regarding potential future revenues from AVXS-101 IT, Zolgensma and branaplam. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that AVXS-101 IT or branaplam will be submitted or approved for sale in any market, or at any particular time. Neither can there be any guarantee that Zolgensma will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that AVXS-101 IT, Zolgensma or branaplam will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis
Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.

Novartis is on Twitter. Sign up to follow @Novartis at https://twitter.com/novartisnews
For Novartis multimedia content, please visit https://www.novartis.com/news/media-library
For questions about the site or required registration, please contact media.relations@novartis.com

# # #

Novartis Media Relations
E-mail: media.relations@novartis.com

Anja von Treskow
Novartis External Communications
+41 79 392 8697 (mobile)
anja.von_treskow@novartis.com



Eric Althoff
Novartis US External Communications
+1 646 438 4335
eric.althoff@novartis.com
Farah Bulsara Speer
SVP, Corporate Communications, Novartis Gene Therapies
+1 312 543 2881 (mobile)
farah.speer@novartis.com



Novartis Investor Relations
Central investor relations line: +41 61 324 7944
E-mail: investor.relations@novartis.com

CentralNorth America
Samir Shah+41 61 324 7944Sloan Simpson+1 862 778 5052
Thomas Hungerbuehler        
Isabella Zinck
+41 61 324 8425
+41 61 324 7188
To view this piece of content from www.globenewswire.com, please give your consent at the top of this page.

About GlobeNewswire by notified

GlobeNewswire by notified
GlobeNewswire by notified
One Liberty Plaza - 165 Broadway
NY 10006 New York

https://notified.com

GlobeNewswire by notified is one of the world's largest newswire distribution networks, specializing in the delivery of corporate press releases financial disclosures and multimedia content to the media, investment community, individual investors and the general public.

Subscribe to releases from GlobeNewswire by notified

Subscribe to all the latest releases from GlobeNewswire by notified by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from GlobeNewswire by notified

Golar LNG Limited - Announcement of filing of Form 20-F Annual Report28.3.2024 22:47:27 CET | Press release

Golar LNG Limited announces that it has filed its Form 20-F for the year ended December 31, 2023 with the Securities and Exchange Commission in the U.S. Form 20-F can be downloaded from the link below, is available on our website (www.golarlng.com) and shareholders may receive a hard copy free of charge upon request. March 28, 2024 The Board of Directors Hamilton, Bermuda Enquiries: Golar Management Limited: + 44 207 063 7900 Stuart Buchanan This information is subject to the disclosure requirements pursuant to Section 5-12 the Norwegian Securities Trading Act Attachment GLNG 2023 Annual Report Form 20-F

Magna Posts 2023 Annual Report28.3.2024 22:26:07 CET | Press release

AURORA, Ontario, March 28, 2024 (GLOBE NEWSWIRE) -- Magna International Inc. (TSX: MG; NYSE: MGA) today announced that its 2023 Annual Report, including Management’s Discussion and Analysis and Audited Consolidated Financial Statements, Annual Information Form (AIF) and Form 40-F, are now available on the company’s website, www.magna.com. Magna has also filed these documents with the Canadian Securities Administrators (accessible through its website at www.sedarplus.ca) and the U.S. Securities and Exchange Commission (accessible through its website at www.sec.gov/edgar). Our 2024 Annual Meeting of Shareholders will be held on Thursday, May 9, 2024, commencing at 10:00 a.m. (Eastern Daylight Time). The meeting is being conducted as a virtual-only meeting accessible at www.virtualshareholdermeeting.com/MGA2024. Magna will provide a paper copy of its audited financial statements as contained in our 2023 Annual Report to Shareholders, free of charge, on request through our website, www.mag

Fortuna files Form 40-F, Annual Report28.3.2024 22:00:00 CET | Press release

VANCOUVER, British Columbia, March 28, 2024 (GLOBE NEWSWIRE) -- Fortuna Silver Mines Inc. (NYSE: FSM) (TSX: FVI) reports that the Company has filed today its fiscal 2023 annual report on Form 40-F with the U.S. Securities and Exchange Commission (“SEC”). The Form 40-F, which includes the Company’s fiscal 2023 annual audited financial statements, management’s discussion and analysis, and annual information form, is available on the Company’s website and on the SEC´s website. Printed copies of the annual financial statements are available free of charge to Fortuna shareholders upon written request. About Fortuna Silver Mines Inc. Fortuna Silver Mines Inc. is a Canadian precious metals mining company with five operating mines in Argentina, Burkina Faso, Côte d'Ivoire, Mexico, and Peru. Sustainability is integral to all our operations and relationships. We produce gold and silver and generate shared value over the long-term for our stakeholders through efficient production, environmental p

Nokia Corporation: Repurchase of own shares on 28.03.202428.3.2024 21:30:00 CET | Press release

Nokia Corporation Stock Exchange Release 28 March 2024 at 22:30 EET Nokia Corporation: Repurchase of own shares on 28.03.2024 Espoo, Finland – On 28 March 2024 Nokia Corporation (LEI: 549300A0JPRWG1KI7U06) has acquired its own shares (ISIN FI0009000681) as follows: Trading venue (MIC Code)Number of sharesWeighted average price / share, EUR*XHEL467,1243.29CEUX--BATE--AQEU--TQEX--Total467,1243.29 * Rounded to two decimals On 25 January 2024, Nokia announced that its Board of Directors is initiating a share buyback program to return up to EUR 600 million of cash to shareholders in tranches over a period of two years. The first phase of the share buyback program in compliance with the Market Abuse Regulation (EU) 596/2014 (MAR), the Commission Delegated Regulation (EU) 2016/1052 and under the authorization granted by Nokia’s Annual General Meeting on 4 April 2023 started on 20 March 2024 and ends by 18 December 2024 with a maximum aggregate purchase price of EUR 300 million. Total cost of

Centessa Pharmaceuticals Reports Business Highlights and Financial Results for the Fourth Quarter and Full-Year 202328.3.2024 21:28:47 CET | Press release

Hemophilia Program: Ongoing registrational PRESent-2 and PRESent-3 studies of SerpinPC for the treatment of hemophilia B; PRESent-2 advancing toward interim analysis planned in 2024Orexin Agonist Program: Clinical proof-of-concept data for ORX750 in sleep-deprived healthy volunteers planned in 2024 LockBody® Technology Platform: Ongoing Phase 1/2a study of LB101 (PD-L1xCD47)for the treatment of solid tumors BOSTON and LONDON, March 28, 2024 (GLOBE NEWSWIRE) -- Centessa Pharmaceuticals plc (Nasdaq: CNTA), a clinical-stage pharmaceutical company, today reported recent business highlights and financial results for the fourth quarter and full-year ended December 31, 2023. “This is an exciting and pivotal time for Centessa,” said Saurabh Saha MD PhD, Chief Executive Officer of Centessa. “We are laser focused on executing the PRESent registrational studies for SerpinPC, a potential first-in-class subcutaneously administered therapy with a differentiated safety profile for individuals with he

HiddenA line styled icon from Orion Icon Library.Eye