GlobeNewswire

Biogen Announces New Data Further Establishing SPINRAZA® (nusinersen) as a Foundation of Care in Spinal Muscular Atrophy for a Broad Range of Patients

Share
  • New results from the NURTURE study demonstrate that pre-symptomatic infants treated with SPINRAZA are achieving motor milestones that are unprecedented in the natural history of the disease, including 100 percent of children sitting without support and 88 percent walking independently
  • Results add to the body of evidence supporting the safety and long-term efficacy of SPINRAZA and demonstrate that treatment results in continuous improvement in attaining motor milestones
  • SPINRAZA is the only treatment approved for infants, children and adults with evidence across a broad range of patients and real-world experience treating more than 7,500 patients

CAMBRIDGE, Mass., July 01, 2019 (GLOBE NEWSWIRE) -- Biogen (Nasdaq: BIIB) today announced new results from the NURTURE study, adding data to the longest study of spinal muscular atrophy (SMA) in pre-symptomatic infants (n=25). These data reported, after up to 45.1 months of analysis, continue to demonstrate efficacy and safety in patients treated pre-symptomatically with SPINRAZA in comparison to the natural history of this disease. These new data also showed that patients treated with SPINRAZA had continuous improvement with the overwhelming majority of patients achieving motor milestones in a normal timeframe.

These data are being presented at the Cure SMA Annual SMA Conference in Anaheim, CA (June 28-July 1, 2019) and the 5th Congress of the European Academy of Neurology (EAN) in Oslo, Norway (June 29-July 2, 2019).

View the multimedia release here: https://www.multivu.com/players/English/8560251-biogen-nurture-study-spinraza-spinal-muscular-atrophy-treatment-data/

“These study results demonstrate the durable impact of pre-symptomatic, proactive treatment on transforming the natural course of this disease. We are seeing an extensive number of patients continually meeting child motor development milestones and making unprecedented gains in a previously hopeless and often fatal condition,” said Darryl De Vivo, M.D., Sidney Carter Professor of Neurology and Pediatrics, Columbia University Irving Medical Center in New York, New York. “SPINRAZA is setting patients on a path toward survival, greater mobility and independence, which is helping improve outcomes for patients of all ages.”

Results from NURTURE, an ongoing, Phase 2, open-label study of 25 pre-symptomatic patients with SMA (most likely to develop SMA Type 1 or 2) who received their first dose of SPINRAZA before six weeks old, demonstrated unparalleled results in comparison to the natural history of SMA. As of March 2019:

  • 100 percent were alive without a need for permanent ventilation.
  • The median age of the study participants was nearly three years old. The majority of untreated patients with SMA Type 1 never reach their second birthday without permanent ventilation.
  • 100 percent of the infants were sitting independently, in comparison to the natural history of this disease where no patients with SMA Type 1 would be able to do so and patients with SMA Type 2 would need assistance.
  • 88 percent of the infants were walking independently with many of them doing so in the normal timeframe for a toddler. In the natural history of SMA, patients with SMA Type 1 or Type 2 are never able to walk independently.
  • Patients were approaching the maximum mean score of 64 on the CHOP INTEND measure of motor function– 63.4 for patients with 3 SMN 2 copies (n=10) and 62.1 for those with 2 SMN 2 copies (n=15), demonstrating the powerful impact of early SPINRAZA treatment.
  • SPINRAZA demonstrated longer term efficacy up to nearly 4 years, with participants continuing to make progress and showing no signs of loss of motor function. 
  • SPINRAZA was well-tolerated with no new safety concerns identified after up to nearly 4 years of treatment.

“A few years ago, SMA patients had no treatment options and faced significant care challenges,” said Kenneth Hobby, President of Cure SMA, a patient advocacy organization dedicated to the treatment and cure of SMA. “However, the future of SMA has changed and especially with early treatment patients now have a very strong chance to reach age appropriate developmental milestones. These new data demonstrate the dramatic impact where children are now walking independently at four years of age, when the usual lifespan would be under two if untreated. This study provides additional evidence on the maintenance of these improvements. It’s critical that research in SMA continues to support the generation of real-world evidence in patients of all ages so that we better understand the long-term implications of SMA and treatment across all types.”

Additional presentations at the two meetings highlight results from the ongoing open-label SHINE extension study of children with infantile and later-onset SMA as well the CS2/CS12 analysis of older patients. Biogen also continues to explore the scientific value of phosphorylated neurofilament heavy chain (pNF-H) and will present new data on the ongoing evaluation of its potential as a biomarker in SMA.

About SPINRAZA® (nusinersen)1-4
SPINRAZA is the first therapy approved to treat infants, children and adults with spinal muscular atrophy (SMA) and is available in more than 40 countries. As of March 31, 2019, more than 7,500 individuals have been treated with SPINRAZA for up to six years, based on patients across the post-marketing setting, Expanded Access Program (EAP) and clinical trial participants. SPINRAZA is the only SMA treatment to combine unsurpassed real-world experience and the highest level of clinical evidence across a broad spectrum of patient populations.

SMA is a rare, genetic, neuromuscular disease that is characterized by a loss of motor neurons in the spinal cord and lower brain stem, resulting in severe, progressive muscle atrophy and weakness. Approximately one in 10,000 live births have a diagnosis of SMA and people of all ages are impacted by the disease. It is a leading genetic cause of infant mortality.

SPINRAZA, a foundation of care in SMA, is an antisense oligonucleotide (ASO) designed to target a root cause of SMA by increasing the amount of full-length survival motor neuron (SMN) protein, which is critical to maintaining motor neurons. It is administered by intrathecal injection into the fluid surrounding the spinal cord where motor neurons reside to deliver the treatment where the disease starts.

SPINRAZA currently maintains the largest clinical data set in SMA based on data from over 300 patients across a broad range of SMA populations demonstrating a favorable benefit:risk profile. SPINRAZA was evaluated in two randomized, double-blind, sham-controlled studies (ENDEAR and CHERISH) in infantile and later-onset SMA patients and supported by open label studies in pre-symptomatic infants (NURTURE) and individuals who were treated into adulthood with later-onset SMA (CS2/CS12). The most common adverse events observed were respiratory infection, fever, constipation, headache, vomiting and back pain. Serious infections of hydrocephalus and meningitis have been observed in the post-marketing setting. Renal toxicity and coagulation abnormalities, including acute severe low platelet counts, have been observed after administration of some ASOs. Laboratory tests can monitor for these signs.

Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), a leader in antisense therapeutics. Biogen and Ionis conducted an innovative clinical development program that moved SPINRAZA from its first dose in humans in 2011 to its first regulatory approval in five years.

About Biogen
At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp, and today has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy and is focused on advancing neuroscience research programs in MS and neuroimmunology, Alzheimer’s disease and dementia, movement disorders, neuromuscular disorders, acute neurology, neurocognitive disorders, pain and ophthalmology. Biogen also commercializes biosimilars of advanced biologics.

We routinely post information that may be important to investors on our website at www.biogen.com. To learn more, please visit www.biogen.com and follow us on social media – TwitterLinkedInFacebookYouTube.

Biogen Safe Harbor 
This news release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, about the potential benefits, safety and efficacy of SPINRAZA; the results of certain real-world data; and the identification and treatment of SMA. These statements may be identified by words such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “plan,” “possible,” “potential,” “will,” “would” and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements or the scientific data presented.

These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation the occurrence of adverse safety events and/or unexpected concerns that may arise from additional data or analysis; failure to obtain regulatory approvals in other jurisdictions; risks of unexpected costs or delays; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; product liability claims; and third party collaboration risks. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this news release. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.

Reference:

  1. Finkel, Richard S., et al. “Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.” New England Journal of Medicine, vol. 377, no. 18, 2017, pp. 1723–1732., doi:10.1056/nejmoa1702752.
  2. Lefebvre S, Burglen L, Reboullet S, et al. Identification and characterization of a spinal muscular atrophy-determining gene. Cell.1995;80(1):155-165.
  3. Mercuri, Eugenio, et al. “Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy.” New England Journal of Medicine, vol. 378, no. 7, 2018, pp. 625–635., doi:10.1056/nejmoa1710504.
  4. Basil T. Darras, et al. “Nusinersen in later-onset spinal muscular atrophy.” Neurology, May 2019, 92 (21) e2492-e2506; DOI:10.1212/WNL.0000000000007527


MEDIA CONTACT:

David Caouette
+ 617 679 4945
public.affairs@biogen.com
INVESTOR CONTACT:

Joe Mara
+1 781 464 2442
IR@biogen.com

About GlobeNewswire

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

https://globenewswire.com

GlobeNewswire 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

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

Latest releases from GlobeNewswire

EVS Broadcast Equipment reports update of share buyback program22.7.2019 17:45:00 CESTPress release

Publication on July 22, 2019, 5.45pm CET Regulated information – reporting share buyback EVS Broadcast Equipment S.A.: Euronext Brussels (EVS.BR), Bloomberg (EVS BB), Reuters (EVSB.BR) EVS reports update of share buyback program EVS Broadcast Equipment reports that the following transactions, conducted within the framework of the share buyback program announced on October 24, 2018, took place between July 15, 2019 and July 17, 2019. Date Number of shares acquired Average price (EUR) Total (EUR) 15/07/2019 1,856 20.3713 37,809 16/07/2019 1,760 20.0932 35,364 17/07/2019 378 20.9000 7,900 As of July 22, 2019, and since the start of the buyback program, EVS has bought 255,028 shares at an average price of EUR 20.8174, representing in total EUR 5,309,024. After aforementioned transactions the total number of own shares amounts now to 333,676 shares as of July 22, 2019 (including 93,144 shares already held by the company before the start of the share buyback program and taking into account t

Change in Amer Sports Corporation’s financial reporting schedule22.7.2019 17:15:00 CESTPress release

Amer Sports Corporation STOCK EXCHANGE RELEASE July 22, 2019 at 6:15 p.m. Change in Amer Sports Corporation’s financial reporting schedule Amer Sports changes the publication date of its half-year financial report 2019. The updated schedule for the half-year report is 30 September, 2019 at approximately 1:00 p.m. Finnish time. The previously announced publication date was July 25, 2019. The change is related to the recent ownership change in the company. ANTA Sports Products Limited consolidates Amer Sports as an associated company in its half-year financial report which will be published on 26 August, 2019. On the same day, Amer Sports will publish a stock exchange release disclosing its financial information included in ANTA Sports’ half-year financial report. On March 28, 2019, Mascot Bidco Oy completed a voluntary recommended public cash tender offer for all the issued and outstanding shares in Amer Sports Corporation and on April 1, 2019 announced that it will exercise its redempt

Changes in Huhtamaki’s Global Executive team22.7.2019 17:00:00 CESTPress release

HUHTAMÄKI OYJ STOCK EXCHANGE RELEASE 22.7.2019 AT 18:00 Changes in Huhtamaki’s Global Executive team Michael Orye, Executive Vice President, Fiber Packaging and a member of Huhtamaki Global Executive Team has decided to leave Huhtamaki to pursue other career opportunities. Eric Le Lay, Executive Vice President Foodservice EAO will assume the leadership and take full responsibility of Fiber Packaging in addition to his current role. This change is effective as of 22.7.2019. “I want to thank Michael for his contribution to Huhtamaki and wish him all success in his future endeavors” says Charles Héaulmé, President and CEO of Huhtamaki. Following the aforementioned change, the members of the Global Executive Team are: Charles Héaulmé (chairman), President and CEO Thomas Geust, Chief Financial Officer; Leena Lie, Senior Vice President, Marketing and Communications; Sami Pauni, Senior Vice President, Corporate Affairs and Legal, Group General Counsel; Teija Sarajärvi, Senior Vice President,

Metso establishes a new business services center in Vilnius22.7.2019 14:00:00 CESTPress release

Metso establishes a new business services center in Vilnius Metso Corporation’s press release on July 22, 2019 at 15:00 EEST Metso has decided to establish a new Metso Business Services (MBS) center in Vilnius, the capital of Lithuania. The investment supports the company’s profitable growth strategy by improving operational excellence and scalability of business services. The Vilnius MBS center will initially offer services for Metso’s finance operations, later followed by logistics. The objective is to centralize the related services to the new center and thus gain benefits from process harmonization, standardization and digitalization. Initially, the center will employ some tens of experts, but the number of personnel is expected to grow in the coming years. As part of the initiative, employee negotiations have been held at Metso Minerals in Tampere, Finland, to review the possible employee arrangements and implications. Metso has decided to transfer its Financial Services operation

Radisson Hospitality AB’s Q2 Financial Report & Webcast on 26 July 201922.7.2019 12:00:00 CESTPress release

Radisson Hospitality AB (publ) will release its Financial Report Q2-2019 on Friday 26 July 2019 at 7:30am CEST. Federico J. González-Tejera, President & CEO, and Sergio Amodeo, CFO, will also host an audio webcast on the same day, at 10:00am CEST. To access the telephone conference, please dial one of the following numbers: LOCATION PHONE NUMBER Belgium National free phone 0800 48740 Belgium +32 (0)2 400 9874 France National free phone 0805 103 028 France +33 (0)1 76 70 07 94 Norway National free phone 800 51874 Norway +47 2396 0264 Sweden National free phone 0200 125 581 Sweden +46 (0)8 5069 2180 Spain National free phone 800 098826 Spain +34 914 146 280 United Kingdom National free phone 0800 376 7922 United Kingdom +44 (0)844 571 8892 USA National free phone 1 866 966 1396 USA +1 631 510 7495 Standard international dial-in +44 (0)207 192 8000 Confirmation Code: 4689049 To follow the webcast, please visit: https://www.radissonhospitalityab.com/investors/financial-information For furt