Business Wire

Biogen’s SPINRAZA® (nusinersen) Data Show Earlier Treatment Initiation May Lead to Improved Motor Function Across a Broad Population of People Living with Spinal Muscular Atrophy

Del

Biogen (NASDAQ: BIIB) presented new data demonstrating that earlier initiation of treatment with SPINRAZA® (nusinersen) may improve motor function outcomes in infants and children with spinal muscular atrophy (SMA). Results continued to reinforce the favorable efficacy and safety profile of SPINRAZA. The data were shared at the 22nd International Annual Congress of the World Muscle Society in Saint Malo, France (October 3-7, 2017).

A new analysis from the Phase 3 ENDEAR study showed infants with SMA who initiated treatment earlier in the disease (shorter disease duration) demonstrated greater benefit and improvement in motor function outcomes.

As measured by the Hammersmith Infant Neurological Examination (HINE), significant differences in motor milestone responders were observed between infants treated with SPINRAZA compared to untreated infants with disease duration less than or equal to 12 weeks (75% vs. 0%; P<.0001) and those with disease duration greater than 12 weeks (32% vs. 0%; P=.0026). There was also a significant benefit in event-free survival in infants treated with SPINRAZA with disease duration less than or equal to 12 weeks (P=.0004).

“These studies contribute to a growing body of evidence that SPINRAZA can make a meaningful difference in the lives of people with SMA regardless of their age or stage of the disease,” said Alfred Sandrock, M.D., Ph.D., executive vice president and chief medical officer at Biogen. “Across studies, we continue to see evidence that earlier initiation of treatment with SPINRAZA can lead to improved clinical and functional outcomes.”

Interim analyses were also presented from the Phase 2 EMBRACE study which was designed to assess the efficacy and safety of SPINRAZA in individuals with infantile- and later-onset SMA who were ineligible for the two earlier pivotal studies.

The EMBRACE interim analysis showed a larger proportion of infants and children treated with SPINRAZA were HINE motor milestone responders compared to those who were untreated. Results from the interim analysis also supported the dosing regimen of four loading doses in the first two months, followed by the administration of SPINRAZA every four months thereafter, for individuals with infantile- and later-onset SMA.

In the ENDEAR and EMBRACE studies SPINRAZA demonstrated a favorable benefit-risk profile. Safety data involving the intrathecal administration of SPINRAZA showed the incidence and nature of the most common lumbar puncture-related adverse events in the clinical studies were similar in children with later-onset SMA with or without scoliosis.

For more information about SPINRAZA and prescribing information in the United States, please visit www.SPINRAZA.com. Prescribing information in the European Union is available at http://www.ema.europa.eu/ema/.

About ENDEAR and EMBRACE
ENDEAR is a randomized, double-blind, sham-procedure controlled 13-month study in patients with infantile-onset SMA. The end of study efficacy analysis included all patients (n=121) who had their final study visit after the interim analysis (n=78) and had the opportunity to attend the six-month study visit assessment. The Hammersmith Infant Neurological Examination (HINE) is a reliable and clinically validated tool to assess motor milestone achievement in infants with SMA.

EMBRACE is a Phase 2, multicenter, randomized, double-blind, sham-procedure controlled 14-month study of SPINRAZA in infants and children not eligible to participate in ENDEAR (symptom onset less than or equal to six months, less than or equal to seven months of age at screening; 2 SMN2 copies) or CHERISH (symptom onset age greater than six months, age 2-12 years at screening).

SPINRAZA Program Status
SPINRAZA is the first approved medicine for the treatment of SMA and is currently approved in the United States, the European Union, Brazil, Japan and Canada. Biogen has submitted regulatory filings in additional countries and plans to initiate additional filings in other countries.

Globally, in 2016, in response to the urgent need for treatment for the most severely affected individuals living with SMA, Biogen sponsored one of the largest, pre-approval Expanded Access Programs (EAP) in rare disease, free of charge.

Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals (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 SMA 1-5
Spinal muscular atrophy (SMA) is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness. Ultimately, individuals with the most severe type of SMA can become paralyzed and have difficulty performing the basic functions of life, like breathing and swallowing.

Due to a loss of, or defect in, the SMN1 gene, people with SMA do not produce enough SMN protein, which is critical for the maintenance of motor neurons. The severity of SMA correlates with the amount of SMN protein. People with Type 1 SMA, the form that requires the most intensive and supportive care, produce very little SMN protein and do not achieve the ability to sit without support or live beyond two years without respiratory support. People with Type 2 and Type 3 SMA produce greater amounts of SMN protein and have less severe, but still life-altering forms of SMA.

About SPINRAZA ® (nusinersen)
SPINRAZA is being developed globally for the treatment of SMA.

SPINRAZA is an antisense oligonucleotide (ASO), using Ionis Pharmaceuticals’ proprietary antisense technology, that is designed to treat SMA caused by mutations or deletions in the SMN1 gene located in chromosome 5q that leads to SMN protein deficiency. SPINRAZA alters the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein.6 ASOs are short synthetic strings of nucleotides designed to selectively bind to target RNA and regulate gene expression. Through use of this technology, SPINRAZA has the potential to increase the amount of full-length SMN protein in individuals with SMA.

SPINRAZA must be administered via intrathecal injection, which delivers therapies directly to the cerebrospinal fluid (CSF) around the spinal cord,7 where motor neurons degenerate in individuals with SMA due to insufficient levels of survival motor neuron (SMN) protein.8

SPINRAZA demonstrated a favorable benefit-risk profile. The most common adverse reactions reported for SPINRAZA were upper respiratory infection, lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients. Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Individuals may be at increased risk of bleeding complications. Renal toxicity has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney.

About Biogen
Through cutting-edge science and medicine, Biogen discovers, develops and delivers innovative therapies worldwide for people living with serious neurological and neurodegenerative diseases. Founded in 1978, Biogen is a pioneer in biotechnology and today the Company has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first and only approved treatment for spinal muscular atrophy and is at the forefront of neurology research for conditions including Alzheimer’s disease, Parkinson’s disease and amyotrophic lateral sclerosis. Biogen also manufactures and commercializes biosimilars of advanced biologics. For more information, please visit www.biogen.com. Follow us on social media – Twitter, LinkedIn, Facebook, YouTube.

Biogen Safe Harbor
This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 relating to the potential benefits, safety and efficacy of SPINRAZA, the results of certain real-world data, the status of current regulatory filings, plans for additional regulatory filings in other jurisdictions, planning and timing for commercial launch, and availability of patient access and reimbursement pathways, which may vary on a country-by-country basis. These forward-looking statements may be accompanied by words such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “except,” “forecast,” “intend,” “may,” “plan,” “potential,” “possible,” “will” and other words and terms of similar meaning. You should not place undue reliance on these statements or the scientific data presented. Drug development and commercialization involve a high degree of risk. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation uncertainty of success in commercialization of SPINRAZA, which may be impacted by, among other things, the level of preparedness of healthcare providers to treat patients, difficulties in obtaining or changes in the availability of reimbursement for SPINRAZA, the effectiveness of sales and marketing efforts, problems with the manufacturing process for SPINRAZA, the occurrence of adverse safety events, unexpected concerns that may arise from additional data or analysis; regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of Biogen’s drug candidates or expansion of product labeling; or Biogen may encounter other unexpected hurdles which may be impacted by, among other things, the occurrence of adverse safety events, failure to obtain regulatory approvals in certain jurisdictions, failure to obtain regulatory approvals in other jurisdictions, failure to protect intellectual property and other proprietary rights; product liability claims; or 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 Biogen’s most recent annual or quarterly report and in other reports Biogen has filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this press 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.

1. Darras B, Markowitz J, Monani U, De Vivo D. Chapter 8 - Spinal Muscular Atrophies. In: Vivo BTD, ed. Neuromuscular Disorders of Infancy, Childhood, and Adolescence (Second Edition). San Diego: Academic Press; 2015:117-145.

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. Mailman MD, Heinz JW, Papp AC, et al. Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2. Genet Med. 2002;4(1):20-26.

4. Monani UR, Lorson CL, Parsons DW, et al. A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2. Hum Mol Genet. 1999;8(7):1177-1183.

5. Peeters K, Chamova T, Jordanova A. Clinical and genetic diversity of SMN1-negative proximal spinal muscular atrophies. Brain.2014;137(Pt 11):2879-2896.

6. Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF, Krainer AR. Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes Dev. 2010 Aug 1; 24(15):16344-44.

7. Evers MM, Toonen LJ, van Roon-Mom WM. Antisense oligonucleotides in therapy for neurodegenerative disorders. Adv Drug Deliv Rev. 2015;87:90-103.

8. Lunn MR, Wang CH. Spinal muscular atrophy. Lancet. 2008;371(9630):2120-2133.

Contact information

Biogen
MEDIA CONTACT:
Hailee Tellier, +1-617-914-6778
public.affairs@biogen.com
or
INVESTOR CONTACT:
Ben Strain, +1-781-464-2442
IR@biogen.com

Om Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(c) 2018 Business Wire, Inc., All rights reserved.

Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.

Følg saker fra Business Wire

Registrer deg med din epostadresse under for å få de nyeste sakene fra Business Wire på epost fortløpende. Du kan melde deg av når som helst.

Siste saker fra Business Wire

"Panasonic Prime Smash!" Has Been Given a Complete Makeover - An iPhone, iPad Game That Awakens Children's Interest in Mathematics26.9.2018 03:05Pressemelding

On August 28, 2018, Panasonic Corporation gave the iPad app, "Panasonic Prime Smash!" a complete makeover. The new release includes the iOS 11 compatible iPhone app as well. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180925006294/en/ The screen of "Panasonic Prime Smash!": In the game, balls with numbers are blown. If it is a prime number, capture it by tapping the ball. (Graphic: Business Wire) [Video] Smartphone game application "Panasonic Prime Smash!" https://channel.panasonic.com/contents/24401/ "Panasonic Prime Smash!" overview "Panasonic Prime Smash!" is a game app that teaches children about the atom of numbers, or prime numbers in a fun, enjoyable way. Balls bearing numbers will appear on the screen one after another. If the number is a prime number, you touch that number. If the number is not a prime number, you "Smash" it, transform it into a prime number, and then touch it. That number will be added to your t

Toshiba's New Global Brand Identity to Promote Growth and Development26.9.2018 02:01Pressemelding

Toshiba Corporation (TOKYO:6502) has today unveiled a new global brand identity the “Essence of Toshiba,” a restatement of its abiding purpose and values. The Essence of Toshiba replaces Toshiba’s previous Group Management Vision, Brand Statement and Brand Tagline. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180925006288/en/ (Graphic: Business Wire) The Essence of Toshiba will be applied across all Toshiba Group companies and in all markets, as part of an initiative to harmonize and integrate the Group’s diverse businesses and operations around the world. As a forward-looking and entrepreneurial Toshiba continues its business transformation, the restated corporate philosophy and new brand identity will provide a strong and consistent framework for all communication. A summation of core management values and the DNA that has shaped the company for 143 years, the Essence of Toshiba will guide Toshiba in building a sustainab

Moody’s Names Mona Breed as Chief Information Officer25.9.2018 20:30Pressemelding

Moody’s Corporation (NYSE:MCO) announced today that Mona Breed will join the company as Chief Information Officer (CIO), effective October 15, 2018. As CIO, Ms. Breed will lead Moody’s global technology infrastructure and programs. She will report to Raymond McDaniel, President and Chief Executive Officer of Moody’s. “With nearly 30 years of experience embedding innovation and technology programs at leading financial and professional services firms, Mona’s insight and expertise will be invaluable as we enhance our technology capabilities and leverage our investments,” said Raymond McDaniel President and Chief Executive Officer of Moody’s. Prior to joining Moody’s, Ms. Breed led IT infrastructure for Oppenheimer Funds, where she drove innovation and transformation, including the firm’s cloud computing, unified communication and collaboration, data center operations and workplace technologies. She has also held senior technology roles at PricewaterhouseCoopers and Apollo. Ms. Breed succe

IWBI Announces First WELL Portfolio Participants25.9.2018 18:00Pressemelding

The International WELL Building Institute™ (IWBI™) announced today the first participants in WELL Portfolio™, a new streamlined pathway for WELL Certification™ for multiple new or existing buildings and tenant spaces in a single portfolio. Being released as part of the WELL v2™ pilot, the latest iteration of the WELL Building Standard™ (WELL™), it is part of a suite of enhancements to WELL that addresses growing demand across the building industry for a program that helps owners, developers and tenants streamline and scale health and wellness upgrades to their real estate assets. The announcement quickly follows the launch of WELL v2, the second iteration of IWBI’s pioneering WELL Building Standard. The addition of an early phase review process enabling projects to seek the WELL D&O™ designation, introduced in WELL v2, allows portfolio projects to move through the certification process in a more streamlined and systematic fashion across their asset base, with a specific focus on polici

Mobile Industry Accelerating Delivery of Sustainable Development Goals, According to Latest GSMA Report25.9.2018 17:00Pressemelding

The global mobile industry is demonstrating significant and measurable impact in contributing to the UN’s Sustainable Development Goals (SDGs) and is strengthening its commitment to improving lives around the world, according to a new GSMA study. The 2018 edition of the GSMA’s annual ‘Mobile Industry Impact’ report, published at the UN General Assembly this week, found that the industry has continued to increase its contribution against all 17 Goals since mobile become the first sector in the world to commit to delivery of the SDGs in 2016. This growing impact reflects mobile’s unprecedented global scale, which provides a platform to connect unconnected communities, reduce poverty, improve access to healthcare and education, and drive sustainable economic growth. “More than two-thirds of the people on the planet are now connected to a mobile network and, for many, mobile is the primary – sometimes only – channel for accessing the internet and life-enhancing services,” commented Mats Gr

PMI: Taking Bold Action for Global Change: a Conversation on Tobacco Today and Transformation for Tomorrow25.9.2018 15:43Pressemelding

During the 2018 Concordia Annual Summit, taking place in September 24 & 25 in New York, André Calantzopoulos, CEO of Philip Morris International, delivered a speech highlighting the need to have a dialogue between the industry, leaders, policy-makers, scientists, medical, and public health professionals in order to provide the 1.1 billion people who smoke with better alternatives to cigarettes. The Concordia Annual Summit, which coincides with the United Nations General Assembly (UNGA) meeting, convenes the world’s most prominent business, government, and nonprofit leaders to foster dialogue and enable effective partnerships for positive social impact. The remarks by André Calantzopoulos follow: “I'm the Chief Executive Officer of a tobacco company. I’m here today because, as global leaders come together in New York for the United Nations General Assembly, this is the time and place to discuss today's problems and their potential solutions. “Smoking tobacco is a behavioral risk factor