Loading, Please Wait...
CAMBRIDGE, Mass., May 01, 2019 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced it will present new data across its industry-leading neuroscience portfolio and advancing clinical development programs at the 71st annual meeting of the American Academy of Neurology (AAN) in Philadelphia, PA. (May 4–11). Of Biogen’s 33 total presentations, key data will focus on spinal muscular atrophy (SMA), multiple sclerosis (MS) and amyotrophic lateral sclerosis (ALS).
“Biogen stands at the forefront of neuroscience, taking on some of the most challenging diseases in history as part of our steadfast commitment to supporting patients with a range of complex and rare neurological disorders,” said Alfred Sandrock, Jr., M.D., Ph.D., executive vice president and chief medical officer at Biogen. “Our data at AAN illustrate our drive to advance critical research for people who have limited or no treatment options.”
Research across Biogen’s clinical and development programs demonstrates the company’s commitment to further understanding neurodegenerative diseases with unmet need. SMA and MS data focus on validating biomarkers that aid in monitoring and detecting disease progression, while pipeline data underscore the company’s emphasis on thoughtful study design and understanding the patient journey.
SMA Data Presentations Reinforce SPINRAZA as Standard of Care in Treating SMA, Underscoring Therapy’s Long-Term Benefit for Infants, Children and Adults
Advancing Biogen’s Pipeline of Investigational Treatments for Neurodegenerative Diseases
Advancing Biogen’s Portfolio of Leading MS Therapies
Highlights of Biogen’s Platform and Poster Presentations:
SPINAL MUSCULAR ATROPHY
Emerging Science (Late Breaking) Presentation
About SPINRAZA® (nusinersen)1-4
SPINRAZA is the first and only approved medicine for the treatment of spinal muscular atrophy (SMA) and is currently available in more than 40 countries. As of March 31, 2019 over 7,500 individuals with SMA are being treated with SPINRAZA worldwide, based on patients across the post-marketing setting, Expanded Access Program (EAP) and clinical trial participants.
SPINRAZA is an antisense oligonucleotide (ASO) developed using Ionis’ proprietary antisense technology that is designed to treat the root cause of SMA. SPINRAZA alters the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein. 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 been shown to increase the amount of full-length SMN protein in individuals with SMA. SPINRAZA is administered via intrathecal injection, which delivers therapies directly into the cerebrospinal fluid (CSF) around the spinal cord, where motor neurons degenerate in individuals with SMA due to insufficient levels of SMN protein.
In the clinical trial program, SPINRAZA demonstrated a favorable benefit-risk profile. The most common adverse reactions that occurred in the SPINRAZA group were 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 ASOs. Individuals may be at increased risk of bleeding complications. Renal toxicity has been observed after administration of some ASOs. SPINRAZA is present in and excreted by the kidney.
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, the largest of its kind in SMA, that moved SPINRAZA from its first dose in humans in 2011 to its first regulatory approval in five years.
About TYSABRI® (natalizumab)
TYSABRI is a well-established relapsing multiple sclerosis (RMS) treatment that has been proven in clinical trials to slow physical disability progression, reduce the formation of new brain lesions and cut relapses. TYSABRI is approved in 80 countries, and nearly 200,000 people worldwide have been treated with TYSABRI, with over 725,000 patient-years of experience, based on clinical trials and prescription data.5 In the U.S., TYSABRI is indicated as monotherapy for the treatment of patients with relapsing forms of MS. In the European Union, it is indicated as single disease modifying therapy (DMT) in adults with highly active relapsing-remitting MS (RRMS) for patients despite a full and adequate course of treatment, with at least one DMT or patients with rapidly evolving severe RRMS.
TYSABRI increases the risk of progressive multifocal leukoencephalopathy (PML), a rare opportunistic viral infection of the brain which has been associated with death or severe disability. Risk factors that increase the risk of PML are the presence of anti-JC virus antibodies, prior immunosuppressant use and longer TYSABRI treatment duration. Patients who have all three risk factors have the highest risk of developing PML.
TYSABRI also increases the risk of developing encephalitis and meningitis caused by herpes simplex and varicella zoster viruses, and serious, life-threatening and sometimes fatal cases have been reported in the post-marketing setting in MS patients receiving TYSABRI. Clinically significant liver injury, including acute liver failure requiring transplant, has also been reported in the post-marketing setting. Other serious adverse events that have occurred in TYSABRI-treated patients include hypersensitivity reactions (e.g., anaphylaxis) and infections, including opportunistic and other atypical infections.
Please click here for Important Safety Information, including Boxed Warning, and full Prescribing Information, including Medication Guide for TYSABRI in the U.S., or visit your respective country’s product website.
About Diroximel Fumarate
Diroximel fumarate (BIIB098) is a novel oral fumarate candidate in development for the treatment of relapsing forms of MS. Diroximel fumarate is designed to rapidly convert to monomethyl fumarate in the body and with a distinct chemical structure, may have the potential to offer differentiated gastrointestinal (GI) tolerability as compared to dimethyl fumarate. Alkermes is conducting the EVOLVE-MS-2 study in patients with relapsing-remitting (RRMS), a five-week, head-to-head study versus dimethyl fumarate to evaluate GI tolerability in addition to the EVOLVE-MS-1 study. Diroximel fumarate is currently under review with the U.S. Food and Drug Administration (FDA) with a PDUFA (Prescription Drug User Fee Act) target action date in the fourth quarter of 2019. Diroximel fumarate will be marketed under the brand name VUMERITY, which has been conditionally accepted by the FDA and will be confirmed upon approval.
Tofersen is an antisense oligonucleotide (ASO) RNase H-mediated inhibitor of SOD1 messenger ribonucleic acid (mRNA) being developed for the treatment of ALS with SOD1 mutations. Tofersen binds to SOD1 mRNA, allowing its degradation by RNase-H and reducing protein production. This is thought to decrease the toxicity of mutant SOD1 and potentially provide therapeutic benefit through improved survival and function to people with ALS with SOD1 mutations. Tofersen demonstrated proof-of-biology and proof-of-concept in a Phase 1 analysis. Biogen licensed tofersen from Ionis Pharmaceuticals, Inc. under a collaborative development and license agreement.
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 and only 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 – Twitter, LinkedIn, Facebook, YouTube.
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, TYSABRI, diroximel fumarate and tofersen; the results of certain real-world data; clinical trial results and plans; our research and development program for the treatment of SMA, MS and ALS and clinical studies on MS, ALS, PSP, stroke and neuropathic pain; the identification and treatment of SMA and MS; risks and uncertainties associated with drug development and commercialization; potential regulatory approval and the timing thereof; the potential of Biogen’s commercial business and pipeline programs, including SPINRAZA, TYSABRI, diroximel fumarate and tofersen; and the anticipated benefits and potential of Biogen’s collaboration arrangements with Ionis and Alkermes;. 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 or results obtained during our clinicial trials, regulatory authorities may require additional information or further studies, or may fail or refuse to approve or may delay approval of our drug candidates; regulatory submissions may take longer or be more difficult to complete than expected; uncertainty of success in the development and potential commercialization of VUMERITY and tofersen; risks relating to the potential launch of VUMERITY, including preparedness of healthcare providers to treat patients, the ability to obtain and maintain adequate reimbursement for VUMERITY and other unexpected difficulties or hurdles; 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; the risk that we may not fully enroll our clinical trials or enrollment will take longer than expected; 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.
1. 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.
2. Finkel R, Chiriboga C, Vajsar J, et al. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet. 2016;388(10063):3017-3026.
3. Evers MM, Toonen LJ, van Roon-Mom WM. Antisense oligonucleotides in therapy for neurodegenerative disorders. Adv Drug Deliv Rev. 2015;87:90-103.
4. Lunn MR, Wang CH. Spinal muscular atrophy. Lancet. 2008;371(9630):2120-2133.
5. Global Natalizumab (TYSABRI) Postmarketing PML Update, March 2019.
+1 617 679 4945
+1 781 464 2442