Biogen and Ionis Expand Strategic Collaboration to Develop Drug Candidates for a Broad Range of Neurological Diseases
- Transaction builds upon the productive collaboration that produced SPINRAZA® as well as two antisense drug candidates currently in the clinic, with the potential to advance up to seven more drug candidates to the clinic within the next two years
- The antisense platform developed by Ionis is an established and validated gene-based therapeutic modality, as evidenced by the success of SPINRAZA, with the potential to address numerous neurological diseases that are currently untreatable
Biogen will pay Ionis
$1 billionin cash, including an equity investment in Ionis stock and an upfront payment, representing one of the most significant discovery stage collaborations in the industry
Under the terms of the collaboration, Biogen will pay Ionis
The companies plan to advance programs for a broad range of neurological diseases for which few treatment options exist today. Disease areas include dementia, neuromuscular diseases, movement disorders, ophthalmology, diseases of the inner ear, and neuropsychiatry. Biogen will have the first choice of neurology targets on which to exclusively collaborate with Ionis. In this collaboration, Ionis will be responsible for the identification of antisense drug candidates based on selected targets, while Biogen will be responsible for and pay for non-clinical studies, clinical development, manufacturing, and commercialization.
“Biogen and Ionis share a commitment and passion to bring new
therapeutic options to those living with neurological diseases where the
unmet medical need is high and growing,” said
“There is no better partner for Ionis to develop new antisense drugs to treat neurological diseases than Biogen. Their demonstrated perseverance, dedication, and commitment to innovation in developing drugs for these challenging diseases is unmatched,” said Stanley T. Crooke, M.D., chief executive officer and chairman of Ionis. “These attributes and Biogen’s investment in neurological diseases have been important to the successes we’ve achieved already in the collaboration. Working with Biogen, we have achieved what neither company could have achieved alone. Together, we believe we can create a robust neurological disease franchise fueled by Ionis’ antisense technology and highlighted by the tremendous success of SPINRAZA. This new collaboration provides the potential opportunity to build an even stronger pipeline for Biogen and Ionis, with the expectation of bringing further benefit to patients in desperate need and value to Ionis’ shareholders. This new collaboration’s financial terms, upfront payment, milestone payments, and royalties are substantially higher compared to our previous collaborations with Biogen, which demonstrates the value we have created in applying Ionis’ antisense technology platform to neurological disease. We believe Biogen’s equity investment reflects its commitment to Ionis, to antisense technology, and to the success of this collaboration.”
“The transformational success of SPINRAZA has given us increased
confidence that antisense therapeutics have the potential to address
many diseases of the central nervous system that were previously
“Our collaboration with Biogen has provided significant value for both
companies. We have and continue to learn more about neurological
diseases and successful strategies for developing drugs for these
diseases. Biogen has worked with us to validate biomarkers and develop
new clinical trial endpoints. Ionis has helped Biogen understand our
antisense platform and the large potential of this powerful drug
modality in neurological diseases,” said Dr.
The transaction is subject to customary closing conditions, including
the expiration of the applicable waiting period under the Hart Scott
Rodino Antitrust Improvements Act of 1976 in
Biogen and Ionis expect the deal to close in the second quarter of 2018.
CONFERENCE CALL AND WEBCAST
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. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by
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Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over 40 drugs in development. SPINRAZA® (nusinersen) has been approved in global markets for the treatment of spinal muscular atrophy (SMA). Biogen is responsible for commercializing SPINRAZA. Inotersen and volanesorsen are two antisense drugs that Ionis discovered and successfully advanced through Phase 3 studies. Inotersen is under regulatory review for marketing approval in the U.S. and EU for the treatment of patients with hereditary ATTR amyloidosis. Volanesorsen is under regulatory review for marketing approval in the U.S., EU, and
SPINRAZA is being developed globally for the treatment of SMA.
SPINRAZA is an antisense oligonucleotide (ASO), using Ionis Pharmaceutical Inc.’s 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.1 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,2 where motor neurons degenerate in individuals with SMA due to insufficient levels of SMN protein.3
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 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.
For additional important safety information, and the U.S. full prescribing information, please visit www.spinraza.com or your respective country’s website.
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 and results that may be achieved through Biogen’s collaboration agreement with Ionis, risks and uncertainties associated with drug development and commercialization, the potential of Biogen’s commercial business and pipeline programs, including potential novel antisense drug candidates for a broad range of neurological disease areas, including dementia, neuromuscular diseases, movement disorders, ophthalmology, diseases of the inner ear, and neuropsychiatry, the anticipated completion and timing of the transaction, and the potential benefits, safety, and efficacy of SPINRAZA. 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. 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: risks that the transaction will be
completed in a timely manner or at all; uncertainty as to whether the
anticipated benefits and potential of Biogen’s collaboration agreement
with Ionis can be achieved; risks of unexpected costs or delays;
uncertainty of success in the development and potential
commercialization of novel antisense drug candidates for a broad range
of neurological disease areas, including dementia, neuromuscular
diseases, movement disorders, ophthalmology, diseases of the inner ear,
and neuropsychiatry, which may be impacted by, among other things, the
occurrence of adverse safety events and/or 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 these drug candidates; Biogen and Ionis
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, or 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 Biogen’s 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
IONIS’ FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding Ionis’ collaboration with Biogen and the development, activity, therapeutic potential, commercial potential and safety of SPINRAZA, and the drugs in development for neurological diseases. Any statement describing Ionis’ goals, expectations, financial or other projections, intentions, or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing, and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Ionis’ forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis’ programs are described in additional detail in Ionis’ annual report on Form 10-K for the year ended
Ionis Pharmaceuticals™ is a trademark of
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
2 Evers MM, Toonen LJ, van Roon-Mom WM. Antisense oligonucleotides in therapy for neurodegenerative disorders. Adv Drug Deliv Rev. 2015;87:90-103.
3 Lunn MR,
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