Biogen Exercises Option with Ionis to Develop and Commercialize Investigational Treatment BIIB067 for a Subtype of Familial Amyotrophic Lateral Sclerosis (ALS) Based on Positive Phase 1 Data

  • Positive interim analysis demonstrated proof-of-biology and proof-of-concept for BIIB067
  • Biogen plans to advance BIIB067 to a pivotal clinical study
  • Biogen paid Ionis Pharmaceuticals a $35 million one-time upfront payment and may pay potential milestone payments and royalties

CAMBRIDGE, MA and CARLSBAD, CA, USA I December 06, 2018 I Biogen Inc (Nasdaq: BIIB) and Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that Biogen exercised its option to obtain from Ionis a worldwide, exclusive, royalty-bearing license to develop and commercialize BIIB067 (IONIS-SOD1RX), an investigational treatment for amyotrophic lateral sclerosis (ALS) with superoxide dismutase 1 (SOD1) mutations. ALS with SOD1 mutations is a subtype of familial ALS and accounts for approximately two percent of all ALS cases.

“Biogen and Ionis have a shared goal of bringing innovative therapies to those living with severe neurological diseases where there is significant unmet medical need,” said Michael Ehlers, M.D., Ph.D., executive vice president, research and development at Biogen. “The progress made to date in the BIIB067 clinical program is a positive step forward. We are committed to our goal of bringing forward a therapy for patients with ALS with SOD1 mutations, who currently have limited or no treatment options.”

The decision to exercise the option was based on a positive interim analysis of a randomized, placebo controlled single- and multiple-ascending dose Phase 1 study (n=70) that achieved proof-of-biology and proof-of-concept for BIIB067. At the highest dose tested (n=10), treatment with BIIB067 over a three month period resulted in a statistically significant lowering of SOD1 protein levels in the cerebrospinal fluid (p=0.002) and a numerical trend towards slowing of clinical decline as measured by the ALS Functional Rating Scale-Revised, both compared to placebo (n=12). The safety and tolerability profile in this analysis supports continued development of BIIB067 in ALS. Based on these results, Biogen plans to advance BIIB067 to a pivotal clinical study.

“The results from this study provide hope to SOD1 ALS patients who are suffering with this devastating disease,” said C. Frank Bennett, Ph.D., senior vice president of research and franchise leader for neurological programs at Ionis. “BIIB067 is the first investigational medicine targeting the known cause of this familial form of ALS to advance towards a pivotal study. Our broad strategic collaborations with Biogen have produced not only SPINRAZA, but six more drugs moving forward in development and a large number of preclinical and research programs. We believe our proprietary antisense technology will continue to bring benefit to patients now and in the future.”

As a part of the option exercise, Biogen made a one-time $35 million payment to Ionis. Future payments may include potential post-licensing milestone payments of up to $55 million and royalties in the low to mid-teen percentages on annual worldwide net sales. Biogen will be solely responsible for the costs and expenses related to the development, manufacturing and commercialization of BIIB067 following the option exercise.

ALS is a rare, fatal neurodegenerative disease characterized by motor neuron loss in the brain and spinal cord that are responsible for controlling voluntary muscle movement. Symptoms may vary depending on the location of the motor neuron failure and may include muscle stiffness and spasticity or spontaneous muscle twitching followed by atrophy. The average life expectancy for an ALS patient is less than five years from the time of diagnosis. Mutations within multiple genes are believed to cause ALS, including mutations in the SOD1 gene.

Final data results from the Phase 1 study are expected to be communicated at a future scientific forum.

About BIIB067
BIIB067 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. BIIB067 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. BIIB067 demonstrated proof-of-biology and proof-of-concept in a Phase 1 interim analysis. Biogen licensed BIIB067 from Ionis Pharmaceuticals under a collaborative development and license agreement.

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. 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 Alzheimer’s disease and dementia, multiple sclerosis and neuroimmunology, movement disorders, neuromuscular disorders, pain, ophthalmology, neuropsychiatry and acute neurology. Biogen also manufactures and 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.

About Ionis Pharmaceuticals, Inc.
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, proprietary antisense technology platform with the potential to treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs – such as children and adults with spinal muscular atrophy (SMA). We created SPINRAZA® (nusinersen)* and are proud to have brought new hope to the SMA community by developing the first and only approved treatment for this disease.

Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 drugs with the potential to treat patients with cardiovascular disease, rare diseases, neurological diseases, infectious diseases and cancer. We created TEGSEDI™ (inotersen) the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy of hereditary transthyretin (TTR) amyloidosis (ATTR) in adult patients that our affiliate Akcea Therapeutics is commercializing. Together with Akcea, we are also bringing new medicines to patients with cardiometabolic lipid disorders.

To learn more about Ionis follow us on twitter @ionispharma or visit http://ir.ionispharma.com/.

*SPINRAZA® is marketed by Biogen.

SOURCE: Biogen

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