The ALS Association

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Carrie Munk
The ALS Association
(571) 319-3047


Isis Pharmaceuticals Announces Initiation of Phase 1/2 Clinical Study of ISIS-SOD1Rx in Patients with ALS

Washington, D.C. (December 11, 2015) — Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today announced that its partner, Biogen, has initiated a Phase 1/2 clinical study of ISIS-SOD1Rx (BIIB067) in patients with amyotrophic lateral sclerosis (ALS). ISIS-SOD1Rx is part of Isis’ strategic collaboration with Biogen to discover and develop antisense drugs to treat neurological diseases. The ALS Association initiated funding for the development of this revolutionary technology in 2003 when the vision for the approach was first established, and has supported its development through continued funding for over a decade.

ISIS-SOD1Rx, is a Gen. 2.0+ antisense drug designed to reduce the production of superoxide dismutase 1 (SOD1). A mutation in the SOD-1 gene results in an inherited form of ALS, referred to as SOD1-ALS. SOD1-ALS is the second most common familial form of ALS, accounting for up to 20 percent of familial ALS. Familial ALS represents approximately 10 percent of all cases of ALS.

“It is extremely exciting to see The ALS Association’s early investment in the development of antisense technology to treat ALS moving forward into the clinic,” said Lucie Bruijn, Ph.D., M.B.A. chief scientist for The ALS Association. “One of the core strategies of our research program is to link academic science closer with industry to promote and de-risk these therapeutic approaches to help facilitate the development of potential treatments for people living with this disease. We are also enthusiastic about the opportunity this technology has brought for not only ALS but other neurodegenerative diseases. Antisense technology would not have been a reality without the vision of Don Cleveland, Ph.D., Richard Smith, M.D., Frank Bennett, M.D., along with the commitment of clinician scientists, Merit Cudkowicz, M.D. and Tim Miller, M.D.

“We are very pleased to advance a new antisense drug into the clinic for the treatment of familial ALS with SOD1 mutation. Our technology provides us with a unique approach to specifically reduce the production of the SOD1 protein, which has the potential of providing therapeutic benefit to patients with SOD1-ALS by slowing or even halting progression of this fatal disease,” said C. Frank Bennett, Ph.D., senior vice president of research at Isis Pharmaceuticals. “We are grateful to The ALS Association for providing some of the initial funding which supported the initial concept of targeting ALS with antisense technology that lead us to this moment.”

“Together with Biogen, we have made significant progress developing antisense drugs to treat neurological and neuromuscular disorders. The advancement of ISIS-SOD1Rx into clinical development is an important milestone for this collaboration and reflects Biogen’s and our commitment to developing drugs for patients with ALS. The advancement of this program is also another example of the broad applicability of our technology to address diseases that are unapproachable by other therapeutic modalities,” said B. Lynne Parshall, chief operating officer of Isis Pharmaceuticals.

ALS is a rare, fatal neurodegenerative disorder. Patients with ALS suffer progressive degeneration of the motor neurons, which results in a declining quality of life and ultimately death. ISIS-SOD1Rx, which targets the best understood genetic cause of familial ALS, is the first antisense drug to treat ALS to enter clinical development under the strategic collaboration between Isis and Biogen.

Biogen is conducting the randomized, placebo-controlled, dose escalation Phase 1/2 clinical study that will evaluate the safety and activity of ISIS-SOD1Rx in patients with ALS, including patients with SOD1-ALS. In this study, ISIS-SOD1Rx will be administered intrathecally as an injection directly into the cerebral spinal fluid. Intrathecal administration of antisense drugs has been shown to be well tolerated in multiple clinical studies. “Biogen is committed to understanding the causes of ALS and developing therapies that may make a difference for patients and their families,” said Donald R. Johns, M.D., vice president, ALS at Biogen. “With our collaborators at ISIS Pharmaceuticals, we are excited to enter the clinic with a new approach and to gain potential insights into how targeting a genetic cause of the disease may change the course of ALS.”

For further study information, please visit and search for ISIS-SOD1Rx

About The ALS Association
The ALS Association is the only national non-profit organization fighting Lou Gehrig’s Disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit our website at

Isis and Biogen have a broad strategic alliance focused on leveraging antisense technology to advance the treatment of neurological and neuromuscular disorders. This alliance combines Isis’ expertise in antisense technology to evaluate potential neurological targets and discover antisense drugs with Biogen’s capability to develop therapies for neurological disorders. Isis is primarily responsible for drug discovery and early development of antisense therapies. Biogen has the option to license each antisense program at a particular stage in development. Current development-stage programs include antisense drugs to treat patients with spinal muscular atrophy (SMA), nusinersen; myotonic dystrophy type 1 (DM1), ISIS-DMPK-2.5Rx; amyotrophic lateral sclerosis (ALS), ISIS-SOD1Rx, and an undisclosed neurodegenerative disease, ISIS- BIIB4Rx. In addition to these four drugs, Isis and Biogen have numerous opportunities to evaluate additional targets for the development of drugs to treat neurological disorders.

Isis 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, Isis has created a large pipeline of first-in-class or best-in-class drugs, with over a dozen drugs in mid- to late-stage development. Drugs currently in Phase 3 development include volanesorsen, a drug Isis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with familial chylomicronemia syndrome and familial partial lipodystrophy; ISIS-TTRRx, a drug Isis is developing with GSK to treat patients with all forms of TTR amyloidosis; and nusinersen, a drug Isis is developing with Biogen to treat infants and children with spinal muscular atrophy. Isis’ patents provide strong and extensive protection for its drugs and technology. Additional information about Isis is available at

This press release includes forward-looking statements regarding Isis' alliance with Biogen, the discovery, development, activity, therapeutic and commercial potential and safety of ISIS- SOD1Rx for the treatment of amyotrophic lateral sclerosis. Any statement describing Isis’ 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. Isis’ 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 Isis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Isis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Isis’ programs are described in additional detail in Isis’ annual report on Form 10-K for the year ended December 31, 2014, and its most recent quarterly report on Form 10-Q, which are on file with the SEC. Copies of these and other documents are available from the Company.

In this press release, unless the context requires otherwise, “Isis,” “Company,” “we,” “our,” and “us” refers to Isis Pharmaceuticals and its subsidiaries.
Isis Pharmaceuticals® is a registered trademark of Isis Pharmaceuticals, Inc. Akcea TherapeuticsTM is a trademark of Isis Pharmaceuticals, Inc.

Isis Pharmaceuticals’ Contacts:
D. Wade Walke, Ph.D.

Vice President, Corporate Communications and Investor Relations

Amy Williford, Ph.D.

Associate Director, Corporate Communications

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