Presentations at the 2018 American Academy of Neurology

Highlight the Broad Potential of Antisense Drugs for Neurological Diseases

— Special conference session focused on therapeutic potential of antisense drugs to treat neurological diseases —
— Fourteen presentations on Ionis’ antisense drugs to treat neurological diseases, including SMA, amyloidosis, Huntington’s disease, Alzheimer’s disease, and ALS —

CARLSBAD, Calif., April 17, 2018– Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, today announced that Ionis and its collaborators will present data from its neurological disease programs at the 70thAmerican Academy of Neurology (AAN) meeting in Los Angeles, California from April 21-27, 2018.

Ionis’ neurological disease programs will be highlighted in 9 presentations and 5 posters. Topics will include:

The benefits SPINRAZA® (nusinersen) provides for individuals with spinal muscular atrophy (SMA) across a broad range of disease.
The long-term efficacy and safety of inotersen in patients with hereditary ATTR.
The potential benefits of IONIS-HTTRx (RG6042) based on Phase 1/2 data in patients with Huntington’s Disease.
Demonstration of robust reductions in MAPT (tau) in the CNS of nonhuman primates with a good safety and tolerability profile for IONIS-MAPTRx, tau-lowering antisense drug, to treat patients with Alzheimer’s disease being evaluated in a Phase 2 study.
The therapeutic potential of antisense therapies against multiple targets for amyotrophic lateral sclerosis (ALS) that demonstrate rescue of disease symptoms in animal models.
Reductions and reversal of disease endpoints in animal models of Lafora disease.
In addition to the topics listed above, the conference will feature a special session entitled, ‘Neuroscience in the Clinic: Antisense Oligonucleotide (ASO) Therapy’, Monday, April 23, 3:30 p.m.-5:30 p.m. During this session, Ionis scientists, as well as scientists and external clinical investigators collaborating with Ionis, will discuss the broad potential of antisense drugs to treat neurological diseases. Dr. Frank Bennett, Ionis’ senior vice president of research and franchise leader for the neurological programs, will provide a scientific overview on antisense therapy and Dr. Morie Gertz will present safety and efficacy data on inotersen.

Stephen Hawking began his last journey to the stars

British astrophysicis Stephen Hawking one of the greatest scientists of all time. He died on Wednesday morning at the age of 76 years. The world-famous scientist suffered from the motor neurological disease Amyotrophic Lateral Sclerosis (ALS), in which the muscles gradually go down.

One of his famous lycris

”There is no heaven or afterlife for broken-down computers; that is a fairy story for people afraid of the dark”.

Our thoughts and prayers go out to family and friends