By creating a better way for drug discovery and development, Ionis is now realizing the full potential of its antisense technology.
With two treatments approved in the last two years and at least four more headed into late-stage clinical trials by the end of 2019, Ionis Pharmaceutical’s novel therapies are treating the untreatable and bringing hope to sick people around the world. The company is focused on using its antisense technology to produce breakthrough therapies to treat a range of diseases that have not before been effectively tackled.
Before Ionis created SPINRAZA®*, and it became the standard of care in the treatment of spinal muscular atrophy (SMA), children born with the condition lived with progressive degeneration and had a drastically shortened life expectancy. Today, babies born with the disease, who are treated pre-symptomatically, are developing similarly to healthy babies. Ionis went on to develop TEGSEDI™, the world’s first RNA-targeting subcutaneous injection to treat polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adults. A degenerative and fatal disease that drastically impacts people’s ability to carry out their basic activities of daily living, hATTR has severely limited the lives of those living with the disease. TEGSEDI significantly reduces levels of the disease-causing protein, offering effective treatment with a simple injection that gives patients the freedom to treat where and when they choose.
This is exactly what Stanley T. Crooke, M.D., Ph.D., Ionis Founder, Chairman and CEO, envisioned when he launched the company in 1989. Dr. Crooke knew that with the right science, the right people, and an unwavering commitment to a better way of discovering and developing drugs, Ionis could deliver truly innovative treatments to patients where no others have proven effective or, in some cases, ever existed.
“We wake up every day thinking about the sick people who depend on us. There is a tremendous sense of gratification that comes from knowing the time spent in the lab can change lives in such a significant way. And this is only the beginning. With potentially 10 programs entering pivotal trials by 2020, the pace of innovation at Ionis is only increasing,” said Dr. Crooke.
Ionis Leading in Innovative RNA-Targeted Therapeutics
This passion is driving unprecedented productivity in the Ionis pipeline that includes 45 potential first-in-class or best-in-class therapies for a broad range of diseases. All of this is being accomplished by a team of scientists who are committed to fully realizing the potential of antisense technology and delivering transformative medicines to those who need them today and long into the future.
“From the rarest of diseases like Prion and Amyotrophic Lateral Sclerosis (ALS) to those that impact millions of people like cardiovascular disease, we never turn down a treatment challenge. If the need is great and we believe our technology is the way forward, we will say yes and work to find new therapies,” said Dr. Crooke.
As the leader in the discovery and development of RNA-targeted therapeutics, Ionis has evolved its novel antisense technology. Antisense medicines interact precisely with specific sequences of RNA that disrupt the disease process and change its course. The next wave of Ionis-developed treatments will continue to address intractable rare diseases, as well as diseases impacting millions of people.
In November, Ionis and its commercial affiliate Akcea announced Phase 2 data evaluating AKCEA-APO(a)-LRx. The trial was the largest study ever conducted in patients with cardiovascular disease (CVD) and elevated lipoprotein(a), also known as Lp(a), high levels of which triple the risk of having a heart attack or stroke at an early age. Results from the study showed that AKCEA-APO(a)-LRx significantly lowered levels of Lp(a) in patients with pre-existing cardiovascular disease.
“Because Lp(a) cannot be easily targeted with small molecules or antibodies, inhibition of apolipoprotein(a) is a great example of how antisense technology can have an impact where no other therapeutic approaches have proven effective. A drug that reduces Lp(a) could be a paradigm shift for the cardiovascular community and the 8 to 10 million treatable patients estimated to be living with cardiovascular disease and elevated levels of Lp(a),” said Brett Monia, Ph.D., Ionis’ chief operating officer.
Also heading into a pivotal Phase 3 trial in 2019 is IONIS-HTTRx (RG6042), the first potentially disease-modifying therapy to be discovered and developed to treat Huntington’s Disease. Huntington’s Disease is a fatal, inherited brain disorder that results in the progressive loss of both mental faculties and physical control. Currently available treatments only mask symptoms but do not slow the underlying progression of the disease. Patients treated with IONIS-HTTRx saw reduced levels of the disease-causing huntingtin protein and for the first time in history, Huntington’s Disease patients have new hope for a drug that treats the underlying cause of the disease.
“We’re on the precipice of finding effective therapies in so many areas. We are constantly working to broaden the utility of antisense technology and fully capitalize on the breadth of its therapeutic potential to bring hope to patients and value to shareholders. This coupled with our innovation-centered business has created a highly efficient, highly productive drug discovery and development engine that will continue to bring transformative therapies to the world for years to come,” said Dr. Crooke.
For more information, visit Ionispharma.com
*SPINRAZA® is marketed by Biogen.