Full Press Release Details
Ionis highlights achievements, commercial strategy and technology advancements at Investor Day
CARLSBAD, Calif., Dec. 8, 2020 - At its Investor Day yesterday, Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS) highlighted the company's significant achievements in 2020 and outlined its strategy to realize the substantial opportunity of its pipeline.
Ionis has been preparing and prioritizing its growing wholly owned pipeline for commercialization in line with its commercial strategy. The
company's commercial priorities are three-fold: (1) Initially focusing its commercial efforts on rare diseases within its prolific neurology and cardiology franchises (2) pioneer new markets where there are no available treatments (3) create new
standards of care where there has been a lack of innovation to optimize patient care.
Delivering on these three priorities will have meaningful impact to patients, their families, and healthcare providers all while reducing the burden on healthcare systems
and driving value for all Ionis' stakeholders including patients and shareholders.
Ionis also has plans to expand opportunistically to new products in additional treatment areas such as hematology, endocrinology and pulmonology.
Ionis projects having the opportunity to launch six or more new products through 2026, with each being ready for launch in a close window, ranging from 18 to 24 months
between each new product launch. The expectation is that the implementation of this strategy will drive double-digit revenue growth and substantial earnings growth.
Brett P. Monia, Ph.D., chief executive officer at Ionis, said, "In 2020, we pursued an aggressive agenda focused on building our commercial plans and capabilities,
progressing the Ionis-owned pipeline, advancing our technology and growing our leadership position in RNA-targeted therapeutics. We are pleased to say that we delivered against all these objectives. We invested in building our commercial plans and
capabilities and began implementation. These actions were accelerated through the acquisition of our commercial affiliate Akcea. We have also progressed and substantially expanded the Ionis-owned pipeline. In addition, we have six Phase 3 trials
underway, initiated 13 Phase 2 trials, achieved multiple, positive clinical proof-of-concept readouts, and advanced new delivery platforms."
Dr. Monia continued, "For years we have been recognized for our excellence in research, early drug development and scientific innovation. We will now add to this by
building a strong and efficient commercial organization of equal excellence. All of which will provide substantial benefit to patients and shareholders for years to come."
Highlights of Investor Day
Akcea Integration Update
The recently completed acquisition of Akcea has created a stronger, more efficient company, further bolstering Ionis' financial strength. The integration of Akcea is
ahead of schedule, delivering cost synergies and efficiencies. It was also announced that Akcea is going to commercialize TEGSEDI and WAYLIVRA in Europe through a distribution agreement with Swedish Orphan Biovitrum AB ("Sobi"), an international
biopharmaceutical company that focuses on rare diseases. Under the terms of this agreement, Akcea retains the marketing authorization ("MAH") for both medicines in Europe. Additionally, Akcea will continue to maintain limited European operations
including regulatory, manufacturing, and the management of relationships with key opinion leaders. Akcea will continue to lead the TEGSEDI and WAYLIVRA global commercial strategy. The agreement provides Ionis the flexibility to reinvest resources
to support its other commercial plans.
Oral Delivery Development Update
Ionis and AstraZeneca are committed to bringing the best possible PCSK9 antisense treatments to patients and have been collaborating on both the subcutaneous and oral
formulations. The subcutaneous formulation of ION449 has a potential best-in-class profile and is advancing rapidly toward Phase 3 development.
Preclinical and early clinical data give Ionis and AstraZeneca confidence that they can achieve effective oral delivery of ION449 and other ASOs. Based on ongoing
research and experience to date, both companies believe they can improve upon the current oral formulation. Therefore, Ionis and AstraZeneca have decided to terminate the Phase 1 PCSK9 oral study. Ionis and AstraZeneca will continue to broadly work
together to further optimize the oral delivery of ASOs, including ION449.
Ionis is expanding its oral delivery to include medicines from its pipeline and has increased its internal investment in oral delivery research. The company plans on
initiating one or more programs from its pipeline within the next year or two. Candidates for consideration include IONIS-TTR-LRx, IONIS-PKK-LRx, ION994 (AGT), and ION547. Success would further enhance the commercial value of
Ionis-owned programs.
Additional details are available in the replay of the webcast. It is available here for a
About Ionis Pharmaceuticals, Inc.
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that
can 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. We created the first and only approved
treatment for children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the
patients we have yet to reach with a pipeline of more than 40 novel medicines designed to potentially treat a broad range of diseases, including neurological, cardio-renal, metabolic, infectious, and pulmonary diseases.
Forward-looking Statements
This presentation includes forward-looking statements regarding our business, financial guidance and the therapeutic and commercial potential of SPINRAZA (nusinersen),
TEGSEDI (inotersen), WAYLIVRA (volanesorsen) and Ionis' technologies and products in development, including the business of Akcea Therapeutics, Inc., Ionis' wholly owned subsidiary. 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, including those related to the impact COVID-19 could have on
our business, and including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and
effective for use as human therapeutics, and in the endeavor of building a business around such medicines. 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 December 31, 2019
and our most recent Form 10-Q quarterly filing, which are on file with the SEC. Copies of this and other documents are available at www.ionispharma.com. In this presentation, unless the context requires otherwise, "Ionis," "Company," "we," "our,"
and "us" refers to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics is a registered trademark of Akcea Therapeutics, Inc. TEGSEDI is a trademark of Akcea
Therapeutics, Inc. WAYLIVRA is a registered trademark of Akcea Therapeutics, Inc. SPINRAZA is a registered trademark of Biogen.
Ionis Pharmaceuticals Investor Contact:
D. Wade Walke, Ph.D.
Vice President, Investor Relations
Ionis Pharmaceuticals Media Contact:
Vice President, Corporate Communications