Ionis highlights achievements, commercial strategy and technology advancements at Investor Day
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.
Highlights of Investor Day
- Ionis estimates the total market opportunity for the indications targeted by its pipeline is well in excess of
$15 billion, with a significant portion from its wholly owned medicines.
- Ionis' cardiovascular franchise includes many potential first-in-class and/or best-in-class medicines targeting a full spectrum of cardiovascular disease risk factors. The company is positioned to potentially launch multiple Ionis-owned cardiovascular medicines through 2026, including:
- AKCEA-APOCIII-LRx: One product with the potential for addressing multiple indications targeting elevated triglycerides and the opportunity to set a new standard of care for triglyceride management
- 91% of patients achieved normal serum triglycerides levels with favorable safety and tolerability in Phase 2
- As announced on
Dec. 1, 2020, a Phase 3 study in patients with familial chylomicronemia syndrome (FCS) is now underway
- Evaluating additional indications with plans to initiate an additional Phase 3 study in 2021
- AKCEA-TTR-LRx: Opportunity to significantly expand ATTR franchise
- Robust target reductions of more than 90% and favorable safety and tolerability demonstrated in Phase 1
- Flexibility of at-home monthly self-administration
- Two Phase 3 studies underway – CARDIO-TTRransform for patients with hereditary or wild type TTR cardiomyopathy and NEURO-TTRansform for patients with hereditary TTR polyneuropathy.
- IONIS-AGT-LRx: Large unmet need in patients with treatment-resistant hypertension (RHTN)
- Two Phase 2 clinical studies: Patients with mild HTN and patients with uncontrolled HTN who are on two (65%) or three (35%) antihypertensive medications
- Positive Phase 2 study in patients in uncontrolled HTN: patients achieved mean reductions of 12 mmHg and 6 mmHg in systolic and diastolic blood pressure from their own baseline, respectively, after eight weeks of once-weekly 80 mg IONIS-AGT-LRx
- IONIS-AGT-LRx has demonstrated a favorable safety and tolerability profile in clinical trials to date
- More detailed results to be presented at an upcoming medical conference
- Ionis' neurology franchise has the potential to establish the standard of care for millions of patients and generate substantial value as it advances its first-in-class medicines to the market. The neurological disease market is a nascent market poised for substantial growth. Ionis believes it can be the catalyst for this growth as it is positioned to launch multiple Ionis-owned medicines through 2026, including:
- ION363: First medicine in development to specifically target FUS-ALS, a rare, rapidly progressing form of ALS
- Pivotal study on track for initiation in 2021
- Potential for a rapid path to the market
- ION716: Potential to be first approved treatment for prion diseases
- Designed to reduce production of prion protein, root cause of prion disease
- Pursuing pre-symptomatic (genetic carriers) and symptomatic (genetic and sporadic) indications
- Pivotal study planned for 2021, design should provide a rapid path to market
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
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 limited time.
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.
To learn more about Ionis visit www.ionispharma.com or follow us on twitter @ionispharma.
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
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Ionis Pharmaceuticals Investor Contact: D. Wade Walke, Ph.D., Vice President, Investor Relations, 760-603-2741; Ionis Pharmaceuticals Media Contact: Roslyn Patterson, Vice President, Corporate Communications, 760-603-2681