On August 5, 2020 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported its financial results for the second quarter of 2020 and recent business highlights (Press release, Ionis Pharmaceuticals, AUG 5, 2020, View Source [SID1234562896]).
"The first half of this year was marked by numerous important achievements. Our Phase 3 programs progressed, we advanced our cardio-renal, metabolic and neurological disease franchises and added new medicines to our Ionis-owned pipeline. Together, these catalysts moved us closer to delivering 10 or more marketing applications through 2025, which we expect to result in a number of new commercial medicines. In the second half of this year, we are expanding the reach of our technology in neurological and pulmonary diseases, as studies in sporadic ALS and COPD get underway. We also look forward to proof-of-concept data from additional mid-stage programs," said Brett P. Monia, Ph.D., chief executive officer at Ionis. "Our achievements, together with our significant financial resources, position us to realize my vision for Ionis – to lead in the delivery of transformational medicines for patients around the world."
Second Quarter 2020 Financial Results and Highlights
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On track to achieve financial guidance of being meaningfully profitable this year
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Net income of $8 million on a non-GAAP basis and a net loss of $32 million on a GAAP basis
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Achieved quarter over quarter growth in commercial and R&D revenues
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Commercial revenue from SPINRAZA (nusinersen) royalties increased to $72 million
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Product sales from TEGSEDI (inotersen) and WAYLIVRA (volanesorsen) increased to $16 million
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R&D revenue increased to more than $55 million, including $26 million from Ionis’ neurological disease franchise, $13 million from the oncology franchise and more than $10 million from the cardio-renal franchise
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Cash position of more than $2.3 billion provides substantial financial resources to continue executing on strategic goals
"We ended the second quarter with net income on a non-GAAP basis, an increase compared to our first quarter results. Looking ahead, we are maintaining our 2020 financial guidance and expect revenue and earnings growth in the second half of this year. With our substantial resources, we are well positioned to continue executing on our ambitious agenda and to deliver increasing value near-term and into the future," said Elizabeth L. Hougen, chief financial officer of Ionis.
All non-GAAP amounts referred to in this press release exclude non-cash compensation expense related to equity awards. Please refer to the reconciliation of non-GAAP and GAAP measures, which is provided later in this release.
Commercial Medicine Highlights
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SPINRAZA: a global foundation-of-care for the treatment of spinal muscular atrophy (SMA) patients of all ages
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$495 million in worldwide sales in the second quarter of this year
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More than 11,000 patients were on SPINRAZA treatment worldwide at the end of the second quarter, including patients across commercial, expanded access and clinical trial settings
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The Phase 4 RESPOND study to evaluate SPINRAZA benefit in patients with a suboptimal clinical response to Zolgensma (onasemnogene abeparvovec) is expected to begin early next year
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The DEVOTE study evaluating a higher dose of SPINRAZA with the potential to deliver even greater efficacy in SMA patients of all ages is progressing
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New clinical data from the NURTURE and SHINE studies, as well as new real-world data, further support SPINRAZA’s durable efficacy and established safety profile across SMA patients of all ages
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TEGSEDI: the only approved at-home subcutaneous therapy for the treatment of hereditary transthyretin amyloidosis (hATTR) with polyneuropathy in adult patients
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Commercially available in 15 countries
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Reimbursement approved in Portugal, Spain, Italy and Austria
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Expanding commercial availability in additional EU countries and in Latin America this year
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WAYLIVRA: the only approved treatment in the EU for adults with genetically confirmed familial chylomicronemia syndrome (FCS) at high risk for pancreatitis
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Launch progressing in Germany, Austria, Greece and through the ATU in France; launching in additional EU countries this year
Filed for marketing approval in Brazil; refiling new drug application for U.S. marketing authorization
Second Quarter 2020 and Recent Pipeline Highlights
Completed enrollment in the global GENERATION HD1 Phase 3 study of tominersen in patients with Huntington’s disease
Progressed multiple neurological disease medicines under Ionis’ broad Biogen collaboration
Published data from the Phase 1/2 study of tofersen in the New England Journal of Medicine
Progressed the IONIS-MAPTRx long-term extension study in patients with Alzheimer’s disease and achieved a $12 million milestone payment
Advanced ION464 into a Phase 1/2 study in patients with multiple system atrophy and achieved an $18 million milestone payment
Advanced medicines for the treatment of cancer and immune-mediated GI disease
Licensed ION736 to AstraZeneca for the treatment of cancer and achieved a $13 million license fee
Initiated a Phase 1 study of ION253 for the treatment of immune-mediated GI disease and achieved a $5 million milestone payment from Janssen
Expanded the Ionis-owned pipeline with the addition of ION363 for the treatment of FUS-ALS
Upcoming Catalysts
Report clinical proof-of-concept results for four or more programs
Present positive Phase 2 results from vupanorsen and AKCEA-APOCIII-LRx
Initiate a Phase 3 study of AKCEA-APOCIII-LRx in patients with FCS
Initiate a registration study of ION363 in patients with FUS-ALS
Initiate a Phase 1/2 study of ION541 in patients with sporadic ALS
Initiate a Phase 2 study of IONIS-ENaC-2.5Rx in patients with chronic obstructive pulmonary disease (COPD)
Initiate a Phase 2 study of IONIS-FXI-LRx in patients with clotting disorders
Initiate a Phase 2 study of IONIS-HBVRx in patients with hepatitis B virus infection
Advance additional new medicines into development
Operating Expenses
Ionis’ operating expenses for the second quarter of 2020 increased compared to the same period in 2019 driven by its investments in advancing its strategic priorities, including advancing the Phase 3 program for AKCEA-TTR-LRx and other medicines in its Ionis-owned pipeline.
Net Loss Attributable to Noncontrolling Interest in Akcea
At June 30, 2020, Ionis owned approximately 76 percent of Akcea. The Company’s net loss attributable to noncontrolling interest in Akcea for the second quarter of 2020 was consistent with the same period last year. The line titled "Net loss attributable to noncontrolling interest in Akcea" on Ionis’ statement of operations reflects the portion of Akcea’s net income or loss attributable to the other owners of Akcea’s common stock.
Net Income (Loss) Attributable to Ionis Common Stockholders
Ionis’ net loss attributable to Ionis’ common stockholders for the second quarter of 2020 was larger compared to the same period in 2019 primarily due to higher revenue and lower operating expenses in the same period last year.
Balance Sheet
Ionis ended June 2020 with cash, cash equivalents and short-term investments of more than $2.3 billion, compared to $2.5 billion at December 31, 2019.
Webcast
Today, at 11:30 a.m. Eastern Time, Ionis will conduct a live webcast to discuss this earnings release and related activities. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address.