On November 4, 2020 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported its financial results for the third quarter of 2020 and recent business highlights (Press release, Ionis Pharmaceuticals, NOV 4, 2020, View Source [SID1234569909]).
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"We took an important step forward in our evolution when we acquired Akcea. This transaction supports our commercial strategy, further enabling us to maximize the value of our Ionis-owned pipeline. As one company, we believe we are stronger and more efficient, with an enhanced ability to achieve even greater future success," said Brett P. Monia, Ph.D., chief executive officer at Ionis. "We made significant progress across our pipeline this year. Recently, we advanced inhaled delivery with IONIS-ENAC-2.5Rx, positioning us to bring new treatment options to patients with pulmonary diseases. We also initiated mid-stage studies for vupanorsen in cardiovascular disease patients and ION541, our medicine to treat nearly all forms of ALS. Additionally, our five Phase 3 studies continue to progress, with our sixth expected to begin by the end of this year. We believe our achievements this year move us closer to delivering 10 or more marketing applications through 2025. Our goal is to bring these medicines to millions of patients around the world."
Third Quarter 2020 Financial Results and Highlights
On track to achieve financial guidance of being meaningfully profitable this year
Net income of $5 million on a non-GAAP basis and a net loss of $31 million on a GAAP basis for the third quarter
Achieved quarter over quarter revenue growth
Commercial revenue from SPINRAZA (nusinersen) royalties of $74 million, in line with the prior quarter
Product sales from TEGSEDI (inotersen) and WAYLIVRA (volanesorsen) increased more than 15 percent
R&D revenue increased more than 15 percent from advancing several partnered programs
Maintained a strong balance sheet with cash of $2.3 billion
Estimated pro forma cash following the Akcea acquisition of $1.8 billion
"Our acquisition of Akcea further strengthens our business and financial position in numerous ways. We now retain more value from Akcea’s rich pipeline and commercial products. We are also able to use Akcea’s current cash and future cash flows to advance Ionis’ strategic priorities. And beginning next year, we expect to realize meaningful cost synergies as we continue to integrate the two companies," said Elizabeth L. Hougen, chief financial officer of Ionis. "Looking ahead, we are maintaining our 2020 financial guidance driven by the significant revenue and earnings growth we expect in the fourth quarter. We have already earned revenue from multiple sources this quarter, including $75 million from Pfizer for advancing vupanorsen. Importantly, we remain well capitalized with the financial resources to achieve our strategic goals."
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
SPINRAZA: a global foundation-of-care for the treatment of spinal muscular atrophy (SMA) patients of all ages
$495 million in worldwide sales in the third quarter
More than 11,000 patients were on SPINRAZA treatment worldwide at the end of the third quarter, including patients across commercial, expanded access and clinical trial settings
The open-label safety cohort of the DEVOTE study of higher-dose SPINRAZA is fully enrolled and the pivotal randomized treatment cohort will begin enrolling patients next
The Phase 4 RESPOND study in patients with a suboptimal clinical response to gene therapy is expected to begin early next year
TEGSEDI: the only approved at-home subcutaneous therapy for the treatment of hereditary transthyretin amyloidosis (hATTR) with polyneuropathy in adult patients
Commercially available in 15 countries
Secured pricing and reimbursement in multiple new EU markets and in Canada in the largest provinces and with multiple private payers
Won 2020 Prix Galien USA Award for the Best Biotechnology Product
WAYLIVRA: the only approved treatment in the EU for adults with genetically confirmed familial chylomicronemia syndrome (FCS) at high risk for pancreatitis
Commercially available in 4 countries
Finalized pricing negotiations in additional EU markets, including in the UK
Third Quarter 2020 and Recent Pipeline Highlights
Positive Phase 2 vupanorsen and AKCEA-APOCIII-LRx results presented at the European Society of Cardiology annual meeting
Advanced multiple programs into key mid-stage studies
Vupanorsen advanced into Phase 2b development with the initiation of the TRANSLATE-TIMI 70 dose-ranging study in statin-treated patients with dyslipidemia, resulting in a $75 million payment from Pfizer
IONIS-FXI-LRx advanced into Phase 2b development in patients with end-stage renal disease
IONIS-HBVRx advanced into Phase 2b development in patients with hepatitis B virus infection
Advanced inhaled delivery
Positive IONIS-ENAC-2.5Rx healthy volunteer results provided support for inhaled antisense medicine delivery
Dosing completed in the IONIS-ENAC-2.5Rx Phase 2 study in patients with cystic fibrosis
Advanced the IONIS-PKK-LRx program
Proof-of-concept data from the PKK development program in patients with hereditary angioedema were reported in the New England Journal of Medicine
Enrollment completed in the IONIS-PKK-LRx Phase 2 study in patients with hereditary angioedema
IONIS-PKK-LRx advanced into an investigator-initiated study in hospitalized COVID-19 patients in Brazil
Progressed multiple neurological disease medicines under Ionis’ broad collaboration with Biogen, earning more than $50 million
ION541 advanced into Phase 1/2 development in patients with nearly all forms of ALS
ION464 advanced into Phase 1/2 development in patients with multiple system atrophy
IONIS-MAPTRx continued to advance in a long-term extension study in patients with Alzheimer’s disease
The U.S. FDA granted orphan drug designation to Ionis-owned medicines for people with Alexander disease, β-thalassemia and Lafora diseases
Upcoming Catalysts
Report clinical data, potentially enabling key programs to advance towards the market:
Subcutaneous and orally delivered ION449 targeting PCSK9 at the American Heart Association Scientific Sessions 2020
IONIS-AGT-LRx Phase 2 studies in patients with hypertension
IONIS-ENAC-2.5Rx Phase 2 study in patients with cystic fibrosis
IONIS-GHR-LRx Phase 2 study in patients with acromegaly
IONIS-PKK-LRx Phase 2 study in patients with hereditary angioedema
Advance the pipeline with numerous study initiations:
AKCEA-APOCIII-LRx Phase 3 study in patients with FCS
ION363 registrational study in patients with FUS-ALS
IONIS-ENAC-2.5Rx Phase 2 study in patients with chronic obstructive pulmonary disease
Phase 1/2 studies of Ionis-owned medicines:
ION251 for patients with multiple myeloma
ION373 for patients with Alexander disease
ION716 for patients with Prion diseases
Expand TEGSEDI and WAYLIVRA commercial availability in the EU and Latin America
Refile the WAYLIVRA application for marketing authorization in the U.S. next year
Operating Expenses
Ionis’ operating expenses for the third quarter of 2020 increased compared to the same period in 2019 driven by the Company’s investments in 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
Prior to completing its acquisition of Akcea in October 2020, Ionis owned approximately 76 percent of Akcea. 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. In October 2020, after the acquisition of Akcea closed, Ionis no longer recognizes any noncontrolling interest in Akcea on its statement of operations.
Net Income (Loss) Attributable to Ionis Common Stockholders
Ionis recognized a net loss attributable to Ionis’ common stockholders for the third quarter of 2020 compared to net income in the same period in 2019 primarily due to higher revenue in 2019, including a $150 million license fee Ionis earned from Novartis. Additionally, Ionis’ operating expenses increased in 2020 compared to the same period last year as described above.
Balance Sheet
Ionis ended September 2020 with cash, cash equivalents and short-term investments of more than $2.3 billion, compared to $2.5 billion at December 31, 2019. In October 2020, Ionis used approximately $545 million of its cash for the Akcea acquisition.
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.