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Ionis reports fourth quarter and full year 2020 financial results and recent business achievements

Published: 2021-02-24 12:00:00 ET
<<<  go to IONS company page

Achieved 2020 financial guidance

2021 guidance reflects increased investments in wholly owned pipeline to drive future revenue growth

Webcast today, February 24, 2021, at 9:00 a.m. Eastern Time

CARLSBAD, Calif., Feb. 24, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today reported its financial results for the fourth quarter of 2020 and recent business highlights.

(PRNewsfoto/Ionis Pharmaceuticals, Inc.)

"Last year, we laid out a bold new vision for the Company and took important steps towards our goal of becoming one of the most successful biotechnology companies. Key to our vision is our strategy to maximize the value of our pipeline by commercializing our wholly owned medicines. Our acquisition of Akcea was an important step in building our commercial capabilities while enabling us to further strengthen our organization," said Brett P. Monia, Ph.D., chief executive officer at Ionis. "Last year, we also advanced our late-stage pipeline and expanded the utility of our technology. Looking ahead, we expect data from multiple wholly owned programs in the first half of this year, followed by Phase 3 tofersen data in patients with SOD1-ALS in the second half. These key upcoming catalysts, together with our recent pipeline and technology achievements, position us well to have 12 or more products on the market in 2026. Importantly, we continue to have the financial strength to expand investment in our wholly owned pipeline and commercial capabilities to drive meaningful and increasing value for patients and shareholders."

2020 Summary Financial Results

  • Achieved 2020 financial guidance
    • $729 million in total revenues, with half from marketed products
    • $640 million of operating expenses on a non-GAAP basis(1) and $901 million on a GAAP basis, reflecting investments in Ionis' wholly owned pipeline
    • Net income of $111 million on a non-GAAP basis(1) and a net loss of $451 million on a GAAP basis
  • Strong balance sheet with cash of $1.9 billion at year-end, enabling increasing investment in advancing the pipeline and technology while also preparing to commercialize the Company's wholly owned medicines

2020 Marketed Products Highlights 

  • SPINRAZA: a global foundation-of-care for the treatment of spinal muscular atrophy (SMA) patients of all ages
    • $2 billion in worldwide sales in 2020   
    • More than 11,000 patients worldwide were on therapy at the end of the fourth quarter across post-marketing, expanded access and clinical trial settings
    • Enrollment began in the RESPOND study evaluating potential SPINRAZA benefit in SMA patients with a suboptimal clinical response to gene therapy
    • Enrollment began in the pivotal randomized treatment cohort of the DEVOTE study evaluating higher doses of SPINRAZA
  • TEGSEDI and WAYLIVRA: transformational medicines approved for the treatment of patients with severe rare diseases
    • Product sales increased more than 65 percent in 2020, compared to 2019
    • Generated growing revenues as major markets launched in 2020
    • Restructured European operations through a distribution agreement with Swedish Orphan Biovitrum AB (Sobi)

Q4 2020 and Recent Pipeline Highlights

  • Phase 3 Pipeline: growing pipeline positioned for 12 or more products on the market in 2026
    • Advanced IONIS-APOCIII-LRx into the Phase 3 BALANCE study in patients with FCS
    • Completed enrollment in the tofersen Phase 3 VALOR study in patients with SOD1-ALS
  • Mid-stage Pipeline: broad and advancing pipeline of potential first-in-class and/or best-in-class medicines
    • Advanced and expanded the IONIS-AGT-LRx development program
      • Reported IONIS-AGT-LRx positive topline Phase 2 results in patients with hypertension uncontrolled with two or three antihypertensive medications
      • Advanced ION904, the follow-on medicine targeting AGT, into Phase 1 development in healthy volunteers
    • Advanced vupanorsen 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
    • Advanced ION449 (AZD8233), targeting PCSK9, into Phase 2b development in patients with dyslipidemia and AstraZeneca licensed ION455, a new investigational medicine for the treatment of nonalcoholic steatohepatitis (NASH), resulting in $50 million from AstraZeneca
    • Unlocked potential new pulmonary disease franchise with positive IONIS-ENAC-2.5Rx data
      • Reported positive healthy volunteer results supporting aerosol antisense medicine delivery to the lung
      • Completed dosing in the Phase 2 study in patients with cystic fibrosis
      • Advanced IONIS-ENAC-2.5Rx into Phase 2 development in patients with chronic obstructive pulmonary disease (COPD)
    • Highlighted IONIS-MAPTRx (BIIB080) Phase 1/2 study in patients with Alzheimer's disease in which IONIS-MAPTRx was generally well tolerated and demonstrated durable, time and dose-dependent reductions in CSF tau protein

Upcoming 2021 Pipeline Catalysts 

Anticipated 2021 Data Readouts(2)

Program

Phase

Anticipated Indication

H1

H2

IONIS-ENAC-2.5Rx

2

Cystic fibrosis

•

IONIS-PKK-LRx

2

Hereditary angioedema (top-line)

•

IONIS-GHR-LRx

2

Acromegaly (top-line)

•

IONIS-AGT-LRx

2

Hypertension

•

Tominersen

OLE/NHS

Huntington's disease

•

IONIS-MAPTRx

1/2

Alzheimer's disease

•

Vupanorsen

2b

sHTG/CVD risk reduction

•

IONIS-C9Rx

1/2

C9-ALS

•

Tofersen

3 (VALOR study)

SOD1-ALS

•

Anticipated 2021 Study Initiations(2)

Program

Phase

Anticipated Indication

H1

H2

ION363

3

FUS-ALS

•

ION373

2

Alexander disease

•

IONIS-AGT-LRx

2b

Resistant hypertension

•

IONIS-AGT-LRx

2

Heart failure with reduced ejection fraction

•

ION224

2b

NASH

•

Tofersen

3 (ATLAS study)

Presymptomatic SOD1-ALS

•

IONIS-APOCIII-LRx

3

Second CV indication

•

IONIS-ENAC-2.5Rx

2

Cystic fibrosis not amenable to CFTR modulators

•

(2) Timing of partnered program catalysts based on partners' most recent publicly available disclosures

2020 Financial Results and 2021 Financial Guidance

"We achieved our 2020 financial guidance, even in the challenging COVID-19 pandemic environment. Moreover, in 2020 we made significant progress toward our goal of creating a stronger, more efficient company focused primarily on advancing our wholly owned medicines to the market. We acquired Akcea enabling us to retain full value from its rich portfolio. We also restructured our European operations. Together, these transactions unlocked substantial cost savings that we plan to reinvest to drive future revenue growth," said Elizabeth L. Hougen, chief financial officer of Ionis. "Our 2021 guidance reflects our new strategy to maximize the value of our wholly owned pipeline, focused primarily on commercializing our rare neurological and cardiometabolic disease programs. Our guidance also reflects the investments we are making in three key areas of our business - advancing and expanding our wholly owned pipeline, building commercial capabilities in support of our rich pipeline and broadening the reach of our technology. We can increase our investments in these areas while only modestly increasing our expenses because of the significant cost savings we realized from acquiring Akcea and restructuring our European operations. Importantly, with nearly $2 billion of cash at the end of last year, we remain well capitalized with the substantial financial resources to achieve our goals."

2021 Financial Guidance

Ionis' full year 2021 financial guidance consists of the following components (on a non-GAAP basis)(1):

Guidance

Revenue

>$600 million

Operating Expenses (1)

$675 million to $725 million

Net Loss (1)