CARLSBAD, Calif., Nov. 4, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.  
(Nasdaq: IONS) today reported its financial results for the third quarter of  
2020 and recent business highlights. 
 
   "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(r) (nusinersen) royalties of $74  
million, in line with the prior quarter 
   *   Product sales from TEGSEDI(r) (inotersen) and WAYLIVRA(r) (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, -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 
 
   Revenue 
 
   Ionis' revenue was comprised of the following (amounts in millions): 
 
                                    Three months ended,  Nine months ended, 
 
                                    September 30,        September 30, 
 
                                    2020      2019       2020      2019 
 
Revenue: 
 
     Commercial revenue: 
 
SPINRAZA royalties                  $74       $82        $212      $212 
 
Product sales, net                  19        12         51        29 
 
Licensing and royalty revenue       2         2          6         11 
 
Total commercial revenue            95        96         269       252 
 
R&D Revenue: 
 
Amortization from upfront payments  19        23         68        100 
 
Milestone payments                  44        12         73        64 
 
License fees                        -         26         15        198 
 
Other services                      2         11         14        15 
 
Total R&D revenue                   65        72         170       377 
 
Total revenue                       $160      $168       $439      $629 
 
   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. 
 
   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. 
 
   To learn more about Ionis visit www.ionispharma.com  or follow us on twitter  
@ionispharma. 
 
   Ionis' Forward-looking Statement 
 
   This press release includes forward-looking statements regarding Ionis'  
business, financial guidance and the therapeutic and commercial potential of  
SPINRAZA (nusinersen), TEGSEDI (inotersen) and 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