Press Releases
Ionis' 2016 Financial Results Outperform Financial Guidance
"2016 was a year of significant accomplishments for Ionis, culminating in the U.S. approval of SPINRAZA, in record time and with a very broad label. The approval of SPINRAZA is a testament to the efficacy, safety and tolerability that SPINRAZA demonstrated in multiple clinical studies in multiple SMA patient populations. We are pleased with Biogen's early launch efforts in the U.S. and their actions directed to making SPINRAZA available to patients around the world as soon as possible," said
"In 2016, we reported positive clinical data from 11 studies with six drugs. We also continued to advance over 36 drugs in development, including our Phase 3 drugs, volanesorsen and IONIS-TTRRx, which will complete pivotal trials shortly. We also added five new drugs to our pipeline, including our first Generation 2.5 LICA drug and our first oral locally acting drug for gastrointestinal autoimmune diseases.
"In the first week of 2017, we and our subsidiary, Akcea, initiated a collaboration with
"In 2017, we expect to be breakeven or profitable at the operating line on a pro forma basis, driven in part by revenue from SPINRAZA. Biogen anticipates EU approval mid-year and has filed for regulatory authorization in
"We believe we have the key elements in place to achieve sustained, long-term financial growth. We have multiple drivers of revenue; a partnership strategy that leverages partner resources; a mature, broad and rapidly advancing clinical pipeline; and an innovative, more efficient drug discovery platform that enables us to continue developing new drugs with the potential for significant commercial opportunity in both rare and more prevalent diseases. We are now closer to achieving our goal of becoming a profitable, multi-product company delivering innovative medicines to patients with serious diseases," concluded Ms. Parshall.
Ionis' 2017 goals and a list of corporate and drug development highlights can be found at the end of this press release prior to the financial tables.
Financial Results
"2016 was marked by continued progress, highlighted by the approval of SPINRAZA. We earned substantial license fees and milestone payments from our many achievements, which led us to significantly improve upon our revenue, pro forma net operating loss and cash guidance for 2016. In 2016, we generated
"We are pleased to add commercial revenue from SPINRAZA to our already significant revenue from partnerships. Over the past five years, we have consistently increased our revenue, reflecting the successes of our partnered programs and drugs. This R&D revenue provides a base of revenue that funds most of our pro forma operating expenses. While in any year the specific sources of our R&D revenue change, the consistent growth in revenue we have achieved over the last five years supports the sustainability of this component of our operating model. In 2017, we expect to continue to have a strong R&D revenue base that funds most of our pro forma operating expenses. To that revenue base, we are adding commercial revenue from SPINRAZA royalties. This revenue is nearly all profit to us, in other words we have only a nominal amount of corresponding expense associated with it. For 2017, we expect our operating expenses to be essentially flat compared to 2016; however, the composition of our expenses will change to reflect the evolution of our business. We plan to continue to increase our commercial spending for volanesorsen as our subsidiary, Akcea, prepares to launch volanesorsen globally in 2018. These increases will be offset by a decrease in our R&D expenses, reflecting the fact that our current Phase 3 programs are coming to a close. Because of the efficiency of our technology, we can advance our earlier stage drugs and add new drugs to our pipeline while still decreasing our research and development expenses. The combination of a solid base of R&D revenue, SPINRAZA commercial revenue and prudent spending, support our projection that we will be breakeven or profitable at the operating line on a pro forma basis for 2017. As we have more visibility on SPINRAZA sales, we will provide more detailed guidance. Already in 2017, we have generated more than
All pro forma amounts referred to in this press release exclude non-cash compensation expense related to equity awards. Please refer to the reconciliation of GAAP to pro forma measures, which is provided later in this release.
Revenue
Ionis' revenue for the three and twelve months ended
$170 million from Biogen forFDA approval, licensing and advancing the Phase 3 program for SPINRAZA;$53 million fromAstraZeneca for advancing and licensing IONIS-KRAS-2.5Rx and selecting IONIS-AZ4-2.5-LRx to move into development;$15 million from Janssen for licensing IONIS-JBI1-2.5Rx and validating an undisclosed target to treat patients with a gastrointestinal autoimmune disease;$15 million from Kastle Therapeutics for acquiring Kynamro;$8 million from Biogen for advancing IONIS-SOD1Rx, IONIS-BIIB4Rx and IONIS-BIIB6Rx;$61 million from the amortization of upfront fees; and$25 million primarily from its partners for the manufacturing services Ionis performed.
In comparison, Ionis' revenue in 2015 included
Ionis' revenue fluctuates based on the nature and timing of payments under agreements with its partners and consists primarily of revenue from the amortization of upfront fees, milestone payments and license fees.
Operating Expenses
Ionis' operating expenses for the three and twelve months ended
Loss on Retirement of Debt
In
Income Tax Expense
Ionis recognized income tax expense of
Net Income (Loss)
Ionis reported net income of
Balance Sheet
As of
Conference Call
At
ABOUT
Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over three dozen drugs in development. SPINRAZA™ (nusinersen) is a drug that has been approved in the U.S. for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. Biogen is responsible for commercialization of SPINRAZA. Drugs currently in Phase 3 development include volanesorsen, a drug Ionis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with either familial chylomicronemia syndrome or familial partial lipodystrophy; and IONIS-TTRRx, a drug Ionis is developing with GSK to treat patients with TTR amyloidosis. Ionis' patents provide strong and extensive protection for its drugs and technology. Additional information about Ionis is available at www.ionispharma.com
FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding
In this release, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to
Ionis Pharmaceuticals™ is a trademark of
In 2017, Ionis plans to achieve the following objectives:
- Support Biogen in expanding access to SPINRAZA
- Assist Biogen with regulatory approval in the EU and filings in multiple countries outside the U.S., including
Japan ,Australia andCanada
- Assist Biogen with regulatory approval in the EU and filings in multiple countries outside the U.S., including
- Advance volanesorsen and IONIS-TTRRx toward the market
- File regulatory submissions for volanesorsen with Akcea, if Phase 3 study is positive
- File regulatory submissions for IONIS-TTRRx with GSK, if Phase 3 study is positive
- Build out U.S. and EU commercial infrastructure and prepare for commercial launch of volanesorsen through Akcea
- Assist GSK in preparing for commercial launch of IONIS-TTRRx
- Advance the pipeline
- Report clinical data on multiple drugs, including:
- Phase 3 data for volanesorsen in patients with familial chylomicronemia syndrome (FCS)
- Phase 3 data for IONIS-TTRRx in patients with transthyretin (TTR) familial amyloid polyneuropathy (FAP)
- Advance volanesorsen Phase 3 study in patients with familial partial lipodystrophy (FPL)
- Initiate multiple clinical studies
- Advance multiple LICA and Generation 2.5 drugs in development
- Report clinical data on multiple drugs, including:
- Broaden the pipeline by adding three to five new drugs into development
- Continue to advance antisense technology
- Achieve break-even or profitability on the operating line on a pro forma basis
(2016 and subsequent activities)
2016 and Recent SPINRAZA Accomplishments:
- Ionis and Biogen achieved
FDA approval of SPINRAZA in three months under Priority Review for the treatment of SMA in pediatric and adult patients. - Biogen filed for marketing authorization in the EU and was granted Accelerated Assessment.
- Biogen filed for marketing authorization in
Canada ,Japan , andAustralia . - Biogen reported positive data from an end of study analysis of the ENDEAR Phase 3 study in patients with infantile-onset (consistent with type 1) SMA at the
British Pediatric Neurology Association annual conference. Ionis previously reported data from an interim analysis of ENDEAR, which along with several other studies, formed the basis for the marketing application for SPINRAZA in the U.S. - Ionis and Biogen reported positive data from an interim analysis of the Phase 3 CHERISH study in patients with later-onset (consistent with Type 2) SMA.
- Ionis and Biogen presented new positive clinical data with SPINRAZA at the
World Muscle Society Congress supporting the companies' efforts to rapidly make SPINRAZA available to patients with SMA, including:- Safety results from the interim analysis of the Phase 3 ENDEAR study in patients with infantile-onset (consistent with Type 1) SMA;
- Encouraging preliminary results from NURTURE, a Phase 2 open-label study in pre-symptomatic infants; and
- A recent analysis of the ongoing Phase 2 open-label study in patients with later-onset SMA.
- Ionis and Biogen reported positive data from an interim analysis of the ENDEAR Phase 3 study in patients with infantile-onset (consistent with Type 1) SMA. Biogen paid Ionis
$75 million to license the drug.
Corporate Highlights:
- Ionis earned more than
$200 million from Biogen in 2016, including payments related to SPINRAZA. - Ionis and Akcea formed a strategic collaboration with
Novartis to develop and co-commercialize AKCEA-APO(a)-LRx and AKCEA-APOCIII-LRx for the treatment of lipid disorders, for which Ionis and Akcea are eligible to receive$225 million in near-term payments, including$100 million Ionis has received and$75 million Ionis expects to receive in the first quarter of 2017. The transaction has a potential value of up to over$1 billion . - Ionis earned
$75 million from Bayer to advance both IONIS-FXIRx and its LICA follow on, IONIS-FXI-LRx. - Ionis sold the global rights to develop and commercialize Kynamro to Kastle Therapeutics and earned a
$15 million upfront payment. - Ionis and
MD Anderson Cancer Center formed a strategic alliance to advance novel cancer therapies. - Ionis added to its pipeline its first oral locally acting drug for gastrointestinal autoimmune diseases for which Ionis earned a
$10 million license fee from Janssen. - Ionis advanced IONIS-KRAS-2.5Rx into development and earned
$28 million fromAstraZeneca . - Ionis advanced IONIS-AZ4-2.5-LRx, its first Generation 2.5 LICA drug, into development and earned
$25 million fromAstraZeneca . - Ionis' CEO, Dr.
Stanley Crooke , received two awards, theE. B. Hershberg Award from theAmerican Chemical Society and the Lifetime Achievement Award from theOligonucleotide Therapeutics Society , recognizing his achievements in the field of oligonucleotide therapeutics.
Drug Development and Technology Highlights
- Ionis continued to advance its pipeline of innovative first-in-class or best-in-class drugs, reported positive data from 11 clinical studies with six drugs. These data and clinical advancements represent the broad applicability of Ionis' technology to address unmet medical needs across multiple disease targets.
- The
FDA and EMA granted orphan drug designation to IONIS-HTTRx for the treatment of patients with Huntington's disease. - Akcea launched IN-FOCUS, a research study to assess the impact of familial chylomicronemia syndrome.
- Akcea published positive clinical data from a Phase 2 study of volanesorsen in patients with high plasma triglyceride levels and type 2 diabetes in Diabetes Care.
- Ionis published a paper in Nature Biotechnology on the novel mechanism of action for antisense drugs that significantly expands therapeutic opportunities for the technology.
- Ionis published a paper in Nucleic Acid Therapeutics on the analysis of its Integrated Safety Database, which demonstrated no class generic effect of 2'-O-methoxyethyl (2'MOE)-modified antisense oligonucleotides (ASOs) on platelet numbers and function.
IONIS PHARMACEUTICALS, INC. SELECTED FINANCIAL INFORMATION Condensed Consolidated Statements of Operations |
|||||||||
Three months ended, |
Year ended, |
||||||||
December 31, |
December 31, |
||||||||
2016 |
2015 |
2016 |
2015 |
||||||
Revenue: |
|||||||||
Research and development revenue under collaborative agreements |
$159,316 |
$50,891 |
$325,898 |
$281,360 |
|||||
Licensing and royalty revenue |
1,033 |
679 |
20,722 |
2,343 |
|||||
Total revenue |
160,349 |
51,571 |
346,620 |
283,703 |
|||||
Expenses: |
|||||||||
Research, development and patent expenses |
101,151 |
101,330 |
344,320 |
322,292 |
|||||
General and administrative |
18,043 |
13,181 |
48,616 |
37,173 |
|||||
Total operating expenses |
119,194 |
114,511 |
392,936 |
359,465 |
|||||
Income (loss) from operations |
41,155 |
(62,940) |
(46,316) |
(75,762) |
|||||
Other income (expense): |
|||||||||
Investment income |
1,548 |
1,355 |
5,416 |
4,302 |
|||||
Interest expense |
(9,934) |
(9,351) |
(38,795) |
(36,732) |
|||||
Gain on investments, net |
13 |
- |
56 |
75 |
|||||
Gain on investment in Regulus Therapeutics, Inc. |
- |
- |
- |
20,211 |
|||||
Loss on early retirement of debt |
(3,983) |
- |
(3,983) |
- |
|||||
Income (loss) before income tax benefit |
28,799 |
(70,061) |
(83,622) |
(87,906) |
|||||
Income tax expense |
(2,934) |
(372) |
(2,934) |
(372) |
|||||
Net income (loss) |
$25,865 |
$(71,433) |
$(86,556) |
$(88,278) |
|||||
Basic net income (loss) per share |
$0.21 |
$(0.59) |
$(0.72) |
$(0.74) |
|||||
Diluted net income (loss) per share |
$0.21 |
$(0.59) |
$(0.72) |
$(0.74) |
|||||
Shares used in computing basic net income (loss) per share |
121,340 |
120,189 |
120,933 |
119,719 |
|||||
Shares used in computing diluted net income (loss) per share |
123,953 |
120,189 |
120,933 |
119,719 |
Ionis Pharmaceuticals, Inc. Reconciliation of GAAP to Pro Forma Basis: Condensed Consolidated Operating Expenses, Income (Loss) From Operations, and Net Income (In Thousands) |
||||||||
Three months ended, December 31, |
Year ended December 31, |
|||||||
2016 |
2015 |
2016 |
2015 |
|||||
As reported operating expenses according to GAAP |
$119,194 |
$114,511 |
$392,936 |
$359,465 |
||||
Excluding compensation expense related to equity awards |
(15,158) |
(17,408) |
(72,108) |
(59,314) |
||||
Pro forma operating expenses |
$104,036 |
$97,103 |
$320,828 |
$300,151 |
||||
As reported income (loss) from operations according to GAAP |
$41,155 |
$(62,940) |
$(46,316) |
$(75,762) |
||||
Excluding compensation expense related to equity awards |
(15,158) |
(17,408) |
(72,108) |
(59,314) |
||||
Pro forma income (loss) from operations |
$56,313 |
$(45,532) |
$25,792 |
$(16,448) |
||||
As reported net income (loss) according to GAAP |
$25,865 |
$(71,433) |
$(86,556) |
$(88,278) |
||||
Excluding compensation expense related to equity awards |
(15,158) |
(17,408) |
(72,108) |
(59,314) |
||||
Pro forma net income (loss) |
$41,023 |
$(54,025) |
$(14,448) |
$(28,964) |
Reconciliation of GAAP to Pro Forma Basis
As illustrated in the Selected Financial Information in this press release, pro forma operating expenses, pro forma income (loss) from operations, and pro forma net income (loss) were adjusted from GAAP to exclude compensation expense related to equity awards, which are non-cash. Ionis has regularly reported non-GAAP measures for operating results as pro forma results. These measures are provided as supplementary information and are not a substitute for financial measures calculated in accordance with GAAP. Ionis reports these pro forma results to better enable financial statement users to assess and compare its historical performance and project its future operating results and cash flows. Further, the presentation of Ionis' pro forma results is consistent with how Ionis' management internally evaluates the performance of its operations.
Ionis Pharmaceuticals, Inc. Condensed Consolidated Balance Sheets (In Thousands) |
|||||
December 31, |
December 31, |
||||
2016 |
2015 |
||||
Assets: |
|||||
Cash, cash equivalents and short-term investments |
$665,223 |
$779,183 |
|||
Contracts receivable |
108,043 |
11,356 |
|||
Investment in Regulus Therapeutics Inc. |
2,414 |
24,792 |
|||
Other current assets |
22,252 |
33,028 |
|||
Property, plant and equipment, net |
92,845 |
90,233 |
|||
Other assets |
21,690 |
20,664 |
|||
Total assets |
$912,467 |
$947,900 |
|||
Liabilities and stockholders' equity: |
|||||
Other current liabilities |
$82,504 |
$81,554 |
|||
Current portion of deferred contract revenue |
51,280 |
67,322 |
|||
1% convertible senior notes |
500,511 |
339,847 |
|||
2 3/4% convertible senior notes |
124 |
49,523 |
|||
Long-term obligations, less current portion |
87,285 |
74,558 |
|||
Long-term deferred contract revenue |
91,198 |
134,306 |
|||
Stockholders' equity |
99,565 |
200,790 |
|||
Total liabilities and stockholders' equity |
$912,467 |
$947,900 |
|||
To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/ionis-2016-financial-results-outperform-financial-guidance-300414629.html
SOURCE
Investor and Media Contact: D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741