Press Releases
Ionis Reports First Quarter 2018 Financial Results
"We have successfully executed our commercialization strategy through the expansion of our strategic relationship with Biogen in neurological diseases and our commercialization of inotersen through our affiliate, Akcea. Our strategy is to create tailored commercial solutions for each of our drugs with the aim to maximize the commercial value of the drug and optimize our financial participation in this value. As our most recent collaboration with Biogen demonstrates, we have substantially increased the value of our antisense technology platform in neurological diseases over the last several years. The economics we achieved with our Bayer and
First Quarter 2018 Financial Highlights
- Revenues increased by 25%, driven by SPINRAZA royalties
- Total revenues were
$144 million , compared to$116 million in Q1 2017 - Commercial revenue from SPINRAZA royalties was
$41 million , compared to$5 million in Q1 2017 - R&D revenue included
$60 million in licensing fees for two drugs discovered by Ionis under its collaboration withAstraZeneca - Beginning in Q2 2018, Ionis' R&D revenue will include revenue from the amortization of the
$500 million technology access fee and equity premium related to Ionis' expanded strategic research collaboration with Biogen - GAAP operating and net loss near breakeven; on track for third consecutive year of pro forma operating profitability
- GAAP operating loss was
$3 million in Q1 2018, compared to GAAP operating income of$19 million for the same period in 2017. Pro forma operating income was$25 million in Q1 2018, compared to$40 million for the same period in 2017 - Operating expenses increased primarily due to higher SG&A expenses as Ionis prepares to commercialize TEGSEDI and WAYLIVRA this year
- Cash will increase to more than
$2 billion , combining Ionis' first quarter cash balance of more than$1 billion with$1 billion expected upon closing of Ionis' expanded collaboration with Biogen - During Q1 2018, Ionis received more than
$155 million in payments from partners
"In the first quarter, we made further progress toward our goal of being a multiproduct, profitable company. We ended the quarter with operating income of
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 pro forma and GAAP measures, which is provided later in this release.
Business Highlights
- Expanded strategic research collaboration with Biogen for neurological diseases – one of the largest research-stage collaborations ever
$1 billion upfront to Ionis, including$625 million to purchase Ionis' stock at a 25% cash premium of$125 million and a$375 million upfront payment- Together, the cash premium and upfront payment represent a
$500 million technology access fee - Ionis is eligible to receive milestone payments and license fees up to
$270 million per drug and royalties up to 20% on net sales - Disease areas include dementia, neuromuscular diseases, movement disorders, ophthalmology, diseases of the inner ear, and neuropsychiatry
- SPINRAZA ® for SMA – one of the most successful orphan disease drug launches in history
- SPINRAZA®, commercialized by Biogen, continued to generate growth with global revenues of
$364 million in Q1 2018 - Increase of over 25% from last quarter in number of patients on SPINRAZA, including a 16% increase in number of patients treated in the U.S. and a more than 50% increase outside the US
- Access expanding outside the U.S. with reimbursement in 24 countries; Biogen expects reimbursement in at least seven more countries by the end of 2018
- Presented data from the SHINE open-label study at the
American Academy of Neurology (AAN) annual meeting demonstrating continued benefit, improved motor function and mobility, and longer event-free survival for the most severely affected patients treated with SPINRAZA® - Presented data from the NURTURE study at the
Muscular Dystrophy Association (MDA) Clinical Conference demonstrating continued benefit in motor function for infants, teens and young adults treated with SPINRAZA® - TEGSEDI (inotersen) for hereditary transthyretin amyloidosis (hATTR) – potential to transform the lives of people with hATTR; on-track to launch in 2018
- Invested in global commercialization of TEGSEDI by licensing TEGSEDI to Ionis' majority-owned affiliate, Akcea
- Optimized Ionis' commercial participation with up to
$1.5 billion in milestone payments and a 60% profit share - Early access program enrolling in the U.S. and
Europe - Global commercial organization staffed and focused on disease education; robust patient support program in place; supply chain in place and launch supplies ready to be labeled
- Presented data from the Phase 3 NEURO-TTR study, the open label extension study and an investigator sponsored Phase 2 study at the International Symposium on Amyloidosis annual meeting and the AAN annual meeting
- WAYLIVRA (volanesorsen) for FCS and FPL – potential first treatment for people with FCS; global on-track to launch in 2018
- Early access program enrolling in the U.S. and
Europe - Global commercial organization staffed and focused on disease education; robust patient support program in place; supply chain in place and launch supplies ready to be labeled
- Positive scientific opinion to initiate Early Access to Medicines Scheme (EAMS) by the
UK's Medicines and Healthcare Products Regulatory Agency (MHRA), for the treatment of people with FCS - Collaboration with
AstraZeneca for Cardiovascular, Renal and Metabolic Diseases - Earned
$60 million for the license of second and third antisense drugs, IONIS-AZ5-2.5Rx and IONIS-AZ6-2.5-LRx, to treat a genetically associated form of kidney disease and nonalcoholic steatohepatitis (NASH), respectively, toAstraZeneca - As IONIS-AZ5-2.5Rx and IONIS-AZ6-2.5-LRx advance, Ionis may receive up to
$300 million for each drug in additional development and regulatory milestone payments, as well as tiered royalties on sales of each drug
Pipeline and Technology Progress
- Presented positive IONIS-HTTRx (RG6042) Phase 1/2 data in people with Huntington's disease (HD) at the annual CHDI HD conference. IONIS-HTTRx is the first drug in development to lower the disease-causing protein in people with HD
- Presented data at the AAN annual meeting that demonstrated broad potential of antisense drugs for neurological diseases with 14 presentations on Ionis' drugs to treat neurological diseases, including SMA, hATTR amyloidosis, Huntington's disease, Alzheimer's disease, and ALS
- Presented additional data from the Phase 1/2 study of IONIS-HTTRx that demonstrated correlations between reductions in mutant huntingtin (mHTT) and improvements in clinical measures of HD
- Published review paper titled, "RNA-targeted Therapeutics" in Cell Metabolism, authored by
Stanley Crooke , M.D., Ph.D.; highlights antisense and other RNA-targeting therapeutics as important platforms for drug discovery across multiple diseases
"This year, we plan to launch two new promising drugs for rare diseases, TEGSEDI and WAYLIVRA. We look forward to adding commercial revenue from these drugs, assuming approval, to our growing revenue from SPINRAZA," said
Expected Events Through 2018
- Launch of TEGSEDI for people with hATTR, assuming approval
- Launch of WAYLIVRA for people with FCS, assuming approval
- Report results from six Phase 2 programs, including data from a study with AKCEA-APO(a)-LRx in people with high Lp(a) and AKCEA-ANGPTL3-LRx for people with rare hyperlipidemias
- Initiate up to nine new clinical studies, including a clinical study of AKCEA-TTR-LRx for hereditary and wild-type forms of ATTR
The recent Biogen transaction is subject to customary closing conditions, including the expiration of the applicable waiting period under the Hart Scott Rodino Antitrust Improvements Act of 1976 in
Revenue
At the beginning of 2018, Ionis adopted the new revenue recognition accounting standard on a retrospective basis. Starting with Ionis' first quarter, all periods presented are shown using the new standard. Ionis has labeled its prior period financial statements "as revised" to indicate the change required under the accounting rules. Whenever Ionis refers to prior period results, they reflect the new accounting rules. This change did not have a significant impact on Ionis' previously reported revenue.
Ionis' revenue in the first quarter of 2018 was
Three months ended, |
||||||
March 31, |
||||||
2018 |
2017 |
|||||
Revenue: |
||||||
Commercial revenue: |
||||||
SPINRAZA royalties |
$41 |
$5 |
||||
Licensing and royalty revenue |
1 |
3 |
||||
Total commercial revenue |
42 |
8 |
||||
R&D Revenue: |
||||||
License fees |
62 |
65 |
||||
Milestone payments |
2 |
3 |
||||
Amortization from: |
||||||
Upfront payments |
27 |
20 |
||||
Milestone payments |
5 |
17 |
||||
Other services |
6 |
3 |
||||
Total R&D revenue |
102 |
108 |
||||
Total revenue |
$144 |
$116 |
License fees in the first quarter of 2018 were
Operating Expenses
Operating expenses for the first quarter on a GAAP basis were $147.7 million and on a pro forma basis were $119.3 million compared to GAAP operating expenses of $96.3 million and pro forma operating expenses of $75.4 million for the same period in 2017. Operating expenses increased in Q1 2018, compared to 2017, principally due to higher SG&A expenses as Ionis and its affiliate Akcea prepare to commercialize WAYLIVRA and TEGSEDI. The Company's SG&A expenses also increased in Q1 2018 compared to Q1 2017 because of fees owed under its in-licensing agreements related to SPINRAZA, which increase as the Company's SPINRAZA revenue increases. R&D expenses accounted for a smaller portion of the increase in operating expenses. R&D expenses increased primarily from medical affairs expenses and manufacturing costs related to TEGSEDI for the planned launch.
Net Income (Loss)
Ionis reported a net loss of $10.8 million for the first quarter of 2018, compared to net income of $9.0 million for the same period in 2017, all according to GAAP. On a pro forma basis, Ionis reported net income of $17.6 million for the first quarter of 2018, compared to net income of
Net Loss Attributable to Noncontrolling Interest in
Akcea sold shares of its common stock to third parties in its IPO in
In
Net Income (Loss) Attributable to Ionis Common Stockholders
Ionis reported a GAAP net loss attributable to Ionis' common stockholders of $1.4 million for the first quarter of 2018, compared to GAAP net income of
Webcast and Conference Call
Today, 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 45 drugs in development. SPINRAZA® (nusinersen) has been approved in global markets for the treatment of spinal muscular atrophy (SMA). Biogen is responsible for commercializing SPINRAZA. TEGSEDI (inotersen) and WAYLIVRA (volanesorsen) are two antisense drugs that Ionis discovered and successfully advanced through Phase 3 studies. TEGSEDI is under regulatory review for marketing approval in the U.S., EU and
Ionis' Forward-looking Statement
This press release includes forward-looking statements regarding business, financial guidance and the therapeutic and commercial potential of SPINRAZA, TEGSEDI (inotersen), WAYLIVRA (volanesorsen) and Ionis' technologies and products in development, including the business of
In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to
IONIS PHARMACEUTICALS, INC. SELECTED FINANCIAL INFORMATION Condensed Consolidated Statements of Operations (In Thousands, Except Per Share Data) |
||||
Three months ended, |
||||
March 31, |
||||
2018 |
2017 |
|||
(as revised) |
||||
Revenue: |
(unaudited) |
|||
Commercial revenue: |
||||
SPINRAZA royalties |
$41,081 |
$5,211 |
||
Licensing and royalty revenue |
942 |
2,590 |
||
Total commercial revenue |
42,023 |
7,801 |
||
Research and development revenue under collaborative agreements |
102,396 |
107,999 |
||
Total revenue |
144,419 |
115,800 |
||
Expenses: |
||||
Research, development and patent expenses |
104,067 |
82,638 |
||
Selling, general and administrative |
43,653 |
13,677 |
||
Total operating expenses |
147,720 |
96,315 |
||
Income (loss) from operations |
(3,301) |
19,485 |
||
Other income (expense): |
||||
Investment income |
3,610 |
2,280 |
||
Interest expense |
(10,938) |
(11,363) |
||
Other expenses |
(168) |
(1,438) |
||
Income (loss) before income tax expense |
(10,797) |
8,964 |
||
Income tax expense |
(15) |
- |
||
Net income (loss) |
$(10,812) |
$8,964 |
||
Net loss attributable to noncontrolling interest in Akcea Therapeutics, Inc. |
$9,392 |
- |
||
Net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders |
$(1,420) |
$8,964 |
||
Basic net income (loss) per share |
$(0.01) |
$0.07 |
||
Diluted net income (loss) per share |
$(0.01) |
$0.07 |
||
Shares used in computing basic net income (loss) per share |
125,330 |
122,861 |
||
Shares used in computing diluted net income (loss) per share |
125,330 |
124,972 |
IONIS PHARMACEUTICALS, INC. SELECTED FINANCIAL INFORMATION Condensed Consolidating Statement of Operations (In Thousands) |
||||||||
Three months ended, |
||||||||
Ionis |
Akcea |
Eliminations |
Ionis Consolidated |
|||||
Revenue: |
||||||||
Commercial revenue: |
||||||||
SPINRAZA royalties |
$41,081 |
$- |
$- |
$41,081 |
||||
Licensing and royalty revenue |
942 |
- |
- |
942 |
||||
Total commercial revenue |
42,023 |
- |
- |
42,023 |
||||
Research and development revenue under collaborative agreements |
85,288 |
17,108 |
- |
102,396 |
||||
Intercompany revenue |
5,229 |
- |
(5,229) |
- |
||||
Total revenue |
132,540 |
17,108 |
(5,229) |
144,419 |
||||
Expenses: |
||||||||
Research, development and patent expenses |
81,356 |
27,970 |
(5,259) |
104,067 |
||||
Selling, general and administrative |
24,188 |
19,465 |
- |
43,653 |
||||
Total operating expenses |
105,544 |
47,435 |
(5,259) |
147,720 |
||||
Income (loss) from operations |
26,996 |
(30,327) |
30 |
(3,301) |
||||
Other income (expense): |
||||||||
Investment income |
2,742 |
868 |
- |
3,610 |
||||
Interest expense |
(10,938) |
- |
- |
(10,938) |
||||
Other expenses |
- |
(168) |
- |
(168) |
||||
Income (loss) before income tax expense |
18,800 |
(29,627) |
30 |
(10,797) |
||||
Income tax expense |
(15) |
- |
- |
(15) |
||||
Net income (loss) |
$18,785 |
$(29,627) |
$30 |
$(10,812) |
||||
Net loss attributable to noncontrolling interest in Akcea Therapeutics, Inc. |
$ - |
$ - |
$9,392 |
$9,392 |
||||
Net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders |
$18,785 |
$(29,627) |
$9,422 |
$(1,420) |
IONIS PHARMACEUTICALS, INC. Reconciliation of GAAP to Pro Forma Basis: Condensed Consolidated Operating Expenses, Income (Loss) From Operations, and Net |
|||||
Three months ended, March 31, |
|||||
2018 |
2017 |
||||
(as revised) |
|||||
(unaudited) |
|||||
As reported operating expenses according to GAAP |
$147,720 |
$96,315 |
|||
Excluding compensation expense related to equity awards |
(28,451) |
(20,912) |
|||
Pro forma operating expenses |
$119,269 |
$75,403 |
|||
As reported income (loss) from operations according to GAAP |
$(3,301) |
$19,485 |
|||
Excluding compensation expense related to equity awards |
(28,451) |
(20,912) |
|||
Pro forma income from operations |
$25,150 |
$40,397 |
|||
As reported net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders according to GAAP |
$(1,420) |
$8,964 |
|||
Excluding compensation expense related to equity awards |
(28,451) |
(20,912) |
|||
Pro forma net income attributable to Ionis Pharmaceuticals, Inc. common stockholders |
$27,031 |
$29,876 |
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) (unaudited) |
|||||
March 31, |
December 31, |
||||
2018 |
2017 |
||||
(as revised) |
|||||
Assets: |
|||||
Cash, cash equivalents and short-term investments |
$1,035,301 |
$1,022,715 |
|||
Contracts receivable |
36,858 |
62,955 |
|||
Other current assets |
71,124 |
83,064 |
|||
Property, plant and equipment, net |
123,188 |
121,907 |
|||
Other assets |
33,089 |
32,133 |
|||
Total assets |
$1,299,560 |
$1,322,774 |
|||
Liabilities and stockholders' equity: |
|||||
Other current liabilities |
$92,516 |
$118,276 |
|||
Current portion of deferred contract revenue |
120,127 |
125,336 |
|||
1% convertible senior notes |
541,635 |
533,111 |
|||
Long-term obligations, less current portion |
72,735 |
72,745 |
|||
Long-term deferred contract revenue |
85,446 |
108,026 |
|||
Total Ionis stockholders' equity |
301,391 |
281,013 |
|||
Noncontrolling interest in Akcea Therapeutics, Inc. |
85,710 |
84,267 |
|||
Total stockholders' equity |
387,101 |
365,280 |
|||
Total liabilities and stockholders' equity |
$1,299,560 |
$1,322,774 |
|||
IONIS PHARMACEUTICALS, INC. |
||||||||||||||||||||
Condensed Consolidating Balance Sheet |
||||||||||||||||||||
(In Thousands) |
||||||||||||||||||||
March 31, 2018 |
||||||||||||||||||||
(unaudited) |
||||||||||||||||||||
Ionis |
||||||||||||||||||||
Ionis |
Akcea |
Eliminations |
Consolidated |
|||||||||||||||||
Assets: |
||||||||||||||||||||
Cash, cash equivalents and short-term investments |
$790,366 |
$244,935 |
$- |
$1,035,301 |
||||||||||||||||
Contracts receivable |
36,858 |
- |
- |
36,858 |
||||||||||||||||
Receivable from Akcea Therapeutics, Inc. |
27,737 |
- |
(27,737) |
- |
||||||||||||||||
Other current assets |
65,496 |
5,628 |
- |
71,124 |
||||||||||||||||
Property, plant and equipment, net |
123,138 |
50 |
- |
123,188 |
||||||||||||||||
Other assets |
305,449 |
1,853 |
(274,213) |
33,089 |
||||||||||||||||
Total assets |
$1,349,044 |
$252,466 |
$(301,950) |
$1,299,560 |
||||||||||||||||
Liabilities and stockholders' equity: |
||||||||||||||||||||
Other current liabilities |
$70,823 |
$49,431 |
$(27,738) |
$92,516 |
||||||||||||||||
Current portion of deferred contract revenue |
71,261 |
48,866 |
- |
120,127 |
||||||||||||||||
1% convertible senior notes |
541,635 |
- |
- |
541,635 |
||||||||||||||||
Long-term obligations, less current portion |
72,725 |
10 |
- |
72,735 |
||||||||||||||||
Long-term deferred contract revenue |
79,286 |
7,859 |
(1,699) |
85,446 |
||||||||||||||||
Total stockholders' equity before noncontrolling interest |
513,314 |
146,300 |
(358,223) |
301,391 |
||||||||||||||||
Noncontrolling interest in Akcea Therapeutics, Inc. |
- |
- |
85,710 |
85,710 |
||||||||||||||||
Total stockholders' equity |
513,314 |
146,300 |
(272,513) |
387,101 |
||||||||||||||||
Total liabilities and stockholders' equity |
$1,349,044 |
$252,466 |
$(301,950) |
$1,299,560 |
SPINRAZA Q1 2017 – Q1 2018 Patient Dynamics |
|||||
U.S. Patient Dynamics* |
Q1:17 |
Q2:17 |
Q3:17 |
Q4:17 |
Q1:18 |
Total patients |
210 |
710 |
1,230 |
1,640 |
1,910 |
New patient starts |
210 |
500 |
530 |
420 |
280 |
Average doses per patient |
2.3 |
2.6 |
1.9 |
1.6 |
1.1 |
% Loading doses |
100% |
100% |
90% |
75% |
60% |
% Maintenance doses |
0% |
0% |
10% |
25% |
40% |
% Free doses |
25% |
20% |
20% |
20% |
20% |
*As announced by Biogen in their Q1:18 earnings call |
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SOURCE
Ionis Pharmaceuticals Investor Contact: D. Wade Walke, Ph.D., Vice President, Investor Relations, 760-603-2741, Ionis Pharmaceuticals Media Contact: Roslyn Patterson, Vice President, Corporate Communications, 760-603-2681