Press Releases
Ionis Pharmaceuticals Reports Financial Results and Highlights for Second Quarter 2016
"The recent announcement to file for regulatory approval of nusinersen based on positive results from an interim analysis of the Phase 3 ENDEAR study was an important next step in our goal to bring this potentially transformational medicine to the patients who desperately need it as quickly as possible. This is the first time a potential treatment for infantile-onset SMA has demonstrated a clinical benefit in a controlled clinical study," said
Biogen has exercised its option to license nusinersen and will be responsible for all development, regulatory and commercialization activities and costs going forward. Over the next several months, Ionis will be working closely with Biogen to transition patients in the ENDEAR and EMBRACE studies to an open-label study that will allow all patients in these studies to have access to nusinersen. Once these patients have been transitioned into an open-label study, Biogen plans to open an expanded access program to make nusinersen available to eligible patients with infantile-onset SMA (consistent with type 1). Ionis will continue to conduct the Phase 3 CHERISH study in childhood-onset SMA patients. Biogen will also continue to conduct the NURTURE study in pre-symptomatic SMA infants.
"We believe that Biogen is the right company to bring nusinersen to patients with this devastating disease. They understand the unique needs of the SMA community and the impact SMA has on patients and their families. Importantly, Biogen has the global commercial infrastructure and expertise needed to successfully launch and commercialize nusinersen. Nusinersen is the first antisense drug from our neurological disease collaboration with Biogen that we expect to advance to regulatory review, and we are excited about the possibility of bringing other therapies to patients with severe neurological diseases with limited or no treatment options," continued Ms. Parshall.
Financial Results
"We finished the second quarter in a strong financial position. In the first half of this year, we earned
"We have continued to advance our Phase 3 programs and to prepare to commercialize volanesorsen through Akcea while prudently managing our expenses. As a result, we finished the first half of 2016 with a GAAP loss from operations 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 GAAP to pro forma measures, which is provided later in this release.
Revenue
Ionis' revenue for the three and six months ended
$15 million from Kastle Therapeutics in an upfront payment for KYNAMRO;$14.5 million from Biogen for advancing the Phase 3 program for nusinersen and advancing IONIS-BIIB4Rx;$1.5 million from GSK for advancing IONIS-HBV-LRx; and$44.3 million primarily from the amortization of upfront fees and manufacturing services Ionis performed for its partners.
Ionis' revenue in the first half of 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. Already in the third quarter of 2016, Ionis has earned
Operating Expenses
Ionis' operating expenses included costs to support the Company's five ongoing Phase 3 studies and three open-label extension studies related to its Phase 3 programs for nusinersen, IONIS-TTRRx and volanesorsen. In addition, Akcea continued to build its operations in preparation for the commercial launch of volanesorsen. As such, Ionis' operating expenses increased for the three and six months ended
Net Income (Loss)
Ionis reported a net loss 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 a dozen drugs in mid- to late-stage development. 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; IONIS-TTRRx, a drug Ionis is developing with GSK to treat patients with all forms of TTR amyloidosis; and nusinersen, a drug Ionis is developing with Biogen to treat infants and children with spinal muscular atrophy. 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
(Q2 2016 and subsequent activities)
- Ionis reported positive data from an interim analysis of the ENDEAR Phase 3 study in infant-onset SMA. Biogen paid Ionis a
$75 million license fee and plans to file marketing applications in the U.S. and E.U. in the next few months, with other countries to follow. - Ionis and Dr.
Merrill Benson reported positive data from the IONIS-TTRRx program at the International Symposium on Amyloidosis (ISA) meeting. In line with previously reported data from his investigator-initiated study, Dr. Benson observed continued evidence of cardiac disease stabilization in eight TTR cardiomyopathy patients treated for 12 months with IONIS-TTRRx. - 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 added to its pipeline its first oral antisense drug acting locally in the GI tract for which Ionis earned a
$10 million license fee from Janssen. - Ionis reported positive results from studies in normal volunteers with IONIS-ANGPTL3-LRx and IONIS-GSK4-LRx that demonstrated these drugs had similar potency to IONIS-APO(a)-LRx, confirming the high potency of the LICA platform.
- Ionis reported positive interim data from a Phase 2 dose-optimization study of IONIS-GCGRRx in patients with type 2 diabetes demonstrating that doses of 75 mg and 50 mg could produce reductions in HbA1c of greater than two percent and one percent, respectively, with minimal to no effects on liver enzyme elevations.
- Ionis reported positive data from a Phase 2 study of IONIS-AR-2.5Rx in patients with prostate cancer showing durable prostate-specific antigen (
PSA ) responses with prolonged stable disease in heavily pre-treated castrate-resistance prostate cancer patients. - Ionis and
MD Anderson Cancer Center formed a strategic alliance to advance novel cancer therapies. - Akcea Therapeutics completed enrollment of the Phase 3 COMPASS trial, a study designed to support volanesorsen regulatory filings that is evaluating the effects of volanesorsen on triglyceride lowering in patients with triglycerides greater than 500 mg/dL.
- Ionis sold the global rights to develop and commercialize KYNAMRO to Kastle Therapeutics and earned a
$15 million upfront payment. - Ionis reported data from its ongoing open-label Phase 2 study of nusinersen in infantile-onset SMA patients at the 2016 AAN meeting as well as the latest progress on multiple new antisense drugs designed to treat neurological diseases.
IONIS PHARMACEUTICALS, INC. |
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SELECTED FINANCIAL INFORMATION |
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Condensed Consolidated Statements of Operations |
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(In Thousands, Except Per Share Data) |
|||||||||
Three months ended, |
Six months ended, |
||||||||
June 30, |
June 30, |
||||||||
2016 |
2015 |
2016 |
2015 |
||||||
Revenue: |
(unaudited) |
(unaudited) |
|||||||
Research and development revenue under collaborative agreements |
$22,455 |
$119,658 |
$57,670 |
$181,551 |
|||||
Licensing and royalty revenue |
16,015 |
770 |
17,675 |
1,461 |
|||||
Total revenue |
38,470 |
120,428 |
75,345 |
183,012 |
|||||
Expenses: |
|||||||||
Research, development and patent expenses |
77,573 |
68,007 |
158,536 |
132,454 |
|||||
General and administrative |
9,824 |
7,775 |
20,386 |
15,241 |
|||||
Total operating expenses |
87,397 |
75,782 |
178,922 |
147,695 |
|||||
Income (loss) from operations |
(48,927) |
44,646 |
(103,577) |
35,317 |
|||||
Other income (expense): |
|||||||||
Investment income |
1,466 |
918 |
2,921 |
1,762 |
|||||
Interest expense |
(9,625) |
(9,127) |
(19,115) |
(18,148) |
|||||
Income (loss) before income tax benefit |
(57,086) |
36,437 |
(119,771) |
18,931 |
|||||
Income tax benefit (expense) |
231 |
(789) |
(1) |
- |
|||||
Net income (loss) |
$(56,855) |
$35,648 |
$(119,772) |
$18,931 |
|||||
Basic net income (loss) per share |
$(0.47) |
$0.30 |
$(0.99) |
$0.16 |
|||||
Diluted net income (loss) per share |
$(0.47) |
$0.29 |
$(0.99) |
$0.15 |
|||||
Shares used in computing basic net income (loss) per share |
120,798 |
119,742 |
120,698 |
119,348 |
|||||
Shares used in computing diluted net income (loss) per share |
120,798 |
127,779 |
120,698 |
124,061 |
Ionis Pharmaceuticals, Inc. |
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Reconciliation of GAAP to Pro Forma Basis: |
||||||||
Condensed Consolidated Operating Expenses, Income (Loss) From Operations, and Net Income (Loss) |
||||||||
(In Thousands) |
||||||||
Three months ended, June 30, |
Six months ended, June 30, |
|||||||
2016 |
2015 |
2016 |
2015 |
|||||
(unaudited) |
(unaudited) |
|||||||
As reported operating expenses according to GAAP |
$87,397 |
$75,782 |
$178,922 |
$147,695 |
||||
Excluding compensation expense related to equity awards |
(19,260) |
(13,605) |
(39,364) |
(26,910) |
||||
Pro forma operating expenses |
$68,137 |
$62,177 |
$139,558 |
$120,785 |
||||
As reported income (loss) from operations according to GAAP |
$(48,927) |
$44,646 |
$(103,577) |
$35,317 |
||||
Excluding compensation expense related to equity awards |
(19,260) |
(13,605) |
(39,364) |
(26,910) |
||||
Pro forma income (loss) from operations |
$(29,667) |
$58,251 |
$(64,213) |
$62,227 |
||||
As reported net income (loss) according to GAAP |
$(56,855) |
$35,648 |
$(119,772) |
$18,931 |
||||
Excluding compensation expense related to equity awards |
(19,260) |
(13,605) |
(39,364) |
(26,910) |
||||
Pro forma net income (loss) |
$(37,595) |
$49,253 |
$(80,408) |
$45,841 |
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 |
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(In Thousands) |
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June 30, |
December 31, |
|||||
2016 |
2015 |
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Assets: |
||||||
Cash, cash equivalents and short-term investments |
$664,090 |
$779,183 |
||||
Investment in Regulus Therapeutics Inc. |
8,217 |
24,792 |
||||
Other current assets |
25,664 |
33,028 |
||||
Property, plant and equipment, net |
90,602 |
90,233 |
||||
Other assets |
22,536 |
20,664 |
||||
Total assets |
$811,109 |
$947,900 |
||||
Liabilities and stockholders' equity: |
||||||
Other current liabilities |
$52,608 |
$81,554 |
||||
Current portion of deferred contract revenue |
58,473 |
67,322 |
||||
1% convertible senior notes |
350,800 |
339,847 |
||||
2 3/4% convertible senior notes |
50,873 |
49,523 |
||||
Long-term obligations, less current portion |
74,706 |
74,558 |
||||
Long-term deferred contract revenue |
112,436 |
134,306 |
||||
Stockholders' equity |
111,213 |
200,790 |
||||
Total liabilities and stockholders' equity |
$811,109 |
$947,900 |
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SOURCE
Investor and Media Contact, D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741