Press Releases
Ionis' 2015 Financial Results Outperform Projections
"We have a pipeline of drugs with the potential to change the lives of patients with many different diseases. In 2015, we completed target enrollment in three Phase 3 studies for our three most advanced drugs in development; nusinersen, IONIS-TTRRx and volanesorsen, bringing these important new medicines one step closer to potentially reaching the market. These drugs are being developed for six different patient populations. We believe the robust development plan for each of these three drugs supports their significant commercial potential. We and our partners are well along in preparing for regulatory filings and for commercialization of nusinersen and IONIS-TTRRx. Our wholly owned subsidiary, Akcea Therapeutics, has begun the key steps necessary to launch volanesorsen.
"The majority of our drugs in Phase 3 and Phase 2 clinical development and our growing number of earlier stage programs provided us with numerous opportunities to highlight the breadth and depth of our pipeline in 2015. We reported positive clinical data from ten drugs in development. We designed these drugs to treat patients with a wide variety of diseases. In addition, in 2015 we advanced several earlier-stage programs into clinical development, including our drug to treat patients with Huntington's disease. We believe these earlier-stage drugs represent the next wave of new medicines with the potential to fundamentally change the treatment of diseases that are currently untreatable," continued Ms. Parshall. "The innovations we have made in our technology have the potential to add significant value to our pipeline. The substantial increase in potency conferred by our LICA technology supports weekly, monthly, quarterly or even less frequent dosing. This is a major advance and broadens the reach of our technology to larger patient populations and new therapeutic areas. The additional potency we achieved with our Generation 2.5 technology also allows us to continue to expand the opportunities for our antisense drugs."
"In 2016, we plan to complete Phase 3 studies for nusinersen, IONIS-TTRRx and volanesorsen and report data from these studies in the first half of 2017. We plan to report additional data from the open-label studies of nusinersen and IONIS-TTRRx. Data from these open-label studies continue to support the potential of these important new drugs. We will continue to provide updates as Akcea builds its commercial infrastructure and prepares to launch volanesorsen. We also plan to report data from many clinical studies, including Phase 2 data from our novel antithrombotic drug, IONIS-FXIRx, and additional clinical data on the LICA drugs we are developing. All of these activities build upon the value we have created and provide us with an event-rich year ahead," concluded Ms. Parshall.
Ionis' 2016 goals and the full list of corporate and drug development highlights can be found at the end of this press release prior to the financial tables.
Financial Results
"Over the past several years, we have ended each year in a better financial position than when we began. During this time, we advanced three important new drugs into Phase 3 development, initiated five Phase 3 studies on these drugs, advanced 12 Phase 2 drugs and multiple Phase 1 drugs, and translated our technological innovations into the next wave of drugs to enter clinical development. Over the last several years, we have consistently increased our revenues and cash balance while decreasing our pro forma NOL reflecting the successes in our partnerships. We continued this trend in 2015 by significantly outperforming both our pro forma NOL and cash guidance. We ended the year with a pro forma NOL of
"In 2016, we plan to continue to progress the drugs we have in development, including our Phase 3 drugs for which we are conducting multiple Phase 3 studies. We also plan to expand our pipeline and continue to invest in advancing our technology. Akcea plans to continue conducting the pre-commercialization activities and building the global medical, marketing and sales infrastructure to successfully commercialize volanesorsen. The efficiency of our technology and our business strategy allows us to do all of this while continuing to be fiscally prudent. We are also able to advance this large agenda while maintaining a strong cash balance because of the cash we generate as our partnered programs progress. As such, we are projecting a pro forma NOL in the low
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.
Revenue
Revenue for the three and twelve months ended
$91.2 million from Bayer in connection with its exclusive license agreement for IONIS-FXIRx;$72.6 million from Biogen for advancing the Phase 3 program for nusinersen, advancing IONIS-DMPK-2.5Rx and IONIS-BIIB4Rx, and validating three new targets for neurological disorders, which have proceeded forward in drug development;$22 million fromRoche for initiating a Phase 1/2 study of IONIS-HTTRx;$20 million from GSK for advancing the Phase 3 program of IONIS-TTRRx and initiating a Phase 1 study of IONIS-GSK4-LRx; and$77.9 million primarily from the amortization of upfront fees and manufacturing services performed for its partners.
Already in the first quarter of 2016, Ionis has generated
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 milestone payments, license fees and upfront fees.
Operating Expenses
In 2015, Ionis had higher operating expenses compared to 2014 primarily due to increased spending to support the Company's ongoing Phase 3 studies for nusinersen, IONIS-TTRRx and volanesorsen, which are in the most expensive stage of development. In addition, Akcea continued to build its operations in preparation for the commercial launch of volanesorsen. As such, Ionis' pro forma operating expenses of
Gain on Investment in
In the third quarter of 2015, Ionis received nearly
Net 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 familial chylomicronemia syndrome and 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 December, 2015, the Company changed its name from
Ionis Pharmaceuticals™ is a trademark of
In 2016, Ionis plans to achieve the following goals itself and with its partners:
- Advance the pipeline:
- Report clinical data on multiple drugs, including
- Phase 2 data for IONIS-FXIRx in patients with end-stage renal disease.
- Phase 2 open-label data for nusinersen in infants with SMA.
- Open-label extension data for IONIS-TTRRx.
- Phase 2 data for IONIS-TTRRx from an investigator initiated study in patients with the cardiomyopathy form of TTR amyloidosis.
- Phase 2 data from two drugs in Ionis' cancer franchise, IONIS-STAT3-2.5Rx and IONIS-AR-2.5Rx.
- Report clinical data on multiple drugs, including
- Initiate multiple clinical studies, including
- CARDIO-TTR, a Phase 3 study of IONIS-TTRRx in patients with the cardiomyopathy form of TTR amyloidosis, which GSK will conduct.
- A Phase 3 study of IONIS-TTRRx in
Japan in patients with FAP. - Numerous additional studies.
- Broaden the pipeline by adding three to five new drugs into development.
- Continue to advance the technology and show its value by reporting clinical data from drugs incorporating Ionis' LICA and Generation 2.5 technologies.
- Continue to successfully advance Ionis' business to generate revenue and cash.
(2015 and subsequent activities)
Corporate Highlights
- Ionis formed a wholly owned subsidiary, Akcea Therapeutics, to develop and commercialize its lipid drugs, volanesorsen, IONIS-APOCIII-LRx, IONIS-APO(a)-LRx and IONIS-ANGPTL3-LRx.
- Ionis licensed IONIS-FXIRx to
Bayer HealthCare to develop and commercialize IONIS-FXIRx for the prevention of thrombosis. - Ionis and
AstraZeneca formed a strategic collaboration to discover and develop antisense therapies for treating cardiovascular and metabolic diseases, primarily focused on targets in the kidney, and renal diseases. - Ionis formed an alliance with Janssen to discover and develop antisense drugs to treat autoimmune disorders of the gastrointestinal tract.
- Ionis received more than
$320 million in payments from partners in 2015. - Isis changed its name to
Ionis Pharmaceuticals, Inc. inDecember 2015 and its stock now trades under the ticker symbol "IONS".
Drug Development Highlights
- Ionis continued to make significant advances in its pipeline and completed target enrollment for three pivotal phase 3 studies, including:
- CHERISH, a Phase 3 study evaluating nusinersen in children with spinal muscular atrophy (SMA).
- NEURO-TTR, a Phase 3 study evaluating IONIS-TTRRx in patients with familial amyloid polyneuropathy (FAP).
- APROACH, a Phase 3 study evaluating volanesorsen in patients with familial chylomicronemia syndrome (FCS).
- Ionis and its partners reported positive data from 13 clinical studies. These data exemplify the broad applicability and potential for antisense drugs to provide therapeutic benefit for many different diseases. These data include:
- Phase 2 data from two ongoing open-label studies in which infants and children with SMA treated with nusinersen experienced increases in muscle function scores. Additionally, there were no events of death or permanent ventilation reported in 2015 in nusinersen-treated infants in the ongoing Phase 2 clinical study.
- Data from the ongoing open-label extension study of NEURO-TTR in which patients with FAP treated with IONIS-TTRRx for at least three months experienced reductions in TTR protein of up to 92 percent with a mean maximum (nadir) reduction of 76 percent compared to baseline.
- Phase 2 data from an ongoing open-label, investigator-initiated study in patients with familial amyloid cardiomyopathy and patients with wild-type transthyretin amyloidosis treated with IONIS-TTRRx for 12 months preliminarily evidencing disease stabilization and sustained TTR reductions.
- Phase 2 data in which patients with high lipoprotein(a), or Lp(a), treated with IONIS-APO(a)Rx experienced reductions in Lp(a) of up to 94 percent.
- Phase 1/2 data in which patients with high Lp(a) treated with IONIS-APO(a)-LRx experienced a greater than 30-fold increase in potency over IONIS-APO(a)Rx, the non-LICA Lp(a) drug. Patients also experienced dose-dependent reductions in Lp(a) of up to 97 percent and 99 percent after a single dose and multiple doses of IONIS-APO(a)-LRx, respectively.
- Clinical and preclinical data in patients with cancer, including advanced/metastatic hepatocellular carcinoma and diffuse large B cell lymphoma, treated with IONIS-STAT3-2.5Rx evidencing antitumor activity.
- Phase 2 data in which patients with type 2 diabetes treated with IONIS-PTP1BRx experienced statistically significant mean reductions in body weight and HbA1c (0.7 percentage point).
- Phase 1 results in which healthy volunteers dosed with IONIS-ANGPTL3Rx experienced significant reductions of up to 93 percent in angiopoietin-like 3 protein, up to 63 percent in triglycerides and up to 46 percent in total cholesterol.
- Phase 1 results in which healthy volunteers dosed with IONIS-PKKRx experienced significant, dose-dependent reductions of prekallikrein of up to 95 percent.
- Phase 3 data from the FOCUS FH study evaluating Kynamro (mipomersen sodium) in patients with severe heterozygous familial hypercholesterolemia. This study met its primary endpoint with a statistically significant reduction of LDL-C.
- Ionis published clinical data from its novel lipid drugs, volanesorsen and IONIS-APO(a)Rx, in the
New England Journal of Medicine and The Lancet , respectively. - The US FDA granted volanesorsen orphan drug designation for the treatment of patients with FCS.
The European Medicines Agency granted IONIS-HTTRx orphan drug designation for the treatment of patients with Huntington's Disease.- Ionis, together with its partners, continued to advance its pipeline of drugs, initiating 11 clinical studies, including one Phase 3 study and six Phase 2 studies.
IONIS PHARMACEUTICALS, INC. |
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Three months ended, |
Year ended, |
||||||||
December 31, |
December 31, |
||||||||
2015 |
2014 |
2015 |
2014 |
||||||
Revenue: |
(unaudited) |
||||||||
Research and development revenue under collaborative agreements |
$50,891 |
$82,539 |
$281,360 |
$202,514 |
|||||
Licensing and royalty revenue |
679 |
2,322 |
2,343 |
11,647 |
|||||
Total revenue |
51,571 |
84,861 |
283,703 |
214,161 |
|||||
Expenses: |
|||||||||
Research, development and patent expenses |
101,330 |
67,953 |
322,292 |
241,751 |
|||||
General and administrative |
13,181 |
6,828 |
37,173 |
20,140 |
|||||
Total operating expenses |
114,511 |
74,781 |
359,465 |
261,891 |
|||||
Income (loss) from operations |
(62,940) |
10,080 |
(75,762) |
(47,730) |
|||||
Other income (expense): |
|||||||||
Investment income |
1,355 |
679 |
4,302 |
2,682 |
|||||
Interest expense |
(9,351) |
(7,305) |
(36,732) |
(22,209) |
|||||
Gain on investments, net |
(125) |
1,116 |
75 |
1,256 |
|||||
Gain on investment in Regulus Therapeutics, Inc. |
- |
19,366 |
20,211 |
19,902 |
|||||
Loss on early retirement of debt |
- |
(8,292) |
- |
(8,292) |
|||||
Income (loss) before income tax (expense) benefit |
(70,061) |
15,644 |
(87,906) |
(54,391) |
|||||
Income tax (expense) benefit |
(372) |
15,409 |
(372) |
15,407 |
|||||
Net income (loss) |
$(71,433) |
$31,053 |
$(88,278) |
$(38,984) |
|||||
Basic net income (loss) per share |
$(0.59) |
$0.26 |
$(0.74) |
$(0.33) |
|||||
Diluted net income (loss) per share |
$(0.59) |
$0.25 |
$(0.74) |
$(0.33) |
|||||
Shares used in computing basic net income (loss) per share |
120,189 |
118,223 |
119,719 |
117,691 |
|||||
Shares used in computing diluted net income (loss)per share |
120,189 |
122,839 |
119,719 |
117,691 |
|||||
Ionis Pharmaceuticals, Inc. Reconciliation of GAAP to Pro Forma Basis: Condensed Consolidated Operating Expenses, Income (Loss) From Operations, and Net Income (Loss) (In Thousands) |
|||||||||||||
Three months ended, December 31, |
Year ended, December 31, |
||||||||||||
2015 |
2014 |
2015 |
2014 |
||||||||||
(unaudited) |
(unaudited) |
||||||||||||
As reported operating expenses according to GAAP |
$114,511 |
$74,781 |
$359,465 |
$261,891 |
|||||||||
Excluding compensation expense related to equity awards |
(17,408) |
(8,488) |
(59,314) |
(31,383) |
|||||||||
Pro forma operating expenses |
$97,103 |
$66,293 |
$300,151 |
$230,508 |
|||||||||
As reported income (loss) from operations according to GAAP |
$(62,940) |
$10,080 |
$(75,762) |
$(47,730) |
|||||||||
Excluding compensation expense related to equity awards |
(17,408) |
(8,488) |
(59,314) |
(31,383) |
|||||||||
Pro forma income (loss) from operations |
$(45,532) |
$18,568 |
$(16,448) |
$(16,347) |
|||||||||
As reported net income (loss) according to GAAP |
$(71,433) |
$31,053 |
$(88,278) |
$(38,984) |
|||||||||
Excluding compensation expense related to equity awards |
(17,408) |
(8,488) |
(59,314) |
(31,383) |
|||||||||
Pro forma net income (loss) |
$(54,025) |
$39,541 |
$(28,964) |
$(7,601) |
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, |
||||
2015 |
2014 |
||||
Assets: |
|||||
Cash, cash equivalents and short-term investments |
$779,183 |
$728,832 |
|||
Investment in Regulus Therapeutics Inc. |
24,792 |
81,881 |
|||
Other current assets |
33,028 |
25,884 |
|||
Property, plant and equipment, net |
90,233 |
88,958 |
|||
Other assets |
28,869 |
30,254 |
|||
Total assets |
$956,105 |
$955,809 |
|||
Liabilities and stockholders' equity: |
|||||
Other current liabilities |
$81,554 |
$63,619 |
|||
Current portion of deferred contract revenue |
67,322 |
51,713 |
|||
1% convertible senior notes |
347,214 |
327,486 |
|||
2 3/4% convertible senior notes |
50,361 |
48,014 |
|||
Long-term obligations, less current portion |
74,558 |
79,400 |
|||
Long-term deferred contract revenue |
134,306 |
127,797 |
|||
Stockholders' equity |
200,790 |
257,780 |
|||
Total liabilities and stockholders' equity |
$956,105 |
$955,809 |
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SOURCE
D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741, or Amy Williford, Ph.D., Associate Director, Corporate Communications, 760-603-2772