Press Releases
Isis Reports Financial Results and Highlights for 2014
"2014 was another year of significant achievements for Isis with successes in every aspect of our business. We continued to advance our large pipeline of first-in-class or best-in-class drugs, including initiating Phase 3 programs for ISIS-APOCIIIRx and ISIS-SMNRx and progressing the Phase 3 study of ISIS-TTRRx. Each of these drugs represents a potential near term commercial opportunity with Phase 3 data planned for the 2016/2017 timeframe. We and our partners reported clinical data from 18 studies in 2014. It is a reflection of the efficiency and effectiveness of our technology that 15 of these studies were positive. Of particular note, the clinical study results from two of our drugs, ISIS-APOCIIIRx and ISIS-FXIRx, were published in two separate papers in the
"We took the next step in the evolution of our business strategy with the formation of our wholly owned subsidiary, Akcea Therapeutics. One of the most important contributions the Akcea team has made to date is the addition of a second ultra-rare indication for ISIS-APOCIIIRx. This new indication is to treat patients with partial lipodystrophy. We believe this new indication could double the initial patient population for ISIS-APOCIIIRx. We plan to initiate a Phase 3 study in patients with partial lipodystrophy rapidly, which should complete roughly in parallel with the Phase 3 FCS study. Both FCS and partial lipodystrophy are ultra-orphan indications with limited or no approved therapeutic options. With the Akcea team's commercial expertise, we believe we can develop a pricing and reimbursement strategy that supports the significant value ISIS-APOCIIIRx can potentially bring to these patients. All of these successes have established a strong base upon which to build in 2015," concluded Ms. Parshall.
"Our financial performance in 2014 was a result of the successful execution of our business strategy, which ensures that we benefit from the achievements of the drugs in our pipeline and our continued innovation in developing RNA-targeted drugs. We received more than
"We are carrying this momentum into 2015. We plan to continue to progress the drugs we have in pivotal Phase 3 studies, initiate additional Phase 3 studies, and advance our drugs that are in or entering early and mid-stage development. We also plan to expand our pipeline and continue to invest in advancing our technology. Although our clinical programs are increasing in size and cost, we are projecting a pro forma NOL in the mid
Financial Results
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
$80 million fromBiogen Idec for advancing ISIS-SMNRx, including initiating two Phase 3 studies, initiating a Phase 1 study of ISIS-DMPK-2.5Rx, validating two undisclosed targets to treat neurological disorders under its neurology collaborations, and advancing a third drug into development;$29 million from GSK for advancing the Phase 2/3 study of ISIS-TTRRx and further advancing ISIS-HBVRx, ISIS-GSK4-LRx, and ISIS-RHO-2.5Rx (formerly ISIS-GSK5-2.5Rx);$22 million fromAstraZeneca for initiating a Phase 1 clinical study of ISIS-AR-2.5Rx and advancing ISIS-STAT3-2.5Rx; and$4 million fromAchaogen whenAchaogen initiated a Phase 3 study of plazomicin.
Isis' revenue in 2014 also included nearly
Operating Expenses
As projected, Isis' pro forma operating expenses of
On a GAAP basis, Isis' operating expenses for the three and twelve months ended
Loss on Retirement of Debt
In
Gain on Investment in
In
Isis' total proceeds from sales of stock in its satellite companies were more than
Income Tax Benefit
Isis recognized a net tax benefit of
Net Income (Loss)
Isis reported net income of
Balance Sheet
As of
In
Drug Development Highlights in 2014 and early 2015
- Isis and its partners reported data from 20 clinical studies of which 17 were positive, including:
- A retrospective analysis from a Phase 3 long-term extension study in which patients with familial hypercholesterolemia treated with KYNAMRO (mipomersen sodium) injection for a mean of one or two years had a significant reduction in major adverse cardiovascular events (MACE) compared to two years prior to therapy.
- Four data presentations from Phase 2 studies in which patients with high to extremely high triglyceride levels treated with ISIS-APOCIIIRx experienced significant reductions in triglycerides, and patients with elevated triglycerides and type 2 diabetes treated with ISIS-APOCIIIRx experienced significant reductions in both triglycerides and HbA1c.
- Phase 2 data from two open-label studies in which infants and children with SMA treated with ISIS-SMNRx experienced time- and dose-dependent increases in muscle function. In addition, the median event-free age of ISIS-SMNRx-treated infants with SMA compared favorably to that of infants with SMA in a PNCR natural history study.
- Phase 2 data in which ISIS-FXIRx-treated patients undergoing total knee replacement surgery experienced a seven-fold lower incidence of venous thromboembolism and numerically fewer bleeding events compared to patients treated with enoxaparin.
- Phase 2 data in which ISIS-GCGRRx-treated patients with type 2 diabetes uncontrolled on stable metformin therapy experienced up to a 2.25 percentage point mean reduction in HbA1c levels after 13 weeks of treatment with ISIS-GCGRRx.
- Phase 2 top line data in which ISIS-PTP1BRx-treated patients with type 2 diabetes who were uncontrolled on metformin with or without sulfonylurea experienced statistically significant mean reductions in body weight and HbA1c (0.7 percentage point) at 36 weeks.
- Phase 1/2 data in which ISIS-STAT3-2.5Rx–treated patients with cancer experienced preliminary evidence of antitumor activity.
- Phase 1 data in which ISIS-APO(a)Rx treatment produced dose-dependent and significant reductions in Lp(a) levels in normal volunteers.
- Phase 2 data in which ISIS-PKKRx treatment produced significant, dose-dependent reductions of prekallikrein, or PKK, of up to 95 percent in healthy volunteers.
- ISIS-DMPK-2.5Rx was granted orphan drug status for the treatment of myotonic dystrophy type 1 by the
U.S. Food and Drug Administration . - Isis, together with its partners, continued to advance its pipeline of drugs, initiating 12 clinical studies, including four Phase 3 studies and four Phase 2 studies.
- Isis added 12 drugs to its pipeline.
Corporate Highlights in 2014 and early 2015
- Isis formed a wholly owned subsidiary,
Akcea Therapeutics Inc. , to develop and commercialize its lipid drugs, ISIS-APOCIIIRx, ISIS-APO(a)Rx, ISIS-ANGPTL3Rx and any follow-on drugs for these programs. - Isis formed an alliance with Janssen to discover and develop antisense drugs to treat autoimmune disorders of the gastrointestinal tract.
- Isis formed an alliance with
AstraZeneca to discover and develop novel delivery methods for antisense oligonucleotides. - Isis and Alnylam formed a new agreement that included a cross-license of intellectual property on four disease targets, providing each company with exclusive RNA therapeutic license rights for two programs.
- Isis generated more than
$250 million in payments from its partners and received proceeds of more than$25 million through the sale of stock it owned in its satellite company partners. - Isis and its partners were recognized by the drug development community for their innovative and collaborative alliances and their commitment to developing drugs to treat patients with serious, unmet medical needs.
- Isis and Genzyme received the 2014 Partners in Progress Corporate Award from the
National Organization for Rare Disorders for the development and approval of KYNAMRO. - Isis' innovative collaboration with
Biogen Idec was voted breakthrough alliance of 2014 by Thomson Reuters Recap.
- Isis and Genzyme received the 2014 Partners in Progress Corporate Award from the
- Isis' senior vice president of research,
Frank Bennett , Ph.D., was awarded the Commitment to a Cure Award by theALS Association for his research and commitment to develop a treatment for amyotrophic lateral sclerosis. - Isis' founder, CEO and chairman of the board of directors,
Stanley T. Crooke , M.D., Ph.D., was recognized with several awards.- The prestigious SCRIP Lifetime Achievement Award.
- The SMA Breakthrough Award by CURE SMA.
2015 Goals
In 2015, Isis plans to achieve the following goals itself and with its partners:
- Advance the pipeline:
- Report clinical data on eight or more drugs, including:
- An update on the Phase 2 open-label study for ISIS-SMNRx in infants with SMA
- Phase 2 data for ISIS-PTP1BRx and ISIS-GCCRRx in patients with type 2 diabetes
- Phase 2 data for ISIS-STAT3-2.5Rx (AZD9150) in patients with lymphoma
- Phase 1/2 data for ISIS-AR-2.5Rx (AZD5312) in patients with androgen receptor tumors, such as prostate cancer
- Phase 3 FOCUS FH study data for KYNAMRO
- Phase 2 data on ISIS-APO(a)Rx in patients with high Lp(a)
- Phase 2 data on ISIS-DMPK-2.5Rx in patients with myotonic dystrophy type 1
- A number of Phase 1 data readouts
- Initiate up to 13 clinical studies, including a Phase 3 study on ISIS-APOCIIIRx in patients with partial lipodystrophy and numerous Phase 2 studies, including:
- ISIS-FGFR4Rx in obese patients
- ISIS-FXIRx in patients with renal disease
- ISIS-GCGRRx in patients with type 2 diabetes (dose optimization)
- ISIS-HBVRx in patients with hepatitis B viral infection
- ISIS-HTTRx in patients with Huntington's Disease
- ISIS-PKKRx in patients with hereditary angioedema
- Report clinical data on eight or more drugs, including:
- Broaden the pipeline by adding up to five new drugs in both partnered and unpartnered programs.
- Continue to successfully execute the business strategy to generate revenue and cash.
Conference Call
At
ABOUT
Isis is exploiting its leadership position in RNA-targeted technology to discover and develop novel drugs for its product pipeline and for its partners. Isis' broad pipeline consists of 38 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, including neurological disorders, and cancer. Isis' partner, Genzyme, is commercializing Isis' lead product, KYNAMRO®, in
FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding
In this press release, unless the context requires otherwise, "Isis," "Company," "we," "our," and "us" refers to
ISIS PHARMACEUTICALS, INC. |
|||||||||
Three months ended, |
Years ended, |
||||||||
December 31, |
December 31, |
||||||||
2014 |
2013 |
2014 |
2013 |
||||||
Revenue: |
(unaudited) |
||||||||
Research and development revenue under collaborative agreements |
$82,539 |
$41,275 |
$202,514 |
$144,194 |
|||||
Licensing and royalty revenue |
2,322 |
973 |
11,647 |
3,091 |
|||||
Total revenue |
84,861 |
42,248 |
214,161 |
147,285 |
|||||
Expenses: |
|||||||||
Research, development and patent expenses |
67,953 |
57,430 |
241,751 |
184,033 |
|||||
General and administrative |
6,828 |
4,676 |
20,140 |
14,918 |
|||||
Total operating expenses |
74,781 |
62,106 |
261,891 |
198,951 |
|||||
Income (loss) from operations |
10,080 |
(19,858) |
(47,730) |
(51,666) |
|||||
Other income (expense): |
|||||||||
Investment income |
679 |
686 |
2,682 |
2,085 |
|||||
Interest expense |
(7,305) |
(4,885) |
(22,209) |
(19,355) |
|||||
Gain on investments, net |
1,116 |
305 |
1,256 |
2,378 |
|||||
Gain on investment in Regulus Therapeutics, Inc. |
19,366 |
- |
19,902 |
- |
|||||
Loss on early retirement of debt |
(8,292) |
- |
(8,292) |
- |
|||||
Income (loss) before income tax benefit |
15,644 |
(23,752) |
(54,391) |
(66,558) |
|||||
Income tax benefit |
15,409 |
(524) |
15,407 |
5,914 |
|||||
Net income (loss) |
$31,053 |
$(24,276) |
(38,984) |
$(60,644) |
|||||
Basic net income (loss) per share |
$0.26 |
$(0.21) |
$(0.33) |
$(0.55) |
|||||
Diluted net income (loss) per share |
$0.25 |
$(0.21) |
$(0.33) |
$(0.55) |
|||||
Shares used in computing basic net income (loss) per share |
118,223 |
116,122 |
117,691 |
110,502 |
|||||
Shares used in computing diluted net income (loss) per share |
122,839 |
116,122 |
117,691 |
110,502 |
Isis 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, |
Years ended, December 31, |
|||||||||
2014 |
2013 |
2014 |
2013 |
|||||||
(unaudited) |
(unaudited) |
|||||||||
As reported operating expenses according to GAAP |
$74,781 |
$62,106 |
$261,891 |
$198,951 |
||||||
Excluding compensation expense related to equity awards |
(8,488) |
(3,101) |
(31,383) |
(11,418) |
||||||
Pro forma operating expenses |
$66,293 |
$59,005 |
$230,508 |
$187,533 |
||||||
As reported income (loss) from operations according to GAAP |
$10,080 |
$(19,858) |
$(47,730) |
$(51,666) |
||||||
Excluding compensation expense related to equity awards |
(8,488) |
(3,101) |
(31,383) |
(11,418) |
||||||
Pro forma income (loss) from operations |
$18,568 |
$(16,757) |
$(16,347) |
$(40,248) |
||||||
As reported net income (loss) according to GAAP |
$31,053 |
$(24,276) |
$(38,984) |
$(60,644) |
||||||
Excluding compensation expense related to equity awards |
(8,488) |
(3,101) |
(31,383) |
(11,418) |
||||||
Pro forma net income (loss) |
$39,541 |
$(21,175) |
$(7,601) |
$(49,226) |
||||||
Reconciliation of GAAP to Pro Forma Basis
As illustrated in the Selected Financial Information in this press release, pro forma operating expenses, pro forma 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. Isis 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. Isis 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 Isis' pro forma results is consistent with how Isis' management internally evaluates the performance of its operations.
Isis Pharmaceuticals, Inc. Condensed Consolidated Balance Sheets |
|||||
(In Thousands) |
|||||
December 31, |
December 31, |
||||
2014 |
2013 |
||||
Assets: |
|||||
Cash, cash equivalents and short-term investments |
$728,832 |
$656,761 |
|||
Investment in Regulus Therapeutics Inc. |
81,881 |
52,096 |
|||
Other current assets |
25,884 |
26,653 |
|||
Property, plant and equipment, net |
88,958 |
86,198 |
|||
Other assets |
30,254 |
25,448 |
|||
Total assets |
$955,809 |
$847,156 |
|||
Liabilities and stockholders' equity: |
|||||
Other current liabilities |
$60,737 |
$49,677 |
|||
Current portion of deferred contract revenue |
54,595 |
48,135 |
|||
1% convertible senior notes |
327,486 |
- |
|||
2 ¾% convertible senior notes |
48,014 |
150,334 |
|||
Long-term obligations, less current portion |
79,400 |
77,830 |
|||
Long-term deferred contract revenue |
127,797 |
142,790 |
|||
Stockholders' equity |
257,780 |
378,390 |
|||
Total liabilities and stockholders' equity |
$955,809 |
$847,156 |
|||
Logo - http://photos.prnewswire.com/prnh/20130807/LA60006LOGO
To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/isis-reports-financial-results-and-highlights-for-2014-300042679.html
SOURCE
D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741 Amy Blackley, Ph.D., Associate Director, Corporate Communications, 760-603-2772