Press Releases
Isis Pharmaceuticals Reports Financial Results and Highlights for Third Quarter 2014
"We have had a very successful year and, with a number of exciting events on the horizon, the remainder of 2014 promises to be equally eventful. Just last month, we reported encouraging results from our ISIS-SMNRx drug in infants and children with spinal muscular atrophy. We initiated the Phase 3 study on ISIS-SMNRx in infants with SMA this summer and plan to initiate the Phase 3 study in children with SMA by the end of this year. We continue to treat patients with the polyneuropathy form of transthyretin amyloidosis in our Phase 3 study of ISIS-TTRRx. Many patients have completed all 15 months of treatment in the Phase 3 ISIS-TTRRx study and are receiving drug in the open-label extension study. We also initiated a Phase 3 clinical trial of ISIS-APOCIIIRx for patients with familial chylomicronemia syndrome, a rare genetic disorder resulting in extremely high triglyceride levels. We are pleased to now be conducting three Phase 3 programs, while we also mature the rest of our pipeline. We have a number of additional activities related to our pipeline to look forward to before the end of the year," said
"The success of the drugs in our pipeline has contributed significantly to our financial performance this year and is strong evidence that our business strategy is working. Our business model is designed to generate substantial amounts of cash and revenue from our partners as our programs succeed. Already this year, including to date in the fourth quarter, we have generated more than
Upcoming Key Milestones
- Initiate an additional Phase 3 clinical study on ISIS-APOCIIIRx in patients with high triglycerides and on ISIS-SMNRx in children with SMA.
- Report the full data analysis of the Phase 2 study of ISIS-FXIRx at an upcoming medical meeting.
- Report Phase 2 data from ISIS-PTP1BRx and ISIS-GCCRRx in patients with type 2 diabetes.
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 nine months ended
Isis also earned
$43 million fromBiogen Idec related to advancing ISIS-SMNRx, initiating a Phase 1 study for ISIS-DMPKRx, and validating an undisclosed target to treat a neurological disorder;$15 million fromAstraZeneca related to initiating a Phase 1 clinical study of ISIS-ARRx;$4.5 million from GSK related to advancing the Phase 3 study of ISIS-TTRRx and advancing ISIS-GSK4Rx; and$4 million fromAchaogen whenAchaogen initiated a Phase 3 study of plazomicin.
Already in the fourth quarter, Isis has earned
Operating Expenses
On a pro forma basis, Isis' operating expenses for the three and nine months ended
Net Loss
Isis reported a net loss of
Balance Sheet
As of
Business Highlights
"We have made significant progress this year maturing our pipeline. Most notably, we have reported a substantial amount of clinical data from drugs to treat patients with a diverse range of diseases, including metabolic, cardiovascular and severe and rare diseases. We and our partners reported Phase 2 results from eight drugs, initiated Phase 3 studies on three drugs, began Phase 1 and 2 studies on six drugs and moved three drugs into development. The maturation of our pipeline ensures that we will have a continuous stream of clinical events to highlight," continued Ms. Parshall.
"As our pipeline of partnered drugs has matured, the potential milestone payments we can earn from our partners become more substantial. This year we have had numerous pipeline advances in many different partnered programs, which have resulted in significant payments from our partners this year. In addition to payments we receive as partnered drugs advance in development, we benefit from the successes of our satellite companies, which expand the application of our technology platform into areas of RNA therapeutics that are outside our core focus. The benefit of this strategy was most recently illustrated by the significant increase in the value of
Corporate and Drug Development Highlights from the Third Quarter and Early Fourth Quarter
- Isis reported positive clinical results for ISIS-SMNRx from two open-label Phase 2 studies in infants and children with spinal muscular atrophy. These data were presented at the
World Muscle Society Congress .- Isis reported that the median event-free age of infants with SMA on
September 2, 2014 compared favorably to that of infants with SMA in the PNCR natural history study. - Isis reported that time- and dose-dependent increases in muscle function scores were observed in both infants and children with SMA.
- Isis presented clinical data showing that ISIS-SMNRx is distributed throughout the spinal cord and neurons with greater amounts of full-length SMN2 mRNA and SMN protein in tissues from ISIS-SMNRx-treated infants compared to the amounts of full-length SMN2 mRNA and SMN protein in the tissues analyzed from untreated SMA infants.
- Isis reported that the median event-free age of infants with SMA on
- Isis' collaborators reported positive clinical results from three drugs in Isis' lipid franchise. These data were presented at the 2014
European Society of Cardiology Congress .- Dr.
John Kastelein , M.D., Ph.D. presented data from a retrospective analysis of 104 patients with familial hypercholesterolemia treated for one year with KYNAMRO® (mipomersen sodium) injection in the long-term extension study showing that patients treated with KYNAMRO experienced a reduction in Major Adverse Cardiovascular Events (MACE) from 25.72/1000 months (in the two years prior to KYNAMRO treatment) to 4.85/1000 months. - Dr.
John Kastelein provided an overview of the Phase 2 program for ISIS-APOCIIIRx in which treatment with ISIS-APOCIIIRx produced consistent, robust and statistically significant reductions in triglycerides, apoC-III and non-HDL-Cholesterol and increases in HDL-Cholesterol in all patient populations evaluated. - Dr.
Sotirios Tsimikas , M.D. presented data from the Phase 1 study in which treatment with ISIS-APO(a)Rx produced dose-dependent and significant reductions in Lp(a) levels in healthy volunteers.
- Dr.
- Isis' partner,
Regulus Therapeutics , reported positive interim results on RG-101, an anti-miR drug in development to treat patients with hepatitis C virus.- Regulus reported a single dose of RG-101 demonstrated a substantial mean reduction in viral load in patients with varied hepatitis C virus (HCV) genotypes and treatment history.
- Together with its partners, Isis continued to advance its pipeline of drugs.
- Isis initiated ENDEAR, the Phase 3 study evaluating ISIS-SMNRx in infants with SMA.
- Isis initiated APPROACH, the Phase 3 study evaluating ISIS-APOCIIIRx in patients with familial chylomicronemia syndrome.
Achaogen initiated a Phase 3 study of plazomicin in patients with serious multi-drug resistant, gram-negative bacterial infections.- Isis added a new drug, ISIS-BIIB3Rx, to its pipeline. ISIS-BIIB3Rx is part of Isis' strategic neurology alliance with
Biogen Idec and is in development to treat an undisclosed neurodegenerative disease. - Isis added a new drug, ISIS-GSK5Rx, to its pipeline. ISIS-GSK5Rx is part of Isis' alliance with GSK and is in development to treat an undisclosed ocular disease
- Isis added a new drug, ISIS-HTTRx, to its pipeline. ISIS-HTTRx is part of Isis' alliance with
Roche and is in development to treat patients with Huntington's Disease.
Conference Call
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ABOUT
Isis is exploiting its leadership position in antisense technology to discover and develop novel drugs for its product pipeline and for its partners. Isis' broad pipeline consists of 34 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, |
Nine months ended, |
|||||||
September 30, |
September 30, |
|||||||
2014 |
2013 |
2014 |
2013 |
|||||
Revenue: |
(unaudited) |
(unaudited) |
||||||
Research and development revenue under collaborative agreements |
$43,798 |
$23,258 |
$119,975 |
$102,543 |
||||
Licensing and royalty revenue |
265 |
327 |
9,325 |
2,493 |
||||
Total revenue |
44,063 |
23,585 |
129,300 |
105,036 |
||||
Expenses: |
||||||||
Research, development and patent expenses |
61,086 |
45,660 |
173,798 |
126,603 |
||||
General and administrative |
4,470 |
3,430 |
13,313 |
10,241 |
||||
Total operating expenses |
65,556 |
49,090 |
187,111 |
136,844 |
||||
Loss from operations |
(21,493) |
(25,505) |
(57,811) |
(31,808) |
||||
Other income (expense): |
||||||||
Investment income |
675 |
434 |
2,003 |
1,400 |
||||
Interest expense |
(4,998) |
(4,867) |
(14,902) |
(14,470) |
||||
Gain on investments, net |
538 |
175 |
675 |
2,073 |
||||
Loss before income tax (expense) benefit |
(25,278) |
(29,763) |
(70,035) |
(42,805) |
||||
Income tax (expense) benefit |
(1,398) |
5,193 |
(2) |
6,437 |
||||
Net loss |
$(26,676) |
$(24,570) |
$(70,037) |
$(36,368) |
||||
Basic and diluted net loss per share |
$(0.23) |
$(0.21) |
$(0.60) |
$(0.33) |
||||
Shares used in computing basic and diluted net loss per share |
117,811 |
115,263 |
117,511 |
108,608 |
Isis Pharmaceuticals, Inc. Reconciliation of GAAP to Pro Forma Basis: Condensed Consolidated Operating Expenses, (Income) Loss From Operations, and Net Loss (In Thousands) |
|||||||||||
Three months ended, September 30, |
Nine months ended, September 30, |
||||||||||
2014 |
2013 |
2014 |
2013 |
||||||||
(unaudited) |
(unaudited) |
||||||||||
As reported operating expenses according to GAAP |
$65,556 |
$49,090 |
$187,111 |
$136,844 |
|||||||
Excluding compensation expense related to equity awards |
(8,118) |
(2,812) |
(22,894) |
(8,318) |
|||||||
Pro forma operating expenses |
$57,438 |
$46,278 |
$164,217 |
$128,526 |
|||||||
As reported loss from operations according to GAAP |
$(21,493) |
$(25,505) |
$(57,811) |
$(31,808) |
|||||||
Excluding compensation expense related to equity awards |
(8,118) |
(2,812) |
(22,894) |
(8,318) |
|||||||
Pro forma income (loss) from operations |
$(13,375) |
$(22,693) |
$(34,917) |
$(23,490) |
|||||||
As reported net loss according to GAAP |
$(26,676) |
$(24,570) |
$(70,037) |
$(36,368) |
|||||||
Excluding compensation expense related to equity awards |
(8,118) |
(2,812) |
(22,894) |
(8,318) |
|||||||
Pro forma net loss |
$(18,558) |
$(21,758) |
$(47,143) |
$(28,050) |
|||||||
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 proforma net 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 |
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(In Thousands) |
|||||
September 30, |
December 31, |
||||
2014 |
2013 |
||||
(unaudited) |
|||||
Assets: |
|||||
Cash, cash equivalents and short-term investments |
$591,986 |
$656,761 |
|||
Investment in Regulus Therapeutics Inc. |
47,426 |
52,096 |
|||
Other current assets |
41,751 |
26,653 |
|||
Property, plant and equipment, net |
88,068 |
86,198 |
|||
Other assets |
24,572 |
25,448 |
|||
Total assets |
$793,803 |
$847,156 |
|||
Liabilities and stockholders' equity: |
|||||
Other current liabilities |
$55,598 |
$49,677 |
|||
Current portion of deferred contract revenue |
51,727 |
48,135 |
|||
2 3/4% convertible senior notes |
155,437 |
150,334 |
|||
Long-term obligations, less current portion |
75,655 |
77,830 |
|||
Long-term deferred contract revenue |
110,614 |
142,790 |
|||
Stockholders' equity |
344,772 |
378,390 |
|||
Total liabilities and stockholders' equity |
793,803 |
$847,156 |
|||
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SOURCE
Isis Pharmaceuticals' Contacts: D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741 or Amy Blackley, Ph.D., Associate Director, Corporate Communications, 760-603-2772