Press Releases
Ionis Pharmaceuticals Reports Financial Results and Highlights for Third Quarter 2016
"This week, we and Biogen announced positive data from an interim analysis of CHERISH, our Phase 3 study in children with later-onset (consistent with Type 2) spinal muscular atrophy (SMA). We are very encouraged with the positive SPINRAZATM data from both of our controlled Phase 3 clinical trials supporting potential benefit not only in infants, but also in children with SMA. We are pleased that our partners at Biogen are already making SPINRAZA available to patients with SMA who have no therapeutic alternatives through a broad Expanded Access Program. In about four weeks after Biogen filed for marketing approval for SPINRAZA, the
"In addition, last week we announced positive results from our Phase 2 study with IONIS-FXIRx in patients with end-stage renal disease on dialysis. Results from the study demonstrated robust, statistically significant reductions in Factor XI activity in treated patients. IONIS-FXIRx also displayed a good safety and tolerability profile. In patients treated with 200 mg or 300 mg of IONIS-FXIRx there were no clinically meaningful platelet declines and no increase in major or clinically relevant non-major bleeding. These results provide further support for the potential benefit IONIS-FXIRx could have for patients who need to prevent clotting but who have increased risk of bleeding," continued Ms. Parshall.
Financial Results
"We ended the third quarter of this year with net income primarily because of the
"Through the first nine months of 2016, we have continued to advance our Phase 3 programs and Akcea is preparing to commercialize volanesorsen while we have prudently managed our expenses. As a result, we finished the first nine months 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 nine months ended
$96.9 million from Biogen for licensing and advancing the Phase 3 program for SPINRAZA and advancing IONIS-BIIB4Rx;$15 million from Kastle Therapeutics for acquiring Kynamro;$10 million from Janssen for licensing IONIS-JBI1-2.5Rx;$1.5 million from GSK for advancing IONIS-HBV-LRx; and$62.9 million primarily from the amortization of upfront fees and manufacturing services Ionis performed for its partners.
Ionis' revenue in the first nine months 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.
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 SPINRAZA, IONIS-TTRRx and volanesorsen. In addition, Akcea continued to build a global organization and prepare for the commercial launch of volanesorsen. As such, Ionis' operating expenses on a GAAP basis for the three and nine months ended
Net Income (Loss)
Ionis reported net income 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 SPINRAZA (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
(Q3 2016 and subsequent activities)
Recent SPINRAZA (formerly nusinersen) Accomplishments:
- The
FDA accepted the NDA filing and granted Priority Review in the U.S. for SPINRAZA for the treatment of patients with SMA. - The EMA validated the MAA and granted Accelerated Assessment in the E.U. for SPINRAZA for the treatment of patients with SMA.
- Biogen and Ionis reported positive data from an interim analysis of the Phase 3 CHERISH study in patients with later-onset (consistent with Type 2) SMA.
- Biogen and Ionis presented new positive clinical data with SPINRAZA at the
World Muscle Society Congress supporting the companies' efforts to rapidly make SPINRAZA available to patients with SMA, including:- Safety results from the interim analysis of the Phase 3 ENDEAR study in patients with infantile-onset (consistent with Type 1) SMA;
- Encouraging preliminary results from NURTURE, a Phase 2 open-label study in pre-symptomatic infants; and
- A recent analysis of the ongoing Phase 2 open-label study in patients with later-onset SMA.
- Ionis reported positive data from an interim analysis of the ENDEAR Phase 3 study in patients with infantile-onset (consistent with Type 1) SMA. Biogen paid Ionis
$75 million to license SPINRAZA.
Recent
- 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. - Akcea and Ionis received Orphan Designation in the E.U. for volanesorsen for the treatment of patients with Familial Partial Lipodystrophy (FPL).
- Ionis reported positive, top-line Phase 2 data with IONIS-FXIRx demonstrating robust, statistically significant reductions in Factor XI activity in patients with ESRD receiving chronic hemodialysis. IONIS-FXIRx was safe and well tolerated in treated patients with no clinically meaningful platelet declines and no increase in major or clinically relevant non-major bleeding.
- 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-resistant prostate cancer patients. - Akcea and Ionis published clinical results with Lp(a)-lowering drugs, IONIS-APO(a)Rx and IONIS-APO(a)-LRx, in The Lancet demonstrating robust reductions in Lp(a) levels, regardless of a patient's starting Lp(a) level.
- 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 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.
Recent Corporate Highlights:
- Ionis' CEO, Dr.
Stanley Crooke , received two awards, theE. B. Hershberg Award from theAmerican Chemical Society and the Lifetime Achievement Award from theOligonucleotide Therapeutics Society recognizing his achievements in the field of oligonucleotide therapeutics. - 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 PHARMACEUTICALS, INC. |
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SELECTED FINANCIAL INFORMATION |
|||||||||
Condensed Consolidated Statements of Operations |
|||||||||
(In Thousands, Except Per Share Data) |
|||||||||
Three months ended, |
Nine months ended, |
||||||||
September 30, |
September 30, |
||||||||
2016 |
2015 |
2016 |
2015 |
||||||
Revenue: |
(unaudited) |
(unaudited) |
|||||||
Research and development revenue under collaborative agreements |
$108,913 |
$48,918 |
$166,583 |
$230,469 |
|||||
Licensing and royalty revenue |
2,014 |
203 |
19,689 |
1,664 |
|||||
Total revenue |
110,927 |
49,121 |
186,272 |
232,133 |
|||||
Expenses: |
|||||||||
Research, development and patent expenses |
84,631 |
88,508 |
243,169 |
220,962 |
|||||
General and administrative |
10,188 |
8,751 |
30,574 |
23,992 |
|||||
Total operating expenses |
94,819 |
97,259 |
273,743 |
244,954 |
|||||
Income (loss) from operations |
16,108 |
(48,138) |
(87,471) |
(12,821) |
|||||
Other income (expense): |
|||||||||
Investment income |
989 |
1,384 |
3,912 |
3,146 |
|||||
Interest expense |
(9,746) |
(9,233) |
(28,861) |
(27,381) |
|||||
Gain on investment in Regulus Therapeutics, Inc. |
- |
20,211 |
- |
20,211 |
|||||
Income (loss) before income tax expense |
7,351 |
(35,776) |
(112,420) |
(16,845) |
|||||
Income tax expense |
- |
- |
(1) |
- |
|||||
Net income (loss) |
$7,351 |
$(35,776) |
$(112,421) |
$(16,845) |
|||||
Basic net income (loss) per share |
$0.06 |
$(0.30) |
$(0.93) |
$(0.14) |
|||||
Diluted net income (loss) per share |
$0.06 |
$(0.30) |
$(0.93) |
$(0.14) |
|||||
Shares used in computing basic net income (loss) per share |
120,989 |
119,979 |
120,795 |
119,560 |
|||||
Shares used in computing diluted net income (loss) per share |
123,378 |
119,979 |
120,795 |
119,560 |
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, September 30, |
Nine months ended, September 30, |
|||||||
2016 |
2015 |
2016 |
2015 |
|||||
(unaudited) |
(unaudited) |
|||||||
As reported operating expenses according to GAAP |
$94,819 |
$97,259 |
$273,743 |
$244,954 |
||||
Excluding compensation expense related to equity awards |
(17,586) |
(14,997) |
(56,950) |
(41,907) |
||||
Pro forma operating expenses |
$77,233 |
$82,262 |
$216,793 |
$203,047 |
||||
As reported income (loss) from operations according to GAAP |
$16,108 |
$(48,138) |
$(87,471) |
$(12,821) |
||||
Excluding compensation expense related to equity awards |
(17,586) |
(14,997) |
(56,950) |
(41,907) |
||||
Pro forma income (loss) from operations |
$33,694 |
$(33,141) |
$(30,521) |
$29,086 |
||||
As reported net income (loss) according to GAAP |
$7,351 |
$(35,776) |
$(112,421) |
$(16,845) |
||||
Excluding compensation expense related to equity awards |
(17,586) |
(14,997) |
(56,950) |
(41,907) |
||||
Pro forma net income (loss) |
$24,937 |
$(20,779) |
$(55,471) |
$25,062 |
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. |
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Condensed Consolidated Balance Sheets |
|||||
(In Thousands) |
|||||
September 30, |
December 31, |
||||
2016 |
2015 |
||||
(unaudited) |
|||||
Assets: |
|||||
Cash, cash equivalents and short-term investments |
$687,848 |
$779,183 |
|||
Investment in Regulus Therapeutics Inc. |
9,382 |
24,792 |
|||
Other current assets |
33,099 |
33,028 |
|||
Property, plant and equipment, net |
90,970 |
90,233 |
|||
Other assets |
22,287 |
20,664 |
|||
Total assets |
$843,586 |
$947,900 |
|||
Liabilities and stockholders' equity: |
|||||
Other current liabilities |
$50,353 |
$81,554 |
|||
Current portion of deferred contract revenue |
56,539 |
67,322 |
|||
1% convertible senior notes |
356,440 |
339,847 |
|||
2 3/4% convertible senior notes |
51,570 |
49,523 |
|||
Long-term obligations, less current portion |
87,214 |
74,558 |
|||
Long-term deferred contract revenue |
101,831 |
134,306 |
|||
Stockholders' equity |
139,639 |
200,790 |
|||
Total liabilities and stockholders' equity |
$843,586 |
$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