Ionis Reports Fourth Quarter and Full Year 2017 Financial Results

Recent Pipeline and Technology Highlights

• SPINRAZA for SMA – one of the most successful orphan drug launches in history
• SPINRAZA^®, commercialized by Biogen, generated 2017 global sales of $884 million
• Results from the ENDEAR study and CHERISH study, in which people with infantile-onset and later-onset SMA, respectively, were treated with SPINRAZA, were published in The New England Journal of Medicine
• Prestigious 2017 Prix Galien USA Award for Best Biotechnology Product awarded to Ionis and Biogen for SPINRAZA
• New collaboration with Biogen initiated to discover new antisense drugs with enhanced properties to treat SMA
• Inotersen for hATTR – potential to transform the lives of people with hATTR
• Marketing applications accepted, no FDA Advisory Committee recommended, Priority Review in the U.S. and Accelerated Assessment in the EU
• Preparations for global launch, planned for mid-2018, progressing
• Phase 3 NEURO-TTR study met both primary endpoints demonstrating benefit compared to placebo in multiple measures of quality of life and disease severity; 50% of inotersen-treated patients experienced improvement from baseline in quality of life
• Volanesorsen for FCS and FPL – potential first treatment for people with FCS
• Marketing applications accepted in the U.S., EU and Canada with Promising Innovative Medicine designation in the UK and Priority Review in Canada
• Preparations for global launch for FCS, planned for mid-2018, progressing
• Phase 3 APPROACH study met primary endpoint of reducing triglyceride levels in people with FCS
• Pipeline Programs (early and mid-stage) – advancing wholly owned and partnered programs
• Positive results from seven Phase 2 studies reported, including:
• Positive data from Phase 1/2 study of IONIS-STAT3-2.5_Rx in combination with AstraZeneca's Imfinzi reported for people with head and neck cancer
• Robust, dose-dependent reductions of mHTT observed in people with Huntington's disease treated with IONIS-HTT_Rx 
• Positive clinical data on five LICA drugs reported, demonstrating consistent, positive performance and sustained target reduction with potential for monthly or less frequent dosing
• Positive results from six Phase 1 studies reported
• Nine Phase 2 studies and four Phase 1 studies initiated across multiple therapeutic areas to treat people with both broad and rare diseases

"In 2017, we and our partners advanced key programs in all of our therapeutic areas, including cardiometabolic, oncology, neurology and, severe and rare disease. Of the eight new drugs we added to the pipeline, six were drugs that use our more potent LICA technology or Generation 2.5 chemistry, and two were drugs to treat neurodegenerative disorders. We also added to our pipeline our second orally delivered, locally acting drug for a GI autoimmune disease. These achievements demonstrate the success of our wholly owned and partnered programs," said Brett P. Monia, Ph.D., chief operating officer and senior vice president of antisense drug discovery and translational medicine at Ionis Pharmaceuticals. "Looking ahead, we plan to pursue only those partnerships with infrastructure and resources that complement our own. Simultaneously, we plan to continue to build our wholly owned pipeline of drugs to treat patients with rare and serious diseases."

Expected Events Through Mid-2018

• Launch of inotersen for people with hATTR, if approved
• Launch of volanesorsen for people with FCS, if approved
• Report results from five Phase 2 programs, including data from studies with IONIS-HTT_Rx in people with Huntington's disease and data from a 6-12 month study with AKCEA-APO(a)-L_Rx in people with high Lp(a) and cardiovascular disease
• Initiate up to six Phase 2 or Phase 3 studies and three Phase 1 studies, including the initiation of a clinical study with follow-on LICA drug, IONIS-TTR-L_Rx

Revenue

Ionis' revenue for the fourth quarter and full year 2017 was $172.3 million and $507.7 million, compared to $160.3 million and $346.6 million for the same periods in 2016. Ionis' revenue in 2017 consisted of the following:

Commercial Revenue:

• $113 million from SPINRAZA royalties; and
• $9 million from other licensing and royalty payments.

R&D Revenue: 

• $118 million in milestone payments from Biogen, including $90 million in milestone payments for SPINRAZA, $15 million for validating two undisclosed neurological disease targets and $10 million for initiating a Phase 1/2 study of IONIS-MAPT_Rx;
• $65 million from Bayer for the license of IONIS-FXI-L_Rx;
• $48 million from Roche primarily for the license of IONIS-HTT_Rx;
• $10 million from Janssen for license of IONIS-JBI2-2.5_Rx and initiation of Phase 1 study of IONIS-JBI1-2.5_Rx;
• $115 million from the amortization of upfront fees; and
• $30 million from services Ionis performed for its partners, of which more than half related to manufacturing services.

Ionis' R&D revenue may fluctuate quarterly 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.

At the beginning of 2018, Ionis adopted the new revenue recognition accounting standard on a retrospective basis, which means that starting with the first quarter, Ionis will begin showing all periods presented using the new standard. The Company does not anticipate that there will be a significant impact to its previously reported revenue. Under the new standard, Ionis' revenue for 2017 will increase by approximately 3%.

Operating Expenses

Operating expenses for the fourth quarter and full year 2017 on a GAAP basis were $174.0 million and $483.1 million, respectively, and on a pro forma basis were $151.7 million and $397.2 million, respectively. This is compared to GAAP operating expenses of $119.2 million and $392.9 million and pro forma operating expenses of $104.0 million and $320.8 million for the same periods in 2016. Operating expenses increased in 2017, compared to 2016, principally due to higher SG&A expenses as Ionis prepares to commercialize volanesorsen and inotersen in 2018. The Company's SG&A expenses also increased in 2017 compared to 2016 because of fees it owes under its in-licensing agreements related to SPINRAZA.

Net Income (Loss)

Ionis reported a net loss of $4.7 million and $17.3 million for the fourth quarter and full year 2017, respectively, compared to net income of $25.9 million and a net loss of $86.6 million for the same periods in 2016, all according to GAAP. On a pro forma basis, Ionis reported net income of $17.7 million and $68.7 million for the fourth quarter and full year 2017, respectively, compared to net income of $41.0 million and a net loss of $14.4 million for the same periods in 2016. Ionis' GAAP and pro forma net loss improved for 2017 compared to 2016 primarily due to the addition of commercial revenue from SPINRAZA royalties and increased R&D revenue.

Additionally, in 2017, Ionis recorded two non-cash, non-recurring items in other expenses, which contributed to the Company's net loss. These two items were the previously capitalized fair value of the potential premium Ionis would have received from Novartis if Akcea had not completed its IPO and the loss the Company recognized on the purchase of its primary R&D facility. In 2016, Ionis recorded a $4.0 million non-cash loss related to the early repurchase of its 2¾ percent convertible senior notes, which contributed to the Company's net loss for 2016.

Net Loss Attributable to Noncontrolling Interest in Akcea Therapeutics, Inc.

Akcea sold shares of its common stock to third parties in its IPO in July 2017. From the closing of the IPO through the end of 2017, Ionis owned 68 percent of Akcea. The shares held by third parties represent an interest in Akcea's equity that Ionis does not control. However, because Ionis continues to maintain overall control of Akcea through its voting interest, Ionis reflects the assets, liabilities and results of operations of Akcea in Ionis' consolidated financial statements. Ionis reflects the noncontrolling interest attributable to other holders of Akcea's common stock in a separate line called "Net loss attributable to noncontrolling interest in Akcea" on Ionis' statement of operations. Ionis' net loss attributable to noncontrolling interest in Akcea for the fourth quarter and full year 2017 was $7.4 million and $11.3 million, respectively. Ionis also added a corresponding account in its stockholders' equity section on its balance sheet called "Noncontrolling interest in Akcea Therapeutics, Inc."  

Net Income (Loss) Attributable to Ionis Common Stockholders

Ionis reported net income attributable to Ionis' common stockholders of $2.7 million for the fourth quarter of 2017, compared to $25.9 million for the same period in 2016. For the full years 2017 and 2016, Ionis reported a net loss attributable to Ionis' common stockholders of $6.0 million and $86.6 million, respectively. For the fourth quarter of 2017, basic and diluted net income per share were $0.02 compared to basic and diluted net income per share of $0.21 for the same period in 2016. For the full year 2017, basic and diluted net income per share were $0.08 compared to basic and diluted net loss per share of $0.72 for the same period in 2016.

Webcast and Conference Call

Today, at 11:30 a.m. Eastern Time, Ionis will conduct a live webcast conference call to discuss this earnings release and related activities. Interested parties may listen to the call by dialing 877-443-5662 or access the webcast at www.ionispharma.com. A webcast replay will be available for a limited time.

ABOUT IONIS PHARMACEUTICALS

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 three dozen drugs in development. SPINRAZA® (nusinersen) has been approved in global markets for the treatment of spinal muscular atrophy (SMA). Biogen is responsible for commercializing SPINRAZA. Drugs that have successfully completed Phase 3 studies include inotersen, an antisense drug Ionis is developing to treat patients with hereditary TTR amyloidosis (hATTR), and volanesorsen, an antisense drug discovered by Ionis and co-developed by Ionis and Akcea Therapeutics to treat patients with familial chylomicronemia syndrome or familial partial lipodystrophy. Akcea, an affiliate of Ionis, is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders. If approved, volanesorsen will be commercialized through Ionis' affiliate, Akcea. Inotersen filings for marketing approval have been submitted in the U.S. and EU. Volanesorsen filings for marketing approval have been submitted in the U.S., EU, and Canada. Ionis' patents provide strong and extensive protection for its drugs and technology. Additional information about Ionis is available at www.ionispharma.com.

IONIS PHARMACEUTICALS' FORWARD-LOOKING STATEMENT

This press release includes forward-looking statements regarding Ionis' business, financial guidance and the therapeutic and commercial potential of SPINRAZA, inotersen, volanesorsen and Ionis' technologies and products in development, including the business of Akcea Therapeutics, Inc., Ionis' majority owned subsidiary. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2016, and its most recent quarterly report on Form 10-Q, which are on file with the SEC. Copies of these and other documents are available from the Company.

In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to Ionis Pharmaceuticals and its subsidiaries.

Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals, Inc. SPINRAZA® is a registered trademark of Biogen.

IONIS PHARMACEUTICALS, INC.
SELECTED FINANCIAL INFORMATION
Condensed Consolidated Statements of Operations
(In Thousands, Except Per Share Data)
		Three months ended,				Year ended,
		December 31,				December 31,
		2017		2016		2017		2016
Revenue:		(unaudited)
Commercial revenue:
SPINRAZA royalties		$52,073		$         883		$112,540		$          883
Licensing and royalty revenue		4,537		150		9,519		19,839
Total commercial revenue		56,610		1,033		122,059		20,722
Research and development revenue under collaborative agreements		115,689		159,316		385,607		325,898
Total revenue		172,299		160,349		507,666		346,620
Expenses:
Research, development and patent expenses		128,285		101,151		374,644		344,320
Selling, general and administrative		45,707		18,043		108,488		48,616
Total operating expenses		173,992		119,194		483,132		392,936
Income (loss) from operations		(1,693)		41,155		24,534		(46,316)
Other income (expense):
Investment income		654		1,561 Werbung Mehr Nachrichten zur Ionis Pharmaceuticals Inc Aktie kostenlos abonnieren E-Mail-Adresse Bitte überprüfe deine die E-Mail-Adresse. Benachrichtigungen von ARIVA.DE (Mit der Bestellung akzeptierst du die Datenschutzhinweise) -1 Vielen Dank, dass du dich für unseren Newsletter angemeldet hast. Du erhältst in Kürze eine E-Mail mit einem Aktivierungslink. Hinweis: ARIVA.DE veröffentlicht in dieser Rubrik Analysen, Kolumnen und Nachrichten aus verschiedenen Quellen. Die ARIVA.DE AG ist nicht verantwortlich für Inhalte, die erkennbar von Dritten in den „News“-Bereich dieser Webseite eingestellt worden sind, und macht sich diese nicht zu Eigen. Diese Inhalte sind insbesondere durch eine entsprechende „von“-Kennzeichnung unterhalb der Artikelüberschrift und/oder durch den Link „Um den vollständigen Artikel zu lesen, klicken Sie bitte hier.“ erkennbar; verantwortlich für diese Inhalte ist allein der genannte Dritte. Andere Nutzer interessierten sich auch für folgende News 24.04.24 - PR Newswire Invesco Select Trust Plc - Cancellation of Treasury Shares 24.04.24 - PR Newswire Oscillate Plc - Holding(s) in Company Solarboom 2024:    5 Gründe die für diese Aktie sprechen… weiterlesen» Anzeige wo-site-teaser-box-16746_site-teaser-box_2024-04-24_08%253A00%253A00_2024-04-25_09%253A00%253A59