SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
SCHEDULE 14A INFORMATION
Proxy Statement Pursuant to Section 14(a) of
the Securities Exchange Act of 1934 (Amendment No. )
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Preliminary Proxy Statement |
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Definitive Proxy Statement |
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Definitive Additional Materials |
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Soliciting Material under §240.14a-12 |
Ionis Pharmaceuticals, Inc. |
(Name of Registrant as Specified In Its Charter) |
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(Name of Person(s) Filing Proxy Statement, if other than the Registrant) |
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Check box if any part of the fee is offset as provided by Exchange Act Rule 0-11(a)(2) and identify the filing for which the offsetting fee was paid previously. Identify the previous filing by registration statement number, or the Form or Schedule and the date of its filing. |
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Ionis had a goal and measure related to SPINRAZA of filing and acceptance of the NDA and MAA for SPINRAZA with Biogen paying us the license fee to exercise its option to take a license to SPINRAZA. Ionis not only achieved the stated goal, Ionis and Biogen achieved FDA approval of SPINRAZA for the treatment of SMA in pediatric and adult patients. Achieving this approval so quickly (approximately 3 months from filing) for the broad patient population was an extraordinary achievement and greatly exceeded expectations for the year, especially since NDA approvals for an orphan drug can typically take between 9-12 months from acceptance of the filing to approval, if at all. SPINRAZA’s approval and the commercial revenue associated with SPINRAZA is a reflection of the evolution of our business and our progress towards sustained profitability.
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Ionis and Akcea formed a strategic collaboration with Novartis to develop and commercialize AKCEA-APO(a)-LRx and AKCEA-APOCIII-LRx, valued at over $1 billion plus potential product royalties, including $225 million in near term payments.
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a profitable first quarter with operating income of $14 million.
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More than $110 million in revenue for the quarter.
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Ionis finished the year with pro forma operating income of $25.8 million, where when setting goals for the year, it had projected a pro forma operating loss in the low $60 million range, representing an improvement of more than 140%.
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Ionis generated $347 million of revenue, compared to its guidance of $240 million, representing an improvement of 45%.
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Ionis ended the year with $665 million in cash, exceeding year end cash guidance by $65 million.
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In 2017 Ionis expects to be breakeven or profitable at the operating line on a pro forma basis, driven in part by the revenue from SPINRAZA.
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Dec-11
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Dec-12
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Dec-13
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Dec-14
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Dec-15
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Dec-16
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Ionis Pharmaceuticals, Inc.
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100.00
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$
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144.80
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552.57
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$
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856.31
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$
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858.95
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663.38
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NASDAQ Composite Index
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100.00
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116.41
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165.47
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188.69
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200.32
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216.54
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NASDAQ Biotechnology Index
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100.00
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134.68
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232.37
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$
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307.67
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$
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328.76
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$
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262.08
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Evaluation of 2016 Corporate Objectives
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Objective & Pre-Approved Measures
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Evaluation
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1
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Prepare for Filing/Approval:
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Ionis exceeded this objective:
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File NDA and MAA for SPINRAZA (filing accepted), achieve Biogen Licensing Fee
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Biogen filed the NDA and MAA for SPINRAZA; and paid Ionis the $75 million license fee. Ionis and Biogen achieved FDA approval of SPINRAZA in 3 months under Priority Review for the treatment of SMA in pediatric and adult patients
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Pass regulatory inspections post filing (GMP and GCP)
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The regulatory inspections supported approval for SPINRAZA
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NDA/MAA sections complete and reviewed for volanesorsen, except Phase 3 data
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Ionis and Akcea completed the NDA/MAA sections for volanesorsen, except Phase 3 data
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Complete all pre-clinical and clinical study reports for IONIS-TTRRx except for the ongoing Neuro-TTR study
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Ionis completed the Pre-Clinical and Clinical study reports for IONIS-TTRRx other than the ongoing Neuro-TTR study
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Complete summary shells and CMC sections for the IONIS-TTRRx NDA to the extent possible
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Ionis substantially completed the summary shells and CMC sections for the IONIS-TTRRx NDA
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Complete position pieces on key issues
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Ionis substantially completed the position pieces for key issues
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Manage process with GSK to enable a smooth transfer for NDA filing for IONIS-TTRRx should GSK exercise its option
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Ionis implemented a process to transfer NDA filing for IONIS-TTRRx should GSK exercise its option
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Ensure smooth transitioning of API site and process changes to GSK/CMO to enable a successful launch for IONIS-TTRRx
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Ionis worked with GSK to transition API manufacturing for IONIS-TTRRx to enable a successful launch for IONIS-TTRRx
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2
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Advance Pipeline:
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Ionis met this objective:
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Screening closed in volanesorsen FPL Phase 3 study
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Due to improved study designs, closing for screening in the volanesorsen FPL Phase 3 Study is planned for 2017
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Positive data for IONIS-DMPK-2.5Rx and Biogen agreement to advance program
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Ionis completed this study, observed encouraging biomarker trends and learned more about the disease. However, Ionis did not achieve the desired potency in muscle
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Positive data for IONIS-FXIRx and Bayer agreement to advance program (milestone payment)
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Ionis reported positive data from the Phase 2 study of IONIS-FXIRx in patients with end-stage renal disease on hemodialysis. Bayer, plans to advance IONIS-FXIRx and expanded the collaboration by licensing the follow-on LICA drug, IONIS-FXI-LRx
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Solidify most rapid route leading to regulatory success and commercialization for IONIS-APO(a)-LRx (finalized plan).
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Ionis and Akcea formed a strategic collaboration with Novartis to develop and commercialize AKCEA-APO(a)-LRx and AKCEA-APOCIII-LRx, which allows Ionis and Akcea to move more rapidly to Phase 3 cardiovascular outcomes studies with both therapies than their original development plans
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Initiate first Phase 2/3 Clinical Trials on ≥3 drugs
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Ionis initiated Phase 2 clinical trials for three drugs
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Positive Phase 2 Clinical data on ≥5 drugs
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Ionis reported positive Phase 2 data for six studies
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Initiate Phase 1 Clinical Trials on ≥4 drugs
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Ionis initiated Phase 1 clinical trials on two drugs
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≥4 new drugs into the pipeline
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Ionis added five new drugs to its development pipeline
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3
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Make Biogen Idec relationship successful:
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Ionis met this objective:
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Positive data for IONIS-DMPK-2.5Rx and Biogen agreement to advance program
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Ionis completed this study, observed encouraging biomarker trends and learned more about the disease. However, Ionis did not achieve the desired potency in muscle
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Initiate Phase 1 clinical trial for IONIS-BIIB4Rx
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Ionis advanced the program but the Phase 1 clinical trial for IONIS-BIIB4Rx did not initiate until the first half of 2017
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Identify ≥1 development candidate and two new target sanctions
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Ionis achieved two target sanctions, and made significant progress towards identifying a development candidate
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Achieve ≥$100 million in revenue across all Biogen collaborations
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Ionis exceeded this measure by generating nearly $210 million in revenue across all Biogen collaborations
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Make AstraZeneca relationship successful:
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Ionis met this objective:
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Successful outcome of STAT3-PDL1 Phase 1/2 study
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Although the study progressed in 2016, we do not expect the study to complete until 2017
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Achieve development candidate approval for IONIS-AZ4-2.5-LRx
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Ionis achieved development candidate approval for IONIS-AZ4-2.5-LRx and received a $25 million milestone
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Successfully complete IONIS-KRAS-2.5Rx nonclinical study and achieve $15 million milestone
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Ionis complete the IONIS-KRAS-2.5Rx nonclinical study and received a $15 million milestone
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Advance two new programs to target sanction
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Ionis advanced two new programs to target sanction
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Demonstrate the value of technology advancements in the clinic:
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Ionis partially met this objective:
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Advance ≥3 LICA drugs through Phase 1 with ED50 ≤20 mg/wk
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Ionis advanced three LICA drugs into Phase 1 and achieved an ED50 ≤20 mg/wk
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≥4 new Gen 2.5 drugs into development
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Ionis advanced two new Gen 2.5 drugs into development
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uORF and TSE approaches fully enabled for drug discovery activities
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Ionis fully enabled drug discovery for uORF and TSE
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Meet budget and financial projections for the year
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Ionis exceeded this objective:
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Ionis significantly improved upon its financial guidance for the year
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Ionis met its budget
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Stock price performance by a percentage greater than or equal to median of the companies listed in the NASDAQ Biotechnology Index; or if SPINRAZA filing successful 110%
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Ionis met this objective:
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Ionis’ stock price outperformed the median stock price change for companies listed in the NASDAQ Biotechnology Index
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Successful Akcea IPO (at prespecified valuation target) assuming reasonable market conditions
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Ionis partially met this objective:
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We made substantial progress preparing for Akcea’s IPO
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Partner IONIS-APO(a)-LRx or alternative successful Akcea transaction
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Ionis exceeded this objective:
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Ionis and Akcea formed a strategic collaboration with Novartis to develop and commercialize AKCEA-APO(a)-LRx and AKCEA-APOCIII-LRx for the treatment of lipid disorder
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Complete one additional partnership either by expanding or adding a new partner
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Ionis exceeded this objective:
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The Novartis transaction noted in item 9 above also included AKCEA-APOCIII-LRx
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Pre-commercial activity for volanesorsen on track for early 2018 launch:
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Ionis met this objective:
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Initial US and EU Commercial infrastructure in place
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Akcea made significant progress regarding its pre-commercial activity for volanesorsen for planned early 2018 launch
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Unplanned Accomplishments for 2016
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12
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The FDA approved SPINRAZA in record time
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13
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Biogen reported positive data from an end of study analysis of the ENDEAR Phase 3 study in patients with infantile-onset (consistent with type 1) SMA at the British Pediatric Neurology Association. annual conference. Ionis previously reported data from an interim analysis of ENDEAR, which along with several other studies, formed the basis for the marketing application for SPINRAZA in the U.S.
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Ionis and Biogen reported positive data from an interim analysis of the Phase 3 CHERISH study in patients with later-onset (consistent with Type 2) SMA
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Ionis and Akcea reported that the Phase 3 COMPASS study for volanesorsen met its primary endpoint
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Ionis sold the global rights to develop and commercialize KYNAMRO to Kastle Therapeutics and earned a $15 million upfront payment
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Ionis and MD Anderson Cancer Center formed a strategic alliance to advance novel cancer therapies
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Ionis completed a satellite company transaction with Dynacure to discover, develop and commercialize drugs to treat neuromuscular diseases
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Ionis received funding from Cystic Fibrosis Foundation for Ionis’ program targeting ENAC for treating cystic fibrosis
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Ionis settled its dispute with OncoGenex on favorable terms
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