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Cytokinetics, Inc. Reports Fourth Quarter 2017 Financial Results

On February 15, 2018 Cytokinetics, Incorporated (Nasdaq:CYTK) reported financial results for the fourth quarter of 2017. Net loss for the fourth quarter was $40.5 million, or $0.75 per basic share and diluted share, respectively, compared to net income for the same period in 2016 of $7.2 million, or $0.18 and $0.16 per basic and diluted share, respectively (Press release, Cytokinetics, FEB 15, 2018, View Source [SID1234524001]). Cash, cash equivalents and investments totaled $285.4 million at December 31, 2017.

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"We had a productive fourth quarter and begin the year with optimism for our growing pipeline of muscle biology directed drug candidates," said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. "In 2018, we are looking forward to results from four mid-stage clinical trials of CK-2127107 in our skeletal muscle activator program which may inform plans to advance to a Phase 3 clinical program under our collaboration with Astellas. Additionally, enrollment in GALACTIC-HF remains on track with planning under our collaboration with Amgen. Our research continues to power innovation with two potential drug candidates expected to move through IND-enabling studies and into Phase 1 trials in 2018."

Recent Highlights and Upcoming Milestones

Cardiac Muscle Program

omecamtiv mecarbil (cardiac myosin activator)

Continued site activation and patient enrollment in GALACTIC-HF, the Phase 3 cardiovascular outcomes clinical trial of omecamtiv mecarbil. Enrollment is proceeding according to plan with patients that have the intended risk profile consistent with the trial design.

Continued protocol development, feasibility assessments, regulatory interactions and other readiness activities for a second Phase 3 clinical trial of omecamtiv mecarbil. This trial which is intended to evaluate the potential of omecamtiv mecarbil to increase exercise performance in patients with heart failure is planned to be conducted by Cytokinetics in collaboration with Amgen.

Announced that a post-hoc responder analysis from COSMIC-HF (Chronic Oral Study of Myosin Activation to Increase Contractility in Heart Failure), a Phase 2 clinical trial evaluating omecamtiv mecarbil in patients with chronic heart failure and left ventricular systolic dysfunction, was presented by John Teerlink, M.D. in an Abstract Rapid Fire Oral presentation at the American Heart Association Scientific Sessions. The proportion of patients achieving various thresholds in the percent reduction of NT-proBNP was larger in patients who received omecamtiv mecarbil than in patients who received placebo.
Skeletal Muscle Program

tirasemtiv (fast skeletal muscle troponin activator (FSTA))

Announced the presentation of results from VITALITY-ALS (Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices after Treatment for a Year in ALS), the international Phase 3 clinical trial of tirasemtiv in patients with amyotrophic lateral sclerosis (ALS), at the 28th International Symposium on ALS and Motor Neurone Disease (MND) in Boston. The presentation, by Jeremy Shefner, M.D., Ph.D., Lead Investigator of VITALITY-ALS, Professor and Chair of Neurology at Barrow Neurological Institute, and Professor and Executive Chair of Neurology at University of Arizona, Phoenix, followed our prior announcement that the trial did not meet the primary endpoint of change from baseline in slow vital capacity which was evaluated at 24 weeks following randomization or any of the secondary endpoints in the trial which were evaluated at 48 weeks.

Continued treatment of patients in VIGOR-ALS (Ventilatory Investigations in Global Open-Label Research in ALS), an open-label clinical trial designed to assess the long-term safety and tolerability of tirasemtiv in patients with ALS who have completed participation in VITALITY-ALS. Currently, there are still over 100 patients who are receiving tirasemtiv in VIGOR-ALS.
CK-2127107, reldesemtiv (next-generation fast skeletal muscle troponin activator)

Received final approval from the World Health Organization and the United States Adopted Name Council for reldesemtiv to be used as the International Nonproprietary Name for CK-2127107.

Continued conduct of our Phase 2 clinical trial of reldesemtiv which is designed to assess its effect on multiple measures of muscle function in both ambulatory and non-ambulatory patients with SMA. This trial has enrolled over 60 patients toward the objective of 72 patients and is being conducted by Cytokinetics in collaboration with Astellas.

Continued site activation and patient enrollment in FORTITUDE-ALS (Functional Outcomes in a Randomized Trial of Investigational Treatment with CK-2127107 to Understand Decline in Endpoints – in ALS), the Phase 2 clinical trial of reldesemtiv which is designed to assess the change from baseline in the percent predicted slow vital capacity (SVC) and other measures of skeletal muscle function after 12 weeks of treatment with reldesemtiv in patients with ALS. This trial has screened over 100 patients and enrolled nearly 90 patients toward the objective of 445 patients and is being conducted by Cytokinetics in collaboration with Astellas.

Continued site activation and patient enrollment in the Phase 2 clinical trial of reldesemtiv in patients with chronic obstructive pulmonary disease (COPD) which is designed to assess its effect on physical function. This trial has enrolled over 30 patients towards the objective of 40 patients and is being conducted by Astellas in collaboration with Cytokinetics.

Continued site activation and patient enrollment in the Phase 1b clinical trial of reldesemtiv in elderly subjects with limited mobility which is designed to assess its effect on measures of physical function. This trial has enrolled over 20 subjects towards the objective of 60 subjects and is being conducted by Astellas in collaboration with Cytokinetics.

Announced the publication of "CK-2127107 Amplifies Skeletal Muscle Response to Nerve Activation in Humans," in Muscle & Nerve, which showed that reldesemtiv increased the force generated by the tibialis anterior muscle versus placebo in response to nerve stimulation in a dose, plasma concentration, and frequency-dependent manner. Single doses of reldesemtiv were well-tolerated in healthy volunteers up to 4000 mg. No serious adverse events were reported and adverse events were all mild or moderate.
Pre-Clinical Research and Development

Advanced a next-generation cardiac muscle activator into development under our collaboration with Amgen. This milestone triggered a $1 million payment from Amgen to Cytokinetics.

Advanced a next-generation skeletal muscle activator into development under our collaboration with Astellas.

Announced that Cytokinetics is advancing an unpartnered cardiac sarcomere directed compound from research into IND-enabling studies in 2018.

Continued collaboration activities under our joint research program with Amgen directed to the discovery of next-generation cardiac muscle activators and under our joint research program with Astellas directed to the discovery of next-generation skeletal muscle activators. Cytokinetics and Astellas recently agreed to extend the joint research program through 2019.

Company scientists continued independent research activities directed to our other muscle biology programs.
Financials

Revenues for the year ended December 31, 2017 included $11.0 million in milestone revenues from Amgen as well as $1.3 million of research and development revenues from our collaboration with Amgen, and $11.9 million of research and development revenues plus $8.8 million of license revenues from our collaboration with Astellas. Revenues for the year ended December 31, 2017 were offset by $20.0 million (out of the total of $40 million) for payments to Amgen related to our option to co-fund the Phase 3 development program of omecamtiv mecarbil in exchange for an increased royalty upon potential commercialization. Revenues in 2016 were primarily due to license revenue from the September 2016 expansion of our collaboration with Astellas and a $26.7 million milestone payment from Amgen.

Research and development expenses for the three months and year ended December 31, 2017 increased to $26.3 million and $90.3 million, respectively, from $18.8 million and $59.9 million for the same periods in 2016, primarily due to increased clinical activity, including activity for VITALITY-ALS, increased clinical trials activity for reldesemtiv, as well as increased personnel.

General and administrative expenses for the three months and year ended December 31, 2017 increased to $10.3 million and $36.5 million, respectively, from $6.7 million and $27.8 million for the same periods in 2016, primarily due to increased personnel, non-cash stock compensation expense and commercial readiness activities.

2018 Financial Guidance

The Company also announced financial guidance for 2018. The company anticipates cash revenue will be in the range of $17 to $23 million, operating expenses will be in the range of $105 to $115 million, and net cash utilization will be approximately $100 million.

2018 Corporate Milestones

Cardiac Muscle Program

omecamtiv mecarbil (cardiac myosin activator)

Expect to complete enrolling patients with chronic heart failure in GALACTIC-HF in approximately one year.

Expect to finalize plans and prepare to start the second Phase 3 trial of omecamtiv mecarbil.
Skeletal Muscle Program

CK-2127107 (reldesemtiv), (next-generation fast skeletal muscle troponin activator (FSTA))

Expect results from a Phase 2 clinical trial of reldesemtiv in patients with SMA in Q2 2018.

Expect results from a Phase 2 clinical trial of reldesemtiv in patients with ALS in 2H 2018.

Expect results from a Phase 2 clinical trial of reldesemtiv in patients with COPD in 2H 2018.

Expect results from a Phase 1b clinical trial of reldesemtiv in adults with limited mobility in 2H 2018.
Pre-Clinical Research

Expect to advance one development compound, under each of our collaborations with Amgen and Astellas, into IND-enabling studies in 2018, one of which may proceed to Phase 1 this year.

Expect to advance an unpartnered cardiac sarcomere directed compound through IND-enabling studies in 2018 to enable initiation of Phase 1 in 2018.
Conference Call and Webcast Information

Members of Cytokinetics’ senior management team will review the company’s fourth quarter results via a webcast and conference call today at 4:30 PM Eastern Time. The webcast can be accessed through the Investors & Media section of the Cytokinetics website at www.cytokinetics.com. The live audio of the conference call can also be accessed by telephone by dialing either (866) 999-CYTK (2985) (United States and Canada) or (706) 679-3078 (international) and typing in the passcode 3665278.

An archived replay of the webcast will be available via Cytokinetics’ website until February 22, 2018. The replay will also be available via telephone by dialing (855) 859-2056 (United States and Canada) or (404) 537-3406 (international) and typing in the passcode 3665278 from February 15, 2018 at 7:30 PM Eastern Time until February 22, 2018.

Cambrex To Present At The 2018 RBC Capital Markets Global Healthcare Conference

On February 15, 2018 Cambrex Corporation (NYSE:CBM), a leading manufacturer of small molecule innovator and generic Active Pharmaceutical Ingredients (APIs), reported that Steven Klosk, President and Chief Executive Officer, will participate in a fireside chat at the 2018 RBC Capital Markets Global Healthcare Conference on February 22, 2018 at 3:05 p.m. EST in New York City (Press release, Cambrex, FEB 15, 2018, View Source [SID1234524000]).

The live audio webcast and slide presentation can be accessed from the Cambrex website at www.cambrex.com in the Investors section under "Webcasts & Presentations", and a replay will be available for 90 days after the live event concludes.

Acorda Provides Financial and Pipeline Update for Fourth Quarter and Year End 2017

On February 15, 2018 Acorda Therapeutics, Inc. (Nasdaq: ACOR) reported its financial and pipeline update for the fourth quarter and full year ended December 31, 2017 (Press release, Acorda Therapeutics, FEB 15, 2018, View Source [SID1234523995]).

"We continue to prepare for the potential approval and launch of INBRIJA, our investigational inhaled levodopa treatment for symptoms of OFF periods in people with Parkinson’s disease. We look forward to working with the FDA during the NDA review process, and to bringing this new treatment option to the Parkinson’s community to help address an important unmet need," said Ron Cohen, M.D., Acorda’s President and CEO. "Based on our continued market research, as well as the strength of our Phase 3 data, we believe INBRIJA’s US market opportunity to be greater than $800 million."

Fourth Quarter 2017 Financial Results

AMPYRA (dalfampridine) Extended Release Tablets, 10 mg – For the quarter ended December 31, 2017, the Company reported AMPYRA net revenue of $167.2 million compared to $132.3 million for the same quarter in 2016.

Royalty Revenue – For the quarter ended December 31, 2017, the Company reported royalty revenue of $16.1 million as compared to $4.4 million for the same quarter in 2016. The Company reported FAMPYRA royalties from sales outside of the U.S. of $3.1 million compared to $2.7 million for the same quarter in 2016. Additionally, the Company completed a transaction that provides a fully paid-up, royalty-free license for Selincro in exchange for $13.0 million which was recorded as royalty revenue in the quarter ended December 31, 2017. During the quarter ended December 31, 2017, the Company completed a royalty purchase transaction for its Fampyra royalty revenue in exchange for an upfront payment of $40 million. The transaction was recorded as a liability in accordance with US GAAP which will be reduced over time as royalty revenue is recognized.

Research and development (R&D) expenses for the quarter ended December 31, 2017 were $35.1 million, including $2.2 million of share-based compensation compared to $53.8 million, including $3.0 million of share-based compensation for the same quarter in 2016.

Sales, general and administrative (SG&A) expenses for the quarter ended December 31, 2017 were $39.5 million, including $5.4 million of share-based compensation compared to $59.0 million, including $6.0 million of share-based compensation for the same quarter in 2016.

The Company recorded non-cash asset impairment charges of $233.5 million for tozadenant as a result of the termination of this program, and $23.8 million for SYN120 as a result of the trial not meeting key primary and secondary endpoints. The Company assessed the valuation assumptions for both programs and determined the assets were fully impaired. Both of these charges were recorded in the quarter ended December 31, 2017.

Benefit from income taxes for the quarter ended December 31, 2017 was $51.9 million, including $2.7 million of cash taxes, compared to a provision for income taxes of $1.0 million, including $0.7 million of cash taxes for the same quarter in 2016.

The Company reported a GAAP net loss attributable to Acorda of $(171.1) million for the quarter ended December

31, 2017, or $(3.70) per diluted share. GAAP net loss in the same quarter of 2016 was $(3.1) million, or $(0.07) per diluted share.

Non-GAAP net income for the quarter ended December 31, 2017 was $28.5 million, or $0.61 per diluted share. Non-GAAP net income in the same quarter of 2016 was $2.5 million, or $0.05 per diluted share. This quarterly non-GAAP net income measure, more fully described below under "Non-GAAP Financial Measures," excludes share-based compensation charges, non-cash interest charges on our debt, restructuring expenses, changes in the fair value of acquired contingent consideration, asset impairment charges, gain on sale of assets and acquisition-related expenses. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

Financial Results – Full Year Ended December 31, 2017

AMPYRA (dalfampridine) Extended Release Tablets, 10 mg – For the full year ended December 31, 2017 net revenue was $543.3 million compared to $492.8 million for full year 2016. Full year 2017 net revenue increased 10.2% over 2016.

Royalty Revenue – For the full year ended December 31, 2017, the Company reported royalty revenue of $29.5 million compared to $17.2 million for the full year 2016. The Company reported FAMPYRA royalties from sales outside of the U.S. of $11.6 million compared to $10.6 million for the full year 2016. Royalty revenue related to the authorized generic version of Zanaflex was $2.6 million compared to $3.9 million for the full year 2016. Additionally, the Company reported $15.3 million in royalties for Selincro for the full year 2017, which includes $13.0 million of royalty revenue related to the Selincro royalty transaction.

Research and development (R&D) expenses for the full year ended December 31, 2017 were $166.1 million, including $9.7 million of share-based compensation, compared to $203.4 million, including $10.6 million of share-based compensation for the full year 2016

Sales, general and administrative (SG&A) expenses for the full year ended December 31, 2017 were $181.6 million, including $23.1 million of share-based compensation, compared to $235.4 million, including $25.8 million of share-based compensation for the full year 2016.

Asset impairment charges for the full year ended December 31, 2017 include $233.5 million for tozadenant, $23.8 million for SYN120, and $39.4 million for Selincro.

Benefit from income taxes for the full year ended December 31, 2017 was $28.5 million, including $14.1 million of cash taxes compared to a benefit from income taxes of $6.7 million, including $4.3 million of cash taxes for the full year 2016.

For the full year ended December 31, 2017, the Company reported a GAAP net loss of $(223.4) million, or $(4.86) per diluted share. GAAP net loss for the full year 2016 was $(34.6) million, or $(0.76) per diluted share.

Non-GAAP net income for the full year ended December 31, 2017 was $80.7 million, or $1.75 per diluted share. Non-GAAP net income for the full year ended December 31, 2016 was $11.5 million, or $0.25 per diluted share. This full year non-GAAP net income measure, more fully described below under "Non-GAAP Financial Measures," excludes share-based compensation charges, non-cash interest charges on our debt, restructuring expenses, changes in the fair value of acquired contingent consideration, asset impairment charges, gain on sale of assets, realized foreign currency loss (gain) and acquisition related expenses. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

At December 31, 2017, the Company had cash and cash equivalents of $307.1 million.

2018 Financial Guidance

R&D expenses for the full year 2018 are expected to be $100-$110 million and include manufacturing expenses associated with INBRIJA. This guidance is a non-GAAP projection that excludes share-based compensation as more fully described below under "Non-GAAP Financial Measures."

SG&A expenses for the full year 2018 are expected to be $170-$180 million. This guidance is a non-GAAP projection that excludes share-based compensation as more fully described below under "Non-GAAP Financial Measures."

Year-end cash balance for 2018 is projected to be over $300 million

Fourth Quarter 2017 Pipeline and Corporate Updates

INBRIJA (levodopa inhalation powder) Next Steps

The Company resubmitted the NDA for INBRIJA in December 2017. The FDA is expected to inform the Company if the submission has been deemed complete and permits a full review in February 2018.

The Company expects to file a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) in Q1 2018.

AMPYRA (dalfampridine) Patent Appeal

In November, 2017, the Company and the defendants filed reply briefs for the appeal to the U.S. Court of Appeals for the Federal Circuit of the District Court’s decision in the AMPYRA patent litigation. The date for oral argument is expected in the first half of 2018.

Both BIO and PhRMA filed amicus briefs in support of the Company’s appeal, raising important issues in conjunction with biopharmaceutical innovation.

Royalty Monetization Transactions/ZANAFLEX (tizanidine hydrochloride) Franchise Sale

In November, 2017, the Company announced royalty monetization transactions of $53 million for FAMPYRA and SELINCRO.

The Company also announced the sale of ZANAFLEX and ZANAFLEX CAPSULES for $4 million.

SYN120 Phase 2 Data in Parkinson’s disease

Data from the Phase 2 proof-of-concept study for SYN120 showed that several of the outcome measures trended in favor of drug versus placebo; neither the primary nor key secondary endpoints achieved statistical significance.

The Company continues to review the data, which will be presented at an upcoming medical meeting.

Tozadenant Program Discontinued

In November, 2017, the Company discontinued its clinical development program for tozadenant, an investigational treatment for Parkinson’s disease. The Company made this decision based on the emergence of serious adverse events in its Phase 3 program.

Webcast and Conference Call

The Company will host a conference call today at 8:30 a.m. ET. To participate, dial

(866) 393-4306 (domestic) or (734) 385-2616 (international); access code 8789908. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 11:30 a.m. ET on February 15, 2018 until 11:59 p.m. ET on March 15, 2018. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international); reference code 8789908. The archived webcast will be available in the Investor Relations section of the Acorda website.

Non-GAAP Financial Measures

This press release includes financial results prepared in accordance with accounting principles generally accepted in the United States (GAAP), and also certain historical and forward-looking non-GAAP financial measures. In particular, Acorda has provided non-GAAP net income, adjusted to exclude the items below, and has provided 2018 guidance for R&D and SG&A expenses on a non-GAAP basis. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, the Company believes the presentation of non-GAAP net income, when viewed in conjunction with our GAAP results, provides investors with a more meaningful understanding of our ongoing and projected operating performance because this measure excludes (i) non-cash compensation charges and benefits that are substantially dependent on changes in the market price of our common stock, (ii) non-cash interest charges related to the accounting for our outstanding convertible debt which are in excess of the actual interest expense owing on such convertible debt as well as non-cash interest related to the Fampyra monetization, non-cash interest charges related to our asset based loan which was terminated in 2017 and acquired Biotie debt, (iii) changes in the fair value of acquired contingent consideration which do not correlate to our actual cash payment obligations in the relevant periods, (iv) acquisition related expenses and related foreign currency losses and gains that pertain to a non-recurring event, (v) corporate restructuring expenses that pertain to a non-recurring event, (vi) asset impairments which are non-cash charges that relate to program terminations that are not routine to the operation of the business, and (vii) gain on sale of assets that pertains to a non-recurring event. The Company believes its non-GAAP net income measure helps indicate underlying trends in the Company’s business and is important in comparing current results with prior period results and understanding projected operating performance. Also, management uses this non-GAAP financial measure to establish budgets and operational goals, and to manage the Company’s business and to evaluate its performance.

In addition to non-GAAP net income, we have provided 2018 guidance for R&D and SG&A expenses on a non-GAAP basis. Due to the forward looking nature of this information, the amount of compensation charges and benefits needed to reconcile these measures to the most directly comparable GAAP financial measures is dependent on future changes in the market price of our common stock and is not available at this time. The Company believes that these non-GAAP measures, when viewed in conjunction with our GAAP results, provide investors with a more meaningful understanding of our ongoing and projected R&D and SG&A expenses. Also, management uses these non-GAAP financial measures to establish budgets and operational goals, and to manage the Company’s business and to evaluate its performance.

Incyte Reports 2017 Fourth-Quarter and Year-End Financial Results, Provides 2018 Financial Guidance and Updates on Key Clinical Programs

On February 15, 2018 Incyte Corporation (Nasdaq: INCY) today reports 2017 fourth-quarter and year-end financial results, highlighting both strong growth in total revenue and the significant progress being made across the product portfolio (Press release, Incyte, FEB 15, 2018, View Source [SID1234523989]).

"2017 was another successful year for Incyte with a fast-growing revenue line and an expanded portfolio of later-stage development candidates that we expect to drive our future growth," stated Hervé Hoppenot, Incyte’s Chief Executive Officer. "As we begin 2018, we look forward to key newsflow events in the first half of the year, including the initial results of the ECHO-301 trial of epacadostat in melanoma and the REACH1 trial of ruxolitinib in steroid-refractory acute GVHD, as well as FDA action on the resubmission of the baricitinib NDA for rheumatoid arthritis."

Portfolio Update

Oncology — key highlights

The pivotal REACH1 trial evaluating ruxolitinib in patients with steroid-refractory acute graft-versus-host disease (GVHD) has completed enrollment and results are expected in the first half of 2018. If successful, Incyte expects to submit an sNDA seeking approval of ruxolitinib in this indication.

Initial results, based on progression-free survival, from the pivotal ECHO-301 trial of epacadostat plus pembrolizumab in patients with unresectable or metastatic melanoma are expected in the first half of 2018. In collaboration with both Merck and Bristol-Myers Squibb, we have recently opened eight new pivotal trials of epacadostat plus PD-1 antagonists.

Initial data from the trial evaluating INCB54828 in patients with cholangiocarcinoma are expected in 2018.

Status updates for Incyte’s most advanced clinical programs are provided below.

Indication

Status Update

Ruxolitinib
(JAK1/JAK2)

Steroid-refractory acute GVHD

Pivotal Phase 2 (REACH1); Phase 3 (REACH2)

Ruxolitinib
(JAK1/JAK2)

Steroid-refractory chronic GVHD

Phase 3 (REACH3)

Ruxolitinib
(JAK1/JAK2)

Essential thrombocythemia

Phase 2 (RESET)

Itacitinib
(JAK1)

Treatment-naïve acute GVHD

Phase 3 (GRAVITAS-301)

Itacitinib
(JAK1)

NSCLC

Phase 1/2 in combination with osimertinib (EGFR)

Epacadostat
(IDO1)

Melanoma

Phase 3 (ECHO-301) in combination with pembrolizumab (PD-1)

Epacadostat
(IDO1)

Renal cancer

Phase 3 (ECHO-302) in combination with pembrolizumab (PD-1)

Epacadostat
(IDO1)

Bladder cancer

Phase 3 (ECHO-303 & ECHO-307) in combination with pembrolizumab (PD-1)

Epacadostat
(IDO1)

Head & neck cancer

Phase 3 (ECHO-304) in combination with pembrolizumab (PD-1)

Epacadostat
(IDO1)

NSCLC

Phase 3 (ECHO-305 & ECHO-306) in combination with pembrolizumab (PD-1)

Epacadostat
(IDO1)

NSCLC

Phase 3 (ECHO-309) in combination with nivolumab (PD-1)

Epacadostat
(IDO1)

Head & neck cancer

Phase 3 (ECHO-310) in combination with nivolumab (PD-1)

Epacadostat
(IDO1)

NSCLC

Phase 3 in combination with durvalumab (PD-L1) expected to begin in H1 2018

MGA012
(PD-1)(1)

Solid tumors

Phase 1 dose-escalation completed, monotherapy expansion cohorts ongoing

INCB50465
(PI3Kō)

DLBCL

Phase 2 (CITADEL-202)

INCB50465
(PI3Kō)

Follicular lymphoma

Phase 2 (CITADEL-203)

INCB50465
(PI3Kō)

Marginal zone lymphoma

Phase 2 (CITADEL-204)

INCB50465
(PI3Kō)

Mantle cell lymphoma

Phase 2 (CITADEL-205)

INCB54828
(FGFR1/2/3)

Bladder cancer

Phase 2 (FIGHT-201)

INCB54828
(FGFR1/2/3)

Cholangiocarcinoma

Phase 2 (FIGHT-202)

Notes:

(1) MGA012 licensed from MacroGenics

A brief status update for Incyte’s earlier-stage clinical candidates is provided below.

Status Update

INCB57643
(BRD)

First-in-man data presented at ASH (Free ASH Whitepaper) 2017, showing optimized PK profile for combination therapy

INCB53914
(PIM)

First-in-man data at ASH (Free ASH Whitepaper) 2017; development expected to focus on combination therapy, including with JAK and PI3Kδ inhibition in hematological malignancies

INCB52793
(JAK1)

150x greater selectivity for JAK1 over JAK2 in preclinical studies; evaluating combination cohorts with azacitadine in AML

INCB59872
(LSD1)

Epigenetic mechanism targeting cell differentiation; evaluating both oncology indications and sickle-cell disease

INCB62079
(FGFR4)

250x greater selectivity for FGFR4 over FGFR1/2/3; initial development expected to focus on hepatocellular carcinoma

INCB81776
(AXL/MER)

Expected to enter clinical trials in 2018

INCB01158
(ARG)(1)

Novel mechanism targeting myeloid cells; development expected to focus on combination therapy, including IDO1, PD-1 and chemotherapy combinations

INCAGN1876
(GITR)(2)

Dose escalation completed; development expected to focus on combination therapy, including IDO1, PD-1 and CTLA-4 combinations

INCAGN1949
(OX40)(2)

Dose escalation completed; development expected to focus on combination therapy, including PD-1 and CTLA-4 combinations

INCAGN2390
(TIM-3)(2)

Expected to enter clinical trials in 2018

INCAGN2385
(LAG-3)(2)

Expected to enter clinical trials in 2018

Notes:

(1) INCB01158 co-developed with Calithera

(2) INCAGN1876, INCAGN1949, INCAGN2390 and INCAGN2385 from discovery alliance with Agenus

Non-oncology

Indication

Status Update

Topical ruxolitinib
(JAK1/JAK2)

Atopic dermatitis, vitiligo

Phase 2

Partnered — key highlights

In December, Lilly announced that it had resubmitted the New Drug Application (NDA) for baricitinib to the U.S. Food & Drug Administration (FDA). This was classified as a Class II resubmission, which began a new six-month review cycle. Lilly also announced that it has initiated a pivotal trial of baricitinib in patients with moderate-to-severe atopic dermatitis.

Novartis has stated that it now anticipates submitting an NDA for capmatinib, a potent and selective MET inhibitor licensed from Incyte, in 2019.

Indication

Status Update

Baricitinib (JAK1/JAK2)(1)

Rheumatoid arthritis

Approved in Europe and Japan; NDA resubmitted to FDA

Baricitinib (JAK1/JAK2)(1)

Atopic dermatitis

Phase 3

Baricitinib (JAK1/JAK2)(1)

Psoriatic arthritis

Lilly expects the Phase 3 program to begin in 2018

Baricitinib (JAK1/JAK2)(1)

Systemic lupus erythematosus

Phase 2

Capmatinib (MET)(2)

Non-small cell lung cancer, liver cancer

Phase 2 in EGFR wild-type, ALK negative NSCLC patients with MET amplification and mutation

Notes:

(1) Baricitinib licensed to Lilly

(2) Capmatinib licensed to Novartis

2017 Fourth-Quarter and Year-End Financial Results (GAAP)

Revenues For the quarter ended December 31, 2017, net product revenues of Jakafi were $302 million as compared to $238 million for the same period in 2016, representing 27 percent growth. For the twelve months ended December 31, 2017, net product revenues of Jakafi were $1.1 billion as compared to $853 million for the same period in 2016, representing 33 percent growth. For the quarter ended December 31, 2017, net product revenues of Iclusig were $19 million as compared to $13 million for the same period in 2016. For the twelve months ended December 31, 2017, net product revenues of Iclusig were $67 million as compared to $30 million for the same period in 2016(1).

For the quarter and twelve months ended December 31, 2017, product royalties from sales of Jakavi, which has been out-licensed to Novartis outside of the United States, were $48 million and $152 million, respectively, as compared to $33 million and $111 million for the same periods in 2016. For the quarter and twelve months ended December 31, 2017, product royalties from sales of Olumiant outside of the United States from Lilly were $5 million and $9 million, respectively.

For the quarter and twelve months ended December 31, 2017, milestone and contract revenues were $70 million and $175 million, respectively, as compared to $43 million and $113 million for the same periods in 2016. The milestone and contract revenues in 2017 relate to milestones earned from our collaborative partners.

For the quarter ended December 31, 2017, total revenues were $444 million as compared to $326 million for the same period in 2016. For the twelve months ended December 31, 2017, total revenues were $1.5 billion as compared to $1.1 billion for the same period in 2016.

(1) In June 2016, Incyte obtained an exclusive license from ARIAD to develop and commercialize Iclusig in Europe and other select ex-U.S. countries.

Year Over Year Revenue Growth

(in thousands, unaudited)

Three Months Ended

Twelve Months Ended

December 31,

2017

2016

Change

2017

2016

Change

Revenues:

Jakafi net product revenues

302,348

237,531

1,133,392

852,816

Iclusig net product revenues

19,461

12,867

—

66,920

29,588

—

Product royalty revenues

52,314

33,225

160,791

110,711

Product-related revenues

374,123

283,623

1,361,103

993,115

Milestone and contract revenues

70,000

42,869

—

175,000

112,512

—

Other revenues

—

113

—

Total revenues

444,156

326,498

1,536,216

1,105,719

Research and development expenses Research and development expenses for the quarter ended December 31, 2017 were $447 million as compared to $162 million for the same period in 2016. For the quarter ended December 31, 2017, research and development expenses were comprised of $150 million related to our collaboration and license agreement with MacroGenics and $297 million of ongoing expenses.

Research and development expenses for the twelve months ended December 31, 2017 were $1.3 billion as compared to $582 million for the same period in 2016. For the twelve months ended December 31, 2017, research and development expenses were comprised of $359 million of upfront consideration and milestone expense related to our collaboration and license agreements with Agenus, Calithera, MacroGenics and Merus, $12 million related to in-process research and development asset impairment and $955 million of ongoing expenses.

Included in ongoing research and development expenses for the quarter and twelve months ended December 31, 2017 were non-cash expenses related to equity awards to our employees of $23 million and $90 million, respectively.

Selling, general and administrative expenses Selling, general and administrative expenses for the quarter and twelve months ended December 31, 2017 were $98 million and $366 million, respectively, as compared to $96 million and $303 million for the same periods in 2016. Increased selling, general and administrative expenses were driven primarily by additional costs related to the commercialization of Jakafi and the geographic expansion in Europe. Included in selling, general and administrative expenses for the quarter and twelve months ended December 31, 2017 were non-cash expenses related to equity awards to our employees of $11 million and $43 million, respectively.

Change in fair value of acquisition-related contingent consideration The change in fair value of acquisition-related contingent consideration for the quarter and twelve months ended December 31, 2017 was $10 million and $8 million, respectively, as compared to $7 million and $17 million for the same periods in 2016. The change in fair value of acquisition-related contingent consideration represents the fair market value adjustments of the Company’s contingent liability related to the acquisition of the European business of ARIAD Pharmaceuticals, Inc.

Unrealized loss on long term investments Unrealized loss on long term investments for the quarter ended December 31, 2017 was $22 million as compared to $24 million for the same period in 2016. The unrealized loss on long term investments for the twelve months ended December 31, 2017 was $24 million as compared to $3 million for the same period in 2016. The unrealized loss on long term investments for the quarter and twelve months ended December 31, 2017 represents the fair market value adjustments of the Company’s investments in Agenus and Merus.

Expense related to senior note conversions Expense related to senior note conversions for the twelve months ended December 31, 2017 was $55 million related to the conversions of certain of our 2018 and 2020 convertible senior notes.

Net income (loss) Net loss for the quarter ended December 31, 2017 was $150 million, or $0.71 per basic and diluted share, as compared to net income of $9 million, or $0.05 per basic and diluted share for the same period in 2016. Net loss for the twelve months ended December 31, 2017 was $313 million, or $1.53 per basic and diluted share, as compared to net income of $104 million, or $0.55 per basic and $0.54 per diluted share for the same period in 2016.

As described below, in 2018 Incyte will begin reporting certain Non-GAAP financial measures, which should be considered in conjunction with Incyte’s GAAP reporting. Under Incyte’s definition of Non-GAAP measures, Non-GAAP net income for the quarter and twelve months ended December 31, 2017 was $4 million and $131 million, respectively.

Cash, cash equivalents and marketable securities position As of December 31, 2017, cash, cash equivalents and marketable securities totaled $1.2 billion as compared to $809 million as of December 31, 2016. The increase in cash, cash equivalents and marketable securities from December 31, 2016 to December 31, 2017 is primarily due to the public offering of 4,945,000 shares of our common stock resulting in net proceeds of $649 million.

Non-GAAP Information

The financial measures other than Non-GAAP net income presented in this press release for the three and twelve months ended December 31, 2017 have been prepared by the Company in accordance with U.S. Generally Accepted Accounting Principles ("GAAP"). Management has chosen to present Non-GAAP net income for the three and twelve months ended December 31, 2017 and to release both GAAP and Non-GAAP financial guidance for the year ending December 31, 2018 in belief that this Non-GAAP information is useful for investors, when considered in conjunction with Incyte’s GAAP financial guidance. Management uses such information internally and externally for establishing budgets, operating goals and financial planning purposes. These metrics are also used to manage the Company’s business and monitor performance. The Company adjusts, where appropriate, for both revenues and expenses in order to reflect the Company’s core operations. The Company believes these adjustments are useful to investors by providing an enhanced understanding of the financial performance of the Company’s core operations. The metrics have been adopted to align the Company with disclosures provided by industry peers. A reconciliation of GAAP net loss to Non-GAAP net income for the three and twelve months ended December 31, 2017 has been included at the end of this press release.

Guidance related to research and development and selling, general and administrative expenses does not include estimates associated with any potential future strategic transactions.

Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used in conjunction with and to supplement Incyte’s operating results as reported under GAAP. Non-GAAP measures may be defined and calculated differently by other companies in our industry.

2018 Financial Guidance

The Company has provided full year 2018 financial guidance, as detailed below.

GAAP and Non-GAAP Jakafi net product revenues

$1,350 – $1,400 million

GAAP and Non-GAAP Iclusig net product revenues

$80 – $85 million

GAAP Cost of product revenues

$85 – $95 million

Non-GAAP Cost of product revenues(1)

$64 – $74 million

GAAP Research and development expenses

$1,200 – $1,300 million

Non-GAAP Research and development expenses(2)

$1,077 – $1,172 million

GAAP Selling, general and administrative expenses

$515 – $535 million

Non-GAAP Selling, general and administrative expenses(3)

$465 – $480 million

GAAP Change in fair value of acquisition-related contingent consideration

$30 million

Non-GAAP Change in fair value of acquisition-related contingent consideration(4)

$0 million

(1) Adjusted to exclude the amortization of licensed intellectual property for Iclusig relating to the acquisition of the European business of ARIAD Pharmaceuticals, Inc.

(2) Adjusted to exclude the estimated cost of stock-based compensation and upfront consideration of approximately $13 million relating to the Syros Pharmaceuticals, Inc. collaboration.

(3) Adjusted to exclude the estimated cost of stock-based compensation.

(4) Adjusted to exclude the change in fair value of estimated future royalties relating to sales of Iclusig in the licensed territory relating to the acquisition of the European business of ARIAD Pharmaceuticals, Inc.

The selling, general and administrative expense guidance includes approximately $125 million in epacadostat GAAP and Non-GAAP pre-launch expenses which we expect to incur in the second half of the year.

Future Non-GAAP financial measures may also exclude upfront and ongoing milestones relating to third-party collaboration partners, impairment of goodwill or other assets, changes in the fair value of equity investments in our collaboration partners, non-cash interest expense related to the amortization of the initial discount on our 2018 and 2020 Senior Notes and the impact on our tax provision of discrete changes in our valuation allowance position on deferred tax assets.

Conference Call and Webcast Information

Incyte will hold its 2017 fourth-quarter and year-end financial results conference call and webcast this morning at 8:00 a.m. ET. To access the conference call, please dial 877-407-3042 for domestic callers or 201-389-0864 for international callers. When prompted, provide the conference identification number, 13675376.

If you are unable to participate, a replay of the conference call will be available for 30 days. The replay dial-in number for the United States is 877-660-6853 and the dial-in number for international callers is 201-612-7415. To access the replay you will need the conference identification number, 13675376.

The conference call will also be webcast live and can be accessed at www.incyte.com in the Investors section under "Events and Presentations".

Acorda Provides Financial and Pipeline Update for Fourth Quarter and Year End 2017

On February 15, 2018 Acorda Therapeutics, Inc. (Nasdaq:ACOR) reported a financial and pipeline update for the fourth quarter and full year ended December 31, 2017 (Press release, Acorda Therapeutics, FEB 15, 2018, View Source [SID1234523984]).

Fourth Quarter 2017 Financial Results

The Company reported a GAAP net loss attributable to Acorda of $(171.1) million for the quarter ended December 31, 2017, or $(3.70) per diluted share. GAAP net loss in the same quarter of 2016 was $(3.1) million, or $(0.07) per diluted share.

Financial Results – Full Year Ended December 31, 2017

Asset impairment charges for the full year ended December 31, 2017 include $233.5 million for tozadenant, $23.8 million for SYN120, and $39.4 million for Selincro.

At December 31, 2017, the Company had cash and cash equivalents of $307.1 million.

2018 Financial Guidance

AMPYRA net revenue is expected to be $330-$350 million. The Company expects to maintain exclusivity of AMPYRA at least through July 30, 2018; this guidance is subject to change based on the appellate court’s decision.
R&D expenses for the full year 2018 are expected to be $100-$110 million and include manufacturing expenses associated with INBRIJA. This guidance is a non-GAAP projection that excludes share-based compensation as more fully described below under "Non-GAAP Financial Measures."
SG&A expenses for the full year 2018 are expected to be $170-$180 million. This guidance is a non-GAAP projection that excludes share-based compensation as more fully described below under "Non-GAAP Financial Measures."
Year-end cash balance for 2018 is projected to be over $300 million
Fourth Quarter 2017 Pipeline and Corporate Updates

INBRIJA (levodopa inhalation powder) Next Steps
The Company resubmitted the NDA for INBRIJA in December 2017. The FDA is expected to inform the Company if the submission has been deemed complete and permits a full review in February 2018.
The Company expects to file a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) in Q1 2018.
AMPYRA (dalfampridine) Patent Appeal
In November, 2017, the Company and the defendants filed reply briefs for the appeal to the U.S. Court of Appeals for the Federal Circuit of the District Court’s decision in the AMPYRA patent litigation. The date for oral argument is expected in the first half of 2018.
Both BIO and PhRMA filed amicus briefs in support of the Company’s appeal, raising important issues in conjunction with biopharmaceutical innovation.
Royalty Monetization Transactions/ZANAFLEX (tizanidine hydrochloride) Franchise Sale
In November, 2017, the Company announced royalty monetization transactions of $53 million for FAMPYRA and SELINCRO.
The Company also announced the sale of ZANAFLEX and ZANAFLEX CAPSULES for $4 million.
SYN120 Phase 2 Data in Parkinson’s disease
Data from the Phase 2 proof-of-concept study for SYN120 showed that several of the outcome measures trended in favor of drug versus placebo; neither the primary nor key secondary endpoints achieved statistical significance.
The Company continues to review the data, which will be presented at an upcoming medical meeting.
Tozadenant Program Discontinued
In November, 2017, the Company discontinued its clinical development program for tozadenant, an investigational treatment for Parkinson’s disease. The Company made this decision based on the emergence of serious adverse events in its Phase 3 program.
Webcast and Conference Call

The Company will host a conference call today at 8:30 a.m. ET. To participate, dial (866) 393-4306 (domestic) or (734) 385-2616 (international); access code 8789908. The presentation will be available on the Investors section of www.acorda.com.

Non-GAAP Financial Measures