On February 13, 2020 Advaxis, Inc. (Nasdaq: ADXS), a clinical-stage biotechnology company focused on the development and commercialization of immunotherapy products reported updated results from the combination arm of KEYNOTE-46 (Part B), the Company’s ongoing Phase 1/2 study investigating ADXS-PSA with KEYTRUDA (pembrolizumab) in patients with metastatic, castrate-resistant prostate cancer (mCRPC) at the ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium in San Francisco, California (Press release, Advaxis, FEB 13, 2020, View Source [SID1234554279]). The KEYNOTE-46 trial was conducted in conjunction with Merck (known as MSD outside the U.S. and Canada) and evaluated ADXS-PSA, one of Advaxis’ Listeria monocytogenes (Lm)-based immunotherapies, alone and in combination with KEYTRUDA, Merck’s anti-PD-1 therapy.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The presented survival data in patients with visceral metastases strengthens our confidence that ADXS-PSA in combination with KEYTRUDA has the potential to provide meaningful increases in median overall survival in patients with advanced, metastatic, castration-resistant prostate cancer," said Kenneth A. Berlin, President and Chief Executive Officer of Advaxis. "Importantly, these demonstrated impacts on survival have not been previously observed with immunotherapy in this advanced patient population leading us to actively assess next steps for the program with the hope of providing a much-needed new treatment for these patients with limited options."

Key findings presented by Mark N. Stein M.D., FACS, Associate Professor of Medical Oncology at Columbia University Medical Center and lead study investigator, titled, "KEYNOTE-046 (Part B): Effects of ADXS-PSA in combination with pembrolizumab on survival in metastatic, castration-resistant prostate cancer patients with or without prior exposure to docetaxel" include:

●Median overall survival (95% CI) of 33.7 months (15.4-NR) for patients treated with ADXS-PSA in combination with KEYTRUDA (n=37)
●Median overall survival (95% CI) of 16.0 months (6.4-34.6) for patients with prior docetaxel (n=20)
●Median overall survival (95% CI) of 16.4 months (4.0-NR) for patients with prior visceral metastasis (n=11; 10 of who had prior docetaxel)
72.4% (21/29) of evaluable patients showed stable disease
●38% of patients had PSA declines and 27% had >= 30% PSA decline from baseline
●The combination of ADXS-PSA and pembrolizumab appeared safe and tolerable in this heavily pretreated, unselected population of patients with MSI-High-negative mCRPC
●Treatment-related adverse events were mostly Grade 1-2, with no additive toxicity observed with combination therapy

Mark N. Stein M.D., FACS said, "These data are encouraging given the advanced nature of the patient population which includes those who have failed next generation hormonal agents and/or docetaxel, and now those with visceral metastasis." He added, "I am particularly enthusiastic to see increases in median overall survival to 16.4 months as compared to standard of care, which tends to be closer to 11 months in patients with measurable disease/visceral metastasis. This improvement, delivered with a generally safe and well-tolerated treatment regimen, warrants additional evaluation in larger studies and I look forward seeing the potential of a continued evaluation of ADXS-PSA in combination with KEYTRUDA."

KEYNOTE-046 was an open-label, multicenter, dose-determining safety and tolerability Phase 1/2 trial of 50 heavily pretreated patients conducted in two parts (Part A and Part B), with a Phase 2 expansion cohort. The objective of the study was to evaluate ADXS-PSA alone (Part A) and in combination with KEYTRUDA (Part B) for primary endpoints that include safety, tolerability and dosing. Secondary endpoints included anti-tumor activity, progression-free survival and overall survival, and exploratory endpoints that include associations between biomarkers of immunologic response (serum PSA) with clinical outcomes. Enrollment in the study has been completed and the database lock occurred on January 28, 2020. The majority of treatment-related adverse events consisted of transient and reversible Grade 1-2 chills/rigors, fever, hypotension, nausea and fatigue. The combination of ADXS-PSA and KEYTRUDA has appeared to be well-tolerated, to date, with no additive toxicity observed.

About KEYNOTE-046

KEYNOTE-046 (NCT02325557) was a Phase 1/2 open-label, multicenter, dose-determination and expansion trial that evaluates the safety, tolerability and preliminary clinical activity of ADXS-PSA as monotherapy (Part A; n=14 [13 treated]), and in combination with KEYTRUDA (Part B; n= 37) in heavily pretreated patients with progressive and refractory mCRPC.

On February 13, 2020 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO) reported business highlights and financial results for the fourth quarter and year ended December 31, 2019 (Press release, Agios Pharmaceuticals, FEB 13, 2020, View Source [SID1234554271]). In addition, Agios highlighted key 2020 corporate milestones and data presentations for its clinical development programs.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"On the heels of a busy and productive 2019, I’m more confident than ever in the strength of our team and our ability to make a meaningful impact on the lives of patients through great science, a deep pipeline and differentiated therapies," said Jackie Fouse, Ph.D., chief executive officer at Agios. "In 2020, our clinical development team is focused on advancing our Phase 3 PK deficiency studies in order to submit a new drug application in 2021, finalizing our pivotal development plan for the PK activation program in thalassemia and establishing proof-of-concept in sickle cell disease. In addition, we are driving enrollment in several Phase 3 studies for our IDH inhibitors in both malignant hematology and solid tumors. Our commercial team is focused on achieving an ambitious revenue target for TIBSOVO and increasing market development activities in preparation for a potential launch in PK deficiency."

ANTICIPATED 2020 KEY MILESTONES

Agios expects the following key milestones in 2020:

Hematologic Malignancies

Deliver full-year U.S. revenue for TIBSOVO of $105-115 million

Receive European Medicines Agency CHMP opinion for TIBSOVO in relapsed or refractory acute myeloid leukemia (AML) with an IDH1 mutation by year-end

Complete enrollment of the Phase 3 AGILE trial of TIBSOVO in combination with azacitidine in adult patients with previously untreated IDH1 mutant AML by year-end
enrollment of the relapsed or refractory myelodysplastic syndrome arm of the TIBSOVO Phase 1 study of IDH1 mutant advanced hematologic malignancies by year-end

Solid Tumors

File supplemental new drug application (sNDA) for TIBSOVO in previously treated IDH1 mutant cholangiocarcinoma by year-end

Rare Genetic Diseases

Announce topline data for ACTIVATE and ACTIVATE-T pivotal trials for mitapivat in adults with pyruvate kinase (PK) deficiency by year-end

Submit updated data from the Phase 2 study of mitapivat in thalassemia for presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress in June and finalize pivotal development strategy by year-end

Achieve proof-of-concept for mitapivat in sickle cell disease by mid-2020

Receive investigational new drug (IND) clearance for AG-946, a next generation PKR activator, and initiate a first-in-human study in healthy volunteers in the first half of 2020

Research

Achieve at least one new development candidate by year-end

FOURTH QUARTER AND FULL YEAR 2019 FINANCIAL RESULTS

Revenue: Total revenue for the fourth quarter of 2019 was $35.4 million, which includes $12.9 million in collaboration revenue, $19.6 million of net product revenue from sales of TIBSOVO and $3.0 million in royalty revenue from net global sales of IDHIFA under our collaboration agreement with Celgene. This compares to $30.0 million for the fourth quarter of 2018, which included $18.4 million in collaboration revenue, $9.4 million of net product revenue from U.S. sales of TIBSOVO and $2.2 million in royalty revenue from net global sales of IDHIFA. Total revenue for the year ended December 31, 2019 was $117.9 million compared to $94.4 million for the year ended December 31, 2018. The increase in 2019 revenue was primarily driven by net U.S. sales of TIBSOVO and were offset by a decline in collaboration revenue due to the recognition of a milestone from Celgene and the upfront payment from CStone in 2018.

Cost of Sales: Cost of sales were $0.3 million for the fourth quarter of 2019 compared to $0.7 million for the fourth quarter of 2018, and $1.3 million for the year ended December 31, 2019 compared to $1.4 million for the comparable period in 2018.

Research and Development (R&D) Expenses: R&D expenses were $106.2 million for the fourth quarter of 2019 compared to $93.8 million for the fourth quarter of 2018 and $410.9 million for the year ended December 31, 2019 compared to $341.3 million for the comparable period in 2018. The increase in R&D expense was primarily attributable to clinical trial activity for mitapivat in PK deficiency and thalassemia; start-up costs for the vorasidenib Phase 3 INDIGO study in low-grade glioma, including required clinical pharmacology studies and companion diagnostic development; and ongoing enrollment in the TIBSOVO Phase 3 AGILE and HOVON frontline AML combination studies. R&D expense also increased as a result of ongoing research efforts across our discovery platform programs.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $34.8 million for the fourth quarter of 2019 compared to $31.9 million for the fourth quarter of 2018, and $132.0 million for the year ended December 31, 2019 compared to $114.1 million for the year ended December 31, 2018. The increase in SG&A expense was primarily attributable to increased investment in marketing activities in preparation for the potential launch of mitapivat and personnel costs related to increased headcount to support growing operations.

Net Loss: Net loss was $102.4 million for the fourth quarter of 2019 compared to $91.8 million for the fourth quarter of 2018, and $411.5 million for the year ended December 31, 2019 compared to a net loss of $346.0 million for the year ended December 31, 2018.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of December 31, 2019 were $717.8 million compared to $805.4 million as of December 31, 2018. The change in cash was primarily driven by expenditures to fund operations of $464.4 million offset by the net proceeds of $277.2 million from the November follow-on offering and cash inflows of $99.3 million from product sales, stock option exercises, royalty revenue, and collaboration reimbursements and milestones. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2019, together with anticipated product and royalty revenue, anticipated interest income, and anticipated expense reimbursements under our collaboration and license agreements, but excluding any additional collaboration-related payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2021.

CONFERENCE CALL INFORMATION

Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss fourth quarter and full year 2019 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and referring to conference ID 4195413. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

On February 13, 2020 MacroGenics, Inc. (Nasdaq: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, reported that the Company will release its financial results for the fourth quarter and full year 2019 after the market closes on Tuesday, February 25, 2020 (Press release, MacroGenics, FEB 13, 2020, View Source [SID1234554270]). MacroGenics will host a conference call to discuss the financial results and recent corporate progress on Tuesday, February 25, 2020 at 4:30 p.m. ET. The conference call can be accessed by dialing (877) 303-6253 (domestic) or (973) 409-9610 (international) five minutes prior to the start of the call and providing the Conference ID 5581596.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The listen-only webcast of the conference call can be accessed under "Events & Presentations" in the Investor Relations section of the Company’s website at View Source A recorded replay of the webcast will be available shortly after the conclusion of the call and archived on the Company’s website for 30 days following the call.

On February 13, 2020 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2019 (Press release, Blueprint Medicines, FEB 13, 2020, View Source [SID1234554269]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"In 2020, we will complete our evolution into a fully-integrated global biopharmaceutical company and fortify our leadership in the field of precision medicine with further expansion of our research pipeline," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "Over the course of the year, we expect a sustained cadence of catalytic milestones across our portfolio, which began with the commercial launch of AYVAKIT in January and will continue with the presentation of updated data for avapritinib in indolent systemic mastocytosis and the completion of our rolling NDA submission for pralsetinib for RET fusion-positive non-small cell lung cancer later this quarter. Ultimately, we believe these and other anticipated achievements throughout the year will transform the profile of our company and create substantial value for patients and healthcare providers."

Fourth Quarter 2019 Highlights and Recent Progress

Avapritinib: gastrointestinal stromal tumors (GIST)

Received U.S. Food and Drug Administration (FDA) approval of AYVAKIT for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Read the press release here and visit www.AYVAKIT.com for full Prescribing Information.

Announced that the FDA has extended the Prescription Drug User Fee Act (PDUFA) date for the company’s new drug application (NDA) seeking accelerated approval of avapritinib for the treatment of adults with fourth-line GIST, by three months from February 14, 2020 to May 14, 2020. Read the press release here.

Avapritinib: systemic mastocytosis (SM)

Reported initial data from Part 1 of the PIONEER trial of avapritinib in patients with indolent SM at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2019 showing rapid and robust reductions in serum tryptase, a measure of mast cell burden, at all dose levels tested. Avapritinib was well-tolerated, and most reported adverse events were Grade 1 or 2. No patients discontinued due to an adverse event. Read the press release here.

Announced updated data from Part 1 of the PIONEER trial will be reported in a late-breaking oral presentation at the American Academy of Allergy, Asthma & Immunology (AAAAI) annual meeting on March 14, 2020.

Pralsetinib: RET-altered cancers

Announced centrally reviewed top-line data from the Phase 1/2 ARROW trial of pralsetinib in patients with RET fusion-positive non-small cell lung cancer (NSCLC) treated with pralsetinib at 400 mg QD, which is the proposed indicated dose. In patients previously treated with platinum-based chemotherapy, the overall response rate (ORR) was 61 percent (95% CI: 50-72%; two responses pending confirmation). In treatment-naïve patients, the ORR was 73 percent (95% CI: 52-88%; all responses confirmed), with 12 percent of patients achieving a complete response. The median duration of response, regardless of prior treatment, was not reached. Pralsetinib was well-tolerated, and most AEs were Grade 1 or 2. Across all patients enrolled in the ARROW trial and treated with pralsetinib at 400 mg QD, only four percent of patients discontinued treatment due to treatment-related adverse events. Read the press release here.

Announced the initiation of a rolling new drug application (NDA) submission to the FDA for pralsetinib for the treatment of RET fusion-positive NSCLC.

Activated the first clinical trial site for the Phase 3 AcceleRET Lung trial in patients with first-line RET fusion-positive NSCLC.

Fisogatinib: hepatocellular carcinoma (HCC)

Dosed the first patient in a Phase 1b/2 trial evaluating fisogatinib in combination with CS1001 for the treatment of locally advanced or metastatic HCC, under Blueprint Medicine’s collaboration with CStone Pharmaceuticals. Read the press release here.

Research portfolio

Nominated a potential first-in-class development candidate for the treatment of resistant EGFR-positive triple mutant NSCLC.

Corporate

Closed an underwritten public offering of 4,710,144 shares of common stock at a public offering price of $69.00 per share. Blueprint Medicines received estimated net proceeds of approximately $308.2 million, after deducting underwriting discounts and commissions and estimated offering expenses.

Key Upcoming Milestones

The company expects to achieve the following milestones in the first half of 2020.

Present updated data from Part 1 of the PIONEER trial of avapritinib in indolent SM at AAAAI Annual Meeting in the first quarter of 2020.

Complete the submission of a rolling NDA to the FDA for pralsetinib for RET fusion-positive NSCLC in the first quarter of 2020.

Report top-line data from the Phase 3 VOYAGER trial of avapritinib in third-line GIST early in the second quarter of 2020.

Gain FDA approval and, if approved, launch avapritinib in fourth-line GIST in the U.S. in the second quarter of 2020.

Report top-line data from the Phase 1/2 ARROW trial of pralsetinib in patients with previously treated RET mutant medullary thyroid cancer (MTC).

Submit an NDA to the FDA for pralsetinib for the treatment of patients with MTC previously treated with an approved multi-kinase inhibitor in the second quarter of 2020.

Submit a marketing authorization application to the European Medicines Agency for pralsetinib for RET fusion-positive NSCLC in the second quarter of 2020.

Initiate a Phase 1 trial of BLU-263, a next-generation KIT inhibitor, in healthy volunteers in the first half of 2020.

Fourth Quarter and Year End 2019 Financial Results

Cash Position: As of December 31, 2019, cash, cash equivalents and investments were $548.0 million, as compared to $494.0 million as of December 31, 2018. This increase was primarily related to $327.5 million in net proceeds received from the company’s April 2019 follow-on underwritten public offering and the $25.0 million upfront cash payment under the license agreement Clementia, partially offset by an increase in cash used in operating activities. Cash, cash equivalents and investments as of December 31, 2019 do not include the estimated net proceeds of approximately $308.2 million from the company’s follow-on underwritten public offering of common stock, which closed in January 2020.

Collaboration Revenues: Collaboration revenues were $51.5 million for the fourth quarter of 2019 and $66.5 million for the year ended December 31, 2019, as compared to $1.0 million for the fourth quarter of 2018 and $44.5 million for the year ended December 31, 2018. Collaboration revenue for the year ended December 31, 2019 consisted primarily of the $25.0 million upfront payment and a $20.0 million cash milestone payment due in the third quarter of 2020 under the license agreement with Clementia, an aggregate of $12.0 million in development and regulatory milestones that were achieved in 2019 under the CStone collaboration agreement and $8.2 million under the Roche collaboration agreement. Collaboration revenue for the year ended December 31, 2018 consisted primarily of the $40.0 million upfront payment under the CStone collaboration agreement and $4.5 million under the Roche collaboration agreement.

R&D Expenses: Research and development expenses were $88.6 million for the fourth quarter of 2019 and $331.5 million for the year ended December 31, 2019, as compared to $70.5 million for the fourth quarter of 2018 and $243.6 million for the year ended December 31, 2018. This increase was primarily due to increased clinical and manufacturing expenses driven by the company’s lead programs and increased personnel expenses. Research and development expenses included $7.6 million in stock-based compensation expenses for the fourth quarter of 2019 and $28.6 million in stock-based compensation expenses for the year ended December 31, 2019.

G&A Expenses: General and administrative expenses were $32.3 million for the fourth quarter of 2019 and $96.4 million for the year ended December 31, 2019, as compared to $13.6 million for the fourth quarter of 2018 and $47.9 million for the year ended December 31, 2018. This increase was primarily related to increased costs and personnel expenses associated with building the company’s commercial infrastructure and to support the overall growth of the business. General and administrative expenses included $8.1 million in stock-based compensation expenses for the fourth quarter of 2019 and $26.1 million in stock-based compensation expenses for the year ended December 31, 2019.

Net Loss: Net loss was $66.3 million for the fourth quarter of 2019 and $347.7 million for the year ended December 31, 2019, or a net loss per share of $1.35 and $7.27, respectively, as compared to a net loss of $80.3 million for the fourth quarter of 2018 and $236.6 million for the year ended December 31, 2018, or a net loss per share of $1.83 and $5.39, respectively.

Financial Guidance

Based on its current operating plans, Blueprint Medicines expects that its existing cash, cash equivalents and investments including the $308.2 million in estimated net proceeds from the January 2020 follow-on public offering, together with anticipated product revenues but excluding any additional potential option fees, milestone payments or other payments under its collaboration or license agreements, will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the second half of 2022.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:30 a.m. ET today to discuss fourth quarter and full year 2019 financial results and recent business activities. The conference call may be accessed by dialing (855) 728-4793 (domestic) or (503) 343-6666 (international) and referring to conference ID 26735762. A webcast of the conference call will be available in the Investors section of the Blueprint Medicines’ website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

On February 13, 2020 Incyte (Nasdaq:INCY) reported 2019 fourth quarter and year-end financial results, announces 2020 guidance and provides a status update on the Company’s development portfolio (Press release, Incyte, FEB 13, 2020, View Source [SID1234554268]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Revenue growth continues to be very strong, driven by robust demand across all three indications for Jakafi," stated Hervé Hoppenot, Chief Executive Officer, Incyte. "As we enter 2020, we have multiple opportunities for additional growth. We recently announced successful initial results from the Phase 3 TRuE-AD program, which we plan to include as part of the NDA in the fourth quarter of 2020 seeking approval of ruxolitinib cream for the treatment of patients with mild-to-moderate atopic dermatitis. We also look forward to the FDA decisions on the potential approvals of pemigatinib and capmatinib later this year. The recent progress made within both oncology and dermatology, as well as the recently announced MorphoSys collaboration for tafasitamab, positions Incyte very well as we deliver on our objectives for diversification and growth."

Portfolio Update

LIMBER – key highlights

The LIMBER program, a key development priority designed to maintain our Leadership In MPNs BEyond Ruxolitinib, is evaluating multiple monotherapy and combination strategies to deliver improved therapies for patients with myeloproliferative neoplasms (MPNs). The program has three key areas of focus: new formulations of ruxolitinib; JAK inhibitor-based combinations; and new targets beyond JAK inhibition.

A once-a-day formulation of ruxolitinib is being developed and is currently being evaluated in clinical pharmacology studies. Following positive proof-of-concept data of ruxolitinib plus parsaclisib in myelofibrosis (MF) patients with a suboptimal response to ruxolitinib monotherapy, a randomized pivotal trial is being prepared in this setting. Additional JAK-based combinations are either ongoing or in preparation.

Recruitment has been discontinued in the RESET trial evaluating ruxolitinib as a potential treatment for patients with essential thrombocythemia.

Oncology beyond MPNs – key highlights

Data from the randomized Phase 3 REACH2 trial of ruxolitinib versus best available therapy (BAT) in patients with steroid-refractory acute graft-versus-host disease (GVHD) have been accepted for presentation within the Presidential Symposium of the 46th annual meeting of the European Society for Blood and Marrow Transplantation (EBMT, March 22-25). As previously announced, the trial, which was conducted in collaboration with Novartis, met its primary endpoint of improving overall response rate (ORR) at Day 28 with ruxolitinib treatment compared to BAT.

REACH3, the Phase 3 trial of ruxolitinib in patients with steroid-refractory chronic GVHD being run in collaboration with Novartis, is ongoing and results are expected in the second half of 2020. If successful, and if approved, steroid-refractory chronic GVHD would be the fourth Jakafi indication available to patients in the US.

In January, it was announced that GRAVITAS-301, the Phase 3 trial of itacitinib as a treatment for patients with newly diagnosed acute GVHD, did not meet the primary endpoint.

Clinical development of itacitinib also includes GRAVITAS-309, a Phase 3 trial of itacitinib as a treatment for patients with newly diagnosed chronic GVHD.

In November, the FDA accepted the New Drug Application (NDA) for pemigatinib in second-line cholangiocarcinoma for Priority Review. The Prescription Drug User Fee Act (PDUFA) target action date is May 30, 2020. In early January, Incyte announced that the Marketing Authorization Application (MAA) seeking approval in Europe for pemigatinib as a second-line treatment for cholangiocarcinoma patients with FGFR2 fusions or rearrangements was validated by the EMA.

The CITADEL program is evaluating parsaclisib as a monotherapy across three types of non-Hodgkin lymphoma: follicular lymphoma; marginal zone lymphoma; and mantle cell lymphoma.

Clinical development of INCMGA0012 includes ongoing evaluation as a monotherapy in niche cancer opportunities as well as a planned program for the first-line treatment of patients with non-small cell lung cancer (NSCLC).

Incyte recently announced a global collaboration with MorphoSys for the development and commercialization of tafasitamab, an anti-CD19 monoclonal antibody. Pending clearance by antitrust authorities, the collaboration agreement is expected to become effective in the first half of 2020.

1)Clinical development of ruxolitinib in GVHD conducted in collaboration with Novartis
2)INCMGA0012 licensed from MacroGenics

Inflammation and Autoimmunity (IAI) – key highlights

TRuE-AD2, the first of two Phase 3 trials in the TRuE-AD development program of ruxolitinib cream in patients with mild-to-moderate atopic dermatitis, met its primary endpoint. The overall efficacy and safety profile observed in TRuE-AD2 was consistent with previous data, and no new safety signals were observed.

The results of TRuE-AD1, the second of two Phase 3 trials required for regulatory submission, are anticipated to be available in the first quarter of 2020. The NDA submission, seeking approval of ruxolitinib cream in atopic dermatitis, is expected in the fourth quarter of 2020 following long-term safety and efficacy data from both pivotal trials.

The two Phase 3 trials in the TRuE-V pivotal program evaluating ruxolitinib cream in patients with vitiligo are recruiting well and results are expected in 2021.

Phase 2 trials of INCB54707 in hidradenitis suppurativa and parsaclisib in autoimmune hemolytic anemia are progressing as planned.

The clinical program of INCB00928, Incyte’s ALK2 inhibitor, is in preparation in patients with fibrodysplasia ossificans progressiva, a disorder in which muscle tissue and connective tissue such as tendons and ligaments are gradually replaced by bone.

The Phase 2 trial of low-dose itacitinib in patients with ulcerative colitis has been discontinued, and initial data from the clinical evaluation of parsaclisib in patients with Sjögren’s syndrome did not warrant continuation of the trial.

Discovery and early development – key highlights

Incyte’s portfolio of earlier-stage clinical candidates is summarized below.

1)INCB01158 development in collaboration with Calithera
2)Discovery collaboration with Agenus
3)MCLA-145 development in collaboration with Merus

Partnered – key highlights

In January, Incyte and Lilly announced initial results of BREEZE-AD4 and BREEZE-AD5, the final two trials in the pivotal program evaluating baricitinib in patients with moderate-to-severe atopic dermatitis. Lilly recently submitted baricitinib for regulatory review in Europe as a treatment for patients with moderate-to-severe atopic dermatitis, and has announced plans to submit for approval in the U.S. and Japan in 2020.

In February, Incyte and Novartis announced that the NDA for capmatinib was accepted for Priority Review by the FDA, seeking approval in patients with NSCLC and MET exon 14 skipping mutations.

(MET)2NSCLC (with MET exon 14 skipping mutations): NDA submitted (by Novartis)

1)Worldwide rights to baricitinib licensed to Lilly: approved as Olumiant in multiple territories globally for certain patients with moderate-to-severe rheumatoid arthritis
2)Worldwide rights to capmatinib licensed to Novartis

2019 Fourth Quarter and Year-End Financial Results

The financial measures presented in this press release for the quarter and year ended December 31, 2019 and 2018 have been prepared by the Company in accordance with U.S. Generally Accepted Accounting Principles ("GAAP"), unless otherwise identified as a Non-GAAP financial measure. Management believes that Non-GAAP information is useful for investors, when considered in conjunction with Incyte’s GAAP disclosures. 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 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.

Beginning in the first quarter of 2019, after reviewing our Reconciliation of GAAP Net Income to Selected Non-GAAP Adjusted Information with the U.S. Securities & Exchange Commission, we no longer adjust for upfront consideration and milestones that are part of collaboration agreements with new or existing partners. This revised methodology is reflected in this press release for the quarter and year ended December 31, 2019 and 2018.

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.

Research and development expenses GAAP and Non-GAAP research and development expense for the quarter ended December 31, 2019 increased 3% and 2%, respectively, compared to the same periods in 2018. This increase was primarily due to our existing pipeline programs progressing to later stages of development and was partially offset by our election to end additional co-funding of development of baricitinib with Lilly effective as of January 1, 2019. GAAP and Non-GAAP research and development expense for the year ended December 31, 2019 decreased 4% and 5%, respectively, compared to the same periods in 2018, primarily due to our election to end additional co-funding of the development of baricitinib and a decrease in upfront and milestone expenses related to collaborative agreements.

Selling, general and administrative expenses GAAP and Non-GAAP selling, general and administrative expenses for the quarter ended December 31, 2019 increased 26% and 27%, respectively, and for the year ended December 31, 2019 increased 8% compared to the same period in 2018, primarily due to the growth in commercialization efforts related to Jakafi.

Other Financial Information

Operating income GAAP and Non-GAAP operating income increased for the quarter and year ended December 31, 2019 compared to the same periods in 2018 due to the growth in total revenues exceeding the growth in operating expenses.

Cash, cash equivalents and marketable securities position As of December 31, 2019 and 2018, cash, cash equivalents and marketable securities totaled $2.1 billion and $1.4 billion, respectively.

2020 Financial Guidance

The Company’s 2020 financial guidance as detailed below excludes the financial impact of the recently announced collaboration with MorphoSys, which is pending clearance by antitrust authorities. In addition, the 2020 financial guidance does not include the impact of any potential future strategic transactions.

Conference Call and Webcast Information

Incyte will hold a conference call and webcast this morning at 8:00 a.m. EST. 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, 13698234.

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, 13698234.

The conference call will also be webcast live and can be accessed at investor.incyte.com.