On February 13, 2020 Alkermes plc (Nasdaq: ALKS) reported financial results for the quarter and year ended Dec. 31, 2019 and provided financial expectations for 2020 (Press release, Alkermes, FEB 13, 2020, View Source [SID1234554281]).

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"2019 was an important year for Alkermes as we took active steps to shape the future of our business and continued to make a real-world impact in the treatment of serious diseases. We made significant progress on three fronts: driving growth in our proprietary product portfolio, advancing and expanding our diversified neuroscience and oncology pipeline, and positioning the business for long-term growth and future profitability," said Richard Pops, Chief Executive Officer of Alkermes. "Looking ahead, our priorities for 2020 are clear as we focus on commercial execution for VIVITROL and ARISTADA, prepare for potential approval and launch of ALKS 3831, advance the development of ALKS 4230, and continue to develop our pipeline of preclinical assets. We remain steadfast in our commitment to be a positive force for change through our science, our medicines, and our advocacy, as we advance patient-centered care."

Quarter Ended Dec. 31, 2019 Financial Highlights

Total revenues for the quarter were $412.7 million. This compared to $315.8 million for the same period in the prior year.

Net loss according to generally accepted accounting principles in the U.S. (GAAP) was $5.4 million for the quarter, or a basic and diluted GAAP loss per share of $0.03. This compared to GAAP net loss of $9.7 million, or a basic and diluted GAAP loss per share of $0.06, for the same period in the prior year.

Non-GAAP net income was $131.4 million for the quarter, or a non-GAAP basic and diluted earnings per share of $0.83. This compared to non-GAAP net income of $54.8 million, or a non-GAAP basic earnings per share of $0.35 and non-GAAP diluted earnings per share of $0.34, for the same period in the prior year.

In October 2019, Alkermes implemented a strategic restructuring plan, which included the elimination of approximately 160 current positions across the organization, a decrease in the company’s expected near-term hiring plans and the implementation of cost-saving measures related to external spend. These efforts are expected to result in cost savings of approximately $150 million in 2020.

In November 2019, Alkermes completed the acquisition of Rodin Therapeutics, Inc. (Rodin), a privately held biopharmaceutical company focused on developing novel, small molecule therapeutics for synaptopathies. At the closing of the transaction, Alkermes made a cash payment of $98.1 million to Rodin’s former security holders. This upfront cash payment was funded by Alkermes’ available cash and was accounted for as an asset acquisition, with $86.6 million of this upfront payment recorded as research and development (R&D) expense in the quarter.

Quarter Ended Dec. 31, 2019 Financial Results

Revenues

Net sales of proprietary products were $149.6 million, compared to $132.7 million for the same period in the prior year.

Net sales of VIVITROL were $92.8 million, compared to $83.8 million for the same period in the prior year, representing an increase of approximately 11%.

Net sales of ARISTADA1 were $56.8 million, compared to $48.8 million for the same period in the prior year, representing an increase of approximately 16%.

Manufacturing and royalty revenues were $107.3 million, compared to $167.4 million for the same period in the prior year.

Manufacturing and royalty revenues from RISPERDAL CONSTA, INVEGA SUSTENNA/XEPLION and INVEGA TRINZA/TREVICTA were $79.1 million, compared to $81.4 million for the same period in the prior year.

Manufacturing and royalty revenues from AMPYRA/FAMPYRA2 were $7.5 million, compared to $38.8 million for the same period in the prior year, due to generic competition to AMPYRA entering the U.S. market in 2018.

Manufacturing and royalty revenues in the fourth quarter of 2018 included $26.7 million related to Alkermes’ share of proceeds from the sale of certain royalty streams by Zealand Pharma A/S related to products using Alkermes technology.

Total revenues also included a $150.0 million milestone payment from Biogen related to the U.S. Food and Drug Administration (FDA) approval of VUMERITY, of which $144.8 million was recorded as license revenue and $5.2 million was recorded as R&D revenue.

R&D revenues were $11.1 million, primarily related to R&D reimbursement from the company’s collaboration with Biogen for VUMERITY and a portion of the milestone payment noted above.

Costs and Expenses

Total operating expenses were $422.7 million, compared to $315.7 million for the same period in the prior year.

R&D expenses were $198.2 million, which included $86.6 million related to the acquisition of Rodin during the fourth quarter. Excluding this R&D charge related to Rodin, R&D expenses were $111.6 million compared to $109.0 million for the same period in the prior year.

Selling, General and Administrative (SG&A) expenses were $154.5 million, compared to $141.2 million for the same period in the prior year, primarily reflecting increased investment in the commercialization of ARISTADA and VIVITROL.

As a result of the restructuring implemented in October 2019, the company recorded a restructuring expense charge of $13.4 million in the fourth quarter of 2019, consisting of one-time termination benefits for employee severance, benefits and related costs.

"Our 2019 results reflect volume growth of VIVITROL and ARISTADA, continued strength of our royalty and manufacturing portfolio and investment in the commercialization of our products and our research and development pipeline," commented James Frates, Chief Financial Officer of Alkermes. "We enter 2020 well positioned to drive growth of our proprietary product portfolio and advance our pipeline of novel oncology and neuroscience candidates. Our financial expectations for 2020 reflect anticipated net

sales growth of our proprietary products and operating expenses in line with the predicted impact of the strategic restructuring that we implemented in the fourth quarter of 2019, reflecting our commitment to non-GAAP profitability while investing in the long-term growth of the company."

Calendar Year 2019 Financial Highlights

Total revenues increased 7% to $1.17 billion in 2019, which included VIVITROL net sales of $335.4 million, ARISTADA net sales of $189.1 million, and the $150.0 million milestone payment from Biogen related to the approval of VUMERITY. This compared to total revenues of $1.09 billion in 2018, which included VIVITROL net sales of $302.6 million, ARISTADA net sales of $147.7 million and license revenues of $48.4 million from Biogen. Please see the tables at the end of this press release for a detailed breakdown of the revenues from our key commercial products.

GAAP net loss was $196.6 million, or a basic and diluted GAAP loss per share of $1.25, for 2019. This compared to a GAAP net loss of $139.3 million, or a basic and diluted GAAP loss per share of $0.90, for 2018.

Non-GAAP net income was $112.2 million, or a non-GAAP basic and diluted earnings per share of $0.71, for 2019, and excludes the impact of the acquisition of Rodin and the restructuring. This compared to non-GAAP net income of $97.8 million, or a non-GAAP basic earnings per share of $0.63 and non-GAAP diluted earnings per share of $0.61, for 2018.

At Dec. 31, 2019, Alkermes recorded cash, cash equivalents and total investments of $614.4 million, compared to $620.0 million at Dec. 31, 2018. At Dec. 31, 2019, the company’s total debt outstanding was $277.1 million, compared to $279.3 million at Dec. 31, 2018.

Recent Events:

ALKS 3831

In January 2020, the FDA accepted for review the company’s New Drug Application (NDA) seeking approval of ALKS 3831 (olanzapine/samidorphan) for the treatment of schizophrenia and for the treatment of bipolar I disorder, and assigned the NDA a Prescription Drug User Fee Act (PDUFA) target action date of Nov. 15, 2020.

VUMERITY

In October 2019, the FDA approved VUMERITY, a novel oral fumarate with a distinct chemical structure, for the treatment of relapsing forms of multiple sclerosis in adults, including clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease. Biogen holds the exclusive worldwide license to commercialize VUMERITY. In November 2019, Alkermes received a $150 million milestone payment from Biogen related to the approval of VUMERITY.

ALKS 4230

In November 2019, Alkermes presented preliminary clinical data from the ARTISTRY-1 phase 1/2 study investigating intravenous administration of ALKS 4230 as monotherapy and in combination with pembrolizumab in adults with advanced solid tumors, and study design details and preliminary safety data from the ARTISTRY-2 phase 1/2 study evaluating subcutaneous administration of ALKS 4230 as monotherapy and in combination with pembrolizumab at the 2019 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting.

HDAC-Inhibitor Platform

In November 2019, Alkermes announced the acquisition of Rodin, a privately held biopharmaceutical company focused on developing novel, small molecule therapeutics

for synaptopathies, which expanded Alkermes’ neuroscience development efforts into a wide range of neurodegenerative disorders.

Financial Expectations for 2020

The following outlines the company’s financial expectations for 2020, which reflect the expected impact of the strategic restructuring implemented in 2019. All line items are according to GAAP, except as otherwise noted.

Revenues: The company expects total revenues to range from $1.03 billion to $1.08 billion. Excluding license and R&D revenues from Biogen of approximately $195 million related to the development and approval of VUMERITY recorded in 2019, this represents revenue growth of approximately 8%. Included in this total revenue expectation, Alkermes expects VIVITROL net sales to range from $340 million to $355 million, and ARISTADA net sales to range from $220 million to $235 million.

Cost of Goods Manufactured and Sold: The company expects cost of goods manufactured and sold to range from $185 million to $195 million.

Research and Development (R&D) Expenses: The company expects R&D expenses to range from $405 million to $430 million.

Selling, General and Administrative (SG&A) Expenses: The company expects SG&A expenses to range from $535 million to $560 million.

Amortization of Intangible Assets: The company expects amortization of intangibles to be approximately $40 million.

Net Interest Expense: The company expects interest expense and interest income to offset one another.

Income Tax Expense: The company expects income tax expense of up to $10 million.

GAAP Net Loss: The company expects GAAP net loss to range from $130 million to $160 million, or a basic and diluted loss per share of $0.82 to $1.01, based on a weighted average share count of approximately 159 million shares outstanding.

Non-GAAP Net Income: The company expects non-GAAP net income to range from $40 million to $70 million, or a non-GAAP basic earnings per share of $0.25 to $0.44, based on a weighted average basic share count of approximately 159 million shares outstanding and a non-GAAP diluted earnings per share of $0.25 to $0.43, based on a weighted average diluted share count of approximately 161 million shares outstanding.

Share-Based Compensation: The company expects share-based compensation of approximately $110 million.

Capital Expenditures: The company expects capital expenditures to range from $45 million to $55 million.

Conference Call

Alkermes will host a conference call and webcast presentation with accompanying slides at 8:00 a.m. ET (1:00 p.m. GMT) on Thursday, Feb. 13, 2020, to discuss these financial results and provide an update on the company. The webcast may be accessed on the Investors section of Alkermes’ website at www.alkermes.com. The conference call may be accessed by dialing +1 877 407 2988 for U.S. callers and +1 201 389 0923 for international callers. In addition, a replay of the conference call will be available from 11:00 a.m. ET (4:00 p.m. GMT) on Thursday, Feb. 13, 2020, through Thursday, Feb. 20, 2020, and may be accessed by visiting Alkermes’ website or by dialing +1 877 660 6853 for U.S. callers and +1 201 612 7415 for international callers. The replay conference ID is 13698323.

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.

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"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.

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"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.

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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]).

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"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.