On February 26, 2019 Epizyme, Inc. (Nasdaq: EPZM), a late-stage company developing novel epigenetic therapies, reported fourth quarter and full year 2018 financial results (Press release, Epizyme, FEB 26, 2019, View Source [SID1234533693]).

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!

"We made tremendous progress last year, leading into what is poised to be one of our most meaningful and value-creating years as a company in 2019, with two successive NDA submissions planned for tazemetostat and a robust clinical expansion strategy," said Robert Bazemore, president and chief executive officer of Epizyme. "Following our meeting with FDA late last year, we are confident in the submission path for accelerated approval for all patients with follicular lymphoma, regardless of EZH2 mutation status, who have been previously treated with two or more therapies. We believe tazemetostat, based on its ongoing safety and efficacy data, would be well-suited to address the unmet need and treatment goals for patients with this indolent disease. Our first NDA submission for tazemetostat for epithelioid sarcoma remains on track to be submitted in the second quarter, and if successful, would make tazemetostat the first commercially available EZH2 inhibitor and the first treatment specifically indicated for epithelioid sarcoma patients. We look forward to submitting both regulatory applications so that we may bring tazemetostat to patients who need it."

2019 Tazemetostat Program Outlook

Tazemetostat NDA Submission for Epithelioid Sarcoma (ES) on Track for Second Quarter: Epizyme is well underway with its preparations to submit its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for accelerated approval of tazemetostat for patients with ES in the second quarter of 2019 based on the ongoing Phase 2 study. ES is an ultra-rare and difficult-to-treat sarcoma with no specifically indicated FDA-approved therapies today. If approved, tazemetostat could be the first treatment specifically indicated for patients with ES and could enhance regulatory efficiencies of future tazemetostat submissions for additional indications. The company anticipates reporting updated data from the Phase 2 study at a medical meeting in mid-2019.
NDA Submission Planned for Fourth Quarter for All-Comer Follicular Lymphoma (FL) Population Based on Fully Enrolled Study: In late 2018, Epizyme met with the FDA to review its planned registration strategy for tazemetostat for patients with FL who have been previously treated with two or more systemic therapies. Based on this interaction, the company has identified a path to a submission for accelerated approval for FL patients with EZH2 activating mutations and those with wildtype EZH2, based on the ongoing, fully enrolled Phase 2 study. The company anticipates reporting updated data from the study at a medical meeting in mid-2019 and submitting an NDA for accelerated approval for this patient population in the fourth quarter of 2019.
Plans Established to Expand Tazemetostat into Earlier FL Treatment Settings: Epizyme is planning to initiate a combination study in mid-2019 of tazemetostat with the chemo-free treatment regimen "R2" (Revlimid plus Rituxan) for the treatment of patients with relapsed/refractory FL who have received at least one prior therapy. The company is also finalizing plans for a trial of tazemetostat in combination with Rituxan for the treatment of patients with relapsed/refractory FL. Further, Epizyme is exploring the opportunity to expand the combination assessment of tazemetostat with R-CHOP into front-line, high-risk patients with FL.
Studies in Prostate Cancer and Platinum-Resistant Solid Tumors to Begin in 2019: Based on strong scientific rationale, as well as research and biomarker data, Epizyme anticipates initiating a combination study in patients with castration-resistant prostate cancer in mid-2019, followed by a combination study with a PARP inhibitor in patients with platinum-resistant solid tumors, such as small-cell lung cancer, triple-negative breast cancer and ovarian cancer, in the second half of 2019.
Pipeline Progress

EZM8266 on Track to Begin Clinical Development: Following the completion of IND-enabling studies, the company is on track to begin clinical development of its first-in-class G9a inhibitor, EZM8266, for the treatment of sickle cell disease in the second half of 2019 with a dose-finding and safety study.
Two Research Programs to Be Advanced under Boehringer Ingelheim Collaboration: In November 2018, Epizyme entered a strategic collaboration with Boehringer Ingelheim focused on the research, development and commercialization of novel small molecule inhibitors directed toward two previously unaddressed epigenetic targets as potential therapies for people with cancer. Specifically, these targets are enzymes within the helicase and histone acetyltransferase (HAT) families. The company received an upfront payment of $15 million and will receive an additional $5 million in research funding in 2019. Epizyme is eligible to receive a total of up to $280 million in additional payments for research, development, regulatory and commercial milestones.
Milestone Payment Earned from GSK: In December 2018, Epizyme earned an $8 million milestone payment from its partner GlaxoSmithKline (GSK) following initiation of patient dosing in a Phase 1 clinical trial of GSK3368715, a first-in-class protein arginine methyltransferase1 (PRMT1) inhibitor discovered by Epizyme and the second program to enter the clinic under the collaboration. The company has earned an aggregate of $89 million in up-front, research and milestone payments to date, and may earn up to an additional $375 million from GSK if all remaining milestones are met.
Financial Guidance

Based on current operating plans, Epizyme expects its current cash runway to extend into the second quarter of 2020.
Fourth Quarter and Full Year 2018 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $240.3 million as of December 31, 2018, as compared to $276.4 million as of December 31, 2017. The decrease is primarily due to operating expenditures for the year off set by milestones received through collaborations and the company’s public offering of common stock that closed in October 2018.
Revenue: Collaboration revenue for the fourth quarter of 2018 was $9.7 million and $21.7 million for the full year ended December 31, 2018, compared to no revenue for the fourth quarter of 2017 and $10.0 million for the full year ended December 31, 2017. The increase in annual collaboration revenue is due to the company’s collaboration with Boehringer Ingelheim, which was initiated in November 2018, and a milestone payment earned from GSK for the initiation of clinical development of a PRMT1 inhibitor discovered at Epizyme.
R&D Expenses: Research and development (R&D) expenses were $21.8 million for the fourth quarter of 2018 and $105.8 million for the full year ended December 31, 2018, compared to $28.9 million for the fourth quarter of 2017 and $109.7 million for the full year ended December 31, 2017. The reductions in expenses are primarily due to decreases in our discovery research expenses and decreases in clinical trial expenses, offset by greater tazemetostat manufacturing costs.
G&A Expenses: General and administrative (G&A) expenses were $12.2 million for the fourth quarter of 2018 and $44.0 million for the full year ended December 31, 2018, compared to $8.4 million for the fourth quarter of 2017 and $37.2 million for the full year ended December 31, 2017. The increase is primarily due to an rise in medical affairs and commercial costs as a result of organizational development in preparation for tazemetostat commercialization.
Net Loss: Net loss was $22.9 million, or $0.29 per share, for the fourth quarter of 2018 and $123.6 million, or $1.72 per share, for the full year ended December 31, 2018, compared to was $36.2 million, or $0.52 per share, for the fourth quarter of 2017 and $134.3 million, or $2.18 per share, for the full year ended December 31, 2017.
Conference Call Information
Epizyme will host a conference call today, Feb. 26, at 8:30 a.m. ET. To participate in the conference call, please dial (877) 844-6886 (domestic) or (970) 315-0315 (international) and refer to conference ID 1088195. A live webcast will be available in the investor section of the company’s website at www.epizyme.com. The webcast and slides will be archived for 60 days following the call and presentation.

On February 26, 2019 MannKind Corporation (NASDAQ:MNKD) reported financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Mannkind, FEB 26, 2019, View Source [SID1234533692]).

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!

Fourth Quarter Results

For the fourth quarter of 2018, total revenues were $16.0 million, reflecting Afrezza net revenue of $5.7 million and collaboration and services revenue of $10.3 million. Afrezza net revenue increased 28% on a GAAP basis compared to $4.5 million for the fourth quarter of 2017. In the fourth quarter of 2017, we recognized a $1.4 million change in estimate to Afrezza net revenue; when this adjustment is excluded, Afrezza net revenue increased 86% (non-GAAP) compared to the fourth quarter of 2017, primarily driven by higher product demand and a more favorable mix of cartridges. Collaborations and services revenue increased $10.2 million primarily attributable to the United Therapeutics licensing and research agreements.

Afrezza cost of goods sold (COGS) was $5.0 million for the fourth quarter of both 2018 and 2017. Afrezza COGS in the fourth quarter of 2018 reflected a one-time charge of $2.0 million related to an amendment fee associated with our insulin supply agreement, offset by lower inventory write-offs in 2018 of $0.8 million and $0.7 million lower spending associated with manufacturing absorption. Afrezza gross profit was $0.7 million for the fourth quarter, the first quarterly gross profit recognized from Afrezza sales. When the one-time charge of $2.0 million related to the amendment fee is excluded, Afrezza gross profit was $2.7 million (non-GAAP) for the fourth quarter.

Research and development (R&D) expenses for the fourth quarter of 2018 were $1.1 million compared to $3.5 million for the fourth quarter of 2017. The decrease of $2.4 million was primarily due to $0.8 million associated with the United Therapeutics research agreement, which was classified as a cost of collaborations and services revenue, and a decrease in spending of $0.8 million related to clinical trials.

Selling, general and administrative (SG&A) expenses were $18.0 million for the fourth quarter of 2018 compared to $23.3 million for the fourth quarter of 2017. The decrease of $5.3 million was primarily due to $5.0 million in selling expenses associated with our first direct-to-consumer television advertising campaign in the fourth quarter of 2017.

Interest expense on notes (facility financing obligation and senior convertible notes) was $0.6 million for the fourth quarter of 2018 compared to $2.1 million for the fourth quarter of 2017. The $1.5 million decrease was primarily due to a reduction in the debt principal balances.

The net loss for the fourth quarter of 2018 was $9.7 million, or $0.06 per share, compared to the $32.8 million net loss in the fourth quarter of 2017, or $0.28 per share. The lower net loss is mainly attributable to an increase in total revenues of $11.5 million and a decrease in total expenses of $9.6 million.

Full Year 2018 Results

For the full year ended December 31, 2018, total revenues were $27.9 million, reflecting Afrezza net revenue of $17.3 million and collaborations and service revenue of $10.6 million. Afrezza net revenue increased 88% compared to $9.2 million for the same period in 2017, primarily reflecting increased product demand and a more favorable mix of cartridges. Collaborations and services revenue increased $10.3 million primarily attributable to the United Therapeutics licensing and research agreements.

Afrezza COGS for the year ended December 31, 2018 was $19.4 million compared to $17.2 million for the year ended December 31, 2017. The increase of $2.2 million was primarily attributable to an increase in costs associated with increased Afrezza sales and a one-time charge of $2.0 million related to an amendment fee associated with our insulin supply agreement, offset by a decrease of $0.8 million in inventory write-offs.

R&D expenses for the year ended December 31, 2018 were $8.7 million compared to $14.1 million for the same period in 2017. This $5.4 million decrease was primarily attributable to lower clinical trials expenses of $2.2 million, a $1.7 million decrease in salary-related expenses and a $0.8 million decrease in research and development supply and services costs.

SG&A expenses were $79.7 million for the year ended December 31, 2018 compared to $75.0 million for the same period in 2017. The $4.7 million increase was primarily due to an increase of $2.9 million in headcount-related expenses associated with commercial operations, an increase in spending of $1.7 million in our human resources, accounting, corporate communications, and office support departments, a $1.4 million increase in medical affairs support, a $1.3 million increase in stock-based compensation expense, and a one-time $1.1 million expense to transition corporate support functions from Connecticut to our headquarters in California, which were partially offset by a decrease in selling expenses of $4.7 million associated with our 2017 direct-to-consumer television advertising campaign.

Interest expense on notes (facility financing obligation and senior convertible notes) was $5.1 million for the year ended December 31, 2018 compared to $9.5 million for the same period in 2017. The $4.4 million decrease was primarily due to a reduction in the debt principal balances.

The net loss for the year ended December 31, 2018 was $87.0 million, or $0.60 per share, compared to $117.3 million for the year ended December 31, 2017, or $1.13 per share. The lower net loss is mainly attributable to an increase in Afrezza net revenue of $8.1 million, an increase in collaboration revenue of $10.3 million and a decrease in total expenses of $15.5 million.

"Our fourth quarter and full year 2018 results showed excellent progress in executing against our Afrezza growth plan and recognized for the first time revenues associated with our license and collaboration agreement with United Therapeutics," said Michael Castagna, Chief Executive Officer of MannKind Corporation. "The fourth quarter of last year was the first time we reported gross profit for Afrezza and we ended the year with a strong cash position thanks to the United Therapeutics deal and a public offering of common stock and warrants in December."

Cash and Cash Equivalents

Cash, cash equivalents and restricted cash at December 31, 2018 was $71.7 million compared to $48.4 million at December 31, 2017. The increase was primarily due to the net proceeds of $26.4 million from a second quarter registered direct offering of common stock and warrants and $37.5 million from a fourth quarter public offering of common stock and warrants, partially offset by the net cash used in operating activities of $37.7 million (inclusive of two payments from United Therapeutics totaling $55.0 million).

Non-GAAP Measures

Certain financial information contained in this press release is presented on both a reported basis (GAAP) and a non-GAAP basis. Reported results were prepared in accordance with GAAP whereas non-GAAP measures exclude items described in the reconciliation tables below. Non-GAAP financial information is intended to portray the results of our baseline performance, supplement or enhance management, analysts and investors overall understanding of our underlying financial performance and facilitate comparisons among current and past periods. The non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP.

Conference Call

MannKind will host a conference call and presentation webcast to discuss these results today at 9:00 a.m. Eastern Time. To participate in the live call by telephone, please dial (888) 394-8218 or (323) 701-0225 and use the participant passcode: 7809405. Those interested in listening to the conference call live via the Internet may do so by visiting the Company’s website at View Source under News & Events.

A telephone replay of the call will be accessible for approximately 14 days following completion of the call by dialing (844) 512-2921 or (412) 317-6671 and use the participant passcode: 7809405#. A replay will also be available on MannKind’s website for 14 days.

On February 26, 2019 OPKO Health, Inc. (NASDAQ: OPK) reported its operating and financial results for the three and twelve months ended December 31, 2018 as well as provide guidance on expected revenues and operating expenses for the first quarter 2019 after the close of the U.S. financial markets on Wednesday, February 27, 2019 (Press release, Opko Health, FEB 26, 2019, View Source [SID1234533691]).

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!

OPKO’s senior management will provide a business update and discuss its financial results in a live conference call and audio webcast beginning at 4:30 p.m. Eastern time on Wednesday, February 27, 2019.

CONFERENCE CALL & WEBCAST INFORMATION

OPKO’s senior management will provide a business update and discuss results in greater detail in a conference call and live audio webcast at 4:30 p.m. Eastern time on Wednesday, February 27, 2019. The conference call dial-in and webcast information is as follows:

DOMESTIC DIAL-IN: 866-634-2258
INTERNATIONAL DIAL-IN: 330-863-3454
PASSCODE: 4254518
WEBCAST: View Source
For those unable to participate in the live conference call or webcast, a replay will be available beginning approximately two hours after the close of the conference call. To access the replay, dial 855-859-2056 or 404-537-3406. The replay passcode is 4254518. The replay can be accessed for a period of time on OPKO’s website at View Source.

On February 26, 2019 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune and viral diseases, reported financial results for the fourth quarter and full year ended December 31, 2018, and recent operational highlights (Press release, Atara Biotherapeutics, FEB 26, 2019, View Source [SID1234533690]).

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!

"2018 was a year of pipeline expansion and strong operational execution for Atara as we advanced our T-cell immunotherapy programs across all three of our major value drivers: tab-cel, multiple sclerosis and next-generation CAR T," said Isaac Ciechanover M.D., Chief Executive Officer and President of Atara Biotherapeutics. "Notably, we successfully executed on our strategy to build a leading next-generation and off-the-shelf, allogeneic CAR T portfolio. Our collaborations with academic leaders leverage technologies at the forefront of CAR T innovation for hematologic malignancies and solid tumors. We also opened a state-of-the-art T-cell operations and manufacturing facility and expanded our R&D, operational and commercial leadership. I am extremely gratified with where the Company is today and wish to acknowledge the many extraordinary contributions by Atara employees that enabled us to reach this point. I anticipate 2019 to be another pivotal year with multiple clinical and regulatory milestones, moving Atara closer to realizing our mission of transforming the lives of patients with serious medical conditions."

Atara continues to progress tab-cel (tabelecleucel) Phase 3 studies for patients with Epstein-Barr virus associated post-transplant lymphoproliferative disease (EBV+ PTLD) and anticipates initial tab-cel Phase 3 results to be available to the company in the first half of 2019.

Discussions with the European Medicines Agency (EMA) and U.S. Food & Drug Administration (FDA) regarding the development of tab-cel are ongoing and Atara’s intention is to align on a global regulatory strategy for patients with EBV+ PTLD. Outcomes of these discussions are expected in the first half of 2019.

Atara plans to submit a tab-cel EU conditional marketing authorization (CMA) application in the second half of 2019. To ensure the integrity of the ongoing, open-label tab-cel Phase 3 studies, the Company anticipates disclosing initial top-line EBV+ PTLD results in the second half of 2019 following submission of the EMA CMA application.

Atara expects initial safety results from the ongoing off-the-shelf, allogeneic ATA188 Phase 1 study in patients with progressive multiple sclerosis (MS) in the first half of 2019. Additional safety and efficacy results from this study are expected in the second half of 2019.

The Company is also rapidly advancing its next-generation chimeric antigen receptor T-cell (CAR T) pipeline across multiple therapeutic areas and expects results to be presented at upcoming scientific conferences.

Recent Highlights and Anticipated Upcoming Milestones

Tab-cel (tabelecleucel)

Two Phase 3 clinical studies are ongoing (MATCH and ALLELE) to evaluate tab-cel for patients with EBV+ PTLD who have failed rituximab following hematopoietic cell transplant (HCT) or solid organ transplant (SOT).
Expanded MATCH and ALLELE study sites, with 30 sites available for enrollment in the United States and Australia, and with additional sites expected to open in the United States and other geographies.
ATA188 & ATA190 for Multiple Sclerosis (MS)

A Phase 1 clinical study of off-the-shelf, allogeneic ATA188 in patients with progressive MS is ongoing across clinical sites in the United States and Australia.
Atara also plans to initiate a randomized autologous ATA190 study in progressive MS patients in the second half of 2019.
Next-Generation CAR T Development Pipeline

Licensed worldwide rights to a mesothelin-targeted chimeric antigen receptor T-cell (CAR T) immunotherapy for solid tumors from Memorial Sloan Kettering Cancer Center (MSK).
Development with MSK will focus on a next-generation, mesothelin-targeted CAR T using novel 1XX CAR signaling domain and PD-1 dominant negative receptor (DNR) checkpoint inhibition technologies for patients with mesothelin-associated solid tumors.
Expect clinical and preclinical results supporting Atara’s next-generation CAR T programs to be presented at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 to be held March 29 to April 3 in Atlanta, Georgia.
First IND submission for Atara’s next-generation CAR T program expected in the fourth quarter of 2019 or first quarter of 2020.
Other Pipeline

Conducting IND-enabling manufacturing process development for ATA621, targeting both JC and BK viruses for patients with progressive multifocal leukoencephalopathy (PML).
Corporate

Atara’s Board of Directors is currently conducting a search for a new Chief Executive Officer following Dr. Ciechanover’s transition plan announced in January. Dr. Ciechanover will remain in his role as President and CEO until the earlier of the appointment of his successor or June 30, 2019.
Fourth Quarter and Full Year 2018 Financial Results

Cash, cash equivalents and short-term investments as of December 31, 2018 totaled $309.6 million, which we believe will be sufficient to fund planned operations to mid-2020. The balance excludes the impact of one-time license fees of $12.5 million paid in the first quarter of 2019 for worldwide rights to the next-generation allogeneic CAR T program targeting mesothelin.
The Company reported net losses of $80.0 million, or $1.75 per share, and $230.7 million, or $5.27 per share, for the fourth quarter and fiscal year 2018, respectively, as compared to $35.3 million, or $1.15 per share, and $119.5 million, or $4.00 per share, for the same periods in 2017.
Total operating expenses include total non-cash expenses of $11.0 million and $37.5 million for the fourth quarter and fiscal year 2018, respectively, as compared to $6.4 million and $24.1 for the same periods in 2017.
Research and development expenses were $62.3 million and $167.5 million for the fourth quarter and fiscal year 2018, respectively, as compared to $24.8 million and $81.2 million for the same periods in 2017. The increases in the fourth quarter and fiscal year 2018 were due to costs associated with the Company’s continuing expansion of research and development activities, including:
clinical study, manufacturing and outside service costs related to the two Phase 3 clinical studies of tab-cel in patients with EBV+ PTLD and the Phase 1 clinical study of allogeneic ATA188 in patients with progressive MS;
one-time license fees of $12.5 million incurred in the fourth quarter of 2018 for exclusive rights to a next-generation allogeneic CAR T program targeting mesothelin from MSK;
higher employee-related and overhead costs from increased headcount and operations, and
an increase in facilities and information technology expenses that are allocated to our research and development function.
Research and development expenses include $5.2 million and $16.2 million of non-cash stock-based compensation expenses for the fourth quarter and fiscal year 2018, respectively, as compared to $2.5 million and $8.8 million for the same periods in 2017.
General and administrative expenses were $19.6 million and $69.7 million for the fourth quarter and fiscal year 2018, respectively, as compared to $11.0 million and $40.3 million for the same periods in 2017. The increases in the fourth quarter and fiscal year 2018 were primarily due to increases in professional services costs and employee-related costs driven by increased headcount to support the Company’s expanding operations.
General and administrative expenses include $4.3 million and $17.6 million of non-cash stock-based compensation expenses for the fourth quarter and fiscal year 2018, respectively, as compared to $3.6 million and $14.3 million for the same periods in 2017.

On February 26, 2019 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, 2018 (Press release, Blueprint Medicines, FEB 26, 2019, View Source [SID1234533689]).

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!

Print

"Following a year of remarkable clinical progress across our portfolio in 2018, we are focused on executing our ‘2020 Blueprint’ vision to transform Blueprint Medicines into a fully-integrated global precision therapy company," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "The cornerstone of this effort is our planned NDA submission for avapritinib for patients with PDGFRA Exon 18 mutant GIST and fourth-line GIST in the second quarter. As we work to bring this new therapy to GIST patients who currently have no approved treatment options, we are also partnering with treating physicians, the patient community and testing companies to evolve the GIST treatment paradigm toward precision medicine, with the shared goals of maximizing patient outcomes, enabling efficient clinical trials and delivering value to the healthcare system."

Fourth Quarter 2018 Highlights and Recent Progress:

Avapritinib: Gastrointestinal stromal tumors (GIST):

Locked the registration database and reported top-line results from the Phase 1 NAVIGATOR trial of avapritinib in patients with PDGFRA Exon 18 mutant GIST and fourth-line GIST in preparation for the planned submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the second quarter of 2019. Read the top-line data here.
Presented updated data from the Phase 1 NAVIGATOR trial across treatment lines at the Connective Tissue Oncology Society 2018 Annual Meeting in November 2018 and disclosed plans to conduct the registration-enabling Phase 3 COMPASS-2L precision medicine trial of avapritinib in second-line GIST. Read the full data here.
Under Blueprint Medicines’ collaboration with CStone Pharmaceuticals, announced the China National Medical Products Administration (NMPA) cleared an Investigational New Drug (IND) application for the ongoing Phase 3 VOYAGER trial of avapritinib in third-line GIST.
Avapritinib: Systemic mastocytosis (SM):

Presented updated data from the Phase 1 EXPLORER trial at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in December 2018. Read the full data here.
Initiated patient dosing in two registration-enabling trials: the Phase 2 PATHFINDER trial in advanced SM and the Phase 2 PIONEER trial in indolent and smoldering SM.
BLU-667: RET-altered solid tumors:

Today announced the FDA has granted Breakthrough Therapy Designation to BLU-667 for the treatment of RET-mutation-positive medullary thyroid cancer (MTC) that requires systemic treatment and for which there are no acceptable alternative treatments.
BLU-554: Hepatocellular carcinoma (HCC):

Under Blueprint Medicines’ collaboration with CStone Pharmaceuticals, announced the China NMPA cleared an IND application for the ongoing Phase 1 trial of BLU-554 as a monotherapy in advanced HCC.
BLU-782: Fibrodysplasia ossificans progressiva (FOP):

Initiated participant dosing in a Phase 1 trial of BLU-782 in healthy volunteers in the first quarter of 2019.
Today announced the FDA has granted Fast Track Designation to BLU-782 for the treatment of FOP.
Corporate:

Announced "2020 Blueprint," a two-year global business strategy under which Blueprint Medicines expects to have two marketed products, four pending marketing applications in the United States or Europe, six clinical-stage therapeutic candidates and eight research programs by the end of 2020.
Announced the promotion of Michael Landsittel to Chief Financial Officer and the promotion of Kate Haviland to Chief Operating Officer in February 2019.
Key Upcoming Milestones:

The company expects to achieve the following milestones by the end of the second quarter of 2019.

Submit an NDA for avapritinib for PDGFRA Exon 18 mutant GIST and fourth-line GIST.
Present the registration dataset for avapritinib in PDGFRA Exon 18 mutant GIST and fourth-line GIST.
Present updated data from the Phase 1 EXPLORER trial of avapritinib in advanced SM.
Present updated data from the Phase 1 ARROW trial of BLU-667 in RET-altered cancers.
Complete enrollment of previously treated NSCLC and MTC patient cohorts in the Phase 1 ARROW trial of BLU-667.
Fourth Quarter and Year End 2018 Financial Results:

Cash Position: As of December 31, 2018, cash, cash equivalents and investments were $494.0 million, as compared to $673.4 million as of December 31, 2017. This decrease was primarily related to cash used in operating activities, partially offset by the $40.0 million upfront payment received in connection with entering into the collaboration with CStone Pharmaceuticals and the $10.0 million milestone payment achieved under the Roche collaboration in June 2018.
Collaboration Revenues: Collaboration revenues were $1.0 million for the fourth quarter of 2018 and $44.5 million for the year ended December 31, 2018, as compared to $1.6 million for the fourth quarter of 2017 and $21.4 million for the year ended December 31, 2017. This increase for the year was primarily due to revenue recognized under the collaboration agreement with CStone Pharmaceuticals, partially offset by the termination of the Alexion agreement in 2017.
R&D Expenses: Research and development expenses were $70.5 million for the fourth quarter of 2018 and $243.6 million for the year ended December 31, 2018, as compared to $43.6 million for the fourth quarter of 2017 and $144.7 million for the year ended December 31, 2017. This increase was primarily due to increased clinical and manufacturing expenses driven by Blueprint Medicines’ lead development candidates and increased personnel-related expenses. Research and development expenses included $4.9 million in stock-based compensation expenses for the fourth quarter of 2018 and $17.0 million in stock-based compensation expenses for the year ended December 31, 2018.
G&A Expenses: General and administrative expenses were $13.6 million for the fourth quarter of 2018 and $47.9 million for the year ended December 31, 2018, as compared to $8.1 million for the fourth quarter of 2018 and $28.0 million for the year ended December 31, 2017. This increase was primarily due to increased personnel-related expenses and increased professional fees, including pre-commercial planning activities. General and administrative expenses included $3.9 million in stock-based compensation expenses for the fourth quarter of 2018 and $13.5 million in stock-based compensation expenses for the year ended December 31, 2018.
Net Loss: Net loss was $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, as compared to a net loss of $49.0 million for the fourth quarter of 2017 and $148.1 million for the year ended December 31, 2017, or a net loss per share of $1.23 and $3.92, respectively.
Financial Guidance:

Based on its current plans, Blueprint Medicines expects that its existing cash, cash equivalents and investments, excluding any potential option fees and milestone payments under its existing collaborations with Roche and CStone Pharmaceuticals, will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the second half of 2020.

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 2018 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.