On August 2, 2018 MannKind Corporation(NASDAQ:MNKD) reported financial results for the second quarter ended June 30, 2018 (Press release, Mannkind, AUG 2, 2018, View Source [SID1234528381]).

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"In 2Q 2018, we experienced our highest Afrezza sales ever due to increased physician trial and adoption. We have doubled our market share over the past year as a result of our commercialization efforts and are excited about our new Afrezza clinical data being released throughout 2018. Additionally, Treprostinil Technosphere (TreT) continues to progress towards Phase 3 and may address an important unmet need for those living with pulmonary arterial hypertension,"said Michael Castagna, Chief Executive Officer of MannKind Corporation.

Second Quarter Results

For the second quarter of 2018, Afrezza net revenue was $3.8 million, an increase of 142% compared to $1.5 million for the second quarter of 2017 resulting from increased volume, price, and favorable cartridge mix. On January 1, 2018, we adopted ASC 606, the new revenue recognition standard, under which we recognize revenue as we sell product to wholesale distributors; revenue for prior periods is recognized on the basis of a model that estimates the sale of Afrezza to patients.

Cost of goods sold remained level for the three months ended June 30, 2018 at $5.0 million compared to the same period in 2017. Lower inventory write-offs offset an increase in cost of goods sold resulting from higher Afrezza sales and excess capacity costs.

Research and development (R&D) expenses for the second quarter of 2018 were $2.9 million compared to $3.1 million for the second quarter of 2017, a decrease of $0.2 million or 5%, reflecting $0.7 million in lower salary related expenses for personnel who were engaged in research and development activities in 2017 who have transitioned to Afrezza medical affairs activities and a $0.3 million decrease in repairs and maintenance on laboratory equipment, offset by a $0.9 million increase in clinical trial costs.

Selling, general and administrative (SG&A) expenses were $21.7 million for the second quarter of 2018 compared to $18.6 million for the second quarter of 2017. The $3.1 million, or 17%, increase was primarily due to a $1.0 million increase in non-cash stock compensation expenses, a $0.8 million increase in consulting fees, a $0.6 million increase in costs related to transitioning certain corporate support functions from Connecticut to our corporate headquarters in California, and a $0.6 million increase in relocation spending for key personnel.

The net loss for the second quarter of 2018 was $22.7 million, or $0.16 per share, compared to the $35.3 million net loss in the second quarter of 2017 or $0.35 per share.

Six Months Ended Results

For the six months ended June 30, 2018, Afrezza net revenue was $7.2 million, an increase of 161% compared to $2.7 million for the same period in 2017 (prior to the adoption of ASC 606).

Cost of goods sold for the six months ended June 30, 2018 was $9.1 million compared to $7.6 million for the six months ended June 30, 2017, an increase of $1.5 million or 19%. This increase was due to the result of higher Afrezza sales of $0.8 million and $1.0 million increase of excess capacity costs. These increases were partially offset by lower inventory write-offs compared to the same period in 2017.

R&D expenses for the six months ended June 30, 2018 were $5.6 million compared to $6.3 million for the six months ended June 30, 2017, a decrease of $0.7 million or 11%, reflecting $0.9 million in lower salary related expenses for personnel who were engaged in research and development activities in 2017 but transitioned to Afrezza medical affairs activities, offset by a $0.3 million increase in clinical trial costs.

SG&A expenses were $42.3 million for the six months ended June 30, 2018 compared to $34.0 million for the six months ended 2017. The $8.3 million or 24% increase was primarily due to a $1.7 million increase in additional headcount-related expenses associated with sales personnel hired in 2018, a $1.1 million increase in additional headcount-related expenses in our human resources, accounting, corporate communications, and office support departments, a $1.7 million increase in non-cash stock compensation expense, a $1.3 million increase in employee benefits, a $1.2 million increase in costs related to transitioning certain corporate support functions from Connecticut to our corporate headquarters in California, and a $0.9 million increase in consulting fees.

The net loss for the six months ended June 30, 2018 was $53.1 million, or $0.41 per share, compared to a $51.7 million for the six months ended June 30, 2017, or $0.53 per share.

Cash and Cash Equivalents

Cash, cash equivalents and restricted cash at June 30, 2018 decreased to $26.7 million compared to $48.4 million at December 31, 2017, primarily due to net cash used in operating activities of $48.8 million in 2018 primarily offset by $26.4 million of net proceeds from a registered direct offering of 14 million shares of common stock and warrants at a purchase price of $2.00 per share and accompanying warrant.

Conference Call

MannKind will host a conference call and presentation webcast to discuss these results today at 5:00 p.m. Eastern Time. To view and listen to the earnings call webcast live via the Internet, visit the Company’s website at www.mannkindcorp.com and click on the "Q2 2018 MannKind Earnings Conference Call" link in the Webcasts section of News & Events. To participate in the live call by telephone, please dial (800) 239-9838 toll-free or (323) 794-2551 toll/international and use the conference passcode: 5195402.

A telephone replay of the call will be accessible for approximately 14 days following completion of the call by dialing (844) 512-2921 toll-free or (412) 317-6671 toll/international and use the replay passcode: 5195402. A replay will also be available on MannKind’s website for 14 days.

On August 2, 2018 argenx (Euronext & Nasdaq: ARGX), a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, reported its second quarter business update and half-year financial results for 2018 (Press release, argenx, AUG 2, 2018, View Source [SID1234528317]).

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These half-year results and this business update will be discussed during a conference call and webcast presentation today at 3 p.m. CEST/ 9 a.m. EDT. To participate in the conference call, please select your phone number below, and use the confirmation code 7669735. The webcast may be accessed on the homepage of the argenx website at www.argenx.com or by clicking here.

"We made significant progress recently in building a pipeline-in-a-product opportunity around our lead candidate efgartigimod (ARGX-113) as we study its potential to treat a range of severe autoimmune diseases, including validation of its mechanism of action across two indications based on the proof-of-concept data from the Phase 2 clinical trial in generalized myasthenia gravis (gMG) and the early evidence of disease control from the first cohort of patients in our Phase 2 pemphigus vulgaris (PV) clinical trial. These results strengthen our conviction that reducing pathogenic autoantibodies may offer an innovative approach to treating gMG and PV and could give rise to potential therapeutic benefits in other conditions that are similarly mediated. We look forward to a productive second half of 2018 around efgartigimod with topline data from the Phase 2 immune thrombocytopenia clinical trial expected before the end of the third quarter and the launch of a Phase 3 clinical trial in gMG before the end of the year," commented Tim Van Hauwermeiren, CEO of argenx.

"We are also advancing a deep pipeline of differentiated antibodies beyond efgartigimod. We continue to enroll the Phase 2 clinical trial of ARGX-110 in acute myeloid leukemia (AML) and expect to report full data from the Phase 1 dose-escalation clinical trial in December around the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. We achieved key milestones in our collaborations with AbbVie and Leo Pharma this quarter and have continued to showcase our ability to grow our pipeline through our productive Innovative Access Program."

SECOND QUARTER 2018 AND RECENT BUSINESS HIGHLIGHTS

Efgartigimod Program

Full Phase 2 Myasthenia Gravis Data: Presented complete data from Phase 2 clinical trial of efgartigimod in gMG at the American Academy of Neurology (AAN) Annual Meeting.

Data showed clinical improvement of efgartigimod over placebo through entire 10-week duration of the trial.

Clinical benefit in the treatment group maximized as of one week after administration of last dose, achieving statistical significance over the placebo group on the Myasthenia Gravis Activity-of-Daily-Living (MG-ADL) score.

All patients in the treatment arm showed reduction of total IgG levels, and clinically meaningful disease improvement was found to correlate with a reduction in pathogenic IgG levels.

Efgartigimod was well-tolerated in all patients, with most adverse events (AEs) characterized as mild and deemed unrelated to the study drug. No serious or severe AEs were reported.

End-of-Phase 2 Meeting with FDA: Received feedback from the U.S. Food and Drug Administration (FDA) during the end-of-Phase 2 meeting on the framework of our Phase 3 program for efgartigimod in gMG.

Global Phase 3 clinical trial expected to evaluate the efficacy of a 10 mg/kg intravenous (IV) dose of efgartigimod in approximately 150 gMG patients over a 26-week period.

argenx expects to enroll in the trial both AChR autoantibody positive patients and AChR autoantibody negative patients whose disease is driven by MuSK and LRP4 autoantibodies, among others.

Patients in the Phase 3 clinical trial would be able to roll over into an open-label extension study for a period of one year.

Interim Data from First Cohort in Phase 2 PV Trial: Reported interim data from the first cohort of our Phase 2 proof-of-concept clinical trial of efgartigimod for the treatment of PV.

Rapid disease control observed in four of the six mild-to-moderate PV patients treated, and efgartigimod was well-tolerated in all treated PV patients.

Strong pharmacodynamic effect correlated with an improvement in Pemphigus Disease Area Index (PDAI) score, characterized by the start of healing of existing lesions and absence of formation of new lesions.

Independent Data Monitoring Committee recommended advancing to cohort 2 with an increased dosing frequency and dosing duration during the maintenance phase.

Phase 1 Data using Subcutaneous Formulation: Announced data from the Phase 1 study of the subcutaneous (SC) formulation of efgartigimod, demonstrating comparable characteristics to the IV formulation, including half-life, pharmacodynamics and tolerability.
Data showed that repeat exposure to SC efgartigimod can maintain IgG suppression at a steady state, with the possibility to dose up or down based on patient needs.
argenx intends to explore various dosing schedules with the SC formulation to best address patient needs, including an IV loading dose followed by SC maintenance as one possible schedule.

ARGX-110 Program

Ongoing Enrollment in Phase 2 Trial of ARGX: argenx expects to enroll an initial 21 patients in the ongoing Phase 2 part of the Phase 1/2 proof-of-concept trial of ARGX-110 in combination with standard of care azacytidine in newly diagnosed, elderly AML and high-risk myelodysplastic syndromes patients who are unfit for chemotherapy. The Company expects to use the selected ARGX-110 dose of 10 mg/kg as determined from the dose-escalation part of the trial.

Corporate Updates

Received second preclinical milestone payment under the development agreement with AbbVie for ARGX-115 targeting novel immune checkpoints in oncology.

Received third preclinical milestone payment from collaboration with LEO Pharma following approval of our clinical trial application (CTA) filing for ARGX-112 to treat inflammatory skin disorders.

Received a milestone payment from the strategic collaboration with Shire triggered by Shire exercising its exclusive option to in-license an antibody discovered and developed using the Company’s proprietary SIMPLE Antibody platform and Fc engineering technologies.

Appointed R. Keith Woods as Chief Operating Officer.

Selected for BEL 20 Index representing the 20 largest companies traded on Euronext Brussels, subject to meeting Euronext Index Family criteria and review.

UPCOMING MILESTONES

Advance efgartigimod into Phase 3 clinical development in gMG before the end of 2018.

Report topline data from the Phase 2 proof-of-concept trial for efgartigimod in immune thrombocytopenia (ITP) before the end of the third quarter of 2018 and present the full dataset at a workshop around the ASH (Free ASH Whitepaper) Annual Meeting.

Report full data from the Phase 2 trial of efgartigimod in PV in the first half of 2019.

Report full data of the dose-escalation phase of the AML Phase 1/2 clinical trial and the cutaneous T-cell lymphoma Phase 2 clinical trial of ARGX-110 around the ASH (Free ASH Whitepaper) Annual Meeting.

Total operating income was €20.5 million for the six months ended June 30, 2018, compared to €23.9 million for the six months ended June 30, 2017. The decrease in operating income in 2018 was primarily due to a decrease of €4.5 million in revenue primarily from the completion of the preclinical activities under our ongoing collaboration with LEO Pharma. The decrease was offset by an increase in other operating income of €1.2 million, mainly driven by an increase in payroll tax rebates for employing certain research and development personnel.

Research and development expenses were €34.4 million for the six months ended June 30, 2018, compared to €25.6 million for the six months ended June 30, 2017. The increase in research and development expenses in 2018 was principally due to (i) an increase of €4.4 million in share-based compensation expense linked to the grant of stock options to our research and development employees (including an increase of €1.3 million in social security costs on stock options granted to certain Belgian and non-Belgian resident employees), (ii) an increase of €4.0 million in costs related to the advancement of the clinical development and manufacturing activities of ARGX-113 and ARGX-110 and (iii) costs associated with a planned increase in research and development headcount.

Selling, general and administrative expenses were €11.5 million for the six months ended June 30, 2018, compared to €5.0 million for the six months ended June 30, 2017. The increase of €6.5 million in selling, general and administrative expenses in 2018 is mainly explained by an increase of €6.1 million of personnel expenses resulting from (i) an increase of €4.9 million in share-based compensation expense linked to the grant of stock options to our selling, general and administrative employees (including an increase of €1.1 million in social security costs on stock options granted to certain Belgian and non-Belgian resident employees) and (ii) the recruitment of additional employees (notably in our US office) to further strengthen our selling, general and administrative activities.

Financial income and exchange gains amounted to €5.3 million for the six months ended June 30, 2018 compared to financial income and exchange losses of €0.8 million for the six months ended June 30, 2017, which was primarily attributable to unrealized exchange rate gains on our cash, cash equivalents and current financial assets position in USD linked to the favorable fluctuation of the USD exchange rate in the six months ended June 30, 2018.

The Group generated a loss for the period and total comprehensive loss of €20.1 million for the six months ended June 30, 2018, compared to a loss for the period and total comprehensive loss of €8.2 million for the six months ended June 30, 2017.

As at June 30, 2018, the Group’s cash, cash equivalents and current financial assets amounted to €338.9 million, compared to €359.8 million as at December 31, 2017.

EXPECTED 2018 FINANCIAL CALENDAR:

October 25, 2018: Third quarter 2018 business update and financial results.

On August 2, 2018 Cerus Corporation (Nasdaq: CERS) reported its financial results for the second quarter ended June 30, 2018, and raised its full year guidance for product revenue (Press release, Cerus, AUG 2, 2018, View Source [SID1234528344]).

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Second Quarter Highlights and Recent Events

Second quarter product revenue of $15.4 million, a 62% increase compared to the second quarter of 2017
Year-over-year worldwide disposable kit volumes increased over 70% in the second quarter of 2018
Cash, cash equivalents and short-term investments of $111.9 million at June 30, 2018
Amended agreement with the Biomedical Advanced Research and Development Authority (BARDA) for an additional $15 million in funding bringing the total potential contract value to over $200 million
Expanded cryoprecipitate manufacturing collaborations with the addition of The Blood Center of New Orleans
"The commercial momentum we experienced at the beginning of the year continued throughout the second quarter with product revenue totaling $15.4 million, exceeding our expectations," said William ‘Obi’ Greenman, Cerus’ president and chief executive officer. "Given the first half outperformance and based on current visibility into our commercial pipeline, we are raising our full year product revenue outlook to a range of $56 million to $58 million compared to our previous guidance range of $53 million to $55 million."

"We believe strong sales performances in France and with the American Red Cross are setting the stage for further market expansion and revenue growth," continued Greenman. "Furthermore, we think that awareness of the benefits of pathogen-reduction is increasing, as evidenced by the discussions during the recent U.S. Food and Drug Administration’s Blood Products Advisory Committee Meeting."

During the quarter, the Company’s development programs continued to progress, and the Company anticipates submitting for CE Mark approval of the INTERCEPT red blood cell system later in 2018. In addition, the Company expanded its cryoprecipitate manufacturing collaborations with the addition of The Blood Center of New Orleans.

Revenue

Product revenue during the second quarter of 2018 was $15.4 million, compared to $9.5 million during the same period in 2017. The increased product revenue was driven by quarter-over-quarter increases across all major product categories led by global platelet kit demand. Year-to-date product revenue totaled $29.0 million, an increase of 75% compared to the same period of the prior year.

Government contract revenue from the Company’s BARDA agreement was $4.0 million during the second quarter of 2018 compared to $1.7 million during the same period in 2017, as a result of increasing INTERCEPT red cell clinical and development activities. Year-to-date government contract revenue totaled $7.5 million compared to $3.1 million in the first half of 2017. BARDA is part of the Office of the Assistant Secretary for Preparedness and Response within the U.S. Department of Health and Human Services.

Gross Margins

Gross margins on product revenue during the second quarter of 2018 were 50%, compared to 54% for the second quarter of 2017. The change in gross margin was primarily attributable to lower selling prices associated with high volume customers. Gross margins through the first half of 2018 were 48% compared to 51% during the first half of 2017.

Operating Expenses

Total operating expenses were $24.3 million for the quarter ended June 30, 2018, compared to $23.0 million for the quarter ended June 30, 2017. Year-to-date, operating expenses totaled $47.4 million compared to $45.9 million in the same period in the prior year.

Selling, general, and administrative (SG&A) expenses for the second quarter of 2018 remained relatively flat at $14.4 million, compared to $14.1 million for the second quarter of 2017, as the Company continued to realize leverage from its existing commercial and back-office investments. Year-to-date SG&A expenses totaled $28.0 million compared to $27.8 million during the first half of 2017.

Research and development (R&D) expenses for the second quarter of 2018 were $9.9 million compared to $8.9 million for the second quarter of 2017. The increase in year-over-year R&D expenses were primarily due to increased activities and costs tied to the development of INTERCEPT red blood cells, including preparation for the planned CE Mark submission, and pursuing expanded usage claims for INTERCEPT platelets and plasma. Year-to-date R&D expenses through the second quarter of 2018 totaled $19.3 million compared to $18.0 million during the first half of 2017.

Operating and Net Loss

Operating loss during the second quarter of 2018 was $12.6 million, compared to $16.2 million during the second quarter of 2017. Year-to-date operating loss was $25.9 million compared to an operating loss of $34.3 million in the same period of the prior year.

Net loss for the second quarter of 2018 was $13.3 million, or $0.10 per diluted share, compared to a net loss of $17.1 million, or $0.16 per diluted share, for the second quarter of 2017. Year-to-date net loss was $27.2 million, or $0.21 per diluted share, compared to a net loss of $35.7 million, or $0.34 per diluted share, in the first half of 2017.

Cash, Cash Equivalents and Investments

At June 30, 2018, the Company had cash, cash equivalents and short-term investments of $111.9 million, compared to $60.7 million at December 31, 2017.

At June 30, 2018 and December 31, 2017, the Company had approximately $29.8 million in outstanding debt under its loan agreement with Oxford Finance.

QUARTERLY CONFERENCE CALL

The Company will host a conference call and webcast at 4:15 P.M. EDT this afternoon, during which management will discuss the Company’s financial results and provide a general business overview and outlook. To access the live webcast, please visit the Investor Relations page of the Cerus website at View Source Alternatively, you may access the live conference call by dialing (866) 235-9006 (U.S.) or (631) 291-4549 (international).

A replay will be available on the Company’s website, or by dialing (855) 859-2056 (U.S.) or (404) 537-3406 (international) and entering conference ID number 8278017. The replay will be available approximately three hours after the call through August 16, 2018.

On August 2, 2018 Omeros Corporation (Nasdaq: OMER) reported that Eckhard Leifke, M.D. has been named Omeros’ Chief Medical Officer and Vice President of Clinical Development (Press release, Omeros, AUG 2, 2018, View Source;p=RssLanding&cat=news&id=2361629 [SID1234528382]). In this role, Dr. Leifke will oversee clinical science & operations, medical affairs and pharmacovigilance. He assumes these corporate responsibilities from J. Steven Whitaker, M.D., J.D., Vice President of Clinical Development, who will now focus primarily on driving the clinical aspects of Omeros’ MASP-2 antibody – OMS721 – toward U.S. and international regulatory approvals in hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).

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"I am excited to join Omeros’ leadership team," said Dr. Leifke. "I am impressed by the quality of Omeros’ pipeline and by the sense of urgency within the company to advance these programs to improve the lives of patients. With input from thought leaders and their patients who are affected by the serious conditions for which Omeros is developing novel therapeutics, my top priority is to continue accelerating the company’s clinical programs toward successful commercialization."

Dr. Leifke brings to Omeros more than 20 years of drug development experience, having built and headed global teams at leading pharmaceutical companies including Bayer, Takeda and, most recently, Sanofi where he was Global Project Head/Vice President of Early Project & External Opportunities – Cardiovascular and Metabolism and Global Head/Vice President of Late-Stage Development Diabetes. He has led the global development of multiple early- and late-stage small-molecule and biologic drug candidates to successful market authorizations in the USA, Europe, Japan and other countries.

"We’re pleased to welcome Eckhard to our senior leadership team, and I expect that his proven track record and thoughtful approach to strategic drug development globally will be valuable assets for Omeros," said Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. "This important and timely expansion of our clinical team underscores Omeros’ progress and our longstanding commitment to providing cutting-edge therapeutics to patients with disabling and life-threatening disorders. I’d like to recognize Steve Whitaker for his ongoing exemplification of that same commitment, and I look forward to his leading OMS721 to a successful outcome in stem-cell TMA, currently one of Omeros’ highest priorities."

Dr. Leifke holds a Medical Doctorate from the University of Freiburg, Germany, and is Board-certified in Internal Medicine and Endocrinology.

On August 1, 2018 Blueprint Medicines Corporation (NASDAQ: BPMC), a leader in discovering and developing targeted kinase medicines for patients with genomically defined diseases, reported financial results and provided a business update for the second quarter ended June 30, 2018 (Press release, Blueprint Medicines, AUG 1, 2018, View Source [SID1234528283]).

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"In the second quarter, Blueprint Medicines continued to advance a broad portfolio, with progress across multiple programs," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "Importantly, we reported updated data from our Phase 1 EXPLORER trial in patients with advanced systemic mastocytosis that showed profound and durable clinical activity in nearly all patients. These data, combined with previously reported data from our ongoing Phase 1 NAVIGATOR trial in advanced gastrointestinal stromal tumors, reinforce our confidence in avapritinib as a potentially transformative therapy across multiple patient populations. By the end of this year, we expect to have four pivotal clinical trials of avapritinib underway, with the potential to rapidly advance toward approval in defined patient populations."

Clinical Programs:

Avapritinib: Gastrointestinal Stromal Tumors (GIST)

In June 2018, Blueprint Medicines announced the dosing of the first patient in its Phase 3 VOYAGER clinical trial, which will evaluate the safety and efficacy of avapritinib compared to regorafenib in patients with third- or fourth-line advanced GIST.
In June 2018, Blueprint Medicines presented data from a retrospective natural history study of patients with advanced PDGFRα D842V-driven GIST at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The data confirmed that patients with advanced PDGFRα D842V-driven GIST are unlikely to respond to currently available tyrosine kinase inhibitors (TKIs), illustrating the high unmet need for new therapies in this patient population with a short survival rate.
Blueprint Medicines continues to evaluate avapritinib in its Phase 1 NAVIGATOR clinical trial and anticipates presenting updated data across multiple patient populations, including PDGFRA-driven GIST, third-line or later GIST and second-line GIST, in the second half of 2018. Additionally, based on data from this trial, the Company plans to submit a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for avapritinib for the treatment of patients with PDGFRA-driven GIST and fourth-line KIT-driven GIST in the first half of 2019.
Avapritinib: Advanced Systemic Mastocytosis (SM)

In June 2018, Blueprint Medicines presented updated clinical data from its ongoing Phase 1 EXPLORER clinical trial of avapritinib in patients with advanced SM at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper). The data showed an overall response rate of 83 percent and durable ongoing responses up to 22 months. All evaluable patients showed marked decreases on one or more objective measures of mast cell burden, regardless of advanced SM subtype, previous treatment or starting dose level. The data also showed that avapritinib was generally well-tolerated. Most adverse events reported by investigators were Grade 1 or 2, and only three patients discontinued due to a treatment-related adverse event. Read the full data here.
Blueprint Medicines plans to initiate screening of patients for enrollment in PATHFINDER, a registration-enabling, open-label, single-arm Phase 2 clinical trial in patients with advanced SM, in the third quarter of 2018, and plans to initiate PIONEER, a registration-enabling, randomized, placebo-controlled Phase 2 clinical trial in patients with indolent and smoldering SM, by the end of 2018.
BLU-667: RET-Altered Solid Tumors

Blueprint Medicines continues to enroll patients in the expansion portion of its ongoing Phase 1 ARROW clinical trial of BLU-667 at a dose of 400 mg once daily. In the expansion, patients are being enrolled in four defined cohorts: RET-altered non-small cell lung cancer (NSCLC) patients previously treated with a TKI; RET-altered NSCLC patients who have not previously received any TKI treatment; patients with medullary thyroid cancer; and patients with other RET-altered solid tumors.
BLU-554: Hepatocellular Carcinoma (HCC)

In June 2018, Blueprint Medicines announced plans to initiate a proof-of-concept clinical trial with CStone Pharmaceuticals in China to evaluate BLU-554 in combination with CS1001, a clinical-stage anti-programmed death ligand-1 (PD-L1) immunotherapy being developed by CStone Pharmaceuticals, as a first-line treatment for patients with HCC. Additionally, the companies plan to expand Blueprint Medicines’ ongoing Phase 1 clinical trial of BLU-554 as a monotherapy to include new sites in Mainland China. The companies expect to submit an investigational new drug (IND) application for BLU-554 to the Chinese health authorities by the end of 2018, and plan to initiate the clinical trial evaluating BLU-554 in combination with CS1001 and the expansion of Blueprint Medicines’ ongoing clinical trial for BLU-554 as a monotherapy in Mainland China in 2019.
Corporate:

In June 2018, Blueprint Medicines announced an exclusive collaboration and license agreement with CStone Pharmaceuticals to develop and commercialize avapritinib, BLU-554 and BLU-667 in Mainland China, Hong Kong, Macau and Taiwan, either as a monotherapy or as part of a combination therapy. Under the terms of the agreement, Blueprint Medicines received an upfront cash payment of $40.0 million, will be eligible to receive up to approximately $346.0 million in potential milestone payments and tiered percentage royalties in the mid-teens to low twenties on annual net sales of each licensed product in the territory.
Blueprint Medicines recently received a $10.0 million milestone payment from Roche following the achievement of a research milestone.
Second Quarter Financial Results:

Cash Position: As of June 30, 2018, cash, cash equivalents and investments were $616.7 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 Blueprint Medicines entering into the collaboration with CStone Pharmaceuticals and the $10.0 million milestone payment received from Roche.
Collaboration Revenues: Collaboration revenues were $41.4 million for the second quarter of 2018, as compared to $5.9 million for the second quarter of 2017. This increase was primarily due to revenue recognized under the collaboration agreement with CStone Pharmaceuticals.
R&D Expenses: Research and development expenses were $58.6 million for the second quarter of 2018, as compared to $33.3 million for the second quarter of 2017. This increase was primarily attributable to increased clinical and manufacturing expenses associated with advancing avapritinib, BLU-554 and BLU-667 further through clinical trials and increased personnel-related expenses. Research and development expenses included $4.3 million in stock-based compensation expenses for the second quarter of 2018.
G&A Expenses: General and administrative expenses were $12.3 million for the second quarter of 2018, as compared to $6.8 million for the second quarter of 2017. This increase was primarily attributable to increased personnel-related expenses and increased professional fees, including pre-commercial planning activities. General and administrative expenses included $3.5 million in stock-based compensation expenses for the second quarter of 2018.
Net Loss: Net loss was $27.0 million the second quarter of 2018, or a net loss per share of $0.62, as compared to a net loss of $33.4 million for the second quarter of 2017, or a net loss per share of $0.86.
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 today at 8:30 a.m. ET. The conference call may be accessed by dialing (855) 728-4793 (domestic) or (503) 343-6666 (international) and referring to conference ID 5597837. A webcast of the conference call will be available in the Investors section of 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.