On August 1, 2019 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 financial results for the first half of 2019 and provided its second quarter business update and the outlook for the remainder of the year (Press release, argenx, AUG 1, 2019, View Source [SID1234537999]).

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"We continue to execute on accelerating and expanding our robust, late-stage innovative clinical development programs, as we invest in forward integration across our organization to maximize value. Efgartigimod is the most advanced and also the broadest FcRn antagonist program with four indications and both intravenous and subcutaneous formulations. Enrollment for the Phase 3 ADAPT trial in generalized myasthenia gravis is progressing as planned and we are on track to initiate before the end of the year the first of two pivotal Phase 3 trials, ADVANCE, for our global primary immune thrombocytopenia program, as well as a Phase 2 trial in chronic inflammatory demyelinating polyneuropathy. Our Phase 2 trial of efgartigimod in pemphigus vulgaris remains a priority, and we expect to report topline data in the first half of 2020," commented Tim Van Hauwermeiren, CEO of argenx. "As we move towards becoming a fully-integrated biotechnology company, we continue to invest in commercial infrastructure with the build-out of our neuromuscular and hematology franchises and the expansion of our global supply chain through our longstanding collaboration with Lonza, which is expected to support the commercial launch of efgartigimod in generalized myasthenia gravis in 2021."

SECOND QUARTER 2019 AND RECENT HIGHLIGHTS

During its 2019 R&D Day in May, argenx announced its plan to become a fully integrated, global immunology company in accordance with its "argenx 2021" vision, which includes building two successful commercial franchises in neuromuscular and hematological disorders.

Efgartigimod (ARGX-113): Potential to be best-in-class with broad applicability

Efgartigimod is a human IgG1 Fc fragment engineered to increase affinity for FcRn versus endogenous IgG, whilst preserving characteristic pH-dependent binding, which may contribute to efgartigimod’s relatively long serum half-life and pharmacodynamic effect, and may promote tissue penetration. Treatment with efgartigimod results in a targeted reduction of IgG autoantibodies and is a rational approach to diseases where IgGs are directly pathogenic. argenx is evaluating efgartigimod as a potential treatment for four high-value indications, including:

Generalized Myasthenia Gravis (gMG)

Global, multi-center Phase 3 ADAPT clinical trial, including ADAPT+ one-year open-label extension study, currently ongoing

With current enrollment on track, topline data are expected in second half of 2020

Results from completed Phase 2 clinical trial were published in Neurology

Primary Immune Thrombocytopenia (ITP)

Global Phase 3 program to include two registration trials that will be run concurrently

First trial (ADVANCE) to evaluate 10 mg/kg intravenous (IV) efgartigimod on top of standard of care medication, with enrollment up to 158 patients; primary endpoint includes achieving sustained platelet count response of at least 50×109/L

Second trial to evaluate 10 mg/kg IV induction period followed by subcutaneous (SC) injections, all on top of standard of care medication, to evaluate potential of SC product to maintain clinical benefit

Phase 3 program was developed following consultation with key regulatory agencies

ADVANCE Phase 3 clinical trial expected to start in second half of 2019

Pemphigus Vulgaris (PV)

Phase 2 proof-of-concept clinical trial ongoing and currently enrolling patients in third cohort with extended dosing of efgartigimod

Data from Phase 2 clinical trial expected in first half of 2020

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Phase 2 clinical trial on track to start in second half of 2019

Key opinion leader (KOL) event planned for fourth quarter 2019 to discuss Phase 2 trial design and unmet needs in CIDP

argenx entered into a global collaboration with Halozyme in February 2019 to develop a SC formulation of efgartigimod using Halozyme’s proprietary ENHANZE drug delivery technology, gaining exclusive rights to the technology for the FcRn target.

First subject dosed in Phase 1 healthy volunteer (HV) trial evaluating safety, pharmacokinetics, pharmacodynamics and bioavailability of ENHANZE SC formulation of efgartigimod

Initiation of study triggered $5 million milestone payment to Halozyme

Data from Phase 1 HV trial are expected by end of 2019 after which argenx will disclose a path forward in patients for ENHANZE SC formulation of efgartigimod

Cusatuzumab (ARGX-110): First-in-class opportunity in acute myeloid leukemia (AML)

Cusatuzumab is a first-in-class monoclonal antibody inhibiting CD70, a target that is uniquely present on both leukemic stem cells and AML blasts but not healthy cells. It is being developed under an exclusive global collaboration and license agreement with Janssen for the treatment of AML, high-risk myelodysplastic syndromes and other hematological malignancies.

Phase 2 and registration-directed clinical trial in AML on track to start in second half of 2019

Trial to enroll up to 150 patients with previously untreated AML and who are not eligible for intensive chemotherapy

In this two-part trial, patients will first be randomized to receive one of two dose levels of cusatuzumab (10 mg/kg and 20 mg/kg) in combination with azacytidine (75 mg/m2) followed by an expansion cohort to evaluate efficacy of the selected dose of cusatuzumab

Early Development Programs

argenx announced during its R&D Day the expansion of its product pipeline with the addition of two new proprietary therapeutic candidates, ARGX-117 and ARGX-118. Both emerged from argenx’s Innovative Access Program, in which it collaborates closely with disease biology experts, bringing the argenx cutting-edge antibody discovery and engineering technologies to the heart of novel target research.

ARGX-117 is a complement-targeting antibody against C2, a component of both the classical and lectin pathways in the complement cascade

Potential therapeutic applications in multiple autoimmune diseases

argenx exercised its second exclusive license to Halozyme’s ENHANZE technology for use with this molecule

Expected to file Clinical Trial Application (CTA) by end of 2019 with first-in-human trial expected to start in first quarter of 2020

ARGX-118 is a highly differentiated antibody against Galectin-10, the protein of Charcot-Leyden crystals (CLCs), which play a major role in severe asthma and the persistence of mucus plugs

Immunology breakthrough in airway inflammation

SIMPLE Antibody observed to have unique crystal-dissolving properties

Currently in final stages of lead optimization work

Data were published in Science by argenx collaborator Dr. Bart Lambrecht from VIB Inflammation Research Center supporting role of CLCs and potential of ARGX-118 in airway inflammation

Corporate Update

Appointed Wim Parys, M.D. as Chief Medical Officer effective July 1, 2019. Most recently, Dr. Parys served as Head of R&D of the Global Public Health group of Janssen.

HALF YEAR 2019 FINANCIAL RESULTS (CONSOLIDATED)

On June 30, 2019, cash, cash equivalents and current financial assets totaled €944.3 million, compared to €564.6 million on December 31, 2018. The increase in cash, cash equivalents and current financial assets resulted primarily from the closing of the exclusive global collaboration and license agreement for cusatuzumab with Janssen which resulted in a $300 million upfront payment and a $200 million equity investment in January 2019.

Total operating income increased by €30.8 million for the six months ended June 30, 2019 to reach €51.3 million, compared to €20.5 million for the six months ended June 30, 2018. The increase is primarily related to (i) a €16.0 million increase in the recognition of milestone payments following the initiation of a first-in-human clinical trial with ABBV-151 (formerly named ARGX-115) under the AbbVie collaboration, which triggered a $30 million milestone payment, (ii) an increase of €7.8 million related to the recognition of research and development service fees under the Janssen collaboration and (iii) an increase of €5.2 million, mainly driven by higher payroll tax rebates for employing certain research and development personnel.

Research and development expenses totaled €78.3 million and €34.4 million for the six months ended June 30, 2019 and 2018 respectively. The increase in the first six months of 2019 resulted primarily from higher external research and development expenses and personnel expenses, reflecting higher clinical trials costs and manufacturing expenses related to the development of argenx’s product candidate portfolio and the recruitment of additional employees to support research and development activities.

Selling, general and administrative expenses totaled €27.5 million and €11.5 million for the six months ended June 30, 2019 and 2018, respectively. The increase of €16.0 million in selling, general and administrative expenses for the six months ended June 30, 2019 primarily resulted from higher personnel expenses and consulting fees related to the preparation of a possible future commercialization of argenx’s lead product candidate efgartigimod.

For the six months ended June 30, 2019, financial income amounted to €7.2 million, compared to €1.3 million for the six months ended June 30, 2018. The increase of €5.9 million in the first six months of 2019 related primarily to an increase in the interest received on cash, cash equivalents and current financial assets.

Exchange gains totaled €2.5 million for the six months ended June 30, 2019, compared to the €4.0 million for the six months ended June 30, 2018 and were mainly attributable to unrealized exchange rate gains on argenx’s cash and current financial assets position in U.S. Dollars due to the favorable fluctuation of the EUR/USD exchange rate in the first six months of 2019.

The total comprehensive loss for the six months ended June 30, 2019 was €45.1 million, compared to €20.1 million for the six months ended June 30, 2018.

The 90 day average number of shares outstanding per June 30, 2019 was 38,026,040.

Financial Outlook

Based on the current objectives of the Company’s business plan, argenx expects that its existing cash, cash equivalents and investments will fund planned operating and capital expense requirements into 2021. With the launch of a second global Phase 3 trial for efgartigimod, the execution of the development plan for cusatuzumab, the build-out of the commercial organization, and the expansion of the Company’s ambition level within its growing business plan, argenx expects operating and capital expense requirements to continue to increase year-over-year.

U.S. SEC and Statutory Financial Reporting

argenx’s primary accounting framework is International Financial Reporting Standards (IFRS) as issued by the International Accounting Standards Board (IASB). Unaudited condensed half yearly interim financial statements prepared in accordance with International Accounting Standards IAS 34 Interim Financial Reporting are available on www.argenx.com.

In addition to reporting financial figures in accordance with IFRS as issued by the IASB, argenx also reports financial figures in accordance with IFRS as adopted by the European Union (EU) for statutory purposes. The unaudited condensed consolidated statement of financial position, the unaudited condensed consolidated statements of profit and loss and other comprehensive income, the unaudited condensed consolidated statements of cashflow, and the unaudited condensed consolidated statement of changes in equity are not affected by any differences between IFRS as issued by the IASB and IFRS as adopted by the EU.

The condensed consolidated statement of profit and loss and other comprehensive income for the six months ended June 30, 2019 presented in this press release is unaudited.

2019 FINANCIAL CALENDAR

October 24, 2019: Q3 2019 business update and financial results

CONFERENCE CALL DETAILS

The half year results will be discussed during a conference call and webcast presentation today at 3:00 pm CEST/9:00 am ET. To participate in the conference call and Q&A session, please select your phone number provided below and use the confirmation code 7539308. The live webcast may be accessed on the homepage of the argenx website at www.argenx.com or by clicking here.

Belgium

+32 (0)2 400 9874

Belgium

0800 48740

France

+33 (0)1 767 00794

France

0805 103028

Netherlands

+31 (0)20 714 3545

Netherlands

0800 0249557

United Kingdom

+44 (0)844 571 8892

United Kingdom

0800 376 7922

United States

+1 (631) 510 7495

United States

+1 (866) 966 1396

On August 1, 2019 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported second quarter 2019 financial results and provided a corporate update (Press release, Kura Oncology, AUG 1, 2019, View Source [SID1234538015]).

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"The past quarter was highlighted by impressive data from our ongoing Phase 2 trial of tipifarnib in peripheral T-cell lymphoma (PTCL)," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "These data support the potential to register tipifarnib in both the angioimmunoblastic T-cell lymphoma (AITL) and PTCL not otherwise specified (PTCL-NOS) populations. In addition, we took further steps toward broadening the potential of farnesyl transferase inhibition to treat other CXCL12-driven indications of high unmet need, including diffuse large B-cell lymphoma (DLBCL), cutaneous T-cell lymphoma (CTCL), acute myeloid leukemia (AML) and pancreatic cancer. These advancements, along with the continued execution of our registration-directed study in HRAS mutant head and squamous cell carcinomas (HNSCC), are important steps toward establishing tipifarnib as a first-in-class farnesyl transferase inhibitor for patients with certain genetically defined cancers."

"As we look toward the second half of this year and early next year, we anticipate a number of milestones across our pipeline programs," continued Dr. Wilson, "including an update from our ongoing Phase 2 trial of tipifarnib in HRAS mutant HNSCC and other squamous cell carcinomas (SCCs), additional Phase 2 data in AITL, completion of the dose-escalation portion of the Phase 1 trial of our ERK inhibitor, KO-947, and initiation of the Phase 1 trial of our menin-MLL inhibitor, KO-539. With our successful public offering in June, we have greater resources and flexibility to continue to create value across our pipeline."

Recent Highlights

Registration-directed trial of tipifarnib in HRAS mutant HNSCC – Kura continues to execute on its initial registration-directed trial of tipifarnib in HRAS mutant HNSCC. The clinical trial has two cohorts: A non-interventional screening and outcomes cohort (SEQ-HN) and a treatment cohort (AIM-HN). AIM-HN is designed to enroll at least 59 evaluable patients with HRAS mutant HNSCC who have received prior platinum-based therapy. AIM-HN initiated in November 2018 and is expected to take approximately two years to fully enroll.

Positive Phase 2 trial of tipifarnib in AITL and CXCL12-driven PTCL – In June 2019, Kura reported positive data from its ongoing Phase 2 trial of tipifarnib in patients with relapsed or refractory PTCL, including clinical proof-of-concept in the trial’s two expansion cohorts: 1) patients with AITL, an aggressive form of T-cell lymphoma often characterized by high levels of CXCL12 expression, and 2) patients with PTCL who lack a single nucleotide variation in the 3’-untranslated region of the CXCL12 gene. The Company believes these data could support the potential registration of tipifarnib in both the AITL and PTCL-NOS patient populations and intends to seek regulatory feedback on next steps for this program. In addition, Kura plans to continue enrolling AITL patients in the trial and expects to provide additional data from this cohort at a medical meeting later this year.

Expanded patent protection for tipifarnib in the U.S. and Europe – The U.S. Patent and Trademark Office recently issued two new patents further protecting tipifarnib, including a method of treating patients with HRAS mutant HNSCC with any farnesyl transferase inhibitor and a method of treating patients with HRAS mutant non-small cell lung carcinoma with tipifarnib. In addition, the European Patent Office granted a patent directed to the use of tipifarnib as a method of treating patients with HRAS mutant HNSCC. The new patents have an expiration date of August 2036, excluding any possible patent term extension.

Dose escalation in Phase 1 trial of KO-947 advancing – Kura continues to evaluate dosing regimens for KO-947, its potent and selective small molecule inhibitor of ERK, in an ongoing Phase 1 trial, with a goal of reaching a recommended Phase 2 dose or maximum tolerated dose. The Company expects to complete the dose-escalation portion of the trial by the end of 2019.

Orphan Drug Designation to KO-539 for treatment of AML – Last week, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to KO-539, Kura’s menin-MLL inhibitor for the treatment of AML. The FDA cleared the investigational new drug application for KO-539 in March 2019, and the Company is in the final stages of study startup for a Phase 1 clinical trial of KO-539 in relapsed or refractory AML.

Successful public offering strengthens cash position – In June 2019, Kura completed a public offering in which the Company sold an aggregate of 6,785,000 shares of common stock at a price of $17.00 per share. Net proceeds from the public offering, after deducting underwriting discounts, commissions and offering expenses, were approximately $108.1 million.
Financial Results

Research and development expenses for the second quarter of 2019 were $11.4 million, compared to $11.5 million for the second quarter of 2018.

General and administrative expenses for the second quarter of 2019 were $4.5 million, compared to $3.8 million for the second quarter of 2018.

Net loss for the second quarter of 2019 was $14.9 million, or $0.38 per share, compared to a net loss of $14.7 million, or $0.45 per share, for the second quarter of 2018.

Cash, cash equivalents and short-term investments totaled $261.4 million as of June 30, 2019, which includes net proceeds of approximately $108.1 million from a public offering completed in June 2019, compared with $179.0 million as of December 31, 2018.

Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund current operations into the second half of 2021.
Upcoming Milestones

Initiation of the Phase 1 trial of KO-539 in relapsed or refractory AML in the second half of 2019

Additional data from the ongoing Phase 2 trial of tipifarnib in HRAS mutant HNSCC and other SCCs in the fourth quarter of 2019

Additional data from the AITL expansion cohort in the ongoing Phase 2 trial of tipifarnib in the fourth quarter of 2019

Completion of the dose-escalation portion of the Phase 1 trial of KO-947 by the end of 2019

Additional data from the ongoing Phase 2 trial of tipifarnib in chronic myelomonocytic leukemia (CMML) in the first half of 2020

Initiation of a proof-of-concept study of tipifarnib in pancreatic cancer in the first half of 2020
Conference Call and Webcast

Kura’s management will host a webcast and conference call today at 4:30 p.m. ET / 1:30 p.m. PT, August 1, 2019, to discuss the financial results for the second quarter 2019 and provide a corporate update. The live call may be accessed by dialing (877) 516-3514 for domestic callers and (281) 973-6129 for international callers and entering the conference code: 5261899. A live webcast of the call will be available from the Investors and Media section of the Company’s website at www.kuraoncology.com, and a replay will be available shortly after the live event.

On August 1, 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 quarter ended June 30, 2019 (Press release, Blueprint Medicines, AUG 1, 2019, View Source [SID1234538031]).

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"The first half of 2019 was a defining period for Blueprint Medicines marked by the submission of our first marketing applications for avapritinib in the United States and Europe, just four years after we initiated clinical development," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "As we prepare for the potential launch of avapritinib, we continue to advance our broader portfolio toward critical program milestones, including multiple planned marketing applications for avapritinib and pralsetinib in 2020. In addition, we plan to present initial clinical data from our ongoing Phase 2 PIONEER trial of avapritinib in patients with indolent systemic mastocytosis in the fourth quarter of 2019 and share an update on our research vision and portfolio strategy at our first R&D Day in November 2019."

Second Quarter 2019 Highlights and Recent Progress:

Avapritinib: Gastrointestinal stromal tumors (GIST)

Submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for avapritinib for the treatment of adult patients with PDGFRA exon 18 mutant GIST, regardless of prior therapy, and fourth-line GIST.
Submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for avapritinib for the treatment of adult patients with PDGFRα D842V mutant GIST, regardless of prior therapy, and fourth-line GIST and received EMA validation of the submission. Validation of the MAA confirms that the application is sufficiently complete to begin the formal review process.
Presented updated clinical data from the ongoing registration-enabling NAVIGATOR trial of avapritinib in patients with PDGFRA Exon 18 mutant GIST and fourth-line GIST at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2019 Annual Meeting. Read the full data here.
Dosed the first patient in China in the ongoing Phase 3 VOYAGER trial of avapritinib in patients with third-line GIST, under Blueprint Medicines’ collaboration with CStone Pharmaceuticals.
Avapritinib: Systemic mastocytosis (SM)

Presented updated clinical data from the ongoing EXPLORER trial of avapritinib in patients with advanced systemic mastocytosis (SM) at the 24thCongress of the European Hematology Association (EHA) (Free EHA Whitepaper). Read the full data here.
Pralsetinib (formerly BLU-667): RET-altered solid tumors

Presented updated clinical data from the ongoing registration-enabling Phase 1/2 ARROW trial of pralsetinib in patients with RET-altered non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC) and other cancers at ASCO (Free ASCO Whitepaper). Read the full data here.
Based on encouraging clinical data for pralsetinib in patients with treatment-naïve RET fusion-positive NSCLC and preliminary FDA feedback, expanded the enrollment target for the ARROW trial cohort for treatment naïve patients with RET-fusion NSCLC to enable potential expedited development in first-line NSCLC.
Fisogatinib (formerly BLU-554): Advanced hepatocellular carcinoma (HCC)

Under Blueprint Medicines’ collaboration with CStone Pharmaceuticals, the China National Medical Products Administration granted approval to conduct a Phase 1b/2 clinical trial evaluating fisogatinib in combination with CS1001, CStone Pharmaceuticals’ anti-PD-L1 inhibitor, in patients with HCC.
Dosed the first patient in China in the ongoing Phase 1 trial of fisogatinib in advanced HCC, under Blueprint Medicines’ collaboration with CStone Pharmaceuticals.
Key Upcoming Milestones:

The company expects to achieve the following milestones by the end of 2019:

Complete enrollment in the Phase 3 VOYAGER trial of avapritinib in third-line GIST.
Initiate the Phase 3 COMPASS-2L precision medicine trial evaluating avapritinib in second-line GIST.
Present initial data from the Phase 2 PIONEER trial of avapritinib in indolent and smoldering SM.
Complete enrollment of the Phase 2 PATHFINDER trial of avapritinib in advanced SM.
Initiate a Phase 3 trial evaluating pralsetinib in first-line RET-fusion NSCLC.
Initiate a Phase 2 trial evaluating pralsetinib in combination with osimertinib in EGFR-mutant NSCLC harboring an acquired RET alteration.
Initiate a Phase 1b/2 trial in China evaluating fisogatinib in combination with CS1001, CStone Pharmaceuticals’ anti-PD-L1 inhibitor, in patients with HCC.
Initiate a Phase 2 trial of BLU-782 in fibrodysplasia ossificans progressiva.
Share research vision and provide portfolio strategy update at an R&D Day on November 5, 2019, including disclosure of up to two new targets.
Nominate at least one new wholly-owned discovery program.
Second Quarter 2019 Financial Results:

Cash Position: As of June 30, 2019, cash, cash equivalents and investments were $667.3 million, as compared to $494.0 million as of December 31, 2018. This increase reflects net proceeds of approximately $327.4 million from the company’s follow-on underwritten public offering of common stock, which closed in April 2019, partially offset by cash used in operations.
Collaboration Revenues: Collaboration revenues were $5.1 million for the second quarter of 2019, as compared to $41.4 million for the second quarter of 2018. This decrease was primarily due to $40.0 million in revenue recognized under the CStone collaboration in the second quarter of 2018 upon execution of the collaboration agreement. During the second quarter of 2019, the company achieved and recognized a $4.0 million development and regulatory milestone payment under the CStone collaboration.
R&D Expenses: Research and development expenses were $87.1 million for the second quarter of 2019, as compared to $58.6 million for the second quarter of 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 $7.5 million in stock-based compensation expenses for the second quarter of 2019.
G&A Expenses: General and administrative expenses were $21.9 million for the second quarter of 2019, as compared to $12.3 million for the second quarter of 2018. This increase was primarily due to increased personnel expenses and increased professional fees for commercial-readiness and other activities. General and administrative expenses included $6.2 million in stock-based compensation expenses for the second quarter of 2019.
Net Loss: Net loss was $99.7 million for the second quarter of 2019, or a net loss per share of $2.04, as compared to a net loss of $27.0 million for the second quarter of 2018, or a net loss per share of $0.62.
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 middle of 2021.

Conference Call Information:

Blueprint Medicines will host a live conference call and webcast at 8:30 a.m. ET today to discuss second quarter 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 1096287. A webcast of the conference call will be available in the Investors section of the Blueprint Medicines’ website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

On August 1, 2019 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the second quarter ended June 30, 2019 (Press release, bluebird bio, AUG 1, 2019, View Source [SID1234538047]).

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"With the approval of our first gene therapy, bluebird bio has entered into an exciting, and potentially transformative time for patients and for the company. We are focused on getting our qualified treatment centers up and running in Europe, ensuring we are prepared to deliver ZYNTEGLO to patients, and advancing the implementation of our value and outcomes-based payment model," said Nick Leschly, chief bluebird. "In the weeks since announcing our approval, we have been encouraged by our progress across these fronts and by the receptivity to our payment model from payers in our initial European launch markets. In the second half of the year, we are focused on executing our clinical studies as well as initiating new studies in sickle cell disease, multiple myeloma, and early stage oncology programs. I am incredibly grateful for our amazing and growing flock of bluebirds who are working to ensure that we do our best for patients every day."

Recent Highlights:

TDT

EU CONDITIONAL MARKETING AUTHORIZATION – In June 2019, bluebird bio announced that the European Commission (EC) granted conditional marketing authorization for ZYNTEGLO (autologous CD34+ cells encoding βA-T87Q-globin gene, known as LentiGlobin for TDT), a gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available. The PRIME and Adaptive Pathway programs allowed for early and enhanced dialogue and accelerated assessment of ZYNTEGLO, which was completed on the shortest timetable for an advanced therapy medicinal product (ATMP) by the EMA to date. ZYNTEGLO is bluebird bio’s first gene therapy to gain regulatory approval. bluebird bio will continue the country-by-country reimbursement process to help ensure access to ZYNTEGLO for appropriate patients.
DATA FROM NORTHSTAR, NORTHSTAR-2 AND NORTHSTAR-3 PRESENTED – At the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress in June 2019, bluebird bio presented new data from its studies of LentiGlobin in patients with TDT: long-term data from the completed Phase 1/2 Northstar study (HGB-204), updated data from the Phase 3 Northstar-2 study (HGB-207) in patients with non-β0/β0 genotypes, and updated data from the Phase 3 Northstar-3 study (HGB-212) in patients with β0/β0 genotypes or an IVS-I-110 mutation.
SCD

DATA FROM HGB-206 PRESENTED – At EHA (Free EHA Whitepaper) in June 2019, bluebird bio presented new data from patients in Group C of its ongoing Phase 1/2 HGB-206 study of the company’s investigational LentiGlobin gene therapy for sickle cell disease (SCD). Group C patients are being treated under an updated study protocol, which includes the implementation of mobilization and apheresis with plerixafor.
TDT & SCD

HGB-205 STUDY COMPLETION – In February 2019, the final patient completed primary follow up in the HGB-205 study of patients with TDT and SCD. All patients will continue to be monitored for long term outcomes.
COMPANY

ANALYST DAY – In May 2019, bluebird bio hosted an analyst day in New York that highlighted significant progress in the company’s emerging immuno-oncology and severe genetic disease pipeline, launch plans for its first gene therapy product, and key aspects of its long-term growth strategy. bluebird bio also announced a new research collaboration with Seattle Children’s Research Institute in Acute Myeloid Leukemia (AML), a planned Phase 1/2 study in Merkel Cell Carcinoma (MCC) in collaboration with the Fred Hutchinson Cancer Research Center and preclinical programs in Diffuse Large B-cell Lymphoma (DLBCL), MAGE-A4 positive solid tumors and Mucopolysaccharidosis (MPSI).
Upcoming Anticipated Milestones:

TDT
Initiation of a rolling Biologics Licensing Application submission to the U.S. FDA for ZYNTEGLO in patients with TDT and non-β0/β0 genotypes by the end of 2019
Presentation of ZYNTEGLO clinical data from the Northstar-2 (HGB-207) clinical study in patients with TDT and non-β0/β0 genotypes by the end of 2019
Presentation of ZYNTEGLO clinical data from the Northstar-3 (HGB-212) clinical study in patients with TDT and a β0/β0 genotype or an IVS-I-110 mutation by the end of 2019
SCD
Initiation of Phase 3 HGB-210 study of LentiGlobin in patients with SCD by the end of 2019
Presentation of LentiGlobin clinical data from the HGB-206 clinical study in patients with SCD by the end of 2019
Multiple Myeloma
ide-cel clinical data update from the registration-enabling KarMMa study in patients with relapsed/refractory multiple myeloma by the end of 2019
Presentation of bb21217 clinical data from the CRB-402 clinical study in patients with relapsed/refractory multiple myeloma by the end of 2019
CALD
Presentation of updated clinical data from the Starbeam (ALD-102) study in patients with cerebral ALD by the end of 2019
Second Quarter 2019 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2019 and December 31, 2018 were $1.54 billion and $1.89 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of normal operating activities and cash used to purchase property, plant and equipment as the company continues the buildout of its manufacturing facility in Durham, North Carolina.
Revenues: Collaboration and license and royalty revenues were $13.3 million for the three months ended June 30, 2019 compared to $7.9 million for the three months ended June 30, 2018. The increase of $5.4 million was primarily attributable to an increase in collaboration revenue under our arrangement with Celgene as well as an increase in license and royalty revenue. Collaboration and license and royalty revenues were $25.8 million for the six months ended June 30, 2019 compared to $23.8 million for the six months ended June 30, 2018. The increase of $2.0 million was primarily attributable to an increase in license and royalty revenue, offset by a decrease in collaboration revenue under our arrangement with Celgene.
R&D Expenses: Research and development expenses were $146.5 million for the three months ended June 30, 2019 compared to $115.0 million for the three months ended June 30, 2018. Research and development expenses were $269.2 million for the six months ended June 30, 2019 compared to $212.1 million for the six months ended June 30, 2018. The increase in both periods was primarily driven by costs incurred to advance and expand the company’s pipeline.
G&A Expenses: General and administrative expenses were $68.6 million for the three months ended June 30, 2019 compared to $41.2 million for the three months ended June 30, 2018. General and administrative expenses were $128.9 million for the six months ended June 30, 2019 compared to $76.1 million for the six months ended June 30, 2018. The increase in both periods was largely attributable to costs incurred to support the company’s overall growth of its pipeline as well as commercial-readiness activities.
Net Loss: Net loss was $195.8 million for the three months ended June 30, 2019 compared to $146.0 million for the three months ended June 30, 2018. Net loss was $360.2 million for the six months ended June 30, 2019 compared to $261.1 million for the six months ended June 30, 2018.

On August 1, 2019 Ziopharm Oncology, Inc. ("Ziopharm") (Nasdaq: ZIOP), reported that it has entered into an agreement with existing investors for the exercise of previously issued warrants to purchase common stock in a private placement that is expected to result in gross proceeds to Ziopharm of approximately $45 million, before deducting placement agent and other offering expenses (Press release, Ziopharm, AUG 1, 2019, View Source [SID1234538072]). The private placement, which strengthens Ziopharm’s balance sheet and provides capital to fund operations into 2021, is being led by existing stockholder, MSD Partners, L.P.

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Pursuant to the terms of the securities issuance agreement, at the closing of the private placement, the investors have agreed to exercise their warrants for an aggregate of 15,015,152 shares of common stock, at an exercise price of $3.01 per share. The warrants being exercised were originally issued by Ziopharm in a private placement Ziopharm closed in November 2018

Concurrently, in this private placement, Ziopharm will issue new warrants to purchase up to 15,015,152 additional shares of common stock. The warrants will become exercisable six months following the date of issuance, will expire on the fifth anniversary of the initial exercise date and have an exercise price of $7.00.

The private placement is expected to close on or about July 30, 2019, subject to the satisfaction of customary closing conditions. Additional details regarding the private placement will be included in a Form 8-K to be filed by Ziopharm with the Securities and Exchange Commission (the "SEC").

The warrants to be issued in the private placement have not been registered under the Securities Act of 1933, as amended (the "Securities Act"). Accordingly, these securities may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act. As part of the private placement, Ziopharm has agreed to file a registration statement with the SEC for the purpose of registering the resale of the shares of Ziopharm common stock to be issued in the private placement as well as the shares of Ziopharm common stock issuable upon exercise of the warrants.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction. Any offering of the securities under the resale registration statement will only be by means of a prospectus.