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.

On August 1, 2019 ArQule, Inc. (Nasdaq: ARQL) reported that Peter Lawrence, President and Chief Operating Officer, and Dr. Brian Schwartz, Chief Medical Officer and Head of Research and Development, reported that it will present at the SVB Leerink Spotlight Series: Rare & Genetic Diseases on August 8, 2019 at 9:00 a.m. ET at the The Boston Harbor Hotel in Boston, MA (Press release, ArQule, AUG 1, 2019, View Source [SID1234538000]).

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On August 1, 2019 Navidea Biopharmaceuticals, Inc. (NYSE American: NAVB) ("Navidea" or the "Company"), a company focused on the development of precision immunodiagnostic agents and immunotherapeutics, reported it will host a conference call and webcast on Thursday, August 8, 2019 at 5:00 p.m. (EDT) to discuss its financial results for the second quarter of 2019 (Press release, Navidea Biopharmaceuticals, AUG 1, 2019, View Source [SID1234538016]).

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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

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Jed Latkin, Chief Executive Officer, Erika Gibson, Director of Finance and Administration, and Dr. Michael Rosol, Chief Medical Officer, of Navidea, will host the call and webcast to discuss the financial results and provide an update on recent developments and clinical progress. Management will be available to answer questions live immediately following the earnings announcement part of the call.

To participate in the call and webcast, please refer to the information below:

Event:

Q2 2019 Earnings and Business Update Conference Call

Date:

Thursday, August 8, 2019

Time:

5:00 p.m. (EDT)

U.S. & Canada Dial-in:

877-407-0312

International Dial-in:

+1 201-389-0899

Conference ID:

13693119

Webcast Link: View Source

A live audio webcast of the conference call will also be available on the investor relations page of Navidea’s corporate website at www.navidea.com. In addition, the recorded conference call can be replayed and will be available for 90 days following the call on Navidea’s website.

About Navidea

Navidea Biopharmaceuticals, Inc. (NYSE American: NAVB) is a biopharmaceutical company focused on the development of precision immunodiagnostic agents and immunotherapeutics. Navidea is developing multiple precision-targeted products based on its Manocept platform to enhance patient care by identifying the sites and pathways of disease and enable better diagnostic accuracy, clinical decision-making, and targeted treatment. Navidea’s