On August 1, 2018 Atara Biotherapeutics, Inc. (Nasdaq:ATRA), a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune and viral diseases reported financial results for the second quarter of 2018 and recent operational highlights (Press release, Atara Biotherapeutics, AUG 1, 2018, View Source [SID1234528632]).

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"The future of T-cell immunotherapy is both off-the-shelf and across multiple therapeutic areas," said Isaac Ciechanover, M.D., Chief Executive Officer and President of Atara Biotherapeutics. "During the second quarter, we continued to advance our robust T-cell immunotherapy pipeline, highlighted by our ongoing Phase 3 studies of tab-cel in patients with EBV+ PTLD and Phase 1 study of ATA188 in patients with progressive multiple sclerosis. In parallel, we continue to build Atara’s global commercial and operational capabilities in anticipation of the first tab-cel Phase 3 results and submission of an EU conditional marketing authorization application in the first half of 2019. We are also preparing to expand our pipeline with the development of the next generation of chimeric antigen receptor T cell (CAR T) technologies. This is an exciting time for Atara as we enter the next phase of the Company’s growth as a leader in off-the-shelf, allogeneic T-cell immunotherapy."

Recent Highlights and Anticipated Upcoming Milestones

Tab-cel (tabelecleucel)

Two Phase 3 clinical studies are ongoing (MATCH and ALLELE) to evaluate tab-cel (tabelecleucel) in patients with Epstein-Barr virus associated post-transplant lymphoproliferative disorder (EBV+ PTLD) who have failed rituximab following hematopoietic cell transplant (HCT) or solid organ transplant (SOT).
11 clinical sites for the MATCH and 13 for the ALLELE studies are now open for enrollment in the U.S. with additional sites expected to open in the U.S. and other geographies.

Presented positive long-term outcomes including durable remissions and encouraging safety findings from two Phase 2 studies of tab-cel in EBV+ PTLD at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper).
One- and three-year overall survival (OS) for tab-cel treated patients with EBV+ PTLD following HCT who failed rituximab (n=35) was 68% and 55%, respectively. Median OS was not reached after a median of 23.3 months of follow-up in this patient group.
In patients with EBV+ PTLD following SOT who failed rituximab, the one- and three-year OS after treatment with tab-cel (n=14) was 64% and 43%, respectively. Median survival in this patient group was 21.3 months.
None of the EBV+ PTLD patients who had complete or partial responses (CR or PR) after treatment with tab-cel died of EBV+ PTLD. Two-year OS for these responding patients was 83% and 86% following HCT (n=24) and SOT (n=7), respectively.
Tab-cel was associated with durable objective response rate (CR plus PR) of 69% and 50% in patients with EBV+ PTLD following HCT and SOT, respectively, who have failed rituximab.

U.S. Food and Drug Administration (FDA) accepted IND to initiate a Phase 1/2 clinical study of tab-cel in combination with Merck’s anti-PD-1 (programmed death receptor-1) therapy, KEYTRUDA (pembrolizumab), in patients with platinum-resistant or recurrent EBV-associated nasopharyngeal carcinoma (NPC) that Atara plans to initiate in the second half of 2018.

Expect to present updated tab-cel results in patients with EBV+ cancers in the second half of 2018.
ATA188 & ATA190 for Multiple Sclerosis (MS)

Announced publication of new research findings advancing the understanding of Epstein-Barr Virus (EBV) infection in the MS-affected brain.
The findings were reported in an article online and published in the July 2018 print issue of Neurology: Neuroimmunology & Neuroinflammation, an official journal of the American Academy of Neurology.

A Phase 1 clinical study to evaluate off-the-shelf, allogeneic ATA188 in patients with progressive MS is also underway across clinical sites in the U.S. and Australia.
The primary objective of the Phase 1 study is to assess the safety of ATA188 in patients followed for at least one year after the first dose. Key secondary endpoints in the study include measures of clinical improvement such as expanded disability status scale (EDSS) and annualized relapse rate (ARR), as well as MRI imaging.
The first results from the ongoing ATA188 Phase 1 study in patients with progressive MS are expected in the first half of 2019.
Plan to initiate a randomized autologous ATA190 study in progressive MS patients in 2019.
Development Pipeline

Plan to rapidly advance novel gene-edited CAR T development programs from recently expanded T-cell immunotherapy collaboration with Memorial Sloan Kettering Cancer Center (MSK), leveraging our existing off-the-shelf T-cell immunotherapy technology platform, manufacturing expertise and research and development capabilities.
Expect to start Phase 1 study for ATA621 targeting both JC and BK viruses in 2019.
Corporate

Commenced operations at Atara T Cell Operations & Manufacturing (ATOM) facility in the second quarter of 2018, with completion to support clinical production expected in 2019.
Appointed Utpal Koppikar as Chief Financial Officer. Utpal has an accomplished track record in global biotechnology financial operations.
In June 2018 we exercised our option under a license agreement with QIMR Berghofer to an exclusive, worldwide license to develop and commercialize additional T-cell immunotherapy programs including ATA190, as well as the option to license additional technology.
Second Quarter 2018 Financial Results

Cash, cash equivalents and short-term investments as of June 30, 2018 totaled $417.0 million, which we believe will enable us to expand our near-term pipeline and accelerate pre-commercial activities as well as fund our previously planned operations to mid-2020. In the second quarter of 2018, we sold approximately 1.0 million shares of common stock pursuant to our "at-the-market" (ATM) facility for net proceeds of $47.6 million, after deducting commissions and other offering expenses.
We reported net losses of $50.9 million, or $1.15 per share, for the second quarter of 2018, as compared to $27.4 million, or $0.94 per share, for the same period in 2017.
Research and development expenses were $33.4 million for the second quarter of 2018, as compared to $18.3 million for the same period in 2017. The increase in the second quarter of 2018 was due to costs associated with our continuing expansion of research and development activities, including:
clinical trial, manufacturing and outside service costs related to the two Phase 3 clinical trials of tab-cel in patients with EBV+ PTLD and the Phase 1 clinical trial of allogeneic ATA188 in patients with MS;
higher payroll and related costs from increased headcount, and
an increase in allocated facilities and information technology expenses.
Research and development expenses include $3.4 million and $2.0 million of non-cash stock-based compensation expenses in the second quarters of 2018 and 2017, respectively.
General and administrative expenses were $19.2 million for the second quarter of 2018, as compared to $9.6 million for the same period in 2017. The increase in the second quarter of 2018 was primarily due to increases in professional services costs and payroll and related costs driven by increased headcount to support the Company’s expanding operations. General and administrative expenses include $4.6 million and $3.7 million of non-cash stock-based compensation expenses in the second quarters of 2018 and 2017, respectively.

On August 1, 2018 VBL Therapeutics (Nasdaq:VBLT), a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class treatments for cancer, reported that it will host a conference call and live audio webcast on Thursday, August 16 at 8:30am Eastern Time to report second quarter ended June 30, 2018 financial results (Press release, VBL Therapeutics, AUG 1, 2018, View Source [SID1234528290]).

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Thursday, August 16th @ 8:30am Eastern Time
US Domestic: 877-222-6394
International: 703-925-2702
Conference ID: 3572709
Webcast: View Source

Replays, Available through August 30, 2018
US Domestic: 855-859-2056
International: 404-537-3406
Conference ID: 3572709

On August 1, 2018 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology, reported its financial results and operational progress for the second quarter of 2018 (Press release, Intellia Therapeutics, AUG 1, 2018, View Source [SID1234528392]).

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"In the first half of 2018, we focused on executing against our priorities, including initiating IND-enabling studies for our lead in vivo liver program, demonstrating that our lipid nanoparticle delivery platform has broad utility, and advancing our ex vivo R&D efforts across our key collaborations. We are excited about our progress, and we remain focused on achieving our 2018 goals," said Intellia President and Chief Executive Officer John Leonard, M.D.

Second Quarter 2018 Operational Highlights

The Company achieved several key operational milestones during the second quarter of 2018, including the following:

Intellia continued progress in non-human primate (NHP) dose-ranging studies that support the Company’s lead in vivo program in transthyretin amyloidosis (ATTR).

At the 21st Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) in Chicago, Intellia and research collaborator, Ospedale San Raffaele, announced the identification of T cell receptors (TCRs) targeting a Wilms’ Tumor 1 protein (WT1) epitope, important for the treatment of acute myeloid leukemia as Intellia’s first ex vivo program. TCRs that target WT1 may also have applicability in solid tumors and other

hematological malignancies, as WT1 is over-expressed on many tumor types. The Company plans to develop these TCRs as part of its first ex vivo product candidate. Intellia anticipates that this program will benefit from ongoing work to develop a truly allogeneic approach for engineered cellular therapy.

In the second quarter, Intellia progressed ex vivo multiplexing efforts to achieve triple knockout edits with greater than 80 percent efficiency in human cells, and observed insertion efficacy of ~50 percent with simultaneous double knockout edits. Capabilities in multiplexing will support and advance ex vivo efforts within the Company’s expanding engineered cell therapy pipeline.

Expanding on the in vivo liver editing achievements announced earlier this year, the Company progressed its primary hyperoxaluria type I (PH1) program utilizing a phenotypic mouse model of the disease. In PH1, excess oxalate produced in the liver crystallizes and accumulates in various organs eventually causing kidney failure. A knockout of the HAO1 gene reduces levels of glyoxylate, a precursor to urinary oxalate, and thereby reduces oxalate accumulation. In a mouse model of the disease, the Company achieved 74 percent editing of HAO1 leading to a ~90 percent protein reduction and a ~55 percent reduction in urinary oxalate after a single dose. This progress reinforces the value and speed of Intellia’s modular lipid nanoparticle delivery platform, including efficient and effective delivery to hepatocytes in the liver.

Intellia continued to expand its fully automated, next-generation sequencing and bioinformatics platform to support the identification of highly active guides with limited to no off-target cutting or unforeseen deletions. In the second quarter, Intellia increased throughput capacity to process greater than 30,000 sequencing samples per week. This capability enables measuring of on- and off-target genome editing, including indels, translocations, excisions and inversions.

Intellia, along with other licensees, announced in June that the U.S. Patent and Trademark Office (USPTO) granted U.S. Patent No. 10,000,772 ("the ’772 patent") to The Regents of the University of California, the University of Vienna and Emmanuelle Charpentier, Ph.D. (collectively, "UC"), co-owners of foundational intellectual property relating to CRISPR/Cas9 genome editing technology. The ’772 patent covers the use of single- and dual-guide RNA formats having certain structural motifs in a region that interacts with the Cas9 enzyme. These guide RNA formats are widely used in the CRISPR/Cas9 field. Intellia anticipates this is the first of many patents to be granted in the U.S. to UC for the CRISPR/Cas9 genome editing intellectual property. Outside of the U.S., UC continues to hold a strong global intellectual property position with applications from this patent estate issued in Europe, the United Kingdom, China, Japan, Australia and various other countries worldwide. The ’772 patent is not involved in the appeal to the U.S. Court of Appeals for the Federal Circuit (CAFC) relating to the February 2017 interference decision from the USPTO’s Patent Trial and Appeal Board. UC’s appeal was heard on April 30, 2018 by the CAFC, and a decision is still pending.

Upcoming Milestones

For the remainder of 2018, Intellia’s expected milestones include the following:

Advance a second liver knockout target in NHPs;

Advance candidates for a second liver indication;

Present additional editing data supporting more complex edits such as insertion and repair;

Prepare for a pre-Investigational New Drug meeting with the U.S. Food and Drug Administration for ATTR;

Expand preclinical data in support of Intellia’s first proprietary ex vivo autoimmune program; and

Identify Intellia’s first hematopoietic stem cell target from the collaboration with Novartis.

Second Quarter 2018 Financial Results

Collaboration Revenue

Collaboration revenue was $7.7 million for the second quarter of 2018, compared to $5.9 million during the second quarter of 2017. The increase in collaboration revenue in 2018 was primarily driven by amounts recognized under Intellia’s collaboration agreement with Regeneron.

Since inception through June 30, 2018, the Company has received $114.1 million in funding from the collaborations with Novartis and Regeneron, excluding amounts received for equity investments, and had an accounts receivable balance of $8.6 million on June 30, 2018.

Operating Expenses

Research and development expenses increased by $7.9 million to $23.5 million during the second quarter of 2018, compared to $15.6 million during the second quarter of 2017. This increase was driven primarily by the advancement of Intellia’s research programs, research personnel growth to support these programs, as well as the expansion of the development organization, and includes laboratory supplies and research materials such as reagents.

General and administrative expenses increased by $1.4 million to $7.8 million during the second quarter of 2018, compared to $6.4 million during the second quarter of 2017. This increase was driven primarily by increased salary and related headcount-based expenses to support Intellia’s larger research and development organization, public company compliance, and administrative obligations.

The Company’s net loss was $22.2 million for the second quarter of 2018, compared to $15.6 million during the second quarter of 2017.

Cash and cash equivalents at June 30, 2018, were $305.5 million, compared to $241.0 million for second quarter in 2017.

Financial Guidance

The Company’s primary uses of capital will continue to be for research and development programs, laboratory and related supplies, compensation costs for current and future employees, consulting, intellectual property related costs, and general operating costs.

As of June 30, 2018, the Company had an accumulated deficit of $159.3 million. The Company expects losses to increase as it continues to incur significant research and development expenses related to the advancement of Intellia’s therapeutic programs and ongoing operations. Based on Intellia’s research and development plans and expectations related to the progress of the Company’s programs, the Company expects that the cash and cash equivalents as of June 30, 2018, as well as technology access and research funding from Novartis and Regeneron, will enable Intellia to fund operating expenses and capital expenditures through mid-2020, excluding any potential milestone payments or extension fees that could be earned and distributed under the collaboration agreements with Novartis and Regeneron or any strategic use of capital not currently in the base-case planning assumptions.

Upcoming Events During the Third Quarter 2018

The Company expects to make presentations at the following upcoming investor conferences:

B. Riley FBR Health Care Conference, Sept. 4, New York City

Citi Biotech Conference, Sept. 5, Boston

Wells Fargo Health Care Conference, Sept. 6, Boston

Jefferies Gene Therapy Summit, Sept. 27, New York City

On August 1, 2018 Immune Design (Nasdaq: IMDZ), an immunotherapy company focused on next-generation therapies in oncology, reported financial results and a corporate update for the second quarter ended June 30, 2018 (Press release, Immune Design, AUG 1, 2018, View Source [SID1234528291]).

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"We have had a solid year of progress so far at Immune Design, with important advancements with CMB305, and now with G100," said Carlos Paya, M.D., Ph.D., President and Chief Executive Officer of Immune Design. "Based on recent interactions with the FDA, we plan to aggressively advance G100 in follicular lymphoma patients. Simultaneously, the CMB305 pivotal Phase 3 SYNOVATE trial is now open for enrollment in the U.S. to patients with synovial sarcoma. We believe these late-stage programs position us well to bring novel therapies to cancer patients with high unmet need."

Pipeline Highlights

G100: moving forward with an ORR-based study in patients with relapsed follicular lymphoma (FL)

G100 is a novel, synthetic TLR4 agonist for intratumoral therapy
Based on feedback from an End of Phase 1 FDA meeting:
FDA notes that relapsed FL patients who have failed three lines of systemic therapy represents an unmet medical need population; and
FDA agrees that a single-arm trial to evaluate objective response rate (ORR) and duration of response (DOR) is appropriate to assess the efficacy of G100 in combination with pembrolizumab with an adaptive design that allows for an interim analysis. Immune Design is working with the FDA on the details of the study and plans to initiate patient enrollment as soon as feasible after the protocol is finalized.
The company intends to use this open label approach to generate data for a potential biological license application and plans to provide an update on the final study design and associated timeline after the ongoing FDA discussions are complete.
CMB305: the SYNOVATE Phase 3 trial is open for enrollment; combination with atezolizumab Phase 2 ongoing
CMB305 is a novel prime-boost cancer vaccine targeting NY-ESO-1+ cancers in patients with soft tissue sarcoma.
Monotherapy:
SYNOVATE study, a randomized, global Phase 3 trial evaluating CMB305 monotherapy versus placebo in synovial sarcoma patients in a post 1st line therapy maintenance setting is open for enrollment.
Immune Design is working on opening additional clinical sites throughout the United States, followed by expansion into Canada, Europe and the Asia Pacific region.
Combination therapy: the Phase 2 study evaluating the combination of CMB305 with atezolizumab in relapsed refractory soft-tissue sarcoma patients continues follow-up to determine overall survival after achieving an estimated 72 events.
Research Programs
Immune Design will shift resources to focus on later-stage programs, specifically for the development of G100 in relapsed FL and beyond. Consequently, the company is pausing further development of its preclinical programs, CA21 and intratumoral ZVex-IL12.
This allocation of resources enables the company to run the planned G100 study at least to the interim analysis with existing capital.
Upcoming Data Presentation
Immune Design plans to present long-term follow-up data from its CMB305 monotherapy trial in soft tissue sarcoma patients at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Congress in October. The ESMO (Free ESMO Whitepaper) presentation will be in the forms of both a poster and poster discussion session.
Financial Results

Immune Design ended the second quarter of 2018 with $120.3 million in cash and cash equivalents, short-term investments, and other receivables compared to $144.2 million as of December 31, 2017. Net cash used in operations for the six months ended June 30, 2018 was $27.3 million.
Net loss and net loss per share for the second quarter of 2018 were $13.8 million and $0.29, respectively, compared to $13.8 million and $0.54, respectively, for the second quarter of 2017.
Revenue for the second quarter of 2018 was $0.8 million and was primarily attributable to $0.4 million in collaboration revenue associated with the Sanofi G103 HSV2 vaccine collaboration and $0.4 million in product sales to collaboration partners. Revenue for the second quarter of 2017 was $0.7 million and was primarily attributable to collaboration revenue associated with the Sanofi G103 collaboration.
Research and development expenses for the second quarter of 2018 were $11.0 million, compared to $10.9 million for the same period in 2017. The $0.1 million increase in research and development expenses was primarily attributable to an increase in personnel-related expenses and an increase in research and development headcount to support the company’s advancing research and clinical pipeline activities. This increase was offset by a slight decrease of $0.1 million in in-licensing royalties and fees and a $0.1 million decrease in research and development supplies and services.
General and administrative expenses for the second quarter of 2018 were $4.0 million, compared to $3.9 million for the same period in 2017. The $0.1 million increase in general and administrative expenses was primarily attributable to an increase in professional fees and services to help support our ongoing operations, which was offset by a decrease in personnel-related expenses in the form of stock-based compensation expense.
Year-to-Date

Net loss and net loss per share for the six months ended June 30, 2018 were $27.1 million and $0.56, respectively, compared to $26.5 million and $1.04, respectively, for the same period in 2017.
Revenue for the six months ended June 30, 2018 was $1.3 million and was primarily attributable to $0.8 million in collaboration revenue associated with the Sanofi G103 collaboration and $0.4 million in product sales to collaboration partners. Revenue for the same period in 2017 was $6.2 million and was primarily attributable to $5.9 million in collaboration revenue associated with the Sanofi G103 collaboration and $0.3 million in product sales to other third parties.
Research and development expenses for the six months ended June 30, 2018 were $21.3 million compared to $24.9 million for the same period in 2017. The $3.6 million decrease in research and development expenses was primarily attributable to a decrease of $4.8 million in costs related to the timing and nature of certain contract manufacturing activities connected to the Sanofi G103 collaboration. Offsetting this decrease was an increase of $1.1 million in personnel-related expenses, which was primarily due to an increase in compensation and benefits and an increase in research and development headcount.
General and administrative expenses did not materially differ over the comparative periods. For the six months ended June 30, 2018, general and administrative expenses were $8.0 million compared to $8.0 million for the same period in 2017. In February 2018, Immune Design recouped $0.8 million from the TVS settlement, which decrease in expense was offset by an increase of $0.6 million in professional fees and services and $0.2 million in compensation and benefits to support ongoing operations.
Cash Guidance

Based on current expectations, Immune Design expects to have cash to fund operations into the second half of 2020.

Conference Call Information

Immune Design will host a conference call and live audio webcast this afternoon at 1:30 p.m. Pacific Time / 4:30 p.m. Eastern Time to discuss second quarter 2018 financial results and provide a corporate update.

The live call may be accessed by dialing 844-266-9538 for domestic callers and 216-562-0391 for international callers. A live webcast of the call will be available online from the investor relations section of the Immune Design website at View Source and will be archived there for 30 days. A telephone replay of the call will be available for five days by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference code 3376676.

An archived copy of the webcast will be available on Immune Design’s website beginning approximately two hours after the conference call. Immune Design will maintain an archived replay of the webcast on its website for at least 30 days after the conference call.

On August 1, 2018 FibroGen, Inc. (NASDAQ: FGEN), a biopharmaceutical company, reported that it will report second quarter 2018 financial results on Tuesday, August 7, 2018, after market close, and will host a conference call to discuss financial results and provide a business update at 5:00 p.m. ET (2:00 p.m. PT) (Press release, FibroGen, AUG 1, 2018, View Source;p=irol-newsArticle&ID=2361527 [SID1234528292]).

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Conference Call and Audio Webcast
Interested parties may access a live audio webcast of the conference call via the investor section of the FibroGen website, www.fibrogen.com. It is recommended that listeners access the website 15 minutes prior to the start of the call to download and install any necessary audio software. A replay of the webcast will be available shortly after the call for a period of two weeks. To access the replay, please dial (888) 843-7419 (domestic) or (630) 652-3042 (international), and use passcode 47339190#.

Dial-In Information
Live (U.S./Canada): (888) 771-4371
Live (International): (847) 585-4405
Confirmation number: 47339190