On May 14, 2019 Precision BioSciences (Nasdaq: DTIL), a genome editing company dedicated to improving life through the application of its proprietary ARCUS genome editing platform, reported that Christopher R. Heery, M.D. has joined Precision BioSciences as its Chief Medical Officer (Press release, Precision Biosciences, MAY 14, 2019, View Source [SID1234536273]).

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"We are thrilled to welcome Chris Heery as our Chief Medical Officer. Chris has exceptional experience in the rapidly evolving field of cell therapies and has led many first-in-human clinical trials during his time at the National Cancer Institute (NCI). We believe his skill set and experience are a perfect fit to guide our off-the-shelf immunotherapy and in vivo gene correction programs through human clinical trials," said Matt Kane, Chief Executive Officer and co-founder of Precision BioSciences.

Chief Development Officer, David Thomson, commented, "Chris brings a highly unique and sought-after skill set to Precision BioSciences. He has been a translational clinical trialist at one of the world’s preeminent research institutions, NCI, where his first-in-human immunotherapy clinical trials included a Phase 1 study of an anti-PD-L1 treatment (avelumab) that is now approved for use in the United States. Chris’s most recent experience as Chief Medical Officer at Bavarian Nordic has given him a strong understanding of regulatory requirements, clinical trial oversight, and leadership that will allow him to play a key role in helping Precision BioSciences build a successful clinical organization. We look forward to advancing our clinical pipeline under Chris’s leadership."

Prior to joining Precision BioSciences, Dr. Heery most recently served as Chief Medical Officer of Bavarian Nordic, where he oversaw clinical development programs for its immune-oncology and infectious diseases portfolios. Prior to this, he was Head of the Clinical Trials Group of the Laboratory of Tumor Immunology and Biology at NCI. He joined the NCI Medical Oncology Branch as a Medical Oncology Fellow in 2009 and also served as an Adjunct Appointment in the Genitourinary Malignancies Branch. He was part of the larger effort of the Laboratory of Tumor Immunology and Biology to create new immunotherapies for the treatment of cancer. Dr. Heery is board certified in Medical Oncology and Internal Medicine and received his M.D. from East Carolina University Brody School of Medicine. He completed his internal medicine residency at the University of Illinois at Chicago. Dr. Heery completed his undergraduate studies at Duke University, where he received a BA in English Literature.

About Precision BioSciences

On May 14, 2019 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported its financial results for the first quarter ended March 31, 2019 (Press release, Curis, MAY 14, 2019, View Source [SID1234536294]).

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"2019 has been a transformational year for Curis as we demonstrate the results of our heightened focus on clinical execution. We are currently on track or ahead of schedule in the execution of all three of our clinical trials," said James Dentzer, President and Chief Executive Officer of Curis. "We look forward to discussing clinical data from all three trials later this year: this summer for CA-4948 and later in the second half for fimepinostat and CA-170."

"Furthermore, with this quarter’s agreement with Oberland Capital, for $65 million upfront and up to $70.7 million in additional milestones, we secured the financial flexibility needed to ensure we can continue to move forward aggressively in our clinical execution of all three programs," he concluded.

First Quarter 2019 Financial Results

Curis reported a net loss of $9.9 million, or $0.30 per share on both a basic and diluted basis for the first quarter of 2019, as compared to a net loss of $10.7 million, or $0.33 per share on both a basic and diluted basis for the same period in 2018.

Revenues for the first quarter of 2019 were $1.8 million, as compared to $2.5 million for the same period in 2018. Revenues for both periods comprise primarily royalty revenues recorded on Genentech and Roche’s net sales of Erivedge.

Operating expenses were $7.3 million for the first quarter of 2019, as compared to $12.4 million for the same period in 2018, and comprised the following:

Costs of Royalty Revenues. Costs of royalty revenues, primarily amounts due to third-party university patent licensors in connection with Genentech and Roche’s Erivedge net sales, were $0.1 million for both the first quarter of 2019 and 2018.

Research and Development Expenses. Research and development expenses were $4.1 million for the first quarter of 2019 as compared to $8.3 million for the same period in 2018. The decrease was primarily due to decreased costs related to ongoing clinical and manufacturing activities for fimepinostat and CA-170. Employee-related expenses decreased from the prior quarter primarily due to a reduction in headcount that occurred in the fourth quarter of 2018. These changes reflect our shift in focus toward clinical development, while down-sizing the in-house discovery research organization. We have also focused the CA-170 program toward the VISTA-expressing mesothelioma indication, which allowed substantial reduction in the number of sites and regions required.

General and Administrative Expenses. General and administrative expenses were $3.1 million for the first quarter of 2019 as compared to $4.0 million for the same period in 2018. The decrease in general and administrative expenses was driven primarily by lower personnel, legal, and stock-based compensation for the period.

Other expense, net was $4.3 million for the first quarter of 2019, as compared to $0.8 million for the same period in 2018. Other expense, net primarily consisted of the loss on extinguishment of $3.5 million and interest expense of $0.8 million related to Curis Royalty’s (a wholly owned subsidiary of Curis) debt obligations.

As of March 31, 2019, Curis’s cash, cash equivalents and investments totaled $42.8 million and there were approximately 33.2 million shares of common stock outstanding.

Recent Operational Highlights

Precision oncology, fimepinostat (HDAC/PI3K inhibitor):

Curis is initiating a study of fimepinostat (a MYC suppressor) with venetoclax (a BCL-2 inhibitor) combination regimen in diffuse large B-cell lymphoma (DLBCL), including patients with Double-Hit/Double-Expressor Lymphoma. In preclinical models, fimepinostat administered in combination with venetoclax resulted in an enhanced benefit relative to each agent alone.

Precision oncology, CA-4948 (IRAK4 Inhibitor; Aurigene collaboration):

In April 2019, Curis advanced to the 200mg BID cohort in the CA-4948 study for treatment of patients with non-Hodgkin lymphoma, including those with MYD88 alterations.

Immuno-oncology, CA-170 (VISTA / PDL1 antagonist; Aurigene collaboration):

In January 2019, Curis dosed the first mesothelioma patient in its ongoing Phase 1 CA-170 trial following evidence supporting high levels of VISTA expression in mesothelioma tumor samples. Recent publications have identified VISTA as a possible resistance mechanism to treatment with anti-PD1 antibodies in several cancer indications.

In May 2019, Curis announced completion of its target enrollment of mesothelioma patients in the ongoing Phase 1 CA-170 trial.

First Quarter 2019 and Recent Corporate Highlights

In March 2019, Curis announced that it sold a portion of its Erivedge royalties to Oberland Capital for up to $135.7 million, including $65 million upfront.

2019 Data Catalysts

For the remainder of the year, Curis expects to:

Report initial data on the combination of fimepinostat and venetoclax regimen in patients with R/R DLBCL, including patients with DH/DE Lymphoma, in the second half of 2019.

Report initial efficacy data from its CA-4948 dose escalation study in patients with NHL in mid-year 2019.

Report initial efficacy data from its CA-170 Phase 1 trial in patients with mesothelioma (high VISTA expressors) in the second half of 2019.

Conference Call Information

Curis management will host a conference call today, May 14, 2019, at 4:30 p.m. EDT, to discuss these financial results, as well as provide a corporate update.

To access the live conference call, please dial 1-888-346-6389 from the United States or 1-412 317-5252 from other locations, shortly before 4:30 p.m. EDT. The conference call can also be accessed on the Curis website at www.curis.com in the Investors section.

On May 13, 2019 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for CLR 131 in fourth line or later relapse/refractory multiple myeloma (Press release, Cellectar Biosciences, MAY 13, 2019, View Source [SID1234536216]). CLR 131 is the company’s small-molecule radiotherapeutic phospholipid drug conjugate (PDC) designed to deliver cytotoxic radiation directly and selectively to cancer cells and cancer stem cells. It is currently being evaluated in Cellectar’s ongoing CLOVER-1 Phase 2 clinical study in patients with relapsed or refractory multiple myeloma and other select B-Cell lymphomas.

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"Fast Track Designation furthers our efforts to expeditiously develop CLR 131 as a new, innovative therapy for patients with relapse/refractory multiple myeloma", said James Caruso, president and CEO of Cellectar. "Patients with third line or later relapsed/refractory multiple myeloma have a poor prognosis and low rates of survival as a result of limited effective treatment options. Based on data in the initial patient cohort from our ongoing CLOVER-1 trial where patients showed a 30% response rate after receiving a single 25.0 mCi/m2 dose as a seventh line of therapy on average, we are optimistic that CLR 131 has the potential to provide a meaningful treatment option for these patients."

Fast Track Designation

Fast Track Designation is granted to drugs being developed for the treatment of serious or life-threatening diseases or conditions where there is an unmet medical need. The purpose of the Fast Track Designation provision is to help facilitate development and expedite the review of drugs to treat serious and life-threatening conditions.

Sponsors of drugs that receive Fast Track Designation have the opportunity for more frequent interactions with the FDA review team throughout the development program. These interactions may include meetings to discuss study design, data required to support approval, or other aspects of the clinical program. Additionally, products that have been granted Fast Track Designation may be eligible for priority review of a New Drug Application (NDA) and the FDA may consider reviewing portions of an NDA before the sponsor submits the complete application (Rolling Review).

About the Phase 2 CLOVER-1 Trial

CLOVER-1 is a Phase 2 study of CLR 131 being conducted in approximately 10 leading cancer centers in the United States in patients with relapsed or refractory B-cell hematologic cancers. The hematologic cancers being studied in the trial include multiple myeloma (MM), chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), lymphoplasmacytic lymphoma (LPL), marginal zone lymphoma (MZL), mantle cell lymphoma (MCL), and diffuse large B-cell lymphoma (DLBCL).

The study’s primary endpoint is clinical benefit response (CBR), with additional endpoints of overall response rate (ORR), progression free survival (PFS), median overall survival (OS) and other markers of efficacy following a fractionated dose of 15.625 mCi/m2 dose of CLR 131 administered on day 1 and day 8, with the option for a second dose cycle approximately 75-180 days later.

In addition to receiving the two fractionated doses of CLR 131, MM patients will receive 40 mg oral dexamethasone weekly for up to 12 weeks. Efficacy responses will be determined by the latest International Multiple Myeloma Working Group criteria. Efficacy for all lymphoma patients will be determined according to Lugano criteria. Cellectar was awarded approximately $2 million in non-dilutive grant funding from the National Cancer Institute to help fund the trial. More information about the trial, including eligibility requirements, can be found at www.clinicaltrials.gov, reference NCT02952508.

About CLR 131

CLR 131 is a small-molecule, cancer-targeting radiotherapeutic PDC designed to deliver cytotoxic radiation directly and selectively to cancer cells and cancer stem cells. CLR 131 is the company’s lead therapeutic PDC product candidate and is currently being evaluated in both Phase 2 and Phase 1 clinical studies. In December 2014, the FDA granted orphan drug designation for CLR 131 for the treatment of multiple myeloma. In 2018, the FDA granted orphan drug and rare pediatric disease designations for CLR 131 for the treatment of neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. In addition to the ongoing Phase 1 dose-escalation study and the Phase 2 CLOVER-1 trial, the company recently initiated a Phase 1 open-label, dose-escalating study in pediatric solid tumors and lymphoma to evaluate the safety and tolerability of a single intravenous administration of CLR 131 in up to 30 children and adolescents with cancers including neuroblastoma, sarcomas, lymphomas (including Hodgkin’s lymphoma) and malignant brain tumors.

On May 13, 2019 GRAIL, Inc., a healthcare company whose mission is to detect cancer early, when it can be cured, reported that its multi-cancer test has been granted Breakthrough Device designation from the U.S. Food and Drug Administration (FDA) (Press release, Grail, MAY 13, 2019, View Source [SID1234536233]). The investigational blood test is in development for the early detection of multiple cancer types in individuals aged 50 or older. The FDA grants Breakthrough designation to devices that have the potential to provide for more effective diagnosis of life-threatening diseases such as cancer. The goal of the FDA’s Breakthrough Devices Program is to provide patients and healthcare providers with timely access to medical devices granted the designation by speeding up their development, assessment, and review.

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"We’re excited the FDA recognizes the potential of our multi-cancer early detection blood test. There are no effective early detection tests for the majority of cancer types, and many deadly cancers are often detected too late. We hope our test may offer a chance to address these challenges," said Jennifer Cook, Chief Executive Officer. "We have made significant progress developing our multi-cancer test and look forward to sharing new data at ASCO (Free ASCO Whitepaper) and other medical conferences this year."

GRAIL previously reported data from the first pre-planned sub-study of its Circulating Cell-free Genome Atlas (CCGA) study, which showed that its three prototype next-generation sequencing (NGS) blood tests were able to detect multiple deadly cancer types from a single blood draw, with a low rate of false positive results (high specificity).1 The company has since selected methylation as its preferred approach and has developed a methylation sequencing blood test that preferentially targets the most informative regions of the genome to both detect the presence of multiple types of cancer and identify the tissue of origin (the part of the body where the cancer originated). The blood test is currently being evaluated in the second pre-planned sub-study of CCGA.

New results from CCGA will be presented at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, including data on the ability of the company’s methylation technology to identify the tissue of origin when cancer is present. An analysis of survival of participants whose cancer was detected by the methylation technology, compared with those whose cancer was not detected by the technology, will also be presented.

About GRAIL’s Clinical Program

GRAIL is conducting what the company believes to be one of the largest clinical research programs ever pursued in genomic medicine. The program consists of three large-scale studies designed to enroll approximately 165,000 participants to create an atlas of genomic cancer signals in the blood, and to develop and evaluate GRAIL’s blood test for the early detection of multiple cancer types. Approximately 115,000 participants have been enrolled to date.

The CCGA study is a prospective, observational, longitudinal study that has completed enrollment of approximately 15,000 people with and without cancer across 142 sites in the United States and Canada. GRAIL is conducting three pre-planned sub-studies within CCGA to discover, train, and validate its multi-cancer early detection test.

The STRIVE study is a prospective, observational, longitudinal cohort study that has completed enrollment of approximately 100,000 women at the time of their screening mammogram across 37 sites in the United States. STRIVE is designed for clinical validation of GRAIL’s multi-cancer test in an intended use population. GRAIL anticipates reporting data from STRIVE in 2020.

The SUMMIT study is a prospective, observational, longitudinal cohort study that is enrolling participants in London in the United Kingdom. The study is designed to enroll approximately 50,000 men and women who do not have a cancer diagnosis at the time of enrollment. Approximately half of the participants will be people at high risk of lung and other cancers due to a significant smoking history, and the other half will be people who are not at high risk for cancer based on smoking history. SUMMIT is designed for clinical validation of GRAIL’s multi-cancer test in a second intended use population and to evaluate clinical utility of the test in a high-risk population.

About GRAIL’s Methylation Technology

GRAIL is developing an NGS blood test for the early detection of multiple deadly cancer types. GRAIL’s methylation technology preferentially targets the most informative regions of the genome and uses machine-learning algorithms to both detect the presence of cancer and identify the tumor’s tissue of origin when cancer is present.

DNA methylation is a natural process used by cells to regulate gene expression. It is a chemical modification to DNA and a well-studied epigenomic feature of the genome. In cancer, abnormal methylation patterns and the resulting changes in gene expression can contribute to tumor growth. For example, hypermethylation can cause tumor-suppressor genes to be inactivated.

On May 13, 2019 Forty Seven Inc. (NASDAQ:FTSV), a clinical-stage, immuno-oncology company focused on developing therapies to activate macrophages in the fight against cancer, reported financial results for the first quarter ended March 31, 2019 and provided a business Update (Press release, Forty Seven, MAY 13, 2019, View Source [SID1234536217]).

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"Our achievements year-to-date reflect our commitment to maximizing the potential of 5F9 as a novel, first-in-class therapeutic for the treatment of cancer," said Mark McCamish, M.D., Ph.D., President and Chief Executive Officer of Forty Seven, Inc. "Building on the promising results observed in our Phase 1b NHL trial, we recently entered into additional clinical collaborations to evaluate 5F9 as part of two triplet regimens for patients with the most aggressive forms of the disease. With these partnerships, we continue to cost-efficiently explore the full therapeutic potential of 5F9 across multiple treatment modalities. Looking ahead, we are eager to report updated data from our Phase 1b/2 trial of 5F9 plus rituximab in r/r NHL at the EHA (Free EHA Whitepaper) and ICML meetings, as well as new clinical data from our Phase 1b trial of 5F9 in combination with azacitidine in AML and MDS at the ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) medical meetings. These data will provide key insights as we chart our long-term development plan for 5F9.

First Quarter and Recent Business Highlights:

Pipeline:

In May 2019, Forty Seven entered into a collaboration with Acerta Pharma, AstraZeneca’s hematology research and development center of excellence, to evaluate 5F9 in combination with rituximab plus AstraZeneca/Acerta Pharma’s Bruton’s tyrosine kinase (BTK) inhibitor, acalabrutinib (CALQUENCE), in patients with diffuse large B-cell lymphoma (DLBCL). This approach aims to optimize the treatment of DLBCL patients by inhibiting BTK. In B-cells, BTK signaling results in the activation of pathways associated with B-cell proliferation, trafficking, chemotaxis and adhesion, and BTK inhibition is a proven strategy for inducing the regression of B-cell lymphomas.

In April 2019, Forty Seven extended its existing collaboration with Genentech, a member of the Roche Group, to include a third clinical trial evaluating 5F9 in combination with rituximab plus Genentech’s anti-PD-L1 antibody, atezolizumab (TECENTRIQ), in patients with DLBCL. This approach aims to optimize the treatment of DLBCL patients whose tumors are associated with high levels of macrophages expressing PD-L1.

Upcoming Presentations:

Forty Seven plans to present updated data from the Phase 1b/2 trial of 5F9 in combination with rituximab in patients with relapsed or refractory non-Hodgkin’s lymphoma (r/r NHL), including safety, efficacy and duration of response across various dosing cohorts, at the 24th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) and the International Conference on Malignant Lymphoma (ICML) meetings in June 2019.

Forty Seven plans to present safety and initial efficacy data from the Phase 1b trial evaluating 5F9 as a monotherapy and in combination with azacitidine in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and the 24th Congress of the EHA (Free EHA Whitepaper) in June 2019.

Key Milestones:

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

Report data from the Phase 1b trial of 5F9 in combination with avelumab in patients with ovarian cancer in the fourth quarter.

Report data from the Phase 1b trial of 5F9 in combination with cetuximab in patients with colorectal cancer in the fourth quarter.

Report preclinical data and complete investigational new drug (IND)-enabling studies for FSI-174, an anti-cKIT antibody with potential use as a novel conditioning regimen for bone marrow transplantation, in the second half.

Complete IND-enabling studies for FSI-189, an anti-SIRPα antibody with therapeutic potential for cancer and other indications, in the second half.

First Quarter 2019 Financial Results:

Cash Position: As of March 31, 2019, cash, cash equivalents and short-term investments were $113.6 million, as compared to $139.0 million as of December 31, 2018. This decrease reflects cash used to fund operating activities, including approximately $7.7 million in an advance payment for contract manufacturing, in the first quarter of 2019. The company expects that its cash, cash equivalents and short-term investments will fund operating expenses and capital expenditure requirements through the first half of 2020.

R&D Expenses: R&D expenses were $19.1 million for the first quarter of 2019, as compared to $11.2 million for the first quarter of 2018. The increase was primarily due to a $3.7 million increase in third-party costs related to advancing the company’s clinical development programs for 5F9 and associated contract manufacturing costs Additionally, there was an increase of $2.8 million in costs associated with the advancement of the company’s preclinical and discovery programs, and an increase of $1.4 million in personnel-related costs, including stock-based compensation.

G&A Expenses: G&A expenses were $4.6 million for the first quarter of 2019, as compared to $3.8 million for the first quarter of 2018. The increase was primarily due to a $0.9 million increase in personnel-related costs driven by an increase in headcount and a $0.2 million increase in directors and officers insurance expense, partially offset by a $0.4 million decrease in accounting and consulting expenses incurred in connection with operating as a public company.

Net Loss: Net loss was $23.0 million for the first quarter of 2019, or a net loss per share of $0.74, as compared to $14.8 million for the first quarter of 2018, or a net loss per share of $2.24.

Conference Call Information:

Forty Seven will host a live conference call and webcast at 4:30 p.m. ET today to discuss first quarter 2019 financial results and recent business activities. The conference call may be accessed by (866) 953-0780 (domestic) or (630) 652-5854 (international), and by referring to conference ID 4283836. A webcast of the conference call will be available in the Investors section of the Forty Seven website at View Source The archived webcast will be available on Forty Seven’s website approximately two hours after the conference call and will be available for 30 days following the call.