On February 25, 2019 Hummingbird Bioscience reported it has been awarded a product development grant totalling $13.1 million from the Cancer Prevention and Research Institute of Texas (CPRIT) (Press release, Hummingbird Bioscience, FEB 25, 2019, View Source [SID1234554020]).

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The grant will support the Phase IA/B clinical trials of the company’s first-in-class anti-VISTA therapeutic antibody for the treatment of VISTA mediated suppression of anti-tumor immunity in solid tumors and lymphomas that are unresponsive to existing therapies. Hummingbird Bioscience is a systems-biology enabled biotech company focused on the discovery and development of novel cancer therapeutics.

The CPRIT review for this grant included an in-depth evaluation of HMBD-002, an antibody that uniquely neutralizes VISTA function, by a panel of scientific, medical, and industry experts. The evaluation included rigorous review of the pre-clinical data, as well as regulatory, research and development, and intellectual property due diligence.

The grant will support the Phase IA/B clinical study and preparatory steps including GMP manufacturing and Investigational New Drug (IND) submission for the HMBD-002 program. The grant will also support the relocation of the company’s U.S. headquarters to Texas. Clinical trials for HMBD-002 are anticipated to begin in 2020. Hummingbird Bioscience was the only successful application of its type for this grant cycle.

The award follows the recent announcement of Nobel Laureate James P. Allison, Ph.D. and Padmanee Sharma, M.D., Ph.D. joining Hummingbird’s Scientific Advisory Board.

"We are delighted to be awarded this highly-selective grant from CPRIT and to start building our operations in Texas," said Piers Ingram, Ph.D., CEO of Hummingbird Bioscience. "Our company applies systems biology approaches to generate uniquely differentiated therapeutic antibodies. We are thrilled CPRIT’s panel of experts agrees that developing this anti-VISTA immuno-oncology program has the potential to significantly benefit patients."

On February 25, 2019 Cellaria, LLC, a scientific innovator that develops revolutionary new models for cancer and other diseases, reported a new distribution partnership with Rockland Immunochemicals, Inc. (Rockland), a life science supplier and manufacturer that specializes in antibodies and antibody-based tools for research applications and assay development (Press release, Rockland, FEB 25, 2019, View Source [SID1234553932]). The agreement gives Rockland the rights to market and sell Cellaria’s high-quality next generation in-vitro disease models and cell culture media worldwide.

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With Rockland, Cellaria adds to its growing list of strategic distribution partners, expanding its global market presence and reach. Cellaria’s novel cell culture media and cancer cell models add to Rockland’s arsenal of products targeting cancer research and drug screening.

Cellaria’s cell models express different biomarkers that are directly correlated to the patient specimens used to create the models. The diversity of biomarker expression within similar categories of patients enables researchers to generate drug response and disease progression data that is highly relevant to those individual patients.

"We allow drug and cancer researchers to make much more informed decisions about which populations of patients are more likely to respond effectively to drugs that are under investigation," said David Deems, CEO of Cellaria. "Rockland’s scientific knowledge and their ability to sell cutting-edge and custom products make them a great fit to represent the innovation that we bring to market."

James Fendrick, CEO of Rockland stated, "Cellaria’s offerings will nicely complement Rockland’s existing line of cell culture products including human melanoma cell lines, fetal bovine serum, lysates and collagens. We are honored that Cellaria chose Rockland to serve as their distribution partner."

On February 25, 2019 ArQule reported the publication of clinical pharmacodynamic, safety and efficacy data in patients with Proteus syndrome (Press release, ArQule, FEB 25, 2019, View Source [SID1234533719]). These data, together with data already presented at ASH (Free ASH Whitepaper)G last year, support miransertib’s further development as a potential first systemic treatment for patients suffering from overgrowth diseases, such as Proteus syndrome. The study, published in the American Journal of Human Genetics, and led by the National Institutes of Health (NIH), demonstrated good target engagement, tolerability and reductions in lesion size and pain, especially in children.

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Highlights from the study include:

Generally well-tolerated safety profile
Reductions in pAKT (activated AKT) in most patients
Reductions in Cerebriform Connective Tissue Nevus (CCTN) lesions in size (measured with standardized photography) but also in firmness, depth of sulci and discomfort (by patient report)
Reduction in pain intensity in all (3 of 3) children in the study

"These data are highly encouraging and support further investigation into the potential use of miransertib for this devastating condition," said Dr. Brian Schwartz, Chief Medical Officer at ArQule. "The reduction in CCTN lesions were particularly striking since these lesions are, in general, relentlessly progressive, and cause severe morbidity, ulcerations and intractable pain. We’d like to thank our academic collaborators and the patients and their families for their support and tremendous dedication to this cause."

The study is available online at: View Source

On February 25, 2019 Cellectis (Euronext Growth: ALCLS; Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on allogeneic gene edited CAR T-cells (UCART), reported the publication of a study in The Journal of Biological Chemistry, identifying Granulocyte Macrophage Colony Stimulating Factor (GMCSF) secreted by Chimeric Antigen Receptor (CAR) T-cells as a key factor promoting cytokine release syndrome (CRS) (Press release, Cellectis, FEB 25, 2019, View Source [SID1234533710]). The accelerated report leverages these findings to elaborate an innovative engineering strategy that paves the way for developing safer UCART products.

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Utilizing these results, Cellectis developed engineered GMCSF Knock-Out CAR T-cells through TALEN-mediated gene inactivation. The inactivation of GMCSF in CAR T-cells was found to prevent secretion of pro-inflammatory cytokines by monocytes, without compromising CAR T-cell anti-tumor activity.

"CAR T-cells have achieved high rates of complete remission in hematological malignancies, however, this ‘living drug’ can show life-threatening inflammatory side effects including CRS and neurotoxicity that need to be addressed," said Mohit Sachdeva, Ph.D., Innovation Project Leader at Cellectis. "Our engineering strategy circumvents such toxic side effects and propose safer, equally potent UCART-cells, to improve patients’ quality of life during treatment."

"Today, tocilizumab or glucocorticoid treatments are considered the standard of care for CRS management," added Julien Valton, Ph.D., Innovation Team Leader at Cellectis. "However, these treatments increase patient medication burden, add substantial costs and lengthen treatment time in intense care settings. To overcome these clinical challenges, we investigated the biogenesis of CRS and based on our findings, developed a CAR T-cell product candidate that could potentially prevent rather than treat CRS symptoms. We hope this approach can bypass CRS symptomatic treatments and improve the overall safety of CAR T-cell therapies for cancer patients."

Julien Valton, Ph.D., Innovation Team Leader, Cellular Engineering & Adoptive CAR T-Cell Immunotherapy

Dr. Julien Valton obtained his Ph.D. at the University Joseph Fourier in Grenoble, France, where he was trained as an enzymologist. He then joined the Yale School of Medicine to apply his knowledge to therapeutic research by investigating the mechanism of inhibition of receptor tyrosine kinases that are involved in the development of gastrointestinal cancer. In 2009, he moved a step further into the field of applied science by joining the Innovation Department of Cellectis, where he actively participated in using and improving TALEN gene editing technology for targeted gene therapy and genome engineering. He is now using TALEN along with protein engineering techniques to develop the next-generation CAR T-cells to treat different malignancies.

Mohit Sachdeva, Ph.D., Innovation Team Senior Scientist

Dr. Sachdeva is an experienced cancer biologist with expertise in immuno-oncology, having authored/co-authored approximately 20 manuscripts in peer-reviewed journals throughout his career. During his time at Cellectis, he has been studying pathways that could be exploited to engineer potent, yet safer, CAR T-cells using gene editing and targeted integration technologies. After receiving his Ph.D. at Southern Illinois University, he completed a successful post-doc at Duke University.

Granulocyte-macrophage colony-stimulating factor inactivation in CAR T-Cells prevents monocyte-dependent release of key cytokine release syndrome mediators

Mohit Sachdeva1*, Philippe Duchateau2, Stéphane Depil2, Laurent Poirot2 and Julien Valton1*

1Cellectis, Inc., 430 East 29th Street, New York, NY 10016, USA

2Cellectis, 8 rue de la Croix Jarry, 75013 Paris, France

On February 25, 2019 Immunomedics, Inc., (NASDAQ: IMMU) ("Immunomedics" or the "Company"), a leading biopharmaceutical company in the area of antibody-drug conjugates (ADC), reported financial results for the Transition Period ended December 31, 2018 (Press release, Immunomedics, FEB 25, 2019, View Source [SID1234533708]). The Company also announced that Dr. Charles Baum, President and Chief Executive Officer (CEO) of Mirati Therapeutics, Inc. has joined the Immunomedics Board of Directors (Board). In addition, the Board has appointed Behzad Aghazadeh, Executive Chairman, while Scott Canute, current member of the Board, has been appointed to Executive Director. At the same time, Michael Pehl has stepped down from the role of President, CEO and member of the Board, due to personal reasons, and interim Chief Financial Officer (CFO), Usama Malik, has been appointed CFO. Please refer to the Company’s Transition Report on Form 10-K filed today with the SEC for more details on the Company’s financial results.

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"I would like to thank Michael for his leadership and service to Immunomedics over the past fifteen months. We wish him all the best as he returns to Germany. At the same time, we are honored to welcome Chuck to our Board. Chuck has a storied career in drug development culminating in his role as President and CEO at Mirati, while also serving on the Board of Array Biopharma. Chuck will bring a strong scientific, drug development, and executive management perspective that will be invaluable to Immunomedics as we continue to execute and expand on our development priorities," said Behzad Aghazadeh, Executive Chairman of Immunomedics.

"Our primary focus is to ensure a high-quality Biologics License Application (BLA) resubmission and gain FDA approval of sacituzumab govitecan for patients with metastatic triple-negative breast cancer (mTNBC). To that end, we have implemented a dedicated Complete Response Letter (CRL) team, led by our Executive Board member, Scott Canute, a recognized expert in Manufacturing and Quality spanning decades in the biopharmaceutical industry," Behzad further commented.

"We are committed to working closely with the FDA to resolve the CMC issues identified in the CRL. Based on various prior experiences with much more complex manufacturing challenges at Eli Lilly and Genzyme, I am confident that our expert teams will drive a high-quality and timely resubmission of sacituzumab govitecan," said Scott Canute, Executive Director of Immunomedics.

The CRL team is finalizing a response strategy to the issues identified in the CRL in advance of a meeting request with the FDA and anticipates providing additional clarity on the BLA resubmission timelines after such meeting has occurred.

"Sacituzumab govitecan has demonstrated a significant clinical benefit in multiple hard-to-treat cancer settings", commented Dr. Baum. "I am thrilled to join the Board of Immunomedics and leverage my experience to help navigate the near-term resubmission of the BLA for mTNBC, and shape the clinical development strategy in other late-stage indications," he added.

Dr. Baum is President, CEO and Board Member of Mirati Therapeutics since November 2012. Prior to joining Mirati, he was Senior Vice President for Biotherapeutic Clinical Research at Pfizer and served as the Head of Oncology Development and as Chief Medical Officer for Pfizer’s Biotherapeutics and Bioinnovation Center. He was responsible for the development of the oncology portfolio, including Axitinib (Inlyta), Crizotinib (Xalkori) and the approval of sunitinib (Sutent) for the treatment of gastrointestinal stromal tumor (GIST) and renal cell carcinoma. Previously, Dr. Baum was responsible for the development of several oncology compounds at Schering-Plough, including temozolomide (Temodar) which was approved for the treatment of patients with advanced brain tumors. Dr. Baum currently serves on the Board of Array BioPharma.

Mr. Scott Canute has more than 36 years of experience in the biopharmaceutical industry, previously serving as President of Global Manufacturing and Corporate Operations of Genzyme Corporation from 2010 until 2011, where he led a major turnaround in manufacturing in order to ensure on-going supply of life saving products that were in short supply and developed and implemented a comprehensive manufacturing strategy, including a revamped global governance system. Prior to joining Genzyme, Mr. Canute spent 25 years at Eli Lilly and Company, where he served as President, Global Manufacturing Operations from 2004 until 2007. During his tenure at Eli Lilly, Mr. Canute directed all manufacturing and supply chain activities for the company’s global operations, which spanned 24 international manufacturing sites and 80+ contract manufacturing operations.

Recent Company Highlights

·The Company received a CRL from the FDA for the BLA seeking accelerated approval of sacituzumab govitecan for the treatment of patients with mTNBC who have received at least two prior therapies for metastatic disease.

·The Company published in the New England Journal of Medicine updated data with sacituzumab govitecan in patients with mTNBC, showing a favorable benefit:risk profile of the ADC, as well as providing additional and consistent safety information on 420 patients with a variety of epithelial cancers.

· In an oral presentation at the 2019 Genitourinary Cancers Symposium, the Company reported updated results from a Phase 1/2 study of sacituzumab govitecan in patients with previously treated metastatic urothelial cancer, showing an overall response rate of 31 percent and a median duration of response of 12.9 months in 45 relapsed/refractory patients.

·Enrollment into the pivotal TROPHY U-01 study continues to progress well, while the Company prepares for the initiation of the registrational Phase 3 study in hormone receptor-positive metastatic breast cancer, as well as the Trop-2-enriched basket study.

· The Company has strengthened its manufacturing partnerships with Johnson Matthey and BSP to further enhance the capacity and scale of its supply chain for sacituzumab govitecan in preparation for future demand driven by additional indications and broadening of geographic footprint.

Results for the Transition Period Ended December 31, 2018

On December 14, 2018, the Company’s Board of Directors approved a change in the Company’s fiscal year end from June 30 to December 31, effective immediately. The reporting period for the six months ended December 31, 2018 is referred to as the "Transition Period."

The Company had no revenues in the Transition Period due primarily to the discontinued sale of LeukoScan during the quarter ended March 31, 2018 in order for the Company to focus on its ADC business. Revenues in the comparable period ended December 31, 2017 were approximately $1.3 million.

Total costs and expenses were $144.5 million for the Transition Period, compared to $52.3 million for the comparable period ended December 31, 2017, due primarily to a $51.1 million increase in research and development expenses, a $23.3 million increase in general and administrative expenses, and a $18.4 million increase in sales and marketing expenses. Most of these increases were attributable to activities related to preparations for the potential approval and commercial launch of sacituzumab govitecan for patients with at least two prior lines of treatment for metastatic TNBC in the United States, and to expanded clinical development of sacituzumab govitecan into other indications.

The Company recognized a $1.4 million non-cash income for the Transition Period, compared to a $59.6 million non-cash expense for the comparable period ended December 31, 2017, due to the net appreciation in the fair value of outstanding warrants and the exercise of warrants. There were no warrants outstanding as of December 31, 2018.

Interest expense was $20.0 million for the Transition Period, compared to $2.9 million for the comparable period ended December 31, 2017. The increase was due primarily to increased debt balances as a result of the agreement with RPI Finance Trust.

Net loss attributable to stockholders was $157.7 million, or $0.84 per share, for the Transition Period, compared to $121.3 million, or $0.88 per share, for the comparable period ended December 31, 2017.

As of December 31, 2018, the Company had $497.8 million in cash, cash equivalents, and marketable securities, which it believes is adequate to support its clinical development plan for sacituzumab govitecan; further build its clinical and manufacturing infrastructure and fund its operations through 2020.

Conference Call

The Company will host a conference call and live audio webcast today at 5:00 p.m. Eastern Time to discuss financial results for the quarter and six months ended December 31, 2018 and provide a corporate update. To access the conference call, please dial (877) 303-2523 or (253) 237-1755 using the Conference ID 6478246. The conference call will be webcast via the Investors page on the Company’s website at View Source Approximately two hours following the live event, a webcast replay of the conference call will be available on the Company’s website for approximately 30 days.