On February 25, 2019 Veracyte, Inc. (Nasdaq: VCYT) reported financial results and business progress for the quarter and full year ended December 31, 2018, and provided financial guidance for 2019 (Press release, Veracyte, FEB 25, 2019, Veracyte Announces Fourth Quarter and Full-Year 2018 Financial Results and Provides 2019 Financial Outlook
[SID1234533639]).

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"We delivered exceptional growth in 2018 and are off to a strong start in 2019," said Bonnie Anderson, Veracyte’s chairman and chief executive officer. "Our 2018 performance was driven by several factors. Our Afirma GSC and Xpression Atlas launch broadened our thyroid offering to inform both diagnosis and treatment decisions, and the first full year of Percepta classifier adoption was strong with the fourth quarter acceleration in growth positioning us for a strong 2019. Clearly, our multi-product sales strategy that is designed to give us the leverage we need to be a profitable enterprise is working."

Anderson added, "In 2018, we also entered into biopharmaceutical collaborations with Loxo Oncology and Johnson & Johnson Innovation and the Johnson & Johnson Lung Cancer Initiative that recognize the value of our novel scientific platform and can help advance our pipeline. As we look to 2019 and beyond, we believe we are well-positioned for continued success as we further increase the number of patients whose lives can be improved by our innovative tests."

Fourth Quarter and Full-Year 2018 Financial Results

For the three- and twelve-month periods ended December 31, 2018, compared to the prior year:

Revenue was $25.8 million and $92.0 million, respectively, an increase of 31% and 28%;
Gross Margin was 66% and 64%, respectively, an increase of 6% and 3%;
Operating Expenses, Excluding Cost of Revenue, were $20.1 million and $81.2 million, respectively, an increase of 12% and 15%;
Net Loss and Comprehensive Loss was ($3.1) million and ($23.0) million, respectively, an improvement of 63% and 26%;
Basic and Diluted Net Loss Per Common Share was ($0.08) and ($0.62), respectively, an improvement of 67% and 32%;
Net Cash Used in Operating Activities was $1.2 million and $13.5 million, respectively, an improvement of 79% and 44%;
Cash Burn1 was $1.7 million and $15.4 million, respectively, an improvement of 73% and 39%; and
Cash and Cash Equivalents were $78.0 million at December 31, 2018.
2018 Full-Year and Recent Business Highlights

Commercial Expansion:

Grew total genomic test volume to 9,154 tests in the fourth quarter of 2018, representing 28% growth over 2017, which resulted in full-year 2018 growth of 22% over 2017, or 31,710 tests.
Transitioned all Afirma customers to the second-generation Afirma Genomic Sequencing Classifier (GSC) platform and launched the Afirma Xpression Atlas to provide a comprehensive solution that informs both thyroid cancer diagnosis and treatment decisions. Notably, 30% of Afirma GSC orders included Xpression Atlas in 2018, ahead of the company’s expectations.
Grew Percepta Bronchial Genomic Classifier volume to nearly 1,550 tests in its first full year of commercialization, with genomic volume accelerating 74% sequentially from the third quarter to the fourth quarter of 2018.
Established 20 leading Early Access Program (EAP) sites across the United States for Envisia in 2018, addressing physician demand for patient access to the classifier which improves idiopathic pulmonary fibrosis (IPF) diagnosis and builds a solid foundation for the company to commercially expand it in 2019.
Biopharmaceutical Collaborations

Executed a long-term strategic collaboration with Johnson & Johnson, LLC and Johnson & Johnson’s Lung Cancer Initiative to advance diagnostics, including a nasal swab test, for early lung cancer detection. Veracyte estimates the combined monetary and non-monetary value of the collaboration to be more than $50 million. The company believes this collaboration expands its addressable lung cancer diagnostic market to a more than $30 billion global opportunity.
Entered into a research collaboration with Loxo Oncology, through which Loxo has access to data from Veracyte’s Afirma Xpression Atlas platform to help in its development of therapies for patients with genetically defined cancers, including thyroid cancer.
Reimbursement Progress:

Received draft Medicare coverage for the Envisia Genomic Classifier through the MolDX program, with a final positive coverage decision expected in early 2019.
Achieved in-network status as a service provider with the last of the major commercial health plans, which Veracyte believes will facilitate coverage and reimbursement for its Percepta and Envisia classifiers.
Evidence Development:

Afirma – Published clinical validation data for the Afirma GSC in JAMA Surgery, demonstrating the next-generation test’s ability to help approximately 70% of patients with indeterminate thyroid nodules avoid unnecessary surgery. Presented 12 Afirma studies at three endocrinology conferences, including real-world data showing that the Afirma GSC is helping even more patients avoid unnecessary surgery than is suggested by the clinical validation study findings.
Percepta – Presented early, interim results at the 2018 CHEST Annual Meeting from the ongoing registry clinical utility study showing the test changed clinical decision-making and reduced invasive procedures at every evaluation time point up to 12 months post-testing.
Envisia – Published a study quantifying and qualifying the challenges in obtaining timely, accurate diagnosis of IPF and other interstitial lung diseases, thus underscoring the clinical need for the Envisia classifier. Presented data at a leading pulmonology conference demonstrating the test’s ability to improve the diagnosis of IPF without the need for surgery.
Financing and Debt Facility:

In July 2018, Veracyte issued and sold 5,750,000 shares of common stock in a registered public offering, including the underwriters’ exercise in full of their option to purchase an additional 750,000 shares, at a price to the public of $10.25 per share. Net proceeds from the offering were approximately $55.0 million.
In January 2019, the company used $12.5 million of cash and cash equivalents to reduce its principal debt balance from $25.0 million to $12.5 million.
2019 Outlook

Veracyte is guiding to full-year 2019 revenue in the range of $113 million to $117 million and full-year 2019 net cash used in operating activities in the range of $4 million to $6 million.

Conference Call and Webcast Details

Veracyte will host a conference call and webcast today at 5:00 p.m. Eastern Time to discuss the company’s financial results and provide a general business update. The conference call will be webcast live from the company’s website and will be available via the following link: View Source The webcast should be accessed 10 minutes prior to the conference call start time. A replay of the webcast will be available for one year following the conclusion of the live broadcast and will be accessible on the company’s website at View Source approximately two hours following the completion of the call.

The conference call can be accessed as follows:

U.S./Canada participant dial-in number (toll-free): (855) 541-0980
International participant dial-in number: (970) 315-0440
Conference I.D.: 5498321

On February 25, 2019 Sesen Bio (Nasdaq: SESN), a late-stage clinical company developing targeted fusion protein therapeutics for the treatment of people with cancer, reported that Company management will host a conference call and webcast on Monday, March 4, 2019 at 8:00 a.m. EST to review operating results for the fourth quarter and full year ended December 31, 2018 and new, preliminary analyses from the Phase 3 VISTA trial of Vicinium for patients with high-risk non-muscle invasive bladder cancer who have been previously treated with bacillus Calmette-Guérin (Press release, Sesen Bio, FEB 25, 2019, View Source [SID1234533640]).

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To participate in the conference call, please dial (844) 831-3025 (domestic) or (315) 625-6887 (international) and refer to conference ID 2179668. The webcast can be accessed in the Investor Relations section of the Company’s website at www.sesenbio.com. The replay of the webcast will be available in the investor section of the Company’s website at www.sesenbio.com for 60 days following the call.

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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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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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 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 Actinium Pharmaceuticals, Inc. (NYSE American: ATNM), reported that new data from the ongoing pivotal Phase 3 SIERRA trial for Iomab-B was reported in a late breaking oral presentation at the 2019 Transplantation & Cellular Therapy Meetings of ASBMT and CIBMTR (TCT Meetings) that was held on February 20th – 24th (Press release, Actinium Pharmaceuticals, FEB 25, 2019, View Source [SID1234533624]). Dr. Sergio Giralt, Chief, Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center presented the late breaking oral presentation. It was reported that all patients who received Iomab-B, received a BMT or Bone Marrow Transplant with 100% (28/28) of patients achieving engraftment and Donor Chimerism. The new data indicated that 92% (26/28) of these patients achieved Full Donor Chimerism prior to day 100, which is defined as at least 95% of donor cells being engrafted in the recipient. Full Donor Chimerism prior to day 100 is a clinically significant outcome that indicates acceptance of donor cells and transplant success.

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"We are excited that data from the SIERRA trial continue to demonstrate strong engraftment, particularly in this patient population who have limited access to BMT, which is the only curative treatment option, with current chemotherapy based conditioning approaches", said Dr. Mark Berger, Actinium’s Chief Medical Officer. "Full Donor Chimerism is an important metric in this setting that indicates patients receiving Iomab-B are having successful transplants, which is significant for the SIERRA trial. I am delighted that we were able to report these new data on strong donor chimerism to the transplant community at the TCT Meetings after reporting at ASH (Free ASH Whitepaper) in December that all patients receiving Iomab-B received a BMT and achieved engraftment. We are motivated by the positive reception that the engraftment, safety and feasibility data have received from trial investigators and referring physicians. These data from the SIERRA trial will serve us well as we work to complete enrollment of the SIERRA trial and bring this important therapy to patients with significant unmet needs."

The SIERRA trial (Study of Iomab-B in Elderly Relapsed or Refractory AML) is a 150-patient pivotal Phase 3 multi-center randomized trial that will compare outcomes of patients who receive Iomab-B and a BMT to those patients receiving physician’s choice of salvage chemotherapy, defined as conventional care, as no standard of care exists for this patient population. The primary endpoint of the SIERRA trial is dCR or durable Complete Remission of 6 months. The SIERRA trial is currently enrolling patients at 18 sites in the U.S and Canada including many of the leading BMT sites based on volume. Patients with active, relapsed or refractory AML have dismal prognoses and are typically not offered potentially curative transplant as an option, largely because salvage treatments have a limited ability to produce a complete remission, which is necessary prior to conventional BMT if conventional BMT is to be successful. However, with Iomab-B targeted conditioning, a complete remission prior to starting the Iomab-B conditioning is not necessary for a successful transplant. Iomab-B is an ARC or Antibody Radiation-Conjugate that targets CD45, an antigen expressed on leukemia, lymphoma and immune cells, and delivers Iodine-131 that kills targeted cells via linear energy transfer. Safety and feasibility data from the first 38 patients (25% of planned enrollment) in the SIERRA trial including donor chimerism data that were presented in a late breaking oral session at TCT can accessed here. Additional safety and feasibility analyses will occur when 50% and 75% of patients have been enrolled. The SIERRA trial also permits ad hoc interim analyses that may be requested at Actinium’s discretion to assess safety and efficacy when 70 and 110 patients have reached the primary endpoint of 6-month dCR. However, these interim analyses will not expend meaningful alpha and repowering of the study is not required as trial size cannot be increased after an Ad-hoc interim analysis.

Key highlights from the SIERRA Trial presented at ASH (Free ASH Whitepaper) and the TCT Meetings include:

All patients receiving a therapeutic dose of Iomab-B engrafted despite active disease with high blast count (median 30%, or median 45% for crossover patients)
15 of 19 (79%) patients in the control arm failed to achieve a complete response
67% (10/15) of patients eligible for crossover successfully transplanted after Iomab-B treatment
Patients receiving Iomab-B received a BMT more quickly post-randomization (28 days) than patients receiving conventional care (67 days)
In the conventional care arm, there was no difference in time to BMT for patients that crossed over to Iomab-B (66 days) compared to those achieving complete remission with conventional care (67 days)
No Grade 3 or 4 Iomab-B infusion related reactions with all Iomab-B infusions completed
No 100-Day non-relapse mortality in patients randomized to Iomab-B arm
All patients receiving Iomab-B and a BMT (28/28) achieved Donor Chimerism prior to day 100
94% of patients initially randomized to receive Iomab-B and a BMT (17/18) achieved Full Donor Chimerism > 95% prior to day 100 with 1 patient achieving 65% donor chimerism
90% of patients who crossed-over to receive Iomab-B and a BMT (9/10), after salvage chemotherapy in the control arm failed to produce a CR or Complete Response, also achieved Full Donor Chimerism > 95% prior to day 100 with 1 patient achieving 86% donor chimerism
Sandesh Seth, Actinium’s Chairman and CEO said, "Iomab-B has been studied extensively across multiple clinical trials and disease indications where it has consistently demonstrated the ability to condition patients for BMT in a well-tolerated manner with high engraftment rates and improved clinical outcomes including a survival benefit. As the lead candidate in our pipeline, it is heartening to see interim safety and feasibility data consistent with prior clinical evidence as the trend of strong engraftment with Iomab-B continues in the pivotal multi-center SIERRA trial. In addition, our other posters at TCT supporting the value proposition of Iomab-B from a healthcare economics perspective are also illuminative of the potential opportunity available. We are also excited to note the strong data from the Iomab-ACT program for targeted lymphodepletion prior to CART and other adoptive cell therapies supportive of clinical advancement that was presented at this TCT Meeting. We look forward to providing additional updates as we continue to build and advance our industry leading, multi-asset, multi-indication targeted conditioning portfolio."