On November 13, 2019 Dragonfly Therapeutics, Inc. ("Dragonfly" or the "Company"), reported that it has patented another TriNKET immuno-therapeutic drug for Celgene Corporation and its affiliated companies. ("Celgene") (Press release, Dragonfly Therapeutics, NOV 13, 2019, View Source;809642739.html [SID1234551223]). Today’s announcement marks Celgene’s third participation since the companies announced a malignant hematology tumor collaboration in June 2017, and follows the announcement in December 2018 by Dragonfly of the participation of Celgene to the first two immuno-therapeutic drug candidates TriNKET .

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"We continue to be impressed by the quality of the candidates developed using Dragonfly’s TriNKET technology," said Rupert Vessey, FRCP DPhil, President of Research and Early Development for Celgene Corporation. "Since choosing the first two Dragonfly candidates in 2018, we have had the opportunity to further evaluate Dragonfly’s TriNKET drug candidates, and we continue to view encouraging potential therapeutic options for patients with of cancer by harnessing the power of NK cells. "

"We are pleased with the pace of our development efforts and the additional validation of our Celgene adoption adoption platform," said Bill Haney , CEO of Dragonfly. "As we move forward with our own programs, including our DF-1001 clinical stage asset, we look forward to seeing the continued progress of our partner programs. We are looking for new potential options for immuno-oncology treatment for cancer patients. "

On November 13, 2019 The Basser Center for BRCA at the Abramson Cancer Center of the University of Pennsylvania reported that it will host its third Basser Jean Bash on November 18th at Cipriani Wall Street (Press release, Basser Center for BRCA, NOV 13, 2019, View Source [SID1234551179]). This year’s Bash celebrates the remarkable progress made by the Basser Center for BRCA, the milestone of the cloning of BRCA1 25 years ago, and the scientific contributions that have advanced the understanding, treatment, and prevention of BRCA-related cancers.

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As part of this year’s event, the Basser Center for BRCA will officially launch the LATINX & BRCA initiative, a comprehensive effort to raise awareness, provide education and resources, and build community around BRCA-related cancers. Among the honorees of this year’s event is Nina Garcia, editor-in-chief of Elle magazine, who announced this year that she has a BRCA mutation and underwent preventative treatment, bravely sharing her story publicly along the way.

The Basser Center is the world’s first comprehensive center to focus solely on the prevention and treatment of cancers associated with inherited mutations of the BRCA genes. Founded by University of Pennsylvania alumni Mindy and Jon Gray, President and Chief Operating Officer at Blackstone, the Basser Center was established in 2012 in honor of Mindy’s sister, Faith Basser, who passed away at the age of 44 of BRCA-related ovarian cancer. The Grays have contributed more than $55 million to fund the mission of the Center, and in total the Basser Center has raised $100 million.

The Basser Jean Bash, which is expected to draw 1100 guests, is being generously underwritten so that every dollar raised will support the life-saving mission of the Basser Center for BRCA. To date, this event has raised $8 million, and this year a limited edition denim tote, customized with the distinct Basser logo, will be available for purchase the evening of the event with 100 percent of the proceeds supporting the Basser Center for BRCA.

On November 13, 2019 Navigation Sciences reported that it will provide an update at the annual Medtech Strategist Innovation Summit San Francisco 2019 Conference on its Image Guided, Video Assisted Surgery (iVATS) technology for the minimally invasive identification and removal of early stage lung cancer, the company (Press release, Navigation Sciences, NOV 13, 2019, View Source [SID1234551178]). The conference will take place in Burlingame, California on November 19, 2019.

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"iVATS is a revolutionary approach to minimally invasive soft tissue surgery that combines surgical instruments with augmented reality software to provide real-time, GPS navigation to precisely remove small tumors with optimal margins while preserving lung function," said Alan Lucas, Navigation Sciences’ co-founder and CEO. "We have made significant progress in product development and pre-clinical testing that puts us on a course to begin a clinical trial in mid-2020. We expect the trial to provide the basis for a 510(k) submission to the FDA for marketing clearance."

The emergence of CT chest scanning as an effective tool in high risk populations to detect lung cancer at an early stage and lower cancer deaths is creating new opportunities to improve lung surgery. Removal of small tumors deep inside the lung with appropriate margins remains a significant challenge, as insufficient distance between the tumor and resection margin is associated with recurrence and death. Current surgical approaches either remove excess tissue via lobectomy, which lowers recurrence risk but results in more impaired lung function, or smaller, wedge resections, which reduces lung impairment but may increase recurrence risk.

The Navigation Sciences solution integrates with multi-modal (imaging and surgery) technology and includes an active, GPS enabled marker (fiducial), proprietary, real-time navigation algorithms, and tumor cutting devices that allow for surgical precision specific to the patient’s anatomy. Radiology images are synchronized with real-time information to enable the clinician to perform surgery with precision guidance. The technology’s proof-of-concept was demonstrated in a Phase I-II clinical trial published in the Journal of Surgical Oncology. In the study, involving 25 patients (23 with early stage lung cancer), there were no significant complications, all nodules were fully resected with negative margins, and the technology was shown to integrate with surgical workflow.

There are an estimated 87,000 patients in the U.S. diagnosed each year with early stage lung cancer and that number is expected to grow with growing recognition of CT X-ray screening’s value in early detection and favorable reimbursement coverage. Beyond lung cancer, the technology has significant potential in other soft tissue cancer surgeries including breast cancer, where an estimated 250,000 lumpectomies are performed annually, as well as liver, thyroid and brain tumors.

The iVATS technology was pioneered by Raphael Bueno, MD Chief, Thoracic Surgery, at Brigham and Women’s Hospital and Professor of Surgery at Harvard Medical School, and Jayender Jagadeesan, PhD, research assistant at BWH and Assistant Professor of Radiology at Harvard Medical School. Drs. Bueno and Jagadeesan are co-founders and shareholders of the company.

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On November 13, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to finding cures for blood cancers and serious blood diseases, reported financial results for the quarter ended September 30, 2019 (Press release, Gamida Cell, NOV 13, 2019, View Source [SID1234551177]). The company also highlighted continued progress in advancing its clinical development candidates: omidubicel, an advanced cell therapy in Phase 3 clinical development as a potential life-saving treatment option for patients in need of bone marrow transplant, and GDA-201, an investigational, natural killer (NK) cell-based cancer immunotherapy in Phase 1 development in patients with non-Hodgkin lymphoma and multiple myeloma.

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Gamida Cell provides a company update and reports financial results for the third quarter of 2019. Webcast at 4:30pm ET here: View Source

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"During the quarter, we made important progress toward our goal of developing next-generation cell therapies with the potential to redefine standards of care for patients with blood cancers and rare, serious hematologic diseases," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "We are nearing completion of patient enrollment in our multi-center, randomized Phase 3 study of omidubicel and are on track to report topline data from the study in the first half of 2020. Positive data from the study would enable us to file our first biologics license application next year."

"Our second cell therapy program, GDA-201, is also advancing, and next month we will report additional data from the ongoing Phase 1 study at the Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper)," Dr. Adams continued. "During the meeting, we will also show new gene expression data that reinforce our understanding of the mechanism of action underlying our NAM technology platform which enabled the generation of both of our development candidates."

Program Highlights

Announced presentation of new data at the 61st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper): Last week, Gamida Cell announced that additional data from the ongoing Phase 1 clinical study of GDA-201 will be presented during an oral session at the ASH (Free ASH Whitepaper) 2019 Annual Meeting, which is being held December 7 – 10 in Orlando, FL. The presentation, "Results of a Phase 1 Trial of GDA-201, Nicotinamide-Expanded Allogeneic Natural Killer Cells (NAM-NK) in Patients with Refractory Non-Hodgkin Lymphoma (NHL) and Multiple Myeloma (MM)" (Abstract #777), will take place on Monday, December 9, at 3:15 p.m. ET.

Additionally, new research on the mechanism of action of Gamida Cell’s NAM-based cell expansion platform, which is designed to enhance the number and functionality of allogeneic donor cells, will also be shared during the meeting. The poster presentation, "Nicotinamide (NAM) Modulates Transcriptional Signature of Ex Vivo Cultured UCB CD34+ Cells (Omidubicel) and Preserves Their Stemness and Engraftment Potential" (Abstract #3718), will take place on Monday, December 9, from 6:00 – 8:00 p.m. ET.
Advanced the Phase 3 clinical study of omidubicel: Patient enrollment continued to progress in the Gamida Cell’s Phase 3 study of omidubicel in patients with high-risk hematologic malignancies. The international, randomized, multi-center study is designed to evaluate the safety and efficacy of omidubicel compared to standard umbilical cord blood for allogeneic bone marrow transplant in approximately 120 patients with no available matched donor. The company anticipates completing patient enrollment by the end of this year with topline data anticipated in first half of 2020.
Initiated health outcomes research for omidubicel: In September, Gamida Cell and the CIBMTR (Center for International Blood and Marrow Transplant Research) announced a research agreement to evaluate outcomes of patients with hematological malignancies who undergo allogeneic hematopoietic stem cell transplant (bone marrow transplant) from various donor sources. The recently launched observational study includes both retrospective and prospective data contemporaneous to the Phase 3 study of omidubicel. The goal of this real-world, observational study is to better understand the variables that influence the health outcomes of patients receiving a transplant from a source other than a fully matched family donor.
Continued to focus on activities required to successfully bring omidubicel to patients: Gamida Cell is continuing to advance key activities required to bring omidubicel to patients in a commercial setting, including building out manufacturing infrastructure, assembling an experienced commercial team with expertise in cell therapy and transplant, establishing hospital services and patient assistance programs, and exploring coverage and reimbursement models to enable access.
Progressed enrollment in the Phase 1/2 study of omidubicel in patients with severe aplastic anemia: Enrollment is ongoing in a Phase 1/2 clinical study of omidubicel in patients with severe aplastic anemia, a rare, life-threatening bone marrow failure disease. Earlier this year, encouraging data from the first cohort of patients were reported at the 2019 Transplantation & Cellular Therapy (TCT) Meeting that demonstrated that all three patients in the cohort successfully underwent a bone marrow transplant consisting of omidubicel plus a haploidentical stem cell graft. The rapid engraftment, sustained hematopoiesis and accelerated immune recovery observed enabled the initiation of a second cohort, where patients will be treated with omidubicel as a stand-alone graft.
Continued to prepare for the next clinical study of GDA-201: The company is continuing its work to enable a Phase 1/2 multi-dose, multi-center study of GDA-201 in patients with non-Hodgkin lymphoma, which is expected to begin in 2020. The decision to focus the next clinical study on non-Hodgkin lymphoma is based on the encouraging clinical data reported at the 2019 TCT Meeting which demonstrated the GDA-201 was generally well tolerated and clinically active, with multiple complete responses observed.
Third Quarter 2019 Financial Results

Research and development (R&D) expenses in the third quarter of 2019 were $7.4 million compared to $5.1 million for the same period in 2018. R&D expenses were higher in the third quarter of 2019 compared to the same period in 2018 due to the advancement of omidubicel and GDA-201.
General and administrative expenses were $4.6 million for the third quarter of 2019, compared to $2.4 million for the same period in 2018. The difference was attributable mainly to a $1.2 million increase in activities related to commercial readiness, as well as $1.0 million increase in professional services and other expenses, including an increase in expenses associated with being a publicly-traded company.
Finance income, net, was $1.7 million for the third quarter of 2019, compared to finance expenses, net, of $2.2 million for the same period in 2018. The net increase was primarily due to non-cash income resulting from the re-valuation of warrants, offset by non-cash expenses from the re-valuation of the Israeli Innovation Authority royalty-bearing grant liability.
Net loss for the third quarter of 2019 was $10.1 million, compared to a net loss of $9.8 million for the same period in 2018.
As of September 30, 2019, Gamida Cell had total cash, cash equivalents and available-for-sale assets of $68.1 million, compared to $60.7 million as of December 31, 2018.
2019 Financial Guidance

Gamida Cell continues to expect cash used for ongoing operating activities in 2019 to range from $35 million to $40 million, reflecting anticipated expenditures to advance the company’s clinical programs.

Gamida Cell expects that its current cash, cash equivalents and available-for-sale securities will support the company’s ongoing operating activities into the fourth quarter of 2020. This cash runway guidance is based on the company’s current operational plans and excludes any additional funding that may be received or business development activities that may be undertaken.

Conference Call Information

Gamida Cell will host a conference call today, November 13, 2019, at 4:30 p.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors" section of Gamida Cell’s website at www.gamida-cell.com. To participate in the live call, please dial 866-930-5560 (domestic) or 409-216-0605 (international) and refer to conference ID number 8653335. A replay of the webcast will be available approximately two hours after the event, for approximately 30 days.

About Omidubicel

Omidubicel (formerly known as NiCord), the company’s lead clinical program, is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU. In a Phase 1/2 clinical study, omidubicel demonstrated rapid and durable time to engraftment and was generally well-tolerated.1 A Phase 3 study evaluating omidubicel in patients with leukemia and lymphoma is ongoing in the U.S., Europe and Asia.2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.3 The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

About GDA-201

Gamida Cell applied the capabilities of its NAM-based cell expansion technology to develop GDA-201 (formerly known as NAM-NK), an innate natural killer (NK) cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.4

Omidubicel and GDA-201 are investigational therapies, and their safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On November 13, 2019 NanoString Technologies, Inc. (NASDAQ:NSTG), a leading provider of life science tools for translational research and molecular diagnostic products, reported that the company’s management is scheduled to present at the Stifel 2019 Healthcare Conference in New York City (Press release, NanoString Technologies, NOV 13, 2019, View Source [SID1234551176]).

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Brad Gray, president and chief executive officer, is scheduled to present on Wednesday, November 20th, 2019 from 1:15-1:45pm ET. Interested parties can access the live webcast with accompanying slides from the investor section of the company’s website at www.nanostring.com. The webcast replay will be available one hour after the conclusion of the live presentation and archived for 60 days.