On February 25, 2020 Noxopharm (ASX: NOX) reported that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application for Veyonda for combination treatment with doxorubicin in patients with soft tissue sarcomas (Press release, Noxopharm, FEB 25, 2020, View Source [SID1234554750]).

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"Based on preclinical and clinical data, the FDA’s approval of this IND is validation of the clinical potential of Veyonda," said Noxopharm CMO Gisela Mautner, M.D., Ph.D. "Further, it means that Veyonda has met stringent FDA standards for safety and tolerability."

"While the Company’s resources and energies are primarily focused on bringing a treatment for late-stage prostate cancer to market, this IND grant for a less common cancer type advances that commercial objective," said Noxopharm CEO Graham Kelly, Ph.D. "The recent IND approval process has familiarized the FDA with Veyonda ahead of IND applications for more advanced DARRT and LuPIN study submissions. It also opens the door to Veyonda’s use in the U.S., where patient demand has been growing."

DARRT and LuPIN are ongoing investigations in which combination therapies with Veyonda are exhibiting promising interim results for the treatment of late-stage, progressive prostate cancer resistant to other treatments.

"This IND grant is an important step toward achieving a strategic partnering arrangement," Kelly said. "With the emerging clinical data and growing evidence that Veyonda is a uniquely acting immuno-oncology drug candidate designed to restore immune function to cold tumors, we are confident that the industry will recognize the considerable opportunities in the adjunctive benefits of Veyonda."

Veyonda is a suppository dosage form of idronoxil, a first-in-class inhibitor of sphingosine-1-phosphate (S1P). By inhibiting the overexpression of S1P-driven pro-survival pathways in solid tumors, idronoxil is designed to sensitize cancer cells to the anti-cancer effects of drugs such as doxorubicin, as well as to radiation.

On February 25, 2020 Proscia, a leading provider of artificial intelligence (AI) enabled digital pathology software, reported that it has released the findings of a new study on the first deep learning system with proven accuracy in real laboratory environments (Press release, Proscia, FEB 25, 2020, View Source [SID1234554749]). Published in Scientific Reports, a journal from Nature Research, the study is the largest AI validation study conducted in pathology to date and supports the growing impact of AI in cancer diagnosis. The paper’s results serve as the foundation for enabling faster and more accurate diagnosis to improve treatment decisions and patient outcomes.

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The antiquated standard of care for diagnosing cancer relies on the pathologist’s assessment of patterns in tissue as viewed under a microscope. This manual and subjective practice cannot keep pace with the growing demand for diagnostic services amid a rapidly declining pathologist population and can lead to a lack of confidence in treatment decisions.

Proscia’s study, titled "Tailored for Real-World: A Whole Slide Image Classification System Validated on Uncurated Multi-Site Data Emulating the Prospective Pathology Workload," describes a deep learning system that achieves 98% accuracy in classifying whole slide images of skin biopsies in real laboratory settings. To achieve this consistent performance, the system was designed using high-quality and diverse de-identified data. It was developed using thousands of images from Dermatopathology Laboratory of Central States, one of the largest dermatopathology laboratories in the United States, and tested on an uncurated set of 13,537 images from Cockerell Dermatopathology, Thomas Jefferson University Hospital, and University of Florida to account for the wide variety of diseases seen in practice. This volume of test data, along with the different methods of biopsy, preparation of tissue, tissue staining procedures, and digital scanning processes used across the test laboratories, indicate that the AI is generalizable across multiple laboratory settings.

"The size, scope, and complexity of our study sets a new standard for demonstrating the real-world viability of artificial intelligence in pathology," said Mike Bonham, MD, Ph.D., Chief Medical Officer of Proscia. "We have finally passed the inflection point in generating scientific evidence that AI can drive accuracy and efficiency gains in practice."

Proscia conducted this study to validate its DermAI application and bring AI into the pathology laboratory. Launched in June 2019, DermAI is the first in a series of AI applications on Proscia’s Concentriq platform.* By using deep learning to automatically classify hundreds of variants of skin diseases, DermAI is driving much-needed confidence, quality, and efficiency gains with capabilities including intelligent workload balancing, case prioritization, automated QA, and 100% AI re-review. This first-of-its-kind pathology solution can reduce costly errors and process added volume to meet the global cancer burden.

Dr. Thomas G. Olsen, founder of Dermatopathology Laboratory of Central States and an investigator in the validation study, collaborated with Proscia on the design and clinical requirements for DermAI. "The true promise of digital pathology lies in deep learning, which will transform the practice of pathology for the first time since the introduction of the microscope," commented Dr. Olsen. "It’s exciting to have been part of moving AI-enabled digital pathology beyond academic efforts and into real-world practice applications."

With additional AI applications forthcoming, Proscia is committed to advancing diagnostics and research for other high-impact areas of pathology. Most recently, the company announced a collaboration with Johns Hopkins School of Medicine to bring AI applications to multiple subspecialties.

"The results of our research show robust generalization to data from multiple labs and scanners. The validation study design was a deliberate departure from the examples we’ve seen in pathology to date," said Julianna Ianni, Ph.D., Proscia’s Director of AI Research. "The burden of proof for AI is higher than it was a year ago, as laboratories are increasingly looking to deploy practical solutions to enhance their workflows."

The full study, published in Scientific Reports on February 21, 2020, can be found online here. To learn more about the study, please register for Proscia’s upcoming webinar, "Overcoming Real-World Variability: Inside Pathology’s Most Comprehensive AI Validation Study," on March 31, 2020 at 12PM ET.

On February 25, 2020 Tarveda Therapeutics, Inc., a clinical stage biopharmaceutical company developing a new class of potent and selective precision oncology medicines, which it refers to as Pentarin miniature drug conjugates, reported that Drew Fromkin, President and Chief Executive Officer, will present at the Cowen & Company 40th Annual Health Care Conference, occurring March 2-4, 2020 in Boston (Press release, Tarveda Therapeutics, FEB 25, 2020, View Source [SID1234554748]). Tarveda presentation details:

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Date: Monday, March 2, 2020
Time: 12:00pm Eastern Time
Location: Boston Marriott Copley Place, Boston, MA

On February 25, 2020 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 full year ended December 31, 2019 (Press release, Gamida Cell, FEB 25, 2020, View Source [SID1234554747]). 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 (NHL) and multiple myeloma.

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"2019 was a key year for Gamida Cell that moved us closer to 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. "In December, we completed patient enrollment in our multi-center, randomized Phase 3 study of omidubicel, the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA). We look forward to reporting topline data from the study in the second quarter of 2020. Positive data would enable us to file a biologics license application for omidubicel with the FDA in the fourth quarter of the year, representing an important step toward bringing potentially curative medicines to patients."

"We also continued to advance GDA-201, our second cell therapy program, which has shown early promise for the treatment of non-Hodgkin lymphoma. We will report additional data from this study in the first half of 2020 and are progressing toward an investigational new drug submission to the FDA by the end of the year," Dr. Adams continued.

Additional Company Highlights

Completed patient enrollment in Phase 3 clinical study of omidubicel: In December 2019, Gamida Cell completed patient enrollment in the 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. Topline data from the study are expected in the second quarter of 2020. Assuming positive data, the company expects to submit a biologics license application to the FDA in the fourth quarter of 2020.
Presented research on mechanism of action for the NAM technology platform at the Transplantation & Cellular Therapy (TCT) Annual Meeting: In February 2020, Gamida Cell reported research on the mechanism of action of its NAM cell expansion platform, which is designed to enhance the number and functionality of allogeneic donor cells. These data provide further scientific rationale for the favorable stem cell engraftment and patient outcomes observed in the Phase 1/2 clinical study of omidubicel.
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. Gamida Cell plans to report additional data from the study in the second half of 2020.
Presented data from Phase 1 clinical study of GDA-201 at 61st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper): In December, additional data were presented from the ongoing Phase 1 study of GDA-201. Results from 22 patients showed that GDA-201 in combination with monoclonal antibodies was generally well tolerated and demonstrated early evidence of clinical activity in heavily pre-treated patients, including five complete responses observed among nine patients with NHL.
Continued to prepare for the next clinical study of GDA-201: Based on the data from the ongoing Phase 1 study of GDA-201, Gamida Cell expects to submit an investigational new drug application to the FDA in the fourth quarter of 2020 to enable the initiation of a multi-center, multi-dose Phase 1/2 clinical study in patients with NHL.
Further strengthened executive team: In January, Gamida Cell announced the appointment of Jas Uppal, Ph.D. to the newly created role of chief regulatory and quality officer. Dr. Uppal brings more than 25 years of global experience in the pharmaceutical industry, including expertise in hematology, immunology and neurology. During her career, she has played key roles in building regulatory organizations and leading multiple successful product launches.
Expected 2020-2021 Milestones
Gamida Cell targets achieving the following milestones during 2020-2021:

Omidubicel

Report topline data from the Phase 3 study in the second quarter of 2020
Present data from the Phase 3 study at a medical meeting in the second half of 2020
Submit the biologics license application to the FDA in the fourth quarter of 2020, assuming positive data
Report additional data from the Phase 1/2 study in patients with severe aplastic anemia in the second half of 2020
Launch omidubicel in 2021, contingent upon FDA approval
GDA-201

Present additional data from the Phase 1 study in the first half of 2020
Submit company-sponsored investigational new drug application to FDA in the fourth quarter of 2020
Initiate a Phase 1/2 clinical study in patients with NHL in 2021
Full Year 2019 Financial Results:

Research and development (R&D) expenses in 2019 were $31.5 million, compared to $22.0 million in 2018. The increase was mainly due to clinical activities relating to the advancement of omidubicel and GDA-201 as well as additional headcount within the R&D organization.
The commercial organization was established in 2019, and commercial expenses for the year were $4.7 million. These expenses were mainly due to $2.4 million of cash and non-cash expenses related to hiring and establishing the commercial organization as well as $2.3 million related to professional services and other expenses.
General and administrative expenses were $12.1 million in 2019, compared to $11.6 million in 2018. The increase was mainly due to a $1.0 million increase in professional services expenses associated with being a publicly traded company and a $1.3 million increase in rent and other expenses, offset by a $1.8 million decrease related to establishing the commercial organization.
Finance income, net, was $13.8 million in 2019, compared to finance expenses, net, of $19.2 million in 2018. The increase was primarily due to non-cash income resulting from revaluation of warrants, offset by non-cash expenses of the Israeli Innovation Authority royalty-bearing grant liability and the implementation of the new IFRS 16 accounting standard.
Net loss for 2019 was $34.4 million, compared to a net loss of $52.9 million for 2018.
At December 31, 2019, Gamida Cell had total cash, cash equivalents and available-for-sale securities of $55.4 million, compared to $60.7 million at December 31, 2018.
2020 Financial Guidance
Gamida Cell expects cash used for ongoing operating activities for the first six months of 2020 to range from $30-$35 million, primarily reflecting anticipated expenditures to advance the company’s clinical programs. The company expects to provide full year financial guidance following the availability of topline Phase 3 data for omidubicel.

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, including the assumption that Gamida Cell will continue to advance all of its clinical programs 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, February 25, 2020, at 8:30 a.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 +1-409-216-0605 (international) and refer to conference ID number 7886498. A recording of the webcast will be available approximately two hours after the event, for approximately 30 days.

About Omidubicel
Omidubicel, 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., South America, 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, 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 February 25, 2020 -Lantheus Holdings, Inc. (the "Company") (NASDAQ: LNTH), parent company of Lantheus Medical Imaging, Inc. ("LMI"), a global leader in the development, manufacture and commercialization of innovative diagnostic imaging agents and products, reported financial results for its fourth quarter and full year 2019 ended December 31, 2019 (Press release, Lantheus Medical Imaging, FEB 25, 2020, View Source [SID1234554746]).

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The Company’s worldwide revenue for the fourth quarter of 2019 totaled $89.3 million, compared with $86.3 million for the fourth quarter of 2018, representing an increase of 3.6% over the prior year period. Full year 2019 worldwide revenues were $347.3 million, compared with $343.4 million for the full year 2018, representing an increase of 1.2% over the prior year period.

The Company’s fourth quarter 2019 net income was $10.5 million, or $0.26 per fully diluted share, as compared to $13.3 million, or $0.34 per fully diluted share for the fourth quarter of 2018, representing a decrease of 21.4% from the prior year period. Full year 2019 net income was $31.7 million, or $0.79 per fully diluted share, as compared to $40.5 million, or $1.03 per fully diluted share for the full year 2018, representing a decrease of 21.8% from the prior year period.

The Company’s fourth quarter 2019 adjusted fully diluted earnings per share were $0.34, as compared to $0.29 for the fourth quarter of 2018, representing an increase of 19.0% over the prior year period. The Company’s full year 2019 adjusted fully diluted earnings per share were $1.17, as compared to $1.13 for the full year 2018, representing an increase of 3.5% over the prior year period.

Lastly, net cash provided by operating activities was $22.4 million and $80.4 million for the fourth quarter and full year 2019. Free Cash Flow was $17.7 million for the fourth quarter of 2019, representing an increase of approximately $7.7 million, or 77.9% over the prior year period. Full year 2019 free cash flow was $58.3 million, as compared to $41.1 million for the full year 2018, representing an increase of approximately $17.3 million, or 42.0% over the prior year period.

"We finished 2019 with robust DEFINITY, earnings and free cash flow performance," said Mary Anne Heino, President and CEO. "Throughout 2019, it was Lantheus team members from across the organization who drove these results. As we look to 2020 and beyond, we see exciting revenue and value creation opportunities for the Company as we add the Progenics’ team with their exciting oncology, precision diagnostic, and other products to our own portfolio."

Outlook

The Company guidance for the first quarter and full year 2020 is as follows:

On a forward-looking basis, the Company does not provide GAAP income per common share guidance or a reconciliation of adjusted fully diluted EPS to GAAP income per common share because the Company is unable to predict with reasonable certainty business development and acquisition-related expenses, purchase accounting fair value adjustments, and any one-time, non-recurring charges. These items are uncertain, depend on various factors, and could be material to results computed in accordance with GAAP. As a result, it is the Company’s view that a quantitative reconciliation of adjusted fully diluted EPS on a forward-looking basis is not available without unreasonable effort.

Internet Posting of Information

The Company routinely posts information that may be important to investors in the "Investors" section of its website at www.lantheus.com. The Company encourages investors and potential investors to consult its website regularly for important information about the Company.

Conference Call and Webcast

As previously announced, the Company will host a conference call on Tuesday, February 25, 2020 at 8:00 a.m. ET. To access the live conference call via telephone, please dial 1-866-498-8390 (U.S. callers) or 1-678-509-7599 (international callers) and provide passcode 7953035. A live audio webcast of the call also will be available in the Investors section of the Company’s website at www.lantheus.com.

A replay of the audio webcast will be available in the Investors section of our website at www.lantheus.com approximately two hours after completion of the call and will be archived for 30 days.

The conference call will include a discussion of non-GAAP financial measures. Reference is made to the most directly comparable GAAP financial measures, the reconciliation of the differences between the two financial measures, and the other information included in this press release, our Form 8-K filed with the SEC today, or otherwise available in the Investor Relations section of our website located at www.lantheus.com.

The conference call may include forward-looking statements. See the cautionary information about forward-looking statements in the safe-harbor section of this press release.