On February 14, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported that it will host a conference call and webcast on Thursday, February 21, 2019, at 8:00 a.m. ET to review the data from its NAM-NK and NiCord programs being presented at the 2019 Transplantation & Cellular Therapy (TCT) Meetings of American Society for Blood and Marrow Transplantation (ASBMT) and Center for International Blood and Marrow Transplant Research (CIBMTR) (Press release, Gamida Cell, FEB 14, 2019, View Source [SID1234533329]). The meeting is taking place in Houston, Texas, February 20 – 24.

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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 conference call, please dial 1-866-930-5560 (domestic) or 1-409-216-0605 (international) five minutes prior to start time. The conference ID number is 9462948. An archived version of the webcast will be available on Gamida Cell’s website for 30 days.

Details about the presentations are as follows:

Time: Wednesday, February 20, 2019, 6:45 p.m. – 7:45 p.m. CT (poster presentation)
Title: First-in-Human Phase I Study of Nicotinamide-Expanded Related Donor Natural Killer Cells for the Treatment of Relapsed/Refractory Non-Hodgkin Lymphoma and Multiple Myeloma
Poster Number: 242
Lead Author: Veronika Bachanova, M.D., Ph.D., associate professor of medicine, division of hematology, oncology and transplantation, University of Minnesota
Location: George R. Brown Convention Center, Level 3, Hall B

Time: Wednesday, February 20, 2019, 6:45 p.m. – 7:45 p.m. CT (poster presentation)
Title: Ex Vivo Nicotinamide-Expanded (NAM-Expanded) Unrelated Cord Blood Transplantation (UCB) for Refractory Severe Aplastic Anemia Results in Rapid Engraftment and Expedites Immune Recovery
Poster Number: 295
Lead Author: Joseph Clara, M.D., Hematology Branch, National Heart, Lung, and Blood Institute
Location: George R. Brown Convention Center, Level 3, Hall B

Time: Saturday, February 23, 2019, 4:45 p.m. – 5:00 p.m. CT (oral presentation)
Title: Rapid and Robust CD4+ and CD8+ T-, NK-, B-Cell, Dendritic Cell, and Monocyte Reconstitution after Nicotinamide-Expanded Cord Blood Transplantation
Abstract Number: 69
Lead Author: Jaap-Jan Boelens, M.D., Ph.D., Chief, Pediatric Stem Cell Transplantation and Cellular Therapies Service, Memorial Sloan Kettering Cancer Center
Location: Hilton Americas Houston, Grand Ballroom G

Abstracts are available on the 2019 TCT Meetings of ASBMT and CIBMTR website.

About NAM-NK
Gamida Cell applied the capabilities of its NAM-based cell expansion technology to highly functional NK cells to develop NAM-NK, an innate immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. NAM-NK 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. NAM-NK is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.1

About NiCord
NiCord, the company’s lead clinical program, is under development as a universal bone marrow transplant solution for patients with high-risk hematologic malignancies. NiCord has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration, making it the first bone marrow transplant alternative to receive this designation. It has also received U.S. and EU orphan drug designation. A Phase 3 clinical study evaluating NiCord in patients with leukemia and lymphoma is ongoing in the United States, Europe and Asia.2 NiCord 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 NiCord. For more information on clinical trials of NiCord, please visit www.clinicaltrials.gov.

NAM-NK and NiCord 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 14, 2019 Vor Biopharma, an immuno-oncology company pioneering engineered hematopoietic stem cell (HSC) therapies for the treatment of hematological malignancies, reported a $42 million Series A financing round led by 5AM Ventures and RA Capital Management (Press release, Vor BioPharma, FEB 14, 2019, View Source [SID1234533328]). Johnson & Johnson Innovation – JJDC, Inc . (JJDC), Novartis Institutes for BioMedical Research (NIBR), and Osage University Partners also participated in the round along with Vor Co-founder PureTech Health. Vor plans to use the proceeds from the financing to advance its lead HSC-based candidate for the treatment of acute myeloid leukemia (AML) towards the clinic, and to further build its pipeline to treat hematologic malignancies.

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"Vor’s unique and patented technology platform for enabling targeted immunotherapies using engineered HSCs has the potential to disrupt the treatment landscape for hematologic malignancies," said Kush Parmar, M.D., Ph.D., Managing Partner at 5AM Ventures who has joined the Vor Biopharma Board of Directors as Executive Chair. "We are excited to work with Vor to drive this next chapter of growth forward."

"The need for new therapies for hematologic malignancies is dire. I am gratified that this discovery from my lab continues to advance towards the clinic. This new platform may enable more patients to benefit from the life-saving potential of targeted immunotherapies," said Siddhartha Mukherjee, M.D., D.Phil, Vor Co-founder and Associate Professor of Medicine at Columbia University. "I look forward to further engaging with the scientific community when we publish our results in peer-reviewed journals."

"We are excited about Vor’s bold and novel approach to potentially revolutionize the way stem cell transplants are used to treat severe hematologic cancers," said Joshua Resnick, M.D., Managing Director at RA Capital who has also joined the Vor Biopharma Board of Directors.

Vor’s engineered HSC technology platform is designed to address fundamental limitations of today’s immunotherapies. Vor’s approach has the potential to expand the reach of targeted immunotherapies to a broad range of patient populations and hematological malignancies by enabling new dosing paradigms for cancer-targeted immunotherapies, which can substantially improve the therapeutic window for efficacy and improve patient safety.

"We are delighted to welcome this terrific syndicate of investors, who share our passion, commitment, and vision for bringing Vor’s potentially life-saving new therapies to patients with acute myeloid leukemia and other hematologic malignancies," said Bharatt Chowrira, J.D., Ph.D., Board of Directors at Vor Biopharma and President and Chief of Business & Strategy at PureTech Health

On February 14, 2019 Bayer reported Results from the pivotal Phase III ARAMIS trial in patients with non-metastatic castration-resistant prostate cancer (nmCRPC) showed a statistically significant improvement in metastasis-free survival (MFS) with the investigational drug darolutamide plus androgen deprivation therapy (ADT) compared to placebo plus ADT (HR=0.41, 95% CI 0.34-0.50; P<0.001) (Press release, Bayer, FEB 14, 2019, View Source [SID1234533327]).2 This translates to a 59 percent reduction in the risk of metastasis or death.1 The median MFS was 40.4 months in the darolutamide arm compared with 18.4 months for the placebo arm – an overall difference in median MFS of 22 months.1

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The incidence of adverse events (AEs) was generally similar in the darolutamide and placebo arms; only fatigue occurred in more than 10 percent of patients (15.8 percent with darolutamide plus ADT versus 11.4 percent with placebo plus ADT).2 The most common grade 3-4 AEs for darolutamide and placebo were 24.7 percent and 19.5 percent, respectively.2 Grade 3-4 AEs occurring in greater than or equal to 2 percent of patients were hypertension (3.1 percent versus 2.2 percent) and urinary retention (1.6 percent versus 2.0 percent) for darolutamide and placebo, respectively.2

These data were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO GU) in San Francisco and published simultaneously in The New England Journal of Medicine.

"These data are exciting for the prostate cancer community, as they show darolutamide’s potential to treat asymptomatic nmCRPC patients and delay spread of the disease," said Karim Fizazi, M.D., Ph.D., Professor of Medicine at the Institut Gustave Roussy, University of Paris Sud, France.

"These data demonstrate that darolutamide may be an option for men with nmCRPC and can potentially fulfill an unmet need for these patients," said Matthew Smith, M.D., Ph.D., Director of the Genitourinary Malignancies Program, Massachusetts General Hospital Cancer Center.

"Bayer is working diligently to bring treatments to patients in need," said Scott Z. Fields, M.D., senior vice president and head of Oncology Development at Bayer’s Pharmaceutical Division. "With the positive results of the ARAMIS trial, we are one step closer to our goal of bringing darolutamide to patients and physicians."

Bayer plans to discuss the data from the ARAMIS trial with health authorities regarding the submission of new drug applications. Bayer has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for darolutamide in men with nmCRPC. Darolutamide is being developed jointly by Bayer and Orion Corporation, a globally operating Finnish pharmaceutical company.

Detailed study results
The ARAMIS trial also included several key secondary endpoints.2 At the time of the first interim analysis, median OS had not yet been reached in either treatment arm.1 However, these interim results demonstrated a trend in favor of darolutamide plus ADT (HR=0.71, 95% CI 0.50-0.99; P=0.045).1 Median time to pain progression was 40.3 months in the darolutamide arm compared to 25.4 months in the placebo arm (HR=0.65, 95% CI 0.53-0.79; P<0.001).1 Median time to cytotoxic chemotherapy was not reached yet in the darolutamide arm compared to 38.2 months in the placebo arm (HR=0.43, 95% CI 0.31-0.60; P<0.001).1 Median time to first symptomatic skeletal event (SSE) was also not reached yet in either treatment arm; however, these interim results demonstrated a trend in favor of darolutamide plus ADT (HR=0.43, 95% CI 0.22-0.84; P<0.001).1 SSE was defined as external beam radiation therapy (EBRT) to relieve skeletal symptoms, new symptomatic pathologic bone fracture, or occurrence of spinal cord compression or tumor-related orthopedic surgical intervention, whichever occurs first.2

Time-to-event exploratory endpoints included progression-free survival (PFS) and time to prostate-specific antigen (PSA) progression.2 Median PFS was 36.8 months in the darolutamide arm compared to 14.8 months in the placebo arm (HR=0.38, 95% CI 0.32-0.45; P<0.001).1 Time to PSA progression was 33.2 months in the darolutamide arm versus 7.3 months in the placebo arm (HR=0.13, 95% CI 0.11-0.16; P<0.001).2

Exploratory PRO-based endpoints (based on the Functional Assessment of Cancer Therapy-Prostate; FACT-P, European Organisation for Research and Treatment of Cancer quality of life; EORTC-QLQ-PR25, and EQ-5D-3L questionnaires) were also evaluated in the ARAMIS trial.1

About the ARAMIS trial design
The ARAMIS trial is a randomized, Phase III, multi-center, double-blind, placebo-controlled trial evaluating the safety and efficacy of oral darolutamide in patients with nmCRPC who are currently being treated with ADT as standard of care and are at high risk for developing metastatic disease. 1,509 patients were randomized in a 2:1 ratio to receive 600 mg of darolutamide twice a day or placebo along with ADT.

The primary endpoint of this trial is MFS defined as time between randomization and evidence of metastasis or death. The secondary endpoints of this trial are OS, time to pain progression, time to initiation of first cytotoxic chemotherapy, time to first SSE, and characterization of the safety and tolerability of darolutamide.

About castration-resistant prostate cancer (CRPC)
Prostate cancer is the second most commonly diagnosed malignancy in men worldwide.3 In 2018, an estimated 1.2 million men were diagnosed with prostate cancer, and about 358,000 died from the disease worldwide.3 Prostate cancer is the fifth leading cause of death from cancer in men.3 Prostate cancer results from the abnormal proliferation of cells within the prostate gland, which is part of a man’s reproductive system.4 It mainly affects men over the age of 50, and the risk increases with age.5 Treatment options range from surgery to radiation treatment to therapy using hormone-receptor antagonists, i.e. substances that stop the formation of testosterone or prevent its effect at the target location.6 However, in nearly all cases, the cancer eventually becomes resistant to conventional hormone therapy.7
CRPC is an advanced form of the disease where the cancer keeps progressing even when the amount of testosterone is reduced to very low levels in the body. The field of treatment options for castration-resistant patients is evolving rapidly, but until recently, there have been no effective treatment options for CRPC patients who have rising PSA levels while on ADT and no detectable metastases. In men with progressive nmCRPC, a short PSA doubling time has been consistently associated with reduced time to first metastasis and death.8

About darolutamide
Darolutamide is an investigational, non-steroidal androgen receptor antagonist with a chemical structure that binds to the receptor and exhibits antagonistic activity, thereby inhibiting the receptor function and the growth of prostate cancer cells. In addition to the Phase III trial ARAMIS in men with nmCRPC, darolutamide is also being investigated in a Phase III study in metastatic hormone-sensitive prostate cancer (ARASENS). Information about these trials can be found at www.clinicaltrials.gov.

On February 14, 2019 Medicenna Therapeutics Corp. ("Medicenna" or the "Company") (TSX: MDNA; OTCQB: MDNAF), a clinical stage immuno-oncology company, reported financial results for the three and nine months ended December 31, 2018 (Press release, Medicenna Therapeutics, FEB 14, 2019, View Source [SID1234533326]).

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The following are the achievements and highlights for the quarter ending December 31, 2018 through to the date hereof:

Presented promising survival data from the ongoing MDNA55 Phase 2b clinical trial for the treatment of recurrent glioblastoma ("rGBM") the most common and uniformly fatal form of brain cancer showing a median overall survival of 15.2 months in the IL4 receptor positive patients who have a more aggressive form of rGBM. Whereas the current standard of care for all rGBM patients provide a median overall survival of only 8.0 months.
Strengthened the balance sheet with a $4.0 million financing in December 2018 and received an additional US$1.2 million in non-dilutive funding in the same month.
Provided an update on MDNA109 demonstrating that it has best-in-class potency toward cancer killing effector T cell and that it potently synergizes with anti-PD-1 or anti-CTLA-4 checkpoint inhibitors to eliminate tumors in the majority of tumor-bearing mice.
Upcoming milestones include:

Completion of patient enrolment in the MDNA55 Phase 2b rGBM is anticipated by the end of Q1 2019
Top line interim results announced in the MDNA55 Phase 2b clinical trial mid-2019
Initiation of Phase 2 clinical trial with MDNA55 for the treatment of newly diagnosed GBM in Q2 2019
Selection of lead MDNA109 candidate in Q1 2019
"2019 is off to an excellent start with compelling median overall survival data in the MDNA55 Phase 2b clinical trial for the treatment of recurrent glioblastoma reported in February as well as the impressive pre-clinical package we are assembling for MDNA109," said Dr. Fahar Merchant, President and CEO of Medicenna. He added that "the clinical and scientific progress we have made in addition to the capital raised and non-dilutive funds received in December has placed Medicenna on an excellent footing to hit key value inflection milestones for 2019."

MDNA55 update
On February 7, 2019 Dr. John H. Sampson, MD, PhD, (Robert H. and Gloria Wilkins Distinguished Professor and Chair of Neurosurgery at Duke University in Durham, NC) presented new clinical study results on MDNA55 for the treatment of rGBM at the 5th Annual Immuno-Oncology 360o Conference held in New York, NY. In a podium presentation entitled, "The IL4 Receptor as a Biomarker and Immunotherapeutic Target for Glioblastoma: Preliminary Evidence with MDNA55, a Locally Administered IL-4 Guided Toxin", Dr. Sampson outlined that following a single treatment with MDNA55 at the low dose, (a) the IL4R positive group showed a remarkable increase in median overall survival ("mOS") of 15.2 months when compared to 8.5 months in the IL4R negative group and (b) irrespective of IL4R expression, mOS was 11.8 months in all patients, substantially exceeding landmark mOS reported for approved drugs for rGBM (mOS is 8 months for Avastin and Lomustine).

On October 22, 2018, the Company presented results and participated in a poster discussion session at the European Society for Medical Oncology Congress held in Munich on October 20, 2018. Based on interim data from patients treated at low doses implemented during the first half of the Phase 2b study of MDNA55, the presentation highlighted the benefits of using of advanced imaging modalities in order to help tumor response evaluation and identify pseudo-progression in some patients which ultimately translates into tumor shrinkage, and potential treatment benefit.

On November 16, 2018, Medicenna presented an update on intratumoral delivery of MDNA55 using MRI-guided convective delivery at the 23rd Annual Meeting of the Society for Neuro-Oncology

MDNA109 update
On February 6, 2019 Dr. Moutih Rafei, PhD, (Associate Professor, Department of Pharmacology and Physiology, Université de Montreal) presented new results on MDNA109 and its long acting variants at the 5th Annual Immuno-Oncology 360o Meeting held in New York, NY. In a podium presentation entitled, "Putting Pedal to the Metal: Combining IL-2 Superkine (MDNA109) with Checkpoint Inhibitors." The presentation outlined that MDNA109, an engineered IL-2 superkine exhibited a 1000-fold enhanced affinity toward the CD122 receptor, has best-in-class potency toward cancer killing effector T cells, was not immunogenic in-vivo and potently synergized with anti-PD-1 or anti-CTLA-4 checkpoint inhibitors to eliminate tumors in the majority of tumor-bearing mice.

On November 9, 2018, Medicenna presented an update on preliminary pre-clinical results on MDNA109 at the 33rd Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) held in Washington, DC.

Operational update
On December 21, 2018, the Company completed a public offering and issued 4,000,000 units of the Company for gross proceeds of CDN$4,000,000.

On December 5, 2018, Medicenna received a US$1.2 million reimbursement of past expenses from the Cancer Prevention and Research Institute of Texas ("CPRIT").

Financial Results
For the three months ended December 31, 2018, Medicenna reported a net loss of $1,723,081 or $0.07 per share compared to a loss of $2,181,022 or $0.09 per share for the three months ended December 31, 2017. For the nine months ended December 31, 2018, Medicenna reported a net loss of $3,658,957 or $0.15 per share compared to a loss of $6,154,946 or $0.25 per share for the nine months ended December 31, 2017.

Research and Development Expenses
Research and development ("R&D") expenses of $2,356,683 were incurred during the nine months ended December 31, 2018, compared with $4,226,141 incurred in the nine months ended December 31, 2017 and were $1,275,896 during the three months ended December 31, 2018, compared with $1,351,703 incurred in the three months ended December 31, 2017. The decrease in the expenses in the current year periods can be primarily attributed to reduced discovery and pre-clinical expenses due to work ongoing and completed in the prior year related to the development of MDNA57as well as lower clinical trial costs due to reduced consulting costs, clinical supplies and CRO fees due to nearing the end of the clinical study and general cost containment. Finally, the variance in recoveries from CPRIT contributed to the changes in net loss of $905,585 in the three months and $3,824,293 in the nine months ended December 31, 2018 compared with $1,884,820 in the three months and $3,334,424 in the nine months ended December 31, 2017

General and Administrative Expenses
General and administrative ("G&A") expenses of $437,218 were incurred in the three months and $1,295,132 in the nine months ended December 31, 2018, compared with $824,007 in the three months and $1,894,230 in the nine months ended December 31, 2017. The decrease in G&A expenses period over period is attributed primarily to lower stock based compensation costs due to timing of grants as well as a lower number of option grants in the current year periods, reduced legal expenses in the current year periods due to expenses related to the graduation from the TSXV to TSX as well as the OTC listing incurred in the prior year periods and lower salary and benefit costs due to headcount reductions and a bonus accrual in the prior year and no comparable accrual in the current year periods

On February 14, 2019 Advaxis, Inc. (NASDAQ:ADXS), a late-stage biotechnology company focused on the discovery, development and commercialization of immunotherapy products, reported it has initiated its Phase 1/2 clinical trial to evaluate ADXS-503, part of the Company’s ADXS-HOT program, in the treatment of non-small cell lung cancer (NSCLC) and has enrolled the first patient in the trial (Press release, Advaxis, FEB 14, 2019, View Source [SID1234533325]). ADXS-HOT is a cancer-type specific immunotherapy program which leverages Advaxis’ proprietary Lm technology platform to target hotspot mutations that commonly occur in specific cancer types as well as other proprietary, tumor-associated antigens. To date, more than 10 drug candidates have been designed for different tumor types under the ADXS-HOT program.

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"Coming on the heels of the presentation of data from our immunotherapy platform at the I/O 360 conference last week, we are excited to announce the enrollment of the first ADXS-HOT patient in our Phase 1/2 trial evaluating ADXS-503 for the treatment of NSCLC. The data presented at I/O 360 suggest that our neoantigen-directed constructs from both our ADXS-NEO and ADXS-HOT programs have the potential to demonstrate best-in-class CD+8 T cell response for neoantigen therapies," said Kenneth A. Berlin, President and Chief Executive Officer of Advaxis. He continued, "We believe our ADXS-HOT drug constructs have significant advantages compared to certain other neoantigen-focused therapies in development as our ADXS-HOT drug constructs are off-the-shelf and immediately available to administer to the patient and have a favorable cost to manufacture." He concluded, "The ADXS-HOT program supports our goal of leveraging the significant experience we have gained from our proprietary platform in order to advance programs in the fight against cancer for patients in need of new therapeutic options and we look forward to studying these constructs in the clinic."

The Phase 1/2 clinical trial for ADXS-503 will seek to establish the recommended dose, safety, tolerability and clinical activity of ADXS-503 administered alone (initially) and in combination with a checkpoint inhibitor in approximately 50 patients with NSCLC in different lines of therapy, at up to 20 centers across the U.S. Advaxis anticipates initial data from the first cohort of ADXS-503 in the first half of 2019.

About ADXS-HOT

ADXS-HOT is a program that leverages the Company’s proprietary Lm technology to target hotspot mutations that commonly occur in specific cancer types. ADXS-HOT drug candidates are designed to target acquired shared or "public" mutations in tumor driver genes along with other proprietary cancer-testes and oncofetal tumor-associated antigens that also commonly occur in specific cancer types. ADXS-HOT drug candidates are an off-the-shelf treatment, designed to potentially treat all patients with a specific cancer type, without the need for pretreatment biomarker testing, DNA sequencing or diagnostic testing.