On December 3, 2018 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported translational data showing that recipients who received NiCord, an investigational cell therapy in Phase 3 clinical development for allogeneic hematopoietic stem cell (bone marrow) transplant had rapid and robust reconstitution of key immune cells (Press release, Gamida Cell, DEC 3, 2018, View Source [SID1234531840]). Successful immune reconstitution is an important factor in the recovery of patients undergoing bone marrow transplant. These translational data from the completed Phase 1/2 study of NiCord were presented in a poster session during the American Society of Hematology (ASH) (Free ASH Whitepaper) 2018 Annual Meeting being held December 1-4 in San Diego, CA.1

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"Immune reconstitution following transplantation is critical for disease and viral control, but historically cord blood transplantation has had limitations in timely immune reconstitution in patients," said Jaap-Jan Boelens, M.D., Ph.D., Chief, Pediatric Stem Cell Transplantation and Cellular Therapies Service, Memorial Sloan Kettering Cancer Center. "We were pleased to see that NiCord treatment resulted in rapid and robust immune reconstitution when compared to younger patients who typically achieve more rapid recovery than adults."

Despite the curative potential of bone marrow transplants, it is estimated that more than 40 percent of eligible patients in the U.S. do not receive one for various reasons, including finding a matched donor.2 While umbilical cord blood provides a source of stem cells for patients who do not have a matched related donor, it provides a smaller number of stem cells, which can delay engraftment and put patients at a greater risk for prolonged hospitalizations and life-threatening infections. NiCord is designed to address these limitations by offering a therapeutic dose of expanded cells while preserving the functional characteristics of stem cells.

Data Presented at ASH (Free ASH Whitepaper) 2018
The poster presentation, "Rapid and robust CD4+ and CD8+ T-, NK-, B- and monocyte cell reconstitution after nicotinamide-expanded cord blood transplantation" (Abstract 2123), described early, in-depth immune reconstitution data from the completed Phase 1/2, multicenter clinical study of NiCord as a stand-alone graft after myeloablative therapy in patients with high-risk hematologic malignancies.3 A random subgroup of 27 patients from this study had extensive immune monitoring evaluated throughout the first year after transplant. The primary endpoint was the probability of achieving CD4+ immune reconstitution (>50×106/L) within the first 100 days, and the secondary endpoints included the recovery of B cells, CD4+ T cells and natural killer (NK) cells during the first year after transplantation. These data were compared to cohorts of adolescent and young adults with hematologic malignancies receiving unmanipulated cord blood transplantation (n=27) or unrelated bone marrow transplantation (n=20). Analyses were performed at the University Medical Centre Utrecht, Laboratory of Translational Immunology.

Key findings from the analysis include the following:

91 percent of patients achieved successful immune reconstitution of CD4+ T cells at 100 days after transplantation with NiCord.
Immune reconstitution of T cells was similar in the NiCord group (median age: 41.5 years) compared to the younger cohorts receiving unmanipulated cord blood and unrelated bone marrow (median ages 15.4 and 14.3 years, respectively).
Immune reconstitution of B cells (p = 0.02) and NK cells (p < 0.001) was significantly faster after transplantation with NiCord compared to the other groups.
Immune reconstitution after NiCord transplantation was associated with recovery of a broad spectrum of T cell, B cell and NK cell subsets representing a range of effector functions similar to that observed with other graft sources.
"These data, combined with the clinical data from our Phase 1/2 study of NiCord in patients with high-risk blood cancers, suggest that NiCord has the potential to be an important treatment option for patients undergoing bone marrow transplant," stated Ronit Simantov, M.D., chief medical officer at Gamida Cell. "We are working to advance our NiCord clinical development program and expect to complete patient enrollment in our ongoing Phase 3 study in the second half of 2019."

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 status 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 study evaluating NiCord in patients with leukemia and lymphoma is ongoing in the United States, Europe and Asia.4 For more information on NiCord clinical trials, please visit www.clinicaltrials.gov.

On December 3, 2018 BerGenBio ASA (OSE: BGBIO), a clinical-stage biopharmaceutical company focused on developing a pipeline of first-in-class AXL kinase inhibitors to treat multiple cancer indications, reported that it presented a comprehensive analysis of monotherapy data from its BGBC003 clinical trial (NCT02488408) with selective AXL inhibitor bemcentinib in patients with relapsed/refractory Acute Myeloid Leukaemia (AML) or high-risk Myelodysplastic Syndrome (MDS) (Press release, BerGenBio, DEC 3, 2018, View Source [SID1234531807]).

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In a poster presentation at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (ASH) (Free ASH Whitepaper) in San Diego entitled: "Comprehensive Analysis of the Dose Escalation, Expansion and Correlates in the Ph I/II Trial BGBC003 with the Selective Oral AXL Inhibitor Bemcentinib (BGB324) in Relapsed/Refractory AML and MDS", Professor Sonja Loges, attending physician at the University Hospital in Hamburg-Eppendorf and lead investigator of the BGBC003 trial, detailed the following:

14 of 26 (54%) of patients found to be AXL positive (denoted by low serum AXL, sAXL, levels at start of treatment).
Response rate of 43% CR/Cri/CRp to bemcentinib monotherapy in AXL positive patients and 22% overall.
Mild and manageable side effect profile with a low incidence of Grade 3/4 events and low incidence of haematological toxicity.
Furthermore, the Company announces that enrolment is complete into the phase II combination cohort of bemcentinib and decitabine in first line AML. Analysis of the activity of the combination will be submitted for presentation at a future medical congress.

Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "I am very encouraged by the data presented today, we are consistently seeing AXL positive patients report a superior response to bemcentinib therapy alone or in combination with standard of care therapy. The value of our biomarkers to identify patients most likely to benefit from bemcentinib is tremendous. Based on our clinical data, more than 50% of AML/MDS patients are AXL positive, of which 43% reported complete response within the first few weeks of starting bemcentinib monotherapy treatment. Bemcentinib is very well tolerated by patients as a single agent and in combination with other drugs, which is an important feature supporting bemcentinib’s broad utility. We are making excellent progress recruiting the chemotherapy combination arms of this study and look forward to reporting the data from these patients in the coming months".

END

About AML
AML is the most common form of acute leukaemia in adults where malignant AML blasts interfere with the normal functioning of the bone marrow leading to a multitude of complications like anaemia, infections and bleeding. AML is diagnosed in over 20,000 patients in the US annually and is rapidly lethal if left untreated. Successful treatment typically requires intensive therapy or bone marrow transplantation, and relapse and resistance are common. Consequently, there is an urgent need for effective novel therapies in relapsed/refractory patients, particularly those that are ineligible for intensive therapy or bone marrow transplant.

About the BGBC003 trial
The BGBC003 trial is a phase Ib/II multi-centre open label study of bemcentinib as a single agent in patients with AML or high risk MDS or in a combination with cytarabine and decitabine in AML patients. Up to 75 patients will be enrolled at centres in the US, Norway, Germany and Italy.

For more information please access trial NCT02488408 at www.clinicaltrials.gov.

About the 60th ASH (Free ASH Whitepaper) Annual Meeting in San Diego, California
The 60th American Society of Haematologist Annual Meeting and Exposition is the most comprehensive haematology conference worldwide and attracts more than 25,000 haematology professionals from every subspecialty.

On December 3, 2018 Merck (NYSE: MRK), known as MSD outside the United States and Canada, reported that Robert M. Davis, chief financial offer and executive vice president, Merck Global Services, is scheduled to present at Citi’s 2018 Global Healthcare Conference in New York on Dec. 5, 2018 at 8:45 a.m. EST (Press release, Merck & Co, DEC 3, 2018, View Source [SID1234531824]).

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Investors, analysts, members of the media and the general public are invited to listen to a live audio webcast of the presentation at View Source

On December 3, 2018 Genprex, Inc. (NASDAQ: GNPX), a clinical-stage gene therapy company developing a new approach to treating cancer based upon a novel proprietary technology platform, reported that its Chairman and Chief Executive Officer, Rodney Varner, will present at the 11th annual LD Micro Main Event at the Luxe Sunset Boulevard Hotel in Los Angeles, CA (Press release, Genprex, DEC 3, 2018, View Source [SID1234531841]).

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Genprex is scheduled to present on Wed., Dec. 5, 2018, at 10:30 a.m. PST. Genprex management will also attend one-on-one meetings with institutional investors throughout the day.

The LD Micro Main Event is the largest independent conference for small/micro-cap companies and will feature 250 companies presenting to an audience of over 1,200 attendees.

On December 3, 2018 Shanghai, Generon BioMed Holding Ltd (Generon), reported at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting the pre-clinical study results of two novel immunotherapies, A-319 (mono-valent) and A-329 (bi-valent), two CD19×CD3 bi-specific antibodies designed using Generon’s ITab platform (Press release, Generon (Shanghai), DEC 3, 2018, View Source [SID1234531890]). A series of studies were conducted to evaluate the bioactivities of A-319 and A-329 in vitro and in vivo. The in vitro data showed that the mono-valent and bi-valent CD19 binding had little effect on the CD3-associated activities including CD3 antigen binding affinity, T cell binding, and T cell activation. In contrast, the bi-valent binding format of A-329 showed better potency compared to the mono-valent format of A-319 in CD19 binding (KD 0.89 nM and 19.4 nM respectively) and in vitro human B cell killing (EC50 0.2 pM and 3.4 pM, respectively). Both A-319 and A-329 were capable of mediating tumor cell lysis with EC50 at 0.03~4.0 pM. A-329 demonstrated a greater killing activity on B cell lines with a low expression of CD19 antigen. In addition, The CD19 bi-valent format of A-329 revealed more B cell killing in monkeys. No significant differences of cytokine induction or liver injuries between A-319 and A-329 were observed. In human PBMC engrafted mouse models, in vivo efficacies of both formats on inhibiting tumor growth or improving animal survival rate were also confirmed. These results demonstrated that both A-319 and A-329 may benefit patients with B cell malignancies with less frequent dosing and lower levels of cytokine inductions than comparator therapies. A-329 especially has the potential to eliminate low CD19 expressing tumor stem cells.

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Dr. David Lacey, chairman of Generon’s scientific advisory board, commented, "The prosecution of A-319 and A-329 ITabs targeting underscores the broadening of Generon’s portfolio into the immune-oncology space. The inherent flexibility of the ITab design and the competitive attributes of its manufacture make this bi-specific platform particularly attractive for CD19 as well as future targets."

Dr. Xiao Qiang Yan, CEO and CSO of Generon, commented, "Targeting CD19 to treat patients with B cell malignancies remains a substantial unmet medical need. Generon has been focusing on developing CD3-activating bi-specific ITabs with the goal to serve patients better through more convenient dosing, improved efficacy and reduced side-effects. The preclinical data suggest that A-319 and A-329 may have unique advantages over other technologies or therapies targeting CD19".

About A-319 and A-329

A-319 and A-329 are T cell activating bi-specific antibodies (BsAb) designed to target CD19 and CD3 (anti-CD19, anti-CD3) and are under development for the treatment of patients with B cell malignancies including B cell leukemia and B cell lymphoma. Both A-319 and A-329 activate T lymphocytes in a patient to kill CD19 expressing malignant B-cells, and A-329 may also activate T lymphocytes in a patient to kill low CD19 expressing malignant B cells. A-319 was recently approved by SFDA to initiate a phase I study in patients with B cell malignancies in China.