On March 19, 2019 OncBioMune Pharmaceuticals, Inc. (OTCQB:OBMP) ("OncBioMune" or the "Company"), a clinical-stage biopharmaceutical company engaged in the development of a proprietary therapeutic prostate cancer vaccine immunotherapy and CD71-targeted cancer therapy, reported that the Phase 2, randomized study of ProscaVax in treatment-naïve patients with clinically localized prostate cancer vs active surveillance is now open for enrollment (Press release, Oncbiomune, MAR 19, 2019, View Source [SID1234534458]).

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The first open site is Beth Israel Deaconess Medical Center, a member of the Dana-Farber/Harvard Cancer Center and a teaching hospital of Harvard Medical School in Boston, MA.

Although many patients with low-risk localized prostate cancer can be safely monitored with an Active Surveillance strategy in lieu of up-front treatment with surgery or radiation, about half of these patients will ultimately have to undergo treatment due to worsening of their disease. Thus, patients with localized prostate cancer need improved therapies to help prevent worsening of their disease and avoid the side effects of surgery or radiation. This study will evaluate the safety and efficacy of ProscaVax (Prostate-specific antigen (PSA) / Interleukin-2 (IL-2) / Granulocyte-macrophage colony-stimulating factor (GM-CSF)), the Company’s flagship therapeutic cancer vaccine, in patients with localized prostate cancer compared to patients monitored on Active Surveillance.

"In general, this population of patients have two very different options. Either go into active surveillance waiting to see if there is progression of their cancer or have intensive local therapy, which can leave these patients with significant side effects. The goal which we are trying to obtain with this immunotherapy approach is to give these patients a third option which has more intervention than Active Surveillance but with less toxicity than surgery, radiation therapy, or hormonal therapy," commented Dr. Brian Barnett, Chief Executive Officer at OncBioMune. "Phase 1 data underscores a strong safety profile for ProscaVax, while potentially moderating disease progression as measured by PSA doubling time. The start of this study represents a major milestone for our company."

The Phase 2 study (randomized 2:1, treatment arm:control group) will enroll 120 adult patients with clinically localized prostate cancer who have received no prior therapy for prostate cancer. For the purpose of this study the primary endpoint will be an analysis of the number and percent of patients with progression at two years in each arm. Statistical assumptions are a 35% progression rate within two years in the control arm and decrease to 15% progression rate in the treatment arm.

On March 19, 2019 TESARO, an oncology-focused business acquired by GlaxoSmithKline plc (LSE/NYSE: GSK), reported the presentation of data from the Phase 1/2 GARNET study evaluating dostarlimab in women with recurrent or advanced endometrial cancer who progressed on or after a platinum-based regimen (Press release, GlaxoSmithKline, MAR 19, 2019, View Source [SID1234534481]). These data were presented at the 2019 Society for Gynecologic Oncology (SGO) Annual Meeting on Women’s Cancer in Honolulu, Hawaii. The preliminary results demonstrate clinically meaningful and durable response rates of dostarlimab (anti-PD-1 antibody, formerly TSR-042), in this patient population, regardless of microsatellite instability status. In addition, the safety findings indicate that dostarlimab is well tolerated with a safety profile consistent with what is expected of anti-PD-1 therapy.

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Further data from the GARNET study will be analysed using the RECIST 1.1 criteria to support regulatory filing for dostarlimab in endometrial cancer at the end of 2019.

Mary Lynne Hedley, Ph.D., President and Chief Operating Officer of TESARO, said, "Currently, treatment options for women with advanced or recurrent endometrial cancer are limited, with only one FDA-approved agent for a subset of these patients. We intend to use these and other data from the GARNET study to seek regulatory approval of dostarlimab to potentially address the critical unmet treatment needs of women whose disease has progressed. The data presented today evaluating dostarlimab in women with recurrent/advanced endometrial cancer, combined with earlier data in patients with non-small cell lung cancer, reinforces the potential of dostarlimab in treating patients with a variety of solid tumours."

Endometrial cancer (EC) is the most common gynaecologic malignancy in the U.S. There are limited treatment options for women whose disease progresses on or after first-line therapy. EC can be classified as microsatellite stable (MSS/75%) or microsatellite instability-high (MSI-H/25%). Currently, there is only one approved therapy in the recurrent EC setting for the subset of patients with MSI-H tumours, and no approved treatments for patients with MSS EC who have recurred after platinum-based chemotherapy.

Key Study Findings
A total of 125 patients were analysed including 41 MSI-H (33%), and 79 MSS (63%) patients, as well as 5 with an unknown MSI-status (4%). Dostarlimab was dosed at 500 mg once every 3 weeks for 4 doses, followed by 1000 mg once every 6 weeks until disease progression.

Determination of MSI status was made based upon a central test using next generation sequencing (NGS). Treatment with dostarlimab monotherapy resulted in a clinically meaningful response rate in recurrent or advanced EC who progressed on or after a platinum-based regimen, regardless of MSI status. Overall response rates by irRECIST in the full population, MSI-H population, and MSS population were 30%, 49%, and 20%, respectively. Disease control rate in the full population, MSI-H population, and MSS population was 53%, 63% and 47%, respectively.

At the time of data cutoff, treatment was still ongoing in 84% of responders, with 89% of the responders (33 of 37) having been on treatment for more than six months and 49% of responders (18 of 37) having been on treatment for more than one year. Durability of response was similar between the MSI-H and MSS cohorts. The median duration of response (DOR) has not yet been reached. All responses were assessed using irRECIST criteria.

The data show that 88 out of 125 (70.4%) patients had at least 1 treatment-emergent adverse event (TEAE). The most commonly reported TEAEs related to dostarlimab were fatigue (14.4%), diarrhea (12.8%), and nausea (12.0%). In general, adverse events were low grade, with only 13.6% of patients experiencing grade 3 or higher adverse events. No deaths occurred due to a treatment-related adverse event. In total, 5.6% of all patients experienced a grade 3 or higher immune related, treatment-related adverse event.

About GARNET
The ongoing Phase 1/2 GARNET trial is evaluating dostarlimab as monotherapy in patients with advanced solid tumours. GARNET included a weight-based dose escalation study (Part 1) and a fixed-dose safety study (Part 2A), both of which have been completed. Results of these studies were used to determine the recommended Phase 2 dose (RP2D; 500 mg Q3W for the first 4 cycles then 1000 mg Q6W). Part 2B of the study includes four expansion cohorts: MSI-H endometrial cancer, MSI-H non-endometrial cancer, MSS endometrial cancer, and non-small cell lung cancer (NSCLC). Data from the NSCLC cohort have been previously presented at Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) in 2018.

About dostarlimab
Dostarlimab (TSR-042) is an investigational humanized anti-programmed death (PD)-1 monoclonal antibody that binds with high affinity to the PD-1 receptor and effectively blocks its interaction with the ligands PD-L1 and PD-L2. If approved, dostarlimab would be the first anti PD-1 therapy administered as monotherapy every 3 weeks for 4 doses then every 6 weeks thereafter. Dostarlimab was developed as part of a collaboration between TESARO and AnaptysBio, Inc. This collaboration was initiated in March of 2014, and is focused on the development of monospecific antibody drugs targeting PD-1, TIM-3 (TSR-022), and LAG-3 (TSR-033), in addition to a bi-specific antibody drug candidate targeting PD-1/LAG-3 (TSR-075).

Dostarlimab is not currently approved for use anywhere in the world.

On March 19, 2019 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, reported financial results and business highlights for the fourth quarter and full year ended December 31, 2018 (Press release, Magenta Therapeutics, MAR 19, 2019, View Source [SID1234534504]).

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"2018 was a transformative year for Magenta, as we progressed our first-in-class programs and achieved multiple clinical and preclinical milestones," said Jason Gardner, D.Phil., Chief Executive Officer and President, Magenta Therapeutics. "As we begin 2019, Magenta is the only company addressing the major barriers to stem cell transplant and gene therapy, with the goal of changing the lives of patients with autoimmune diseases, blood cancers and genetic diseases through curative therapies. We are looking forward to building on this progress as we advance our conditioning, mobilization and cell therapy programs."

Upcoming Anticipated Milestones:

The Company plans to achieve the following key milestones in 2019:

Present preclinical data on C100 anti-CD45 targeted conditioning program in autoimmune diseases and declare development candidate

Present preclinical data on C200 anti-CD117 targeted conditioning in gene therapy, and advance development candidate

Begin Phase 1 study of MGTA-145 first-line mobilization agent in healthy volunteers in the first half of 2019, and present clinical data in the second half of 2019

Present additional clinical data from the Phase 2 study of MGTA-456 in IMDs

Recent Business Highlights:

Updated preclinical data for C100 conditioning program showed potent depletion of hematopoietic stem cells and immune cells: At the Transplant and Cellular Therapy (TCT) meeting in February 2019, Magenta presented data from its C100 targeted conditioning program, showing potent stem and immune cell depletion with an anti-CD45 amanitin antibody-drug conjugate (ADC) that was well tolerated at efficacious doses in non-human primates. The Company expects to declare a development candidate for this program in 2019 and intends to develop C100 in both autoimmune diseases and blood cancers.

Declared development candidate in C200 targeted conditioning program and presented preclinical data: At the end of 2018, Magenta declared a development candidate in its C200 targeted conditioning program, which is designed to deplete stem cells in the bone marrow. The Company presented data at the TCT meeting in February 2019 on the development candidate, an anti-CD117 amanitin ADC, showing that it potently and selectively depleted hematopoietic stem cells in non-human primates while preserving the immune system. The ADC was well tolerated at the efficacious doses. Magenta has begun investigational new drug (IND)-enabling studies with this ADC and plans to develop it as a conditioning agent for stem cell gene therapy, in patients with genetic disorders such as sickle cell disease, where current conditioning regimens are toxic.

Updated preclinical data for MGTA-145 first-line mobilization therapy showed differentiated efficacy from standard of care: In data presented at the TCT meeting in February 2019, Magenta showed that a single dose of MGTA-145 plus plerixafor mobilized two to three times more stem cells in non-human primates than a multi-day regimen of current standard of care, G-CSF. The cells mobilized with MGTA-145 plus plerixafor rapidly engrafted in non-human primates following autologous transplant. A subset of the MGTA-145-mobilized cells from non-human primates was also shown to suppress graft-vs.-host-disease and extend survival in preclinical models. The company expects to initiate a Phase 1 study of MGTA-145 in the first half of 2019 and share clinical results in the second half of 2019.

Updated clinical data for MGTA-456 cell therapy showed early signs of clinical benefit in IMDs: Magenta presented updated data from the Phase 2 clinical study of MGTA-456 in patients with IMDs at the TCT meeting in February 2019. Patients with cerebral adrenoleukodystrophy (cALD) treated with MGTA-456 in the study showed persistent resolution of brain inflammation and stable disease scores. Patients with Hurler syndrome treated with MGTA-456 showed correction of enzyme deficiency and decrease in toxic metabolites. These early clinical benefits are associated with improved long-term disease outcomes in patients undergoing stem cell transplant for IMDs. The Company will next present data from this study at the American Academy of Neurology (AAN) annual meeting in May. A Phase 2 investigator-initiated study of MGTA-456 in blood cancers began in December 2018. After careful review of comprehensive transplant outcomes data in sickle cell disease, the Company will evaluate development of MGTA-456 in sickle cell disease as less toxic conditioning becomes available.

Fourth Quarter Financial Results:

Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2018, were $142.6 million compared to $51.4 million on December 31, 2017. The increase is primarily driven by net proceeds from the $52.2 million Series C preferred stock financing completed in April 2018, and net proceeds of $89.9 million from Magenta’s IPO completed in June 2018, offset by $57.6 million in net loss during 2018. Magenta anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund operations and capital expenditures through 2020 on the Company’s current business plan.

Research and Development Expenses: Research and development (R&D) expenses were $12.4 million in the fourth quarter of 2018, compared to $5.6 million for the same period in 2017. The increase was primarily due to increased costs related to drug discovery efforts in our conditioning programs, preclinical costs, toxicology studies and manufacturing to support our mobilization program, the advancement of the MGTA-456 Phase 2 clinical trial, continued progression of the Company’s pipeline and increased costs associated with the growth of the Company.

General and Administrative Expenses: General and administrative (G&A) expenses were $5.5 million for the fourth quarter of 2018, compared to $2.6 million for the same period in 2017. The increase was primarily due to increased G&A personnel and facility costs associated with the growth of the Company.

Net Loss: Net loss was $16.7 million for the fourth quarter of 2018, compared to net loss of $8.0 million for the same period in 2017.

On March 19, 2019 Context Therapeutics, a clinical-stage biotechnology company focused on hormone driven cancers, reported it will present at the following upcoming investor conferences (Press release, Context Therapeutics, MAR 19, 2019, View Source [SID1234534482]).

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Needham and Company 18th Annual Health Care Conference in New York City on Tuesday, April 9, 2019 at 2:30 p.m. ET.

On March 19, 2019 VBL Therapeutics (Nasdaq: VBLT), reported the presentation of human data indicating that the viral anti-cancer investigational therapy VB-111 has potential to stimulate the immune system to induce a strong and durable response against ovarian tumors (Press release, VBL Therapeutics, MAR 19, 2019, View Source [SID1234534461]). Administration of VB-111 was associated with an infiltration of immune cells within the tumor, which was followed by tumor necrosis and sustained clinical response. VBL’s study entitled "VB-111, an anti-cancer gene therapy, induces immunotherapeutic effect in platinum resistant ovarian cancer" was presented last evening at the SGO 50th Annual Meeting on Women’s Cancer at the Hawaii Convention Center in Honolulu, Hawaii. The presentation was delivered by Dr. Ronnie Shapira-Frommer from Sheba Medical Center, Tel Aviv and Dr. Richard Penson from Massachusetts General Hospital, Boston.

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Ofranergene obadenovec (VB-111) is a targeted anti-cancer investigational gene therapy with a dual mechanism: disruption of the tumor vasculature and induction of an anti-tumor directed immune response. In a Phase 2 clinical trial, VB-111 in combination with chemotherapy (paclitaxel) prolonged survival in patients with recurrent platinum resistant ovarian cancer (median OS 498 days for therapeutic dose VB-111 + paclitaxel combination therapy, compared to 172 days for low dose VB-111 + paclitaxel (p=0.03)). OVAL, VBL’s ongoing pivotal Phase 3 trial, is designed to repeat the successful Phase 2 protocol, with the primary endpoint of improvement in overall survival. An interim analysis of the OVAL study is expected at year-end 2019.

"Cancer cells utilize various mechanisms to proliferate and survive, including silencing of, and escape from, the immune system. We believe that treatment strategies should therefore aim both to target the cancer cells and to stimulate the immune system to attack the tumor," said Dror Harats, M.D., Chief Executive Officer of VBL Therapeutics. "While we initially designed VB-111 as anti-tumor therapy targeting tumor blood supply, it is now evident that VB-111 can also drive immune cells into immunologically `cold` tumors turning the tumors ‘hot,` enabling the immune system to fight the tumor. These clinical findings provide encouraging evidence of the therapeutic potential of VB-111, as we continue to advance our clinical program in ovarian cancer and additional indications."

For a link to VBL’s poster at the SGO 50th Annual Meeting on Women’s Cancer refer to: SGO Poster

About Ofranergene Obadenovec (VB-111)
VB-111, a potential first-in-class anticancer therapeutic candidate, is the Company’s lead oncology product currently being studied in the OVAL potential-registration Phase 3 pivotal trial for ovarian cancer (ClinicalTrials.gov Identifier: NCT03398655). VB-111 has received orphan drug designation in both the US and Europe, and fast track designation in the US for prolongation of survival in patients with rGBM. In addition, VB-111 successfully demonstrated proof-of-concept and survival benefit in Phase 2 clinical trials in radioiodine-refractory thyroid cancer and recurrent platinum-resistant ovarian cancer (NCT01711970). VB-111 has received an Orphan Designation for the treatment of ovarian cancer from the European Commission.