On January 6, 2017 Rgenix, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics using its unique target discovery platform, reported treatment of the first patient with its lead candidate RGX-104 in a Phase 1a/b study (Press release, Rgenix, JAN 6, 2017, View Source [SID1234517299]). The 80-patient study consists of an initial dose-escalation stage involving patients with advanced solid malignancies and lymphoma, in which an optimal biological dose is being defined using pharmacokinetic, pharmacodynamic, biomarker and safety studies. This will be followed by an expansion stage in at least four cancer types, in which detection of preliminary efficacy signals will be determined.

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RGX-104 is an orally administered small molecule that selectively targets the nuclear receptor LXR to activate the expression of the ApoE protein that becomes silenced in human cancers as they grow, become invasive, and metastasize. ApoE silencing is related to reduced survival in cancer patients. The LXR-ApoE pathway was originally identified as a cancer target using a novel microRNA-based discovery platform developed by Rgenix’s scientific co-founders at The Rockefeller University who demonstrated that the pathway regulates the ability of cancers to evade the immune system and recruit blood vessels.

Rgenix’s scientists showed that RGX-104 reverses the immune suppressive and pro-angiogenic effects of cancers by potently activating ApoE expression in cancer and highly specific circulating immune cells, leading to the elimination of these immunosuppressive cells and blood vessels in the tumor microenvironment. RGX-104 treatment confers robust anti-cancer activity and suppression of metastases in a wide range of well-established animal models of multiple human cancer types, including drug-resistant malignancies. In addition to its profound and diverse actions as a single agent, RGX-104 blocks resistance to immune checkpoint inhibitors, providing a rationale to combine RGX-104 with these therapies in patients who either do not initially respond to immune checkpoint inhibitor therapy alone, or those who at first benefited, but soon after developed resistance. RGX-104 was granted Orphan Drug Designation by the FDA for the treatment of several cancer types of high unmet need.

"RGX-104 represents the first LXR-ApoE targeting drug in clinical development for cancer," said Masoud Tavazoie, MD, PhD, Chief Executive Officer and co-founder of Rgenix. He added, "Because of its ability to inhibit tumor growth and metastasis as a single agent in drug-resistant cancer types via a novel mechanism, RGX-104 has the potential to transform the treatment landscape for several common cancers types, addressing urgent unmet needs in oncology."

Dr. Michael Postow, a member of the Melanoma and Immunotherapeutics Service at Memorial Sloan Kettering Cancer Center, one of the RGX-104 study sites, commented, "We are excited about RGX-104’s potential to treat a broad array of cancers given its unique effect on the immune system and strong pre-clinical activity both as a single agent and in combination with a checkpoint inhibitor."

The same discovery platform used to identify the ApoE pathway in cancer has been used to discover several other key regulators of cancer progression that Rgenix is targeting with first-in-class small-molecule and antibody drug candidates as part of its novel therapeutics pipeline.

For more information about the clinical trial visit: View Source

On January 6, 2017 VBL Therapeutics (NASDAQ:VBLT),reported that it has completed enrollment in the GLOBE Phase 3 study evaluating the efficacy of its lead candidate ofranergene obadenovec (VB-111) in patients with recurrent glioblastoma (rGBM) (Press release, VBL Therapeutics, JAN 6, 2017, View Source [SID1234517297]). Enrollment in the study, 256 patients in total, has been completed five months ahead of schedule.

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"We are very encouraged by the high level of interest shown by both patients and physicians in GLOBE, resulting in completion of enrollment five months ahead of plan," said Professor Dror Harats, M.D., Chief Executive Officer of VBL. "VB-111 has generated positive data in several tumor types, including a statistically significant overall survival benefit in rGBM. We are hopeful that the GLOBE trial will confirm these positive findings and generate the data required to support registration."

VBL has also received FDA approval for adjustments in the GLOBE protocol. These major modifications relate to the triggers for the interim and final analyses. The SPA covering GLOBE remains in place. Originally, the interim analysis in GLOBE was to be conducted after 91 deaths. The modified protocol specifies that it will be conducted after 105 deaths, and after 50% of the patients have more than 12 months potential follow up, whichever occurs later. The final analysis will be conducted at 189 deaths (75% of events), versus the original planned for 151 deaths (60% of events).

These adjustments to the GLOBE protocol are intended to provide better powering and increase the probability for a clearer efficacy signal. Given the fast recruitment pace and completion of recruitment ahead of schedule, the company continues to expect that the interim analysis will occur in mid-2017 and that the top-line results from the full dataset will be available in early 2018.

On January 6, 2017 Sangamo Therapeutics, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, reported that it has changed its corporate name, from "Sangamo BioSciences, Inc." to "Sangamo Therapeutics, Inc." Sangamo common stock will continue to trade on the NASDAQ Global Select Market under the current ticker symbol: "SGMO (Filing, 8-K, Sangamo BioSciences, JAN 6, 2017, View Source [SID1234517296])." The new corporate name underscores Sangamo’s focus on clinical development of genomic therapies using the Company’s industry-leading platform technologies across genome editing, gene therapy, gene regulation and cell therapy.

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"Our new name, Sangamo Therapeutics, reflects our commitment to advance our groundbreaking science into the clinic for the development of transformative therapies for serious, genetically tractable diseases," said Sandy Macrae, M.B., Ch.B., Ph.D., Sangamo’s newly appointed CEO. "Our scientists are pioneers in their fields, and over the last two decades, they have developed the most advanced, flexible and precise tools available for genomic therapies. With new leadership, a flattened organizational structure, overhauled management processes and clear direction from our board, Sangamo now has the focus and capabilities in clinical science and product development to advance this powerful scientific platform into clinical development."
Macrae continued: "In 2017, we expect to enroll four clinical trials for our lead programs developing valuable new therapies for patients living with hemophilia A and B and lysosomal storage disorders MPS I and II. We will also work closely with our collaborator Bioverativ, the planned spin-off of Biogen’s hemophilia business, to advance development of our ZFN-mediated genome editing programs in two related genetic disorders, sickle cell disease and beta thalassemia."
Recent accomplishments

• Appointed Sandy Macrae as president and CEO in June 2016

• Appointed other new senior leaders including a chief medical officer and head of clinical development, a chief business officer, as well as heads of technical operations and manufacturing, product development, and communications. The Company also hired new staff, expanding capabilities in commercial planning, clinical development and medical affairs.
• Received acceptance from the U.S. Food and Drug Administration of the IND application for the AAV Factor 8 cDNA vector program (SB-525) in Hemophilia A

• Established new processes for target evaluation and program progression

• Manufactured and released cGMP materials for all currently planned clinical trials

• Opened recruitment for SB-FIX Phase 1/2 clinical trial for treatment of hemophilia B
Priorities and expectations for 2017

• Enroll Phase 1/2 clinical trials for Sangamo’s four lead programs with data expected potentially in late 2017 or early 2018, once the Company has gathered sufficient quantity of information from each study to understand clinical relevance:

• Hemophilia A: SB-525, AAV Factor 8 cDNA in vivo gene therapy

• Hemophilia B: SB-FIX, in vivo genome editing

• Mucopolysaccharidosis (MPS) I: SB-318, in vivo genome editing

• MPS II: SB-913, in vivo genome editing

• Extend technological advantages of our zinc finger nuclease (ZFN) platform for genome editing

• Advance novel delivery methods, including lipid nanoparticles, toward clinical development

• Work closely with collaborator Bioverativ, the planned spin-off company of Biogen’s hemophilia business, on the development of our ZFN-mediated genome editing programs for two rare blood disorders, sickle cell disease and beta thalassemia
In line with previously stated guidance, Sangamo expects to report a year-end 2016 balance of cash and cash equivalents of at least $140 million, which will fund the Company’s operations beyond the completion of all four Phase 1/2 clinical trials.
Sangamo Therapeutics today has also revealed a new logo and updated website, www.sangamo.com, reflecting the Company’s mission to translate ground-breaking science into genomic therapies that transform patients’ lives.

Sangamo will be participating in the JP Morgan Healthcare Conference being held next week in San Francisco. A presentation at the conference by CEO Sandy Macrae is scheduled for Wednesday, January 11th, 2017 at 4:30 pm Pacific Time. A live webcast of the presentation will be accessible through a link on the Investors + Media section of the company’s website, www.sangamo.com.

On January 6, 2017 Genmab A/S (Nasdaq Copenhagen: GEN) reported that it has achieved the first sales volume milestone in its DARZALEX (daratumumab) collaboration with Janssen. The USD 25 million milestone was triggered by sales of DARZALEX reaching USD 500 million in a calendar year (Press release, Genmab, JAN 6, 2017, View Source [SID1234517293]). In August 2012, Genmab granted Janssen Biotech, Inc. an exclusive worldwide license to develop, manufacture and commercialize DARZALEX.

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"We have seen an extremely robust launch of DARZALEX since its approval in the U.S in November 2015 and in Europe in May 2016, with very rapid sales uptake. We are very pleased to receive the first commercial sales volume milestone so quickly," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

The milestone payment was included in Genmab’s financial guidance published on December 20, 2016.

About DARZALEX (daratumumab)
DARZALEX (daratumumab) injection for intravenous infusion is indicated in the United States in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a proteasome inhibitor (PI) and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.1 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (FDA) approval to treat multiple myeloma. DARZALEX is indicated in Europe for use as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy. For more information, visit www.DARZALEX.com.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a person’s own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).1,2,3,4,5

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. Five Phase III clinical studies with daratumumab in relapsed and frontline multiple myeloma settings are currently ongoing, and additional studies are ongoing or planned to assess its potential in other malignant and pre-malignant diseases on which CD38 is expressed, such as smoldering myeloma, non-Hodgkin’s lymphoma, NKT-cell lymphoma, amyloidosis, Myelodysplastic Syndromes and solid tumors. Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA, for multiple myeloma, as both a monotherapy and in combination with other therapies.