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Puma Biotechnology Expands Cohort in Phase II SUMMIT Trial of PB272 in HER2 Mutation-Positive Cancer Patients

On January 6, 2017 Puma Biotechnology, Inc. (Nasdaq: PBYI), a biopharmaceutical company, reported it has expanded the fourth cohort from its Phase II SUMMIT clinical trial of its lead drug candidate PB272 (neratinib) as a single agent in patients with solid tumors who have an activating HER2 mutation (basket trial) (Press release, Puma Biotechnology, JAN 6, 2017, View Source [SID1234517291]). The cohort that has been expanded is the cohort that includes patients with metastatic cervical cancer and whose tumors have a HER2 mutation.

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The Phase II SUMMIT basket trial is an open-label, multicenter, multi-histology, international study to evaluate the safety and efficacy of PB272 administered daily to patients who have solid tumors with activating ERBB mutations including EGFR, HER2 and HER3. The cohorts included in the basket study receiving neratinib monotherapy are hormone receptor-negative breast cancer; biliary tract cancers; endometrial cancer; gastric/esophageal cancer; ovarian cancer; and all other solid tumors with a HER2 mutation. The cohorts receiving combination treatment are hormone receptor-positive breast cancer (neratinib plus fulvestrant) and bladder cancer (neratinib plus paclitaxel). The cervical cancer patients initially entered the study in the "other solid tumors with a HER2 mutation" cohort, and due to the preliminary activity seen in the trial, the Company has expanded a separate cervical cancer cohort pursuant to the protocol for the trial. The expanded HER2-mutant cervical cancer cohort will now enroll a total of 18 patients.

Dr. David Hyman, Director, Developmental Therapeutics at Memorial Sloan Kettering Cancer Center and principal investigator of the trial, stated, "We are pleased to expand our evaluation of neratinib in metastatic cervical cancer, an orphan and deadly disease with few treatment options. We believe this once again demonstrates the value of the basket study approach, in particular for developing targeted therapy for rare diseases with clinically-actionable mutations. We look forward to presenting the full results of the SUMMIT trial at a medical meeting in 2017."

"We are pleased to expand the fourth cohort in the basket trial," said Alan H. Auerbach, Chief Executive Officer and President of Puma. "Although it is early, we are pleased with the initial activity that we are seeing in the patients with HER2 mutated cervical cancer in the trial. We look forward to continuing enrollment into this expanded cohort and look forward to presenting the full results from the SUMMIT study in 2017."

Atreca, Inc. to Participate in Immuno-Oncology Panel at 9th Annual Biotech Showcase

On January 6, 2017 Atreca, Inc., a biotechnology company focused on developing novel therapeutics based on a deep understanding of the human immune response, reported that Tito A. Serafini, Ph.D., Atreca’s President, Chief Executive Officer, and CoFounder, will participate in a panel discussion on immuno-oncology (IO) during the 9th Annual Biotech Showcase Conference on Wednesday, January 11, 2017, at 11:00a.m. Pacific Time (Press release, Atreca, JAN 6, 2017, View Source [SID1234522958]).

The panel, titled "The Overflowing IO Buffet: Next Gen IO Antibodies, Oncolytic Viruses, Bispecifics, Adoptive Cell Therapies and Oral Immunometabolism / Immuno-Epigenetic Agents," will be held at the Hilton San Francisco Union Square in Continental Room 2.

Rgenix Announces Treatment of First Patient in Phase 1 Trial of RGX-104, its Lead Immuno-Oncology Therapeutic

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.

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

VBL Therapeutics Announces Completion of Enrollment in the Pivotal GLOBE Study

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.

"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.

SANGAMO BIOSCIENCES CHANGES NAME TO SANGAMO THERAPEUTICS, UNDERSCORING FOCUS ON CLINICAL DEVELOPMENT OF GENOMIC THERAPIES

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.

"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.