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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.

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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.

Genmab Achieves USD 25 Million Sales Milestone in DARZALEX® (daratumumab) Collaboration with Janssen

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.

"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.

TARIS Biomedical® Announces Positive Results from Ph1b Trial of TAR-200 (GemRISTM) in Patients with Muscle Invasive Bladder Cancer

On January 6, 2017 TARIS Biomedical LLC, a company developing targeted new treatments for millions of patients suffering from difficult-to-treat bladder diseases, reported that it has closed initial enrollment in its Phase 1b clinical trial of TAR-200 (GemRIS) following highly positive results (Press release, TARIS Biomedical, JAN 6, 2017, View Source [SID1234517284]). TAR-200, a drug-device combination product utilizing the TARIS System, is designed to release gemcitabine continuously into the bladder over 7 days. This open-label study assessed the safety and tolerability of TAR-200 when used in patients with MIBC following initial diagnosis and prior to radical cystectomy. In addition, the study evaluated anti-tumor activity at Day 28 of the study. The treatment regimen employed in this study included two system deployments separated by a 14-day rest period.

Preliminary results indicate that the system was well tolerated over two 7-day treatment periods, with no local or systemic tolerability findings. To be eligible for enrollment, patients were required to have visible bulky residual tumor of at least 3 cm in size, clinical stage T2 or T3. Striking tumor responses, including complete tumor ablation or substantial shrinkage, were observed visually at the time of cystectomy in 8 of 10 patients. While the study was originally designed to include up to 20 subjects, the company closed enrollment early following these positive results in order to accelerate the clinical advancement of TAR-200. The company expects complete data from this study, including histopathological assessment, to be presented at a major upcoming medical meeting.

"The results of this study are very exciting," said Siamak Daneshmand, M.D., Associate Professor of Urology, University of Southern California, and Principal Investigator for the TAR-200-101 study. "TAR-200 appears to be remarkably well-tolerated in patients who have recently undergone extensive transurethral resection of the bladder. Moreover, the unexpected activity observed in these heavily diseased subjects in just 28 days indicates the significant potential of this product in a broad population of patients with MIBC, often a very difficult disease to treat."

"TARIS is extremely excited about the compelling results from this study, and plans to rapidly advance the product into later-stage clinical trials in bladder cancer in 2017," said Purnanand Sarma, Ph.D., President & CEO of TARIS. "The tumor response we have seen to date suggests TAR-200 may offer a fundamentally new treatment option for patients with MIBC, where the current standard of care includes bladder removal. We share the excitement of the study investigators about the potential impact that TAR-200 may have on the lives of patients across the spectrum of bladder cancer."

About the TAR-200-101 Phase 1b Trial
The Phase 1b open-label study was designed to assess whether continuous local exposure to gemcitabine is safe and tolerable in patients with MIBC. The study was initially designed to enroll up to 20 subjects across three sites in the US. Enrolled subjects had bulky residual clinical stage T2 or T3b MIBC tumors following initial transurethral resection of bladder tumor (TURBT), and were ineligible to receive cisplatin-based combination neoadjuvant chemotherapy. Subjects received two placements of TAR-200 during Days 1-7 and 21-28 in the 28-day window between TURBT and radical cystectomy. Subjects were evaluated for safety, tolerability, and evidence of antitumor effects.

About TAR-200
TAR-200 (GemRIS) is TARIS Biomedical’s first program in bladder cancer. TAR-200 is a drug-device combination product designed to release gemcitabine continuously into the bladder over 7 days. Gemcitabine is commonly used to treat multiple cancers alone and in combination with other chemotherapeutic drugs, and is routinely given intravenously1. TARIS believes TAR-200 has the potential to set a new standard of care in bladder cancer, with enhanced efficacy and minimal systemic side effects compared to current approaches. TARIS is developing TAR-200 to address unmet needs in both muscle invasive and non-muscle invasive bladder cancer.

About Muscle Invasive Bladder Cancer
Bladder cancer affects roughly 2.7 million people worldwide, including nearly 600,000 in the United States.2 The National Cancer Institute estimates that there will be a total of nearly 77,000 new cases and 16,000 deaths due to this disease in 2016.3 When measured as a cumulative lifetime per patient cost, the expense to treat bladder cancer exceeds all other forms of cancer.4 The estimated U.S. national expenditure on bladder cancer was $4.3 billion in 2014. 5

Muscle Invasive Bladder Cancer (MIBC) is an advanced form of the disease, representing 25-30% of the newly diagnosed cases. MIBC tumors, which have progressed into the muscle of the bladder wall and potentially beyond, may lead to metastases and death. The standard of care for treatment of MIBC includes radical cystectomy, or complete removal of the bladder, with or without neoadjuvant chemotherapy. Radical cystectomy is a major, life changing surgery and many patients are medically unfit and/or unwilling to undergo this procedure.

ARTSaVIT Ltd. Completes $6.3 Million Series A Financing

On January 5, 2017 ARTSaVIT LTD reported that it has completed a $6.3 million Series A round of financing led by Arkin Bio Ventures and Pontifax, with participation of M Ventures, Carmel Innovation and Carmel – Haifa University Economic Corporation Ltd (Press release, ARTSaVIT, JAN 5, 2017, View Source [SID1234561841]).

ARTSaVIT was co-founded by Carmel, the economic corporation of the University of Haifa, Carmel Innovations Fund and Professor Sarit Larisch from the University of Haifa, Israel. Prof. Larisch has identified and characterized ARTS, a protein which regulates the levels of several important anti-apoptotic proteins by promoting their degradation. Apoptosis is a highly regulated process of natural cell death. Faulty regulation of apoptosis is implicated in many human diseases, including cancer. Moreover, resistance to apoptosis is a hallmark of most human cancers.

The insights gathered by Prof. Larisch and the unique function of ARTS led to the establishment of the company, which is developing small molecule ARTS mimetics designed to selectively induce apoptosis in cancer cells. The company received seed investment from the Carmel Innovations Fund, which supported the research and development of the company to its current stage.

ARTSaVIT will move from its facilities at Carmel, University of Haifa, to the state-of-the-art facilities at the M Ventures Israel BioIncubator, which will support the development of the start-up with its infrastructure and a wide range of incubation facilities and services.

Dr. Rom Eliaz, Head of the M Ventures Israel BioIncubator commented: "We are excited to join forces with Arkin Bio Ventures, Pontifax and Carmel and would like to welcome ARTSaVit to our BioIncubator. Following the completion of this fundraising, the company is now well positioned to reach its next value inflection point".

Elka Nir, CEO of Carmel Ltd, the economic corporation of the Haifa University and CEO of Carmel Innovations Fund, noted: "We are proud and excited that M Ventures together with Arkin Bio Ventures and Pontifax invested in ARTSaVIT. It demonstrates the excellent quality and potential of the research and researchers at the University of Haifa. It is another great success for the Carmel Innovation Fund and its business model, which is funding seed companies, supporting them to a stage of significant value "

Avidity Biosciences Raises $16 Million in Series B Financing to Advance Antibody-siRNA Conjugate Platform

On January 5, 2017 Avidity Biosciences reported the completion of a $16 million Series B financing round to support the development of its Antibody-siRNA Conjugate (ASC) platform (Press release, Avidity Biosciences, JAN 5, 2017, View Source [SID1234521096]). The Series B round includes investment of $10 million in new capital and conversion of $6 million in convertible debt. Takeda Pharmaceuticals, through its venture group, led the Series B round, and both new and existing investors participated, including Alethea Capital, Alexandria Real Estate Equities, Brace Pharma, EcoR1 Capital, F-Prime Capital, Moore Venture Partners and Tavistock Life Sciences.

"Our ASC platform unites two of the most impactful innovations over the past twenty years – monoclonal antibodies and oligonucleotides – to create a new class of precision medicines," said Troy Wilson, Ph.D., J.D., president and chief executive officer of Avidity Biosciences. "Although siRNA-based therapeutics have demonstrated significant clinical and commercial promise, conventional approaches are limited to targeting diseases of the liver. Because ASCs use antibodies to overcome barriers of delivering siRNA, they have the potential to impact a broader range of therapeutic areas. Our goal is to partner with leading pharmaceutical and biotechnology companies to deliver a pipeline of ASCs targeting genetic drivers of disease."

ASCs link a monoclonal antibody—designed against a specific molecular target—with a siRNA therapeutic payload, allowing the conjugate to have unmatched specificity and selectivity. In preclinical models, ASCs have shown potential to knockdown messenger RNA levels in multiple important cell types and tissues, including tumor, muscle, heart, lung, liver and B cells. In addition, ASCs have drug-like properties comparable to antibodies and antibody-drug conjugates.

In connection with the financing, Avidity Biosciences annouced that Michael Martin, Ph.D., global head of Takeda Ventures Inc., and Todd Brady, director of finance and investments of Brace Pharma Capital, will join its board of directors. Tony Hsu, founder and chief investment officer of Alethea Capital, will also join the board as a non-voting member.

"We believe Avidity’s ASC platform offers a compelling approach that builds on successes with antibodies, ADCs and oligonucleotide-based therapeutics," said Dr. Martin. "Avidity has recruited a top-notch team and made significant progress against its scientific and business goals. We look forward to working with the company to realize the promise of ASCs as a new class of precision medicines."