ACAD
34.93
-0.08
-0.23%
AEMD
3.65
-0.32
-8.13%
APRI
2.26
+0.04
+1.80%
ARNA
1.43
-0.01
-0.69%
ATEC
2.68
-0.12
-4.29%
CNAT
4.4
-0.02
-0.45%
CRXM
0.21
0.00
0.00%
CYTX
1.61
-0.03
-1.83%
DXCM
76.9
+0.41
+0.54%
GNMK
13.02
+0.14
+1.09%
HALO
13.49
+0.2
+1.50%
ILMN
167.79
+4.29
+2.62%
INNV
0.104
+0.001
+0.971%
INO
5.92
+0.06
+1.02%
ISCO
1.75
+0.15
+9.38%
ISIS
57.56
0.00
0.00%
LGND
104.07
+0.02
+0.02%
LPTN
2.93
0.00
0.00%
MBVX
2.45
-0.07
-2.78%
MEIP
1.54
-0.04
-2.53%
MNOV
5.8
-0.01
-0.17%
MRTX
5.15
-0.05
-0.96%
MSTX
0.098
-0.005
-4.6921%
NBIX
41.72
-0.05
-0.12%
NUVA
74.55
+0.42
+0.57%
ONCS
1.26
-0.03
-2.33%
ONVO
3.01
0.00
0.00%
OREX
4
-0.08
-1.96%
OTIC
13.15
+0.9
+7.35%
QDEL
21.31
-0.17
-0.79%
RCPT
231.96
0.00
0.00%
RGLS
1.25
+0.05
+4.17%
RMD
71.29
+0.57
+0.81%
SPHS
2.55
+0.01
+0.39%
SRNE
4.15
0.00
0.00%
TROV
1
-0.08
-6.98%
VICL
2.18
+0.02
+0.93%
VOLC
18
0.00
0.00%
ZGNX
10.35
0.00
0.00%
ACAD
34.93
-0.08
-0.23%
AEMD
3.65
-0.32
-8.13%
APRI
2.26
+0.04
+1.80%
ARNA
1.43
-0.01
-0.69%
ATEC
2.68
-0.12
-4.29%
CNAT
4.4
-0.02
-0.45%
CRXM
0.21
0.00
0.00%
CYTX
1.61
-0.03
-1.83%
DXCM
76.9
+0.41
+0.54%
GNMK
13.02
+0.14
+1.09%
HALO
13.49
+0.2
+1.50%
ILMN
167.79
+4.29
+2.62%
INNV
0.104
+0.001
+0.971%
INO
5.92
+0.06
+1.02%
ISCO
1.75
+0.15
+9.38%
ISIS
57.56
0.00
0.00%
LGND
104.07
+0.02
+0.02%
LPTN
2.93
0.00
0.00%
MBVX
2.45
-0.07
-2.78%
MEIP
1.54
-0.04
-2.53%
MNOV
5.8
-0.01
-0.17%
MRTX
5.15
-0.05
-0.96%
MSTX
0.098
-0.005
-4.6921%
NBIX
41.72
-0.05
-0.12%
NUVA
74.55
+0.42
+0.57%
ONCS
1.26
-0.03
-2.33%
ONVO
3.01
0.00
0.00%
OREX
4
-0.08
-1.96%
OTIC
13.15
+0.9
+7.35%
QDEL
21.31
-0.17
-0.79%
RCPT
231.96
0.00
0.00%
RGLS
1.25
+0.05
+4.17%
RMD
71.29
+0.57
+0.81%
SPHS
2.55
+0.01
+0.39%
SRNE
4.15
0.00
0.00%
TROV
1
-0.08
-6.98%
VICL
2.18
+0.02
+0.93%
VOLC
18
0.00
0.00%
ZGNX
10.35
0.00
0.00%
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San Diego biotech news from BioSpace, Xconomy, PR Newswire, Marketwired and other sources, click on headlines to read the full story.

Update on Full Six-Patient Cohort Confirms Patients with Complete Paralysis Can Experience Meaningful Recovery of Function by Six Months Following AST-OPC1 Treatment

March 21, 2017 – 3:00 am

-Newly reported data on sixth and final patient in cohort further confirms previously announced improvements in hand and arm function following dosing-

FREMONT, Calif., March 21, 2017 /PRNewswire/ — Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company pioneering the field of regenerative medicine, reported that including the sixth and final patient in the AIS-A 10 million cell cohort in the company’s ongoing SCiStar Phase 1/2a clinical trial has further confirmed previously-announced motor function improvements at 6-months following administration of AST-OPC1. 

“We are excited to see the sixth and final patient in the AIS-A 10 million cell cohort show upper extremity motor function improvement at 3 months and further improvement at 6 months, especially because this particular patient’s hand and arm function had actually been deteriorating prior to receiving treatment with AST-OPC1,” stated Dr. Edward Wirth III, Chief Medical Officer. “We are very encouraged by the meaningful improvements in the use of arms and hands seen in the SciStar study to date since such gains can increase a patient’s ability to function independently following complete cervical spinal cord injuries.”

Improvements in upper extremity motor function are being measured using the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) scale, widely used to quantify functional status of patients with spinal cord injuries.  Both subjects and physicians consistently report that improvements in upper extremity motor function are the most desirable functional improvement target in the quadriplegic population, as they can have a significant impact on functional independence, quality of life and cost of care from even relatively modest changes.   The SCiStar study is monitoring two separate ISNCSCI measurements of upper extremity motor function.  The upper extremity motor score (UEMS), is a linear scale used to quantify motor function at each of five upper extremity muscle groups driving arm and hand function; these scores are also used to calculate “motor levels”, which define the level within the cord above which a subject has a certain minimum level of function.

The UEMS improvement at 6-months for the final patient was 9 points and in line with the median and average 6-month UEMS improvement for all six patients in this cohort (9.7 average; 9 median). In all six patients, administration of AST-OPC1 was followed by early improvements in UEMS at 3-months that were sustained or further increased through their most recent follow-up assessment.

In addition to the motor score gains, the final patient in the AIS-A 10 million cell cohort has so far achieved a one motor level improvement over baseline on both sides of his body.   All six patients in this cohort have now achieved at least a one motor level improvement over baseline on at least one side.

“These results are quite encouraging, and suggest that there are meaningful improvements in the recovery of functional ability in patients treated with the 10 million cell dose of AST-OPC1 versus spontaneous recovery rates observed in a closely matched untreated patient population,” said Steve Cartt, Chief Executive Officer of Asterias. “We look forward to reporting additional efficacy and safety data for this cohort, as well as for the currently-enrolling AIS-A 20 million cell and AIS-B 10 million cell cohorts, later this year.”

The trial results to date continue to indicate a positive safety profile for AST-OPC1. There have been no serious adverse events related to AST-OPC1 and data from the study indicate that AST-OPC1 can be safely administered to patients in the subacute period after severe cervical spinal cord injury.

Each year in the U.S. more than 17,000 people suffer a severe, debilitating spinal cord injury. These injuries can be devastating to quality of life and ability to function independently. Lifetime healthcare costs for these patients can often approach $5 million. Improvements in arm, hand and finger functional capabilities in these patients can result in lower healthcare costs, significant improvements in quality of life, increased ability to engage in activities of daily living, and increased independence.

About the SCiStar Trial

The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with sub-acute, C-5 to C-7, motor complete (AIS-A or AIS-B) cervical SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A patients have lost all motor and sensory function below their injury site, while AIS-B patients have lost all motor function but may retain some minimal sensory function below their injury site. AST-OPC1 is being administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.

The study is being conducted at six centers in the U.S. and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment. Clinical sites that have enrolled and dosed patients in the study include the Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) in Los Angeles, Rush University Medical Center in Chicago and Santa Clara Valley Medical Center in San Jose.

Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provides $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.

Additional information on the Phase 1/2a trial, including trial sites, can be found at www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).

About AST-OPC1

AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells, has been shown in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed at the injury site of a spinal cord injury. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site. In preclinical animal testing, AST-OPC1 administration led to remyelination of axons, improved hindlimb and forelimb locomotor function, dramatic reductions in injury-related cavitation and significant preservation of myelinated axons traversing the injury site.

In a previous Phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. They also received low levels of immunosuppression for the next 60 days. Delivery of AST-OPC1 was successful in all five subjects with no serious adverse events associated with AST-OPC1. No evidence of rejection of AST-OPC1 was observed in detailed immune response monitoring of all patients. In four of the five patients, serial MRI scans indicated that reduced spinal cord cavitation may have occurred. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with complete cervical spine injuries, which represents the first targeted population for registration trials.

About Asterias Biotherapeutics

Asterias Biotherapeutics, Inc. is a biotechnology company pioneering the field of regenerative medicine. The company’s proprietary cell therapy programs are based on its immunotherapy and pluripotent stem cell platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC1 (antigen-presenting autologous dendritic cells) is undergoing continuing development by Asterias based on promising efficacy and safety data from a Phase 2 study in Acute Myeloid Leukemia (AML), with current efforts focused on streamlining and modernizing the manufacturing process. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy. The company’s research partner, Cancer Research UK, plans to begin a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at www.asteriasbiotherapeutics.com.   

FORWARD-LOOKING STATEMENTS

Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as “will,” “believes,” “plans,” “anticipates,” “expects,” “estimates”) should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias’ filings with the Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.

 

SOURCE Asterias Biotherapeutics, Inc.

Waving White Flag, Cerulean Merges With Dare Bio, Sells Cancer Drugs

March 20, 2017 – 6:41 am

Cerulean Pharma may have been able to survive one brush with death, but the second has proven fatal. Today, the Waltham, MA, company announced plans to merge with a privately held women’s health…

[[Click headline to continue reading.]]

Global Genomics Group Announces Breakthrough Diagnostic Blood Test for Plaque Detection in the Heart

March 20, 2017 – 5:00 am

ATLANTA, March 20, 2017 /PRNewswire/ — Global Genomics Group (“G3”), a precision-medicine-based biopharmaceutical company developing genetically validated therapeutics and diagnostics for common, chronic and orphan diseases announced today that the Company’s management team will be presenting updated data on their first precision diagnostic biomarker, “knowPLAQUETM“, for the detection of atherosclerotic coronary artery disease at the 2017 Biomarker Summit in San Diego, CA, on March 20, 2017. “knowPLAQUETM” is the lead product in the Company’s diagnostic biomarker portfolio; it is a mass-spectrometry-based metabolomics diagnostic biomarker signature for the detection of atherosclerotic coronary artery disease, based on a simple mass spectrometry measurement from a standard blood draw.

“knowPLAQUETM” is the first diagnostic blood test that was specifically designed and developed for the identification of patients with any plaque in their coronary arteries. Atherosclerosis is the underlying malevolent abnormality responsible for coronary artery disease, which can result in heart attack, stroke and sudden cardiac death. When patients present with chest pain in the outpatient setting, the test can be used to “rule out” the presence of any plaque in the heart. Positioning this test as a “gatekeeper” to the current clinical evaluation paradigm can eliminate a significant number of unnecessary nuclear stress tests and invasive angiograms, and could save approximately 46% in the costs associated with the evaluation of chest pain patients – possibly saving several billion dollars to the US healthcare system.

“We are very pleased with the outstanding diagnostic performance of “knowPLAQUETM“, said Szilard Voros, MD, founder and CEO of Global Genomics Group, who is presenting data on the test at the 2017 Biomarker Summit. “We completed the scientific discovery and validation of this diagnostic biomarker signature, and we are in discussions for the implementation of the test. It is terrific to see that novel, cutting-edge scientific insights can be translated to patient care, which can significantly impact and improve the detection of cardiovascular disease.”

“knowPLAQUETM” was discovered and validated in Global Genomics Group’s own GLOBAL (Genetic Loci and the Burden of Atherosclerotic Lesions; NCT01738828) clinical study, which recruited a total of approximately 7,500 patients, and the test was developed and validated in a total of 1,096 subjects. The study was specifically designed based on the Institute of Medicine Guidelines and the American Heart Association guidelines for biomarker discovery and validation. Initial results from the study were presented last year at the Society of Cardiovascular Computed Tomography meeting in Orlando, FL.

About Global Genomics Group (G3)

G3 is a global leader in pan-omics and investigates biological networks that lead to the development of disease to identify novel biomarkers and therapeutics. G3 combines precision phenotyping with pan-omics (genomics, epigenomics, transcriptomics, proteomics, metabolomics, lipidomics and lipoprotein proteomics) and systems-biology driven bioinformatics to investigate the development and progression of conditions such as atherosclerosis in cardiovascular disease.

For additional information, please visit www.globalgenomicsgroup.com

G3 Contact:
Idean Marvasty
VP, Finance and Operations
idean.marvasty@globalgenomicsgroup.com

SOURCE Global Genomics Group (G3)

Arena Pharmaceuticals Appoints Dr. Preston Klassen, M.D., M.H.S. as Executive Vice President, Research and Development and Chief Medical Officer

March 20, 2017 – 4:30 am

SAN DIEGO, March 20, 2017 /PRNewswire/ — Arena Pharmaceuticals, Inc. (NASDAQ: ARNA), a biopharmaceutical company focused on developing novel, small molecule drugs across multiple therapeutic areas, today announced the appointment of Dr. Preston Klassen, M.D., M.H.S. as Executive Vice President, Research and Development and Chief Medical Officer. Dr. Klassen will report to Amit Munshi, Arena’s President and Chief Executive Officer.  

“We are thrilled to have Preston join Arena at this exciting time,” stated Mr. Munshi. “Having previously wo­­­­rked closely with Preston at Amgen on the global development and launch of multiple products, I am confident in his ability to lead our product development efforts as we strive to deliver critical data on our three Phase 2 assets in 2017.”

Dr. Klassen has over 20 years of experience in biopharmaceutical product development. Most recently, he was Chief Medical Officer of Laboratoris Sanifit S.L. and prior to that was Executive Vice President, Head of Global Development at Orexigen Therapeutics, Inc. Previously, Dr. Klassen held several positions of increasing responsibility at Amgen Inc., including Therapeutic Area Head for Nephrology. Prior to joining Amgen, he was a faculty member in the Division of Nephrology at Duke University Medical Center. Dr. Klassen received his medical degree from the University of Nebraska College of Medicine and completed his residency in internal medicine, fellowship in nephrology, and masters in health sciences degree at Duke University. 

Inducement Equity Award

In connection with the hiring of Dr. Klassen, the Compensation Committee of Arena’s Board of Directors approved an inducement stock option grant to Dr. Klassen to purchase 1,293,500 shares of Arena common stock. The option grant will be effective on March 20, 2017, and will have an exercise price per share equal to the closing price of Arena’s common stock on that date. The non-qualified stock option will have a 7-year term and will vest over four years, with 25% of the shares subject to the option vesting one year after the commencement of Dr. Klassen’s employment and the remainder of the shares vesting monthly over the following three years in equal installments, subject to Dr. Klassen’s continued service with Arena through the applicable vesting dates. The vesting of the option is subject to acceleration in certain circumstances as provided in Arena’s Amended and Restated Severance Benefit Plan. The stock option grant is subject to the terms and conditions of Arena’s 2013 Long-Term Incentive Plan, as amended, and the stock option agreement pursuant to which the option is granted.

The stock option is granted as an inducement material to Dr. Klassen entering into employment with Arena in accordance with NASDAQ listing Rule 5635(c)(4).

About Arena Pharmaceuticals
Arena Pharmaceuticals is a biopharmaceutical company focused on developing novel, small molecule drugs with optimized receptor pharmacology designed to deliver broad clinical utility across multiple therapeutic areas. Our proprietary pipeline includes potentially first- or best-in-class programs for which we own global commercial rights. Our three most advanced investigational clinical programs are ralinepag (APD811) in Phase 2 evaluation for pulmonary arterial hypertension (PAH), etrasimod (APD334) in Phase 2 evaluation for multiple autoimmune indications, and APD371 entering Phase 2 evaluation for the treatment of pain associated with Crohn’s disease. In addition, Arena has collaborations with the following pharmaceutical companies: Eisai Co., Ltd. and Eisai Inc. (commercial stage), Axovant Sciences (Phase 2 candidate), and Boehringer Ingelheim International GmbH (preclinical candidate).

Forward-Looking Statements
Certain statements in this press release are forward-looking statements that involve a number of risks and uncertainties. Such forward-looking statements include statements about the timing and significance of Dr. Klassen’s appointment as an executive officer; Dr. Klassen’s experience and his expected contribution; the timing and terms of Dr. Klassen’s equity award; expected results, data readouts and timing relating to ongoing clinical trials; and Arena’s focus, clinical utility, primary programs and collaborations. For such statements, Arena claims the protection of the Private Securities Litigation Reform Act of 1995. Actual events or results may differ materially from Arena’s expectations. Factors that could cause actual results to differ materially from the forward-looking statements include those disclosed in Arena’s filings with the Securities and Exchange Commission. These forward-looking statements represent Arena’s judgment as of the time of this release. Arena disclaims any intent or obligation to update these forward-looking statements, other than as may be required under applicable law.

Contact: Arena Pharmaceuticals, Inc.
Kevin R. Lind, Chief Financial Officer
klind@arenapharm.com  
858.210.3636

 

SOURCE Arena Pharmaceuticals, Inc.

Biocept Announces Revised Presentation Time at the 27th Annual Oppenheimer Healthcare Conference

March 20, 2017 – 4:05 am

Company presentation set for March 22 at 1:00 p.m. ET

SAN DIEGO, March 20, 2017 /PRNewswire/ — Biocept, Inc. (NASDAQ: BIOC), a leading commercial provider of liquid biopsy tests designed to provide physicians with clinically actionable information to improve the outcomes of cancer patients, announces a revised time for its presentation at the 27th Annual Oppenheimer Healthcare Conference. David Moskowitz, Vice President of Strategy and Corporate Communications, is now scheduled to present a corporate overview on Wednesday, March 22, 2017 at 1:00 p.m. Eastern time (10:00 a.m. Pacific time). The conference is being held at The Westin New York Grand Central in New York City.

A live webcast of the corporate presentation will be available on the Company’s website at ir.biocept.com. A replay of the presentations will be available for 90 days.

About Biocept

Biocept, Inc. is a molecular diagnostics company with commercialized assays for lung, breast, gastric, colorectal and prostate cancers, and melanoma. The Company uses its proprietary liquid biopsy technology to provide physicians with clinically actionable information for treating and monitoring patients diagnosed with cancer. The Company’s patented Target Selector™ liquid biopsy technology platform captures and analyzes tumor-associated molecular markers in both circulating tumor cells (CTCs) and in circulating tumor DNA (ctDNA). With thousands of tests performed, the platform has demonstrated the ability to identify cancer mutations and alterations to inform physicians about a patient’s disease and therapeutic options. For additional information, please visit www.biocept.com.

 

SOURCE Biocept, Inc.

Edico Genome Expands Intellectual Property Portfolio with an Additional Five Patents Issued for DRAGEN Platform

March 20, 2017 – 4:00 am

Company Holds Largest IP Portfolio Encompassing Processor-Based Analysis of Next-Generation Sequencing Data

SAN DIEGO, March 20, 2017 /PRNewswire/ — Edico Genome, creator of the world’s first processor designed to analyze next-generation sequencing data, today announced the United States Patent and Trademark Office has issued an additional five, foundational patents surrounding the DRAGEN™ platform for analysis and storage of genomic data. Edico’s intellectual property portfolio now contains eight issued patents, which provide protection up to 2033. An additional sixteen patents are pending.

All issued patents broadly cover the technology core to DRAGEN, including execution of sequence analysis pipelines and storage for genomic data on the hardware processing platform and providing for both onsite and cloud deployment. The patents cover any type of processor, including application-specific integrated circuits (ASICs), graphical processing units (GPUs) and field-programmable gate arrays (FPGAs), which are utilized by Edico’s highly reconfigurable DRAGEN processor. Entitled “Bioinformatics Systems, Apparatuses, And Methods Executed On An Integrated Circuit Processing Platform,” the patent numbers are as follows: 9,576,104; 9,576,103; 9,529,967; 9,519,752; and 9,483,610. Numbers for previously announced U.S. patents are as follows: 9,342,652; 9,235,680 and 9,014,989.

“Our robust and growing intellectual property portfolio surrounding DRAGEN reflects the innovation of our company. DRAGEN is a cutting-edge, unique solution for the big data bottleneck that slows clinical and research applications of genomics,” said Pieter van Rooyen, Ph.D., chief executive officer of Edico Genome. “Edico remains committed to delivering innovative, best-in-class infrastructure for researchers, companies and clinicians working in genomics that accelerates the industry’s maturation from data to diagnosis.”

The DRAGEN end-to-end platform is an ultra-rapid, fully automated solution for analysis of next-generation sequencing data both onsite and in the cloud. Powered by FPGA-centric technology that features optimized algorithms for mapping, alignment, sorting, variant calling and more, DRAGEN enables a whole genome to be analyzed in only 20 minutes onsite, or even under 10 minutes in a single cloud instance, while maintaining high accuracy and significantly lowering costs. DRAGEN also features hyper-efficient lossless, real-time compression of data files, reducing genomic data footprint and long-term storage costs significantly.

Multiple end-to-end, clinical-grade pipelines are available from Edico, including genome/exome, cancer, transcriptome/RNA-seq, structural variant, copy number variant, epigenome/methyl-seq, metagenome/microbiome, joint genotyping and third-party pipelines such as GATK 3.6. The platform is flexible and allows for customization of algorithms and existing pipelines. This system is managed through the browser-based DRAGEN portal, which features an easy-to-use, “drag and drop” interface, or fully parameterizable command line scripting. Best-in-class solutions for onsite, cloud or hybrid cloud analysis have been created through partnerships with top technology companies, including Intel, IBM, Dell EMC, and Amazon Web Services.

To date Edico’s customers have processed more than ten petabytes of data using DRAGEN for a range of activities, including processing of whole genomes, exomes or analyzing 3D genome structures.

About Edico Genome
The use of next-generation sequencing is growing at an unprecedented pace, creating a need for easy to implement infrastructure that enables rapid, accurate and cost-effective processing and storage of this big data. Edico Genome has created an end-to-end platform solution for analysis of next-generation sequencing data, DRAGEN™, which speeds whole genome data analysis from hours to minutes while maintaining high accuracy and reducing costs. Top clinicians and researchers are utilizing the platform to achieve faster diagnoses for critically ill newborns, cancer patients and expecting parents waiting on prenatal tests, and faster results for scientists and drug developers. For more information, visit www.EdicoGenome.com or follow @EdicoGenome.

 

SOURCE Edico Genome

Audentes Therapeutics Announces Presentation Of Data From RECENSUS, A Medical Chart Review Of Patients With X-Linked Myotubular Myopathy (XLMTM)

March 20, 2017 – 4:00 am

Data confirm and expand understanding of significant medical burden associated with XLMTM

SAN FRANCISCO, March 20, 2017 /PRNewswire/ — Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases, today announced that data from RECENSUS, a medical chart review of patients with X-linked Myotubular Myopathy (XLMTM), will be presented at the 2017 Muscular Dystrophy Association (MDA) Scientific Conference, which will be held in Arlington, Virginia from March 19 to 22, 2017. These data provide new insights into the significant medical burden for children with XLMTM, their families and caregivers.

A Multicenter, Retrospective Medical Record Review of Patients with X-Linked Myotubular Myopathy (XLMTM): The RECENSUS Study” will be presented by Alan Beggs, PhD, Director of The Manton Center for Orphan Disease Research at Boston Children’s Hospital, Sir Edwin and Lady Manton Professor of Pediatrics at Harvard Medical School, and Principal Investigator of the RECENSUS study.

This initial analysis of 112 male patients is the first publication to describe the substantial humanistic and economic burden on the lives of XLMTM children, their families and the healthcare system. Consistent with previous studies, RECENSUS data show that XLMTM is a devastating, life-threatening disease manifesting early in the neonatal period with considerable, ongoing unmet medical need. Key observations include:  

  • Overall mortality was 44% (64% of patients ≤18 months of age; 32% of patients >18 months of age)
  • In the first year of life, infants with XLMTM spent 35% of their time in the hospital and underwent an average of 3.7 surgeries
  • At birth, 95% of the boys were hypotonic and 90% required respiratory support
  • 48% of the boys required 24-hour ventilation and 60% had received a tracheostomy. Those patients that were not ventilated 24-hours per day still spent an average of 8.5 hours daily on a ventilator
  • The majority of patients for whom data were available were receiving the most invasive forms of ventilatory support (67% – CPAP/BiPAP, and 64% – IPPV/SIMV/Pressure support)

The data also demonstrate that the time from presentation of symptoms to a confirmed diagnosis of XLMTM is declining, which likely represents an increasing physician awareness of XLMTM, coupled with improved diagnostic techniques. Since the discovery of the MTM1 gene in 1996, the mean age at diagnosis has dropped from 35.1 months in the period 1996-2000, to 4.4 months in the period 2011-2014.

“RECENSUS has established one of the largest data sets of XLMTM in the world, and this analysis makes a vital contribution to our understanding of this terrible disease,” stated Dr. Alan Beggs. “The RECENSUS study more completely defines the disease burden and management of XLMTM, and demonstrates the devastating impact that an XLMTM diagnosis has on the lives of patients and their families.”

While retrospective studies of observational data must be interpreted with caution, they are particularly important in the rare disease setting, where large populations of patients are not available for enrollment in prospective studies. Such studies are critical for exploring the signs, symptoms, management, and burden of rare diseases, and have previously been used to provide historical control populations for new therapies undergoing regulatory approval. In addition, data from the RECENSUS study provide important information to aid in selecting endpoints for interventional studies of XLMTM, including measures of survival, respiratory function, and burden of illness.

AT132 for X-Linked Mytubular Myopathy
AT132 is the Audentes product candidate being developed to treat XLMTM, a rare monogenic disease characterized by extreme muscle weakness, respiratory failure and early death, with an estimated 50% mortality rate by 18 months of age. XLMTM is caused by mutations in the MTM1 gene, which encodes a protein called myotubularin. Myotubularin plays an important role in the development, maintenance and function of skeletal muscle cells. AT132 is comprised of an AAV8 vector containing a functional copy of the MTM1 gene. Multiple studies in animal models of XLMTM have demonstrated that a single administration of AT132 was well tolerated and significantly improved disease symptoms and survival rates. In one study these effects have lasted more than four years to date.

About Audentes Therapeutics, Inc.
Audentes Therapeutics (Nasdaq: BOLD) is a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases. We have four product candidates in development, AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM), AT342 for the treatment of Crigler-Najjar Syndrome, AT982 for the treatment of Pompe disease, and AT307 for the treatment of the CASQ2 subtype of Catecholaminergic Polymorphic Ventricular Tachycardia (CASQ2-CPVT). We are a focused, experienced and passionate team committed to forging strong, global relationships with the patient, research and medical communities.

For more information regarding Audentes, please visit www.audentestx.com

Forward Looking Statements
This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the interpretation of the data from RECENSUS and whether it contributes to the overall understanding of the burden of illness and burden of care of XLMTM, whether a study such as RECENSUS can be used to establish a historical control for AT132 as it seeks regulatory approval, and whether RECENSUS will be valuable in establishing or interpreting endpoints in future clinical trials of AT132. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Although the company believes that the expectations reflected in such forward-looking statements are reasonable, the company cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the company’s actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the company’s ability to advance its product candidates, obtain regulatory approval of and ultimately commercial its product candidates, the timing and results of pre-clinical and clinical trials, the company’s ability to fund development activities and achieve development goals, the company’s ability to protect intellectual property and other risks and uncertainties described under the heading “Risk Factors” in documents the company files from time to time with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.

Audentes Contacts:

Investor Contact:
Thomas Soloway, CFO
415.818.1040
ir@audentestx.com

Media Contact:
Jeffrey Gruis
415.818.1015
media@audentestx.com

 

SOURCE Audentes Therapeutics, Inc.

Genetic profile of treatment-resistant lung cancer more variable than previously understood

March 17, 2017 – 4:33 pm

Study finds that acquired resistance mechanisms to targeted therapies often changed over time and that some patients demonstrated simultaneous genetic mutations

SAN FRANCISCO, March 17, 2017 /PRNewswire-USNewswire/ — The genetic mutations underlying treatment resistance in non-small cell lung cancer (NSCLC) are more complex and dynamic than previously thought. Analysis of 355 biopsied tumors from patients who acquired resistance to EGFR inhibitors, the most common form of targeted therapy for NSCLC, found that mutations frequently varied between biopsies and that nearly one in five patients harbored more than one type of genetic resistance to treatment. Findings will be presented today at the 2017 Multidisciplinary Thoracic Cancers Symposium.

Between 10 and 30 percent of NSCLC cases are driven by mutations in the epidermal growth factor receptor (EGFR) gene. These patients typically are treated with drugs that inhibit activation of the gene, but nearly all tumors eventually develop resistance to these therapeutic agents. Currently, many patients who acquire resistance to EGFR-targeted therapy undergo a tumor biopsy and are designated as either positive or negative for specific secondary mutations. This prognosis guides decisions for subsequent courses of targeted therapy, and further biopsies are not routinely obtained, given the invasive nature of rebiopsy in the lungs. Recent studies have indicated, however, that resistance in EGFR-mutant NSCLC can vary and that a permanent designation based on a single biopsy may be insufficient.

“In the past, resistance mechanisms were usually thought of as static. Our study demonstrates, conversely, that a binary designation of resistance may oversimplify cancer’s true biology. These genetic mutations can fluctuate over time, and some patients can harbor more than one mutation,” said Zofia Piotrowska, MD, lead author of the study and a thoracic oncologist at the Massachusetts General Hospital Cancer Center in Boston.

“In an era where cancer treatment is increasingly personalized to the biology of each individual patient, our findings highlight the importance of molecular testing and the need for improved, noninvasive methods for serial testing.”

To better understand the biological diversity of EGFR-resistant NSCLC, researchers retrospectively analyzed 355 biopsies of NSCLC tumors obtained from 221 patients after they acquired resistance to first-line EGFR inhibitor therapy. All patients were seen at a single institution between April 2008 and May 2016. The median patient age was 59 years (range 28-88), and 69 percent of patients were female. Researchers examined tumor samples for various genetic causes of EGFR resistance, including T790M, the most common secondary mutation.

All patients received at least one biopsy at first resistance to EGFR inhibitors. Nearly one in five (19%) of these 221 biopsies demonstrated more than one resistance mechanism simultaneously. The overall distribution of resistance mechanisms was consistent with previous research. The T790M mutation was found in 61 percent of patients, and the proportions of other mutations were as expected. Specifically, 18 percent of patients had EGFR amplification, 2 percent acquired PIK3CA mutations, 5 percent had amplification of the MET gene, 3 percent transformed non-small cell to small cell disease and 1 percent acquired BRAF mutations.

Eighty-three patients (37%) underwent two biopsies during post-resistance treatment. For half of these patients (49%), resistance mechanisms varied between biopsies, including both “gain” and “loss” of the T790M mutation (20% and 11% of two-biopsy patients, respectively). Three of the 17 patients who “lost” T790M demonstrated a new resistance mechanism on the second biopsy.

Researchers also sought to determine whether serial biopsies were safe and feasible for NSCLC patients. Two of the 307 biopsies (0.7%) resulted in clinically significant complications, and 13 percent of patients (n = 28) underwent three or more post-resistance biopsies.

“As much as half of the time, the dominant resistance mechanisms observed on one biopsy may no longer be relevant on a second biopsy, and that second biopsy may uncover a new, potentially targetable, resistance mechanism,” said Dr. Piotrowska. “We hope that these findings will prompt clinicians to consider rebiopsy when selecting a new therapy. While we observed that biopsies were generally safe and feasible, noninvasive testing methods, such as liquid biopsies that analyze tumor DNA circulating in the patient’s blood, may provide another method to more easily characterize resistance over time.”

The abstract, “Heterogeneity and variation in resistance mechanisms among 223 EGFR-mutant NSCLC patients with > 1 post-resistance biopsy,” will be presented in detail during the plenary session at the 2017 Multidisciplinary Thoracic Cancers Symposium in San Francisco (full details below). To schedule an interview with Dr. Piotrowska or an outside expert, contact the ASTRO media relations team at press@astro.org or 703-286-1600.

ATTRIBUTION TO THE 2017 MULTIDISCIPLINARY THORACIC CANCERS SYMPOSIUM REQUESTED IN ALL NEWS COVERAGE.

Abstract and Presentation Details

  • Heterogeneity and Variation in Resistance Mechanisms among 223 EGFR-mutant NSCLC Patients with > 1 Post-resistance Biopsy
  • News Briefing: Thursday, March 16, 12:30 – 1:45 p.m. Pacific time, Foothill F, Briefing slides and audio
  • Plenary Session, Thursday, March 16, 10:30 a.m.12:00 p.m. Pacific time, Yerba Buena Salon 9
  • This news release contains additional and/or updated information from the study author(s). Full original abstract and author disclosures available from press@astro.org or thoracicsymposium.org.

Resources on Lung Cancer and Radiation Therapy

ABOUT THE SYMPOSIUM
The 2017 Multidisciplinary Thoracic Cancers Symposium, co-sponsored by the American Society for Radiation Oncology (ASTRO), the American Society of Clinical Oncology (ASCO) and The Society of Thoracic Surgeons (STS), features the latest advances in surgery, radiation therapy, chemotherapy and novel molecular biologic therapies for thoracic malignancies such as lung cancer. The symposium will be held March 16-18, 2017, at the San Francisco Marriott Marquis. For more information about the symposium, visit www.thoracicsymposium.org. For press registration and news briefing information, visit www.astro.org/thoracicpress.

ABOUT ASTRO
The American Society for Radiation Oncology (ASTRO) is the premier radiation oncology society in the world, with more than 10,000 members who are physicians, nurses, biologists, physicists, radiation therapists, dosimetrists and other health care professionals who specialize in treating patients with radiation therapies. As the leading organization in radiation oncology, the Society is dedicated to improving patient care through professional education and training, support for clinical practice and health policy standards, advancement of science and research, and advocacy. ASTRO publishes three medical journals, International Journal of Radiation Oncology • Biology • Physics (www.redjournal.org), Practical Radiation Oncology (www.practicalradonc.org) and Advances in Radiation Oncology (www.advancesradonc.org); developed and maintains an extensive patient website, RT Answers (www.rtanswers.org); and created the Radiation Oncology Institute (www.roinstitute.org), a nonprofit foundation to support research and education efforts around the world that enhance and confirm the critical role of radiation therapy in improving cancer treatment. To learn more about ASTRO, visit www.astro.org.

ABOUT ASCO
Founded in 1964, the American Society of Clinical Oncology (ASCO) is committed to making a world of difference in cancer care. As the world’s leading organization of its kind, ASCO represents more than 40,000 oncology professionals who care for people living with cancer. Through research, education, and promotion of the highest-quality patient care, ASCO works to conquer cancer and create a world where cancer is prevented or cured, and every survivor is healthy. ASCO is supported by its affiliate organization, the Conquer Cancer Foundation. Learn more at www.ASCO.org, explore patient education resources at www.Cancer.Net, and follow us on Facebook, Twitter, LinkedIn, and YouTube.

ABOUT STS
Founded in 1964, The Society of Thoracic Surgeons is a not-for-profit organization representing approximately 7,200 cardiothoracic surgeons, researchers, and allied health care professionals worldwide who are dedicated to ensuring the best possible outcomes for surgeries of the heart, lung, and esophagus, as well as other surgical procedures within the chest. The Society’s mission is to enhance the ability of cardiothoracic surgeons to provide the highest quality patient care through education, research, and advocacy.

Contact: Liz Gardner
703-286-1600
liz.gardner@astro.org

Leah Kerkman Fogarty
703-839-7336
leah.fogarty@astro.org

 

SOURCE American Society for Radiation Oncology

Proton therapy offers new treatment possibility for recurrent lung cancer

March 17, 2017 – 4:17 pm

Advanced form of image-guided radiation therapy is an option for patients who previously had none

SAN FRANCISCO, March 17, 2017 /PRNewswire-USNewswire/ — A new study offers hope for patients with recurrent lung cancer, who historically have been considered ineligible for curative treatment. In the largest analysis to date of reirradiation using intensity-modulated proton therapy (IMPT) for lung and other thoracic tumors, more than three-fourths of patients were free from local recurrence at one year following retreatment, and fewer than one in ten patients experienced severe side effects. The study will be presented today at the 2017 Multidisciplinary Thoracic Cancers Symposium.

Lung cancer causes more deaths in the United States than any other type of cancer, due in part to its aggressive nature and likelihood of recurrence. Historically, recurrences have been challenging to treat, because many of these patients are not candidates for surgery. Moreover, concerns about cumulative radiation doses to essential organs near thoracic tumors (e.g., heart, lungs, esophagus) may limit the use of curative radiation therapy (RT) for patients who received thoracic RT in the past. Accordingly, patients whose lung cancer recurs are generally offered only palliative options to manage pain and other symptoms.

Proton therapy, an advanced type of RT, allows radiation oncologists to spare critical normal tissues while delivering escalated, curative doses of radiation to nearby tumors. In particular, IMPT is able to exactly target a tumor, even if the tumor wraps around a critical normal tissue structure, such as a lung.

“Treating patients who have already received a prior course of thoracic radiation is a common clinical scenario, and it is particularly challenging to subsequently provide strong enough radiation doses to eliminate the new tumor without causing significant harm to normal tissues,” said Jennifer Ho, MD, lead author of the study and a resident in radiation oncology at the University of Texas MD Anderson Cancer Center in Houston. “Our study is the first to show that IMPT can be safe and effective for these patients—and that it offers these patients a chance for lasting cancer control without adding significant toxicity.”

Researchers retrospectively examined the records of 27 patients who received reirradiation for thoracic tumors using the IMPT technique through prospective clinical trials at a single institution between 2011 and 2016. Twenty-two patients (81%) had non-small cell lung cancer (NSCLC). All patients had previously received curative thoracic RT.

Time to reirradiation ranged from 0.1 months to 212 months, with a median of 29.5 months. The median radiation dose was 66 Gray (Gy), with a range of 43.2 to 84 Gy, and it was delivered in 2 Gy fractions. The median follow-up for all patients in the study was 11.2 months.

The median overall survival (OS) for patients in this study was 18 months following IMPT reirradiation. At one year following retreatment, the majority of patients were free from local and regional relapse (freedom from local failure = 78%; local-regional relapse = 61%). Just over half of the patients were free from disease progression (progression-free survival, PFS = 51%), and the one-year OS rate was 54 percent. Four of the patients (15%) experienced a local recurrence within one year of retreatment.

Patients who were prescribed a higher dose of IMPT reirradiation were even less likely to experience recurrence or progression. At one-year follow-up, patients who received IMPT reirradiation doses at or above the population median were twice as likely to be free from local failure (100% vs. 49%, p = 0.01) and nearly four times as likely to be free from local-regional failure (84% vs. 23%, p = 0.035). The one-year PFS rate was five times higher for these patients, as well (76% vs. 14%, p = 0.05). Several disease characteristics, namely higher T stage at diagnosis, squamous histology and higher recurrent tumor volume, were associated with worse OS rates.

Reirradiation with IMPT was well-tolerated among the patients. Only two patients (7%) experienced moderate to severe long-term lung toxicity (i.e., grade 3 or higher side effects). There were no severe long-term esophageal side effects nor any life-threatening toxicities among these patients.

“Historically, 20 to 30 percent of patients have experienced moderate or severe side effects, and even fatal side effects, following reirradiation. We knew that IMPT would allow us to generate much more precise radiation treatment plans that spared normal tissue, but we weren’t sure if this would translate into excellent clinical outcomes,” said Joe Y. Chang, MD, senior author of the study and a professor of radiation oncology at MD Anderson. “Our findings demonstrate that the use of IMPT resulted in better local control and survival, with very minimal toxicity, compared to other radiation types, suggesting that IMPT is the optimal treatment modality for re-treatment of thoracic cancers.”

The abstract, “Reirradiation of thoracic cancers with intensity modulated proton therapy,” will be presented in detail during the plenary session at the 2017 Multidisciplinary Thoracic Cancers Symposium in San Francisco (full details below). To schedule an interview with Dr. Ho, Dr. Chang or an outside expert, contact the ASTRO media relations team at press@astro.org or 703-286-1600.

ATTRIBUTION TO THE 2017 MULTIDISCIPLINARY THORACIC CANCERS SYMPOSIUM REQUESTED IN ALL NEWS COVERAGE.

Abstract and Presentation Details

  • Reirradiation of Thoracic Cancers with Intensity Modulated Proton Therapy
  • News Briefing: Friday, March 17, 1:00 – 2:00 p.m. Pacific time, Foothill F, Briefing slides and audio
  • Plenary Session, Thursday, March 16, 10:30 a.m.12:00 p.m. Pacific time, Yerba Buena Salon 9
  • This news release contains additional and/or updated information from the study author(s). Full original abstract and author disclosures available from press@astro.org or thoracicsymposium.org.

Resources on Lung Cancer and Radiation Therapy

ABOUT THE SYMPOSIUM
The 2017 Multidisciplinary Thoracic Cancers Symposium, co-sponsored by the American Society for Radiation Oncology (ASTRO), the American Society of Clinical Oncology (ASCO) and The Society of Thoracic Surgeons (STS), features the latest advances in surgery, radiation therapy, chemotherapy and novel molecular biologic therapies for thoracic malignancies such as lung cancer. The symposium will be held March 16-18, 2017, at the San Francisco Marriott Marquis. For more information about the symposium, visit www.thoracicsymposium.org. For press registration and news briefing information, visit www.astro.org/thoracicpress.

ABOUT ASTRO
The American Society for Radiation Oncology (ASTRO) is the premier radiation oncology society in the world, with more than 10,000 members who are physicians, nurses, biologists, physicists, radiation therapists, dosimetrists and other health care professionals who specialize in treating patients with radiation therapies. As the leading organization in radiation oncology, the Society is dedicated to improving patient care through professional education and training, support for clinical practice and health policy standards, advancement of science and research, and advocacy. ASTRO publishes three medical journals, International Journal of Radiation Oncology • Biology • Physics (www.redjournal.org), Practical Radiation Oncology (www.practicalradonc.org) and Advances in Radiation Oncology (www.advancesradonc.org); developed and maintains an extensive patient website, RT Answers (www.rtanswers.org); and created the Radiation Oncology Institute (www.roinstitute.org), a nonprofit foundation to support research and education efforts around the world that enhance and confirm the critical role of radiation therapy in improving cancer treatment. To learn more about ASTRO, visit www.astro.org.

ABOUT ASCO
Founded in 1964, the American Society of Clinical Oncology (ASCO) is committed to making a world of difference in cancer care. As the world’s leading organization of its kind, ASCO represents more than 40,000 oncology professionals who care for people living with cancer. Through research, education, and promotion of the highest-quality patient care, ASCO works to conquer cancer and create a world where cancer is prevented or cured, and every survivor is healthy. ASCO is supported by its affiliate organization, the Conquer Cancer Foundation. Learn more at www.ASCO.org, explore patient education resources at www.Cancer.Net, and follow us on Facebook, Twitter, LinkedIn, and YouTube.

ABOUT STS
Founded in 1964, The Society of Thoracic Surgeons is a not-for-profit organization representing approximately 7,200 cardiothoracic surgeons, researchers, and allied health care professionals worldwide who are dedicated to ensuring the best possible outcomes for surgeries of the heart, lung, and esophagus, as well as other surgical procedures within the chest. The Society’s mission is to enhance the ability of cardiothoracic surgeons to provide the highest quality patient care through education, research, and advocacy.

Contact: Liz Gardner
703-286-1600
liz.gardner@astro.org

Leah Kerkman Fogarty
703-839-7336
leah.fogarty@astro.org

SOURCE American Society for Radiation Oncology

With New Data, Amgen Promises Refund If Cholesterol Drug Doesn’t Work

March 17, 2017 – 4:55 am

Stung by slow sales of its next-generation anti-cholesterol drug, Amgen hopes new clinical data, released this morning, will spur doctors to boost prescriptions and—perhaps more…

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