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MedNess: bite-size biopharma and medtech news

11th February, 2020
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Regulatory News
PDUFA date extension of 3 months announced for NDA of Avapritinib in 4L Gastrointestinal Stromal Tumor patients
  • FDA extended the PDUFA action date by three months from February 14, 2020 to May 14, 2020
  • FDA has requested top-line data from ongoing Ph 3 VOYAGER trial of avapritinib vs. regorafenib to inform the pending action on the NDA for fourth-line GIST
  • Top-line data expected early in the second quarter of 2020 to enable the FDA to take action by the May 14, 2020 PDUFA date
DSMB confirms initial safety of weekly GEN-1 dosing at 100 mg/m² in 15 advanced ovarian cancer patients
"This latest DSMB review of GEN-1 at 100 mg/m² confirmed that there were no dose limiting toxicities detected in any of the six evaluable patients (those patients who received at least four doses of GEN-1) and that intraperitoneal GEN-1 administration is well tolerated even when given with standard NACT," said Nicholas Borys, M.D., executive vice president and chief medical officer of Celsion. “Of the fifteen patients treated in the Phase I portion of the OVATION 2 Study, nine patients were treated with GEN-1 plus NACT and six patients were treated with NACT only. After the final six patients in the Phase I portion of the Study have completed their interval debulking surgery, we will be reporting surgical results and overall tumor response rates for all fifteen patients from the Phase I portion of the trial later this quarter.”
Click Here for more Regulatory News
Trial Results
New Ph 2b data for personalized TLPLDC vaccine in high-risk melanoma patients presented at ASCO-SITC meeting
"Patients with Stage III and IV melanoma who have completed initial treatment have a very high risk of having their disease return, representing a serious unmet medical need," said Mark B. Faries, M.D., co-director of the Melanoma Program and head of Surgical Oncology at The Angeles Clinic and Research Institute, an affiliate of Cedars-Sinai and principal investigator of the study. "Achieving a 73 percent disease-free survival rate in this extremely challenging stage of disease is significant. Importantly, in this study we see that checkpoint inhibitors, when combined with a cell-based vaccine like TLPLDC, may produce a synergistic anti-tumor response. The goal of this type of approach is to increase patient responses and prevent disease recurrence."
Updated data from Ph 2 MERECA trial of Ilixadencel in Kidney Cancer presented at ASCO-SITC meeting
“The updated data emphasize that both tumor responses and the durability of patient response with ilixadencel treatment as part of a combination regimen were better compared to sunitinib alone. The addition of ilixadencel did not increase either the frequency or the severity of side effects. However, longer follow-up is required before we with certainty can comment on any differences in long-term survival,” stated Associate Professor Magnus Lindskog, clinical oncologist at Uppsala University Hospital, Sweden and principal investigator in the MERECA study who presented the results.
Click Here for Trial Results
Trial/Program Status
  • Ph 1b dose escalation and expansion clinical study of niraparib + MGD013 in advanced or metastatic gastric adenocarcinoma or gastroesophageal junction adenocarcinoma (collectively as gastric cancer) patients who failed prior treatment
  • Registrational bridging study of margetuximab + chemotherapy in metastatic HER2-positive breast cancer patients
Patient recruitment completes in Ph 2 GBM trial of AV-GBM-1, a novel immunotherapy targeting autologous tumor-initiating cells
“An extremely high manufacturing success rate combined with a very low cost of goods makes this approach highly desirable from both a clinical and commercial viability perspective,” said Bob Dillman, M.D., Chief Medical Officer of AIVITA. “We’re already exploring new ways to further increase efficiency and expand the applicability of this approach.”
Collated by : Richa Tewari, PhD
Medness @HealthIT
Cracking down on fraud with AI-police
According to the estimates of National Heath Care Anti-Fraud Association, health care fraud costs the US about $68 billion annually; which is about 3 percent of the nation's $2.26 trillion in health care spending. Lesser conservative estimates may go up as high as 10 percent of annual health care expenditure, or $230 billion. However, a new tool of preventing healthcare fraud is here in the form of artificial intelligence, as cleverly utilized by Highmark Inc.’s Financial Investigations and Provider Review (FIPR) department. It has utilized AI to generate over $260 million in savings that are associated with fraud, waste, and abuse in 2019, and has saved over $850 million in the last five years. 
The insurance organization Highmark is taking advantage of advanced AI tools to: 1) Detect fraudulent activity at a high speed and 2) Limit financial exposure of its customers. Using an internal team of registered nurses, investigators, accountants, former law enforcement agents, clinical coders, and programmers, it is performing audits to detect unusual claims, coding reviews, and investigations that evaluate the appropriateness of provider payments. Get the details on the story with HealthIT Analytics. 
Precision medicine gets a boost with chip-technology
University of Houston has reported a significant advancement on a microfluidic chip technology previously developed in Akay lab. This newer version of the chip allows multiple-simultaneous drug administration, as well as a parallel testing of drug response for glioblastoma (GBM) patients. Of note, GBM accounts for the most common malignant brain tumor (50%) with a five-year survival rate of only 5.6%. As published in the inaugural issue of the IEEE Engineering in Medicine & Biology Society's Open Journal of Engineering in Medicine and Biology, Metin Akay emphasized on the rapidity of assessment of a drug/drug combination via this chip, which is approximately 2 weeks, as opposed to months of animal research. "The new chip generates tumor spheroids, or clusters, and provides large-scale assessments on the response of these GBM tumor cells to various concentrations and combinations of drugs. This platform could optimize the use of rare tumor samples derived from GBM patients to provide valuable insight on the tumor growth and responses to drug therapies," Dr. Akay reports. 
The idea is to take a piece of a tumor biopsy, culture it and put it in the microfluidic chip, followed by addition of chemotherapy drugs to the chip's microvalves to determine the efficacy and best proportion of drug combinations lethal to the tumor cells. Get the details on the story from Science Daily.
Collated by : Debarati Banik
Medness Reviews
2019 Corona Virus Status
The Wuhan corona virus outbreak has claimed 813 lives globally as of February 8, 2020 pushing the death toll above the 2003 severe acute respiratory syndrome (SARS) outbreak. The day saw 89 deaths in mainland China, the highest number for a single day since the outbreak that began in January. Several new cases have also been emerging in countries across the world, with more than 34,800 people globally.
The World Health Organization has made an announcement that it will be sending a team to China for investigating the virus. The team leader is expected to touch down on Monday.
In light of the current global health crisis due to the spread of coronavirus infection, the FDA has issued an expedited approval of a new diagnostic test. Around 200 test kits of the nCoV test developed by Novacyte, a French Biotech company will be sent to qualified domestic and international labs. This expedited approval for corona virus approval follows other such approvals for MERS, Ebola and Zika.
Click here for the situation summary issued by Centers for Disease Control and Prevention
Massive Genomics Project Generates Insights into The Genetic Basis on Cancer
In a series of six research papers published in Nature, the Pan Cancer Analysis of Whole Genome (PCAWG) Consortium has presented the most comprehensive meta-analysis of cancer genomes. 
The consortium, involving an interdisciplinary group of scientists from across four continents adopted a whole genome analysis for scrutinizing important aspects of cancer genetics. The researchers, who were divided into 16 groups, each focusing on distinct aspects of cancer genomics performed integrative analyses of 38 tumor types. They sequenced 2,658 whole-cancer genomes, alongside matched samples of non-cancerous cells from the same individuals. These data were complemented by 1,188 transcriptomes.
The PCAWG efforts are an excellent example of how cloud computing can help make massive international collaboration possible for data intensive fields, since the study required involved extensive quality control and coordinated data processing, as well as massive, systematic experimental validation of the computational pipelines used to detect mutations.
These six papers, the first giving an overview of the PCAWG dataset and the five other papers delving into each aspect of the dataset in detail, represent a milestone in cancer and cloud genomics.
Biogen gets FDA approval to relaunch Aducanumab trial
Biogen and Esai Co., Ltd, have the U.S. Food and Drug Administration’s approval (2020) for a re-dosing trial of Aducanumab (human monoclonal antibody to amyloid-β), on 2044 early Alzheimer’s patients previously enrolled in their identical phase 3 clinical trials ENGAGE and EMERGE (2018-2019). Futility re-assessment of the trials (October 2019), showed statistically significant reduction in CDR-SB for EMERGE (P=0.01) and for a subset of ENGAGE patients, relative to placebo. The reversal of Aducanumab’s efficacy status was attributed to the cumulative effects of higher dosing and exposure time. Amidst divided opinion, the sceptics view the failure of ENGAGE as inconclusive of the positive effects of Aducanumab and consider the ENGAGE subset data analysis as a contentious method of data interpretation. More positive opinions rely on the fact that Aducanumab is the first anti-amyloid to elicit complete reversal of amyloid plaques, which is unprecedented amidst previous anti-amyloids.
At the JP Morgan Healthcare meeting (13th January 2020), Biogen spoke hopefully about gaining approval from FDA, European and Japanese regulatory authorities for the global commercial launch of Aducanumab, manufactured by company units in Switzerland and North Carolina. While already working on Aducanumab pricing, Biogen speculates that a potentially successful redosing trial will increase R&D investments in Alzheimer’ research. Biogen stock prices are currently up by 3%. A lot is now pegged, for the company and the Alzheimer’s community, on the new open-label re-dosing trial, which will administer 10 mg/kg of Aducanumab, to all patients over 100 weeks.
Collated by :  Divyaanka Iyer and Ananda Ghosh, PhD
Medness Business
Onco-News
$10.0 million development milestone payment made for the initiation of Ph 2 trial of CD27 agonist MK-5890 in NSCLC
“We are thrilled Merck is continuing to advance MK-5890 through development with the initiation of a Phase 2 clinical trial in NSCLC,” said Andrea van Elsas, Ph.D., chief scientific officer of Aduro. “The achievement of this milestone would not be possible without the efforts of the exceptional team we built at Aduro Biotech Europe and the B-select technology that created several monoclonal antibodies, including MK-5890 and BION-1301, an anti-APRIL antibody we are currently developing for IgA nephropathy.”
Collated by : Richa Tewari, PhD
Editors' Desk
Richa Tewari, PhD
Oncology News
Esha Sehanobish, PhD
MedNess Plus
Arundithi Ananthanarayanan
MedNess Reviews
Debarati Banik
HealthIT
Divyaanka Iyer
MedNess Reviews
Nisha Peter, PhD
Managing Editor
Mayur Vadhvani, PhD
Consulting Editor
Abhi Dey
Consulting Editor
Ananda Ghosh, PhD
Founder
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The editors take care to share authentic information.  In case of any discrepancies please write to medness.newsletter@gmail.com
The sponsors do not have any influence on the nature or kind of the news/analysis reported in MedNess. The views and opinions expressed in this article are those of the authors and do not necessarily reflect the official policy or position of Medness. Examples of analysis performed within this article are only examples. They should not be utilized in real-world analytic products as they are based only on very limited and dated open source information. Assumptions made within the analysis are not reflective of the position of anyone volunteering or working for Medness. This blog is strictly for news and information. It does not provide medical advice, diagnosis or treatment nor investment suggestions. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
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