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MedNess: bite-size biopharma and medtech news

5th May, 2020
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MedNess This Week
HIGHLIGHTS
Our Sponsors
Drug Approvals
FDA approves Zejula (niraparib) as 1L maintenance treatment for women with platinum-responsive advanced ovarian cancer regardless of BRCA status
Dr. Hal Barron, Chief Scientific Officer and President R&D, GSK, said: “Women with advanced ovarian cancer have a five-year survival rate of less than 50%. This expanded indication means that many more women with this devastating disease can receive earlier treatment with Zejula, which can extend the time it takes for their cancer to progress.”
FDA Approves KEYTRUDA® (pembrolizumab) for Use at an Additional Recommended Dose of 400 mg Every Six Weeks for All Approved Adult Indications
“The important social distancing measures for COVID-19 have created a number of challenges for people with cancer, including keeping to planned treatment schedules,” said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. “Today’s approval of an every six-week dosing schedule for KEYTRUDA gives doctors an option to reduce how often patients are at the clinic for their treatment.”
Regulatory News
FDA grants Breakthrough Therapy Designation for Mobocertinib (TAK-788) for the Treatment of NSCLC Patients with EGFR Exon 20 Insertion Mutations
“We are pleased that the FDA has recognized the therapeutic potential mobocertinib offers for patients with EGFR exon 20 insertion-mutant NSCLC who are desperately in need of effective treatment options,” said Christopher Arendt, Head, Oncology Therapeutic Area Unit, Takeda. “At Takeda, we are committed to developing novel medicines for hard-to-treat diseases. Establishing Breakthrough Therapy Designation for mobocertinib is one step forward in our efforts to help change the current standard of care for this underserved population.”
Priority Review granted to CC-486 for Maintenance Treatment of Adult Patients in Remission from AML based on data from Ph 3 QUAZAR® AML-001 trial
“Often, newly diagnosed adult patients with AML achieve a complete response with induction therapy, however many patients will relapse and experience a poor outcome. Patients in remission are seeking treatment options that decrease the likelihood of relapse and extend overall survival,” said Noah Berkowitz, M.D., Ph.D., senior vice president, Global Clinical Development, Hematology, Bristol Myers Squibb. “Today’s acceptance of our submission for CC-486 represents an important step towards a potential new maintenance treatment to address an urgent medical need for AML patients and we look forward to working with the FDA during its review of CC-486.”
Click Here for more Regulatory News
Trial Results
Primary endpoint of OS met in Ph 3 trial of Libtayo® (cemiplimab) vs platinum-doublet chemotherapy in 1L PD-L1+ve NSCLC patients; trial to stop early
"While demonstrating a survival benefit in first-line NSCLC has been challenging for immunotherapies, the one FDA-approved anti-PD-1 monotherapy has changed the therapeutic paradigm," said George D. Yancopoulos, M.D., Ph.D., Co-Founder, President and Chief Scientific Officer of Regeneron. "We are pleased with the results of this trial that demonstrate the survival benefit of Libtayo in these patients and hope it may become a potential alternative for physicians and patients."
FAILED TRIAL: Phase 3 VOYAGER Trial of Avapritinib vs Regorafenib in 3L+ GIST Patients did not meet primary endpoint of PFS improvement
"While we are disappointed by the outcome of the VOYAGER trial, we are deeply grateful to the patients, investigators and clinical site staff who contributed to the completion of this global study. We hope these data will reveal important insights to improve the scientific understanding of the disease and inform future innovations in GIST, and we are committed to sharing the results at a future medical meeting," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "At Blueprint Medicines, we strive to advance science, building on successes and learning from setbacks, to create new medicines for patients with difficult-to-treat cancers and rare diseases. With a deep portfolio of precision therapies and a strong financial position, we will continue to advance our pipeline with clear near-term priorities in systemic mastocytosis and RET-altered cancers."
Click Here for more Trial Results
Trial/Program Status
First Patient Dosed in Ph 3 Trial of TJ202/MOR202 in RRMM patients in China
"TJ202/MOR202 is a front-runner candidate that adequately demonstrates our fast-to-market development strategy, representing a highly differentiated clinical development approach to provide new treatment options for unmet medical needs," said Dr.Joan Shen, CEO of I-Mab. "The phase 3 study is the second registrational trial of TJ202 as a potential second line treatment option for patients with multiple myeloma in Greater China."
First patient dosed with BCMA-targeting TriTAC HPN217 in Ph 1/2 trial in RRMM patients
“The initiation of a clinical trial for our third product candidate is another significant milestone for Harpoon and demonstrates the outstanding progress we are making in advancing our innovative oncology pipeline,” said Gerald McMahon, Ph.D., President and CEO of Harpoon Therapeutics. “The $50 million milestone payment adds additional financial resources to support the HPN217 program and other development activities that we are conducting. The first patient was treated at Colorado Blood Cancer Institute and the Sarah Cannon Research Institute at HealthONE’s Presbyterian St. Luke’s Medical Center. We are excited to be advancing HPN217 in the clinic and look forward to reporting our progress as this program matures.”
Ph 2 trial of BXCL701 + PD-1 Inhibitor initiated in Treatment Emergent Neuroendocrine Prostate Cancer patients
“Identifying the recommended Phase 2 dose of BXCL701, in combination with KEYTRUDA®, is a critical step for our immuno-oncology program, as it allows us to evaluate the anti-tumor activity in patients with tNEPC and other cancer types,” commented Vincent J. O’Neill, M.D., Senior Vice President and Chief Medical Officer of BTI. “tNEPC is a rare and highly aggressive form of prostate cancer, the most common malignancy in men, with no current standard treatments. With the DPP8/9 target amplified in some tNEPC tumors, we believe BXCL701 has the potential to facilitate a strong adaptive immune response, making tumors more responsive to immunotherapies, including the PD-1 inhibitor pembrolizumab. We believe BXCL701 has potential to treat this subpopulation, and if successfully developed and approved, could fill the gap in prostate cancer treatment.”
Collated by : Richa Tewari, PhD
MedNess @ HealthIT
COVID-19 specific sequencing for developing precision medicine
The patients that were initially tested positive for COVID-19 from northern and southern Nevada may help the scientists to understand the phylogenetic tree of the virus as well as the factors associated with the severity of the disease. The Nevada Genomics Center at the University of Nevada, Reno (UNR) is conducting genomic sequencing on samples in order to decipher the susceptibility factors and difference within the viral strain. NSPHL is the first public health lab in the nation to construct its own COVID-19 sample collection kits which takes advantage of NGS from a minute amount of patient sample. The manufacturing process involves creating 3D printed testing swabs and producing viral transport media (VTM), which is part of the testing kit. The process has helped the state of Nevada to achieve testing capacity above the national average of tests per 100,000 residents. According to the director of the Nevada Genomics Center, Paul Hartley, PhD, “The coronavirus genome can acquire mutations, and with the right amount of data, one can do epidemiological studies to understand how the virus may have spread.”
Imaging dataset by RSNA will help characterize brain hemorrhage
The Radiological Society of North America (RSNA) has created a public medical imaging dataset of expert-annotated brain hemorrhage CT scans, which will lead to the development of machine learning algorithms helping detect and characterize this condition. RSNA initiated this effort in response to the most recent edition of its Artificial Intelligence Challenge, and instead of using an existing dataset, as they used in the past challenges, this time they created a brain hemorrhage CT scan dataset from scratch. The three institutes making seminal contribution to generate the data set are Stanford University in Palo Alto, California, Universidade Federal de São Paulo in São Paulo, Brazil, and Thomas Jefferson University Hospital in Philadelphia, Pennsylvania. RSNA then partnered with the American Society of Neuroradiology (ASNR) to curate the dataset while calling for volunteers within the ASNR membership to annotate the images. They gathered 140 volunteers within a day and half, 60 of which were then selected to annotate a collection of 874,035 brain hemorrhage CT images in 25,312 unique exams. RSNA has released the dataset under a non-commercial license to make it freely available to all AI researchers for non-commercial use and further refinement and enrichment of the dataset.
Collated by :  Debarati Banik
MedNess Reviews

Single domain antibodies and bacterial superglue provide a new twist to anti-viral immunotherapy
In a recent Elife study, Schreur et al, describe an inventive antibody intervention by gluing together single domain antibodies (sdAbs) with bacterial superglue peptides for effective Bunyavirus neutralization.
sdAbs are miniature antibodies that contain only the heavy chain fragment and an extended CDR3 region which serves as an antibody binding domain. They are less bulky than standard antibodies, making them easier to fold and amenable to genetic manipulation. sdAbs, bind unique epitopes on antigen surfaces, not accessible by standard antibodies. Llama derived sdAbs (VHHs) have been shown to have nano to picomolar affinities to a broad spectrum of antigens and have been successfully applied to viral neutralization.
Bacterial superglues are peptides derived from Streptococcus pyogens that form spontaneous and irreversible isopeptide bonds. These amide bonds are formed by the nucleophilic attack of lysine on an asparagine residue between 2 peptide chains, in the presence of a catalytic glutamic acid residue. Isopeptide bonds confer proteolytic, thermal and pH stability to joined molecules.
In this study, monovalent VHHs were genetically engineered to contain either the lysine bearing ‘Spy-tag’ or the asparagine bearing ‘snoop tag’ which irreversibly connected in the presence of a catcher fragment (elastin-like-protein or ELP fragment containing the glutamic acid residue), coded separately. The tethered VHHs now had multimeric valency and a high degree of flexibility (due to ELP), resulting in greater degrees of freedom in spatial arrangements of the antigen binding domains.
In comparison to monovalent llama antibodies, the multimeric llama antibodies achieved complete neutralization of Rift Valley fever virus, reducing invasion of monkey kidney cells in culture as well as in mice infected with a lethal dose of the virus. In the latter experiment, control mice perished within 5 days of infection while all of the mice treated with superglued llama antibodies survived even after 10 days of observation.
This study presents a broadly applicable method of sdAbs-based biotherapeutics, especially in the case of challenging viral infections.
Collated by :  Divyaanka Iyer
Conference Coverage - ASCO 2020
ABSTRACTS
  1. Karyopharm to present Data from Ph 3 BOSTON Study
  2. BeiGene to present data from Tislelizumab and Zanubrutinib trials
  3. AVEO Oncology to Present Final OS Analysis from Ph 3 TIVO-3 Trial of Tivozanib in RCC
  4. Genmab to present Epcoritamab, Tisotumab Vedotin and Daratumumab results
  5. Calithera Biosciences to present Telaglenastat Ph 2 KEAPSAKE Trial data
  6. Celyad to Present Update from CYAD-101 for Advanced CRC and Next-Gen shRNA Platform
  7. Iovance Biotherapeutics to Present Updated melanoma Data from C-144-01 trial
  8. Allogene Therapeutics & Servier to present data from dose escalation Phase 1 ALPHA study of ALLO-501 in R/R NHL patients
  9. Merck to present 25 abstracts to Showcase Significant Clinical Advances in Cancer Care
Medness Business
Onco-News

Puma Biotechnology and Bixink Therapeutics to Commercialize NERLYNX® (neratinib) in South Korea
“Our new agreement with Bixink demonstrates our commitment to bringing NERLYNX to patients around the world while continuing to focus our commercial resources on the U.S. market,” stated Alan H. Auerbach, Chief Executive Officer and President of Puma. “We are excited about the potential to provide South Korean breast cancer patients with access to NERLYNX.”
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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Images : Twitter , Unsplash.com
Content Editors: Richa Tewari , Esha Sehanobish , Mayur Vadhvani and Abhinav Dey 
Concept and Design: Ananda Ghosh and Nisha Peter
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