View this email in your browser

MedNess: bite-size biopharma and medtech news

16th February, 2022
Subscribe here
MedNess This Week
HIGHLIGHTS
 
  Regulatory News

NDA for poziotinib accepted for review by the U.S. FDA
“The NDA acceptance is a major step toward advancing the treatment for patients with HER2 exon 20 insertion mutations in lung cancer,” said Tom Riga, President and Chief Executive Officer of Spectrum Pharmaceuticals. “This remains an area of high unmet medical need as there are no treatments specifically approved for these patients. We are actively working with the agency to support the review process.”

FDA ODAC Committee Voted Against sintilimab Over Concerns Trials Conducted Only In China
“At a time when the FDA and the industry are trying to increase diversity in clinical trials to ensure they are representative of the patient population to be treated, it makes no sense to move in the opposite direction with this application,” said David Mitchell, a committee and founder of Patients for Affordable Drugs. 
Trial Results
ADCETRIS® Combination Significantly Improves Overall Survival in Newly Diagnosed Patients with Advanced Hodgkin Lymphoma
“These groundbreaking results are important for patients with advanced classical Hodgkin lymphoma given that an improvement in overall survival has rarely been shown in frontline treatment of this disease,” said Roger Dansey, M.D., Chief Medical Officer at Seagen. “We look forward to presentation of the results at an upcoming medical meeting.”
Ph 3 SIENDO Study Meets Primary Endpoint with Statistically Significant Increase in PFS in Patients with Advanced or Recurrent Endometrial Cancer
"As an oral, chemotherapy-free treatment, selinexor has the potential to transform the way advanced or recurrent endometrial cancer is treated and I am intrigued to learn more about the patients with the wild-type p53," said Professor Ignace Vergote, principal investigator and gynecologist oncologist, ENGOT and the Belgium and Luxembourg Gynaecological Oncology Group (BGOG), University of Leuven, Leuven Cancer Institute, Leuven, Belgium. "This study brings us one step closer to offering patients a treatment option that can give them more time with their friends and families."
Click here for more Trial Results
Trial/Program Status
First Patient Dosed in Ph 2 Study of NT-I7 (efineptakin alfa) with PD-L1 checkpoint inhibitor in High-Risk Skin Cancers
“We are excited to move into the Phase 2 portion of this important clinical trial, now that the safety and tolerability of the combination has been demonstrated,” said Se Hwan Yang, Ph.D., President and CEO of NIT. “NT-I7’s established ability to amplify T cells coupled with its excellent safety profile gives us great confidence that combinations with checkpoint inhibitor therapies like atezolizumab could improve clinical outcomes. We look forward to watching this next phase of the trial unfold and potentially set the foundation for a new immunotherapeutic option for the many patients living with these forms of skin cancer.”
Ph 2 trial of Elraglusib Plus FOLFIRINOX As First Line Therapy for Advanced Pancreatic Cancer to be initiated
“Novel approaches for patients with advanced pancreatic cancer are urgently required,” said Dr Weekes. “The pre-clinical and clinical data being generated with elraglusib in a spectrum of cancers, including pancreatic cancer, is extremely encouraging and we are delighted to have initiated this study with elraglusib. Elraglusib is the first clinically relevant specific GSK-3β inhibitor that we can thoroughly investigate. In preclinical models, elraglusib has multiple biologic effects relevant to targeting pancreatic cancer including direct cytotoxicity, reversal of chemoresistance, reversal of pathologic fibrosis, and there is increasing evidence of its immunemodulatory activity. In our study, we are particularly focused on elraglusib’s potential to synergize with TGF-β suppression mediated by Losartan. This study builds on the work of our investigative teams demonstrating the roles of TGF-β and GSK-3β in acquired chemotherapy resistance. This study uniquely attempts to harness the mechanisms that pancreatic cancer utilizes to combat the effects of chemotherapy as an Achilles heel for therapeutic intent. We believe that a multi-pronged attack as represented by elraglusib plus Losartan is a potentially sophisticated approach to a complex, often lethal, situation. It is an honour to lead this multicenter collaboration with my clinical and pre-clinical colleagues across the US and Europe. We are very grateful for the critical support of this program by the Lustgarten Foundation”
Click here for more Trial/Program Statuses
Collated by: Richa Tewari, PhD 
Genes and Therapy
Lentiviral gene therapy shows long-term efficacy in Wiskott-Aldrich syndrome
Genethon has announced long-term efficacy and safety data for its lentiviral gene therapy for Wiskott-Aldrich syndrome (WAS). WAS is a rare and severe complex immune deficiency, caused by a mutation in the WAS gene in hematopoietic progenitor cells. This inherited disease affects only boys and results in hemorrhages, repeated severe infections, severe eczema and, in some patients, autoimmune reactions and development of cancers. Results from the longer term follow-up of eight patients for a median of 7.6 years confirm the stability of the transplanted genetically modified cells and their safety and efficacy. The gene therapy corrected major disease symptoms, improved or eliminated bleeding and signs of autoimmunity, and restored T-cell (or immune system) function.
Positive preliminary data from Pompe Disease gene therapy
Astellas Pharma’s AT845, an investigational adeno-associated virus (AAV) gene replacement therapy for Late-Onset Pompe Disease (LOPD) showed positive interim safety data from FORTIS, the Phase I/II clinical trial evaluating to deliver a functional alpha-glucosidase (GAA) gene to express acid alpha-glucosidase (GAA) directly in muscle cells in adult patients. GAA is responsible for metabolizing glycogen, and dysfunction or absence of this protein results in the accumulation of glycogen, primarily in the skeletal and cardiac muscles, where it causes damage to tissue structure and function. AT845 is a one-time peripheral intravenous infusion, followed by one year of frequent monitoring of safety, clinical and biochemical endpoints including GAA activity and protein level in muscle and four additional years of long-term safety monitoring. 

Positive interim data for diabetic retinopathy
REGENXBIO’s ongoing Phase II ALTITUDE™ trial of RGX-314 for the treatment of diabetic retinopathy yielded positive interim data after six months of one-time injection. REGENXBIO is investigating RGX-314 in collaboration with AbbVie as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 includes the NAV AAV8 vector containing a gene encoding for a monoclonal antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.
Editors' Desk
Richa Tewari, PhD
Oncology News
Debarati Banik
HealthIT
Sohini Dutta
BioPharma News
MedNess Plus
Manoj Mishra
Neuroimmunology and immunotherapies
Darpan Chakraborty
Social Media Manager
IP & BioPharma News
 
Nisha Peter, PhD
Consulting Editor

 
Shalini Roy Choudhury
Genes and Therapy
Managing Editor
Share Share
Tweet Tweet
Forward Forward
Subscribe
Disclaimer
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.
Copyright © 2019 MedNess , All rights reserved.
You are receiving this MedNess Newsletter as a subscriber on the list.

Cover Image : iStock
Images : Twitter , Unsplash.com
Content Editors: Richa Tewari , Esha Sehanobish , Rinki Saha ,  Shilpa Rawal, PhD ,  Debarati Banik  , Divyaanka Iyer , Arundithi Ananthanarayanan and Abhinav Dey 
Concept and Design: Ananda Ghosh and Nisha Peter
Our mailing address is:
MedNess
1150 First Ave. King of Prussia,, PA 19406

Want to change how you receive these emails?
You can update your preferences or unsubscribe from this list.