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MedNess: bite-size biopharma and medtech news

29th July, 2020

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HIGHLIGHTS
Our Sponsors
COVID19 Special
FDA grants fast track designation for two investigational mRNA-based vaccine candidates from BioNTech and Pfizer
Pfizer and BioNTech recently announced that two of the companies’ vaccine candidates were granted a Fast Track designation by the FDA. Two of the candidates that received this designation belong to a group of four such candidate vaccine in the BNT162 mRNA-based vaccine program which aims to develop a vaccine against SARS-CoV-2. The two candidates, BNT162b1 and BNT162b2, are currently involved in ongoing Phase 1/2 clinical studies in the US and Germany.
“We are pleased to have received Fast Track designation from the FDA for two of our vaccine candidates and look forward to working closely with the FDA, along with out partner Pfizer, to expedite the clinical development path forward,” said Özlem Türeci, Chief Medical Officer at BioNTech. “The FDA’s decision to grant these two COVID-19 vaccine candidates Fast Track designation signifies an important milestone in the efforts to develop a safe and effective vaccine against SARS-CoV-2,” said Peter Honig, Senior Vice President, Global Regulatory Affairs, Pfizer. “We look forward to continue working closely with the FDA throughout the clinical development of this program, Project Lightspeed, to evaluate the safety and efficacy of these vaccine candidates.”
Moderna announces the publication of an interim analysis of the Phase 1 study of mRNA-1273, its vaccine candidate against COVID-19
In mid-July, Moderna, a clinical stage biotechnology company, announced the publication of an interim analysis of the of the Phase 1 study of their candidate vaccine against COVID-19, mRNA-1273. The results were published in The New England Journal of Medicine.
“These positive Phase 1 data are encouraging and represent an important step forward in the clinical development of mRNA-1273, our vaccine candidate against COVID-19, and we thank the NIH for their ongoing collaboration. The Moderna team continues to focus on starting our Phase 3 study this month and, if successful, filing a BLA,” said Stéphane Bancel, Chief Executive Officer of Moderna. “We are committed to advancing the clinical development of mRNA-1273 as quickly and safely as possible while investing to scale up manufacturing so that we can help address this global health emergency.” “These Phase 1 data demonstrate that vaccination with mRNA-1273 elicits a robust immune response across all dose levels and clearly support the choice of 100 µg in a prime and boost regimen as the optimal dose for the Phase 3 study,” said Tal Zaks, M.D., Ph.D., Chief Medical Officer of Moderna. “We look forward to beginning our Phase 3 study of mRNA-1273 this month to demonstrate our vaccine’s ability to significantly reduce the risk of COVID-19 disease.”
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Collated by :  Esha Sehanobish, PhD
Drug Approvals
FDA approves Tecartus™ for R/R MCL patients based on ZUMA-2 trial results
“Despite promising advances, there are still major gaps in treatment for patients with MCL who progress following initial therapy,” said Michael Wang, MD, ZUMA-2 Lead Investigator and Professor, Department of Lymphoma and Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center. “Many patients have high-risk disease and are more likely to keep progressing, even after subsequent treatments. The availability of Tecartus as the first-ever cell therapy for patients with relapsed/refractory MCL provides an important option with a response rate of nearly 90 percent and early clinical evidence suggesting durable remissions in later lines of therapy.”
Regulatory News
FDA grants Fast Track Designation to CMP-001 + PD-1 Blockade in the Treatment of Certain Types of Metastatic or Unresectable Melanoma
“These FDA designations for CMP-001 are testaments to the critical need for new drugs designed to treat patients with melanoma,” said Barry Labinger, CEO of Checkmate. “We look forward to continued engagement with the FDA in advancing the development of CMP-001 in combination with PD-1 blockade in melanoma and head and neck squamous cell carcinoma.”
Calquence recommended for approval in the EU by CHMP for CLL based on Ph 3 ELEVATE TN and ASCEND trial results
José Baselga, Executive Vice President, Oncology R&D said: “With its outstanding efficacy and tolerability profile, Calquence can offer important advantages to patients with chronic lymphocytic leukaemia who are typically older, facing multiple comorbidities and often require treatment for many years. This positive recommendation brings us closer to providing a much-needed new treatment option to patients in Europe who are suffering from this chronic blood cancer.”
Positive Input Received from FDA on Ph 3 Trial of VERU-111 in mCRPC
“Input from the FDA was very positive and constructive regarding the VERU-111 pivotal Phase 3 trial design. We received clarity that the indication, metastatic castration and novel androgen blocking agent resistant prostate cancer, which is a prechemotherapy population, was acceptable, and that an open label, randomized, active control study using an alternative novel androgen blocking agent as the active control is reasonable, and that the primary endpoint will be radiographic progression-free survival,” said Mitchell Steiner, MD, Chairman, President and CEO of Veru. “By allowing radiographic progression-free survival as an endpoint, the sample size of the Phase 3 study could be much smaller—potentially between 200 and 300 men. We plan to submit the final Phase 3 protocol to FDA in the fourth quarter of calendar year 2020. We plan to also meet with the EMA. We anticipate starting the Phase 3 pivotal study in the first quarter of calendar year 2021.”
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Trial Results
Results Reported from TG4001 + Avelumab trial in Advanced HPV-Positive Cancers
“Patients with HPV16-induced cancers still do not have access to approved treatments designed to address the viral origin of their disease. We believe these data establish the clinical proof-of-concept of combining TG4001 with an immune checkpoint inhibitor. Based on these promising findings, Transgene intends to continue the clinical development of TG4001 in a larger, controlled confirmatory study and provide a better treatment option to this patient population,” added Dr. Maud Brandely, MD, PhD, Chief Medical Officer of Transgene.
Positive preliminary interim data announced from Ph 1 portion of the TC-210 Ph 1/2 trial for mesothelin-expressing solid tumors
“We are delighted that our very first dose of TC-210 induced consistent tumor regression and clinical benefit in heavily pre-treated cancer patients,” said Garry Menzel, Ph.D., President and Chief Executive Officer of TCR2 Therapeutics. “There are very few options for patients with solid tumors and those expressing mesothelin represent a significant frontier of unmet medical need. While these are early data requiring further study, we are encouraged by the potential of our TRuC-T cells as we continue to enroll and treat patients with the goal of quickly finding a recommended Phase 2 dose for TC-210.”
Trial/Program Status
FDA places clinical hold on Ph 1 studies of PRS-343 in HER2+ve solid tumors
"We share FDA's commitment to product quality and will continue to engage with the Agency to initiate and complete the requested in-use and compatibility study with the highest priority," said Stephen S. Yoder, President and Chief Executive Officer of Pieris. "Pending satisfactory completion of this laboratory study, we remain committed to continuing the development of PRS-343, including the initiation of a phase 2 study in second-line gastric cancer in combination with the standard of care this year, as previously communicated. We also remain on track to present comprehensive data from both the monotherapy and atezolizumab combination phase 1 studies at a medical conference later this year."
Ph 3 CONTACT-03 Trial of Cabozantinib + Atezolizumab initiated in Previously Treated mRCC patients
“The treatment landscape for metastatic kidney cancer is rapidly evolving as the use of immune checkpoint inhibitor-based regimens move to earlier lines of therapy,” said Gisela Schwab, M.D., President, Product Development and Medical Affairs and Chief Medical Officer, Exelixis. “More data are needed to better understand the sequential use of treatments for this patient community, and we look forward to learning more about the potential role of the combination of cabozantinib and atezolizumab following checkpoint inhibitor therapy in this pivotal trial with our partner Roche.”
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Collated by : Richa Tewari, PhD
MedNess Reviews

Researchers Identify Potential Lung Cancer Marker by Studying Changes in Chromatin Structure
Scientists at the Massachusetts Institute of Technology and Harvard University have discovered a protein that, when elevated in human lung tumors could indicate tumor metastasis. They believe this finding could point to drug targets or new diagnostics.
The researchers observed structural changes in the chromosomes referred to as “epigenomic states” and defined 11 such states that cancer cells can pass through as they become more aggressive. They found that as the structure of tumor cells’ chromatin changed, transcription factors tended to target genes that would help the cells to lose their original identity and become less differentiated. Many of the cells were eventually able to leave their original locations and seed new tumors — a process largely controlled by a transcription factor called RUNX2. In more aggressive cancer cells, RUNX2 promotes the transcription of genes for proteins secreted by cells. These proteins help remodel the tumor environment to make it easier for cancer cells to escape. They reported their findings in the journal
Cancer Cell.
However, the researchers acknowledged that designing medicines that target transcription factors would be challenging. So the team is now trying to identify enzymes involved in chromatin changes that may be easier to block with drugs.
In the meantime, their discovery could be used in lung cancer diagnostics to patient outcomes and facilitate early intervention.

FDA approves Abbott’s neurostimulator-controlling iPhone app

The FDA recently approved an iOS-compatible Patient Controller smartphone app from Abbott that allows people to personalize and manage their own neuromodulation therapies. This would include regimens for chronic pain and movement disorders such as Parkinson’s disease.
With this app approval, the need to carry a separate patient programmer device is eliminated, streamlining the patient’s experience, and seamlessly integrating therapy management into their life. This is an important step in the integration and connection of digital health tools that ultimately help patients live fuller, healthier lives.
The Patient Controller App will be integrated into Abbott’s NeuroSphere Digital Care connected care management platform. This integration across all Abbott neuromodulation technologies allows physicians to treat the individual needs of each patient more easily within their practice for those who have an Abbott device and Apple smartphone and is particularly important in the context of the COVID-19 crisis.
The NeuroSphere management platform, first launched in May, is compatible with Abbott’s
Infinity DBS system aimed at Parkinson’s and essential tremor, as well as the company’s Proclaim XR and Proclaim DRG  devices for chronic pain.

Medness Business
Onco-News

AstraZeneca and Daiichi Sankyo enter collaboration to develop and commercialise TROP2-targeting ADC DS-1062
Pascal Soriot, Chief Executive Officer, said: “We see significant potential in this antibody drug conjugate in lung as well as in breast and other cancers that commonly express TROP2. We are delighted to enter this new collaboration with Daiichi Sankyo and to build on the successful launch of Enhertu to further expand our pipeline and leadership in Oncology. We now have six potential blockbusters in Oncology with more to come in our early and late pipelines.”

Cullinan Amber launched to pioneer Next-Gen Cytokine Fusion Proteins for Cancer Therapy
“Historically, numerous studies have shown that cytokine combinations, including IL-12 and IL-2, can synergistically enhance both innate and adaptive immunity, and mediate impressive antitumor activity across a range of preclinical tumor models,” stated Jon Wigginton, Chief Medical Officer of Cullinan Oncology. “Clinically, however, many cytokines have been limited by systemic toxicity, and were developed without the benefit of key learnings regarding dosing, supportive care and patient selection that have emerged in the field of immuno-oncology.”
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Collated by : Richa Tewari, PhD
Editors' Desk
Richa Tewari, PhD
Oncology News
Esha Sehanobish, PhD
MedNess Plus
Arundithi Ananthanarayanan
MedNess Reviews
Tanmoy Samaddar
MedNess Reviews
Divyaanka Iyer
MedNess Reviews
Debarati Banik
HealthIT
Abhi Dey
Consulting Editor
Nisha Peter, PhD
Managing 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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Content Editors: Richa Tewari , Esha Sehanobish , Mayur Vadhvani and Abhinav Dey 
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