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MedNess: bite-size biopharma and medtech news

24th November, 2021

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MedNess This Week
HIGHLIGHTS
 
Drug Approvals
FDA Approves KEYTRUDA® as Adjuvant Therapy for Patients With Renal Cell Carcinoma RCC at Intermediate-High or High Risk of Recurrence Following Nephrectomy, or Following Nephrectomy and Resection of Metastatic Lesions
“Despite decades of research, limited adjuvant treatment options have been available for earlier-stage renal cell carcinoma patients who are often at risk for recurrence. In KEYNOTE-564, pembrolizumab reduced the risk of disease recurrence or death by 32%, providing a promising new treatment option for certain patients at intermediate-high or high risk of recurrence,” said Dr. Toni K. Choueiri, director, Lank Center for Genitourinary Oncology, Dana-Farber Cancer Institute, and professor of medicine, Harvard Medical School. “With this FDA approval, pembrolizumab may address a critical unmet treatment need and has the potential to become a new standard of care in the adjuvant setting for appropriately selected patients.”
European Commission approves Gavreto (pralsetinib) for the treatment of adults with RET fusion-positive advanced NSCLC
“Today’s approval represents an important step forward in delivering precision medicine to people with RET fusion-positive advanced non-small cell lung cancer, for whom treatment options have historically been limited," said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “By using cancer genomic profiling upfront, healthcare professionals may identify specific genetic alterations that predict clinical benefit of targeted treatment options like Gavreto in the first-line setting.”
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Regulatory News

IND of MRTX1719 submitted to U.S. FDA to Treat MTAP-Deleted Cancers
"We are excited to take this next step in the advancement of MRTX1719 to clinical trials," said James Christensen, Ph.D., chief scientific officer, Mirati Therapeutics, Inc. "MRTX1719 was discovered and developed internally at Mirati, representing an important milestone for the Company's continued expansion of its pipeline and further reinforcing our differentiated end-to-end drug discovery and development capabilities. We look forward to further evaluating MRTX1719 as a potentially best-in-class treatment for patients living with MTAP-deleted cancers."

FDA Fast Track Designation for FixVac Candidate BNT111 in Advanced Melanoma
“The Fast Track Designation underlines the potential of our FixVac platform to address current treatment challenges of pre-treated and immune checkpoint blocker experienced melanoma with limited standard of care therapy options left. This is an important step to pave the way for this versatile new treatment approach in a high medical need setting,” said Özlem Türeci, M.D., Co-founder and Chief Medical Officer of BioNTech. “With the Fast Track status and support by the FDA, we aim to expedite the further development of the BNT111 program to provide a new therapeutic option for patients with life-threatening, hard-to-treat melanoma.”
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Trial Results
Quizartinib + Chemotherapy Demonstrated Superior OS Compared to Chemotherapy Alone in Adult Patients with 1L FLT3-ITD Positive AML
“The results of the phase 3 QuANTUM-First trial showed that adding quizartinib, a potent and selective FLT3 inhibitor, to chemotherapy significantly prolonged overall survival in patients with newly diagnosed FLT3-ITD positive AML,” said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. “We look forward to sharing the QuANTUM-First data with the hematology community and will initiate discussions with global regulatory authorities.”
2-THE-TOP Ph 2 Trial Reports Positive Top-Line Results in 1L Glioblastoma
“We are very encouraged by the results of the 2-THE-TOP study, especially in light of the poor prognostic factors of the patient population,” said William Doyle, Novocure’s Executive Chairman. “Dr. Tran’s research is an important continuation of our exploration of synergies between TTFields and immunotherapy agents. We would like to thank Dr. Tran and the patients enrolled in the 2-THE-TOP trial for their ingenuity and courage.”
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Trial/Program Status
TROPION-Breast01 Ph 3 Trial of Datopotamab Deruxtecan Initiated in Patients with HR+ve, HER2neg Metastatic Breast Cancer
“There are no TROP2 directed therapies currently approved for HR positive, HER2 negative breast cancer and we are encouraged by the emerging clinical profile of datopotamab deruxtecan in patients with breast cancer,” said Gilles Gallant, BPharm, PhD, FOPQ, Senior Vice President, Global Head, Oncology Development, Oncology R&D, Daiichi Sankyo. “TROPION-Breast01 is the first pivotal trial of datopotamab deruxtecan in breast cancer and the third pivotal study in our clinical development program, underscoring our efforts to accelerate development of this TROP2 directed ADC in breast and lung cancer.”
Enrollment Completed of Ph 3 Registration Trial of Uproleselan in Patients with R/R AML
“We believe that uproleselan is clearly a novel and potent inhibitor of E-selectin. Should the ongoing registrational trial prove positive, we will have created a foundational paradigm shift that has the potential to significantly impact outcomes for our patients with relapsed or refractory AML,” commented Daniel J. DeAngelo, M.D., Ph.D., Dana Farber Cancer Institute in Boston, who is the Principal Investigator of this multinational Phase 3 trial.
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Collated by: Richa Tewari, PhD 
Genes and Therapy
bluebird bio announces FDA Priority review of Biologics License Application for its β-thalassemia gene therapy
On 22nd November, bluebird bio announced that the FDA had accepted the Biologics License Application (BLA) and given priority review for Zynteglo, its gene therapy for the rare blood disorder β-thalassemia. Although priority review does not guarantee acceptance; if accepted, Zyntelgo (betibeglogene autotemcel or beti-cel) will be the first one-time treatment that addresses the underlying genetic cause of disease for patients living with β-thalassemia in the U.S.—offering an alternative to regular RBC transfusions and iron chelation therapy. 
Saint-Gobain LifeSciences and denovoMATRIX enter collaboration to develop technology that advances cell-based therapies
Saint-Gobain LifeSciences and denovoMATRIX have entered into a Master Service Agreement to optimize the performance of new products for adherent cell culture and autologous therapies. This collaboration will facilitate product development for the cell-based therapy market. The collaboration will utilize research and development, and laboratory facilities from both companies, including in the United States and Germany.
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MedNess Business
  Onco-News
Gilead Exercises Options to Three Arcus Biosciences Clinical-Stage Programs and Adds Research Collaboration
“Gilead is pursuing some of the most promising mechanisms of action in oncology today, with the aim of achieving better treatment outcomes for more patients,” said Daniel O’Day, Chairman and Chief Executive Officer, Gilead Sciences. “The addition of three mid- to late-stage clinical programs into our oncology pipeline significantly expands the number of transformational medicines we can potentially deliver to people with cancer, while also enabling our pursuit of novel combinations.”
SOTIO Expands its Antibody-Drug Conjugate Pipeline with Exclusive Collaboration and License Agreement with LegoChem Biosciences
“At SOTIO we are building an innovative pipeline of ADC programs and plan IND filing for our lead program SOT102 by the end of 2021. The licensing agreement with our new, experienced partner LegoChem allows us to broaden our oncology pipeline with additional programs and solid tumor targets. We are looking forward to using the potential of LegoChem’s ADC technology platform and to develop innovative ADCs for patients in need,” said Radek Spisek, M.D., Ph.D., chief executive officer of SOTIO.
 
  BioPharma and MedTech
Avilar Therapeutics launches with $60 million to degrade extracellular targets
Avilar Therapeutics is joining the growing targeted degrader crowd. But whereas most companies like Arvinas, Frontier Medicines in that area are advancing small molecules that break down intracellular proteins, Avilar is only the second to publicly take on extracellular troublemakers.
The Massachusetts-based startup is formally launching with $60 million in seed financing from RA Capital Management, a life science–focused investment firm, to build on the concept of bifunctional degraders. The startup is named ATACs (ASGPR Targeting Chimeras). These small molecules typically have two “arms” with different chemical functionality: one to bind a target of interest and another to bind a protein called an E3 ligase. The E3 ligase tags the target for degradation by a cellular trash disposal system called the proteasome. RA Capital started exploring this extracellular opportunity in 2019. The degrader strategy its team landed on also relies on two-armed molecules, but it harnesses a different cellular disposal system, called the endolysosome, to do the degradative dirty work. One arm again binds a therapeutic target; the other binds to a cell surface receptor called an asialoglycoprotein receptor (ASGPR). ASGPR then drags the tethered extracellular protein into an intracellular endolysosome for disposal. ASGPRs typically bind complex sugars, including galactose and its derivative N-acetylgalactosamine (GalNAc). While Avilar was in stealth mode, one of its priorities was the design of better ASGPR-binding small molecules. “The team identified opportunities to not just harness this pathway, but to improve upon nature itself,” Avilar CEO Dan Grau says.
“We see a very large scale of opportunity here, with many potential targets,” says Avilar CEO Dan Grau. “There are so many targets that are either poorly served by existing modalities, or frankly in some cases, not served at all.” Avilar has not yet disclosed which targets or diseases it is working on. “But the space is large,” Grau says, spanning hundreds of extracellular proteins. “It wouldn’t surprise me if others begin to move in, just because of the scale of the opportunity. There’s enough room for a few companies in this very, very large space,” he adds.
BridgeBio Pharma Secures Debt Financing Of $750M
Palo Alto-based BridgeBio Pharma has secured credit assistance worth as much as $750 million to move forward with its research and development efforts for the treatment of cancers and genetic diseases. Based on BridgeBio's current pipeline of programs, this total amount will be sufficient to carry over 30 drug development and discovery programs well into 2024. The definitive credit facility agreement it signed with a syndicate of lenders allows it to borrow up to $750 million, effectively bringing its spending capacity to more than $1.2 billion. The company reported its existing cash balance of $599.6 million in September.
"Since our founding, we have believed in the power and importance of innovative financing approaches to advance critical biomedical research and drug development, and we are grateful that our broad diversified pipeline enables us to do this. By bringing on this additional capital, we have the potential to help more people living with genetic diseases and cancers as quickly as possible," said Brian Stephenson, Ph.D., CFA, the chief financial officer of BridgeBio, in a statement. Immediately after the loan, BridgeBio Pharma And Helsinn Group Announce Strategic Collaboration To Co-Develop And Co-Commercialize BridgeBio’s Novel GPX4 Inhibitor In Multiple Cancer Tumor Types.
Click here for more on mergers, acquisition and business news
Editors' Desk
Richa Tewari, PhD
Oncology News
Shilpa Rawal, PhD
Onco I-Analyse
Arundithi Ananthanarayanan
MedNess Reviews
Debarati Banik
HealthIT
Darpan Chakraborty
Social Media Manager
IP & BioPharma News
 
Nisha Peter, PhD
Consulting Editor


 
Rinki Saha
BioPharma News
Managing Editor
Shalini Roy Choudhury
Genes and Therapy
Managing Editor
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Content Editors: Richa Tewari , Esha SehanobishRinki Saha ,  Shilpa Rawal, PhD ,  Debarati Banik  , Divyaanka Iyer , Arundithi Ananthanarayanan and Abhinav Dey 
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