The New England Journal of Medicine

Amylyx Pharmaceuticals Provides Update on Ongoing Regulatory Review of AMX0035 for the Treatment of ALS in the European Union

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Tuesday, May 30, 2023
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“We disagree with the current view expressed by the CHMP and remain confident in the data from the CENTAUR trial.

Key Points: 
  • “We disagree with the current view expressed by the CHMP and remain confident in the data from the CENTAUR trial.
  • Should a negative opinion ultimately be issued, we intend to request a formal re-examination procedure,” said Tammy Sarnelli, Global Head, Regulatory Affairs and Clinical Compliance at Amylyx.
  • The CHMP is expected to adopt a formal opinion on the MAA at its next meeting, which will be held June 19-22, 2023.
  • ALS affects approximately 29,000 people in the U.S. and more than 30,000 people are estimated to be living with ALS in Europe (European Union and United Kingdom).

BRUKINSA® Approved in Canada for the Treatment of Chronic Lymphocytic Leukemia

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Tuesday, May 30, 2023
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BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global biotechnology company, announced today that BRUKINSA (zanubrutinib), a Bruton's tyrosine kinase inhibitor (BTKi), has been approved by Health Canada for the treatment of adult patients with chronic lymphocytic leukemia (CLL).

Key Points: 
  • BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global biotechnology company, announced today that BRUKINSA (zanubrutinib), a Bruton's tyrosine kinase inhibitor (BTKi), has been approved by Health Canada for the treatment of adult patients with chronic lymphocytic leukemia (CLL).
  • “BRUKINSA now has four approved indications in Canada, demonstrating our commitment to bring this innovative BTKi treatment option to more patients across the world,” said Mehrdad Mobasher, M.D., M.P.H., Chief Medical Officer, Hematology at BeiGene.
  • The approval of zanubrutinib represents a significant advance for patients who can benefit from an additional treatment option,” says Raymond Vles, Board Chair, CLL Canada.
  • “More importantly, becoming informed and connecting with the CLL patient community will enable people with CLL to discuss with their doctor the treatment and dosing options that will work best for them and their lifestyle.”

Equify Health Receives $1 Million Grant to Launch Transformative Education Workshop to Accelerate Health Care Equity

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Wednesday, May 31, 2023
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MENLO PARK, Calif., May 31, 2023 /PRNewswire-PRWeb/ -- Equify Health, an innovation firm working to transform patient-provider experiences, was awarded a $1 million grant by leading biotechnology company Genentech. The grant will support the deployment of a multidisciplinary education program to train medical providers to shed unconscious biases in clinical practice. The ultimate goal behind the grant is to raise the quality of care for minority populations.

Key Points: 
  • "Negative barriers impacting minority patients are real," says Sylvie Leotin, the founder and CEO of Equify Health.
  • The new grant will allow Equify Health to expand its education workshop to doctors and nurses at academic hospitals and health care organizations across the United States.
  • With this second grant, Equify Health will expand its pilot workshop to additional institutions beyond Emory and enroll doctors across multiple specialties beyond cancer.
  • We believe this transformative education workshop will empower and engage medical providers to connect with marginalized patients in a more empathetic and inclusive way.

FDA green-lights bionic pancreas studied at UT Health Science Center San Antonio, University Health

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Tuesday, May 30, 2023
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SAN ANTONIO, May 30, 2023 /PRNewswire-PRWeb/ -- While it won't cure children of type 1 diabetes, the "bionic pancreas," an artificial intelligence-powered system cleared May 19 by the U.S. Food and Drug Administration, will relieve youth and their parents of constant worries over estimating insulin doses and carb intake from food.

Key Points: 
  • The University of Texas Health Science Center at San Antonio, with its clinical partner, University Health, is one of a few U.S. sites to offer children the "bionic pancreas," an artificial intelligence-powered system cleared Friday, May 19, by the U.S. Food and Drug Administration.
  • The bionic pancreas should relieve youth and their parents of constant worries over estimating insulin doses and carb intake from food.
  • It is AI for insulin delivery," said Jane Lynch, MD , professor of pediatric endocrinology and diabetes at The University of Texas Health Science Center at San Antonio.
  • The FDA reviewed the bionic pancreas and associated AI software through the 510(k) premarket clearance pathway.

Quibim, a company leveraging artificial intelligence to improve cancer diagnostics, makes key move into U.S. by hiring Boston-based chief medical officer

Retrieved on: 
Friday, May 26, 2023
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BOSTON, May 26, 2023 /PRNewswire/ -- Catching cancer earlier and achieving more accurate initial diagnostics are two of the key benefits of breakthrough artificial intelligence technology that a European company is now bringing to U.S. patients. For Quibim, a leading provider of imaging technology in the E.U., that path began with the recent approval they received from the U.S. FDA to apply their patented A.I. powered technology to domestic prostate cancer diagnostics.

Key Points: 
  • The move signals a key juncture in the proliferation and mainstreaming of A.I.-based technology into the U.S. patient experience.
  • Now, the company is seeking to broaden those impacts in the U.S. with a chief medical officer at the helm who has deep ties to Massachusetts.
  • According to data adapted from the American Cancer Society and other sources by Cancer.net , prostate cancer is the most common cancer among men, except for skin cancer.
  • "Harmonization of medical solutions, particularly in imaging, to enhance real-world evidence interpretation and clinical trial reporting is critical.

Data Demonstrating Long-Term Improvement in Depression in Cancer Patients Following Single Dose of COMP360 Psilocybin Therapy to be Presented at ASCO 2023

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Friday, May 26, 2023
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More than half of the patients (16/28) treated with a single dose of 25mg COMP360 psilocybin with psychological support remained in remission from their depression at 18 months post administration.

Key Points: 
  • More than half of the patients (16/28) treated with a single dose of 25mg COMP360 psilocybin with psychological support remained in remission from their depression at 18 months post administration.
  • The investigator-initiated study, led by Manish Agrawal, MD, at Sunstone Therapies , is believed to be the longest study of psilocybin therapy ever conducted.
  • The trial was a phase 2, single-center, fixed-dose, open-label study in 30 patients with curable and incurable cancer and major depressive disorder.
  • COMPASS is currently conducting a phase 3 clinical program of COMP360 psilocybin therapy in treatment-resistant depression (TRD), the largest of its kind ever conducted.

SpringWorks Therapeutics Announces Presentation of Additional Data from Phase 3 DeFi Trial of Nirogacestat in Adults with Desmoid Tumors at the 2023 ASCO Annual Meeting

Retrieved on: 
Thursday, May 25, 2023
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STAMFORD, Conn., May 25, 2023 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that additional data from the Phase 3 DeFi trial assessing the impact of nirogacestat, an investigational gamma secretase inhibitor, on pain, tumor volume and T2 hypersensitivity in adults with desmoid tumors will be presented at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting, being held June 2-6, 2023. Data from the DeFi trial were previously presented at the European Society for Medical Oncology Congress in September 2022 and published in the March 9, 2023 edition of the New England Journal of Medicine.1

Key Points: 
  • ET)
    As previously reported, in the DeFi trial ( NCT03785964 ), nirogacestat met its primary endpoint of significantly improving progression-free survival compared to placebo in adult patients with progressing desmoid tumors (hazard ratio: 0.29 [95% CI, 0.15–0.55]; P
  • Nirogacestat also achieved a significant and clinically meaningful reduction in pain severity, a key secondary endpoint, compared with placebo at Cycle 10 (P
  • “Many patients with desmoid tumors live with severe, chronic pain that significantly impacts their quality of life,” said Winette T.A.
  • Exploratory analyses were conducted to evaluate changes in MRI-assessed desmoid tumor volume and T2 signal intensity in the Phase 3 DeFi trial.

Bristol Myers Squibb Receives Positive CHMP Opinion Recommending Approval for Opdivo (nivolumab) with Chemotherapy as Neoadjuvant Treatment of Resectable Non-Small Cell Lung Cancer at High Risk of Recurrence in Patients with Tumor Cell PD-L1 Expre...

Retrieved on: 
Friday, May 26, 2023
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The European Commission (EC), which has the authority to approve medicines for the European Union (EU), will now review the CHMP recommendation.

Key Points: 
  • The European Commission (EC), which has the authority to approve medicines for the European Union (EU), will now review the CHMP recommendation.
  • “The devastating reality is that despite progress in lung cancer treatment, many patients still ultimately end up relapsing and potentially dying of their disease,” said Abderrahim Oukessou, M.D., vice president, thoracic cancers development lead, Bristol Myers Squibb.
  • Opdivo-based treatments have shown improved efficacy in the neoadjuvant or adjuvant treatment of four tumor types: lung cancer, bladder cancer, esophageal/gastroesophageal junction cancer and melanoma.
  • Bristol Myers Squibb thanks the patients and investigators involved in the CheckMate -816 clinical trial.

Lumicell Announces FDA Acceptance and Priority Review of New Drug Application for LUMISIGHT™ Optical Imaging Agent for Breast Cancer

Retrieved on: 
Monday, May 22, 2023
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Lumicell, Inc ., a privately held company focused on innovative fluorescence-guided imaging technologies for cancer surgery, today announced the U.S. Food and Drug Administration (FDA) has accepted and granted priority review for the New Drug Application (NDA) for the LUMISIGHT™ Optical Imaging Agent and accepted the Premarket Approval (PMA) application for the Lumicell™ Direct Visualization System (DVS).

Key Points: 
  • Lumicell, Inc ., a privately held company focused on innovative fluorescence-guided imaging technologies for cancer surgery, today announced the U.S. Food and Drug Administration (FDA) has accepted and granted priority review for the New Drug Application (NDA) for the LUMISIGHT™ Optical Imaging Agent and accepted the Premarket Approval (PMA) application for the Lumicell™ Direct Visualization System (DVS).
  • The Lumicell DVS and LUMISIGHT are limited by Federal (or United States) law to investigational use only.
  • The Lumicell DVS is intended for use with the investigational optical imaging agent LUMISIGHT (pegulicianine) for fluorescence imaging of the lumpectomy cavity.
  • “Priority Review designation is further recognition of the potential of our system to significantly improve the effectiveness of breast cancer treatment.

Dupixent® (dupilumab) Late-breaking Phase 3 COPD Results Presented at ATS and Simultaneously Published in the New England Journal of Medicine

Retrieved on: 
Sunday, May 21, 2023
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TARRYTOWN, N.Y. and PARIS, May 21, 2023 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today presented positive Phase 3 results evaluating the investigational use of Dupixent® (dupilumab) compared to placebo in adults currently on maximal standard-of-care inhaled therapy (triple therapy) with uncontrolled chronic obstructive pulmonary disease (COPD) and evidence of type 2 inflammation. The Dupixent Phase 3 results were presented in the 2023 American Thoracic Society (ATS) International Conference session “New England Journal of Medicine and JAMA. Discussion on the Edge: Reports of Recently Published Pulmonary Research” and simultaneously published in the New England Journal of Medicine (NEJM). These results will also be presented in the “Breaking News: Clinical Trial Results in Pulmonary Medicine” session on May 22.

Key Points: 
  • The Dupixent Phase 3 results were presented in the 2023 American Thoracic Society (ATS) International Conference session “New England Journal of Medicine and JAMA.
  • Discussion on the Edge: Reports of Recently Published Pulmonary Research” and simultaneously published in the New England Journal of Medicine (NEJM).
  • These results will also be presented in the “Breaking News: Clinical Trial Results in Pulmonary Medicine” session on May 22.
  • The results presented at ATS and published in NEJM are from the BOREAS trial, which met the primary and all key secondary endpoints.