BTD

FDA Grants Breakthrough Therapy Designation to Sunvozertinib for the First-Line Treatment of Patients with advanced Non-Small Cell Lung Cancer Harboring EGFR Exon 20 Insertion Mutations

Retrieved on: 
Sunday, April 7, 2024
PART, EGFR, Mutation, BTD, NSCLC, Disease, Progression-free survival, Patient, Heel, Corr, NDA, Breakthrough, Non-Euclidean geometry, Doctor of Philosophy, CDE, Society, ESMO, Safety, ASCO, FDA, Annual general meeting, Food, Pharmaceutical industry

SHANGHAI, April 7, 2024 /PRNewswire/ -- Dizal (688192.SH) today announced that the U.S. Food and Drug Administration ("FDA") has granted Breakthrough Therapy Designation (BTD) to its sunvozertinib as the first-line treatment for patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring epidermal growth factor receptor (EGFR) exon 20 insertion (Exon20ins) mutations.

Key Points: 
  • SHANGHAI, April 7, 2024 /PRNewswire/ -- Dizal (688192.SH) today announced that the U.S. Food and Drug Administration ("FDA") has granted Breakthrough Therapy Designation (BTD) to its sunvozertinib as the first-line treatment for patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring epidermal growth factor receptor (EGFR) exon 20 insertion (Exon20ins) mutations.
  • This Breakthrough Therapy Designation (BTD) approval was based on results from the global multi-center phase I/II study (WU-KONG1).
  • This new BTD will enable us work more closely with the FDA and accelerate its clinical development and regulatory submission."
  • Affecting roughly 2%-4% of NSCLC patients, EGFR Exon20ins mutations have been difficult to treat due to their unique spatial conformation, diverse mutation subtypes, and high heterogeneity.

Vertex Advances Inaxaplin (VX-147) into Phase 3 Portion of Adaptive Phase 2/3 Clinical Trial for the Treatment of APOL1-Mediated Kidney Disease

Retrieved on: 
Monday, April 1, 2024
Biotechnology, Other Health, Health, Pharmaceutical, Clinical Trials, BTD, Orphan drug, APOL1, RPD, Disease, Patient, Apolipoprotein L1, FSGS, Standard of care, EMA, UPCR, European Medicines Agency, Vertex Pharmaceuticals, Clinical trial, Vertex, Proteinuria, Food, FDA, IDMC, VRTX, Pharmaceutical industry

The clinical trial is designed to assess the impact of inaxaplin on kidney function and proteinuria for people living with proteinuric kidney disease mediated by two variants in the APOL1 gene, known as AMKD.

Key Points: 
  • The clinical trial is designed to assess the impact of inaxaplin on kidney function and proteinuria for people living with proteinuric kidney disease mediated by two variants in the APOL1 gene, known as AMKD.
  • In addition, the trial has been expanded to include adolescents with AMKD ages 10 to 17 years.
  • The IDMC also recommended enrolling adolescents with AMKD ages 10 to 17 years in the Phase 3 portion of the study.
  • The European Medicines Agency (EMA) has also granted inaxaplin Priority Medicines (PRIME) and Orphan Drug designations for AMKD.

Cybin Announces Positive End-of-Phase 2 Meeting with FDA for CYB003 in Major Depressive Disorder and Phase 3 Program Design

Retrieved on: 
Thursday, March 14, 2024
Mental Health, Finance, FDA, Clinical Trials, Professional Services, Biotechnology, Alternative Medicine, Health, Pharmaceutical, Depression, Week, DEA, BTD, Inc., MDD, Patient, Accelerated approval (FDA), DSM-IV codes, Suicidal ideation, Behavior, Priority review, Drug development, Major depressive disorder, Pharmaceutical industry

The Company has received minutes from its End-of-Phase-2 meeting with the FDA and reached alignment on its Phase 3 program design.

Key Points: 
  • The Company has received minutes from its End-of-Phase-2 meeting with the FDA and reached alignment on its Phase 3 program design.
  • The Company intends to commence enrollment for the multinational, multisite Phase 3 program in mid-year 2024.
  • The Company has engaged Worldwide Clinical Trials (“Worldwide”), a global, full-service contract research organization with deep expertise managing clinical trials for mental health conditions, including major depressive disorder.
  • Having aligned on key features of the pivotal program, we look forward to initiating a multisite, multinational Phase 3 program around mid-year,” said Doug Drysdale, Chief Executive Officer of Cybin.

Cybin Receives FDA Breakthrough Therapy Designation for its Novel Psychedelic Molecule CYB003 and Announces Positive Four-Month Durability Data in Major Depressive Disorder

Retrieved on: 
Wednesday, March 13, 2024
Alternative Medicine, Mental Health, Health, FDA, Clinical Trials, Pharmaceutical, Biotechnology, JAMA, Depression, Disability, Conference, BTD, Breakthrough therapy, Massachusetts General Hospital, Inc., Psychiatry, Clinical, Grammatical mood, Appetite, MDD, Data analysis, Patient, Knowledge, Disease, Causality, Sleep, Anhedonia, DNB, DSM-IV codes, MBBS, Suicidal ideation, Behavior, Cognition, Event, Hospital, Drug development, Safety, Injury, Multimedia, Plasma protein binding, Sadness, Lancet, BMJ, Pharmaceutical industry

If approved by the FDA, CYB003 would be the first known adjunctive psychedelic-based therapeutic for the treatment of MDD.

Key Points: 
  • If approved by the FDA, CYB003 would be the first known adjunctive psychedelic-based therapeutic for the treatment of MDD.
  • The designation of CYB003 as a breakthrough therapy acknowledges the significant unmet medical need for more effective treatments of MDD and supports CYB003’s potential for significant improvements over existing therapies.
  • With the robust durability data from our Phase 2 study in hand, we are ready to move forward expeditiously.
  • (2022) Response to acute monotherapy for major depressive disorder in randomized, placebo-controlled trials submitted to the US Food and Drug Administration: individual participant data analysis.

Merus Announces Financial Results for the Fourth Quarter and Full Year 2023 and Provides Business Update

Retrieved on: 
Wednesday, February 28, 2024
Cetuximab, Research, Castration, PRS, Intellectual property, Patient, Pancreatic cancer, HNSCC, EAP, Eli Lilly and Company, CRPC, Drug design, BLA, FTD, HER2, MET, Congress, Afatinib, Partnership, NSCLC, CD3, EGFR, ESMO, BTD, Betta, CRC, AACR, Safety, Biomarker, RECIST, Clinical trial, Oncology, Prostate cancer, Neoplasm, FDA, Pembrolizumab, Lung, Disease, Travel, Pharmaceutical industry

Merus also continues to evaluate patients with untreated advanced PD-L1+ HNSCC treated with petosemtamab 1500 mg in combination with pembrolizumab.

Key Points: 
  • Merus also continues to evaluate patients with untreated advanced PD-L1+ HNSCC treated with petosemtamab 1500 mg in combination with pembrolizumab.
  • Merus plans to report initial interim efficacy and safety data from this cohort in the second quarter of 2024.
  • Merus achieved a milestone and received a payment of $2.5 million related to the advancement of this program in the third quarter of 2023.
  • Merus also achieved an additional milestone of $1 million for candidate nomination in the fourth quarter of 2023.

U.S. FDA Accepts for Priority Review the Supplemental Biologics License Application for Epcoritamab (EPKINLY®) for Difficult-to-Treat Relapsed or Refractory Follicular Lymphoma

Retrieved on: 
Tuesday, February 27, 2024
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, ABBV, BTD, ASH, Food, AbbVie, PDUFA, Exposition, Annual general meeting, Safety, Society, Patient, Diagnosis, Priority review, Pharmaceutical industry

The FDA grants Priority Review to investigational therapies that, if approved, may offer significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.

Key Points: 
  • The FDA grants Priority Review to investigational therapies that, if approved, may offer significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.
  • “The acceptance of the epcoritamab application for Priority Review marks an important milestone toward potentially providing a new treatment option to patients affected by R/R follicular lymphoma.
  • The FDA previously granted Breakthrough Therapy Designation (BTD) to epcoritamab for the treatment of adult patients with R/R FL after two or more lines of systemic therapy.
  • The companies will share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization.

Nuvalent Receives U.S. FDA Breakthrough Therapy Designation for NVL-520

Retrieved on: 
Tuesday, February 27, 2024
NSCLC, BTD, ROS1, FDA, Food, TRK, Brain, CNS, Patient, Diagnosis, TKI, Cancer

There remains no clear standard of care for patients who have been previously treated with two or more ROS1 TKIs.

Key Points: 
  • There remains no clear standard of care for patients who have been previously treated with two or more ROS1 TKIs.
  • "We're very encouraged by today's announcement of FDA breakthrough therapy designation for NVL-520, as it recognizes the continued need for innovation for patients with ROS1-positive NSCLC who have exhausted available therapies.
  • We look forward to providing an update from the ARROS-1 trial of NVL-520 at a medical meeting later this year."
  • Under the designation, the FDA provides intensive guidance, organizational commitment involving senior managers, and eligibility for rolling review and other actions to expedite review.

Medicenna Therapeutics Reports Third Quarter Fiscal 2024 Financial Results and Corporate Update

Retrieved on: 
Wednesday, February 14, 2024
IPR, Research, CMO, Decompression (diving), PR, Society, Bifunctional, MNP, PRS, Bank statement, Melanoma, OTCQB, ICI, Toronto Stock Exchange, SITC, CBV, Annual general meeting, European Medicines Agency, Partnership, BTD, Highlight, AACR, Conference, Safety, Clinical trial, Survival, Neurology, CFO, EMA, Enzyme, MD, ECA, FDA, TSX, Cancer, EDGAR, Pembrolizumab, Response, MDNA, SNO, Patient, Pharmaceutical industry

Today, the Company reports promising clinical data from the on-going monotherapy escalation and expansion arms of the ABILITY-1 study.

Key Points: 
  • Today, the Company reports promising clinical data from the on-going monotherapy escalation and expansion arms of the ABILITY-1 study.
  • Clinical update from the combination arm of the ABILITY-1 study evaluating MDNA11 in with KEYTRUDA® expected in H1 and H2 of 2024.
  • Research and development expenses of $3.0 million were incurred during the quarter ended December 31, 2023, compared with $2.9 million incurred in the quarter ended December 31, 2022.
  • General and administrative expenses of $1.8 million were incurred during the quarter ended December 31, 2023, compared with $2.0 million during the quarter ended December 31, 2022.

CymaBay Announces FDA Acceptance of NDA and Priority Review for Seladelpar for the Treatment of Primary Biliary Cholangitis

Retrieved on: 
Monday, February 12, 2024
Alkaline phosphatase, Liver, Child, Ursodeoxycholic acid, New Drug Application, BTD, Cirrhosis, Inflammation, FDA, Quality of life, Food, Breakthrough, Chief, Conditional sentence, PDUFA, ALP, PBC, Seladelpar, Therapy, Fatigue, Patient, Itch, Mortality, NDA, Pharmaceutical industry

NEWARK, Calif., Feb. 12, 2024 /PRNewswire/ -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a biopharmaceutical company focused on innovative therapies for patients with liver and other chronic diseases, today announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) for seladelpar, an investigational treatment for the management of primary biliary cholangitis (PBC) including pruritus in adults without cirrhosis or with compensated cirrhosis (Child Pugh A) who are inadequate responders or intolerant to ursodeoxycholic acid. The FDA has granted priority review and set a Prescription Drug User Fee Act (PDUFA) target action date of August 14, 2024 and notified the company that it is not currently planning to hold an advisory committee meeting to discuss the application.

Key Points: 
  • PBC is a rare, chronic condition that can lead to inflammation and eventually liver cirrhosis.
  • People living with PBC can also experience symptoms that significantly impact their quality of life such as pruritus (itch) and fatigue.
  • Seladelpar is an investigational, potent, selective, orally active PPARδ agonist, or delpar, in development for PBC treatment.
  • "We are encouraged by the agency's decision to grant priority review for seladelpar, and its recognition of the significant need for new treatment options for people living with PBC.

RadioMedix and Orano Med receive FDA Breakthrough Therapy Designation for AlphaMedixTM in gastroenteropancreatic neuroendocrine tumors First Targeted Alpha Therapy to receive a Breakthrough Therapy Designation

Retrieved on: 
Monday, February 12, 2024
Oncology, Health, FDA, Clinical Trials, Radiology, Pharmaceutical, Toxicity, BTD, MD, GEP-NET, News, Breakthrough therapy, Breakthrough, Safety, Patient, RECIST, Alpha, PRRT, Pharmaceutical industry

AlphaMedixTM is a Targeted Alpha Therapy currently in Phase 2 clinical development, which consists of an SSTR-targeting peptide complex radiolabeled with lead-212 (212Pb) that serves as an in vivo generator of alpha particles.

Key Points: 
  • AlphaMedixTM is a Targeted Alpha Therapy currently in Phase 2 clinical development, which consists of an SSTR-targeting peptide complex radiolabeled with lead-212 (212Pb) that serves as an in vivo generator of alpha particles.
  • AlphaMedixTM is the first Targeted Alpha Therapy to receive Breakthrough Therapy Designation.
  • “The FDA's Breakthrough Therapy Designation underscores AlphaMedixTM potential as an innovative treatment that could redefine how patients with neurendocrine tumors are treated.
  • “Receiving FDA Breakthrough Therapy Designation for AlphaMedixTM is a great achievement for everyone involved and confirms the strong interest of the medical community for Targeted Alpha Therapies with lead-212.