Accelerated approval (FDA)

EQS-News: AdrenoMed Receives FDA Fast Track Designation for Enibarcimab for Treatment of Septic Shock

Retrieved on: 
Wednesday, April 10, 2024
Sepsis, Communication, Patient, Public, Fast Track, Adrenomedullin, Biomarker, Accelerated approval (FDA), Mortality, Septic shock, Death, DPP3, Septic, Medicine, Priority review, Organ dysfunction, Intensive care medicine, CMO, Collection, FDA, Infection, RCI, Food, Pharmaceutical industry

AdrenoMed is now preparing a confirmatory Phase IIb/III clinical trial to confirm the reduced septic shock mortality under enibarcimab treatment employing a precision medicine approach.

Key Points: 
  • AdrenoMed is now preparing a confirmatory Phase IIb/III clinical trial to confirm the reduced septic shock mortality under enibarcimab treatment employing a precision medicine approach.
  • AdrenoMed’s CEO Dr. Richard Jones commented: “We are very pleased that enibarcimab has received Fast Track designation from the FDA, recognizing its potential as an innovative biomarker-guided treatment against septic shock to fill the unmet medical need in this very serious condition with a high death toll.
  • With a mortality rate of 20-30% for sepsis [1] and 30%-50% for septic shock in developed countries, [2] sepsis represents an enormous public health burden and is responsible for almost 20% of all deaths worldwide.
  • Dr. Stephan Witte, CMO of AdrenoMed, said: “We are very confident that the use of enibarcimab in combination with two biomarkers, Adrenomedullin (bio-ADM) and circulating dipeptidyl peptidase 3 (cDPP3), holds the promise to become the first effective targeted treatment against septic shock.

Inozyme Pharma Announces Positive Topline Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ABCC6 Deficiency (PXE) and ENPP1 Deficiency

Retrieved on: 
Monday, April 8, 2024
Patient, Cerebrovascular disease, Stroke, Accelerated approval (FDA), European Medicines Agency, Doctor of Philosophy, Osteomalacia, PROMIS, Therapy, Conditional, Natural history, CTX, Pros, ADAS, Risk, DSM-IV codes, Cohort, Aes, Alkaline phosphatase, Rickets, EMA, PXE, Safety, Epilepsy, Clinical trial, Food, Hospital, Disability, BSAP, Cardiovascular disease, Disease, PD, Paresis, Consultant, Hypophosphatemia, Fatigue, Survivor, Sclera, FDA, Observational study, Pyrophosphate, Choroid, Incidence, Retina, Biomarker, Life, Event, Medical imaging

BOSTON, April 08, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced positive topline safety, pharmacokinetic (PK), pharmacodynamic (PD) and exploratory efficacy data from the Company’s ongoing Phase 1/2 clinical trials of INZ-701 in adults with ABCC6 Deficiency (PXE, pseudoxanthoma elasticum) and ENPP1 Deficiency.

Key Points: 
  • “We are excited by the excellent safety and preliminary efficacy profile of INZ-701 in adults with ABCC6 Deficiency,” said Douglas A. Treco, Ph.D., CEO of Inozyme Pharma.
  • The patients were assigned to three dose cohorts of INZ-701: 0.2 mg/kg (n=3), 0.6 mg/kg (n=3), and 1.8 mg/kg (n=4).
  • For trial design details, please see the section entitled “INZ-701 in ABCC6 Deficiency Phase 1/2 Clinical Trial Design” below.
  • For trial design details, please see the section entitled “INZ-701 in ENPP1 Deficiency Phase 1/2 Clinical Trial Design” below.

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.

Verastem Oncology Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Business Updates

Retrieved on: 
Thursday, March 14, 2024
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Strategic management, Diagnosis, Initiation, Research and development, Conference, Immunotherapy, Part, FAK, New Drug Application, Patient, Cancer, RAMP, Accelerated approval (FDA), NDA, Table, SGO, BRAF, Society, IND, Abstract, Bank statement, AACR, Pancreatic cancer, Investment, Verastem Oncology, MEK, Therapy, Safety, GAAP, Gynecologic Oncology (journal), Food, FDA, Annual general meeting, Pharmaceutical industry

Verastem Oncology ended the fourth quarter of 2023 with cash, cash equivalents and investments of $137.1 million.

Key Points: 
  • Verastem Oncology ended the fourth quarter of 2023 with cash, cash equivalents and investments of $137.1 million.
  • Research & development expenses for the 2023 Quarter were $22.5 million, compared to $10.7 million for the 2022 Quarter.
  • Selling, general & administrative expenses for the 2023 Quarter were $8.6 million, compared to $6.1 million for the 2022 Quarter.
  • These non-GAAP financial measures exclude certain amounts or expenses from the corresponding financial measures determined in accordance with GAAP.

Verastem Oncology Receives Orphan Drug Designation from FDA for Avutometinib Alone or in Combination With Defactinib in Recurrent Low-Grade Serous Ovarian Cancer

Retrieved on: 
Tuesday, March 5, 2024
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, NRAS, Woman, Recurrence, Food, Pain, FAK, ODD, Verastem Oncology, Accelerated approval (FDA), Patient, Survival, Disease, NDA, Cancer, Pharmaceutical industry

“The FDA Orphan Drug Designation for avutometinib alone or in combination with defactinib in low-grade serous ovarian cancer is an important step in recognizing this rare cancer as a distinct disease that currently has no FDA-approved treatments,” said Dan Paterson, president and chief executive officer of Verastem Oncology.

Key Points: 
  • “The FDA Orphan Drug Designation for avutometinib alone or in combination with defactinib in low-grade serous ovarian cancer is an important step in recognizing this rare cancer as a distinct disease that currently has no FDA-approved treatments,” said Dan Paterson, president and chief executive officer of Verastem Oncology.
  • “We are rapidly advancing the development program for avutometinib and defactinib in low-grade serous ovarian cancer with our ongoing Phase 3 clinical trial to deliver this new combination treatment to patients as quickly as possible.
  • LGSOC is a highly recurrent, chemotherapy-resistant cancer, associated with slow tumor growth and high mortality rate.
  • While chemotherapy is the standard of care for this disease, there are no treatments specifically approved by the FDA to treat LGSOC.

Indapta Therapeutics Receives U.S. FDA Fast Track Designation for Lead Clinical Drug Candidate IDP-023 for Non-Hodgkin’s Lymphoma and Myeloma

Retrieved on: 
Thursday, February 29, 2024
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, FDA, Clinical Trials, Fast Track, CMV, BLA, Cell, Biologics license application, Cytomegalovirus, Interleukin, Safety, Therapy, Cytokine, Accelerated approval (FDA), Patient, NK, Clinical trial, Multiple myeloma, Rituximab, Lymphoma, Cancer, Priority review, Pharmaceutical industry

Companies granted Fast Track designation may be eligible for more frequent interactions with the FDA, Accelerated Approval and Priority Review, and Rolling Review of a Biologic License Application (BLA).

Key Points: 
  • Companies granted Fast Track designation may be eligible for more frequent interactions with the FDA, Accelerated Approval and Priority Review, and Rolling Review of a Biologic License Application (BLA).
  • “This designation highlights the promise of Indapta’s highly potent NK cell platform and will further accelerate clinical development of our lead drug candidate, IDP-023, for two of the largest unmet needs in B-cell driven blood cancers, non-Hodgkin’s lymphoma and multiple myeloma,” said Dr. Mark Frohlich, CEO of Indapta.
  • Patients being enrolled now in Indapta’s Phase 1 clinical trial are receiving up to three planned doses of IDP-023 with or without interleukin-2.
  • To generate IDP-023, Indapta preferentially expands g-NK cells from healthy donors with increased numbers of g-NK cells, with low donor-to-donor variability.

FDA Approves First Cellular Therapy to Treat Patients with Unresectable or Metastatic Melanoma

Retrieved on: 
Friday, February 16, 2024
Human services, BRAF, Skin cancer, Neoplasm, Fever, Immune system, Melanoma, Cosmetics, Multimedia, Febrile neutropenia, Immunotherapy, Diarrhea, Hair loss, Dietary supplement, FDA, Food, Infection, Growth, Swelling, Tachycardia, PD-1, Edema, Cell, Hypoxia, Vaccine, Risk, Antibody, Animal, Metastasis, Safety, Heated tobacco product, Fatigue, Accelerated approval (FDA), Death, Rash, Public, BRAF V600, Patient, Protein, Chills, Sunlight, Hypotension, MEK, Cancer, Pharmaceutical industry

"Unresectable or metastatic melanoma is an aggressive form of cancer that can be fatal," said Peter Marks, M.D., Ph.D., director of the FDA's Center for Biologics Evaluation and Research (CBER).

Key Points: 
  • "Unresectable or metastatic melanoma is an aggressive form of cancer that can be fatal," said Peter Marks, M.D., Ph.D., director of the FDA's Center for Biologics Evaluation and Research (CBER).
  • Melanoma can spread to other parts of the body if not detected and treated early, resulting in metastatic disease.
  • Treatment for unresectable or metastatic melanoma may include immunotherapy using PD-1 inhibitors, which are antibodies targeting certain proteins in the body to help the immune system fight off cancer cells.
  • Those patients whose melanoma has progressed with these therapies have a high unmet medical need.

Nurix Therapeutics Reports Fourth Quarter and Fiscal Year 2023 Financial Results and Provides a Corporate Update

Retrieved on: 
Thursday, February 15, 2024
Fast Track, BTK, MCL, Research, B-cell lymphoma, ASH, NHL, Inflammation, FDA, CLL, Calendar, Mantle cell lymphoma, Manuscript, Medicinal chemistry, Lymphocytosis, BCL2, Lymphoma, Society, Chronic lymphocytic leukemia, Therapy, Pharmacology, Accelerated approval (FDA), Rheumatoid arthritis, Lymphoid leukemia, Paclitaxel, Non-Hodgkin lymphoma, Patient, Journal, Clinical trial, Science, IND, Publication, Trial of the century, DLBCL, Cancer, Priority review, Pharmaceutical industry

NX-2127 exhibited dose-dependent PK, leading to robust and sustained degradation of BTK and biologically relevant degradation of IKZF1 (Ikaros).

Key Points: 
  • NX-2127 exhibited dose-dependent PK, leading to robust and sustained degradation of BTK and biologically relevant degradation of IKZF1 (Ikaros).
  • NX-2127 had a manageable safety profile that was consistent with previous reports for BTK-targeted and immunomodulatory therapies.
  • During the year ended November 30, 2023, Nurix achieved research milestones under its collaborations with Gilead and Sanofi totaling $12.5 million and $7.0 million, respectively.
  • Cash, cash equivalents and marketable securities was $295.3 million as of November 30, 2023, compared to $268.7 million as of August 31, 2023.

Verastem Oncology Outlines Key 2024 Strategic Priorities and Upcoming Catalysts for Advancing Avutometinib and Defactinib and Broader Pipeline in RAS Pathway-Driven Cancers

Retrieved on: 
Monday, January 29, 2024
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Verastem Oncology, Patient, Accelerated approval (FDA), Toxicology, GLP, BIW, Part, Diagnosis, Safety, RAMP, Cancer, NSCLC, IND, Food, NDA, RAS, Lung cancer, FDA, Avutometinib, QD, Pancreatic cancer, Pharmaceutical industry

Verastem Oncology (Nasdaq:VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, today outlined key 2024 strategic priorities and upcoming catalysts to support advancement of its clinical programs in RAS pathway-driven cancers.

Key Points: 
  • Verastem Oncology (Nasdaq:VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, today outlined key 2024 strategic priorities and upcoming catalysts to support advancement of its clinical programs in RAS pathway-driven cancers.
  • “We have made significant progress in our commitment to advancing new solutions for RAS pathway-driven cancers and look forward to an exciting and catalyst-filled year ahead.
  • Safety and tolerability continued to be favorable and consistent with previously reported data.
  • Entered into a discovery and development collaboration with GenFleet Therapeutics (“GenFleet”) to advance three oncology discovery programs targeting RAS pathway-driven cancers.

Conversations on Cancer: Transforming Patient Lives by Therapeutic and Regulatory Innovations, European Medicines Agency, Amsterdam, the Netherlands, from 1 February 2024, 15:30 (CET) to 1 February 2024, 17:00 (CET)

Retrieved on: 
Friday, January 19, 2024
Role, Patient, OCE, Accelerated approval (FDA), Melanoma, European Medicines Agency, EMA, Outline, Location, Multiple myeloma, Therapy, Acute leukemia, Pharmaceutical industry

The conversation will focus on the introduction of certain innovative therapeutic classes in the past two decades that have revolutionized the care for patients with chronic myeloid leukemia, multiple myeloma, and melanoma.

Key Points: 
  • The conversation will focus on the introduction of certain innovative therapeutic classes in the past two decades that have revolutionized the care for patients with chronic myeloid leukemia, multiple myeloma, and melanoma.
  • The discussion will provide an overview of regulatory programs that expedite access to patients, including FDA’s Breakthrough therapy designation and Accelerated Approval; and EMA’s Prime and Conditional Approval.
  • The panel of patients, investigators and regulators aims to highlight successes from development to approval.
  • We’ll specifically outline:
    - Patient experiences with new innovative therapeutic classes;
    - Investigator approaches in development;
    - Role of regulatory collaboration and programs to expedite development and approval of novel, safe, and effective therapeutics.