Breakthrough therapy

HUTCHMED and Innovent Jointly Announce NDA Acceptance in China for Fruquintinib Combination with Sintilimab for the Treatment of Advanced Endometrial Cancer with Priority Review Status

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화요일, 4월 2, 2024
Toxicity, Radiation, Sintilimab, NMPA, Endometrial cancer, Disease, DCR, New Drug Application, Patient, NDA, Breakthrough therapy, Senior, Priority review, HKEX, Dor, IRC, Prognosis, PFS, Pharmaceutical industry

HONG KONG, SHANGHAI, China & FLORHAM PARK, N.J., April 02, 2024 (GLOBE NEWSWIRE) -- HUTCHMED (China) Limited (“HUTCHMED”) (Nasdaq/AIM:​HCM; HKEX:​13) and Innovent Biologics, Inc. (“Innovent”) (HKEX:1801) today jointly announce that the New Drug Application (“NDA”) for the combination of fruquintinib and sintilimab for the treatment of patients with advanced endometrial cancer with pMMR1 or non-MSI-H2 tumors that have failed prior systemic therapy but are not candidates for curative surgery or radiation has been accepted and granted priority review by the China National Medical Products Administration (“NMPA”).

Key Points: 
  • The NDA is supported by data from FRUSICA-1, the endometrial cancer registration cohort of a multi-center, open-label Phase II study investigating fruquintinib in combination with sintilimab in endometrial cancer patients who experienced disease recurrence, disease progression or intolerable toxicity with treatment on platinum-based doublet chemotherapy.
  • Data from FRUSICA-1 will be submitted for presentation at an upcoming medical conference.
  • “This is the first regulatory filing for the combination of fruquintinib and the immune checkpoint inhibitor sintilimab.
  • “Endometrial cancer remains one of the most common gynecological malignancies.

ArriVent BioPharma Reports Full Year 2023 Financial Results

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목요일, 3월 28, 2024
Research, EGFR, Conference, Breakthrough therapy, NSCLC, Patient, Food and Drug Administration, Lung cancer, Therapy, PACC, Safety, ADC, FDA, SHP2, Food, Pharmaceutical industry

In September 2023, ArriVent and its partner, Shanghai Allist Pharmaceuticals Company, Ltd. ("Allist”), presented interim Phase 1b results at the World Conference on Lung Cancer.

Key Points: 
  • In September 2023, ArriVent and its partner, Shanghai Allist Pharmaceuticals Company, Ltd. ("Allist”), presented interim Phase 1b results at the World Conference on Lung Cancer.
  • In January 2024, ArriVent appointed Winston Kung as Chief Financial Officer and Treasurer, bringing over 20 years of extensive leadership experience, most recently as Chief Financial Officer and Chief Operating Officer at PMV Pharmaceuticals.
  • Research and development expenses were $64.9 million and $30.4 million for the years ended December 31, 2023 and 2022, respectively.
  • Net loss was $69.3 million and $37.0 million for the years ended December 31, 2023 and 2022, respectively.

Tonix Pharmaceuticals Announces Pricing of $4.4 Million Registered Direct Offering

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목요일, 3월 28, 2024
Failure, Madison Avenue, Exercise, Fibromyalgia, Infection, Cocaine, Autoimmunity, Central nervous system, Form, Telephone, Patient, CNS, Research, Sale, NDA, Disease, Cancer, Long, Security (finance), Breakthrough therapy, Nature, Risk, Annual report, Therapy, Series, CD40L, FDA, Sumatriptan, Government, Food

The closing of the offering is expected to take place on or about April 1, 2024, subject to the satisfaction of customary closing conditions.

Key Points: 
  • The closing of the offering is expected to take place on or about April 1, 2024, subject to the satisfaction of customary closing conditions.
  • The gross proceeds of the offering will be approximately $4.4 million before deducting placement agent fees and other estimated offering expenses payable by the Company.
  • A.G.P./Alliance Global Partners is acting as sole placement agent for the offering.
  • This offering is being made pursuant to an effective shelf registration statement on Form S-3 (File No.

SAB Biotherapeutics Announces Clinical Partnership with Naval Medical Research Center to Advance Potential Influenza Treatment

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월요일, 3월 25, 2024
Disease, Development, Immunotherapy, Partnership, T1D, Patient, NMRC, Research, Mortality, Death, Breakthrough therapy, Infection, CDC, Vaccination, Safety, SAB, Therapy, Vaccine, Immunoglobulin G, Food

The NMRC Clinical Trials Center, located in Bethesda, Maryland, will be conducting this PK study under the leadership of Cmdr.

Key Points: 
  • The NMRC Clinical Trials Center, located in Bethesda, Maryland, will be conducting this PK study under the leadership of Cmdr.
  • SAB has utilized its proprietary DiversitAb™ platform to manufacture SAB-176, fully human polyclonal antibodies targeting influenza from Transchromosomic (Tc) Bovine™.
  • SAB-176 is a novel multi-target biologic that has shown sustained neutralization activity across multiple virus strains of Influenza A and B.
  • Influenza is responsible for approximately 226,000 excess hospitalizations and 30,000 to 50,000 deaths each year in the United States alone.

IgA Nephropathy Pipeline Dynamics Signal Further Treatment Evolution on the Horizon

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목요일, 3월 14, 2024
Nephrology, Indoor Living, Novartis, Patient, Alnylam Pharmaceuticals, Perception, SGLT2, Immunoglobulin A, Budesonide, NDA, Record, Breakthrough therapy, UPCR, Atrasentan, Kidney, EGFR, Alexion, FDA, CHMP, Physician, Health, Pharmaceutical industry

For several years, Spherix Global Insights has been closely tracking the IgAN market and recently released its fourth annual US patient chart audit study.

Key Points: 
  • For several years, Spherix Global Insights has been closely tracking the IgAN market and recently released its fourth annual US patient chart audit study.
  • Spherix is the leading authority on IgAN market dynamics and will continue to provide insight into how nephrologists are treating and approaching care for their IgAN patients.
  • Patient Chart Dynamix™ is an independent, data-driven service unveiling real patient management patterns through rigorous analysis of large-scale patient chart audits.
  • Insights reveal the “why” behind treatment decisions, include year over year trending to quantify key aspects of market evolution, and integrate specialists’ attitudinal & demographic data to highlight differences between stated and actual treatment patterns.

VOYDEYA™ approved in the US as add-on therapy to ravulizumab or eculizumab for treatment of extravascular hemolysis in adults with the rare disease PNH

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월요일, 4월 1, 2024
Biotechnology, FDA, Health, Pharmaceutical, Clinical Trials, Factor D, Arthralgia, Chest pain, Confusion, Neuropsychological test, Infection, Eculizumab, Risk, Headache, ALPHA, PNH, C5, Patient, Orphan, Mitigation, Vaccine, Light, Paroxysmal nocturnal hemoglobinuria, EVH, Bacteria, University, Chills, MD, Breakthrough therapy, Cough, European Medicines Agency, Fatigue, Nausea, Rash, Fever, Priority, Safety, Immune system, REMS, Division, Vomiting, Hematology, Patient safety, Medicine

VOYDEYA has also been granted Orphan Drug Designation in the US, European Union (EU) and Japan for the treatment of PNH.

Key Points: 
  • VOYDEYA has also been granted Orphan Drug Designation in the US, European Union (EU) and Japan for the treatment of PNH.
  • You must complete or update meningococcal vaccine(s) and streptococcus vaccine(s) at least 2 weeks before your first dose of VOYDEYA.
  • If you have been vaccinated against these bacteria in the past, you might need additional vaccinations before starting VOYDEYA.
  • Carry it with you at all times during treatment and for 1 week after your last VOYDEYA dose.

InnoCare Announces Dosing of First Patient in Clinical Study of the Combination of SHP2 Inhibitor ICP-189 and EGFR Inhibitor Furmonertinib

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금요일, 3월 29, 2024
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Lung cancer, EGFR, SRC, NSCLC, Patient, Breakthrough therapy, Trial of the century, DLT, Clinical trial, PACC, HKEX, FDA, SHP2, Food, Pharmaceutical industry

In July 2023, InnoCare and ArriVent BioPharma, Inc. (Nasdaq: AVBP) announced a clinical development collaboration, accelerating the clinical trial of ICP-189 in combination with furmonertinib in patients with advanced or metastatic NSCLC in China.

Key Points: 
  • In July 2023, InnoCare and ArriVent BioPharma, Inc. (Nasdaq: AVBP) announced a clinical development collaboration, accelerating the clinical trial of ICP-189 in combination with furmonertinib in patients with advanced or metastatic NSCLC in China.
  • Furmonertinib is being advanced by ArriVent in global studies in patients with advanced or metastatic NSCLC with EGFR mutations, including exon 20 insertion mutations.
  • Furmonertinib is also being evaluated for the treatment of NSCLC patients with EGFR P-loop alpha-c helix compressing (PACC) mutations.
  • We will accelerate the clinical study and expect this innovative therapy to benefit more NSCLC patients early.”

FDA Approves Merck’s WINREVAIR™ (sotatercept-csrk), a First-in-Class Treatment for Adults with Pulmonary Arterial Hypertension (PAH, WHO* Group 1)

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화요일, 3월 26, 2024
Cardiology, Biotechnology, Pharmaceutical, Oncology, Health, FDA, Clinical Trials, Other Health, MSD, FC, Exercise, Breakthrough therapy, Blood, Risk, Patient, Orthostatic hypertension, MRK, World Health Organization, Heart, WHO, Multimedia, PAH, Food, Pharmaceutical industry

“The Pulmonary Hypertension Association welcomes the development of new therapies for those with PAH,” said Matt Granato, president and chief executive officer, Pulmonary Hypertension Association.

Key Points: 
  • “The Pulmonary Hypertension Association welcomes the development of new therapies for those with PAH,” said Matt Granato, president and chief executive officer, Pulmonary Hypertension Association.
  • In the WINREVAIR treatment group, the placebo-adjusted median increase in 6MWD was 41 meters (95% CI: 28, 54; p
  • The median treatment difference in PVR between WINREVAIR and placebo was -235 dynes*sec/cm5 (95% CI: -288, -181; p
  • The median treatment difference in NT-proBNP between WINREVAIR and placebo was -442 pg/mL (95% CI: -574, -310; p

Cybin Initiates Phase 2 Proof-of-Concept Study of CYB004 in Generalized Anxiety Disorder

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금요일, 3월 15, 2024
Research, Mental Health, FDA, Other Health, Biotechnology, General Health, Pharmaceutical, Health, Science, Other Science, GAD, Selective serotonin reuptake inhibitor, Breakthrough therapy, Inc., Patient, Disease, Human, Anxiety, DMT, Epidemiology, Psychiatry, Anxiety disorder, Safety, E36, Food, Pharmaceutical industry

“Initiation of the Phase 2 study of CYB004 for GAD is an exciting step forward for our deuterated DMT program,” said Doug Drysdale, Chief Executive Officer of Cybin.

Key Points: 
  • “Initiation of the Phase 2 study of CYB004 for GAD is an exciting step forward for our deuterated DMT program,” said Doug Drysdale, Chief Executive Officer of Cybin.
  • The CYB004-002 Phase 2 study is a randomized, double-blind study which will evaluate the safety and efficacy of CYB004 in participants with GAD, with concomitant antidepressant/anxiolytic treatment and co-morbid depression allowed.
  • The study will enroll participants with moderate to severe GAD and a score of ≥10 on the GAD-7 anxiety scale.
  • Global, regional and national burden of anxiety disorders from 1990 to 2019: results from the Global Burden of Disease Study 2019.

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

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수요일, 3월 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.