Breakthrough therapy

Vaxcyte Provides Clinical and Regulatory Progress Update on Potential Best-in-Class Pneumococcal Conjugate Vaccine (PCV) Franchise

Retrieved on: 
Thursday, January 4, 2024
Licensure, Food, PCV, End, Breakthrough therapy, FDA, CMC, Ageing, Phase 10, SAN, Pivotal, Biologics license application, Advisory Committee, PCV20, Breakthrough, Data monitoring committee, Immunization, Initiation, IPD, PCV15, Infant, Safety, Tolerability, Genetically modified bacteria, BLA, Vaccine

-- Enrollment in Ongoing VAX-24 Infant Phase 2 Study Continues to Progress; Topline Data from Primary Immunization Series Expected by End of First Quarter of 2025, Followed by Topline Data from Booster Dose by End of 2025 --

Key Points: 
  • Vaxcyte’s carrier-sparing PCV franchise candidates, including VAX-24, a 24-valent PCV proceeding to Phase 3, and VAX-31, the Company’s next-generation 31-valent PCV, are being studied for the prevention of invasive pneumococcal disease (IPD).
  • The VAX-24 Phase 2 infant study continues to enroll participants in the second and final stage of the study.
  • This is a randomized, observer-blind, dose-finding two-stage clinical study evaluating the safety, tolerability and immunogenicity of VAX-24 in healthy infants.
  • Topline safety, tolerability and immunogenicity data from the ongoing adult Phase 1/2 study in the third quarter of 2024.

INOVIO Plans to Submit a BLA for INO-3107 as a Potential Treatment for RRP in Second Half of 2024 Under Accelerated Approval Program

Retrieved on: 
Wednesday, January 3, 2024
European Commission, Food, Priority review, Breakthrough therapy, FDA, Type, Health, DNA, Cancer, RRP, Recurrence, Biologics license application, Patient, Infection, Food and Drug Administration, BLA, Pharmaceutical industry

This announcement follows an Initial Comprehensive Multidisciplinary Breakthrough Therapy (Type B) Meeting with the FDA on critical aspects of the data package required to submit a BLA under the agency's accelerated approval program.

Key Points: 
  • This announcement follows an Initial Comprehensive Multidisciplinary Breakthrough Therapy (Type B) Meeting with the FDA on critical aspects of the data package required to submit a BLA under the agency's accelerated approval program.
  • "Based on productive discussions with the FDA, we believe we now have established a path to submitting a BLA for INO-3107 under the accelerated approval program," said Dr. Jacqueline Shea, INOVIO's President & Chief Executive Officer.
  • "Our plan is to complete the submission of our BLA in the second half of 2024 and request a Priority Review.
  • Concurrently, we will continue advancing our commercial plans, with the goal of being ready to launch INO-3107 in 2025."

Tonix Pharmaceuticals to Present at Two Upcoming Investor Conferences in January: Focus is on TNX-102 SL for the Management of Fibromyalgia Following Positive Topline Results in Second Pivotal Phase 3 Trial

Retrieved on: 
Wednesday, January 3, 2024
Long COVID, Annual report, Hydrogen chloride, Upsher-Smith Laboratories, Long, Failure, Infection, Breakthrough therapy, NIAID, IND, Government, Research, FDA, Sumatriptan, Prader–Willi syndrome, NDA, Cancer, CD40L, Orphan drug, Risk, Disease, COVID-19, Fibromyalgia, Smallpox, Union, NIH, Patient, Therapy, Security (finance), Nature, PWS, Autoimmunity, Pharmaceutical industry

CHATHAM, N.J., Jan. 03, 2024 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a biopharmaceutical company with marketed products and a pipeline of development candidates, announced today that Seth Lederman, M.D., President and Chief Executive Officer of Tonix Pharmaceuticals, will present and host investor meetings at the following January investor conferences:

Key Points: 
  • Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering.
  • Tonix’s priority is to submit a New Drug Application (NDA) to the FDA for TNX-102 SL (cyclobenzaprine HCl sublingual tablet), which has completed two positive Phase 3 studies for the management of fibromyalgia.
  • Tonix intends to meet with the FDA in the first half of 2024 and submit an NDA for the approval of TNX-102 SL for the management of fibromyalgia in the second half of 2024.
  • TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA.

Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies, European Medicines Agency, Amsterdam, the Netherlands, 26 November 2018

Retrieved on: 
Tuesday, January 2, 2024
PRIME, Industry, FDA, EMA, Patient, Safety, European Medicines Agency, Breakthrough therapy, Location, Pharmaceutical industry

Participants looked at specific industry case studies covering chemicals, biologicals and advanced therapies and experiences across different regions.

Key Points: 
  • Participants looked at specific industry case studies covering chemicals, biologicals and advanced therapies and experiences across different regions.
  • EMA and the United States Food and Drug Administration (FDA) launched PRIME and Breakthrough Therapy schemes to strengthen their support to the development of medicines for unmet medical needs to help patients to benefit from these therapies as early as possible.
  • This workshop is a joint collaboration between EMA and the FDA.
  • EMA has published a report on the workshop, containing recommendations from participants on next steps and areas to be further explored by EMA and the FDA.

MAPS PBC Announces New American Medical Association CPT III Codes for Psychedelic-Assisted Therapies Take Effect

Retrieved on: 
Tuesday, January 2, 2024
Continuity, Food, Priority review, Breakthrough therapy, Physician, MAPS, FDA, Intervention, Psychotherapy, NDA, SAN, MDMA, Mental health, PTSD, American Medical Association, Post-traumatic stress disorder, DEA, CPT, Safety, Pharmaceutical industry

SAN JOSE, Calif., Jan. 2, 2024 /PRNewswire/ -- MAPS Public Benefit Corporation ("MAPS PBC"), a clinical-stage company dedicated to changing the way mental health conditions are treated, announced that the American Medical Association ("AMA") current procedural terminology ("CPT") III codes for psychedelic-assisted therapies, first announced in July 2023, are now in effect. The new CPT codes will provide physicians and other qualified healthcare providers a means to seek coverage and reimbursement for delivering psychedelic-assisted therapy if approved by the U.S. Food and Drug Administration ("FDA").

Key Points: 
  • SAN JOSE, Calif., Jan. 2, 2024 /PRNewswire/ -- MAPS Public Benefit Corporation ("MAPS PBC"), a clinical-stage company dedicated to changing the way mental health conditions are treated, announced that the American Medical Association ("AMA") current procedural terminology ("CPT") III codes for psychedelic-assisted therapies, first announced in July 2023, are now in effect.
  • MAPS PBC and COMPASS Pathways collaborated to work with the AMA to create these new CPT III codes.
  • On March 3, 2023, the AMA published the Summary of Panel Actions which included the acceptance of the new CPT III codes.
  • With Breakthrough Therapy designation given to MDMA in 2017, MAPS PBC has requested the FDA grant Priority Review of the NDA.

BioNTech and DualityBio Receive FDA Breakthrough Therapy Designation for Antibody-Drug Conjugate Candidate BNT323/DB-1303 in Endometrial Cancer

Retrieved on: 
Thursday, December 21, 2023
Food, HER2, Breakthrough therapy, ASCO, Collection, FDA, ESGO, Mortality, Gene amplification, BNTX, Incidence, Endometrial cancer, BioNTech, Breakthrough, Fast track (FDA), Patient, Uterine cancer, Data, ADC, Pharmaceutical industry

The designation is based on encouraging topline data from a Phase 1/2 study ( NCT05150691 ) with BNT323/DB-1303 in patients with HER2-expressing advanced endometrial cancer.

Key Points: 
  • The designation is based on encouraging topline data from a Phase 1/2 study ( NCT05150691 ) with BNT323/DB-1303 in patients with HER2-expressing advanced endometrial cancer.
  • Endometrial or uterine cancer is the second most common gynecologic cancer globally, with over 400,000 cases occurring each year1,2 and both incidence and mortality are increasing3,4.
  • “The Breakthrough Therapy designation for BNT323/DB-1303 shows the potential of our ADC candidate to address current treatment challenges for patients with advanced HER2-expressing endometrial cancer who progressed under several lines of systemic therapy.
  • The BNT323/DB-1303 program received FDA Fast Track designation for the treatment of endometrial cancer in January 2023.

Inventiva announces the randomization of the first patient in China in the NATiV3 clinical trial and provides an update on its clinical development program

Retrieved on: 
Wednesday, December 20, 2023
Phase, Marketing, GLP1, Food, MRI, Risk, NASH, Euronext Paris, FDA, Breakthrough therapy, DMC, Disease, European, Diabetes, Clinical trial, Glycated hemoglobin, LEGEND, Lipid, Fatty liver disease, NMPA, EIB, Data monitoring committee, Type 2 diabetes, SGLT2, European Investment Bank, BofA Securities, Patient, T2D, Safety, Fibrosis, Data collection, Magnetic resonance imaging, F4, Scan (company), Pharmaceutical industry

The first patient was randomized in China in the NATiV3 Phase III clinical trial, triggering a milestone payment of $3 million from CTTQ to Inventiva.

Key Points: 
  • The first patient was randomized in China in the NATiV3 Phase III clinical trial, triggering a milestone payment of $3 million from CTTQ to Inventiva.
  • Analysis of the baseline characteristics of all patients randomized in the main cohort suggests a patient profile aligned with those of patients randomized in the NATIVE Phase II clinical trial.
  • Lanifibranor continues to show a favorable tolerability profile as confirmed by the third Data Monitoring Committee2 of November 2023.
  • Topline results of the proof-of-concept Phase II clinical trial, LEGEND, evaluating lanifibranor in combination with empagliflozin are expected in the first quarter of 2024.

Travere Therapeutics Initiates Pivotal Phase 3 Clinical Trial of Pegtibatinase for the Treatment of Classical Homocystinuria (HCU)

Retrieved on: 
Thursday, December 14, 2023
Patient, Food, Breakthrough therapy, Caregiver, Homocysteine, Research, FDA, Plasma, Intellectual, SAN, Therapy, Stroke, Orphan drug, CBS, Doctor of Philosophy, HCU, Diagnosis, Safety, Pharmaceutical industry

SAN DIEGO, Dec. 14, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (Nasdaq: TVTX) today announced the Company has opened enrollment in the HARMONY Study, a global, randomized pivotal Phase 3 clinical trial of pegtibatinase, a novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocystinuria (HCU).

Key Points: 
  • SAN DIEGO, Dec. 14, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (Nasdaq: TVTX) today announced the Company has opened enrollment in the HARMONY Study, a global, randomized pivotal Phase 3 clinical trial of pegtibatinase, a novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocystinuria (HCU).
  • Classical HCU is a rare genetic metabolic disorder caused by a deficiency in the enzyme cystathionine beta synthase (CBS).
  • “Classical HCU is a devastating rare disease that often manifests in childhood and can lead to serious complications due to toxic levels of homocysteine.
  • Participants will be randomized 1:1 to receive 2.5 mg/kg of pegtibatinase or placebo, administered subcutaneously, for a 24-week blinded treatment duration.

Tonix Pharmaceuticals to Present at The National Academies Committee on the Current State of Research, Development, and Stockpiling of Smallpox Medical Countermeasures Public Meeting

Retrieved on: 
Monday, December 11, 2023
Long COVID, Hydrogen chloride, Long, Upsher-Smith Laboratories, Failure, National academy, Infection, Breakthrough therapy, NIAID, Binge eating disorder, Government, Research, Psychiatry, Sumatriptan, FDA, Pain, Prader–Willi syndrome, Cancer, CD40L, Orphan drug, Tonix Pharmaceuticals, Social anxiety disorder, Risk, COVID-19, Disease, Smallpox, CNS, Fibromyalgia, Obesity, Neurology, NIH, Central nervous system, Therapy, Security (finance), Annual report, Autoimmunity, Pharmaceutical industry

Dr. Lederman will participate in a panel discussion on Vaccine Research & Development taking place from 2:00 - 2:45 p.m.

Key Points: 
  • Dr. Lederman will participate in a panel discussion on Vaccine Research & Development taking place from 2:00 - 2:45 p.m.
  • A webcast of the meeting can be found here and will be available under the IR Events tab of the Tonix website at www.tonixpharma.com following the presentation.
  • Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering.
  • Zembrace SymTouch and Tosymra are each indicated for the treatment of acute migraine with or without aura in adults.

Osteal Therapeutics’ VT-X7 Receives FDA’s Breakthrough Therapy Designation for the treatment of periprosthetic joint infection of the hip and knee and completes enrollment of the pivotal APEX-2 trial

Retrieved on: 
Tuesday, December 5, 2023
Hip, Food, Breakthrough therapy, Knee, FDA, Disease, Mortality, APEX, Patient, Safety, Pharmaceutical industry

DALLAS, Dec. 05, 2023 (GLOBE NEWSWIRE) -- The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for VT-X7, Osteal Therapeutics’ (“Osteal’s”) investigational drug therapy for periprosthetic joint infection (“PJI”) of the hip and knee.

Key Points: 
  • DALLAS, Dec. 05, 2023 (GLOBE NEWSWIRE) -- The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for VT-X7, Osteal Therapeutics’ (“Osteal’s”) investigational drug therapy for periprosthetic joint infection (“PJI”) of the hip and knee.
  • Breakthrough Therapy Designation helps to accelerate commercialization of new drugs for the treatment of serious conditions when early clinical evidence shows the potential to provide a significant improvement of current standard of care.
  • Previously, FDA granted VT-X7 Orphan Drug, Qualified Infectious Disease Product and Fast Track designations.
  • The company also announced completion of enrollment in APEX-2 the pivotal clinical trial for VT-X7.