Breakthrough therapy

Palvella Therapeutics and Ligand Pharmaceuticals Expand Strategic Partnership to Accelerate Phase 3 Development of QTORIN™ rapamycin for Microcystic Lymphatic Malformations and Additional High Unmet Need Clinical Indications

Retrieved on: 
Friday, December 1, 2023
Patient, Partnership, FDA, Breakthrough therapy, Ligand Pharmaceuticals, Standard of care, Disease, Therapy, Patent, Skin, Standard, Pharmaceutical industry

WAYNE, Pa., Dec. 01, 2023 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc., a clinical-stage biopharmaceutical company developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no FDA-approved therapies, and Ligand Pharmaceuticals Incorporated (Nasdaq: LGND), a biopharmaceutical company enabling scientific advancement through supporting the clinical development of high-value medicines, today announced the expansion of their strategic partnership to accelerate Phase 3 development of QTORIN™ rapamycin for the treatment of Microcystic Lymphatic Malformations (Microcystic LMs).

Key Points: 
  • “We are pleased to expand our strategic partnership with Ligand Pharmaceuticals, a recognized leader with an established track record in partnering with rare disease companies to accelerate development of high-value therapies,” said Wes Kaupinen, Founder and Chief Executive Officer of Palvella.
  • In return, Ligand’s existing tiered royalty on worldwide commercial sales of QTORIN rapamycin increased to 8.0–9.8%.
  • The U.S. FDA granted Breakthrough Therapy Designation to QTORIN rapamycin for the treatment of Microcystic Lymphatic Malformations in November 2023.
  • In addition to Breakthrough Therapy Designation, the FDA previously granted Fast Track Designation and Orphan Drug Designation to QTORIN rapamycin for the treatment of Microcystic LMs.

Tonix Pharmaceuticals to Present at Two Upcoming Investor Conferences in December

Retrieved on: 
Tuesday, November 28, 2023
Long COVID, Hydrogen chloride, Upsher-Smith Laboratories, Long, Failure, Infection, Breakthrough therapy, NIAID, Binge eating disorder, Government, Psychiatry, Research, Sumatriptan, FDA, Pain, Prader–Willi syndrome, Cancer, CD40L, Orphan drug, Social anxiety disorder, Risk, Disease, COVID-19, Fibromyalgia, Obesity, CNS, Smallpox, Virtual, Neurology, NIH, Central nervous system, Therapy, Security (finance), Annual report, Autoimmunity, Pharmaceutical industry

Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering.

Key Points: 
  • 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.
  • Tonix’s development portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates.
  • Tonix’s CNS development portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions.

Dupixent® (dupilumab) Significantly Reduced COPD Exacerbations in Second Positive Phase 3 Trial, Accelerating FDA Submission and Confirming Potential to Become First Approved Biologic for This Serious Disease

Retrieved on: 
Monday, November 27, 2023
Patient, Regeneron Pharmaceuticals, Food, Smoker, Aes, Breakthrough therapy, Death, Upper respiratory tract infection, Diarrhea, FDA, Hypertension, Headache, Common, Inflammation, COVID-19, European Medicines Agency, Doctor of Philosophy, International development, Back pain, Phenotype, Safety, History, Pharmaceutical industry

TARRYTOWN, N.Y. and PARIS, Nov. 27, 2023 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the second Dupixent® (dupilumab) investigational Phase 3 chronic obstructive pulmonary disease (COPD) trial (NOTUS) showed that Dupixent significantly reduced (34%) exacerbations, confirming positive results from the landmark Phase 3 BOREAS trial. The NOTUS trial also confirmed that treatment with Dupixent led to rapid and significant improvements in lung function by 12 weeks and were sustained at 52 weeks.

Key Points: 
  • “These results demonstrate the important role of type 2 inflammation in yet another chronic and debilitating disease, and the ability of Dupixent to address this inflammation.
  • Patients receiving Dupixent compared to placebo experienced:
    34% reduction in moderate or severe acute COPD exacerbations over 52 weeks (p=0.0002), the primary endpoint.
  • BOREAS results showed:
    30% reduction in moderate or severe acute COPD exacerbations over 52 weeks (p=0.0005), the primary endpoint.
  • The safety and efficacy of Dupixent in COPD are currently under clinical investigation and have not been evaluated by any regulatory authority.

Press Release: Dupixent® significantly reduced COPD exacerbations in second positive Phase 3 trial, accelerating FDA submission and confirming potential to become first approved biologic for this serious disease

Retrieved on: 
Monday, November 27, 2023
Patient, Regeneron Pharmaceuticals, Sanofi, Food, Smoker, Death, Aes, Breakthrough therapy, Upper respiratory tract infection, Diarrhea, FDA, Hypertension, Headache, Disease, Common, Inflammation, COVID-19, European Medicines Agency, Back pain, Phenotype, Safety, History, Pharmaceutical industry

The second Dupixent® (dupilumab) investigational Phase 3 chronic obstructive pulmonary disease (COPD) trial (NOTUS) has shown that Dupixent significantly reduced (34%) exacerbations, confirming positive published results from the landmark Phase 3 BOREAS trial.

Key Points: 
  • The second Dupixent® (dupilumab) investigational Phase 3 chronic obstructive pulmonary disease (COPD) trial (NOTUS) has shown that Dupixent significantly reduced (34%) exacerbations, confirming positive published results from the landmark Phase 3 BOREAS trial.
  • These results were from an interim analysis and, given the overwhelming positive efficacy of the primary endpoint, will be considered the primary analysis of the trial.
  • Patients receiving Dupixent compared to placebo experienced:
    34% reduction in moderate or severe acute COPD exacerbations over 52 weeks (p=0.0002), the primary endpoint.
  • The safety and efficacy of Dupixent in COPD are currently under clinical investigation and have not been evaluated by any regulatory authority.

Patritumab Deruxtecan Granted Priority Review in the U.S. for Certain Patients with Previously Treated Locally Advanced or Metastatic EGFR-Mutated Non-Small Cell Lung Cancer

Retrieved on: 
Friday, December 22, 2023
Biotechnology, Pharmaceutical, Health, Oncology, MSD, Senior, Food, Leukopenia, Priority review, Hypokalemia, Daiichi Sankyo, FDA, Breakthrough therapy, PDUFA, EGFR, ILD, Anemia, Fatigue, HER3, Neutropenia, MD, Biologics license application, Recurrence, Patritumab, Weakness, Thrombocytopenia, Merck & Co., DXD, Patient, Safety, Medicine, MRK, ADC, NSCLC, BLA, Pharmaceutical industry

The Prescription Drug User Fee Act (PDUFA) date, the FDA action date for their regulatory decision, is June 26, 2024.

Key Points: 
  • The Prescription Drug User Fee Act (PDUFA) date, the FDA action date for their regulatory decision, is June 26, 2024.
  • The Priority Review follows receipt of Breakthrough Therapy Designation granted by the FDA in December 2021.
  • RTOR allows the FDA to review components of an application before submission of the complete application.
  • Twelve patients (5.3%) had confirmed treatment-related interstitial lung disease (ILD) as determined by an independent adjudication committee.

Palvella Therapeutics Announces U.S. FDA Breakthrough Therapy Designation Granted to QTORIN™ 3.9% Rapamycin Anhydrous Gel (QTORIN™ rapamycin) for the Treatment of Microcystic Lymphatic Malformations

Retrieved on: 
Thursday, November 16, 2023
Disease, Natural history, Food, European Medicines Agency, Therapy, Cellulitis, Patient, Breakthrough therapy, Infection, FDA, Breakthrough, DSM-IV codes, Skin, Bleeding, Cyst, Orphan, Life, Patent, Pharmaceutical industry, Microcystic lymphatic malformation

WAYNE, Pa., Nov. 16, 2023 (GLOBE NEWSWIRE) -- Palvella Therapeutics, Inc., a clinical-stage biopharmaceutical company developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no FDA-approved therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to QTORIN rapamycin™ for the treatment of Microcystic Lymphatic Malformations (Microcystic LMs). QTORIN rapamycin is a novel, 3.9% rapamycin anhydrous gel currently under development by Palvella for the treatment of Microcystic LMs and other serious, functionally debilitating skin diseases driven by the overactivation of the mammalian target of rapamycin (mTOR) pathway.

Key Points: 
  • QTORIN rapamycin is a novel, 3.9% rapamycin anhydrous gel currently under development by Palvella for the treatment of Microcystic LMs and other serious, functionally debilitating skin diseases driven by the overactivation of the mammalian target of rapamycin (mTOR) pathway.
  • Microcystic LMs is a rare, chronically debilitating genetic disease caused by dysregulation of the PI3K/mTOR pathway.
  • In addition to Breakthrough Therapy Designation, the FDA previously granted Fast Track Designation and Orphan Drug Designation to QTORIN rapamycin for the treatment of Microcystic LMs.
  • The European Medicines Agency has also granted Orphan Drug Designation to QTORIN rapamycin for the treatment of Microcystic LMs.

AnHeart Therapeutics and Foundation Medicine Announce Collaboration to Develop Tissue-Based and Liquid-Based Companion Diagnostics for Taletrectinib, a ROS1 Inhibitor

Retrieved on: 
Thursday, December 14, 2023
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Foundation Medicine, Food, Patient, Liquid, Breakthrough therapy, ROS1, FDA, Lung cancer, MD, Cancer, Doctor of Philosophy, NGS, Physician, NSCLC, Pharmaceutical industry

Taletrectinib is a potential best-in-class ROS1 inhibitor being evaluated for the treatment of patients with advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC), an underserved group of lung cancer patients in need of new options.

Key Points: 
  • Taletrectinib is a potential best-in-class ROS1 inhibitor being evaluated for the treatment of patients with advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC), an underserved group of lung cancer patients in need of new options.
  • “We are excited to collaborate with Foundation Medicine on the development of both tissue- and liquid-based companion diagnostics to help identify patients who may benefit from taletrectinib,” said Lian Li, MD, PhD, AnHeart’s U.S. Chief Medical Officer.
  • Foundation Medicine is the global leader in companion diagnostic approvals.
  • The company has approximately 60% of all U.S. companion diagnostic approvals for next-generation sequencing (NGS) testing.

Verastem Oncology Announces Initiation of a Confirmatory Phase 3 Trial of Avutometinib and Defactinib in Recurrent Low-Grade Serous Ovarian Cancer

Retrieved on: 
Wednesday, December 13, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, NDA, Paclitaxel, Woman, Institute of Cancer Research, Research, Safety, Food, Avutometinib, Cancer, Letrozole, Disease, Verastem Oncology, Patient, Anastrozole, GOG, PFS, Consultant, RAMP, Breakthrough therapy, Part, FDA, NHS foundation trust, FRAME, ORR, The Institute, Ovarian cancer, FRCP, Doctor of Philosophy, Physician, Accelerated approval (FDA), Royal Marsden Hospital, Pharmaceutical industry, MBBS, Topotecan

“Patients and treating physicians have advocated for more research and development in support of LGSOC.

Key Points: 
  • “Patients and treating physicians have advocated for more research and development in support of LGSOC.
  • At Verastem Oncology, we are moving with urgency to offer a Phase 3 study specifically directed at this disease in an effort to address this need,” said Dan Paterson, President and Chief Executive Officer, Verastem Oncology.
  • RAMP 301 is a global trial with enrollment open in the U.S. and planned enrollment in Canada, the United Kingdom, Europe, Australia, and Korea.
  • Dr. Grisham and Dr. Banerjee are paid consultants for Verastem Oncology.

CG Oncology Receives Both FDA Fast Track and Breakthrough Therapy Designation for Cretostimogene Grenadenorepvec in High-Risk BCG-Unresponsive Non-Muscle Invasive Bladder Cancer

Retrieved on: 
Tuesday, December 5, 2023
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, BCG, Woman, Food, NMIBC, BCAN, Carcinoma, Breakthrough therapy, Disease, Bladder cancer, Patient, Fast track (FDA), Caregiver, Cancer, SUO, Society, Bacillus, Pharmaceutical industry

CG Oncology, Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted both Fast Track Designation and Breakthrough Therapy Designation for cretostimogene grenadenorepvec in high-risk Bacillus Calmette-Guérin (BCG)-unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC) with carcinoma in situ with or without Ta or T1 (papillary) tumors.

Key Points: 
  • CG Oncology, Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted both Fast Track Designation and Breakthrough Therapy Designation for cretostimogene grenadenorepvec in high-risk Bacillus Calmette-Guérin (BCG)-unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC) with carcinoma in situ with or without Ta or T1 (papillary) tumors.
  • “Receiving both FDA Fast Track and Breakthrough Therapy Designation is an important milestone in the development of cretostimogene grenadenorepvec and for patients with bladder cancer who urgently need more therapeutic options,” said Ambaw Bellete, President & Chief Operating Officer, CG Oncology.
  • CG Oncology looks forward to working with the FDA to advance cretostimogene grenadenorepvec as a potential backbone therapy in bladder cancer.
  • “The Bladder Cancer Advocacy Network (BCAN) is grateful for the expedited review of this potential treatment option for bladder cancer patients with high-risk BCG-unresponsive NMIBC.

FDA Grants Priority Review for Supplemental Biologics License Application (sBLA) of PADCEV® (enfortumab vedotin-ejfv) with KEYTRUDA® (pembrolizumab) for First-Line Treatment of Advanced Bladder Cancer

Retrieved on: 
Thursday, November 30, 2023
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, FDA, Clinical Trials, Cisplatin, Priority review, Safety, Food, Progression-free survival, Breakthrough therapy, Transitional cell carcinoma, Carcinoma, Ureter, Bladder cancer, Patient, PFS, Pembrolizumab, Brentuximab vedotin, PDUFA, Inc., Pharmaceutical industry

Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of May 9, 2024.

Key Points: 
  • Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of May 9, 2024.
  • The FDA is reviewing the application under its Real-Time Oncology Review (RTOR) program, which aims to explore a more efficient review process to ensure that safe and effective treatments are available to patients as early as possible.
  • If approved, this combination would be the first treatment option for cisplatin eligible and ineligible patients.
  • In February 2020, PADCEV in combination with KEYTRUDA was granted Breakthrough Therapy designation by the FDA and the EV-103 sBLA received Priority Review designation in December 2022.