Breakthrough therapy

Ligand Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Tuesday, February 27, 2024
Oncology, Health, Clinical Trials, General Health, Pharmaceutical, Biotechnology, MRK, Research, CRL, New Drug Application, Investment, Eisai, Growth, Sale, Parent, Novin, Focal segmental glomerulosclerosis, Streptococcus, LGND, FSGS, Breakthrough therapy, BLA, AED, GAAP, Therapy, NASH, Liver, NDA, Depreciation, Breast cancer, Total, Forecasting, Perampanel, Shanda, Telecanthus, ID, Retirement, PDUFA, Vaccine, Symantec Endpoint Protection, Type 2 diabetes, Safety, VRNA, Molluscum contagiosum, F2, Acquisition, Caregiver, Merck, AASLD, FDA, Telephone, Food, Pneumonia, Income tax, Hepatology, COVID-19, Element, Patient, SPA, Pharmaceutical industry

Royalties for the fourth quarter of 2023 were $22.5 million, compared with $22.0 million for the same period in 2022.

Key Points: 
  • Royalties for the fourth quarter of 2023 were $22.5 million, compared with $22.0 million for the same period in 2022.
  • Costs of Captisol sales were $1.6 million for the fourth quarter of 2023, compared with $21.6 million for the same period in 2022, with the decrease due to lower total Captisol sales during the fourth quarter of 2023 and $9.8 million in accelerated depreciation on Captisol manufacturing equipment during the fourth quarter of 2022.
  • Amortization of intangibles was $8.3 million for the fourth quarter of 2023, compared with $8.5 million for the same period in 2022.
  • Travere also announced fourth quarter 2023 results reporting that it received 459 new patient start forms for FILSPARI in the fourth quarter of 2023 and net product sales of $14.9 million for the fourth quarter.

Bayer Receives U.S. FDA Breakthrough Therapy Designation for BAY 2927088 for Non-Small Cell Lung Cancer Harboring HER2 Activating Mutations

Retrieved on: 
Monday, February 26, 2024
Research, FDA, Clinical Trials, Biotechnology, Health, Pharmaceutical, General Health, Science, Oncology, NSCLC, EGFR, Neoplasm, Physician, Food, MIT, Harvard University, Breakthrough, Broad Institute, HER2, Safety, Bayer, Pharmacokinetics, Patient, Lung cancer, Mutation, Breakthrough therapy, ERBB2, TKI, Pharmaceutical industry

Bayer announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for BAY 2927088 for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have received a prior systemic therapy.

Key Points: 
  • Bayer announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for BAY 2927088 for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have received a prior systemic therapy.
  • The Breakthrough Therapy designation is supported by preliminary clinical evidence from the Phase I, open-label, multicenter first-in-human study (NCT05099172) evaluating the safety, pharmacokinetics and preliminary efficacy of BAY 2927088 in adult patients with advanced NSCLC harboring HER2 or EGFR.
  • “Early clinical evidence suggests that BAY 2927088, our investigational novel oral tyrosine kinase inhibitor, has the potential to benefit patients with NSCLC harboring a HER2 mutation that have progressed on a prior systemic therapy and currently have no other approved treatment available,” said Dominik Ruettinger, M.D., Ph.D., Head of Research and Early Development for Oncology at Bayer’s Pharmaceuticals Division.
  • “This Breakthrough Therapy designation is a significant milestone in our relentless efforts to develop innovative therapies for the treatment of lung cancer characterized by specific genomic markers.

U.S. Food and Drug Administration (FDA) Grants Priority Review of Epcoritamab (EPKINLY®) for Difficult-to-Treat Relapsed or Refractory Follicular Lymphoma (FL)

Retrieved on: 
Tuesday, February 27, 2024
ABBV, EMA, ASH, FDA, Food, Follicular lymphoma, AbbVie, Breakthrough therapy, Exposition, Cytokine release syndrome, Risk, Annual general meeting, Safety, Hematology, Society, European Medicines Agency, CRS, Patient, Diagnosis, Priority review, DLBCL

NORTH CHICAGO, Ill., Feb. 27, 2024 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review of the supplemental Biologics License Application (sBLA) for epcoritamab-bysp, a subcutaneously administered T-cell engaging bispecific antibody for the treatment of adult relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of therapy. If approved, epcoritamab-bysp (EPKINLY®) will be the first and only subcutaneous bispecific antibody to treat adults with R/R FL after two lines of prior therapy, marking its second indication following FDA and European Medicines Agency (EMA) approval of R/R third-line diffuse large B-cell lymphoma (DLBCL) treatment.

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.
  • This designation shortens the review period to six months compared to 10 months for Standard Review.
  • "Together with our partner Genmab, we are committed to further advancing epcoritamab as a core lymphoma therapy."
  • Both companies will pursue additional international regulatory approvals for the investigational R/R FL indication and additional approvals for the R/R DLBCL indication.

Sibeprenlimab Receives U.S. FDA Breakthrough Therapy Designation for the Treatment of Immunoglobulin A Nephropathy

Retrieved on: 
Friday, February 16, 2024
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Clinical Trials, Chronic kidney disease, Commercialization, Caregiver, Multimedia, Food, BLA, Medication, IgA nephropathy, Risk, RAAS, OPDC, Life expectancy, Otsuka Pharmaceutical, Patient, Immunoglobulin A, Disease, Breakthrough therapy, Biology, Kidney failure, Pharmaceutical industry

Otsuka Pharmaceutical Development & Commercialization, Inc., (OPDC); Otsuka Pharmaceutical, Co. Ltd. (Otsuka); and Visterra, Inc., an Otsuka group company announce that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for the investigational drug sibeprenlimab for the treatment of immunoglobulin A nephropathy (IgAN; Berger’s disease).

Key Points: 
  • Otsuka Pharmaceutical Development & Commercialization, Inc., (OPDC); Otsuka Pharmaceutical, Co. Ltd. (Otsuka); and Visterra, Inc., an Otsuka group company announce that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for the investigational drug sibeprenlimab for the treatment of immunoglobulin A nephropathy (IgAN; Berger’s disease).
  • This press release features multimedia.
  • Breakthrough therapy designation allows the agency to prioritize and focus resources on the most promising products for which no satisfactory treatment option exists.
  • “We are encouraged by the FDA’s decision to grant breakthrough therapy status for the sibeprenlimab program,” said John Kraus, M.D., Ph.D., executive vice president and chief medical officer at Otsuka.

Galderma Announces Regulatory Filing Acceptance for Nemolizumab in Prurigo Nodularis and Atopic Dermatitis in the U.S. and EU

Retrieved on: 
Wednesday, February 14, 2024
Lifestyle, Biotechnology, FDA, Health, Pharmaceutical, Cosmetics, Research, Retail, Consumer, Science, Clinical Trials, Nemolizumab, Quality of life, Atopic dermatitis, Food, Prurigo nodularis, File, Safety, Prurigo, European Medicines Agency, Sleep, Patient, Diagnosis, Breakthrough therapy, Itch, Priority review

This follows its designation as a Breakthrough Therapy for the treatment of pruritus associated with prurigo nodularis, originally granted in December 2019 and reconfirmed in March 2023.

Key Points: 
  • This follows its designation as a Breakthrough Therapy for the treatment of pruritus associated with prurigo nodularis, originally granted in December 2019 and reconfirmed in March 2023.
  • Priority Review is granted for medicines that would significantly improve the treatment, diagnosis or prevention of serious conditions.
  • The European Medicines Agency has also accepted Galderma’s Marketing Authorization Applications for nemolizumab in both prurigo nodularis and atopic dermatitis.
  • “The relentless itch experienced by many people living with prurigo nodularis and atopic dermatitis has a significant impact on their overall quality of life.

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.

Madrigal Pharmaceuticals Announces Publication of the Phase 3 MAESTRO-NASH Trial of Resmetirom in the New England Journal of Medicine

Retrieved on: 
Thursday, February 8, 2024
Principal, ALT, Liver failure, Fatty liver disease, Cirrhosis, Hepatotoxicity, Nausea, GGT, Death, Therapy, Biomarker, Patient, Breakthrough therapy, Safety, Incidence, FDA, NAFLD, Lipoprotein, Priority review, Diarrhea, NAS, Woman, University of Oxford, Disease, Drug development, Fibrosis, Biopsy, Risk, Week, Heart rate, PDUFA, CAP, NASH, Medicine, Liver cancer, Liver, Pharmaceutical industry

NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally.

Key Points: 
  • NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally.
  • Resmetirom received Breakthrough Therapy designation from the FDA and is under review to become the first medicine approved to treat patients with NASH with liver fibrosis.
  • Approximately 50% of patients treated with resmetirom 100 mg with biopsies at Week 52 showed either NASH resolution or fibrosis improvement.
  • More than 80% of patients with biopsies at Week 52 had either fibrosis reversal or no progression of fibrosis.

Bionomics Provides a Review of 2023 and of 2024 Plans

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Monday, January 22, 2024
MSD, Schizophrenia, BNC210, Deficit spending, Patient, Module (mathematics), PAMS, PTSD, Post-traumatic stress disorder, Sleep, Partnership, Merck & Co., GAD, NAM, Anxiety, Neuropsychiatry, SAD, Alzheimer's disease, Central nervous system, Australian Securities Exchange, FDA, DSM-IV codes, CIAS, Breakthrough therapy, Panic, Merck, Phase, Data analysis, Data, CNS, Social anxiety disorder, Pharmaceutical industry

ADELAIDE, Australia and CAMBRIDGE, Mass., Jan. 22, 2024 (GLOBE NEWSWIRE) -- Bionomics Limited (Nasdaq: BNOX) (Bionomics or Company), a clinical-stage biotechnology company developing novel, first-in-class, allosteric ion channel modulators to treat patients suffering from serious central nervous system (CNS) disorders with high unmet medical need, today provided a 2023 review and its outlook for 2024.

Key Points: 
  • ADELAIDE, Australia and CAMBRIDGE, Mass., Jan. 22, 2024 (GLOBE NEWSWIRE) -- Bionomics Limited (Nasdaq: BNOX) (Bionomics or Company), a clinical-stage biotechnology company developing novel, first-in-class, allosteric ion channel modulators to treat patients suffering from serious central nervous system (CNS) disorders with high unmet medical need, today provided a 2023 review and its outlook for 2024.
  • “Bionomics continued its evolution into a U.S.-focused, late-stage clinical company last year.
  • Voluntarily delisted from the Australian Stock Exchange in August 2023 and is currently solely listed on the NASDAQ Global Market.
  • Bionomics continues to plan the initiation of its first Phase 3 study of BNC210 in patients with SAD.

ENHERTU® Granted Priority Review in the U.S. for Patients with Metastatic HER2 Positive Solid Tumors

Retrieved on: 
Monday, January 29, 2024
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, Orbis, HER2, Immunohistochemistry, Neoplasm, Patient, IHC, MD, ADC, Daiichi Sankyo, Survival, Breakthrough therapy, Medicine, Cancer, Food, Safety, Priority review, Doctor of Philosophy, Breast, AstraZeneca, PDUFA, Pharmaceutical industry

ENHERTU is a specifically engineered HER2 directed antibody drug conjugate (ADC) being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca.

Key Points: 
  • ENHERTU is a specifically engineered HER2 directed antibody drug conjugate (ADC) being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca.
  • The Priority Review follows receipt of Breakthrough Therapy Designation granted by the FDA in August 2023 for ENHERTU in metastatic HER2 positive solid tumors.
  • Data from other supporting trials in patients with HER2 positive IHC 3+ tumors in the ENHERTU clinical development program, including DESTINY-Lung01 and DESTINY-CRC02 , also were included in the submission.
  • We will continue working closely with the FDA to bring this potential first tumor agnostic HER2 targeted medicine to patients as quickly as possible.”

HUTCHMED Announces NDA Acceptance in China for Sovleplenib for the Treatment of Primary Immune Thrombocytopenia with Priority Review Status

Retrieved on: 
Thursday, January 11, 2024
The Lancet, Patient, Bone marrow, Quality of life, NDA, Safety, ITP, Breakthrough therapy, New Drug Application, NMPA

HONG KONG and SHANGHAI and FLORHAM PARK, N.J., Jan. 10, 2024 (GLOBE NEWSWIRE) -- HUTCHMED (China) Limited (“HUTCHMED”) (Nasdaq/AIM:​HCM; HKEX:​13) today announces that the New Drug Application (“NDA”) for sovleplenib for the treatment of adult patients with primary immune thrombocytopenia (“ITP”) has been accepted for review and granted priority review by the China National Medical Products Administration (“NMPA”). Sovleplenib is a novel, selective, oral inhibitor targeting spleen tyrosine kinase (“Syk”), being developed for the treatment of hematological malignancies and immune diseases.

Key Points: 
  • HONG KONG and SHANGHAI and FLORHAM PARK, N.J., Jan. 10, 2024 (GLOBE NEWSWIRE) -- HUTCHMED (China) Limited (“ HUTCHMED ”) (Nasdaq/AIM:​HCM; HKEX:​13) today announces that the New Drug Application (“NDA”) for sovleplenib for the treatment of adult patients with primary immune thrombocytopenia (“ITP”) has been accepted for review and granted priority review by the China National Medical Products Administration (“NMPA”).
  • Sovleplenib is a novel, selective, oral inhibitor targeting spleen tyrosine kinase (“Syk”), being developed for the treatment of hematological malignancies and immune diseases.
  • “Our submission includes data from the successful Phase III ESLIM-01 trial in China which demonstrated a durable response rate of sovleplenib for patients.
  • There is a significant need for new therapies in adult primary ITP which can significantly impair the quality of life for patients.”