ODD

EQS-News: Heidelberg Pharma granted orphan drug designation by FDA for its proprietary ATAC candidate HDP-101

Retrieved on: 
Wednesday, April 10, 2024
ODD, Diagnosis, RRMM, BCMA, Patient, Multiple myeloma, Spacecraft, Amanitin, AACR, Antibody, Safety, HPHA, FDA, B-cell maturation antigen, ATAC, Food, Pharmaceutical industry

Ladenburg, Germany, 27 March 2024 – Heidelberg Pharma AG (FSE: HPHA), a clinical stage biotech company developing innovative Antibody Drug Conjugates (ADCs), is pleased to announce that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for the treatment of multiple myeloma to its lead candidate HDP-101.

Key Points: 
  • Ladenburg, Germany, 27 March 2024 – Heidelberg Pharma AG (FSE: HPHA), a clinical stage biotech company developing innovative Antibody Drug Conjugates (ADCs), is pleased to announce that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for the treatment of multiple myeloma to its lead candidate HDP-101.
  • Heidelberg Pharma is investigating the candidate in a clinical Phase I/IIa study for the treatment of relapsed/refractory multiple myeloma (RRMM).
  • Prof. Dr. Andreas Pahl, Chief Executive Officer at Heidelberg Pharma, commented: “We are delighted that our proprietary ATAC candidate HDP-101 has been granted Orphan Drug Designation by the FDA, providing further validation of its potential benefit as a therapeutic for patients with multiple myeloma.
  • The designation provides significant incentives to promote the development of the drug including tax credits for qualified clinical trials, prescription drug user-fee exemptions, and potential seven-year marketing exclusivity upon FDA approval.

Cardiol Therapeutics Announces Year-End 2023 Update on Operations

Retrieved on: 
Tuesday, April 2, 2024
Hypertrophy, Heart, CRDL, Patient, Mayo Clinic, PPAR, Agent handling, Cleveland Clinic, Fibrosis, European, Therapy, Myocarditis, Analysis, American Heart Association, Human, Massachusetts General Hospital, Pericarditis, MMT, Heart failure, American College, Safety, ODD, Cardiology, Clinical professor, Disease, Pericardium, Research, World Heart Federation, Management, University, Death, Pericardial effusion, Maverick, Reactive oxygen species, TSX, Priority review, FDA, Conversation, Financial statement, Inflammation, Fast Track, Calcium, Wound healing, API, Pharmaceutical industry

"Cardiol Therapeutics made important progress in 2023 and early 2024 as we pursued our primary objective of providing new therapeutic options to patients with poorly served heart diseases," said David Elsley, President and Chief Executive Officer of Cardiol Therapeutics.

Key Points: 
  • "Cardiol Therapeutics made important progress in 2023 and early 2024 as we pursued our primary objective of providing new therapeutic options to patients with poorly served heart diseases," said David Elsley, President and Chief Executive Officer of Cardiol Therapeutics.
  • In January 2023, Cardiol announced the first patient had been enrolled in the Company's Phase II open-label pilot study ("MAvERIC-Pilot") investigating the tolerance, safety, and efficacy of CardiolRx™ in patients with recurrent pericarditis.
  • In November 2023, Cardiol announced that it had exceeded 50% of the patient enrollment target for the MAvERIC-Pilot study.
  • In January 2024, Cardiol announced that ARCHER had exceeded 50% patient enrollment and was progressing ahead of the original study timeline.

NurExone Reports Fourth Quarter and Year Ended December 31, 2023 Financial Results and Provides Company Update

Retrieved on: 
Tuesday, April 2, 2024
ODD, Research, Growth, Drug discovery, Exercise, NRX, CFO, RNA, Laboratory, Contract research organization, Regenerative medicine, Investigational New Drug, Safety, Spinal cord injury, Intellectual property, Plasma protein binding, Small RNA, FDA, Food, Cryptocurrency

TORONTO and HAIFA, Israel, April 02, 2024 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX) (Germany: J90) (the “Company” or “NurExone”), a pioneering biopharmaceutical company, developing regenerative medicine therapies, today provided a business update and reported financial results for the fourth quarter and financial year ended December 31, 2023.

Key Points: 
  • TORONTO and HAIFA, Israel, April 02, 2024 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX) (Germany: J90) (the “Company” or “NurExone”), a pioneering biopharmaceutical company, developing regenerative medicine therapies, today provided a business update and reported financial results for the fourth quarter and financial year ended December 31, 2023.
  • For the fourth quarter of 2023, expenses were US$0.31 million, compared to US$0.39 million in the previous year.
  • For the fourth quarter of 2023, expenses were US$0.40 million, compared to US$0.46 million in the previous year.
  • For the fourth quarter of 2023, expenses were US$0.02 million, compared to US$0.17 million in the previous year.

Unicycive Therapeutics Announces Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, March 28, 2024
ODD, Research, Hyperphosphatemia, DGF, CRRT, ERA, Administration, Congress, Conference, OLC, CKD, Patient, AKI, National Kidney Foundation, Mitochondrion, NDA, Kidney transplantation, Insurance, Kidney disease, Critical care, Acute kidney injury, Clinical trial, Therapy, Drug development, Safety, Chronic kidney disease, Dialysis, LOS, Research and development, SAD, FDA, KATP, Food, Pharmaceutical industry

LOS ALTOS, Calif., March 28, 2024 (GLOBE NEWSWIRE) -- Unicycive Therapeutics, Inc. (Nasdaq: UNCY) (the “Company” or “Unicycive”), a clinical-stage biotechnology company developing therapies for patients with kidney disease, today announced its financial results for the year ended December 31, 2023, and provided a business update.

Key Points: 
  • LOS ALTOS, Calif., March 28, 2024 (GLOBE NEWSWIRE) -- Unicycive Therapeutics, Inc. (Nasdaq: UNCY) (the “Company” or “Unicycive”), a clinical-stage biotechnology company developing therapies for patients with kidney disease, today announced its financial results for the year ended December 31, 2023, and provided a business update.
  • Completed a private placement with new and existing healthcare institutional investors that generated $50 million in gross proceeds to Unicycive.
  • Cash Position: As of December 31, 2023, cash and cash equivalents totaled $9.7 million.
  • Subsequent to year end, in March 2024, Unicycive completed a private placement of preferred stock which generated $50 million in gross proceeds.

Marker Therapeutics Reports Year-End 2023 Corporate and Financial Results

Retrieved on: 
Monday, March 25, 2024
ODD, Research, Food and Drug Administration, U.S. FDA, Drug discovery, AML, International, Patient, Committee, SBIR, Female, Acute myeloid leukemia, Sale, INN, Marker, EMA, OTS, NIH, IND, Myelodysplastic syndrome, European Medicines Agency, Counsel, Therapy, MRD, Bone marrow, USAN, MDS, APOLLO, FDA, DLBCL, Press release, CD19, Pharmaceutical industry

Marker also executed a comprehensive non-dilutive agreement with Cell Ready which included a sale of select cell manufacturing assets from Marker for approximately $19 million in cash.

Key Points: 
  • Marker also executed a comprehensive non-dilutive agreement with Cell Ready which included a sale of select cell manufacturing assets from Marker for approximately $19 million in cash.
  • Granted ODD from the Committee for Orphan Medicinal Products of the EMA for the treatment of patients with AML in 2023.
  • On June 26, 2023, Marker completed a non-dilutive transaction with Cell Ready, under which Cell Ready purchased certain cell manufacturing assets from Marker for approximately $19 million in cash.
  • Cash Position and Guidance: At December 31, 2023, Marker had cash and cash equivalents of $15.1 million.

Cadrenal Therapeutics Receives FDA Orphan Drug Designation for Tecarfarin for Prevention of Thromboembolism and Thrombosis in Patients with LVADs, RVADs, Biventricular Assist Devices, and Total Artificial Hearts

Retrieved on: 
Tuesday, April 9, 2024
ODD, Warfarin, Thrombosis, VADS, Risk, Vitamin K antagonist, New Drug Application, Patient, Apixaban, Stroke, Anticoagulant, Splenic infarction, Death, Therapy, Bleeding, LVAD, FDA, VKA, Pharmaceutical industry

PONTE VEDRA, Fla., April 9, 2024 /PRNewswire/ -- Cadrenal Therapeutics, Inc. (Nasdaq: CVKD), a biopharmaceutical company developing tecarfarin, a late-stage novel oral and reversible anticoagulant (blood thinner) designed to prevent heart attacks, strokes, and deaths due to blood clots in patients with rare cardiovascular conditions, announced today that the United States Food and Drug Administration (FDA) has granted tecarfarin Orphan Drug Designation (ODD) for the prevention of thromboembolism and thrombosis in patients with an implanted mechanical circulatory support device (left ventricular assist device (LVAD), right ventricular assist device (RVAD), collectively known as ventricular assist devices (VADs), biventricular assist device, and total artificial heart).

Key Points: 
  • "This second orphan drug designation highlights the expanded need for tecarfarin where existing anticoagulation therapies are inadequate," said Quang Pham, Founder, Chairman and Chief Executive Officer of Cadrenal Therapeutics.
  • "We are dedicated to advancing tecarfarin through clinical development options as swiftly as possible."
  • Since its adoption in 1983, the Orphan Drug Act has helped countless individuals living with these conditions gain access to life-enhancing and life-saving therapies.
  • The designation is made to promote safe and efficacious products for the treatment of rare conditions.

Cabaletta Bio Announces FDA Granted Orphan Drug Designation to CABA-201 for Treatment of Systemic Sclerosis

Retrieved on: 
Wednesday, March 20, 2024
ODD, Diagnosis, Face, Survival, Disease, Patient, Scleroderma, Clinical trial, Skin, Systemic scleroderma, Marketing, FDA, Food, Pharmaceutical industry

PHILADELPHIA, March 20, 2024 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, for the treatment of systemic sclerosis (SSc).

Key Points: 
  • PHILADELPHIA, March 20, 2024 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, for the treatment of systemic sclerosis (SSc).
  • CABA-201 is in development as a potential treatment for autoimmune diseases driven by B cells.
  • “Based on the role of B cells and the recently published academic clinical data with CD19-CAR T therapy in systemic sclerosis, we believe CABA-201 may transform the treatment for systemic sclerosis.
  • This designation qualifies Cabaletta for certain incentives, which may include partial tax credit for clinical trial expenditures, waived user fees and potential eligibility for seven years of marketing exclusivity.

Pharming Group reports fourth quarter and full year 2023 financial results

Retrieved on: 
Thursday, March 14, 2024
Patient, Health care, Epidemiology, Executive Committee, Laboratory, Microsoft Access, Lymphocyte, Clinical trial, European Commission, APDS, MHLW, AEG, Welfare, Ageing, Committee, EMA, Trial of the century, PID, CTLA4, Safety, Pharmacokinetics, Ministry, CHMP, ODD, U.S. FDA, Prevalence, PTEN, Disease, HAE, Immunodeficiency, VUS, Literature, PIK3R1, PMDA, FDA, DSM-IV codes, Diagnosis, Civil service commission, IRP, Haploinsufficiency, Genetic testing, MAA, Autoimmunity, World Games, Iberian languages, Marketing, MHRA, Euronext Amsterdam, PIK3CD, Physician, Pharmaceutical industry

The U.S. market contributed 97% of 2023 revenues, while the EU and Rest of World contributed 3%.

Key Points: 
  • The U.S. market contributed 97% of 2023 revenues, while the EU and Rest of World contributed 3%.
  • Revenues increased to US$7.9 million in the fourth quarter of 2023, driven by the continued increase in patients on paid therapy, and revenues were US$18.2 million for 2023.
  • Pharming made continued progress in the fourth quarter of 2023 on leniolisib regulatory filings for APDS patients 12 years of age and older in key global markets.
  • Pharming filed regulatory submissions in Canada and Australia in the third quarter of 2023, and Israel in the second quarter.

Faron’s Financial Statement Release January 1 to December 31, 2023

Retrieved on: 
Wednesday, March 13, 2024
Head, Q&A, Survival, Immunotherapy, Patient, Medicine, Standard of care, CMML, Macrophage, Faron Pharmaceuticals, Neoplasm, EET, Rheumatology, PD-1, TME, PR, ASH, AML, Cancer, Johnson & Johnson, Safety, Azacitidine, MDS, BLA, Decitabine, Food, ODD, Nasdaq First North, PD, Tumor microenvironment, Manuscript, CRS, SICAV, MD, Interferon beta-1a, Haematopoietic system, GMT, FDA, AIM, Neurotoxicity, Infection, EUR, Sanofi, SOC, Metabolism, European Hematology Association, EDT, MBA, IPF, Facility, Treasury, Society, Cytokine release syndrome, CPA, Pharmaceutical industry

As at March 13, 2024, the Company is in compliance with all IPF financial covenants as agreed with the waiver letter.

Key Points: 
  • As at March 13, 2024, the Company is in compliance with all IPF financial covenants as agreed with the waiver letter.
  • Loss for the period for the financial year ended December 31, 2023, was EUR 30,9 million (2022: EUR 28,7 million).
  • In June 2023, Faron conducted a placement of 2,601,510 newly issued treasury shares to investors to raise EUR 6,6 million gross.
  • In October 2023, the Company successfully raised EUR 7,1 million gross through the issuance of 2,491,998 ordinary shares to investors.

Belite Bio Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Tuesday, March 12, 2024
ODD, Retina, Stargardt disease, Research, Conference, Vitamin A, Exercise, RPD, Eye, Patient, RBP4, Tissue, ARVO, ATM, Safety, Paratriathlon, Therapy, Macular degeneration, Blood, FDA, DRAGON, Food, Pharmaceutical industry

ET

Key Points: 
  • ET
    SAN DIEGO, March 11, 2024 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite Bio” or the “Company”), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced its audited financial results for full-year 2023 and the unaudited and unreviewed financial results for the quarter ended December 31, 2023, and provided a business update.
  • The decrease resulted primarily from higher R&D expenses in the fourth quarter of 2022 as the DRAGON trial reached certain milestones in such quarter.
  • Belite Bio will host a webcast on Tuesday, March 12, 2023, at 4:30 p.m. Eastern Time to discuss the Company’s financial results and provide a business update.
  • A replay will be available for approximately 90 days following the event at the Company’s Investor Relations website at https://investors.belitebio.com/presentations-events/events .