Aplastic anemia

New US product, ORMALVI™ (dichlorphenamide) tablets, launches for Primary Periodic Paralysis (PPP) treatment

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Mercoledì, Maggio 8, 2024
FDA, General Health, Pharmaceutical, Health, Fetus, Cardiomyopathy, Torrent, Acidosis, Respiratory acidosis, Full, Diclofenamide, Risk, Pregnancy, Cognitive disorder, Milk, Aspirin, Metabolic acidosis, Dysgeusia, Hyperkalemia, Aplastic anemia, Partnership, PPP, Agranulocytosis, Anorexia nervosa, Human, Carbonic anhydrase inhibitor, SVP, Tablet, Incidence, Patient, Nursing, Lethargy, Serum, Hyperkalemic periodic paralysis, Diagnosis, Myopathy, Hypokalemic periodic paralysis, Paralysis, Paresthesia, Cycle, Stevens–Johnson syndrome, Hypokalemia, Quality of life, Argyreia nervosa, Sulfonamide, Coma, Pharmaceutical industry

We want to provide them with the support that they require to effectively manage their PPP.

Key Points: 
  • We want to provide them with the support that they require to effectively manage their PPP.
  • ORMALVI™ is an oral carbonic anhydrase inhibitor indicated for the treatment of primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variants.
  • Simply Stated: Research Updates in Periodic Paralysis - Quest | Muscular Dystrophy Association [online] Available at: https://mdaquest.org/simply-stated-research-updates-in-periodic-paralysis Accessed 23 Nov. 2023.
  • The impact of permanent muscle weakness on quality of life in periodic paralysis: a survey of 66 patients.

Amneal Launches PEMRYDI RTU®, the First Ready-to-Use Version of Important Oncology Injectable Medicine

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Martedì, Aprile 16, 2024
Biotechnology, Hospitals, Health, Pharmaceutical, Oncology, Radiation, CMS, Pharmacy, Refrigeration, Hospital, Pembrolizumab, Cisplatin, Aplastic anemia, Mesothelioma, Disease, Restrictive lung disease, Pemetrexed, Patient, Medicare, Kidney, ALK, Medication, Amneal Pharmaceuticals, RTU, Pharmaceutical industry

Amneal Pharmaceuticals, Inc. (Nasdaq: AMRX) (“Amneal” or the “Company”) today announced the launch of PEMRYDI RTU®, the first and only ready-to-use presentation of pemetrexed for injection.

Key Points: 
  • Amneal Pharmaceuticals, Inc. (Nasdaq: AMRX) (“Amneal” or the “Company”) today announced the launch of PEMRYDI RTU®, the first and only ready-to-use presentation of pemetrexed for injection.
  • This product does not require reconstitution, dilution, or refrigeration, which is different than other versions of pemetrexed for injection.
  • “The launch of PEMRYDI RTU highlights Amneal’s deep commitment to oncology as we invest to develop injectable products that are important to healthcare providers and patients,” said Sean McGowan, Vice President, Biosimilars and Branded Oncology.
  • PEMRYDI RTU® injectable is available in two vial sizes: 100mg/10mL and 500 mg/50mL.

Astex Pharmaceuticals Announce Key Data Presentations at the American Association For Cancer Research (AACR) 2024 Annual Meeting

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Mercoledì, Aprile 3, 2024
Toxicity, Aplastic anemia, AACR, Astex Pharmaceuticals, MDM2, Half-life

ASTX295 is an oral, potent inhibitor of the p53-MDM2 protein-protein interaction that was discovered by Astex using its proprietary structure-based drug design approach.

Key Points: 
  • ASTX295 is an oral, potent inhibitor of the p53-MDM2 protein-protein interaction that was discovered by Astex using its proprietary structure-based drug design approach.
  • The compound was specifically designed to overcome the on-target toxicity seen in the first generation MDM2 antagonist compounds which have shown dose-limiting haematological toxicities in the clinic.
  • In contrast, ASTX295 is a potent MDM2 antagonist with a clean CYP/hERG profile and a shorter human half-life allowing for pulsatile pathway modulation while avoiding myelosuppression.
  • ASTX295 therefore has bone-marrow sparing characteristics which permit a differentiated safety profile to be presented at AACR.

Aprea Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Provides a Business Update

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Martedì, Marzo 26, 2024
Ovarian cancer, Cyclin, IND, Therapy, Anemia, Research, University, Cell cycle, MD Anderson Cancer Center, PLK1, DDR, Investigational New Drug, Safety, Pharmacokinetics, Synthetic lethality, WEE1, U.S. FDA, Mutation, Acceleration, Part, HERG, ATR, KOL, Acquisition, Cancer, AACR, Research and development, Poster, FDA, Cyclin E, Toxicity, Aplastic anemia, Administration, Conference, APRE, PLK3, PLK2, Division, Data, Cardiotoxicity, Pharmaceutical industry

DOYLESTOWN, Pa., March 26, 2024 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc. (Nasdaq: APRE) (“Aprea”, or the “Company”), a clinical-stage biopharmaceutical company focused on precision oncology through synthetic lethality, today reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a business update.

Key Points: 
  • “Aprea had a very productive 2023 with significant progress across our diversified pipeline of synthetic lethality-based cancer therapeutics.
  • An update from Part 1 of the trial was featured in a poster presentation at the AACR-NCI-EORTC International Conference in October 2023.
  • Select Financial Results for the Fourth Quarter ended December 31, 2023
    As of December 31, 2023, Aprea reported cash and cash equivalents of $21.6 million.
  • Research and Development (R&D) expenses were $2.0 million for the quarter ended December 31, 2023, compared to $0.5 million for the fourth quarter of 2022.

Syros Receives Fast Track Designation from the FDA for Tamibarotene for the Treatment of Newly Diagnosed Unfit AML with RARA gene overexpression

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Martedì, Aprile 9, 2024
Biotechnology, FDA, Health, Pharmaceutical, Clinical Trials, Toxicity, Aplastic anemia, Syros, Tamibarotene, AML, Patient, Fast Track, Venetoclax, Acute myeloid leukemia, Azacitidine, Prognosis, MDS, Bone marrow, RARA, Pharmaceutical industry

Tamibarotene, an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist, is currently being evaluated in combination with venetoclax and azacitidine for the treatment of newly diagnosed AML patients with RARA gene overexpression.

Key Points: 
  • Tamibarotene, an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist, is currently being evaluated in combination with venetoclax and azacitidine for the treatment of newly diagnosed AML patients with RARA gene overexpression.
  • “We are pleased to receive Fast Track designation for tamibarotene for the treatment of AML.
  • Syros is evaluating tamibarotene in combination with venetoclax and azacitidine in newly diagnosed, unfit AML patients with RARA overexpression in the ongoing SELECT-AML-1 Phase 2 trial.
  • In January 2023, the FDA granted Fast Track Designation to tamibarotene for the treatment of HR-MDS patients with RARA overexpression.

Syros Reports Fourth Quarter and Full Year 2023 Financial Results and Provides a Corporate Update

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Mercoledì, Marzo 27, 2024
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Research, Aplastic anemia, Pfizer, AML, Webcast, Patient, Venetoclax, NDA, G&A, Azacitidine, ID, MDS, Bone marrow, RARA, Pharmaceutical industry

Gross proceeds to Syros were approximately $45.0 million, before underwriting discounts and commissions and offering expenses payable by Syros.

Key Points: 
  • Gross proceeds to Syros were approximately $45.0 million, before underwriting discounts and commissions and offering expenses payable by Syros.
  • Cash, cash equivalents and marketable securities as of December 31, 2023, were $139.5 million, as compared with $202.3 million on December 31, 2022.
  • ET to discuss the fourth quarter and full-year 2023 financial results and provide a corporate update.
  • A webcast of the call will also be available on the Investors & Media section of the Syros website at www.syros.com .

MEI Pharma Reports Update from Clinical Study Evaluating Oral CDK9 Inhibitor Voruciclib in Combination with Venetoclax in Patients with Relapsed and Refractory Acute Myeloid Leukemia

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Martedì, Marzo 26, 2024
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Toxicity, Aplastic anemia, Salvage therapy, CLL, AML, CRi, Patient, Venetoclax, Biomarker, Diarrhea, BCL2, University, Acute myeloid leukemia, ELN, Safety, Pharmacokinetics, CDK9, Phase 1, Associate professor, Pharmaceutical industry

The study is currently enrolling a 12-patient expansion cohort evaluating voruciclib administered at 300 mg daily for two weeks in a four-week cycle in combination with venetoclax.

Key Points: 
  • The study is currently enrolling a 12-patient expansion cohort evaluating voruciclib administered at 300 mg daily for two weeks in a four-week cycle in combination with venetoclax.
  • A total of 29 patients with R/R AML, median age 67 years (range 34-89), enrolled in the dose escalation stage of the study evaluating voruciclib in combination with venetoclax.
  • The primary objectives of the study are to determine the safety and biologic effective dose of voruciclib monotherapy or voruciclib in combination with venetoclax.
  • Secondary objectives of the study include assessing the preliminary efficacy, pharmacokinetics, pharmacodynamics, and biomarkers of voruciclib monotherapy or voruciclib in combination with venetoclax.

Cotchett, Pitre & McCarthy Files Lawsuit on Behalf of Residents of Maywood, CA, for Exposure to Cancer Causing Ethylene Oxide

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Lunedì, Marzo 18, 2024
Professional Services, Legal, Environment, Bone marrow, Public health, Inhalation, National Cancer Institute, Breast, Ethylene oxide, Cancer, Leukemia, Ethylene, Aplastic anemia, Complaint, Atmosphere, Breast cancer, Partner, Superior court, Family, Stomach, Spice, Los Angeles Times, Lymphoma, Stomach cancer, Oil

Cotchett, Pitre & McCarthy LLP filed a lawsuit on behalf of many longtime residents of Maywood, CA, next to Vernon, an industrial area, for their decades long exposure to cancer caused by carcinogenic Ethylene Oxide being released into the air.

Key Points: 
  • Cotchett, Pitre & McCarthy LLP filed a lawsuit on behalf of many longtime residents of Maywood, CA, next to Vernon, an industrial area, for their decades long exposure to cancer caused by carcinogenic Ethylene Oxide being released into the air.
  • Ethylene Oxide is used to sterilize medical equipment and spices but, during the process, some of the Ethylene Oxide escapes the facility into the surrounding air.
  • Ethylene Oxide is odorless and colorless, so nearby Maywood residents never knew it was in the air surrounding their homes.
  • According to the National Cancer Institute, and other protective health agencies around the world, Ethylene Oxide is associated with breast cancer, stomach cancer, lymphoma, and leukemia.

A Historic Downtown Oakland Hotel Re-Emerges as 130 Stylish Residences

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Martedì, Marzo 19, 2024
Mojave Desert, Jack London, Legislation, White Wolf Publishing, Partnership, Nike, Public policy, History, Policy, Entitlement, Jack London Square, Aplastic anemia, Housing, Olympic Games, Warrior, System, Palm, Interior design

OAKLAND, Calif., March 19, 2024 /PRNewswire/ -- Converting existing buildings into multifamily housing can be a significant step in alleviating California's housing crisis. In one prominent example, Riaz Capital's newly completed multifamily project, ArtHaus Jack London, has been refashioned from the former Z-Hotel, an Oakland icon. This stylish 130 residence project makes entry-level housing more affordable for city residents, while preserving a slice of history in Oakland's Jack London Square.

Key Points: 
  • In one prominent example, Riaz Capital's newly completed multifamily project, ArtHaus Jack London, has been refashioned from the former Z-Hotel, an Oakland icon.
  • This stylish 130 residence project makes entry-level housing more affordable for city residents, while preserving a slice of history in Oakland's Jack London Square.
  • It takes inspiration from the Saguaro Hotel in Palm Springs, whose color palette is derived from the flora of the Mojave Desert.
  • This law enabled us to increase the ArtHaus Jack London unit count from 102 hotel rooms to 130 entry-level apartments.

Injectable Hematology Growth Factors Report 2024 - Evolution in Therapeutic Care - ResearchAndMarkets.com

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Martedì, Febbraio 13, 2024
Pharmaceutical, Health, Medical Devices, Hormone, Economics, Safety, Hematology, Antibody, Anemia, Thrombocytopenia, Kidney disease, Aplastic anemia, Patient, Neutropenia, Vaccine

The "Injectable Hematology Growth Factors Report" has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Injectable Hematology Growth Factors Report" has been added to ResearchAndMarkets.com's offering.
  • This report is a comprehensive evaluation and analysis of the technology, products, and participants providing the driving force behind this evolving segment of the healthcare sector.
  • Provides detailed analysis of injectable hematology growth factors delivery and device strategies, and product development factors.
  • Analyzes therapeutic demand drivers and evaluates injectable drug products in a number of key therapeutic segments.