US regulators have approved CSL Behring’s Haegarda, the first C1 esterase inhibitor for subcutaneous administration to prevent hereditary (HAE) attacks in adolescent and adults.
Biopharmaceutical companies are regaining interest in metabolic dysfunction-associated steatohepatitis (MASH) innovator drug development. MASH innovator drugs witnessed over $2.5 billion increase in the total value of partnership deals from 2020 to 2024 year-to-date (YTD), with more than $2 billion forged in Q1 2024 alone, reveals GlobalData. MASH, previously known as nonalcoholic steatohepatitis (NASH), is a disease characteriSed by liver inflammation and damage caused by the accumulation of fat. Madrigal Pharmaceuticals’ Rezdiffra (resmetirom), a small molecule THRB agonist, was the first drug approved by the FDA for MASH in March 2024. Alison Labya, Business Fundamentals Analyst at GlobalData, comments, “Interest in MASH has returned in light of the FDA approval of Madrigal’s Rezdiffra, as well as the success of GLP-1 obesity drugs and their potential efficacy in MASH, as demonstrated by Eli Lilly’s Zepbound (tirzepatide; also known as Mounjaro for type 2 diabetes) in its Phase II SYNERGY-NASH trial readout.” However, ...
Small cell lung cancer (SCLC), a rapidly progressing neuroendocrine malignancy, exhibits low survival rates. Despite its molecular and clinical heterogeneity, SCLC is presently treated as a single entity, without the use of predictive biomarkers, which leads to poor patient outcomes. Recent research has proposed dividing SCLC into four subtypes—labeled “A”, “N”, “P”, and “I”—each characterized by distinct molecular signatures and treatment vulnerabilities. Initially, this classification relied on gene expression (RNA-seq) data. Further studies indicated that the same categorization could be recapitulated through the use of a reduced-representation bisulfite sequencing (RRBS) methylation profile. Although this classification system effectively predicts treatment responses, including to immunotherapy, in retrospective analyses, both RNAseq and RRBS techniques are too labor-intensive and slow for quick treatment decisions in an aggressive malignancy. Now, a pilot study published in the journal Cancer Cell has demonstrated the feasibility of a simple PCR assay to accurately differentiate between SCLC SCLC subtypes. ...
Amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease) and frontotemporal dementia (FTD) are two progressively neurodegenerative conditions that damage essential nerve cells. ALS compromises nerve cells in the brain and spinal cord, leading to movement impairment, while FTD damages brain areas responsible for personality, behavior, and language. Studies indicate that in ALS or FTD patients, TAR DNA-binding protein 43 (TDP-43) malfunctions. This disruption prevents the correct splicing of ribonucleic acid (RNA), essential for generating proteins necessary for nerve growth and function. Instead, RNA strands become riddled with erroneous code sequences, known as “cryptic exons,” which are linked to an elevated risk of developing ALS and FTD. The timing of this anomaly in the disease progression of ALS and FTD was previously unknown. Now, researchers from Johns Hopkins Medicine (Baltimore, MD, USA) have developed a method for identifying a specific protein, hepatoma-derived growth factor-like 2 [HDGFL2], linked to TDP-43 ...
Clasp Therapeutics’ novel T cell engagers could offer advantages over others in this class of cancer immunotherapies. The startup is based on the research of Johns Hopkins University scientist Bert Vogelstein. By FRANK VINLUANCancer immunotherapy comes in several forms, and one area of growing research interest is a type of drug called a T cell engager. These drugs bind to a cancer cell and a T cell simultaneously, bringing both of them together to spark a therapeutic effect. Robert Ross, CEO of Clasp Therapeutics, likens T cell engagers to the chaperone at a middle school dance who grabs a boy and a girl in each hand, getting them to dance together. But in cancer drugs, just as in school dances, sometimes the pairing just isn’t right. The wrong pairing in cancer happens when a drug that’s targeted to a tumor also hits healthy cells, causing toxic effects. Clasp is developing ...
The FDA has approved Madrigal Pharmaceuticals drug Rezdiffra as a treatment for the fatty liver disease NASH (also called MASH). It’s the first treatment for the chronic condition, which has been growing in prevalence. By FRANK VINLUAN A chronic metabolic disease in which fat buildup leads to worsening liver function that can ultimately require an organ transplant now has its first FDA-approved therapy, a once-daily pill developed by Madrigal Pharmaceuticals. The FDA decision Thursday is for adults with noncirrhotic non-alcoholic steatohepatitis, or NASH. The approval specifically covers those who have reached the point of moderate-to-advanced liver scarring, which is also called fibrosis. The Conshohocken, Pennsylvania-based company’s drug, known in development as resmetirom, will be marketed under the brand name Rezdiffra. “We’ve had patients waiting and waiting for something that could impact underlying fibrosis in the setting of NASH, and this truly represents a milestone achievement that frankly I, and many ...
Davy James BeiGene’s Brukinsa is a small molecule Bruton’s tyrosine kinase inhibitor indicated as a monotherapy and in combination with other treatments for various B-cell malignancies. The FDA has granted accelerated approval to BeiGene’s Brukinsa (zanubrutinib) in combination with Roche’s Gazyva (obinutuzumab) for patients with relapsed or refractory follicular lymphoma (FL) following two or more lines of systemic therapy.1 Brukinsa is a small molecule Bruton’s tyrosine kinase (BTK) inhibitor indicated as a monotherapy and in combination with other treatments for various B-cell malignancies, such as chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma; Waldenström’s macroglobulinemia; patients with mantle cell lymphoma (MCL) previously treated with at least one prior therapy; and patients with relapsed or refractory marginal zone lymphoma previously treated with at least one anti-CD20-based regimen.1 Gazyva is a CD20-directed cytolytic antibody indicated in combination with chlorambucil for the treatment of patients with previously untreated CLL; in combination with chemotherapy ...
BeiGene’s Brukinsa (zanubrutinib) has been granted accelerated approval by the US Food and Drug Administration (FDA) to treat relapsed or refractory follicular lymphoma. The BTK inhibitor has specifically been authorised for use alongside Roche’s anti-CD20 monoclonal antibody Gazyva (obinutuzumab) in adult patients who have received at least two prior lines of systemic therapy. The FDA’s decision was supported by positive results from the mid-stage ROSEWOOD study, which demonstrated an overall response rate of 69% for the Brukinsa combination arm compared to 46% for Gazyva alone. An 18-month duration of response of 69% for the Brukinsa combination was also observed, BeiGene said, adding that safety results were consistent with previous studies of both medicines. Mehrdad Mobasher, chief medical officer, haematology at BeiGene, said: “This accelerated approval of Brukinsa represents an important advancement, offering the first and only BTK inhibitor treatment for follicular lymphoma patients in the US who have either not ...
The US Department of Health and Human Services (HHS), as part of the Centers for Medicare and Medicaid Services (CMS), has announced that all ten pharmaceutical manufacturers taking part in the Medicare drug price negotiations have submitted counteroffers. The announcement comes after the HHS sent initial offers on 1 February in an ongoing effort to lower drug prices in the US as part of the Inflation Reduction Act (IRA) and negotiations will continue over the next several months. First selected last August, the ten medicines subject to the first round of Medicare pricing negotiations included therapies for diabetes, heart failure, immunological disorders and cancer, as well as anticoagulants. The medicines selected included Bristol Myers Squibb/Pfizer’s Eliquis (apixaban), Novartis’ Entresto (valsartan/sacubitril), AstraZeneca’s Farxiga (dapagliflozin), AbbVie/Johnson & Johnson’s Imbruvica (ibrutinib), Eli Lilly/Boehringer Ingelheim’s Jardiance (empagliflozin), and Johnson & Johnson’s Stelara (ustekinumab) and Xarelto (rivaroxaban). According to the HHS, collectively, the drugs were ...
Australia-based global biopharmaceutical company Telix Pharmaceuticals has agreed to acquire US-based QSAM Biosciences for $123.1m.The deal comes after the companies signed a conditional non-binding term sheet for the prospective acquisition.Telix will also acquire QSAM’s lead investigational drug, Samarium-153-DOTMP (153Sm-DOTMP), a new kit-based bone-seeking targeted radiopharmaceutical candidate. The acquisition terms include an upfront payment of $33.1m (A$50.8m), payable as 4,369,914 ordinary shares of Telix.The deal also comprises contingent value rights that could see additional payments of $90m on meeting clinical and commercial milestones. These payments may be made in cash and/or shares.QSAM focuses on the development of therapeutic radiopharmaceuticals for primary and metastatic bone cancer. Its 153Sm-DOTMP for bone cancer has applications in pain management and therapy for bone metastases and osteosarcoma, including for paediatric patients.It complements Telix’s focus on oncology therapies and has shown promising safety, efficacy and commercial potential in early trials. The drug also has an improved safety ...
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