Applied Therapeutics saw its stock price climb by over two-thirds after an encouraging update on the US Food and Drug Administration review of govorestat. 19 September 2024
TG Therapeutics yesterday shared new five-year data from the ULTIMATE I & II Phase III trials evaluating Briumvi (ublituximab-xiiy) in patients with relapsing forms of multiple sclerosis (RMS). 19 September 2024
Canada’s NanoVation Therapeutics, a privately-held platform company developing innovative technologies to overcome the barriers of nucleic acid delivery, has announced a multi-year partnership with Danish drugmaker Novo Nordisk. 18 September 2024
AstraZeneca’s Fasenra (benralizumab) has been approved in the USA for the treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA, the UK pharma major announced today. 18 September 2024
US biotech Capricor Therapeutics has entered into a binding term sheet with Japanese drugmaker Nippon Shinyaku for the commercialization and distribution in Europe of Capricor’s lead asset, deramiocel. 17 September 2024
Dystonia is a neurological movement disorder characterized by involuntary muscle contractions that cause repetitive movements, abnormal postures, or twisting. 17 September 2024
The US Food and Drug Administration (FDA) has approved Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq) for the treatment of relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS), from Swiss pharma giant Roche. 16 September 2024
The Gastrointestinal Drugs Advisory Committee (GIDAC) of the US Food and Drug Administration (FDA) met on Friday to discuss Intercept Pharmaceuticals’ supplemental New Drug Application (sNDA) for Ocaliva (obeticholic acid, OCA). 16 September 2024
Boehringer Ingelheim today announced that the FIBRONEER-IPF trial met its primary endpoint, which was the absolute change from baseline in Forced Vital Capacity [mL] (FVC) at week 52 versus placebo. FVC is a measure of lung function. 16 September 2024
Oligonucleotide therapeutics are rapidly emerging as a promising class of drugs capable of addressing a variety of genetic and complex diseases by targeting RNA sequences.1 16 September 2024
Thursday was a wretched day for Fulcrum Therapeutics, a US biopharma company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases. 13 September 2024
California, USA-based biotech BridgeBio Pharma saw its shares dip 4% to $29.60 yesterday on disappointing clinical results and the company’s decision to drop an early-stage candidate. 11 September 2024
Santhera Pharmaceuticals has signed an exclusive distribution agreement with GENESIS Pharma for Agamree (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in 20 markets in Central and Eastern Europe. 10 September 2024
Cartesian Therapeutics today announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to Descartes-08 for the treatment of juvenile dermatomyositis (JDM). 9 September 2024
Last week’s research news included French pharma major Sanofi releasing mixed Phase III results for it tolebrutinib in progressive multiple sclerosis. Also of note, US vaccine developer Vaxcyte announced strong early-stage trial results for its VAX-31 in pneumococcal disease. On the regulatory front, US drugmaker Travere Therapeutics gained full approval from the US Food and Drug Administration (FDA) for its kidney disease drug Filspari. US cancer drug developer IN8bio announced a prioritization of its R&D pipeline to focus on INB-100 in acute myeloid leukemia, along with a workforce cutback. 8 September 2024
The Institute for Clinical and Economic Review (ICER) has posted its revised Evidence Report assessing the comparative clinical effectiveness of three medicines for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). 6 September 2024
San Diego, USA-based Travere Therapeutics saw its shares close up more than 8% at $10.75 yesterday on positive news form the US regulator. 6 September 2024
The European Medicines Agency’s (EMA) human medicines committee (CHMP) recommended two novel medicines for approval at its June 2023 meeting. 23 June 2023
The US Food and Drug Administration (FDA) yesterday approved Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy for the treatment of pediatric patients aged four through five years of age with Duchenne muscular dystrophy (DMD). 23 June 2023
The US Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for lovotibeglogene autotemcel (lovo-cel) for priority review, said the drug’s developer, bluebird bio. 22 June 2023
Promising clinical results from the 12-week portion of the PIVOT-HD Phase II study of PTC518 in Huntington's disease (HD) lifted US pharma group PTC Therapeutics. 22 June 2023
The appointment of a new chief executive at immune-mediated disease specialist Veralox Therapeutics coincides with a fresh investment of $24 million from new and existing backers. 20 June 2023
CDKL5 deficiency disorder (CDD), which has an incidence of 1 in 40,000 to 60,000 newborns, is a developmental disorder caused by either the presence of a non-functional cyclin-dependent kinase-like 5 (CDKL5) protein known as serine-threonine kinase (STK9), or its complete absence.1,2 20 June 2023
Oxford University, UK, biotech spinout Evox Therapeutics has purchased USA-base Codiak Biosciences’ engEx-AAV technology platform, including all intellectual property rights therein, as well as secured exclusive access to certain associated engineering and manufacturing rights. 20 June 2023
Shares of Santhera Pharmaceuticals were up almost 8% at 0.97 francs in early trading, after the Swiss rare diseases company revealed a lucrative licensing deal with Catalyst Pharmaceuticals for its vamorolone in the North America. 20 June 2023
Texas, USA-based drug developer Reata Pharmaceuticals says that the US Food and Drug Administration (FDA) accepted for filing the Prior Approval Supplement (PAS) to update the drug substance specification for Skyclarys (omaveloxolone). 16 June 2023
Japanese drugmaker Chugai Pharmaceutical said today it has filed a regulatory application with the Ministry of Health, Labor and Welfare (MHLW) for the anti-C5 antibody crovalimab for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). 14 June 2023
Israel-based NeuroSense Therapeutics announced in a Securities and Exchange Commission that it has entered into a collaborative evaluation agreement with a unit of US biotech major Biogen. 14 June 2023
Just months after France’s Ipsen bought liver disease specialist Albireo (Nasdaq: ALBO), the company’s main product Bylvay (odevixibat) has won a new US nod. 14 June 2023
Shares of US gene and cell therapy company Abeona Therapeutics closed down 6.5% at $4.01 on Friday, when it announced receipt of feedback from the US Food and Drug Administration (FDA) regarding EB-101. 12 June 2023
As usual, the American Society of Clinical Oncology (ASCO) meeting held last week attracted a great deal of attention, among which were presentations related to AstraZeneca’ Tagrisso in lung cancer and Blueprint Medicines new VELA trial of BLU-222 in breast and lung cancer. Outside of ASCO, Mereo BioPharma and partner Ultragenyx released new data showing their setrusumab induced rapid bone production in patients with osteogenesis imperfecta. US biotech FibroGen released disappointing Phase III result for its Duchenne muscular dystrophy (DMD) candidate pamrevlumab. Also of note last week, US pharma giant Merck & Co filed a lawsuit challenging the USA’s Inflation Reduction Act. 11 June 2023
San Diego, USA-base next-gen gene therapy developer Kate Therapeutics (KateTx) has emerged from stealth mode with a $51 million Series A financing. 9 June 2023
The US Food and Drug Administration (FDA) has accepted the Biologics License Applications (BLAs) for the investigational treatment exagamglogene autotemcel (exa-cel) for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) that has been submitted by US biotech Vertex Pharmaceutical. 9 June 2023
Californian biopharma FibroGen suffered a setback in part of its development of pamrevlumab, sending its shares 11% lower in Wednesday’s trading. 8 June 2023