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Pharmaceutical
Swiss pharma giant Novartis today announced an upgrade to its mid-term guidance, in advance of its Meet Novartis Management event for investors and analysts in London. 21 November 2024
Biotechnology
US biotech major Amgen has announced that Dr Howard Chang will join the company as senior vice president of research, effective December 16, 2024. 21 November 2024
Biotechnology
Australia’s CSL Limited plans to shut down its Californian R&D facility focused on cell and gene therapies by January 2025, signaling a shift away from ex vivo lentiviral-based technology. 19 November 2024
Biotechnology
Cellular immunotherapy specialist Fate Therapeutics soared as much as 73%, and closed up 12.45 at $2.27 yesterday, after it released early-stage data on its FT819 in lupus. 19 November 2024
Pharmaceutical
US antiviral giant Gilead Sciences on Friday unveiled two-and-a-half-year interim analysis from the ongoing Phase III ASSURE study of its Livdelzi (seladelpar). 16 November 2024
Pharmaceutical
French drugmaker Ipsen announced late-breaking data for Iqirvo (elafibranor 80mg tablets) from an interim analysis of the ongoing open-label extension of the Phase III ELATIVE study at the American Association for the Study of Liver Disease (AASLD) congress. 16 November 2024
Biotechnology
Oxford, UK-based rare respiratory diseases focused AlveoGene today revealed it has been granted a Rare Pediatric Disease designation (RPDD) by the US Food and Drug Administration (FDA) for AVG-002, its novel, inhaled gene therapy for lethal neonatal surfactant protein B (SP-B) deficiency. 15 November 2024
Biotechnology
Shares of US genetic meds company MeiraGTx Holdings were up 7% at $7.12 in early trading after it announced important developments along with its third-quarter 2024 financials, reporting a $0.54 per share loss. 13 November 2024
Biotechnology
Dutch firm Argenx and China’s Zai Lab have received an approval from the National Medical Products Administration (NMPA) for Vyvgart Hytrulo (efgartigimod alfa). 13 November 2024
Pharmaceutical
AstraZeneca and Merck & Co announced that their jointly developed drug Koselugo (selumetinib) has shown positive outcomes in the KOMET Phase III trial. 12 November 2024
Biotechnology
Geron Corporation, a biopharma company focused on telomerase inhibition therapies, has announced a $375 million financing agreement aimed at supporting the commercial rollout of Rytelo (imetelstat). 11 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program, according to Sarah Wicks and James Valentine at US law firm Hyman, Phelps & McNamara. 11 November 2024
Biotechnology
Sarepta Therapeutics has announced it will cease development of SRP-5051, an experimental drug for Duchenne muscular dystrophy, citing safety issues, feedback from the Food and Drug Administration, and the shifting Duchenne treatment landscape. 11 November 2024
Pharmaceutical
Research results featured heavily in the news last week, first with Viking Therapeutics releasing promising early-stage data for its investigational medicine VK2738 at the ObesityWeek meeting. US biotech Beam Therapeutics released early-stage data on its sickle cell disease candidate BEAM-101. Also of note, Arcus Biosciences and Gilead Sciences announced promising Phase III results for their TIGIT inhibitor domvanalimab in combination with zimberelimab. Denmark’s Novo Nordisk released financial results that attracted interest. 10 November 2024
Pharmaceutical
Italian pharma major Recordati saw its shares dip 3.75% to 50.10 euros this morning, despite posting a set of strong financial results. 8 November 2024
Biotechnology
Asklepios BioPharmaceutical (AskBio) today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation and orphan-drug designation from the US Food and Drug Administration (FDA) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). 7 November 2024
Biotechnology
The global market for Duchenne muscular dystrophy (DMD) therapeutics is projected to grow by $4.4 billion between 2024 and 2028, fueled by artificial intelligence and strong demand for new treatments. 7 November 2024
Pharmaceutical
US drugmaker Sarepta Therapeutic saw its shares gain 2.4% to $127.97 as it reported financial results for the third quarter 2024. 7 November 2024
Pharmaceutical
USA-based Acadia Pharmaceuticals has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million upon the closing of the transaction. 6 November 2024
Biotechnology
UK health technology assessor the National Institute for Health and Care Excellence (NICE) issued final guidance recommending reimbursement of Ayvakyt (avapritinib) from USA-based Blueprint Medicines. 6 November 2024
Biotechnology
USA-based LogicBio Therapeutics has announced a strategic collaboration and option agreement with China-based CANbridge Pharmaceuticals leveraging LogicBio’s gene editing and gene delivery platforms. 27 April 2021
Pharmaceutical
Swiss pharma giant Novartis today released first-quarter 2021 financial results, which failed to meet analysts’ expectations. 27 April 2021
Biotechnology
Having a treatment that, the data show, can save the lives of babies and children facing an otherwise grim outlook is an excellent starting point for ensuring swift reimbursement for a product, but by no means is it a guarantee. 26 April 2021
Biotechnology
Notable news last week included Australia’s Kazia Therapeutics entering a licensing deal with Germany’s Evotec for worldwide rights to cancer candidate EVT801. French vaccine specialist Valneva has started Phase III evaluation of its COVID-19 candidate VLA200 in the UK. US biotech bluebird bio gave an update on its genetic disease portfolio last Tuesday and said it was withdrawing its beta thalassemia drug Zynteglo from the German market due to failed price negotiations. GlaxoSmithKline was granted US Food and Drug Administration approval for its checkpoint blocker Jemperli. Also, on Friday the FDA lifted its pause on the use of Johnson & Johnson’s COVID-19 Vaccine Janssen. 25 April 2021
Biotechnology
The European Medicines Agency’s Committee for Medicinal Products for Human US (CHMP) recommended eight medicines for approval at its April 2021 meeting, five of which were novel drugs. 23 April 2021
Biotechnology
Horizon Therapeutics has announced new data on Uplinza (inebilizumab-cdon) were presented at the American Academy of Neurology’s 73rd Annual Meeting being held virtually April 17-22, 2021 (AAN 2021). 23 April 2021
Biotechnology
US biotech major Biogen today reported first quarter 2021 financial results which, though well below 2020 comparisons, beat analysts’ expectations. 22 April 2021
Biotechnology
Guernsey and Switzerland based Pureos Bioventure has announces the final closing of its first fund BB Pureos Bioventures LP with a committed capital of $205 million. 22 April 2021
Biotechnology
US RNAi therapeutics company Alnylam Pharmaceuticals has announced full positive results from the HELIOS-A Phase III study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, which met its primary and both secondary endpoints at nine months in patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy. 20 April 2021
Biotechnology
US biotech major Biogen today announced new data from the Spinraza (nusinersen) clinical development program aimed at optimizing outcomes for people with spinal muscular atrophy (SMA) and advancing understanding of the disease. 19 April 2021
Biotechnology
Swiss pharma giant Novartis has announced new post hoc data from the Phase III ASCLEPIOS trials of Kesimpta (ofatumumab). 16 April 2021
Biotechnology
Roche today announced new Ocrevus (ocrelizumab) analyses supporting its significant benefit on disease progression in early-stage relapsing-remitting multiple sclerosis (RRMS) and primary progressive MS (PPMS) as well as demonstrating high persistence and strong adherence to twice-yearly (six-monthly) dosing. 16 April 2021
Biotechnology
The National Institute for Care and Health Excellence’s (NICE) has recommended Ultomiris (ravulizumab) for use in the National Health Service (NHS) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). 15 April 2021
Biotechnology
PTC Therapeutics has announced new data showing continued improvements in motor milestones in infants with type 1 spinal muscular atrophy (SMA) after two years of treatment with Evrysdi (risdiplam). 15 April 2021
Pharmaceutical
Russian drugmaker Biocad says it has completed one of the stages of development of its new drug, which is designed for the treatment of spinal muscular atrophy (SMA), reports The Pharma Letter’s local correspondent. 13 April 2021
Biotechnology
The Institute for Clinical and Economic Review (ICER) has announced that it is using observational real-world evidence (RWE) to update its 2018 assessment of three therapies for long-term prophylaxis against hereditary angioedema (HAE) attacks. 12 April 2021
Biotechnology
Novartis today announced that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), alongside the recent European Commission, has granted a marketing authorization for the use of Kesimpta (ofatumumab) for the treatment of adult patients with relapsing forms of multiple sclerosis (RMS) with active disease, defined by clinical or imaging features. 8 April 2021
Biotechnology
Sino-American biotech firm BeiGene today announced approval from the China National Medical Products Administration (NMPA) for BeiGene to begin manufacturing commercial supply of its approved anti-PD-1 antibody, tislelizumab, at its state-of-the-art biologics facility in Guangzhou, China. 8 April 2021
Pharmaceutical
US biotech Cogent Biosciences has named John Robinson the company’s chief scientific officer (CSO). 7 April 2021
Pharmaceutical
Paul Hudson, chief executive of French pharma major Sanofi today outlined several key projects that the company will implement to increase the impact of its corporate social responsibility (CSR) strategy. 7 April 2021
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