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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
US biopharma Insmed yesterday announced progress across its early-stage research programs, at the company's investor and analyst event, The Future of Rare at Insmed: Functional Genes, AI-Enhanced Proteins, Glowing Algae, and More. 10 May 2023
Biotechnology
A new Fabry disease therapy, PRX-102 (pegunigalsidase alfa), has been given marketing authorization by the European Medicines Agency. 9 May 2023
Biotechnology
Last week, the US Food and Drug Administration (FDA) approved Qalsody (tofersen) to treat amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). 3 May 2023
Pharmaceutical
A third of the value of Travere Therapeutics stock has been wiped out following the failure of the Phase III DUPLEX study of sparsentan. 3 May 2023
Biotechnology
Total investment in biopharma M&A in the first quarter of 2023 was over four times the equivalent value for first quarter 2022, including the first megadeal since December 2020 with Pfizer paying $43 billion to acquire Seagen. 3 May 2023
Biotechnology
Maze Therapeutics, a privately-held Californian company translating genetic insights into new precision medicines, has announced the signing of an exclusive worldwide license agreement with French pharma major Sanofi. 2 May 2023
Pharmaceutical
Big pharma financials dominated the news last week, first among which were from Swiss pharma giant Novartis, whose first-quarter 2023 figures beat Wall Street expectations. Also, GSK released results, along with a promise to strengthen its pipeline. On the research front, Ionis and partner AstraZeneca reported positive Phase III data on their eplontersen in the treatment of polyneuropathy. Meantime, Nektar revealed that Eli Lilly had returned rights to the Lupus treatment rezpegaldesleukin (rezpeg). 30 April 2023
Pharmaceutical
The European medicines Agency’s (EMA) human medicines committee (CHMP) recommended six novel medicines for approval at its April 2023 meeting. 28 April 2023
Pharmaceutical
UK pharma major AstraZeneca this morning reported first-quarter 2023 financial results, with total revenues of $10.879 billion, a decline of 4% (1% increase at constant exchange rates) impacted by lower sales of COVID-19 medicines. 27 April 2023
Pharmaceutical
Shares of US drugmaker Vertex Pharmaceuticals closed down 2.6% at $323.30 yesterday, despite the firm announcing that the US Food and Drug Administration (FDA) has approved the expanded use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor). 27 April 2023
Biotechnology
With another set of strong data under its belt, Swiss pharma giant Novartis is now looking to filing regulatory applications for its investigational oral monotherapy iptacopan. 26 April 2023
Biotechnology
The US Food and Drug Administration (FDA) yesterday approved Qalsody (tofersen) to treat patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). 26 April 2023
Pharmaceutical
Swiss pharma giant Roche says that, in the first three months of 2023, group sales declined by 3% (-7% in Swiss franc term) to 15.3 billion francs ($17.2 billion). 26 April 2023
Biotechnology
US biotech company Biogen beat analysts’ expectations with its first-quarter 2023 financial results, exceeding forecasts with both sales and earnings figures. 25 April 2023
Biotechnology
Idorsia’s shares were down more than 9% at 8.76 Swiss francs by early afternoon, as the Switzerland-based biotech announced its financial results for the first quarter of 2023. 25 April 2023
Biotechnology
Detailed results have been presented from the NEURO-TTRansform Phase III trial in hereditary transthyretin-mediated amyloid polyneuropathy testing AstraZeneca and Ionis’ eplontersen. 25 April 2023
Biotechnology
Shares of New York, USA-based rare disease firm Applied Therapeutics closed up more than 16% yesterday at $1.10, despite releasing disappointing results from the ACTION-Galactosemia Kids study of govorestat (AT-007), a novel, oral, small molecule, central nervous system (CNS) penetrant aldose reductase inhibitor (ARI). 25 April 2023
Pharmaceutical
The European Medicines Agency (EMA) has opened a public consultation on a reflection paper that discusses key concepts for single-arm clinical trials submitted as pivotal evidence in support of marketing authorization applications. 21 April 2023
Biotechnology
For EC Awareness month, The Pharma Letter examines the treatment landscape of esophageal cancer (EC), a rare but deadly malignancy that is the sixth most common cause of cancer-related deaths in the world.1 18 April 2023
Biotechnology
Significant research news last week included US firm Horizon Therapeutics reporting positive Phase IV data on its thyroid eye disease medication Tepezza, while mRNA specialist Moderna released an ambivalent analysis of its influenza vaccine program. Also, French biotech Genfit announced important developments in its R&D pipeline, notably on elafibranor in cholangitis and non-alcoholic steatohepatitis (NASH), and on ezurpimtrostat. On the regulatory front, Germany’s Merck KGaA had a setback with its multiple sclerosis candidate evobrutinib last week, when the US Food and Drug Administration (FDA) slapped a partial clinical hold on its Phase II trial. 16 April 2023
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