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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
Pharmaceutical
Last week’s notable news includes US biotech Nkarta pleasing investors on Monday, when it released new clinical data on its NKX101 and NKX019 candidates for hematological malignancies. Also, Mundipharma and Cidara Therapeutics last week released positive new data on their antifungal rezafungin, with hopes of a regulatory filing mid-2022. There was negative news for Molecular Partners last Tuesday, when partner Amgen said it was returning rights to immuno-oncology candidate MP0310. On the regulatory front, Bristol Myers Squibb was granted Food and Drug Administration (FDA) approval for its cardiomyopathy drug mavacamten, now given the trade name Camzyos. Novo Nordisk presented first-quarter financials showing that the Danish company is making sales progress with its newest weight management drug Wegovy, as Eli Lilly touted its rival tirzepatide as strong potential challenger. 1 May 2022
Biotechnology
Fibrodysplasia ossificans progressiva (FOP) is a rare connective tissue disease characterized by heterotopic ossification, where bone growth occurs in areas where bone typically should not be present.1 30 April 2022
Pharmaceutical
Soaring AstraZeneca sales beat estimates in 2022’s first quarter, but the company’s full-year guidance remains unchanged amid an uncertain Covid-19-product sales outlook and Chinese market challenges. 29 April 2022
Biotechnology
People with Duchenne muscular dystrophy (DMD) have been given renewed hope that an investigational gene therapy under development by Pfizer may yield results. 29 April 2022
Pharmaceutical
Swiss pharma giant Roche today announced new three-year data from the FIREFISH study, including one-year data from the open label extension, reinforcing the long-term efficacy and safety of Evrysdi (risdiplam) in infants with symptomatic Type 1 spinal muscular atrophy (SMA). 29 April 2022
Biotechnology
RNAi therapeutics company Alnylam Pharmaceuticals today reported its consolidated financial results for the first quarter ended March 31, 2022. 28 April 2022
Biotechnology
Now the first and only long-acting C5 complement inhibitor in gMG, Ultomiris (ravulizumab-cwvz), has gained an additional approval in the USA. 28 April 2022
Biotechnology
The European Medicines Agency (EMA) has granted access to the Priority Medicines (PRIME) scheme for BCX9250, a novel, oral activin receptor-like kinase-2 (ALK-2) inhibitor discovered and developed by BioCryst Pharmaceuticals for the treatment of fibrodysplasia ossificans progressiva (FOP). 27 April 2022
Biotechnology
Boston, USA-based biotech start-up Jnana Therapeutics today announced the appointment of Doug Pagán, as both chief financial and chief operating officer. 26 April 2022
Pharmaceutical
The Russian government will allocate additional 23 billion roubles ($305 million) for the procurements of high-priced imported drugs as part of 14 high-cost nosologies (VZN) state program, reports The Pharma Letter’s local correspondent. 25 April 2022
Pharmaceutical
The European Medicines Agency’s human medicines committee (CHMP) recommended four medicines for approval in the European Union (EU) at its April 2022 meeting. 23 April 2022
Biotechnology
Rare diseases powerhouse Alexion was last year acquired for $39 billion by the Anglo-Swedish pharma major AstraZeneca, becoming part of one of big pharma’s most successful companies in recent years. 22 April 2022
Pharmaceutical
Japanese drugmaker Astellas Pharma has announced that it will book an impairment loss in both the fourth quarter of the fiscal year ended on March 31, and in the subsequent quarter. 22 April 2022
Pharmaceutical
Results from a pooled analysis of Rezurock (belumosudil) for the treatment of chronic graft-versus-host disease (cGVHD) show certain organ clinical responses correlated with clinically-meaningful changes in patient-reported outcomes (PROs). 21 April 2022
Pharmaceutical
Florida, USA-based biotech Hesperos has published early-stage research into a novel drug development technology that has caught the attention of French pharma major Sanofi. 21 April 2022
Biotechnology
Global Market Insights (GMI) has predicted 5% compound annual growth rate (CAGR) in the global hemophilia market between 2022 and 2028. 20 April 2022
Pharmaceutical
Boston, USA-based Sionna Therapeutics, a life sciences company founded in 2019 to develop highly effective and differentiated treatments for cystic fibrosis (CF), has announced the official launch of the company and the closing of a $111 million Series B financing. 20 April 2022
Biotechnology
In a filing with the US Securities and Exchange Commission (SEC), Boston-based rare disease specialist Imara has disclosed savage job cuts as part of an extensive restructuring. 19 April 2022
Biotechnology
US biotech TG Therapeutics saw its shares plunge almost 32% to $6.03, after it announced the voluntary withdrawal of the pending Biologics License Application (BLA)/supplemental New Drug Application (sNDA) for the combination of ublituximab and Ukoniq (umbralisib) - combination referred to as U2 - for the treatment of adult patients with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL). 19 April 2022
Biotechnology
USA-based Be Biopharma (“Be Bio”), a pioneer in the discovery and development of Engineered B Cell Medicines (BeCM) founded in October 2020, has announced the closing of a $130 million financing, bringing the total investment in the company to over $180 million. 14 April 2022
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