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Biotechnology
Shares of US gene and cell therapy company Abeona Therapeutics hit a high of $4.80 yesterday but closed 13% lower at $3.87, after it announced positive top-line data from its pivotal Phase III VIITAL study assessing the safety and efficacy of EB-101 for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB), as well as a $35 million financing. 4 November 2022
Generics
Israeli generics company Teva Pharmaceutical Industries was trading more than 2% lower late on Thursday, following the firm’s presentation of its third quarter 2022 financial results. 3 November 2022
Biotechnology
New top-line data released this morning by US pharma major Bristol Myers Squibb suggest that its already-marketed drug Reblozyl (luspatercept-aamt) should be able to address a larger proportion of myelodysplastic syndromes (MDS) patients. 31 October 2022
Biotechnology
Sino-American biotech BeiGene claims significant progress in its efforts to unlock global opportunities for its Brukinsa (zanubrutinib) with recent regulatory approvals in six Latin American countries. 31 October 2022
Pharmaceutical
This month, we discuss Rett syndrome and pharmacological advancements in the treatment of this rare condition. 29 October 2022
Biotechnology
Alnylam has announced that it will not presently be taking its drug Amvuttra (vutrisiran) into a Phase III trial for Stargardt disease. 28 October 2022
Pharmaceutical
Ipsen has reported a 16% rise in its revenue for the third quarter of 2022. 27 October 2022
Pharmaceutical
Japan’s largest drugmaker Takeda Pharmaceutical’s shares edged 1.8% higher to $12.97 in early US trading, after it reported financial results for the first half of its fiscal year ended September 2022. 27 October 2022
Biotechnology
Anglo-Swedish pharma major AstraZeneca has presented new results on Ultomiris (ravulizumab) in neuromyelitis optica spectrum disorder (NMOSD). 27 October 2022
Pharmaceutical
Rare diseases specialist Swedish Orphan Biovitrum saw its shares fall more than 3% to 204.40 kronor this morning after releasing third-quarter 2022 financial result. 27 October 2022
Biotechnology
British pharma major AstraZeneca has reported mixed top-line results from a trial of its monoclonal antibody Fasenra (benralizumab). 25 October 2022
Biotechnology
The market for spinal muscular atrophy (SMA), a progressive neuromuscular disease that can be fatal, has attracted many of biopharma’s biggest names. 25 October 2022
Biotechnology
US biotech major Biogen today reported third quarter 2022 financial results that were better than investors expected, but the stock fell 2.3% to $267.84 in early trading. 25 October 2022
Biotechnology
Japanese pharma major Astellas Pharma has announced a strategic investment to support the advancement of Texas, USA-based Taysha Gene Therapies’ adeno-associated virus (AAV) gene therapy development programs for the treatment of Rett syndrome and giant axonal neuropathy (GAN). 25 October 2022
Biotechnology
US biotech Mirum Pharmaceuticals edged up 2.6% to $20.02 yesterday, as it released positive top-line data from its Phase III MARCH-PFIC trial evaluating Livmarli (maralixibat) in rare pediatric disease progressive familial intrahepatic cholestasis (PFIC). 25 October 2022
Biotechnology
Novartis saw its shares edge up 1% to 77.33 francs this morning, after the Swiss pharma giant released top-line clinical trial results showing the its hemoglobinuria treatment candidate iptacopan showed superiority over Alexion and AstraZeneca’s blockbuster drugs Soliris (eculizumab) and Ultomiris (ravulizumab), the current standard of care treatments for the condition. 24 October 2022
Biotechnology
New Jersey, USA-based rare disease drug developer PTC Therapeutics saw its shares close down 11% yesterday and fall a further almost 12% to $43.00 pre-market today, after it revealed it has paused US enrollment in a Phase II study of PTC518 in Huntington's disease. 19 October 2022
Biotechnology
Cambridge, USA-based clinical-stage biotech Synlogic has announced positive top-line data from its Phase II Synpheny-1 study in phenylketonuria (PKU). 19 October 2022
Pharmaceutical
US neurological diseases specialist Acorda Therapeutics saw its shares go stratospheric yesterday, after it revealed it has been awarded $16.5 million by an arbitral panel following a dispute with Ireland-incorporated drugmaker Alkermes over licensing royalties for the multiple sclerosis drug Ampyra (dalfampridine). 18 October 2022
Biotechnology
Biotech major Gilead Sciences has entered an exclusive option and collaboration agreement with fellow USA-based Macrogenics to develop MGD024, an investigational, bispecific antibody that binds CD123 and CD3 using MacroGenics’ DART platform, and two additional bispecific research programs. 17 October 2022
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
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