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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
Italian drugmaker Recordati has appointed Milan Zdravkovic as its executive vice president of Research & Development (R&D). 17 October 2023
Biotechnology
Swiss pharma giant Roche subsidiary Genentech has announced new data from the Phase II FENopta study showing that investigational, oral fenebrutinib is brain penetrant and reduces brain lesions in people with relapsing multiple sclerosis (RMS), with a consistent safety profile to other fenebrutinib trials. 16 October 2023
Pharmaceutical
At its October monthly meeting, the European Medicines Agency’s (EMA) human medicines committee (CHMP) recommended five novel medicines for approval. 14 October 2023
Biotechnology
Israel-based cancer and rare diseases drug developer BioLineRx today announced the closing of an exclusive license agreement with Guangzhou Gloria Biosciences (GloriaBio) and an associated investor for the development of motixafortide across all indications in Asia, with the news sending BioLineRx’ shares up 6.7% to $1.60. 12 October 2023
Biotechnology
Genentech announced new clinical and real-world data on its anti-CD20 therapy Ocrevus (ocrelizumab) at the 9th Joint ECTRIMS-ACTRIMS Meeting (European and Americas Committees for Treatment and Research in Multiple Sclerosis) that it said “continue to deepen our understanding of multiple sclerosis (MS).” 12 October 2023
Pharmaceutical
Swedish company Egetis Therapeutics has raised around $16 million in a private share placement led by US healthcare investor Frazier Life Sciences. 11 October 2023
Pharmaceutical
German life sciences and pharma firm Merck KGaA today announced the presentation of new analyses from the Mavenclad (cladribine) MAGNIFY-MS study. 10 October 2023
Pharmaceutical
The situation with the availability of modern, original drugs continues to deteriorate in Russia, which is mainly due to ever-tightening sanctions against the country and its growing isolation in the international arena, reports The Pharma Letter’s local correspondent. 9 October 2023
Biotechnology
US biotech giant Amgen has at last completed its acquisition of Ireland-headquartered Horizon Therapeutics for $116.50 per share in cash, representing a transaction equity value of around $27.8 billion, that was announced in December last year. 7 October 2023
Pharmaceutical
Californian biotech Neurocrine Biosciences has announced positive top-line data from the Phase III CAHTALYST study of crinecerfont. 6 October 2023
Biotechnology
Dutch gene therapy company uniQure saw its shares fall 11.6% to $6.73 yesterday, after it announced a strategic reorganization. 6 October 2023
Biotechnology
Shares of US and UK-based gene therapy company Orchard Therapeutics almost doubled to $16.03 in pre-market activity today, on the news that it has received a takeover offer from mid-size Japanese drugmaker Kyowa Kirin. 5 October 2023
Pharmaceutical
Rare diseases specialist Swedish Orphan Biovitrum, also known as Sobi, today announced that Lydia Abad-Franch has been appointed as senior vice president, head of research, development and medical affairs (RDMA), and chief medical office, a role which she has held on an interim basis since June. 5 October 2023
Pharmaceutical
Shares of Melbourne, Australia-based Dimerix shot up 154% to A$0.16 on the news of a licensing deal with privately-held UK-based drugmaker Advanz Pharma. 5 October 2023
Biotechnology
Positive results have been presented from the primary analysis of the ongoing RAINBOWFISH study assessing the efficacy and safety of Evrysdi (risdiplam) in babies with pre-symptomatic spinal muscular atrophy (SMA), aged from birth to six weeks. 4 October 2023
Biotechnology
The US Food and Drug Administration (FDA) has approved Rivfloza (nedosiran) injection, a once-monthly subcutaneous ribonucleic acid interference (RNAi) therapy, from Danish diabetes care giant Novo Nordisk. 3 October 2023
Pharmaceutical
US pharma company PTC Therapeutics has announced a further strategic prioritization and associated reduction in its workforce. 2 October 2023
Biotechnology
Among important research news last week, US clinical-stage biotech Immunovant leapt 80% after it released early data on its IMVT-1402 autoimmune disease candidate. Also, Ionis Pharmaceuticals announced positive Phase III results for its investigational rare disease drug olezarsen. Troubled US biotech Intercept Pharmaceuticals has accepted an around $800 million takeover bid from Italian privately-owned drugmaker Alfasigma. Last week saw pharma major AbbVie pull out of another licensing collaboration, this time with Caribou Biosciences on two programs on allogenic CAR-T products. And, on the regulatory front, the US Food and Drug Administration (FDA) finally approved Amicus Therapeutics’ Pompe disease combination of Pombiliti plus Opfolda. 1 October 2023
Biotechnology
After several delays, the US Food and Drug Administration (FDA) has finally approved Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) for the treatment of late onset Pompe disease/LOPD. 29 September 2023
Biotechnology
Information from research group GlobalData suggests there is a pressing need for innovation and collaboration in China to address an unmet medical need in Fabry disease. 29 September 2023
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