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Biotechnology
US ultra-rare diseases focussed biotech firm Ultragenyx Pharmaceutical and Japan’s Kyowa Hakko Kirin have announced that the Phase III study of Crysvita (burosumab) met its primary endpoint 17 May 2018
Pharmaceutical
Eiger BioPharmaceuticals has expanded its licensing agreement with Merck & Co to include rights to develop the investigational farnesyltransferase inhibitor lonafarnib for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria), a rare and fatal genetic condition characterized by accelerated aging in children. 16 May 2018
Pharmaceutical
Following the finalization of his firm’s $62 billion purchase of Dubkin’s Shire, Takeda Pharamceutical chief executive Christophe Weber says the deal will start to pay dividends almost immediately. 14 May 2018
Pharmaceutical
What looks likely to be one of the biggest pharma industry M&A deals for several year, and certainly the largest acquisition by a Japanese company ever, seems set to move forward, with the agreement between Ireland-based Shire and Japan’s Takeda Pharmaceutical. Also on the M&A front, Eli Lilly agreed to acquire immuno-oncology drug developer Arma Biosciences and its lead candidate pegilodecakin. Among research news attracting attention was mixed results for Johnson & Johnson’s depression drug candidate esketamine, and elsewhere there was transformative news from Valeant Pharmaceuticals International, including a name change to Bausch Health Companies. 13 May 2018
Biotechnology
Loyal investors in Faron Pharmaceuticals hoping that there might be a straight forward explanation for the failure of the Phase III INTEREST trial of traumakine might be disappointed in a statement issued by the company on Friday. 11 May 2018
Biotechnology
Safety concerns could not stop the majority of a US Food and Drug Administration (FDA) committee voting in favor of approving volanesorsen for familial chylomicronemia syndrome (FCS), a rare lipid disorder with no therapeutic options. 11 May 2018
Pharmaceutical
Rare and orphan disease specialist Amryt Pharma has announced an agreement with the Noverion Therapeutics subsidiary Aegerion Pharmaceuticals to expand its exclusive license agreement for Lojuxta (lomitapide). 10 May 2018
Pharmaceutical
Cardiovascular and rare disease specialist Recordati has published first quarter results showing the Italian firm took in 366.5 million euros ($434 million), up 7.2% from the same period last year, in line with analysts’ expectations. 9 May 2018
Pharmaceutical
Takeda and Shire have reached agreement on the Japanese pharma major’s takeover of the rare disease drugmaker. 8 May 2018
Biotechnology
Alexion Pharmaceuticals has revealed new Phase III study results for ALXN1210, an investigational long-acting C5 inhibitor the firm is trialling against a serious ultra-rare blood disorder called paroxysmal nocturnal hemoglobinuria (PNH). 8 May 2018
Biotechnology
CEVEC Pharmaceuticals and Australian biotechnology leader CSL Limited have entered into an exclusive licensing agreement for the development, manufacture and commercialization of recombinant C1 esterase Inhibitor (C1-INH) proteins for hereditary angioedema (HAE) and other potential indications using CEVEC's proprietary CAP Go technology. 8 May 2018
Biotechnology
The US Food and Drug Administration has granted a Rare Pediatric Disease designation to the company’s gene therapy candidate OTL-200, for the treatment of patients with metachromatic leukodystrophy (MLD). 3 May 2018
Pharmaceutical
Ironwood Pharmaceuticals’ shares climbed 5%, to about $19 apiece, in midday trading on Tuesday, after it announced that its board of directors has authorized an intent to separate into two independent, publicly traded companies (Ironwood and “R&D Co”). 3 May 2018
Biotechnology
California biotech firm Rigel Pharmaceuticals today announced that the American Journal of Hematology has published positive results from the Fostamatinib in Thrombocytopenia (FIT) Phase III clinical program of Tavalisse (fostamatinib disodium hexahydrate) for the treatment of adults with chronic immune thrombocytopenia (ITP). 30 April 2018
Pharmaceutical
Mergers and acquisitions continued at the forefront of news again last week, with Ireland-based Shire reaching preliminary agreement to recommend Takeda Pharmaceutical’s fifth revised bid of some £46 billion, though observers warn that the deal could be scuppered because of the huge level of debt that will ensue. Also attracting attention was Fresenius’ declaration that it wants to pull out of the 2017 deal to acquire Akorn. In regulatory news, there was a mixed Food and Drug Administration panel vote for Eli Lilly and Incyte’s rheumatoid arthritis drug baricitinib, and on research, the implications of Prothena’s failed trial with rare disease drug NEOD001. 29 April 2018
Biotechnology
Shares in US drugmaker Alexion Pharmaceuticals closed 15% up on Thursday at $121.42 as markets digested an encouraging set of first quarter results and some strong clinical data. 27 April 2018
Pharmaceutical
Despite being upbeat about future growth, the shares of French pharma major Sanofi fell 1.84% to 65.09 euros by mid-morning, after the company released first-quarter 2018 financial results that disappointed. 27 April 2018
Pharmaceutical
In a quarter that was dominated by speculation over the protracted courtship dance of Takeda Pharmaceutical, London-listed Shire managed to deliver strong revenues of $3.8 billion, a 5% increase on the same period last year. 26 April 2018
Pharmaceutical
Nordic drugmaker Swedish Orphan Biovitrum saw its shares rise 7.05% to 170.15 Swedish kronor this morning, after it announced strong first-quarter 2018 results. 26 April 2018
Pharmaceutical
After a four-week courtship and four rebuttals, Shire has finally succumbed to Takeda Pharmaceutical’s fifth offer to acquire the Ireland-based rare diseases company. 25 April 2018
Biotechnology
Japanese drugmaker Chugai, which is majority owned by Roche, On Friday revealed that Zenyaku Kogyo has obtained regulatory approval from the Ministry of Health, Labor and Welfare (MHLW), for an additional dosage and administration of an anti-CD20 monoclonal antibody Rituxan (rituximab) intravenous injection 100mg and 500mg, which is co-marketed by both companies, for chronic idiopathic thrombocytopenic purpura (ITP) in children. 23 November 2024
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
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