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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
French drugmaker Sanofi has revealed the results from the Phase III LUNA 3 trial of rilzabrutinib, an oral bruton tyrosine kinase (BTK) inhibitor, in persistent or chronic immune thrombocytopenia (ITP). 6 November 2024
Biotechnology
In 2023, more than 50% (28 of 55) of the novel drugs reviewed by the US Food and Drug Administration (FDA) received orphan drug designation for the prevention, diagnosis, or treatment of a rare disease.1 20 February 2024
Biotechnology
M&A continued to feature in the news last week, as Gilead Sciences announced plans to acquire fellow USA-based CymaBay Therapeutics and its liver disease candidate seladelpar for around $4.3 billion. On a negative development, AN2 Therapeutics saw its market capitalization crash 74% after it revealed it is pausing enrollment of a Phase III study of epetraborole in lung cancer, Also of note, Brii Biosciences announced another research collaboration with VBI Vaccines in lung cancer. Sarepta Therapeutics announced that the US Food and Drug Administration (FDA) has accepted its application for an extended indication for its Duchenne muscular dystrophy drug Elevidys. 18 February 2024
Biotechnology
The Food and Drug Administration (FDA) has accepted and filed the efficacy supplement to the Biologics License Application (BLA) for Elevidys (delandistrogene moxeparvovec-rokl) submitted by US precision genetic medicines firm Sarepta Therapeutics. 16 February 2024
Biotechnology
The UK's National Institute for Health and Care Excellence (NICE) has issued a Final Draft Guidance (FDG) recommending GSK’s Omjjara (momelotinib) for myelofibrosis-related splenomegaly or symptoms in adults with moderate to severe anaemia who have not had a JAK-inhibitor or had Jakafi (ruxolitinib). 15 February 2024
Biotechnology
Positive Phase II clinical trial demonstrated frexalimab significantly slowed disease activity in people with relapsing multiple sclerosis (MS). 15 February 2024
Biotechnology
French pharma major Sanofi is angry that its interleukin (IL)-13 and IL-4 blocking antibody Dupixent (dupilumab) has been recommended for the treatment of moderate-to-severe prurigo nodularis (PN) in Scotland but not in England and Wales. 14 February 2024
Pharmaceutical
KalVista Pharmaceuticals shares, which closed Monday at $16.04, were recently up 37% to $22 in pre-market activity, but fell back almost 13% to $14.01 by late morning today, after it released new research data. 13 February 2024
Biotechnology
Rare diseases specialist Swedish Orphan Biovitrum (also known as Sobi) has set up a joint venture with South Korean firm Handok to commercialize its drugs in the latter’s home market. 13 February 2024
Pharmaceutical
The European Commission (EC) has authorized Biogen’s Skyclarys (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older. 13 February 2024
Pharmaceutical
UK-based Poolbeg Pharma has announced that research conducted on its behalf confirms a market opportunity of at least $10 billon for POLB 001 in cancer immunotherapy-induced cytokine release syndrome (CRS) as an orally-delivered preventative therapy. 12 February 2024
Biotechnology
M&A was a feature again last week, with two deals raising antitrust concerns. Under a two-part transaction by Novo Holdings, which has a major ownership role in Denmark’s diabetes care and obesity giant Novo Nordisk, announced it intends to acquire contract manufacturer Catalent for $16.5 billion, with Novo Nordisk in turn paying $11 billion for three of Catalent’s production sites, aiming to relieve supply shortages for Ozempic and Wegovy. Also, following earlier rumors, Novartis announced that it has made a $2.9 billion bid for Germany’s MorphoSys and its two investigation drugs pelabresib and tulmimetostat, but Incyte did a separate deal to buy rights for MorphoSys’ Monjuvi. On the licensing deal-making front, US clinical-stage biotech Immunome entered into as asset purchase deal with Ayala Pharmaceuticals for a Phase III cancer candidate to treat desmoid tumors. Also, the USA’s BridgeBio Pharma announced a licensing deal with Japan’s Kyowa Kirin for the development of infigratinib that earns it an upfront of 100 million. 11 February 2024
Biotechnology
US biopharma Synlogic is to discontinue Synpheny-3, its ongoing pivotal study of labafenogene marselecobac in phenylketonuria (PKU), with the firm’s board now evaluating strategic options for the company. 9 February 2024
Biotechnology
Crovalimab, a humanized complement inhibitor C5 monoclonal antibody discovered by Japan’s Chugai Pharmaceutical (TYO: 4519), has been approved by China’s National Medical Products Administration (NMPA) for treatment of PNH. 8 February 2024
Biotechnology
California, USA-based biotech BridgeBio Pharma today announced a partnership wherein its affiliate, QED Therapeutics, grants Japan’s Kyowa Kirin an exclusive license for infigratinib. 7 February 2024
Pharmaceutical
French pharma major Sanofi has announced that Rippon Ubhi has been appointed as country lead for the UK and Ireland, taking over immediately from Jessamy Baird. 7 February 2024
Biotechnology
The UK's Medicines and Healthcare Regulatory Agency (MHRA) has licensed subcutaneous (SC) injectable Vyvgart (efgartigimod alfa) as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody positive. 7 February 2024
Biotechnology
The long and winding road of biopharma development has taken another turn for Rallybio, with the firm opting to slash almost half of its workforce to preserve cash. 7 February 2024
Biotechnology
British gene specialist Silence Therapeutics as raised around $120 million in a private share placement. 6 February 2024
Biotechnology
US healthcare giant Johnson & Johnson late Monday announced top-line results from the pivotal Phase III VIVACITY study of nipocalimab in adults living with generalized myasthenia gravis (gMG), as well as the Phase II DAHLIAS study of nipocalimab in adults with Sjögren’s disease (SjD). 6 February 2024
Biotechnology
US biopharma company Vertex Pharmaceuticals showcased a year of robust growth and significant progress in its latest earnings call. 6 February 2024
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