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US rare diseases specialist Fulcrum Therapeutics lost more than half of its stock market value on Friday. 27 February 2023
Pharmaceutical
Coming back from a major partnership disappointment last week, Boston, USA-based biotech Blueprint Medicines has now released positive results from the PIONEER trial of Ayvakit (avapritinib) in patients with indolent systemic mastocytosis (SM). 27 February 2023
Biotechnology
Detailed Phase III results for a new long-term prophylactic treatment for hereditary angioedema (HAE) have been presented at the annual meeting of the American Academy of Allergy Asthma & Immunology (AAAAI). 27 February 2023
Biotechnology
Last week’s important regulatory news included the US Food and Drug Administration (FDA) approving Travere Therapeutics’ Filspari for the reduction of proteinuria in adults with primary IgAN. Among research news, Actinium Pharmaceuticals released positive Phase III data on its myeloid leukemia candidate Iomab-12. On the M&A front, troubled US drug developer Jounce Therapeutics found a ‘white knight’, entering a reverse merger with the UK’s Redx Pharma. Deal-making saw AstraZeneca sign a licensing agreement with China-based KYM Biosciences for CMG901, a potential gastric cancer treatment to expand its own portfolio. Also, having already forked out around $1 billion in upfront and milestone payments, Swiss pharma giant Roche terminated its agreement with US biotech Blueprint Medicines on the cancer drug Gavreto. 26 February 2023
Biotechnology
At its February meeting, the European Medicines Agency’s human medicines committee CHMP recommended approval of eight novel medicines, one of which is a biosimilar. 24 February 2023
Biotechnology
The US Food and Drug Administration (FDA) has approved Altuviiio (efanesoctocog alfa), a high-sustained factor VIII replacement therapy. 24 February 2023
Biotechnology
Today, Dutch gene therapy company uniQure announced that the prestigious New England Journal of Medicine (NEJM) has published data from the pivotal Phase III HOPE-B study evaluating the efficacy, durability and safety of Hemgenix (etranacogene dezaparvovec-drlb), the first gene therapy approved for the treatment of adults with hemophilia B. 23 February 2023
Biotechnology
Two more American biotech companies have been forced to announce plans to restructure, including mandatory redundancies, to avoid looming financial trouble. 23 February 2023
Biotechnology
US biotech Actinium Pharmaceuticals saw its shares rise as much as 21%, after it announced positive results for the primary and secondary endpoints from its pivotal Phase III SIERRA trial of Iomab-B in patients age 55 and above with active relapsed or refractory acute myeloid leukemia (r/r AML). 22 February 2023
Pharmaceutical
Karyopharm Therapeutics and privately-held Italian drugmaker Menarini Group today announced that the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted full Marketing Authorization for Nexpovio (selinexor) in combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy. 21 February 2023
Biotechnology
The European Commission has granted conditional marketing authorization (CMA) for Hemgenix (etranacogene dezaparvovec), the first and only one-time gene therapy for the treatment of severe and moderately severe hemophilia B (congenital Factor IX deficiency) in adults without a history of Factor IX inhibitors. 21 February 2023
Biotechnology
HealthCap VIII portfolio company Hemab Therapeutics has closed an oversubscribed $135 million Series B financing. 21 February 2023
Biotechnology
Research news featured last week, first with US pharma giant Bristol Myers Squibb and 2seventy bio presenting Phase III results for their CAR-T cell therapy Abecma (ide-cel) in multiple sclerosis. Also, G1 Therapeutics announced it was terminating a trial of its Cosela (trilaciclib) in colorectal cancer. Additionally, Pfizer released new Phase III data on its Talzenna in combination with Xtandi that missed one of its objectives. On the deal-making front, US drug developer Cullinan Oncology entered into a collaboration with China-based Harbour BioMed for the development of a potential cancer treatment, HBM7008/CLN-418, and USA-based Arrowhead Pharma revealed that Johnson & Johnson subsidiary Janssen has pulled out of a 2018 collaboration on the development of non-alcoholic steatohepatitis (NASH) candidate ARO-PNPLA3, formerly JNJ-75220795. 19 February 2023
Pharmaceutical
Spanish producer of blood plasma-based products Grifols has joined the ranks of pharma and biotech companies cutting their budgets and workforces, in a move that was welcomed by investors, with the firm’s shares rising 9% Wednesday and a further 8.6% to 14.76 euros this morning. 16 February 2023
Biotechnology
Hematologic diseases specialist Disc Medicine has announced a $62.5 million financing. 15 February 2023
Pharmaceutical
US rare diseases specialist Soligenix closed 19% lower after Tuesday’s trading following news of a setback with its lead drug candidate. 15 February 2023
Biotechnology
Australian biotech company CSL Limited , the world's No 2 influenza vaccine provider in sales, today released financial results for the fist half of fiscal year 2022/23, showing that total revenues were up 19% to US$7.184 billion, US$889 million of which is attributable to CSL Vifor. 14 February 2023
Biotechnology
Positive Phase III data for Bristol Myers-Squibb’s Abecma (idecabtagene vicleucel) underscore the potential of the firm’s CAR-T cell therapy in multiple myeloma. 13 February 2023
Biotechnology
US biotech Magenta Therapeutics has announced the outcome of a review of its business, including the status of its programs, resources, and capabilities. 10 February 2023
Biotechnology
Adding to a string of strong clinical trials reports for its already-marketed drug, Gilead Sciences subsidiary Kite today announced the three-year follow-up results from the pivotal ZUMA-3 study of its CAR T-cell therapy Tecartus (brexucabtagene autoleucel). 9 February 2023
Biotechnology
San Diego-based Kura Oncology and Japan’s Kyowa Kirin have entered into a global strategic collaboration to develop and commercialize ziftomenib. 21 November 2024
Biotechnology
The European Commission (EC) has granted marketing authorization for Hympavzi (marstacimab), from US pharma giant Pfizer. 20 November 2024
Pharmaceutical
Ascentage Pharma will see its investigational BCL-2 selective inhibitor, lisaftoclax, assessed under China’s Priority Review scheme. 20 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) has approved Syndax Pharmaceuticals’ Revuforj (revumenib). 18 November 2024
Biotechnology
UK drugmaker GSK might be trading lower following Thursday’s nomination of vaccine sceptic Robert F Kennedy Jr as the USA’s next health secretary, but the day brought good news for the group elsewhere in the business. 15 November 2024
Biotechnology
Adaptimmune Therapeutics reported promising data Wednesday from its pivotal Phase II trial of lete-cel in certain solid tumors with few effective treatments. 14 November 2024
Biotechnology
Sanofi has announced that the UK's health technology assessor will conduct a new review of its Sarclisa (isatuximab) combo therapy for relapsed or refractory multiple myeloma. 13 November 2024
Biotechnology
Syndax Pharmaceuticals has announced that its Phase II AUGMENT-101 trial of experimental drug revumenib achieved its primary endpoint. 13 November 2024
Biotechnology
VarmX, a Dutch biotech developing innovative approaches for the bypass of direct oral anticoagulants targeting activated factor Xa (FXa DOACs) and inherited coagulation disorders, has appointed industry veteran John Glasspool (pictured, above) as chief executive. 12 November 2024
Biotechnology
On November 8, the US Food and Drug Administration approved Aucatzyl (obecabtagene autoleucel; obe-cel) from US biopharma Autolus Therapeutics' 11 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
Biotechnology
US healthcare giant Johnson & Johnson today revealed it has submitted applications with the US Food and drug Administration (FDA) and European Medicines Agency (EMA) to expand the approved indications for its Darzalex (daratumumab) subcutaneous (SC) formulation for the treatment of high-risk smouldering multiple myeloma (SMM). 8 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
Beam Therapeutics will detail new clinical and preclinical data on its sickle cell disease (SCD) therapies at the upcoming American Society of Hematology (ASH) meeting. 6 November 2024
Pharmaceutical
An impressive set of quarterly financial results and an improved outlook for the rest of the year were announced by Bristol Myers Squibb on Thursday. 31 October 2024
Biotechnology
Maryland, USA–based glycobiology specialist GlycoMimetics has entered into an acquisition agreement with Crescent Biopharma, a privately held biotechnology company advancing a pipeline of oncology therapeutics designed to treat solid tumors. 30 October 2024
Pharmaceutical
Novartis late yesterday revealed that Scemblix (asciminib) was granted accelerated approval by the US Food and Drug Administration (FDA) for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). 30 October 2024
Biotechnology
Last week Vertex Pharmaceuticals released positive Phase III results for its suzetrigine for moderate-to-severed pain. Belgium’s UCB revealed that Swiss pharma giant Roche was terminating its collaboration on the Alzheimer’s candidate bepranemab. The UK’s health technology assessor the Institute for Health and Clinical Excellence (NICE) declined to recommend a second Alzheimer’s treatment, this time Eli Lilly’s Kisunla. Also of note, Lyell Immunopharma announced its planned acquisition of ImmPACT Bio and reprioritization of its own clinical pipeline. 27 October 2024
Biotechnology
California, USA-based Lyell Immunopharma (Nasdaq: LYEL) has entered into a definitive agreement to acquire privately-held US CAR-T company ImmPACT Bio. 26 October 2024
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