Sanofi adds to hemophilia arsenal with FDA nod for Qfitlia
Sanofi SA gained U.S. FDA approval for fitusiran as a first-in-class siRNA therapy for hemophilia. Branded Qfitlia, the antithrombin-lowering therapy is indicated for use as a prophylactic treatment to prevent or reduce bleeding episodes in people with hemophilia A or B, with or without inhibitors, and joins the small but growing group of non-factor therapy options, with the advantage of a broad label and a convenient dosing regimen.

Sanofi adds to hemophilia arsenal with FDA nod for Qfitlia
Sanofi SA gained U.S. FDA approval for fitusiran as a first-in-class siRNA therapy for hemophilia. Branded Qfitlia, the antithrombin-lowering therapy is indicated for use as a prophylactic treatment to prevent or reduce bleeding episodes in people with hemophilia A or B, with or without inhibitors, and joins the small but growing group of non-factor therapy options, with the advantage of a broad label and a convenient dosing regimen.
Alzheimer’s drug Kisunla rejected by European regulators
The EMA has rejected the Alzheimer’s disease therapy Kisunla (donanemab) from Eli Lilly and Co., saying the benefits of the anti-amyloid antibody do not outweigh the risks of edema and hemorrhage in the brain, known as amyloid-related imaging abnormalities (ARIA).
Novo Nordisk licenses Lexicon’s preclinical obesity drug in $1B deal
As companies continually search for next-generation obesity prospects, one of the leaders in the disease space, Novo Nordisk A/S, has obtained an exclusive license to a preclinical-stage, first-in-class, small-molecule inhibitor of Acyl-CoA synthetase 5 (ACSL5) developed by Lexicon Pharmaceuticals Inc.
Myst it again: Milestone gets a CRL for its heart spray
Milestone Pharmaceuticals Inc.’s heart rhythm-restoring calcium channel blocker for paroxysmal supraventricular tachycardia (PSVT) has yet another obstacle in its way.
Australia looks to Asia as Trump administration threatens funding
Australia’s top universities are looking more to Asia for research collaborations following threats from the Trump administration to stop funding research at institutions that don’t comply with U.S. narratives.
Daewoong sees end of CS Pharma deal; new starts in cancer, DTx
It was a mixed day for Daewoong Pharmaceutical Co. Ltd., which disclosed separately on March 28 both the termination of a $336 million licensing deal with CS Pharmaceuticals Ltd. and upbeat plans to unveil three new oncology assets at the upcoming American Association for Cancer Research in April.
February’s phase III wins include Qyuns’ ankylosing spondylitis data
In February 2025, BioWorld recorded 148 updates from phase I to phase III trials, marking a decline from the 171 updates tracked in January.

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Analysis and data insight

Bifunction conjunction afoot; multiple players competing
Cancer
Bispecific antibodies have gotten plenty of press in recent years, but less prominent in headlines are bifunctional ones – i.e., those that boast one binding site for an antigen and another site for a non-antibody molecule such as a toxin or drug, taking aim at a single target and bringing a...
Checkpoint inhibitor journey traces Chinese firms’ path to global revenue
Immuno-oncology
Chinese pharmaceutical companies are making significant inroads into the global oncology market, particularly with the development and approval of immune checkpoint inhibitors. These treatments, which have shown strong efficacy in various cancer indications, are not only transforming the oncology...
Nature Cell wins FDA breakthrough designation of Jointstem
Regulatory
Nature Cell Co. Ltd. won U.S. FDA breakthrough therapy designation of its autologous adipose-derived mesenchymal stem cell therapy, Jointstem, March 20, becoming the first Korean company to earn the label in the field of cell therapy.
Report shows increased pharma innovation despite rising R&D costs
Science
The obesity market is driving pharma’s return on investment (ROI) upward, with the projected return rising to 5.9% overall in 2024, but only reaching 3.8% if GLP-1 assets are excluded. At the same time, the average cost of developing a drug continues to rise and now stands at $2.23 billion,...

Financings

Hillstar launches with $67M series A to target TRBV9+ T cells
Newco news
Hillstar Bio came out of stealth mode, announcing a $67 million series A financing round with investors including Droia Ventures, Frazier Life Sciences, Novo Holdings A/S and Lifearc Ventures.

Science

Ten days of normal survival of a pig liver in a human being
Drug design, drug delivery and technologies
Transplanting an animal organ into a human is now a closer reality following the successful xenotransplantation of a genetically modified pig liver into a patient diagnosed with brain death in China. The operation was intended to evaluate organ function over a 10-day period. This is a complex...

EU seeking talented scientists amid US funding woes
Europe
Science ministers from 13 countries in Europe are calling on the European Union to offer a home to researchers affected by the Trump Administration’s cuts. They have written to EU research and innovation commissioner Ekaterina Zaharieva asking her to organize a welcome “for brilliant talents from...
Hepatic stellate cells shape and control liver function
Gastrointestinal
Researchers at the German Cancer Research Center Deutsches Krebsforschungszentrum (DKFZ) and their collaborators have cast new light on the mechanisms by which hepatic stellate cells control liver metabolism and regeneration. The work builds on the concept of angiocrine signaling, established 15...

Newco news

Astellas-Yaskawa JV to build robot-based cell production platform
Deals and M&A
Astellas Pharma Inc. is setting up a joint venture with Yaskawa Electric Corp. to develop a new cell production platform using Yaskawa’s dual-arm humanoid robot called Maholo.
Hummingbird migrates ADCs, tech to newco Callio’s $187M debut
Financings
Antibody-drug conjugate (ADC)-focused Callio Therapeutics debuted on March 3 with the closing of a $187 million series A financing round based on cancer drug technology and assets in-licensed from Hummingbird Bioscience Pte Ltd.
Harbour Biomed newco HBM Alpha pens $395M hyperplasia drug deal
Analysis and data insight
HBM Alpha Therapeutics Inc. signed a potential $395 million licensing deal Feb. 26 with an unnamed “business partner” for its endocrine asset, HAT-001, adding another contender to the congenital adrenal hyperplasia space.
Ligachem gets rights to Daan’s antibody for solid tumor ADCs
Deals and M&A
Ligachem Biosciences Inc. landed another exclusive licensing deal to develop and commercialize antibody-drug conjugates (ADCs), this time with T-cell receptor therapy specialist Daan Biotherapeutics Inc. for a cancer-targeting antibody.
Biomissile’s multispecific antibodies harness NK cell engagers
Cancer
Biomissile Pharmaceuticals Co. Ltd. is developing multispecific antibodies that overcome resistance associated with antibody-drug conjugates via its next-generation natural killer (NK) cell engagers. “ADCs are very efficacious, but they do have a drawback with side effects and resistance, because...

U.S.

Disease-modifying Alzheimer’s drugs are ‘drivers of change’
Aging
The first disease modifying therapies for Alzheimer’s may have limited utility in some senses, but they will be a force for change, providing momentum and altering the way governments as payers, and health systems as carers, think about the disease.

Europe

UK trade association says drug rebate hurting industry
Pharma companies in the U.K. said the rebate they are required to make on drug sales is making the country “un-investible,” prompting staff cuts and leading clinical research partnerships to be unwound. Rather than the 15.3% rebate on branded drugs companies expected to pay this year, the rate has...

WHO looking to fill funding gap left by the US
Regulatory
The U.S. foreign aid cuts and freezes that are taking place under President Donald Trump are putting at risk the global public health gains that have been made against diseases such as malaria, HIV and tuberculosis over the past two decades, World Health Organization (WHO) Director-General Tedros...
Torqur’s actinic keratosis data catches pharma eye, says CEO
Clinical
Term sheets are being drawn up with potential pharma partners after Torqur AG reported positive interim results from an ongoing phase II trial of bimiralisib topical gel in the treatment of actinic keratosis.

Clinical

Come what May: Equillium marches on with itolizumab in GVHD
Immune
Equillium Inc. is forging ahead with regulatory plans for itolizumab in first-line treatment of patients with acute graft-vs.-host disease (GVHD), despite a phase III disappointment. Shares of the La Jolla, Calif.-based firm (NASDAQ:EQ) closed...
Riding the Wave, exon skipper sails toward DMD filing
Musculoskeletal
Wave Life Sciences Ltd. plans to file with the U.S. FDA for accelerated approval of WVE-N531, an exon skipping oligonucleotide for boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping, a decision based on favorable data...
Opthea in freefall after phase III fail in wet AMD
Ocular
Opthea Ltd. is considering its options after lead candidate sozinibercept (OPT-302) failed the phase III Coast trial in wet age-related macular degeneration (AMD). The global Coast phase III trial evaluated the efficacy and safety of...
Taimed’s long-acting HIV treatment offers daily ART alternative
Immune
Taimed Biologics Inc.’s TMB-365/TMB-380 long-acting combination of broadly neutralizing antibodies (bnAbs) regimen for HIV maintenance could reshape HIV treatment management, offering a viable alternative to daily combination antiretroviral...
Elevation buries claudin 18.2 ADC for cancer, slashes staff by 70%
Cancer
Elevation Oncology Inc. has nixed its lead pipeline product, a claudin 18.2 antibody-drug conjugate (ADC) called EO-3021, on disappointing phase I data, sending shares tumbling by 42% and placing its preclinical HER3 ADC to the forefront of...
En garde, Vyvgart: Immunovant rolls out MG data
Immune
Roivant Sciences Ltd. CEO Matt Cline said the firm’s unit Immunovant Inc. with FcRn blocker batoclimab has established “frankly a new bar” in myasthenia gravis (MG) as the New York-based firm reported top-line results from its phase III study and...

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podcast microphone, sound waves on purple background Breakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.

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