Clinical research - Today Stories
Regeneron’s experimental siRNA therapy, cemdisiran, successfully met the main objectives in a Phase III trial for adults with generalized myasthenia gravis. In the 24‑week study, cemdisiran monotherapy led to a 74 percent reduction in complement protein C5 levels and delivered measurable improvement in patients’ daily activities such as talking and eating. When combined with Regeneron’s approved C5 blocker pozelimab (Veopoz), C5 suppression increased to nearly 99 percent, though cemdisiran alone performed slightly better on key disease‑specific outcomes. No patients discontinued treatment due to adverse events. The company now plans to submit an FDA marketing application in early 2026. This development positions cemdisiran as a promising competitor in the field, potentially offering at‑hom…
Lundbeck is advancing bexicaserin - an oral 5‑HT₂C receptor agonist initially developed by Longboard Pharmaceuticals - for the treatment of severe developmental and epileptic encephalopathies (DEEs), including Dravet syndrome and Lennox–Gastaut syndrome. Lundbeck acquired the asset through a ~$2.6 billion takeover of Longboard in late 2024.
In a 12‑month open‑label extension (OLE) of the Phase 1b/2a PACIFIC trial, bexicaserin delivered a sustained and clinically meaningful reduction in motor seizures—median decrease of 59.3% from baseline. Participants who had been on bexicaserin from the trial start achieved a 60.4% reduction; those switching from placebo saw a 58.2% decrease. The safety profile remained favourable, with most participants completing the year-long study and common adverse…
Tiziana Life Sciences has reached a key milestone in its Phase 2a clinical trial of intranasal Foralumab for non‑active secondary progressive multiple sclerosis (na‑SPMS). Patient dosing has now commenced at Yale’s Multiple Sclerosis Center, expanding the study’s reach beyond its initial sites and underscoring progress in the multicentre, randomized, double‑blind, placebo‑controlled investigation.
Launched in late 2023, the trial explores two dosing regimens of Foralumab against placebo, assessing effects on microglial activation via PET imaging, alongside standard measures of disability, cognition, fatigue, and quality of life. The inclusion of Yale adds to a network of major US research centres (including Johns Hopkins, UMass, Brigham and Women’s Hospital, and more) enhancing the study’s…
Stoke Therapeutics and Biogen have initiated the global Phase III EMPEROR trial of zorevunersen (previously known as STK‑001) for children and adolescents with Dravet syndrome. This pioneering antisense oligonucleotide is designed to boost expression of the healthy SCN1A gene, potentially addressing not just seizure frequency but also cognitive and behavioral impairments. The study will enroll approximately 150 patients across the United States, United Kingdom, Europe, and Japan. Participants will undergo 8 weeks of baseline monitoring followed by 52 weeks of treatment, beginning with two 70 mg loading doses and transitioning to two 45 mg maintenance doses. The primary outcome measure is the reduction in major motor seizure frequency, with secondary outcomes assessing improvements in behav…
Pfizer and Astellas announced encouraging interim results from a late stage trial combining its antibody drug conjugate Padcev with Merck’s immunotherapy Keytruda in patients with muscle invasive bladder cancer. The combination, administered before and after surgery, delivered significant improvements in both event‑free survival (how long patients remained recurrence‑free) and overall survival compared to surgery alone. Johanna Bendell, Pfizer’s oncology chief development officer, emphasised the potential for this treatment to reshape standard care for this aggressive cancer, which accounts for around 25 percent of new bladder cancer cases. Padcev, approved in 2019 for metastatic urothelial cancer, generated $1.59 billion in sales last year. Pfizer and Astellas now plans to engage with glo…
AstraZeneca’s experimental hypertension drug, baxdrostat, has achieved a major milestone. The company announced that its BaxHTN Phase III trial met both primary and secondary endpoints, demonstrating a statistically significant and clinically meaningful reduction in systolic blood pressure after 12 weeks in patients with uncontrolled or treatment-resistant hypertension. The trial involved two once‑daily doses (1 mg and 2 mg) alongside standard therapy, compared to placebo, and showed strong efficacy and favourable safety profiles.
Developed through AstraZeneca’s 2023 acquisition of CinCor Pharma, baxdrostat represents a novel class of aldosterone synthase inhibitors that target hormonal drivers unrelated to traditional ACE inhibitors. The treatment‑resistant hypertension market affects ove…
AbbVie has entered into an exclusive licensing agreement with Ichnos Glenmark Innovation (IGI), a joint venture between Glenmark Pharmaceuticals and Ichnos Sciences, to develop and commercialize ISB 2001—a trispecific T-cell engager targeting BCMA, CD38, and CD3—for relapsed or refractory multiple myeloma.
Under the terms, AbbVie will acquire rights to ISB 2001 across North America, Europe, Japan, and Greater China, providing IGI with a $700 million upfront payment and up to $1.225 billion in milestone payments.
ISB 2001, currently in Phase 1 trials, has demonstrated an overall response rate of 84% in patients naive to CAR-T or T-cell engager therapies, and 71% in those previously treated, including those refractory to anti-CD38 therapy.
This collaboration enhances AbbVie's oncology pipe…
Epitopea, a transatlantic cancer immunotherapeutics company, has announced the formation of a dedicated clinical team and the expansion of its research team as it accelerates the development of its RNA-based immunotherapies. The company is focused on creating off-the-shelf treatments targeting Cryptigen™ tumor-specific antigens (TSAs), which are aberrantly expressed antigens derived from non-coding regions of the genome, commonly referred to as "junk DNA."
The establishment of a clinical team marks a significant milestone in Epitopea's transition from a preclinical to a clinical-stage company. This move is supported by the company's proprietary CryptoMap™ platform, which enables the identification of novel Cryptigen™ TSAs shared across patients with the same tumor type. These antigens serv…
FDA Grants Accelerated Approval to Lynozyfic for Advanced Multiple Myeloma
Regeneron Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Lynozyfic™ (linvoseltamab-gcpt) to treat adults with relapsed or refractory multiple myeloma (R/R MM) who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.
Lynozyfic is a bispecific antibody that targets B-cell maturation antigen (BCMA) on myeloma cells and CD3 on T cells, facilitating T-cell activation and cancer cell killing. It is the first FDA-approved BCMAxCD3 bispecific antibody with a response-adapted dosing regimen, allowing for dosing every two weeks starting at week 14, and every fo…
Pfizer has discontinued its Phase 1b/2 trial of maplirpacept (PF-07901801), a CD47-blocking agent, in combination with tafasitamab and lenalidomide for relapsed or refractory diffuse large B-cell lymphoma (DLBCL) patients ineligible for stem cell transplantation. The trial, which began in August 2023, enrolled only six participants before being terminated due to recruitment challenges. Pfizer stated that the decision was not related to safety or efficacy concerns.
Maplirpacept, acquired through Pfizer's $2.3 billion purchase of Trillium Therapeutics in 2021, is designed to block CD47—a protein that cancer cells use to evade immune detection. Despite this setback, Pfizer continues to explore maplirpacept's potential in other blood cancers, including multiple myeloma and acute myelocytic leu…
CSL Behring has achieved a significant milestone with the German Institute for Quality and Efficiency in Health Care (IQWiG) issuing a positive benefit assessment for garadacimab, its once-monthly prophylactic treatment for hereditary angioedema (HAE) in patients aged 12 years and older.
IQWiG's evaluation, based on a methodologically sound indirect comparison using placebo as a common comparator, concluded that garadacimab offers a "hint of considerable additional benefit" in reducing monthly HAE attack rates. This marks a precedent in the AMNOG process since its inception in 2011.
The assessment also noted minor additional benefits in general health status (EQ-VAS) and disease-specific quality of life (AE-QoL). CSL Behring's submission included data from the VANGUARD study, comparing gar…
Oracle has announced major advancements in its Randomization and Trial Supply Management (RTSM) solution, introducing vendor-agnostic interoperability and drug pooling capabilities. These innovations enable real-time drug inventory management across multiple clinical trials and vendors—cutting waste and accelerating timelines.
Sponsors and CROs can now use the same investigational product across different studies, with seamless integration to leading supply providers like SAP, Almac, Fisher Clinical Services, Catalent, and PMD. Built on Oracle Cloud Infrastructure, RTSM supports end-to-end automation in drug supply ordering, tracking, and allocation.
The enhancements reflect Oracle’s commitment to open, connected systems and its broader vision to unify clinical and health applications. Wit…
Sanofi announced today that the U.S. FDA has granted orphan drug designation to rilzabrutinib for the treatment of sickle cell disease, marking its fourth orphan status for this investigational Bruton's tyrosine kinase (BTK) inhibitor. The oral, reversible BTK inhibitor targets immune system modulation and aims to reduce vaso-occlusive crises—a major source of pain and complications in sickle cell patients.
Rilzabrutinib is also under regulatory review in the US, EU, and China for immune thrombocytopenia (ITP), with an FDA decision expected by August 29, 2025. Previous designations include approvals for ITP, warm autoimmune hemolytic anemia (wAIHA), and IgG4-related disease.
Preclinical data presented at ASH 2024 demonstrated rilzabrutinib’s potential to reduce inflammation and blood vesse…
21 May 2025 – Ramsey, Isle of Man – Juvenescence Ltd., a clinical-stage biotech leveraging AI to target age-related diseases, has announced the successful first close of its Series B-1 financing round, raising $76 million. The round was led by Abu Dhabi-based M42, which also enters a strategic partnership with Juvenescence to advance AI-driven therapeutics.
The fresh capital will propel Juvenescence’s pipeline of age-related disease therapies into later-stage clinical trials, with the second tranche of the Series B-1 round expected to close in Q3 2025.
This financing follows the announcement of a landmark collaboration between Juvenescence and M42, aimed at establishing a cutting-edge drug development hub in Abu Dhabi. The initiative will merge M42’s capabilities in digital health, genomic…
Researchers at the University of Turku, Finland, have taken a step towards more personalised and effective cancer treatments. A new study has identified the conditions under which the antibody drug bexmarilimab can activate the body's own defence system against cancer, and how to identify patients who will benefit from the drug. Bexmarilimab has the ability to “wake up” macrophages, a type of immune cell, to act against cancer. The study found that this activation occurs especially when the tumour microenvironment is immunologically silent. Moreover, in the healthy tissue adjacent to the tumour, bexmarilimab was shown to trigger B cell–mediated immune responses, regardless of how sensitive the neighbouring tumour was to the treatment. This suggests that the drug may have immunological effe…
Mulhouse, France, April 20, 2025 – CellProthera, a regenerative cell therapy developer specializing in ischemic diseases, has chosen CELLforCURE by SEQENS, as its contract development and manufacturing organization (CDMO) partner for its planned Phase 3 trial for ProtheraCytes®, its autologous expanded CD34+ stem cell-based therapy aimed at improving heart failure event-free survival following a severe heart attack. CellProthera will tech transfer to CELLforCURE by SEQENS this year, with plans to produce clinical batches starting in 2026.
With promising results from a Phase 1/2b study of its lead ProtheraCytes®, CellProthera has begun preparations for its late stage of clinical development. Last year, the company presented its Phase 3 design to FDA and received favorable advice. Tech tran…
A new report reveals disparities in chimeric antigen receptor (CAR) T-cell therapy delivery across Europe, with healthcare system utilisation rates of the therapy as low as 11% for eligible patients. The independent research ‘Achieving CAR T-Cell Therapy Health System Readiness: An assessment of barriers and opportunities’ by the IQVIA Institute for Human Data Science, funded by Gilead and Kite, highlights challenges across countries that can lead to uneven access to CAR T-cell therapy. The report, which assessed seven countries (Australia, Canada, France, Germany, Italy, Spain, the United Kingdom), found that factors such as complex referral pathways, limited treatment centers and reimbursement challenges are hindering access to this potentially life-saving treatment for eligible patient…
Adoption allows ICON to take full advantage of the Medidata “Data Experience,” leveraging AI to unify data sources, standardize outputs, and reduce reconciliation efforts
New York – March 20, 2025 – Medidata, a Dassault Systèmes brand and leading provider of clinical trial solutions to the life sciences industry, today announced that ICON plc, (NASDAQ: ICLR) a world-leading clinical research organization (CRO) powered by healthcare intelligence, will immediately begin utilizing Medidata Clinical Data Studio on its studies, differentiating ICON as the first large CRO to fully infuse this technology into its clinical workflows.
The integration of Clinical Data Studio into ICON’s service offerings helps unlock the full potential of the Medidata Data Experience, merging information from both M…
RESEARCH TRIANGLE PARK, N.C. – February 24, 2025 – IQVIA (NYSE:IQV), a leading global provider of advanced analytics, technology solutions and clinical research services to the life sciences and healthcare industries, today announced that IQVIA AI Assistant has been awarded a 2024 PM360 Innovation Award in the Artificial Intelligence category.
Launched in September 2024, IQVIA AI Assistant revolutionizes insight generation by providing rapid, relevant and precise answers to complex business questions. Using a conversational interface with advanced data science, it makes complex analytics accessible to a wide variety of users and provides near real-time insights.
IQVIA AI Assistant builds upon IQVIA’s decade of experience in artificial intelligence and developing Healthcare-grade AI™, a pro…
Enterprise Ireland grant awarded to ICON to further enhance artificial intelligence capabilities
Dublin, Ireland – 30 January 2025 – ICON plc, (NASDAQ: ICLR) a world-leading clinical research organisation powered by healthcare intelligence, today announced the expansion of its portfolio of artificial intelligence (AI) tools that deliver efficiencies across the clinical trial process, including study startup, document management, resource forecasting and metrics reporting.
ICON has invested significantly in its AI and digital capabilities through the establishment of its AI Centre of Excellence. The centre, which comprises a dedicated team of data scientists, engineers and domain experts, is responsible for developing and deploying AI solutions that accelerat…
