Clinical research - Today Stories

Thermo Fisher Scientific has entered into a definitive agreement to acquire Clario Holdings, Inc., a leader in endpoint data solutions for clinical trials, in a transaction valuing Clario at approximately US$8.9 billion in cash, with additional performance-based payments possible. The acquisition is expected to close by mid-2026, subject to regulatory approvals and customary closing conditions. Clario’s platform collects and analyzes clinical trial data from devices, sites and patients thereby enabling sponsors to derive deeper insights and make faster decisions.  Thermo Fisher says the move will strengthen its offerings for pharmaceutical and biotech customers seeking to accelerate drug development and bring therapies to patients more efficiently.  In financial terms the deal is expecte…

Novartis has announced that its oral complement-factor B inhibitor Fabhalta® (iptacopan) has met the primary endpoint of the Phase III APPLAUSE‑IgAN study in adults with IgA nephropathy (IgAN). In the final analysis of the trial, patients receiving Fabhalta showed a statistically significant improvement in the annualised slope of estimated glomerular filtration rate (eGFR) decline compared with placebo over two years, indicating slowed kidney-function deterioration. According to Novartis, Fabhalta is the first and only approved complement inhibitor for adult IgAN and the new data support its potential to delay disease progression. The study included adults with biopsy-confirmed primary IgAN who had proteinuria and were on maximally tolerated renin-angiotensin system inhibitors with or with…

Innovative technology platform reshapes how sponsors, CRO, and clinical sites manage complex clinical trials at scale   Almac Group has today announced a $48 million investment in eClinical technologies that includes the launch of Almac Trial Coordinator™ – a first-in-class interoperable clinical trial technology platform, enhancements to its eClinical ecosystem capabilities, and creation of over 100 advanced technology roles globally. This investment addresses the top technology challenges facing sponsors, CROs, and clinical sites through the solution’s market-disrupting and interoperable approach to integration, enabling high-quality, efficient trials with full visibility, reduced risk, and enhanced patient engagement.   Almac Trial Coordinator™ is purpose-built to unify operational proc…

Discovery Park in Sandwich, Kent, has announced the launch of a new specialist sixth form for science-focused students aged 16 to 18. Named Carbon 6 Academy of Science, the school will open in September 2026 and will deliver a dedicated STEM curriculum. Located within the Discovery Park innovation campus, Carbon 6 will give students direct access to professional laboratories and real-world scientific environments. The academy will offer A-levels in Biology, Chemistry, Physics and Mathematics, with options for Further Maths and an Extended Project Qualification. Students will benefit from close connections to the science and pharmaceutical companies based at Discovery Park, gaining opportunities for mentoring, guest lectures, and research placements. The programme is designed to prepare lea…

AI-native biotech startup expands to Oxford to develop predictive models for safer, faster drug discovery Fukuoka, Japan – October 3, 2025 – Tres Alchemix, a cutting-edge biotechnology company applying artificial intelligence (AI) to drug discovery, has closed a $4.4 million Series A funding round (approx. ¥650 million JPY), led by DNX Ventures, with support from Waseda University Ventures. The funding will fuel the development of Tres Alchemix’s human in silico drug-design platform, a technology that integrates multi-omic datasets, quantum-chemical calculations, and AI modeling to simulate drug interactions in the human body. This enables direct predictions of both therapeutic effects and potential adverse reactions, dramatically improving the speed and safety of early drug discovery. The…

University College London is leading a new clinical trial called ADAPT (Alzheimer’s Disease Diagnosis and Plasma p-tau217) that could transform how Alzheimer’s is diagnosed in the UK. The trial centres on a blood test that detects a protein marker p-tau217, which reflects the build-up of amyloid and tau proteins in the brain, two key hallmarks of Alzheimer’s disease. The goal is to see whether this test can increase diagnostic accuracy from around 70 percent with current clinical assessments to over 90 percent. Over 1,000 people with suspected dementia will be recruited from about 20 NHS memory clinics. Participants will be randomised into two groups: half will receive results within three months, the other half after 12 months. Researchers will examine whether earlier feedback improves di…

Novartis announced it will acquire Tourmaline Bio for approximately $1.4 billion, offering $48 per share, a significant premium over recent stock prices. The acquisition will provide Novartis access to pacibekitug, a promising anti-inflammatory drug candidate nearing Phase III trials for atherosclerotic cardiovascular disease. Both companies’ boards have approved the deal, and Novartis will initiate a tender offer to acquire all outstanding shares. The acquisition is expected to close in the fourth quarter of 2025, after which Tourmaline will become an indirect, wholly owned subsidiary of Novartis. The move bolsters Novartis’ growing cardiovascular disease portfolio and aligns with its broader strategic focus. Analysts see the timing as logical, given recent strong Phase 2 data for pacibek…

Clerkenwell Health has launched the UK’s first fully NHS‑embedded commercial research site dedicated to brain and mental health, in partnership with Rotherham Doncaster and South Humber NHS Foundation Trust (RDaSH). Located within an existing NHS facility in Doncaster, the site serves over 1.3 million people across South Yorkshire. This initiative offers NHS patients quicker access to cutting‑edge investigational treatments while addressing long‑standing delays that have discouraged global sponsors from conducting trials in the UK. By utilising underused NHS infrastructure and collaborating closely with NHS clinicians, the site also creates local jobs, enhances research capability through upskilling, and boosts workforce development. Rather than a pilot, it is the first in a planned nation…

United Therapeutics recently announced that its pivotal TETON-2 clinical trial of nebulized Tyvaso (treprostinil) inhalation solution for idiopathic pulmonary fibrosis achieved its primary endpoint. The study revealed a significant improvement in absolute forced vital capacity compared with placebo. Encouraging results were also seen across most secondary endpoints, such as time to first clinical worsening and quality of life measures. Strong outcomes from TETON-2 are intended to support a supplemental New Drug Application to the US Food and Drug Administration for an expanded indication in IPF. The company also intends to consult with the FDA later this year to explore opportunities to accelerate regulatory review. Additionally, more detailed findings are expected to be shared at the upco…

The National Institute for Health and Care Excellence has approved a novel first‑line treatment for adults with unresectable or metastatic urothelial cancer who are eligible for platinum-based chemotherapy. The regimen combines enfortumab vedotin (Padcev) with pembrolizumab (Keytruda) and is now available on the NHS in England and Wales. Clinical trials revealed that the new combination nearly doubled progression‑free survival—from 6.3 to 12.5 months—and extended overall survival dramatically, with median overall survival reaching 33.8 months compared to 15.9 months with chemotherapy alone. Around 30 percent of patients had no detectable cancer after treatment, markedly higher than rates seen with traditional chemotherapy. Oncology experts have hailed the development as a transformational…

Roche, together with its partner Alnylam, is propelling the RNA interference therapy zilebesiran into a Phase III cardiovascular outcomes trial following compelling Phase II results. At the European Society of Cardiology Congress 2025, data from the KARDIA‑3 study were presented. While the trial did not meet its primary endpoint due to statistical constraints, the 300 mg dose achieved a placebo‑adjusted systolic blood pressure reduction of 5 mmHg at Month 3 (p = 0.0431), with sustained benefits through Month 6. Safety data were encouraging, and subgroup analysis suggested enhanced benefit when zilebesiran was combined with a diuretic. The comprehensive insights from the KARDIA‑1 through KARDIA‑3 studies guided the decision to launch a global Phase III cardiovascular outcomes trial, targete…

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…