The first half of 2025 is poised to be transformative for clinical research, with pivotal trials spanning oncology, neurology, metabolic diseases, and gene therapies.
Alongside notable efforts highlighted in this report, additional groundbreaking trials—such as Eli Lilly’s Orforglipron for obesity, BioNTech’s BNT327 for cancer, and MindMed’s LSD-based therapy for anxiety—underscore the relentless innovation reshaping healthcare. These advancements signal the growing importance of precision medicine, novel biotechnologies, and holistic approaches to address complex diseases.
Collectively, the outcomes of these trials will shape the future of global healthcare and provide new hope for patients worldwide.
The landscape of clinical trials in 2025 demonstrates unprecedented innovation across diverse therapeutic areas. This report identifies key trials to monitor during the first half of the year, with a focus on oncology, neurodegenerative disorders, psychiatry, rare diseases, and metabolic and cardiovascular health. Additionally, newly emerging studies such as Eli Lilly’s Orforglipron for obesity, Summit Therapeutics’ Ivonescimab for lung cancer, and Beam Therapeutics’ BEACON trial for sickle cell disease further highlight the rapid pace of progress in precision medicine, advanced therapies, and mental health research.
Together, these efforts illustrate the dynamic intersection of science and healthcare to address critical unmet medical needs.
ONCOLOGY
•Arvinas: Veritac-2 trial testing vepdegestrant for estrogen receptor-positive breast cancer.
•AstraZeneca and Daiichi Sankyo: TROPION-Breast01 trial of datopotamab deruxtecan for HR-positive, HER2-low breast cancer.
•Bristol Myers Squibb: CheckMate-76K trial of nivolumab for early-stage melanoma.
•Merck: KEYNOTE-671 trial of pembrolizumab in resectable non-small cell lung cancer (NSCLC).
•Mirati Therapeutics: Trials of adagrasib for KRAS G12C-mutated cancers.
•BioNTech’s BNT327 Trials: BioNTech is advancing BNT327, an oncology candidate that could potentially rival existing treatments like Merck’s Keytruda. This trial signifies BioNTech’s expansion beyond mRNA based vaccines into cancer therapeutics.
•Summit Therapeutics’ Ivonescimab Trials: Summit Therapeutics is expected to launch Ivonescimab, a first-in-class bispecific antibody targeting PD-1 and VEGF, initially for non-small cell lung cancer (NSCLC), with potential expansion into breast cancer.
NEURODEGENERATIVE DISEASES
Alzheimer’s Disease
•Eisai and Biogen: Clarity AD and AHEAD trials testing lecanemab.
•Roche: GRADUATE trials for gantenerumab and semorinemab (anti-tau antibody).
•Novo Nordisk: Evoke and Evoke+ trials testing oral semaglutide for Alzheimer’s.
•Eli Lilly: TRAILBLAZER-ALZ 2 trial of donanemab and LUCIDITY trial of solanezumab.
Parkinson’s Disease
•Denali Therapeutics: TAK-994 and DNL151 (LRRK2 inhibitors).
•Voyager Therapeutics: VY-AADC gene therapy for advanced Parkinson’s.
•Biogen: BIIB122 (in partnership with Denali) targeting LRRK2.
•BlueRock Therapeutics: DA01 stem cell therapy for dopamine restoration.
Amyotrophic Lateral Sclerosis (ALS)
•Biogen and Ionis Pharmaceuticals: Tofersen for SOD1-ALS.
•Amylyx Pharmaceuticals: AMX0035 (Relyvrio) for broader ALS use.
•Clene Nanomedicine: CNM-Au8 nanocatalytic therapy for ALS.
Huntington’s Disease
•Wave Life Sciences: PRECISION-HD1 and HD2 trials of allele-specific oligonucleotide therapies.
•Roche and Ionis Pharmaceuticals: GENERATION HD1 trial of tominersen.
•Sangamo Therapeutics: ST-501 zinc-finger gene therapy.
RARE NEURODEGENERATIVE DISEASES
•TauRx Therapeutics: LMTX targeting tauopathies.
•Biogen and Ionis Pharmaceuticals: BIIB080 antisense therapy for tau disorders.
•Prothena: PRX005 anti-alpha-synuclein antibody for multiple system atrophy and Parkinson’s.
•Ionis Pharmaceuticals: ION582 for Angelman syndrome.
PSYCHIATRY AND BEHAVIORAL HEALTH
•Compass Pathways: NCT05624268 trial of synthetic psilocybin for treatment-resistant depression.
•MAPS Public Benefit Corporation: MDMA-assisted therapy trials for PTSD.
•Atai Life Sciences: PCN-101 (R-ketamine) for treatment-resistant depression.
•MindMed’s LSD-Based Therapy Trials: MindMed is preparing Phase 3 clinical trials for its LSD-based therapy targeting generalized anxiety disorder (GAD). The first study, involving 200 participants, is set to commence later this year, with a second study planned for early 2025.
RARE DISEASES
•Beam Therapeutics: NCT06389877 trial testing gene-editing for alpha-1 antitrypsin deficiency.
•Vertex and Moderna: NCT05668741 trial of mRNA-522 for cystic fibrosis.
•Sarepta Therapeutics: EMBARK trial testing SRP-9001 for Duchenne muscular dystrophy (DMD).
•bluebird bio: lovotibeglogene autotemcel (lovo-cel) for beta-thalassemia.
•Rocket Pharmaceuticals: Gene therapy trials for Danon Disease.
•Orchard Therapeutics: OTL-203 for mucopolysaccharidosis type I (Hurler syndrome).
AUTOIMMUNE DISEASES
•Eli Lilly: Emerald-2 trial of MORF-057 for ulcerative colitis.
•Janssen: TREMFYA (guselkumab) combination trials for ulcerative colitis.
•Pfizer: Etrasimod trials for inflammatory bowel diseases like Crohn’s disease.
•Vera Therapeutics: Origin 3 trial of atacicept for IgA nephropathy (IgAN).
GENE AND CELL THERAPIES
•CRISPR Therapeutics and Vertex Pharmaceuticals: Exa-cel gene-editing for sickle cell disease and beta-thalassemia.
•Editas Medicine: EDIT-301 for sickle cell disease using CRISPR-based approaches.
•Sana Biotechnology: SC291 allogeneic CAR-T therapy for B-cell malignancies.
•Beam Therapeutics’ BEACON Trial: Beam Therapeutics is conducting the BEACON trial, utilizing base editing technology to treat sickle cell disease. Initial results are anticipated in February 2025, potentially marking a significant advancement in gene therapy.
INFECTIOUS DISEASES
•Pfizer: Trials for next-generation pneumococcal and meningococcal vaccines.
•Moderna: Trials for mRNA-based flu and RSV vaccines.
•Gilead Sciences: Lenacapavir trials for long-acting HIV treatment.
•Sanaria’s PfSPZ-LARC2 Vaccine Trials: Sanaria is preparing for first-in-human clinical trials of PfSPZ-LARC2 Vaccine, a genetically attenuated malaria vaccine, in Seattle, Washington; Tübingen, Germany; and Burkina Faso. These trials aim to assess the vaccine’s safety and efficacy in preventing malaria.
METABOLIC DISEASES AND OBESITY
•Eli Lilly: Surpass-CVOT trial of tirzepatide (Mounjaro) for cardiovascular outcomes in diabetes.
•Eli Lilly: SURMOUNT-3 and SURMOUNT-4 trials of tirzepatide for weight management.
•Novo Nordisk: SELECT trial of semaglutide in cardiovascular outcomes for overweight/obese patients.
•Eli Lilly’s Orforglipron Trials: Eli Lilly is conducting Phase 3 trials for Orforglipron, an oral GLP-1 receptor agonist aimed at treating obesity and type 2 diabetes. This oral formulation could offer a more convenient alternative to injectable therapies, with results expected in August 2025.
•Novo Nordisk’s CagriSema Trials: Novo Nordisk is testing CagriSema, a combination of semaglutide and cagrilintide, for weight management. Despite recent underwhelming results, further trials are ongoing to assess its efficacy.
CARDIOVASCULAR DISEASE
•Novartis: Lp(a) Horizon trial testing pelacarsen to reduce cardiovascular events.
•Amgen: Olpasiran trials for reducing lipoprotein(a) and lowering cardiovascular risk.
•Esperion Therapeutics: CLEAR Outcomes trial of bempedoic acid for patients intolerant to statins.
•Verve Therapeutics: Heart-2 trial using base-editing to target PCSK9 for lifelong cholesterol reduction.
The clinical trials scheduled for early 2025 highlight a remarkable breadth of innovation aimed at improving patient outcomes and advancing therapeutic frontiers.
With the inclusion of additional promising studies, such as Sanaria’s malaria vaccine, MindMed’s anxiety therapy, and BioNTech’s oncology pipeline, the research landscape is expanding beyond traditional approaches to embrace cutting-edge technologies and multidisciplinary strategies.
The success of these initiatives could redefine therapeutic paradigms, offering novel solutions for both prevalent and rare conditions. These trials affirm the vital role of collaboration and innovation in achieving a healthier future, marking 2025 as a pivotal year in medical research.
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Author: Dr. Jean Chatellier, PhD
Partner, EVP & Managing Director
KYBORA
Email: jean@kybora.com
