Q1 Clinical trials round-up: Jan-Mar 2025

The first quarter of 2025 saw a wave of significant advancements in clinical research across oncology, neurology, dermatology, respiratory, and rare diseases.

Several biotech and biopharma companies reported promising results from late-stage trials, while others initiated first-in-human studies, marking pivotal moments in their development pipelines.

Radiopharmaceutical breakthroughs in oncology

ITM Isotope Technologies Munich SE announced compelling Phase 3 results from its COMPETE trial evaluating ITM-11 (n.c.a. 177Lu-edotreotide) in patients with Grade 1 or 2 gastroenteropancreatic neuroendocrine tumours (GEP-NETs). The therapy significantly improved progression-free survival compared to everolimus, with favourable safety outcomes. ITM plans a US regulatory submission in 2025 and continues to explore ITM-11 in other tumour types, including glioblastoma and renal cell carcinoma.

Genetic medicine and siRNA therapies in dermatology

Alys Pharmaceuticals dosed its first patient in a Phase 2a trial of ALY-101, a JAK1-targeting siRNA therapy for Alopecia Areata. The trial follows IND clearance from both the FDA and Health Canada. ALY-101, developed in collaboration with UMass Chan Medical School, is part of a broader pipeline targeting inflammatory skin diseases with long-acting, infrequent dosing regimens.

Gene therapy for cystic fibrosis

Boehringer Ingelheim, in partnership with the UK Respiratory Gene Therapy Consortium and OXB, launched the LENTICLAIR 1 Phase I/II trial of BI 3720931, a first-in-class inhaled lentiviral gene therapy for cystic fibrosis. This therapy aims to benefit patients who cannot use CFTR modulators due to genetic incompatibility or intolerance, potentially offering long-term correction of CFTR function.

Neurodegenerative disease advances

Annovis Bio initiated a pivotal Phase 3 trial of buntanetap in early Alzheimer’s disease. The study will assess both symptomatic and disease-modifying effects over 18 months. The trial builds on earlier data showing cognitive improvements and no safety concerns, with funding secured for the initial phase.

Pulmonary fibrosis and respiratory innovation

Boehringer Ingelheim also reported positive Phase 3 results from the FIBRONEER-ILD trial of nerandomilast in progressive pulmonary fibrosis. The drug met its primary endpoint of improved lung function and showed a consistent safety profile. Regulatory submissions are planned globally.

Ophthalmology and glaucoma

Qlaris Bio shared positive Phase 2 results for QLS-111, a novel therapy targeting episcleral venous pressure in glaucoma. The drug demonstrated significant intraocular pressure reductions, both as monotherapy and in combination with latanoprost, with excellent tolerability and no serious adverse events.

Immunotherapy in ovarian cancer

IMUNON presented translational data from its OVATION 2 Phase 2 study of IMNN-001, a DNA-based IL-12 immunotherapy for advanced ovarian cancer. The therapy showed dose-dependent increases in IL-12 and downstream cytokines, with a favourable safety profile and plans to initiate a Phase 3 trial.

Paediatric haemophilia A

Novo Nordisk’s FRONTIER3 trial interim results showed that 74.3% of children on once-weekly Mim8 prophylaxis experienced zero treated bleeds. The bispecific antibody demonstrated strong efficacy and was well-tolerated, with many families preferring it over previous treatments.

Antimicrobial innovation

Centauri Therapeutics selected its first clinical candidate from the ABX-01 programme targeting Gram-negative bacterial lung infections. The dual-action Alphamer molecule combines immune recruitment with direct antibacterial effects and is progressing toward Phase I trials.

Duchenne muscular dystrophy

Avidity Biosciences announced positive topline data from its Phase 1/2 EXPLORE44 trial of del-zota, an AOC therapy for DMD amenable to exon 44 skipping. The therapy showed significant increases in dystrophin and exon skipping, with reductions in creatine kinase and a favourable safety profile. A BLA submission is planned for late 2025.

Cannabis use disorder

Aelis Farma released final Phase 2B results for AEF0117, a CB1-SSi compound for cannabis use disorder. While the primary endpoint was not statistically significant in the overall population, significant reductions in cannabis use were observed in highly motivated patients, supporting further development at higher doses.

Lung cancer survival milestone

Johnson & Johnson’s MARIPOSA Phase 3 trial showed that the combination of amivantamab and lazertinib significantly improved overall survival in EGFR-mutated NSCLC compared to osimertinib. The combination also delayed symptomatic progression and reduced brain metastases, marking a potential new standard of care.

Stargardt disease gene therapy

SpliceBio dosed the first patient in its ASTRA Phase 1/2 trial of SB-007, a dual-AAV gene therapy for Stargardt disease. The therapy aims to restore full-length ABCA4 protein expression and is the first of its kind to enter clinical development for this condition.

ALS and neuroinflammation

Tiziana Life Sciences filed an IND for a Phase 2 trial of intranasal foralumab in ALS, supported by a grant from the ALS Association. The therapy targets neuroinflammation and is also being studied in multiple sclerosis and Alzheimer’s disease.

Chronic cough relief

Trevi Therapeutics reported positive Phase 2a results from the RIVER trial of Haduvio in refractory chronic cough. The therapy achieved a 57% placebo-adjusted reduction in cough frequency and showed rapid onset and strong patient-reported outcomes.

Wound healing innovation

SolasCure began a new Phase II trial of Aurase Wound Gel, a maggot-derived enzyme therapy for venous leg ulcers. The CLEANVLU2 study will assess higher concentrations of Tarumase, aiming to improve debridement and healing outcomes.

Setback in wet AMD

Opthea announced that its COAST Phase 3 trial of sozinibercept in wet AMD failed to meet its primary endpoint. The company is reviewing its development strategy and financial obligations under its funding agreement.

That’s it for 2025’s Q1 clinical trials round-up. If you have a study that could fit the bill for our next column, please send press releases to editorial@pharmaphorum.com, and check back for the Q2 round-up, coming soon.