Biotech companies are exploring new treatment approaches across autoimmune diseases, depression and complications of cancer treatment, with trials expected to report results in 2027.
Drug development has seen considerable activity in 2026, including several first-time FDA approvals. Regeneron’s Otarmeni became the first approved gene therapy for OTOF-related hearing loss, while Ionis Pharmaceuticals’ Zanvastro became the first approved treatment for Alexander disease.
Eli Lilly’s oral glucagon-like peptide-1 (GLP-1) receptor agonist orforglipron also received FDA approval for weight management in April. Meanwhile, Mineralys Therapeutics’ investigational hypertension drug lorundrostat is under FDA review, with a decision expected by December 22.
However, clinical development also brought setbacks. Novartis’ pelacarsen lowered lipoprotein(a) levels but did not significantly reduce cardiovascular events in Phase III, while a Phase III lung cancer trial of sacituzumab govitecan with pembrolizumab missed its primary endpoints. In October, Geron discontinued its Phase III IMpactMF trial of imetelstat in myelofibrosis after independent monitors determined it was unlikely to demonstrate an overall survival benefit.
With more clinical trial results expected in 2027, here are studies to follow, covering engineered CAR T cells, gene therapy, biomarker-guided treatment and antibody combinations.
1. Cartesian Therapeutics: Descartes-08 for Myasthenia Gravis
Trial: AURORA (NCT06799247) | Phase III | Expected topline: Q1 2027
Descartes-08 is an investigational chimeric antigen receptor (CAR) T-cell therapy for generalized myasthenia gravis, an autoimmune disease that causes muscle weakness.
The therapy uses a patient’s own T cells, engineered with messenger RNA (mRNA) to target cells expressing B-cell maturation antigen (BCMA). Unlike conventional CAR T-cell treatments, it is designed to be administered without prior chemotherapy.
The Phase III AURORA trial compares Descartes-08 with placebo, with the primary endpoint measuring the proportion of patients who achieve at least a three-point improvement in daily functioning at four months.
What’s next: Cartesian plans to submit a biologics license application by mid-2027 if the findings support filing.
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2. Alto Neuroscience: ALTO-300 for Major Depressive Disorder
Trial: NCT05922878 | Phase IIb | Expected topline: First half of 2027
Alto Neuroscience is studying ALTO-300, also known as agomelatine, as an add-on treatment for major depressive disorder (MDD) in patients who have not responded adequately to antidepressants.
The oral drug activates melatonin receptors and blocks a serotonin receptor involved in mood regulation. Agomelatine is already approved for depression in Europe and Australia but remains investigational in the US.
The study uses electroencephalography (EEG), which measures electrical activity in the brain, to identify patients who may be more likely to respond. Researchers are assessing changes in depression severity after six weeks among patients with the selected EEG biomarker.
What’s next: Alto expects topline results next year, which will help assess how patients with the selected biomarker respond to treatment.
3. MeiraGTx: AAV2-hAQP1 Gene Therapy for Radiation-Induced Dry Mouth
Trial: AQUAx2 (NCT05926765) | Phase II | Expected readout: Q2 2027
MeiraGTx is developing AAV2-hAQP1, an investigational gene therapy for radiation-induced xerostomia, or chronic dry mouth, following treatment for head and neck cancer.
Radiation can damage the salivary glands and reduce saliva production, making it hard to eat, speak and swallow. AAV2-hAQP1 aims to restore fluid secretion by delivering a gene encoding aquaporin-1, a protein that helps water move through cells.
The Phase II AQUAx2 trial is evaluating safety and effectiveness, including changes in patient-reported dry mouth symptoms. The FDA granted AAV2-hAQP1 Breakthrough Therapy designation in March 2026.
What’s next: Findings from the AQUAx2 trial could support a regulatory submission for AAV2-hAQP1.
4. Spyre Therapeutics: SKYLINE for Ulcerative Colitis
Trial: SKYLINE Part B (NCT07012395) | Phase II | Expected topline: Q3 2027
Spyre Therapeutics is testing whether combinations of antibodies can improve outcomes in moderate to severe ulcerative colitis, a chronic inflammatory bowel disease.
Its investigational therapies target three pathways involved in intestinal inflammation: α4β7 integrin, TL1A and IL-23.
After initially evaluating the antibodies individually, the Phase II SKYLINE trial is now testing three combinations — SPY120, SPY130 and SPY230 — alongside the individual therapies and placebo.
What’s next: The SKYLINE Part B results will help assess whether combining the antibodies provides additional benefits compared with using them individually.
5. Zura Bio: Tibulizumab for Systemic Sclerosis
Trial: TibuSURE (NCT06843239) | Phase II | Expected topline: First half of 2027
Zura Bio is developing tibulizumab, an investigational antibody designed to block two immune signaling proteins: IL-17 and B-cell activating factor (BAFF).
The treatment is being studied in systemic sclerosis, an autoimmune disease involving inflammation, tissue scarring and, in some patients, damage to internal organs.
The Phase II TibuSURE trial enrolled 91 patients with early diffuse cutaneous systemic sclerosis. Researchers are measuring changes in skin thickening after 24 weeks using the modified Rodnan Skin Score, alongside assessments of lung involvement.
What’s next: Topline results which will help guide further development of tibulizumab in systemic sclerosis.
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