clinical trial

Corcept Therapeutics presented Phase 3 ROSELLA trial data at ESMO 2025, showing relacorilant plus nab-paclitaxel significantly improved progression-free and overall survival in platinum-resistant ovarian cancer patients, including those progressing on PARP inhibitors. The combination also demonstrated a favorable safety profile. Corcept is expanding the Phase 2 BELLA trial to include platinum-sensitive ovarian and endometrial cancer arms, with initial results expected in late 2026. The FDA has granted relacorilant priority review for platinum-resistant ovarian cancer with a PDUFA date of July 11, 2026.

Regeneron’s DB-OTO gene therapy showed significant hearing improvements in children with OTOF-related hearing loss in the CHORD trial. 11 of 12 participants demonstrated clinically meaningful hearing gains, with 3 achieving normal hearing. The trial met its primary endpoint, and improvements were durable up to 72 weeks. DB-OTO was generally well-tolerated. Regeneron plans to file for U.S. regulatory approval later in 2025, pending FDA discussions. DB-OTO holds Orphan Drug and other FDA designations.

C4 Therapeutics announced positive Phase 1 results for cemsidomide in relapsed/refractory multiple myeloma. The drug demonstrated a 50% overall response rate at the highest dose, a favorable safety profile with no treatment discontinuations, and promising immunomodulatory effects. The company plans to initiate Phase 2 trials in Q1 2026 for fourth-line therapy and Phase 1b trials in Q2 2026 for second-line therapy in combination with BCMA BiTE, pursuing two potential accelerated approval pathways. The trial included 72 heavily pre-treated patients.

The SURPASS-PEDS Phase 3 trial evaluated Mounjaro (tirzepatide) in pediatric patients (10-17) with type 2 diabetes. Mounjaro significantly reduced A1C and BMI compared to placebo after 30 weeks, improvements sustained through 52 weeks. 86.1% of subjects on 10mg Mounjaro achieved an A1C target ≤6.5%. The safety profile was consistent with adult studies. Lilly seeks regulatory approval to expand Mounjaro’s label for this younger population, addressing a critical unmet need given the rise of type 2 diabetes in children.

Sensorion reported positive H1 2025 results, highlighting progress in gene therapies. The Audiogene Phase 1/2 trial for SENS-501 (OTOF-GT) showed a positive safety profile and early hearing improvement signs. Enrollment in SENS-401’s Phase 2a Cisplatin-Induced Ototoxicity study is complete, with topline data expected in H2 2025. GJB2-GT is on track for a Q1 2026 CTA filing. The company’s cash position of €57.1m funds operations into Q3 2026. R&D expenses increased slightly, reflecting intensified clinical trial activities. Net loss was -€16.0 million.

Kyverna Therapeutics will present interim Phase 2 KYSA-6 trial results for KYV-101 in myasthenia gravis at the AANEM meeting in October 2025. Data from six patients with up to 9 months follow-up will be shared. KYV-101, a CD19 CAR T-cell therapy, aims for durable remission after a single dose. The study has been upgraded to a registrational Phase 2/3 trial, with Phase 3 initiation planned by year-end 2025. The presentation by Dr. Srikanth Muppidi will be on October 29th.

Monopar Therapeutics will present long-term data on ALXN1840 for Wilson disease at the ANA Annual Meeting on September 14-15, 2025. The pooled analysis of clinical trials (n=255 efficacy, n=266 safety) with a median 2.6-year treatment duration showed sustained neurological and psychiatric improvements. Patients switching from standard of care to ALXN1840 also demonstrated improvement. The drug exhibited a favorable safety profile. ALXN1840 offers a novel mechanism of action by directly binding copper, potentially preventing its accumulation.

HCW Biologics (HCWB) reported mixed Q2 2025 results. A $5M equity raise helped shore up finances and debt was extinguished. However, revenue plummeted to $6,550, and the Wugen License Agreement was suspended. R&D spending decreased while G&A expenses increased. Despite reducing its net loss, the company expresses doubt about its ability to continue without additional funding. They are seeking new licensing deals and clinical trials are planned. HCWB regained Nasdaq compliance but remains under Panel Monitor.

Hemogenyx Pharmaceuticals (LSE:HEMO, OTC:HOPHF) has successfully treated the third patient in its Phase I clinical trial of HG-CT-1, a CAR-T cell therapy for relapsed/refractory acute myeloid leukemia (R/R AML), completing the first adult dose cohort. The FDA granted special permission for the treatment. The first two patients remain alive at six and three months post-treatment. Hemogenyx plans to initiate a second adult dose cohort and a pediatric trial arm, evaluating safety and anti-leukemic activity.

Apogee Therapeutics will announce pivotal 16-week data from its Phase 2 APEX trial for APG777, a novel inflation drug. The data, expected on July 7, 2025, could reveal differentiated efficacy and new dosing regimens for conditions like atopic dermatitis, asthma, and COPD. The company will host a conference call to discuss the findings.