Aprea, Gain, and Grace Report Quarterly Results with Clinical Milestones

Aprea Therapeutics, Gain Therapeutics, and Grace Therapeutics reported quarterly financial results and provided corporate updates, highlighting clinical progress across oncology, Parkinson's disease, and aneurysmal subarachnoid hemorrhage programs.

Aprea Therapeutics reported two partial responses in the ongoing Phase 1 dose escalation ACESOT-1051 trial of oral WEE1 inhibitor APR-1051. One uterine carcinosarcoma patient with PPP2R1A-mutation treated at the 220 mg dose level achieved a 50% reduction in target lesion size per RECIST v1.1 criteria and a significant reduction in CA-125 levels at the first imaging assessment; at the confirmatory second imaging assessment, an additional 9.5% reduction in target lesion size was observed, along with a further decline in CA-125 to 40.2 U/mL from 362 U/mL at baseline, and the patient remains on study with an ongoing partial response. An unconfirmed partial response was also observed in a second patient with PPP2R1A-mutated uterine serous carcinoma treated at the 150 mg dose level. A total of 28 patients have been treated at doses ranging from 10 mg to 300 mg once daily, with six patients achieving best overall response of stable disease. APR-1051 has been well tolerated, with the two most common adverse events being Grade 1 or 2 nausea and fatigue. Supported by an oversubscribed $30 million private placement that closed on March 31, 2026, the company is expanding enrollment in ACESOT-1051 to include at least 50 patients with uterine serous carcinoma and patients with cyclin E-overexpressing, platinum-resistant ovarian cancer. Additional clinical data from ACESOT-1051 will be provided at the ASCO 2026 Annual Meeting on May 30, 2026. Aprea reported $46.5 million in cash and cash equivalents as of March 31, 2026, with anticipated cash runway into Q1 2028.

Gain Therapeutics reported that in the Phase 1b trial of GT-02287 for Parkinson's disease, CSF GluSph decreased by an average of 81% after 90 days, and MDS-UPDRS scores remained stable over 150 days of dosing. The company expects to begin the GT-02287 Phase 2 trial in Parkinson's disease in Q3 2026, with Phase 1b results readout expected in Q4 2026. Gain also declared GT-04686 ready for IND-enabling studies in neurological disorders. For the first quarter ended March 31, 2026, Gain reported R&D expenses of $2.8 million, G&A expenses of $2.6 million, a net loss of $0.13 per share, and cash, cash equivalents and marketable securities of $16.5 million. The company continues Investigational New Drug engagement with the FDA in preparation for the GT-02287 Phase 2 clinical trial.

Grace Therapeutics announced that the FDA established April 23, 2026 as the PDUFA target date for review of the submission seeking approval for GTx-104 in the treatment of patients with aneurysmal subarachnoid hemorrhage (aSAH). Phase 3 STRIVE-ON safety trial data were presented at the 2025 Society of Vascular and Interventional Neurology Annual Meeting. The company continues pre-commercial planning in anticipation of potential FDA approval of the NDA submission. For the three months ended December 31, 2025, Grace reported a net loss of approximately $2.3 million, or $0.14 per share, compared to a net loss of $4.2 million, or $0.36 per share, for the same period in 2024. Total research and development expenses were $0.5 million, down from $2.2 million, primarily due to completion of the GTx-104 pivotal Phase 3 STRIVE-ON safety clinical trial. General and administrative expenses were $2.0 million, up from $1.5 million, due to costs for GTx-104 pre-commercial planning. As of December 31, 2025, cash and cash equivalents were $18.7 million, and the company believes its existing cash will be sufficient to sustain planned operations through at least 12 months from the date of the press release.