Rezolute Completes upLIFT Enrollment, Targets Mid-December Data
Cash, cash equivalents and investments in marketable securities were $90.0 million as of September 30, 2026.
Rezolute, Inc. (RZLT), a late-stage ultra-rare metabolic disease company developing ersodetug for refractory hypoglycemia from hyperinsulinism, completed enrollment in its Phase 3 upLIFT study of tumor hyperinsulinism and said topline results are expected by mid-December 2026.
The single-arm, open-label trial is designed for up to 16 hospitalized participants. The company said 7 of the 8 participants enrolled in the first half of the study met the responder criterion for the primary endpoint, up from 6 of 8 reported in June. Each of those responders also achieved complete discontinuation of intravenous glucose with ersodetug. The eighth first-half participant discontinued study drug shortly after enrollment to pursue hospice care and died one week later of cancer progression; the company counts that participant as a non-responder.
Ersodetug has been well-tolerated in upLIFT thus far, with no drug-related safety findings reported, the company said.
On congenital hyperinsulinism, Rezolute said the FDA reviewed additional study data it had provided at the agency’s request and, after a pre-BLA meeting, recommended the company file a Biologics License Application for that indication. That followed a period in which Phase 3 sunRIZE data remained under FDA review and the company said it would await feedback.
As previously reported, sunRIZE, a Phase 3, multicenter, double-blind, randomized, placebo-controlled study, demonstrated glycemic improvements but did not meet its primary endpoint because reductions in hypoglycemia events by finger-stick self-monitored blood glucose were not statistically significant versus placebo. The company said consistent and clinically relevant improvements were observed across multiple continuous-glucose-monitoring hypoglycemia endpoints in both treatment arms. The open-label extension of sunRIZE is ongoing, with a high participation rate and several indicators of improved glycemic control, including a notable reduction in the use of background standard-of-care therapies.
At a Guggenheim Healthcare Spotlight Series virtual Q&A on October 29, 2026, the company discussed commercial outlook and launch strategy for ersodetug, citing a potential $5 billion combined market opportunity and a plan to submit a BLA for one or potentially both indications in 2027.
Cash, cash equivalents and investments in marketable securities were $90.0 million as of September 30, 2026, compared with $107.8 million as of June 30, 2026. Working capital was $84.37 million and total assets were $96.01 million. Accumulated deficit widened to $499.93 million from $481.44 million; stockholders’ equity was $85.95 million.
For the three months ended September 30, 2026, research and development expenses were $12.8 million, compared with $13.1 million a year earlier. The company said the decrease was primarily due to lower manufacturing costs for ersodetug and patient-affairs costs, partially offset by higher employee-related stock-based compensation. R&D included $1.5 million of share-based compensation. General and administrative expenses were $6.6 million, versus $6.7 million a year earlier, primarily due to lower professional fees, partially offset by higher stock-based compensation of $1.8 million.
Net loss was $18.5 million, or $0.18 a share, compared with a net loss of $18.2 million, or $0.18 a share, for the same period a year ago. Non-operating income, net, was $898 thousand. Total operating expenses were $19.39 million.
The company said there can be no assurance the FDA will agree that the totality of sunRIZE evidence demonstrates a clinically meaningful benefit or is sufficient to support approval of ersodetug for congenital HI.
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