RZLT 最新10-K变化

相对上期新增的文字 · 来源:10-K · 2026-09-24

• On December 11, 2025, we announced that the sunRIZE study did not meet its primary endpoint (hypoglycemia events by finger stick self-monitored blood glucose “SMBG”) or its key secondary endpoint (time in hypoglycemia by continuous glucose monitoring “CGM”) at the defined Week 24 evaluable window. A…

• While we believe the pronounced placebo/study effect confounded the results, with a particular impact on the primary endpoint of hypoglycemia events by SMBG due to measurement bias associated with infrequent measurement, the totality of the data further supports previous clinical evidence that ersod…

• sunRIZE topline data as well as pre-specified and post-hoc data updates were shared by oral presentation at the Pediatric Endocrine Society Annual Meeting held on May 1, 2026, including CGM-based outcomes that demonstrated significant and consistent improvements in glycemic control in ersodetug trea…

• ●Average daily percent time in hypoglycemia by CGM: clinically relevant and nominally statistically significant reductions of >50% (Full Analysis Set [“FAS”]) and ~60-80% (Per Protocol Set [“PPS”]), compared to placebo across multiple timepoints

• ●Average weekly hypoglycemia events by CGM: clinically relevant and nominally statistically significant reductions of ~50-65% (FAS) and ~50-80% (PPS), compared to placebo across multiple timepoints

相对上期删除的文字 · 来源:10-K · 2025-09-17

• We completed enrollment in May 2025 of a pivotal Phase 3 clinical study (the “sunRIZE” study) of ersodetug for the treatment of hypoglycemia in participants with congenital HI, an ultra-rare pediatric genetic disorder characterized by excessive production of insulin by the pancreas. The study was to…

• The sunRIZE study is a global, randomized, double-blind, placebo-controlled, parallel arm evaluation of ersodetug in participants 3 months of age and older with congenital HI who are not adequately responding to standard of care medical therapies. Specifically, the study is evaluating the safety and…

• On July 14, 2025, we presented “Preliminary Patient Demographics and Baseline Characteristics From a Phase 3 Study (sunRIZE) of Ersodetug for Hypoglycemia Due to Congenital Hyperinsulinism: Trial in Progress” at the Annual Meeting of the Endocrine Society (“ENDO”). Comparable to what we witnessed in…

• Congenital HI is the most common cause of recurrent and persistent hypoglycemia in children. Individuals with congenital HI typically present with signs or symptoms of hypoglycemia shortly after birth. Hypoglycemia can result in significant brain injury and death if not recognized and managed approp…

• standard of care treatments are suboptimal. The current treatments used by physicians include glucagon, diazoxide, somatostatin analogues and pancreatectomy. We estimate that in the U.S. alone the addressable market for congenital HI is more than 1,500 individuals.

相对上期新增的文字 · 来源:10-K · 2026-09-24

• The supplemental information provided to the FDA to support our belief in the effectiveness of ersodetug in the treatment of congenital HI might not result in a viable path forward.

• As discussed in this Annual Report, on March 17, 2026, we met with the FDA to discuss the results of the sunRIZE trial and were asked to submit comprehensive analysis datasets and summary outcomes for the Agency’s independent evaluation. There can be no guarantee that after it analyzes the supplemen…

• There is no guarantee that the FDA will not consider the results of the sunRIZE trial when it considers additional regulatory steps related to ersodetug and its treatment of other conditions.

• As discussed in this Annual Report, we have previously asked the FDA whether it will consider the results of the sunRIZE trial when considering additional regulatory steps related to ersodetug, including whether the results of the sunRIZE trial will impact the FDA’s assessment of any results that we…

• studying, marketing or monetizing any future potential applications of ersodetug, we may be required to make significant operational changes.

相对上期删除的文字 · 来源:10-K · 2025-09-17

• Before obtaining approval from the government authorities or professional bodies with authority to grant regulatory approval for our drug candidates in a particular country, such as the EMA, the FDA and analogous authorities in other jurisdictions outside of the United States (“Regulatory Authoritie…

• manufacturing requirements; or seize or detain products, refuse to permit the import or export of products, or require us to initiate a product recall.

• The results of preclinical studies and initial clinical trials of our product candidates do not necessarily predict the results of later-stage clinical trials. Product candidates in later stages of clinical trials may fail to show the desired safety and efficacy despite having progressed through ini…

• We have a history of losses and may not achieve profitability in the future. We will need substantial additional capital to fund our operations. If we fail to obtain additional capital, we will be unable to sustain operations.

• We incurred net losses of $74.4 million and $68.5 million for the fiscal years ended June 30, 2025 and 2024, respectively. As of June 30, 2025, we had an accumulated deficit of $403.9 million. Cash used in our operating activities amounted to $69.1 million and $57.4 million for the fiscal years ende…

相对上期新增的文字 · 来源:10-K · 2026-09-24

• Our priorities going into the second half of 2026 and first half of 2027 are to: (i) achieve alignment with the FDA on the path forward in congenital HI following the completion of the sunRIZE study, (ii) complete enrollment and announce topline data for the registrational Phase 3 upLIFT study in tu…

• Our focus as a Company is advancing ersodetug as a potential treatment for refractory hypoglycemia caused by all forms of HI, specifically in two Phase 3 clinical studies for congenital HI and tumor HI. In December 2025, we announced topline results from the Phase 3 sunRIZE study of ersodetug in pat…

• forward for ersodetug in congenital HI, and no specific timeline has been established for completion of this review. See Item 1A of this Annual Report for the related risks.

• The upLIFT study in tumor HI is currently enrolling in the U.S. and Europe. At a meeting held with FDA on August 19, 2025, the Agency agreed to modifications to the design of the study including removing the need to conduct a double-blind randomized placebo-controlled trial. The truncated study will…

• We have not generated any meaningful revenues since our inception in March 2010. Over the last several years, we have conducted private placements and public offerings to raise additional capital, conducted pre-clinical and clinical trials, and conducted other research and development activities on …

相对上期删除的文字 · 来源:10-K · 2025-09-17

• Our priorities going into the second half of 2025 and first half of 2026 are to execute across our two Phase 3 clinical trials. Our goals include (i) complete the sunRIZE study (as defined below) to enable topline data in December 2025, (ii) continue enrollment in the registrational tumor HI study, …

• Our focus as a Company is advancing ersodetug as a potential treatment for all forms of HI, specifically in two Phase 3 clinical studies for congenital HI and tumor HI. To that end, we have completed enrollment in the pivotal Phase 3 sunRIZE clinical study of ersodetug, which is a randomized, double…

• The upLIFT study in tumor HI is currently enrolling in the U.S. and Europe. At a meeting held with FDA on August 19, 2025, the agency agreed to modifications to the design of the study including removing the need to conduct a double-blind randomized placebo-controlled trial. The truncated study will…

• single-arm open-label portion of the upLIFT study. Topline results from the study are anticipated to be available in the second half of 2026.

• Appointment of Chief Commercial Officer. On August 18, 2025, the Board of Directors approved the appointment of Sunil Karnawat to serve as our Chief Commercial Officer. In connection with the appointment, we extended Mr. Karnawat an employment offer letter (the “Offer Letter”). The Offer Letter prov…