Rezolute

DelveInsight's analysis reveals 4+ key companies are developing 6+ therapies for congenital hyperinsulinism, with emerging treatments including CRN-04777, HM 15136, RZ358, and dasiglucagon expected to significantly impact the market.

• Rezolute, Inc. has priced an underwritten public offering of common stock and pre-funded warrants at $3.25 per share, expected to generate approximately $90 million in gross proceeds. • The offering attracted significant participation from notable investors including Federated Hermes Kaufmann Funds, Blackstone Multi-Asset Investing, and Great Point Partners, with closing anticipated on April 24, 2025. • Proceeds will support Rezolute's research and development initiatives for novel therapies targeting serious rare diseases, along with general corporate expenses and working capital needs.

• XOMA Royalty doubled its portfolio to over 120 royalty assets through five strategic transactions in 2024, strengthening its position as a biotech royalty aggregator. • The company completed two whole company acquisitions and celebrated FDA approvals for Day One's OJEMDA™ (tovorafenib) and Zevra's MIPLYFFA™ (arimoclomol), generating significant milestone payments. • Despite reporting a net loss of $13.8 million for 2024, XOMA Royalty received $46.3 million in cash receipts and maintains over $100 million in cash, positioning it for sustainable cashflow from royalties.

• Pharmaceutical companies warn in SEC filings that Trump administration's FDA staffing and budget cuts could significantly delay or halt new drug approvals and commercialization processes. • Recent layoffs of hundreds of FDA employees have sparked industry-wide concerns about disruptions to clinical trials, grant applications, and regulatory oversight activities. • Multiple biotech firms, including Xenon Pharmaceuticals and Rezolute, report that reduced FDA capacity could negatively impact their drug development timelines and business operations.

iECURE's ECUR-506, an in vivo gene insertion therapy, demonstrated a complete clinical response in an infant with neonatal-onset Ornithine Transcarbamylase (OTC) deficiency.

The FDA granted Breakthrough Therapy Designation to Rezolute's ersodetug for treating hypoglycemia due to congenital hyperinsulinism.

Precision BioSciences' PBGENE-HBV demonstrated a favorable safety profile in initial Phase 1 trial results, with no serious adverse events reported in the first cohort.

• The FDA has granted Orphan Drug Designation to Rezolute's ersodetug for treating hypoglycemia caused by tumor hyperinsulinism (HI). • Ersodetug, a fully human monoclonal antibody, targets insulin receptor over-activation, addressing both islet cell and non-islet cell tumors. • The designation provides Rezolute with seven years of market exclusivity and potential expedited approval pathways. • A Phase 3 registrational trial for ersodetug in tumor HI patients is anticipated to begin in 2025, building on demonstrated real-world benefits.

• Sidra Medicine has successfully conducted Qatar's first industry-sponsored pharmaceutical trial for pediatric congenital hyperinsulinism, marking a significant advancement in treating this rare condition. • The Phase 3 sunRIZE trial, led by Rezolute, aims to evaluate a new, safer, and non-invasive treatment option, potentially reducing the need for invasive pancreatic surgery in affected children. • The trial focuses on clinical trials that will benefit children in the Arab region, particularly those with rare diseases that lack effective treatment, offering new hope for patients and families. • Sidra Medicine was selected as one of 22 leading centers worldwide to participate in the sunRIZE Phase 3 clinical trial, highlighting its growing prominence in pediatric endocrinology.

• Rezolute is advancing ersodetug, a monoclonal antibody, in two Phase 3 trials for congenital and tumor hyperinsulinism (HI). • The sunRIZE study for congenital HI is enrolling patients outside the U.S., with U.S. enrollment expected to begin in early 2025. • A Phase 3 study for tumor HI is anticipated to commence in the first half of 2025, building on the success of the Expanded Access Program. • Topline results from the sunRIZE study are expected in the second half of 2025, and tumor HI study results are expected in the second half of 2026.