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RZLT Stock Up as FDA Lifts Clinical Holds on Rare Disease Study

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Rezolute, Inc. (RZLT - Free Report) announced that the FDA has removed the partial clinical holds on the phase III study evaluating its lead pipeline candidate RZ358 (ersodetug) for the treatment of hypoglycemia caused by congenital hyperinsulinism (HI), an ultra-rare pediatric genetic disorder.

Shares of the company were up 22.1% on Sept. 9 following the announcement of the news.

The double-blind, placebo-controlled phase III sunRIZE study is designed to evaluate the safety and efficacy of RZ358 in patients with congenital HI. Participants aged between three months to 45 years will be eligible for enrollment in the study.

RZLT is gearing up to begin the start-up activities for the above mentioned study in the United States. Enrollment of participants in the United States is expected to begin in early 2025, with top-line data from the sunRIZE study anticipated in the second half of 2025.

Year to date, shares of Rezolute have skyrocketed 435% against the industry’s decline of 2.8%.

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Development Activities for RZLT’s RZ358

In December 2023, the company initiated the pivotal phase III sunRIZE study evaluating RZ358 in participants with cHI, the most common cause of recurrent hypoglycemia in children.

However, the FDA’s Office of Cardiology, Hematology, Endocrinology and Nephrology placed partial clinical holds as preclinical data evaluating RZ358 in Sprague Dawley rats demonstrated liver toxicity.

Per management, the FDA has now come to the conclusion that the liver toxicity observed in Sprague Dawley rats is strain-specific and not otherwise relevant to humans.

Previously, the phase IIb RIZE study investigated the safety, tolerability, pharmacokinetics and glycemic efficacy of RZ358 in patients with congenital HI whose hypoglycemia was not adequately controlled on standard-of-care therapies. Data from the study showed that treatment with RZ358 led to highly significant improvements in hypoglycemia levels in the given patient population.

Rezolute started the phase III sunRIZE study following the success of the phase II RIZE study.

Last month, the FDA cleared Rezolute’s investigational new drug (IND) application for RZ358 for treating hypoglycemia in patients with tumor HI. Enrollment of patients in this phase III study is expected to begin in the first half of 2025.

In the absence of a marketed product currently, the successful development of RZ358, along with other pipeline candidates, remains the key focus for Rezolute.

Zacks Rank & Stocks to Consider

Rezolute currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are Illumina, Inc. (ILMN - Free Report) , Krystal Biotech, Inc. (KRYS - Free Report) and Fulcrum Therapeutics, Inc. (FULC - Free Report) , each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for Illumina’s 2024 earnings per share have moved up from $1.18 to $3.62. Earnings per share estimates for 2025 have improved from $2.93 to $4.50. Year to date, shares of ILMN have lost 12%.

ILMN’s earnings beat estimates in each of the trailing four quarters, the average surprise being 463.46%.

In the past 60 days, estimates for Krystal Biotech’s 2024 earnings per share have increased from $1.98 to $2.38. Earnings per share estimates for 2025 have improved from $4.33 to $7.31. Year to date, shares of KRYS have risen 51.3%.

KRYS’ earnings beat estimates in three of the trailing four quarters while missing on the remaining occasion, the average surprise being 45.95%.

In the past 60 days, estimates for Fulcrum Therapeutics’ 2024 loss per share have narrowed from $1.24 to 48 cents. Loss per share estimates for 2025 have narrowed from $1.71 to $1.51. Year to date, shares of FULC have jumped 27.9%.

FULC’s earnings beat estimates in each of the trailing four quarters, the average surprise being 393.18%.

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