Lingdolinurad

Plus, news about Anavex, Alnylam, Rapport Therapeutics, Dianthus Therapeutics, Ultragenyx, Atom Therapeutics, Alchemab, BioMarin, Amgen, Kyowa Kirin and Bristol Myers Squibb: 🤧 Regeneron’s allergy drugs clear Phase 3 trials: The company’s allergen-blocking antibody candidates met the primary endpoint of improvement in ocular itch in two separate registrational studies in people with allergies to cats and birch. Participants in the cat study had an average 52% reduction in ocular itch, while those in the birch study had an average 51% reduction. — Ayisha Sharma 💉 Ascletis Pharma’s long-acting GLP-1 could be given quarterly: The Hong Kong biotech said a subcutaneous depot version of ASC30 had a half life of 75 days in obesity patients in a US-based Phase 1b study. There were no grade 3 or higher adverse events (AEs), and gastrointestinal AEs were mild among ASC30 recipients. Ascletis said the formulation, which is intended for maintenance of weight loss, was the most clinically advanced incretin that could be given once every three months. The company is also trialing once-monthly shot and once-daily pill forms of ASC30. — Elizabeth Cairns 🧠 Anavex releases analysis from Phase 2b/3 Alzheimer’s study: It found that early Alzheimer’s patients who lacked two genetic mutations saw their disease progress more slowly than the full trial population after one year. The post-hoc analysis comes after Anavex shared mixed data last year for its pill known as blarcamesine. Its stock $AVXL was up about 4% on Tuesday morning. — Max Gelman 💰 Alnylam to raise $500M in convertible senior notes: The company said the private offering will be for “qualified institutional buyers” and will mature on Sept. 15, 2028. Alnylam will use part of the funds to pay off older convertible notes. — Max Gelman 💵 Rapport Therapeutics is aiming to raise up to $287.5 million in a public offering. The biotech on Monday detailed promising open-label data for its epilepsy pill. — Jaimy Lee 🏦 Dianthus Therapeutics also launched a public offering the day after disclosing that its experimental antibody hit multiple endpoints in a Phase 2 trial in generalized myasthenia gravis, an increasingly popular therapeutic category for drugmakers. It’s seeking to bring in up to $172.5 million. — Jaimy Lee 🧬 Ultragenyx details two-year results for glycogen storage disease gene therapy: The rare disease drug developer said its experimental treatment cut nighttime cornstarch by 70% to 75% in two groups. The company is developing DTX401, a gene therapy for glycogen storage disease type 1a. People with the disease can’t process glycogen properly and need a continuous glucose supply, which cornstarch provides. Ultragenyx said participants maintained low levels of low blood sugar despite reductions in cornstarch intake. — Lei Lei Wu 💊 Atom Therapeutics toplines Phase 2b/3 gout trial: The company said lingdolinurad, an oral URAT1 inhibitor, hit the primary endpoint of lowering serum uric acid levels after six months. All secondary endpoints were also met. Specific data were not disclosed. — Max Gelman 💸 Alchemab raises $32M in Series A extension: The funds are expected to support the advancement of ATLX-2847 into clinical development for muscle atrophy. Alchemab has also started a first-in-human trial of a separate asset called ATLX-1282 that is partnered with Eli Lilly. The drug is being developed for amyotrophic lateral sclerosis and other neurodegenerative conditions. — Ayisha Sharma 🏷️  BioMarin to ask FDA to expand PKU drug label to teens: Over the weekend, the biotech said a Phase 3 trial showed its enzyme replacement therapy for phenylketonuria cut levels of phenylalanine in the blood by 49.7% in a study with children between the ages of 12 and 17 years old. Phenylalanine is a protein building block that people with PKU can’t break down. BioMarin’s therapy, which delivers a version of an enzyme that breaks the amino acid down, is currently approved for adults with PKU as Palynziq. BioMarin plans to ask global drug regulators to expand that indication to include adolescents. — Lei Lei Wu 🩺 Amgen, Kyowa Kirin tout long-term eczema data: In a Phase 3 study, patients who took rocatinlimab, an anti-OX40 monoclonal antibody, continued to see few side effects and sustained benefit. The companies followed patients for one year. The full data are set to be released at a future medical conference. — Max Gelman 🏛 Bristol Myers Squibb to bring its IRA fight to the Supreme Court: A company spokesperson told Endpoints News that Bristol Myers is disappointed that an appellate court rejected its challenge to Medicare drug price negotiations. It now plans to appeal to the Supreme Court. The company also praised a dissenting opinion by Third Circuit Judge Thomas Hardiman that claimed the Inflation Reduction Act “forces the companies to turn over their property to Medicare beneficiaries by threatening them with ruinous excise tax liability.” The spokesperson said the company appreciates “that the dissenting opinion recognized the merits of our arguments.”  — Nicole DeFeudis Editor’s note: An earlier version of this article had an incorrect figure for Rapport’s public offering. It plans to raise up to $287.5 million.

Atom Therapeutics\' CEO said combined efficacy and safety data position ABP-671 as a “leading candidate for best-in-class therapeutic in this area.”\n Atom Therapeutics is touting data from a phase 2b/3 study as evidence that its gout drug has the efficacy and safety profile to stand out from the competition.The Chinese biotech evaluated the oral URAT1 inhibitor, called lingdolinurad, against placebo or the approved gout drug allopurinol in a trial conducted across the U.S., Europe, Latin America and Australia. The study met all primary and secondary endpoints, Atom explained in a Sept. 8 release.When compared to the allopurinol and placebo cohorts, patients who received ABP-671 experienced a significant reduction in the relative risk of acute gout attacks, with “a maximum risk reduction of 42% within the period of 15-28 weeks,” Atom said.Not only did the study show that patients receiving ABP-671 were more likely to see their serum uric acid levels fall below 6 mg/dL than those on allopurinol, but a higher proportion of the ABP-671 cohort also saw their levels fall below 4 mg/dL.Atom said that an “increasing number of clinical consensus guidelines” now support maintaining uric acid levels at around 4-5 mg/dL, below the 6-mg/dL threshold recommended by the American College of Rheumatology for reducing gout flares and long-term joint damage.Gout can result in small lumps of urate crystals called tophi developing under the skin. Atom said ABP-671 demonstrated “good efficacy” at dissolving tophi over six months of treatment, with a “remarkable reduction in gouty stone compared to baseline levels.”The response rate of a reduction in tophus diameter by Week 28 reached 91%, the company added. Unlike some approved urate-lowering gout drugs like febuxostat and benzbromarone, the trial didn’t link ABP-671 to any cardiovascular risks or liver toxicity, Atom stressed.In a statement, Atom CEO William Dongfang Shi, Ph.D., said the combined efficacy and safety data from the study position ABP-671 as a “leading candidate for best-in-class therapeutic in this area.”“The six-month global phase 2b/3 clinical trial provided compelling evidence that ABP-671 is associated with clinically meaningful improvement in reducing the risk of acute gout attacks and promoting tophus dissolution,” he added. “These findings lay the groundwork for the design and planning of our upcoming pivotal clinical studies.”A relatively recent addition to the gout treatment arsenal was Horizon Therapeutics’ Krystexxa, which Amgen took on through its $27.8 billion acquisition of the drugmaker two years ago. However, Krystexxa hasn’t performed quite as well as expected, with $236 million of sales in the first quarter of 2025 falling $57 million shy of analyst expectations.