Sydnexis and Centessa Advance Innovative Small Molecules with Positive Phase 3 and Phase 2a Data, Demonstrating Best-in-Class Potential in Myopia and Narcolepsy

Significantly Slows Myopia Progression Over Time! Results of Innovative Small Molecule Phase 3 Trial Announced

Sydnexis recently announced positive results from the phase 3 STAR study of its investigational low-dose atropine formulation SYD-101 (0.01%) for slowing the progression of myopia in children. According to the press release, STAR is one of the largest global clinical programs for childhood myopia completed to date, with the trial enrolling 847 children aged 3–14 years at treatment initiation. In the overall population, SYD-101 met the primary efficacy endpoint at 36 months:Compared with the placebo group, the proportion of subjects reaching a confirmed myopia progression of -0.75D was lower in the SYD-101 group (139 cases in the placebo group vs 111 cases in the SYD-101 group; p=0.0226). The treatment effect was evident from the 12th month and persisted until the 36th month., and more significant benefits were observed in subjects who were younger, had a history of myopia progression, or started medication earlier in the disease. The therapy was well-tolerated, with no unexpected drug-related adverse events reported.

SYD-101 is a proprietary 0.01% atropine formulation developed by Sydnexis based on enhanced ocular delivery properties and room temperature stability, aiming to optimize tolerability, stability, and clinical performance. The therapy demonstrated higher ocular tissue permeability than other formulations in preclinical animal models and can be stably stored at room temperature for up to three years.Nearly neutral pH, which may help enhance ocular safety and comfort.

Showcasing "Best-in-Class" Potential! Small Molecule to Enter Pivotal Registration Study

Centessa Pharmaceuticals recently announced the latest progress of its OX2R agonist development program, including initial dosing cohort data from the ongoing CRYSTAL-1 study for ORX750 in subjects with Type 1 Narcolepsy (NT1), Type 2 Narcolepsy (NT2), and Idiopathic Hypersomnia (IH) in Phase 2a, as well as Phase 1 study data for ORX142 in healthy volunteers. In the initial dose cohort of NT1 subjects, the study utilized once-daily dosing with a 2-week randomized crossover design. Results showed that ORX750 at doses of 1.0 mg and 1.5 mg demonstrated statistically significant, clinically meaningful, and dose-dependent improvements compared to placebo in the Maintenance of Wakefulness Test (MWT).Especially in the 1.5 mg dose group (n=6), the average sleep onset latency (MSL) of patients improved by more than 20 minutes from baseline at week 2 (p=0.0026), with half of the subjects showing an improvement in sleep onset latency of over 30 minutes. ORX750 demonstrates the potential of a "best-in-class" therapy.Currently, research is advancing dose escalation, including once-daily and fractionated dosing.

Meanwhile, based on data as of October 3, 2025, a total of 89 healthy volunteers have participated in the Phase 1 clinical study of ORX142. The results show that ORX142 has a rapid onset of action, differentiated pharmacokinetic characteristics, and demonstrated good tolerability at all tested doses. The company stated,The Phase 2a CRYSTAL-1 study for ORX750 is still ongoing, with plans to initiate a registrational study in the first quarter of 2026; the Phase 1 study for ORX142 is also proceeding smoothly and is expected to enter the patient study phase in the first quarter of 2026.

Nearly $200 Million Series A Financing! Newcomer to Advance Small Molecules into Global Late-Stage Clinical Development

Braveheart Bio Announces Exit from Stealth Mode and Completion of $185 Million Series A FinancingBraveheart Bio today announced its exit from stealth mode and the completion of a $185 million Series A financing round. This round was participated in by several seasoned life science investment institutions, including Andreessen Horowitz (a16z Bio + Health), Forbion, OrbiMed, Enavate Sciences, and Frazier Life Sciences. The company will use the funds obtained to advance the global development and commercialization layout of its core projects.

The company's leading small molecule candidate drug, BHB-1893, is a selective cardiac myosin inhibitor designed to improve cardiac pumping and filling functions in patients with hypertrophic cardiomyopathy (HCM). The pathological characteristics of HCM include myocardial thickening and gradual decline in heart function due to excessive myocardial contraction.BHB-1893 has been extensively studied in clinical trials, including a Phase 2 dose-finding study in symptomatic obstructive HCM (oHCM), a Phase 2 study in non-obstructive HCM (nHCM), multiple clinical pharmacology studies (including a bridging study in Australia), and an ongoing Phase 3 study in oHCM in China.PublicThe company plans to initiate the global late-stage clinical development of BHB-1893 in 2026.

References:

[1] Sydnexis Announces Topline Pivotal Data from Phase 3 STAR Trial of SYD-101 for Patients with Pediatric Progressive Myopia Presented at AMCP Nexus 2025. Retrieved November 5, 2025 from https://sydnexis.com/sydnexis-announces-topline-pivotal-data-from-phase-3-star-trial-of-syd-101-for-patients-with-pediatric-progressive-myopia-presented-at-amcp-nexus-2025/

[2] Centessa Pharmaceuticals Reports Financial Results for the Third Quarter of 2025 and Provides Update on Potential Best-in-Class Orexin Receptor 2 (OX2R) Agonist Program. Retrieved November 5, 2025 from https://investors.centessa.com/news-releases/news-release-details/centessa-pharmaceuticals-reports-financial-results-third-0

[3] Braveheart Bio Launches with $185M Series A Aiming to Transform Care of Hypertrophic Cardiomyopathy. Retrieved November 5, 2025 from https://www.prnewswire.com/news-releases/braveheart-bio-launches-with-185m-series-a-aiming-to-transform-care-of-hypertrophic-cardiomyopathy-302604764.html

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