Research Updates

The FDA granted approval to Stealth BioTherapeutics' elamipretide (brand name Forzinity) for the treatment of Barth syndrome, a rare X-linked mitochondrial cardiomyopathy affecting primarily males. This marks a landmark regulatory milestone — the first FDA-approved therapy specifically for Barth syndrome and one of the very few approved mitochondria-targeting peptides in clinical use.

Elamipretide (SS-31) works by selectively targeting cardiolipin in the inner mitochondrial membrane, stabilising mitochondrial cristae architecture and improving electron transport chain efficiency. The approval was supported by the Phase III TAZPOWER extension trial, which demonstrated sustained improvements in skeletal muscle strength and exercise capacity over 36 weeks.

Post-marketing surveillance data for bremelanotide (Vyleesi), the FDA-approved subcutaneous peptide for hypoactive sexual desire disorder (HSDD) in premenopausal women, was compiled and analysed across multiple US healthcare systems. Real-world data broadly corroborated Phase III trial findings, with nausea remaining the most commonly reported adverse effect (occurring in roughly 40% of users) and transient blood pressure elevations noted in a subset of patients with pre-existing cardiovascular risk factors.

Prescribers reported that patient counselling on the nausea profile and on-demand dosing expectations significantly improved adherence and satisfaction outcomes. The data reinforced current label guidance recommending against use in patients with cardiovascular disease.

A pooled meta-analysis of 11 randomised and quasi-randomised trials (n = 1,847 patients) examined thymosin alpha-1 (Zadaxin) as an adjunct therapy in moderate-to-severe COVID-19. The analysis, published in a peer-reviewed infectious disease journal, found a statistically significant reduction in 28-day mortality (RR 0.71, 95% CI 0.58–0.87) and shorter ICU length of stay in the thymosin alpha-1 arms compared with standard-of-care controls.

The authors noted significant heterogeneity in dosing protocols (1.6 mg vs. 3.2 mg, twice weekly vs. daily) and cautioned that most trials were conducted in China, where Zadaxin is approved and commercially available, limiting generalisability. No serious adverse events attributable to the peptide were reported across the pooled cohort.

A Phase I dose-escalation safety trial for oral BPC-157 was registered and initiated, representing the first prospective controlled human trial for this widely researched peptide. The trial, sponsored by a Croatian academic consortium, enrolled healthy adult volunteers to evaluate pharmacokinetics, tolerability, and bioavailability of an orally administered BPC-157 formulation across three dose cohorts.

Prior to this trial, human use of BPC-157 had been exclusively anecdotal or extrapolated from an extensive animal literature. The initiation of a formal Phase I study is a significant step toward generating regulated safety data, though results are not expected until 2026 at the earliest.

Investigators published extended follow-up data from a two-year open-label continuation of an earlier MK-677 (ibutamoren) trial in older adults with GH deficiency. The analysis confirmed durable increases in IGF-1 and lean body mass but documented a clinically meaningful incidence of insulin resistance (fasting glucose elevation ≥10% from baseline in 23% of participants) and fluid retention requiring dose adjustment in 18%.

The data support prior short-term findings while reinforcing that metabolic monitoring — particularly fasting glucose and HbA1c — is essential during extended MK-677 use. The compound remains investigational and is not approved in any jurisdiction for anti-aging or body composition indications.

Researchers using transcriptomic profiling (RNA-seq) in human fibroblast and keratinocyte cell lines documented that GHK-Cu modulates expression of over 4,000 human genes — including upregulation of genes involved in antioxidant defence, DNA repair, and collagen synthesis, and downregulation of pro-inflammatory and oncogenic pathways.

While this in vitro work reinforces the pleiotropic signalling biology of GHK-Cu, the authors cautioned that gene expression changes in isolated cell lines do not directly translate to clinical outcomes. In vivo and clinical studies remain limited. The findings are consistent with earlier landmark work by Pickart et al. and extend the mechanistic rationale for topical and systemic GHK-Cu research.

Registry-based outcome analyses from Russian neurological centres where Semax (Met-Glu-His-Phe-Pro-Gly-Pro) is an approved pharmaceutical provided updated clinical data on its use in ischaemic stroke rehabilitation. Pooled registry data from 14 centres (n = 2,340 patients) reported that Semax adjunct therapy was associated with significantly improved neurological deficit scores (NIHSS) at 30 days and reduced rates of post-stroke cognitive impairment at 6 months compared with matched controls receiving standard rehabilitation alone.

This remains observational registry data rather than a randomised controlled trial, and Semax is not approved outside Russia and several former Soviet states. Independent replication in controlled Western trial settings is lacking.

Paradigm Biopharmaceuticals reported top-line Phase IIb results for intra-articular AOD-9604 (Pentosan Polysulfate Sodium — PPS) in knee osteoarthritis. The trial met its primary endpoint of pain reduction at 12 weeks (VAS score), with the 3-injection PPS group showing a statistically significant improvement over placebo (p = 0.031). Secondary endpoints of WOMAC function scores also trended favourably, though did not reach pre-specified significance thresholds.

Note: Paradigm's clinical programme primarily focuses on Pentosan Polysulfate Sodium rather than the AOD-9604 fragment peptide specifically; the two are distinct compounds that are sometimes conflated in online peptide communities. AOD-9604 itself (a lipolytic GH fragment) has no active regulated clinical programme as of 2024.