Written By: Pharmacally Medical News Desk
PeptiDream Inc., a Japanese biotech firm specializing in peptide therapeutics, has received a milestone payment from Alexion Pharmaceuticals, Inc. (part of AstraZeneca Rare Disease) following the initiation of the Phase 2 clinical trial for ALXN2420 in patients with acromegaly. This development marks a key advancement in treating the rare endocrine disorder characterized by excess growth hormone production.
The ASTERIA trial (NCT07037420) is a Phase 2, randomized, double-blind, placebo-controlled study evaluating subcutaneous ALXN2420 combined with somatostatin analogs (SSAs) in adults with acromegaly who show partial response to SSA therapy. Participants receive daily injections for 15 weeks in the primary period, followed by an open-label extension up to 52 weeks, with primary endpoint focusing on Insulin-like Growth Factor 1 (IGF-1) level reduction from baseline. Eligible patients must have confirmed acromegaly diagnosis, stable once-monthly SSA use for at least six months, and over 20% IGF-1 reduction on SSA.
This milestone underscores the progress in Alexion’s pipeline for rare diseases, with the trial enrolling about 60 participants across multiple sites and expecting completion by late 2027
ALXN2420 acts as a novel small peptide growth hormone receptor (GHR) antagonist, blocking GHR activation to lower IGF-1 levels and address both hepatic and extra-hepatic effects of elevated GH in acromegaly patients. Preclinical data demonstrated its additive efficacy with SSAs, potentially improving quality of life in patients with persistent symptoms despite normalized IGF-1. Developed via PeptiDream peptide discovery platform, ALXN2420 completed Phase 1 trials in healthy volunteers, confirming safety, tolerability, and favorable pharmacokinetics for daily subcutaneous dosing prior to Phase 2 initiation.
“We are excited to see ALXN2420 advance into Phase 2,” said Dr. Patrick C. Reid, CEO of PeptiDream. “This milestone highlights our peptide platform’s strength and successful collaborations with Amolyt Pharma and Alexion, offering hope for better acromegaly outcomes.”
PeptiDream licensed ALXN2420 (formerly AZP-3813) exclusively to Amolyt Pharma worldwide in 2021. AstraZeneca acquired Amolyt for $1.05 billion in 2024, so Alexion now drives its clinical development, manufacturing, and commercialization.
For PeptiDream, the payment highlights successful partnership outcomes from their prior agreement with Alexion, potentially unlocking further milestones tied to development progress. Acromegaly treatments remain limited, making GHR antagonists like ALXN2420 a promising option for SSA non-fully responsive patients.
References
PeptiDream Receives Milestone Payment upon Initiation of Phase 2 Clinical Trial for ALXN2420 in Patients with Acromegaly, 26 November 2025, PeptiDream, https://contents.xj-storage.jp/xcontents/45870/bf1e7456/1b98/4ab8/a6c4/a8702b2e4f11/20251126170744633s.pdf
ALXN2420 versus Placebo in Combination with Somatostatin Analogs in Participants With Acromegaly, UCH Clinical Trials, https://clinicaltrials.ucbraid.org/trial/NCT07037420
ALXN2420 versus Placebo in Combination with Somatostatin Analogs in Participants with Acromegaly (ASTERIA), ClinicalTrials.gov ID NCT07037420, https://www.clinicaltrials.gov/study/NCT07037420