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Were any Phase 3 clinical trials conducted for Memoblast and what were the endpoints and outcomes?
Executive summary
Available reporting shows Mesoblast has conducted multiple Phase 3 trials of its cell‑therapy candidates (including rexlemestrocel‑L/revascor/rexlemestrocel‑L variants) and at least one Phase 3 trial was used to support regulatory action for remestemcel‑L (Ryoncil) in graft‑versus‑host disease; specific trial names, primary endpoints and detailed outcomes are described in the cited coverage below (e.g., DREAM‑HF published Phase 3 results; MSB‑GVHD001 uncontrolled Phase 3 reported 70% ORR and 30% CR at 28 days) [1] [2] [3]. Coverage also documents ongoing and planned Phase 3 programs—rexlemestrocel‑L for chronic low back pain (300‑patient pivotal trial) and new pivotal trials of Ryoncil in adults—though full endpoint lists and complete outcomes are variably reported across sources [4] [5] [6] [7].
1. Mesoblast has run multiple Phase 3 trials — the portfolio and publicity
Mesoblast’s public materials and industry reporting list a portfolio of Phase 3 product candidates and describe completed and ongoing Phase 3 trials across indications including chronic heart failure (Revascor/rexlemestrocel‑L), chronic low back pain (rexlemestrocel‑L), and acute steroid‑refractory graft‑versus‑host disease (remestemcel‑L / Ryoncil) [8] [4] [3]. The company’s DREAM‑HF Phase 3 trial in heart failure had results published in a major cardiology journal, and Mesoblast has promoted Phase 3 activity across multiple programs [1] [3].
2. DREAM‑HF: a Phase 3 with published outcomes in heart failure
The DREAM‑HF Phase 3 trial of Mesoblast’s cell therapy for chronic heart failure reported that a single intra‑myocardial intervention with rexlemestrocel‑L reduced a composite of cardiovascular death, myocardial infarction or stroke over a mean follow‑up of about 30 months; these Phase 3 results were publicly announced and published, per clinical coverage [1]. That publication is cited in industry press as evidence of a positive Phase 3 readout for that program [1].
3. MSB‑GVHD001 and Ryoncil: Phase 3 data used in regulatory filings
Reporting indicates Mesoblast relied on an uncontrolled Phase 3 trial, MSB‑GVHD001, to support regulatory submissions for remestemcel‑L (Ryoncil) in pediatric steroid‑refractory acute GVHD; that trial reportedly showed about 70% overall response rate (ORR) and 30% complete response (CR) at 28 days in the uncontrolled dataset [2]. Follow‑on reporting and regulatory interactions led to further planned randomized pivotal trials in adults conducted by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) [2] [6].
4. Ongoing and planned pivotal trials — back pain and adult GVHD programs
Mesoblast began enrollment in a pivotal Phase 3 trial of rexlemestrocel‑L for chronic low back pain due to inflammatory degenerative disc disease, a 300‑patient study intended to confirm durable pain reduction observed previously [4] [5]. Separately, Mesoblast and the NIH‑funded BMT CTN planned a pivotal randomized Phase 3 adding Ryoncil to ruxolitinib in adults with severe steroid‑refractory aGvHD, with enrollment targeted in 2026 per press items [6] [7].
5. What were the endpoints and what outcomes are reported?
Available reporting gives primary or composite endpoints in some trials but not a uniform, detailed endpoint list across programs. For DREAM‑HF the reported outcome was a reduction in a composite of cardiovascular death, MI or stroke over ~30 months [1]. For MSB‑GVHD001 the reported efficacy metrics were ORR and CR at 28 days (70% ORR; 30% CR reported by industry analysis) [2]. For the chronic low back pain Phase 3 Mesoblast describes evaluation of durable pain reduction and safety over 24 months in investigator and site materials, but specific prespecified primary endpoint wording or statistical results are not fully detailed in the available press coverage [5] [4].
6. Limitations, disagreements and regulatory context
Some trial reports were uncontrolled (MSB‑GVHD001) which drew FDA requests for randomized data in prior interactions; Mesoblast later planned randomized adult trials to address that regulatorily‑driven gap [2] [6]. Media and company releases emphasize positive signals; independent peer review is explicit for DREAM‑HF via journal publication [1], but for other trials the publicly available summaries are from company or trade press and may omit full protocol and statistical detail [5] [4] [8]. The ISCT committee commentary frames the FDA approval of remestemcel‑L as a notable development in MSC therapeutics, reflecting both clinical interest and ongoing debate about evidentiary standards for cell therapies [9].
7. Takeaway and what’s not found in current reporting
In sum: yes — Mesoblast has conducted several Phase 3 trials; key reported endpoints include composite cardiovascular events (DREAM‑HF) and short‑term ORR/CR in GVHD (MSB‑GVHD001), and Mesoblast is running/initiating additional pivotal trials in low back pain and adult aGvHD [1] [2] [4] [6]. Available sources do not mention full, verbatim primary endpoint definitions or complete statistical outcome tables for every Phase 3 trial (not found in current reporting), nor do they include full regulatory review documents in these excerpts; for those, clinicaltrials.gov protocol entries and peer‑reviewed publications should be consulted [10].