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Asset Brief

ARG-007

AGN·Argenica Therapeutics Limited·Traumatic brain injury (preclinical stage; completed Phase 2 trial was in acute ischaemic stroke)·Preclinical (TBI); Phase 2 completed (acute ischaemic stroke)·[ TODO: fill in ]

Plain-language Brief

The trial in plain terms

The disease: Traumatic brain injury (TBI) occurs when an external force causes damage to the brain, triggering a cascade of injury processes including cell death, inflammation, and loss of blood-brain barrier integrity. It is a leading cause of death and disability globally. There are currently no approved neuroprotective drug treatments that directly limit the brain damage occurring after a traumatic injury. ARG-007 is listed by Argenica Therapeutics as a target indication for the asset, though the programme remains at preclinical stage for TBI specifically (per ASX announcements).
What the asset is trying to do: ARG-007, also referred to by Argenica as Xaranetide (per ASX announcement 2026-06-10), is a peptide therapeutic, meaning it is a short chain of amino acids designed to interact with specific biological targets. The precise molecular target of ARG-007 is not disclosed in the data available for this brief. Peptide therapeutics are generally designed to mimic or block naturally occurring protein interactions involved in disease processes such as cell death signalling. The mechanism by which ARG-007 is intended to act in TBI is .
What the trial is measuring, and why it matters: No clinical trial registration for ARG-007 in TBI has been identified in the data provided for this brief, and no primary endpoints for a TBI trial are therefore available. The completed Phase 2 trial (ACTRN12621000385922) was conducted in acute ischaemic stroke, not TBI. Primary and secondary endpoints for any planned or ongoing TBI study are . As of the announcements available, Argenica has established a Clinical Advisory Committee to advance a Phase 2b programme (per ASX announcement 2026-05-13) and has been progressing an IND (Investigational New Drug) application with the FDA (per ASX announcements 2026-05-20 and 2026-06-10), though the indication for that IND is .
Efficacy benchmarks in this setting: No approved neuroprotective pharmacotherapy specifically for acute TBI currently exists, making direct efficacy benchmarking difficult. In related acute brain injury research, therapies targeting cell death pathways have been studied extensively without regulatory approval in TBI. In acute ischaemic stroke, the thrombolytic agent alteplase (tPA) has demonstrated benefit when administered within 4.5 hours, with trials such as ECASS III reporting a modified Rankin Scale score of 0-1 at 90 days in approximately 52% of treated patients versus 45% for placebo (Hacke et al, NEJM 2008). These figures are from a different indication and a different mechanism class and are not statistically comparable to any results from ARG-007 in any setting.

Trial Design

Design summary

Phase: Preclinical (TBI); Phase 2 completed (acute ischaemic stroke)
Design type: [ TODO: fill in ] — no clinical trial registration for ARG-007 in TBI has been identified in the provided data.
Primary endpoint: [ TODO: fill in ]
Key secondary endpoints: [ TODO: fill in ]
Enrolment target: [ TODO: fill in ]
Current enrolment status: [ TODO: fill in ] — asset is at preclinical stage for TBI indication per available data.
Sites: [ TODO: fill in ]
Expected readout window: [ TODO: fill in ] — no trial start date or enrolment data is available for a TBI clinical trial at the time of this brief.

Asset Track Record

Did AGN deliver against its own guidance?

Past catalysts on this ticker, reconciled against the announcement parse pipeline. Hover any row for the parsed outcome detail.

15 July 2026otherARG007 Shows Significant Neuroprotection in Concussion Studyhitsrc ASX ↗
30 June 2026regulatory decisionWHO INN recommended status publicationoverduesrc ASX ↗
10 June 2026regulatory decisionARG-007 (Xaranetide) FDA — ARG007 (Xaranetide) Completes All Three FDA Requested Assayshit · positivesrc ASX ↗
9 June 2026otherWHO Confirms Xaranetide as INN for ARG-007hitsrc ASX ↗
7 May 2026conference presentationARG-007 Phase 2 SEANCON results at ESOC 2026 (Maastricht)hitASX ↗
1 Feb 2026regulatory decisionARG-007 EMA — Successful Applicaton for EMA Paediatric Waiverhit · positivesrc

Class Catalyst Calendar

What's landed, what's next for ARG-007 and peers

ARG-007's own upcoming catalysts alongside class-defining historical readouts and upcoming primary completion dates in the same mechanism classes. Each event moves the broader thesis for ARG-007. Showing past 6 months and next 12 months · 9 later hidden.

ConferenceAACR Annual Meeting 2026· ChicagoApr 2026
ConferenceASCO Annual Meeting 2026· ChicagoMay 2026
Today
BCAAs in Concussion 2.0
Akiva Cohen·Small Molecule Other

Trial design comparison

How ARG-007 reads against active peer trials

Same axes, same units. Discontinued and historical pivotal trials excluded; see Competitor Landscape below for the full set.

Trial	Sponsor	Class	Phase / Line	Design	Primary endpoint	Dosing	Key eligibility	n
ARG-007	Argenica Therapeutics Limited	Peptide Therapeutic	Preclinical (TBI); Phase 2 completed (acute ischaemic stroke)[ TODO: fill in ]	[ TODO: fill in ] — no clinical trial registration for ARG-007 in TBI has been identified in the provided data.	[ TODO: fill in ]	[ TODO: fill in ]	[ TODO: fill in ]	0
Ketamine in Acute Brain Injury Patients.

Competitor Landscape

Competing programmes by mechanism and phase

Read-across commentary attached per trial. Phase ordering preserved within each group.

Mechanism trade-offs· 1 classes comparedexpand ›

Mechanism	Speed to patient	Manufacturing	Best approved result	Durability	Moat
Mesenchymal Stem CellAllo MSC	Off-the-shelf	Batch (donor)	Ryoncil (GvHD only)	Transient	Low

Peptide TherapeuticASX peers ›

Curated Commentary

Third-party coverage(none curated yet)

expand ›

External analyst notes, journalism, and industry commentary cited with attribution.

No third-party coverage curated for ARG-007 yet. Broker coverage of Argenica Therapeutics Limited is thin; named analyst notes will appear here as they become available. For now, see the company's ASX announcements and the linked sponsor / class sources elsewhere on this page.

Asset Track Record

Did AGN deliver against its own guidance?

Past catalysts on this ticker, reconciled against the announcement parse pipeline. Hover any row for the parsed outcome detail.

15 July 2026otherARG007 Shows Significant Neuroprotection in Concussion Studyhitsrc ASX ↗
30 June 2026regulatory decisionWHO INN recommended status publicationoverduesrc ASX ↗
10 June 2026regulatory decisionARG-007 (Xaranetide) FDA — ARG007 (Xaranetide) Completes All Three FDA Requested Assayshit · positivesrc ASX ↗
9 June 2026otherWHO Confirms Xaranetide as INN for ARG-007hitsrc ASX ↗
7 May 2026conference presentationARG-007 Phase 2 SEANCON results at ESOC 2026 (Maastricht)hitASX ↗
1 Feb 2026regulatory decisionARG-007 EMA — Successful Applicaton for EMA Paediatric Waiverhit · positivesrc

Class Catalyst Calendar

What's landed, what's next for ARG-007 and peers

ConferenceAACR Annual Meeting 2026· ChicagoApr 2026
ConferenceASCO Annual Meeting 2026· ChicagoMay 2026
Today
BCAAs in Concussion 2.0
Akiva Cohen·Small Molecule Other

Trial design comparison

How ARG-007 reads against active peer trials

Same axes, same units. Discontinued and historical pivotal trials excluded; see Competitor Landscape below for the full set.

Trial	Sponsor	Class	Phase / Line	Design	Primary endpoint	Dosing	Key eligibility	n
ARG-007	Argenica Therapeutics Limited	Peptide Therapeutic	Preclinical (TBI); Phase 2 completed (acute ischaemic stroke)[ TODO: fill in ]	[ TODO: fill in ] — no clinical trial registration for ARG-007 in TBI has been identified in the provided data.	[ TODO: fill in ]	[ TODO: fill in ]	[ TODO: fill in ]	0
Ketamine in Acute Brain Injury Patients.

Competitor Landscape

Competing programmes by mechanism and phase

Read-across commentary attached per trial. Phase ordering preserved within each group.

Mechanism trade-offs· 1 classes comparedexpand ›

Mechanism	Speed to patient	Manufacturing	Best approved result	Durability	Moat
Mesenchymal Stem CellAllo MSC	Off-the-shelf	Batch (donor)	Ryoncil (GvHD only)	Transient	Low

Peptide TherapeuticASX peers ›

ARG-007n = 0

Argenica Therapeutics Limited·Peptide Therapeutic·Preclinical (TBI); Phase 2 completed (acute ischaemic stroke)·[ TODO: fill in ]

Design: [ TODO: fill in ] — no clinical trial registration for ARG-007 in TBI has been identified in the provided data.
Primary endpoint: [ TODO: fill in ]
Dosing: [ TODO: fill in ]
Eligibility: [ TODO: fill in ]

Ketamine in Acute Brain Injury Patients.n = —

Geert Meyfroidt, MD, PhD·Small Molecule Other·Phase 4

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Early Post-Traumatic Seizures Prevention Trial (E-PTS Trial)n = —

All India Institute of Medical Sciences, Jodhpur·Other·Phase 4

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Seizure Prevention in Traumatic Brain Injury With Levetiracetam and Lacosamiden = —

Wake Forest University Health Sciences·Small Molecule Other·Phase 4

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Effects of Recombinant Human Growth Hormone in Elderly Patients With Moderate to Severe Acute Brain Injury (GH-ABI-RCT)n = —

Anhui Provincial Hospital·Other·Phase 4

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Methylprednisolone for Moderate to Severe Traumatic Brain Injuryn = —

Zhangjiagang First People's Hospital·Small Molecule Other·Phase 4

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Buspirone and Melatonin for Depression Following Traumatic Brain Injuryn = —

Massachusetts General Hospital·Small Molecule Other·Phase 4

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

The Anaesthetic Ketamine as Treatment for Patients With Severe Acute Brain Injuryn = —

Rigshospitalet, Denmark·Small Molecule Other·Phase 4

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Study to Compare the Efficacy and Safety of NT 201 (Botulinum Toxin) With Placebo for the Treatment of Lower Limb Spasticity Caused by Stroke or Traumatic Brain Injuryn = —

Merz Pharmaceuticals GmbH·Other·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Pharmacological Management of Seizures Post Traumatic Brain Injuryn = —

Cambridge University Hospitals NHS Foundation Trust·Other·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

The NIPA Study Naloxegol Administration to Prevent Opioids Induced Gastrointestinal Motility Disturbance in Brain Injured PAtientsn = —

University Hospital, Brest·Small Molecule Other·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

PROphylaxis for Venous ThromboEmbolism in Severe Traumatic Brain Injury (PROTEST)n = —

Sunnybrook Health Sciences Centre·Other·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

ErythroPOietin Alfa to Prevent Mortality and Reduce Severe Disability in Critically Ill TRAUMA Patientsn = —

Australian and New Zealand Intensive Care Research Centre·Small Molecule Other·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Clinical Randomisation of an Anti-fibrinolytic in Symptomatic Mild Head Injury in Older Adultsn = —

London School of Hygiene and Tropical Medicine·Small Molecule Other·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Growth Hormone Replacement Therapy in Veterans With Mild Traumatic Brain Injury (mTBI) and Adult Growth Hormone Deficiency (AGHD)n = —

VA Office of Research and Development·Peptide Therapeutic·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

The Use of Tranexamic Acid in the Treatment of Symptomatic Subdural Hematoman = —

Unity Health Toronto·Small Molecule Other·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Tranexamic Acid in Traumatic Brain Injuryn = —

Assiut University·Small Molecule Other·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Quetiapine to Reduce Post Concussive Syndrome After Mild Traumatic Brain Injury (mTBI)n = —

Foundation for Advancing Veterans' Health Research·Small Molecule Other·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Tranexamic Acid to Prevent OpeRation in Chronic Subdural Hematoman = —

Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)·Small Molecule Other·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Efficacy of Nucleo CMP Forte in Traumatic Brain Injury in Pediatricsn = —

Sherief Abd-Elsalam·Other·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Traumatic Injury Clinical Trial Evaluating Tranexamic Acid in Children: An Efficacy Studyn = —

Daniel Nishijima, MD, MAS·Small Molecule Other·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Memantine as an Additive Therapy in Patients With Traumatic Brain Injuryn = —

Mansoura University·Small Molecule Other·PHASE2/PHASE3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Effects of Dexmedetomidine on Agitation in Critically Ill TBI Patientsn = —

Centre Integre Universitaire de Sante et Services Sociaux du Nord de l'ile de Montreal·Small Molecule Other·PHASE2/PHASE3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

FFP In Traumatic BRAin INjury (FIT-BRAIN) Trialn = —

Northwestern University·Other·PHASE2/PHASE3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Valproic AcId for Traumatic BRAin INjury Trialn = —

Northwestern University·Small Molecule Other·PHASE2/PHASE3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Autologous Stem Cell Study for Adult TBI (Phase 2b)n = —

The University of Texas Health Science Center, Houston·Mesenchymal Stem Cell·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Mesenchymal Stromal Cells for Traumatic Brain Injuryn = —

Fondazione IRCCS San Gerardo dei Tintori·Mesenchymal Stem Cell·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Study to Assess the Safety of Amantadine Hydrochloride (HCl) Intravenous (IV) Solution (MR-301) in Patients With Severe Traumatic Brain Injury (TBI).n = —

SHINKEI Therapeutics, Inc·Small Molecule Other·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Randomised, Double-blind, Placebo-controlled Phase 2a Pilot Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ONP-002 in Adults With Mild Traumatic Brain Injuryn = —

Oragenics, Inc.·Other·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Cannabidiol as a Treatment for PTSD and PTSD Comorbid With TBIn = —

NYU Langone Health·Small Molecule Other·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Treatment of Acute PTH With a CGRP Receptor mAb in Military Service Members and Civilians With mTBIn = —

Henry M. Jackson Foundation for the Advancement of Military Medicine·Monoclonal antibody·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Baricitinib for Moderate and Severe Traumatic Intracerebral Hemorrhage/Contusionsn = —

Tang-Du Hospital·Kinase Inhibitor·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

BCAAs in Concussion 2.0n = —

Akiva Cohen·Small Molecule Other·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Improving Neurotrauma by Depolarization Inhibition With Combination Therapy (INDICT)n = —

University of Cincinnati·Other·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Understanding and Treating Traumatic Brain Injury (TBI) Associated Photophobia With Botulinum Toxin Type A (BoNT-A)n = —

University of Miami·Other·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Autologous Adipose-Derived Mesenchymal Stem Cells for Chronic Traumatic Brain Injuryn = —

Hope Biosciences LLC·Mesenchymal Stem Cell·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Ketamine Treatment for PTSD and MDD in TBIn = —

Minneapolis Veterans Affairs Medical Center·Small Molecule Other·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Antisecretory Factor In Severe Traumatic Brain Injuryn = —

Peter Siesjö·Peptide Therapeutic·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Allopregnanolone in Chronic Complex Traumatic Brain Injuryn = —

VA Office of Research and Development·Small Molecule Other·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Effects of Intravascular Administration of Mesenchymal Stromal Cells Derived from Wharton's Jelly of the Umbilical Cord on Systemic Immunomodulation and Neuroinflammation After Traumatic Brain Injury.n = —

Assistance Publique - Hôpitaux de Paris·Mesenchymal Stem Cell·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Ketamine, SGB and Combination Treatment for TBIn = —

Northwestern University·Small Molecule Other·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Sildenafil for Microvasculopathy in Chronic TBIn = —

University of Pennsylvania·Small Molecule Other·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Inhaled Nitric Oxide for Microvascular Dysfunction in Traumatic Brain Injuryn = —

University of Pennsylvania·Small Molecule Other·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Multi-Arm Multi-Stage Adaptive Platform Trial (APT) for the Acute Treatment of Traumatic Brain Injuryn = —

University of California, San Francisco·Other·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Vitamin D3 for Moderate to Mild Traumatic Brain Injury: A Randomized Trial on Inflammation and Recovery (VIMOT)n = —

Lagos State University·Small Molecule Other·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Tranexamic Acid Therapy For The Treatment of Subdural Hematomasn = —

Baylor College of Medicine·Small Molecule Other·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Sodium Lactate in Severe TBIn = —

University of California, Los Angeles·Small Molecule Other·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Evaluating the Effect of Tranexamic Acid on the Clinical Outcomes in Patients With Traumatic Brain Injuryn = —

Mansoura University·Small Molecule Other·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Safety and Efficacy of Angiotensin (1-7) in Persons With Moderate to Severe Traumatic Brain Injuryn = —

University of Arizona·Peptide Therapeutic·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Small Molecule OtherASX peers ›

28 activeexpand ›

GM
Ketamine in Acute Brain Injury Patients.
Phase 4
Geert Meyfroidt, MD, PhD·Recruiting·Primary completion 31 Dec 2025·NCT05097261
WF
Seizure Prevention in Traumatic Brain Injury With Levetiracetam and Lacosamide
Phase 4
Wake Forest University Health Sciences·Recruiting·Primary completion 1 Apr 2027·NCT06866691
ZF
Methylprednisolone for Moderate to Severe Traumatic Brain Injury
Phase 4
Zhangjiagang First People's Hospital·Not yet recruiting·Primary completion 1 Oct 2027·NCT07180277
MG
Buspirone and Melatonin for Depression Following Traumatic Brain Injury
Phase 4
Massachusetts General Hospital·Recruiting·Primary completion 31 July 2028·NCT04400266
RD
The Anaesthetic Ketamine as Treatment for Patients With Severe Acute Brain Injury
Phase 4
Rigshospitalet, Denmark·Recruiting·Primary completion 15 Sept 2028·NCT05095857
UH
The NIPA Study Naloxegol Administration to Prevent Opioids Induced Gastrointestinal Motility Disturbance in Brain Injured PAtients
Phase 3
University Hospital, Brest·Recruiting·Primary completion 15 Mar 2026·NCT05008926
AA
ErythroPOietin Alfa to Prevent Mortality and Reduce Severe Disability in Critically Ill TRAUMA Patients
Phase 3
Australian and New Zealand Intensive Care Research Centre·Recruiting·Primary completion 31 Dec 2026·NCT04588311
LS
Clinical Randomisation of an Anti-fibrinolytic in Symptomatic Mild Head Injury in Older Adults
Phase 3
London School of Hygiene and Tropical Medicine·Recruiting·Primary completion 31 Dec 2026·NCT04521881
UH
The Use of Tranexamic Acid in the Treatment of Symptomatic Subdural Hematoma
Phase 3
Unity Health Toronto·Recruiting·Primary completion 30 Mar 2027·NCT05713630
AU
Tranexamic Acid in Traumatic Brain Injury
Phase 3
Assiut University·Recruiting·Primary completion 10 Sept 2027·NCT05323149
FF
Quetiapine to Reduce Post Concussive Syndrome After Mild Traumatic Brain Injury (mTBI)
Phase 3
Foundation for Advancing Veterans' Health Research·Recruiting·Primary completion 1 June 2028·NCT06333990
AM
Tranexamic Acid to Prevent OpeRation in Chronic Subdural Hematoma
Phase 3
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)·Recruiting·Primary completion 30 Apr 2029·NCT03582293
DN
Traumatic Injury Clinical Trial Evaluating Tranexamic Acid in Children: An Efficacy Study
Phase 3
Daniel Nishijima, MD, MAS·Not yet recruiting·Primary completion 31 Mar 2031·NCT04387305
MU
Memantine as an Additive Therapy in Patients With Traumatic Brain Injury
PHASE2/PHASE3
Mansoura University·Not yet recruiting·Primary completion 30 Aug 2025·NCT06914310
CI
Effects of Dexmedetomidine on Agitation in Critically Ill TBI Patients
PHASE2/PHASE3
Centre Integre Universitaire de Sante et Services Sociaux du Nord de l'ile de Montreal·Not yet recruiting·Primary completion 1 June 2026·NCT06620393
NU
Valproic AcId for Traumatic BRAin INjury Trial
PHASE2/PHASE3
Northwestern University·Not yet recruiting·Primary completion 1 Feb 2030·NCT07166393
S,
Study to Assess the Safety of Amantadine Hydrochloride (HCl) Intravenous (IV) Solution (MR-301) in Patients With Severe Traumatic Brain Injury (TBI).
Phase 2
SHINKEI Therapeutics, Inc·Active, not recruiting·Primary completion 31 July 2025·NCT06253923
NL
Cannabidiol as a Treatment for PTSD and PTSD Comorbid With TBI
Phase 2
NYU Langone Health·Recruiting·Primary completion 1 Mar 2026·NCT04550377
AC
BCAAs in Concussion 2.0
Phase 2
Akiva Cohen·Recruiting·Primary completion 1 Aug 2026·NCT06829498
MV
Ketamine Treatment for PTSD and MDD in TBI
Phase 2
Minneapolis Veterans Affairs Medical Center·Not yet recruiting·Primary completion 1 Dec 2026·NCT06228391
VO
Allopregnanolone in Chronic Complex Traumatic Brain Injury
Phase 2
VA Office of Research and Development·Not yet recruiting·Primary completion 1 June 2027·NCT04003285
NU
Ketamine, SGB and Combination Treatment for TBI
Phase 2
Northwestern University·Recruiting·Primary completion 30 Dec 2027·NCT06608277
UO
Sildenafil for Microvasculopathy in Chronic TBI
Phase 2
University of Pennsylvania·Recruiting·Primary completion 31 Dec 2027·NCT05782244
UO
Inhaled Nitric Oxide for Microvascular Dysfunction in Traumatic Brain Injury
Phase 2
University of Pennsylvania·ENROLLING_BY_INVITATION·Primary completion 28 Mar 2028·NCT05616910
LS
Vitamin D3 for Moderate to Mild Traumatic Brain Injury: A Randomized Trial on Inflammation and Recovery (VIMOT)
Phase 2
Lagos State University·Not yet recruiting·Primary completion 30 Dec 2028·NCT07269457
BC
Tranexamic Acid Therapy For The Treatment of Subdural Hematomas
Phase 2
Baylor College of Medicine·Not yet recruiting·Primary completion 1 Oct 2029·NCT06718751
UO
Sodium Lactate in Severe TBI
Phase 2
University of California, Los Angeles·Not yet recruiting·Primary completion 30 Dec 2035·NCT06701760
MU
Evaluating the Effect of Tranexamic Acid on the Clinical Outcomes in Patients With Traumatic Brain Injury
PHASE1/PHASE2
Mansoura University·Recruiting·Primary completion 1 Mar 2025·NCT06330935

Mesenchymal Stem CellASX peers ›

4 activeexpand ›

Class profileexpand ›

Description

Allogeneic mesenchymal stromal/stem cells expanded from donor bone marrow and cryopreserved as an off-the-shelf product. They are not thought to engraft or replace tissue; instead they act transiently through paracrine anti-inflammatory and immunomodulatory signalling before being cleared. That mechanism is distinct from CAR-T or gene therapy and is, in principle, applicable across inflammation-driven indications (graft-versus-host disease, cardiovascular, orthopaedic, autoimmune). Note that one MSC platform can spawn several distinct products: Mesoblast markets remestemcel-L (Ryoncil) for GvHD but uses a separate candidate, rexlemestrocel-L (Revascor), in cardiovascular and back-pain programmes.

Class benchmarks

One MSC product is FDA-approved — remestemcel-L (Ryoncil, Mesoblast), cleared December 2024 for paediatric steroid-refractory acute GvHD after multiple prior complete-response letters. Beyond GvHD the class has a long history of Phase 3 disappointment: large trials in chronic heart failure (Mesoblast DREAM-HF), stroke and ARDS (Athersys MultiStem) and orthopaedic disease have repeatedly missed primary endpoints or produced subgroup-only signals. The recurring pattern is a biologically plausible anti-inflammatory rationale that does not convert into a hard clinical-event benefit at scale.

Commercial track record

Off-the-shelf logistics and a benign safety profile are the commercial attractions; the chronic obstacle is demonstrating a durable, statistically robust effect on hard endpoints. Ryoncil gives the class its first commercial proof point and some manufacturing and reimbursement precedent, but each new indication is effectively a fresh efficacy bet rather than a platform read-through.

Capital intensity

Batch (allogeneic) manufacture from donor marrow scales better on cost-of-goods than autologous cell therapy, but lot-to-lot potency/consistency and cryopreservation logistics are the operational risks. No per-dose economics exist in cardiovascular because no product has reached that market.

Regulatory posture

Contested and slow. Mesoblast received multiple complete-response letters on remestemcel-L (CMC/potency-assay and efficacy-evidence concerns) before the 2024 approval. The FDA has signalled it wants a validated potency assay tying mechanism to clinical effect, and a single positive trial has generally not sufficed for the class. Expect demands for confirmatory hard-endpoint data in any new indication.

Class sources (4)

Ryoncil (remestemcel-L) prescribing information · FDA · accessed 29 May 2026
DREAM-HF chronic heart failure trial (NCT02032004) · ClinicalTrials.gov · accessed 29 May 2026
Mesoblast — investor relations and pipeline · Mesoblast · accessed 29 May 2026
Galipeau J, Sensébé L — Mesenchymal Stromal Cells: Clinical Challenges and Therapeutic Opportunities (Cell Stem Cell 2018) · Cell Stem Cell · accessed 29 May 2026

TU
Autologous Stem Cell Study for Adult TBI (Phase 2b)
Phase 2
The University of Texas Health Science Center, Houston·Active, not recruiting·Primary completion 1 July 2024·NCT02525432
FI
Mesenchymal Stromal Cells for Traumatic Brain Injury
Phase 2
Fondazione IRCCS San Gerardo dei Tintori·Recruiting·Primary completion 1 Dec 2024·NCT06163833
HB
Autologous Adipose-Derived Mesenchymal Stem Cells for Chronic Traumatic Brain Injury
Phase 2
Hope Biosciences LLC·Recruiting

OtherASX peers ›

11 activeexpand ›

AI
Early Post-Traumatic Seizures Prevention Trial (E-PTS Trial)
Phase 4
All India Institute of Medical Sciences, Jodhpur·Recruiting·Primary completion 15 Mar 2027·NCT07072624
AP
Effects of Recombinant Human Growth Hormone in Elderly Patients With Moderate to Severe Acute Brain Injury (GH-ABI-RCT)
Phase 4
Anhui Provincial Hospital·Not yet recruiting·Primary completion 30 Sept 2027·NCT07481656
MG
Study to Compare the Efficacy and Safety of NT 201 (Botulinum Toxin) With Placebo for the Treatment of Lower Limb Spasticity Caused by Stroke or Traumatic Brain Injury
Phase 3
Merz Pharmaceuticals GmbH·Active, not recruiting·Primary completion 19 June 2025·NCT03992404
CU
Pharmacological Management of Seizures Post Traumatic Brain Injury
Phase 3
Cambridge University Hospitals NHS Foundation Trust·Not yet recruiting·Primary completion 1 Mar 2026·NCT04573803
SH
PROphylaxis for Venous ThromboEmbolism in Severe Traumatic Brain Injury (PROTEST)
Phase 3
Sunnybrook Health Sciences Centre·Recruiting·Primary completion 1 Dec 2026·NCT03559114
SA
Efficacy of Nucleo CMP Forte in Traumatic Brain Injury in Pediatrics
Phase 3
Sherief Abd-Elsalam·Recruiting·Primary completion 1 Dec 2030·NCT04499755
NU
FFP In Traumatic BRAin INjury (FIT-BRAIN) Trial
PHASE2/PHASE3
Northwestern University·Recruiting·Primary completion 1 Aug 2028·NCT06062888
OI
A Randomised, Double-blind, Placebo-controlled Phase 2a Pilot Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ONP-002 in Adults With Mild Traumatic Brain Injury
Phase 2
Oragenics, Inc.·Not yet recruiting·Primary completion 1 Jan 2026·NCT06870240
UO
Improving Neurotrauma by Depolarization Inhibition With Combination Therapy (INDICT)
Phase 2
University of Cincinnati·Recruiting·Primary completion 1 Sept 2026·NCT05337618
UO
Understanding and Treating Traumatic Brain Injury (TBI) Associated Photophobia With Botulinum Toxin Type A (BoNT-A)
Phase 2
University of Miami·Recruiting·Primary completion 30 Sept 2026·NCT06293300
UO
Multi-Arm Multi-Stage Adaptive Platform Trial (APT) for the Acute Treatment of Traumatic Brain Injury
Phase 2
University of California, San Francisco·ENROLLING_BY_INVITATION·Primary completion 31 Aug 2028·NCT05826912