/Company Directory

/

Asset Brief

NYR-BI03

NYR·Nyrada·Traumatic brain injury (TBI)·Phase 1 (completed); Phase 2 planned·NCT06894862·Primary completion: 24 July 2025

Plain-language Brief

The trial in plain terms

The disease: Traumatic brain injury (TBI) occurs when an external mechanical force damages the brain, triggering a cascade of secondary injury processes including inflammation, cell death, and oedema (swelling) that can worsen outcomes beyond the initial physical harm. TBI ranges in severity from concussion to severe brain injury and is a leading cause of death and disability worldwide. There are currently no approved pharmacological treatments that specifically target the secondary injury cascade following TBI. Management remains largely supportive.
What the asset is trying to do: NYR-BI03 is described by Nyrada as a neuroprotective agent administered by infusion (per Nyrada public disclosures). The asset is classified as a small molecule. The specific molecular target of NYR-BI03 has not been publicly disclosed. Beyond the infusion route and neuroprotective intent, the mechanism of action has not been described in detail in available public sources; further detail is .
What the trial is measuring, and why it matters: The completed Phase 1 first-in-human (FIH) study (NCT06894862) was designed to assess safety, tolerability, and pharmacokinetics (PK, meaning how the drug moves through and is processed by the body) of NYR-BI03 in healthy participants, using single ascending dose (SAD) and multiple ascending dose (MAD) cohorts (per ClinicalTrials.gov NCT06894862). Safety and tolerability endpoints capture whether the drug causes adverse effects at increasing dose levels, while PK data establish the dosing parameters needed to design an efficacy study. The design in healthy volunteers is a standard first step before testing in patients with TBI.
Efficacy benchmarks in this setting: No pharmacological therapy has received regulatory approval specifically for the treatment of secondary injury in TBI in major markets including the United States or Australia, meaning there is no approved-drug benchmark for this indication. In the broader neuroprotection space, multiple agents have been evaluated in Phase 2 and Phase 3 TBI trials without demonstrating statistically significant benefit on functional outcome measures such as the Glasgow Outcome Scale Extended (GOSE); for example, a Phase 3 trial of progesterone in TBI (the SYNAPSE trial) did not meet its primary endpoint of improved outcome at six months (Wright et al, New England Journal of Medicine, 2014). Early-stage FIH data from NYR-BI03 in healthy volunteers are not statistically comparable to efficacy figures from trials conducted in TBI patients.
Commercial context: No pharmacological treatment for the secondary injury phase of TBI has been approved by the US Food and Drug Administration (FDA) or the Australian Therapeutic Goods Administration (TGA), leaving an area with no currently approved competitors (per publicly available FDA and TGA approval databases). NYR-BI03 is one of at least two pipeline assets disclosed by Nyrada; the company's other programmes include Xolatryp (a cardioprotection asset with a Phase IIa trial recently initiated, per ASX announcement 2026-03-18) and a cholesterol-lowering programme. No licensing deals, partnership terms, or manufacturing cost data specific to NYR-BI03 have been publicly disclosed. Nyrada received an A$2.455 million R&D tax incentive rebate in 2026 (per ASX announcement 2026-05-28).

Sources for this brief (5)

A Study of the Safety, Tolerability, and Pharmacokinetics of NYR-BI03 in Healthy Participants (NCT06894862) · ClinicalTrials.gov · accessed 15 June 2026
Progesterone for the Treatment of Traumatic Brain Injury (SYNAPSE Trial) · New England Journal of Medicine (Wright et al, 2014) · accessed 15 June 2026
Nyrada ASX Announcements Page · Australian Securities Exchange (ASX) · accessed 15 June 2026
Nyrada Investor Presentation (June 2026) · Nyrada Inc / ASX · accessed 15 June 2026
Traumatic Brain Injury: Hope After a Decade of Failed Clinical Trials · The Lancet Neurology · accessed 15 June 2026

Trial Design

Design summary

Phase: Phase 1 (completed); Phase 2 planned
Design type: Randomised, placebo-controlled, single ascending dose (SAD) and multiple ascending dose (MAD), first-in-human study in healthy participants (per ClinicalTrials.gov NCT06894862).
Primary endpoint: Safety and tolerability of NYR-BI03 (per ClinicalTrials.gov NCT06894862).
Key secondary endpoints: Pharmacokinetics (PK) of NYR-BI03 (per ClinicalTrials.gov NCT06894862)
Enrolment target: 48 participants (per ClinicalTrials.gov NCT06894862)
Current enrolment status: Study completed (per ClinicalTrials.gov NCT06894862, primary completion 2025-07-24)
Sites: [ TODO: fill in ]
Expected readout window: The Phase 1 study primary completion date was 2025-07-24 (per ClinicalTrials.gov NCT06894862); Nyrada has indicated it is moving to Phase 2, but no Phase 2 trial registration or readout timeline has been publicly disclosed as of the most recent available announcements.

Asset Track Record

Did NYR deliver against its own guidance?

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

30 June 2026data readoutNYR-BI03 Phase 1 brain injury final resultsoverdue
15 June 2026data readoutPROTECT-MI Phase IIa Clinical Trial Updatehitsrc ASX ↗
19 May 2026otherPause in Tradinghitsrc ASX ↗
19 May 2026regulatory decisionXolatryp USPTO — Nyrada Receives Notice of Allowance for Xolatryp US Patenthit · positivesrc ASX ↗
5 July 2026commercial milestonePROTECT-MI Phase IIa first patient dosinghit · positivesrc ASX ↗
18 Mar 2026data readoutXolatryp Phase 2a PROTECT-MI first patient dosedhitsrc ASX ↗
22 Apr 2026data readoutFirst Site Activated in Nyradas PROTECT-MI Phase IIa Trialhitsrc ASX ↗
31 Mar 2026commercial milestonePROTECT-MI Phase IIa site initiation visits(delayed 1×)hitsrc

Class Catalyst Calendar

What's landed, what's next for NYR-BI03 and peers

NYR-BI03'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 NYR-BI03. Showing past 6 months and next 12 months · 12 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 NYR-BI03 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
NYR-BI03	Nyrada	Small Molecule Other	Phase 1 (completed); Phase 2 planned[ TODO: fill in ]	Randomised, placebo-controlled, single ascending dose (SAD) and multiple ascending dose (MAD), first-in-human study in healthy participants (per ClinicalTrials.gov NCT06894862).	Safety and tolerability of NYR-BI03 (per ClinicalTrials.gov NCT06894862).	[ TODO: fill in ]	48 participants (per ClinicalTrials.gov NCT06894862)	48

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

Small Molecule OtherASX 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 NYR-BI03 yet. Broker coverage of Nyrada 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 NYR deliver against its own guidance?

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

30 June 2026data readoutNYR-BI03 Phase 1 brain injury final resultsoverdue
15 June 2026data readoutPROTECT-MI Phase IIa Clinical Trial Updatehitsrc ASX ↗
19 May 2026otherPause in Tradinghitsrc ASX ↗
19 May 2026regulatory decisionXolatryp USPTO — Nyrada Receives Notice of Allowance for Xolatryp US Patenthit · positivesrc ASX ↗
5 July 2026commercial milestonePROTECT-MI Phase IIa first patient dosinghit · positivesrc ASX ↗
18 Mar 2026data readoutXolatryp Phase 2a PROTECT-MI first patient dosedhitsrc ASX ↗
22 Apr 2026data readoutFirst Site Activated in Nyradas PROTECT-MI Phase IIa Trialhitsrc ASX ↗
31 Mar 2026commercial milestonePROTECT-MI Phase IIa site initiation visits(delayed 1×)hitsrc

Class Catalyst Calendar

What's landed, what's next for NYR-BI03 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 NYR-BI03 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
NYR-BI03	Nyrada	Small Molecule Other	Phase 1 (completed); Phase 2 planned[ TODO: fill in ]	Randomised, placebo-controlled, single ascending dose (SAD) and multiple ascending dose (MAD), first-in-human study in healthy participants (per ClinicalTrials.gov NCT06894862).	Safety and tolerability of NYR-BI03 (per ClinicalTrials.gov NCT06894862).	[ TODO: fill in ]	48 participants (per ClinicalTrials.gov NCT06894862)	48

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

Small Molecule OtherASX peers ›

NYR-BI03n = 48

Nyrada·Small Molecule Other·Phase 1 (completed); Phase 2 planned·[ TODO: fill in ]

Design: Randomised, placebo-controlled, single ascending dose (SAD) and multiple ascending dose (MAD), first-in-human study in healthy participants (per ClinicalTrials.gov NCT06894862).
Primary endpoint: Safety and tolerability of NYR-BI03 (per ClinicalTrials.gov NCT06894862).
Dosing: [ TODO: fill in ]
Eligibility: 48 participants (per ClinicalTrials.gov NCT06894862)

Ketamine in Acute Brain Injury Patients.n = —

Geert Meyfroidt, MD, PhD·Small Molecule 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·Small Molecule 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·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·Small Molecule Other·Phase 3

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

Role of Non-Selective Beta-Adrenergic Blocker in Severe TBIn = —

Tanta University·Small Molecule 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: —

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·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·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: —

Efficacy of Early Continuous Infusion of HSS on the Neurological Outcome at 6 Months in TBI Patients.n = —

Nantes University Hospital·Other·Phase 3

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

Hyperbaric Oxygen Treatment for Veterans With Traumatic Brain Injuryn = —

University of South Florida·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: —

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·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: —

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·Small Molecule Other·PHASE1/PHASE2

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

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
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
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
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
TU
Role of Non-Selective Beta-Adrenergic Blocker in Severe TBI
Phase 3
Tanta University·Not yet recruiting·Primary completion 20 Sept 2025·NCT06870370
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
VO
Growth Hormone Replacement Therapy in Veterans With Mild Traumatic Brain Injury (mTBI) and Adult Growth Hormone Deficiency (AGHD)
Phase 3
VA Office of Research and Development·Recruiting·Primary completion 28 Feb 2027·NCT04867317
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
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
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
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
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
UO
Safety and Efficacy of Angiotensin (1-7) in Persons With Moderate to Severe Traumatic Brain Injury
PHASE1/PHASE2
University of Arizona·Recruiting·Primary completion 1 Sept 2027·NCT06282965

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 ›

12 activeexpand ›

RD
The Anaesthetic Ketamine as Treatment for Patients With Severe Acute Brain Injury
Phase 4
Rigshospitalet, Denmark·Recruiting·Primary completion 15 Sept 2028·NCT05095857
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
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
NU
Efficacy of Early Continuous Infusion of HSS on the Neurological Outcome at 6 Months in TBI Patients.
Phase 3
Nantes University Hospital·Recruiting·Primary completion 1 June 2028·NCT07319208
UO
Hyperbaric Oxygen Treatment for Veterans With Traumatic Brain Injury
Phase 3
University of South Florida·Recruiting·Primary completion 15 Aug 2028·NCT06581003
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
NU
Ketamine, SGB and Combination Treatment for TBI
Phase 2
Northwestern University·Recruiting·Primary completion 30 Dec 2027·NCT06608277
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