TACT Reviews

A non-confidential summary of all the TACT reports is detailed below however interested parties and potential funders are encouraged to contact the researcher directly to request a copy of the full TACT report.

April 2017

Tamoxifen

DMD

Dirk Fischer
University Children's Hospital, Basel

Tamoxifen in Duchenne muscular dystrophy

 

 

Rycal®

DMD

Alexia Blesius
Servier

Clinical development of Rycal® in the ambulant DMD population

 

MYOPAX

DMD

Rita Perlingeiro
University of Minnesota

 

iPS cell derivedmyogenic progenitor cells (MYOPAX) for the treatment of Muscular Dystrophies

October
2016

Ezutromid

DM1

Jon Tinsley
Summit

Utrophin modulation as a potential treatment for Becker muscular dystrophy

 

Tideglusib

DM1

Mike Snape
AMO Pharma

An inhibitor of the kinase GSK3β

 

VAL-0620

MTM1

Deborah Ramsdell
Valerion Therapeutics

Muscle-targeted enzyme replacement therapy

 



April
2016

Simvastatin

DMD

Jorge Quiroz, Solid Biosciences

Use of simvastatin as a potential treatment for Duchenne muscular dystrophy

 



MTB-1

DMD

George Mulligan
Mitobridge

MTB-1 - a selective gene regulator, for the treatment of Duchenne muscular dystrophy

 


GSK3350916A

DMD

Patrick Eidam GlaxoSmithKline
Development of the hematopoetic prostaglandin D synthase inhibitor, GSK3350916A, for the treatment of Duchenne muscular dystrophy



December
2015

Vasomera (PB1046)

DMD

Lynne Georgopolous, PhaseBio Pharmaceuticals Inc.

Development of PB1046 (Vasomera), as an adjunctive therapy for the treatment and prevention of cardiomyopathy associated with dystrophinopathies; Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD), and X-linked dilated cardiomyopathy (XL-dCMP).

 

BBRM2 (Intrathecal Azythromycin)

SMA

Dr. Gliko-Kabir
BioBlast Pharma Ltd.

BBRM2 (Intrathecal Azythromycin) for the treatment of Spinal Muscular Atrophy



March
2015

Rimeporide (EMD 87580)

DMD

Florence Porte-Thomé, EspeRare Foundation

Rimeporide (EMD 87580)- a potential disease modifying drug for Duchenne muscular dystrophy (DMD)

 

The NHE-1 (sodium-hydrogen exchanger 1) blocker KR-33028

DMD

Stefan Schäfer,
Peacock Pharma

The NHE-1 (sodium-hydrogen exchanger 1) blocker KR-33028 for the treatment of muscular dystrophies

 

AAV vector carrying TYMP gene

MNGIE

Ramon Marti,
Vall d'Hebron Research Institute (VHIR)

Gene Therapy for mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) using a new orphan drug consisting of an adeno-associated virus vector carrying the TYMP gene. Phase I/II clinical trial



October
2014

AAV8.Des.MTM1

MTM

Suyash Prasad
Audentes Therapeutics

A Phase 2 Single Arm, Open Label, Clinical Study to Evaluate the Safety and Efficacy of AAV8 Delivered Gene Therapy Delivered to Children with X-Linked Myotubular Myopathy (XLMTM)

FG-3019

DMD

Seth Porter
FibroGen

Clinical Trial of FG-3019 in patients with Duchenne muscular dystrophy (DMD)

Naproxcinod HCT3012

DMD

Gloria Vigliani Naproxcinod for the treatment of Duchenne muscular dystrophy



May
2014

TXA127

NMD

Dr Richard Franklin
Tarix Orphan LLC
TXA127 for the treatment of muscular dystrophies


AAV9 viral vector

SMA

Prof Mimoun Azzouz
University of Sheffield
Viral vector containing DNA coding for the human SMN protein; for treatment of 5q spinal muscular atrophy



November
2013

Anti-GDF8

DMD

Rare Disease Research Unit, Pfizer Inc. Anti-GDF8 antibody for treatment of Duchenne Muscular Dystrophy


iNOS blockers

IBM

Dr. Jens Schmidt
University Medical Centre Göttingen
Blockade of nitric oxide-related cell stress as potential treatment for inclusion body myositis


Nemo binding domain peptide

DMD

Dr Denis Guttridge
Ohio State University
NBD Therapy for Duchenne Muscualr Dystrophy



April
2013

SMT C1100

DMD

Jon Tinsley
Summit PLC
Utrophin modulator for the treatment of Duchenne muscular dystrophy



Wnt7a

DMD

Peter Flynn
Fate Therapeutics

Analog Protein Therapeutic for the Treatment of Muscular Dystrophy






October
2012


P2X7

DMD

Dariusz C Gorecki
University of Portsmouth

P2X7 purinoceptor as a target for pharmacotherapy of Duchenne Muscular Dystrophy






Myostin

DMD

Patricio Sepulveda Myostin Therapeutics Pty Ltd
Novel myostatin antagonist peptides to enhance muscle function





ReveraGen

DMD

Erica Reeves ReveraGen BioPharma Inc VBP15 for the treatment of DMD




Catabasis

DMD

Joanne M. Donovan
Catabasis Pharmaceuticals, Inc
CAT-1004, a novel anti-inflammatory agent for treatment of Duchenne Muscular Dystrophy



April
2012


Satisma

SMA

Fred Marin
GMP-Orphan SAS

A new drug formulation development program of sodium phenylbutyrate in SMA patients






Tamoxifen

DMD

Urs Ruegg &
Olivier Dorchies
University of Geneva

Proposal for clinical investigation of tamoxifen in
DMD boys







Biglycan

DMD

Joel Braunstein
Tivorsan Pharmaceuticals

Recombinant Biglycan for Treatment of Duchenne and Becker Muscular Dystrophy

 

Givinostat

DMD

Paolo Bettica
Italfarmaco SpA
A two part study to assess safety and tolerability, pharmacokinetics, effects on histology and on different clinical parameters of Givinostat in ambulant children with Duchenne Muscular Dystrophy



October
2011


HT-100 - Halofuginone

DMD

Marc B Blaustein MPP
Halo Therapeutics LLC

A randomised, double-blind, placebo-controlled, multiple-dose, dose-escalation study to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of HT-100 in patients with Duchenne muscular dystrophy





Naproxcinod - HCT 3012

BMD

Fabrizio Dolfi
NicOx SA

A 6-month multicenter, randomised, double-blind, placebo-controlled, Phase IIa proof of principle study of naproxcinod (HCT 3012) 750 mg bid in patients with Becker Muscular Dystrophy.





Human Embryonic Stem Cell Derived Motor Neuron Progenitors

SMA

Chris N Airriess
California Stem Cell

Human Embryonic Stem Cell Derived Motor Neuron Progenitors for the Treatment of Motor Neuron Disease.



January
2011


Tetracycline Derivatives

SMA

Dr Higgins
Paratek Pharmaceuticals

Tetracycline Derivatives as SMN2 Splicing Modifiers for the Treatment of SMA. Screen of a tetracycline library -identified a group of potential candidates. One of these will be selected as a final candidate.





GsMTx4

DMD

Dr Sachs
Rose Pharmaceuticals

Therapy for Muscular Dystrophy by Inhibition of Mechanosensitive Ion Channels.



June
2010


N-Acetyl cysteine

CMD

Dr Ferreiro
Institute of Myology

Oxigem: AntiOXIdants for a GEnetic Myopathy. The First Clinical Trial for a Congenital Muscle Disorder: N-Acetyl cysteine (NAC) Treatment for SEPN1 Related Myopathy.





P-188

DMD

Dr Symons
Phrixus Pharmaceuticals

An open-label, fixed dose, exploratory study to assess the efficacy and safety of P-188 NF on left ventricular volume changes in patients with Duchenne muscular dystrophy (DMD)





Laminin-111

DMD

Dr Hodges
Prothelia Inc

Recombinant human Laminin-111 for treatment of Duchenne Muscular Dystrophy



February
2010


lisinopril / Losartan

DMD

Dr Chris Spurney
Children's National
Medical Center

Treatment of early cardiac systolic dysfunction in Duchenne muscular dystrophy with lisinopril or Losartan: a prospective, randomized, blinded, crossover trial.






Isosorbide dinitrate plus Ibuprofen

DMD

Professor Clementi &
Dr D'Angelo
L Sacco University Hospital

A pharmacological treatment for muscular dystrophy combining NO-releasing and non steroidal anti-inflammatory drugs.





Flavocoxid

DMD

Professor Vita &
Dr Messina
University of Messina

Randomized double-blind placebo-controlled trial of flavocoxid in Duchenne muscular dystrophy.
 
17 Oct 2017