The latest news related to research, industry and clinical trials. You can also visit our Research News section for more detailed research stories.
Myonexus receives another FDA designation
17 May 2018
The US Food and Drug Administration (FDA) has granted Rare Paediatric Disease designation to MYO-101, for the treatment of limb girdle muscular dystrophy 2E. Dependant on clinical data, this designation could help speed up the approval process of MYO-101.
Myonexus, who recently received orphan drug designation, plans to initiate a Phase 1/2a trial to evaluate MYO-101 later on this year.
For more information, please read Myonexus’ press release.
Novartis completes acquisition of AveXis
16 May 2018
Novartis has announced it has successfully acquired AveXis – a gene therapy company developing a treatment for spinal muscular atrophy (SMA). AveXis’ lead candidate, AVXS-101, is currently being evaluated in multiple SMA clinical trials and has shown promising results.
Novartis already has a strong gene therapy programme – this acquisition will bring together the expertise from both companies which will help to advance SMA research.
Find out more, please read Novartis’ press release.
Sarepta and Myonexus form partnership
04 May 2018
Sarepta Therapeutics has announced it is partnering with Myonexus Therapeutics – a gene therapy company developing potential treatments for limb girdle muscular dystrophy (LGMD). Myonexus’ pipeline includes five different LGMD gene therapies, three of which are currently being evaluated in clinical trials.
Sarepta is developing potential therapies – including gene therapies – for Duchenne muscular dystrophy. This new partnership between Sarepta and Myonexus brings together expertise that will help to advance LGMD gene therapy research.
For more information, please read Sarepta’s press release.
FSHD drug receives fast track designation
01 May 2018
Acceleron Pharma has announced the US Food and Drug Administration (FDA) has granted Fast Track Designation to ACE-083, for the treatment of facioscapulohumeral muscular dystrophy (FSHD). This status will help speed up the development and regulatory review process of the drug.
ACE-083 is currently being evaluated in a Phase 2 trial in individuals with FSHD.
For more information, please read Acceleron’s press release.
Update on SMA gene therapy trials
26 April 2018
AveXis has announced updates on its AVXS-101 gene therapy trials. The first participant has been dosed in SPRINT, a trial for young babies with spinal muscular atrophy (SMA) who are not yet showing symptoms. This will assess whether AVSX-101 has a greater benefit if given earlier in life. The trial continues to recruit at sites worldwide.
PTC announces preliminary data from SMA trial
25 April 2018
PTC Therapeutics has released clinical data from part one of FIREFISH; a two-part study in babies with spinal muscular atrophy (SMA) type 1. The study is investigating RG7916, an oral drug that increases the levels of the SMN protein. Preliminary data shows RG7916 was well tolerated and it increased the levels of the SMN protein in a dose-dependent manner. In addition, no babies have required a tracheostomy or permanent ventilation and all babies have retained their ability to swallow.
Recruitment for the second part of FIREFISH is ongoing; it will evaluate the efficacy of RG7916.
For more information please read PTC’s press release.