FDA Grants Orphan Drug Status to SRK-015 for SMA

Discussion in 'Human Growth Hormone and Peptides' started by Roco Bama, Oct 3, 2018.

  1. Roco Bama

    Roco Bama Member

    More companies are pushing to develop myostatin inhibitors. SRK-015 is another antibody that selectively inhibits myostatin.
    Phase 1 Trial of SRK-015, Potential SMA Therapy, Completes 1st Dosing, Scholar Rock Reports

    Scholar Rock announced it has completed dosing a first group of healthy volunteers in its Phase 1 clinical study of SRK-015, a spinal muscular atrophy(SMA) treatment candidate.

    SRK-105 selectively blocks the activation of myostatin, a growth factor produced primarily in skeletal muscle cells that helps to maintain a healthy muscle mass. Excessive activation of myostatin is associated with muscle atrophy. Prior trials of compounds targeting the mature form of myostatin found that they can affect other closely related growth factors, possibly causing side effects.

    In preclinical studies, SRK-105 prevented additional atrophy in mice with muscle wasting and increased healthy animals’ muscle mass and function. The company believes SRK-105 could become the first muscle-directed treatment to reverse or prevent additional muscle atrophy in SMA patients.

    “The initiation of this Phase 1 clinical trial of SRK-015 is a momentous milestone for Scholar Rock and we are one step closer to potentially bringing the first muscle-directed therapy to patients suffering from SMA,” Nagesh Mahanthappa, president and CEO at Scholar Rock, said in a press release. The company is based in Cambridge, Massachusetts.

    The placebo-controlled, double-blind Phase 1 study is assessing the safety, tolerability and pharmacological profile of single- and multiple-ascending doses of intravenous SRK-015 in healthy adult volunteers.

    If successful, Scholar Rock plans to open a Phase 2 study in early next year to test the efficacy and safety of SRK-015 in patients with later-onset SMA.

    The therapy will be assessed either as a stand-alone therapy or in combination with an approved SMN upregulator therapy as a background standard of care.

    The U.S. Food and Drug Administration granted SRK-015 orphan drug status for the treatment of muscle atrophy SMA, and in January 2018 Scholar Rock announced $47 million in new financing for the compound’s clinical testing.

    The Phase 1 trial is continuing to enroll healthy volunteers, but more information about the trial — including test location(s) was not available.
     
    Millard Baker, master.on and TRT like this.
  2. TRT

    TRT Member

    I thought I read that there are 2 different myostatins that control muscle growth. Maybe I'm mistaken. Never read much about them. But if they can make a really effective one then I feel like you could take just that and make gains since it uncaps the amount of muscle you can put on.
     
    MindlessWork likes this.
  3. master.on

    master.on Member

    Does orphan drug means that it can't be patented?

    If so, is SRK-015 available in other countries?
     
  4. Roco Bama

    Roco Bama Member

    Read Orphan Drug Act of 1983.
     
  5. TRT

    TRT Member

    No. Spoon feed me.
     
  6. TRT

    TRT Member

    "The Orphan Drug Act of 1983 is a law passed in the United States to facilitate development of orphan drugs — drugs for rare diseases such as Huntington's disease, myoclonus, ALS, Tourette syndrome and muscular dystrophy which affect small numbers of individuals residing in the United States. "

    Easy enough.
     
    Roco Bama likes this.
  7. TRT

    TRT Member

    @Roco Bama so I'm aware of myostatin peptides and all their hype. What I wanna know is if this drug a first of it's kind to be tested on humans? If not what are some other ones?
    I don't know much about these and that's why I ask. I can you read up on them.
     
  8. Roco Bama

    Roco Bama Member

    SRK is an antibody that selectively blocks myostatin. 1 of the first of its kind to be tested on humans. Pfizer is also currently testing a monoclonal antibody that blocks myostatin.
     
  9. TRT

    TRT Member

    How do you think this will change bodybuilding if any at all. I feel like with this the natural limit will go up and could have some changes to the AAS use.
     
  10. Roco Bama

    Roco Bama Member

    this stuff will be a great stack with AAS.
     
  11. JackSmooth

    JackSmooth Member

    If myostatin is blocked wouldn’t that lead to other muscles such as the heart growing quicker and larger also. Would certainly lead to greater complications if it did being as this community has a tendency of abusing dosages.
     
  12. TRT

    TRT Member

    Your dame right they do. Haha
     
    MindlessWork and JackSmooth like this.
  13. MindlessWork

    MindlessWork Member AnabolicLab.com Supporter

    Looks promising indeed, but won’t you lose the gains once you stop the SRK?
     
    TRT likes this.
  14. TRT

    TRT Member

    I'm no expert but from what I read yes. But because of the illnees the drug is trying to cure then it would need to be taken for years straight. So the drug needs to come out of testing good for long term use.

    Wonder what kind of side effects there are to that. Can't see there being any but there always is.
     
  15. MindlessWork

    MindlessWork Member AnabolicLab.com Supporter

    Also we don’t know if there would be any negative interactions with AAS. Agreed that long term use might also have its downside.
     
    TRT likes this.
  16. Roco Bama

    Roco Bama Member

    We don't know yet