"A hint in the datasuggesting potential use in combination with dystrophin restoration drugs was particularly interesting. Questions around efficacy for these treatments remains, while a number of sources suggest inflammation may play a functional role in the newly produced dystrophin not being able to penetrate the muscles effectively and lowering functional efficacy rates being observed in these patients."
Let’s hear a man on the inside said about Sarepta’s drug:
“What Antisense is doing is quite different to the Sarepta drug.”
“There (Sarepta) we had a disease modifying drug that modified protein expression in the body and changed the course of the disease,” Mr Goolsbee said.
But in a vicious circle, while the Sarepta drug may slow the progression of the disease it can’t stop the disease-created inflammation that keeps attacking the muscle.
The Antisense treatment is designed to treat the inflammation that exacerbates muscle fibre damage.
This can be managed by using cortico-steroids but these come with a whole range of side effects including excessive weight gain — precisely the wrong thing for boys who already have limited movement.
“What we want to do is add our treatment ATL1102 on top of that drug and eliminate a substantial portion of continued muscle degeneration, so whatever the period of ambulation is for that boy would be longer – years is our expectation,” Mr Goolsbee said.
Ever wonder why Sarepta's drugs are so controversial? Based on some of the info out there :
- 3 approved drugs using the same "questionable" biomarker data
- Dystrophin data without showing actual clinical benefits such as improvement on disease progression.
- Sarepta has not presented any post-approval data to confirm the drug’s benefit - FDA has repeatedly said with all three drugs that the dystrophin production data are “reasonably likely to predict clinical benefit.” Sarepta has yet to produce outcomes data to prove that benefit. And regulators still don’t know if any of them work
See the below picture, INFLAMMATION has an important impact on DMD!
Now, ATL1102 - This is the REAL DEAL!!
ATL1102 is a Phase IIb‐ready asset with demonstrated safety and efficacy data in non‐ambulatory DMD patientsATL1102 has a novel MOA to reduceinflammation in DMD patients
- Anti‐inflammatory steroids, dystrophinrestoration technologies and gene therapies are ONLY being tested in ambulantpatients
- ATL1102’snovel mechanism in targeting CD49d suggests potential for drug to be used in combinationwith steroid anti‐ inflammatory agents
- ATL1102 has potential to be synergisticwith other projects in development reducing competitive pressure of otherpotential product launches
- ATL1102 has a mechanism appears effective across all genetic subtypes of DMD ‐ a key differentiator among the exon skipping therapies which increases the addressable patient pool
