Interesting recent article (28 April 2022)... I had forgot the...

Interesting recent article (28 April 2022)... I had forgot the trail was done in Australia - the next set is expanded (two dose volumes and placebo) and in multiple countries!

https://www.valuewalk.com/the-next-stage-of-drug-development-how-orphan-drugs-are-giving-drugmakers-new-life/

"If the trial results are sufficiently positive, they could allow the company to seek approval in the EU. The Australian study found that, after six months of treatment with ATL1102, the boys saw a mean improvement in the function and strength of their upper limbs compared to the baseline measured before they started receiving the drug.

"This was an exciting outcome for us," Antisense Therapeutics CEO Mark Diamond said in an interview. "Usually in DMD, we would see the boys' upper limb function and strength decline even when they are being treated with corticosteroids."

He explained that slowing the decline of the loss of function of the upper limbs is the main goal in the clinical development of an effective treatment for non-ambulant DMD boys.

"We were also very encouraged by MRI data, which showed that the fat levels in the boys' muscles had stabilized," Diamond said. "Increasing levels of fat in the muscle are a sign of disease progression where muscle cells die and are replaced by fat cells. In fact, their lean muscle mass had increased. We don't believe this has ever been seen in Duchenne's boys taking corticosteroids.""

FUNDING (any extra helps) - Australia provided ANP funding (Tax funding in April 2022) but the US can also provide as well.

"Some agencies like the U.S. Food and Drug Administration give grants and tax credits to drug companies to support development of orphan drugs. Orphan drug status also offers extended market exclusivity.The FDA will also offer companies developing drugs for rare pediatric diseases such as DMD "Rare Pediatric Disease Designation," which entitles these companies to apply for a Pediatric Review Voucher (PRV). The company can use the PRV to secure accelerated approval or sell it to another drug developer that may wish to use it to accelerate approval of their own program. PRVs can trade for more than $100 million.Antisense Therapeutics has been awarded Rare Pediatric Disease Designation for ATL1102 in DMD."