Ann: Additional Phase 1 data suggest MPL may inhibit MND, page-317

A new deal done in the ALS space , for a preclinical drug, for up to USD$580M (~ A$900m ) between Takeda and AcuraStem. for upfront and milestone payments.

The post from Michael is below , and the press release from AcuraStem.

From the release

"AcuraStem’s therapeutic strategy for PIKFYVE focuses on addressing neurodegeneration by expelling toxic protein aggregates and protecting healthy neuronal function."
“We aim to develop transformative treatments for some of society’s most debilitating neurological diseases including ALS. Whilst recent treatment advances have brought new hope to some patients, there remains a significant unmet need,” said Sarah Sheikh, BM BCh, MSc, MRCP, Head, Neuroscience Therapeutic Area Unit at Takeda. “We believe AS-202 has the potential to address this unmet need through its unique dual mechanism of action, which addresses TDP-43 aggregation and improves TDP-43 function, the pathological hallmark of ALS and other TDP-43 proteinopathies including certain forms of dementia."

Bring on the results for MPL trial. The value of what we have if we demonstrate stable disease and that MPL can likely clear the TDP-43 aggregates through induced autophagy, is mind boggling.

And some folks are selling ... go figure !