The Final Charge , Phase I/II MND Results & FDA ODD Approval Catalyst, page-961

The ALS Drug development summit is fast approaching, May 2024.
MT is attending.
Amylyx might play hide & seek, but Novartis, Sanofil, Biogen, Takeda and Abbvie are all serious players, and potential partners ( or buyers ) for PharmAust.






Biogen completed an acquisition for Reata Pharmaceuticals, a company focused on developing therapeutics that regulate cellular metabolism and inflammation in serious neurologic diseases, in Sept 2023 for USD$7.2B

An interesting case study for PharmAust - if one can imagine us completing phase 2/3 study, or perhaps after 24 weeks with stellar results, 1st half 2025.
Similar patient cohort in size and lack of therapeutic options, neuro related rare disease, ODD etc.
The transaction was completed after FDA approval for SKYCLARY, but before European approval.

BTW, USD$7.2B ~ AUD$11B, 500M SOI = $22 per share.

I know there is still plenty of water to pass under the bridge before this type of transaction becomes a possibility for PharmAust, but I reckon if we ask the 12 patients that are on the OLE study ( or about to be .. hurry up Macquarie Uni ) if MPL is working, and if success is likely, we would get an emphatic and resounding YES.

The drug in question is SKYCLARYS:

As a result of the transaction, Biogen has now acquired SKYCLARYS^® (omaveloxolone), as well as other clinical and preclinical pipeline programs.

SKYCLARYS^®, Reata Pharmaceuticals’ lead asset, was approved for the treatment of Friedreich’s ataxia (FA), a rare neuromuscular disorder, in the United States earlier this year. FA is genetic, progressive, life-shortening, debilitating, and degenerative, affecting an estimated 5,000 diagnosed patients within the United States¹. The commercial launch of SKYCLARYS^® is underway in the United States and European regulatory review is ongoing. As of the closing date, over 1,000 patient start forms for SKYCLARYS^® have been submitted in the United States.

Looking at data on therapeutic benefit of omaveloxolone of Friedreich's ataxia, looks similar to our path on ALS ( rating scores, life extension etc. ).



And an interesting insight into the negotiating process.






Exciting times ahead, for those of us patient enough to see the journey through.