Company Interview / Alterity clears critical FDA hurdle in race to treat incurable brain disease

Key points:

FDA alignment on phase three design, endpoint and 50mg dose for MSA candidatePhase two data viewed by Stamler as robust, with substantial slowing of disease progressionPeak sales for MSA treatment estimated internally at about US$2.4 billionPartnering, M&A or licensing seen as key to funding global phase three and commercialisation

Alterity Therapeutics (ASX:ATH) is preparing for a pivotal phase three trial in multiple system atrophy (MSA), after what David Stamler describes as US Food and Drug Administration alignment on trial design, primary endpoint and a 50mg dose. Stamler states that phase two data indicate robust efficacy with around 50% slowing of disease progression and good tolerability, which in his view materially de-risks the next stage of development.

Stamler characterises MSA as a rare, highly aggressive Parkinsonian disorder, affecting speech, swallowing, fine motor skills and mobility, with patients often progressing from first symptoms to death within seven to eight years. He estimates up to 5,000 patients in the United States and suggests a similar order of magnitude in Australia. He sees the commercial opportunity as substantial, citing internal research with 100 US neurologists and projecting potential peak annual sales of about US$2.4 billion on conservative assumptions.

Funding for phase three is expected to come primarily from a partnering transaction. Stamler reports strong inbound interest from large and mid-size pharma and indicates that both M&A and licensing structures are on the table. He also highlights upside optionality in Parkinson’s disease, where he considers the lead molecule to have significant potential supported by ongoing patent strategy work.