Health Check: Alterity angles for fast-track FDA approval after positive trial results tackling Parkinson’s-like disorder

Alterity plans to seek US FDA fast-track approval after posting positive phase II results for multiple system atrophy treatment.

Alterity's multiple system atrophy treatment trial results have been dubbed "incredibly compelling". Pic: Getty Images

• Alterity shares double after the company claims “statistically significant and meaningful” phase II study results

• EBR Systems points to an increasingly user-friendly US Food & Drug Administration

• Hydrix shares have been on a romp, but the company’s still worth … not much

Following “positive” top-line phase II trial results, neurodegenerative disease house Alterity Therapeutics (ASX:ATH) will approach the US Food & Drug Administration (FDA) for fast-track approval of its treatment for multiple system atrophy (MSA).

Alterity shares soared up to 112% this morning after the company said the 77-patient, placebo-controlled study in early-stage MSA patients achieved “statistical significance”, with up to 48% showing slowing of the debilitating disease.

MSA is a so-called Parkinsonian disorder, marked by similar gait problems, shuffling, tremors and lack of bladder and bowel control.

In the US there are only around 50,000 sufferers, compared with more than one million Parkinson’s patients. The FDA classes the incurable MSA as an orphan disease.

Alterity’s lead compound ATH-434 targets excess iron in the brain, which leads to neurons ‘clumping’ and losing their ability to communicate.

ATH-434 acts as a “chaperone” which takes the excess iron and deposits it in less harmful parts of the body.

With the trial, the patient improvement was measured by an accepted scale known as UMSARS, which assesses the ability of patients to undergo daily activities.

On the UMSARS measure, 48% of patients showed a “slowing of clinical progression” on a 50-milligram dose at week 52, relative to placebo.

Somewhat oddly, the 75mg dose showed a 62% slowing at week 26, but only a 29% response at week 52.

Yep – sometimes less is more.

"There are no definitive answers ," says Alterity chief Dr David Stamler. "That's a question obviously we are very interested in continuing to explore."

He says achieving statistical significance on the UMSARS score was "extremely meaningful, because it assesses the functional areas affected in MSA."

More pertinently, the FDA wants a good UMSARS score before it will mull approval.

Alterity also used key biomarkers to measure iron stabilisation in MSA-affected regions of the brain.

The results suggest reduced iron accumulation with both the 50mg and 75mg doses, with “preservation of brain volume” also evident.

“We are thrilled that ATH-434 has demonstrated significant slowing of clinical progression and an excellent safety profile in this rare, rapidly progressive disease,” Stamler says.

“Based on the strength of the , we look forward to engaging with the FDA as quickly as possible to discuss the path forward for accelerating  the development of ATH-434,  given the tremendous unmet need for treating MSA.”

The trial's lead investigator, professor Daniel Claassen from Vanderbilt University Medical Centre, dubs the results as "incredibly compelling".

ATH-434 could also be relevant for other ‘orphan’ neurological diseases such as Friedreich’s ataxia as well as the big prizes of Parkinson’s disease and Alzheimer’s disease.

EBR says relax, the FDA will be OK

Today’s reading of the tea leaves as to what the Trump Administration might or might not do with healthcare comes courtesy of EBR Systems (ASX:EBR), which is awaiting FDA approval for its novel leadless pacemaker device.

EBR chief John McCutcheon notes the FDA has become more transparent and customer friendly under successive presidencies, including Trump 1.0.

“Under Obama, Trump and Biden the FDA has been really consistent, it doesn’t seem to ebb and flow with the changes in administration,” he says.

“What will be interesting is that (health secretary nominee) RFK Junior is anti vaxx and anti pharma, but none of his rhetoric has been about medical technology.

“I don’t anticipate any negative effect on the FDA side."

As for reimbursement, Trump 1.0 was favourably disposed to the medical device industry and there was bi-partisan Obama-Trump support.

“Going by history I don’t expect anything and our guys expect if anything Trump might be more pro-industry on the reimbursement side.”

There’s still a chance RFK won’t get the top job, having been roughed up in the first Senate confirmatory hearing (albeit by mainly Democrat senators).

Not to mention the undiplomatic character assessment from former diplomat cousin Caroline.

Whatever the case, EBR looks to have approval in the bag and will know for sure on or before April 13.

Circle that one in the diary, folks.

Hydrix emerges from penny-dreadful penury

Quiet Achiever Award goes to Hydrix (ASX:HYD) , with its shares soaring up to 130% since last Thursday’s announcement of a European contract win.

The Melbourne-based Hydrix designs and develops medical devices and invests in disruptive cardiac-related med techs.

Its subsidiary Hydrix Services has entered a $2.8m contract extension with European medical device company Paul Hartmann AG (Hartmann).

While details are scarce, it’s part of a multi-stage product development program, with the revenue recognised by the end of 2025.

It’s relatively small beer but put in context Hydrix reported December quarter revenue of $2.7 million, 5% lower year on year and $5.6 million in the December half (down 6.7%).

The company also disclosed sales orders of $4.3m for the quarter, up 84%, with $40m of  “anticipated sales pertaining to future stages of programs across 15 clients.”

Hydrix had a quarterly cash operating loss of $390,000 and a $2.09m net loss for the half, with December end cash on hand of $1.1m.

After today's pull-back, Hydrix bears a paltry $5.7m market valuation.

At Stockhead, we tell it as it is. While EBR Systems is a Stockhead advertiser, the company  did not sponsor this article

Originally published as Health Check: Alterity angles for fast-track FDA approval after positive trial results tackling Parkinson’s-like disorder