Health Check: Percheron ‘closes the book’ on failed childhood rare disease program 

Percheron Therapeutics says it will abandon its lead drug candidate altogether, following December’s Duchenne muscular dystrophy trial flop.

It's the end – and nighty night – for Percheron's lead drug program. Pic via Getty Images

• Percheron has detailed why its Duchenne muscular dystrophy trial flopped – and why it needs to find a new drug candidate

• Imugene’s multiple oncology and inflammation programs aren’t helping its valuation

• Impedimed and Micro-X raise funds and Alterity is on the hunt.

 

“In the spirit of transparency”, Percheron Therapeutics (ASX:PER) has outlined the gory details as to why its phase II Duchenne muscular dystrophy (DMD) trial failed so badly.

CEO Dr James Garner says at the time of the December 18 trial bombshell that knocked 85% off the value of Percheron shares, the company only had limited top-line data to go by.

Now with 95% of the data analysed, the company is confident of drawing more definitive conclusions.

The upshot? The company is definitively “closing the book” on its lead – and sole – drug candidate ATL-1102 (avicursen).

The company already has terminated the DMD trial.

“One way or other, we don’t think ATL-1102 can remain the centerpiece of the story put forward to investors,” Garner told investors in a webinar this morning.

In summary, the trial failed to detect any meaningful difference between the boys actively treated with ATL-1102, compared with the placebo cohort.

On some measures of the key endpoint, the Performance of Upper Limb (PUL) test, the placebo worked better.

Management posits the placebo group may have had a longer duration of treatments with steroids – the flawed standard of care – or that the dose was too low.

Or perhaps the PUL test wasn’t sensitive enough.

But these factors alone can’t explain the failure of the trial results, which flew in the face of earlier trial results.

Management also points out that at least two other non-Percheron DMD programs flopped, unexpectedly, at a late stage.

DMD is a devastating genetic disease affecting boys, which means they can’t produce enough dystrophy for muscle growth.

Eventually they become wheelchair bound, with a life expectancy as early as in their 20s.

Today’s update comes ahead of a March 4 shareholder meeting board brought on by dissident shareholders, who want to remove Dr Garner and chairman Dr Charmaine Gittleson.

With clinical trials, less can be more

Percheron’s fate shows the hazards of focusing on one clinical program, but the share valuation of immune-ocology house Immutep (ASX:IMM) shows that multiple trials might not impress investors, either.

Immutep's investor prezzo released yesterday doesn’t have any market-sensitive revelations, but it highlights the company’s slate of no fewer than four clinical programs in mid-to-advanced stage.

These programs are either partnered or off Immutep's own bat.

The most progressed is a phase III trial for non-small cell lung cancer, in collaboration with big pharma MSD.

Aiming for 750 patients across 150 sites in 25 countries, the study road-tests Immutep’s candidate, Efti, in conjunction with the checkpoint inhibitor Keytruda and chemotherapy.

Efti targets the Lag-3 protein which regulates immune response.

Readouts from the study are expected in “2025 and beyond”.

Immutep has other phase II studies for lung cancer and head neck and shoulder cancer.

Using a different mechanism of action, other studies are on the go for soft-tissue sarcoma, psoriasis and ulcerative colitis.

If even one of the key programs were a winner, Immutep would hit the jackpot.

But the company’s current market cap stands at $470 million, less $149 million of cash on hand (enough to last until calendar 2026).

The shares have lost around 7% year to date. This attests to the sector's perverse trend of valuations falling as trials become more advanced – and more expensive to run.

Raising funds is a capital idea

When shares surge on the back of good news, a capital raising follows just as night follows day (or until Donald Trump declares otherwise).

Following last month’s triumphant results for its muscular system atrophy (MSA) trial, Alterity Therapeutics (ASX:ATH) has asked for a trading halt until Monday, pending a raising.

On January 30 Alterity shares more than doubled after posting “statistically significant and meaningful” phase II study results for its study into muscular system atrophy, a Parkinson’s-like disorder.

The company is angling for fast-track US Food and Drug Administration approval for the ‘orphan’ disease.

Portable X-ray device developer Micro-X (ASX:MX1) today unveiled a “strategic equity investment” from Billion Prima, a Malaysian baggage, parcel and vehicle scanning specialist.

The first stanza involves Billion Prima subscribing for $2.4 million of Micro-X shares 9 cents per share, a 15% premium – not, not discount – to the last traded price of 7.8c per share.

In total, the compact delivers “aggregate cash” of $5.6 million over time.

The company has been riding high since its December 4 announcement of US$8.2 million development contracts with a US agency.

This is to develop a full-body portable computed tomography (CT) scanner over two years.

Arovella Therapeutics (ASX:ALA) promises more news on a $20 million placement announced in January.

Meanwhile, device maker ImpediMed (ASX:IPD) prefers debt over equity, today unveiling a US$15 million, five-year facility with the healthcare-focused SWK Funding LLC.

Impedimed chair Christine Emmanuel-Donnelly says the deal will enable funding flexibility, without diluting existing shareholders. The shares have lost one third of their value over the past 12 months.

However, the facility also includes attached warrants which might convert to shares.

The funds will support Impedimed’s ongoing rollout of  Sozo, a device for measuring lymphodema – swelling of the limbs – in cancer patients.

The Sozos replace a current sophisticated tech known as a tape measure.

Originally published as Health Check: Percheron ‘closes the book’ on failed childhood rare disease program