Health Check: Percheron board addresses shareholders after failed phase 2b trial

Percheron’s board has written a letter to shareholders following failure of its phase 2b trial of lead candidate avicursen into non-ambulant boys with Duchenne muscular dystrophy.

The Percheron board has spoken out following the failed phase 2b trial. Pic via Getty Images

• Percheron Therapeutics board addresses shareholders following failed phase 2b trial 

• EBR's US FDA manufacturing pre-approval inspection scheduled to start January 14 as share price rockets

• Regis Healthcare inks a deal to a acquire the business and assets of BodeWell Community Care

The Percheron Therapeutics (ASX:PER) board of directors has written a letter to shareholders following failure of its phase 2b trial of lead candidate avicursen (ATL-1102) into non-ambulant (unable to walk unassisted) boys with Duchenne muscular dystrophy (DMD).

Percheron's share price fell almost 90% on December 18 after announcing the trial failed to achieve its primary endpoint, which measured upper limb function at week 25 of treatment, compared with placebo.

"This has understandably come as an enormous disappointment for all our shareholders, as indeed it has to all those of us who work in the company on your behalf," the board noted in the letter.

"We write to you collectively as a board to outline some of the implications for Percheron and to explain how we plan to rebuild and restore the company following this setback."

The company said it would for now proceed on three fronts with its first priority to better understand results of the avicursen trial and to see if there were convincing reasons to continue development in DMD.

"Companies in our position must have the courage to recognise futility when it occurs, and our assumption at this stage is that the drug will not move forward in DMD," the board said.

"However, we owe it to all those who have invested their time, money, energy, and hope in avicursen for DMD to make sure that we do not abandon it prematurely."

Percheron expects to receive further data from the trial and plan to complete its analyses by the end of Q1 CY25.

"To manage expectations, we should be clear that not all trial results are entirely explicable – sometimes drugs simply don’t work – but we think it is important to learn everything we can so that we are able to make rational and informed decisions about the program’s future, and to benefit future research in DMD," the board explained.

Secondly, the board revealed it would undertake a broad strategic review of the company’s pipeline to more comprehensively evaluate alternative sources of value within the current assets.

"Avicursen has shown evidence of activity in a range of other conditions, and we have always been clear that, while DMD was the lead indication for the drug, it was not the only use case," the board said.

"However, if we are to pursue an alternative indication, we will need to be convinced that the negative result in DMD does not prejudice the chances of success in another illness."

Meanwhile, Percheron said it had plans to make a definitive decision about the future of atesidorsen (ATL1103) in CY25 and it was likely it would need to diversify its pipeline and add one or more new programs to its portfolio.

"We have already begun exploring opportunities that may provide a faster and more secure path to restoring shareholder value and we have initiated discussions with several potential partners."

The company noted that recent capital raises enabled "choices in respect of what Percheron may become" and the board had an open mind.

"In the meantime, we have taken aggressive measures to conserve the company’s cash.

"Our most substantial cost has been the clinical trial itself, and we are moving to finalise its termination as quickly as possible.

"We are also making substantial reductions in our operating expenditure."

The board added that analysis by the Biotechnology Innovation Organization calculates the probability that a drug entering phase I human trials will become a marketed product at 7.9%.

"We are not the first company whose drug candidate has failed to meet hopes expectations and, like so many of our peers, it falls to us now to adapt and reorient our business so as to place it back on the path to success," Percheron's board said.

"We recognise that moments such as this in the life of a biotech company place immense demands on the endurance and fortitude of shareholders."

EBR pre-approval inspection for mid-January as share price soars 30%

EBR Systems (ASX:EBR) has announced that the US Food and Drug Administration (FDA) has scheduled the manufacturing pre-approval inspection (PAI) to start on Tuesday, January 14, after previously being expected to start the week of January 6.

2025 is shaping up to being a big year for the developer of the WiSE CRT (cardiac resynchronisation therapy) system, which holds the distinction of being the world’s first leadless pacemaker for the heart’s left ventricle.

EBR said the updated PAI schedule won't affect the expected FDA regulatory approval timing, which remains on track for Q1 2025, with commercial launch later in the year.

The purpose of the PAI is to confirm EBR’s manufacturing procedures are compliant with quality system regulations and to ensure EBR can consistently produce devices that meet approved specifications.

FDA Breakthrough Device designation for the WiSE CRT System enables EBR to receive prioritised review and interactive communication with the FDA to the completion of the review process.

The company has submitted the final module of its premarket approval (PMA) for Wise with a substantive review underway.

Meanwhile, EBR also responded to a price query from the ASX with its share price up more than 30% in the past five days and 48% in the past month.

"EBR is not aware of any information concerning it that has not been announced which, if known, could be an explanation for recent trading in its securities," the company said.

Regis makes another key acquisition

Aged care provider Regis Healthcare (ASX:REG) is continuing its acquisition trail in 2025, inking a deal to a acquire the business and assets of BodeWell Community Care, a privately owned home care operator providing Home Care Packages (HCP), Commonwealth Home Support Programme (CHSP), and private home care.

Just how much the deal is costing Regis has not yet been disclosed but the company said its home care business is expected to double to ~$30 million in revenue with more than 2,500 clients following completion.

The company said the deal would be funded from existing cash and expected to be completed in Q4 FY25, subject to regulatory approvals and other customary conditions.

Regis CEO Dr Linda Mellors said BodeWell was a well-regarded home care service which enhances the company's existing operations by providing greater scale in Melbourne and extending operations into South-East Queensland.

"We expect the expanded home care business will benefit from an ageing population, increased government funding, and the shift in consumer preference to age in place," she said.

In December Regis announced it had completed the acquisition of two residential aged care homes from Ti Tree Operations Pty Ltd, located on the Mornington Peninsula in Victoria.

The acquisition expanded Regis’ national portfolio to 69 homes and ~7,750 beds.

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