Health Check: It’s thumbs up from the FDA for two ASX Car-T cancer drug developers
Cell therapy oncology drug developers Imugene and Chimeric Therapeutics have won FDA support – albeit in different ways.
Imugene says the agency supports the company’s approach to a pivotal phase III blood cancer trial
Chimeric’s solid cancer cell therapy wins FDA ‘orphan’ designation
CSL reports positive five-year data for its single dose haemophilia B therapy
Imugene (ASX:IMU) is planning a phase III blood cancer trial, after the US Food and Drug Administration (FDA) delivered positive feedback on the company’s lead asset, Azer-cel.
In effect, the agency has given its blessing to the structure and endpoints of the trial, which would be conducted to support US marketing approval application.
Azer-cel is based on Car-T cells (chimeric antigen receptor T-cells), which fight cancer by targeting CDH-17, an antigen expressed on tumours.
The mooted study will enrol patients with advanced diffuse large B cell lymphoma (DLBCL), including those who have relapsed after previous autologous Car-T therapies.
Under the autologous approach, clinicians take the patient’s own blood cells and engineer them with Car-T cells to bolster their cancer-busting abilities.
Imugene says the FDA is happy with the company’s proposed use of overall response rate (ORR) and progression-free survival (PFS) as endpoints.
ORR is the rate of overall cancer disappearance; PFS refers to the time the disease remains stable, or doesn’t get worse.
Currently, Imugene is carrying out an ongoing, open-label phase Ib trial across sites in the US and here.
The study focuses on relapsed DLBCL Car-T patients but has broadened to ‘naïve’ (previously untreated) patients with other lymphomas.
The company cites an 82% ORR in the Car-T relapsed patients and an 83% response in Car-T naïve niche indications.
Imugene CEO Leslie Chong says the FDA’s written feedback “gives us confidence as we continue to design our pivotal study plans”.
Chimeric is happy to be an orphan
Meanwhile the FDA has bestowed ‘orphan’ drug designation on fellow cancer drug developer Chimeric Therapeutics' (ASX:CHM) lead program.
The asset, CHM CDH-17 also harnesses Car-T cells and – as the name suggests – targets CDH-17.
Chimeric has enrolled nine gastric and neuroendocrine cancer patients in a phase I/II trial and treated nine of them. The company expects to enroll 15 patients in the first stage, aimed at dose selection and expansion.
Orphan status confers benefits such as tax advantages and seven years’ marketing exclusivity in the US.
Chimeric CEO Rebecca McQualter dubs the orphan status “a great step forward in the development of CHM CDH-17 to serve patients with gastric cancer, where there is a significant unmet need”.
Five years on, CSL’s haemophilia trial looks HOPE-ful
CSL (ASX:CSL) has reported positive five-year data from a phase III trial of its single-infusion hemophilia B treatment, Hemgenix.
As published in the august New England Journal of Medicine, 51 of the 54 patients – 94% – “remained free from the burden of continuous prophylaxis treatment” over the five years.
This demonstrated “sustained therapeutic benefit”.
In 2021 CSL acquired Hemgenix, the world’s first gene therapy for haemophilia B, from Dutch outfit Uniqure for around $500 million.
Hemophilia B is a life-threatening rare disease caused by gene mutation. This results in low levels of a blood clotting agent, called functional clotting factor IX.
Patients are vulnerable to bleeds in their joints, muscles, and internal organs. This leads to pain, swelling, and joint damage.
Hemgenix stimulates production factor IX, using a non-infectious virus called AAV5.
Dubbed HOPE B, the open-label study enrolled 54 adult male participants with severe or moderately severe hemophilia B. These candidates were “with or without” pre-existing AAV5 antibodies.
At year five, mean factor IX activity levels “remained strong” at 36.1%. Hemgenix also continued to show favourable safety, reinforcing its “durable efficacy”.
Trial participants can continue on a trial extension for up to 15 years.
Regulators in geographies including the US, Australia, Canada and the UK have approved Hemgenix. To date, more than 75 patients have received Hemgenix in “real-world' settings.
Led by the standard of care Idelvion, CSL’s haemophilia franchise contributed just under US$1.5 billion of revenue in the year to June 2025, 13% of the total.
Share relief for last week’s IPO flops
Unlike with the Australians’ clinical flogging of the Poms with both the ball and the willow, investors have taken pity on Saluda Medical (ASX:SLD) and Epiminder (ASX:EPI) following their train-wreck debuts last week.
Saluda shares plunged 50% on Friday’s listing, but bounced 11 cents (9%) this morning.
Epiminder shares had lost one third of their value since last Monday’s listing, but today recovered 18 cents (18%).
Saluda has developed – and commercialised – an implanted device for pain management. Last year the company generated US$71 million of revenue and expects to manage US$82 million this year.
But Saluda also lost US$128 last year and expects a US$145 million deficit this year.
In pre revenue stage, Epiminder is developing a sub-scalp epilepsy management device.
The message is that investors are becoming receptive to biotech IPOs after a prolonged drought, but don’t get too ambitious on pricing.
Record order for Audeara
Hearing-technology outfit Audeara (ASX:AUA) is ending the year with an audible bang, having secured its biggest ever purchase order of $560,000.
To be recognised as revenue in the March quarter, the order derives from a “major Australian wholesale customer”.
The win brings wholesale revenue for the year to date to around $1.75 million, 50% higher year to date.
Meanwhile, the company’s offshore AUA Technology arm is in advanced negotiations with existing customers, in view of repeat orders.
This company has progressed to the second and final shipment for a “previously announced global music industry leader”.
“This follow-on shipment is expected to be recognised in the current quarter and highlights the increasing contribution of AUA Technology revenues to Audeara’s financial performance,” the company says.
In August, Audeara struck a licensing deal with the Taiwan-based Eastech. This involves Eastech manufacturing hearing aids using Audeara’s tech, with the finished product sold under a third-party brand.
At Stockhead, we tell it is like it is. While Audeara is a Stockhead advertiser, the company did not sponsor this article.
Originally published as Health Check: It’s thumbs up from the FDA for two ASX Car-T cancer drug developers
