Health Check: Clinical studies hit key milestones, but the long drive continues

• Amplia reports more positive responses in its pancreatic cancer study
• Is Immutep’s lung cancer study futile or not? We’ll soon know
• Cryosite comes in from the cold?

Major clinical studies are not a case of enrolling a patient one day and announcing a cure the next. They involve incremental advances over a period of years, hopefully leading to an inflexion point where it is clear the therapy works.

In this vein, four drug developers today delivered trial updates that would never move the market in isolation. But they are crucial milestones towards the ultimate destination.

The clinicians can only take a quick coffee stop before getting back to work.

More pancreatic promise from Amplia

We’ll start with Amplia (ASX:ATX), which earlier this year piqued investor interest with two ‘complete responses’ in its pancreatic cancer trial, dubbed Accent.

Given pancreatic cancers are one of the deadliest and difficult to treat, complete responses – the tumours disappears – were unexpected.

Today, Amplia reported a further confirmed partial response and unconfirmed partial response, taking the objective response rate (ORR) to 33%.

This was “notably superior” to the 23% rate for standard-of-care chemotherapy.

ORR measures the percentage of trial patients that see their cancers shrink (partial response), or a complete response.

The average ‘duration on trial’ for the patients has reached 219 days, ‘substantially better’ than chemo alone.

Duration on trial measures the ability of a drug to halt cancer progression.

Combined, the confirmed and unconfirmed response rate was 42%.

FAK, there’s hope after all

Dubbed Accent, the trial is testing Amplia’s focus kinase (FAK) inhibitor narmafotinib in combination with two chemotherapies.

FAKs are overexpressed in pancreatic tumours.

Enrolling nine patients, the trial combines narmafotinib with chemo.

“Notably, seven patients have remained on the study for at least 12  months, and two patients have continued for more than 18 months,” the company says.

Earlier, Amplia reported a median progression-free survival of 7.6 months – a two-month improvement compared to chemo alone.

Amplia CEO Dr Chris Burns says the results suggest narmafotinib “enhances the response to chemotherapy by improving efficacy and durability, whilst being well tolerated in this patient group”.

The company plans further trial updates in “coming months”, with overall survival data due out in the March quarter.

Amplia is conducting the trial at sites locally and in South Korea.

Immutep hits ‘futility analysis’ stage

Fellow cancer drug developer Immutep (ASX:IMM) says it has enrolled more than 170 patients in its phase III non-small cell lung cancer (NSCLC) trial, Tacti-004.

The company says the number is significant because it is enough to carry out a ‘futility’ analysis. This is the point at which trial custodians decide whether a study is worth continuing.

The trial evaluates Immutep’s novel immunotherapy eftilagimod alfa (efti), in combination with the approved immunotherapy Keytruda and chemo.

The company intends efti to be used as  first-line treatment for advanced or metastatic NSCLC.

“We are very pleased with the pace of enrolment in our pivotal phase III trial that we believe has the potential to change the treatment landscape in non-small cell lung cancer,” Immutep CEO Marc Voigt says.

Previously the company completed two combination studies, with 165 patients enrolled.

These efforts saw “high response rates and strong progression-free survival.”

The company hopes an independent data monitoring committee will complete the futility analysis in the March quarter.

Still the deadliest cancer

Lung cancer is the biggest cause of cancer deaths, with NSCLC accounting for 80-85% of all diagnoses.

The condition is often diagnosed at a late stage, and fewer than 30% of patients last for more than five years.

Tacti-004 targets enrolling 756 patients at more than 150 clinical sites in more than  25 countries. Half the patients receive a placebo.

In a busy slate, Immutep also is trialling efti for head and neck squamous cell  carcinoma  (HNSCC), soft tissue sarcoma and breast cancer.

The US Food and Drug Administration (FDA) has granted Efti has fast-track designation for NSCLC and HNSCC.

Immutep shares this morning gained up to 2.5 cents, or 9%.

PYC’s dosing study passes safety hurdles

In the non-cancer sphere, genetic disease specialist PYC Therapeutics (ASX:PYC) says a safety review has cleared its eye disease drug candidate to progress to a further study.

The company’s drug candidate PYC-001 addresses a blinding eye disease, called autosomal dominant optic atrophy (ADOA).

The company has completed three cohorts of a single ascending dose (safety) study. A safety committee reviewed data from the strongest-dose cohort after four weeks’ treatment and found no safety concerns.

The study thus will continue to phase I/II stage, in which the patients receive multiple doses.

The company expects the trial to kick off in the current quarter.

Alterity leis it all out at Hawaiian jamboree

Meanwhile, Alterity Therapeutics (ASX:ATH) aired further data from its multiple system atrophy (MSA) trial, at the 2025 International Congress of Parkinson’s Disease and Movement Disorders in Honolulo.

Alterity’s 77 patient, phase II effort tests its therapy ATH-434 for MSA, a Parkinsonian disorder.

The additional data supports previously reported “efficacy signals” at the 52-week treatment mark.

“This new analysis continues to support our belief that ATH-434 has great potential to treat this devastating disease,” Alterity chief Dr David Stamler says.

The therapy relates to excess iron levels in the brain.

Research house warms to Cryosite

Overlooked veteran cryogenics and clinical logistics play Cryosite (ASX:CTE) trades at a material discount to its true worth, according to Research as a Service (RaaS).

This makes the company a prime takeover target.

The independent research house believes Cryosite is well poised for the “explosive growth” in cell and gene therapies, genetic medicine and biological sample management.

Founded 25 years ago and listed since 2002, Cryosite provides end-to-end storage and logistics services to the life sciences sector.

“It is the market leader in Australia, and strong cash flows and a debt-free balance sheet have allowed the company to grow revenues organically to date,” RaaS says.

The company is expanding its South Granville site to a second facility at Auburn, both in western Sydney.

“CTE’s established footprint may be highly attractive to global peers targeting the Asia-Pacific market,” RaaS says.

Cryogenics also is well protected from competition, given the sector is highly regulated and approvals would take years.

Cryosite trades at a multiple of eleven times last year’s underlying earnings of $3.4 million, compared with 21 to 74 times for its global peers.

Seeing you ask, these include the Nasdaq listed Azenta (20 times), Biolife Solutions (74 times) and the loss-making Cryoport.

The RaaS report doesn’t mention the unprofitable Vitrafy Life Sciences (ASX:VFY) , which listed on the ASX in November last year after a $35 million raising.

Vitrafy is focused on bovine artificial insemination, aquaculture and blood platelet storage but wants to expand into the cell gene sector.

Vitrafy bears a $55 million market cap compared to Cryosite’s $42 million – which once again shows that investors prefer the new toy.