Top 2 Biotech Penny Stocks on the ASX With Breakthrough Potential

Biotech penny stocks can feel binary—until the science clicks and commercial momentum takes off. That’s exactly why two small caps are drawing outsized attention right now. Neuren Pharmaceuticals is compounding rare‑disease royalties while opening new global markets for its first approved drug, and Amplia Therapeutics is producing early clinical signals that could reshape pancreatic cancer treatment. Here’s a clear, data‑driven look at the latest progress, catalysts to watch, and the risks that come with the ride.

Big science, bigger optionality

The best small-cap biotechs pair credible clinical data with tangible commercial pathways. Neuren’s royalty engine from an approved rare‑disease therapy is ramping quarter by quarter, funding a pipeline in neurology. Amplia’s targeted FAK inhibitor is generating response signals in combination therapy for one of oncology’s hardest tumours—pancreatic ductal adenocarcinoma (PDAC). Together, they offer diversified shots on goal across commercial execution and clinical inflection.

Neuren Pharmaceuticals (ASX: NEU): Rare‑disease royalties compounding, global expansion in sight

Neuren earns tiered royalties from US sales of DAYBUE (trofinetide) for Rett syndrome via its partner, Acadia Pharmaceuticals. Momentum is building as the field footprint grows and patient access broadens, while ex‑US pathways add meaningful upside.

1. Royalty ramp: Quarterly royalty income reached about $14.7 million in Q2 FY2025, up roughly 16% year on year and 9% sequentially, supported by a record ~987 US patients shipped and an expanded commercial field force (around 30% larger at partner level).
2. Ex‑US catalysts: A European marketing application for trofinetide was filed in January 2025, with potential EU approval targeted for Q1 2026. Health Canada approved in October 2024, with first Canadian sales expected in Q3 2025. Each region layers additional royalty-bearing revenue streams on top of the US base.
3. Japan pathway: Plans for a supportive local study ahead of a regulatory filing keep Japan in view as another high‑value market.
4. Second asset (NNZ‑2591): Positive Phase 2 signals across multiple rare paediatric neurodevelopmental syndromes (including Pitt Hopkins, Angelman, and Phelan‑McDermid) set the stage for Phase 3 planning. Any fast‑track designations, pivotal trial initiations, or interim updates can be material value inflections.

Why it matters: Neuren blends a proven commercial royalty with a diversified neurology pipeline—an uncommon profile for an ASX biotech. The expanding DAYBUE patient base funds development without heavy dilution, while new geographies and potential milestones from the partner add torque to earnings through 2025–2026.

What to watch next (6–12 months):

1. Successive quarterly increases in US patient numbers and net sales for DAYBUE.
2. Canada launch dynamics and progress of the EU review; any early access or named‑patient supply updates.
3. Program design and timing for NNZ‑2591 Phase 3 across target indications, plus any regulatory interactions that expedite timelines.

Key risks:

1. Commercial execution in rare diseases depends on diagnosis, payer coverage, caregiver persistence, and patient adherence—royalty trajectories can flatten if real‑world persistence dips.
2. Pipeline transitions to late‑stage trials bring larger, costlier studies with higher execution risk, even when Phase 2 signals are encouraging.

Amplia Therapeutics (ASX: ATX): FAK inhibitor shows compelling early signals in pancreatic cancer

Amplia is advancing narmafotinib (AMP945), a selective focal adhesion kinase (FAK) inhibitor designed to remodel the tumour microenvironment and enhance chemotherapy effectiveness. PDAC is notoriously resistant; combining targeted stromal modulation with standard chemo could be a step change if efficacy and tolerability hold.

1. ACCENT Phase 1b/2a update: In the ongoing study of AMP945 plus gemcitabine/Abraxane, investigators have reported 15 confirmed partial responses to date, with 21 of 55 patients still on study; final data readout is expected mid‑Q3 2025. Early read‑throughs suggest superiority on response metrics versus chemo alone.
2. Scientific rationale: Amplia presented the basis for pulsed dosing at a major US pancreatic cancer meeting, aligning pharmacokinetics, tissue target engagement, and safety to support once‑daily oral scheduling. Prior Phase 1 data demonstrated FAK inhibition in human tissue with a manageable safety profile.
3. Next steps: Depending on the final ACCENT dataset (response rate, durability, PFS/OS signals, safety), Amplia plans to engage on a randomized expansion and discuss potential registrational routes, supported by biomarker insights embedded in the current design.

Why it matters: PDAC remains an area of profound unmet need, with modest gains from existing regimens. A small molecule that safely enhances standard-of-care chemo could unlock a substantial market and attract expedited pathways or partnerships if mid‑stage efficacy holds up.

What to watch next (6–12 months):

1. Final ACCENT efficacy/safety data, detailed durability analyses, and subgroup outcomes (e.g., baseline tumour burden, biomarkers).
2. Conference presentations and peer‑reviewed publications that raise external confidence.
3. Clarity on randomized study design, geographies, endpoints, and whether a breakthrough/fast‑track regulatory dialogue is feasible.

Key risks:

1. Early signal risk: Open‑label Phase 1b/2a outcomes can fade in larger, randomized settings; robust design and powering are crucial.
2. Competitive landscape and funding: Multiple approaches in PDAC (including immunotherapy combinations and stroma‑targeted agents) compete for patients and capital; sustained financing is critical through the next phase.

What could push these stocks higher in the next 6–12 months

1. Neuren

Continued US patient growth and expanding field effectiveness reflected in quarterly royalties.

Progress in Canada launch metrics and concrete steps toward EU approval; any early named‑patient or access programs outside the US.

NNZ‑2591 regulatory interactions, pivotal trial initiations, or designations across indications.

2. Amplia

A strong final ACCENT readout with durable responses and a clean safety profile.

Inclusion at top oncology congresses and favorable KOL commentary.

Moving into a randomized study with sightlines to registrational endpoints and potential partnering.

Risks to keep front‑of‑mind

1. Neuren: Royalty curves require durable real‑world adherence, smooth reimbursement, and ongoing physician education; any plateau in patient adds or persistence can dampen growth. Pipeline success is not guaranteed, and multi‑indication programs stretch resources.
2. Amplia: Scaling from promising signals to registrational clarity in PDAC is a high bar. Timelines, competitive pressures, and capital availability will shape the path and potential dilution.

Bottom line

Neuren is a rare ASX biotech combining an accelerating rare‑disease royalty stream with a growing neurology pipeline—turning science into cash that can fund its next wave. Amplia’s focused FAK strategy is generating the kind of mid‑stage signals in pancreatic cancer that, if sustained and confirmed in randomized settings, could be transformative. For investors comfortable with higher risk in pursuit of outsized upside, these two penny biotechs offer credible, near‑term catalysts and differentiated routes to breakthrough potential.

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