Here are 3 ASX biotechs with major milestones coming right up!

Investing in ASX biotechs is a risky business, even more than most other companies on the market. Unless you own one of the large cap biotechs, you are betting that major milestones (such as clinical trial completions and decisions on regulatory approval) will occur right on cue and that they will be favourable to the company. But it doesn’t hurt to know which companies have these milestones upcoming, because at least you’ll get an answer sooner.

We attended the Bioshares Summit in Perth last week and heard from plenty of interesting companies. Some companies had success to crow about – like Dimerix and Telix – others remain a work in progress. But there were some with clear milestones coming in the next 12 months and we thought they were worth highlighting. So, here are 3 ASX biotechs with major milestones coming up.

1. Opthea (ASX:OPT)

It has been more than 5 years since Opthea’s wet AMD treatment (Sozinibercept) passed Phase 2 – a fairly long wait, even for a biotech. But the company is expecting results from 2 Phase 3 trials in the first half of CY25. This is expected to be the trial that will help the drug gain regulatory approval.

For those who have forgotten, Phase 2 was a flying success for the company. In simple terms, the average patient receiving Sozinibercept could read 14.2 letters on a typical eye chart whilst other patients (those receiving Lucentis’ treatment ranibizumab, the placebo) could only read 10.8 letters. Non-headline results (such as patients who gained 15 letters or more from the baseline) were good too. And it was a trial of 366 patients across 110 sites worldwide – so the result clearly was no mere fluke. If this result is replicated in Phase 3, there will be shareholder value created.

2. EBR Systems (ASX:EBR) 

EBR Systems has a wireless device that is the size of a cooked grain of rice that can treat heart failure by providing pacing stimulation directly inside the heart’s left ventricle. The device, the WiSE Cardiac Resynchronisation Therapy (CRT) passed clinical trials with flying colours, showing improved heart function, a decrease in left ventricular end systolic volume of 16.4% (vs 9.4% for the placebo) and with 80.9% of patients free of type 1 complications.

The company has been making its case to the FDA, expecting to the final part of its data necessary in the current quarter, and is expecting a response in the March quarter of 2025. If its rollout is anything near as successful as Telix (ASX:TLX) and Neuren (ASX:NEU), shareholders in EBR will do nicely. The company thinks it has an initial addressable market of US$3.6bn.

3. Neuren (ASX:NEU)

Wait? Hasn’t this company had its time in the sun. It is true that Neuren has had plenty of success, launching its drug (Daybue or trofinetide) in North America in April 2023 for Rett Syndrome. Neuren has >1,300 patients having initiated therapy as of May 2024. It achieved US$177m in the first 9 months of commercial sales, expecting US$370-420m in CY23.

But this may not be the end of this company’s hot run. Neuren has advanced the drug against other neurological indications. Results in Phase 2 from NNZ-2591 are expected in Q3 2024 for Angelman and entry into Phase 3 for Phelan-McDermid and Pitt Hopkins is expected as there already is Phase 2 data. Furthermore, the company has not licensed its drug out for these conditions yet, enabling either a licensing deal to deliver upside. Companies like Dimerix (ASX:DXB) have realised shareholder value prior to successful clinical trial results when such deals are done.

Oh, and did we mention that there could be further approvals for Rett Syndome in other markets? The company is planning to make its case to regulators in Europe and Japan in the next 12 months

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