PYC Therapeutics (ASX:PYC): Its been raising ~$650m to fund 4 clinical assets for multiple years!

PYC Therapeutics (ASX:PYC) is currently in the Top 25 ASX healthcare stocks and is the 4th largest biotech, only trailing Telix, Mesoblast and Neuren. Clearly this is one worth looking into and this was before a capital raising that would be $650m and purportedly fund the company into next decade.

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PYC Therapeutics: focused on RNA drugs

PYC Therapeutics, which is headquartered in Perth, has a focus on RNA drugs. Accordingly, we cannot go further without explaining (at an elementary level) the underlying science. RNA therapeutics work by intervening earlier in the biology. Instead of trying to block or stimulate a protein after it already exists, RNA drugs act at the level of gene expression — telling the cell to make less of a harmful protein, more of a missing one, or a corrected version of it. That shift alone opens up huge territory.

One reason RNA drugs are compelling is that they can target diseases that are genetically well understood but chemically un-druggable. Roughly 80–90% of human proteins lack the kinds of pockets or structures that small-molecule drugs need to bind effectively. RNA doesn’t care about protein shape. If you know the genetic sequence driving the disease, you can often design an RNA construct to modulate it. That’s why monogenic diseases, in particular, are such a natural fit.

Other benefits include specificity (i.e. they can be designed to act on a single gene, splice variant, or even a mutant version of a gene while leaving the normal version untouched) as well as predictability in design (once you have a delivery system that works, designing a new RNA therapy is more like engineering than discovery chemistry – in other words, you’re not searching through thousands of compounds hoping one sticks, you’re rationally designing something to match a known sequence).

In terms of limitations, RNA therapeutics originally had three big problems: instability, delivery, and immune activation. Naked RNA degrades quickly in the body, struggles to cross cell membranes, and can trigger unwanted immune responses. These issues are why early RNA approaches failed repeatedly in the 2000s.

What’s changed is that many of those barriers have been partially or substantially overcome. Chemical modifications, backbone changes, conjugates, lipid nanoparticles, and peptide-based delivery systems have dramatically improved stability and cellular uptake. This is what made mRNA vaccines viable and what underpins modern antisense, siRNA, and RNA-editing approaches. Delivery is still the hardest problem, but it’s no longer a showstopper in certain tissues like liver, eye, kidney, and central nervous system.

That said, RNA is not magic. It still faces real constraints. Most RNA drugs require repeat dosing, sometimes for life. Delivery outside of well-accessed tissues remains challenging. Manufacturing is complex and expensive. And while off-target effects are rarer than with small molecules, they can still happen, particularly if delivery is imperfect or expression patterns aren’t fully understood.

There’s also a practical limitation: RNA therapies tend to work best when the disease biology is simple and direct — a missing protein, a toxic gain-of-function, or a splicing error. Complex, multifactorial diseases like diabetes or Alzheimer’s are much harder to tackle this way, at least with today’s technology.

PYC’s science

PYC was established to pioneer a new generation of precision RNA therapeutics aimed at treating rare monogenic diseases — genetic disorders caused by mutations in a single gene where no or few therapies currently exist.

The company’s scientific foundation is built around its proprietary delivery technology, which uses cell-penetrating peptide conjugated morpholino oligomers (PPMOs) to improve intracellular delivery of RNA-modulating compounds, overcoming a key limitation of many traditional RNA therapies. This platform underpins all its lead drug candidates and defines PYC’s strategy of targeting the root causes of disease rather than symptoms.

PYC’s pipeline

PYC’s pipeline comprises four major programs in various stages of development, each addressing a different genetic disorder:

• PYC-003 — Polycystic Kidney Disease (PKD): This candidate targets the underlying genetic drivers of autosomal dominant PKD, a relatively common inherited condition affecting around 1 in 1,000 people. The program has progressed into early human clinical trials, including a Phase 1a/1b study with dosing completed in several cohorts. The goal of these early studies is to establish safety and early indication of biological effect, with data expected as the program moves toward multiple-dose studies and eventually registrational trials.

• PYC-002 — Phelan-McDermid Syndrome (PMS): This candidate is designed to address a severe neurodevelopmental disorder caused by insufficient expression of the SHANK3 gene. Preclinical work demonstrated the ability to restore SHANK3 expression in disease models, supporting progression toward first-in-human studies. Clinical advancement is targeted around the first half of 2026 as the company prepares to enter the regulatory and clinical phases.
• VP-001 — Retinitis Pigmentosa Type 11 (RP11): VP-001 is PYC’s lead ocular program focused on this form of inherited retinal degeneration. The company has already reported early clinical data showing improvements in visual function in treated patients and has aligned with the U.S. Food and Drug Administration (FDA) on a registrational trial design. The next big milestones for VP-001 involve longer-term efficacy data from ongoing Phase 1/2 studies and the potential initiation of a registrational Phase 2/3 trial, guided by FDA feedback.
• PYC-001 — Autosomal Dominant Optic Atrophy (ADOA): PYC-001 aims to treat ADOA, a progressive blinding disorder caused by mutations in the OPA1 gene. After completing early cohort dosing and receiving regulatory clearances, PYC has started a global repeat-dose Phase 1b study to assess safety, tolerability, and preliminary efficacy. Readouts from this study are anticipated in the second half of 2026, with the intention of progressing toward larger registrational trials in 2027.

The commonality

You can see the company’s focus is on monogenic diseases with major unmet medical needs and no approved treatments. PYC often highlights that monogenic indications statistically have a higher likelihood of clinical success and regulatory approval than many other types of therapeutic targets.

The company’s investor materials reference published genetic drug development data to support the idea that drug targets with human genetic validation can have up to five times the probability of success compared to broader industry averages. Now of course, this is a general research observation rather than something guaranteed for PYC’s specific programs, but it would be fair to say it is lower risk compared to single-asset biotechs, especially those at a pre-clinical phase.

As we observed, key milestones are expected through 2026 and into 2027 that could materially affect the company’s valuation and scientific credibility. By ‘materially affect’, we mean the company will live or die on them. Of course, it will not fall apart on the basis of one bad result with multiple assets, but it will be receiving crucial clinical data on several of its assets.

These include human safety and early efficacy data for PYC-001 (ADOA), regulatory approvals and first-in-human trials for PYC-002 (PMS), ongoing Phase 1/2 and potential registrational work for VP-001 (RP11), and advancing PYC-003 (PKD) into deeper clinical evaluation. Collectively, these readouts are typical biotech binary outcomes, where positive or negative data can significantly move market sentiment and the share price.

Conclusion