LIFE-CHANGING RESEARCH

Translating scientific advances into meaningful treatments
An image of a woman wearing a white lab coat, clear goggles and blue gloves in a lab. Her hair is plaited and lying across her right shoulder. She is standing in front of a scientific machine and her right hand is resting on the machine while she smiles at the camera.
OUR CLINICAL TRIALS

We are committed to applying our breakthrough RNA technology to the development of life-changing therapies.

We are initially focused on three core therapeutic areas – the ocular, central nervous and renal systems – although our proprietary drug delivery platform has the potential to revolutionise treatment for many genetic diseases.

Patient trials are essential to the development of all novel therapeutics. At PYC, we conduct responsible patient trials to ensure that our treatments are safe and effective. These trials rely on patient volunteers and are crucial to the development of disease-modifying therapies for genetic diseases.

Current trials

An up-to-date overview of our current clinical trials.

Autosomal Dominant Optic Atrophy (ADOA)

ADOA Phase 1a study

Trial Status: Enrolling

Location: Australia

Contact for participation: [email protected]

PYC is conducting a Phase 1a open-label, single ascending dose study to evaluate the safety and tolerability of intravitreally administered PYC-001 in participants with confirmed OPA1 mutation-associated autosomal dominant optic atrophy.

SRC: safety review committee.

This is a first-in-human multi-centre, prospective, Phase 1a, single ascending dose (SAD) interventional study of PYC-001 in participants with confirmed OPA1 mutation (haploinsufficiency) associated ADOA.

If you are interested in learning more about this clinical trial, visit clinicaltrials.gov for more information or email [email protected].

ADOA Phase 1b study

Trial Status: Not Yet Recruiting (starting Sep 2025)

Location: Australia and United Kingdom

Contact for participation: [email protected]

 PYC is conducting Phase 1b Open-Label, Randomized, Single Dose and Repeat Dose Study to Evaluate the Single and Repeat Dose Safety and Tolerability of Intravitreally Administered PYC-001 in Participants with Confirmed OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (ADOA)

 Who may be eligible?

A patient may be able to participate in the clinical trial if they meet eligibility criteria including (but not limited to):

  • Adults at least 18 years of age
  • A recent (within 5 years) genetic diagnosis of OPA1 mutation associated ADOA
  • A BCVA of between ≤20/40 and ≥20/200

If you are interested in learning more about this clinical trial, visit clinicaltrials.gov  for more information or email [email protected].

Autosomal Dominant Polycystic Disease (ADPKD)

ADPKD Phase 1 study

Trial status: Enrolling

Location: Australia

Contact for participation: [email protected]

PYC is conducting a Phase 1 study to evaluate the safety, tolerability and pharmacokinetics of intravenously administered PYC-003 in healthy volunteers and ADPKD patients.

SRC: safety review committee.

This is a first-in-human multi-centre Phase 1a and Phase 1b, single ascending dose (SAD) interventional study of PYC-003 in healthy volunteers and patients with confirmed PKD1 mutation-associated Autosomal Dominant Polycystic Kidney Disease.

If you are interested in learning more about this clinical trial, visit clinicaltrials.gov or email [email protected].

Retinitis Pigmentosa type 11 (RP11)

Clinical trial information

PYC’s RP11 program have closed enrolment for the Natural History Study and Phase 1 clinical trials.

Future trials are being planned. For more information, please email [email protected].

Phelan-McDermid Syndrome (PMS)

Clinical trial information

PYC’s PMS program is expected to advance into clinical trials in 2026.

For more information please email [email protected].

Expanded access policy

PYC Therapeutics is a clinical-stage biopharma company dedicated to the discovery and development of RNA therapeutics for genetic diseases.

Our lead program is in ophthalmology, to treat retinitis pigmentosa type 11(RP11) with our proprietary molecule VP-001. We have recently received Fast Track Designation for this program from the US FDA. We are committed to conducting clinical trials to evaluate the safety and efficacy of VP-001 to obtain the necessary marketing approvals that allow physicians to prescribe them so patients may have broad access.

In some circumstances, a treating physician may make a request to provide an unapproved therapy to a qualifying individual, which must be approved by the U.S. Food and Drug Administration (FDA) and supported by the pharmaceutical manufacturer. This is called an expanded access program (EAP), sometimes known as ‘compassionate use’, and refers to a potential pathway for a patient with a serious or life-threatening disease or condition to try an investigational therapy outside of a clinical trial when there is no comparable or satisfactory therapy available.

While we understand that some patients may wish to access our unapproved investigational drugs, our goal is to provide access to our RNA therapeutics at the appropriate time and in a manner that is most beneficial to the relevant patient population. At this stage of our development, we do not have sufficient evidence of clinical safety and efficacy of our investigational drug, VP-001, to support an expanded access program. Therefore, PYC Therapeutics does not currently offer an expanded access program. We believe enrolment in our ongoing clinical trials is the safest and most effective way of achieving this goal and we encourage all patients and physicians who are interested in accessing our investigational treatments to visit the clinicaltrials.gov website to find out about enrolling.

PYC Therapeutics may revise this expanded access policy at any time. Additionally, the posting of this policy by PYC does not serve as a guarantee of access to any specific investigational new drug by any individual patient.

If you have any questions, please reach out to us at [email protected].