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.

Retinitis Pigmentosa type 11 (RP11)

VP-001 Phase 1 trial

Trial status: Recruiting

Location: United States

Contact for participation: [email protected]

PYC is conducting a Phase 1 open-label study of RP11 patients who have a genetic mutation in the PRPF31 gene.

The primary objective of the study is to assess the safety and tolerability of VP-001.

Patients who participated in the RP11 natural history study are eligible to participate in this trial.

If you are interested in learning more about this clinical trial, visit clinicaltrials.gov for a list of active sites and contact the site’s study coordinator for more information.

Single ascending dose Phase 1 study

A diagram that depicts the RP11 single ascending dose phase clinical trial. The 3 participating patients each received a 3ug dose follow by a safety review after 4 weeks. They then received a 10ug with a safety review 4 weeks later, then a 30ug dose with a safety review 4 weeks later and finally a 75ug dose with a safety review 4 weeks later. After 24 weeks from the final dose, all patients received a follow up assessment.
SRC: safety review committee.

Multiple ascending dose Phase 1 study

SRC: safety review committee.

RP11 natural history study

Trial status: Enrolling

Location: United States, Western Australia

Contact for participation: [email protected]

PYC is conducting a study to characterise the natural history of disease through the systemic evaluation of subjects identified with PRPF31 mutation-associated retinal dystrophy, or RP11.

This is a non-interventional trial with assessments that will be completed to measure and evaluate structural and functional visual changes, including those impacting patient quality of life associated with this inherited retinal condition and observing how these changes evolve over time.

Results of this 4-year study will be utilised in conjunction with the VP-001 clinical trials to assess efficacy. If you are interested in learning more about this clinical trial, visit clinicaltrials.gov for a list of active sites and contact the site’s study coordinator for more information.

Autosomal Dominant Optic Atrophy (ADOA)

ADOA Phase 1 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 a list of active sites and contact the site’s study coordinator for more information.

ADOA natural history study

Trial status: Enrolling

Location: United States, Australia, Europe

Contact for participation: [email protected]

PYC is conducting a natural history study of patients with genetically confirmed diagnosis of autosomal dominant optic atrophy (ADOA), caused by OPA1 mutation.

This is a multi-centre, longitudinal, prospective, observational natural history study of patients with confirmed OPA1 mutation (haploinsufficiency) associated ADOA.

Natural history data will be collected prospectively and will include ophthalmic examinations, imaging studies and electrophysiological testing. Assessments will be conducted approximately every 3 months in the first year and every 6 months in the second year of the study after each participant’s baseline visit (Visit 2).

If you are interested in learning more about this clinical trial, visit clinicaltrials.gov for a list of active sites and contact the site’s study coordinator for more information.

CLINICAL TRIAL PARTICIPATION

Express your interest

We believe in the importance of collaborating closely with patients, their families, patient advocacy organisations, physicians, researchers and regulatory authorities to help achieve our goals together.

If you would like to learn more about patient trial participation with PYC, register your interest below.

You can also find more information about PYC Therapeutics’ investigational products that are currently undergoing clinical studies at clinicaltrials.gov.

For general information, please contact [email protected].

    A photo of two female scientists wearing white lab coats, goggles and blue gloves. One scientist is standing in front and is placing a slide under a microscope. The other scientist is standing behind her and watching.

    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].