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We are a Phase 3 clinical biotech developing drug candidates to treat neurodegenerative diseases. (Incorporated in the Cayman Islands)
We are a clinical-stage biotechnology company committed to protecting patients’ brain health and changing clinical outcomes for a broad range of neurodegenerative diseases by developing novel, highly sensitive and selective diagnostic tools and novel therapeutics.
Our name, APRINOIA, merges “apricum,” the Latin word for sunlight, and “noia,” the Greek suffix for the mind, reflecting our mission to shed light on ways to better diagnose and treat dreaded neurodegenerative diseases with a vision to offer a brighter future for patients with novel diagnostic tools and targeted therapeutics worldwide.
Neurodegenerative diseases are relentless and largely fatal, resulting from progressive loss of nerve cells in the brain and, depending on the specific disorders, can affect a broad range of cognitive, behavioral, and motor functions, such as memory, thinking, speaking, walking, and breathing. Although our understanding of the underlying pathophysiology has grown over the past decade due to advances in neuroscience, a significant unmet need remains for both accurate, safe and personalized diagnostics and effective and safe treatments.
We are developing PET imaging agents for the diagnosis and tracking of neurodegenerative disorders such as AD, PSP and related tauopathies and synucleinopathies. Our diagnostic pipeline includes PET tracers that target abnormal tau and α-Syn protein aggregates and enable (i) the visualization of their distribution in brain regions and (ii) their quantification.
Currently, early diagnostic markers for most neurodegenerative diseases do not exist. This is particularly true for patients who may have PSP or one of its variants, where in most cases clinicians cannot differentiate among these disorders or make specific diagnoses without pathological confirmation (i.e., autopsy). Our diagnostic product candidates, if approved, may provide clinicians with powerful and quantifiable tools to make more accurate clinical diagnoses earlier in the course of a neurodegenerative disease, potentially enable differentiation among different neurodegenerative disorders that clinically can resemble each other, such as PSP and PD, and importantly, enable clinicians to intervene with novel therapies that may be more likely effective at earlier stages of the disorder. These tracers may also improve the probability of success of clinical trials, by enabling the recruitment of patients at earlier stages of disease, who are more likely respond to treatment as was recently shown in the Phase 3 clinical trials for lecanemab and donanemab.
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Our lead diagnostic product candidate (APN-1607). APN-1607 is our 3R/4R tau PET tracer and most clinically advanced diagnostic product candidate. APN-1607 is designed as a new generation tau PET tracer to achieve a higher specificity for the pathological tau aggregates. We believe that APN-1607, if approved, has the potential to be a powerful enabling tool for the diagnosis of various tauopathies, as it has shown low non-specific binding to other brain proteins, and the ability to detect different forms of tau in clinical studies. APN-1607 may therefore potentially be used in more precise diagnosis and stage classification of various tauopathies, including PSP, AD and PiD.
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APN-1607 is being studied in PSP, for which we hold an ODD. In July 2023, the FDA agreed that we could conduct a single Phase 3 clinical trial of APN-1607 as a basis for approval without a pathology study, normally required for these types of programs. In lieu of a second trial, we will provide confirmatory evidence to support the diagnostic accuracy of the APN-1607 using other data sources such as the existing clinical imaging data obtained from our investigator-initiated studies in PSP patients.
On November 9, 2023, we filed an IND with the FDA to launch a single Phase 3 global clinical trial for APN-1607 in subjects suspected to have PSP. On December 8, 2023, the FDA issued a Study May Proceed letter, allowing us to conduct a Phase 3 trial to evaluate the efficacy and safety of APN-1607 as a diagnostic marker for PSP. We plan to launch a prospective multicenter trial in approximately 130 patients with suspected PSP, atypical Parkinsonism disorder, and PD. A visual read method will be used to establish the diagnosis of PSP in those patients who show the expected pattern of APN-1607 uptake in brain regions typically affected in PSP. We plan to implement this trial in the fourth quarter of 2024 in the United States, Europe, the United Kingdom, Japan and Taiwan and submit results from this trial, if positive, in an NDA as a basis for regulatory approval in the United States in the fourth quarter of 2026, and subsequently seek marketing authorization in Europe and Asia. We have also initiated a Phase 2 clinical trial of APN-1607 in AD in the United States, Japan and Taiwan, which is active and not recruiting for the time being due to a reprioritization of resources, with the last patient enrolled on July 11, 2022.
APN-1607 is also being studied in a Phase 3 clinical trial in AD in mainland China through our collaboration with Yitai, a wholly-owned subsidiary of Yantai Dongcheng Biochemicals Co., Ltd (together with its subsidiaries, “Dongcheng Pharma”). The trial has met its target enrollment of 230 patients, with the last patient dosed on December 21, 2023. Contingent on the results of this trial, Yitai plans to submit an NDA to the NMPA for the marketing approval of APN-1607 for the diagnosis of AD in mainland China.
We also plan to combine (i) the results from APN-1607’s Phase 2 clinical trial in AD in the United States, Japan and Taiwan, (ii) the results from APN-1607’s Phase 3 clinical trial in AD in mainland China together with (iii) imaging data of APN-1607 collected from investigator-initiated studies in over 3,300 patients to seek concurrence from the FDA for a single pivotal trial of APN-1607 as a diagnostic marker for AD in the United States and potentially other markets.
To support Aprinoia’s investigator-initiated and our programs, we have established relationships with contract manufacturing organization (“CMOs”) at 20 sites across Asia, Europe and the United States to produce and distribute APN-1607 to clinical trial sites.
(Note: APRINOIA Therapeutics filed its S-1/A on Feb. 26, 2024, disclosing the terms for its IPO: The company plans to offer 2.0 million shares at a price range of $10.00 to $14.00 to raise $24.0 million.)
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