Full Press Release Details
HAT-301 Registrational Trial for PAX-101
HAT-301 is the Pivotal Efficacy Study to Support Upcoming NDA Filing
Top Line Results Planned for Release in July 2023
June 1, 2023 - PaxMedica, Inc. (Nasdaq: PXMD), a clinical stage biopharmaceutical company focusing on the development of novel anti-purinergic
drug therapies (APT) for the treatment of Autism Spectrum Disorder (ASD), and other serious conditions with intractable neurologic symptoms,
today announced that the eligibility review and final enrollment for the company's Real World Evidence study (HAT-301) has
been completed. Patient eligibility was assessed for inclusion by an Independent Study Adjudication Committee which included a total of
three subject matter expert medical reviewers.
The comparative arms for this pivotal Phase 3
Real World Evidence study included:
A total of 145 patients treated with suramin in Uganda and Malawi between 2000 - 2022, and
A total of 204 natural history cases from patients in Uganda and Malawi between 1900-1910, prior to the availability of suramin.
The HAT-301 study is the first and only retrospective
clinical study of suramin in the treatment of Rhodesian African Sleeping Sickness, also known as Stage
1 Trypanosoma Brucei Rhodesiense Human African Trypanosomiasis (TBR HAT), a rare and universally fatal infectious disease. Suramin
has been the standard of care for treating TBR HAT for many decades, solely based on empirical evidence derived during mass epidemics
The official database lock for HAT-301 will occur
in June 2023, with top-line data for this study expected to be available in July 2023, and pave the way for filing an NDA for the use
of PAX-101 in TBR HAT in 2024. If approved, suramin will be the first drug indicated for the treatment of TBR HAT in the United States,
and would possibly qualify PaxMedica to receive a Priority Review Voucher (PRV) under section 524 of the Food, Drug, and Cosmetics Act
(FD&C Act). A PRV, once granted, is an independently valued asset (see GAO report 20-251), granted to a sponsor company after NDA
approval and, according to section b)2) of the act, can be sold by that sponsor to any biopharmaceutical company to obtain FDA priority
review in a future filing of any NDA.
Howard Weisman, Chief Executive Officer of PaxMedica,
commented, "Database lock for the HAT-301 study is a significant threshold event for PAX-101. With this achievement, PaxMedica has
completed a critical step in filing for potential U.S. market approval for this important, life-saving treatment. The opportunity to raise
significant non-dilutive funding, as a result of our potential successful participation in the Neglected Rare Tropical Disease Program
through receipt of a PRV, may enable future large-scale trials of PAX-101 in Autism, and other important conditions, in the very near
future. This remains central to our strategy to build and support sustainable long term shareholder value."
About the Phase 3 HAT-301 Trial
Launched in November 2022, the Phase 3 HAT-301
trial is a pivotal, retrospective, controlled analysis of suramin for the treatment of the rare and fatal tropical disease, TBR HAT. The
study has been underway at multiple primary HAT treatment sites in Uganda and Malawi using exclusively licensed, retrospective clinical
data that is being reviewed, and compared with a natural history controlled dataset comprised of exclusively licensed source data.
The primary objective of the study is to demonstrate
that the standard of care treatment using suramin, as currently practiced in Uganda and Malawi, leads to better clinical outcomes in patients
with Stage 1 Trypanosoma Brucei Rhodesiense Human African Trypanosomiasis than observed in
an untreated natural history cohort, that had documented illness prior to availability of suramin. The World Health Organization lists
suramin, discovered in 1916, and found to be effective against Stage 1 Trypanosoma Brucei Rhodesiense
Human African Trypanosomiasis since at least 1920, as a standard of care for Trypanosoma b. Rhodesiense.
PaxMedica is a clinical stage biopharmaceutical
company focusing on the development of anti-purinergic drug therapies ("APT") for the treatment of disorders with intractable
neurologic symptoms, ranging from neurodevelopmental disorders, including Autism Spectrum Disorder ("ASD"), to Myalgic Encephalomyelitis/Chronic
Fatigue Syndrome ("ME/CFS"), a debilitating physical and cognitive disorder believed to be viral in origin and now with rising
incidence globally due to the long term effects of SARS-CoV-2 ("COVID-19"). One of PaxMedica's primary points of focus
is the development and testing of its lead program, PAX-101, an intravenous formulation of suramin, in the treatment of ASD and the advancement
of the clinical understanding of using that agent against other disorders such as ME/CFS and Long COVID-19 Syndrome, a clinical diagnosis
in individuals who have been previously infected with COVID-19. For more information, visit www.paxmedica.com.
Forward-Looking Statements
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our anticipated clinical program, the timing and success of our anticipated data announcements, pre-clinical and clinical trials and regulatory
filings, statements about the strength of our balance sheet. These forward-looking statements involve known and unknown risks and uncertainties
and are based on the Company's current expectations and projections about future events that the Company believes may affect its
financial condition, results of operations, business strategy and financial needs. Such risks and uncertainties include, but are not limited
to, risks associated with the Company's development work, including any delays or changes to the timing, cost and success of the
Company's product development and clinical trials, risk of insufficient capital resources, cash funding and cash burn and risks
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Although the Company believes that the expectations expressed in these forward-looking statements are reasonable, it cannot assure you
that such expectations will turn out to be correct, and the Company cautions investors that actual results may differ materially from
the anticipated results and encourages investors to review other factors that may affect its future results described in the Company's
"Risk Factors" section and other sections in its Annual Report on Form 10-K, most recent quarterly filings and other filings
with the U.S. Securities and Exchange Commission.