PaxMedica Completes 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
TARRYTOWN, NY, June 01, 2023 (GLOBE NEWSWIRE) -- via NewMediaWire – 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 of TBR HAT.
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.
About PaxMedica
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.
