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 positive top line
data for the Company’s PAX-HAT-301 Retrospective Analysis of
Suramin Treatment for Stage 1 Trypanosoma Brucei Rhodesiense Human
African Trypanosomiasis (S1 TBR HAT).
The conclusions of the study confirmed that the retrospective,
non-randomized, externally controlled, interventional efficacy and
safety study of suramin for the treatment of Stage 1 TBR HAT
demonstrated better health outcomes when compared with a natural
history control group of patients evaluated and treated from
1900-1910, prior to the availability of suramin in Africa. The
adverse event profile of suramin observed in the study was
consistent with what has been widely reported in published medical
and clinical literature.
PaxMedica is expecting to file an NDA with the U.S. Food and
Drug Administration (FDA) for the use of PAX-101 to treat Stage 1
African Sleeping Sickness (also known as Human African
Trypanosomiasis Brucei Rhodesiense or 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 potentially
qualify PaxMedica to receive a Priority Review Voucher (PRV) under
the Neglected Rare Tropical Disease Program in 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,
“We’re excited to have completed this important real world evidence
study that demonstrated both statistically significant and
clinically meaningful results, and confirmed over 100 years of
clinical experience with suramin as a life-saving medication in
this potentially fatal infection. Completion of this study is an
important step on the path to bring suramin to the United States
under the Neglected Rare Tropical Disease Program and will enable
PaxMedica to accelerate the clinical development of PAX-101 for the
treatment of the core symptoms of ASD.”
The PAX-HAT-301 Study
The PAX-HAT-301 study is a retrospective, non-randomized,
externally controlled, interventional efficacy and safety study
comparing medical records data from a cohort of patients with Stage
1 Trypanosoma Brucei Rhodesiense (S1 TBR HAT) evaluated and treated
from about 2000 – 2020 at one medical site in Uganda and two
medical sites in Malawi (referred to as the suramin-treated
cohort), with medical records data from a cohort of patients from
1900-1910 evaluated and treated during the TBR HAT epidemic in
Uganda (referred to as the natural history cohort). These records
included data from a few weeks of hospitalization while they were
being evaluated and the diagnosis of TBR HAT confirmed. As their
conditions began to deteriorate, patients were often treated with
arsenic or related compounds, sent to a Sleeping Sickness Hospital,
or sent home to die with their families. The natural history
records do not include long term outcomes data for many of the
patients. The study was designed in consultation with the U.S. Food
and Drug Administration (FDA) and to ensure that the historical
control group of patients had TBR HAT (not the chronic TBG form)
and were in Stage 1 of the disease.
The primary objective of the study was to determine whether
standard of care treatment with suramin, as currently practiced in
Uganda and Malawi, from 2000-2020, led to better health outcomes in
patients with S1 TBR HAT, than outcomes observed in a natural
history cohort from the epidemic >100 years ago. The secondary
objective was to evaluate the safety and tolerability of suramin.
The primary endpoint of the study was survival and not meeting any
of the supportive descriptive criteria (i.e., death, progression of
the disease from Stage 1 to Stage 2, or becoming “moribund”
[discharged to a sleeping sickness hospital, physician or patient
giving up hope, or being close to death with no hope of recovery]).
An independent study adjudication committee was established to
review the suramin-treated and natural history cases for study
eligibility, and to confirm the clinical endpoints. The committee
was comprised of three physicians experienced in the treatment of
TBR HAT in Malawi and Uganda.
The PAX-HAT-301 Study Results
The outcomes observed in the suramin-treated cohort were both
statistically significant and clinically meaningfully different
from the outcomes observed in the natural history cohort. The
suramin-treated patients had a far lower rate of death or
progression to Stage 2 compared with the natural history cohort. In
addition, many of the longer-term outcomes from the natural history
cohort pointed towards death as the inevitable outcome of TBR HAT
without the benefit of suramin treatment.
In the study population there were 349 patients, 145 in the
suramin-treated cohort and 204 patients in the natural history
cohort. There were 121 suramin-treated and 42 natural history
patients with sufficient data and that met all eligibility criteria
for the primary analysis. The suramin-treated patients had a mean
age of 31.1 years (range from 2 to 85 years) and 64% male. The
natural history patients had a mean age of 22 (range from 3 to 40
years) and 79% male. Racial and ethnicity data were not available
and weight was only available for about half of the suramin-treated
patients.
The suramin-treated patients presented with a variety of
commonly reported HAT related symptoms. The most common symptoms
were fever/chills, severe headache, aching joints, extreme fatigue,
and swollen lymph nodes. The natural history cohort patients had
presenting symptoms recorded in 27/42 (64%) of cases. The reported
symptoms were similar including headache, “feeling ill”,
drowsiness, cough, weakness, chest pain, diarrhea, and enlarged
lymph nodes. One suramin-treated patient tested positive for HIV
(only 23% tested) and 16/76 (21%) of patients tested were positive
for malaria. No comparable data is available for the natural
history cohort.
The outcomes for the two cohorts differed substantially. Of the
suramin patients, 114 (94%) survived and successfully completed the
treatment. Three patients (2%) had “Other” listed as the reason for
stopping suramin treatment and 4 (3%) had no reason for stopping
suramin treatment recorded. No patients required rescue medications
for progression from Stage 1 to Stage 2.
In the natural history cohort, 6 (14%) were recorded as cured,
improved, or discharged. Three (7%) patients died, 10 (24%)
experienced clinical worsening, and 17 (40%) achieved moribund
status (near death and in terminal clinical decline). It is
anticipated that if all of these patients were followed for up to 6
months, that nearly all of them would have died.
The primary efficacy analysis revealed that the health outcomes
in the suramin-treated cohort were statistically significantly
better than those in the natural history cohort. According to the
definition of the primary endpoint, the proportion of patients in
the suramin-treated group that was alive and not meeting any
supportive descriptive criteria of death, any clinical worsening or
moribund status was 92% vs. 50% in the natural history cohort. The
estimated proportion (95% CI) was 0.442 (0.277, 0.600). The
two-sided p-value for the Fisher’s exact test was <0.001
Background Information
Human African Trypanosomiasis (HAT), also known as sleeping
sickness, is caused by infection with vector-borne protozoan
parasites of the two species of Trypanosoma brucei: Trypanosoma
brucei gambiense and Trypanosoma brucei rhodesiense (TBR). TBR HAT
is an acute form of HAT with the symptoms emerging within weeks,
and death occurring within six months, if untreated. Between 1900
and 1920, an epidemic of the disease occurred in Uganda resulting
in the death of an estimated 250,000 people. At the time of the
epidemic, there were no known treatments. Physicians tried to treat
patients with arsenic, and some arsenic-related compounds, with
limited success and significant side effects.
Suramin was introduced in 1922 and is still frequently used as
first-line treatment for Stage 1 TBR HAT. Suramin is unsuitable for
use in confirmed Stage 2 TBR HAT because it does not readily cross
the blood-brain barrier. It has been manufactured by Bayer and
distributed to countries endemic for TBR HAT through the World
Health Organization. Suramin was never approved for use in the U.S.
or EU, and it is difficult to procure quickly, although it is often
prescribed to US travelers who contract TBR HAT after a trip to an
endemic area in Africa.
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. 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
advancing the clinical understanding of using that agent against
other disorders such as ME/CFS.
For more information, visit www.paxmedica.com.
Forward-Looking Statements
This press release contains “forward-looking statements.”
Forward-looking statements reflect our current view about future
events. Investors can identify these forward-looking statements by
words or phrases such as “may,” “will,” “could,” “expect,”
“anticipate,” “aim,” “estimate,” “intend,” “plan,” “believe,”
“is/are likely to,” “propose,” “potential,” “continue” or similar
expressions. These forward-looking statements include 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 associated with
intellectual property and infringement claims. The Company
undertakes no obligation to update or revise publicly any
forward-looking statements to reflect subsequent occurring events
or circumstances, or changes in its expectations, except as may be
required by law. 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 most recent Annual Report on Form 10-K,
and subsequent quarterly and other filings with the U.S. Securities
and Exchange Commission.
Contact:Stephanie PrincePCG
Advisorysprince@pcgadvisory.com (646) 863-6341
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