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PaxMedica Receives FDA Orphan Drug Designation for Use of PAX-101 (IV suramin) in a Rare Tropical Disease

PaxMedica, Inc.
·4 min read

Company Pursuing Development of PAX-101 as Treatment for Autism Spectrum Disorder and Other Neurodevelopmental Conditions

WOODCLIFF LAKE, N.J., Feb. 02, 2021 (GLOBE NEWSWIRE) -- via NewMediaWire -- PaxMedica, Inc. ("PaxMedica" or the “Company”), a biopharmaceutical company focused on the development and commercialization of innovative treatments for unmet needs in neurodevelopmental disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PAX-101 (IV suramin) for the treatment of Human African Trypanosomiasis (HAT), a rare infectious disease.

“Receiving this designation from the FDA is an important milestone for PaxMedica and the treatment of HAT,” said Howard Weisman, CEO of PaxMedica. “Despite HAT being a very rare disease in the U.S., there continue to be isolated cases of this infection in people returning from travel in certain areas of East Africa. As there are no FDA approved treatments for HAT, access to effective medicines has been difficult and inconsistent for US citizens and residents. This is especially problematic in a condition like HAT, where time is of the essence when treatment is needed.”

Human African Trypanosomiasis (HAT) is a neglected tropical disease commonly known as African sleeping sickness. It is endemic to regions of sub-Saharan Africa and is a serious disease that is fatal if not treated quickly with the appropriate medication. HAT is caused by infection with protozoan parasites of the species Trypanosoma brucei and is vector-borne, i.e., transmitted through the bite of the tsetse fly. Suramin is the only treatment that has proved effective in curing East African HAT infection in its early stages.

PaxMedica has exclusively licensed patient data from East African Ministries of Health and hospitals where patients with the disease are treated. The Company expects to use these patient data, in addition to conducting necessary supplemental nonclinical toxicology, and in vitro and in vivo clinical studies, as the basis for an NDA submission for the use of PAX-101 in HAT infections. An FDA Approval of PAX-101 in HAT could qualify for New Chemical Entity exclusivity, in addition to Orphan Drug exclusivity, and could also result in PaxMedica being awarded a Priority Review Voucher (PRV) under the FDA’s Tropical Disease Priority Review Voucher Program.

Suramin has been in continuous use as the standard of care for the treatment of early-stage East African sleeping sickness (Trypanosoma Brucei Rhodesiense) for more than 100 years and is listed on the Centers for Disease Control (CDC) formulary and supplied by the World Health Organization (WHO), although it has never been approved by the FDA for use in the U.S. for any indication.

PaxMedica is separately pursuing the development of PAX-101 (IV suramin) as a treatment for Autism Spectrum Disorder and other neurodevelopmental conditions and will report findings from its just completed clinical trial in the near future.

About PaxMedica, Inc.

PaxMedica is a clinical-stage biopharmaceutical company focused on developing innovative treatments for unmet needs in neurodevelopmental disorders. Our lead programs are focused on two of the world’s most challenging conditions – Autism Spectrum Disorder (ASD) and Fragile X-associated Tremor / Ataxia (FXTAS). There are no FDA approved treatments for the core symptoms of ASD and no FDA approved treatments at all for FXTAS. We exist in order to change that. For more information, visit https://www.paxmedica.com/.

Forward-Looking Statements

This press release contains certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934 and Private Securities Litigation Reform Act, as amended, including those relating to the Company's product development, clinical and regulatory timelines, market opportunity, competitive position, possible or assumed future results of operations, business strategies, potential growth opportunities and other statements that are predictive in nature. These forward-looking statements are based on current expectations, estimates, forecasts and projections about the industry and markets in which we operate and management's current beliefs and assumptions.

These statements may be identified by the use of forward-looking expressions, including, but not limited to, "expect," "anticipate," "intend," "plan," "believe," "estimate," "potential, "predict," "project," "should," "would" and similar expressions and the negatives of those terms. These statements relate to future events or our financial performance and involve known and unknown risks, uncertainties, and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Such factors include those set forth in the Company's filings with the Securities and Exchange Commission. Prospective investors are cautioned not to place undue reliance on such forward-looking statements, which speak only as of the date of this press release. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise.

For more information, contact:

PaxMedica, Inc.
Howard Weisman
Chief Executive Officer
hweisman@paxmedica.com

Investor Relations:
Stephanie Prince
PCG Advisory
(646) 863-6341
sprince@pcgadvisory.com

Media Relations:
Tony Russo
Russo Partners, LLC
(212) 845-4251
Tony.Russo@russopartnersllc.com