Neuren receives US$40m milestone payment
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Neuren (NEU) – ASX Announcement
7 June 2023
Neuren receives US$40m milestone payment
Melbourne, Australia: Neuren Pharmaceuticals (ASX: NEU) today reported that it has received
the milestone payment of US$40 million earned following the first commercial sale of DAYBUE™
(trofinetide) by Neuren’s North America partner Acadia Pharmaceuticals (NASDAQ: ACAD).
DAYBUE was approved by the US Food and Drug Administration (FDA) on 10 March 2023 and is
the first and only drug approved by the FDA for the treatment of Rett syndrome.
Neuren and Acadia have an exclusive license agreement for Acadia to develop and
commercialise trofinetide for the treatment of Rett syndrome and other indications in North
America. Neuren is eligible to receive ongoing royalties on net sales of trofinetide in North
America, plus milestone payments of up to US$350 million on achievement of a series of four
thresholds of total annual net sales, plus one third of the market value of the Rare Pediatric
Disease Priority Review Voucher that was awarded to Acadia by the FDA upon approval of the
NDA, with the one third share estimated by Neuren as US$33 million. No royalties or similar
costs are payable by Neuren to third parties, which means that Neuren’s revenue from Acadia
will flow through to pre-tax profit.
About Neuren
Neuren is developing new drug therapies to treat multiple serious neurological disorders that emerge in
early childhood and have no or limited approved treatment options.
DAYBUE™ (trofinetide) is approved by the US Food and Drug Administration (FDA) for the treatment of
Rett syndrome in adult and pediatric patients two years of age and older. Neuren has granted an
exclusive licence to Acadia Pharmaceuticals Inc. for the development and commercialisation of
trofinetide in North America, while retaining all rights outside North America.
Neuren is conducting Phase 2 trials of its second drug candidate, NNZ-2591, for each of PhelanMcDermid syndrome, Angelman syndrome, Pitt Hopkins syndrome and Prader-Willi syndrome.
Recognising the urgent unmet need, all programs have been granted “orphan drug” designation in the
United States. Orphan drug designation provides incentives to encourage development of therapies for
rare and serious diseases.
Contact:
Jon Pilcher, CEO: jpilcher@neurenpharma.com; +61 438 422 271
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ASX Listing Rules information
This announcement was authorized to be given to the ASX by the board of directors of Neuren
Pharmaceuticals Limited, Suite 201, 697 Burke Road, Camberwell, VIC 3124
Forward-looking Statements
This announcement contains forward-looking statements that are subject to risks and uncertainties. Such
statements involve known and unknown risks and important factors that may cause the actual results,
performance or achievements of Neuren to be materially different from the statements in this
announcement.
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