Monopar Therapeutics: Rare Pediatric Disease Designation a Positive Ahead of NDA Submission

Research evidence excerpt

Monopar Therapeutics: Rare Pediatric Disease Designation a Positive Ahead of NDA Submission

Equity Research

U.S. Small & Mid Cap Biotechnology

30 June 2026

Monopar Therapeutics

Rare Pediatric Disease Designation

a Positive Ahead of NDA First Look

MNPR OVERWEIGHTSubmission

U.S. Small & Mid Cap POSITIVE

FDA may grant MNPR a priority review voucher upon Biotechnology Price Target USD 125.00

potential approval of ALXN1840. Price (29-Jun-26) USD 94.56

Potential Upside/Downside +32.2%

Source: Bloomberg, Barclays Research

MNPR announced via press release that FDA has granted ALXN1840 Rare Pediatric Disease (RPD)

designation for the treatment of Wilson disease. The designation comes ahead of MNPR's

planned New Drug Application (ND) submission for ALXN1840 in mid-year and gives MNPR a U.S. Small & Mid Cap Biotechnology

potential priority review voucher (PRV) upon an approval. PRVs are fully transferable and have Etzer Darout, PhD, MBA

+1 212 526 6940been sold for as much as $205M, which could provide non-dilutive capital to MNPR to cover

etzer.darout@barclays.com

commercialization expenses or fund R&D for the company's radiotherapeutics pipeline. RPD

BCI, US

does not automatically grant MNPR a priority review of ALXN1840, so we maintain our projected

launch of 2Q27 for the drug. We reiterate our Overweight rating for MNPR. Lukas Shumway, PhD

+1 212 526 7293

Recent data disclosure continues to support case for ALXN1840 in Wilson disease lukas.shumway@barclays.com

MNPR recently presented a poster at the European Academy of Neurology (EAN) conference in

Geneva with analysis of the Phase 3 FoCus trial that evaluated ALXN1840 versus standard of care Jordan Becker, PhD

in patients with Wilson disease, in a subset of patients that had neurologic symptoms at +1 212 526 3960