6 Stocks Likely to Move Big on Rare/Genetic Disease Readouts

A number of rare/genetic disease readouts are set this Q3 2025. Some of them are likely to move big. See table below for 6 of the anticipated readouts. 📈 📉

6 stocks likely to move big on rare/genetic disease readouts in Q3 ‘25 👇 

$ARCT

ARCT-032 / Cystic Fibrosis / Phase 2

» Safety findings

→ No SAEs, severe AEs, or dose-limiting toxicities

→ Dose-related increase in transient, mild, post-dose respiratory symptoms

» PK findings: Very low systemic exposure

→ mRNA: all plasma specimens BLQ

→ LNP lipid components sporadically detected in low concentrations (<1.0 ng/mL)

Prior results & Ph2 trial overview 📸

$IONS

Donidalorsen / Hereditary angioedema / PDUFA

» Robust Data Supports Potential Preferred Treatment for HAE Prophylaxis

» Sustained Reduction in HAE attacks that continue to improve with extended treatment

Prior trial results 📸

$FBIO ✔️

CUTX-101 (Copper Histidinate) / Menkes Disease / PDUFA

» Showed significant clinical benefit in both CuHis-ET (early tx) and CuHis-LT (late tx) cohorts

→ 75-79% reduction in risk of death compared to untreated Historical Control (HC-ET and HC-LT) arms

→ increase in Median OS from 1.3 years to 14.8 years in the ET cohort

Prior results 📸 👇

$KALA 

KPI-012 / Persistent Corneal Epithelial Defect / Phase 2b

» Top line results in efficacy cohort

→ 8/8 showed improvement; 6/8 patients had complete healing

» All healed patients remained healed through end of follow-up

» Well-tolerated with no safety issues observed

Phase 1b results 📸 👇

$HRMY

Zygel (ZYN002 CBD Gel) / Fragile X syndrome / Phase 3

» 40% Median improvement in socially avoidant behaviors

» Sustained Improvement in Patients With Complete Methylation of FMR1

Ph3 RECONNECT trial overview & prior results 📸 👇

$LRMR ✔️

Nomlabofusp (CTI-1601) / Friedreich’s Ataxia / Phase 2

» Generally well tolerated

» Dose dependent increases of frataxin levels in tissues tested (skin and buccal cells)

» FXN levels increased to 33% to 59% of the average of the healthy volunteers after OD x 14 days dosing

Prior results 📸 👇

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#ARCT #IONS #FBIO #KALA #HRMY #LRMR

This article is not investment, tax, or legal advice. Please do your own diligence and seek advice from professional advisors representing your interests.

Article history:

First published 07/16/25 EJV, JD, AV, DG