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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 plas

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. 📈 📉

APLS

APLS This stock is worth a closer look at the current enterprise value/stock price. Pros → 2 Approved early-stage drugs, → 7/28 PDUFA with solid data for next rare disease indication for EMPAVELI, → More attractive entry point for buying stock (~$2.25B MC and EV), ⚬ Valuation is now 1/2 where it was 6 months ago → ~$150M/quarter in product revenue with early products, → Early stage SiRNA asset to enhance effectiveness of SYFOVRE has exciting potential, → SYFOVRE safety issu

Our Top 50 StR portfolio targets have been substantially updated. See new targets at the link below.

Another interesting company to diligence is Dyne Therapeutics (DYN). Their shares were hit pretty hard this week, apparently because they changed primary outcomes for their upcoming DMD readout. Did the market over-react to that, and in the wrong direction? That is something to dig deeper into.

An interesting company to diligence is ACRS (Aclaris) - they have 2 Big Mover readouts coming this month for 2 different assets, and their market cap is less than $200M, yet 7 hedge funds hold them. It's very rare for this many hedge funds to hold a company with that market cap. However, in 2021 their market cap was 10X current, and we find that biopharma-focused hedge funds are not in a rush to pull out of holdings where they have lost big. We have not traced the hedge fund

An interesting company to diligence is ACRS (Aclaris) - they have 2 Big Mover readouts coming this month for 2 different assets, and their market cap is less than $200M, yet 7 hedge funds hold them. It's very rare for this many hedge funds to hold a company with that market cap. However, in 2021 their market cap was 10X current, and we find that biopharma-focused hedge funds are not in a rush to pull out of holdings where they have lost big. We have not traced the hedge fund

If you are looking for another interesting company to diligence, check out COGT. They are trying to develop a therapeutic that would compete with BPMC's lead drug AYVAKIT® (avapritinib) to treat Systemic Mastocytosis (SM). BPMC announced yesterday that it agreed to be acquired by SNY for ~$9B, whereas COGT has a current market cap around $650M and EV ~$500M. Thus, if COGT were successful to develop a therapeutic to displace AYVAKIT, investors would be handsomely rewarded. COG

It was good to see SCPH up ~25% on their quarterly update in May. Company still only ~$200M market cap, so plenty of room to run up this year.