Fulcrum Therapeutics (FULC)
Update on former net cash FULC
I mentioned Fulcrum Therapeutics (FULC) here as another net cash pharma last winter. It was the last of the attractive net cash pharma’s I could find back then.
Others from that article include Aclaris ACRS and Seer SEER. Seer still has $203 mil in net cash with a market cap of $123 mil. ACRS has slightly lower cash now and doesn’t make the cut currently of net cash anymore.
The stock went up from $3.30’s up 110% to $7.00 this past month. I see this as an opportunity to sell it all. I don’t have any specialized knowledge in these drugs or what the possibility of success truly is. Perhaps you, reading this, have more insights here. So, I will give a brief summary of things in this article.
Fulcrum is up a huge percentage here but many hurdles remain in the lead candidate. The lead program is Pociredir (FTX-6058) for Sickle Cell Disease (SCD) in phase 1b.
Pociredir is an oral small molecule designed to increase fetal hemoglobin (HbF) expression, offering potential as a best-in-class therapy for SCD.
While early data are promising, Phase 1b results must be replicated in larger trials. The prior clinical hold underscores the need for robust safety data to mitigate concerns about EED inhibitors.
The firm’s analysts have increased their gross peak sales estimates for Fulcrum to approximately $600 million, up from the previous $300 million, and doubled the probability-of-success rate to 50%.
It looks like there is cash runway into 2027.
Market Dynamics
The withdrawal of Pfizer’s Oxbryta (voxelotor) and the slow uptake of gene therapy alternatives have created a “scarcity value” in the SCD treatment landscape. This opens greater market potential for an effective oral small molecule like pociredir.
Fulcrum is advancing preclinical programs targeting inherited aplastic anemias, such as DBA, Shwachman-Diamond syndrome, and Fanconi anemia.
An Investigational New Drug (IND) application for DBA is planned for Q4 2025.
I've seen enough small-cap pharma stocks big runs reverse over the years to know it's wise to take profits on them on huge spikes. This is a clear place to lock in profits on the stock to me. Perhaps just leaving a small tranche of shares on as the drug results play out. That way, if the tiny tranche say ,10% of the original position drops the loss can be cut and the gain remains. There are always more opportunities in value stocks that will come about.
As I have mentioned previously, in the past I would adamantly avoid biotech and pharma stocks. The recent technology breakthroughs got me open to them. The net cash Somalogic worked out well and quickly so I kept some focus in the industry just for net cash. They keep working. The record low valuations industry-wide pushed them into basically a thematic.