To say the phase 1 results of FTX-6058, Fulcrum Therapeutics’ (FULC) potential treatment for sickle cell disease (SCD) excited investors would be a serious understatement. Shares took off by over 180% last week as the drug displayed signs it could be beneficial for those suffering from SCD, a rare disease and one with a high unmet medical need.
The candidate is based on Fulcrum’s proprietary FulcrumSeek technology, a pioneering method for the discovery of new drugs.
In the early-stage testing, healthy volunteers showed significant signs of fetal hemoglobin (HbF) production after swallowing FTX-6058 tablets once a day over a period of two weeks. For volunteers who were given the highest dosage, the critical oxygen-transporting protein’s levels increased more than four fold. Apart from showing potential to be an effective therapy, FTX-6058 was also well tolerated.
“We believe the data provides strong proof-of-concept, proving that the targeting of EED is a viable target for the treatment of SCD,” said H.C. Wainwright’s Andrew Fein. “While early, we believe the Phase 1 data puts the FTX-6058 program on the radar for investors and makes the program a formidable candidate for approval, based on the earlier efficacy and novel mechanism-of-action.”
Based on the the clinical data, the 5-star analyst notes “approval may be warranted” as long as its efficacy and safety “continue to translate well” from preclinical studies.
Full results of the ongoing Phase HV study should see the light of day in 4Q21, with a Phase 1b SCD trial also to be initiated in the fourth quarter. The study will be an open-label multiple dose trial beginning at 6mg QD with the treatment administered over a 3-month period. The analyst anticipates this study will “further garner positive signals of safety, tolerability, PK, and PD effects (HbF protein induction) in SCD.” These will be used to form the basis of the Phase 2/3 SCD trial.
Overall, Fein rates Fulcrum a Buy and has a $20 price target for the shares. However, following last week’s gains, the figure implies ~11% downside from current levels. It will be interesting to see whether the analyst downgrade his rating or upgrade the price target over the coming months. (To watch Fein’s track record, click here)
All of Fein’s colleagues agree FULC stock is a Buy, and the addition of 4 other recent positive reviews make for a Strong Buy consensus rating. The forecast is for additional returns of 14%, given the average price target stands at $25.50. (See FULC stock analysis on TipRanks)
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Disclaimer: The opinions expressed in this article are solely those of the featured analyst. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.
