Fulcrum Therapeutics' (FULC) FTX-6058 Data is Strong Start, Sets Stage for In-Patient Trial in 4Q21 - Analyst
Earlier today, Fulcrum Therapeutics, Inc. (Nasdaq: FULC) announced positive interim results from its ongoing single- and multiple-ascending dose (SAD and MAD) Phase 1 trial with FTX-6058 in healthy adult volunteers.
Results from the MAD portion of the trial demonstrated proof of biology as evidenced by a dose proportional induction in HBG mRNA and accompanying increases in HbF-containing reticulocytes (F-reticulocytes). At 10mg, the highest dose studied to date, the mean changes were 4.5-fold and 4.2-fold, respectively. The increases in F-reticulocytes indicate that the HBG mRNA increases observed with FTX-6058 treatment are translating to HbF protein production.
Additionally, all FTX-6058 doses in the MAD portion of the trial achieved maximal target engagement as evidenced by decreases in trimethylation at lysine 27 of histone H3 (H3K27me3), a downstream target of the polycomb repressive complex 2 (PRC2). These proof of mechanism results were achieved with once-daily, oral administration of FTX-6058 for 14 consecutive days, which was also generally well-tolerated in all SAD and MAD cohorts completed to date.
Reacting to the news, Stifel analyst Dae Gon Ha says that the data readout makes him “incrementally more bullish on FTX-6058.”
The analyst notes that “certain biological reasons would suggest in-patient efficacy could be better than HVs that were reported today.” He is encouraged by the drug’s efficacy at every dose regimen and notes that “HV data partially de-risks FTX-6058 ahead of Ph.1b patient trial wherein the outcome will help select the go-forward dose into the subsequent Ph.2/3 trial (possibly a 2023 initiation).”
The analyst currently models in-patient trial will commence in 4Q21 and raises overall probability of success for FTX-6058 to 30% from 20%.
Stifel reiterates a “Buy” rating on the shares and increases its price target to $45 from $39.
FULC Shares are gaining over 140% following the announcement.
Written by Vlad Schepkov | [email protected]