Geron Corporation (GERN) announced on Thursday that the FDA has lifted a clinical hold on one trial of lead drug candidate, imetelstat, in myelofibrosis. Though this is a step in the right direction, the lift pertains to an investigator-sponsored myelofibrosis (MF) trial at the Mayo Clinic; the company’s own IND remains under full clinical hold. Geron cannot begin dosing any new patients or being new trials with imetelstat. GERN climbed 26% in morning trading, opening at $3.24 from a $2.56 close on Wednesday, before sliding through the afternoon back to the low $3-range.
To lift the IND hold, the FDA has requested 1) clinical follow-up in patients who experience LFT (liver function testing) abnormalities until changes are resolved to normal or baseline; and 2) information regarding reversibility of the liver toxicity after chronic drug administration in animals.
Geron is working to amend protocols and obtain patient-informed consent for longer follow-up to collect LFT and other relevant information. On the non-clinical front, the company is exploring toxicity studies of chronic imetelstat administration in mice and cynomolgus monkeys. Studies to date have shown no clinical or anatomical changes indicative of injury and no clear sign of LFT abnormalities.
Following a full or partial lift on the company’s sponsored trials, Geron expects to pursue development of imetelstat in MF, essential thrombocythemia/polycythemia vera, and in multiple myeloma. Future indications may include myelodysplastic syndromes (MDS) or acute myelogenous leukemia (AML).
The lift is a step forward for Geron, but the hold won’t be resolved rapidly. We note that Geron ended the first quarter with $153.5 million in cash and equivalents, roughly $1.00 per share.
