Shares of Amicus Therapeutics, Inc. (NASDAQ:FOLD) ended Monday session in green amid volatile trading. The shares closed up +0.36 points or 4.95% at $7.64 with 142.14 million shares getting traded. Post opening the session at $7.34, the shares hit an intraday low of $7.32 and an intraday high of $7.94 and the price vacillated in this range throughout the day. The company has a market cap of $1.12 billion and the numbers of outstanding shares have been calculated to be 142.14 million shares.
Amicus Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines for various rare and orphan diseases. Its principal product is the migalastat HCl, a small molecule, which has completed Phase III studies that can be used as a monotherapy and in combination with enzyme replacement therapy (ERT) for Fabry disease. The company is also developing SD-101, which is in Phase III clinical study for the treatment of the genetic connective tissue disorder epidermolysis bullosa; ATB200 that is in Phase II studies to treat pompe disease; AT2221, which is in Phase II studies for the treatment of pompe disease; and AT3375 to treat Parkinson’s disease. The company has strategic alliance with GlaxoSmithKline plc to develop and commercialize migalastat as a monotherapy and in combination with ERT for Fabry disease. Amicus Therapeutics, Inc. was founded in 2002 and is headquartered in Cranbury, New Jersey.
Corporate GovernanceShares of Geron Corporation (NASDAQ:GERN) ended Monday session in green amid volatile trading. The shares closed up +0.12 points or 6.38% at $2.00 with 159.14 million shares getting traded. Post opening the session at $2.00, the shares hit an intraday low of $1.94 and an intraday high of $2.05 and the price vacillated in this range throughout the day. The company has a market cap of $303.74 million and the numbers of outstanding shares have been calculated to be 159.14 million shares.
Geron Corporation (GERN) on Sept. 12, 2016 provided updates on the clinical trials being conducted by Janssen Research & Development, LLC, of the telomerase inhibitor imetelstat. Planned internal reviews of initial data from both trials have been completed by Janssen, and both trials are continuing in order to evaluate additional and more mature data.
IMbarkTM (NCT02426086) was originally designed as a Phase 2 clinical trial to evaluate two dose levels of imetelstat (either 4.7 mg/kg or 9.4 mg/kg administered every three weeks) in approximately 200 patients (approximately 100 patients per dosing arm) with Intermediate-2 or High risk myelofibrosis (MF) who have relapsed after or are refractory to prior treatment with a JAK inhibitor. The co-primary efficacy endpoints for the trial are spleen response rate and symptom response rate at 24 weeks. To date, over 90 patients have been enrolled in the trial across both dosing arms.
To inform an assessment of the appropriate dose and schedule for relapsed or refractory MF patients in IMbarkTM, Janssen conducted a planned internal interim review of safety, efficacy and pharmacokinetic data from 20 patients from each dosing arm who have been followed on the trial for at least 12 weeks. Based on this first internal review at the early 12-week time point, the following has been determined by Janssen:
- The safety profile was consistent with previous imetelstat clinical trials in hematologic myeloid malignancies. No new safety signals were identified.
- Activity in the 4.7 mg/kg dosing arm does not warrant further investigation of that dose and this arm will be closed to new patient enrollment. An amendment to the trial protocol is planned to allow eligible patients in this arm to increase their dose to 9.4 mg/kg per investigator discretion.
- In the 9.4 mg/kg dosing arm, even though at the week 12 data assessment an insufficient number of patients met the protocol defined interim criteria, this arm warrants further investigation because encouraging trends in the efficacy data were observed. Patients already enrolled in this arm may continue to receive imetelstat. New enrollment in this arm will be suspended while the trial continues in order to obtain additional and more mature data that includes a longer follow-up of patients at 24 weeks, consistent with the co-primary efficacy endpoints. The number of patients enrolled to date is expected to be sufficient to inform potential future development of this dose.
- Janssen plans to conduct an additional internal data review in the second quarter of 2017 to include a longer follow-up of patients at 24 weeks. Potential outcomes of the second internal review at the 24-week time point could include resuming enrollment in the 9.4 mg/kg dosing arm, with or without changes to the dosing regimen; adding a new dosing arm; or closing the trial.
- Any protocol amendments will be subject to review by health authorities around the world.