Tekmira Pharmaceuticals Corporation has announced an update on the TKM-Ebola Phase I clinical hold. The Company has received the clinical hold letter from the U.S. Food and Drug Administration (FDA) and is preparing a Complete Response to the Agency. The Company anticipates this matter will be resolved by Q4, 2014.
Tekmira's other clinical development programs are unaffected by the TKM-Ebola clinical hold and all remain on track. The key milestones for these programs in the second half of 2014 are:
• Presentation of TKM-HBV Pre-Clinical data
• Filing IND (or equivalent) for TKM-HBV
• Interim Phase IIa TKM-PLK1 data
• Nomination of the next product development candidate
The clinical hold letter confirms that the FDA is seeking data to elucidate the mechanism of potential cytokine release and a modification to the protocol for the multiple ascending dose portion of the trial to ensure the safety of healthy volunteers.
"It is important to highlight that the study protocol for the TKM-Ebola Phase I trial called for an interim review of the data from the single ascending dose portion of the trial before proceeding to the multiple ascending dose portion of the study. I wish to emphasize this trial is unique. It represents the first RNAi study involving the daily treatment of healthy volunteers, without steroid pre-medication or any other type of pre-medication, and with multiple ascending doses," said Dr. Mark Murray, President and CEO of Tekmira Pharmaceuticals. "Furthermore, the multiple ascending dose portion of the study, as originally proposed, reflects the intense dosing regimen that would be used in patients lethally infected with Ebola virus."
On May 21, 2014, the Company disclosed the results of the single ascending dose portion of the study which demonstrated the administration of TKM-Ebola in the absence of any steroid-containing pre-medication was well-tolerated at a dose level of 0.3 mg/kg, determined to be the maximum tolerated dose in the absence of steroid cover.
At that time, Dr. Murray said, "These (TKM-Ebola Phase I) results are significant as they establish the safety of 'third generation' LNP formulations and confirm that dosing at efficacious levels may be accomplished without the need for pre-medication."
The Company is assembling the data requested by the FDA and shares its commitment to patient safety. Dr. Murray added, "The mechanism for cytokine release is understood and we will be modifying our study protocol to further ensure subject safety. Our team is working expediently to respond to the FDA. We are mindful of the need for this important therapeutic in situations such as the ongoing Ebola outbreak in West Africa. However, TKM-Ebola is currently an unapproved agent and the regulatory framework to support its use in Africa has not been established at this time."
The Company will provide a full business update at the upcoming earnings release on August 13, 2014.