By Grant Zeng, CFA
Tekmira Provides Update on U.S. Government Sponsored TKM-Ebola Program
On May 8, 2013, Tekmira Pharmaceuticals Corporation (Nasdaq-small:TKMR) announced that its contract with the U.S. Department of Defense (DoD) has been modified to support development plans that integrate advancements in Tekmira's lipid nanoparticle (LNP) formulation and manufacturing technologies, and provide for additional funding for the TKM-Ebola program.
Ebola is a virus, which, for many years, has been associated with periodic outbreaks of hemorrhagic fever in human populations with mortality rates reaching 90%. Currently there are no approved treatments for Ebola or other hemorrhagic fever viruses.
TKM-Ebola is an anti-Ebola viral therapeutic, being developed under a contract with the U.S. DoD's Joint Project Manager Transformational Medical Technologies (JPM-TMT) Office with a total contract value of approximately $140 million.
The Development History of TKM-Ebola
In May 2010, Tekmira published a series of preclinical studies demonstrating the ability of an RNAi therapeutic utilizing its LNP technology to protect non-human primates from Ebola virus. Tekmira conducted the studies in collaboration with infectious disease researchers from Boston University and the United States Army Medical Research Institute for Infectious Diseases (USAMRIID) and were funded in part by the U.S. Government’s Transformational Medical Technologies (TMT) program.
These preclinical studies demonstrated that when siRNA targeting the Ebola virus and delivered by Tekmira's LNP technology were used to treat previously infected non-human primates, the result was 100 percent protection from an otherwise lethal dose of Zaire Ebola virus.
On July 14, 2010, Tekmira signed a contract with the United States Department of Defense (DoD) to advance an RNAi therapeutic utilizing the Company’s LNP technology to treat Ebola virus infection. In the initial phase of the contract, Tekmira is eligible to receive up to US$34.8 million. This initial funding is for the development of TKM-Ebola, including completion of preclinical development, filing an IND application with the FDA and the completion of a Phase I human safety clinical trial. The United States DoD has the option of extending the contract beyond the initial funding period to support the advancement of TKM-Ebola through to the completion of clinical development and FDA approval. Based on the budget for the extended contract this would provide the Company with a total of up to US$140.0 million in funding for the entire program.
In November 2011, an Investigational New Drug (IND) application for TKM-Ebola was approved by the FDA. In February 2012, Tekmira initiated the TKM-Ebola Phase I clinical trial, which is a placebo-controlled, single-blind, single-ascending dose study with additional multiple-ascending dose cohorts in healthy human volunteers. The objective of the Phase I trial is to assess the safety and tolerability of TKM-Ebola and evaluate the pharmacokinetics and systemic exposure following both a single-ascending dose and multiple-ascending doses of TKM-Ebola.
On August 6, 2012, Tekmira received a temporary stop-work order from the United States DoD with respect to the TKM-Ebola program. On October 2, 2012, Tekmira disclosed that the temporary stop-work order was lifted by the United States DoD and work will continue on development of the TKM-Ebola product.
During the course of the development of TKM-Ebola, the LNP technology has been significantly improved. Therefore, Tekmira has submitted a modification request to the existing contract to the United States DoD in order to integrate recent advancements in LNP formulation and manufacturing technology, including lyophilization, in the TKM-Ebola development program. The program will utilize an LNP formulation that is more than 10-fold more potent than previous formulations and more potent than all other LNP formulations currently being evaluated in clinical trials.
Under the modification to the existing contract, funding has been increased by $6.9 million, from $34.8 million to $41.7 million for the first phase of the contract.
Tekmira has initiated pre-clinical and chemistry, manufacturing and control studies that support the use of these improvements in the program. This development strategy will be accommodated by modifications to the existing contract, allowing both Tekmira and TMT to benefit from the significant advancements in LNP formulation technology made by Tekmira since the commencement of the TMT-funded program in July 2010. It is expected that the LNP formulation work will be completed and submitted to the FDA in the second half of 2013 in order to initiate a new Phase I clinical trial.
New data from the TKM-Ebola program will be presented at the 15th Annual TIDES Summit: Oligonucleotide and Peptide(R) Therapeutics from Research through Commercialization taking place in Boston, MA from May 12-15, 2013.
TKM-Ebola, Accelerated Program Could Provide Significant Near-Term Cash Flow
TKM-Ebola is being developed under specific FDA regulatory guidelines called the “Animal Rule.” The Animal Rule provides that under certain circumstances, where it is unethical or not feasible to conduct human efficacy studies, the FDA may grant marketing approval based on adequate and well-controlled animal studies when the results of those studies establish that the drug is reasonably likely to produce clinical benefit in humans. Demonstration of the product's safety in humans is still required.
We think the “Animal Rule” means a lot for Tekmira, because this can accelerate the development of TKM-Ebola. Once approved by the FDA, Tekmira will have the opportunity to negotiate a stock-pile contract with the US government. These stock-pile or procurement contracts have been very lucrative for other companies supplying similar drugs to the US government.
By Grant Zeng, CFA