This morning clinical-stage biotechnology company Moderna Inc. (MRNA:NASDAQ), which states it is pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines, announced positive data from the three-month interim analysis of safety and immunogenicity of the Phase 1 study of its investigational cytomegalovirus (CMV) vaccine (mRNA-1647).
Based on these data, the firm advised that it is advancing mRNA-1647 to a dose-confirmation Phase 2 study in the near term. Preparation has also begun for a pivotal Phase 3 study designed to evaluate the efficacy of mRNA-1647 against primary CMV infection. The firm indicates that mRNA-1647 is a vaccine combining six mRNAs in a single vial, which encode for two antigens on the surface of CMV: five mRNAs encoding the subunits that form the membrane-bound pentamer complex and one mRNA encoding the full-length membrane-bound glycoprotein B (gB).
Tal Zaks, M.D., Ph.D., chief medical officer at Moderna commented, "I am very encouraged by the ability of mRNA-1647 to induce high levels of durable immune responses that can reach or exceed the levels generated by natural CMV infection...We recognize there is an urgent need for a preventative vaccine against congenital CMV and will be advancing mRNA-1647 into a Phase 2 study in the near term to confirm the appropriate dose, while we plan for a pivotal Phase 3 study."
Sallie Permar, M.D., Ph.D., associate dean of physician scientist development and professor of pediatrics, immunology, and molecular genetics and microbiology at Duke Medical School, added, "Cytomegalovirus is the leading infectious cause of birth defects, and there is a great need for a vaccine that blocks transmission of the virus from the mother to the fetus...These interim data are exciting because mRNA-1647 has shown the ability to induce immune responses in seronegative individuals that are greater than what is seen in those naturally infected with CMV, which is important in that natural immunity is not completely protective against congenital CMV transmission."
The Phase 1 study, which has completed enrollment, is evaluating the safety and immunogenicity of mRNA-1647 in 169 healthy adult volunteers. The study population includes those who were naïve to CMV infection (CMV-seronegative) and those who had previously been infected by CMV (CMV-seropositive). A safety analysis indicated that the vaccine was generally well tolerated and there were no vaccine-related serious adverse events.
CMV is a common pathogen and member of the herpesvirus family. Congenital CMV infection results when infected mothers transmit the virus to their unborn child, and it is the leading infectious cause of birth defects in the U.S., with approximately 25,000 newborns in the U.S. infected every year. There is currently no approved vaccine for the prevention of CMV infection.
The company also today announced positive data in the first analysis of safety and activity in its Phase 1 study evaluating escalating doses of mRNA-1944 administered via intravenous infusion in healthy adults. mRNA-1944 encodes for an antibody (CHKV-24) with activity against chikungunya virus.
The data from the study shows that at all three dose levels, the administration of mRNA-1944 led to detectable levels of CHKV-24 antibody in all participants, ranging from 1 µg/mL to 14 µg/mL. The firm states that these results mark the first systemic mRNA therapeutic to show production of a secreted protein in humans. mRNA-1944 is being developed with financial support from the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense. mRNA-1944 is the first development candidate from the company's systemic therapeutics modality to start clinical testing and utilizes the same lipid nanoparticle (LNP) formulation as the company's rare disease program for methylmalonic acidemia (mRNA-3704). A total of 22 healthy adults have been enrolled in the study to date.
Dr. Zaks advised, "These Phase 1 data represent a significant scientific breakthrough: this study shows for the first time the ability to generate therapeutic levels of a complex protein in humans through systemic administration of an mRNA, essentially instructing the body to make its own medicines."
Moderna's CEO Stéphane Bancel stated, "These data represent another critical milestone for the validation of Moderna's mRNA platform in humans...This is the fifth modality for which we have shown translation from preclinical research to humans and the first demonstration of mRNA as a systemic therapeutic capable of creating high levels of protein at a well-tolerated dose. We believe these results further validate our approach, the scientific platform we have built and the potential of mRNA to become a new class of medicines. We look forward to learning from the ongoing Phase 1/2 study of mRNA-3704 for methylmalonic acidemia, the first of our rare disease programs to enter the clinic, as it utilizes the same technology demonstrated in this chikungunya study."
Chikungunya is a mosquito-borne virus that poses a significant public health problem in tropical and subtropical regions. The disease is characterized by an acute onset of fever, rash, muscle pain and sometimes debilitating pain in multiple joints. There are no vaccines approved to prevent chikungunya infection or disease.
Moderna Inc. is a clinical stage biotechnology company headquartered in Cambridge, Mass., engaged in transformative medicines based on messenger ribonucleic acid (mRNA). It is pursuing mRNA science to minimize the undesirable activation of the immune system by mRNA and to maximize the potency of mRNA once in the target cells. The company has a diverse development pipeline of 22 programs with multiple clinical studies underway. Its therapeutics and vaccine development programs span infectious diseases, oncology, cardiovascular diseases and rare genetic diseases. The company currently has strategic alliances for development programs with AstraZeneca, Plc. and Merck, Inc. and the Defense Advanced Research Projects Agency.
MRNA share opened today at $17.34 (+$1.42, +8.92%) over yesterday's $15.92 closing price. The stock has traded today between $16.23 and $18.46/share and at present is trading at $16.84 (+$0.92, +5.78%).[NLINSERT]
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