Re­flect­ing on More Than a Decade of Progress on mR­NA Sci­ence

Sep­tem­ber marked the 12th an­niver­sary of Mod­er­na’s found­ing, when we set out to study whether it is pos­si­ble to in­struct a pa­tient’s own cells to pro­duce pro­teins that may have the po­ten­tial to treat a wide range of con­di­tions with mR­NA.

Since then, we have built a di­verse clin­i­cal port­fo­lio of mR­NA vac­cines and ther­a­peu­tics across sev­en modal­i­ties. We have pi­o­neered the de­vel­op­ment of our mR­NA plat­form and have ex­pand­ed our mR­NA pipeline to 47 pro­grams in de­vel­op­ment in­clud­ing 31 in clin­i­cal stud­ies.

We be­lieve our mR­NA plat­form could po­ten­tial­ly solve some of the world’s great­est health chal­lenges—from dis­eases im­pact­ing mil­lions to med­i­cines per­son­al­ized down to the in­di­vid­ual lev­el.

What is mR­NA?

mR­NA is a nat­u­ral­ly oc­cur­ring part of the hu­man body, and it ex­ists in our cells. It func­tions as an in­for­ma­tion mol­e­cule, teach­ing the body how to make its own med­i­cines in the form of pro­tein.  Each mR­NA car­ries in­struc­tions like a blue­print to make a spe­cif­ic pro­tein. Cells in­ter­pret this blue­print and put the pro­tein to­geth­er. Once mR­NA does its job, it is bro­ken down by the body. Mod­er­na’s mR­NA tech­nol­o­gy al­lows its sci­en­tists to cre­ate in­struc­tions to make dif­fer­ent pro­teins that could help the body fight in­fec­tions or pre­vent dis­eases.

Per­son­al­ized Can­cer Vac­cine (PCV)

Per­son­al­ized can­cer vac­cines tar­get an in­di­vid­ual pa­tient’s unique tu­mor mu­ta­tions to se­lec­tive­ly treat their can­cer. Mod­er­na’s PCV pro­gram is be­ing de­vel­oped in col­lab­o­ra­tion with Mer­ck and is de­signed to stim­u­late an im­mune re­sponse by boost­ing T cells, which are be­lieved to be nec­es­sary for re­cur­rence-free sur­vival.

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The Phase 2 study¹ is ful­ly en­rolled and pri­ma­ry da­ta are ex­pect­ed in the fourth quar­ter of 2022. The Phase 2 study will de­ter­mine if the in­ves­ti­ga­tion­al PCV in com­bi­na­tion with KEYTRU­DA (pem­brolizum­ab) can im­prove re­cur­rence-free sur­vival (at 12 months) in pa­tients with re­sect­ed melanoma at high risk of re­cur­rence, com­pared to KEYTRU­DA alone.

mR­NA Ther­a­peu­tic Can­di­dates for Rare Dis­eases

In rare dis­eases, our pro­grams aim to de­liv­er mR­NA in­to tar­get or­gan cells as a ther­a­peu­tic ap­proach for dis­eases caused by a miss­ing or de­fec­tive pro­tein. We are ex­cit­ed about a num­ber of clin­i­cal tri­als in this ther­a­peu­tic area.

The Phase 1/2 study² of our Pro­pi­onic Acidemia (PA) can­di­date is on­go­ing. The Phase 1/2 study³ of our Methyl­malonic Acidemia (MMA) pro­gram is on­go­ing and the first two groups of pa­tients are ful­ly en­rolled. We are re­cruit­ing par­tic­i­pants in the Unit­ed King­dom, Cana­da, and the U.S. Ini­tial da­ta from the tri­al are ex­pect­ed by 2023.

We are eval­u­at­ing⁴ the safe­ty, tol­er­a­bil­i­ty, and phar­ma­col­o­gy of a sin­gle IV dose of our ther­a­peu­tic can­di­date for GSD1a in adult par­tic­i­pants in a Phase 1 study. En­roll­ment is on­go­ing and ini­tial da­ta are ex­pect­ed in 2023.

Based on proof-of-con­cept da­ta and lever­ag­ing our learn­ings from our rare dis­ease pro­grams, we re­cent­ly an­nounced a new de­vel­op­ment can­di­date for or­nithine tran­scar­bamy­lase (OTC) de­fi­cien­cy, which us­es the same lipid nanopar­ti­cle (LNP) as our GSD1a pro­gram. This is our sixth rare dis­ease can­di­date.

Late-Stage Res­pi­ra­to­ry Vac­cine Can­di­dates

Our late-stage res­pi­ra­to­ry vac­cine pipeline con­tin­ues to progress. The Phase 3 study⁵ of one of our sea­son­al in­fluen­za vac­cine can­di­dates in the South­ern Hemi­sphere is ful­ly en­rolled with ap­prox­i­mate­ly 6,000 par­tic­i­pants. We are al­so prepar­ing to launch a Phase 3 ef­fi­ca­cy tri­al in the North­ern Hemi­sphere to test ef­fi­ca­cy com­pared to a cur­rent­ly li­censed sea­son­al in­fluen­za vac­cine.

The Phase 3 study⁶ of our RSV vac­cine can­di­date in adults over 60 years of age has en­rolled more than 24,000 of the 34,000-par­tic­i­pant tar­get. The study has been de­signed to pro­vide an ef­fi­ca­cy read­out ex­pect­ed in the 2022-2023 win­ter sea­son. Since RSV al­so caus­es a sig­nif­i­cant dis­ease bur­den in chil­dren, we are study­ing our RSV vac­cine can­di­date in an on­go­ing Phase 1 tri­al in pe­di­atric pop­u­la­tions.

Com­bi­na­tion Vac­cines Can­di­dates

We are al­so pro­gress­ing sev­er­al com­bi­na­tion res­pi­ra­to­ry vac­cine can­di­dates. The Phase 1/2 study⁷ of our com­bi­na­tion vac­cine can­di­date tar­get­ing SARS-CoV-2 and in­fluen­za is ful­ly en­rolled. We ex­pect to be­gin a clin­i­cal tri­al for a com­bi­na­tion vac­cine can­di­date tar­get­ing SARS-CoV-2, in­fluen­za, and RSV this year. By pur­su­ing com­bi­na­tion vac­cines to pro­tect against a range of dis­eases, we can po­ten­tial­ly help de­crease mor­bid­i­ty and mor­tal­i­ty from res­pi­ra­to­ry dis­ease, low­er sys­temic costs and in­crease health se­cu­ri­ty glob­al­ly.

Late-Stage La­tent Virus Vac­cine Can­di­date

Once a hu­man is in­fect­ed by a la­tent virus, virus re­mains in the body and can lead to life­long med­ical com­pli­ca­tions. We are com­mit­ted to de­vel­op­ing a port­fo­lio of vac­cine and ther­a­peu­tic can­di­dates against these la­tent virus­es. To date, we have en­rolled more than 40% of an­tic­i­pat­ed par­tic­i­pants in the Phase 3 study⁸ of our cy­tomegalovirus (CMV) vac­cine can­di­date. En­roll­ment is on­go­ing in the U.S. and in­ter­na­tion­al­ly.

The Fu­ture Po­ten­tial of mR­NA

Be­cause mR­NA is an in­for­ma­tion mol­e­cule, our hy­poth­e­sis has al­ways been this: if we in­vest in sci­ence over time, we may be able to cre­ate many ap­pli­ca­tions of this tech­nol­o­gy, po­ten­tial­ly bring­ing mR­NA to dif­fer­ent cell types. More than a decade lat­er, we be­lieve Mod­er­na will be­come one of the most im­pact­ful health­care com­pa­nies in the world.

For­ward-Look­ing State­ment Dis­claimer

This post con­tains for­ward-look­ing state­ments with­in the mean­ing of the Pri­vate Se­cu­ri­ties Lit­i­ga­tion Re­form Act of 1995, as amend­ed, in­clud­ing re­gard­ing: the po­ten­tial launch and ap­proval of ad­di­tion­al prod­ucts by Mod­er­na, in­clud­ing com­bi­na­tion vac­cines; the tim­ing for the re­lease of da­ta re­lat­ed to mR­NA-4157; the safe­ty and tol­er­a­bil­i­ty of Mod­er­na’s prod­ucts in clin­i­cal de­vel­op­ment; and the ad­vance­ment of clin­i­cal tri­als. The for­ward-look­ing state­ments in this post are nei­ther promis­es nor guar­an­tees, and you should not place un­due re­liance on these for­ward-look­ing state­ments be­cause they in­volve known and un­known risks, un­cer­tain­ties, and oth­er fac­tors, many of which are be­yond the Com­pa­ny’s con­trol and which could cause ac­tu­al re­sults to dif­fer ma­te­ri­al­ly from those ex­pressed or im­plied by these for­ward-look­ing state­ments. These risks, un­cer­tain­ties, and oth­er fac­tors in­clude those oth­er risks and un­cer­tain­ties de­scribed un­der the head­ing “Risk Fac­tors” in the Com­pa­ny’s most re­cent An­nu­al Re­port on Form 10-K or its Quar­ter­ly Re­port on Form 10-Q for the quar­ter end­ed March 30, 2022, each filed with the U.S. Se­cu­ri­ties and Ex­change Com­mis­sion (“SEC”) and in sub­se­quent fil­ings made by the Com­pa­ny with the SEC, which are avail­able on the SEC’s web­site at www.sec.gov. Ex­cept as re­quired by law, Mod­er­na dis­claims any in­ten­tion or re­spon­si­bil­i­ty for up­dat­ing or re­vis­ing any for­ward-look­ing state­ments con­tained in this post in the event of new in­for­ma­tion, fu­ture de­vel­op­ments or oth­er­wise. These for­ward-look­ing state­ments are based on Mod­er­na’s cur­rent ex­pec­ta­tions and speak on­ly as of the date here­of.

 Pro­grams out­lined in this ar­ti­cle are in clin­i­cal de­vel­op­ment and have not been ap­proved by FDA or any oth­er reg­u­la­to­ry agency for any in­di­ca­tion.

Ref­er­ences:

¹https://clin­i­cal­tri­als.gov/ct2/show/NCT03897881
²https://clin­i­cal­tri­als.gov/ct2/show/NCT04159103
³https://clin­i­cal­tri­als.gov/ct2/show/NCT04899310
⁴https://clin­i­cal­tri­als.gov/ct2/show/NCT05095727
⁵https://clin­i­cal­tri­als.gov/ct2/show/NCT05415462
⁶https://clin­i­cal­tri­als.gov/ct2/show/NCT05127434
⁷https://clin­i­cal­tri­als.gov/ct2/show/NCT05375838
⁸https://clin­i­cal­tri­als.gov/ct2/show/NCT05085366