Metagenomi Reports Business Updates and Full Year 2023 Financial Results

– Demon­strat­ed non­hu­man pri­mate proof of con­cept in Hemo­phil­ia A pro­gram; pro­gress­ing towards Devel­op­ment Can­di­date nom­i­na­tion by mid-year; 12-month non­hu­man pri­mate dura­bil­i­ty data in 2H 2024 –

– Proof of capa­bil­i­ty demon­strat­ed with CRISPR-asso­ci­at­ed trans­posase (CAST) sys­tem being devel­oped to enable >10,000 base pair, tar­get­ed genom­ic integrations –

– Ultra-small base edit­ing sys­tems (SMART) are the small­est nick­ase-based sys­tems char­ac­ter­ized to-date, designed to enable more effi­cient deliv­ery via sin­gle ade­no-asso­ci­at­ed virus­es (AAVs) –

– Clos­ing of ini­tial pub­lic offer­ing pro­vides cash run­way to sup­port 2 INDs and 2 addi­tion­al Devel­op­ment Can­di­date nominations –

Metageno­mi, Inc. (Nas­daq: MGX), a pre­ci­sion genet­ic med­i­cines com­pa­ny com­mit­ted to devel­op­ing cura­tive ther­a­peu­tics for patients using its pro­pri­etary, com­pre­hen­sive metage­nomics-derived gene edit­ing tool­box, today report­ed finan­cial results for the full year end­ed Decem­ber 31, 2023, and addi­tion­al busi­ness updates. 

We con­tin­ue to exe­cute across all aspects of our busi­ness. Through­out 2023, we achieved key ther­a­peu­tic mile­stones, includ­ing pre­clin­i­cal proof of con­cept in non­hu­man pri­mates for our lead pro­gram in Hemo­phil­ia A. Addi­tion­al­ly, we announced a series of indus­try firsts on our tech­nol­o­gy plat­form, includ­ing the demon­stra­tion of large, com­plex gene cor­rec­tions,” said Bri­an C. Thomas, Chief Exec­u­tive Offi­cer of Metageno­mi. Look­ing ahead, we are plan­ning for addi­tion­al non­hu­man pri­mate data read­outs over the course of this year, includ­ing 12 month data for our Hemo­phil­ia A pro­gram, expect­ed in the sec­ond half of 2024.” 

Recent Program Highlights:

Wholly-owned Pipeline:

In Q4 2023, we achieved inte­gra­tion of a func­tion­al Fac­tor VIII gene in non­hu­man pri­mates, result­ing in clin­i­cal­ly rel­e­vant cir­cu­lat­ing lev­els of Fac­tor VIII up through 4.5 months of follow-up.

We believe this mile­stone is ground­break­ing for the hemo­phil­ia com­mu­ni­ty, val­i­dates our edit­ing tech­nol­o­gy, and pro­vides a ready plat­form for sta­ble deliv­ery of oth­er secret­ed proteins.

We are pro­gress­ing towards a Devel­op­ment Can­di­date nom­i­na­tion for this pro­gram, antic­i­pat­ed in the mid­dle of 2024, and plan to present 12-month dura­bil­i­ty data for Fac­tor VIII expres­sion in the sec­ond half of 2024.

Key partnership updates:

Ion­is com­plet­ed tar­get selec­tion of all 4 tar­gets in Wave 1 of our col­lab­o­ra­tion, nom­i­nat­ing the remain­ing 2 tar­gets in addi­tion to Transthyretin Amy­loi­do­sis and Car­dio­vas­cu­lar Dis­ease (AGT).

Recent Technology Platform Highlights:

The abil­i­ty of our metage­nomics dis­cov­ery plat­form to iden­ti­fy nov­el enzymes with high activ­i­ty makes it pos­si­ble to rapid­ly devel­op dif­fer­en­ti­at­ed tech­nol­o­gy that sur­pass­es first-gen­er­a­tion systems.

Big RIGS (RNA-mediated integration systems):

Using pro­pri­etary reverse tran­scrip­tases we demon­strat­ed what we believe to be the first-ever tar­get­ed inte­gra­tion of >900 bp in human cells with our big RIGS with all-RNA delivery.

We con­sid­er this to be a major step for­ward in the gene edit­ing space, as these sys­tems could be deliv­ered entire­ly as RNA, com­pat­i­ble with cur­rent LNP deliv­ery tech­nolo­gies, and could enable large, tar­get­ed exoge­nous gene integrations.

CAST (CRISPR-associated transposases):

We demon­strat­ed ini­tial pre­clin­i­cal proof-of-capa­bil­i­ty for our CASTs with a tar­get­ed inte­gra­tion of a large DNA tem­plate in the genome of mam­malian cells. Our CASTs are being devel­oped in order to enable large (>10,000 base pairs), tar­get­ed genom­ic inte­gra­tions for ther­a­peu­tic applications.

Base Editing, including ultra-small (SMART) systems: 

Our base edi­tors are high­ly active and pro­gress­ing towards in vivo ther­a­peu­tic appli­ca­tions. Using PAM inter­act­ing domain engi­neer­ing, we expand­ed the genome tar­getabil­i­ty of our base edi­tors by 5‑fold com­pared to SpCas9 base editors. 

We iden­ti­fied a series of nov­el, high­ly effi­cient ultra-small nucle­as­es and demon­strat­ed our abil­i­ty to engi­neer these sys­tems into ultra-small base edi­tors and prime edi­tors. We believe our SMART base edi­tors which are as small as 623 amino acids, rep­re­sent some of the small­est edit­ing sys­tems in the indus­try. As the size of these sys­tems are well-with­in the pack­ag­ing lim­its of AAV vec­tors, our SMART base edi­tors could great­ly expand both the deliv­ery and tar­getabil­i­ty options of these sys­tems for dis­ease tar­gets out­side the liver.

Recent Corporate Highlights:

Initial public offering:

On Feb­ru­ary 13, 2024, Metageno­mi announced the clos­ing of its ini­tial pub­lic offer­ing of 6,250,000 shares of its com­mon stock at a price to the pub­lic of $15.00 per share. The aggre­gate gross pro­ceeds to Metageno­mi from the offer­ing were approx­i­mate­ly $93.75 mil­lion, before deduct­ing under­writ­ing dis­counts and com­mis­sions and offer­ing expens­es. Our cash and cash equiv­a­lents and avail­able-for-sale mar­ketable secu­ri­ties, includ­ing the pro­ceeds from our ini­tial pub­lic offer­ing, pro­vide us with a cash run­way to sup­port 2 INDs and 2 addi­tion­al Devel­op­ment Can­di­date nominations.

Our shares began trad­ing on the Nas­daq Glob­al Select Mar­ket on Feb­ru­ary 9, 2024 under the tick­er sym­bol​“MGX”.

Strengthened leadership team with key appointments:

Over the course of 2023, we strength­ened our lead­er­ship team with the appoint­ment of sev­er­al key exec­u­tives, focused on dri­ving for­ward our entry into clin­i­cal devel­op­ment, con­tin­u­ing our lead­er­ship in gene edit­ing and expand­ing busi­ness oper­a­tions. These include appoint­ments of Sarah Noon­berg, MD, PhD, as Chief Med­ical Offi­cer, Luis Borges, PhD, as Chief Sci­en­tif­ic Offi­cer and Pamela Wap­nick, MBA, as Chief Finan­cial Officer.

Full Year 2023 Financial Results:

Cash Posi­tion: Cash and cash equiv­a­lents and avail­able-for-sale mar­ketable secu­ri­ties were $271.2 mil­lion as of Decem­ber 31, 2023, which does not include the $81.1 mil­lion in net pro­ceeds from our IPO com­plet­ed in Feb­ru­ary 2024. In addi­tion, cash used to fund our oper­a­tions was $91.4 mil­lion for the year end­ed Decem­ber 312023

R&D Expens­es: Research and devel­op­ment (R&D) expens­es were $94.4 mil­lion for the full year end­ed Decem­ber 312023.

G&A Expens­es: Gen­er­al and admin­is­tra­tive (G&A) expens­es were $28.8 mil­lion for the full year end­ed Decem­ber 312023.

Please refer to FY 2023 con­densed finan­cial state­ments in the full press release here: https://​www​.globe​newswire​.com/​n​e​w​s​-​r​e​l​e​a​s​e​/​2024​/​03​/​27​/​2853262​/​0​/​e​n​/​M​e​t​a​g​e​n​o​m​i​-​R​e​p​o​r​t​s​-​B​u​s​i​n​e​s​s​-​U​p​d​a​t​e​s​-​a​n​d​-​F​u​l​l​-​Y​e​a​r​-​2023​-​F​i​n​a​n​c​i​a​l​-​R​e​s​u​l​t​s​.html

About Metageno­mi

Metageno­mi is a pre­ci­sion genet­ic med­i­cines com­pa­ny com­mit­ted to devel­op­ing cura­tive ther­a­peu­tics for patients using its pro­pri­etary, com­pre­hen­sive metage­nomics-derived tool­box. Metageno­mi is har­ness­ing the pow­er of metage­nomics, the study of genet­ic mate­r­i­al recov­ered from the nat­ur­al envi­ron­ment, to unlock four bil­lion years of micro­bial evo­lu­tion to dis­cov­er and devel­op a suite of nov­el edit­ing tools capa­ble of cor­rect­ing any type of genet­ic muta­tion found any­where in the genome. Its com­pre­hen­sive genome edit­ing tool­box includes pro­gram­ma­ble nucle­as­es, base edi­tors, and RNA and DNA-medi­at­ed inte­gra­tion sys­tems (includ­ing prime edit­ing sys­tems and clus­tered reg­u­lar­ly inter­spaced short palin­dromic repeat asso­ci­at­ed trans­posas­es. Metageno­mi believes its diverse and mod­u­lar tool­box posi­tions the com­pa­ny to access the entire genome and select the opti­mal tool to unlock the full poten­tial of genome edit­ing for patients. For more infor­ma­tion, please vis­it https://​metageno​mi​.co.

Cau­tion­ary Note Regard­ing Forward‐​Looking Statements

This press release con­tains​“for­ward-look­ing state­ments” with­in the mean­ing of Sec­tion 27A of the Secu­ri­ties Act of 1933 and Sec­tion 21E of the Secu­ri­ties Exchange Act of 1934, each as amend­ed. Such state­ments, which are often indi­cat­ed by terms such as​“antic­i­pate,”​“believe,”​“could,”​“esti­mate,”​“expect,”​“goal,”​“intend,”​“look for­ward to,”​“may,”​“plan,”​“poten­tial,”​“pre­dict,”​“project,”​“should,”​“will,”​“would” and sim­i­lar expres­sions, include, but are not lim­it­ed to, any state­ments relat­ing to our growth strat­e­gy and prod­uct devel­op­ment pro­grams, includ­ing the tim­ing of and our abil­i­ty to con­duct IND-enabling stud­ies, make reg­u­la­to­ry fil­ings such as INDs and oth­er appli­ca­tions and to obtain reg­u­la­to­ry approvals for our prod­uct can­di­dates, state­ments con­cern­ing the poten­tial of ther­a­pies and prod­uct can­di­dates, and any oth­er state­ments that are not his­tor­i­cal facts. For­ward look­ing state­ments are based on management’s cur­rent expec­ta­tions and are sub­ject to risks and uncer­tain­ties that could neg­a­tive­ly affect our busi­ness, oper­at­ing results, finan­cial con­di­tion, and stock val­ue. Fac­tors that could cause actu­al results to dif­fer mate­ri­al­ly from those cur­rent­ly antic­i­pat­ed include: risks relat­ing to our growth strat­e­gy; our abil­i­ty to obtain, per­form under, and main­tain financ­ing and strate­gic agree­ments and rela­tion­ships; risks relat­ing to the results of research and devel­op­ment activ­i­ties; risks relat­ing to the tim­ing of start­ing and com­plet­ing clin­i­cal tri­als; uncer­tain­ties relat­ing to pre­clin­i­cal and clin­i­cal test­ing; our depen­dence on third par­ty sup­pli­ers; our abil­i­ty to attract, inte­grate and retain key per­son­nel; the ear­ly stage of prod­ucts under devel­op­ment; our need for sub­stan­tial addi­tion­al funds; gov­ern­ment reg­u­la­tion; patent and intel­lec­tu­al prop­er­ty mat­ters; com­pe­ti­tion; as well as oth­er risks described in​“Risk Fac­tors,” in our most recent Form 10‑K on file with the SEC. We express­ly dis­claim any oblig­a­tion or under­tak­ing to release pub­licly any updates or revi­sions to any for­ward-look­ing state­ments con­tained here­in to reflect any change in our expec­ta­tions or any changes in events, con­di­tions or cir­cum­stances on which any such state­ment is based, except as required by law, and we claim the pro­tec­tion of the safe har­bor for for­ward-look­ing state­ments con­tained in the Pri­vate Secu­ri­ties Lit­i­ga­tion Reform Act of 1995.

Investor Con­tact:

Simon Harnest — CIO, SVP Investor Relations


Media Con­tact:

Ash­lye Hodge — Com­mu­ni­ca­tions Manager