French can­cer-fo­cused mi­cro­bio­me play­er is flush with €18M Se­ries B in­jec­tion

Last year, the death of an im­muno-com­pro­mised el­der­ly pa­tient in a fe­cal mi­cro­bio­ta trans­plan­ta­tion tri­al — due to a do­na­tion that con­tained a rare type of E. coli bac­te­ria — sent shiv­ers across the field. The in­ci­dent marred an oth­er­wise ex­plod­ing field of drug de­vel­op­ment that backed re­plen­ish­ing the gut with good bac­te­ria as a safe and ef­fec­tive means to for­ti­fy the im­mune sys­tem to fight dis­ease.

Hervé Affa­gard

For France’s MaaT Phar­ma, an on­col­o­gy-fo­cused mi­cro­bio­me com­pa­ny, the set­back was al­most re­as­sur­ing.

“Bot­tom line is if they would have used our prod­uct, this pa­tient would not have died,” chief Hervé Affa­gard said in an in­ter­view with End­points News, sug­gest­ing that MaaT’s rig­or­ous test­ing stan­dards in­clude the screen­ing of drug-re­sis­tant bac­te­ria that would have pre­clud­ed the use of the rogue do­na­tion.

On Wednes­day the com­pa­ny — whose lead ex­per­i­men­tal prod­uct is an en­e­ma for­mu­la­tion de­signed to help pa­tients with acute graft-ver­sus-host dis­ease that have un­der­gone stem cell trans­plan­ta­tion — scored €18 mil­lion in Se­ries B fi­nanc­ing as it works on prov­ing its met­tle in a field crowd­ed with com­peti­tors fo­cused on a raft of dis­eases.

Mi­cro­bio­me-based ther­a­peu­tics to­day is a fe­cund field for drug de­vel­op­ers — big and small — cap­i­tal­iz­ing on sci­ence that sug­gests flush­ing ‘good’ gut bac­te­ria in­to the sys­tem can treat a pletho­ra of con­di­tions — from C. diff in­fec­tions to obe­si­ty — us­ing dif­fer­ent ther­a­peu­tic modal­i­ties, some of which are de­signed to side­step the “ick” fac­tor as­so­ci­at­ed with tra­di­tion­al stool trans­fer or fe­cal mi­cro­bio­ta trans­plan­ta­tion (FMT).

The con­cept of FMT was orig­i­nal­ly doc­u­ment­ed in Chi­na and has been used in the Unit­ed States since the 1950s with lit­tle reg­u­la­to­ry scruti­ny. In the last decade, the FDA sanc­tioned the use of FMT as a last re­sort mea­sure for re­cur­rent C. diff, but the agency con­tin­ues to con­sid­er it an in­ves­ti­ga­tion­al treat­ment. Glob­al­ly, hun­dreds of tri­als are now un­der­way test­ing the po­ten­tial of FMT for pa­tients suf­fer­ing from var­i­ous ill­ness­es, from autism to can­cer.

A few years ago, the spec­tac­u­lar fail­ure of Seres Ther­a­peu­tics’ sem­i­nal ef­fort in­to de­vel­op­ing a “crap­sule” — donor-de­rived processed fe­cal ma­te­r­i­al en­cap­su­lat­ed in a pill — de­railed an emerg­ing field work­ing to har­ness the in­sights gained from gut mi­cro­bio­ta to de­vel­op drugs. How­ev­er, the suc­cess of fe­cal trans­plant ther­a­pies to treat stub­born­ly re­cur­rent C. diff in­fec­tions has gained trac­tion, at­tract­ing a buck­et of biobucks and even in­spir­ing the takeover of a key play­er, Re­bi­otix.

MaaT Phar­ma prides it­self on its metic­u­lous process of donor screen­ing, qual­i­ty con­trol and di­ver­si­ty of bac­te­ria in its prod­uct — which like many oth­ers is for­mu­lat­ed us­ing fe­ces from healthy donors.

The Ly­on-based com­pa­ny’s lead for­mu­la­tion — MaaT013 — is cur­rent­ly in a mid-stage study in GvHD pa­tients. Da­ta from this tri­al are ex­pect­ed by the end of the year.

“It’s a kind of an im­muno-restora­tion, in­stead of im­muno­sup­pres­sion,” Affa­gard said of MaaT013, not­ing that all the drugs that have been de­vel­oped so far for GvHD pa­tients are im­muno­sup­pres­sive.

Late last year, MaaT re­port­ed en­cour­ag­ing MaaT013 da­ta from 8 pa­tients whose GvHD per­sist­ed de­spite up to five pre­vi­ous sys­temic treat­ments as part of a com­pas­sion­ate use pro­gram in hos­pi­tals. Each pa­tient ex­pe­ri­enced at least a par­tial re­sponse af­ter re­ceiv­ing MaaT013, while 3 out of 8 pa­tients at­tained a com­plete re­sponse, the com­pa­ny said.

“So we know our project is work­ing,” Affa­gard said. “Those pa­tients they’re re­ceiv­ing chemother­a­py, stem cell trans­plan­ta­tion…their mi­cro­bio­me was de­stroyed many times dur­ing their jour­ney.”

Next-gen­er­a­tion se­quenc­ing plat­forms and ad­vanced bioin­for­mat­ics ap­proach­es have stim­u­lat­ed re­search eval­u­at­ing the role of the gut mi­cro­bio­me in can­cer. In 2019, a con­sor­tium of ex­perts con­vened to dis­cuss the ev­i­dence un­der­ly­ing the as­so­ci­a­tion and found that while there are plau­si­ble mech­a­nisms and sup­port­ive ev­i­dence from in vit­ro, murine and cross-sec­tion­al hu­man stud­ies —di­rect ev­i­dence from large lon­gi­tu­di­nal co­hort stud­ies is lack­ing. In ef­fect, the role of the hu­man mi­cro­bio­me in the cause and sub­se­quent de­vel­op­ment of can­cer re­mains un­proven, the pan­el con­clud­ed, al­though a ma­jor­i­ty of pan­elists nev­er­the­less agreed with the hy­poth­e­sis.

MaaT is go­ing to use the fresh in­jec­tion of funds to prove its bet on the as­so­ci­a­tion. The mon­ey will be used for the on­go­ing Phase II en­e­ma study, as well as a cap­sule for­mu­la­tion that is set to be test­ed in a tri­al this year. The com­pa­ny is al­so ex­plor­ing the po­ten­tial of “restor­ing a bal­anced mi­cro­bio­me” to im­prove the clin­i­cal out­comes of check­point in­hibitors, with plans for a com­bi­na­tion tri­al in sol­id tu­mors.

The Se­ries B round in­clud­ed the par­tic­i­pa­tion of US in­vestor Sym­Bio­sis as well as sup­port from MaaT’s ex­ist­ing in­vestors Sev­en­ture Part­ners, Crédit Mutuel In­no­va­tion and Biocodex.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

Ab­b­Vie slapped with age dis­crim­i­na­tion law­suit, fol­low­ing oth­er phar­mas

Add AbbVie to the list of pharma companies currently facing age discrimination allegations.

Pennsylvania resident Thomas Hesch filed suit against AbbVie on Wednesday, accusing the company of passing him over for promotions in favor of younger candidates.

Despite 30 years of pharma experience, “Hesch has consistently seen younger, less qualified employees promoted over him,” the complaint states.

Nashville-based CD­MO nets a $65M Se­ries B to ex­pand fa­cil­i­ty and ca­pa­bil­i­ties

Another $65 million is music to the ears of the team at August Bioservices, a contract manufacturer in Nashville.

The company announced the Series B round last week, which will fund equipment in a new building expected to open in 2023, according to CEO Jenn Adams. It was led by Oak HC/FT, the same firm that led August’s Series A round in July 2020.

August Bioservices, a producer of materials such as prefilled syringes, IV bags and vials, was formed back in 2020 after the acquisition of PMI BioPharma Solutions, also based in Nashville. Adams said the goal was to build a business that could “address the scarcity of supply relative to sterile injectable manufacturing based in the US” and provide a broad range of manufacturing services.

Rami Elghandour, Arcellx CEO

Up­dat­ed: Gilead, Ar­cel­lx team up on an­ti-BC­MA CAR-T as biotech touts a 100% re­sponse rate at #ASH22

Gilead and Kite are plunking down big cash to get into the anti-BCMA CAR-T game.

The pair will shell out $225 million in cash upfront and $100 million in equity to Arcellx, Kite announced Friday morning, to develop the biotech’s lead CAR-T program together. Kite will handle commercialization and co-development with Arcellx, and profits in the US will be split 50-50.

Concurrent with the deal, Arcellx revealed its latest cut of data for the program known as CART-ddBCMA, ahead of a full presentation at this weekend’s ASH conference — a 100% response rate among patients getting the therapy. Investors jumped at the dual announcements, sending Arcellx shares $ACLX up more than 25% in Friday’s morning session.

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WIB22: Lead­ing NK cell re­searcher re­flects on roots in Iran, the UK and Texas

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

In a small but widely-cited 11-person study published in NEJM in 2020, seven patients saw signs of their cancer completely go away after getting a new therapy made from natural killer cells. The study was one of the earliest to provide clinical proof that the experimental treatment method had promise.

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