French biotech lands man­u­fac­tur­ing deal in step to­ward com­mer­cial­iza­tion of rare bone dis­ease drug

As a start­up, it can some­times be tough to think too far ahead in the fu­ture. How­ev­er, with a ma­jor man­u­fac­tur­ing part­ner­ship an­nounced Wednes­day, CTO Ca­r­ole Schwint­ner and her team at MaaT Phar­ma are tak­ing the first steps to­ward es­tab­lish­ing a long-term game plan for the sev­en-year-old com­pa­ny.

The French biotech and CD­MO Skyephar­ma will part­ner to build the largest spe­cial­ized man­u­fac­tur­ing fa­cil­i­ty for mi­cro­bio­me-based ther­a­peu­tics. The plant will help MaaT in­crease its out­put by ten-fold by 2030, with the aim to pro­duce sev­er­al thou­sand en­e­mas of the lead can­di­date MaaT-013, to treat Graft-ver­sus-Host dis­ease (GvHD), which hap­pens when do­nat­ed bone mar­row or stem cells view the re­cip­i­ent’s body as for­eign, and do­nat­ed cells and bone mar­row at­tack the body. It will al­so pro­duce “sev­er­al hun­dreds of thou­sands cap­sules” of MaaT-033, for pa­tients who re­ceive an al­lo­gene­ic stem cell trans­plan­ta­tion.

Ca­r­ole Schwint­ner

Through the part­ner­ship, the two com­pa­nies will build a 1,500-square-me­ter man­u­fac­tur­ing site in France, with the po­ten­tial to dou­ble in size if need­ed to in­crease man­u­fac­tur­ing ca­pac­i­ty. The let­ter of in­tent was signed in Sep­tem­ber 2021, the com­pa­nies dis­closed in a re­lease, and fi­nan­cial terms are still un­der wraps. The site will be op­er­a­tional by 2023 and will host MaaT Phar­ma’s team and equip­ment. Skyephar­ma, mean­while, will lend MaaT ex­per­tise in prod­uct qual­i­ty, reg­u­la­to­ry af­fairs and large-scale pro­duc­tion. MaaT al­so will ben­e­fit from Skyephar­ma’s ex­per­tise on de­layed-re­lease for­mu­la­tion. While MaaT could have fo­cused on its own man­u­fac­tur­ing plans, the new deal al­lows MaaT to fo­cus on what it knows best: its pipeline.

“(Skyephar­ma has) al­ready many years of ex­pe­ri­ence. Their sites have been al­ready in­spect­ed by the French, Amer­i­cans, and sev­er­al oth­er in­spec­tors,” Schwint­ner said in an in­ter­view with End­points News. “It’s im­por­tant for us to have some­thing re­al­ly sol­id, and they bring this part of ex­per­tise on top of it…Rather than try­ing to build an en­tire­ly new thing, they have some­thing al­ready its been chal­lenged and val­i­dat­ed.”

Last month, MaaT com­plet­ed its Phase II/III tri­als ear­ly, and da­ta from four out of five in­tend­ed co­horts showed sat­is­fac­to­ry safe­ty and “good mi­cro­bio­me en­graft­ment” de­ter­mined by the pres­ence of spe­cif­ic gut bac­te­ria.

Through the deal, MaaT will ac­cel­er­ate MaaT-03X, which us­es a mi­cro­bio­me ecosys­tem co-fer­men­ta­tion tech­nol­o­gy, and re­cent­ly was award­ed two pub­lic grants.

MaaT has moved can­di­dates in­to six dif­fer­ent clin­i­cal tri­als in its sev­en years of ex­is­tence and should be sign­ing up its first par­tic­i­pant in MaaT-013’s Phase III tri­al any day now. Its can­di­date us­es mi­cro­bio­me-based ther­a­peu­tics to flush good gut bac­te­ria in­to a pa­tient’s sys­tem through an en­e­ma for­mu­la­tion. The com­pa­ny prides it­self on the pre­ci­sion of its donor screen­ing, qual­i­ty con­trol and di­ver­si­ty of bac­te­ria in its prod­uct. That is for­mu­lat­ed us­ing fe­ces from healthy donors.

The com­pa­ny land­ed €18 mil­lion in its Se­ries B fi­nanc­ing round in Feb­ru­ary 2020.

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.

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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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.

David Light, Valisure CEO

Val­isure in the hot seat: New Form 483 over a 2021 in­spec­tion as CEO fires back

The notorious drug testing company Valisure, which has made a name for itself by forcing FDA’s hand with some of its safety-related uncoverings, received a letter this week after the FDA uncovered violations at its Connecticut-based testing lab in 2021.

The letter, which was sent on Dec. 5, stated that the FDA is “concerned” that Valisure was not aware of  drug supply chain security requirements.

Bags of shred­ded docs: In­di­an drug­mak­er Lupin hand­ed a Form 483 by FDA in­spec­tors

The generics manufacturer Lupin has been given another Form 483 from the FDA this year.

US regulators inspected Lupin’s pharmaceutical manufacturing site in the town of Mandideep, India from Nov. 14 through Nov. 23, with the 14-page report marking 16 observations.

The inspection report stated that the site did not have the appropriate controls over its computer systems to ensure that changes in “master production” or records are only done by authorized personnel, along with written procedures not being established to conduct annual reviews of records associated with drug batches.

Mark Cuban (Jed Jacobsohn/AP Images)

Mov­ing to the em­ploy­er side of health­care, Mark Cuban's Cost Plus Drugs part­ners with a PBM

From “Shark Tank” to direct-to-consumer generic drugs, Mark Cuban has made another inroad in the ongoing battle over prescription drug prices. His cost-plus-15% generic drug company, frequently undercutting many competitors, now has its sights set on the employer healthcare market.

Cost Plus Drugs, which originally pledged to cut out PBMs, has now partnered with the PBM EmsanaRx, majority owned by the Purchaser Business Group on Health, to launch a supplemental drug discount program designed specifically for self-funded employers, the company announced Thursday.

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.

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.