Man­u­fac­tur­ing roundup: Lon­za fin­ish­es ex­pand­ing Visp site; CBM to pro­duce jCyte’s cell ther­a­py

Lon­za has opened the doors to an ex­pan­sion at one of its pri­ma­ry lo­ca­tions.

The ex­pan­sion oc­curred at Lon­za’s high­ly po­tent API (HPA­PIs) suite at its core man­u­fac­tur­ing site in the city of Visp, Switzer­land. The ex­pan­sion adds room for the de­vel­op­ment and man­u­fac­tur­ing of Lon­za’s ADC prod­ucts, which are be­com­ing more pop­u­lar.

“We ex­pand­ed our HPA­PI ca­pa­bil­i­ties at our Visp site to meet the grow­ing de­mand for AD­Cs. This de­mand is dri­ven by the need for im­proved an­ti­cancer ther­a­peu­tics with few­er side ef­fects. Al­though every com­po­nent of the ADC is es­sen­tial to its ther­a­peu­tic ac­tiv­i­ty, the pay­load is the one that kills can­cer cells.

“These pay­loads are al­most al­ways small mol­e­cule high­ly po­tent APIs,” said Gio­van­na Li­bralon, se­nior di­rec­tor for com­mer­cial de­vel­op­ment of small mol­e­cules at Lon­za in a state­ment to End­points News.

While the ex­act cost and size of the fa­cil­i­ty were not dis­closed to End­points, Li­bralon did state that the project is ex­pect­ed to gen­er­ate more jobs, how­ev­er, no hard num­bers were giv­en.

The Cen­ter for Break­through Med­i­cine to man­u­fac­ture jCyte’s cell ther­a­py

The Cen­ter for Break­through Med­i­cine (CBM), a CD­MO spe­cial­iz­ing in cell and gene ther­a­py, has land­ed a mul­ti-year con­tract to pro­duce jCyte’s cell ther­a­py.

The deal will have CBM pro­duce the ma­te­ri­als for the biotech’s Phase III and man­u­fac­ture the project once it gets BLA ap­proval. It will al­so pro­vide CMC work for jC­tye as it goes through the reg­u­la­to­ry and com­mer­cial phas­es for its treat­ment.

The biotechs ther­a­py is de­signed to treat pa­tients with re­tini­tis pig­men­tosa (RP), a rare ge­net­ic dis­ease main­ly found in chil­dren that can lead to vi­sion loss. The ther­a­py al­so has an RMAT des­ig­na­tion from the FDA.

“We have made tremen­dous progress in ad­vanc­ing our in­no­v­a­tive cell ther­a­py pro­gram to this key stage of clin­i­cal de­vel­op­ment, and CBM’s broad man­u­fac­tur­ing ca­pa­bil­i­ties, ded­i­cat­ed and ex­pe­ri­enced team, flex­i­bil­i­ty to fit our needs, and abil­i­ty to pro­vide ful­ly ded­i­cat­ed man­u­fac­tur­ing suites and an­a­lyt­i­cal test­ing ser­vices tai­lored to our fi­nal drug man­u­fac­tur­ing process made them an ide­al part­ner to sup­port our pro­gram,” said Shan­non Blalock, jCyte CEO in a state­ment.

More funds go­ing to­ward large-scale cell and gene ther­a­py man­u­fac­tur­ing in Cana­da

Man­u­fac­tur­ers in Cana­da are re­ceiv­ing a fund­ing boost to get cell and gene ther­a­py man­u­fac­tur­ing on a wider scale.

Next Gen­er­a­tion Man­u­fac­tur­ing Cana­da (NGen) will con­tribute $10.5 mil­lion to a $34.8 mil­lion project led by Om­ni­aBio and its part­ners. The project aims to de­vel­op tech­nolo­gies for the man­u­fac­tur­ing of cell and gene ther­a­pies in Cana­da and five biotechs have been brought on to the ini­tia­tive.

The project is de­signed to have part­ner­ing com­pa­nies ad­vance cell and gene ther­a­py pro­grams to move to com­mer­cial pro­duc­tion. The ef­fort will try to prove that Cana­da can han­dle large-scale cell and gene ther­a­py man­u­fac­tur­ing. How­ev­er, the part­ner­ship has not dis­closed when pro­duc­tion is ex­pect­ed to progress for­ward.

The in­vest­ment will how­ev­er pro­vide a re­source to pro­vide train­ing in this par­tic­u­lar kind of man­u­fac­tur­ing to the wider work­force.

“This in­vest­ment in crit­i­cal bio­man­u­fac­tur­ing in­fra­struc­ture en­sures that we will be able to lever­age our strengths in re­search and tech­nol­o­gy, build a work-ready tal­ent pipeline, and pro­vide the right con­di­tions for our do­mes­tic CGT ven­tures to scale up in Cana­da,” said Jayson My­ers, CEO of NGen, in a state­ment.

Om­ni­aBio an­nounced ear­li­er this year that it is build­ing a $580 mil­lion cell and gene ther­a­py man­u­fac­tur­ing cen­ter in Hamil­ton, On­tario.

Ther­mo Fish­er part­ners up with a re­gion­al or­ga­ni­za­tion to in­crease life sci­ences in the Philadel­phia area

While Ther­mo Fish­er has been busy open­ing new plants, its next move sees it part­ner­ing to in­vest in fu­ture life sci­ence ini­tia­tives.

The com­pa­ny has part­nered with the Penn­syl­va­nia Biotech­nol­o­gy Cen­ter (PABC) to spon­sor its new cam­pus in Doylestown, PA, a sub­urb of Philadel­phia. Ther­mo Fish­er will pro­vide ex­per­tise and tech­nol­o­gy to biotechs at the cam­pus and an­oth­er in­cu­ba­tor run by PABC.

“Through their in­cu­ba­tor sites, PABC can ac­cel­er­ate the path to com­mer­cial­iza­tion, and our in­vest­ment en­sures that these com­pa­nies start with the com­mer­cial-scale tech­nolo­gies and work­flows that will be re­quired as they move from R&D to the clin­ic,” said Ab­hi­nav Akhoury, VP of cor­po­rate ac­counts at Ther­mo Fish­er, in a state­ment.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Bo Cumbo, new Solid Bio CEO

Sol­id Bio gets a new CEO, $75M cash and drops lead drug as big in­vestors ju­ry-rig a merg­er deal

Three months after Endpoints News broke the story that gene therapy outfit AavantiBio had gutted its CMC group in a reorganization, the biotech’s CEO has helped engineer a merger with struggling penny stock player Solid Bio. And he’s going to remain in charge of the combined operation, as Solid founder Ilan Ganot steps aside.

The merger news this morning features some high-profile investors.

Perceptive Advisors, RA Capital Management and Bain Capital Life Sciences are leading a $75 million raise to add to the pool of cash Solid will have after the tie-up. That will leave Solid $215 million in cash as Bain’s Adam Koppel jumps on the board — enough to pay for ops and get through some key data milestones on their way into 2025.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.