Work taking place in the clean rooms at Vor (Credit: Vor)

Vor Bio opts to keep man­u­fac­tur­ing op­er­a­tions in-house for de­vel­op­ing stem cell, CAR-T ther­a­pies

While it is not un­com­mon for a biotech to go down the route of hav­ing the prod­uct man­u­fac­tured by a con­tract or­ga­ni­za­tion, one small biotech is look­ing to keep its card close to its chest.

Vor Bio­phar­ma has start­ed man­u­fac­tur­ing op­er­a­tions at an in-house fa­cil­i­ty at its HQ in Cam­bridge, MA af­ter be­gin­ning con­struc­tion last sum­mer.

Ac­cord­ing to the biotech, the fa­cil­i­ty aims to de­vel­op Vor’s hematopoi­et­ic stem cells (eHSCs) and CAR-T ther­a­pies for pa­tients with blood can­cers. The site will ini­tial­ly man­u­fac­ture a clin­i­cal sup­ply of its can­di­date VCAR33al­lo to sup­port its IND, which is slat­ed to be sub­mit­ted in the first half of next year. It al­so plans to trans­fer the pro­duc­tion of VOR33 to the fa­cil­i­ty. Vor is get­ting to work quick­ly as en­gi­neer­ing runs for VCAR33al­lo has start­ed this week.

Michael Pin­aud

In an email to End­points News, Michael Pin­aud, the head of GMP op­er­a­tions for Vor said that the fa­cil­i­ty is around 9,000 square feet. Due to the type of man­u­fac­tur­ing that the com­pa­ny is un­der­tak­ing, it can man­u­fac­ture its eHSCs with a small foot­print. Vor did not dis­close the fi­nan­cial fig­ures of the fa­cil­i­ty, but the site did not use any gov­ern­men­tal funds.

“We be­lieve that the up­front in­vest­ment is well worth the long-term ad­van­tages of hav­ing full con­trol over our man­u­fac­tur­ing ca­pac­i­ty, time­lines and costs,” Pin­aud said in the email to End­points.

The site is made up of four clean rooms along with two qual­i­ty con­trol lab­o­ra­to­ries, six an­a­lyt­i­cal lab­o­ra­to­ries and ware­hous­ing space. It is look­ing to scale up quick­ly as Pin­aud said the site is look­ing to hire in the ar­eas of qual­i­ty, man­u­fac­tur­ing op­er­a­tions and process de­vel­op­ment over the next few years. How­ev­er, no de­tails were giv­en on the num­ber of em­ploy­ees the com­pa­ny plans to add.

As to why the com­pa­ny is not con­tract­ing out its man­u­fac­tur­ing work, Pin­aud stressed that it al­lows Vor to main­tain full con­trol over its prod­uct.

“In­te­grat­ing in­ter­nal re­search, process de­vel­op­ment, an­a­lyt­i­cal de­vel­op­ment, man­u­fac­tur­ing and qual­i­ty con­trol test­ing ca­pa­bil­i­ties un­der one roof en­ables us to pro­vide ef­fi­cient trans­fer of knowl­edge and main­tain strate­gic con­trol of man­u­fac­tur­ing ca­pac­i­ty, and re­duce the time and cost re­quired to man­u­fac­ture com­plex cell ther­a­py clin­i­cal can­di­dates,” Pin­aud said in a state­ment.

Pin­aud al­so told End­points that the fa­cil­i­ty can sup­port all its pro­grams in its pipeline, but that Vor would even­tu­al­ly need to eval­u­ate ad­di­tion­al space to sup­port fu­ture com­mer­cial sup­ply.

When the site was an­nounced last year, then CTO Sadik Kas­sim told End­points in an in­ter­view that out­sourc­ing could give away trade se­crets, among oth­er rea­sons for bring­ing things un­der one roof.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; 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.

Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

Eu­ro­pean doc­tors di­al up dig­i­tal com­mu­ni­ca­tion with phar­mas, but still lean to­ward in-per­son med meet­ings, study finds

As in-person sales rep access declines in the big five European countries, a corresponding uptick in virtual rep access is happening. It’s not surprising, but it does run counter to pharma companies’ assessment – along with long-held sales rep sway in Europe – that in-person access hadn’t changed.

CMI Media Group and Medscape’s recent study reports that 75% of physicians in the EU5 countries of Spain, Germany, Italy, France and the UK already limit engagements with pharma sales reps, and 25% of those surveyed plan to decrease time with reps.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

Judge al­lows ex­pert tes­ti­mo­ny in GSK tri­al al­leg­ing Zan­tac link to can­cer

A California judge will allow a plaintiff in a state court case to introduce expert testimony connecting a potential carcinogen in former blockbuster medicine Zantac to cancer.

The order was handed down on Thursday from state judge Evelio Grillo, who is now allowing both parties to introduce expert testimony in an upcoming trial after what’s known as a Sargon hearing, where a judge determines the admissibility of expert witnesses and expert opinions.

Mark Womack, BioCina CEO

Q&A: BioCi­na’s new CEO Mark Wom­ack on the CD­MO he says is 'worth trav­el­ing over'

A handful of CDMOs have made changes at the top over the past few weeks, including Genezen and Curia.

That also includes Australian CDMO BioCina, which announced last week that Mark Womack would be taking the helm. Womack previously served as chief business officer at AGC Biologics, CEO of Indian manufacturer Stelis Biopharma and most recently, CEO at CDMO KBI Biopharma and Selexis SA.

BioCina completed the takeover of a Pfizer manufacturing facility in Adelaide in 2021 and is now prepping for wider growth. Endpoints News sat down with Womack to discuss his new role, plans for the future, and how to compete in the wider CDMO market. This interview has been edited for brevity and clarity.