Ying Huang, Legend CEO

Lentivi­ral vec­tor ramp-up: J&J and Leg­end to in­vest $500M in New Jer­sey man­u­fac­tur­ing to sup­port Carvyk­ti

In re­sponse to a ques­tion on man­u­fac­tur­ing scale at Leg­end Biotech’s R&D day yes­ter­day, the com­pa­ny’s top ex­ec said its part­ner­ship with John­son & John­son will be dou­bling its in­vest­ment in its New Jer­sey man­u­fac­tur­ing cen­ter and will be in­vest­ing a to­tal of $500 mil­lion.

With an eye on their BC­MA-di­rect­ed CAR-T ther­a­py Carvyk­ti (cil­ta-cel), ap­proved in Feb­ru­ary as a fifth-line treat­ment for mul­ti­ple myelo­ma, Leg­end CEO Ying Huang said that the ramp-up in pro­duc­tion and the de­ci­sion to man­u­fac­ture its own lentivi­ral vec­tors — cur­rent­ly in short­age world­wide — means they won’t have to deal with that short­age.

“So far, Leg­end and Janssen have in­vest­ed $250 mil­lion in Rar­i­tan [New Jer­sey] and we will in­vest an­oth­er $250 mil­lion joint­ly,” a Leg­end spokesper­son added via email. “The sec­ond $250 mil­lion will be spent over the next 2-3 years to ex­pand the build­out of our New Jer­sey site to sup­port peak ca­pac­i­ty.”

The build­out is part of ef­forts to put the com­pa­nies in po­si­tion to ac­com­mo­date for $5 bil­lion-plus in peak pro­ject­ed sales for Carvyk­ti.

Huang said ear­li­er this year that the es­ti­mate, which is con­sid­er­ably high­er than Bris­tol My­ers Squibb’s es­ti­mate of $1 bil­lion for peak Abec­ma sales, is due to the ther­a­py’s po­ten­tial to reach ear­li­er lines of treat­ment.

SVB Se­cu­ri­ties an­a­lysts on Tues­day called the $500 mil­lion an­nounce­ment “the ex­treme side of cGMP fa­cil­i­ty ex­pens­es,” not­ing that the ex­pan­sion will help them

reach scale for their ear­li­er line tri­als, cit­ing an­tic­i­pat­ed ap­proval for cil­ta-cel in the 2L+ set­ting (based on CAR­TI­TUDE-4) as the pro­posed dead­line for the build-out. With this build-out, Leg­end plans to dou­ble their ap­proved treat­ment cen­ters to 70-80 cen­ters.

Carvyk­ti stole the show at AS­CO 2017 when Leg­end CSO Frank Fan and his team re­port­ed an im­pres­sive 100% over­all re­sponse rate among mul­ti­ple myelo­ma pa­tients. That was enough for Janssen to jump in with a $350 mil­lion cash deal to part­ner on the big can­cer can­di­date.

SVB Se­cu­ri­ties added, while es­ti­mat­ing a launch date for BMS and 2sev­en­ty’s Abec­ma (ide-cel) in third to fifth line mul­ti­ple myelo­ma in the first half of 2023, on the man­u­fac­tur­ing front:

Nei­ther 2sev­en­ty / BMY or Leg­end / JNJ have guid­ed COGS ex­pec­ta­tions for BC­MA CAR-T in the long term. 2sev­en­ty man­age­ment has guid­ed that ide-cel will be prof­itable to­wards the end of 2022 or ear­ly 2023, as they scale more dos­es on the base in­fra­struc­ture. We as­sume pro­gram prof­itabil­i­ty for ide-cel in 1Q23 and then COGS ramp-down, with in­creased ide-cel man­u­fac­tur­ing scale. For us, the speed and ex­tent of op­er­a­tional ef­fi­cien­cies are high­ly un­cer­tain.

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.