As gene edit­ing ex­plodes, a new re­port from Gold­man says Chi­nese groups are seiz­ing the lead on CRISPR and CAR-T stud­ies

Con­trary to what you might be­lieve, bio­phar­ma com­pa­nies in the US are not the lead­ers in clin­i­cal tri­als us­ing gene edit­ing tech. While a slate of US/Eu­ro­pean pi­o­neers have been lin­ing up their first hu­man tri­als, in­ves­ti­ga­tors in Chi­na are al­ready well ahead in test­ing CRISPR-edit­ed cells in hu­mans, ac­cord­ing to a deep dive on gene ther­a­pies from Gold­man Sachs an­a­lyst Salveen Richter.

“As of the end of Feb­ru­ary 2018,” Richter and her team re­port­ed, “there were nine reg­is­tered clin­i­cal stud­ies test­ing CRISPR-edit­ed cells to treat var­i­ous can­cers and HIV in­fec­tion in Chi­na, vs. on­ly one study in the U.S. All of the stud­ies were ini­ti­at­ed / spon­sored by top-tier pub­lic hos­pi­tals across Chi­na, and >80 pa­tients were re­port­ed as be­ing treat­ed by these in­ves­ti­ga­tion­al genome med­i­cines.”

Last year alone, Richter adds, the Na­tion­al Nat­ur­al Sci­ence Foun­da­tion of Chi­na pro­vid­ed fund­ing for more than 90 CRISPR projects — more than 270 over the last 4 years. With no reg­u­la­tions on these gene edit­ing projects, hos­pi­tals in Chi­na in par­tic­u­lar have been quick to ac­cel­er­ate their CRISPR work, a sit­u­a­tion that might change soon if Chi­na’s main drug agency steps in and ap­plies the brakes, as Gold­man ex­pects will hap­pen.

And Chi­nese in­ves­ti­ga­tors are al­ready ri­val­ing the US in the to­tal num­ber of CAR-T stud­ies that are be­ing done. As of Feb­ru­ary, Gold­man Sachs count­ed 153 CAR-T tri­als in Chi­na, com­pared to 164 in the US, 73 in Eu­rope and 56 in all of the rest of the world com­bined.

The one com­pa­ny fur­thest out in front is Leg­end Biotech­nol­o­gy, which J&J paid $350 mil­lion to part­ner with as it en­tered the glob­al race to de­vel­op new ther­a­pies in the wake of his­toric ap­provals for Kym­ri­ah (No­var­tis) and Yescar­ta (Gilead/Kite). And if these com­pa­nies and tri­als hit pay-dirt da­ta, as they promise to, Gold­man be­lieves that Chi­na will reap a con­sid­er­able ben­e­fit by sell­ing these ther­a­pies at a much low­er rate than the pi­o­neers on the mar­ket, spurring a sig­nif­i­cant amount of med­ical tourism.

What is this kind of mar­ket worth for the ri­vals look­ing to com­pete in it?

That de­pends on the sup­ply of pa­tients.

If your new genome med­i­cine ei­ther promis­es to cure a dis­ease or de­lay any fur­ther ther­a­py for a lengthy pe­ri­od of time, notes the Gold­man re­port, then your mar­ket could play out quick­ly as you race through the pa­tient pool that is avail­able. That’s what Gilead ex­pe­ri­enced when it came up with a pain­less cure for he­pati­tis C.

Not on­ly did the drugs from Gilead shrink the pool, Gold­man re­ports in a cold analy­sis of the num­bers, they al­so re­duced the num­ber of car­ri­ers present to keep spread­ing the dis­ease, fur­ther shrink­ing the mar­ket. From the re­port:

The po­ten­tial to de­liv­er “one shot cures” is one of the most at­trac­tive as­pects of gene ther­a­py, ge­net­i­cal­ly-en­gi­neered cell ther­a­py and gene edit­ing. How­ev­er, such treat­ments of­fer a very dif­fer­ent out­look with re­gard to re­cur­ring rev­enue ver­sus chron­ic ther­a­pies, par­tic­u­lar­ly in cer­tain dis­eases where it is pos­si­ble to cure a large pro­por­tion of the preva­lent pa­tient pool (or at least pre­vent an ad­di­tion­al dose from be­ing re­quired for an ex­tend­ed pe­ri­od). While this propo­si­tion car­ries tremen­dous val­ue for pa­tients and so­ci­ety, it could rep­re­sent a chal­lenge for genome med­i­cine de­vel­op­ers look­ing for sus­tained cash flow. Gilead is a case in point, where the suc­cess of its he­pati­tis C fran­chise has grad­u­al­ly ex­haust­ed the avail­able pool of treat­able pa­tients. 

The most lu­cra­tive dis­eases for these new cu­ra­tive ther­a­pies, Richter adds, would be big fields like he­mo­phil­ia or ar­eas where there’s a large sup­ply of new pa­tients each year — like can­cer. Spinal mus­cu­lar at­ro­phy, which af­flicts a stan­dard set of in­fants each year, is al­so vi­able. 

That may not be the kind of math that bio­phar­ma ex­ecs like to dis­cuss in pub­lic, but it’s cer­tain­ly the kind of equa­tions they re­view care­ful­ly while de­cid­ing how to spend R&D bud­gets.

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; 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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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Eliot Forster, F-star CEO (Rachel Kiki for Endpoints News)

F-star gets down to the wire with $161M sale to Chi­nese buy­er as na­tion­al se­cu­ri­ty con­cerns linger

With the clock ticking on F-star Therapeutics’ takeover by a Chinese buyer, the companies are still scrambling to remove a hold on the deal from the US government’s Committee on Foreign Investment in the United States.

F-star and invoX Pharma said they are “actively negotiating” with CFIUS “about the terms of a mitigation agreement to address CFIUS’s concerns regarding potential national security risks posed by the transaction.”

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CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

Rodney Rietze, iVexSol CEO

Bris­tol My­ers, Charles Riv­er join Se­ries A fund­ing for iVex­Sol

Massachusetts-based iVexSol has secured funding to the tune of $23.8 million in its latest Series A round. The new investors include Bristol Myers Squibb, manufacturer Charles River Laboratories and Asahi Kasei Medical.

iVexSol is a manufacturer of lentiviral vectors (LVV), used in making gene therapies, and this latest round of fundraising brings its total Series A total over $39 million, which will be used to recruit more employees and bolster its technology.

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John Rim, Samsung Biologics CEO (Samsung/PR Newswire)

Sam­sung Bi­o­log­ics spells out ex­pan­sion plans in South Ko­rea and US

The CDMO arm of one of South Korea’s largest conglomerates has posted its year-end results and plans for 2023, which include new construction.

Samsung Biologics netted north of KRW 3 trillion ($2.4 billion) in 2022 revenue and an operating profit of KRW 983.6 billion ($799 million), which the company touted on Friday as “record-high earnings.” The revenue boost was 55% compared to 2021.

No­var­tis' ap­proved sick­le cell dis­ease drug fails to beat place­bo in PhI­II

Novartis’ sickle cell drug, approved in 2019 and branded as Adakveo, has failed an ongoing Phase III, according to preliminary results.

The Swiss pharma giant unveiled early data from the ongoing STAND Phase III study on Friday, saying that crizanlizumab showed no statistically significant difference between the drug at two different dose levels compared to placebo in annualized rates of vaso-occlusive crises that lead to a healthcare visit over the first year since being randomized into the trial.