Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of piv­otal da­ta on Au­to­lus Ther­a­peu­tics’ CAR-T is in, and ex­ecs are ready to plot a path to mar­ket.

With an over­all re­mis­sion rate of 70% at the in­ter­im analy­sis fea­tur­ing 50 pa­tients, the re­sults set the stage for a BLA fil­ing by the end of 2023, said CEO Chris­t­ian Itin.

Per­haps more im­por­tant­ly — giv­en that Au­to­lus’ drug, obe-cel, is go­ing af­ter an in­di­ca­tion that Gilead’s Tecar­tus is al­ready ap­proved for — the biotech high­light­ed “en­cour­ag­ing safe­ty da­ta” in the tri­al, with a low per­cent­age of pa­tients ex­pe­ri­enc­ing se­vere im­mune re­spons­es.

Au­to­lus sold $150 mil­lion worth of its stock im­me­di­ate­ly af­ter the read­out, as part of a pre­vi­ous­ly filed shelf reg­is­tra­tion. It is al­so col­lect­ing $70 mil­lion in mile­stone pay­ments from Black­stone per a $250 mil­lion col­lab­o­ra­tion and fi­nanc­ing deal they struck late last year.

The piv­otal Phase II tri­al, dubbed FE­LIX, en­rolled pa­tients with re­lapsed/re­frac­to­ry adult acute lym­phoblas­tic leukemia.

Jae Park, a prin­ci­pal in­ves­ti­ga­tor at Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter, called it an “un­der­served dis­ease set­ting” char­ac­ter­ized by “the ex­plo­sive growth of the leukemia and the poor con­di­tion of many pa­tients.”

Out of 50 pa­tients eval­u­at­ed for ef­fi­ca­cy, 70% went in­to ei­ther com­plete re­mis­sion or com­plete re­mis­sion with in­com­plete blood count re­cov­ery, as as­sessed by an in­de­pen­dent re­sponse re­view com­mit­tee. Among this group, obe-cel showed “com­pa­ra­ble ex­pan­sion and ini­tial per­sis­tence” to a pre­vi­ous Phase I study at me­di­an fol­low-up of 6.4 months, Au­to­lus re­port­ed, sug­gest­ing con­sis­tent da­ta.

When re­searchers look at all 92 pa­tients who have been dosed so far, 3% ex­pe­ri­enced grade 3 or high­er cy­tokine re­lease syn­drome, and 8% ex­pe­ri­enced grade 3 or high­er im­mune ef­fec­tor cell-as­so­ci­at­ed neu­ro­tox­i­c­i­ty syn­drome (ICANS), com­mon side ef­fects as­so­ci­at­ed with CAR-T ther­a­py. All told, 23% of pa­tients ex­pe­ri­enced any grade ICANS.

“We look for­ward to sup­ple­ment­ing this in­ter­im da­ta with longer fol­low up da­ta to more ful­ly ex­plore the clin­i­cal ben­e­fit of obe-cel,” said Itin, who leads a crew scat­tered across Lon­don, Mu­nich, Basel and Rockville, MD.

Shares of the com­pa­ny, though, plum­met­ed 27% to $2.17 in pre-mar­ket trad­ing, which Mizuho an­a­lyst Mara Gold­stein chalked up to the pub­lic of­fer­ing. She al­so not­ed over­all con­cerns about the com­mer­cial CAR-T mar­ket.

Obe-cel is de­signed to hit CD19, a fa­mil­iar tar­get in the CAR-T space, but Au­to­lus be­lieves that its en­gi­neer­ing ap­proach can lim­it T cell ac­ti­va­tion, keep­ing the im­mune sys­tem from go­ing hay­wire while al­so pre­vent­ing T cell ex­haus­tion.

In the r/r ALL set­ting, it will be go­ing up against Gilead’s Tecar­tus, which post­ed an ORR of 65% among 54 pa­tients at me­di­an fol­low-up of 12.3 months.

Au­to­lus plans to present more de­tails at a con­fer­ence in mid-2023.

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.

Ying Huang, Legend CEO

J&J, Leg­end say Carvyk­ti beat stan­dard ther­a­py in ear­li­er-line blood can­cer

J&J and Legend Biotech’s next step in turning their CAR-T therapy Carvykti into a potential megablockbuster has succeeded, the companies said Friday.

Carvykti achieved the primary endpoint — progression-free survival — in an open-label Phase III study testing the treatment in second- to fourth-line multiple myeloma patients. The CARTITUDE-4 trial, for which there aren’t any hard data yet, represents the biggest development for Carvykti’s ability to compete with Bristol Myers Squibb’s Abecma since its approval last February.

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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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.

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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FDA ap­proves an­oth­er in­di­ca­tion for Keytru­da, this time in the ad­ju­vant NSCLC set­ting

Merck’s blockbuster cancer treatment Keytruda has been handed another indication by the FDA.

The US regulator announced on Thursday that it has approved Keytruda to serve as an adjuvant treatment for non-small cell lung cancer (NSCLC), which is its fifth indication in NSCLC and 34th indication overall.

According to a Merck release, the approval is based on data from a Phase III trial, dubbed Keynote-091, which measured disease-free survival in patients who received chemotherapy following surgery. The data from Merck displayed that Keytruda cut down on the risk of disease recurrence or death by 27% versus placebo.

Steve Harr, Sana Biotechnology CEO

Four years in, Sana gets first FDA go-ahead to bring can­cer treat­ment in­to the clin­ic

Sana Biotechnology is finally headed to the clinic.

Thursday afternoon, the biotech announced the FDA had cleared its application to start a clinical trial for its allogeneic, or “off-the-shelf,” CAR-T cell therapy targeting the antigen CD19 for patients with B-cell lymphomas and leukemias. Sana said its therapy, dubbed SC291, was designed to evade the immune system, which could help cell therapy produce a more durable response in patients, a concern that has followed such off-the-shelf therapies that use donor cells as opposed to a patient’s own cells.

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