Rami Elghandour, Arcellx CEO

With its lead CAR-T flash­ing some PhI da­ta, Ar­cel­lx read­ies a jump to Nas­daq

Near­ly a year af­ter un­veil­ing a megaround Se­ries C, Ar­cel­lx is head­ed to­ward Nas­daq.

The Gaithers­burg, MD-based biotech pen­ciled in a $100 mil­lion IPO raise last Fri­day, aim­ing to take its clas­sic CAR-T ther­a­py and ex­per­i­men­tal, con­trol­lable CAR-T to the pub­lic mar­ket. The lead pro­gram, a clas­si­cal CAR-T which gar­nered the ex­cite­ment for last April’s hefty $115 mil­lion raise, will like­ly head to a Phase II study with the IPO funds, Ar­cel­lx not­ed in its S-1.

Biotech IPOs proved a hot mar­ket for the first year or so of the Covid-19 pan­dem­ic, smash­ing in­dus­try records in to­tal fundraise and num­ber of de­buts both last year and in 2020. But the pace slowed down to­ward the end of last year and the slow move­ment ap­pears to have trick­led in­to the new year, with on­ly a hand­ful of biotechs fil­ing their SEC pa­per­work the last few weeks.

On top of that, many of the bio­phar­mas that did file in the lat­ter half of 2021 end­ed up down­siz­ing their ex­pect­ed deals and pric­ing to­ward the low­er ends of their ranges. As 2022 be­gan, an­a­lysts have tak­en note of the slow­down and writ­ten that most in­vestor in­ter­est has shift­ed to ear­li­er stage in­vest­ments.

Ar­cel­lx is one of a small group of biotechs aim­ing to de­vel­op a CAR-T ther­a­py that can be ma­nip­u­lat­ed af­ter be­ing in­fused in­to the pa­tient. Though CAR-Ts have shown dra­mat­ic ef­fi­ca­cy across a range of blood can­cers, most pa­tients even­tu­al­ly re­lapse as the can­cer mu­tates, and re­searchers are hop­ing this ap­proach can help coun­ter­act such mu­ta­tions down the road.

Those pro­grams are still in the pre­clin­i­cal phase, but Ar­cel­lx, un­like much of the 2021 biotech IPO crowd, is head­ing to Nas­daq with some clin­i­cal da­ta al­ready in hand. At ASH last month, the biotech re­vealed some Phase I da­ta from its clas­sic CAR-T can­di­date for re­lapsed or re­frac­to­ry mul­ti­ple myelo­ma, ob­serv­ing all 19 evalu­able pa­tients as of a No­vem­ber cut­off date re­spond­ed to the ther­a­py.

Ad­di­tion­al­ly, 13 of the 19 pa­tients saw a com­plete re­sponse or a “strin­gent” com­plete re­sponse, Ar­cel­lx re­port­ed, with an­oth­er three notch­ing a “very good” par­tial re­sponse (Ar­cel­lx said it is us­ing the CR and PR de­f­i­n­i­tions laid out by the 2016 In­ter­na­tion­al Myelo­ma Work­ing Group re­sponse cri­te­ria).

On­ly one pa­tient ex­pe­ri­enced grade 3 cy­tokine re­lease syn­drome, while two oth­ers re­port­ed im­mune ef­fec­tor cell-as­so­ci­at­ed neu­ro­tox­i­c­i­ty syn­drome (ICANS). All in­ci­dents re­solved af­ter nor­mal in­ter­ven­tion, Ar­cel­lx said.

The biotech didn’t dish any de­tails on ex­act­ly how much of the IPO raise will go to­ward this pro­gram, but it’s like­ly to be at least a plu­ral­i­ty. Two of its con­trol­lable CAR-T pro­grams, an an­ti-BC­MA can­di­date for r/r MM and an an­ti-CD123 pro­gram for r/r acute myeloid leukemia and myelodys­plas­tic syn­drome, will al­so get some of the cash.

Once Ar­cel­lx goes pub­lic, it plans to trade un­der the tick­er $ACLX.

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Phar­ma com­pa­ny con­tin­ues its FDA law­suit spree, this time af­ter agency de­nies fast-track des­ig­na­tion

Vanda Pharmaceuticals is making a name for itself, at least in terms of suing the FDA.

The DC-headquartered firm on Monday filed its latest suit against the agency, with the company raising concerns over the FDA’s failure to grant a fast track designation for Vanda’s potential chronic digestive disorder drug tradipitant, which is a neurokinin 1 receptor antagonist.

Specifically, Vanda said FDA’s “essential point” in its one-page denial letter on the designation pointed to “the lack of necessary safety data,” which was “inconsistent with the criteria for … Fast Track designation.”

Mod­er­na seeks to dis­miss Al­ny­lam suit over Covid-19 vac­cine com­po­nent, claim­ing wrong venue

RNAi therapeutics juggernaut Alnylam Pharmaceuticals made a splash in March when it sued and sought money from both Pfizer and Moderna regarding their use of Alnylam’s biodegradable lipids, which Alnylam claims have been integral to the way both companies’ mRNA-based Covid-19 vaccines work.

But now, Moderna lawyers are firing back, telling the same Delaware district court that Alnylam’s claims can only proceed against the US government in the Court of Federal Claims because of the way the company’s contract is set up with the US government. The US has spent almost $10 billion on Moderna’s Covid-19 vaccine so far.

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Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

The discordant effects of the Covid-19 pandemic on pharma reputation continues. While the overall industry still retains a respectable halo from its Covid-19 quick response and leadership, a new patient group study reveals a different story emerging in the details.

On one hand, US patient advocacy groups rated the industry higher-than-ever overall. More than two-thirds (67%) of groups gave the industry a thumbs up for 2021, a whopping 10 percentage point increase over the year before, according to the PatientView annual study, now in its 9th year.

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Michael Corbo, Pfizer CDO of inflammation & immunology

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

Pfizer has laid out the detailed results behind its boast that etrasimod — the S1P receptor modulator at the center of its $6.7 billion buyout of Arena Pharma — is the winner of the class, potentially leapfrogging an earlier entrant from Bristol Myers Squibb.

Pivotal data from the ELEVATE program in ulcerative colitis — which consists of two Phase III trials, one lasting 52 weeks and the other just 12 weeks — illustrate an “encouraging balance of efficacy and safety,” according to Michael Corbo, chief development officer of inflammation & immunology at Pfizer. The company is presenting the results as a late breaker at Digestive Disease Week.

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Robert Califf (Michael Brochstein/Sipa USA via AP Images)

House Re­pub­li­cans at­tack Chi­na-on­ly da­ta in FDA sub­mis­sions, seek new in­ves­ti­ga­tion in­to re­search in­spec­tions

Three Republican representatives are calling on the FDA to take a closer look at the applications including only clinical data from China.

The letter to FDA commissioner Rob Califf late last week comes as the agency recently rejected Eli Lilly’s anti-PD-1 antibody, which attempted to bring China-only data but ran into a bruising adcomm that may crush the hopes of any other companies looking to bring cheaper follow-ons based only on Chinese data.

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Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; 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.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”

Delaware court rules against Gilead and Astel­las in years-long patent case

A judge in Delaware has ruled against Astellas Pharma and Gilead in a long-running patent case over Pfizer-onwed Hospira’s generic version of Lexiscan.

The case kicked off in 2018, after Hospira submitted an Abbreviated New Drug Application (ANDA) for approval to market a generic version of Gilead’s Lexiscan. The drug is used in myocardial perfusion imaging (MPI), a type of nuclear stress test.