A car­dio­vas­cu­lar cell ther­a­py play­er grabs $54M for a new be­gin­ning — where next-gen CAR-T ap­proach fea­tures promi­nent­ly

Be­fore the ad­vent of CAR-T, the term cell ther­a­py con­jured up very dif­fer­ent im­ages. Sure, there were can­cer im­munother­a­pies like Den­dreon’s Provenge, but more com­mon were the var­i­ous fla­vors of stem cell ther­a­pies and cell trans­plan­ta­tion.

None of that has gone away, even if they’ve been nudged out of the spot­light — and a lit­tle biotech has gar­nered $54 mil­lion (£40 mil­lion) to show that both the old and new ideas of cell (and gene) ther­a­py can mesh to­geth­er to form a spe­cial breed of plat­form com­pa­ny.

Joe Dupere

Ix­a­ka is launch­ing with quite a bit of his­to­ry. For­mer­ly Rex­gen­ero, it had li­censed a tech­nol­o­gy known as con­cen­trat­ed mul­ti-cell ther­a­pies from the An­dalu­sian Re­gion­al Min­istry of Health in Seville, Spain to get start­ed in 2015 and last June ex­pand­ed its fo­cus by bag­ging France’s aratin­ga.bio and its tar­get­ed nanopar­ti­cles — which puts a twist to off-the-shelf CAR-T ther­a­pies. Through it all, the com­pa­ny’s head­quar­ters had re­mained in Lon­don.

While the two ini­tial pro­grams — a Phase III mul­ti-cell ther­a­py de­signed to treat the se­ri­ous car­dio­vas­cu­lar con­di­tion chron­ic limb-threat­en­ing is­chemia, and a pre­clin­i­cal CD19 can­cer drug — may seem un­re­lat­ed to each oth­er, CEO Joe Dupere be­lieves that they re­quire the same un­der­ly­ing ex­per­tise to de­liv­er. That spans leg­isla­tive knowl­edge, man­u­fac­tur­ing knowhow, reg­u­la­to­ry deft­ness, lo­gis­ti­cal ex­pe­ri­ence and more.

“So there are a num­ber of fac­tors which are very, very com­mon be­tween cell and gene ther­a­pies, to­geth­er with some spe­cif­ic fac­tors around in­di­vid­ual in­di­ca­tions,” he told End­points News. “So we have ca­pa­bil­i­ties and a good net­work in the car­dio­vas­cu­lar area, in the on­col­o­gy area, but we very much view that the ca­pa­bil­i­ties we have from a sci­en­tif­ic, med­ical, clin­i­cal per­spec­tive en­able us to de­sign so­lu­tions that we can ap­ply to a whole range of dif­fer­ent in­di­ca­tions but uti­liz­ing our fun­da­men­tal un­der­stand­ing of the bi­ol­o­gy, med­i­cine, and how do you bring these prod­ucts to mar­ket.”

The new fund­ing, which comes from ex­ist­ing share­hold­ers, will see Ix­a­ka to the end of its on­go­ing Phase III for REX-001, the car­dio­vas­cu­lar prod­uct.

To date, more than 150 peo­ple had been treat­ed with the au­tol­o­gous ther­a­py, Dupere not­ed. Clin­i­cians first ex­tract bone mar­row from pa­tients, then trans­port­ed to a fa­cil­i­ty where the most ac­tive cells (stem cells, T cells, mono­cytes, gran­u­lo­cytes, and so on) are picked out and pack­aged in­to a sy­ringe that then gets in­fused back, tar­get­ing the ves­sels in the low­er leg.

The idea is that the cells would then re­store the im­bal­ance of cells at those ves­sels and, ac­cord­ing to Ix­a­ka, it had looked promis­ing in Phase II tri­als. Up to 80% of those small­er groups had re­spond­ed as hoped, with blood flow re­stored, “ul­cers healed, pain re­lieved.”

With an in­ter­im analy­sis slat­ed for lat­er this year, Dupere said Ix­a­ka will al­so plan for a sec­ond Phase III — this time not just in Eu­rope but al­so in the US — as well as scal­ing up man­u­fac­tur­ing and get­ting ready for com­mer­cial­iza­tion and mar­ket ac­cess, even though ap­proval isn’t ex­pect­ed un­til 2024 or 2025.

For the sec­ond lead pro­gram, dubbed Celtic, the Se­ries A should be suf­fi­cient to fu­el an­i­mal proof of con­cept. Some­what like Umo­ja, a fel­low up­start based out of Seat­tle, Ix­a­ka is aim­ing to pro­duce a bi­o­log­ic that lets can­cer pa­tients make their own CAR-T in vi­vo.

The tools they’re us­ing are poly­mer-based nanopar­ti­cles that con­tain a mod­i­fied, in­ert lentivirus and a cell-spe­cif­ic pro­mot­er as the ge­net­ic car­go. Ix­a­ka will start by lever­ag­ing the well-known CD19 tar­get to see if it can prove faster, cheap­er and even more ef­fec­tive than the first (and, by the time it gets fur­ther, like­ly sec­ond) wave of CAR-T prod­ucts. In both pro­grams, Dupere added, the cost of goods is a frac­tion of what peo­ple cur­rent­ly as­so­ciate with CAR-T cell ther­a­pies.

“That gives you much more scope to go for dif­fer­ent in­di­ca­tions which wouldn’t po­ten­tial­ly sup­port such high lev­els,” he said — in­di­ca­tions that Ix­a­ka is now in the process of “triag­ing.”

Bob Nelsen (Photo by Michael Kovac/Getty Images)

With stars aligned and cash in re­serve, Bob Nelsen's Re­silience plans a makeover at 2 new fa­cil­i­ty ad­di­tions to its drug man­u­fac­tur­ing up­start

Bob Nelsen’s new, state-of-the-art drug manufacturing initiative is taking shape.

Just 3 months after gathering $800 million of launch money, a dream team board and a plan to shake up a field where he found too many bottlenecks and inefficiencies for the era of Covid-19, Resilience has snapped up a pair of facilities now in line for a retooling.

The company has acquired a 310,000-square-foot plant in Boston from Sanofi along with a 136,000-square-foot plant in Ontario to add to a network which CEO Rahul Singhvi says is just getting started on building his company’s operations up. The Sanofi deal comes with a contract to continue manufacturing one of its drugs.

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Paul Sekhri

The next big biotech su­per­star? Paul Sekhri has some thoughts on that

It occasionally occurs to Paul Sekhri that if they pull this off, his company will be on the front page of the New York Times and a lead story in just about every major news outlet on the planet. He tries not to dwell on it, though.

“I just want to be laser-focused on getting to that point,” Sekhri says, before acknowledging, “Yes, it absolutely crossed my mind.”

Sekhri, a longtime biopharma executive with tenures at Sanofi and Novartis, is now entering year three as CEO of eGenesis, the biotech that George Church protégé Luhan Yang founded to genetically alter pigs so that they can be used for organ transplants. He led them through one megaround and has just closed another, raising $125 million from 17 different investors to push the first-ever (humanized) pig to human transplants into the clinic.

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UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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Pascal Soriot, AstraZeneca CEO (AP Images)

Pas­cal So­ri­ot cash­es in As­traZeneca’s chips on Mod­er­na for $1.2B cash in­jec­tion

While still working to prove its own Covid-19 vaccine, AstraZeneca has reportedly capitalized on the success of another.

The company has sold off its 7.7% stake in Moderna and turned it into $1.2 billion in cash, according to the Times, beefing up the reserves just as Pascal Soriot is wrapping up his $39 billion acquisition of Alexion and its rare disease pipeline.

AstraZeneca’s stock sale follows a similar move by Merck in December. But like its pharma brethren, the British giant is keeping its R&D collaborations with Moderna.

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Amit Munshi, Arena

One of Are­na's top drugs flops in a PhI­Ib study for IBS pain. But re­searchers tease out a pos­si­ble path for­ward as CEO ex­plores 's­trate­gic op­tion­s'

Four years ago, when Arena CEO Amit Munshi cut its ties to a troubled weight drug and doubled down on the pipeline, a cannabinoid receptor 2 agonist figured prominently in the biotech’s future. On Tuesday evening, however, Munshi’s high hopes for the drug took a nasty hit after it failed a Phase IIb study for patients with irritable bowel syndrome pain.

Put through a randomized pace with 273 patients, researchers said it flat failed the primary endpoint among the large group with abdominal pain. But they quickly went on to highlight subgroup data, always a tricky and controversial ploy, where they spotlighted a positive p value for patients with moderate to severe pain who received the high dose of the drug — one of 3 provided in the study.

Af­ter bail­ing on Covid-19 vac­cines, Mer­ck will team up with J&J to pro­duce its shot as part of un­usu­al Big Phar­ma pact

Merck took a big gamble when it opted to jump into the Covid-19 vaccine race late, and made an equally momentous decision to back out in late January. Now, looking to chip in on the effort, Merck reportedly agreed to team up with one of the companies that has already crossed the finish line.

President Joe Biden on Tuesday is expected to announce a partnership between drugmakers Merck and Johnson & Johnson to jointly produce J&J’s recombinant protein Covid-19 vaccine that received the FDA’s emergency use authorization Saturday, the Washington Post reported.

Ab­b­Vie tees up a biotech buy­out af­ter siz­ing up their Parkin­son's drug spun out of Ke­van Shokat's lab

AbbVie has teed up a small but intriguing biotech buyout after looking over the preclinical work it’s been doing in Parkinson’s disease.

The company is called Mitokinin, a Bay Area biotech spun out of the lab of UCSF’s Kevan Shokat, whose scientific explorations have formed the academic basis of a slew of startups in the biotech hub. One of Shokat’s PhD students in the lab, Nicholas Hertz, co-founded Mitokinin using their lab work on PINK1 suggesting that amping up its activity could play an important role in regulating the mitochondrial dysfunction contributing to Parkinson’s disease pathogenesis and progression.

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Fi­bro­Gen shares skid low­er as a sur­prise ad­comm rais­es risks on roxa OK

FibroGen will likely have to delay its US rollout for roxadustat once again.

In an unexpected move, the FDA is convening its Cardiovascular and Renal Drugs Advisory Committee to review the NDA in an advisory committee meeting. The date is yet to be confirmed.

Just a few weeks ago, SVB Leerink analyst Geoffrey Porges predicted that the roxa approval could come ahead of the PDUFA date on March 20 — effusive despite already being let down once by the FDA’s extension of its review back in December. AstraZeneca, which is partnered with FibroGen on the chronic kidney disease-related anemia drug, disclosed regulators had requested further clarifying analyses of clinical data.

In­tro­duc­ing End­pointsF­DA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.