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.”

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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So what hap­pened with No­var­tis' gene ther­a­py group? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Af­ter sell­ing to Genen­tech, the old Je­cure team is back at an RNA-fo­cused start­up — and more en­thu­si­as­tic than ever

When Genentech swooped in to buy NASH-focused Jecure Therapeutics back in 2018, a handful of the startup’s executives weren’t quite ready to disperse.

It had been just three years since Jecure launched with a preclinical portfolio of NLRP3 inhibitors — and the takeover came sooner than anyone, including CEO Jeff Stafford, had expected. So he got talking with James Veal and Gretchen Bain, two serial entrepreneurs in charge of Jecure’s R&D.

Lat­est news: It’s a no on uni­ver­sal boost­ers; Pa­tient death stuns gene ther­a­py field; In­side Tril­li­um’s $2.3B turn­around; and more

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