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.”

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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David Ricks, Eli Lilly CEO (David Paul Morris/Bloomberg via Getty Images)

Eli Lil­ly set to in­vest $2.1B in home state man­u­fac­tur­ing boost

Eli Lilly is looking to expand its footprint in its home Hoosier State by making a major investment in manufacturing.

The pharma is investing $2.1 billion in two new manufacturing sites at Indiana’s LEAP Lebanon Innovation and Research District in Boone County, northwest of Lilly’s headquarters in Indianapolis.

The two new facilities will expand Lilly’s manufacturing network for active ingredients and new therapeutic modalities, including genetic medicines, according to a press release.

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US sees spike in Paxlovid us­age as Mer­ck­'s mol­nupi­ravir and As­traZeneca's Evusheld are slow­er off the shelf

New data from HHS show that more than 162,000 courses of Pfizer’s Covid-19 antiviral Paxlovid were administered across the US over the past week, continuing a streak of increased usage of the pill, and signaling not only rising case numbers but more awareness of how to access it.

In comparison to this week, about 670,000 courses of the Pfizer pill have been administered across the first five months since Paxlovid has been on the US market, averaging about 33,000 courses administered per week in that time.

Pfiz­er and CD­MOs ramp up Paxlovid man­u­fac­tur­ing with Kala­ma­zoo plant ex­pan­sion lead­ing the way

As the Covid-19 pandemic continues to evolve, pharma companies and manufacturers are exploring how to step up production on antivirals.

Pfizer is planning to expand its Kalamazoo-area facility to increase manufacturing capabilities for the oral Covid-19 antiviral Paxlovid, according to a report from Michigan-based news site MLive. The expansion of the facility, which serves as Pfizer’s largest manufacturing location, is expected to create hundreds of “high-skilled” STEM jobs, MLive reported. No details about the project’s cost and timeline have been released, but according to MLive, Pfizer will announce the details of the expansion at some point in early June.

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FDA spells out the rules and re­stric­tions for states seek­ing to im­port drugs from Cana­da

The FDA is offering more of an explanation of the guardrails around its program that may soon allow states to import prescription drugs in some select circumstances from Canada, but only if such imports will result in significant cost reductions for consumers.

While the agency has yet to sign off on any of the 5 state plans in the works so far, and PhRMA’s suit to block the Trump-era rule allowing such imports is stalled, the new Q&A guidance spells out the various restrictions that states will have to abide by, potentially signaling that a state approval is coming.

Simba Gill, CEO of Evelo Biosciences

While down 87% YOY, Evelo gets Flag­ship and oth­ers to in­fuse new cap­i­tal for come­back hope

Just four years after Flagship spinout Evelo Biosciences went public in an IPO worth $85 million, the biotech has seen its share price tank from $13 a share this time last year (ultimately reaching a peak of over $17) to now under $1.50. And today, it looks like Flagship still thinks the fledging biotech, in a down market, is still worth something after initial pre-IPO backing from the likes of Google’s GV, Celgene, Mayo Clinic and Alexandria Venture.

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Peter Thompson, Terremoto Biosciences interim CEO

For­mer Prin­cip­ia team looks to shake up co­va­lent small mol­e­cules again, this time at 'earthquake' scale

Terremoto Biosciences goes back a long ways, in a sense, to about a dozen years ago when Principia Biopharma was founded by UCSF professor Jack Taunton. Peter Thompson initially helmed the biotech.

The company helped expand covalent small molecule inhibitors beyond oncology and into autoimmune disease by targeting cystine. But that amino acid is uncommon in a lot of proteins, offering fewer drug targets than, say, lysine, which is present in most proteins of interest. So, over the years, Taunton went back to the drawing board to check out that second amino acid.

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Pfizer CEO Albert Bourla at the World Economic Forum (Gian Ehrenzeller/Keystone via AP Images)

All about ac­cess: Pfiz­er moves to a non-prof­it mod­el for drug sales in 45 low­er-in­come coun­tries

Leading the way to increase access to cheaper drugs worldwide, Pfizer said Wednesday it will provide all current and future patent-protected medicines and vaccines available in the US or EU on a not-for-profit basis to about 1.2 billion people in 45 lower-income countries.

Rwanda, Ghana, Malawi, Senegal and Uganda are the first five countries to sign on to this accord, which will also seek to blaze new paths for quick and efficient regulatory and procurement processes to reduce the usual delays in making new medicines and vaccines available in these countries.

Almirall is tapping artificial intelligence on behalf of its sales force for insights and efficiencies. (via Shutterstock)

Almi­rall rolls out sales rep ar­ti­fi­cial in­tel­li­gence sys­tem, cut­ting pre-call prep and 'wind­shield time'

Dermatology specialty pharma Almirall is making its sales reps smarter. Not with extra training or educational courses, but instead with artificial intelligence tools.

It began a soft launch of a sales rep AI and machine learning platform it calls Polaris last August in one of its 7 US coverage regions. The platform from Aktana gathers information from across Almirall internal sources and external ones – such as claims and prescribing data – to generate insights for reps. Now, instead of spending hours prepping for a sales call, Polaris can generate details about a physician’s preferences, past behaviors and prescription habits for reps in minutes, said Almirall head of commercial operations Vincent Cerio.

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