Up­dat­ed: FDA gives Cel­lec­tis all clear af­ter pa­tient death halt­ed off-the-shelf CAR-T tri­al

Four months af­ter a pa­tient death forced Cel­lec­tis to halt one of their off-the-shelf CAR-T tri­als, the FDA has giv­en them the OK to start dos­ing pa­tients again.

The Paris-based biotech said they changed the study’s pro­to­col to ac­com­mo­date the agency’s con­cerns, al­though they will still have to work with in­ves­ti­ga­tors to ob­tain lo­cal ap­proval to restart the tri­al and start re­cruit­ing pa­tients again. Pri­or its halt, the Phase I had sites in New York, New Jer­sey and Texas.

Cel­lec­tis did not dis­close what the changes were, but af­ter the ini­tial hold, an­a­lysts sug­gest­ed the com­pa­ny might mon­i­tor pa­tient cy­tokine lev­els more close­ly, swap out of one of the chemother­a­py drugs used or pre­vent pa­tients who have pre­vi­ous­ly re­ceived an ex­per­i­men­tal mul­ti­ple myelo­ma CAR-T from en­rolling.

The com­pa­ny’s stock {CLLS} rose 5.6% on the news, from $19.70 to $20.80.

Though part­nered with the new up­starts such as Arie Bellde­grun’s Al­lo­gene, Cel­lec­tis, a 21-year-old com­pa­ny, has been work­ing on off-the-shelf ver­sions to the first CAR-Ts since be­fore those CAR-Ts were ap­proved.

Car­rie Brown­stein

One of three dif­fer­ent so­lo projects now in the clin­ic, the halt­ed study, MELANI-01, is test­ing a cell ther­a­py for mul­ti­ple myelo­ma. If ef­fec­tive, it could serve as a po­ten­tial­ly eas­i­er way to man­u­fac­ture and ad­min­is­ter an al­ter­na­tive to the mul­ti­ple myelo­ma CAR-Ts now near­ing ap­proval from J&J and Leg­end Biotech and from blue­bird bio and Bris­tol My­ers Squibb.

When they an­nounced the hold, Cel­lec­tis CMO Car­rie Brown­stein said that the com­pa­ny had al­ready in­de­pen­dent­ly de­cid­ed to move for­ward with dose lev­el 1 in the study. The pa­tient who died had been the first to re­ceive dose lev­el 2, an in­fu­sion of 3 mil­lion cells per kilo­gram. They de­vel­oped cy­tokine re­lease syn­drome, the hall­mark side ef­fect of CAR-T ther­a­py, and died of car­diac ar­rest 25 days lat­er.

The dose 1 lev­el in­volves 1 mil­lion cells per kilo­gram.

The FDA has been on high alert for safe­ty is­sues in cell ther­a­py tri­als, and this wasn’t the first time they’ve paused a Cel­lec­tis study. Back in 2017, an­oth­er pa­tient death paused de­vel­op­ment of UCART123.

Af­ter some pro­to­col ad­just­ments, the ther­a­py was al­lowed to go back in­to pa­tients. It is now in a Phase I for acute myeloid leukemia.

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.

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.

Ex­elix­is pulls a sur­prise win in thy­roid can­cer just days ahead of fi­nal Cabome­tyx read­out

Exelixis added a thyroid cancer indication to its super-seller Cabometyx’s label on Friday — months before the FDA was expected to make a decision, and days before the company was set to unveil the final data at #ESMO21.

At a median follow-up of 10.1 months, differentiated thyroid cancer patients treated with Cabometyx (cabozantinib) lived a median of 11 months without their disease worsening, compared to just 1.9 months for patients given a placebo, Exelixis said on Monday.

Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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Den­mark's Gubra to col­lab­o­rate with Bay­er on pep­tides; Sam­sung and Bio­gen re­ceive FDA ap­proval for Lu­cen­tis biosim­i­lar

Danish biotech Gubra announced a research collaboration and license agreement with Bayer to develop peptide therapeutics to treat cardiorenal diseases. The collaboration will utilize Gubra’s peptide drug discovery platform to identify potential candidates.

This is not the first time Gubra has partnered with a company on peptide therapeutics — they partnered with Boehringer Ingelheim back in 2017 to create peptide therapeutics to treat obesity.

Ex-My­lan em­ploy­ee pleads guilty to in­sid­er trad­ing, il­le­gal­ly deal­ing on FDA ap­provals, earn­ings and Up­john merg­er

A former Mylan IT executive pleaded guilty Friday to an insider trading scheme where he bought and sold stock options on another executive’s advice.

Prosecutors secured the plea from Dayakar Mallu, Mylan’s former VP of global operations information technology, after uncovering the plan. Mallu collaborated with an unnamed “senior manager,” the SEC said, to trade options ahead of Mylan public announcements regarding FDA approvals, revenue reports and its merger with the Pfizer generics subsidiary Upjohn. The two subsequently shared profits.

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Time for round 2: Il­lu­mi­na-backed VC snags $325M for its next fund

Illumina Ventures closed off its second investment fund with a total commitment of $325 million, offering fresh fuel to back a slate of startups that have already included a smorgasbord of companies, covering everything from diagnostics to biotech drug development and genomics.

Fund II brings the total investment under Illumina Ventures’ oversight to $560 million, which has been focused on early-stage companies. And it has a transatlantic portfolio that includes SQZ, Twist and Encoded Therapeutics.

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.