Arie Belldegrun speaking at UKBIO19

Pa­tient death mars lat­est cut of da­ta from Al­lo­gene's off-the-shelf CAR-T

Al­lo­gene has been rid­ing high since AS­CO in May, when ear­ly da­ta showed their off-the-shelf CAR-T worked in a hand­ful of pa­tients and that no one re­ject­ed their for­eign cells. It was a key mo­ment in a field that could re­make cell ther­a­py and treat­ment for cer­tain can­cers.

On Wednes­day, though, the Arie Bellde­grun-found­ed biotech re­leased re­sults for their mul­ti­ple myelo­ma CAR-T and, al­though the re­spons­es were there, so was a red flag: Four pa­tients con­tract­ed a se­ri­ous in­fec­tion, and one of them died.

Al­lo­gene $AL­LO shares im­me­di­ate­ly fell from $33.79 to $28.78 Wednes­day morn­ing, al­though they would re­bound to $31.06 by the time mar­kets close.

The news is trag­ic on its own and a set­back for an ap­proach that Al­lo­gene is try­ing to prove can com­pete with or even out­per­form tra­di­tion­al mul­ti­ple myelo­ma CAR-Ts from J&J and Leg­end and from Bris­tol My­ers Squibb and blue­bird. Al­though Al­lo­gene and star­tups such as Po­sei­don have point­ed to the po­ten­tial for off-the-shelf ap­proach­es to be more scal­able and al­low for re­peat dos­ing, J&J and blue­bird have al­ready shown strong ef­fi­ca­cy in hu­man tri­als, set­ting the bar high.

Still, an­a­lysts were di­vid­ed on how large a role Al­lo­gene’s treat­ment played in the pa­tient’s death, or how con­cerned they should be if they are. The vol­un­teers in the Al­lo­gene tri­al were very sick, with many on their fifth-line of ther­a­py for a dead­ly can­cer, and pa­tients have died through­out the last decade of CAR-T tri­als, in­clud­ing in the blue­bird stud­ies.

Cowen’s Marc Frahm said it was “un­sur­pris­ing” news. “We would note that sev­er­al oth­er CAR T cell tri­als [saw] in­fec­tions in­clud­ing fa­tal events,” he wrote in a note to in­vestors.

The pa­tient was di­ag­nosed with se­vere pneu­mo­nia eight days af­ter re­ceiv­ing the ther­a­py, and in­ves­ti­ga­tors at­trib­uted it to the in­tense chemother­a­py pa­tients un­der­go di­rect­ly be­fore re­ceiv­ing the ther­a­py. They added that, as of the da­ta cut­off for the ab­stract, they had not seen oth­er ma­jor ad­verse events, in­clud­ing graft ver­sus host dis­eases — one com­mon and dam­ag­ing side ef­fect from oth­er form of cell trans­plants.

Jef­feries’ Biren Amin not­ed that, more broad­ly, “high in­fec­tion rate is a key fac­tor for mor­tal­i­ty in myelo­ma pa­tients.”

An­a­lysts, mean­while, were large­ly sat­is­fied with the ef­fi­ca­cy re­sults. Al­though it’s still ear­ly in Phase I, they ar­gued it showed the po­ten­tial to even­tu­al­ly com­pete with the J&J and blue­bird CAR-Ts.

In the high-dose arm of the dose-find­ing study, three out of five pa­tients re­spond­ed. One of the three had a strin­gent com­plete re­sponse and an­oth­er had a very good par­tial re­sponse.

That’s a pret­ty small sam­ple size to draw per­cent­ages from, but Frahm ar­gued it looked “sim­i­lar” to the rough­ly 65% VG­PR/CR re­sponse blue­bird saw in ear­ly stud­ies and the 88% VG­PR re­sponse J&J saw.

Amin said it showed high dos­es are need­ed for the ther­a­py to work. He said longer term, they’ll be look­ing for Al­lo­gene to match blue­bird’s Phase I re­sults. In that study, he not­ed, pa­tients across dose co­horts who had high BC­MA ex­pres­sion had an 89% re­sponse rate and a 22% com­plete re­sponse rate.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Paul Hastings, Nkarta CEO

With no up­front pay­ment or mile­stones on the line, Nkar­ta and CRISPR join forces on CAR-NK search

Most deals in biotech come with hefty upfront payments attached, and the promise of big biobucks if a program works out. Not this one.

Nkarta has struck what CEO Paul Hastings calls a “real collaboration” with CRISPR Therapeutics to co-develop and commercialize two CAR-NK therapies, in addition to an NK+T program. The duo will split all R&D costs — and any worldwide profits — 50/50, Hastings said.

Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.

Eric Kelsic, Dyno Therapeutics CEO

Dyno's Er­ic Kel­sic fills the tank in his quest for bet­ter AAV with a group of big-name sup­port­ers on board

Adeno-associated viruses (AAV) for gene therapy have received a ton of scrutiny throughout the field’s history after a smattering of safety scares and their limited therapeutic range. Hoping to crack the field wide open through a capsid design revolution, Eric Kelsic and his team at Dyno have drummed up immense excitement — and now a hefty war chest.

Dyno Therapeutics has bagged a $100 million Series A with backing from the likes of round leader Andreessen Horowitz and new investor Casdin Capital in its quest to use AI to design better AAV capsids for gene therapy, the company said Thursday.