Pos­i­tive piv­otal cell graft da­ta bring No­var­tis-backed Boston biotech step clos­er to fin­ish line

Gami­da Cell — the No­var­tis-backed Is­raeli biotech de­vel­op­ing a cell graft to help pa­tients with blood can­cer ad­dress­ing lim­i­ta­tions as­so­ci­at­ed with bone mar­row trans­plants — has its first taste of late-stage da­ta, paving the way for US ap­proval.

The ther­a­py, called omidu­bi­cel, is com­prised of stem cells, prog­en­i­tor cells as well as den­drit­ic cells ex­tract­ed from the um­bil­i­cal cord. It was test­ed in 125 pa­tients aged 12-65 years suf­fer­ing from high-risk lethal blood can­cers. One group of pa­tients un­der­go­ing a bone mar­row trans­plant re­ceived omidu­bi­cel, while the com­para­tor group was giv­en a stan­dard um­bil­i­cal cord blood trans­plant.

The study was de­signed this way be­cause many pa­tients in need of bone mar­row trans­plants do not get matched donors — and while un­re­lat­ed donor um­bil­i­cal cord blood can pro­vide a suf­fi­cient­ly matched donor to prac­ti­cal­ly all pa­tients, such trans­plants are as­so­ci­at­ed with a high risk of graft fail­ure.

The main goal of the study was time to neu­trophil en­graft­ment: Neu­trophils are the most com­mon type of white blood cell, and en­graft­ment refers to a process by which cells be­gin to grow and re­pro­duce new blood cells. Typ­i­cal­ly, en­graft­ment be­gins about 20 days post a bone mar­row trans­plant, ac­cord­ing to the NIH.

The me­di­an time to neu­trophil en­graft­ment was sta­tis­ti­cal­ly sig­nif­i­cant­ly short­er (p<0.001) for pa­tients who re­ceived omidu­bi­cel at 12 days, ver­sus 22 days in the com­para­tor group, the com­pa­ny said, adding that among pa­tients who were trans­plant­ed per pro­to­col, 96% of pa­tients who re­ceived omidu­bi­cel achieved suc­cess­ful neu­trophil en­graft­ment, com­pared to 88% in the com­para­tor group.

Ju­lian Adams

The graft was well-tol­er­at­ed, and de­tailed da­ta will be dis­closed at a lat­er date.

An ap­pli­ca­tion to mar­ket omidu­bi­cel, which has been grant­ed the FDA’s break­through ther­a­py sta­tus, is ex­pect­ed to be sub­mit­ted on a rolling ba­sis be­gin­ning the fourth quar­ter of this year, said CEO Ju­lian Adams, a well-known sci­en­tist whose biotech ex­pe­ri­ence spans stints at Mil­len­ni­um and In­fin­i­ty.

Shares of the com­pa­ny, which went pub­lic in a $50 mil­lion IPO in 2018, shot up more than 46% to $6.64 in morn­ing trad­ing.

As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Ku­ra flash­es pos­i­tive HRAS da­ta on once-failed J&J drug

Troy Wilson was working with J&J on their KRAS inhibitor and periodically thumbing through their publications when he spotted an old drug called tipifarnib that looked promising. So promising, in fact, that the large pharma had run it through over 5,000 patients across 70 trials, hoping they would at some point be able to nail down who were the small slice of patients who responded in some studies.

Af­ter star­ring at ASH last fall, Gilead’s new Forty Sev­en crew col­ors in more promis­ing da­ta for ma­grolimab at AS­CO

We now know the full, early-stage story behind the drug that inspired Gilead CEO Dan O’Day’s recent $5 billion acquisition of Forty Seven.

Following up on their ASCO abstract from a couple of weeks ago, the team at Forty Seven is making their return appearance this week holding clearly promising early-stage data on their lead drug magrolimab as they ponder whether they should roll on a quest to obtain an accelerated approval.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Pfiz­er, Mer­ck KGaA ce­ment Baven­cio blad­der can­cer win with OS da­ta — while carv­ing an­oth­er niche in rare can­cer

Pfizer and Merck KGaA have detailed the Phase III data that inspired FDA regulators to designate Bavencio a “breakthrough” for first-line advanced bladder cancer and offered an early glance at how the PD-L1 can help patients with a rare gynecological cancer — carving out niches in the checkpoint space for itself after being shut out of numerous others.

In JAVELIN Bladder 100, Bavencio led to a 31% reduction in risk of death compared to standard care alone. It also extended median survival by more than seven months — a historic feat in this setting, according to investigators at Queen Mary University of London.

Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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