David Mazzo, Caladrius Biosciences CEO

Blam­ing Covid dis­rup­tion, sup­ply short­ages, mi­cro­cap biotech hits the brakes on cell ther­a­py tri­al

More than two years in­to the Covid-19 pan­dem­ic, we’re not done see­ing its dis­rup­tion of biotech.

That’s ac­cord­ing to Cal­adrius ex­ecs, who cit­ed the pan­dem­ic as one rea­son for sus­pend­ing its Phase IIb tri­al of a cell ther­a­py as a treat­ment for coro­nary mi­crovas­cu­lar dys­func­tion (CMD). The com­pa­ny plans to go ahead on an in­ter­im analy­sis be­fore de­cid­ing what to do with the pro­gram, which was built on a li­cens­ing deal with Shire in 2018.

In ret­ro­spect, the writ­ing may have been on the wall last month when Cal­adrius an­nounced its merg­er with Cend Ther­a­peu­tics, a lit­tle-known biotech fo­cused on sol­id tu­mors. Cal­adrius had plans to re­name it­self in­to Lisa­ta and make Cend’s can­cer work the “em­pha­sis” of its R&D group mov­ing for­ward.

Over its 16-year his­to­ry — it was found­ed as NeoStem and al­ready re­brand­ed once, in 2015 — Cal­adrius has tried and failed to bring for­ward cell ther­a­pies for a num­ber of dis­eases, and its shares have been lan­guish­ing in pen­ny stock ter­ri­to­ry.

Xow­na, the drug be­ing test­ed in its halt­ed tri­al, com­pris­es an in­fu­sion of au­tol­o­gous CD34+ cells.

Cal­adrius had pre­vi­ous­ly com­plet­ed a Phase IIa open-la­bel, proof-of-con­cept study in CMD. The Phase IIb FREE­DOM study was meant to con­firm those re­sults in a con­trolled tri­al — as well as bet­ter gauge how big the treat­ment ef­fect might be, as mea­sured by clin­i­cal end­points the FDA would like­ly be look­ing for in a lat­er piv­otal tri­al.

But while the study kicked off as planned in Oc­to­ber 2020 and treat­ed the first pa­tient the next Jan­u­ary, Cal­adrius ran in­to se­ri­ous trou­ble en­rolling pa­tients. On top of the im­pact of Covid-19, the biotech said sup­ply chain is­sues made it tough to get the catheters need­ed for both the di­ag­no­sis and ad­min­is­tra­tion of Xow­na, as the man­u­fac­tur­er stopped mak­ing the equip­ment orig­i­nal­ly spec­i­fied in the tri­al to qual­i­fy pa­tients for the study while a com­mon­ly used con­trast agent was al­so in short­age.

Thrown off the orig­i­nal sched­ule, the com­pa­ny said the re­vised time­line is sim­ply “not vi­able for fi­nan­cial and com­mer­cial rea­sons and an al­ter­na­tive de­vel­op­ment plan must be con­sid­ered.”

De­spite the mul­ti­ple pro­to­col amend­ments to ad­dress these ob­sta­cles, along with an in­creased num­ber of sites in the study, the FREE­DOM tri­al has on­ly en­rolled ap­prox­i­mate­ly one third of the tar­get­ed 105 pa­tients, and at this rate, more than four years would like­ly be re­quired to reach the pri­ma­ry end­point fol­low-up at 6 months post-treat­ment for all sub­jects.

CEO David Maz­zo added that paus­ing en­roll­ment now and tak­ing a look at the in­ter­im re­sults is in the “best in­ter­est” of the com­pa­ny.

Re­sults of the in­ter­im analy­sis are ex­pect­ed in Au­gust.

Ed­i­tor’s note: This sto­ry has been up­dat­ed to clar­i­fy the ori­gin of the Xow­na pro­gram in CMD. While Cal­adrius li­censed a no-op­tion re­frac­to­ry dis­abling angi­na pro­gram from Shire, it for­mu­lat­ed and owns the IP for CMD.

Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)

FDA side­lines Paul Hud­son's $3.7B MS drug af­ter es­tab­lish­ing link to liv­er dam­age

One of Sanofi CEO Paul Hudson’s top picks in the pipeline — picked up in a $3.7 billion buyout 2 years ago — has just been sidelined in the US by a safety issue.

The pharma giant put out word early Thursday that the FDA has put their Phase III studies of tolebrutinib in multiple sclerosis and myasthenia gravis on partial clinical hold, halting enrollment and suspending dosing for patients who have been on the drug for less than 60 days. Patients who have completed at least 60 days of treatment can continue therapy as researchers explore a “limited” — but unspecified in Sanofi’s statement — number of cases of liver injury.

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Phar­ma re­acts to post-Roe; Drug­mak­ers beef up cy­ber de­fense; Boehringer, Roche qui­et­ly axe drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As a reminder, we are off on Monday for the Fourth of July. I hope this recap will kick off your (long) weekend well and that the rest of it will be just what you need. See you next week for a shortened edition!

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Eric Hughes, incoming Teva EVP of global R&D and CMO

Te­va chief raids Ver­tex for his new glob­al head of re­search and de­vel­op­ment

Teva CEO Kåre Schultz has found his new R&D chief and CMO in Vertex’s ranks.

The global generics giant, which has some 3,500 staffers in the R&D group, has named Eric Hughes to the top research spot in the company. He’ll be replacing Hafrun Fridriksdottir, who held the role for close to five years, on Aug. 1.

Hughes hasn’t been at Vertex for long, though. He jumped from Novartis less than a year ago, after heading the immunology, hepatology & dermatology global development unit. Before that, he completed a five-year stint as head of early clinical research for the specialty discovery medicine department in the exploratory clinical & translational research group at Bristol Myers Squibb, according to his LinkedIn profile.

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#BIO22: Man­ag­ing a biotech in tur­bu­lent times. 'There's a per­fect shit­show out there'

On Tuesday, June 14, Endpoints News EIC John Carroll sat down with a group of biotech execs to discuss the bear market for industry stocks and how they were dealing with it. Here’s the conversation, which has been lightly edited for brevity.

Martin Meeson, sponsor opening:

Thank you, John. Hello everyone. My name’s Martin Meeson, I’m the CEO of Fujifilm Diosynth. For those of you who don’t know Fujifilm Diosynth, we operate in the development of clinical and commercial product scale up, we have facilities in Europe and the US, and around about 4,000 employees. We run on average about 150 programs, so when it comes to managing in turbulent times over the last two years, we’ve had quite a lot of experience of that. Not just keeping the clinical pipelines and the commercial pipelines open, but also our response to the pandemic and the molecules that we’ve had within there. One of the phrases that I coined probably about a year ago when we were talking at JP Morgan, was I talked about managing through turbulent times. Well, it’s become the fact that we are not managing and leading through these times, we are managing in them, which is why that’s really the purpose of and the topic that we’ve got today.

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Bo Cumbo, AavantiBio CEO

Scoop: A small gene ther­a­py biotech, flush with ex-Sarep­ta and blue­bird lead­ers, guts CMC

En route to entering the clinic with its first AAV-based gene therapy for a rare neuromuscular disease, AavantiBio has let go of 30 employees, Endpoints News has learned.

The move comes after a year stacking its executive bench with ex-Sarepta and bluebird bio leaders and inking multiple partnerships with the likes of Aldevron, Catalent and Resilience. The biotech also formed a scientific advisory board in February.

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Susan Galbraith, AstraZeneca EVP, oncology R&D

Catch­ing up with Bris­tol My­ers and Mer­ck, As­traZeneca de­clares neoad­ju­vant win for PD-L1/chemo com­bo

When AstraZeneca started the Phase III AEGEAN trial for Imfinzi in 2018, it was, alongside several Big Pharma brethren, hoping to push the use of PD-(L)1 therapies into earlier lines of treatment. Three and a half years later, the British drugmaker has nabbed promising data in a type of lung cancer.

Topline results from an interim analysis showed that adding Imfinzi to chemotherapy before surgery spurred a “statistically significant and meaningful” improvement in pathologic complete response for patients with resectable non-small cell lung cancer compared to chemotherapy alone.

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Nassim Usman, Catalyst Biosciences CEO

Fac­ing set­backs for months and an ac­tivist at­tack, Cat­a­lyst Bio­sciences pre­pares to call it quits

After downsizing for several months, Catalyst Biosciences is getting ready to tap out.

The San Francisco biotech announced Wednesday that it would be liquidating and distributing cash back to shareholders, with total proceeds expected to reach $65 million. Catalyst intends to return the money “as soon as practicable,” the company said, as it has ceased all R&D activities, CEO Nassim Usman said in a statement.

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Amgen's taking social media followers around the globe as it introduces the many different

From Tam­pa to Mu­nich, Am­gen’s ‘Places’ cam­paign in­tro­duces its lo­ca­tions around the world

Amgen is taking social media followers around the world with its latest corporate campaign. Called “Places of Amgen,” the twice monthly posts highlight the biopharma’s different offices and sites – and the people who work there.

Each post runs on LinkedIn, Facebook and Instagram with details about the work Amgen does in that location, when it was established, comments from people who work there and other interesting facts. The most recent one about Paris, France, for example, notes that Amgen France last year signed a French association charter committed to the inclusion of LBGT+ people in the workplace.

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On Friday, Lonza announced plans to construct a large-scale commercial drug product fill and finish facility in the town of Stein, Switzerland.

Lon­za to in­vest $500M+ on fill-fin­ish fa­cil­i­ty on its home turf

Lonza has been expanding its reach across the globe, bringing sites in China and the US online this year, but now they are looking closer to home for their next major investment.

The Swiss manufacturer on Friday announced plans to construct a large-scale commercial drug fill and finish facility in the town of Stein, Switzerland. The new facility will be delivered through an investment of approximately CHF 500 million, or $519 million, and is expected to be completed in 2026. The facility will also be constructed on the same campus as Lonza’s current clinical drug product facility.