Bel­gium's liv­er-fo­cused stem cell ther­a­py de­vel­op­er Promethera rais­es $44M+ in lat­est round

Bet­ting on its stem cell ther­a­py for end-stage liv­er dis­ease, Bel­gium-based Promethera Bio­sciences has got €39.7 mil­lion (about $44.4 mil­lion) in the bank to de­vel­op the drug as an al­ter­na­tive to of­ten hard-to-pro­cure trans­plants.

Es­tab­lished in 2009, Promethera’s lead drug is an ex­per­i­men­tal stem cell ther­a­py called He­paStem for pa­tients with a dead­ly syn­drome called acute-on-chron­ic liv­er fail­ure, or ACLF, for whom trans­plants are the on­ly ex­ist­ing res­cue treat­ment. De­rived from hu­man liv­ers, the ther­a­py is for­mu­lat­ed us­ing mes­enchy­mal stem cells (MSCs) re­plete with liv­er mark­ers and liv­er fea­tures. At the In­ter­na­tion­al Liv­er Con­gress last month, Promethera pre­sent­ed da­ta from an on­go­ing dose-es­ca­la­tion Phase IIa study, which showed the treat­ment is safe and sug­gest­ed “trends in ef­fi­ca­cy.”

“The hope is to de­vel­op the tech­nol­o­gy as an al­ter­na­tive to liv­er trans­plan­ta­tion in ACLF pa­tients — not all of them are get­ting trans­plants be­cause a lot of the pa­tients are al­co­holics,” com­pa­ny chief John Tchelin­ger­ian said in an in­ter­view with End­points News.

John Tchelin­ger­ian

“Our cells have these nat­ur­al fea­tures — they are com­ing from the liv­er and go­ing back in­to the liv­er — af­ter their in­tra­venous ad­min­is­tra­tion in pa­tients. They are pro­duc­ing high­ly po­tent fac­tors that are nec­es­sary for liv­er bal­ance and liv­er main­te­nance…these fac­tors are tai­lored to act specif­i­cal­ly on liv­er cells and re­pair the le­sion or act against the in­flam­ma­tion oc­cur­ring in the liv­er.”

In 2016, Promethera ac­quired the as­sets of Durham-based Cynotet, who had over a decade of ex­pe­ri­ence deal­ing with US-based or­gan pro­cure­ment or­ga­ni­za­tions (OPOs). This es­tab­lished in­fra­struc­ture makes Promethera the sec­ond port of call for the OPOs — if the liv­er is non-trans­plantable, Tchelin­ger­ian said.

Once the do­na­tion is pro­cured — the liv­er is processed at a GMP-com­pli­ant US Promethera fa­cil­i­ty, which pro­duces a liv­er cell sus­pen­sion that is en­riched with he­pa­to­cytes — this con­sti­tutes the start­ing ma­te­ri­als to pro­duce He­paStem.  This sus­pen­sion is put in­to sealed bags, frozen and shipped to the com­pa­ny’s Bel­gium fac­to­ry, where He­paStem is then for­mu­lat­ed and cryo-pre­served, he ex­plained. “The process takes about two months — to pro­duce a batch of bil­lions of stem cells from a sin­gle bag that con­tains a few hun­dred mil­lions cells. We have many runs per year. From one liv­er to­day — we can treat about 300 to 350 pa­tients suf­fer­ing from ACLF.”

“We’re the op­po­site of the CAR-Ts — who went from bench to pa­tient very quick­ly show­ing ef­fi­ca­cy…we have done the re­verse, I think that’s a very good way to bring our prod­ucts to the max­i­mum num­ber of pa­tients world­wide, be­cause we con­trol the full val­ue chain from the donor to the end prod­uct.”

No­var­tis’ $NVS pi­o­neer­ing CAR-T ther­a­py Kym­ri­ah was ap­proved amidst great fan­fare in 2017, but sales have suf­fered due to man­u­fac­tur­ing is­sues. In the fourth quar­ter, the drug gen­er­at­ed a pal­try $28 mil­lion. How­ev­er, No­var­tis is do­ing its best to shore up man­u­fac­tur­ing, hav­ing bought cell and gene ther­a­py man­u­fac­tur­er Cell­for­Cure.

Promethera’s on­go­ing He­paStem Phase IIa will be com­plet­ed this sum­mer — and if the pos­i­tive find­ings are con­firmed, that da­ta will pro­vide the ground to launch a Phase IIb tri­al. That study is ex­pect­ed to kick off by the end of 2019, with about 200+ pa­tients and should take 2.5 years to com­plete.

Mean­while, Promethera in­tends to ini­ti­ate a NASH tri­al in very late-stage pa­tients some­time this year and will make a de­ci­sion on whether to pur­sue He­paStem in less-se­vere pa­tients, based on the re­sults of oth­er NASH read­outs ex­pect­ed this year.

So far, the com­pa­ny has raised rough­ly €110 mil­lion — in­clud­ing the Tues­day se­ries D raise, which was co-led by new Japan­ese in­vestors Itochu and Shin­sei Cap­i­tal Part­ners.

Mer­ck is tak­ing the ax to its US op­er­a­tions, cut­ting 500 jobs in its lat­est re­or­ga­ni­za­tion

Merck is cutting 500 jobs in its US sales and headquarters commercial teams in its latest effort to find new ways to streamline the operation.

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Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

Flu Virus (Source: CDC)

FDA ex­pands Xofluza ap­proval as Roche strug­gles to catch loom­ing flu mar­ket

As a potentially powerful flu season looms, so does a big test for Roche and its new flu drug, Xofluza. The Swiss giant just got a small boost in advance of that test as the FDA expanded Xofluza’s indication to include patients at high risk of developing flu-related complications.

Xofluza (baloxavir marboxil) was approved last October in the US, the first landmark flu drug approval in 20 years and a much-needed green light for a company that had watched its leading flu drug Tamiflu get eaten alive by generics. Like its predecessor, the pill offered a reduction in flu symptoms but not a cure.

EMA backs sev­en ther­a­pies, in­clud­ing Mer­ck­'s Ebo­la vac­cine

The first-ever Ebola vaccine is on the precipice of approval after the European Medicine’s Agency (EMA) backed the Merck product in this week’s roster of recommendations.

The drugmaker $MRK began developing the vaccine, christened Ervebo, during the West African outbreak that occurred between 2014 and 2016, killing more than 11,000.

The current outbreak in the Democratic Republic of Congo (DRC) has shown case fatality rates of approximately 67%, the agency estimated. Earlier this year, the WHO declared the outbreak — which so far has infected more than 3,000 people — a public health emergency of international concern.

Ronald Herb­st fol­lows Med­Im­mune ex­o­dus to Pyx­is CSO post; Jeff God­dard to suc­ceed CEO of AIT Bio­science

→ The outflow of top execs from MedImmune continues to fill the leadership ranks of smaller biotechs. The latest to take off is Ronald Herbst, the head of oncology research, who’s assuming the CSO post at Pyxis Oncology.  

Herbst was part of the old MedImmune organization AstraZeneca CEO Pascal Soriot restructured earlier this year, reorganizing the company and eliminating the storied subsidiary as a separate organization.

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UP­DAT­ED: J&J's Xarel­to, Amar­in's Vas­cepa are cost-ef­fec­tive, not bud­get friend­ly — ICER

ICER, an increasingly influential cost-effectiveness watchdog in the United States, has concluded in its review of treatments for cardiovascular disease that while the cost of J&J’s Xarelto and Amarin’s Vascepa meet its benchmark for value pricing — the two treatments will not likely treat as many patients as hoped without surpassing the annual budget threshold calculated by ICER for each therapy.

Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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