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.

Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can created herd immunity in the US by the middle of next year, with Europe following a few months later.

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J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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FDA hands Mor­phoSys and In­cyte a quick OK on their po­ten­tial block­buster CAR-T al­ter­na­tive

Nearly three years after okaying the CAR-Ts Yescarta and Kymriah, the FDA has approved a new CD19 therapy.

MorphoSys’ Monjuvi, or tafasitamab-cxix, was cleared Friday for use in refractory diffuse large B-cell lymphoma (DBLCL). The approval sets up both MorphoSys and their commercial partner Incyte to compete with Gilead and Novartis in the ultra-competitive indication, where similar trial results and far easier delivery could allow them to cut a fair share of the market.

Rich Heyman (ARCH)

Rich Hey­man joins PMV Phar­ma, a p53 biotech, as it adds $70 mil­lion in Se­ries D

Less than a year after pulling in an impressive $62 million Series C round, PMV Pharma is back at it again.

The Cranbury, NJ-based biotech announced Monday an additional $70 million in Series D financing as it seeks to develop cancer therapies targeting p53 mutations. Additionally, PMV also introduced longtime biotech entrepreneur Rich Heyman as chairman of the board of directors.

“This financing provides PMV Pharma with the resources to expand our pipeline and to potentially advance multiple p53 therapies into the clinic,” said PMV president and CEO David Mack in a statement.

Covid-19 roundup: Eli Lil­ly retro­fits RVs for first-of-its-kind an­ti­body tri­al with NIH; Am­gen, Ab­b­Vie, Take­da team on a drug

Eli Lilly and the NIH are about to start a first-of-its-kind trial that researchers and developers have talked about for months as a way of providing temporary immunity to the most at-risk populations.

Lilly announced this morning that it will start a 2,400-person trial with the National Institute for Allergy and Infectious Diseases to test whether its experimental Covid-19 neutralizing antibody can prevent people in nursing homes and assisted living facilities from developing the disease. The idea, known as passive immunity, is that rather than waiting on a vaccine to induce people to develop antibodies, doctors can give them lab-grown antibodies. Ideally, those antibodies will either attack the new SARS-CoV-2 infection, if the patient has recently been exposed, or persist in the blood for several weeks and prevent infection or disease for that period.

So Covid-19 leader BioN­Tech has a can­cer vac­cine in de­vel­op­ment? Yes, and Re­gen­eron just jumped in for the PhII com­bo study

Before the coronavirus global emergency stole the R&D show in biopharma, the leaders in the race to develop new mRNA therapies had a big interest in determining if their tech could be used to create an effective cancer vaccine after all the first-gen tries had failed to impress. So perhaps it’s not surprising that an early cut of the data at frontrunner BioNTech went largely unnoticed.

Unless you were at Regeneron.

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Sanofi un­der for­mal in­ves­ti­ga­tion for De­pakine al­le­ga­tions; Beam li­cens­es CAR-T tech from Ox­ford Bio­med­ica

Sanofi is facing a formal investigation on manslaughter charges, due to accusations that its epilepsy drug Depakine caused birth malfunctions and slow neurological development when taken during pregnancy.

The French pharma was formally charged in February, years after evidence surfaced that the drug, sodium valproate, posed neurodevelopmental risks. Sodium valproate first hit the market in 1967 for the treatment of epilepsy and bipolar disorder, and is currently prescribed in more than 100 countries.

Tony Coles, Cerevel Therapeutics CEO

Adding $445M, Tony Coles and his big Pfiz­er neu­ro spin­out hitch a ride to Wall Street on Per­cep­tive’s SPAC

Two years ago, after Pfizer abruptly shut down its entire neuroscience division, Bain Capital bet $350 million that those assets were still worth something and packaged them into a new biotech: Cerevel Therapeutics. A year later, they got seasoned executive Tony Coles, who had recently jumped back into the C-suite of another neuroscience startup, to run the company.

Now Coles is steering Cerevel public, in what he says is the largest ever transaction of its kind. Cerevel has agreed to merge with Perceptive Advisors’ specialty acquisition company ARYA II. Between the roughly $125 million Perceptive raised through ARYA and an additional investment of $320 million Bain Capital, Perceptive and — yes, really — Pfizer, among others, Cerevel will now move forward with an added $445 million in its coffers.

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Days af­ter seal­ing Sanofi pact, Kymera beats a path to the Nas­daq with $100M IPO pitch

Back in March, when Kymera Therapeutics closed $102 million in Series C funding led by Biotechnology Value Fund and Redmile Group, CEO Nello Mainolfi noted the protein degradation player was “at the cusp of transitioning” into a fully integrated R&D company. Five months and a major Sanofi pact later, he’s back asking for another little push to get there.

Kymera has penciled in $100 million in its first IPO pitch — although given the public market’s seemingly insatiable appetite for biotechs these days the final figure is anyone’s guess.