On a mis­sion to make or­gan trans­plants safer and more durable, Black­stone gifts Ta­laris a new CEO, leads $100M round

As a trans­plant sur­geon by train­ing, Suzanne Ild­stad un­der­stood the plight of her pa­tients all too well: The risk of or­gan re­jec­tion forces them to adopt im­muno­sup­pres­sion reg­i­mens, which in turn ex­pose them to in­fec­tions and car­dio­vas­cu­lar prob­lems they have to con­tend with. She knew that if the bone mar­row from a donor takes, any sub­se­quent or­gan trans­plant would, too.

“We dis­cov­ered a cell in the bone mar­row called the fa­cil­i­tat­ing cells that is CD8 pos­i­tive and T cell re­cep­tor neg­a­tive, and it helps the stem cell to take,” Ild­stad, di­rec­tor of the Uni­ver­si­ty of Louisville’s In­sti­tute of Cell Ther­a­py, said. “From that we de­vel­oped a method to process the bone mar­rows from hu­mans to es­sen­tial­ly take out the bad cells and leave in the good cells.”

That was 15 years ago. Ild­stad start­ed Re­generex, per­fect­ing the cell pro­cess­ing method on NIH and De­part­ment of De­fense grants. Their re­search even­tu­al­ly caught the eye of No­var­tis, which played a hand in up­grad­ing the man­u­fac­tur­ing fa­cil­i­ties and shap­ing a Phase II pro­to­col. But when the phar­ma gi­ant dis­band­ed its cell and gene ther­a­pies unit, Ild­stad found her­self search­ing for new fund­ing sources to bring the now Phase III-ready ther­a­py over the fin­ish line.

Dis­cus­sions even­tu­al­ly brought her to Clarus (now Black­stone Life Sci­ences), a ven­ture play­er with an ap­petite for just this type of deal, breath­ing life in­to a nom­i­nal­ly new com­pa­ny dubbed Ta­laris with $100 mil­lion to spend.

Aside from a new iden­ti­ty, Ta­laris is al­so un­veil­ing some Phase II that CEO Scott Re­quadt — who is tak­ing his first biotech ex­ec­u­tive role in 13 years as Clarus’ man­ag­ing di­rec­tor — calls “ex­cep­tion­al­ly ro­bust.”

Im­age: Scott Re­quadt (Ta­laris)

Ad­min­is­ter­ing Ta­laris’ per­son­al­ized ther­a­py, FCR001, to 37 pa­tients a day af­ter they re­ceived a liv­ing donor kid­ney trans­plant, in­ves­ti­ga­tors found that 26 — or 70% — of them were able to achieve im­mune tol­er­ance of their new or­gans and be free of im­muno­sup­pres­sion. And hav­ing fol­lowed those pa­tients for an av­er­age of five (longest 10) years, Re­quadt has the con­fi­dence to say: “We have 100% dura­bil­i­ty.”

“Since the mid 50s — since kid­ney trans­plants have been done — there’s less than 100 doc­u­ment­ed cas­es of that hav­ing oc­curred and there’s about 25,000 kid­ney trans­plants every year in the US alone. So you can do the math there,” he tells me. “So to get a 70% rate of be­ing able to be off im­muno­sup­pres­sion and to show no signs of re­jec­tion of the or­gan and have that be durable is quite re­mark­able.”

Ta­laris plans to ini­ti­ate a Phase III by the end of this year with an ex­pand­ed team of 50, with an eye to beef­ing up the cell pro­cess­ing team in Louisville and adding a few hires to the clin­i­cal de­vel­op­ment team in Boston, where Re­quadt spends two-thirds of his time.

In fact, the com­pa­ny has al­ready added a few se­nior lead­ers, in­clud­ing chief med­ical of­fi­cer Nan­cy Krieger, the for­mer clin­i­cal lead for FCR001 at No­var­tis. Car­los Yuraszeck is join­ing from Cel­gene as SVP of tech­ni­cal op­er­a­tions.

There’s more. The Se­ries A — com­ing from Lon­gi­tude Cap­i­tal and Qim­ing Ven­ture Part­ners USA along­side Black­stone Life Sci­ences — will fund two Phase II stud­ies what Ta­laris calls de­layed tol­re­ance and in au­toim­mune dis­eases, re­spec­tive­ly.

In the de­layed tol­er­ance tri­al, the com­pa­ny plans to re­cruit pa­tients who’ve pre­vi­ous­ly re­ceived a liv­ing kid­ney do­na­tion and are on im­muno­sup­pres­sion and see if they can go back to the kid­ney donor, take some stem cells, and cook up a per­son­al­ized con­coc­tion that can in­duce tol­er­ance af­ter the fact.

As for au­toim­mune dis­eases, Re­quadt says he’s en­cour­aged by the sub­set of pa­tients in the Phase II tri­al who didn’t see their au­toim­mune dis­eases — which led to the trans­plant in the first place — re­cur fol­low­ing ther­a­py.

“So it’s a small dataset, on­ly 7 pa­tients, but it gives us some great hope that this could have a broad­er ap­plic­a­bil­i­ty in help­ing peo­ple with a se­vere au­toim­mune or im­mune-me­di­at­ed dis­or­der to hit the re­set but­ton,” he said, mak­ing this “much more of a pipeline in a prod­uct.”

Top im­age: Suzanne Ild­stad. TA­LARIS

UP­DAT­ED: Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

Back at the beginning of 2018, Clay Siegall snagged a cancer drug called tucatinib with a $614 million cash deal to buy Cascadian. It paid off today with a solid set of mid-stage data for HER2 positive breast cancer that will in turn serve as the pivotal win Siegall needs to seek an accelerated approval in the push for a new triplet therapy.

And if all the cards keep falling in its favor, they’ll move from 1 drug on the market to 3 in 2020, which is shaping up as a landmark year as Seattle Genetics prepares for its 23rd anniversary on July 15.

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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?

Pfiz­er gets some en­cour­ag­ing PhI­II news on a fran­chise sav­ior, but is a dos­ing ad­van­tage worth the $295M up­front?

Close to 3 years after Opko tried to defend itself as shares tumbled on the news that its long-acting growth hormone had failed to outperform a placebo, the Pfizer partner $PFE is back. And this time they’re pitching Phase III data that demonstrates their drug is non-inferior — or maybe a tad better — than their well-known but fading standard in the field.
The comparator drug here is Genotropin, which earned a marginal $142 million for Pfizer last year — down 9% from the year before. Approved 24 years ago, biosimilars are now in development that Pfizer would like to stay out in front of.
The new data, says researchers, underscore that a weekly injection of somatrogon performed as well or slightly better than Genotropin (somatropin) in young children with growth hormone deficiency. Investigators tracked height velocity at 10.12 cm/year, edging out the older drug’s 9.78 cm/year. That 0.33 difference may not prove compelling to payers, though, who have been known to overlook dosing advantages in favor of lower costs.
That message may have weighed on the stock reaction this morning, with a 30%-plus hike $OPK giving way to more marginal gains.
Back in late 2016, Opko had to defend itself against a devastating Phase III setback as their initial late-stage trial failed against a sugar pill. Opko later blamed that setback on outliers in the study, though it wasn’t able to expunge the failure.

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As­traZeneca's Farx­i­ga scores FDA nod to cut risk of hos­pi­tal­iza­tion for heart fail­ure in di­a­bet­ics

While the FDA recently spurned an application to allow AstraZeneca’s blockbuster drug Farxiga for type 1 diabetes that cannot be controlled by insulin, citing safety concerns — the US regulator has endorsed the use of the SGLT2 treatment to reduce the risk of hospitalisation for heart failure in patients with type-2 diabetes and established cardiovascular disease or multiple CV risk factors.

IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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Alex­ion clinch­es aHUS ap­proval for Ul­tomiris as the clock ticks on Soliris con­ver­sion

Alexion has racked up a second approval for Ultomiris, the successor therapy to Soliris, as its mainstay blockbuster therapy faces a patent review process that could drastically shorten its patent exclusivity.

The FDA OK for atypical hemolytic uremic syndrome (aHUS) on Friday was widely expected after Alexion posted a full slate of positive Phase III data in January. But regulators also flagged concerns about serious meningococcal infections, slapping a black box warning on the label and mandating a REMS.

FDA ap­proval lets Foamix set its maid­en ac­ne ther­a­py on course for US mar­ket launch

Months ago, Foamix leaned on its biggest shareholders — Perceptive Advisors and OrbiMed — to financially grease its wheels, ahead of the FDA decision date for its acne therapy. On Friday, that approval came in — and the topical formulation of the antibiotic minocycline is set for a January launch.

The therapy, Amzeeq (formerly known as FMX101), was approved to treat inflammatory lesions of non-nodular moderate-to-severe acne vulgaris in patients aged 9 and older.

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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Christine Bunt, Robert Langer. Verseau

Armed with Langer tech and $50M, Verseau hails new check­point drugs un­leash­ing macrophages against can­cer

The rising popularity of CD47 has propelled the “don’t-eat-me” signal to household name status in the immuno-oncology world: By blocking that protein, the theory goes, one can stop cancer cells from fooling macrophages. But just as PD-(L)1 merely represents the most fruitful of all checkpoints regulating T cells, Verseau Therapeutics is convinced that CD47 is one of many regulators one can modulate to stir up or tame the immune system.