Michael Yang, ViaCyte CEO

Af­ter years of fail­ure, Vi­a­Cyte shows off first proof-of-con­cept for di­a­betes stem cell trans­plants

Sev­en years af­ter they first tried it, Vi­a­Cyte fi­nal­ly has da­ta show­ing that their stem cell treat­ment for di­a­betes — a po­ten­tial cure — can work in a pa­tient.

The da­ta are ex­treme­ly lim­it­ed: They come from a sin­gle pa­tient. But they pro­vide, out­side ex­perts say, the first proof-of-con­cept for an ap­proach that com­pa­nies, in­clud­ing Ver­tex, and di­a­betes re­search foun­da­tions are bet­ting could even­tu­al­ly pro­vide a func­tion­al cure for many pa­tients with type 1 di­a­betes and — down the road — the mil­lions more with type 2 di­a­betes.

The lim­it­ed re­sults were par­tic­u­lar­ly note­wor­thy be­cause Vi­a­Cyte’s first at­tempt at trans­plant­i­ng lab-grown, in­sulin-pro­duc­ing in­to pa­tients be­gin­ning in 2014 had been a fail­ure.

“It’s ac­tu­al­ly very im­pres­sive — I was not im­pressed by their first tri­al, but this is a ma­jor im­prove­ment,” Camil­lo Ri­cor­di, di­rec­tor of the Di­a­betes Re­search In­sti­tute at the Uni­ver­si­ty of Mi­a­mi, told End­points News. “It’s a crit­i­cal first step, it shows that they can work.”

At the same time, the re­sults fell short of Vi­a­cyte’s am­bi­tions to cre­ate a func­tion­al cure. De­spite the new cells, the pa­tient still need­ed to take in­sulin to con­trol her dis­ease.

“I would think it’s not hav­ing enough of an ef­fect,” Jeanne Lor­ing, a pro­fes­sor emer­i­tus at Scripps Re­search, told End­points.

Lor­ing, who launched a fore­run­ner of Vi­a­cyte in 1998 and main­tains a mod­icum of stock, said the re­sults were good but un­sur­pris­ing. “With any of these cell re­place­ment ther­a­pies, the wish is to have a com­plete cure,” she added, “but at least un­til they get bet­ter, it is like­ly to just re­duce the need for in­sulin.”

The pro­ce­dure, first con­ceived decades ago, in­volves tak­ing a bank of stem cells, ma­nip­u­lat­ing them to be­come pan­cre­at­ic pre­cur­sor cells and then im­plant­i­ng them in­to a pa­tient. There, they de­vel­op in­to in­sulin-pro­duc­ing be­ta cells, re­plac­ing the ones that are lost in type 1 di­a­betes pa­tients.

The ap­proach was meant as a scal­able al­ter­na­tive to islet cell trans­plant, an op­er­a­tion that has treat­ed thou­sands of pa­tients, many of who were able to go off in­sulin, but whose reach is lim­it­ed by the lim­it­ed num­ber of avail­able donors.

That was eas­i­er said than done, though. When Vi­a­Cyte first put their cells in­to hu­mans in 2014, en­cap­su­lat­ed in a de­vice meant to shield the lab-grown cells from the pa­tient’s im­mune sys­tem, most of the cells didn’t en­graft. The pa­tients saw lit­tle, if any, change to their dis­ease. The de­vice “ob­vi­ous­ly did not work,” Ri­cor­di said.

Reel­ing from the fail­ure and fac­ing new com­pe­ti­tion from a start­up out of Har­vard called Sem­ma, Vi­a­Cyte tried an eas­i­er ap­proach. They im­plant­ed the cells in a porous de­vice that al­lows blood ves­sels to reach through. That means the cells are ex­posed to the im­mune sys­tem and pa­tients have to take im­muno­sup­pres­sants — lim­it­ing the ther­a­py to on­ly the sick­est 10% of di­a­bet­ics — but it al­so al­lows the cells to ful­ly en­graft.

Nine months af­ter un­der­go­ing ther­a­py, the pa­tient in the study, a woman who had di­a­betes for 36 years and was con­sid­ered “brit­tle,” had mul­ti­ple in­di­ca­tors of high lev­els of in­sulin pro­duc­tion. C-Pep­tide, a bio­mark­er of in­sulin, had in­creased from 0.1 nanogram per mililiter, to 0.8.

Her HbA1C, a mea­sure of blood sug­ar con­trol, dropped from 7.4% to 6.6%, putting her al­most be­low di­a­bet­ic lev­els. She went from hav­ing her blood sug­ar in a healthy range 54% of the time to 84% of the time.

“This case is sem­i­nal,” CEO Michael Yang told End­points. “It’s re­al­ly the first time that it’s ever been shown that an im­plant­ed set of cells in a pa­tient can pro­duce glu­cose re­spons­es, in­sulin and im­pact clin­i­cal mea­sures in a mean­ing­ful way.”

Still the da­ta fall well short of what Yang says is the ul­ti­mate goal: a cure, ef­fec­tive­ly an ar­ti­fi­cial pan­creas. It’s just one pa­tient — a re­sult, in part, of the pan­dem­ic shut­ter­ing en­roll­ment in their study — and that pa­tient re­mains on in­sulin. And at best, Yang said, the treat­ment will work for 2 to 5 years.

Yang said they have more pa­tients ready to en­roll soon and, af­ter fin­ish­ing a 10-12 per­son study, will talk to the FDA about launch­ing a piv­otal tri­al. But they are now in a close com­pe­ti­tion with Ver­tex, who spent $900 mil­lion to buy Sem­ma in 2019, and was cleared ear­li­er this year to launch their own tri­al.

Al­though both now re­ly on im­muno­sup­pre­sants, they are each de­vel­op­ing new ap­proach­es to shield the cells from the im­mune sys­tem, which would open the treat­ment to far more pa­tients. Oth­er com­pa­nies, Ri­cor­di not­ed, are al­so de­vel­op­ing safer and more tol­er­a­ble al­ter­na­tives to im­muno­sup­pre­sants that could be used in tan­dem with the stem cell ther­a­pies.

With more re­sources, Ver­tex could beat Vi­a­Cyte to mar­ket — a prod­uct, in part, of their de­ci­sion to start with the failed en­cap­su­la­tion de­vice in 2014. But Yang, who joined the com­pa­ny ear­li­er this year, said he doesn’t have re­grets.

“I’m a front wind­shield kind of guy,” he said. “So the fact that we have this da­ta, I’m look­ing for­ward to see­ing what we can do.”

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls

Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Paul Hudson, Sanofi CEO (ROMUALD MEIGNEUX/Sipa via AP Images)

Sanofi and Am­gen are bring­ing cash to cov­er the ta­ble stakes for the Hori­zon M&A game

With the market cap on Horizon Therapeutics $HZNP pushed up to the $23 billion mark today, one of the Big Pharmas in the hunt for a major league buyout deal signaled it’s playing the M&A game with cash.

Paris-based Sanofi, where CEO Paul Hudson has been largely focused on some risky biotech acquisitions to win some respect for its future pipeline prospects, issued a statement early Friday — complying with rule 2.12 of the Irish takeover rules — making clear that while the certainty or size of an offer can’t be determined, any offer “will be solely in cash.” And Amgen CEO Robert Bradway came right in behind him, filing a statement on the London Stock Exchange overnight that any offer they may make will “likely” be in cash as well.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

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SQZ Biotech slash­es head­count by 60% as founder/CEO hits ex­it — while Syn­log­ic lays off 25%

It’s a tough time for early-stage companies developing highly promising, but largely unproven, new technologies.

Just ask SQZ Biotechnologies and Synlogic. The former is bidding farewell to its founder and CEO and slashing the headcount by 60% as it pivots from its original cell therapy platform to a next-gen approach; the latter — a synthetic biology play founded by MIT’s Jim Collins and Tim Lu — is similarly “optimizing” the company to focus on lead programs. The resulting realignment means 25% of the staffers will be laid off.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and DN­Di aim to elim­i­nate sleep­ing sick­ness in Africa with promis­ing Ph II/III re­sults for new drug

The Drugs for Neglected Diseases initiative (DNDi) and Sanofi today said that their potential sleeping sickness treatment saw success rates of up to 95% from a Phase II/III study investigating the safety and efficacy of single-dose acoziborole.

The potentially transformative treatment for sleeping sickness would mainly be targeted at African countries, according to data published today in The Lancet Infectious Diseases medical journal. The clinical trial was led by DNDi and its partners in the Democratic Republic of the Congo (DRC) and Guinea, with the authors noting: