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.”

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

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Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

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Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

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Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Reshma Kewalramani, Vertex CEO (Vertex via YouTube)

Bat­tling a line­up of skep­tics, Ver­tex claims an­oth­er ear­ly clin­i­cal win — this time in kid­ney dis­ease

Vertex claimed its second early-stage win of the fall Wednesday, announcing positive results in a small study on a genetically defined form of kidney disease.

The 16-patient, Phase II trial focused on patients with focal segmental glomerulosclerosis, a rare disease where kidneys are unable to filter blood properly. Over 13 weeks on an experimental pill, the level of protein in the patients’ urine fell by an average of 47.6%.

As first Omi­cron case in US crops up, re­searchers won­der: which an­ti­bod­ies, vac­cines will hold up?

As Covid-19 drug and vaccine developers race to figure out which of their products might be hampered by the new variant, the CDC on Wednesday afternoon announced the first confirmed case of the Omicron variant (B.1.1.529) in the US, found in San Francisco.

The unidentified individual was a traveler who returned from South Africa on Nov. 22, 2021, was fully vaccinated, and had mild symptoms that the CDC described as improving. All close contacts have been contacted and have tested negative, the centers said.

Mod­er­na los­es lat­est bat­tle in key vac­cine de­liv­ery patent fight as fed­er­al ap­peal falls flat

The US Court of Appeals for the Federal Circuit on Wednesday rejected Moderna’s attempt to overturn key patents related to the delivery vehicle for its Covid-19 vaccine after the biotech sought to preempt a potentially risky infringement lawsuit.

For years, Moderna has been battling a tiny Pennsylvania biotech known as Arbutus over patents for a technology required to deliver its mRNA drugs and vaccines, known as lipid nanoparticles or LNP. Moderna is concerned there’s a substantial risk that Arbutus will assert the ’069 patent in an infringement suit targeting Moderna’s Covid-19 vaccine, particularly as Arbutus has boasted of its patent protection and refused to grant a covenant not to sue Moderna.

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Jeff Baxter, VBI Vaccines CEO (ChromaDex)

VBI Vac­cines ex­ecs tout ap­proval of new triple-anti­gen he­pati­tis B shot. Can they sell it?

VBI Vaccines celebrated a new approval Wednesday morning, announcing the FDA has greenlighted its hepatitis B vaccine for adults. But questions remain on how well the new shot will sell.

The biotech intends to hit the market in the first quarter of next year, joining three other adult hepatitis B vaccines from Merck, Dynavax and GlaxoSmithKline. CEO Jeff Baxter said in an analyst call Wednesday the price of VBI’s shot, branded as PreHevbrio, won’t be revealed until commercialization, but claimed it would be “highly competitive.”