Af­ter a long, hard slog to the clin­ic, stem cell play­er Vi­a­Cyte fu­els up with $105M for its next big at­tack on di­a­betes

Over the 14 years since 3 com­pa­nies merged to­geth­er to cre­ate the stem cell play­er we now call Vi­a­Cyte in San Diego, the biotech has been op­er­at­ing on a steady stream of cash from the Cal­i­for­nia In­sti­tute of Re­gen­er­a­tive Med­i­cine as well as the non-prof­it JDRF or­ga­ni­za­tion. 

But with one stem cell prod­uct in the clin­ic and a next-gen prod­uct about to go back in­to hu­man test­ing af­ter a re­vamp — plus a new­ly formed col­lab­o­ra­tion with the gene edit­ing spe­cial­ists at CRISPR Ther­a­peu­tics — CEO Paul Laikind is ready to dou­ble down on a strate­gic bet that the crew at Vi­a­Cyte can make some ma­jor clin­i­cal progress over the next 2 to 3 years.

Paul Laikind, Vi­a­Cyte

To­day Laikind is un­veil­ing an $80 mil­lion ven­ture round, the first in years, that brings to­geth­er a set of new in­vestors — Bain Cap­i­tal Life Sci­ences and joined by TPG and RA Cap­i­tal Man­age­ment —who are join­ing Sander­ling and some in­di­vid­ual back­ers on the next leg of the jour­ney. To­geth­er with the $15 mil­lion CRISPR paid them to forge their tie-up and an­oth­er $10 mil­lion from part­ners at WL Gore and As­so­ci­ates, Laikind’s team now has $105 mil­lion to fund this next leg of prod­uct de­vel­op­ment.

“With this new fund­ing we’re re­al­ly open­ing the gate, say­ing ‘lets get this done,’” says the CEO.

“We stuck to our guns of what we want to ac­com­plish here,” adds Laikind. “It’s tak­en 7-8 years to de­vel­op the tech­nol­o­gy for dif­fer­en­ti­at­ing the cells in a reg­u­la­to­ry-com­pli­ant way.” 

Pe­ter Kolchin­sky

Laikind has been woo­ing RA’s Pe­ter Kolchin­sky for some time now. And Kolchin­sky and Bain’s Adam Kop­pel have a long­stand­ing re­la­tion­ship forg­ing deals to­geth­er, which helped get this syn­di­cate to gel.

Like a lot of the stem cell sur­vivors in Cal­i­for­nia, Vi­a­Cyte has seen the burst of glo­ry and hype suc­ceed­ed by the bleak­ness of the marathon run that stem cell R&D has proven to be — just like many oth­er new tech­nolo­gies in biotech. And it’s man­aged to keep plug­ging away at it to get to the re­newed era of op­ti­mism that has been lift­ing the sur­vivor’s prospects over the last cou­ple of years.

Adam Kop­pel

The lead prod­uct at Vi­a­Cyte is called PEC-Di­rect, de­signed to fer­ry pan­cre­at­ic prog­en­i­tor cells from stem cells that can se­crete in­sulin. It’s prob­lem­at­ic, though, trig­ger­ing the kind of im­mune re­sponse that re­quires im­muno­sup­pres­sion — which nec­es­sar­i­ly lim­its its us­es to 10% of high-risk Type 1 di­a­betes pa­tients will­ing to put up with the risks.

PEC-En­cap is an en­cap­su­lat­ed prod­uct that us­es Gore-tex like ma­te­r­i­al to de­liv­er the same pan­cre­at­ic prog­en­i­tor cells with­out spark­ing the for­eign body re­sponse that spurs the body to coat their de­vice with cells. And this can be used with­out im­mune sup­pres­sion, which would sig­nif­i­cant­ly ex­pand the mar­ket for Vi­a­Cyte.

CRISPR Ther­a­peu­tics $CR­SP, one of the lead­ers in CRISPR/Cas9 gene edit­ing with a big in­ter­est in re­gen­er­a­tive med­i­cine, is step­ping in to use their im­mune-eva­sive tech to cre­ate a new line of off-the-shelf cells that can do the job with­out kick­ing in the re­jec­tion re­sponse.

There are 55 staffers at the biotech, which the CEO says will grow now that he has his big fi­nanc­ing pack­age com­plet­ed. Vi­a­Cyte does its own man­u­fac­tur­ing work, and that team will need to ex­pand along with a spe­cial group ded­i­cat­ed to the part­ner­ship with CRISPR.

Charles Baum, Mirati CEO

Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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