Can a mag­net­ic cell ther­a­py re­place corneal trans­plan­ta­tion? As eight-year jour­ney leads to the clin­ic, two broth­ers un­veil bold vi­sion

Jeff Gold­berg was get­ting ac­quaint­ed with a brand new way to do corneal trans­plants when an even new­er, even bold­er idea hit him.

Jeff Gold­berg

It was al­most 10 years ago, and Gold­berg was in his first fac­ul­ty po­si­tion at Bas­com Palmer Eye In­sti­tute at the Uni­ver­si­ty of Mi­a­mi. Sci­en­tists had de­vel­oped a new way to do cornea trans­plants where in­stead of sewing a whole donor cornea — a decades-old prac­tice — they were just en­graft­ing the in­ner lay­er of cells.

“We were kind of work­ing through that surgery, and I had what I would de­scribe as one of my on­ly a-ha mo­ments in re­search,” Gold­berg, now the chair of oph­thal­mol­o­gy at Stan­ford’s By­ers Eye In­sti­tute, told End­points News.

With the mag­net­ic nanopar­ti­cles that he had been study­ing in his labs, he won­dered, couldn’t he make these cells mag­net­ic such that they can be brought to where they need to be af­ter a sim­ple in­jec­tion, skip­ping the com­plex and risky pro­ce­dure al­to­geth­er?

He told his broth­er Roger, a reti­nal spe­cial­ist who was al­so do­ing his train­ing at Bas­com Palmer. The younger Gold­berg was im­me­di­ate­ly in­trigued — enough to launch a biotech spin­off dubbed Em­me­cell to­geth­er. And eight years lat­er, they’re ready to pull the cur­tain on it.

The tech, he re­called, had broad po­ten­tial ap­pli­ca­tions and it was es­pe­cial­ly promis­ing for corneal ede­ma, which is of­ten caused by de­gen­er­a­tion in the cornea lead­ing to ex­tra flu­id get­ting caught. Too of­ten, pa­tients must bear with blur­ry vi­sion and painful blis­ters un­til their dis­ease is bad enough to jus­ti­fy a surgery.

Roger Gold­berg

“It’s a dis­ease that has no FDA-ap­proved treat­ment, there is on­ly corneal trans­plan­ta­tion avail­able with more ad­vanced dis­ease, and corneal trans­plan­ta­tion is a ma­jor surgery, it’s tech­ni­cal­ly very com­pli­cat­ed, and ac­tu­al­ly on­ly a se­lect group of sub­spe­cial­ist oph­thal­mol­o­gists even per­form these tech­ni­cal chal­leng­ing surg­eries,” he said.

Over the years the broth­ers have been work­ing with a group of aca­d­e­m­ic col­lab­o­ra­tors to ad­vance the tech. Noelia Kun­ze­vitzky, a for­mer PhD stu­dent and co-founder, took up the clin­i­cal and reg­u­la­to­ry charge, even­tu­al­ly fol­low­ing Gold­berg from Mi­a­mi to Men­lo Park.

Em­me­cell is now head­ed to the clin­ic, af­ter the FDA green­lights a Phase I tri­al for the lead prod­uct, EO2002.

Noelia Kun­ze­vitzky

The cell ther­a­py es­sen­tial­ly com­pris­es three parts: corneal cells, mag­net­ic nanopar­ti­cles tagged to them, and a shield con­tain­ing mag­nets that are small­er than the knuck­le of a pinky but strong enough to erase a cred­it card. Af­ter the cells are in­ject­ed, pa­tients would put the mag­net­ic shield in front of their closed eye­lids for a cou­ple of hours — and the nanopar­ti­cles should go in­to the blood­stream and get ex­cret­ed in urine while the cells stay.

So far, Jeff Gold­berg added, Em­me­cell has been fund­ed by pri­vate eq­ui­ty. The up­com­ing tri­al should con­firm whether the treat­ment is safe for peo­ple and if it can ben­e­fit a broad swath of pa­tients — even mild and mod­er­ate ones who wouldn’t have qual­i­fied for a surgery.

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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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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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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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.

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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Pfiz­er scoops up an an­tibi­ot­ic in rare M&A deal, bag­ging a vir­tu­al start­up op­er­at­ing on a shoe­string bud­get

Pfizer is stepping up with a rare antibiotics buyout deal today, grabbing Palo Alto, CA-based Arixa Pharmaceuticals in a bid to add a new oral version of avibactam, a beta lactamase inhibitor — or BLI — approved back in 2015 as part of the IV treatment Avycaz.

The Arixa acquisition follows some encouraging Phase I responses demonstrating that 60% to 80% of the oral drug is absorbed into the bloodstream. Only 7% of the IV version is absorbed orally, far below the 30% threshold Arixa has pointed to as a therapeutic threshold. The buyout gives Pfizer’s hospital group a line on a new oral combo with antibiotics like ceftibuten to go after drug-resistant cases of urinary tract infections and other ailments.

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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.

CRISPR Ther­a­peu­tics gets a snap­shot of off-the-shelf CAR-T suc­cess in B-cell ma­lig­nan­cies — marred by the death of a pa­tient

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

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