A fast-mov­ing en­try in the gene edit­ing race is speed­ing to the clin­ic, fu­eled by an $83.5M round

The last time I talked to Arthur Tzian­a­bos a lit­tle more than a year ago, the CEO at Ho­mol­o­gy Med­i­cines had a team of about 10 and a new ap­proach to in vi­vo gene trans­fer and gene edit­ing that was still in pre­clin­i­cal liftoff mode. To­day, Tzian­a­bos is whip­ping the cov­er off a whop­ping $83.5 mil­lion ven­ture round with some A-lis­ter sup­port so his team of 40-plus can take their work in­to hu­mans for the first time.

“We re­al­ly have made a tremen­dous amount of progress,” Tzian­a­bos tells me by way of catch­ing up.

There’s a lead in­di­ca­tion now — an in­born er­ror of me­tab­o­lism in­volv­ing the liv­er is as spe­cif­ic as he wants to get right now — with a long list of pre­clin­i­cal projects in CD34 dis­eases, cys­tic fi­bro­sis, CNS dis­eases, Duchenne mus­cu­lar dy­s­tro­phy and so on. When you’re a pre­clin­i­cal biotech work­ing on the foun­da­tion for a plat­form tech that seeks to ed­it and cor­rect genes us­ing your own ade­no-as­so­ci­at­ed virus vec­tors, you can think about what you want to keep and what you want to part­ner.

Ho­mol­o­gy is one of the up­starts trav­el­ing one step be­hind the pi­o­neer­ing crews that boot­ed up gene edit­ing in a big way. That group in­cludes Ed­i­tas, CRISPR Ther­a­peu­tics and In­tel­lia — which all made vir­tu­al­ly overnight de­buts on­to the pub­lic mar­kets with a set of IPOs that at­tract­ed a tremen­dous amount of in­vestor at­ten­tion — and which sagged as the long clin­i­cal road to an ac­tu­al ther­a­py came in­to view.

These days, the pi­o­neers al­so are spend­ing a sig­nif­i­cant amount of time wran­gling over patents as they work to get in­to the clin­ic. It’s no easy task birthing a new tech­nol­o­gy, as Ed­i­tas found out when it had to de­lay its time­line re­cent­ly in or­der to work through a de­lay on the man­u­fac­tur­ing side.

Tzian­a­bos and his team are learn­ing from the lead­ers, and may al­so be mov­ing faster to­ward the clin­ic than the out-front ri­vals know.

“We want to be there in a short pe­ri­od of time,” says the CEO about hu­man tri­als. (No, he isn’t be­ing more spe­cif­ic than that.) He has plen­ty of cash now to get through a proof-of-con­cept tri­al, and a chance to sign up a part­ner or two to help ex­pand the pipeline work more quick­ly.

Tzian­a­bos gives his Bed­ford, MA-based biotech high scores for their pre­clin­i­cal ef­fort on fine tun­ing gene trans­fer and edit­ing, start­ing with a page out of the book of na­ture on the way DNA is re­paired to point to an in vi­vo ap­proach that can be a sim­pler and more di­rect means of po­ten­tial­ly cur­ing ge­net­ic dis­eases.

But he’s al­so start­ing to look at what needs to hap­pen on man­u­fac­tur­ing to pre­vent fu­ture foul-ups.

“I think peo­ple got a lit­tle over their skis on that point,” he says about the field. And he has built up in­ter­nal and ex­ter­nal man­u­fac­tur­ing ops.

“CMC, bi­o­log­ics man­u­fac­tur­ing is com­pli­cat­ed any­way,” says Tzian­a­bos, point­ing to the cri­sis that Gen­zyme went through be­fore Sanofi ac­quired the com­pa­ny. The new gene edit­ing com­pa­nies are com­ing straight out of acad­e­mia, he adds, and that kind of tech al­ways takes time to in­dus­tri­al­ize.

At Ho­mol­o­gy, says the CEO, “that was one of our main fo­cus­es right out of the gate.”

Add it all up, he says, and “I would put us not too far be­hind where these guys (the pi­o­neers) are. That’s what at­tract­ed this team to the tech­nol­o­gy.”

It is al­so what at­tract­ed Deer­field, which led the round. New in­vestors in­clude Fi­deli­ty Man­age­ment and Re­search Com­pa­ny, HBM Health­care In­vest­ments, Mav­er­ick Ven­tures, No­var­tis, Rock Springs Cap­i­tal, Vi­da Ven­tures, Vi­vo Cap­i­tal and Alexan­dria Ven­ture In­vest­ments. Ex­ist­ing in­vestors 5AM Ven­tures, ARCH Ven­ture Part­ners and Temasek al­so jumped back in.

As of now, Ho­mol­o­gy has raised a to­tal of $127 mil­lion in a lit­tle more than a year, of­fer­ing an­oth­er ex­am­ple of the kind of funds that are be­ing spent to pur­sue a rad­i­cal new tech­nol­o­gy. It’s still ear­ly stages, with years of clin­i­cal work ahead, but Ho­mol­o­gy is mov­ing fast.

Amarin CEO John Thero discussing the company's plans for Vascepa, August 2019 — via Bloomberg

Amarin wins a block­buster ap­proval from the FDA. Now every­one can shift fo­cus to the patent

For all those people who could never quite believe that Amarin $AMRN would get an expanded label with blockbuster implications, the stress and anxiety on display right up to the last minute on Twitter can now end. But new, pressing questions will immediately surface now that the OK has come through.

On Friday afternoon, the FDA stamped its landmark approval on the industrial strength fish oil for reducing cardio risks for a large and well defined population of patients. The approval doesn’t give Amarin everything it wants in expanding its use, losing out on the primary prevention group, but it goes a long way to doing what the company needed to make a major splash. The approval was cited for patients with “elevated triglyceride levels (a type of fat in the blood) of 150 milligrams per deciliter or higher. Patients must also have either established cardiovascular disease or diabetes and two or more additional risk factors for cardiovascular disease.”

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Sarep­ta was stunned by the re­jec­tion of Vyondys 53. Now it's stun­ning every­one with a sur­prise ac­cel­er­at­ed ap­proval

Sarepta has a friend in the FDA after all. Four months after the agency determined that it would be wrong to give Sarepta an accelerated approval for their Duchenne MD drug golodirsen, regulators have executed a stunning about face and offered the biotech a quick green light in any case.

It was the agency that first put out the news late Thursday, announcing that Duchenne MD patients with a mutation amenable to exon 53 skipping will now have their first targeted treatment: Vyondys 53, or golodirsen. Having secured the OK via a dispute resolution mechanism, the biotech said the new drug has been priced on par with their only other marketed drug, Exondys 51 — which for an average patient costs about $300,000 per year, but since pricing is based on weight, that sticker price can even cross $1 million.

Sarepta shares $SRPT surged 23% after-market to $124.

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UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Arie Belldegrun (Photo: Jeff Rumans for Endpoints News)

Ju­ry finds Gilead li­able for $585M and big roy­al­ties in Kite CAR-T patent case

A Kite deal that’s already become a burden on Gilead’s back just got heavier as a California jury has ruled Gilead must pay Bristol-Myers Squibb and Sloan Kettering $585 million plus a 27.6% royalty for patent infringement committed by its subsidiary. The ruling is almost certain to be appealed.

Kite Pharma — founded by Arie Belldegrun, now focused on a next-gen CAR-T company — has been facing a lawsuit since the day its first CAR–T therapy won approval in October, 2017. Juno Therapeutics and Sloan Kettering filed a complaint saying Kite had copied its technology. Gilead acquired Kite in June of that year for $11.9 billion.  Juno was acquired the following year by Celgene for $9 billion, before Celgene was acquired by Bristol-Myers Squibb in 2019.

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Paul Biondi (File photo)

Paul Biondi's track record at Bris­tol-My­ers cov­ered bil­lions in deals of every shape and size. Here's the com­plete break­down

Paul Biondi was never afraid to bet big during his stint as business development chief at Bristol-Myers Squibb. And while the gambles didn’t all pay out, by any means, his roster of pacts illustrates the broad ambitions the pharma giant has had over the last 5 years — capped by the $74 billion Celgene buyout.

On Thursday, we learned that Biondi had exited the company. And Chris Dokomajilar at DealForma came up with the complete breakdown on every buyout, licensing pact and product purchase Bristol-Myers forged during his tenure in charge of the BD team at one of the busiest companies in biopharma.

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FDA ex­pert pan­el unan­i­mous­ly rec­om­mends ap­proval for Hori­zon Ther­a­peu­tics eye drug

An FDA advisory committee noted with concern a small safety database but unanimously endorsed a Horizon Therapeutics drug for a rare eye autoimmune disease that can blind patients: teprotumumab for thyroid eye disease (TED).

“It was a pretty easy vote,” said Erica Brittain, an NIH biostatistician and one of the 12 panelists on FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee.

This image shows a lab technician measuring the zone of inhibition during an antibiotic sensitivity test, 1972. The zone of inhibition is measured and compared to a standard in order to determine if an antibiotic is effective in treating the bacterial infection. (Gilda Jones/CDC via Getty Images)

Bio­phar­ma has aban­doned an­tibi­ot­ic de­vel­op­ment. Here’s why we did, too.

Timing is Everything
When we launched Octagon Therapeutics in late 2017, I was convinced that the time was right for a new antibiotic discovery venture. The company was founded on impressive academic pedigree and the management team had known each other for years. Our first program was based on a compelling approach to targeting central metabolism in the most dangerous bacterial pathogens. We had already shown a high level of efficacy in animal infection models and knew our drug was safe in humans.

Shehnaaz Suli­man dives back in­to Alzheimer's at Alec­tor; Pyx­is re­cruits Spring­Works founder Lara Sul­li­van as CEO

Amid Shehnaaz Suliman’s lengthy resume it could be easy to miss her stint leading early-stage Alzheimer’s R&D at Genentech, where she oversaw a program for the ill-fated crenezumab and initiated one of the first prevention studies around the devastating neurodegenerative disease. But it is this experience that she — after thinking long and hard about her next career move over the past months — will be leaning heavily on as the first president and COO of Alector.

PhII fail­ure in rare neu­rode­gen­er­a­tive dis­ease? No mat­ter, Bio­gen will mo­tor on in Alzheimer's

Biogen’s fierce focus on disorders of the brain has hit another roadblock.

On Friday, the US drugmaker — which recently resurrected its amyloid-targeting Alzheimer’s drug, aducanumab — said its anti-tau drug, gosuranemab, failed a mid-stage study in patients with progressive supranuclear palsy (PSP), a rare brain disorder that results from deterioration of brain cells that control movement and thought.