Gene edit­ing up­start backed by Broad star Feng Zhang nabs $135M, blue­print­ing a plat­form biotech for the long haul

Im­age: John Evans. BEAM

Last May, when Beam Ther­a­peu­tics had its com­ing out par­ty, com­plete with $87 mil­lion in in­vest­ment back­ing, a sol­id core of 15 staffers and a dream team of sci­en­tif­ic co-founders, CEO John Evans was sure they were set for sev­er­al years of R&D work.

But that’s not the way it played out. 

At a time when the mon­ey spig­ot is wide open for the right biotech plans and teams, Beam is an­nounc­ing that it went back to the ven­ture well to flesh out its syn­di­cate and added a whop­ping $135 mil­lion mon­ster round to the bank. That core team has swelled to 70, with more hires on the way as the ex­ec­u­tive team pre­pares to host a staff of 130. And Evans says they are well on their way to cre­at­ing a pipeline of pro­grams be­gin­ning with the 10 pre­clin­i­cal ef­forts they have un­der­way in new­ly opened labs.

“We de­cid­ed to go broad,” says Evans. “There are like­ly a lot of places for this be­ing the best op­tion for pa­tients.”

The big idea at Beam is that in­stead of us­ing a spe­cial gene edit­ing cut­ting tool to do per­ma­nent sur­gi­cal al­ter­ations of DNA, they’re pen­cilling in their ed­its with base edit­ing en­zymes that mod­i­fy nu­cle­obas­es  — cor­rect­ing or mod­i­fy­ing dis­ease caus­ing genes or writ­ing in code for ge­net­ic vari­a­tions that can pre­vent dis­ease. It’s one of a group of next-wave gene edit­ing out­fits that have come along in the wake of pi­o­neers like Ed­i­tas, In­tel­lia or CRISPR Ther­a­peu­tics. 

The Broad In­sti­tute’s Feng Zhang, who has backed Ed­i­tas and oth­er up­starts in the field, joined with Broad col­league David Liu and Har­vard Med’s Kei­th Joung to pro­vide the sci­en­tif­ic in­spi­ra­tion.


Im­age: David Liu, Kei­th Joung, Feng Zhang. BEAM

At this point in Beam’s ex­is­tence, go­ing broad means stick­ing with gen­er­al­i­ties and avoid­ing specifics about the work they’re do­ing in the lab. That’s stan­dard op­er­at­ing pro­ce­dure in start­up land. The de­tails can start to come lat­er as they be­gin to pub­lish their work and line up INDs for the first clin­i­cal pro­grams now in the works.

The plan now is to tran­si­tion from their plat­form con­cept in­to a pipeline sto­ry, says Evans. And they’re adding AAV vec­tors, lipid nanopar­ti­cles and oth­er kinds of tech that can get their ed­its wher­ev­er they need to go in the body.

“We’re not go­ing to throw this over the wall to a phar­ma com­pa­ny,” adds the CEO, adding that the team at Beam in­tends to build it all in house from end-to-end — much like they did at Agios, where he was SVP of cor­po­rate de­vel­op­ment.

Go­ing all the way means in­vest­ing heav­i­ly in man­u­fac­tur­ing ear­ly on and lay­ing the foun­da­tion for an even­tu­al shift to com­mer­cial­iza­tion. And that means plan­ning for the long haul, with lots of cap­i­tal re­quired to make it a re­al­i­ty.

That takes a syn­di­cate of back­ers ready for the long haul as well. And here’s who’s back­ing the B round: new in­vestors Red­mile Group, LLC; Cor­morant As­set Man­age­ment; GV; Al­ti­tude Life Sci­ence Ven­tures, and “ad­di­tion­al undis­closed in­vestors.” Then there’s al­so new mon­ey from ex­ist­ing in­vestors: F-Prime Cap­i­tal, ARCH Ven­ture Part­ners, Eight Roads Ven­tures, and Omega Funds.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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