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.

Ryan Watts, Denali CEO

Bio­gen hands De­nali $1B-plus in cash, $1B-plus in mile­stones to part­ner on late-stage Parkin­son’s drug

Biogen is handing over more than a billion dollars cash to partner with the up-and-coming neurosciences crew at Denali on a new therapy for Parkinson’s. And the big biotech is ready to pile on more than a billion dollars more in milestones — if the alliance is a success.

For Biogen $BIIB, the move on Denali’s small molecule inhibitors of LRRK2 puts them in line to collaborate on a late-stage program for DNL151, which is scheduled to start next year.

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Bio­haven adds near­ly $1B in Nurtec deals with Roy­al­ty Phar­ma, Sixth Street

Biohaven just added nearly $1 billion to their balance sheet.

On Friday morning, the neuroscience biotech announced a pair of creative agreements with Royalty Pharma and the investment firm Sixth Street to bolster the commercial launch of their new migraine drug, Nurtec. Biohaven will sell a sliver of its royalties on Nurtec and 3% of the royalties on their experimental migraine drug zavegepant to Royalty Pharma as part of a larger agreeement that will pay $450 million. At the same time, the company announced they took out a $500 million loan from Sixth Street.

Ab­b­Vie set­tles in­sur­ance fraud suit, agrees to tweak nurse am­bas­sador pro­gram; CStone aims for NSCLC OK with pos­i­tive PhI­II da­ta

AbbVie has resolved a California lawsuit alleging insurance fraud in the promotion of its cash cow Humira, paying $24 million to settle things with the state’s insurance regulator.

The settlement comes almost four years after a whistleblower first reported AbbVie’s practice of deploying registered nurses to visit patients at home or call them by phone to ensure that Humira prescriptions are filled. AbbVie was also charged with providing illegal kickbacks to doctors in an attempt to encourage them to prescribe Humira for a range of anti-inflammatory diseases.

Per­cep­tive fields SPAC #3 as an­oth­er group of biotechs scoops up $364M in lat­est Nas­daq romp

There’s no sign that the windfall of cash dropping biotech’s way on Wall Street is abating. Three more biopharmas priced IPOs on Thursday and Friday morning, riding a historic boom with a $364 million payoff.

London-based biotech Freeline Therapeutics took home the lion’s share of the cash with $159 million after pricing 8,823,529 shares at $18 a pop. Checkmate Pharmaceuticals, of Cambridge, MA, raised $75 million with an offer of 5 million shares at $15 — right at the midpoint of its range. And Arya Sciences Acquisition Corp III, the third in a series from Perceptive, priced 13,000,000 shares at $10 per share.

Covid-19 roundup: Gates Foun­da­tion pours $150M in­to In­dia’s Serum In­sti­tute; Pfiz­er teams with Gilead on remde­sivir

By CEO and scion Adar Poonawalla’s estimation, the Serum Institute in India has already poured hundreds of millions of dollars into scaling up the unproven Covid-19 vaccine being developed by AstraZeneca and Oxford for use in low and middle income countries. It’s meant taking on a risk that other companies, including AstraZeneca, have mitigated with huge amounts of government funding.

Now, for the first time, Poonawalla is getting some outside help. The Gates Foundation has agreed to pay the institute $150 million to supply 100 million vaccines to India and other emerging economies next year, Reuters reported. That includes both the vaccine being developed by AstraZeneca and the one being developed by Novavax. Those vaccines will be available in 92 countries and be priced at $3 per dose.

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President Trump speaks with members of the media before boarding Marine One (AP Images)

'Oc­to­ber is com­ing,' and every­one still wants to know if a Covid-19 vac­cine will be whisked through the FDA ahead of the elec­tion

Right on the heels of a lengthy assurance from FDA commissioner Stephen Hahn that the agency will not rush through a quick approval for a Covid-19 vaccine, the President of the United States has some thoughts on timing he’d like to share.

In an exchange with Fox News’ Geraldo Rivera on Thursday, President Trump allowed that a vaccine could be ready to roll “sooner than the end of the year, could be much sooner.”

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UP­DAT­ED: No­vavax her­alds the lat­est pos­i­tive snap­shot of ear­ly-stage Covid-19 vac­cine — so why did its stock briefly crater?

High-flying Novavax $NVAX became the latest of the Covid-19 vaccine players to stake out a positive set of biomarker data from its early-stage look at its vaccine in humans.

Their adjuvanted Covid-19 vaccine was “well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera,” the company noted. According to the biotech:

All subjects developed anti-spike IgG antibodies after a single dose of vaccine, many of them also developing wild-type virus neutralizing antibody responses, and after Dose 2, 100% of participants developed wild-type virus neutralizing antibody responses. Both anti-spike IgG and viral neutralization responses compared favorably to responses from patients with clinically significant COVID‑19 disease. Importantly, the IgG antibody response was highly correlated with neutralization titers, demonstrating that a significant proportion of antibodies were functional.

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Alex­ion cre­ates new post for chief di­ver­si­ty of­fi­cer; Bar­ry Greene step­ping down at Al­ny­lam, Yvonne Green­street named as suc­ces­sor

Alexion has carved out a new position for chief diversity officer and filled it with an inside promotion.

Uzair Qadeer will now be responsible for their “diversity, inclusion and belonging” strategy, looking to reshape the biotech’s corporate culture. A veteran of Deloitte and Bristol Myers Squibb, Qadeer was working on executive coaching and helping create the diversity program he now leads.