Cedric Francois, Apellis CEO (Optum via YouTube)

Apel­lis, Beam team up to test base edit­ing against the broad field of com­ple­ment dis­or­ders

Last sum­mer, as Apel­lis was prepar­ing for the ap­proval of its po­ten­tial block­buster PNH drug Em­paveli, CEO Cedric Fran­cois sat down with his team and be­gan plot­ting the post-Em­paveli fu­ture.

Apel­lis had spent most of its 12-year his­to­ry de­vel­op­ing that one drug. The pipeline list­ed on­line con­sist­ed of sev­en items, six of which were dif­fer­ent for­mu­la­tions or ap­pli­ca­tions of the mol­e­cule. But in 2018, they start­ed div­ing deep­er in­to com­ple­ment bi­ol­o­gy — the broad im­munol­o­gy sub-field where Em­paveli op­er­ates — in hopes of ex­pand­ing their reach.

By 2020, the team had al­most every­thing it need­ed in place.

“We looked at what we were hop­ing to cre­ate, and we saw one miss­ing piece of the puz­zle,” Fran­cois told End­points News. “And that was gene edit­ing.”

So Fran­cois struck a deal. On Wednes­day Apel­lis an­nounced it’s team­ing with Beam Ther­a­peu­tics to de­vel­op gene edit­ing ther­a­pies that tar­get the com­ple­ment path­way, the an­cient branch of the im­mune sys­tem re­spon­si­ble for help­ing clear out pathogens and im­pli­cat­ed in a range of au­toim­mune dis­or­ders.

Un­der the five-year col­lab­o­ra­tion, Apel­lis will pay Beam $75 mil­lion cash — $50 mil­lion up­front and $25 mil­lion on the one-year an­niver­sary of the deal — along with un­spec­i­fied mile­stones in ex­change for pre­clin­i­cal work on six dif­fer­ent pro­grams. These will be base edit­ing ap­proach­es to dif­fer­ent genes in the com­ple­ment sys­tem in three dif­fer­ent or­gans that are ac­ces­si­ble with cur­rent de­liv­ery tech­nolo­gies: the eye, brain and liv­er. Apel­lis will han­dle sub­se­quent de­vel­op­ment, al­though Beam has the right to go 50-50 de­vel­op­ing and com­mer­cial­iz­ing one of them in the US.

John Evans

It’s the sec­ond col­lab­o­ra­tion Beam has signed since it launched out of David Liu’s Har­vard lab, af­ter its car­dio­vas­cu­lar-fo­cused deal with Verve. CEO John Evans said the com­pa­ny will look to sign more in the fu­ture, ap­ply­ing its base edit­ing tech­nol­o­gy to dis­eases where that tech could help but where they lack the re­sources or ex­per­tise to de­vel­op the drug alone.

“There are these ar­eas where we may have the tech­no­log­i­cal ex­per­tise to do some­thing but we cer­tain­ly lack the bi­ol­o­gy in­sight,” Evans told End­points. “This is ex­act­ly the kind of deal we want to do more of.”

Apel­lis and Beam are re­main­ing tight-lipped about how ex­act­ly they’ll de­ploy gene edit­ing on the com­ple­ment sys­tem, al­though Fran­cois said the com­pa­ny would try to use it both in ar­eas where they can be “con­fi­dent it will work” and ar­eas that are more ex­plorato­ry.

Ef­fec­tive­ly, that means try­ing both to cre­ate one-and-done treat­ments for dis­eases where re­searchers have al­ready proven, with con­ven­tion­al ther­a­pies, that block­ing the com­ple­ment sys­tem is ef­fec­tive (i.e. PNH) and dis­eases where there isn’t that proof of con­cept.

The deal is part of a grow­ing push to move CRISPR gene edit­ing out of rare ge­net­ic dis­or­ders and in­to more com­mon and more com­plex ail­ments. Fran­cois and Evans said Apel­lis picked Beam be­cause its base edit­ing tech­nol­o­gy al­lows for sub­tler, base-by-base al­ter­ations of DNA than tra­di­tion­al CRISPR. It was par­tic­u­lar­ly suit­ed for com­ple­ment dis­or­ders, they said, where the goal is less to knock out any par­tic­u­lar gene — as con­ven­tion­al CRISPR tech­nolo­gies do well — than to re­store a com­plex, im­muno­log­i­cal sys­tem to bal­ance.

“These are the con­se­quences of a dis­reg­u­lat­ed home­o­sta­t­ic sys­tem,” Fran­cois said. “With a gene edit­ing ap­proach, you can turn the but­tons in a way where that home­o­sta­t­ic sys­tem can re-find its na­ture bal­ance. Which may take time — may take one year or two years — but re­al­ly be a cu­ra­tive ap­proach.”

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Marianne De Backer (L) and Jeff Hatfield

Bay­er nabs star biotech Vi­vid­ion with a $2B buy­out and an ‘arms-length’ pact, pulling a part­ner out of the IPO con­ga line

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

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Tadataka Yamada (Photographer: Kiyoshi Ota/Bloomberg via Getty Images)

Sci­ence pi­o­neer, phar­ma re­search chief, glob­al health ad­vo­cate and biotech en­tre­pre­neur Tadata­ka ‘Tachi’ Ya­ma­da has died

Tadataka Yamada, a towering physician-scientist who made his name in academia before transforming drug development at GlaxoSmithKline and developing vaccines for malaria and meningitis at the Gates Foundation, died unexpectedly of natural causes at his home in Seattle Wednesday morning.

He was 76. Frazier Healthcare Partners’ David Socks confirmed his death.

Known widely by the mononym “Tachi,” Yamada had a globetrotting career and arrived in industry relatively late in life. A 2004 Independent article noted GSK had asked Yamada to stay on beyond his approaching 60th birthday, the company’s usual retirement age. Yamada would continue working for the next 17 years, steering the Gates Foundation’s global health division for 6 years, funding Jim Wilson’s gene therapy work when few would touch it, launching Takeda Vaccines and co-founding a series of high-profile biotechs.

Josh Hoffman, outgoing Zymergen CEO (Zymergen)

UP­DAT­ED: Syn­bio uni­corn Zymer­gen jet­ti­sons found­ing CEO, cuts guid­ance as cus­tomers re­port lead prod­uct does­n't work

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

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Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

UP­DAT­ED: Sanofi buys mR­NA play­er Trans­late Bio for $3.2B. And the price fits a pop­u­lar range for biotech M&A

Sanofi CEO Paul Hudson is dead serious about his intention to vault directly into contention for the future of mRNA vaccines.

A year after paying Translate Bio $TBIO a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines, Sanofi closed the deal with a buyout early Tuesday, spending $38 a share in a $3.2 billion buyout.

Translate’s stock $TBIO soared after the market closed Monday when Reuters reported the first word of the acquisition just hours ahead of the formal announcement. The wire service, though, didn’t have a price to report in its scoop, and investors chased the stock up 78% in the wild ride that followed. Once the price was announced, gains shriveled to 29% ahead of the bell.

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Matt Gline (L) and Pete Salzmann

UP­DAT­ED: Roivant bumps stake in Im­muno­vant with a $200M deal. But with M&A off the ta­ble, shares crater

Roivant has worked out a deal to pick up a chunk of stock in its majority-owned sub Immunovant $IMVT, but the stock buy falls far short of its much-discussed thoughts about buying out all of the 43% of shares it doesn’t already own.

Roivant, which recently inked a SPAC move to the market at a $7 billion-plus valuation, has forged a deal to boost its ownership in Immunovant by 6.3 points, ending with 63.8% of the biotech’s stock following a $200 million injection. That cash will bolster Immunovant’s cash reserves, giving it a $600 million war chest to fund a slate of late-stage studies for its big drug: the anti-FcRn antibody IMVT-1401.

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Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

Zymer­gen's sud­den im­plo­sion shocked biotech. A lin­ger­ing loan could make things even worse

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.

Bio­gen, Ei­sai are push­ing for an­oth­er ac­cel­er­at­ed Alzheimer's OK — this time for BAN2401

Now that the door at the FDA has been opened wide for Alzheimer’s drugs that can demonstrate a reduction in amyloid, Biogen and its partners at Eisai are pushing for a quick OK on the next drug to follow in the controversial path of aducanumab.

In a presentation to analysts, Eisai neurology chief Ivan Cheung outlined some bullish expectations for their newly-approved treatment and set the stage for what he believes will be a fast follow for BAN2401 (lecanemab) — after a dry spell in new drug development that’s lasted close to 20 years.

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Masayoshi Son, SoftBank CEO (glen photo/Shutterstock)

Soft­Bank claims $5B stake in Roche amid grand plans to in­vest in biotech — re­ports

SoftBank has made a somewhat surprising choice in its bid to pour billions into biotech and healthcare.

The Japanese behemoth has quietly acquired a $5 billion stake in Roche, making it one of the Swiss pharma giant’s largest investors, Bloomberg reported. The Financial Times later confirmed the news.

The move marks a significant departure from SoftBank’s previous strategy of channeling biotech investments through its Vision Fund into early-stage startups such as Vir, Sana, XtalPi, Umoja and Exscientia. It also placed a sizable bet on Roivant back in 2017, leading a $1.1 billion round, long before Vivek Ramaswamy and his successor, Matt Gline, landed a SPAC merger at a valuation of $7 billion.