Third Rock seeds cell ther­a­py start­up with $95M in bid to cure MS

In 2018, for­mer Bio­gen ex­ec­u­tives Saman­tha Singer and Richard Ran­so­hoff were work­ing with Third Rock on a new way to treat au­toim­mune dis­eases when one of the VC’s part­ners asked what Ran­so­hoff thought of a wild­ly dif­fer­ent ap­proach to tack­ling those be­dev­il­ing dis­or­ders.

Richard Ran­so­hoff

Third Rock want­ed to build a com­pa­ny that would turn a pro­tec­tive class of im­mune cells called reg­u­la­to­ry T cells in­to ther­a­pies, and an­oth­er team had spent the last year and a half try­ing to find the best con­di­tion it would work for. Fi­nal­ly, they set­tled on mul­ti­ple scle­ro­sis, a dis­ease Ran­so­hoff had stud­ied for three decades as an aca­d­e­m­ic. They want­ed his opin­ion: What do you think?

“I said I don’t think any­thing,” Ran­so­hoff re­called. But he be­gan read­ing their da­ta and won­der­ing about pa­tients with the most ad­vanced forms of MS, those whose symp­toms grad­u­al­ly progress.

“There was ac­tu­al­ly a eu­re­ka mo­ment,” he told End­points News. It was “the on­ly thing I could think of that might ben­e­fit [these] peo­ple.”

In time, Singer and Ran­so­hoff’s first project would fiz­zle, the vi­sion prov­ing too ear­ly for its era, and the pair would set about turn­ing the cell ther­a­py idea in­to a com­pa­ny, en­list­ing top aca­d­e­mics as founders and hir­ing out a team. The start­up, Aba­ta Ther­a­peu­tics, emerged from stealth Wednes­day with $95 mil­lion in a Third Rock-led Se­ries A round, three pro­grams and a plan to be in the clin­ic by 2025.

Aba­ta re­lies on reg­u­la­to­ry T cells. The bounc­ers of the im­mune sys­tem, they rec­og­nize the body’s own pro­teins and tell oth­er im­mune cells not to at­tack when they see cells stud­ded with those re­cep­tors.

Saman­tha Singer

In au­toim­mune dis­or­ders, though, that sys­tem goes awry; the im­mune sys­tem at­tacks healthy cells. Aba­ta is bet­ting it can cor­rect by tak­ing a pa­tients’ own reg­u­la­to­ry T cells  — some­times called Tregs — and equip­ping them with a re­cep­tor for the pro­tein that the body is at­tack­ing. In MS, with the help of some oth­er tweaks, those cells would set­tle in­to the cen­tral ner­vous sys­tem and pump out no-go sig­nals to the rest of the im­mune ar­se­nal.

The goal, Singer said, would be to not on­ly pre­vent fur­ther dam­age but al­so al­low the ner­vous sys­tem to re­gen­er­ate the myelin sheaths that pa­tients lose dur­ing the course of the dis­ease.

A Treg’s “na­tive job is to pre­vent au­toim­mu­ni­ty,” Singer told End­points News.  This is “in some ways, very much lever­ag­ing the nat­ur­al abil­i­ties of the cell.”

Singer will serve as the com­pa­ny’s per­ma­nent CEO, her first chief gig af­ter a pair of at top op­er­at­ing po­si­tions at Bio­gen and the Broad In­sti­tute. Ran­so­hoff will serve as CMO.

Aba­ta is one of a small hand­ful of star­tups that have cropped over the past two years to bring cell ther­a­py in­to au­toim­mune dis­eases, join­ing Sono­ma, Gen­tiBio, and Or­ca Bio, among oth­ers. None have pre­sent­ed much da­ta so far, though, and the field re­mains nascent, com­pared to cell ther­a­pies for can­cer or, in the au­toim­mune field, an­ti­bod­ies and bis­pecifics — the ap­proach that has gen­er­at­ed the most ven­ture and phar­ma back­ing in the past half-decade.

They’ll look to keep an edge in part through a part­ner­ship with David Hal­lal’s El­e­vate Bio to man­u­fac­ture their cell ther­a­pies. They now have 15 em­ploy­ees, with plans to dou­ble it by the end of the year.

In mul­ti­ple scle­ro­sis, they will start by test­ing the ther­a­py on the most se­vere pa­tients, where they hope the drug could pro­vide a one-time cure.  Be­yond MS, Aba­ta is de­vel­op­ing Treg treat­ments for type 1 di­a­betes and in­clu­sion body myosi­tis, a pain­less mus­cle-wast­ing dis­ease.

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.

Tien Lee, Aardvark Therapeutics CEO

Emerg­ing from stealth mode, Aard­vark rounds up enough cash to put its lead drug through Prad­er-Willi PhII

When Aardvark Therapeutics CEO Tien Lee started his work on the biotech’s lead candidate, appetite suppression was the goal for the small molecule.  Soon after, his team started to see added benefits with lower blood glucose levels and anti-inflammatory activity. On the tail end of that, the company has emerged from stealth mode and announced today that they’ve raised enough cash in the B round to cover mid-stage development work.

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.

Covid-19 roundup: Pfiz­er im­pos­es vac­cine man­date for US work­ers; WHO calls for mora­to­ri­um on boost­ers, while some coun­tries make plans any­way — re­port

As the US struggles to keep pace with the fast-spreading Delta variant, big companies like Walmart and Disney are imposing vaccine mandates for some workers. It may come as no surprise that Pfizer — the Big Pharma behind the US’ first authorized Covid-19 vaccine — is joining them.

Pfizer will start requiring all US employees and contractors to get vaccinated, or participate in weekly Covid-19 testing, spokesperson Pamela Eisele told Reuters. Workers outside the US are strongly urged to get a vaccine if they can, according to the report. And those with medical conditions or religious objections can seek accommodations.

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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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.

UK re-in­ves­ti­gates Pfiz­er's eye-pop­ping price goug­ing on an epilep­sy drug

When a drugmaker raises the price of a drug in the US by more than 2,000% overnight, and without any particular reason for that increase, nothing typically happens to the company. No fines, no court orders, just business as usual.

Martin Shkreli’s decades-old anti-parasitic drug Daraprim was the perfect example — massive price spike on an old drug, lots of media attention, public outcry, congressional committees dragging his former company through multiple hearings, and at the end of it? Nothing happened to the price or the company (until generic competition came).

Thomas Lingelbach, Valneva CEO

A small vac­cine de­vel­op­er fa­vored by the UK gov­ern­ment in Covid-19 touts a PhI­II first in chikun­gun­ya

Before Valneva garnered the favor of the UK government as a potential supplier of Covid-19 vaccines, the French biotech prided itself on being the first company to bring a chikungunya vaccine into Phase III.

It now has positive pivotal results to back up the breakthrough therapy designation the FDA granted just weeks ago.

There are currently no approved jabs to prevent chikungunya virus infection despite decades of R&D efforts, a fact that underscores just how arduous traditional vaccine development can be, particularly for neglected tropical disease. In a absence of a major commercial market, the US government and NGOs such as CEPI have deployed various grants and incentives to spur on a small crew of academics and industry players, with Merck, via its acquisition of Themis, claiming a spot in that race.