Pfiz­er biotech spin­out nabs $125M megaround as Bain-backed syn­di­cate bets on its late-stage game plan

Spring­Works Ther­a­peu­tics launched in the fall of 2017 with a whop­ping $103 mil­lion round and some well-de­vel­oped Pfiz­er as­sets that the com­pa­ny had high hopes for. And now Bain, Pfiz­er and the rest of the found­ing in­vestors have joined hands with an even big­ger group of in­vestors led by Per­cep­tive to bankroll a pair of late-stage stud­ies ready to be­gin in a mat­ter of weeks.

The Se­ries B weighs in at a heavy­weight $125 mil­lion, bring­ing their to­tal raise to $228 mil­lion, with ac­cess to more if need­ed.

The fo­cus of this lat­est megaround is cen­tered on two drugs that are be­ing re­pur­posed for rare dis­eases: nirogace­s­tat, a gam­ma sec­re­tase in­hibitor for the treat­ment of desmoid tu­mors (rare soft tis­sue tu­mors), and PD-0325901, a MEK in­hibitor for the treat­ment of neu­rofi­bro­mato­sis type 1-as­so­ci­at­ed plex­i­form neu­rofi­bro­mas. 

Both of these tar­gets are well de­fined, with mul­ti­ple tri­als for re­lat­ed drugs over the past decade or more. The gam­ma sec­re­tase in­hibitor was born in the Alzheimer’s field, where Eli Lil­ly ex­pe­ri­enced a colos­sal dis­as­ter years ago. That MEK in­hibitor, Spring­Works ex­ecs say, al­so has po­ten­tial to make good as a back­bone com­bi­na­tion drug in on­col­o­gy, fol­low­ing oth­er ap­proved ther­a­pies.

Im­age: Saqib Is­lam. CHECK­RARE via YOUTUBE

The big fi­nanc­ing takes the com­pa­ny all the way through the two late-stage stud­ies, says CEO Saqib Is­lam, with a clas­sic cross­roads that could ar­rive in H2 of next year if they do an in­ter­im analy­sis and if it’s pos­i­tive, set­ting up a pos­si­ble FDA ap­pli­ca­tion. The gam­ma sec­re­tase study should read out in ear­ly 2021.

The com­pa­ny, which now has 41 staffers head­ed to about 70 at the end of the year, says the CEO, has the ca­pac­i­ty to go the dis­tance by it­self in rare dis­eases. And as a vet of Alex­ion, Is­lam — who more re­cent­ly did deals for Mod­er­na — says they are ready to do what’s nec­es­sary to launch com­mer­cial op­er­a­tions. Larg­er in­di­ca­tions could be cov­ered with part­ner­ships, and he point­ed to a tie-up they have with BeiGene, com­bin­ing their RAF dimer in­hibitor li­fi­rafenib (BGB-283) with the MEK.

More deals are clear­ly in the works, he adds, which could trig­ger a faster ex­pan­sion of the staff and ca­pa­bil­i­ties at Spring­Works, which has the re­search team in RTP and the cor­po­rate staff now head­quar­tered in Stam­ford, CT.

I asked Is­lam the ob­vi­ous ques­tion: With a syn­di­cate this size with these play­ers, and plans to push through piv­otal tri­als, an IPO would seem to be in the cards as long as the mar­ket holds up. He laughed a lit­tle and lim­it­ed him­self to the stan­dard re­frain: All fi­nanc­ing op­por­tu­ni­ties are un­der re­view.

Pfiz­er has helped craft sev­er­al new com­pa­nies like this re­cent­ly. There’s Bain-backed Cerev­el as well as Al­lo­gene from Arie Bellde­grun and David Chang. In every case, Pfiz­er ex­ecs of­floaded as­sets they no longer want­ed in the pipeline, but which they felt had re­al com­mer­cial prospects in the right hands. Is­lam al­so not­ed that they are in­clud­ed in Pfiz­er’s port­fo­lio re­view process, which could al­so trig­ger more deals down the road as Pfiz­er con­tin­ues to shed projects.

Per­cep­tive Ad­vi­sors led the show for the Se­ries B. New in­vestors par­tic­i­pat­ing in this fi­nanc­ing in­clude Box­er Cap­i­tal of Tavi­s­tock Group, HBM Health­care In­vest­ments, BVF Part­ners, Sur­vey­or Cap­i­tal, Sam­sara Bio­Cap­i­tal, Ar­row­Mark Part­ners, Glax­o­SmithK­line, and Lau­ri­on Cap­i­tal Man­age­ment, as well as “sev­er­al oth­er long-term in­sti­tu­tion­al in­vestors.” All of the com­pa­ny’s ex­ist­ing in­vestors – Or­biMed, Bain Cap­i­tal, Pfiz­er, via Pfiz­er Ven­tures, and LifeArc – al­so par­tic­i­pat­ed.

It’s cu­ri­ous to see GSK in­clud­ed here as a di­rect in­vestor. In re­cent months its cor­po­rate ven­ture arm SR One has been ne­go­ti­at­ing to spin out from un­der the phar­ma gi­ant, which is more in­ter­est­ed in us­ing all of its cash in di­rect line with the R&D game plan set by Hal Bar­ron.

Is­lam said he could of­fer on­ly lim­it­ed in­sight in­to GSK’s cor­po­rate in­volve­ment, but added that “they could be a fab­u­lous part­ner for a cou­ple of things down the road.”

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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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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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 with any issues.

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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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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.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.