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.”

Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

UP­DAT­ED: In a land­mark first glimpse of hu­man da­ta from Ver­tex, CRISPR/Cas9 gene ther­a­py sig­nals ear­ly ben­e­fit

Preliminary data on two patients with blood disorders that have been administered with Vertex and partner CRISPR Therapeutics’ gene-editing therapy suggest the technology is safe and effective, marking the first instance of the benefit of the use of CRISPR/Cas9 technology in humans suffering from disease.

Patients in these phase I/II studies give up peripheral blood from which hematopoietic stem and progenitor cells are isolated. The cells are tinkered with using CRISPR/Cas9 technology, and the edited cells — CTX001 — are infused back into the patient via a stem cell transplant. The objective of CTX001 is to fix the errant hemoglobin gene in patents with two blood disorders: beta-thalassemia and sickle cell disease, by unleashing the production of fetal hemoglobin.

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UP­DAT­ED: Make that 2 ap­proved RNAi drugs at Al­ny­lam af­ter the FDA of­fers a speedy OK on ul­tra-rare dis­ease drug

Seventeen years into the game, Alnylam’s pivot into commercial operations is picking up speed.
The bellwether biotech $ALNY has nabbed their second FDA OK for an RNAi drug, this time for givosiran, the only therapy now approved for acute hepatic porphyria. This second approval came months ahead of the February deadline — even after winning priority review following their ‘breakthrough’ title earlier.
AHP is an extremely rare disease, with some 3,000 patients in Europe and the US, not all diagnosed, and analysts have projected peak revenue of $600 million to $700 million a year. The drug will be sold as Givlaari.

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David Ricks. Eli Lilly

Eli Lil­ly touts $400M man­u­fac­tur­ing ex­pan­sion, 100 new jobs to much fan­fare in In­di­anapo­lis — even though it's been chop­ping staff

Eli Lilly is pouring in $400 million to beef up manufacturing facilities at its home base of Indianapolis. The investment, which was lauded by the city’s mayor, is expected to create 100 new jobs.

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Am­gen chops 172 more staffers in R&D, op­er­a­tions and sales amid neu­ro­science ex­it, rev­enue down­turn

Neuroscience wasn’t the only unit that’s being hit by a reorganization underway at Amgen. As well as axing 149 employees in its Cambridge office, the company has disclosed that 172 others nationwide, including some from its Thousand Oaks, CA headquarters, are being let go.

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Stephen Hahn (via Senate HELP Committee)

Stephen Hahn gets through Sen­ate’s soft­ball job in­ter­view — but most­ly plays dodge­ball on the is­sues fac­ing the FDA

Anyone looking for fresh insights on what kind of FDA commissioner Stephen Hahn will be got precious few clues during Wednesday’s Senate hearing on the nomination.

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Op­di­vo/Yer­voy com­bo for melanoma fails in key pa­tient pop­u­la­tion

Bristol-Myers Squibb’s efforts to expand their checkpoint inhibitor combination have run into another recalcitrant cancer.

The NJ-based pharma announced that a combination of Yervoy and Opdivo didn’t beat out Opdivo alone in patients with resected high-risk melanoma who had very low levels of PD-L1. The drug combo couldn’t improve recurrence-free survival in these post-surgery patients.

Ver­tex's stel­lar quar­ter car­ries on with French re­im­burse­ment deal

Vertex’s golden quarter just got brighter. About a month after the US drugmaker finally clinched a deal with UK authorities to cover its slate of cystic fibrosis (CF) drugs following years of protracted negotiations, the company on Wednesday secured a deal with France for its CF therapy, Orkambi.

After the UK, France has one of the largest CF populations outside the United States. Achieving French reimbursement unlocks an ~7000-patient CF population, around ~2500-3000 of which will likely be eligible to receive (and be reimbursed for) Orkambi, Stifel’s Paul Matteis wrote in a note.

Nello Mainolfi, Kymera via Youtube

Kymera hands the helm to No­var­tis vet — and found­ing CSO — Nel­lo Main­olfi

Kymera Therapeutics is turning to a co-founder to run the company.
The protein degradation specialist with a deep-pocket syndicate behind them has opted to give the helm officially to Nello Mainolfi. The new CEO is a veteran of the Novartis Institutes for Biomedical Research. He joined Atlas Venture in their entrepreneur-in-residence program and helped launch Kymera as the CSO three years ago with Atlas’ Bruce Booth.
The boast at Kymera is that they’re angling to create a new class of protein degraders, a popular field where there’s been a variety of startups. One of its chief advocates is NIBR head Jay Bradner, who launched C4 just ahead of joining Novartis, where he’s also been doing new work in the field.