Pfiz­er spins off drug or­phans in­to a PhI­II-ready start­up backed by Bain to the tune of $103M

Like a lot of Big Phar­ma com­pa­nies with a big pipeline, Pfiz­er can’t de­vel­op all the drugs it has. So what do you do with the good drugs that still can’t make the cut?

In Pfiz­er’s case, you spin a line­up of your best, fur­thest ad­vanced ex­per­i­men­tal meds in­to a new com­pa­ny, while look­ing to add more when the time is ripe.

Lara Sul­li­van, an R&D strat­e­gy ex­ec, has gained the com­pa­ny’s sup­port to split off from the phar­ma gi­ant with four of its clin­i­cal-stage or­phans, cre­at­ing a new com­pa­ny called Spring­Works Ther­a­peu­tics. And with con­sid­er­able help from two big Bain funds, they’re start­ing out with a mega-round of $103 mil­lion for the Se­ries A.

Lara Sul­li­van

Sul­li­van, the new­ly un­veiled pres­i­dent of Spring­Works, says she gained con­sid­er­able sup­port for this new ven­ture from Pfiz­er’s chief med­ical of­fi­cer Fre­da Lewis-Hall, who is tak­ing a board spot on Spring­Works to help over­see the fu­ture of these drugs, with Pfiz­er lend­ing its fi­nan­cial sup­port along­side Bain Cap­i­tal Life Sci­ences, Bain Cap­i­tal Dou­ble Im­pact, Or­bimed and LifeArc.

It’s no easy task grab­bing four drugs out of the pipeline at a glob­al op­er­a­tion like Pfiz­er, even if they haven’t made the cut on R&D fund­ing.

“I’ve got a cou­ple of more gray hairs than I did a few years ago,” Sul­li­van tells me, when she got start­ed pur­su­ing this project. Lewis-Hall helped cham­pi­on the ef­fort, and then they built sup­port among the com­pa­ny’s lawyers, ac­coun­tants and sci­en­tists, who are on­ly too aware that even in a top 10 R&D out­fit like Pfiz­er  there are far more de­vel­op­ment projects than mon­ey to fund the work.

Sul­li­van tells me they’re still grow­ing the staff, but the top po­si­tions are oc­cu­pied by some high-pro­file fig­ures in the in­dus­try — from Chair­man Dan Lynch, to Bain Cap­i­tal Dou­ble Im­pact’s De­val Patrick, the for­mer gov­er­nor of Mass­a­chu­setts, on the board. Stephen Squin­to, a co-founder and for­mer R&D chief of Alex­ion — now a ven­ture part­ner at Or­biMed — is step­ping in as act­ing head of re­search at the up­start. Jeff Schwartz, a gen­er­al part­ner at Bain’s re­cent­ly cre­at­ed life sci­ences group along­side Adam Kop­pel, is al­so tak­ing a spot on the board.

Spring­Works is a di­rect out­growth of an in­creas­ing­ly com­mon strat­e­gy that is find­ing an abun­dance of deep-pock­et play­ers — in­clud­ing some ma­jor out­fits that are new to the game — who are ready to gam­ble hun­dreds of mil­lions of dol­lars on drug R&D.

Just like Vivek Ra­maswamy, who’s built a grow­ing biotech en­ter­prise with mul­ti­ple ten­ta­cles around Roivant Sci­ences with the castoffs to be found in Big Phar­ma, Whar­ton grad Sul­li­van found a way to jump­start a ma­ture biotech with a pipeline of ad­vanced as­sets with hu­man da­ta. And with a slate of new CEOs tak­ing over ma­jor R&D shops, shift­ing fo­cus and re­al­lo­cat­ing funds, this is one trend that seems to be gain­ing mo­men­tum.

It doesn’t stop here, ei­ther, for Spring­Works. Sul­li­van and her team are work­ing with Pfiz­er and oth­ers in the in­dus­try and acad­e­mia to find more clin­i­cal-stage drugs they can add to the pipeline to grow the com­pa­ny even fur­ther.

Spring­Works has plen­ty on its plate to get start­ed. Pfiz­er, like most of the ma­jor phar­ma com­pa­nies, tends to stick to its late-stage pipeline when they talk up R&D, so these drugs have been fly­ing large­ly un­der the radar.

  • The lead drug is nirogace­s­tat (PF-03084014), a gam­ma-sec­re­tase in­hibitor which will be moved in­to Phase III now for rare cas­es of desmoid tu­mors, a slow-grow­ing non-metasta­t­ic tu­mor of con­nec­tive tis­sue cells, which can cause se­vere mor­bid­i­ty, pain and loss of func­tion.
  • There’s a MEK 1/2 in­hibitor, PF-0325901, for the ge­net­ic dis­or­der NF1, one of three ge­net­ic con­di­tions known as neu­rofi­bro­mato­sis. It’s been through Phase I/II and will now head in­to a piv­otal tri­al.
  • An­oth­er rare con­di­tion, hered­i­tary xe­ro­cy­to­sis — char­ac­ter­ized by de­hy­drat­ed red blood cells — will be tar­get­ed by sen­i­capoc (PF-05416266).
  • An FAAH in­hibitor (PF-0445784) is be­ing de­vel­oped for post-trau­mat­ic stress dis­or­der.

To get in­to the Spring­Works pipeline, the drugs had to pass two fa­mil­iar chal­lenges in the biotech world: Was it tar­get­ing a dis­ease where there are no good drugs to choose from? And was there enough hard sci­ence to back up their po­ten­tial in mak­ing the grade with piv­otal da­ta?

Based on their work at Pfiz­er so far, there are plen­ty more like this out there.

“We an­tic­i­pate there will be plen­ty of more com­pounds,” says Sul­li­van.

At this stage, there’s no telling ex­act­ly how long the $103 mil­lion will last, though they ex­pect at a min­i­mum to get through the late-stage work on mid-stage proof-of-con­cept stud­ies on the 4. The com­pa­ny has a set of big names be­hind it, but the staff is still un­der 10, though grow­ing fast.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

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US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Ivan Cheung, Eisai US chairman and CEO

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One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

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Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

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Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

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The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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Marcelo Bigal, Ventus Therapeutics CEO

No­vo Nordisk joins No­var­tis, Roche in NL­RP3 are­na, bet­ting $70M cash on NASH, car­diometa­bol­ic us­es

As a drug target, the NLRP3 inflammasome has drawn serious interest from Big Pharma, inspiring a series of M&A deals from Novartis and Roche on top of venture investments by others. Now Novo Nordisk is jumping on the bandwagon — and the Danish pharma giant is taking the target where it knows best.

Novo Nordisk is getting its NLRP3 inhibitors from Ventus Therapeutics, a Versant-backed startup that set out to make some of the best NLRP3 drugs out there by incorporating new insights into the structure of the target complex.

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