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.

What Will it Take to Re­al­ize the Promise and Po­ten­tial of Im­mune Cell Ther­a­pies?

What does it take to get to the finish line with a new cancer therapy – fast? With approvals in place and hundreds of immune cell therapy candidates in the pipeline, the global industry is poised to create a fundamental shift in cancer treatments towards precision medicine. At the same time, unique challenges associated with cell and process complexity present manufacturing bottlenecks that delay speed to market and heighten cost of goods sold (COGS) — these hurdles must be overcome to make precision treatments an option for every cancer patient. This series of articles highlights some of the key manufacturing challenges associated with the production of cell-based cancer therapies as well as the solutions needed to transcend them. Automation, process knowledge, scalability, and assured supply of high-quality starting material and reagents are all critical to realizing the full potential of CAR-based therapies and sustaining the momentum achieved in recent years. The articles will highlight leading-edge technologies that incorporate these features to integrate across workflows, accelerate timelines and reduce COGS – along with how these approaches are enabling the biopharmaceutical industry to cross the finish line faster with new treatment options for patients in need.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Lat­est news: It’s a no on uni­ver­sal boost­ers; Pa­tient death stuns gene ther­a­py field; In­side Tril­li­um’s $2.3B turn­around; and more

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President Biden and Pfizer CEO Albert Bourla (Patrick Semansky/AP Images)

Chaot­ic ad­comm sees Pfiz­er/BioN­Tech boost­ers re­ject­ed for gen­er­al pop­u­la­tion, but rec­om­mend­ed for old­er and high-risk pop­u­la­tions

With just days before President Joe Biden’s Covid-19 booster rollout is set to go into effect, an FDA advisory committee appeared on the verge of not recommending boosters for anyone in the US before a last-minute change of wording laid the groundwork for older adults to have access to a third dose.

The FDA’s adcomm on Vaccines and Related Biological Products (VRBPAC) roundly rejected Pfizer/BioNTech booster shots for all individuals older than 16 by a 16-2 vote Friday afternoon. Soon after, however, the agency posed committee members a new question limiting booster use to the 65-and-older population and individuals at high risk of disease due to occupational exposure or comorbidities.

As­traZeneca, Dai­ichi Sanky­o's ADC En­her­tu blows away Roche's Kad­cy­la in sec­ond-line ad­vanced breast can­cer

AstraZeneca and Japanese drugmaker Daiichi Sankyo think they’ve struck gold with their next-gen ADC drug Enhertu, which has shown some striking data in late-stage breast cancer trials and early solid tumor tests. Getting into earlier patients is now the goal, starting with Enhertu’s complete walkover of a Roche drug in second-line breast cancer revealed Saturday.

Enhertu cut the risk of disease progression or death by a whopping 72% (p=<0.0001) compared with Roche’s ADC Kadcyla in second-line unresectable and/or metastatic HER2-positive breast cancer patients who had previously undergone treatment with a Herceptin-chemo combo, according to interim data from the Phase III DESTINY-Breast03 head-to-head study presented at this weekend’s #ESMO21.

Merck Research Laboratories CMO Roy Baynes

Mer­ck­'s Keytru­da un­corks full da­ta on lat­est ad­ju­vant win — this time in melanoma — adding bricks to ear­ly can­cer wall

In recent months, the battle for PD-(L)1 dominance has spilled over into early cancer with Merck’s Keytruda and Bristol Myers Squibb’s Opdivo all alone on the front lines. Keytruda now has another shell in its bandolier, and it could spell a quick approval.

Keytruda cut the risk of relapse or death by 35% over placebo (p=0.00658) in high-risk, stage 2 melanoma patients who had previously undergone surgery to remove their tumors, according to full data from the Phase III KEYNOTE-716 presented Saturday at #ESMO21.

Mer­ck flesh­es out Keytru­da win in first-line cer­vi­cal can­cer, adding more fire­pow­er to its ear­ly can­cer push

Merck has worked hard to bring its I/O blockbuster Keytruda into earlier and earlier lines of therapy, and now the wonder drug appears poised to make a quick entry into early advanced cervical cancer.

A combination of Keytruda and chemotherapy with or without Roche’s Avastin cut the risk of death by 33% over chemo with or without Avastin (p=<0.001) in first-line patients with persistent, recurrent or metastatic cervical cancer, according to full data from the Phase III KEYNOTE-826 study presented Saturday at #ESMO21.

Back-to-back piv­otal fail­ures force Ther­a­vance to lay off 270 staffers, prune R&D fo­cus

If it all went well, Q3 was supposed to be harvest time for Theravance.

Both of its lead drugs — the pan-JAK inhibitor izencitinib and blood pressure drug ampreloxetine — were slated for crucial readouts. The biotech was, as SVB Leerink analyst Geoffrey Porges put it, “entering the most important period of validation events in its history.”

Instead, izencitinib flopped a key Phase IIb trial in ulcerative colitis, putting the J&J partnership around it in jeopardy. A month later, Theravance is reporting that the Phase III trial testing ampreloxetine in symptomatic neurogenic orthostatic hypotension is also a failure, imploding the company’s entire pipeline and forcing a rethink on R&D strategy.

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Skin tu­mors in mice force Pro­tag­o­nist to halt lead pro­gram, crush­ing stock

Protagonist Therapeutics just can’t catch a break.

Six months after the Newark, CA-based biotech unveiled grand plans to launch its lead candidate for blood disorders into a Phase III trial, the FDA has slapped the program with a clinical hold. The halt — which applies to all trials involving the candidate, rusfertide — comes after skin tumors were discovered in mice treated with the drug, according to Protagonist.

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