Saqib Islam, SpringWorks CEO

Pfiz­er spin­out Spring­Works will ship its first drug to the FDA be­fore year’s end with PhI­II win

Spring­Works Ther­a­peu­tics thinks it has ce­ment­ed the back­bone for its first “pipeline-in-a-prod­uct” on­col­o­gy treat­ment and will send it to the FDA be­fore the clock strikes 2023 with a Phase III win on Tues­day.

The oral gam­ma sec­re­tase in­hibitor, dubbed nirogace­s­tat, beat place­bo on the pri­ma­ry goal of pro­gres­sion-free sur­vival in adults with pro­gress­ing desmoid tu­mors.

The soft-tis­sue tu­mors can lead to long-last­ing pain, dis­fig­ure­ment and am­pu­ta­tion, and there are cur­rent­ly no ap­proved meds for the rare on­col­o­gy in­di­ca­tion. The tu­mors typ­i­cal­ly im­pact pa­tients aged 20 to 44 years old and dis­pro­por­tion­ate­ly af­fect women at rates 2 to 3 times high­er, with up to a to­tal of 1,650 new cas­es di­ag­nosed in the US an­nu­al­ly, ac­cord­ing to Spring­Works.

The news sent the Stam­ford, CT biotech’s stock $SWTX soar­ing 18% at one point be­fore the open­ing bell Tues­day. In the in­vestor call and pre­sen­ta­tion, CEO Saqib Is­lam not­ed patent pro­tec­tion on the drug lasts un­til 2039.

In the 142-pa­tient De­Fi tri­al, the treat­ment showed a 71% re­duc­tion in the risk of dis­ease pro­gres­sion, the biotech said. The drug hit sec­ondary goals of sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments over place­bo in ob­jec­tive re­sponse rate and pa­tient-re­port­ed out­comes, ac­cord­ing to Spring­Works. The ran­dom­ized, dou­ble-blind study took place at sites in North Amer­i­ca and Eu­rope.

Spring­Works said the drug, which was giv­en twice dai­ly, was “gen­er­al­ly well tol­er­at­ed with a man­age­able safe­ty pro­file.”

But mul­ti­ple ques­tions sur­round­ing re­pro­duc­tive ad­verse events went large­ly unan­swered dur­ing the com­pa­ny’s in­vestor call.

“The ma­jor­i­ty of women of child­bear­ing po­ten­tial had ad­verse events con­sis­tent with ovar­i­an dys­func­tion,” the com­pa­ny said in its press re­lease.

On the in­vestor call, Is­lam was asked mul­ti­ple times about this safe­ty point, and he re­peat­ed­ly said it was a “class ef­fect as re­lat­ed to ovar­i­an dys­func­tion” and not­ed a few times that more spe­cif­ic da­ta will be re­leased at a med­ical con­fer­ence lat­er this year. Ex­ec­u­tives de­clined to dis­close what per­cent­age of the tri­al en­rollees were women.

Asked if it was “fair to as­sume you haven’t reached a me­di­an pro­gres­sion free sur­vival in the treat­ment arm,” the CEO re­spond­ed by say­ing it is “pre­ma­ture to reach that con­clu­sion.” The drug did not meet that mea­sure in ei­ther the Phase I or Phase II stud­ies.

The drug is in near­ly a dozen clin­i­cal tri­als, flush with mul­ti­ple com­bo ther­a­py stud­ies test­ing nirogace­s­tat along­side BC­MA drugs from the who’s who of Big Phar­ma and biotech alike: GSK, John­son & John­son, Pfiz­er, Ab­b­Vie, Re­gen­eron, Seagen, Pre­ci­sion Bio­Sciences and Al­lo­gene Ther­a­peu­tics. All those stud­ies are in re­lapsed/re­frac­to­ry mul­ti­ple myelo­ma. Spring­Works is teamed up with Chil­dren’s On­col­o­gy Group for a mid-stage pe­di­atric desmoid tu­mor monother­a­py study.

Nirogace­s­tat comes from the cof­fers of Pfiz­er. The Big Phar­ma spun out the drug and oth­er as­sets in­to Spring­Works in 2017. The biotech hit the ground run­ning at the time with an ini­tial $103 mil­lion from in­vestors like Bain. Is­lam, who joined the lead­er­ship team at the be­gin­ning, was pre­vi­ous­ly Mod­er­na’s CBO.

Spring­Works lead­er­ship re­it­er­at­ed on the call that it had more than $380 mil­lion to spend at the end of last quar­ter, which gives run­way “com­fort­ably” in­to 2024 for its 18 R&D pro­grams.

Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)

FDA side­lines Paul Hud­son's $3.7B MS drug af­ter es­tab­lish­ing link to liv­er dam­age

One of Sanofi CEO Paul Hudson’s top picks in the pipeline — picked up in a $3.7 billion buyout 2 years ago — has just been sidelined in the US by a safety issue.

The pharma giant put out word early Thursday that the FDA has put their Phase III studies of tolebrutinib in multiple sclerosis and myasthenia gravis on partial clinical hold, halting enrollment and suspending dosing for patients who have been on the drug for less than 60 days. Patients who have completed at least 60 days of treatment can continue therapy as researchers explore a “limited” — but unspecified in Sanofi’s statement — number of cases of liver injury.

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Phar­ma re­acts to post-Roe; Drug­mak­ers beef up cy­ber de­fense; Boehringer, Roche qui­et­ly axe drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As a reminder, we are off on Monday for the Fourth of July. I hope this recap will kick off your (long) weekend well and that the rest of it will be just what you need. See you next week for a shortened edition!

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Eric Hughes, incoming Teva EVP of global R&D and CMO

Te­va chief raids Ver­tex for his new glob­al head of re­search and de­vel­op­ment

Teva CEO Kåre Schultz has found his new R&D chief and CMO in Vertex’s ranks.

The global generics giant, which has some 3,500 staffers in the R&D group, has named Eric Hughes to the top research spot in the company. He’ll be replacing Hafrun Fridriksdottir, who held the role for close to five years, on Aug. 1.

Hughes hasn’t been at Vertex for long, though. He jumped from Novartis less than a year ago, after heading the immunology, hepatology & dermatology global development unit. Before that, he completed a five-year stint as head of early clinical research for the specialty discovery medicine department in the exploratory clinical & translational research group at Bristol Myers Squibb, according to his LinkedIn profile.

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#BIO22: Man­ag­ing a biotech in tur­bu­lent times. 'There's a per­fect shit­show out there'

On Tuesday, June 14, Endpoints News EIC John Carroll sat down with a group of biotech execs to discuss the bear market for industry stocks and how they were dealing with it. Here’s the conversation, which has been lightly edited for brevity.

Martin Meeson, sponsor opening:

Thank you, John. Hello everyone. My name’s Martin Meeson, I’m the CEO of Fujifilm Diosynth. For those of you who don’t know Fujifilm Diosynth, we operate in the development of clinical and commercial product scale up, we have facilities in Europe and the US, and around about 4,000 employees. We run on average about 150 programs, so when it comes to managing in turbulent times over the last two years, we’ve had quite a lot of experience of that. Not just keeping the clinical pipelines and the commercial pipelines open, but also our response to the pandemic and the molecules that we’ve had within there. One of the phrases that I coined probably about a year ago when we were talking at JP Morgan, was I talked about managing through turbulent times. Well, it’s become the fact that we are not managing and leading through these times, we are managing in them, which is why that’s really the purpose of and the topic that we’ve got today.

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Susan Galbraith, AstraZeneca EVP, oncology R&D

Catch­ing up with Bris­tol My­ers and Mer­ck, As­traZeneca de­clares neoad­ju­vant win for PD-L1/chemo com­bo

When AstraZeneca started the Phase III AEGEAN trial for Imfinzi in 2018, it was, alongside several Big Pharma brethren, hoping to push the use of PD-(L)1 therapies into earlier lines of treatment. Three and a half years later, the British drugmaker has nabbed promising data in a type of lung cancer.

Topline results from an interim analysis showed that adding Imfinzi to chemotherapy before surgery spurred a “statistically significant and meaningful” improvement in pathologic complete response for patients with resectable non-small cell lung cancer compared to chemotherapy alone.

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Nassim Usman, Catalyst Biosciences CEO

Fac­ing set­backs for months and an ac­tivist at­tack, Cat­a­lyst Bio­sciences pre­pares to call it quits

After downsizing for several months, Catalyst Biosciences is getting ready to tap out.

The San Francisco biotech announced Wednesday that it would be liquidating and distributing cash back to shareholders, with total proceeds expected to reach $65 million. Catalyst intends to return the money “as soon as practicable,” the company said, as it has ceased all R&D activities, CEO Nassim Usman said in a statement.

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Amgen's taking social media followers around the globe as it introduces the many different

From Tam­pa to Mu­nich, Am­gen’s ‘Places’ cam­paign in­tro­duces its lo­ca­tions around the world

Amgen is taking social media followers around the world with its latest corporate campaign. Called “Places of Amgen,” the twice monthly posts highlight the biopharma’s different offices and sites – and the people who work there.

Each post runs on LinkedIn, Facebook and Instagram with details about the work Amgen does in that location, when it was established, comments from people who work there and other interesting facts. The most recent one about Paris, France, for example, notes that Amgen France last year signed a French association charter committed to the inclusion of LBGT+ people in the workplace.

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On Friday, Lonza announced plans to construct a large-scale commercial drug product fill and finish facility in the town of Stein, Switzerland.

Lon­za to in­vest $500M+ on fill-fin­ish fa­cil­i­ty on its home turf

Lonza has been expanding its reach across the globe, bringing sites in China and the US online this year, but now they are looking closer to home for their next major investment.

The Swiss manufacturer on Friday announced plans to construct a large-scale commercial drug fill and finish facility in the town of Stein, Switzerland. The new facility will be delivered through an investment of approximately CHF 500 million, or $519 million, and is expected to be completed in 2026. The facility will also be constructed on the same campus as Lonza’s current clinical drug product facility.

Credit: Shutterstock

Bio­haven takes mi­graine cam­paign to pa­tients' Twit­ter feeds, months ahead of Pfiz­er takeover

Two weeks ago, Biohaven hit an all-time high in weekly Nurtec prescriptions. CEO Vlad Coric attributes at least some of that success to a new interactive Twitter campaign that encourages patients to free their feed of potential migraine triggers.

Earlier this month, Biohaven in partnership with Twitter launched the #RelieveYourFeed campaign that allows users to customize their app settings based on their migraine triggers.