Sol-Gel's rosacea drug clears late-stage hur­dle; uniQure's he­mo­phil­ia gene ther­a­py im­press­es with PhII da­ta

Alon Seri-Levy Sol-Gel

→ Is­rael’s Sol-Gel Tech­nolo­gies‘ mi­croen­cap­su­la­tion plat­form — de­signed to en­trap ac­tive in­gre­di­ents in porous sil­i­ca mi­cro­cap­sules to ad­dress the lim­i­ta­tions of top­i­cal drug de­liv­ery by sta­bi­liz­ing the in­gre­di­ents, ex­tend­ing drug de­liv­ery time and re­duc­ing po­ten­tial ir­ri­ta­tion — was val­i­dat­ed when its papu­lo­pus­tu­lar rosacea prod­uct made of mi­croen­cap­su­lat­ed ben­zoyl per­ox­ide, Ep­so­lay, cleared twin late-stage stud­ies. “While we ex­pect­ed to see strong ef­fi­ca­cy and tol­er­a­bil­i­ty with Ep­so­lay, the rapid ef­fi­ca­cy was a stand­out in our Phase 3 stud­ies,” said Alon Seri-Levy, Sol-Gel’s chief, in a state­ment.

→ Months af­ter un­veil­ing it had start­ed dos­ing pa­tients with its he­mo­phil­ia B gene ther­a­py, AMT-061, in a late-stage study — uniQure $QURE on Fri­day un­veiled da­ta from three pa­tients treat­ed in its on­go­ing Phase IIb study. Fol­low up da­ta 36 weeks af­ter ther­a­peu­tic ad­min­is­tra­tion showed that each pa­tient ex­pe­ri­enced sus­tained in­creas­es in fac­tor IX (FIX) lev­els. “Mean FIX ac­tiv­i­ty for the three pa­tients at 36 weeks af­ter ad­min­is­tra­tion was 45% of nor­mal, with the first pa­tient achiev­ing FIX ac­tiv­i­ty of 54% of nor­mal, the sec­ond pa­tient achiev­ing FIX ac­tiv­i­ty of 30% of nor­mal and the third pa­tient achiev­ing FIX ac­tiv­i­ty of 51% of nor­mal,” the com­pa­ny dis­closed. “We re­main op­ti­mistic that uniQure has the best-in-class he­mo­phil­ia B gene ther­a­py prod­uct and will re­port pos­i­tive re­sults from the on­go­ing Phase 3 HOPE-B study (we have re­struc­tured our pay­ment mod­el and in­creased the PoS to 90% from 75%),” Can­tor Fitzger­ald an­a­lysts wrote in a note.

Mike Grey Linkedin

Mirum Phar­ma­ceu­ti­cals has set terms for its IPO $MIRM as it plots a quick flip to late-stage stud­ies for a cou­ple of as­sets bought back from Shire. With 5 mil­lion shares priced at $14 to $16 each, the biotech — led by Mike Grey and his for­mer crew at Lu­me­na — would raise $80 mil­lion in the best case sce­nario.

Third Rock-backed Ful­crum Ther­a­peu­tics has spelled out a price range be­tween $16 and $18 for its IPO, in which it plans to sell 4.5 mil­lion shares, reach­ing for $93 mil­lion at max­i­mum. The pro­ceeds will go to­ward its lead drug, losmapi­mod, an ex­per­i­men­tal drug for fa­cioscapu­lo­humer­al mus­cu­lar dy­s­tro­phy.

→ More he­mo­phil­ia da­ta flowed in on Mon­day. Cat­a­lyst Bio­sciences$CBIO sub­cu­ta­neous he­mo­phil­ia ther­a­py, marzep­tacog al­fa, for pro­phy­lax­is sig­nif­i­cant­ly re­duc­ing the an­nu­al­ized bleed rate (ABR) in pa­tients with he­mo­phil­ia A or B with in­hibitors, meet­ing the main goal in a mid-stage study.

→ South San Fran­cis­co-based RAPT Ther­a­peu­tics is gun­ning for a $86 mil­lion IPO. The ear­ly-stage com­pa­ny, which is de­vel­op­ing ther­a­pies for can­cer and in­flam­ma­to­ry dis­eases, is hop­ing to list on the Nas­daq un­der the sym­bol RAPT.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Lit­tle Leo Phar­ma steps to­ward the prize-fight ring with pos­i­tive PhI­II atopic der­mati­tis da­ta. Now they just have to beat Dupix­ent

A day after new Sanofi CEO Paul Hudson staked his reputation and the future of the pharma giant on making Dupixent a megasuccess story, little Leo Pharma is throwing down the gauntlet on atopic dermatitis.

Three years ago Leo paid AstraZeneca $115 million to buy up rights to use tralokinumab against atopic dermatitis — with $1 billion more on the table in milestones — the Danish company says their drug has swept up positive results for all primary and secondary endpoints in three Phase III trials. Now they plan to start the final push for regulatory approvals so they can challenge the heavyweight champions in this slugfest.

Otello Stampacchia. Omega Funds

Af­ter sev­er­al high pro­file start­up launch­es, om­niv­o­rous Omega Funds clos­es $438M fund to pur­sue more deals

Omega Funds likes to work backwards. It invests with the end game — denoted by the Greek letter in its name — in mind, and it keeps tabs on the number of marketed medical products that culminate from its ventures: 37 in 16 years.

So when founder and managing director Otello Stampacchia declares it’s the most exciting time to be investing in life sciences in a generation, it’s perhaps only natural that Omega has closed its largest fund to date. With $438 million in total commitments for Fund VI, the firm will continue injecting capital into a broad swath of companies across the US and Europe.

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Janet Woodcock (Credit: CQ Roll Call/AP)

For­eign drug in­spec­tions de­cline as FDA hir­ing strug­gles con­tin­ue

The US reliance on imported pharmaceuticals and ingredients is rising as foreign drug facility inspections decreased by about 10% from 2016 to 2018. Part of the reason for the decline: The US Food and Drug Administration (FDA) said it’s still struggling to hire new inspectors.

Testifying before a House subcommittee on Tuesday, Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, defended the agency’s approach and discussed some of the vulnerabilities of its foreign inspection program. She said FDA is looking to hire 50 new inspectors, but there are difficulties.

Paul Hudson, Sanofi

Paul Hud­son promis­es a bright new fu­ture at Sanofi, kick­ing loose me-too drugs and fo­cus­ing on land­mark ad­vances. But can he de­liv­er?

Paul Hudson was on a mission Tuesday morning as he stood up to address Sanofi’s new R&D and business strategy.

Still fresh into the job, the new CEO set out to convince his audience — including the legions of nervous staffers inevitably devoting much of their day to listening in — that the pharma giant is shedding the layers of bureaucracy that had held them back from making progress in the past, dropping the duds in the pipeline and reprioritizing a more narrow set of experimental drugs that were promised as first-in-class or best-in-class.  The company, he added, is now positioned to “go after other opportunities” that could offer a transformational approach to treating its core diseases.

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Left top to right: Mark Timney, Alex Denner, Vas Narasimhan. (The Medicines Company, Getty, AP/Endpoints News)

In a play-by-play of the $9.7B Med­Co buy­out, No­var­tis ad­mits it over­paid while of­fer­ing a huge wind­fall to ex­ecs

A month into his tenure at The Medicines Company, new CEO Mark Timney reached out to then-Novartis pharma chief Paul Hudson: Any interest in a partnership?

No, Hudson told him. Not now, at least.

Ten months later, Hudson had left to run Sanofi and Novartis CEO Vas Narasimhan was paying $9.7 billion for the one-drug biotech – the largest in the string of acquisitions Narasimhan has signed since his 2017 appointment.

The deal was the product of an activist investor and his controversial partner working through nearly a year of cat-and-mouse negotiations to secure a deal with Big Pharma’s most expansionist executive. It represented a huge bet in a cardiovascular field that already saw two major busts in recent years and brought massive returns for two of the industry’s most eye-raising names.

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Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

This is the second biotech buyout pact today, marking a brisk tempo of M&A deals in the lead-up to the big JP Morgan gathering in mid-January. It’s no surprise the acquisitions are both for cancer drugs, where Sanofi will try to make its mark while Merck beefs up a stellar oncology franchise. And bolt-ons are all the rage at the major pharma players, which you could also see in Novartis’ recent $9.7 billion MedCo buyout.

ArQule — which comes out on top after their original lead drug foundered in Phase III — highlighted early data on ‘531 at EHA from a group of 6 chronic lymphocytic leukemia patients who got the 65 mg dose. Four of them experienced a partial response — a big advance for a company that failed with earlier attempts.

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Am­gen puts its foot down in shiny new South San Fran­cis­co hub as it re­or­ga­nizes R&D ops

Amgen has signed up to be AbbVie’s neighbor in South San Francisco as it moves into a nine-story R&D facility in the booming biotech hub.

The arrangement gives Amgen 240,000 square feet of space on the Gateway of Pacific Campus, just a few minutes drive from its current digs at Oyster Point. The new hub will open in 2022 and house the big biotech’s Bay Area employees working on cardiometabolic, inflammation and oncology research.