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.

A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Su­per-se­cre­tive an­ti-ag­ing biotech Cal­i­co tees up the first vis­i­ble clin­i­cal tri­al of an ex­per­i­men­tal drug. And it’s for can­cer?

Over the past 7 years, Calico has been so much more than your average, run-of-the-mill secretive biotech players. It’s a riddle, wrapped in a mystery, inside an enigma, to repurpose an old Winston Churchill line dating from the time he confronted the Iron Curtain surrounding Stalin’s thoughts.

Launched by industry legend Art Levinson of Genentech fame, with the infinitely deep pockets of Google for support, one of the few big headlines the anti-aging biotech has sparked focused on a major alliance with AbbVie — a giant outfit that conversely likes to show off its drug prospects whenever it can. Together, they’ve been focused on diseases that limit life span — quite an arc of ailments.

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RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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Af­ter near­ly a year of de­bate, the Covid-19 vac­cine chal­lenge tri­als are of­fi­cial­ly com­ing

After nearly a year of public advocacy and often rancorous ethical debate, human challenge trials for Covid-19 vaccines are getting off the ground in London.

The UK government’s Covid-19 Vaccine Taskforce and the contract research firm Open Orphan announced today £10 million ($13 million) plan to test experimental Covid-19 vaccines in volunteers whintentionally exposed to the novel coronavirus. The studies, which won’t launch until early 2021, come after 9 months of debate over whether such studies were safe and would actually hasten vaccine development, and they follow a long history of researchers using challenge models to study other respiratory viruses, including flu and the coronaviruses that cause the common cold.

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Derek Chalmers, Cara Ther

Cara lines up a $440M deal for US rights to its late-stage drug for se­vere itch, with $150M cash on the ta­ble

With plans afoot to file an NDA for what could be its first approved drug, Cara Therapeutics is pivoting its focus to commercialization. And Swiss company Vifor Pharma is willing to surrender up to $440 million to market the candidate in the US.

Cara $CARA CEO Derek Chalmers said an NDA submission is coming this quarter for their intravenous drug Korsuva in chronic kidney disease-associated pruritus (CKD-aP), a severe itching condition. The Stamford, CT-based biotech read out positive topline data from a Phase III pivotal study back in April, and announced plans to approach EMA regulators shortly after filing with the FDA.

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News brief­ing: Ab­b­Vie and Roche's Ven­clex­ta scores an­oth­er FDA OK; Im­muno­Gen nabs Chi­na deal with $40M cash

AbbVie and Roche’s Venclexta has gotten a new FDA thumbs up.

The pair announced Monday that regulators have approved the drug in combination with azacitidine or low-dose cytarabine for newly-diagnosed acute myeloid leukemia in adults who are 75 or older or those who can’t undergo intensive chemotherapy. This follows the drug’s accelerated approval in 2018 and positive data from two Phase III confirmatory trials.

Covid-19 roundup: Pars­ing Bourla, a top an­a­lyst sees im­proved chances for Pfiz­er vac­cine; Fau­ci: No sur­prise that Trump was hit by Covid-19

With a medley of adverse events hobbling the late-stage development of vaccines and drugs, Pfizer CEO Albert Bourla’s latest — extended — timeline for the mRNA approach they’re working on with BioNTech is giving some top analysts added confidence that the pharma giant can come up with the regulatory goods next month.

Parsing Bourla’s language in his comments last week, SVB Leerink’s Geoffrey Porges notes that Bourla’s decision to say they “may” be able to nail down the positive efficacy of their vaccine in a matter of days — a big change from his earlier certainty — may also indicate a delay on that to early November.

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Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Gilead feels the heat as close al­ly Gala­pa­gos re­ports a big set­back on one of their top ex­per­i­men­tal drugs

The bad news keeps stacking up at Galapagos — which quite likely just lost control of a billion-dollar deal — and by extension their close partners at Gilead.

The biotech $GLPG reported after the bell Thursday that GLPG1972, one of their top development programs, flat failed a mid-stage study for osteoarthritis, flunking the primary and all secondary endpoints.

Testing 3 different doses of their drug, which relies on ADAMTS-5 inhibition, investigators concluded that none of them triggered a statistically significant response — as measured by cartilage thickness.

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IN8bio CEO William Ho (IN8bio)

Bring­ing their ge­net­i­cal­ly mod­i­fied gam­ma delta T cells to Nas­daq, IN8bio files for $86M IPO

The biotech IPO parade continues marching forward as 2020 turns toward the fourth quarter.

IN8bio, a New York-based company focused on genetically modified gamma delta T cell therapies, filed to go public Friday seeking an $86 million raise. The company has two clinical-stage candidates being studied in glioblastoma and leukemia, respectively.

By any stretch of the imagination, 2020 has already been a huge year for biotech, and nowhere does it appear more obvious than the vast amounts of companies hitting the public market.