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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Re­searchers move clos­er to de­ci­pher­ing blood clots from As­traZeneca, J&J's Covid-19 vac­cines

Researchers may be nearing an answer for the mysterious and life-threatening blood clots that appeared on very rare occasions in people who received the J&J or AstraZeneca Covid-19 vaccine.

The new work builds on an early hypothesis researchers in Norway put forward last spring, when the cases first cropped up. They proposed the events were similar to blood clots that can occur in a small subset of patients who receive heparin, one of the most commonly used blood thinners.