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