Takeda (NYSE: TAK) confirmed Sunday August 30, 2026 that commercial launch of Mimrylo (rusfertide, the first-in-class subcutaneous hepcidin mimetic peptide approved Friday August 28 for erythrocytosis in adults with polycythemia vera) is underway across the U.S., with the product available at specialty pharmacies within 48 hours of FDA approval. The commercial launch approach reflects Takeda's rare-blood-cancer specialty commercial infrastructure built up around Iclusig (ponatinib) for chronic myeloid leukemia and the recent Fruzaqla (fruquintinib) launch in colorectal cancer. Jefferies analyst projections of $2 billion peak sales assume rapid coverage decisions from major U.S. payers by end of 2026 plus European approval on the same clinical dataset in H1 2027. The Mimrylo launch establishes the hepcidin mimetic peptide category and sets a commercial template that Disc Medicine's DISC-0974 (an anti-hemojuvelin antibody targeting hepcidin from a different angle) and Silence Therapeutics' SLN124 (siRNA targeting TMPRSS6, an upstream hepcidin regulator) will need to differentiate against.
Silence Therapeutics (NASDAQ: SLN) announced the closing of its upsized $201.3 million underwritten public offering of American Depositary Shares. Terms: priced August 11 at $13.50 per ADS for 12,962,963 ADSs. The underwriters (Jefferies, Morgan Stanley, Cantor Fitzgerald, and William Blair as joint book-running managers) fully exercised their 30-day option to purchase an additional 1,944,444 ADSs, bringing the total offering to 14,907,407 ADSs and gross proceeds to approximately $201.3 million. The financing follows the August 10 Phase 2 SANRECO trial win in polycythemia vera with divesiran (first-in-class TMPRSS6-targeting siRNA that increases hepcidin production to restrict iron availability to bone marrow) achieving an 88% response rate versus 19% on placebo (P<0.0001, 69% placebo-adjusted response rate) in 48 phlebotomy-dependent patients over 36 weeks. Use of proceeds: advance divesiran into Phase 3 (planned initiation H1 2027 evaluating Q12W dosing versus placebo) plus broader pipeline progression including hepatic-target GalNAc-conjugated siRNA portfolio across TMPRSS6, complement component 3 (SLN-124/SLN-501), and other liver-targeted RNAi candidates. Divesiran has FDA Fast Track and Orphan Drug designations for polycythemia vera.
Silence Therapeutics (NASDAQ: SLN) closed its upsized $175 million underwritten public offering of American Depositary Shares (ADSs) following the August 10, 2026 Phase 2 SANRECO trial win in polycythemia vera. Trial anchor: divesiran achieved 88% response versus 19% on placebo (P<0.0001, 69% placebo-adjusted response rate) in 48 phlebotomy-dependent patients over 36 weeks. Offering mechanics: priced August 11 at $13.50 per ADS for 12,962,963 ADSs. The underwriters (Jefferies, Morgan Stanley, Cantor Fitzgerald, and William Blair as joint book-running managers) fully exercised their 30-day overallotment option for an additional 1,944,444 ADSs, bringing the total offering to 14,907,407 ADSs and gross proceeds to approximately $175 million. The financing provides the capital to advance divesiran into Phase 3 (planned initiation H1 2027) plus broader pipeline progression including the GalNAc-conjugated hepatic-target siRNA portfolio across TMPRSS6, complement component 3 (SLN-124/SLN-501), and other hepatic and extrahepatic targets. Divesiran has FDA Fast Track and Orphan Drug designations for polycythemia vera; the transferable-voucher secondary market and the recently completed Arrowhead $215 million Priority Review Voucher purchase provide comparable references for the broader siRNA regulatory-instrument landscape.
Silence Therapeutics (NASDAQ: SLN) announced positive topline results from the Phase 2 SANRECO trial of divesiran, a first-in-class TMPRSS6-targeting siRNA product candidate developed from the company's proprietary mRNAi GOLD platform. The trial enrolled 48 phlebotomy-dependent adults with polycythemia vera (PV) and evaluated divesiran 6 mg/kg administered subcutaneously every 6 weeks (Q6W) or every 12 weeks (Q12W) versus placebo over a 36-week randomized double-blind period. Results: 88% of divesiran-treated patients achieved a response versus 19% on placebo (P<0.0001), corresponding to a 69% placebo-adjusted response rate. How divesiran works: it silences TMPRSS6 (transmembrane serine protease 6) expressed almost exclusively in the liver; TMPRSS6 is a negative regulator of hepcidin, the body's master regulator of iron metabolism. By silencing TMPRSS6, divesiran increases hepcidin production and release by liver hepatocytes, which restricts iron availability to bone marrow and reduces the excessive red blood cell production that drives PV symptoms. Divesiran has FDA Fast Track and Orphan Drug designations for PV. Silence Therapeutics anticipates initiating a Phase 3 trial evaluating divesiran Q12W versus placebo in the first half of 2027. Shares rose sharply on the news, touching a 52-week high. The read-through extends the broader siRNA cardiometabolic and rare-disease franchise landscape that also includes Alnylam's Amvuttra and Arrowhead's Redemplo (plozasiran) in adjacent hepatic-target siRNA categories.