Clinical Trials
16
6 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
1992
Active, not recruiting
6
37.5%
Completed
6
37.5%
Recruiting
3
18.8%
Withdrawn
1
6.3%
No approval data available
- Silence Therapeutics achieved positive Phase II results for zerlasiran in ASCVD patients, demonstrating greater than 90% reduction in Lp(a) levels. - Phase I results for divesiran in polycythemia vera showed elimination of phlebotomy need in well-controlled patients, earning orphan drug designation. - The company reported 2024 revenue of $43.3 million, up from $31.6 million in 2023, with net loss improving to $45.3 million from $54.2 million. - Phase III for zerlasiran will only commence after securing a partnership, as Silence prioritizes rare disease programs and extends cash runway into 2027.
- A comprehensive analysis reveals over 40 companies are developing more than 90 RNA interference therapeutics, highlighting the sector's rapid expansion and therapeutic potential. - Recent strategic partnerships include SanegeneBio's global licensing agreement with Genentech and Arrowhead Pharmaceuticals' collaboration with Novartis for Parkinson's disease treatment. - RNAi therapies offer promising alternatives to conventional drugs, particularly for infectious diseases and genetic disorders where traditional treatments face limitations. - Key marketed therapies like Lumasiran (OXLUMO) for primary hyperoxaluria and investigational drugs like Cemdisiran in Phase III trials demonstrate the technology's clinical advancement.
- Silence Therapeutics has completed enrollment of 48 patients in the SANRECO Phase 2 study evaluating divesiran, a first-in-class siRNA targeting TMPRSS6, for polycythemia vera treatment. - The randomized, double-blind, placebo-controlled trial aims to demonstrate divesiran's ability to maintain hematocrit levels below 45% without phlebotomies between weeks 18 and 36. - Phase 1 results showed divesiran's potential to maintain target hematocrit levels with infrequent dosing every six weeks without requiring phlebotomies. - Initial topline results from the Phase 2 study are anticipated in the third quarter of 2026, addressing significant unmet medical needs in this rare blood cancer.
- Silence Therapeutics presented updated Phase 1 data for divesiran at EHA 2025, showing the siRNA therapy essentially eliminated the need for phlebotomies in 21 polycythemia vera patients with a combined history of 79 prior phlebotomies. - The SANRECO Phase 1 study demonstrated that divesiran maintained hematocrit levels at ≤45% across all dose cohorts while increasing hepcidin and ferritin levels, with no dose-limiting toxicities observed. - The company announced that its Phase 2 SANRECO study has exceeded 50% enrollment and remains on track for full enrollment by year-end 2025. - Divesiran represents a first-in-class siRNA therapy targeting TMPRSS6 to regulate iron metabolism and reduce excessive red blood cell production in polycythemia vera patients.
- Eli Lilly's experimental drug lepodisiran demonstrated up to 95% reduction in lipoprotein(a) levels with a single 400mg dose in midstage trials, potentially addressing a significant cardiovascular risk factor prevalent in South Asian populations. - The once-yearly injectable targets LPA cholesterol particles that are found in elevated levels in approximately 25% of South Asians and are not addressed by current cholesterol-lowering medications like statins. - With an estimated 64 million Indians suffering from heart conditions and above-average death rates, lepodisiran represents a targeted intervention that could significantly impact cardiovascular disease management in the region.
- Silence Therapeutics has paused the Phase III development of zerlasiran, its RNA-silencing therapy for cardiovascular diseases, until securing a strategic partnership, extending their cash runway into 2027. - The company's lead candidate zerlasiran demonstrated promising results with approximately 90% median maximum Lp(a) reduction at 48 weeks in high-risk cardiovascular patients. - The strategic pivot allows Silence to focus resources on rare disease programs, including divesiran for polycythemia vera, with Phase II SANRECO study enrollment expected by year-end.
- Zerlasiran, a gene-silencing therapy, demonstrated sustained reductions in lipoprotein(a) [Lp(a)] levels through 60 weeks in the ALPACAR-360 trial. - The Phase 2 trial showed zerlasiran reduced Lp(a) levels by 81.3% to 85.6% at 36 weeks, with consistent reductions maintained through 60 weeks. - Zerlasiran was well-tolerated, with mostly mild injection site reactions, suggesting its potential as a long-term, infrequent-dose therapy for high Lp(a). - The trial's findings support the progression of zerlasiran into Phase 3 trials for patients with elevated Lp(a) and high cardiovascular risk.
- Zerlasiran, a novel siRNA, significantly reduced time-averaged lipoprotein(a) levels by over 80% in a Phase 2 trial, offering a potential treatment for elevated Lp(a). - The ALPACAR-360 trial demonstrated the efficacy and safety of zerlasiran with infrequent dosing in patients at high risk of atherosclerotic cardiovascular disease (ASCVD). - The study's findings support the advancement of zerlasiran into Phase 3 trials as a promising gene-silencing approach for individuals with genetically determined high Lp(a) levels. - Common treatment-related adverse effects were mild injection site reactions, with no serious adverse events linked to zerlasiran, highlighting its tolerability.
- Zerlasiran, a GalNAc-conjugated siRNA, significantly reduced lipoprotein(a) levels by 80-85% in patients at high risk of atherosclerotic cardiovascular disease. - The Phase II trial showed that zerlasiran, administered subcutaneously every 16 or 24 weeks, also lowered LDL-C by 25-30% and apoB by 10-15%. - Zerlasiran targets the LPA gene, offering a precise approach to reduce cardiovascular risk associated with elevated lipoprotein(a) levels, an unmet need in current therapies. - The investigational gene-silencing therapy was well-tolerated, suggesting potential for less frequent dosing in Phase III trials and improved patient adherence.
- Zerlasiran, a GalNAc-conjugated siRNA, significantly reduced lipoprotein(a) levels in patients at high risk of atherosclerotic cardiovascular disease. - Subcutaneous injections of zerlasiran (300mg or 450mg) every 16 or 24 weeks led to an 80-85% reduction in time-averaged Lp(a) levels over 36-60 weeks. - The Phase II trial also demonstrated that zerlasiran lowered time-averaged LDL-C by 25-30% and apoB by 10-15%, with good tolerability and no significant safety concerns. - Zerlasiran addresses the unmet need for therapies specifically targeting Lp(a), potentially improving patient adherence and long-term cardiovascular outcomes.
