相关临床试验
25
6 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
2016
进行中（未招募）
5
20.0%
已完成
9
36.0%
Enrolling By Invitation
1
4.0%
招募中
5
20.0%
终止
5
20.0%
暂无批准数据
- Fulcrum Therapeutics and Slate Medicines announced a definitive all-stock merger agreement, with the combined company operating as Slate Medicines and trading on Nasdaq under the ticker "SLTE." - Slate's lead candidate SLTE-1009 is a clinical-stage subcutaneous anti-PACAP/VIP monoclonal antibody designed for the preventative treatment of migraine and other headache disorders. - A concurrent oversubscribed $245 million private placement, led by Frazier Life Sciences, is expected to fund operations into 2029 and advance SLTE-1009 through Phase 1 and Phase 2 studies. - Pre-merger Fulcrum stockholders will own 5% of the combined company and receive a cash dividend estimated at $270 million, with the transaction expected to close in the fourth quarter of 2026.
- Fulcrum Therapeutics discontinued development of pociredir for sickle cell disease after FDA expressed heightened concerns about cancer risk associated with PRC2 complex inhibitors. - The FDA's decision was triggered by the global withdrawal of Tazverik (tazemetostat) in March 2026 due to unexpectedly high rates of secondary hematologic malignancies. - With pociredir representing the company's only clinical-stage asset, Fulcrum has initiated a strategic review to explore merger, acquisition, or other transactions to maximize shareholder value. - Company shares plummeted 49% in after-hours trading following the announcement, leaving Fulcrum with no near-term pipeline after previous program failures.
- AstronauTx has appointed Dr. Michelle Mellion as Chief Medical Officer, bringing over 20 years of neurology and clinical development experience to advance the company's sleep architecture-targeting therapeutics. - The company is developing first-in-class therapies that target sleep architecture as a potential disease-modifying strategy for Alzheimer's disease and other neurological disorders. - AstronauTx's innovative approach uses EEG-based sleep architecture metrics and advanced data analytics to improve slow-wave sleep quality and potentially slow cognitive decline. - The appointments strengthen the company's clinical capabilities as it prepares to initiate clinical development of its pipeline following a $61 million Series A financing in October 2023.
- The FSHD Society, SOLVE FSHD, and FSHD CTRN announced the launch of a first-of-its-kind multi-stakeholder Industry Collaborative to improve clinical trial design and execution for facioscapulohumeral muscular dystrophy. - The initiative brings together industry experts, academic researchers, and technology partners to analyze biomarker and clinical endpoint data, aiming to reduce drug development risk and improve patient selection strategies. - Founding sponsors include Scholar Rock developing apitegromab, with Fulcrum Therapeutics donating Phase 2 and Phase 3 losmapimod trial data after program suspension in 2024. - The collaborative operates as a pre-competitive effort across multiple datasets while protecting sponsor confidentiality, building on prior FDA engagements to address regulatory expectations for FSHD therapeutics.
- Eight biotech companies raised a record $3.5 billion in a single night following the American Society of Hematology conference, marking the busiest day ever in biotech fundraising. - Terns Pharmaceutical led the surge with a $650 million offering after presenting Phase I leukemia trial data showing 64% response rates, with potential peak sales projected at $2.5 billion. - Fulcrum Therapeutics raised $175 million following positive Phase Ib results for its sickle cell disease treatment in expectant mothers, demonstrating meaningful increases in fetal hemoglobin levels. - The fundraising wave reflects strong biotech sector performance with the NYSE Arca Biotechnology Index up 27% year-to-date, driven by positive trial results and increased M&A activity.
- Fulcrum Therapeutics reported interim results showing their experimental pill increased fetal hemoglobin levels in sickle cell disease patients, offering a potential oral treatment alternative. - The study demonstrated positive outcomes in key disease markers, though experts noted the impact on fetal hemoglobin levels was modest rather than transformative. - While the pill-based approach could simplify treatment compared to existing therapies, researchers emphasize more studies are needed to confirm long-term efficacy and patient benefits.
- Dyne Therapeutics' DYNE-302 demonstrated functional improvement in a severe FSHD mouse model, with a single intravenous dose restoring treadmill running ability at peak muscle weakness. - The siRNA-based therapy targets DUX4 mRNA and showed correction of muscle damage and inflammation markers in skeletal muscle tissue analysis. - Results suggest that preexisting and severe skeletal muscle disease in FSHD may be reversible through DUX4 mRNA targeting, offering hope for the estimated 16,000-38,000 affected individuals in the United States. - The findings will be presented at the 32nd Annual FSHD Society's International Research Congress in Amsterdam on June 13, 2025.
- Epicrispr Biotechnologies has raised $68 million in Series B funding to develop EPI-321, a first-in-class epigenetic therapy targeting facioscapulohumeral muscular dystrophy (FSHD). - New Zealand's Medsafe has approved Epicrispr's clinical trial application, with the first-in-human study of EPI-321 expected to begin in 2025 to evaluate its safety and biological activity in adults with FSHD. - EPI-321 uses CRISPR technology to silence the DUX4 gene that drives FSHD progression and has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations.
- Fulcrum Therapeutics has successfully enrolled 10 patients in the 12mg dose cohort of the Phase 1b PIONEER trial for pociredir in sickle cell disease, with data expected by mid-2025. - The company maintains a strong financial position with $241 million in cash reserves, providing runway into at least 2027 to support ongoing clinical development programs. - Pociredir, an oral EED inhibitor, shows promise in increasing fetal hemoglobin levels and could potentially transform SCD treatment by offering a differentiated oral therapy option.
• Pfizer has voluntarily withdrawn Oxbryta (voxelotor) from all global markets due to safety concerns, citing an imbalance in vaso-occlusive crises and fatal events. • The decision follows a review of clinical data that indicated the overall benefit of Oxbryta no longer outweighs the risks for sickle cell disease patients. • Regulatory agencies, including the EMA and FDA, have alerted healthcare professionals and patients about the withdrawal, advising discussions on alternative treatments. • The withdrawal impacts ongoing clinical trials and expanded access programs, leaving the sickle cell community seeking alternative treatment options and raising concerns about trust.
