Clinical Trials
34
19 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
1966
Active, not recruiting
19
55.9%
Completed
7
20.6%
Recruiting
4
11.8%
Terminated
3
8.8%
Withdrawn
1
2.9%
No approval data available
- China's NMPA has approved Milestone Pharmaceuticals' etripamil nasal spray for paroxysmal supraventricular tachycardia, the first regulatory approval for the drug anywhere. - The approval triggered a $2 million milestone payment to Milestone Pharmaceuticals, strengthening the company's liquidity position and highlighting the Chinese market's importance. - Etripamil remains under regulatory review in Western markets including the United States, with the China decision seen as validation of the clinical data.
- Milestone Pharmaceuticals reported an operating loss of $62 million over the last twelve months with an operating margin of -4019%, signaling severe profitability challenges. - Mirum Pharmaceuticals posted a $70 million operating loss (LTM) despite a 130% stock price surge over the past 12 months, raising valuation concerns. - Both companies remain cash-flow negative, with Milestone's free cash flow margin at -3192% and Mirum's at -32%, underscoring unsustainable burn rates. - Share-based compensation remains elevated at both firms, reaching 483% of revenue at Milestone and 16% at Mirum, potentially diluting shareholder value.
- The FDA has approved CARDAMYST (etripamil) nasal spray, marking the first new treatment option for paroxysmal supraventricular tachycardia (PSVT) in over 30 years for more than 2 million Americans with the condition. - The novel calcium channel blocker allows patients to self-administer treatment outside healthcare settings, potentially avoiding emergency department visits and providing greater control over unpredictable PSVT episodes. - Clinical trials demonstrated that patients using CARDAMYST were twice as likely to convert to normal heart rhythm within 30 minutes compared to placebo, with a median conversion time of 17 minutes versus 54 minutes for placebo. - The approval is supported by robust safety data from over 1,800 participants across multiple trials, with most adverse events being mild and transient nasal-related symptoms.
- Milestone Pharmaceuticals will present poster data on etripamil's efficacy, safety, and tolerability for self-administered treatment of paroxysmal supraventricular tachycardia at AHA Scientific Sessions 2025. - The presentation will combine results from multiple clinical trials examining etripamil's performance in treating acute PSVT episodes. - The company has recently submitted a New Drug Application to the FDA for etripamil as a treatment for PSVT, an abnormal heart rhythm condition. - Dr. James Ip from Weill Cornell Medicine will present the combined clinical trial data on November 10, 2025, in New Orleans.
- DelveInsight's 2025 pipeline report reveals over 12 active companies developing 15+ novel atrial fibrillation therapies, with promising candidates including Abelacimab, Etripamil, and THRV-1268 advancing through clinical trials. - Major pharmaceutical players including Novartis, Bayer, Bristol-Myers Squibb, and Johnson & Johnson are actively evaluating new treatments to address the growing market driven by aging populations and rising AF prevalence. - Recent regulatory milestones include Thryv Therapeutics receiving FDA IND clearance for THRV-1268 and Bayer initiating Phase I trials for BAY 3670549, demonstrating accelerating development momentum. - The pipeline features diverse therapeutic approaches including Factor XI inhibitors, multi-ion channel blockers, and calcium channel antagonists, targeting various aspects of AF management from acute episodes to long-term stroke prevention.
• The FDA has issued a Complete Response Letter for Milestone Pharmaceuticals' Cardamyst (etripamil) nasal spray, citing chemistry, manufacturing, and controls issues rather than safety or efficacy concerns. • TD Cowen downgraded Milestone Pharmaceuticals from Buy to Hold and suspended its price target, citing regulatory uncertainty and the company's limited capital reserves of $69.7 million. • Milestone expects results from ongoing nitrosamine impurity assays in April and plans a Type A meeting with the FDA in May or June to determine the path forward for resubmission.
• The FDA has rejected Milestone Pharmaceuticals' nasal spray Cardamyst for paroxysmal supraventricular tachycardia, citing chemistry, manufacturing and controls issues, causing company shares to plummet over 60%. • Cardamyst, a novel calcium channel blocker, showed promising Phase III results being "twice as effective and three times as fast as placebo" in normalizing heart rhythm for the 2 million Americans affected by PSVT. • Despite the setback, Milestone plans to request a Type A meeting with the FDA to address concerns, while continuing development of Cardamyst for atrial fibrillation in Phase II trials.
- Milestone Pharmaceuticals received a Notice of Allowance from the USPTO for a new patent covering the repeat dose regimen of etripamil nasal spray, extending potential IP protection to July 2042. - The patent supports the New Drug Application (NDA) for CARDAMYST™ (etripamil) currently under FDA review, with a PDUFA target date of March 27, 2025. - Milestone is actively preparing for a mid-2025 commercial launch of CARDAMYST for Paroxysmal Supraventricular Tachycardia (PSVT), pending FDA approval. - A Phase 3 trial of etripamil nasal spray for Atrial Fibrillation with Rapid Ventricular Rate (AFib-RVR) is planned to commence in the first half of 2025.
- Ji Xing Pharmaceuticals' Phase III trial of etripamil nasal spray met its primary endpoint, showing a statistically significant conversion to normal heart rhythm within 30 minutes compared to placebo. - The trial, conducted across 40 hospitals in China, demonstrated that 40.5% of patients converted to sinus rhythm with etripamil versus 15.9% with placebo. - Secondary efficacy endpoints were also met, with treatment-emergent adverse events being comparable between the etripamil and placebo groups, and no serious adverse events reported. - Ji Xing Pharmaceuticals plans to engage with regulatory authorities to advance the new drug application for etripamil, aiming to provide a self-managed treatment option for PSVT patients.
- The FDA has accepted Milestone Pharmaceuticals' NDA for Cardamyst (etripamil) nasal spray, a potential treatment for paroxysmal supraventricular tachycardia (PSVT). - Cardamyst is designed for patient self-administration, offering a rapid-response therapy to manage PSVT episodes without immediate medical oversight. - The PDUFA target date for the FDA's decision on Cardamyst is set for 10 months from the NDA acceptance date of May 26, 2024. - PSVT affects an estimated two million people in the U.S., and Cardamyst aims to provide a convenient and effective treatment option.
