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RNA Stock Moves More Than 30% in a Week: What's Driving This Rally?
ZACKS· 2025-08-25 19:26
Key Takeaways Avidity shares jumped 36% after reports that Novartis is interested in acquiring the biotech.FT noted talks are in early stages with no guarantee of a deal and other suitors may emerge.Avidity develops RNA therapies for rare muscular diseases and has major pharma partnerships.Shares of Avidity Biosciences (RNA) have soared 36% in the past month, all thanks to a report issued by the Financial Times (FT), which stated that pharma giant Novartis (NVS) is interested in acquiring the company.Per th ...
Avidity Biosciences Receives FDA Breakthrough Therapy Designation for Delpacibart Zotadirsen (del-zota) for the Treatment of DMD in People with Mutations Amenable to Exon 44 Skipping
Prnewswire· 2025-07-23 13:00
Core Insights - Avidity Biosciences has received Breakthrough Therapy designation from the FDA for delpacibart zotadirsen (del-zota) aimed at treating Duchenne muscular dystrophy (DMD) in patients with mutations suitable for exon 44 skipping [1][4][7] - The company is on track for a Biologics License Application (BLA) submission by the end of 2025, with ongoing preparations for a potential U.S. launch of del-zota following FDA approval [5][4] - Del-zota is currently in the Phase 2 EXPLORE44 Open-Label Extension trial, building on positive results from the Phase 1/2 trial that showed significant improvements in biomarkers related to DMD [2][4][10] Company Overview - Avidity Biosciences focuses on developing a new class of RNA therapeutics known as Antibody Oligonucleotide Conjugates (AOCs™) [13] - The company aims to address rare neuromuscular diseases, including DMD, myotonic dystrophy type 1 (DM1), and facioscapulohumeral muscular dystrophy (FSHD) [13] - Avidity's proprietary AOC platform allows for targeted delivery of RNA into muscle tissue, which is a significant advancement in the field of RNA therapeutics [13] Product Development - Del-zota is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to specifically skip exon 44 of the dystrophin gene, facilitating the production of near-full length dystrophin [3][12] - The Phase 1/2 EXPLORE44 trial demonstrated statistically significant increases in exon skipping and dystrophin production, along with a reduction in creatine kinase levels to near normal [4][12] - The ongoing EXPLORE44-OLE study will evaluate the long-term safety and efficacy of del-zota, with a total treatment duration of approximately 24 months [9][10] Regulatory Designations - In addition to Breakthrough Therapy designation, del-zota has received Orphan designation, Rare Pediatric Disease designation, and Fast Track designation from the FDA [7][12] - These designations highlight the drug's potential to significantly improve treatment options for patients with DMD, a condition with a high unmet medical need [11][6]
Avidity Biosciences (RNA) Earnings Call Presentation
2025-06-09 12:20
Del-brax Development and Regulatory Pathway - FDA has confirmed an accelerated approval pathway for delpacibart braxlosiran (del-brax) in the US [8] - The primary endpoint for accelerated approval is the reduction in the circulating biomarker cDUX [10, 59, 76] - A global confirmatory Phase 3 FORWARD trial has been initiated with 200 participants in a 1:1 randomized, double-blind, placebo-controlled design [10, 79] - The company anticipates topline data from the biomarker cohort of the FORTITUDE trial in Q2 2026 and a BLA submission in H2 2026 [10, 74] - The confirmatory Phase 3 FORWARD trial is designed to support full approval, with approximately 45 global sites [80, 83] FORTITUDE Trial Data - 12-month topline data from the FORTITUDE trial showed improved functional mobility in del-brax treated participants compared to placebo, as measured by 10MWRT and TUG [35, 39, 57] - Participants treated with del-brax also demonstrated improved muscle strength measured by QMT and improved upper limb function measured by RWS compared to placebo [42, 44, 57] - Del-brax showed a favorable long-term safety and tolerability profile in the FORTITUDE trial and its open-label extension (OLE) [10, 19, 57] - Over 60% of patients express that slowing or stopping the loss of muscle function would be the most meaningful outcome [30] cDUX Biomarker - cDUX is elevated 6- to 9-fold in people living with FSHD compared to healthy volunteers [59] - Del-brax treatment resulted in rapid and statistically significant reductions in cDUX and creatine kinase (CK) levels [59, 64]
Avidity Biosciences Announces Positive Topline Phase 1/2 FORTITUDE™ Data Demonstrating Consistent Improvement Across Multiple Functional Measures Compared to Placebo in Del-Brax Treated FSHD Participants
Prnewswire· 2025-06-09 11:01
Core Insights - Avidity Biosciences announced positive topline data from the Phase 1/2 FORTITUDE™ program for delpacibart braxlosiran (del-brax) in treating Facioscapulohumeral Muscular Dystrophy (FSHD), showing improvements in function, strength, and patient-reported outcomes compared to placebo [1][3][6] - The company plans to submit a Biologics License Application (BLA) for accelerated approval in the second half of 2026 [1][3] - Del-brax is the first investigational therapy targeting the underlying cause of FSHD by directly addressing the DUX4 gene, with no approved therapies currently available for this condition [2][13] Group 1: Clinical Data and Results - The FORTITUDE™ trial included a randomized, placebo-controlled, double-blind study evaluating 39 participants on doses of 2 mg/kg or 4 mg/kg of del-brax over 12 months [5][10] - Results indicated consistent improvements in functional mobility and muscle strength, as measured by the 10-Meter Walk-Run test, Timed Up and Go, and quantitative muscle testing [6][9] - Significant reductions in biomarkers such as KHDC1L and creatine kinase were observed, indicating muscle damage [6][7] Group 2: Safety and Regulatory Pathway - Del-brax demonstrated favorable long-term safety and tolerability, with most adverse events being mild or moderate, and no serious adverse events reported [7][9] - The U.S. FDA has opened the accelerated approval regulatory pathway for del-brax, and a global Phase 3 FORWARD™ study has been initiated [3][4] Group 3: Future Developments - The ongoing biomarker cohort of the FORTITUDE trial aims to assess the impact of del-brax on KHDC1L levels, with topline data expected in Q2 2026 [7][11] - Avidity is advancing its clinical development pipeline, focusing on RNA therapeutics through its proprietary Antibody Oligonucleotide Conjugates (AOCs™) platform [16]
Wave Life Sciences Announces Appointment of Dr. Christopher Wright as Chief Medical Officer
Globenewswire· 2025-05-28 12:00
Core Insights - Wave Life Sciences Ltd. has appointed Dr. Christopher Wright as Chief Medical Officer to lead the clinical development of its RNA medicines pipeline [1][2] - Dr. Wright brings extensive experience in global development, having worked with regulatory agencies in both the US and EU, and has a strong track record in drug development for various diseases [2][3] - The company has a robust therapeutic pipeline and has delivered positive clinical datasets over the past year, indicating the potential of its RNA medicines [4] Company Overview - Wave Life Sciences is a biotechnology company focused on RNA medicines, utilizing its PRISM platform to address both rare and common disorders [5] - The company's RNA-targeting modalities include editing, splicing, RNA interference, and antisense silencing, providing a comprehensive toolkit for disease treatment [5] - Wave's pipeline includes clinical programs for Alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy, Huntington's disease, and obesity, along with several preclinical programs [5] Leadership Background - Dr. Wright has held significant roles in various biotechnology companies, including CMO at Ring Therapeutics and AavantiBio, and has experience in leading global development functions [2][3] - He has a strong academic background, having served as a board-certified neurologist and Associate Professor of Neurology at Harvard Medical School [3] Equity Grant - In connection with his appointment, Dr. Wright received a share option to purchase 300,000 ordinary shares of Wave, with an exercise price of $5.94 per share, vesting over four years [4]
TransCode Therapeutics Appoints Dr. Phillip D. Zamore to Science Advisory Board
Prnewswire· 2025-05-28 12:00
Company Overview - TransCode Therapeutics, Inc. is a clinical-stage oncology company focused on developing RNA-targeted therapeutics for cancer treatment [1][4] - The company aims to combat metastatic disease through the intelligent design and effective delivery of RNA therapeutics using its proprietary TTX nanoparticle platform [4] Key Appointment - Dr. Phillip D. Zamore has been appointed to the Scientific Advisory Board of TransCode Therapeutics [1] - Dr. Zamore is recognized for his pioneering work in RNA interference (RNAi) and co-founded Alnylam Pharmaceuticals, which developed the first FDA-approved RNAi drug [1][6] Research Contributions - Dr. Zamore's research has advanced the understanding of non-coding RNA and its implications in health and disease, particularly in the processing of microRNAs [2] - His contributions have earned him recognition from prestigious institutions, including the National Academy of Sciences and the National Academy of Medicine [2] Strategic Vision - The company emphasizes the importance of Dr. Zamore's expertise in RNA biology to enhance its pipeline of RNA-targeted cancer therapies [3] - Dr. Zamore expressed enthusiasm for contributing to the development of effective RNA-based treatments for cancer patients [3] Product Focus - TransCode's lead therapeutic candidate, TTX-MC138, targets metastatic tumors that overexpress microRNA-10b, a biomarker associated with metastasis [4] - The company has a portfolio of first-in-class RNA therapeutic candidates aimed at overcoming RNA delivery challenges to access novel genetic targets for various cancers [4]
TransCode Therapeutics Successfully Completes Initial Dosing of Patients in Cohort 4 of Phase 1a Clinical Trial; No Dose Limiting Toxicities Reported
Prnewswire· 2025-05-08 12:05
Core Viewpoint - TransCode Therapeutics is advancing its Phase 1a clinical trial for TTX-MC138, a novel RNA therapeutic aimed at treating metastatic cancers, with positive safety and pharmacokinetic data supporting further development [1][3][5]. Group 1: Clinical Trial Progress - The third patient in Cohort 4 has received the initial dose of TTX-MC138, with all cohorts having at least three patients dosed [1]. - A total of 15 patients have been treated across four dose levels (0.8 mg/kg to 4.8 mg/kg), with no significant safety or dose-limiting toxicities reported [2][8]. - Ten patients remain on study, receiving doses every 28 days, with two patients showing stable disease after seven doses over approximately seven months [2][8]. Group 2: Safety and Efficacy Data - Preliminary pharmacokinetic (PK) and pharmacodynamic (PD) analyses indicate a predictable dose-response relationship, with evidence of miR-10b target engagement at 24 hours post-infusion [2][4][8]. - The observed tolerability profile supports the advancement of the trial into the Phase 1b dose expansion stage to further evaluate safety and potential anti-tumor activity [3]. Group 3: About TTX-MC138 - TTX-MC138 is designed to inhibit microRNA-10b, which is implicated in the progression of many metastatic cancers, and has shown promising delivery and activity in preclinical studies [4][7]. - The drug candidate has demonstrated a broad therapeutic window, suggesting potential for effective treatment of metastatic tumors [4]. Group 4: Trial Design - The Phase 1 clinical trial is a multicenter, open-label study aimed at evaluating the safety and tolerability of TTX-MC138 in patients with various metastatic solid cancers [5]. - The trial consists of an initial dose-escalation stage followed by a dose-expansion stage to further assess safety, tolerability, and anti-tumor activity [5].
TransCode Therapeutics Reports Further Progress on Phase 1a Clinical Trial with No Dose Limiting Toxicities Reported in Patients with Metastatic Cancer
Prnewswire· 2025-05-01 13:01
Core Insights - TransCode Therapeutics is advancing its Phase 1a clinical trial of TTX-MC138, a first-in-class therapeutic candidate targeting microRNA-10b, which is believed to play a significant role in metastatic cancer progression [1][4][7] - The trial has treated 13 patients with doses ranging from 0.8 mg/kg to 4.8 mg/kg, with no significant safety issues reported [1][8] - Preliminary pharmacokinetic (PK) and pharmacodynamic (PD) data indicate a favorable profile for TTX-MC138, supporting its progression to the Phase 1b trial [2][3] Company Overview - TransCode Therapeutics focuses on RNA therapeutics for treating metastatic diseases, utilizing its proprietary TTX nanoparticle platform [7] - The lead candidate, TTX-MC138, aims to inhibit microRNA-10b, a biomarker associated with metastasis [7] Clinical Trial Details - The Phase 1 clinical trial is designed as a multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety and tolerability of TTX-MC138 in patients with various metastatic solid cancers [5] - The trial consists of an initial dose-escalation stage followed by a dose-expansion stage, with the primary objective of assessing safety and tolerability [5] Treatment Progress - Eight patients are currently receiving ongoing treatment, with two patients demonstrating stable disease after seven months of treatment [2][8] - The trial has shown a predictable dose-response relationship in PK data, with evidence of target engagement for miR-10b in 5 out of 6 patients analyzed [2][8]
TransCode Therapeutics Announces Initial Dosing in Fourth Cohort of Phase 1 Clinical Trial with TTX-MC138
Prnewswire· 2025-03-27 20:37
Core Insights - TransCode Therapeutics has initiated treatment for the first patient in Cohort 4 of its Phase I clinical trial for TTX-MC138, a therapeutic candidate targeting microRNA-10b, which is associated with metastatic cancer [1][3] - The trial has shown no significant safety or dose-limiting toxicities among the ten patients treated so far, with seven remaining on study for continued treatment [2][8] - The ongoing analysis of pharmacokinetic and pharmacodynamic data suggests that TTX-MC138 aligns with preclinical results, indicating a promising safety profile [2][4] Company Overview - TransCode Therapeutics is focused on developing RNA therapeutics to treat metastatic diseases, utilizing its proprietary TTX nanoparticle platform [7] - TTX-MC138 is the lead candidate designed to inhibit microRNA-10b, which is believed to drive metastatic disease [4][7] - The company aims to address challenges in RNA delivery to unlock therapeutic access to various genetic targets relevant for cancer treatment [9] Clinical Trial Details - The Phase 1 clinical trial is a multicenter, open-label, dose-escalation and dose-expansion study aimed at evaluating the safety and tolerability of TTX-MC138 in patients with metastatic solid cancers [5] - The trial consists of an initial dose-escalation phase followed by a dose-expansion phase, with the primary objective of assessing safety and tolerability [5] - Cohort 4's treatment is expected to provide insights for dose expansion and initial evidence of clinical activity as the trial progresses [3][5]