| Over a week ago | ||||
Dyne Therapeutics… Dyne Therapeutics announced that the European Medicines Agency, EMA, has granted orphan drug designation for DYNE-101. DYNE-101 is being evaluated in the Phase 1/2 ACHIEVE global clinical trial in adults with myotonic dystrophy type 1, DM1. "We are pleased to receive orphan drug designation from the EMA for DYNE-101, further supporting our efforts to develop a potentially transformative therapy for DM1," said Wildon Farwell, M.D., MPH, chief medical officer of Dyne. "The DM1 community has waited far too long for a therapy that addresses the underlying cause of this devastating rare muscle disease. We are committed to advancing DYNE-101 as quickly as possible and anticipate our first clinical data readout from our ACHIEVE trial later this year." | ||||
Dyne Therapeutics is… Dyne Therapeutics is delivering an oral presentation today at the American Society of Gene & Cell Therapy 26th Annual Meeting in Los Angeles highlighting new preclinical data demonstrating the FORCE platform achieved delivery to the central nervous system in non-human primates and robust pharmacological effects in the brain in a model of myotonic dystrophy type 1. The presentation will be available in the Scientific Publications & Presentations section of Dyne's website following the session. Data being presented at ASGCT show that intravenous administration of FORCE conjugate, a TfR1-binding Fab antibody conjugated to an antisense oligonucleotide, achieved delivery to the CNS via TfR1 in both non-human primates and the innovative hTfR1/DMSXL mouse model. The hTfR1/DMSXL model, developed by Dyne, expresses the human TfR1 and carries a human DMPK gene with more than 1,000 CTG repeats that represents a severe DM1 phenotype. In these studies, FORCE conjugate was well tolerated. Highlights from the ASGCT data include: In NHPs, FORCE conjugate achieved superior delivery compared to unconjugated ASO when both were administered via IV. In addition, IV administration of FORCE showed broader distribution throughout the brain compared to intrathecal administration of unconjugated ASO. FORCE conjugate also delivered to the brain of hTfR1/DMSXL mice and demonstrated robust knockdown of toxic human nuclear DMPK RNA and foci reduction. | ||||
"We are making… "We are making excellent progress in both our ACHIEVE and DELIVER trials and are on track to report initial data from both in the second half of 2023, including evaluating the key disease-driving biomarkers of splicing in DM1 and dystrophin in DMD," said Joshua Brumm, president and chief executive officer of Dyne. "Additionally, we are pleased to be presenting on our FORCE platform and clinical programs at premiere scientific meetings, engaging with the communities treating and living with DM1 and DMD. In particular, we are excited about the preclinical data that are being shared at ASGCT this month which, for the first time, demonstrate that FORCE leveraged TfR1 to deliver to the CNS, reinforcing the potential broad applicability of the platform to address multiple clinical manifestations of neuromuscular diseases." | ||||
| Over a month ago | ||||
Meetings to be held March… Meetings to be held March 30-31 hosted by Oppenheimer. | ||||
Meetings to be held March… Meetings to be held March 30-31 hosted by Oppenheimer. | ||||
Dyne Therapeutics… Dyne Therapeutics announced that DYNE-251, an investigational therapeutic for Duchenne muscular dystrophy mutations amenable to exon 51 skipping, was granted U.S. Food and Drug Administration, FDA, orphan drug and rare pediatric disease designations. DYNE-251 is being evaluated in the Phase 1/2 DELIVER clinical trial. "These regulatory designations highlight the urgent and critical need for new and better therapeutic options for people living with this fatal disease," said Wildon Farwell, M.D., MPH, chief medical officer of Dyne. "We are excited about DYNE-251 which we believe has the potential to transform the lives of people with DMD. We continue to advance our DELIVER clinical trial and look forward to sharing initial clinical data later this year." | ||||
The FDA granted Dyne… The FDA granted Dyne Therapeutics orphan status for its treatment of Duchenne muscular dystrophy. Reference Link | ||||
Chardan analyst Keay… Chardan analyst Keay Nakae raised the firm's price target on Dyne Therapeutics to $20 from $17 and keeps a Buy rating on the shares post the Q4 results. The analyst believes the initial safety and efficacy data from the DYNE-101 and DYNE-251 programs will provide important clinical proof of concept for the Dyne's Force platform to successfully deliver to ex-hepatic targets. | ||||
| Over a quarter ago | ||||
Cash, cash equivalents… Cash, cash equivalents and marketable securities were $256.0 million as of December 31, 2022, which is anticipated to fund operations through 2024. | ||||
"2022 was a… "2022 was a momentous year for Dyne and our commitment to the rare muscle disease community as we advanced not one, but two programs from our FORCE platform into clinical trials. This exciting transition into a clinical-stage company required important work across the organization to engage with individuals and families living with DM1 and DMD, clinicians treating these diseases, regulators and other stakeholders," said Joshua Brumm, president and chief executive officer of Dyne. "We remain focused on driving to meaningful clinical data readouts for our ACHIEVE and DELIVER trials anticipated in the second half of 2023, including evaluating key biomarkers of dystrophin in DMD and splicing in DM1. With cash runway expected through 2024 and strong fundamentals across the business, we believe we are well positioned to achieve our near-term milestones and to advance our mission of delivering life-transforming therapies for people with serious muscle diseases." | ||||
