Updates include expected timelines for trials for the treatment of wet AMD, DME, GA, and inherited retinal diseases.
4D Molecular Therapeutics (4DMT) has shared clinical pipeline progress, updates to near-term milestones, and organizational updates as the company looks ahead to 2024. The updates included expected timelines for trials of 4D-150 for Wet AMD and DME as well as 4D-175 for geographic atrophy. The company also noted the progress expected for 4D-110 for Choroideremia and 4D-125 for X-Linked Retinitis Pigmentosa.
David Kirn, MD, Co-founder and Chief Executive Officer of 4DMT summarized 2023 and noted the company’s intended progress for the coming year in his statement. In the news release1 he said, “2023 was a landmark year for 4DMT, highlighted by rapid enrollment for 4D-150 in wet AMD and DME, strong CFTR protein expression data for 4D-710 in lungs of people with CF, multiple value-generating business development partnerships, and key additions to the Executive Team. Looking forward to 2024, we plan to release multiple important datasets from our lead programs in wet AMD, DME and CF. We also plan to share guidance on the design and timing of Phase 3 clinical trials. In addition, we expect to initiate clinical development of 4D-175 in geographic atrophy by the second half of the year. By the end of 2024, we intend to progress clinical development in four large market diseases, positioning us well in our strategy to become a fully integrated large market genetic medicines company.”
4D-150 for Wet AMD
According to the company's press release1, phase 2 PRISM Clinical Trial has a randomized Dose Expansion arm (N=50) in advanced high treatment need patients. Initial interim 24-week landmark data on this arm is set to be presented at the Angiogenesis, Exudation, and Degeneration 2024 Conference on Saturday, February 3, 2024, followed by a corporate webcast with details to be announced at a future date.
The Phase 2 PRISM clinical trial also has a population extension arm (N≤45) in broad population (non-advanced, standard treatment-need).Enrollment update for this arm is expected in Q1 2024 and an initial interim 24-week landmark data analysis is expected in H2 2024.
The Phase 2 PRISM trial received both RMAT and PRIME in Q4 2023, increasing the collaboration between the FDA and EMA on regulatory approval planning, in addition to the opportunity for expedited product development.
An update on Phase 3 trial plans expected in February 2024 along with the interim randomized Phase 2 PRISM trial data.
4D-150 for DME
The Phase 2 SPECTRA clinical trial: Part 1 is at the dose confirmation stage and completed enrollment in Q4 2023. 4DMT expected Initial interim 24-week landmark data in H2 2024.1
4D-175 for Geographic Atrophy
The company expected to file an IND with the FDA in Q2 2024. Phase 1 for this treatment is currently planned in H2 2024.1
Rare Inherited Retinal Disorders: 4D-110 for Choroideremia and 4D-125 for X-Linked Retinitis Pigmentosa
All enrolled patients are expected to reach 24 months of follow-up in 2024. The company plans to assess the magnitude and durability of key imaging and functional endpoint changes in evaluable patients. Updates on this program will likely be shared in 2024.1
The appointment of a new Chief Scientific Officer (CSO) and Scientific Advisory Board (SAB)
In addition to the pipeline updates provided in the news release, 4DMT has appointed Noriyuki (Nori) Kasahara, MD, PhD, as Chief Scientific Officer after previously serving as a member of 4DMT’s Board of Directors and chaired the Board’s Science & Technology Committee from September 2022 to December 2023.1
4DMT has also formed a scientific advisory board (SAB) whos members include key opinion leaders in AAV gene therapy, immunology, and core 4DMT therapeutic areas. More details on the 4DMT board members can be found on the SAB section of the 4DMT website.