It’s been a busy week for research on rare diseases, with several clinical trials from AnaptysBio, Regenxbio, Sangamo and more posting results from ongoing activities.
Anyone tracking the clinical trial blotter this Labor Day weekend would have seen positive news from Vertex, Boehringer Ingelheim, MannKind Corporation and more. For those who were basking in the sun instead, BioSpace has the details below.
Labor Day Weekend
Vertex announced The FDA approved expanded use of cystic fibrosis drug Orkambi to include treatment of pediatric patients under 2 years of age. The approval will allow for the treatment of patients who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene. Orkambi was previously approved for use in cystic fibrosis patients 2 years and older with two copies of the F508del mutation.
With this approval, approximately 300 children with two copies of the F508del mutation will have a medicine to treat the underlying cause of their disease for the first time, the company said in its announcement.
Cystic fibrosis is caused by a defective or missing CFTR protein that is inherited from both parents. Carmen Bozic, chief medical officer at Vertex, said treating children with cystic fibrosis as early as possible is critically important because it can slow its progression.
MannKind Corporation completed its Phase I program assessing clofazimine inhalation (MNKD-101) suspension as a potential treatment for nontuberculous mycobacterial (NTM) lung disease.
Data from the Phase I study of 24 healthy adults showed MNKD-101 was generally well tolerated at daily doses of up to 90 mg over a seven-day period. The trial assessed three different dosing levels of 30 mg, 60 mg and 90 mg. There were no lab abnormalities, QT prolongation or serious adverse events identified, the company said. MannKind is in talks with the FDA regarding the results and future development of MNKD-101.
Additional data collected during the MKC-CI-001 study is currently undergoing final analysis. Detailed data findings will be presented in upcoming publications and scientific conferences.
MNKD-101 has been designated by the FDA as both an orphan lung and a qualified infectious disease product for the treatment of pulmonary NTM infections.
Boehringer Ingelheim released positive data from its Phase III INpedILD study assessing the pharmacokinetics (dosing) and safety profile of Ofev (nintedanib. The drug is being assessed in pediatric patients ages 6 to 17 who have clinically significant fibrosing interstitial lung disease.
Investigators said the trial data supports the potential use of Ofev as a treatment for these patients. Results showed the weight-based dosing regimen of Ofev “resulted in comparable exposure” to adults assessed with the medication. Ofev posted an acceptable safety and tolerability profile with no new safety signals observed, the company announced.
Data from the study was published in the European Respiratory Journal and will be shared at the European Respiratory Society (ERS) International Congress in Spain.
Based on these findings, Boehringer Ingelheim intends to seek regulatory approval in both Europe and the United States.
September 2
Novartis announced that it is showcasing the latest data from some of the leading immunology products in its pipeline at the European Academy of Dermatology and Venereology congress and the 15th International Symposium of Sjögren’s Syndrome, both happening this month.
A total of 37 abstracts will be featured across the two events, covering areas such as psoriasis, hidradenitis suppurativa, chronic spontaneous urticaria and Sjögren’s Syndrome. Highlights include late-breaking results from trials for Cosyentyx for HS, psoriasis and spondyloarthritis, ianalumab for Sjögren’s Syndrome, remibrutinib for chronic spontaneous urticaria and more.
Immuneering Corporation submitted an Investigational New Drug application to the FDA to support its trial of IMM-1-104, an oral once-daily treatment for advanced RAS mutant solid tumors. The Phase I/IIa clinical trial evaluates a third generation MEK inhibitor that’s made for broad pan-RAS as well as other MAPK-activated tumors.
Preclinical data has, so far, shown that IMM-1-104 has robust anti-tumor capabilities across various in vitro and in vivo tumor models. This is driven by MAPK pathway events, including animal models of KRAS mutant pancreatic cancer, KRAS mutant colorectal cancer, KRAS mutant lung cancer and NRAS mutant melanoma. This all while maintaining a good safety profile regardless of the specific mutation upstream of MEK.
The FDA will assess the IND application before the company is allowed to commence a clinical trial. Immuneering is also preparing for the Phase I/IIa trial of IMM-1-104 for advanced solid tumors with RAS mutations, with the goal to recruit patients at five globally recognized clinical sites in the U.S.
August 31
AnaptysBio failed to meet primary and key secondary efficacy endpoints in its study of imsidolimab for hidradenitis suppurativa. Topline data from the HARP P2b trial, which enrolled 149 patients in Europe and North America, tested the drug for moderate to severe HD. The participants received subcutaneous monthly doses of imsidolimab at either 400 mg/200 mg, 200 mg/100 mg or a placebo, followed by a 16-week extension period.
While the drug demonstrated safety and tolerability throughout, it did not achieve the primary endpoint of a mean change in AN lesion count from baseline by the 16th week. It also did not meet the secondary endpoint based on the Hidradenitis Suppurativa Clinical Response, which regulatory agencies typically use to evaluate potential treatments for HS.
Regenxbio shared additional positive interim results from its Phase I/II/III trial of candidate RGX-121 for patients under five years old with Hunter Syndrome (mucopolysaccharidosis Type II, MPS II). Data from the CAMPSITE trial demonstrated the drug’s ability to meet the primary endpoint of safety, with new data showing the largest median reduction in CSF glycosaminoglycans in patients at 48 weeks. RGX-121 also continued to be well-tolerated across all three dose levels evaluated. Details were presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) annual symposium.
August 30
Sangamo Therapeutics released preliminary Phase I/II data from its trial of isaralgagene civaparvovec (ST-920), showing that the candidate drug for Fabry disease continued to be well tolerated across three dose cohorts. The five longest treated participants in the study demonstrated elevated α-Gal A activity for up to 15 months as of the February 14 cutoff date, prompting the researchers to advance the Phase I/II STAAR trial into the dose expansion phase. The α-Gal A levels ranged from almost threefold to nearly 17-fold above the mean normal.
