Safety and Efficacy ATYR1923, a Novel Immunomodulator for Pulmonary Sarcoidosis: Results of a Phase 1b/2a Randomized Placebo-Controlled Trial

Sporn, Peter H. S.; Culver, Daniel; Barney, Joseph B.; Maier, Lisa; Hsia, CC; Sweiss, Nadera J.; Aryal, Shambhu; Ndili, Ogugua; Tabassomi, M.; Marts, Lucian T.; Walker, G.; Carey, Leeanne M.; Shukla, S.; Baughman, R.P.

doi:10.1164/ajrccm-conference.2022.205.1_meetingabstracts.a3932

Cited by 2 publications

(4 citation statements)

References 0 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…In a Phase II study, Efzofitimod was tested in patients with pulmonary sarcoidosis. The biologic was found to be safe and well tolerated, with no cases of immunogenicity (150). Efzofitimod is currently undergoing a Phase III multicentre double-blind placebo-controlled RCT in pulmonary sarcoidosis patients (NCT05415137) (151).…”

Section: Pulmonary Sarcoidosismentioning

confidence: 99%

Biologic drugs in the treatment of chronic inflammatory pulmonary diseases: recent developments and future perspectives

2023

View full text Add to dashboard Cite

Chronic inflammatory diseases of the lung are some of the leading causes of mortality and significant morbidity worldwide. Despite the tremendous burden these conditions put on global healthcare, treatment options for most of these diseases remain scarce. Inhaled corticosteroids and beta-adrenergic agonists, while effective for symptom control and widely available, are linked to severe and progressive side effects, affecting long-term patient compliance. Biologic drugs, in particular peptide inhibitors and monoclonal antibodies show promise as therapeutics for chronic pulmonary diseases. Peptide inhibitor-based treatments have already been proposed for a range of diseases, including infectious disease, cancers and even Alzheimer disease, while monoclonal antibodies have already been implemented as therapeutics for a range of conditions. Several biologic agents are currently being developed for the treatment of asthma, chronic obstructive pulmonary disease, idiopathic pulmonary fibrosis and pulmonary sarcoidosis. This article is a review of the biologics already employed in the treatment of chronic inflammatory pulmonary diseases and recent progress in the development of the most promising of those treatments, with particular focus on randomised clinical trial outcomes.

show abstract

Section: Pulmonary Sarcoidosismentioning

confidence: 99%

Biologic drugs in the treatment of chronic inflammatory pulmonary diseases: recent developments and future perspectives

2023

View full text Add to dashboard Cite

show abstract

“…Next, patients on medium to high dose steroids may develop steroid withdrawal symptoms unrelated to the efficacy of the trial drug and these may confound trial results. These concerns notwithstanding, several studies in pulmonary sarcoidosis have shown statistically significant steroid-sparing ( 132 , 133 ) and it remains an important outcome to patients dealing with sarcoidosis and the toxic effects of steroids ( 5 , 21 , 22 ). Measures to limit the extent of steroid toxicities or to pro-actively manage their onset may be necessary for patients on placebo who require escalating steroid doses ( 28 ).…”

Section: Challenges Affecting Drug Trials and Drug Development In Sar...mentioning

confidence: 99%

“…Three of the studies are reported to have an unknown status, however, on further literature review, one of these studies has been completed with results published ( 131 ). An additional ten studies are reported as completed; six of these have preliminary results yet only three of these study results have been published in peer-reviewed journals ( 106 , 128 , 211 ) and one in abstract form ( 132 ). Of the 15 active and/or anticipated studies, 11 are evaluating drug molecules and four are evaluating alternative treatment approaches using already established drugs as listed in Table 1 .…”

Section: Ongoing Future and Recently Concluded Clinical Drug Trials I...mentioning

confidence: 99%

“…The study will also monitor for the development of anti-Jo1 antibodies in study participants. A recently completed phase 2 study of Efzofitimod (ATYR1923) in patients with pulmonary sarcoidosis showed that this drug was safe and well tolerated and there was no signal of increased immunogenicity or formation of anti-Jo1 antibodies (study results published in abstract form) ( 132 ).…”

Section: Studies In Pulmonary Sarcoidosismentioning

confidence: 99%

See 1 more Smart Citation

Sarcoidosis: Updates on therapeutic drug trials and novel treatment approaches

et al. 2022

View full text Add to dashboard Cite

Sarcoidosis is a systemic granulomatous inflammatory disease of unknown etiology. It affects the lungs in over 90% of patients yet extra-pulmonary and multi-organ involvement is common. Spontaneous remission of disease occurs commonly, nonetheless, over 50% of patients will require treatment and up to 30% of patients will develop a chronic progressive non-remitting disease with marked pulmonary fibrosis leading to significant morbidity and death. Guidelines outlining an immunosuppressive treatment approach to sarcoidosis were recently published, however, the strength of evidence behind many of the guideline recommended drugs is weak. None of the drugs currently used for the treatment of sarcoidosis have been rigorously studied and prescription of these drugs is often based on off-label” indications informed by experience with other diseases. Indeed, only two medications [prednisone and repository corticotropin (RCI) injection] currently used in the treatment of sarcoidosis are approved by the United States Food and Drug Administration. This situation results in significant reimbursement challenges especially for the more advanced (and often more effective) drugs that are favored for severe and refractory forms of disease causing an over-reliance on corticosteroids known to be associated with significant dose and duration dependent toxicities. This past decade has seen a renewed interest in developing new drugs and exploring novel therapeutic pathways for the treatment of sarcoidosis. Several of these trials are active randomized controlled trials (RCTs) designed to recruit relatively large numbers of patients with a goal to determine the safety, efficacy, and tolerability of these new molecules and therapeutic approaches. While it is an exciting time, it is also necessary to exercise caution. Resources including research dollars and most importantly, patient populations available for trials are limited and thus necessitate that several of the challenges facing drug trials and drug development in sarcoidosis are addressed. This will ensure that currently available resources are judiciously utilized. Our paper reviews the ongoing and anticipated drug trials in sarcoidosis and addresses the challenges facing these and future trials. We also review several recently completed trials and draw lessons that should be applied in future.

show abstract

Safety and Efficacy ATYR1923, a Novel Immunomodulator for Pulmonary Sarcoidosis: Results of a Phase 1b/2a Randomized Placebo-Controlled Trial

Cited by 2 publications

References 0 publications

Biologic drugs in the treatment of chronic inflammatory pulmonary diseases: recent developments and future perspectives

Biologic drugs in the treatment of chronic inflammatory pulmonary diseases: recent developments and future perspectives

Sarcoidosis: Updates on therapeutic drug trials and novel treatment approaches

Contact Info

Product

Resources

About