Processa Pharmaceuticals will explore and further develop the anti-fibrotic, anti-inflammatory compound HT-100 as a potential treatment for rare fibrotic-related diseases in adults, including focal segmental glomerulosclerosis, scleroderma, and idiopathic pulmonary fibrosis (IPF).
The company announced that it has acquired the exclusive global rights to develop and commercialize HT-100 from its initial developer, Akashi Therapeutics.
“The Processa strategy is to add drugs to our portfolio which already have some clinical evidence of efficacy,” David Young, PharmD, PhD, CEO of Processa, said in a press release. “This enables our team to efficiently develop these drugs for patients with a high unmet medical need condition while the risk of failure associated with the clinical trials is decreased.”
“Since the quality of life for these patients is so often impaired given the lack of treatment options, the more efficiently we can develop these products, the sooner patients will experience the positive impact of these drugs on their lives,” Young added.
HT-100, or delayed-release halofuginone, is an orally available small molecule that was first discovered by Mark Pines at the Volcani Institute in Israel. Preclinical pivotal studies demonstrated that HT-100 had therapeutic potential due its ability to reduce fibrosis and inflammation, as well as to promote healthy muscle fiber regeneration.
Akashi started to assess HT-100 as a potential treatment for people with Duchenne muscular dystrophy (DMD), a rare degenerative disease that affects muscles fibers. The therapy was granted orphan drug designation in both the U.S. and the E.U., and fast-track designation in the U.S.
Preliminary results of a Phase 1b/2 trial showed promising improvements in muscle strength in non-ambulant pediatric DMD patients. However, the U.S. Food and Drug Administration (FDA) placed a clinical hold on the trial in 2016 after a serious adverse event was reported in one participant.
After a thorough data review, the FDA allowed the company to resume the trial with defined recommendations on how it should be conducted.
However, Processa now says it “will revisit potential pediatric indications, such as DMD, at a later time.”
The company instead plans to further explore HT-100 as a potential strategy to improve the survival and quality of life of those affected by rare fibrotic-related diseases, including IPF.
“Processa needs to learn more about the safety and dose response of HT-100 while we determine the best way to clinically manage patients on this anti-fibrotic drug,” said Sian Bigora, PharmD, the company’s chief development officer.
“In early 2020 we hope to begin to define, in collaboration with the FDA, an efficient way to develop HT-100 for all those patients who would benefit from this drug,” Bigora concluded.