Two drugs face trials
Mesoblast Ltd., a Melbourne, Australia-based company, has a new drug in phase 2 trials. It relies on proprietary allogeneic mesenchymal precursor cells with immunosuppressive and tissue repair properties.
CEO Silviu Itescu says the medication is the result of a high number of patients who are resistant to or suffer major side effects from anti-TNF medications—about one-third of the 30% of patients using the drugs. “We saw a need for a safe effective agent,” he says.
The new drug, he says, targets multiple pathways—TNF, IL6 and IL17 (inducers of inflammation) and switches off the disease at the core. It has an advantage because it is derived from a single donor, generating a consistent, uniform and reproductible product.
The Mesoblast product has been tested among 48 patients in a 12-week trial comparing two different doses and a placebo. The results indicate improvement in function for those on the drug and proof that higher doses are safe, effective, and durable, Itescu says. Another trial ran for 52 weeks comparing a single dose with a placebo and resulted in a lower disease activity score over placebo.
Itescu anticipates moving into phase 3 trials by the end of the year.
Another drug in trial is evobrutinib, a Bruton tyrosine kinase inhibitor (BTKi) from Merck KGaA, Darmstadt, Germany. The oral small molecule therapy is currently in phase 2b studies and is targeting multiple immunological indications including RA, lupus, and multiple sclerosis.
BTK inhibition is thought to suppress key immune cells, which preclinical research suggests may be therapeutically useful in certain autoimmune diseases.
Alise Reicin, head of global clinical development, Merck KGaA, says that evobrutinib represents one of the largest clinical development programs established for an investigational BTK inhibitor.
Early data have provided encouraging results to support continued development, including a study in mouse models of RA that displayed robust efficacy with a marked reduction in disease severity.
Results from a small phase 2a, signal-generating study met the predefined criteria for progressing to a dose-finding study; however, the study did not reach the statistical criteria across all patients enrolled, but there was a clear positive outcome in the pre-specified patient population conventionally studied across RA pivotal trials.
The estimated primary completion date for evobrutinib is March 2019 for RA. Launch timing is dependent on the design and extent of phase 3 studies, Reicin says.
She says it is premature to determine pricing for the new drug and whether payers would elect to put it on formulary. “We believe in value-based pricing, and Merck KGaA, Darmstadt, Germany, has an ongoing partnership with payers and providers to ensure fast and broad access to its treatments, as well as patient support programs to help assist patients in need.” Reicin says.
Huntsman says the same thing about Sanofi. “Regeneron and Sanofi have a shared goal with patients, physicians and payers to reduce the burden of RA and an ongoing commitment to continuing to work with payers/insurers to provide access to Kevzara,” she says.
The U.S. list price (wholesale acquisition cost) of Kevzara is $39,000/year for the 200 mg and 150 mg doses. “The price of the drug reflects the clinical value that it provides as a treatment for RA, and the list price is comparable to those of other biologics in the RA marketplace,” Huntsman says. â¨
Mari Edlin, a frequent contributor to Managed Healthcare Executive, is based in Sonoma, California.