Nohla Therapeutics, a clinical stage biopharmaceutical company focused on the development of universal donor, off- the -shelf cell therapies to treat cancer and other critical diseases, announced the European Commission (EC) has granted Orphan Drug Designation to NLA101 as an orphan medicinal product for the treatment in haematopoietic stem cell transplantation.

NLA101 is a haematopoietic stem and progenitor stem cell therapy currently being evaluated in a phase 2b multi -center, open -label randomized study (NCT01690520) of  myeloablative cord blood transplantation with or without infusion of NLA101 in patients with hematologic malignancies.

The primary endpoint of the phase 2b study is time to engraftment with an absolute neutrophil count (ANC) = 500. Secondary endpoints for this study include survival, non -relapse mortality, incidence of severity of graft versus host disease (GVHD) and other healthcare economic outcomes such as days in the hospital and rates of severe infections. Over 90% of this 160-patient trial has been enrolled.

“NLA101 has shown encouraging results when used in combination with a cord blood transplant ,” commented Katie Fanning, president and chief executive officer of Nohla. “We believe NLA101 has the potential to make a meaningful difference for patients with hematologic malignancies who are candidates for an allogeneic transplant and we look forward to reporting results from the phase 2b randomized trial later this year.”

Orphan drug designation from the EC provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or very serious conditions that affect no more than 5 in 10, 000 people in the European Union (EU), and where there is an unmet medical need . In addition to a 10-year period of marketing exclusivity in the EU upon product approval, orphan drug designation provides fee waivers, protocol assistance, and marketing authorization under the centralized procedure in all EU countries.