ReAlta gains FDA orphan drug and fast track status for GvHD therapy

The US Food and Drug Administration (FDA) has recently accorded orphan drug designation and fast track designation to ReAlta Life Sciences’ pegtarazimod (RLS-0071) for treating steroid-refractory acute graft-versus-host disease (aGvHD). This development is a significant step in the ongoing Phase II AURORA trial (NCT06343792) exploring pegtarazimod for this condition. These FDA designations ensure ReAlta will enjoy seven years of market exclusivity for pegtarazimod upon its approval and will streamline the review process.

Acute graft-versus-host disease (aGvHD) is a severe, often fatal disorder that arises after haematopoietic stem cell transplantation. It occurs when donor immune cells attack the recipient's tissues. Typically, steroid treatments are the first line of defense, but for those who do not respond to steroids, the outlook is grim with limited therapeutic alternatives and a high risk of mortality. In the US, EU, and Japan, approximately 4,000 patients fall into this category of steroid-refractory aGvHD.

Pegtarazimod is developed from ReAlta’s proprietary EPICC peptide platform, which is inspired by the human astrovirus HAstV-1. This virus is known to cause a mild, non-inflammatory gastroenteritis by modulating the immune system. Dr. Kenji Cunnion, ReAlta's Chief Medical Officer, emphasized the FDA's decision as a validation of pegtarazimod's potential. He pointed out the novel dual mechanism-of-action of RLS-0071 as a promising solution for patients facing this severe condition.

The market for aGvHD treatments is currently dominated by several major pharmaceuticals, including Johnson & Johnson’s Imbruvica (ibrutinib), Novartis’s Jakavi or Jakafi (ruxolitinib phosphate), and Bristol Myers Squibb’s Orencia (abatacept). According to projections by GlobalData, these treatments are expected to generate significant annual sales by 2030, with Imbruvica at $2.56 billion, Jakavi at $2.25 billion, and Orencia at $3.44 billion.

ReAlta Life Sciences, based in Virginia, is not only focusing on aGvHD but also exploring pegtarazimod's potential in treating other conditions. The company has secured $20 million from Series A financing in 2021 to support its ongoing research. In addition to the AURORA trial, ReAlta is conducting Phase II trials for pegtarazimod in other indications, including treatment for newborns with hypoxic-ischemic encephalopathy (NCT05778188) and patients suffering from acute exacerbations of chronic obstructive pulmonary disease (NCT06175065).

Moreover, ReAlta concluded a Phase I trial examining RLS-0071's effectiveness against asthma (NCT05351671). They had also initiated a Phase I trial to explore its use in Covid-19-associated acute lung injury (NCT04574869), though this trial was later withdrawn due to low enrollment prospects within the study drug's expiration period, as indicated on ClinicalTrials.gov. Earlier this year, ReAlta partnered with the US National Institute of Allergy and Infectious Diseases (NIAID) to assess the potential of RLS-0071 in treating acute radiation syndrome.

In summary, ReAlta Life Sciences’ pegtarazimod shows considerable promise for treating steroid-refractory aGvHD. Supported by FDA designations and a robust pipeline of ongoing trials, ReAlta is well-positioned to make significant advancements in this crucial area of medical need.