Pegtarazimod is being studied as a potential treatment to aGvHD patients after haematopoietic stem cell transplant where steroid therapy is ineffective. Image: Shutterstock / Gorodenkoff.

The US Food and Drug Administration (FDA) has granted an orphan drug designation and fast track designation to ReAlta Life Sciences’ lead candidate pegtarazimod (RLS-0071) to treat steroid-refractory acute graft-versus-host disease (aGvHD).

These designations come as the company continues its open label Phase II AURORA trial (NCT06343792) with pegtarazimod in this indication. The FDA designations will grant ReAlta seven years of market exclusivity for pegtarazimod upon approval and expedite the review process.

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aGvHD is an often fatal complication following haematopoietic stem cell transplantation in which donor immune cells attack the recipient’s tissue. Standard treatment involves steroid regimens, but patients who are unresponsive to steroid therapy face limited further options and a high risk of mortality. There are an estimated 4,000 such patients in the US, EU, and Japan.

Pegtarazimod is based on ReAlta’s EPICC peptide platform. The technology is based on the human astrovirus HAstV-1, which causes non-inflammatory, self-limiting gastroenteritis by inhibiting components of the immune system.

Dr. Kenji Cunnion, chief medical officer at ReAlta, said the FDA decision underscored, “the significant potential of RLS-0071 and its novel dual mechanism-of-action to address critical unmet needs for patients with this life-threatening condition”.

If successfully developed and approved, pegtarazimod is set to compete in a market inhabited by Johnson & Johnson’s Imbruvica (ibrutinib), Novartis’s Jakavi or Jakafi (ruxolitinib phosphate), and Bristol Myers Squibb’s Orencia (abatacept) among others. These therapies are projected by GlobalData to generate $2.56bn, $2.25bn, and $3.44bn in annual sales by 2030, respectively.

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The Virginia-based ReAlta is currently investigating their lead therapeutic candidate’s application in a variety of indications, fuelled in part by $20m raised in Series A financing in 2021. Alongside the AURORA trial, the company is conducting Phase II trials to determine the efficacy of pegtarazimod in newborns with hypoxic-ischemic encephalopathy (NCT05778188) and in patients with acute exacerbations of chronic obstructive pulmonary disease (NCT06175065).

ReAlta also completed a Phase I trial to investigate the therapeutic application of RLS-0071 against asthma (NCT05351671) and was conducting a Phase I trial exploring the drug’s use in Covid-19-associated acute lung injury (NCT04574869) before the trial was withdrawn due to the “low probability of enrolling patients prior to expiry of study drug”, as per ClinicalTrials.gov. Earlier this year, the company collaborated with US National Institute of Allergy and Infectious Diseases (NIAID) to evaluate the drug’s use in acute radiation syndrome.