Golike is being commercialized for the dietary management of phenylketonuria (PKU), a rare inherited disorder affecting approximately 350,000 patients in the world’s key markets.  PKU is caused by a defect of the enzyme needed to break down phenylalanine, leading to a toxic buildup of phenylalanine when eating foods that contain protein or aspartame that can eventually lead to serious health problems. Excessive levels of phenylalanine in the blood cause accumulation in the brain, which hampers proper brain development and results in neurophysiological dysfunction. For the rest of their lives, people with PKU — from infancy through adulthood — need to follow a diet that limits phenylalanine.

Patients with PKU require supplementation of amino acid-based foods for special medical purposes (FSMP) to prevent protein deficiency and optimize metabolic control. Many of these FSMPs can result in poor dietary compliance due to their taste and odor. Furthermore, the unpleasant odor and aftertaste of current amino acid supplements can become a barrier to social interaction for PKU patients.

Golike is the first controlled-release amino acid mix product with effective taste and odor masking. With these characteristics, Golike is a uniquely differentiated product, offering improved metabolic management and better compliance for PKU patients of all age groups. In the U.S., Golike has been granted Orphan Drug Designation, and Relief intends to assess options to pursue approval of Golike as a prescription product. In various European  countries, Golike is available as a fully reimbursed treatment option for PKU patients.

Relief is planning to expand the Golike commercial infrastructure beyond the current countries where APR is present and aims to strengthen the commercial  activities to increase and accelerate future growth.

APR’s physiomimic technology is a proprietary, patented drug delivery technology to control and prolong the release of multiple active ingredients simultaneously. Beyond Golike, APR is developing optimized amino acid mix-based products for other rare metabolic disorders, such as tyrosinemia, homocystinuria and maple syrup urine disease (MSUD). For MSUD, such a product is expected to be highly complementary to Relief’s ACER-001, which is in development for treatment of this disease and possesses effective taste-masking properties.

The APR acquisition brings to Relief a pipeline of product candidates at various stages of development.

A promising example of a program for potential further clinical development is APR-TD011 for the treatment of epidermolysis bullosa (EB). EB is a group of rare, genetic, life-threatening connective tissue disorders characterized by skin blistering throughout the body and the risk of severely impacting internal organs. There are an estimated 250,000 patients with EB worldwide, with an estimated 30,000 patients in the European Union (EU) and 20,000 patients in the U.S.

APR TD011 is a sprayable solution that combines strong antimicrobial action with anti-inflammatory properties. APR TD011 is designed to be a complete treatment for EB patients to prevent or reduce infections and inflammation through modulation of the wound microenvironment to support a faster physiological wound healing. The product was granted Orphan Drug Designation in late 2019 by the U.S. FDA.

In a preliminary clinical trial, EB patients administered with APR TD011 demonstrated improvement in skin blistering and tissue repair within just two weeks of treatment, and the product candidate was shown to be well tolerated with a favorable safety profile. Relief plans to discuss next development steps with regulatory authorities in the near future, with the goal of initiating a Phase 2 proof-of-concept study quickly thereafter.

Relief is carefully evaluating the entire APR clinical development pipeline and will focus on advancing the development of those assets and indications that offer an optimal strategic fit combined with differentiated product treatment benefits  that offer strong growth potential. 

Beyond the programs that Relief will commercialize internally, the APR pipeline also provides promising value-added out-licensing opportunities. One example is Sentinox, an intranasal spray containing a well-known, safe and effective antiviral which can block transmission of the SARS-CoV-2 virus from the respiratory tract and limit or reduce the effects of COVID-19. Sentinox has already received EU marketing clearance.