FDA Grants Medivir Rare Pediatric Disease Designation for MIV-711 and Orphan Drug Designation for the Treatment of Legg-Calvé-Perthes Disease

Stockholm, April 25, 2024 /PRNewswire/—— Mediville AB (NASDAQ: MVIR) (STO: MVIR)is a pharmaceutical company focused on developing innovative cancer treatments in areas of high unmet medical need, today announced that its selective cathepsin K inhibitor MIV-711 has received Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) for the treatment of Legg-Calvé-Perthes disease (LCPD).

The FDA defines a rare pediatric disease as a serious or life-threatening condition that primarily affects individuals from birth to 18 years of age.Pediatric disease considered 'rare' affects fewer than 200,000 people USA. With RPDD, MIV-711 is eligible for expedited review, and the company may be eligible for an FDA priority review voucher upon LCPD's marketing approval. This coupon can be redeemed to receive priority review for any subsequent marketing applications, or it can be sold to another company for their use.

– “LCPD is a disorder that has a significant impact on daily life and carries the risk of long-term sequelae for which there is currently no effective treatment. Children with LCPD are more likely to suffer from obesity and depression due to being forced to remain immobile. For this disease, in which more than half of children have affected legs that are shorter than their healthy legs and approximately 50% of children end up with hip joint deformity that eventually develops osteoarthritis, we are excited that MIV-711 may be the first to be approved. An approved treatment option, we believe collaboration is an attractive way to advance MIV-711 to ensure speed of development.

In order to receive a RPDD, there must be supporting data showing that the drug may be effective against the disease. MIV-711 was shown to prevent femoral head deformity and positively affect bone degradation biomarkers in LCPD-specific animal models without negatively affecting normal bone formation. Likewise, clinical studies have shown that MIV-711 prevents bone degradation in patients with osteoarthritis, further supporting the potential clinical benefit of LCDP.

Cathepsin K is a major cysteine ​​protease that participates in bone resorption and cartilage degradation by osteoclasts by breaking down key bone matrix proteins. Cathepsin K inhibition can protect damaged bone by reducing bone resorption and promoting bone formation, thus addressing the key mechanisms causing pathological changes in LCPD, potentially minimizing long-term negative effects. Selective inhibition of cathepsin K has the potential to provide clinical benefit in diseases characterized by excessive bone resorption, including other childhood skeletal disorders.

For more information please contact:Magnus ChristensenChief Financial Officer, Medivir AB
Telephone: +46 8 5468 3100.
Email: magnus.christensen@medivir.com

About Legg-Calvé-Perthes Disease

Legg-Calvé-Perthes disease (LCPD) is a childhood hip disorder caused by disruption of blood flow to the femoral head. Due to lack of blood flow, the bone dies (osteonecrosis or avascular necrosis) and stops growing. Over time, healing occurs as new blood vessels penetrate and remove the dead bone, causing bone loss and weakness of the femoral head. This condition most commonly occurs in children ages 4 to 8, but can also occur in children ages 2 to 15. USA. It causes permanent deformity of the femoral head in many children, increasing the risk of osteoarthritis in adults.

About MIV-711

MIV-711 is a potent and selective inhibitor of cathepsin K, the major protease involved in breaking down collagen in bone and cartilage. It has been shown to slow, stop or reverse the progressive degeneration of joints affected by osteoarthritis. By inhibiting cathepsin K and increased/excessive osteoclast activity, MIV-711 has the potential to prevent bone and cartilage degradation, counteract osteonecrosis caused by insufficient blood flow, prevent femoral head deformity and long-term negative effects, and positively impact quality of life. .

About Mediville

Medivir develops innovative medicines focused on cancers with high unmet medical need. Drug candidates target indication areas where available therapies are limited or missing and have a strong opportunity to provide significant improvements to patients. Medivir is focused on developing fostroxacitabine brapamide (fostrox), a smart targeted chemotherapy drug designed to selectively treat liver cancer cells and minimize side effects. Collaborations and partnerships are an important part of Medivir's business model, and drug development is conducted by Medivir or partners. Medivir's shares (ticker symbol: MVIR) are listed on Nasdaq SmallCap Stockholm. www.medivir.com.

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