Vivjoa, the first FDA-approved product for Mycovia, is an azole antifungal indicated to reduce the incidence of recurrent vulvovaginal candidiasis (RVVC) in females with a history of RVVC who are NOT of reproductive potential. Oteseconazole received FDA Qualified Infectious Disease Product and Fast-Track designations to become the first FDA-approved therapy for RVVC. In 2019, Mycovia licensed oteseconazole to Jiangsu Hengrui Pharmaceuticals Co., Ltd., to develop and commercialize oteseconazole in China, including mainland China, Hong Kong, Macau and Taiwan, and Gedeon Richter Plc., a Hungary-based pharmaceutical company, to commercialize and manufacture oteseconazole in Europe, Russia, the Commonwealth of Independent States, Latin America and Australia. Mycovia also recognizes a tremendous potential for its oral fungal inhibitors and a growing need to treat a range of multi-drug resistant fungal pathogens.
Camzyosis the FIRST AND ONLY cardiac myosin inhibitor approved by the FDA indicated for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms. Camzyos is an allosteric and reversible inhibitor selective for cardiac myosin. Camzyos modulates the number of myosin heads that can enter “on actin” (power-generating) states, thus reducing the probability of force-producing (systolic) and residual (diastolic) cross-bridge formation. Excess myosin actin cross-bridge formation and dysregulation of the super-relaxed state are mechanistic hallmarks of HCM. Camzyos shifts the overall myosin population towards an energy-sparing, recruitable, super-relaxed state. In HCM patients, myosin inhibition with Camzyos reduces dynamic LVOT obstruction and improves cardiac filling pressures.
Ztalmy, a neuroactive steroid that acts as a positive allosteric modulator of the GABAA receptor, is taken three times daily. Ztalmy is the FIRST ANDONLY FDA-approved treatment indicated specifically for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD) in patients two years of age and older.
PLUVICTO™ (Lutetium Lu 177 vipivotide tetraxetan)
Pluvictois a PSMA targeted radioligand therapy that delivers DNA-breaking radiation directly to the PSMA positive bone, nodal and visceral metastases. It is the FIRST AND ONLY PSMA-targeted radioligand therapy for men with PSMA+ mCRPC who have been treated with androgen receptor pathway inhibitors and a taxane-based chemotherapy.
Sickle Cell Disease (SCD) is a genetic disease that is a form of inherited anemia arising from problems with the structure and function of red blood cells. It is an inherited disease caused by a defect, a single mutation, in the beta-globin gene that helps make adult hemoglobin. Abnormal hemoglobin, called hemoglobin S, causes sickle cell disease. The signs and symptoms of sickle cell disease include swelling of the hands and feet, anemia, fatigue, extreme tiredness, jaundice, and skin discoloration. Sickle cell disease can lead to complications such as infections, delayed growth, and episodes of pain, acute chest syndrome, and stroke. According to the Centres for Disease Control and Prevention (CDC), approximately 100,000 Americans are affected by sickle cell disease. Sickle Cell Disease occurs among about 1 out of every 365 Black or African-American births. About 1 in 13 Black or African-American babies is born with sickle cell trait (SCT).
Several pharmaceutical companies such as AstraZeneca, Global Blood Therapeutics, Bluebird bio, Micelle BioPharma Inc, Novartis Pharmaceuticals, Cyclerion Therapeutics, and Imara, Inc. are developing the drugs for the treatment of sickle cell disease.
Global Blood Therapeutics (Voxelotor)
Voxelotor is an oral therapy taken once daily that directly inhibits sickle hemoglobin polymerization, which causes red blood cells (RBCs) to deform and become sickle-shaped. Voxelotor acts by binding to hemoglobin and stabilizing RBCs in an oxygenated state. This increases hemoglobin’s affinity for oxygen and inhibits polymerization and the resultant sickling and destruction of RBCs. The potential of Voxelotor to slow the progression of sickle cell disease and its devasting downstream effects across a wide range of studies, including the HOPE-KIDS 2 post-approval confirmatory study using transcranial doppler (TCD) flow velocity to evaluate the reduction in stroke risk in children 2 to 15 years of age. Voxelotor was granted Breakthrough Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA) for the treatment of patients with Sickle cell disease, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). The European Commission (EC) has designated Voxelotor as an orphan medicinal product for the treatment of patients with Sickle cell disease.
Bluebird bio (LentiGlobin /BB305/HGB-210)
LentiGlobin BB305 drug is being developed for the treatment of beta-thalassemia major and severe sickle cell disease by inserting a fully functional human beta-globin gene into the patient’s own hematopoietic stem cellsex-vivo and then transplanting those modified cells into the patient through infusion, also known as autologous stem cell transplantation.
SC411(Altemia) is the proprietary product candidate that is being developed for the treatment of Sickle Cell Disease. Altemia consists of a complex proprietary mixture of various fatty acids, primarily in the form of Ethyl Cervonate (Micelle’s proprietary blend of docosahexaenoic acid and other omega-3 fatty acids), and surface-active agents formulated using ALT specifically to address the treatment of SCD. The drug is encapsulated in a soft gelatin capsule for oral administration. Altemia is well-positioned to deliver a narrow, therapeutic dose of certain lipids directly to the membrane of red blood cells of sickle cell patients. The combination of ALT drug delivery technology and highly purified lipids may reduce VOC significantly. It has the potential to address the inflammatory symptoms of SCD and to assist in reducing sickle cell events in general. Altemia is effective in reducing the sickle cell crisis events and related mortality. The double-blind, randomized, multicenter phase II study of SC411 (Altemia) in children with sickle cell disease (SCOT Trial). The SCOT trial investigated the effect of three different doses of SC411 on selected clinical and biochemical endpoints in 67 children with SCD aged 5-17 years old.
Novartis Pharmaceuticals (Crizanlizumab)
Crizanlizumab (SEG101) is an investigational humanized monoclonal antibody blocking P-selectin mediated multicellular adhesion that is in late-stage development for the prevention of vaso-occlusive crises (VOCs) in patients with sickle cell disease (SCD). Crizanlizumab acts by binding to the molecule called P-selectin on the surface of platelets and endothelium in the blood vessels. It inhibits the interactions between endothelial cells, platelets, red blood cells, sickled red blood cells, and leukocytes.
Cyclerion Therapeutics (IW-1701/ Olinciguat)
Olinciguat is an orally administered, once-daily, vascular sGC stimulator designed for the treatment of sickle cell disease (SCD). Olinciguat has the potential to manifest various properties such as to improve local blood flow to organs, reduce vascular inflammation, and reduce the proportion of sickled cells. Olinciguat has been granted Orphan Drug Designation for Sickle Cell Disease by the U.S. Food and Drug Administration. It is currently in a Phase II study in patients with SCD, the STRONG-SCD study.
Imara, Inc. (IMR-687)
IMR-687 is an oral, once-a-day, potentially disease-modifying treatment for sickle cell disease (SCD) and β-thalassemia. IMR-687 is a highly selective, potent small-molecule inhibitor of PDE9. It acts primarily on red blood cells (RBCs). It has the potential to act on white blood cells (WBCs), adhesion mediators, and other cell types that are implicated in sickle cell disease.
Modus Therapeutics AB (Sevuparin)
Sevuparin is an innovative, proprietary polysaccharide drug with anti-adhesive, anti-aggregate, and anti-inflammatory effects due to its multimodal mechanism of action. The drug candidate has the potential to restore blood flow and prevent further microvascular obstructions in a number of diseases. Modus has completed a global Phase II study of sevuparin in hospitalized sickle cell disease (SCD) patients. The randomized, double-blinded study included 144 SCD-patients at clinical sites across Europe, the Middle East, and the Caribbean. The study compared intravenously (IV) administered sevuparin with a placebo in patients admitted to the hospital with an acute vaso-occlusive crisis (VOC) associated with SCD. The study also assessed several pain-related secondary endpoints.