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Rhythm PHARMACEUTICALS Rhythm Pharmaceuticals Reports Second Quarter 2021 Financial Results August 3 , 2021 -- Received European Commission authorization of IMCIVREE for treatment of obesity and control of hunger associated with POMC , PCSK1 and LEPR deficiencies -- -- Setmelanotide selected for evaluation as a " Highly Specialised Technology " by Great Britain's National Institute for Health and Care Excellence -- On track to submit sNDA to FDA and MAA Type Il amendment to EMA for setmelanotide in BBS and Alström syndromes in 2H 2021 -- -- Five new Phase 2 and 3 clinical trials planned to initiate in 2H 2021 -- -- Management to host conference call at 8 a.m. ET -- BOSTON , Aug. 03 , 2021 ( GLOBE NEWSWIRE ) -- Rhythm Pharmaceuticals , Inc. ( Nasdaq : RYTM ) , a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity , today reported financial results and provided a business update for the second quarter ended June 30 , 2021 . " We have made tremendous progress in the second quarter towards our goal of transforming the care of patients with rare genetic diseases of obesity globally , " said David Meeker , M.D. , Chair , President and Chief Executive Officer of Rhythm . " We are pleased with our first full quarter of IMCIVREEⓇ ( setmelanotide ) commercial availability in the United States with positive engagements with patients , prescribers and payors . We recently secured European Commission marketing authorization for the treatment of obesity and the control of hunger associated with genetically confirmed loss - of- function biallelic POMC , including PCSK1 , deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above , enabling us to expand patient access to IMCIVREE . From a market access standpoint , we are encouraged that regulatory authorities , such as National Institute for Health and Care Excellence ( NICE ) in the United Kingdom , recognize these obesities as rare genetic diseases for which there are no available treatment options . " Dr. Meeker continued , “ In parallel , we are executing on our clinical development and regulatory strategy to bring setmelanotide to substantially more patients suffering from rare genetic diseases of obesity . We look forward to completing supplementary regulatory submissions in the second half of this year to both the U.S. Food and Drug Administration ( FDA ) and European Medicines Agency ( EMA ) , seeking marketing authorization for setmelanotide for Bardet - Biedl and Alström syndromes , and we are excited to continue advancing our broad clinical development program in additional patient populations . With agreement from both the FDA and EMA , we are poised to initiate five clinical trials of setmelanotide : the pivotal Phase 3 EMANATE trial with five sub - studies in heterozygous POMC , PCSK1 or LEPR deficiency obesities and SRC1 and SH2B1 deficiency obesities , the Phase 2 DAYBREAK trial in 31 additional genes each with strong or very strong ties to the MC4R pathway , as well as a Phase 3 pediatrics trial for children younger than 6 and two registrational trials for our weekly formulation of setmelanotide . Taken together , we believe these efforts may enable us to help many more people with rare genetic diseases of obesity with a potential treatment for their insatiable hunger or hyperphagia and early - onset , severe obesity . " Second Quarter and Recent Business Highlights : Pipeline and Business Developments : POMC and LEPR Deficiency Obesities : • In July 2021 , Rhythm announced that the European Commission ( EC ) granted marketing authorization to IMCIVREE for the treatment of obesity and the control of hunger associated with genetically confirmed loss - of - function biallelic proopiomelanocortin ( POMC ) , including PCSK1 , deficiency or biallelic leptin receptor ( LEPR ) deficiency in adults and children 6 years of age and above . • In Great Britain , Rhythm's marketing authorisation application for IMCIVREE is under review by the Medicines & Healthcare Products Regulatory Agency ( MHRA ) . The Company today announced that IMCIVREE has been selected for evaluation as a " Highly Specialised Technology " ( HST ) by the National Institute for Health and Care Excellence ( NICE ) . HST is a specific status reserved for rare and severe diseases . Bardet - Biedl Syndrome and Alström Syndrome : • Today , Rhythm provided an update on its regulatory strategy for setmelanotide for the treatment of Alström syndrome . Based on feedback from both the FDA and EMA , Rhythm now plans to submit a supplemental New Drug Application ( SNDA ) to the FDA and Type II variation marketing authorization application ( MAA ) to the EMA in the second half of 2021 , which will cover both BBS and Alström syndrome . • Also in July 2021 , Rhythm announced a collaborative research agreement with the Clinical Registry Investigating Bardet - Biedl Syndrome ( CRIBBS ) to initiate a population study focused on the natural history of weight gain , hyperphagia and quality of life in patients with BBS . Additional Clinical Development Updates :