공시 • Jun 14
Rhythm Pharmaceuticals Presents Initial Data from Long-Term Extension Trial Evaluating Setmelanotide in Rare Genetic Diseases of Obesity at ENDO 2022
Rhythm Pharmaceuticals, Inc. announced new data from the Company’s long-term extension (LTE) trial, which show continued body mass index (BMI) and weight reductions in patients with SH2B1 or SRC1 deficiency obesity, or with POMC or LEPR insufficiency obesity (heterozygous). Rhythm and its collaborators delivered these data in one oral presentation and two posters at the Endocrine Society Annual Meeting & Expo (ENDO), being held June 11-14, 2022 in Atlanta. SH2B1 Deficiency Obesity: A total of 19 patients with obesity due to SH2B1 deficiency caused by a variant in the SH2B1 gene or 16p11.2 deletion encompassing the SH2B1 gene (SH2B1 deficiency obesity) who were treated with setmelanotide in the Phase 2 Basket Trial continued into the LTE trial. These data were presented in a poster, “Body Mass Index and Weight Reduction in Patients with SH2B1 Deficiency Obesity After 1 Year of Setmelanotide,” by Sadaf Farooqi, M.D., Ph.D, the Wellcome-MRC Institute of Metabolic Science at the University of Cambridge. Highlights include: Nineteen (19) patients with SH2B1 deficiency entered the LTE trial, with 19, 15, and 14 of those patients having received at least 6, 9, and 12 months of treatment as of Oct. 29, 2021, respectively. Across all patients, including those who did not achieve 5% weight loss in the Phase 2 Basket Trial but chose to enter the LTE, the mean (SD) % change in BMI was -3.4% (8.1%; n=19), -5.9% (10.0%; n=15), and -9.7% (8.0%; n=14) at 6, 9, and 12 months on therapy, respectively; Responders were defined as patients who achieved =5% weight loss at Month 3: Across responders, the mean % change in BMI was -8.8% (n=8), -9.1% (n=9) and -12.3% (n=9) at 6, 9 and 12 months on therapy, respectively; and Across non-responders, the mean % change in BMI was 0.6% (n=11), -1.1% (n=6) and -4.9% (n=5) at 6, 9 and 12 months on therapy, respectively. Seventeen (17) patients were continuing to receive setmelanotide therapy in the LTE trial as of the data cut off, while two patients had discontinued voluntarily. No patients discontinued due to adverse events. SRC1 Deficiency Obesity: A total of 17 patients with obesity due to SRC1 deficiency caused by a variant in the NCOA1 gene who were treated with setmelanotide in Rhythm’s exploratory Phase 2 Basket Trial continued into the LTE trial. These data were presented in a poster, “Body Mass Index and Weight Reductions in Patients with SRC1 Deficiency Obesity After 1 Year of Setmelanotide,” by Jesús Argente, M.D., Ph.D., Universidad Autónoma de Madrid in Spain. A deficiency in the SRC1 protein may be due to a variant on the NCOA1 gene. Highlights include: Seventeen (17) patients with SRC1 deficiency entered the LTE trial, with 18, 13 and 10 of those patients having received at least 6, 9, and 12 months of treatment as of Oct. 29, 2021, respectively. Across all patients, including those who did not achieve 5% weight loss in the Phase 2 Basket Trial but chose to enter the LTE, the mean (SD) % change in BMI was -5.7% (5.6%; n=16), -7.8% (5.8%; n=11), and -10.1% (9.4%; n=8) at 6, 9, and 12 months on therapy, respectively; Responders were defined as patients who achieved =5% weight loss at Month 3: Across responders, the mean % change in BMI was -10.1% (n=7), -10.5% (n=6) and -14.3% (n=3) at 6, 9 and 12 months on therapy, respectively; and Across non-responders, the mean % change in BMI was -2.3% (n=9), -4.7% (n=5) and -7.5% (n=5) at 6, 9 and 12 months on therapy, respectively. Fifteen (15) of 17 patients with SRC1 deficiency were continuing to receive setmelanotide therapy in the LTE trial as of the data cut off, while two patients had discontinued voluntarily. No patients discontinued due to adverse events. POMC or LEPR insufficiency obesity (heterozygous): A total of 17 patients with obesity caused by heterozygous variants in the POMC, PCSK1 or LEPR genes who were treated with setmelanotide in the Phase 2 Basket Trial continued into the LTE trial. These data were delivered orally by Brieana Buckley, PharmD, M.S., Vice President of Medical Affairs at Rhythm, in a presentation entitled, “Body Mass Index and Weight Reductions in Patients with Obesity Due to Heterozygous Variants in POMC, PCSK1, or LEPR After 1 Year of Setmelanotide.” Highlights include: Seventeen (17) patients with POMC or LEPR insufficiency obesity reached 12 months on therapy as of Oct. 29, 2021. Across all patients, the mean (SD) % change in BMI was -7.9% (7.2%; n=16), -9.0% (8.6%; n=17), and -8.7% (8.2%; n=17) at 6, 9, and 12 months on therapy, respectively; and Patients 18 years old and older (n=15) achieved a mean (SD) % change in body weight of -8.9% (6.8%; n=15), -11.7% (7.3%; n=14), and -10.2% (7.9%; n=15) at 6, 9, and 12 months on therapy, respectively; and 53.3% of patients 18 years old and older achieved greater than 10% weight loss at 12 months on therapy. Seventeen (17) patients with POMC or LEPR insufficiency obesity reached 12 months on therapy as of Oct. 29, 2021. Fifteen of those patients had enrolled in the LTE, five of those patients had discontinued voluntarily, and two patients who reached 12 months on therapy in the Basket Trial did not opt into the LTE. No patients discontinued due to adverse events. Consistent with prior clinical observations, setmelanotide was generally well tolerated in the LTE trial across all indications and no new safety signals were observed.
