Rhythm Pharmaceuticals, Inc. (RYTM) is a biopharmaceutical company focused on the development and commercialization of therapeutics for rare genetic disorders of obesity. It is currently evaluating the efficacy and safety of setmelanotide, the company’s first-in-class MC4R agonist in Phase 3 studies, in patients who suffer from POMC deficiency obesity, LEPR deficiency obesity, BBS and Alström Syndrome.
It is obvious that this firm has taken advantage of the advancement in genomics to improve the understanding of severe obesity that results from specific genetic disorders.
Among Rhythm’s tasks is opening the minds of obese people to understanding the reasons behind their insatiable hunger; helping the obese to learn about the genetic causes of their obesity and guiding them towards understanding their disease, while giving them hope for a treatment that targets the pathway of their obesity.
Having said that, we inform that the Rhythm has just announced it will outline its expected 2019 milestones and review updated clinical data from its Phase 2 basket studies using setmelanotide on patients with BBS in a presentation at the 37th Annual J.P. Morgan of Healthcare Conference on Thursday, January 10, 2019.
Setmelanotide is a first-in-class melanocortin-4 receptor (MC4R) agonist.
Rhythm Chief Executive Office Expectations
Keith Gottesdiener, M.D., Chief Executive Officer of Rhythm expects 2019 to be an important year for Rhythm regarding the advancement of setmelanotide and the expected submission of the firm’s first NDA for POMC and LEPR deficiency obesity. Regarding 2018 achievements which led to the man’s great expectations. Dr. Gottesdiener cited the completion of the pivotal enrollment in Phase 3 trials in POMC and LEPR deficiency obesity, the initiation of additional pivotal trials in BBS and Alström Syndrome and the building of an integrated patient community. These achievements, he said, brought the firm closer to providing a first-in-class therapeutic option for people living with rare genetic disorders of obesity.
Dr. Gottesdiener reminded that these people have no approved treatments for reducing hunger or body weight.
Updated BBS Data from Phase 2 Basket Studies and Pivotal Phase 3 Trial Design
Rhythm announced updated clinical data from two adolescent patients in its Phase 2 basket studies evaluating setmelanotide for the treatment of BBS who previously had only short-term results (18 & 15 weeks of total treatment). New data is now available for 47 & 41 weeks of treatment where these patients have lost 11.2% & 15.5% of their body weight and experienced hunger score reductions of 66% & 21%, respectively.
Treatment with setmelanotide continues to be well tolerated and safety data were consistent with previous clinical studies. In total, six out of the nine BBS patients enrolled in the Phase 2 basket studies have now achieved a clinically meaningful weight loss of 10% change from baseline, which is the primary endpoint for the company’s pivotal Phase 3 clinical trial.
One additional patient with Type-1 diabetes responded with marked improvements in hunger score and blood sugar levels.
In December 2018, Rhythm began treating patients in a combined pivotal Phase 3 clinical trial evaluating setmelanotide in BBS and Alström Syndrome and expects to complete pivotal enrollment of at least 20 patients with BBS and at least 6 patients with Alström Syndrome in the second half of 2019.
Progress in Phase 3 Clinical Trials in POMC and LEPR Deficiency Obesity
The ongoing, separate pivotal Phase 3 clinical trials evaluating setmelanotide in POMC and LEPR deficiency obesity continue to progress, with topline data expected in the third quarter of 2019.
Pending positive results, the company expects to submit an NDA filing for each of POMC and LEPR deficiency obesity to the U.S. Food and Drug Administration (FDA) in late 2019 or early 2020.
Additional Development Pipeline Updates
Rhythm continues to evaluate setmelanotide for the treatment of additional rare genetic disorders of obesity in its Phase 2 basket studies, including, POMC and other MC4R pathway deficiency heterozygous obesities, as well as POMC epigenetic disorders.
The company expects to announce updated interim data from these disorders in the first quarter of 2019.
In addition, Rhythm is continuing to expand the evaluation of setmelanotide into additional MC4R pathway disorders in its Phase 2 basket studies.
Rhythm will webcast its corporate presentation from the 37th Annual J.P. Morgan Healthcare Conference in San Francisco on Thursday, January 10, 2019, at 10:00 a.m. PST, 1:00 p.m. EST. Live webcasts of the presentation and subsequent breakout session can be accessed under “Events & Presentations” in the Investors & Media section of the company’s website at www.rhythmtx.com. A replay of the webcasts will be available on the Rhythm website for 30 days.
To assess the value of the Rhythm Pharmaceutical treatment of obesity, in these trials, one has to understand the drug the firm is using to treat the obesities in the clinical trial mentioned above.
The product used, Setmelanotide, is a first-in-class MC4R agonist.
Why this drug?
Setmelanotide activates MC4R, which is part of the biological pathway that independently regulates energy expenditure and appetite. Genes within the MC4R pathway are associated with persistent hunger and severe, early-onset obesity. Rhythm is currently developing setmelanotide as a replacement therapy for patients with monogenic defects upstream of MC4R.
There are no effective or approved therapies for these patients, which led the FDA to grant Breakthrough Therapy designation to setmelanotide for obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway. This includes POMC deficiency obesity, LEPR deficiency obesity, BBS and Alström Syndrome.
The European Medicines Agency has also granted Priority Medicines (PRIME) designation for setmelanotide for obesity and for the control of hunger associated with deficiency disorders of the MC4R pathway.
Bottomline: The product is expected to be an effective replacement therapy. Until today, we have not heard any bad news from any of the trials using setmelanotide in treating various genetic-derived diseases. We saw no reason for skepticism or a stock selloff.