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Biopharma Co. Posts 17% BMI Reduction in Ph. 2 Trial
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Shares of Rhythm Pharmaceuticals Inc. rose by over 30% after the company posted positive interim results from its Phase 2 clinical study of setmelanotide in hypothalamic obesity. The data shows that after 16 weeks of treatment with setmelanotide, patients experienced a 17.2% mean reduction in BMI and a mean weight loss of 15.8%.

Commercial-stage biopharma firm Rhythm Pharmaceuticals Inc. (RYTM:NASDAQ), which concentrates its work on developing and commercializing treatments for patients living with hyperphagia and severe obesity resulting from rare melanocortin-4 receptor (MC4R) pathway diseases, yesterday announced "positive interim results from a Phase 2 clinical trial evaluating setmelanotide, the company's MC4R agonist, for the treatment of severe obesity and hyperphagia in people living with hypothalamic obesity."

The company advised that based upon the highly encouraging results gathered in the study, it is now planning to consult with various regulatory agencies in order to move forward into Phase 3 clinical trials.

An investigator in the Phase 2 trial, M. Jennifer Abuzzahab, M.D. of the McNeely Pediatric Diabetes Center and Endocrine Clinic at Children's Minnesota, remarked, "Therapeutic options are very limited for patients with hypothalamic obesity, which is the result of damage to the medial hypothalamic region of the brain where MC4R pathway signaling is impaired due to certain tumors and their treatment. Surgical intervention, radiation treatment or even the growth and position of the tumor itself can leave patients with aggressive, rapid and debilitating weight gain."

Dr. Abuzzahab added, "I believe these interim results for setmelanotide represent a major milestone for the hypothalamic obesity community. In this 16-week trial, patients receiving setmelanotide achieved a remarkable reduction in body weight, suggesting this precision therapy has the potential to transform the care of this rare, acquired obesity."

Rhythm Pharmaceuticals noted that the data released pertains to 11 patients in the Phase 2 trial who met the parameters for assessment as of the data cutoff date of May 6, 2022. Of these 11 patients, 9 completed the full 16-week program and 2 patients discontinued treatment early due to treatment-related adverse events.

The company advised that the interim results were positive with treated patients demonstrating a 17.2% average (mean) reduction in BMI with decreases ranging from 6.7% to 37.2%. Participants additionally achieved a 15.8% average (mean) loss in body weight, equating to an average (mean) loss of 35.1 lb. from baseline weight. The firm pointed out that in the study setmelanotide also produced meaningful overall improvements in hunger scores.

The company's Chair, President and CEO David Meeker, M.D. commented, "We are highly encouraged by these initial results, which reinforce the importance of the MC4R pathway in regulating hunger, caloric intake, energy expenditure and ultimately body weight, as well as the potential role of setmelanotide in the management of diseases where this pathway is impaired…We look forward to engaging with regulatory agencies to finalize our Phase 3 development plans in the months ahead, as we aim to expand our reach and make setmelanotide available to the approximately 5,000 to 10,000 people living with hypothalamic obesity in the U.S."

The company indicated that the Phase 2 setmelanotide study is a multi-center clinical trial which included 18 pediatric and adult patients between the ages of 6 and 28 years old who were identified as having hypothalamic obesity. Patients in the study were given a once daily injection of setmelanotide over a period of 16 weeks. The firm advised that the trial's primary endpoint was to measure "the percentage of patients who achieve more than 5% percent reduction in BMI from baseline after 16 weeks of treatment compared to a historic control of less than 5% in this population."

The company explained that "IMCIVREE (setmelanotide) is a melanocortin-4 receptor (MC4R) agonist, which is a part of the key biological pathway that regulates hunger, caloric intake and energy expenditure." Rhythm is actively developing setmelanotide as a targeted therapy with the objective of restoring impaired MC4R pathway functioning and believes that by doing so, will unlock the ability of patients with rare genetic diseases of obesity to raise energy expenditure, reduce hunger and ultimately lose weight.

The firm described hypothalamic obesity as "a rare, acquired form of extreme obesity that occurs following damage to the hypothalamic region of the brain, which is responsible for controlling physiological functions such as hunger and weight regulation." The condition frequently arises subsequent to growth or surgical removal of certain types of brain tumors. Patients with hypothalamic obesity typically experience rapid increases in weight caused by a decrease in energy and an increase in hunger during the first 6-12 months after surgery to remove the tumors. The company stated that presently there are about 5,000 to 10,000 individuals in the U.S, who are living with the disease.

Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical firm headquartered in Boston, Mass. The company focusses on developing and commercializing therapies for treating rare genetic obesity-related diseases such as hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases.

The company advised that its leading precision medicine, IMCIVREE (setmelanotide), has been approved by the U.S. Food and Drug Administration (FDA) for use in chronic weight management in adults and children six years of age and older who have been diagnosed with "monogenic or syndromic obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS)." The firm claims that by using its Rhythm Engine, it has assembled the largest known obesity DNA database which contains over 45,0000 sequencing samples from individuals with severe obesity. The company noted that it has collected this data to aid in its efforts to improve understanding, diagnosis, patient care and potential treatments for rare genetic obesity-related deficiencies.

Rhythm Pharmaceuticals started the day Wednesday with a market cap of around $336.1 million with ~50.4 million shares outstanding and a short interest of about 6.3%. RYTM shares opened 5% lower at $6.34 (-$0.33, -4.95%) from the previous day's $6.67 closing price. The stock traded between $6.34 and $8.8799 per share and closed for trading at $8.77 (+$2.10, +31.48%).

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