Rhythm Pharmaceuticals Announces Completion of Screening for Enrollment in Setmelanotide Phase 3 Hypothalamic Obesity Trial and Additional Updates

-- Phase 3 hypothalamic obesity top-line data expected in 1H2025 --

-- Positive reimbursement decision achieved for IMCIVREE® (setmelanotide) to treat patients with Bardet-Biedl syndrome and POMC/LEPR deficiencies in Spain --

-- IND application for RM-718 accepted by the FDA --

BOSTON, Jan. 04, 2024 -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, provided a business update and announced it has completed screening for enrollment in the pivotal, Phase 3 clinical trial evaluating setmelanotide in patients with acquired hypothalamic obesity.

Rhythm closed screening and anticipates overenrolling the ongoing phase 3 hypothalamic obesity trial with more than 140 patients consented and going through active screening and baseline assessments. In this trial, patients with acquired hypothalamic obesity aged 4 years or older will be randomized 2:1 to setmelanotide therapy or placebo for a total of 60 weeks, including up to eight weeks for dose titration. The Company expects to obtain top-line study results in H1 2025.

“2023 was a truly transformational year for Rhythm, as we made excellent progress with the execution of our IMCIVREE® (setmelanotide) commercial plan while also advancing important development efforts to expand the number of patients living with hyperphagia and severe obesity that could potentially benefit from this therapy,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. “We believe the rapid over enrollment in less than one year and execution of our Phase 3 hypothalamic obesity trial is a strong indicator of the unmet need for an effective therapy for patients who have no approved therapeutic options and the community’s excitement around setmelanotide’s potential to make a meaningful difference in their lives.”... Rhythm Pharmaceuticals' Press Release -

Keros Therapeutics to Develop KER-065 for the Treatment of Obesity

Keros commenced a randomized, double-blind, placebo-controlled, two-part Phase 1 clinical trial to evaluate single and multiple ascending doses of KER-065 in healthy volunteers

Keros expects to report initial data from this Phase 1 clinical trial in the first quarter of 2025

Preclinical data showed potential proof-of-mechanism of KER-065 for the treatment of obesity

Keros believes these preclinical data support developing KER-065 for the treatment of obesity, and Keros plans to initiate a proof-of-concept trial of KER-065 in obese patients following completion of this Phase 1 clinical trial

LEXINGTON, Mass., Jan. 03, 2024 - Keros Therapeutics, Inc. (“Keros” or “we”) (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta (“TGF-ß”) family of proteins, announced that it plans to develop KER-065, a novel ligand trap designed to bind to and inhibit TGF-ß ligands, including myostatin (GDF8) and activin A, for the treatment of obesity.

“Obesity is a complex and chronic disease associated with numerous comorbidities and a growing prevalence in patients. We believe there is a need for additional treatment options, including one that leads to weight loss without an associated loss of muscle and a potential for frailty. Based on our preclinical data, we believe that KER-065 has the potential to treat obesity without those limitations, by increasing skeletal muscle, reducing fat mass through an increase in energy expenditure, improving insulin sensitivity and improving cardiac function,” said Jasbir S. Seehra, Ph.D., President and Chief Executive Officer of Keros. “To that end, we recently commenced our Phase 1 clinical trial evaluating KER-065 in healthy volunteers and, following its successful completion, plan to initiate a proof-of-concept trial of KER-065 in obese patients.”... Keros Therapeutics' Press Release - 

Resalis Therapeutics Raises €10 Million Series A to Complete First Clinical Trial for RES-010 in Obesity

January 04, 2024- TORINO, Italy- Resalis Therapeutics announced the closing of a €10 million ($11 million) Series A financing round led by Sunstone Life Science Ventures with participation from existing investors including Claris Ventures and angel investors. The proceeds will be used to initiate and complete the first-in-human Phase 1 clinical trial and reach Phase 2 readiness for Resalis’ lead program, RES-010, in obesity. RES-010 is a non-coding RNA-based compound designed to provide a disease-modifying approach in obesity with longer-lasting weight reduction and the ability to extend treatment durability in combination with approved therapeutics, such as GLP-1 receptor agonists. In conjunction with the financing, Claus Andersson, PhD, General Partner of Sunstone Life Science Ventures, will join Resalis’ Board of Directors.

“The successful close of our Series A is an important milestone for Resalis because it will enable us to reach the clinic and to achieve the next significant inflection point with our lead candidate, RES-010. With RES-010 we aim to provide a therapeutic that precisely reduces fat mass in obesity with the potential to extend the durability of existing therapies. We value the trust and support of our new and current investors, and eagerly look forward to this next phase of development,” said Alessandro Toniolo, CEO of Resalis Therapeutics.

“Resalis’ therapeutic approach is based on groundbreaking science that has rapidly led to the development of a lead candidate, now on the cusp of clinical evaluation. Preserving muscle mass is key to provide a sustained and meaningful clinical benefit, and we share the team’s vision that this non-GLP1/GIP therapeutic will significantly improve both effect and tolerability of today’s therapies. This investment underscores our belief in the company’s vision and the transformative potential of its lead candidate to make a meaningful impact for obese patients,” said Claus Andersson, General Partner at Sunstone Life Science Ventures.

Pietro Puglisi, Managing Partner at Claris Ventures and Chairman of the Board at Resalis Therapeutics, added: “In recent years, there has been remarkable progress in the treatment of metabolic disorders. Yet, there persists a critical need for innovative solutions that can both provide orthogonal therapeutic effects and pave the way for combination therapies. I look forward to the continued collaboration with the outstanding Resalis team and welcoming Claus as a new member of the Resalis Board.”

Resalis has a deep understanding of the field of ncRNAs and RNA-targeted therapeutics in human health and metabolic disorders. The company’s lead candidate, RES-010, is an antisense oligonucleotide that targets miR-22, a central player in the regulation of lipid metabolism and energy consumption. In multiple proof-of-concept studies in large and small animal models, RES-010 has shown the potential to provide a safe and longer-lasting therapeutic effect, alone or in combination with approved drugs. Resalis plans to initiate a Phase 1 clinical study evaluating the safety and efficacy of RES-010 in the first half of 2024. RES-010 is patent-protected in the US, Japan and China with patents pending in the EU... Resalis Therapeutics' Press Release [PDF] -