Rhythm Pharmaceuticals, Inc. (NASDAQ: RYTM) has announced a successful third quarter in 2024, marked by significant revenue growth attributed to increased sales of IMCIVREE for Bardet-Biedl Syndrome (BBS). The company's earnings call on November 5, 2024, highlighted financial performance, clinical trial advancements, and strategic initiatives, with a 48% year-over-year increase in global net revenue, reaching $33.3 million. The company anticipates sufficient cash to fund operations into 2026.
IMCIVREE Drives Revenue Growth
IMCIVREE, Rhythm's key drug, continues to be a major revenue driver. In Q3 2024, global net revenue from IMCIVREE reached $33.3 million, a 48% increase from Q3 2023. The U.S. contributed $23.3 million, representing 70% of total sales, while international revenue grew to $10 million, a 35% increase from the previous quarter. IMCIVREE is now available in over 15 countries, with significant contributions from France and Germany. A successful BBS launch is expected in the UK in Q4, following a NICE recommendation.
Advancing Clinical Programs
Rhythm is actively advancing its clinical programs. The company expects to complete enrollment in a Phase II trial for bivamelagon and a Phase I trial for RM-718 in early 2025. Topline data from a Phase III trial for acquired hypothalamic obesity is expected in the first half of 2025. Early access programs for hypothalamic obesity in France and Italy have shown positive outcomes. The company is optimistic about completing enrollment in various clinical trials by year-end and starting dosing for new indications in early 2025.
IMCIVREE Label Expansion
A PDUFA date for expanding IMCIVREE's label to include children ages two to six is set for December 26, 2024. The European Commission has already expanded the marketing authorization for IMCIVREE to include children as young as two years of age. The FDA has accepted with priority review Rhythm's sNDA for the same expansion.
Financial Performance
Rhythm reduced its 2024 operating expense guidance to $245-$255 million and reported $298.4 million in cash reserves. Operating expenses for Q3 were $35.4 million, with R&D expenses at $37.9 million. The company anticipates sufficient cash to fund operations into 2026.
DAYBREAK Trial Results
Full results from the DAYBREAK trial were presented at ObesityWeek. The trial evaluated patients with genetic variants in genes linked to the MC4R pathway. Patients who lost 5% or more after 16 weeks were eligible for a double-blind, randomized withdrawal phase. Results showed a mean decrease in BMI of 12.4% in patients on continuous setmelanotide therapy for 40 weeks, compared to 29% of patients randomized to placebo during the 24-week stage two of the trial.
Hypothalamic Obesity Data
Real-world data from initial French patients with hypothalamic obesity showed that eight adult patients had a mean BMI decrease of 5.6% and 12.8% at one and three months, respectively, after initiating setmelanotide treatment. Patients have continued to lose weight, with five patients who have reached the six-month time point experiencing a 21.3% average decrease in their BMI.
Company Outlook
Rhythm Pharmaceuticals continues to focus on enhancing patient engagement, expanding access to treatments, and navigating the complexities of trial recruitment and market entry. The company remains optimistic about its direction and its potential for international growth, particularly in the context of hypothalamic obesity and other indications.
