Full Press Release Details
Rhythm Pharmaceuticals Reports Second Quarter
2024 Financial Results and Business Update
-- Second quarter 2024 net revenue from global
sales of IMCIVREE (setmelanotide) of $29.1 million --
-- Dosed first patients in supplemental,
12-patient Japanese cohort in global Phase 3 trial evaluating setmelanotide in hypothalamic obesity; On track for topline data
from 120-patient, pivotal cohort in 1H 2025 --
-- Completed sNDA submission seeking to expand
U.S. label of IMCIVREE to treat pediatric patients as young as 2 years old in approved indications --
-- Cash on-hand expected to support planned
operations into 2026 --
-- Management to host conference call today
BOSTON, August 6, 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, today reported financial results and provided a business update for the second quarter ended
"We delivered a strong quarter executing on our global strategy
to bring IMCIVREE (setmelanotide) to patients with rare melanocortin-4 receptor (MC4R) diseases to treat their hyperphagia
and severe obesity," said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. "This included steady
growth in global sales of IMCIVREE, and the achievement of regulatory milestones in Europe and the United States that brings us closer
to expanding its availability to patients as young as 2 years old. We believe this pediatric expansion will improve long-term clinical
outcomes by treating these genetically-caused diseases when hyperphagia and obesity begin."
Dr. Meeker continued, "We are making rapid progress in
developing our MC4R portfolio assets to bring much needed therapeutic options to patients with rare MC4R diseases, starting with acquired
hypothalamic obesity. We remain on track to report topline data from the pivotal cohort of our ongoing, global Phase 3 trial of setmelanotide
for this indication in the first half of 2025, and we recently began dosing patients in a supplemental cohort in Japan, where the prevalence
rate is higher than in the United States and Europe. In addition, we dosed the first patients with hypothalamic obesity in the Phase
2 trial evaluating an oral MC4R agonist, LB54640, and we are advancing our dose-finding, Phase 1 trial of the weekly RM-718."
Second Quarter and Recent
Anticipated Upcoming Milestones
Rhythm expects to achieve the following near-term milestones:
Second Quarter 2024 Financial Results:
Year to Date 2024 Financial Results:
Financial Guidance: For the year ending December 31, 2024,
Rhythm anticipates approximately $250 million to $270 million in Non-GAAP Operating Expenses. Non-GAAP Operating Expenses are derived
Non-GAAP Operating Expenses is defined as
GAAP operating expenses excluding stock-based compensation and fixed consideration related to in-licensing (see below under "Non-GAAP
Financial Measures" for more details).
Based on its current operating plans, Rhythm
expects that its existing cash, cash equivalents and short-term investments as of June 30, 2024, will be sufficient to fund its
operating expenses and capital expenditure requirements into 2026.
Conference Call Information
Pharmaceuticals will host a live conference call and webcast at 8:00 a.m. ET today to review its second quarter 2024 financial results
and recent business activities. Participants may register for the conference call here. It is recommended that participants join the
call ten minutes prior to the scheduled start.
A webcast of the call will also be available under "Events and
Presentations" in the Investor Relations section of the Rhythm Pharmaceuticals website at https://ir.rhythmtx.com/. The archived
webcast will be available on Rhythm Pharmaceuticals' website approximately two hours after the conference call and will be available
for 30 days following the call.
About Rhythm Pharmaceuticals
Rhythm is a commercial-stage
biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases.
Rhythm's lead asset, IMCIVREE (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity,
is approved by the U.S. Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age
and older with monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1)
or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS).
Both the European Commission (EC) and the UK's Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized
setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed
loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above.
Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as RM-718 and
a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm's headquarters is in Boston, MA.
Setmelanotide Indication
In the United States, setmelanotide
is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity
due to POMC, PCSK1 or LEPR deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1 or LEPR genes that
are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS) or BBS.
In the European Union, setmelanotide is indicated for the treatment
of obesity and the control of hunger associated with genetically confirmed Bardet-Biedl syndrome (BBS) or genetically confirmed loss-of-function
biallelic proopiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children
6 years of age and above.
In the United States and Europe,
Setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.
Setmelanotide is not indicated for the treatment of patients with
the following conditions as setmelanotide would not be expected to be effective:
WARNINGS AND PRECAUTIONS
Skin Monitoring: Setmelanotide may lead to generalized
increased skin pigmentation and darkening of pre-existing naevi because of its pharmacologic effect. Full body skin examinations should
be conducted annually to monitor pre-existing and new skin pigmentary lesions before and during treatment with setmelanotide.
Heart rate and blood pressure monitoring: Heart rate and
blood pressure should be monitored as part of standard clinical practice at each medical visit (at least every 6 months) for patients
treated with setmelanotide.
Prolonged penile erection: Spontaneous penile erections
have been reported in clinical trials with setmelanotide. Patients who have a penile erection lasting longer than 4 hours should be instructed
to seek emergency medical attention for potential treatment of priapism.
Depression: In clinical trials, depression has been reported
in patients treated with setmelanotide. Patients with depression should be monitored at each medical visit during treatment with setmelanotide.
Consideration should be given to discontinuing setmelanotide if patients experience suicidal thoughts or behaviors.
Pediatric Population: The prescribing physician should
periodically assess response to setmelanotide therapy. In growing children, the impact of weight loss on growth and maturation should
be evaluated. The prescribing physician should monitor growth (height and weight) using age- and sex-appropriate growth curves.
Excipients: This medicinal product contains 10 mg benzyl
alcohol in each ml. Benzyl alcohol may cause allergic reactions. Patients who are pregnant or breastfeeding should be advised of the
potential risk from the excipient benzyl alcohol, which might accumulate over time and cause metabolic acidosis. This medicinal product
should be used with caution in patients with hepatic or renal impairment, because of the potential risk from the excipient benzyl alcohol
which might accumulate over time and cause metabolic acidosis.
Sodium: This medicinal product contains less than 1 mmol
sodium (23 mg) per dose, that is to say essentially "sodium-free."
The most frequent adverse reactions are hyperpigmentation (51%), injection
site reaction (39%), nausea (33%), and headache (26%).
USE IN SPECIFIC POPULATIONS
There are no data from the use of setmelanotide in