Full Press Release Details
Rhythm Pharmaceuticals Rhythm Pharmaceuticals
Reports First Quarter 2024 Financial Results and Business Update
-- First quarter 2024 net revenue from global
sales of IMCIVREE (setmelanotide) of $26.0 million --
-- On track to submit sNDA to the FDA to treat
pediatric patients between 2 and younger than 6 years old in approved indications in second quarter of 2024 --
-- Phase 3 trial evaluating setmelanotide in
hypothalamic obesity remains on track for topline data readout in 1H 2025; dosing of first patients in Japan anticipated in 2Q 2024 --
-- Secured $150 million in convertible preferred
stock financing, funding planned operations into 2026 --
-- Management to host conference call today
BOSTON, May 7, 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 first
quarter ended March 31, 2024.
"We continue to execute our global strategy of delivering IMCIVREE
(setmelanotide) to patients with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases,"
said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. "We had another solid quarter marked by steady
growth in global sales of IMCIVREE, which is now available in 14 markets."
Dr. Meeker continued, "We remain focused on expanding the
global opportunity for setmelanotide in additional indications, most notably in hypothalamic obesity with our ongoing, global Phase 3
trial. This pivotal trial is progressing as planned, and we anticipate dosing the first patients with hypothalamic obesity in Japan in
the second quarter of 2024. In addition, we expect to submit a supplementary New Drug Application (sNDA) to the FDA seeking a label expansion
for IMCIVREE to treat pediatric patients between 2 and younger than 6 years old in approved indications in the second quarter of 2024.
While we work to maximize the setmelanotide opportunity, we are also advancing our broader MC4R portfolio with continued progress with
our RM-718 and LB54640 programs."
First Quarter and Recent Business
Anticipated Upcoming Milestones
Rhythm expects to achieve the following near-term milestones:
First Quarter Financial
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 from:
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 March 31, 2024 along with the proceeds from the
sale of $150 million in preferred stock, will be sufficient to fund its operating expenses and capital expenditure requirements into 2026.
Rhythm Pharmaceuticals will host a live conference
call and webcast at 8:00 a.m. ET today to review its first 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
A live 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
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
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 pregnant women.
Animal studies do not indicate direct harmful effects with respect to reproductive toxicity. However, administration of setmelanotide