Full Press Release Details
Rhythm Pharmaceuticals Reports Fourth Quarter
2023 Financial Results
-- Fourth quarter 2023 net revenue from global
sales of IMCIVREE (setmelanotide) of $24.2 million --
-- Enrollment completed in 120-patient, pivotal
cohort of Phase 3 setmelanotide trial in hypothalamic obesity; top-line data on track for 1H2025 --
-- Announced Phase 3 development plans for setmelanotide
in hypothalamic obesity in Japan --
-- Spain and Italy authorities approve reimbursement
for IMCIVREE to treat patients with Bardet-Biedl syndrome -
-- Acquired global rights to oral MC4R agonist
LB54640 from LG Chem --
-- IND application for RM-718 accepted
-- Management to host conference call today
BOSTON, February 22, 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 fourth quarter and full year ended December 31, 2023.
"2023 was a strong year for us as we delivered IMCIVREE
(setmelanotide) and provided support for patients and families living with hyperphagia and severe obesity caused by rare melanocortin-4
receptor (MC4R) pathway diseases in 14 countries, including the United States," said David Meeker, M.D., Chair, President and Chief
Executive Officer of Rhythm. "We began this year by meaningfully strengthening our pipeline with RM-718 ready to enter the clinic
and with the acquisition of the global rights to LB54640."
He continued, "Importantly, we remain focused on developing setmelanotide
to treat patients with hypothalamic obesity, for whom there are no approved therapies. We are pleased to have completed enrollment in
our global Phase 3 trial in hypothalamic obesity and to have achieved alignment with Japanese authorities on an efficient path to develop
setmelanotide for patients with hypothalamic obesity."
Fourth Quarter and Recent
Corporate and Commercial Updates
| On February 7, 2024, the Company announced the Italian Medicine Agency (AIFA) approved reimbursement for IMCIVREE) for the treatment of obesity and control of hunger associated with BBS. | ||
| On January 4, 2024, Rhythm announced it entered into a global licensing agreement with LG Chem, Ltd. ("LG Chem") for LB54640, an investigational oral small molecule MC4R agonist in Phase 2 clinical trials. | ||
| On January 4, 2024, the Company also announced that federal healthcare authorities in Spain approved reimbursement for IMCIVREE for the treatment of obesity and control of hunger associated with BBS or biallelic POMC, PCSK1 or LEPR deficiency. |
Clinical, Research and Regulatory Updates
| Today, Rhythm also announced that it completed enrollment in the pivotal, 120-patient cohort in its global Phase 3 trial of setmelanotide in hypothalamic obesity with patients, aged 4 years or older with hypothalamic obesity randomized 2:1 to setmelanotide therapy or placebo for a total of 60 weeks, including up to eight weeks for dose titration. As agreed to with both the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA), Rhythm's regulatory submissions would be based on data from this cohort. The Company remains on track to obtain top-line study results in the first half of 2025. | ||
| Rhythm today also announced its clinical development plan of setmelanotide for hypothalamic obesity in Japan. The Company will add a supplemental Japanese patient cohort - designed to enable registration there - to its Phase 3 trial following an agreement with Japan's Pharmaceuticals and Medical Devices Agency (PMDA), with first patients expected to be dosed during the third quarter of 2024. Rhythm estimates that hypothalamic obesity has a prevalence of 5,000 to 8000 patients in Japan. | ||
| On January 4, 2024, Rhythm announced it submitted a Type II variation application to the EMA seeking regulatory approval and authorization for setmelanotide to treat obesity and control hunger in pediatric patients between 2 and younger than 6 years old with BBS or POMC, PCSK1 or LEPR deficiency in the European Union. | ||
| On January 4, 2024, Rhythm also announced that the FDA accepted the Company's Investigational New Drug (IND) application for RM-718 for administration as a weekly therapy treatment. RM-718 is designed to be more targeted and potent than setmelanotide with the potential to not cause hyperpigmentation. | ||
| On December 6, 2023, Rhythm announced new data from its 52-week, Phase 3 pediatrics trial in patients between 2 and younger than 6 years old (N=12) that showed setmelanotide achieved the primary endpoint with a 3.04 mean reduction in BMI-Z score (a measure of body mass index deviations from what is considered normal) and 18.4 percent mean reduction in BMI. | ||
| On December 6, 2023, the Company also announced data from the open-label part of its exploratory Phase 2 DAYBREAK trial that demonstrated potential efficacy in patients in multiple genetically-defined cohorts. A total of 49 patients who completed Stage 1 with a response to setmelanotide were randomized into Stage 2 of the trial, a 24-week, double-blind, placebo-controlled withdrawal study. These patients were stratified into genetically defined cohorts and randomized 2:1 to receive setmelanotide or placebo. |
Corporate and Anticipated Upcoming Milestones
Rhythm expects to achieve the following near-term milestones:
Fourth Quarter and Full Year 2023 Financial
Financial Guidance: For
the year ended December 2023, the Company had GAAP total operating expenses of $261.8 million. The Company today reported non-GAAP
Operating Expenses for the year ended December 31, 2023 of $219.9 million, which is derived from GAAP total costs and expenses of
$261.8 million less $9.3 million in cost of sales and less $32.6 million in stock-based compensation.
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
| o | SG&A expenses of $105 million to $110 million; and | |
| o | R&D expenses of $145 million to $160 million, |
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 December 31, 2023, will be sufficient to fund its
operating expenses and capital expenditure requirements into the second half of 2025.
Rhythm Pharmaceuticals will host a live conference call and webcast at 8:00 a.m. ET today to review its
fourth quarter and year end 2023 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 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
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
Setmelanotide Indication
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.
United States and Europe, Setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying
Setmelanotide is not indicated for the treatment of patients with the
following conditions as setmelanotide would not be expected to be effective:
| Obesity due to suspected POMC, PCSK1 or LEPR deficiency with POMC , PCSK1 or LEPR variants classified as benign or likely benign | ||
| Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, or BBS, including obesity associated with other genetic syndromes and general (polygenic) obesity. |
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