Full Press Release Details
Rhythm Pharmaceuticals Reports Third Quarter
2023 Financial Results
-- Third quarter 2023 net revenue from global
sales of IMCIVREE (setmelanotide) of $22.5 million --
-- More than 100 international patients on reimbursed
-- Phase 3 hypothalamic obesity trial on track
to be fully enrolled by the end of 2023 --
-- 25.5% mean BMI reduction achieved at one
year of setmelanotide therapy in patients with hypothalamic obesity (n=12) who transitioned to long term extension from Ph 2 trial; three
of 11 pediatric patients achieving normal body weight at one year on setmelanotide treatment --
-- Cash on-hand of $299.3 million sufficient
to fund planned operations into 2026 --
-- Company to provide update on R&D programs
for investors and analysts at an event planned for Dec. 6 --
-- Management to host conference call today
BOSTON, November 7, 2023 -
Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming
the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway
diseases, today reported financial results and provided a business update for the third quarter ended September 30, 2023.
"We have achieved remarkable progress this year in expanding
access to IMCIVREE (setmelanotide) and delivering it to patients on a global level," said David Meeker, M.D., Chair,
President and Chief Executive Officer of Rhythm. "We remain pleased by continued strong performance more than one year into our
U.S. commercial launch in Bardet-Biedl syndrome (BBS). Outside the U.S., we continue to expand access with pre-approval, reimbursed early-access
for hypothalamic obesity in France and a positive recommendation for public reimbursement in Canada for patients with BBS."
"In addition, we are excited by the strength of our 12-month
LTE data in patients with hypothalamic obesity where we reported a mean body mass index (BMI) reduction of more than 25% in patients on
therapy for one year, with several patients trending towards or achieving normal body weight. These data and continued enrollment progress
with our ongoing Phase 3 study reinforce our confidence as we advance this high potential program. We look forward to providing an R&D
update, including our RM-718 program, data from the open label part of the setmelanotide Phase 2 DAYBREAK study and data from our Phase
3 pediatrics trial, during an investor event in December."
Third Quarter and Recent Business
Clinical Development Updates
Corporate and Anticipated Upcoming Milestones
Rhythm also expects to achieve the following near-term milestones:
Today, Rhythm announced plans to provide an
update on several R&D programs during a breakfast event for investors and analysts on December 6, 2023 in Boston.
Third Quarter 2023 Financial Results:
Year to Date 2023 Financial Results:
Guidance: For the year ending December 31, 2023, Rhythm anticipates approximately $210 million to $220 million in Non-GAAP
Operating Expenses comprised of $125 million to $130 million from R&D expenses and $85 million to $90 million from S,G&A expenses.
Non-GAAP operating expenses is defined as GAAP operating expenses excluding stock-based compensation (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 September 30, 2023, 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 third quarter 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
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 hyperphagia
and severe obesity caused by rare melanocortin-4 receptor (MC4R) diseases. Rhythm's lead asset, IMCIVREE (setmelanotide),
an MC4R agonist designed to treat hyperphagia and severe obesity caused by rare MC4R pathway diseases, 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 MC4R pathway diseases, as well as a preclinical suite of small molecules
for the treatment of congenital hyperinsulinism. Rhythm's headquarters is in Boston, MA.
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:
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