Full Press Release Details
Rhythm Pharmaceuticals Reports Second Quarter
2021 Financial Results
-- Received European Commission authorization
of IMCIVREE for treatment of obesity and control of hunger associated with POMC, PCSK1 and LEPR deficiencies -
-- Setmelanotide selected for evaluation as
a "Highly Specialised Technology" by Great Britain's National Institute for Health and Care Excellence -
-- On track to submit sNDA to FDA and MAA Type
II amendment to EMA for setmelanotide in BBS and Alstr m syndromes in 2H 2021 --
-- Five new Phase 2 and 3 clinical trials planned
to initiate in the second half of 2021--
-- Management to Host Conference Call at 8:00
BOSTON, August 3, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals,
Inc. (Nasdaq: RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic
diseases of obesity, today reported financial results and provided a business update for the second quarter ended June 30, 2021.
"We have made tremendous progress in the second quarter towards
our goal of transforming the care of patients with rare genetic diseases of obesity globally," said David Meeker, M.D., Chair, President
and Chief Executive Officer of Rhythm. "We are pleased with our first full quarter of IMCIVREE (setmelanotide) commercial
availability in the United States with positive engagements with patients, prescribers and payors. We recently secured European Commission
marketing authorization for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function
biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above, enabling us
to expand patient access to IMCIVREE. From a market access standpoint, we are encouraged regulatory authorities, such as National Institute
for Health and Care Excellence (NICE) in the United Kingdom, recognize these obesities as rare genetic diseases for which there are no
available treatment options."
Dr. Meeker continued, "In parallel, we are executing on our clinical
development and regulatory strategy to bring setmelanotide to substantially more patients suffering from rare genetic diseases of obesity.
We look forward to completing supplementary regulatory submissions in the second half of this year to both the U.S. Food and Drug Administration
(FDA) and European Medicines Agency (EMA), seeking marketing authorization for setmelanotide for Bardet-Biedl and Alstr m syndromes,
and we are excited to continue advancing our broad clinical development program in additional patient populations. With agreement from
both the FDA and EMA, we are poised to initiate five clinical trials of setmelanotide: the pivotal Phase 3 EMANATE trial with five sub-studies
in heterozygous POMC, PCSK1 or LEPR deficiency obesities and SRC1 and SH2B1 deficiency obesities, the Phase 2 DAYBREAK trial in 31 additional
genes each with strong or very strong ties to the MC4R pathway, as well as a Phase 3 pediatrics trial for children younger than 6 and
two registrational trials for our weekly formulation of setmelanotide. Taken together, we believe these efforts may enable us to help
many more people with rare genetic diseases of obesity with a potential treatment for their insatiable hunger or hyperphagia and early-onset,
Second Quarter and Recent Business Highlights:
Pipeline and Business Developments:
POMC and LEPR Deficiency Obesities:
Bardet-Biedl Syndrome and Alstr m Syndrome:
Additional Clinical Development Updates:
Today, Rhythm announced it has reached agreement with the FDA and EMA
on five new Phase 2 and Phase 3 clinical trials, all of which the Company expects to initiate in the second half of 2021:
Phase 3 study in patients currently on daily setmelanotide therapy ("switch study"), that is a randomized, double-blind
trial to evaluate the efficacy of daily and weekly formulations of setmelanotide in patients with obesity due to biallelic POMC, PCSK1
or LEPR deficiency or BBS;
Phase 3, randomized, double-blind study in patients na ve to setmelanotide therapy ("de novo study"),
that includes a cross-over period to evaluate the weekly formulation of setmelanotide in patients with BBS.
Key Upcoming Milestones:
Rhythm expects to achieve the following milestones in 2021:
Regulatory Milestones:
Additional Clinical Milestones:
Second Quarter 2021 Financial Results:
Year to Date 2021 Financial Results:
Financial Guidance: Based on its current operating plans, Rhythm
expects that its existing cash, cash equivalents and short-term investments as of June 30, 2021, will be sufficient to fund its operating
expenses and capital expenditure requirements into at least the second half of 2023.
Conference Call Information
Rhythm Pharmaceuticals will host a live conference call and webcast
at 8:00 a.m. ET today to discuss this update, as well as review its second quarter 2021 financial results and recent business
activities. The conference call may be accessed by dialing (844) 498-0570 (domestic) or (409) 983-9726 (international), and referring
to conference ID 6776764. A webcast of the call will be available under "Events and Presentations" in the Investor Relations
section of the Rhythm Pharmaceuticals website at http://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
About Rhythm Pharmaceuticals
Rhythm is a commercial-stage
biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity. The
Company's precision medicine, IMCIVREE (setmelanotide), was approved in November 2020 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 obesity due to POMC, PCSK1 or LEPR
deficiency confirmed by genetic testing and by the European Commission (EC) in July 2021 for the treatment of obesity and the control
of hunger associated with genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency
in adults and children 6 years of age and above. IMCIVREE is the first-ever FDA-approved and EC-authorized therapy for these rare genetic
diseases of obesity. Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity.
The Company is leveraging the Rhythm Engine and the largest known obesity DNA database - now with approximately 37,500 sequencing samples
- to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. The company
is based in Boston, MA.
IMCIVREE (setmelanotide) Indication
States, IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due
to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The condition
must be confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that
are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).
In the EU, IMCIVREE is indicated for the
treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic POMC, including
PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. IMCIVREE should be prescribed and
supervised by a physician with expertise in obesity with underlying genetic etiology.
IMCIVREE is not indicated for the treatment
of patients with the following conditions as IMCIVREE would not be expected to be effective:
Important Safety Information
WARNINGS AND PRECAUTIONS
Disturbance in Sexual Arousal: Sexual adverse reactions
may occur in patients treated with IMCIVREE. Spontaneous penile erections in males and sexual adverse reactions in females occurred in
clinical studies with IMCIVREE. Instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.
Depression and Suicidal Ideation: Some drugs that target
the central nervous system, such as IMCIVREE, may cause depression or suicidal ideation. Monitor patients for new onset or worsening of
depression. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors.
Skin Pigmentation and Darkening of Pre-Existing Nevi: IMCIVREE
may cause generalized increased skin pigmentation and darkening of pre-existing nevi due to its pharmacologic effect. This effect is reversible
upon discontinuation of the drug. Perform a full body skin examination prior to initiation and periodically during treatment with IMCIVREE
to monitor pre-existing and new skin pigmentary lesions.
Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative
in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants.
USE IN SPECIFIC POPULATIONS
Discontinue IMCIVREE when pregnancy is recognized unless the benefits