Full Press Release Details
Rhythm Pharmaceuticals
Reports Fourth Quarter and Full Year 2020 Financial Results
FDA approval of IMCIVREE (setmelanotide), the first-ever therapy for chronic weight management in patients with obesity
due to POMC, PCSK1 or LEPR deficiency --
positive topline data from Phase 3 pivotal trial evaluating setmelanotide in Bardet-Biedl and Alstr m syndromes; on-track
to complete regulatory submissions to FDA and EMA for BBS in 2H 2021 --
data from five cohorts in Phase 2 Basket Study demonstrating proof-of-concept in HET POMC, PCSK1 or LEPR deficiencies, and obesity
due to SRC1 and SH2B1 deficiencies --
broad development program for setmelanotide, with five Phase 2 and 3 clinical studies expected to initiate in 2021 in MC4R pathway-related
rare genetic diseases of obesity --
financial position with net proceeds of $98.5M from sale of Rare Pediatric Disease PRV and $161.6M from public offering, extending
cash runway through at least the second half of 2023 -
Boston, MA - March 1, 2021 -
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 fourth
quarter and full year ended December 31, 2020.
"We enter 2021 with tremendous momentum
on our journey to transform the care of people with rare genetic diseases of obesity," said David Meeker, M.D., Chair, President
and Chief Executive Officer of Rhythm. "In recent months, we secured our first approval of IMCIVREE, validating the melanoticortin-4
receptor (MC4R) pathway as an important therapeutic target and bringing the first-ever therapy to people suffering from obesity
due to POMC, PCSK1 or LEPR deficiency. The patients with defects in the MC4R pathway we have studied to date, including those with
our approved indications and patients who participated in our Phase 2 Basket Study, have severe obesity and have largely failed
other treatment modalities."
Dr. Meeker continued, "We also announced
positive topline data from our pivotal, Phase 3 trial in Bardet-Biedl syndrome (BBS), and we achieved proof-of-concept in multiple
additional genetic diseases of obesity, paving the way for a potentially registration-enabling trial this year. On the heels of
these recent achievements, we are entering our next phase as we begin to commercialize IMCIVREE and work to expand setmelanotide's
reach to address the unmet needs of many more people with a range of genetic variants in the MC4R pathway. We look forward to completing
regulatory submissions to both the FDA and the EMA seeking marketing authorization for setmelanotide for the treatment of obesity
in patient with BBS in the second half of 2021 and, in parallel, initiating new trials in a broad clinical program. Following the
sale of our priority review voucher (PRV) and recent public offering, we are well-funded, with sufficient resources to advance
the development of setmelanotide while supporting the patient community and driving awareness of genetic testing to identify and
properly diagnose people with these rare conditions."
Fourth Quarter and Recent Business Highlights:
Pipeline and Recent Developments:
Key Upcoming Milestones:
Rhythm expects to achieve the following
Commercial and Regulatory Milestones:
| Make IMCIVREE commercially available in the United States for obesity due to POMC, PCSK1 and LEPR deficiencies in the first quarter of 2021. | ||
| Obtain regulatory approval from the European Commission and make IMCIVREE commercially available in Europe in POMC, PCSK1 and LEPR deficiency obesities in the second half of 2021. | ||
| Complete regulatory submissions to both the FDA and the EMA seeking marketing authorization for setmelanotide for the treatment of obesity in patient with BBS in the second half of 2021. |
Clinical Milestones:
| Initiate a Phase 2 clinical trial of setmelanotide in hypothalamic obesity in the first half of 2021. | ||
| Announce new top-line data from the ongoing exploratory Phase 2 Basket Study evaluating setmelanotide in MC4R-recusable patients in the first half of 2021. | ||
| Announce full data from the pivotal Phase 3 trial evaluating setmelanotide in Bardet-Biedl and Alstr m Syndromes in the first half of 2021. | ||
| Initiate a Phase 2 clinical trial of setmelanotide in pediatric patients aged two to six years old in the second half of 2021. | ||
| Pending FDA feedback, initiate a pivotal Phase 3 MC4R pathway trial of setmelanotide in patients with HET obesity, as well as SRC1 and SH2B1 deficiency obesities, in the second half of 2021. | ||
| Initiate an expanded Phase 2 Basket Study of setmelanotide in patients with variants in one of 31 additional genes with strong or very strong MC4R pathway relevance in the second half of 2021. | ||
| Initiate a Phase 3 potentially registration-enabling trial for the weekly formulation of setmelanotide in the second half of 2021. |
Fourth Quarter and Full Year 2020 Financial
Financial Guidance: Based on its
current operating plans, Rhythm expects that its existing cash, cash equivalents and short-term investments as of December 31,
2020, together with an aggregate of approximately $260.1 million in net proceeds from the February 2021 sale of its Rare Disease
PRV and the February 2021 follow-on public offering, will be sufficient to fund its operating expenses and capital expenditure
requirements through at least the second half of 2023.
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), has been approved by the 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. IMCIVREE is
the first-ever FDA approved 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. For healthcare professionals, visit www.UNcommonObesity.com
for more information. For patients and caregivers, visit www.LEADforRareObesity.com for more information. The company is based
IMCIVREE (setmelanotide) Indication
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
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
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 of therapy outweigh the potential risks to the fetus.
Treatment with IMCIVREE is not recommended for use while breastfeeding.
To report SUSPECTED ADVERSE REACTIONS,
contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
See Full Prescribing
Information for IMCIVREE.
Forward-Looking Statements
This press release contains forward-looking statements
within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release
that do not relate to matters of historical fact should be considered forward-looking statements, including without
limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide,
including the anticipated timing for initiation of clinical trials and release of clinical trial data and our expectations
surrounding potential regulatory submissions, approvals and timing thereof, our business strategy and plans, including
regarding commercialization of setmelanotide, management changes, our participation in upcoming events and presentations, and
the sufficiency of our cash, cash equivalents and short-term investments to fund our operations. Statements using word such
as "expect", "anticipate", "believe", "may", "will" and similar
terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but
not limited to, the impact of our management transition, our ability to enroll patients in clinical trials, the design and
outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks
associated with data analysis and reporting, our liquidity and expenses, the impact of the COVID-19 pandemic on our business