-- Preparations continue for U.S. commercial launch in June 2022 in BBS and Alström syndrome, pending FDA approval --
-- First commercial sales of IMCIVREE in Germany and France expected in 2Q2022 –
-- Enrollment completed in Phase 2 trial evaluating setmelanotide in patients with hypothalamic obesity --
-- IMCIVREE fourth quarter sales of $1.8M and 2021 sales of $3.2M –
-- Management to host conference call today at 8:00 a.m. ET --
BOSTON, March 01, 2022 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company committed to transforming the care of people living with rare genetic diseases of obesity, today reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2021.
“2021 was a pivotal year for Rhythm, with tremendous progress across clinical development and regulatory efforts globally, as we deliver on our promise to transform the treatment of rare genetic diseases of obesity,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. “We first made IMCIVREE® (setmelanotide) commercially available last year to people living with obesity due to POMC, PCSK1 or LEPR deficiency, and now we look forward to our first commercial sales in Europe in the second quarter of this year. With a potential U.S. approval for IMCIVREE in BBS and Alström syndrome now set for June, our commercial team continues to drive patient identification and pre-launch activities as we prepare to deliver IMCIVREE to the waiting BBS and Alström syndrome communities at approval.”
Dr. Meeker continued, “We also significantly expanded our clinical development efforts with the Phase 3 EMANATE and Phase 2 DAYBREAK trials, the pediatrics trial for patients between the ages of 2 and 6, and the switch study evaluating a once-weekly formulation of setmelanotide. We are excited to have completed enrollment in our Phase 2 hypothalamic obesity trial with plans to read out data in the first half of this year. These studies have the potential to meaningfully expand the reach of setmelanotide and address the severe obesity and hyperphagia associated with rare genetic diseases of obesity, and, in the case of hypothalamic obesity, an acquired deficiency in the hypothalamic melanocortin pathway.”
Fourth Quarter and Recent Business Highlights:
Commercial Readiness for Bardet Biedl
International Updates
Clinical Development Updates:
Regulatory Updates:
Bardet-Biedl Syndrome and Alström Syndrome:
Key Upcoming Milestones:
Fourth Quarter and Full Year 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 December 31, 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 fourth quarter and full year 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 9840866. 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 following the call.
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. Rhythm’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 in July and September 2021, respectively, by the European Commission (EC) and Great Britain’s Medicines & Healthcare Products Regulatory Agency (MHRA) 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- and MHRA-authorized therapy for patients with these rare genetic diseases of obesity. The Company submitted a supplemental New Drug Application (sNDA) to the FDA, which was accepted for filing in November 2021 and is currently assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 16, 2022, for the treatment of obesity and control of hunger in adult and pediatric patients six years of age and older with Bardet-Biedl Syndrome (BBS) or Alström syndrome. A Type II variation application to the European Medicines Agency seeking regulatory approval and authorization for setmelanotide to treat obesity and control of hunger in adult and pediatric patients 6 years of age and older with BBS also is under review. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity and is leveraging the Rhythm Engine and the largest known obesity DNA database -- now with approximately 45,000 sequencing samples -- to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythm’s headquarters is in Boston, MA.
IMCIVREE® (setmelanotide) Indication
In the United 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, confirmed by an FDA-approved genetic test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS).
In the EU and Great Britain, 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.
Limitations of Use
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.
ADVERSE REACTIONS
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, EU SmPC and MHRA SmPC 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, sales of our lead product candidate IMCIVREE, 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 and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and the other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2021 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.
Corporate Contact:
David Connolly
Head of Investor Relations and Corporate Communications
Rhythm Pharmaceuticals, Inc.
857-264-4280
[email protected]
Investor Contact:
Hannah Deresiewicz
Stern Investor Relations, Inc.
212-362-1200
[email protected]
Media Contact:
Adam Daley
Berry & Company Public Relations
212-253-8881
[email protected]
Rhythm Pharmaceuticals, Inc. Condensed Consolidated Statements of Operations (in thousands, except share and per share data) (Unaudited) | ||||||||||||||||
Three months ended December 31, | Year ended December 31, | |||||||||||||||
2021 | 2020 | 2021 | 2020 | |||||||||||||
Product revenue, net | $ | 1,817 | $ | — | $ | 3,154 | $ | — | ||||||||
Costs and expenses: | ||||||||||||||||
Cost of sales | 236 | — | 599 | — | ||||||||||||
Research and development | 31,574 | 21,954 | 104,128 | 90,450 | ||||||||||||
Selling, general, and administrative | 20,997 | 13,119 | 68,486 | 46,125 | ||||||||||||
Total costs and expenses | 52,807 | 35,073 | 173,213 | 136,575 | ||||||||||||
Loss from operations | (50,990) | (35,073) | (170,059) | (136,575) | ||||||||||||
Other income: | ||||||||||||||||
Other income | — | — | 100,000 | — | ||||||||||||
Interest income, net | 134 | 176 | 447 | 2,579 | ||||||||||||
Total other income, net | 134 | 176 | 100,447 | 2,579 | ||||||||||||
Loss before taxes | (50,856) | (34,897) | (69,612) | (133,996) | ||||||||||||
Benefit from income taxes | (7,989) | — | — | — | ||||||||||||
Net loss | $ | (42,867) | $ | (34,897) | $ | (69,612) | $ | (133,996) | ||||||||
Net loss per share, basic and diluted | $ | (0.85) | $ | (0.79) | $ | (1.40) | $ | (3.04) | ||||||||
Weighted-average common shares outstanding, basic and diluted | 50,270,801 | 44,216,694 | 49,600,294 | 44,127,220 |
Rhythm Pharmaceuticals, Inc. Condensed Consolidated Balance Sheets (in thousands, except share and per share data) (Unaudited) | ||||||||
December 31, | December 31, | |||||||
2021 | 2020 | |||||||
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 59,248 | $ | 100,854 | ||||
Short-term investments | 235,607 | 71,938 | ||||||
Accounts receivable | 1,025 | — | ||||||
Prepaid expenses and other current assets | 12,507 | 8,876 | ||||||
Total current assets | 308,387 | 181,668 | ||||||
Property and equipment, net | 2,813 | 3,195 | ||||||
Right-of-use asset | 1,522 | 1,807 | ||||||
Intangible assets, net | 4,658 | — | ||||||
Restricted cash | 328 | 403 | ||||||
Other long-term assets | 11,815 | — | ||||||
Total assets | $ | 329,523 | $ | 187,073 | ||||
Liabilities and stockholders’ equity | ||||||||
Current liabilities: | ||||||||
Accounts payable | $ | 5,737 | $ | 4,900 | ||||
Accrued expenses and other current liabilities | 30,084 | 12,559 | ||||||
Contract liability | 7,000 | — | ||||||
Lease liability | 606 | 535 | ||||||
Total current liabilities | 43,427 | 17,994 | ||||||
Long-term liabilities: | ||||||||
Lease liability | 1,945 | 2,551 | ||||||
Total liabilities | 45,372 | 20,545 | ||||||
Stockholders’ equity: | ||||||||
Preferred Stock, $0.001 par value: 10,000,000 shares authorized; no shares issued and outstanding at December 31, 2021 and December 31, 2020 | — | — | ||||||
Common stock, $0.001 par value: 120,000,000 shares authorized; 50,283,574 and 44,235,903 shares issued and outstanding December 31, 2021 and December 31, 2020, respectively | 50 | 44 | ||||||
Additional paid-in capital | 813,041 | 625,762 | ||||||
Accumulated other comprehensive (loss) income | (1) | 49 | ||||||
Accumulated deficit | (528,939) | (459,327) | ||||||
Total stockholders’ equity | 284,151 | 166,528 | ||||||
Total liabilities and stockholders’ equity | $ | 329,523 | $ | 187,073 |
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