Latest News by Industry
-- U.S. launch of IMCIVREE®(setmelanotide) for Bardet-Biedl Syndrome progresses with strong demand with more than 120 prescriptions since FDA approval --
-- EC authorization for IMCIVREE expanded to include BBS; UK launch in POMC and LEPR deficiencies underway --
-- Setmelanotide received FDA Breakthrough Therapy Designation for hypothalamic obesity; Phase 3 trial expected to be initiated in early 2023 --
--Successfully completed $140 million public offering with exercise of underwriters’ option, extending cash runway into 2025 --
-- Management to host conference call today at 8:00 a.m. ET --
BOSTON, Nov. 08, 2022 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM) today reported financial results and provided a business update for the third quarter ended September 30, 2022.
“The third quarter of 2022 was transformative for Rhythm and our global strategy to deliver IMCIVREE® (setmelanotide) as the first precision medicine to address the needs of patients and families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. “We are pleased with our U.S. commercial launch and the initial reception of IMCIVREE for Bardet-Biedl syndrome (BBS), and we look forward to continuing to build on early market access successes in Europe.”
Dr. Meeker continued, “We also are excited by the potential of setmelanotide to achieve a profound, consistent and sustained reduction in body weight in patients with hypothalamic obesity as demonstrated in our Phase 2 and long-term-extension trials. Following recent feedback from the U.S. Food and Drug Administration (FDA), we look forward to initiating our pivotal Phase 3 trial early in 2023.”
Third Quarter and Recent Business Highlights:
Development, Regulatory and Commercial Updates:
Hypothalamic Obesity
Bardet-Biedl Syndrome
POMC and LEPR Deficiency Obesities
Clinical Development Updates:
Corporate:
Key Upcoming Milestones:
Rhythm expects to achieve the following near-term milestones:
Third Quarter 2022 Financial Results:
Year to Date 2022 Financial Results:
Financial Guidance: Based on its current operating plans, Rhythm expects that its existing cash and cash equivalents and short-term investments as of September 30, 2022 will be sufficient to fund operations into 2025.
Conference Call Information
Rhythm Pharmaceuticals will host a live conference call and webcast at 8:00 a.m. ET today to review its third quarter 2022 financial results and recent business activities. Participants may register for the conference call here. While not required, 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 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 lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases. Rhythm’s precision medicine, setmelanotide, 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). The European Commission (EC) has 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.
The UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) authorized setmelanotide 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. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare MC4R pathway diseases, including acquired hypothalamic 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.
Setmelanotide Indication
In the United 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 pro-opiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 6 years of age and above.
Limitations of Use
In the United States and Europe, Setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.
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 setmelanotide.
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 growth curves.
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.”
ADVERSE REACTIONS
The most frequent adverse reactions are hyperpigmentation (51%), injection site reaction (39%), nausea (33%), and headache (26%).
USE IN SPECIFIC POPULATIONS
Pregnancy
There are no data from the use of setmelanotide in pregnant women. Animal studies do not indicate direct harmful effects with respect to reproductive toxicity. However, administration of setmelanotide to pregnant rabbits resulted in decreased maternal food consumption leading to embryo-foetal effects. As a precautionary measure, setmelanotide should not be started during pregnancy or while attempting to get pregnant as weight loss during pregnancy may result in fetal harm. If a patient who is taking setmelanotide has reached a stable weight and becomes pregnant, consideration should be given to maintaining setmelanotide treatment as there was no proof of teratogenicity in the nonclinical data. If a patient who is taking setmelanotide and still losing weight gets pregnant, setmelanotide should either be discontinued, or the dose reduced while monitoring for the recommended weight gain during pregnancy. The treating physician should carefully monitor weight during pregnancy in a patient taking setmelanotide.
Breast-feeding
It is unknown whether setmelanotide is excreted in human milk. A nonclinical study showed that setmelanotide is excreted in the milk of nursing rats. No quantifiable setmelanotide concentrations were detected in plasma from nursing pups. A risk to the newborn/infant cannot be excluded. A decision must be made whether to discontinue breastfeeding or to discontinue/abstain from setmelanotide therapy taking into account the benefit of breastfeeding for the child and the benefit of therapy for the mother.
Fertility
No human data on the effect of setmelanotide on fertility are available. Animal studies did not indicate harmful effects with respect to fertility.
To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337. See Summary of Product Characteristics’ APPENDIX V for a list of European national reporting systems to communicate adverse reactions.
Please see the full Prescribing Information for additional Important Safety Information.
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, our expectations surrounding potential regulatory submissions, approvals and timing thereof, our business strategy and plans, including regarding commercialization of setmelanotide in Europe, the United Kingdom, Canada, the United States and other international regions, our ability to reach certain milestones and receive additional investment amounts under our financing agreement with HealthCare Royalty, our participation in upcoming events and presentations, the intended use of proceeds from our public offering, management changes, 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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2022 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.
Rhythm Pharmaceuticals, Inc.
Condensed Consolidated Statements of Operations and Comprehensive (Loss) Income
(in thousands, except share and per share data)
(Unaudited)
| Three months ended September 30, | Nine months ended September 30, | ||||||||||||||||
| 2022 | 2021 | 2022 | 2021 | ||||||||||||||
| Product revenue, net | $ | 4,284 | $ | 1,028 | $ | 8,094 | $ | 1,337 | |||||||||
| License revenue | — | — | 6,754 | — | |||||||||||||
| Costs and expenses: | |||||||||||||||||
| Cost of sales | 497 | 222 | 1,105 | 363 | |||||||||||||
| Research and development | 21,116 | 27,539 | 85,082 | 72,554 | |||||||||||||
| Selling, general, and administrative | 21,938 | 17,507 | 65,715 | 47,490 | |||||||||||||
| Total costs and expenses | 43,551 | 45,268 | 151,902 | 120,407 | |||||||||||||
| Loss from operations | (39,267 | ) | (44,240 | ) | (137,054 | ) | (119,070 | ) | |||||||||
| Other income: | |||||||||||||||||
| Other expense | (370 | ) | — | (370 | ) | 100,000 | |||||||||||
| Interest expense | (2,144 | ) | — | (2,190 | ) | — | |||||||||||
| Interest income | 920 | 138 | 988 | 313 | |||||||||||||
| Total other (expense) income, net | (1,594 | ) | 138 | (1,572 | ) | 100,313 | |||||||||||
| Loss before taxes | (40,861 | ) | (44,102 | ) | (138,626 | ) | (18,757 | ) | |||||||||
| (Benefit from) provision for income taxes | — | (8,995 | ) | — | 7,989 | ||||||||||||
| Net loss | $ | (40,861 | ) | $ | (35,107 | ) | $ | (138,626 | ) | $ | (26,746 | ) | |||||
| Net loss per share, basic and diluted | $ | (0.79 | ) | $ | (0.70 | ) | $ | (2.73 | ) | $ | (0.54 | ) | |||||
| Weighted-average common shares outstanding, basic and diluted | 51,400,922 | 50,246,303 | 50,712,452 | 49,374,336 | |||||||||||||
| Other comprehensive (loss) income: | |||||||||||||||||
| Net loss | $ | (40,861 | ) | $ | (35,107 | ) | $ | (138,626 | ) | $ | (26,746 | ) | |||||
| Unrealized loss, net on marketable securities and other long-term assets | 567 | — | (338 | ) | (107 | ) | |||||||||||
| Comprehensive loss | $ | (40,294 | ) | $ | (35,107 | ) | $ | (138,964 | ) | $ | (26,853 | ) | |||||
Rhythm Pharmaceuticals, Inc.
Condensed Consolidated Balance Sheets
(in thousands, except share and per share data)
(Unaudited)
| September 30, | December 31, | |||||||
| 2022 | 2021 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 185,087 | $ | 59,248 | ||||
| Short-term investments | 162,708 | 235,607 | ||||||
| Accounts receivable, net | 3,328 | 1,025 | ||||||
| Prepaid expenses and other current assets | 10,751 | 12,507 | ||||||
| Total current assets | 361,874 | 308,387 | ||||||
| Property and equipment, net | 2,426 | 2,813 | ||||||
| Right-of-use asset | 1,273 | 1,522 | ||||||
| Intangible assets, net | 8,097 | 4,658 | ||||||
| Restricted cash | 328 | 328 | ||||||
| Other long-term assets | 15,616 | 11,815 | ||||||
| Total assets | $ | 389,614 | $ | 329,523 | ||||
| Liabilities and stockholders’ equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 4,933 | $ | 5,737 | ||||
| Accrued expenses and other current liabilities | 25,741 | 30,084 | ||||||
| Deferred revenue | 1,760 | 7,000 | ||||||
| Lease liability | 664 | 606 | ||||||
| Total current liabilities | 33,098 | 43,427 | ||||||
| Long-term liabilities: | ||||||||
| Deferred royalty obligation | 72,961 | — | ||||||
| Lease liability | 1,440 | 1,945 | ||||||
| Derivative liability | 920 | — | ||||||
| Total liabilities | 108,419 | 45,372 | ||||||
| Stockholders’ equity: | ||||||||
| Preferred Stock, $0.001 par value: 10,000,000 shares authorized; no shares issued and outstanding at September 30, 2022 and December 31, 2021 | — | — | ||||||
| Common stock, $0.001 par value: 120,000,000 shares authorized; 55,756,256 and 50,283,574 shares issued and outstanding at September 30, 2022 and December 31, 2021, respectively | 56 | 50 | ||||||
| Additional paid-in capital | 949,043 | 813,041 | ||||||
| Accumulated other comprehensive loss | (339 | ) | (1 | ) | ||||
| Accumulated deficit | (667,565 | ) | (528,939 | ) | ||||
| Total stockholders’ equity | 281,195 | 284,151 | ||||||
| Total liabilities and stockholders’ equity | $ | 389,614 | $ | 329,523 | ||||
Corporate Contact:
David Connolly
Head of Investor Relations and Corporate Communications
Rhythm Pharmaceuticals, Inc.
857-264-4280
dconnolly@rhythmtx.com
Investor Contact:
Hannah Deresiewicz
Stern Investor Relations, Inc.
212-362-1200
hannah.deresiewicz@sternir.com
Media Contact:
Adam Daley
Berry & Company Public Relations
212-253-8881
adaley@berrypr.com