Full Press Release Details
Rhythm Pharmaceuticals Reports Third Quarter
2024 Financial Results and Business Update
-- Third quarter 2024 net revenue from global
sales of IMCIVREE (setmelanotide) of $33.3 million --
-- Adult patients with acquired hypothalamic
obesity (N=8) achieved mean BMI reduction of 12.8% on setmelanotide therapy at three months in French early-access program --
-- Top-line data readout for Phase 3 trial evaluating
setmelanotide in 120 patients with acquired hypothalamic obesity on track for 1H 2025 --
-- sNDA to expand IMCIVREE label to include
patients as young as 2 years old in approved indications accepted by FDA for Priority Review with PDUFA goal date of Dec. 26, 2024
-- Cash on-hand expected to support planned
operations into 2026 --
-- Management to host conference call today
BOSTON, November 5, 2024 - Rhythm Pharmaceuticals, Inc.
(Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine
diseases, today reported financial results and provided a business update for the third quarter ended September 30, 2024.
"Rhythm continued our strong clinical and commercial execution
aimed at bringing treatments to patients with rare melanocortin-4 receptor (MC4R) pathway diseases to address hyperphagia and severe obesity,"
said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. "Growth in global sales of IMCIVREE
(setmelanotide) - primarily driven by prescriptions for patients with Bardet-Biedl syndrome (BBS) - remains steady, and we
look forward to a potential expansion of its availability to patients as young as 2 years old, pending U.S. Food and Drug Administration
(FDA) approval. We believe being able to treat children at a younger age can improve long-term clinical outcomes by addressing the underlying
cause of hyperphagia and early-onset, severe obesity."
Dr. Meeker continued, "We are particularly excited about
new data from the early-access program in France for adult patients with hypothalamic obesity presented today at ObesityWeek .
Importantly, these real-world data support the potential for success of our Phase 3 trial and highlight the opportunity for adult patients
who have lived with acquired hypothalamic obesity for a decade or more."
Third Quarter and Recent Business
| o | Real-world data from patients participating in an early-access program in France that showed eight adult patients with acquired hypothalamic obesity achieved a mean BMI reduction of 12.8% at three months on setmelanotide therapy; five of these patients achieved a mean BMI reduction of 21.3% after six months on therapy. These results demonstrate the clinical benefit of setmelanotide in adult patients who had lived with hypothalamic obesity for an average of 12.1 years; | |
| o | Data from the extension phase of the Company's Phase 2 trial of setmelanotide showed that patients with acquired hypothalamic obesity who were on therapy for more than one year (n=11) achieved a mean overall percent reduction in fat mass of 29.6%, versus a reduction in lean muscle mass of 7.7%; and four male patients between 11 and 14 years old at baseline exhibited an increase in muscle mass; and | |
| o | Encouraging data from the Company's exploratory Phase 2 DAYBREAK study showed that 27 of 32 patients on setmelanotide, or 84% of patients, achieved or maintained 5% BMI reduction from baseline through stage 2, compared to 5 of 17 patients, or 29.4% of patients, who transitioned to placebo for stage 2; also in DAYBREAK, a 12.4% mean percent BMI reduction was observed for all patients on continuous setmelanotide therapy over 40 weeks (n=32). |
| On October, 24, 2024, the Company announced a joint research collaboration with Axovia Pharmaceuticals, led by its CEO and Co-founder Professor Philip Beales, in order to better understand the global epidemiology of BBS, how various symptoms impact patients and their families' lives, the underlying genetics of this disease, and the need for improved diagnosis and additional treatment options. | ||
| On August 26, 2024, the Company announced that the FDA accepted for Priority Review its supplemental New Drug Application (sNDA) for IMCIVREE to treat children as young as 2 years old in its approved indications. The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of December 26, 2024. |
Anticipated Upcoming Milestones
Rhythm expects to achieve the following near-term milestones:
| Complete enrollment in the supplemental, 12-patient Japanese cohort of its global Phase 3 trial evaluating setmelanotide in acquired hypothalamic obesity by the end of 2024; | ||
| Complete enrollment in two substudies in the Phase 3 EMANATE trial evaluating setmelanotide in genetically caused MC4R pathway diseases by the end of 2024; | ||
| Complete enrollment in the Phase 2 trial evaluating oral MC4R agonist bivamelagon (LB54640) in acquired hypothalamic obesity in the first quarter of 2025; | ||
| Following submission and approval of a protocol amendment, begin dosing patients with acquired hypothalamic obesity in Part C of the Phase 1 trial evaluating the weekly, MC4R agonist RM-718 in the first quarter of 2025; and | ||
| Announce top-line data in the Phase 3 trial evaluating setmelanotide in acquired hypothalamic obesity in the first half of 2025. |
Third Quarter 2024 Financial Results:
Cash Position: As of September 30,
2024, cash, cash equivalents and short-term investments were approximately $298.4 million, as compared to $275.8 million as of December 31,
Revenue: Net product revenues relating
to global sales of IMCIVREE were $33.3 million for the third quarter of 2024, as compared to $22.5 million for the third quarter of 2023.
R&D Expenses: R&D expenses
were $37.9 million in the third quarter of 2024, as compared to $33.6 million in the third quarter of 2023. The year-over-year increase
was primarily due to increased costs associated with certain clinical product manufacturing activity, ongoing clinical trials and increased
headcount. This increase was partially offset by decreased costs in other trials.
SG&A Expenses: SG&A expenses
were $35.4 million for the third quarter of 2024, as compared to $30.5 million for the third quarter of 2023. The year-over-year increase
was primarily due to increased headcount, marketing and promotions costs and expenses for professional services.
Other income (expense), net: Other
income (expense), net was ($0.1) million for the third quarter of 2024.
Net Loss: Net loss attributable to
common stockholders was ($45.0) million for the third quarter of 2024, or a net loss per basic and diluted share of ($0.73), as compared
to a net loss attributable to common stockholders of ($44.2) million for the third quarter of 2023, or a net loss per basic and diluted
Year to Date 2024 Financial Results:
Revenue: Net product revenues relating
to sales of IMCIVREE were $88.3 million for the nine months ended September 30, 2024, as compared to $53.2 million for the nine months
ended September 30, 2023.
R&D Expenses: R&D expenses
were $196.8 million for the nine months ended September 30, 2024, as compared to $105.1 million for the nine months ended September 30,
2023. This increase was primarily due to costs associated with the acquisition and development of bivamelagon (LB54640), increased headcount
and certain clinical trial costs. This increase was partially offset by one-time, non-recurring costs associated with the acquisition
of Xinvento B.V. in 2023.
SG&A Expenses: SG&A expenses
were $106.2 million for the nine months ended September 30, 2024, as compared to $85.2 million for the nine months ended September 30,
2023. The increase was primarily due to increased headcount, professional services fees, marketing and data analytics, and general office
expenses to accommodate a growing workforce.
Other income (expense), net: Other
income (expense), net was $7.4 million for the nine months ended September 30, 2024, as compared to $0.4 million for the nine months
ended September 30, 2023. The increase was primarily due to a gain on settlement of the forward contract associated with the issuance
of convertible preferred stock during the second quarter of 2024, the change in fair value of the embedded derivative of the debt royalty
obligation to HealthCare Royalty Partners (HCR) and increased interest income based on higher investment balances. This increase was partially
offset by accretion of the non-current liability payable to LG Chem due in July 2025 and non-cash interest expense related to amortization
of debt discount from the HCR milestone payment received in September 2023.
Net Loss: Net loss attributable to
common stockholders was ($219.9) million for the nine months ended September 30, 2024, or a net loss attributable to common stockholders
per basic and diluted share of $(3.62), as compared to a net loss attributable to common stockholders of ($143.0) million for the nine
months ended September 30, 2023, or a net loss per basic and diluted share of ($2.50).
Financial Guidance: For the year ending
December 31, 2024, Rhythm today reduced its anticipated Non-GAAP Operating Expenses to approximately $245 million to $255 million
from $250 million to $270 million. Non-GAAP Operating Expenses are derived from:
| o | S,G&A expenses of approximately $113 million; and | |
| o | R&D expenses of approximately $137 million; |
| o | Stock-based compensation, and | |
| o | $92.4 million in fixed consideration related to in-licensing of global rights to bivamelagon (LB54640), which was recognized in the first quarter of 2024. |
Non-GAAP Operating Expenses is defined as
GAAP operating expenses excluding stock-based compensation and fixed consideration related to in-licensing (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, 2024, will be sufficient to fund
its operating expenses and capital expenditure requirements into 2026.
Conference Call Information
Rhythm Pharmaceuticals will host a live conference
call and webcast at 5:00 p.m. ET today to review its third quarter 2024 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 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
Rhythm is a commercial-stage biopharmaceutical
company committed to transforming the lives of patients living with rare neuroendocrine diseases. Rhythm's lead asset, IMCIVREE
(setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, 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 &