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UNITED STATES
SECURITIES AND
EXCHANGE COMMISSION
Washington, D.C.
20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported):
November 7, 2023
RHYTHM PHARMACEUTICALS, INC.
(Exact name of registrant as specified in
its charter)
Delaware |
|
001-38223 |
|
46-2159271 |
(State or other jurisdiction
of incorporation) |
|
(Commission
File Number) |
|
(IRS Employer
Identification Number) |
222 Berkeley Street
12th Floor
Boston, MA 02116
(Address of principal executive offices)
(Zip Code)
Registrant’s telephone number, including
area code: (857) 264-4280
N/A
(Former name or former address, if changed since
last report)
Check the appropriate box below if the
Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
¨ Written
communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
¨ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
¨ Pre-commencement
communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
¨ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
Securities registered pursuant to Section 12(b) of the Act:
Title
of each class |
Trading
Symbol(s) |
Name
of each exchange on which registered |
Common Stock, $0.001 par value per share |
RYTM |
The Nasdaq Stock Market LLC (Nasdaq Global Market) |
Indicate by check mark whether the registrant
is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or
Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company ¨
If an emerging growth company, indicate by check
mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting
standards provided pursuant to Section 13(a) of the Exchange Act. ¨
Item 2.02. Results of Operations and Financial Condition.
On November 7, 2023, Rhythm Pharmaceuticals, Inc.
(the “Company”) announced its financial results for the quarter ended September 30, 2023. The full text of the press release
issued by the Company in connection with the announcement is furnished as Exhibit 99.1 to this Current Report on Form 8-K.
The information contained in Item 2.02 of this
Current Report on Form 8-K (including Exhibit 99.1 attached hereto) shall not be deemed “filed” for purposes of Section 18
of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section,
nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except
as expressly provided by specific reference in such a filing.
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits
The following Exhibit 99.1 relates to Item 2.02, and shall be deemed
to be furnished, and not filed:
SIGNATURES
Pursuant to the requirements
of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned
hereunto duly authorized.
|
RHYTHM PHARMACEUTICALS,
INC. |
|
|
|
Date: November 7, 2023 |
By: |
/s/
Hunter Smith |
|
|
Hunter Smith |
|
|
Chief Financial Officer |
Exhibit 99.1
Rhythm Pharmaceuticals Reports Third Quarter
2023 Financial Results
and Business Update
-- Third quarter 2023 net revenue from global
sales of IMCIVREE® (setmelanotide) of $22.5 million --
-- More than 100 international patients on reimbursed
IMCIVREE therapy --
-- Phase 3 hypothalamic obesity trial on track
to be fully enrolled by the end of 2023 --
-- 25.5% mean BMI reduction achieved at one
year of setmelanotide therapy in patients with hypothalamic obesity (n=12) who transitioned to long term extension from Ph 2 trial; three
of 11 pediatric patients achieving normal body weight at one year on setmelanotide treatment --
-- Cash on-hand of $299.3 million sufficient
to fund planned operations into 2026 --
-- Company to provide update on R&D programs
for investors and analysts at an event planned for Dec. 6 --
-- Management to host conference call today
at 8:00 a.m. ET --
BOSTON, November 7, 2023 –
Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming
the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway
diseases, today reported financial results and provided a business update for the third quarter ended September 30, 2023.
“We have achieved remarkable progress this year in expanding
access to IMCIVREE® (setmelanotide) and delivering it to patients on a global level,” said David Meeker, M.D., Chair,
President and Chief Executive Officer of Rhythm. “We remain pleased by continued strong performance more than one year into our
U.S. commercial launch in Bardet-Biedl syndrome (BBS). Outside the U.S., we continue to expand access with pre-approval, reimbursed early-access
for hypothalamic obesity in France and a positive recommendation for public reimbursement in Canada for patients with BBS.”
“In addition, we are excited by the strength of our 12-month
LTE data in patients with hypothalamic obesity where we reported a mean body mass index (BMI) reduction of more than 25% in patients on
therapy for one year, with several patients trending towards or achieving normal body weight. These data and continued enrollment progress
with our ongoing Phase 3 study reinforce our confidence as we advance this high potential program. We look forward to providing an R&D
update, including our RM-718 program, data from the open label part of the setmelanotide Phase 2 DAYBREAK study and data from our Phase
3 pediatrics trial, during an investor event in December.”
Third Quarter and Recent Business
Highlights
Commercial Updates
| · | Today, Rhythm announced that more than 120 new prescriptions for IMCIVREE
for BBS have been written by U.S. prescribers and that the Company has received payor approval for reimbursement for 80 prescriptions
during the third quarter of 2023. |
| · | Today, Rhythm also announced that more than 100 international patients across
11 countries cumulatively have initiated reimbursed IMCIVREE therapy, as of October 27, 2023. |
| · | In November, Rhythm announced that the Canadian Agency for Drugs and Technologies
in Health (CADTH) recommended IMCIVREE® (setmelanotide) be reimbursed by CADTH-participating public drug plans for weight
management in adult and pediatric patients 6 years of age and older with obesity due to BBS. |
| · | In September 2023, the Company announced that a new International Classification
Diseases, Tenth Revision (ICD-10) diagnosis code for BBS was approved by the Centers for Disease Control and Prevention (CDC), and effective
October 1, 2023. This BBS-specific code may improve patient identification and yield data to improve understanding of disease progression,
and diagnostic and treatment journey. |
| · | In August 2023, the Company announced that the French National Agency
for Medicines and Health Products Safety (ANSM) and French National Authority for Health (HAS) granted pre-marketing early access authorization
AP1 (Autorisation d’Accès Précoce), for IMCIVREE® (setmelanotide) for patients with lesional hypothalamic
obesity. Products included in the AP1 programs are fully covered by France’s National Health System and Rhythm can expect to be
reimbursed for any patients receiving treatments through this program. |
Clinical Development Updates
| · | Today, Rhythm provided an update on progress of its pivotal, Phase 3 clinical
trial evaluating setmelanotide in patients with acquired hypothalamic obesity. Approximately two-thirds of planned patients have been
screened with a very low screen-failure rate observed. Rhythm affirms its expectation to complete enrollment in the fourth quarter of
2023. This Phase 3 trial is designed to enroll 120 patients aged 4 years or older randomized 2:1 to setmelanotide therapy or placebo for
a total of 60 weeks, including up to eight weeks for dose titration. |
| · | During The Obesity Society’s ObesityWeek® October 14
– 17 in Dallas, Rhythm and its collaborators delivered a total of six presentations, including data showing that setmelanotide therapy
resulted in sustained and deepened weight loss in patients with severe obesity caused by rare MC4R pathway diseases. Long-term extension
study data in patients with hypothalamic obesity (n=12) demonstrate a 25.5% reduction in mean BMI from baseline on setmelanotide therapy
at one year. |
| · | The
Company also presented data that showed intervention with setmelanotide may reduce the risk
of future metabolic syndrome, cardiovascular disease (CVD) and type 2 diabetes mellitus (T2DM)
in patients with obesity due BBS or due to POMC or LEPR deficiency obesity; and research
that showed severity of obesity was associated with increased prevalence of cardiac, endocrine/diabetes,
and renal outcomes early in life based on an analysis of 318 pediatric patients with BBS
enrolled in the Clinical Registry Investigating BBS (CRIBBS). Researchers concluded that
timely diagnosis and early implementation of hyperphagia and weight management strategies
in pediatric patients with BBS may reduce the risk and burden associated with these comorbidities.
See the Company’s Oct. 17, 2023 press release for more. |
| · | During
the 61st Annual European Society for Paediatric Endocrinology (ESPE)
meeting September 21-23 in The Hague, Netherlands, Rhythm delivered four oral
presentations, including data presentations on genetic testing results and the effects of
setmelanotide on the metabolic syndrome severity score in pediatric patients with BBS and
in pediatric patients with POMC or LEPR deficiencies. See the Company’s Sept. 22,
2023 press release for more. |
| · | In September 2023, Rhythm announced that the European Medicines Agency
(EMA) issued a positive opinion on the Company’s orphan drug designation request for setmelanotide as a treatment for acquired hypothalamic
obesity. |
Corporate and Anticipated Upcoming Milestones
Rhythm also expects to achieve the following near-term milestones:
| · | Complete patient enrollment in the pivotal Phase 3 clinical trial in hypothalamic
obesity in the fourth quarter of 2023; |
| · | Complete submission to the U.S. Food and Drug Administration of an investigational
new drug application for RM-718, a new, weekly, MC4R-specific agonist, in the fourth quarter of 2023. RM-718 is designed to be more targeted
and potent than setmelanotide, and designed to be MC1R sparing, with the potential to not cause hyperpigmentation; |
| · | Announce preliminary data from the open-label part of the Phase 2 DAYBREAK
trial from approximately five genetically-defined cohorts in the fourth quarter of 2023; |
| · | Announce topline data from the ongoing Phase 3, open-label pediatrics trial
evaluating one year of setmelanotide therapy in patients with MC4R pathway deficiencies between the ages of 2 and 6 years old in the fourth
quarter of 2023; and |
| · | Provide pharmacokinetic and tolerability data from the ongoing Phase 3 switch
trial evaluating a weekly formulation of setmelanotide in the fourth quarter of 2023. |
Today, Rhythm announced plans to provide an
update on several R&D programs during a breakfast event for investors and analysts on December 6, 2023 in Boston.
Third Quarter 2023 Financial Results:
| · | Cash Position: As of September 30, 2023, cash, cash equivalents
and short-term investments were approximately $299.3 million, as compared to $333.3 million as of December 31, 2022. |
| · | Revenue: Net product revenues relating to global sales of IMCIVREE
were $22.5 million for the third quarter of 2023, as compared to $4.3 million for the third quarter of 2022. For the third quarter ended
September 30, 2023, 80% of the Company’s product revenue was generated in the United States. |
| · | R&D Expenses: R&D expenses were $33.6 million in the third
quarter of 2023, as compared to $21.1 million in the third quarter of 2022. The year-over-year increase was primarily due to increased
costs associated with certain clinical trials and pre-clinical studies, increased headcount and increased fees for professional services
and consulting. |
| · | S,G&A Expenses: S,G&A expenses were $30.5 million for the
third quarter of 2023, as compared to $21.9 million for the third quarter of 2022. The year-over-year increase was primarily due to increased
headcount in the United States and internationally, professional services and other expenses. |
| · | Other
income (expense), net. Other income (expense), net was $0.2 million for the third quarter
of 2023. Total other income (expense), net for the three months ended September 30, 2023
consists of interest income of $3.5 million earned on short-term investments, other expense
of $0.2 million from net foreign currency losses and the change in fair value of the Royalty
Interest Financing Agreement (RIFA) embedded derivative and $3.1 million of interest expense
related to the Company’s RIFA with HealthCare Royalty Partners. |
| · | Net Loss: Net loss was ($44.2) million for the third quarter of 2023,
or a net loss per basic and diluted share of ($0.76), as compared to a net loss of ($40.9) million for the third quarter of 2022, or a
net loss per basic and diluted share of ($0.79). |
Year to Date 2023 Financial Results:
| · | Revenue: Net product revenues relating to sales of IMCIVREE were $53.2
million for the nine months ended September 30, 2023, as compared to $8.1 million for the nine months ended September 30,
2022. |
| · | License Revenue: The Company did not report license revenue relating
to out-license arrangements in the nine months ended September 30, 2023. License revenue relating to the Company’s out-license
arrangement with RareStone was $6.8 million for the nine months ended September 30, 2022. |
| · | R&D Expenses: R&D expenses were $105.1 million for
the nine months ended September 30, 2023, as compared to $85.1 million for the nine months ended September 30, 2022. This
increase was primarily due to the acquisition of Xinvento B.V. and increased costs associated with headcount, certain clinical trials,
pre-clinical studies and gene sequencing and was partially offset by decreased costs associated with less manufacturing of clinical materials. |
| · | S,G&A Expenses: S,G&A expenses were $85.2 million for the
nine months ended September 30, 2023, as compared to $65.7 million for the nine months ended September 30, 2022. The increase
was primarily due to increased headcount to support business and commercial operations in the United Sates and internationally, professional
services and other expenses and was partially offset by decreased marketing activities associated with the BBS U.S. launch during the
prior year. |
| · | Other
income (expense), net: Other income (expense), net was $0.4 million for the nine months
ended September 30, 2023. Total other income (expense), net for the nine months ended September
30, 2023 consists of interest income of $10.1 million earned on our short-term investments,
other expense of $0.4 million from net foreign currency losses and the change in fair value
of the RIFA embedded derivative and $9.3 million of interest expense related to the RIFA
with HealthCare Royalty Partners. |
| · | Net Loss: Net loss was ($143.0) million for
the nine months ended September 30, 2023, or a net loss per basic and diluted share of $(2.50), as compared to a net loss of ($138.6)
million for the nine months ended September 30, 2022, or a net loss per basic and diluted share of ($2.73). |
Financial
Guidance: For the year ending December 31, 2023, Rhythm anticipates approximately $210 million to $220 million in Non-GAAP
Operating Expenses comprised of $125 million to $130 million from R&D expenses and $85 million to $90 million from S,G&A expenses.
Non-GAAP operating expenses is defined as GAAP operating expenses excluding stock-based compensation (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, 2023, 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 8:00 a.m. ET today to review its third quarter 2023 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
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 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 and their families living with hyperphagia
and severe obesity caused by rare melanocortin-4 receptor (MC4R) diseases. Rhythm’s lead asset, IMCIVREE (setmelanotide),
an MC4R agonist designed to treat hyperphagia and severe obesity caused by rare MC4R pathway diseases, 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 & Healthcare Products Regulatory Agency (MHRA) have 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. Additionally, Rhythm is advancing
a broad clinical development program for setmelanotide in other rare MC4R pathway diseases, as well as a preclinical suite of small molecules
for the treatment of congenital hyperinsulinism. 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 proopiomelanocortin (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:
| · | Obesity due to suspected POMC, PCSK1 or LEPR deficiency with POMC, PCSK1 or LEPR variants
classified as benign or likely benign |
| · | Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, or
BBS, including obesity associated with other genetic syndromes and general (polygenic) obesity. |
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-fetal
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, including the anticipated IND application for RM-718, our business strategy and plans, including regarding commercialization
of setmelanotide in certain international regions, expectations surrounding sales and reimbursement of IMCIVREE, our anticipated financial
performance and financial position, including estimated Non-GAAP Operating Expenses for the year ending December 31, 2023, the sufficiency
of our cash, cash equivalents and short-term investments to fund our operations , and our participation in upcoming events and presentations.
Statements using word such as “expect”, “anticipate”, “believe”, “may”, “will”,
"aim" and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties,
including, but not limited to, risks relating to our liquidity and expenses, our ability to enroll patients in clinical trials, the design
and outcome of clinical trials, the ability to achieve necessary regulatory approvals, risks associated with data analysis and reporting,
failure to identify and develop additional product candidates, unfavorable pricing regulations, third-party reimbursement practices or
healthcare reform initiatives, risks associated with the laws and regulations governing our international operations and the costs of
any related compliance programs, the impact of competition, risks relating to product liability lawsuits, inability to maintain our collaborations,
or the failure of these collaborations, our reliance on third parties, risks relating to intellectual property, our ability to hire and
retain necessary personnel, the impact of the COVID-19 pandemic and general economic conditions on our business and operations, including
our preclinical studies, clinical trials and commercialization prospects, failure to realize the anticipated benefits of our acquisition
of Xinvento B.V. or significant integration difficulties related to the acquisition, and the other important factors discussed under the
caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2023 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.
Non-GAAP Financial Measures
This press release includes Non-GAAP Operating
Expenses, a supplemental measure of our performance that is not required by, or presented in accordance with, U.S. GAAP and should not
be considered as an alternative to operating expenses or any other performance measure derived in accordance with GAAP.
We define Non-GAAP Operating Expenses as GAAP
operating expenses excluding stock-based compensation.
We caution investors that amounts presented
in accordance with our definition of Non-GAAP Operating Expenses may not be comparable to similar measures disclosed by our competitors
because not all companies and analysts calculate this non-GAAP financial measure in the same manner. We present this non-GAAP financial
measure because we consider it to be an important supplemental measure of our performance and believe it is frequently used by securities
analysts, investors, and other interested parties in the evaluation of companies in our industry. Management believes that investors’
understanding of our performance is enhanced by including this non-GAAP financial measure as a reasonable basis for comparing our ongoing
results of operations.
Management uses this non-GAAP financial measure
for planning purposes, including the preparation of our internal annual operating budget and financial projections; to evaluate the performance
and effectiveness of our operational strategies; and to evaluate our capacity to expand our business. This non-GAAP financial measure
has limitations as an analytical tool, and should not be considered in isolation, or as an alternative to, or a substitute for operating
expenses or other financial statement data presented in accordance with GAAP in our consolidated financial statements.
Rhythm has not provided a quantitative reconciliation
of forecasted Non-GAAP Operating Expenses to forecasted GAAP operating expenses because the Company is unable, without making unreasonable
efforts, to calculate the reconciling item, stock-based compensation expenses, with confidence. This item, which could materially affect
the computation of forward-looking GAAP operating expenses, is inherently uncertain and depends on various factors, some of which are
outside of Rhythm's control.
Corporate Contact:
David Connolly
Executive Director, Investor Relations
and Corporate Communications
Rhythm Pharmaceuticals, Inc.
857-264-4280
dconnolly@rhythmtx.com
Media Contact:
Adam Daley
Berry & Company Public Relations
212-253-8881
adaley@berrypr.com
Rhythm Pharmaceuticals, Inc.
Condensed Consolidated
Statements of Operations and Comprehensive Loss
(in thousands, except
share and per share data)
(Unaudited)
| |
Three months ended September 30, | | |
Nine months ended September 30, | |
| |
2023 | | |
2022 | | |
2023 | | |
2022 | |
Revenues: | |
| | |
| | |
| | |
| |
Product revenue, net | |
$ | 22,504 | | |
$ | 4,284 | | |
$ | 53,194 | | |
$ | 8,094 | |
License revenue | |
| — | | |
| — | | |
| — | | |
| 6,754 | |
Total revenues | |
| 22,504 | | |
| 4,284 | | |
| 53,194 | | |
| 14,848 | |
Costs and expenses: | |
| | | |
| | | |
| | | |
| | |
Cost of sales | |
| 2,412 | | |
| 497 | | |
| 6,069 | | |
| 1,105 | |
Research and development | |
| 33,570 | | |
| 21,116 | | |
| 105,059 | | |
| 85,082 | |
Selling, general, and administrative | |
| 30,475 | | |
| 21,938 | | |
| 85,158 | | |
| 65,715 | |
Total costs and expenses | |
| 66,457 | | |
| 43,551 | | |
| 196,286 | | |
| 151,902 | |
Loss from operations | |
| (43,953 | ) | |
| (39,267 | ) | |
| (143,092 | ) | |
| (137,054 | ) |
Other income (expense): | |
| | | |
| | | |
| | | |
| | |
Other income (expense), net | |
| (159 | ) | |
| (370 | ) | |
| (369 | ) | |
| (370 | ) |
Interest expense | |
| (3,149 | ) | |
| (2,144 | ) | |
| (9,342 | ) | |
| (2,190 | ) |
Interest income | |
| 3,466 | | |
| 920 | | |
| 10,126 | | |
| 988 | |
Total other income, net | |
| 158 | | |
| (1,594 | ) | |
| 415 | | |
| (1,572 | ) |
(Loss) income before taxes | |
| (43,795 | ) | |
| (40,861 | ) | |
| (142,677 | ) | |
| (138,626 | ) |
Provision for income taxes | |
| 368 | | |
| — | | |
| 368 | | |
| — | |
Net loss | |
$ | (44,163 | ) | |
$ | (40,861 | ) | |
$ | (143,045 | ) | |
$ | (138,626 | ) |
Net loss per share, basic and diluted | |
$ | (0.76 | ) | |
$ | (0.79 | ) | |
$ | (2.50 | ) | |
$ | (2.73 | ) |
Weighted-average common shares outstanding, basic and diluted | |
| 57,874,960 | | |
| 51,400,922 | | |
| 57,154,803 | | |
| 50,712,452 | |
| |
| | | |
| | | |
| | | |
| | |
Other comprehensive loss: | |
| | | |
| | | |
| | | |
| | |
Net loss | |
$ | (44,163 | ) | |
$ | (40,861 | ) | |
$ | (143,045 | ) | |
$ | (138,626 | ) |
Reclassification of losses on RareStone equity into net loss | |
| | | |
| 300 | | |
| | | |
| | |
Foreign currency translation adjustment | |
| 76 | | |
| — | | |
| 49 | | |
| — | |
Unrealized gain (loss), net on marketable securities | |
| (175 | ) | |
| 267 | | |
| (70 | ) | |
| (338 | ) |
Comprehensive loss | |
$ | (44,262 | ) | |
$ | (40,294 | ) | |
$ | (143,066 | ) | |
$ | (138,964 | ) |
Condensed Consolidated Balance Sheets
(in thousands, except share and per share data)
(Unaudited)
| |
September 30, | | |
December 31, | |
| |
2023 | | |
2022 | |
Assets | |
| | | |
| | |
Current assets: | |
| | | |
| | |
Cash and cash equivalents | |
$ | 64,593 | | |
$ | 127,677 | |
Short-term investments | |
| 234,667 | | |
| 205,611 | |
Accounts receivable, net | |
| 14,541 | | |
| 6,224 | |
Inventory | |
| 7,762 | | |
| 2,917 | |
Prepaid expenses and other current assets | |
| 7,638 | | |
| 11,807 | |
Total current assets | |
| 329,201 | | |
| 354,236 | |
Property and equipment, net | |
| 1,545 | | |
| 2,197 | |
Right-of-use asset | |
| 888 | | |
| 1,182 | |
Intangible assets, net | |
| 7,242 | | |
| 7,883 | |
Restricted cash | |
| 328 | | |
| 328 | |
Other long-term assets | |
| 14,995 | | |
| 16,655 | |
Total assets | |
$ | 354,199 | | |
$ | 382,481 | |
Liabilities and stockholders’ equity | |
| | | |
| | |
Current liabilities: | |
| | | |
| | |
Accounts payable | |
$ | 3,839 | | |
$ | 4,797 | |
Accrued expenses and other current liabilities | |
| 44,271 | | |
| 32,894 | |
Deferred revenue | |
| 1,286 | | |
| 1,434 | |
Lease liability | |
| 748 | | |
| 684 | |
Total current liabilities | |
| 50,144 | | |
| 39,809 | |
Long-term liabilities: | |
| | | |
| | |
Deferred royalty obligation | |
| 104,699 | | |
| 75,810 | |
Lease liability | |
| 692 | | |
| 1,260 | |
Derivative liability | |
| 1,190 | | |
| 1,340 | |
Total liabilities | |
| 156,725 | | |
| 118,219 | |
Stockholders’ equity: | |
| | | |
| | |
Preferred stock, $0.001 par value: 10,000,000 shares authorized; no shares issued and outstanding at September 30, 2023 and December 31, 2022 | |
| — | | |
| — | |
Common stock, $0.001 par value: 120,000,000 shares authorized; 59,089,352 and 56,612,429 shares issued and outstanding at September 30, 2023 and December 31, 2022, respectively | |
| 59 | | |
| 56 | |
Additional paid-in capital | |
| 1,050,631 | | |
| 974,356 | |
Accumulated other comprehensive loss | |
| (113 | ) | |
| (92 | ) |
Accumulated deficit | |
| (853,103 | ) | |
| (710,058 | ) |
Total stockholders’ equity | |
| 197,474 | | |
| 264,262 | |
Total liabilities and stockholders’ equity | |
$ | 354,199 | | |
$ | 382,481 | |
v3.23.3
Cover
|
Nov. 07, 2023 |
Cover [Abstract] |
|
Document Type |
8-K
|
Amendment Flag |
false
|
Document Period End Date |
Nov. 07, 2023
|
Entity File Number |
001-38223
|
Entity Registrant Name |
RHYTHM PHARMACEUTICALS, INC.
|
Entity Central Index Key |
0001649904
|
Entity Tax Identification Number |
46-2159271
|
Entity Incorporation, State or Country Code |
DE
|
Entity Address, Address Line One |
222 Berkeley Street
|
Entity Address, Address Line Two |
12th Floor
|
Entity Address, City or Town |
Boston
|
Entity Address, State or Province |
MA
|
Entity Address, Postal Zip Code |
02116
|
City Area Code |
857
|
Local Phone Number |
264-4280
|
Written Communications |
false
|
Soliciting Material |
false
|
Pre-commencement Tender Offer |
false
|
Pre-commencement Issuer Tender Offer |
false
|
Title of 12(b) Security |
Common Stock, $0.001 par value per share
|
Trading Symbol |
RYTM
|
Security Exchange Name |
NASDAQ
|
Entity Emerging Growth Company |
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