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
InformationRhythm 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
PharmaceuticalsRhythm 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
IndicationIn 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 UseIn 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 REACTIONSThe most frequent
adverse reactions are hyperpigmentation (51%), injection site
reaction (39%), nausea (33%), and headache (26%).
USE IN SPECIFIC POPULATIONS
PregnancyThere 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-feedingIt 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.
FertilityNo 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
StatementsThis 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 MeasuresThis 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 ConnollyExecutive Director, Investor
Relations and Corporate CommunicationsRhythm Pharmaceuticals,
Inc.857-264-4280dconnolly@rhythmtx.com
Media
Contact:Adam DaleyBerry & Company Public
Relations212-253-8881adaley@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 |
|
Rhythm Pharmaceuticals (NASDAQ:RYTM)
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