Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage
biopharmaceutical company committed to transforming the care of
people living with rare genetic diseases of obesity, today reported
financial results and provided a business update for the first
quarter ended March 31, 2022.
“We are excited by the progress we are making towards our
long-term vision of transforming the care of individuals living
with rare genetic diseases of obesity,” said David Meeker, M.D.,
Chair, President and Chief Executive Officer of Rhythm. “We
continue to build out our U.S. and international commercial
organizations while advancing patient identification efforts to
support a successful U.S. launch for setmelanotide in Bardet-Biedl
and Alström syndromes in June, pending U.S. Food and Drug
Administration (FDA) approval. We achieved our first European sales
in France during the quarter and continue to have constructive
market access discussions with authorities in several key European
markets.”
Dr. Meeker continued, “Additionally, our robust clinical
development program is advancing with patients enrolled in multiple
phase 2 and 3 clinical trials. We have optimized our ongoing Phase
3 EMANATE trial to focus exclusively on patient populations with
the highest probability of responding to setmelanotide and narrowed
our Phase 2 DAYBREAK trial to more efficiently evaluate rare
variants associated with ten prioritized genes. In the months
ahead, we look forward to reporting interim data from our Phase 2
trial in hypothalamic obesity and our Phase 2 Basket Study in
obesity due to a variant in the melanocortin-4 receptor
(MC4R).”
First Quarter and Recent
Business Highlights:
Update on Bardet-Biedl and Alström
Syndromes:
- Rhythm continues to execute on ongoing physician and patient
education and engagement efforts, as well as patient identification
efforts and additional strategies in support of a potential U.S.
commercial launch of setmelanotide for the treatment of obesity and
hyperphagia in patients with Bardet-Biedl syndrome (BBS).
- The Company’s supplemental New Drug Application (sNDA) for
IMCIVREE® (setmelanotide) for the treatment of obesity and control
of hunger in adult and pediatric patients 6 years of age and older
with BBS or Alström syndrome is under review by the FDA, with a
Prescription Drug User Fee Act (PDUFA) goal date of June 16,
2022.
- Rhythm’s Type II variation application to the European
Medicines Agency (EMA) for setmelanotide for the treatment of
obesity and control of hunger in adult and pediatric patients 6
years of age and older with BBS is also under review. Rhythm
anticipates that the EMA’s Committee for Medicinal Products for
Human Use (CHMP) will make its recommendation on this application
in the third quarter of 2022.
- The Company also announced today that on April 22, 2022, the
CHMP recommended the European Commission (EC) approve a
modification to the summary product characteristics (SmPC) for
IMCIVREE for biallelic POMC, PSCK1 or LEPR deficiency that would
allow for treating patients who have moderate and severe renal
impairment with an adjustment for more gradual dose escalation and
a lower maximum dose. The final EC decision on this amendment is
anticipated to come in July 2022, and this same dosing modification
request is being considered as part of the scheduled review for
BBS.
International Updates:
- Today, Rhythm announced that the first patients have been
treated with IMCIVREE in France under a paid early access program.
The French Haute Autorité de Santé (HAS) granted paid early access
for IMCIVREE for patients with POMC, PCSK1 or LEPR deficiency
obesity in January 2022.
Clinical Development Updates:
- In April 2022, Rhythm announced that the first patient was
enrolled in its Phase 3 EMANATE trial. With recently announced
modifications to the trial design, EMANATE now includes four
independent sub-studies evaluating setmelanotide in patients with
obesity due to a heterozygous variant of the POMC or PCSK1 gene,
the LEPR gene, the SRC1 gene or the SH2B1 gene. These modifications
were intended to optimize the design of EMANATE, with the goal of
focusing on rare patient populations that the Company believes have
the highest likelihood of success.
- In April 2022 at the Pediatric Endocrine Society Annual Meeting
(PES), Rhythm and its collaborators presented new data from the
Company’s Phase 3 clinical trial in BBS, which showed that
setmelanotide improved body weight measures, as well as total
cholesterol, high-density lipoprotein cholesterol, low-density
cholesterol and triglycerides in treated patients. Also at PES,
Rhythm presented data demonstrating clinically beneficial
reductions in BMI Z score and BMI in pediatric and adolescent
patients with BBS; characterizing the negative impacts of
hyperphagia and the resulting food-seeking behaviors on the lives
of patients with BBS and their caregivers; and highlighting the
safety and tolerability of setmelanotide across the 561 patients
treated across the setmelanotide clinical development program.
- Also in April 2022, Rhythm announced modifications to optimize
the design of its ongoing Phase 2 DAYBREAK trial to focus initially
on rare variants associated with 10 prioritized MC4R-relevant
genes, which the Company and key opinion leaders believe have the
highest probability of success.
Key Upcoming Milestones:
- Rhythm today announced that six abstracts have been accepted
for presentation at the Endocrine Society Annual Meeting & Expo
(ENDO), being held June 11-14, 2022 in Atlanta, GA. Dr. Jesús
Argente, Universidad Autónoma de Madrid in Spain, will deliver an
oral presentation of 12-month data from long-term extension trials
of setmelanotide in patients with obesity due to heterozygous
variants in POMC, PCSK1, and LEPR. In addition, five abstracts will
be delivered as poster presentations, including:
- Long-term Efficacy of Setmelanotide in Patients With Obesity
Due to POMC, PCSK1, and LEPR Biallelic Deficiency;
- Setmelanotide in Patients with Heterozygous POMC, LEPR, SRC1,
or SH2B1 Obesity: Design of EMANATE – A Placebo-Controlled Phase 3
Trial;
- Long-term Efficacy of Setmelanotide in Patients with
Bardet-Biedl Syndrome;
- Body Mass Index and Weight Reductions in Patients with SRC1
Genetic Variant Obesity After 1 Year of Setmelanotide; and
- Body Mass Index and Weight Reduction in Patients with SH2B1
Genetic Variant Obesity After 1 Year of Setmelanotide.
- Also in mid-2022, Rhythm anticipates announcing:
- Preliminary data from the ongoing Phase 2 study in patients
with hypothalamic obesity.
- New data from the ongoing exploratory Phase 2 Basket Study
evaluating setmelanotide in patients with obesity due to a variant
in the MC4 receptor.
First Quarter 2022 Financial Results:
- Cash Position: As of March 31, 2022,
cash, cash equivalents and short-term investments were
approximately $241.0 million, as compared to $294.9
million as of December 31, 2021.
- Revenue: Product net revenues relating to
sales of IMCIVREE were $1.5 million for the first quarter of 2022,
as compared to $35.0 thousand for the first quarter of 2021.
IMCIVREE became commercially available in late March 2021.
- R&D Expenses: R&D expenses
were $32.5 million in the first quarter of 2022, as
compared to $19.9 million in the first quarter of 2021.
The year-over-year increase was due to an increase of $9.4 million
in clinical trial costs associated with new and planned clinical
trials, including the Phase 2 DAYBREAK and Phase 3 EMANATE trials,
Phase 3 pediatrics trial, QTc study, Phase 2 hypothalamic obesity
study and increased enrollment in the long-term extension study; an
increase of $3.8 million due to increased purchases of clinical
supply material; an increase of $1.0 million in development
milestones earned by Camurus related to the weekly formulation of
setmelanotide and an increase of $0.4 million related to increased
number of genetic testing performed. These increases were partially
offset by the conclusion of prior studies, as well as a decrease of
$2.0 million in costs associated with medical affair
consulting.
- S,G&A Expenses: S,G&A expenses
were $21.4 million for the first quarter of 2022, as compared to
$14.5 million for the first quarter of 2021. The year-over-year
increase was primarily due to an increase of $3.3 million due to
increased compensation and benefits-related costs associated with
additional headcount to support expanding business operations, as
well as to establish commercial operations in the United
States and internationally; an increase of $2.3 million
related to marketing activities for IMCIVREE; an increase of $1.0
million due to increased costs associated with office support and
insurance costs for Rhythm’s expanding workforce; and an increase
of $0.2 million due to increased professional fees and consulting
services to support the build out of commercial operations in the
United States and internationally, as well as corporate legal and
consulting support for international expansion.
- Net (Loss)/Income: Net loss was $52.8
million for the first quarter of 2022, or a net loss per basic and
diluted share of ($1.05), as compared to a net income of $43.8
million for the first quarter of 2021, or a net income per basic
and diluted share of $0.92 and $0.90, respectively.
Financial Guidance: Based on its current
operating plans, Rhythm expects that its existing cash, cash
equivalents and short-term investments as of March 31, 2022
will be sufficient to fund its operating expenses and capital
expenditure requirements into at least the fourth quarter of
2023.
Conference Call InformationRhythm
Pharmaceuticals will host a live conference call and webcast
at 8:30 a.m. ET today to discuss this update, as well as
review its first quarter 2022 financial results and recent business
activities. The conference call may be accessed by dialing (866)
374-5140 (domestic) or (404) 400 0571 (international) and referring
to conference ID 79122322. A webcast of the call will be available
under "Events and Presentations" in the Investor Relations section
of the Rhythm Pharmaceuticals website
at http://ir.rhythmtx.com/. The archived webcast will be
available on Rhythm Pharmaceuticals’ website
approximately two hours after the conference call and will be
available for 30 days following the call.
About Rhythm PharmaceuticalsRhythm is a
commercial-stage biopharmaceutical company committed to
transforming the treatment paradigm for people living with rare
genetic diseases of obesity. Rhythm’s precision medicine, IMCIVREE
(setmelanotide), was approved in November 2020 by
the U.S. Food and Drug Administration (FDA) for chronic
weight management in adult and pediatric patients 6 years of age
and older with obesity due to POMC, PCSK1 or LEPR deficiency
confirmed by genetic testing and in July and September 2021,
respectively, by the European Commission (EC) and Great
Britain’s Medicines & Healthcare Products Regulatory
Agency (MHRA) for the treatment of obesity and the control of
hunger associated with genetically confirmed loss-of-function
biallelic POMC, including PCSK1, deficiency or biallelic LEPR
deficiency in adults and children 6 years of age and above.
IMCIVREE is the first-ever FDA-approved and EC- and MHRA-authorized
therapy for patients with these rare genetic diseases of obesity.
The Company submitted a supplemental New Drug Application (sNDA) to
the FDA, which was accepted for filing in November
2021 and is currently assigned a Prescription Drug User Fee
Act (PDUFA) goal date of June 16, 2022, for the treatment of
obesity and control of hunger in adult and pediatric patients six
years of age and older with Bardet-Biedl Syndrome (BBS) or Alström
syndrome. A Type II variation application to the European
Medicines Agency seeking regulatory approval and authorization
for setmelanotide to treat obesity and control of hunger in adult
and pediatric patients 6 years of age and older with BBS also is
under review. Additionally, Rhythm is advancing a broad clinical
development program for setmelanotide in other rare genetic
diseases of obesity and is leveraging the Rhythm Engine and the
largest known obesity DNA database -- now with approximately 45,000
sequencing samples -- to improve the understanding, diagnosis and
care of people living with severe obesity due to certain genetic
deficiencies. Rhythm’s headquarters is in Boston, MA.
IMCIVREE®
(setmelanotide) IndicationIn the United
States, IMCIVREE is indicated for chronic weight management in
adult and pediatric patients 6 years of age and older with obesity
due to proopiomelanocortin (POMC), proprotein convertase
subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR)
deficiency. The condition must be confirmed by genetic testing
demonstrating variants in POMC, PCSK1,
or LEPR genes that are interpreted as pathogenic, likely
pathogenic, or of uncertain significance (VUS).
In the EU and Great Britain, IMCIVREE is indicated for the
treatment of obesity and the control of hunger associated with
genetically confirmed loss-of-function biallelic POMC, including
PCSK1, deficiency or biallelic LEPR deficiency in adults and
children 6 years of age and above. IMCIVREE should be prescribed
and supervised by a physician with expertise in obesity with
underlying genetic etiology.
Limitations of UseIMCIVREE is not indicated for
the treatment of patients with the following conditions as IMCIVREE
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, including obesity associated with other genetic
syndromes and general (polygenic) obesity.
Important Safety Information
WARNINGS AND PRECAUTIONS
Disturbance in Sexual Arousal: Sexual
adverse reactions may occur in patients treated with IMCIVREE.
Spontaneous penile erections in males and sexual adverse reactions
in females occurred in clinical studies with IMCIVREE. Instruct
patients who have an erection lasting longer than 4 hours to seek
emergency medical attention.
Depression and Suicidal Ideation: Some
drugs that target the central nervous system, such as IMCIVREE, may
cause depression or suicidal ideation. Monitor patients for new
onset or worsening of depression. Consider discontinuing IMCIVREE
if patients experience suicidal thoughts or behaviors.
Skin Pigmentation and Darkening of Pre-Existing
Nevi: IMCIVREE may cause generalized increased skin
pigmentation and darkening of pre-existing nevi due to its
pharmacologic effect. This effect is reversible upon
discontinuation of the drug. Perform a full body skin examination
prior to initiation and periodically during treatment with IMCIVREE
to monitor pre-existing and new skin pigmentary lesions.
Risk of Serious Adverse Reactions Due to Benzyl Alcohol
Preservative in Neonates and Low Birth Weight
Infants: IMCIVREE is not approved for use in neonates
or infants.
ADVERSE REACTIONS
- The most common adverse reactions (incidence ≥23%) were
injection site reactions, skin hyperpigmentation, nausea, headache,
diarrhea, abdominal pain, back pain, fatigue, vomiting, depression,
upper respiratory tract infection, and spontaneous penile
erection.
USE IN SPECIFIC POPULATIONSDiscontinue IMCIVREE
when pregnancy is recognized unless the benefits of therapy
outweigh the potential risks to the fetus.
Treatment with IMCIVREE is not recommended for use while
breastfeeding.
To report SUSPECTED ADVERSE REACTIONS, contact Rhythm
Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088
or www.fda.gov/medwatch.
See Full Prescribing Information, EU SmPC
and MHRA SmPC for IMCIVREE.
Forward-Looking
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 and our expectations surrounding potential
regulatory submissions, approvals and timing thereof, our business
strategy and plans, including regarding commercialization of
setmelanotide, sales of our lead product candidate IMCIVREE, our
participation in upcoming events and presentations, and the
sufficiency of our cash, cash equivalents and short-term
investments to fund our operations. Statements using word such as
“expect”, “anticipate”, “believe”, “may”, “will” and similar terms
are also forward-looking statements. Such statements are subject to
numerous risks and uncertainties, including, but not limited to,
the impact of our management transition, our ability to enroll
patients in clinical trials, the design and outcome of clinical
trials, the impact of competition, the ability to achieve or obtain
necessary regulatory approvals, risks associated with data analysis
and reporting, our liquidity and expenses, the impact of the
COVID-19 pandemic on our business and operations, including our
preclinical studies, clinical trials and commercialization
prospects, and general economic conditions, and the other important
factors discussed under the caption “Risk Factors” in our Quarterly
Report on Form 10-Q for the quarter ended March 31, 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.
Corporate
Contact:David ConnollyHead of Investor Relations and
Corporate CommunicationsRhythm Pharmaceuticals,
Inc.857-264-4280dconnolly@rhythmtx.com
Investor
Contact:Hannah DeresiewiczStern Investor Relations,
Inc.212-362-1200hannah.deresiewicz@sternir.com
Media Contact:Adam
DaleyBerry & Company Public
Relations212-253-8881adaley@berrypr.com
Rhythm Pharmaceuticals,
Inc.Condensed Consolidated Statements of
Operations and Comprehensive (Loss) Income(in
thousands, except share and per share
data)(Unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three months ended March 31, |
|
|
2022 |
|
|
2021 |
|
Product revenue, net |
|
$ |
1,498 |
|
|
$ |
35 |
|
Costs and expenses: |
|
|
|
|
|
|
Cost of sales |
|
|
230 |
|
|
|
4 |
|
Research and development |
|
|
32,510 |
|
|
|
19,911 |
|
Selling, general, and administrative |
|
|
21,449 |
|
|
|
14,518 |
|
Total costs and expenses |
|
|
54,189 |
|
|
|
34,433 |
|
Loss from operations |
|
|
(52,691 |
) |
|
|
(34,398 |
) |
Other income: |
|
|
|
|
|
|
Other income |
|
|
— |
|
|
|
100,000 |
|
Interest income, net |
|
|
(73 |
) |
|
|
154 |
|
Total other income, net |
|
|
(73 |
) |
|
|
100,154 |
|
(Loss) income before
taxes |
|
|
(52,764 |
) |
|
|
65,756 |
|
Provision for income
taxes |
|
|
— |
|
|
|
22,006 |
|
Net (loss) income |
|
$ |
(52,764 |
) |
|
$ |
43,750 |
|
Net (loss) income per
share |
|
|
|
|
|
|
Basic |
|
$ |
(1.05 |
) |
|
$ |
0.92 |
|
Diluted |
|
$ |
(1.05 |
) |
|
$ |
0.90 |
|
Weighted-average common shares
outstanding |
|
|
|
|
|
|
Basic |
|
|
50,326,627 |
|
|
|
47,638,565 |
|
Diluted |
|
|
50,326,627 |
|
|
|
48,501,697 |
|
|
|
|
|
|
|
|
Other comprehensive (loss)
income: |
|
|
|
|
|
|
Net (loss) income |
|
$ |
(52,764 |
) |
|
$ |
43,750 |
|
Unrealized (loss) on marketable securities |
|
|
(628 |
) |
|
|
(107 |
) |
Comprehensive (loss)
income |
|
$ |
(53,392 |
) |
|
$ |
43,643 |
|
Rhythm
Pharmaceuticals, Inc.Condensed Consolidated
Balance Sheets(in thousands, except share and per
share data)(Unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
March 31, |
|
December 31, |
|
|
2022 |
|
|
2021 |
|
|
|
|
|
|
|
|
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
78,537 |
|
|
$ |
59,248 |
|
Short-term investments |
|
|
162,427 |
|
|
|
235,607 |
|
Accounts receivable |
|
|
862 |
|
|
|
1,025 |
|
Prepaid expenses and other current assets |
|
|
11,623 |
|
|
|
12,507 |
|
Total current assets |
|
|
253,449 |
|
|
|
308,387 |
|
Property and equipment,
net |
|
|
2,722 |
|
|
|
2,813 |
|
Right-of-use asset |
|
|
1,442 |
|
|
|
1,522 |
|
Intangible assets, net |
|
|
8,527 |
|
|
|
4,658 |
|
Restricted cash |
|
|
328 |
|
|
|
328 |
|
Other long-term assets |
|
|
18,356 |
|
|
|
11,815 |
|
Total assets |
|
$ |
284,824 |
|
|
$ |
329,523 |
|
Liabilities and
stockholders’ equity |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Accounts payable |
|
$ |
12,010 |
|
|
$ |
5,737 |
|
Accrued expenses and other current liabilities |
|
|
25,199 |
|
|
|
30,084 |
|
Contract liability |
|
|
9,440 |
|
|
|
7,000 |
|
Lease liability |
|
|
625 |
|
|
|
606 |
|
Total current liabilities |
|
|
47,274 |
|
|
|
43,427 |
|
Long-term liabilities: |
|
|
|
|
|
|
Lease liability |
|
|
1,781 |
|
|
|
1,945 |
|
Total liabilities |
|
|
49,055 |
|
|
|
45,372 |
|
Stockholders’ equity: |
|
|
|
|
|
|
Common stock, $0.001 par value: 120,000,000 shares authorized;
50,393,731 and 50,283,574 shares issued and outstanding at
March 31, 2022 and December 31, 2021,
respectively |
|
|
50 |
|
|
|
50 |
|
Additional paid-in capital |
|
|
818,051 |
|
|
|
813,041 |
|
Accumulated other comprehensive loss |
|
|
(629 |
) |
|
|
(1 |
) |
Accumulated deficit |
|
|
(581,703 |
) |
|
|
(528,939 |
) |
Total stockholders’ equity |
|
|
235,769 |
|
|
|
284,151 |
|
Total liabilities and stockholders’ equity |
|
$ |
284,824 |
|
|
$ |
329,523 |
|
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