Full Press Release Details
Rhythm Pharmaceuticals
Reports Second Quarter 2020 Financial Results
Appointed David Meeker, M.D., as President and Chief Executive Officer -
FDA granted rare pediatric disease designations for setmelanotide for the treatment of POMC and LEPR deficiency obesities --
Submitted MAA to EMA for setmelanotide in POMC and LEPR deficiency obesities --
Received FDA acceptance of NDA for setmelanotide for POMC and LEPR deficiency obesities for filing; assigned PDUFA goal date of
On track to announce topline data from pivotal Phase 3 trial of setmelanotide in BBS and Alstr m syndrome in fourth quarter
of 2020 or early in the first quarter of 2021 --
Boston, MA - August 3, 2020 -
Rhythm Pharmaceuticals, Inc. (Nasdaq:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies
for the treatment of rare genetic disorders of obesity, today reported financial results and provided a business update for the
second quarter ended June 30, 2020.
"Rhythm has made tremendous regulatory
and clinical progress on its path to bring the first approved therapy to individuals living with rare genetic disorders of obesity,"
said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. "The potential approval of setmelanotide
for the treatment of pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesities will validate the melanocortin-4
receptor (MC4R) pathway as an important therapeutic target, and we look forward to furthering our ongoing efforts, with pivotal
data from our Phase 3 clinical trial in Bardet-Biedl syndrome (BBS) and Alstr m syndromes expected in the fourth quarter
or early in 2021 and the continued expansion of our ongoing Phase 2 Basket study."
Dr. Meeker continued, "We are energized
by the opportunity to help individuals living with rare genetic disorders of obesity, who currently have no meaningful treatment
options for their severe obesity or insatiable hunger. Looking ahead, we are eager to complete our transformation into an integrated,
patient-focused organization while continuing to advance our community building and patient engagement efforts."
Second Quarter 2020 and Recent Business
Pipeline and Recent Developments:
POMC and LEPR Deficiency Obesities
Additional Development Pipeline Updates
Upcoming Milestones:
| Rhythm is on track to report topline data from its combined pivotal Phase 3 trial evaluating setmelanotide in BBS and Alstr m syndrome in the fourth quarter of 2020 or early in the first quarter of 2021. | ||
| Rhythm expects to announce additional data in 2020 from its ongoing Phase 2 Basket Study of setmelanotide in high-impact heterozygous (HET) obesity and additional data from one or more of the other disorders being studied in its Phase 2 Basket Study. | ||
| Rhythm expects to provide an update on its genetic sequencing efforts in 2020. |
Second Quarter 2020 Financial Results:
| R&D Expenses: R&D expenses were $22.9 million for the second quarter of 2020 as compared to $35.3 million for the second quarter of 2019. The decrease in R&D spending was primarily attributed to a $6.3 million reduction in clinical trial expenses associated with the Company's GO-ID genotyping study and the once-weekly formulation study incurred with site initiations in 2019, a decrease of $4.0 million related to genetic sequencing with expected lower volume in the same study, and a $3.4 million decrease in expenses from purchases of setmelanotide API in the same quarter last year. These decreases were partially offset by an increase of $1.7 million in spending related to the Phase 2 Basket Study and a $2.0 million milestone payment associated with the license agreement with Ipsen on filing the NDA for setmelanotide for the treatment of POMC and LEPR deficiency obesities. | ||
| S,G&A Expenses : S,G&A expenses were $8.9 million for the second quarter of 2020 as compared to $8.8 million for the second quarter of 2019. This modest increase was primarily due to increased consulting and professional services fees. | ||
| Net Loss: Net loss was $31.1 million for the second quarter of 2020, or a net loss per basic and diluted share of $0.71, as compared to a net loss of $42.8 million for the second quarter of 2019, or a net loss per basic and diluted share of $1.24. |
Year to Date 2020 Financial Results:
Rhythm Pharmaceuticals
Rhythm is a late-stage biopharmaceutical
company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity.
The FDA has accepted for filing an NDA for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity
with Priority Review and assigned a PDUFA goal date of November 27, 2020. Rhythm also submitted an MAA for setmelanotide to treat
individuals living with POMC deficiency obesity or LEPR deficiency obesity to the EMA in June 2020. Rhythm is also evaluating
setmelanotide for reduction in hunger and body weight in a pivotal Phase 3 trial in people living with Bardet-Biedl and Alstr m
syndromes, with topline data from this trial expected in the fourth quarter of 2020 or early in the first quarter of 2021. Rhythm
is leveraging the Rhythm Engine -- comprised of its Phase 2 basket study, TEMPO Registry, GO-ID genotyping study and Uncovering
Rare Obesity program -- to improve the understanding, diagnosis and potentially the treatment of rare genetic disorders of obesity.
For healthcare professionals, visit www.UNcommonObesity.com for more information. For patients and caregivers, visit www.LEADforRareObesity.com
for more information. The company is based in Boston, MA.
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, expectations regarding
regulatory approval, the anticipated timing for release of clinical trial data, our ongoing efforts related to patient identification
and genetic sequencing and timing thereof, and the 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 quarterly period ended June 30, 2020 and our other filings with the Securities and Exchange Commission.
Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this
release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of
new information, future developments or otherwise.
Rhythm Pharmaceuticals, Inc.
Condensed Consolidated Statements
of Operations and Comprehensive Loss
(in thousands, except
share and per share data)
| Three months ended June 30, | Six months ended June 30, | |||||||||||||||
| 2020 | 2019 | 2020 | 2019 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 22,997 | $ | 35,308 | $ | 45,501 | $ | 58,069 | ||||||||
| Selling, general, and administrative | 8,921 | 8,841 | 21,717 | 16,600 | ||||||||||||
| Total operating expenses | 31,918 | 44,149 | 67,218 | 74,669 | ||||||||||||
| Loss from operations | (31,918 | ) | (44,149 | ) | (67,218 | ) | (74,669 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Interest income, net | 801 | 1,353 | 1,937 | 2,899 | ||||||||||||
| Total other income, net | 801 | 1,353 | 1,937 | 2,899 | ||||||||||||
| Net loss | $ | (31,117 | ) | $ | (42,796 | ) | $ | (65,281 | ) | $ | (71,770 | ) | ||||
| Net loss per share, basic and diluted | $ | (0.71 | ) | $ | (1.24 | ) | $ | (1.48 | ) | $ | (2.08 | ) | ||||
| Weighted-average common shares outstanding, basic and diluted | 44,098,860 | 34,452,661 | 44,074,352 | 34,435,023 | ||||||||||||
| Other comprehensive loss: | ||||||||||||||||
| Net loss | $ | (31,117 | ) | $ | (42,796 | ) | $ | (65,281 | ) | $ | (71,770 | ) | ||||
| Unrealized gain on marketable securities | 567 | - | 630 | - | ||||||||||||
| Comprehensive loss | $ | (30,550 | ) | $ | (42,796 | ) | $ | (64,651 | ) | $ | (71,770 | ) |
Rhythm Pharmaceuticals, Inc.
Condensed Consolidated Balance Sheets
(in thousands, except
| June 30, | December 31, | |||||||
| 2020 | 2019 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 59,091 | $ | 62,294 | ||||
| Short-term investments | 169,535 | 230,165 | ||||||
| Prepaid expenses and other current assets | 9,193 | 9,945 | ||||||
| Total current assets | 237,819 | 302,404 | ||||||
| Property and equipment, net | 3,331 | 3,671 | ||||||
| Right-of-use asset | 1,932 | 2,045 | ||||||
| Restricted cash | 403 | 403 | ||||||
| Total assets | $ | 243,485 | $ | 308,523 | ||||
| Liabilities and stockholders' equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 4,761 | $ | 10,415 | ||||
| Accrued expenses and other current liabilities | 9,640 | 13,530 | ||||||
| Lease liability | 503 | 472 | ||||||
| Total current liabilities | 14,904 | 24,417 | ||||||
| Long-term liabilities: | ||||||||
| Lease liability | 2,828 | 3,086 | ||||||
| Total liabilities | 17,732 | 27,503 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders' equity: | ||||||||
| Preferred Stock, $0.001 par value: 10,000,000 shares authorized; no shares issued and outstanding at June 30, 2020 and December 31, 2019 | - | - | ||||||
| Common stock, $0.001 par value: 120,000,000 shares authorized; 44,115,612 and 43,996,753 shares issued and outstanding at June 30, 2020 and December 31, 2019, respectively | 44 | 44 | ||||||
| Additional paid-in capital | 615,691 | 606,307 | ||||||
| Accumulated other comprehensive income | 630 | - | ||||||
| Accumulated deficit | (390,612 | ) | (325,331 | ) | ||||
| Total stockholders' equity | 225,753 | 281,020 | ||||||
| Total liabilities and stockholders' equity | $ | 243,485 | $ | 308,523 |
Head of Investor Relations and Corporate Communications
Rhythm Pharmaceuticals, Inc.
Stern Investor Relations, Inc.
Berry & Company Public Relations