-- Launched Uncovering Rare Obesity to facilitate diagnosis of rare genetic disorders of obesity --
-- Announced positive opinion by EMA on Orphan Drug Designation for setmelanotide for the treatment of patients with BBS --
-- Topline data from pivotal Phase 3 trials evaluating setmelanotide in POMC and LEPR deficiency obesity expected in the third quarter of 2019, expected to be followed by NDA submission --
BOSTON, July 29, 2019 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq:RYTM), a biopharmaceutical company focused on the development and commercialization of therapeutics 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, 2019.
“In recent months, we have made great strides toward achieving our foundational goals of changing the treatment paradigm for rare genetic disorders of obesity and delivering setmelanotide as the first-approved therapy for people living with these conditions,” said Keith Gottesdiener, M.D., Chief Executive Officer of Rhythm. “We look forward to building on this momentum in the months ahead. We are on track to announce topline data from our two pivotal trials in POMC and LEPR deficiency obesity in the third quarter of 2019, and we are actively preparing for our first new drug application.”
Dr. Gottesdiener continued, “We are making significant progress across the Rhythm Engine. Our GO-ID genotyping study is enrolling patients ahead of schedule, with several new sites coming online in the second quarter. We are receiving additional sequencing data through new collaborations with multiple biobanks, and our recently launched Uncovering Rare Obesity program is supporting patient identification and helping identify underlying genetic causes of obesity. As we expand our efforts to educate physicians and patient advocacy groups on these conditions and the potential benefits of genetic sequencing, we are building a new community of patients, families and health care providers focused on rare genetic disorders of obesity in both the United States and Europe.”
Second Quarter and Recent Business Highlights:
Upcoming Milestones:
Second Quarter 2019 Financial Results:
Year to Date Financial Results:
About Rhythm Pharmaceuticals
Rhythm is a biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. Rhythm is currently evaluating the efficacy and safety of setmelanotide, the company’s first-in-class MC4R agonist, in Phase 3 studies in patients with Pro-opiomelanocortin (POMC) deficiency obesity, Leptin receptor (LEPR) deficiency obesity, Bardet-Biedl syndrome, and Alström syndrome. The company 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.
Forward-Looking Statements
This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties, including statements regarding Rhythm’s anticipated timing for enrollment and design of clinical trials, the timing for filing of a new drug application, its ongoing efforts related to patient identification, the release of results of clinical trials, and its sufficiency of cash. 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, 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, and expenses, and other risks as may be detailed from time to time in our Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other reports we file 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, | ||||||||||||||||
2019 | 2018 | 2019 | 2018 | ||||||||||||||
Operating expenses: | |||||||||||||||||
Research and development | $ | 35,308 | $ | 8,584 | $ | 58,069 | $ | 20,870 | |||||||||
Selling, general, and administrative | 8,841 | 6,437 | 16,600 | 11,152 | |||||||||||||
Total operating expenses | 44,149 | 15,021 | 74,669 | 32,022 | |||||||||||||
Loss from operations | (44,149 | ) | (15,021 | ) | (74,669 | ) | (32,022 | ) | |||||||||
Other income (expense): | |||||||||||||||||
Interest income, net | 1,353 | 609 | 2,899 | 1,151 | |||||||||||||
Total other income: | 1,353 | 609 | 2,899 | 1,151 | |||||||||||||
Net loss and comprehensive loss | $ | (42,796 | ) | $ | (14,412 | ) | $ | (71,770 | ) | $ | (30,871 | ) | |||||
Net loss attributable to common stockholders | $ | (42,796 | ) | $ | (14,412 | ) | $ | (71,770 | ) | $ | (30,871 | ) | |||||
Net loss per share attributable to common stockholders, basic and diluted | $ | (1.24 | ) | $ | (0.52 | ) | $ | (2.08 | ) | $ | (1.12 | ) | |||||
Weighted average common shares outstanding, basic and diluted | 34,452,661 | 27,960,664 | 34,435,023 | 27,624,271 |
Rhythm Pharmaceuticals, Inc.
Condensed Consolidated Balance Sheets
(in thousands, except share and per share data)
June 30, | December 31, | |||||||
2019 | 2018 | |||||||
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 63,118 | $ | 49,542 | ||||
Short-term investments | 132,049 | 202,519 | ||||||
Prepaid expenses and other current assets | 8,334 | 6,628 | ||||||
Total current assets | 203,501 | 258,689 | ||||||
Property and equipment, net | 3,901 | 1,120 | ||||||
Right-of-use asset | 3,085 | — | ||||||
Restricted cash | 401 | 401 | ||||||
Total assets | $ | 210,888 | $ | 260,210 | ||||
Liabilities and stockholders’ equity | ||||||||
Current liabilities: | ||||||||
Accounts payable | $ | 6,565 | $ | 7,640 | ||||
Accrued expenses and other current liabilities | 19,146 | 5,942 | ||||||
Lease liability | 379 | — | ||||||
Total current liabilities | 26,090 | 13,582 | ||||||
Long-term liabilities: | ||||||||
Lease liability | 3,331 | — | ||||||
Deferred rent | — | 372 | ||||||
Total liabilities | 29,421 | 13,954 | ||||||
Commitments and contingencies | ||||||||
Stockholders’ equity: | ||||||||
Preferred Stock, $0.001 par value: 10,000,000 shares authorized; no shares issued and outstanding at June 30, 2019 and December 31, 2018, respectively | — | — | ||||||
Common stock, $0.001 par value: 120,000,000 shares authorized; 34,497,542 and 34,410,725 shares issued and outstanding June 30, 2019 and December 31, 2018, respectively | 34 | 34 | ||||||
Additional paid-in capital | 437,805 | 430,824 | ||||||
Accumulated deficit | (256,372 | ) | (184,602 | ) | ||||
Total stockholders’ equity | 181,467 | 246,256 | ||||||
Total liabilities and stockholders’ equity | $ | 210,888 | $ | 260,210 |
Corporate Contact:
David Connolly
Head of Investor Relations and Corporate Communications
Rhythm Pharmaceuticals, Inc.
857-264-4280
dconnolly@rhythmtx.com
Investor Contact:
Hannah Deresiewicz
Stern Investor Relations, Inc.
212-362-1200
hannah.deresiewicz@sternir.com
Media Contact:
Adam Daley
Berry & Company Public Relations
212-253-8881
adaley@berrypr.com