Full Press Release Details
Rhythm Pharmaceuticals Reports Fourth
Quarter and Full Year 2019 Financial Results
to complete rolling NDA submission to the FDA for setmelanotide in POMC and LEPR deficiency obesities in the first quarter of 2020
drug designation by the European Medicines Agency for setmelanotide in Alstr m syndrome --
full enrollment in Phase 3 trial of setmelanotide in Bardet-Biedl and Alstr m syndromes in December; topline data expected
in the fourth quarter of 2020 or early in the first quarter of 2021 -
2 Basket study actively enrolling multiple cohorts across five rare MC4R pathway-related disorders with potential U.S. prevalence
greater than 80,000 patients; data updates expected in 2020 --
sequencing efforts have sequenced more than 25,000 people with early-onset, severe obesity --
- March 2, 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 fourth quarter and full year ended December 31, 2019.
"We believe Rhythm is entering 2020
in a position of strength as we continue to make significant progress in the science and understanding of rare genetic disorders
of obesity and prepare to deliver setmelanotide as the first therapeutic option for people living with these conditions,"
said Keith Gottesdiener, M.D., Chief Executive Officer of Rhythm.
Murray Stewart, M.D., Chief Medical Officer,
added, "We expect to submit our first New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the coming
weeks. In parallel, we are focused on advancing the development of setmelanotide in five additional melanocortin-4 receptor (MC4R)
pathway-related disorders in our Phase 2 Basket study. We are also working to build a robust community of patients, caregivers
and healthcare providers in order to drive patient identification, increase genetic testing and improve the understanding of these
conditions. These efforts are critically important to our progress in Bardet-Biedl syndrome (BBS) and Alstr m syndrome, for
which we expect topline pivotal data in the fourth quarter of 2020 or early in the first quarter of 2021. Lastly, our ongoing sequencing
efforts continue to demonstrate that a significant percentage of severely obese individuals may be suffering from MC4R-related
disorders of severe obesity and unrelenting hunger, indicating a need for a therapy like setmelanotide that has the potential to
treat people living with these conditions."
Fourth Quarter and Recent Business Highlights:
Pipeline and Recent Developments:
Upcoming Milestones:
| - | Rhythm expects to announce additional data from its ongoing Phase 2 Basket Study of setmelanotide in high-impact heterozygous (HET) obesity and additional data from one or more of its other ongoing Phase 2 Basket Study indications in 2020. | |
| - | Rhythm expects to provide a clinical development update for its once-weekly formulation of setmelanotide in 2020. | |
| - | Rhythm expects to submit an investigational new drug (IND) application for RM-853, its ghrelin o-acyltransferase (GOAT) inhibitor for the treatment of Prader-Willi syndrome, to the FDA in 2020. | |
| - | Rhythm expects to provide an update on its genetic sequencing efforts in 2020. |
Fourth Quarter and Full Year 2019 Financial
Rhythm Pharmaceuticals
late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic
disorders of obesity. The company recently announced positive topline results from pivotal Phase 3 clinical trials of setmelanotide,
its MC4R agonist, in people living with POMC deficiency obesity and LEPR deficiency obesity, and plans to complete its first rolling
NDA submission to the FDA in the first quarter of 2020. Rhythm is also evaluating setmelanotide 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 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 an NDA, submission of an investigational
new drug application and submission of an MAA, its ongoing efforts related to patient identification and genetic sequencing, the
release of results of clinical trials and updates on patient enrollment, 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.
Consolidated Statements of Operations
(in thousands, except share and per
| Three months ended December 31, | Year ended December 31, | |||||||||||||||
| 2019 | 2018 | 2019 | 2018 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 24,810 | $ | 18,763 | $ | 109,450 | $ | 50,337 | ||||||||
| Selling, general, and administrative | 9,414 | 8,388 | 36,550 | 28,080 | ||||||||||||
| Total operating expenses | 34,224 | 27,151 | 146,000 | 78,417 | ||||||||||||
| Loss from operations | (34,224 | ) | (27,151 | ) | (146,000 | ) | (78,417 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Interest income, net | 1,268 | 1,644 | 5,271 | 4,353 | ||||||||||||
| Total other income: | 1,268 | 1,644 | 5,271 | 4,353 | ||||||||||||
| Net loss | $ | (32,956 | ) | $ | (25,507 | ) | $ | (140,729 | ) | $ | (74,064 | ) | ||||
| Net loss per common share, basic and diluted | $ | (0.78 | ) | $ | (0.74 | ) | $ | (3.86 | ) | $ | (2.39 | ) | ||||
| Weighted average common shares outstanding, basic and diluted | 42,213,180 | 34,400,916 | 36,422,450 | 31,004,047 |
Consolidated Balance Sheets
(in thousands, except share and per
| December 31, | December 31, | |||||||
| 2019 | 2018 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 62,294 | $ | 49,542 | ||||
| Short-term investments | 230,165 | 202,519 | ||||||
| Prepaid expenses and other current assets | 9,945 | 6,628 | ||||||
| Total current assets | 302,404 | 258,689 | ||||||
| Property and equipment, net | 3,671 | 1,120 | ||||||
| Right-of-use asset | 2,045 | - | ||||||
| Restricted cash | 403 | 401 | ||||||
| Total assets | $ | 308,523 | $ | 260,210 | ||||
| Liabilities and stockholders' equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 10,415 | $ | 7,640 | ||||
| Accrued expenses and other current liabilities | 13,530 | 5,942 | ||||||
| Lease liability | 472 | - | ||||||
| Total current liabilities | 24,417 | 13,582 | ||||||
| Long-term liabilities: | ||||||||
| Lease liability | 3,086 | - | ||||||
| Deferred rent | - | 372 | ||||||
| Total liabilities | 27,503 | 13,954 | ||||||
| Stockholders' equity: | ||||||||
| Preferred Stock, $0.001 par value: 10,000,000 shares authorized; no shares issued and outstanding at December 31, 2019 and 2018 | - | - | ||||||
| Common stock, $0.001 par value: 120,000,000 shares authorized; 43,996,753 and 34,410,725 shares issued and outstanding December 31, 2019 and 2018, respectively | 44 | 34 | ||||||
| Additional paid-in capital | 606,307 | 430,824 | ||||||
| Accumulated deficit | (325,331 | ) | (184,602 | ) | ||||
| Total stockholders' equity | 281,020 | 246,256 | ||||||
| Total liabilities and stockholders' equity | $ | 308,523 | $ | 260,210 |
Head of Investor Relations and Corporate Communications
Rhythm Pharmaceuticals, Inc.
Stern Investor Relations, Inc.
Berry & Company Public Relations