Rhythm Pharmaceuticals Reports Fourth Quarter and Full Year 2019 Financial Results
-- On track to complete rolling NDA submission to the FDA for setmelanotide in POMC and LEPR deficiency obesities in the first quarter of 2020 --
-- Granted orphan drug designation by the
-- Completed full enrollment in Phase 3 trial of setmelanotide in Bardet-Biedl and Alström syndromes in December; topline data expected in the fourth quarter of 2020 or early in the first quarter of 2021 --
--Rhythm Phase 2 Basket study actively enrolling multiple cohorts across five rare MC4R pathway-related disorders with potential
-- Ongoing genetic sequencing efforts have sequenced more than 25,000 people with early-onset, severe obesity --
“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
Fourth Quarter and Recent Business Highlights:
Pipeline and Recent Developments:
- Today, Rhythm announced that its ongoing genetic sequencing programs have now sequenced more than 25,000 people with early-onset, severe obesity. Rhythm’s sequencing efforts seek to uncover more rare genetic disorders of obesity and develop a better understanding of those disorders currently under study in its pivotal trials and Phase 2 Basket Study. Rhythm expects to provide an update on its genetic sequencing efforts in 2020.
- Today, Rhythm announced that the
European Commissionadopted the European Medicines Agency’s (EMA’s) Committee for Orphan Medicinal Products’ positive opinion and designated setmelanotide as an orphan medicinal product for the treatment of patients with Alström syndrome.
December 2019, Rhythm announced the completion of enrollment in its pivotal Phase 3 clinical trial evaluating setmelanotide in BBS and Alström syndrome. The Company enrolled 32 individuals with BBS and six individuals with Alström syndrome in the pivotal cohort.
November 2019, Rhythm presented late-breaking data from its two pivotal Phase 3 trials evaluating setmelanotide in POMC deficiency obesity and LEPR deficiency obesity at ObesityWeek 2019®, which highlighted the effect of setmelanotide on body mass index scores and certain cardiovascular parameters.
January 2020, Rhythm announced that Dr. Gottesdienerintends to step down as Chief Executive Officer, President and a member of the Company’s Board of Directors, following the NDA submission for POMC deficiency obesity and LEPR deficiency obesity, expected in the first quarter of 2020. Rhythm’s Board of Directors has initiated a search for a new chief executive officer.
October 2019, Rhythm completed a public offering of 9,324,324 shares of its common stock at a public offering price of $18.50per share, for aggregate gross proceeds of approximately $172.5 million, before underwriting discounts, commissions, and offering expenses.
- Rhythm remains on track to complete submission of its rolling NDA to the FDA for setmelanotide in patients with POMC deficiency obesity and LEPR deficiency obesity in the first quarter of 2020.
- Rhythm expects to submit a Marketing Authorization Application (MAA) to the EMA for setmelanotide in patients with POMC deficiency obesity and LEPR deficiency obesity in the second quarter of 2020.
- Rhythm expects to report topline data from its combined Phase 3 trial evaluating setmelanotide in BBS and Alström syndrome in the fourth quarter of 2020 or early in the first quarter of 2021.
- 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 Results:
- Cash Position: As of
December 31, 2019, cash, cash equivalents and short-term investments were $292.5 million, as compared to $252.1 millionas of December 31, 2018. This increase reflects net proceeds of $161.4 millionfrom Rhythm’s public offering of common stock in October 2019, partially offset by cash used to fund operating activities in 2019. Based on its current clinical development plans, Rhythm expects that its existing cash and cash equivalents and short-term investments will enable it to fund operations through at least the end of 2021.
- R&D Expenses: R&D expenses were
$24.8 millionin the fourth quarter of 2019 and $109.5 millionfor the year ended December 31, 2019, as compared to $18.8 millionin the fourth quarter of 2018 and $50.3 millionfor the year ended December 31, 2018. The year-over-year increase was primarily due to an increase of $34.6 millionrelated to Rhythm’s clinical trials, including an expansion of enrollment and opening of new trial sites for the ongoing Phase 3 study of setmelanotide in patients with BBS and Alström syndrome, the Phase 2 Basket Study, and the GO-ID genotyping study; as well as an increase of $11.8 millionrelated to translational research and genetic sequencing efforts designed to improve identification of patients with MC4R pathway deficiencies and pathway validation efforts; and an increase of $6.8 milliondue to the hiring of additional personnel in Medical Affairs as well as research and development. This increase was partially offset by a decrease of $4.4 milliondue to the non-cash expense related to the license acquired from Takedafor RM-853in March 2018.
- S,G&A Expenses: S,G&A expenses were
$9.4 millionfor the fourth quarter of 2019 and $36.6 millionfor the year ended December 31, 2019, as compared to $8.4 millionfor the fourth quarter of 2018 and $28.1 millionfor the year ended December 31, 2018. The year-over-year increase was primarily due to an increase of $6.9 millionin employee-related costs in connection with the full-year impact of employees hired in 2018, as well as new personnel in 2019, to support planned commercial activities, operations and finance.
- Net Loss: Net loss was
$33.0 millionfor the fourth quarter of 2019 and $140.7 millionfor the year ended December 31, 2019, or a net loss per basic and diluted share of $0.78and $3.86, respectively, as compared to a net loss of $25.5 millionfor the fourth quarter of 2018 and $74.1 millionfor the year ended December 31, 2018, or a net loss per basic and diluted share of $0.74and $2.39, respectively.
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 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 Alström 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
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
|Consolidated Statements of Operations
(in thousands, except share and per share data)
|Three months ended
|Research and development||$||24,810||$||18,763||$||109,450||$||50,337|
|Selling, general, and administrative||9,414||8,388||36,550||28,080|
|Total operating expenses||34,224||27,151||146,000||78,417|
|Loss from operations||(34,224||)||(27,151||)||(146,000||)||(78,417||)|
|Other income (expense):|
|Interest income, net||1,268||1,644||5,271||4,353|
|Total other income:||1,268||1,644||5,271||4,353|
|Net loss per common share, basic and diluted||$||(0.78||)||$||(0.74||)||$||(3.86||)||$||(2.39||)|
|Weighted average common shares outstanding, basic and diluted||42,213,180||34,400,916||36,422,450||31,004,047|
|Consolidated Balance Sheets
(in thousands, except share and per share data)
|Cash and cash equivalents||$||62,294||$||49,542|
|Prepaid expenses and other current assets||9,945||6,628|
|Total current assets||302,404||258,689|
|Property and equipment, net||3,671||1,120|
|Liabilities and stockholders’ equity|
|Accrued expenses and other current liabilities||13,530||5,942|
|Total current liabilities||24,417||13,582|
|Additional paid-in capital||606,307||430,824|
|Total stockholders’ equity||281,020||246,256|
|Total liabilities and stockholders’ equity||$||308,523||$||260,210|
Head of Investor Relations and Corporate Communications
Stern Investor Relations, Inc.
Berry & Company Public Relations
Source: Rhythm Pharmaceuticals, Inc.