Rhythm Pharmaceuticals Reports First Quarter 2018 Financial Results
-- Entered Licensing Agreement with Takeda for
“Our achievements year-to-date reflect our dual commitment to addressing the unmet medical needs of people living with rare genetic disorders of obesity and raising awareness and understanding of these conditions,” said
Dr. Gottesdiener continued, “We are also pleased that the
Recent Business Highlights:
- Rhythm today announced that the
FDAhas agreed that BBS and Alström Syndrome, two rare genetic disorders of obesity for which there are currently no effective or approved therapeutics, are included under the previously granted BTD for setmelanotide. The FDApreviously granted BTD to setmelanotide for the treatment of POMC and LEPR deficiency obesity.
May 2018, longer-term data from Rhythm’s Phase 2 open-label study of setmelanotide in patients with LEPR deficiency obesity were published online in Nature Medicine. All three patients treated with setmelanotide experienced reductions in excess hunger, also referred to as hyperphagia, and body-weight. The data show that setmelanotide was well-tolerated. The publication also detailed the results of in vitro analyses that provide insight into setmelanotide’s activity at the MC4R, suggesting that it may rescue specific MC4R mutations where the natural ligand cannot.
May 2018, the results of new genetic epidemiological analyses of known and predicted loss of function variants in the POMC, PCSK1 and LEPR genes were published online in the Journal of Clinical Endocrinology and Metabolism. This publication builds on data previously presented by Rhythm in a late-breaking poster presentation at ENDO2018 in April, suggesting that there are potentially more than 12,000 people living in the U.S. with rare genetic disorders of obesity.
April 2018, Rhythm acquired exclusive, worldwide rights from Takeda Pharmaceutical Company Limitedto develop and commercialize RM-853, a potent, orally available GOAT inhibitor currently in preclinical development for PWS. In preclinical research, RM-853prevented body weight gain and reduced fat mass in high fat-fed mice, with a favorable pharmacokinetic, pharmacodynamic, and safety profile. Rhythm continues to assess opportunities to further evaluate setmelanotide in PWS and plans to pursue those in parallel with the development of RM-853. Additionally, given setmelanotide and RM-853’s distinct mechanisms of action, Rhythm will explore opportunities to evaluate the two compounds in combination, as there may be complementary effects.
- Rhythm expects to complete enrollment of ten patients in its ongoing pivotal Phase 3 trial of setmelanotide in patients with POMC deficiency obesity in the first half of 2018. Rhythm expects to announce initial data from the trial in the first half of 2019, and to subsequently file an NDA with the
FDAbased on one-year data from the cohort of ten patients.
- Rhythm expects to complete enrollment of ten patients in its ongoing pivotal Phase 3 trial of setmelanotide in patients with LEPR deficiency obesity by the end of 2018.
- Rhythm expects to announce initial data from its ongoing Phase 2 proof-of-concept basket study of setmelanotide in each of Alström Syndrome, POMC epigenetic disorders, and POMC heterozygous deficiency obesity in the first half of 2018.
- Rhythm expects to initiate a pivotal Phase 3 clinical trial evaluating setmelanotide for the treatment of BBS in 2018.
- Rhythm expects to complete preclinical studies of
RM-853and file an IND with the FDAin the first quarter of 2020.
First Quarter 2018 Financial Results:
- Cash Position: As of
March 31, 2018, cash, cash equivalents and short-term investments were $136.5 million, as compared to $148.1 millionas of December 31, 2017. This decrease reflects cash used to fund operating activities in the first quarter of 2018.
- R&D Expenses: R&D expenses were
$12.3 millionfor the first quarter of 2018 as compared to $4.9 millionfor the first quarter of 2017. The increase was primarily due to non-cash expenses related to the license acquired from Takeda for RM-853, a $1 millionmilestone expense associated with the license agreement with Ipsen Pharma S.A.S. and the hiring of additional personnel in the clinical and development departments in the second half of 2017.
- S,G&A Expenses: S,G&A expenses were
$4.7 millionfor the first quarter of 2018 as compared to $1.5 millionfor the first quarter of 2017. The increase was primarily due to increased headcount, the development and building of our commercial organization to drive patient identification, as well as increased professional and consulting fees associated with being a public company.
- Net Loss: Net loss was
$16.5 millionfor the first quarter of 2018, or a net loss per basic and diluted share of $0.60, as compared to a net loss of $7.5 millionfor the first quarter of 2017, or a net loss per basic and diluted share of $0.74.
- Based on its current clinical development plans, Rhythm expects that its existing cash and cash equivalents and short-term investments will be sufficient for Rhythm to fund its operations into the second half of 2019.
Rhythm is a biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. Rhythm is currently evaluating the efficacy and safety of setmelanotide, the Company’s first-in-class melanocortin-4 receptor (MC4R) agonist, in Phase 3 studies in patients with pro-opiomelanocortin (POMC) deficiency obesity (which includes deficiencies in both the POMC and PCSK1 genes) and leptin receptor (LEPR) deficiency obesity. Rhythm also supports
This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties, including statements regarding Rhythm’s expectations regarding development of
RHYTHM PHARMACEUTICALS, INC.
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(in thousands, except share and per share data)
|Three months ended March 31,|
|Research and development||$||12,286||$||4,873|
|Selling, general, and administrative||4,715||1,516|
|Total operating expenses||17,001||6,389|
|Loss from operations||(17,001||)||(6,389||)|
|Other income (expense):|
|Interest income, net||542||29|
|Total other income (expense):||542||29|
|Net loss and comprehensive loss||$||(16,459||)||$||(6,360||)|
|Net loss attributable to common stockholders||$||(16,459||)||$||(7,526||)|
|Net loss attributable to common stockholders per common share, basic and diluted||$||(0.60||)||$||(0.74||)|
|Weighted average common shares outstanding, basic and diluted||27,284,140||10,196,292|
RHYTHM PHARMACEUTICALS, INC.
CONSOLIDATED BALANCE SHEETS
(in thousands, except share and per share data)
|Cash and cash equivalents||$||43,167||$||34,236|
|Prepaid expenses and other current assets||1,961||2,589|
|Total current assets||138,414||150,671|
|Property, plant and equipment, net||779||840|
|Liabilities, convertible preferred stock and stockholders’ equity|
|Accrued expenses and other current liabilities||3,038||4,210|
|Total current liabilities||5,579||6,720|
|Commitments and contingencies|
|Convertible Preferred Stock, $0.001 par value: 10,000,000 shares authorized; no shares issued and outstanding at March 31, 2018 and December 31, 2017, respectively||—||—|
|Common stock, $0.001 par value: 120,000,000 shares authorized; 27,284,140 shares issued and outstanding March 31, 2018 and December 31, 2017, respectively||27||27|
|Additional paid-in capital||260,342||255,013|
|Total stockholders’ equity||133,658||144,788|
|Total liabilities, convertible preferred stock and stockholders’ equity||$||139,443||$||151,736|
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