resTORbio Reports Fourth Quarter and Full Year 2019 Financial Results and Provides Corporate Update
“Although we experienced a setback following the discontinuation of our PROTECTOR program of RTB101 in preventing clinically symptomatic respiratory illness in older adults, we are encouraged by the recent positive interim results of the ongoing Phase 1b/2a trial of RTB101 alone and in combination with sirolimus in Parkinson’s disease,” said
Recent Corporate Highlights
Phase 1b/2a Trial of RTB101 alone and in combination with sirolimus in Parkinson’s disease
Interim results of the multicenter, 2:1 randomized, double-blind, placebo-controlled Phase 1b/2a trial evaluating the safety and tolerability of RTB101 alone or in combination with escalating doses of sirolimus once weekly for 4 weeks in patients with Parkinson’s disease indicated that the first 3 dosing regimens were well tolerated and RTB101 300 mg once weekly was observed to cross the blood brain barrier. The concentrations of RTB101 in cerebrospinal fluid (CSF) in subjects dosed with RTB101 300 mg once-weekly monotherapy were higher than expected and based on preclinical models, have the potential to induce autophagy in the brain. Sirolimus at the dose of 2 mg, alone or in combination with RTB101, was not detected in the CSF. Concentrations of RTB101 observed in the CSF four hours after dosing were highest when RTB101 was given as a monotherapy in the first three cohorts in the study. To date, patients have been enrolled in three cohorts and dosed once weekly with 300 mg of RTB101 alone, 2 mg of sirolimus alone, or a combination of 300 mg RTB101 and 2 mg of sirolimus. Enrollment of the RTB101 300 mg in combination with sirolimus 4 mg once weekly cohort is ongoing with results expected by mid-year 2020.
Fourth Quarter and Full Year 2019 Financial Results
- R&D Expenses: Research and development (R&D) expenses were $26.1 million for the three months ended December 31, 2019 and $73.6 million for the year ended December 31, 2019, as compared to
$4.3 millionfor the three months ended December 31, 2018and $31.1 millionfor the year ended December 31, 2018. The increase in R&D expenses year-over-year was primarily due to the Company’s Phase 3 PROTECTOR program for clinically symptomatic respiratory illness, now discontinued, and, to a lesser extent, the ongoing Phase 1b/2a for Parkinson’s disease.
- G&A Expenses: General and administrative (G&A) expenses were $3.3 million for the three months ended December 31, 2019 and $11.8 million for the year ended December 31, 2019, as compared to
$2.0 millionfor the three months ended December 31, 2018and $8.6 millionfor the year ended December 31, 2018. The increase in G&A expenses year-over-year was primarily due to an increase in headcount and facilities-related expenses.
- Net Loss: Net loss was $28.9 million, or $0.79 per share, for the three months ended December 31, 2019, and $82.7 million, or $2.41 per share, for the year ended December 31, 2019. Net loss was
$5.8 million, or $0.21per share, for the three months ended December 31, 2018, and $37.6 million, or $1.42per share, for the year ended December 31, 2018.
Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities were $91.5 million as of December 31, 2019, as compared to $108.0 millionas of December 31, 2018. The Company expects that current cash, cash equivalents and marketable securities as of December 31, 2019 will be sufficient to fund its operating expenses at least into 2022.
Strategic Review Progress Update
RTB101 is an oral, selective, and potent TORC1 inhibitor product candidate that inhibits the phosphorylation of multiple targets downstream of TORC1. Inhibition of TORC1 has been observed to extend lifespan and healthspan in aging preclinical species and to improve the function of aging organ systems, including neurologic function, suggesting potential benefits in several aging-related diseases.
resTORbio, Inc. is a clinical-stage biopharmaceutical company developing innovative medicines that target the biology of aging to treat aging-related diseases. resTORbio’s lead program selectively inhibits TORC1, an evolutionarily conserved pathway that contributes to the decline in function of aging organ systems, including neurologic function. Learn more about resTORbio, Inc. at www.resTORbio.com.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Investors are cautioned that statements in this press release which are not strictly historical statements, including, without limitation, our proposed timing and anticipated results of our Phase 1b/2a clinical trial of RTB101 alone or in combination with sirolimus in patients with mild to severe Parkinson’s disease, including the timing of the initiation and anticipated results of this trial; our future plans to develop RTB101 alone or in combination with rapalogs, such as everolimus or sirolimus, including the therapeutic potential and clinical benefits thereof; our expectations on the potential patient populations that may be addressed by our product candidates and the patient populations that may be addressed by our product candidates; our ability to replicate results achieved in our clinical trials in any future trials; the intended regulatory path for our product candidates and interactions with regulatory authorities; our engagement of JMP Securities LLC and our plans to explore and evaluate strategic alternatives and external opportunities; and our expectations regarding our uses of capital, expenses, future accumulated deficit and other 2019 financial results, and our ability to fund our operating expense at least into 2022, constitute forward-looking statements identified by words such as, but not limited to, “believe,” “expect,” “may,” “will,” “should,” “seek,” “anticipate,” or “could” and similar words or expressions.
Any forward-looking statements in this statement are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated, including, without limitation, risks associated with: our ongoing Phase 1b/2a clinical trial of RTB101 alone or in combination with sirolimus in Parkinson’s disease, including the announcement of interim results; the timing and anticipated results of our clinical trials; the risk that the results of our clinical trials will be predictive of future results in connection with future clinical trials; our ability to explore and evaluate strategic alternatives and external opportunities; the timing and outcome of our planned interactions with regulatory authorities; and obtaining, maintaining and protecting our intellectual property as well as those risks more fully discussed in the section entitled “Risk Factors” in the Annual Report on Form 10-K filed by resTORbio, Inc. with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements are neither historical facts nor assurances of future performance. Instead, they represent our beliefs, expectations, assumptions and views only as of today and should not be relied upon as representing our beliefs, expectations, assumptions and views as of any subsequent date. resTORbio explicitly disclaims any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except per share data)
|Three Months Ended December 31,||Year Ended December 31,|
|Research and development||$||26,111||$||4,349||$||73,634||$||31,065|
|General and administrative||3,325||2,011||11,823||8,640|
|Total operating expenses||29,436||6,360||85,457||39,705|
|Loss from operations||(29,436||)||(6,360||)||(85,457||)||(39,705||)|
|Other income (expense), net||551||629||2,754||2,117|
|Loss before income taxes||(28,885||)||(5,731||)||(82,703||)||(37,588||)|
|Income tax expense||5||26||36||26|
|Net loss per share —basic and diluted||$||(0.79||)||$||(0.21||)||$||(2.41||)||$||(1.42||)|
|Weighted-average number of common shares used in net loss per share — basic and diluted||36,444||28,051||34,306||26,439|
CONDENSED CONSOLIDATED BALANCE SHEETS
|December 31,||December 31,|
|Cash, cash equivalents and marketable securities||$||91,473||$||108,028|
|Prepaid expenses and other current assets||1,780||1,506|
|Total current assets||93,253||109,534|
|Property and equipment, net||414||321|
|Liabilities and stockholders' equity|
|Total current liabilities||12,199||5,716|
|Additional paid-in capital||235,777||175,635|
|Accumulated other comprehensive income (loss)||49||(41||)|
|Total stockholders' equity||81,698||104,204|
|Total liabilities and stockholders' equity||$||93,912||$||109,939|
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