Eidos Therapeutics Reports Third Quarter 2019 Financial Results and Business Update
“We continue to focus on executing our Phase 3 program studying AG10 in patients with transthyretin (TTR) amyloidosis (ATTR). Our phase 3 study in ATTR-cardiomyopathy (ATTR-CM) is enrolling patients in the US and
Recent Achievements and Upcoming Milestones
Alexion Pharmaceuticals, Inc.an exclusive license to develop and commercialize AG10 in Japanfor an upfront payment of $25 millionand an equity investment of $25 million.
- Continued enrollment in Phase 3 study of AG10 in ATTR-CM (ATTRibute-CM).
- Plan to present interim analysis of the ongoing Phase 2 open label extension study of AG10 in patients with TTR amyloid cardiomyopathy at the AHA 2019 Scientific Sessions in a Late-Breaking Featured Science Oral Presentation.
- Plan to initiate Phase 3 study of AG10 in ATTR-PN (ATTRibute-PN) in the first quarter of 2020.
Eidos also announced the transition plan of
Third Quarter Financial Results
Cash and cash equivalents totaled
Eidos reported a net income attributable to common stockholders of $6.9 million or
Research and development expenses for the third quarter of 2019 were $12.0 million, as compared to $8.4 million for the same period in the prior year. Research and development expenses for the period included costs related to contract manufacturing, and the preparation for, and the increase in, activity related to our clinical trials.
General and administrative expenses for the third quarter of 2019 were $6.0 million, as compared to $2.6 million for the same period in the prior year. The increase in general and administrative expense in these periods was due primarily to an increase in one-time charges, professional service fees, salaries and employee-related expense primarily due to an increase in headcount to support the growth of our operations, and other administrative expenses.
Nine Months Ended
Eidos reported a net loss attributable to common stockholders of $18.9 million or
Research and development expenses for the nine months ended
General and administrative expenses for the nine months ended
AG10 is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin, or TTR, thereby halting at its outset the series of molecular events that give rise to amyloidosis, or ATTR. In a Phase 2 clinical trial in subjects with symptomatic ATTR-CM, AG10 was generally well tolerated, demonstrated >90% average TTR stabilization at day 28, and increased serum TTR concentrations, a prognostic indicator of survival in a retrospective study of ATTR-CM patients, in a dose-dependent manner. AG10 is currently being studied in an open-label extension of a Phase 2 clinical trial in patients with ATTR-CM, and patient enrollment is ongoing for a Phase 3 clinical trial of AG10 in patients with ATTR-CM (ATTRibute-CM).
AG10 was designed to mimic a naturally-occurring variant of the TTR gene (T119M) that is considered a rescue mutation because co-inheritance has been shown to prevent ATTR in individuals also inheriting a pathogenic, or disease-causing, mutation in the TTR gene. To our knowledge, AG10 is the only TTR stabilizer in development that has been observed to mimic the stabilizing structure of this rescue mutation.
About transthyretin amyloidosis (ATTR)
ATTR represents a significant unmet medical need with a large patient population and an inadequate current standard of care. ATTR is caused by the destabilization of TTR due to inherited mutations or aging and is commonly divided into three distinct categories: wild-type ATTR cardiomyopathy (ATTRwt-CM), mutant ATTR cardiomyopathy (ATTRm-CM), and ATTR polyneuropathy (ATTR-PN). The worldwide prevalence of each disease is approximately 400,000 patients, 40,000 patients and 10,000 patients, respectively.
All three forms of ATTR are progressive and fatal. For patients with ATTRwt-CM and ATTRm-CM, symptoms usually manifest later in life (age 50+), with median survival of three to five years from diagnosis. ATTR-PN either presents in a patient's early 30s or later (age 50+), and results in a median life expectancy of five to ten years from diagnosis. Progression of all forms of ATTR causes significant morbidity, impacts productivity and quality of life, and creates a significant economic burden due to the costs associated with progressively greater patient needs for supportive care.
This release includes forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act. All statements other than statements of historical facts, including the statements about the potential therapeutic and clinical benefits of AG10, the potential to accelerate the development and registration of AG10, our ability to enroll patients in and conduct the ATTRibute-CM trial in accordance with our plans, our plan to initiate a Phase 3 study of AG10 in ATTR-PN, our ability to generate data from the open label extension of our Phase 2 study of AG10 in ATTR-CM, future clinical and regulatory milestones of AG10, our chief financial officer transition plan, the timing of these events, the indications we intend to pursue and our possible clinical or other business strategies, and our ability to fund our clinical development plans, are forward-looking statements. Forward-looking statements can be identified by terms such as “believes,” “expects,” “plans,” “potential,” “would” or similar expressions and the negative of those terms. These forward-looking statements are based on our management’s current beliefs and assumptions about future events and on information currently available to management. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These risks include, but are not limited to, risks and uncertainties related to: our limited operating history and historical losses, our liquidity to fund the development of AG10 through current and future milestones, our ability to raise additional funding to complete the development of AG10, our dependence on the success of AG10, our ability to enroll patients in the ATTRibute-CM trial, results from our clinical trials and pre-clinical studies and those of third parties working in the same area as our product candidate, our ability to advance AG10 in clinical development in accordance with our plans, and our dependence on third parties in connection with our manufacturing, clinical trials and pre-clinical studies. Additional risks and uncertainties that could affect our future results are included in the section titled “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our Quarterly Report on Form 10-Q for the quarter ended
Condensed Statements of Operations
(In thousands, except share and per share data)
|Three Months Ended||Nine months ended|
|September 30,||September 30,|
|Cost of license revenue||2,500||2,500|
|Research and development||11,987||8,369||33,033||20,216|
|General and administrative||5,953||2,619||12,285||6,858|
|Total operating expenses||20,440||10,988||47,818||27,074|
|Income (loss) from operations||6,251||(10,988||)||(21,127||)||(27,074||)|
|Other income (expense), net||680||374||2,272||(3,797||)|
|Net income (loss) and comprehensive income (loss)||6,931||(10,614||)||(18,855||)||(30,871||)|
|Deemed dividend related to redemption feature embedded in Convertible Promissory Notes payable to stockholders||-||-||-||(6,523||)|
|Gain on extinguishment of Convertible Promissory Notes payable to stockholders||-||-||-||7,436|
|Net income (loss) attributable to common stockholders||$||6,931||$||(10,614||)||$||(18,855||)||$||(29,958||)|
|Net income (loss) per share attributable to common stockholders, basic||$||0.19||$||(0.30||)||$||(0.52||)||$||(1.83||)|
|Net income (loss) per share attributable to common stockholders, diluted||$||0.18||$||(0.30||)||$||(0.52||)||$||(1.83||)|
|Weighted-average shares used in computing net income (loss) per share attributable to common stockholders, basic||36,581,786||35,965,790||36,356,675||16,361,349|
|Weighted-average shares used in computing net income (loss) per share attributable to common stockholders, diluted||37,710,734||35,965,790||36,356,675||16,361,349|
|* Includes stock-based compensation as follows|
|Research and development||$||626||$||251||$||1,630||$||872|
|General and administrative||969||443||2,095||829|
|Total stock-based compensation expense||$||1,595||$||694||$||3,725||$||1,701|
Condensed Balance Sheets
|September 30,||December 31,|
|Cash and cash equivalents||$||165,822||$||157,147|
|Related party receivable||83||34|
|Prepaid expenses and other current assets||5,402||1,789|
|Total current assets||171,307||158,970|
|Property and equipment, net||1,199||209|
|Operating lease, right of use asset||4,121||-|
|Liabilities and Stockholders’ Equity|
|Related party payable||372||256|
|Accrued expenses and other current liabilities||5,665||2,577|
|Total current liabilities||10,075||4,789|
|Lease liabilities, non-current||4,736||-|
|Additional paid-in capital||248,041||220,240|
|Total stockholders’ equity||163,954||155,007|
|Total liabilities and stockholders’ equity||$||178,894||$||160,112|
Source: Eidos Therapeutics, Inc.