Translate Bio Announces Third Quarter 2018 Financial Results and Reviews Recent Highlights
Continued progress with ongoing Phase 1/2 clinical trial in CF and towards IND submission for OTC Deficiency
“This past quarter, we made significant progress in advancing our programs, primarily focused on continuing to enroll and dose patients in our Phase 1/2 clinical trial of MRT5005 in cystic fibrosis,” said Ronald Renaud, Chief Executive Officer of Translate Bio. “We also completed the IND-enabling studies for MRT5201 and are compiling the Investigational New Drug Application which will support the anticipated Phase 1/2 clinical trial initiation in patients with OTC deficiency in the first half of 2019.”
Renaud continued, “Related to our discovery efforts, we are assessing data from our work in additional disease areas with the goal of advancing additional programs and leveraging the broad potential of our mRNA therapeutic platform.”
Third Quarter 2018 and Recent Updates
- Dosed multiple patients in Phase 1/2 clinical trial of MRT5005 for the treatment of CF:
Translate Biohas continued to enroll and dose patients in a first-in-human clinical trial of MRT5005, an mRNA therapeutic product candidate that is designed to treat all patients with cystic fibrosis (CF) by addressing the underlying cause of the disease, including those with limited or no cystic fibrosis transmembrane conductance regulator (CFTR) protein. The primary endpoint of the randomized, double-blind, placebo-controlled trial will be the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization. The trial is being conducted in collaboration with the Cystic Fibrosis Therapeutics Development Network.
- Completed IND-enabling studies of MRT5201 for the treatment of OTC Deficiency: With the goal of initiating a Phase 1/2 clinical trial in the first half of 2019,
Translate Biohas completed its IND-enabling studies for MRT5201, a first-in-class treatment designed to directly address the underlying cause of ornithine transcarbamylase (OTC) deficiency by providing mRNA encoding the fully functional OTC enzyme in patients with the disease. MRT5201 has been granted Orphan Drug Designation for for the treatment of OTC deficiency in the US and EU.
- Presented preclinical data supporting ongoing clinical trial at NACFC: Translate Bio’s Chief Medical Officer, Dr.
Ann Barbier, presented preclinical in vivo data in October 2018at the 32nd Annual North American Cystic Fibrosis Conference(NACFC) demonstrating MRT5005’s ability to reach the lungs in animal studies, including in a disease model with increased mucus accumulation, resulting in functional protein expression. These data support the potential use of MRT5005 as a treatment for all patients with CF, including those with limited or no CFTR protein.
- Closed collaboration and licensing agreement with
Sanofi Pasteurto develop mRNA vaccines for infectious diseases: In July 2018, Translate Bioannounced the closing of a multi-year research and development collaboration and exclusive licensing agreement with Sanofi Pasteurto develop mRNA vaccines for up to five undisclosed infectious disease pathogens. Upon closing in July, Translate Bioreceived an upfront payment of $45 million.
- MRT5005 (CF): Initiate multiple-ascending dose portion of the Phase 1/2 clinical trial by year end 2018
- MRT5201 (OTC Deficiency): Initiate Phase 1/2 clinical trial in the first half of 2019
- Identify lead preclinical candidates for additional lung and liver disease targets
- 6th International mRNA Conference on
Tuesday, November 13, 2018at 10:45 a.m. ET, Cambridge, MA(presentation)
- Stifel 2018 Healthcare Conference on Wednesday, November 14, 2018 at 8:00 a.m. ET, New
York, NY(presentation and meetings) Evercore ISI Healthcare Conferenceon Wednesday, November 28, 2018 at 12:50 p.m. ET, Boston, MA(presentation and meetings) Citi Global Healthcare Conferenceon Wednesday, December 5, 2018, New York, NY(meetings only)
Third Quarter 2018 Financial Results and Financial Guidance
Collaboration revenue was
Operating expenses for the quarter ended
- Research and development expenses of $12.9 million during the third quarter of 2018, compared to $11.1 million for the same period in 2017. The increase is primarily due to increases in personnel-related costs and an increase in costs associated with the continued development of the Company’s product candidates.
- General and administrative expenses of
$6.0 millionduring the third quarter of 2018, compared to $3.6 millionfor the same period in 2017. The increase is primarily due to an increase in personnel-related costs, professional fees and insurance costs.
- In the three months ended
September 30, 2018and 2017, the Company recognized operating expenses of $26.8 millionand $6.1 million, respectively, for changes in the fair value of the contingent consideration liabilities related to future potential milestone and earnout payment obligations and anti-dilution rights with respect to common stock issued to Shire that the Company recorded in connection with its acquisition of the MRT Program in December 2016. The $20.7 millionincrease in expense was attributed primarily to an increase in the fair value of the contingent consideration liability for future earnout payments that could be due.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, those regarding: the potential for MRT5005 to treat patients with CF; Translate Bio’s plans to continue to enroll and dose patients in its Phase 1/2 clinical trial of MRT5005 in CF and to initiate the multiple-ascending dose part of the study by the end of 2018; Translate Bio’s plans to submit an IND and initiate a Phase 1/2 clinical trial of MRT5201 in the first half of 2019; its plans to advance additional programs and leverage its mRNA therapeutic platform, including its plans to identify lead preclinical candidates for additional lung and liver disease targets; and the period in which
|TRANSLATE BIO, INC.|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS|
|Three Months Ended September 30,||Nine Months Ended September 30,|
|Research and development||12,933||11,097||40,854||34,224|
|General and administrative||5,957||3,607||16,726||9,706|
|Change in fair value of contingent consideration||26,829||6,132||39,589||10,731|
|Total operating expenses||45,719||20,836||97,169||54,661|
|Loss from operations||(45,481||)||(20,836||)||(96,931||)||(54,661||)|
|Other income (expense):|
|Other income (expense), net||(7||)||—||(52||)||59|
|Total other income (expense), net||311||72||447||289|
|Loss before benefit from income taxes||(45,170||)||(20,764||)||(96,484||)||(54,372||)|
|Benefit from income taxes||2,524||2,299||5,126||4,020|
|TRANSLATE BIO, INC.|
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|September 30,||December 31,|
|Cash and cash equivalents||$||38,864||$||48,058|
|Prepaid expenses and other current assets||4,194||3,014|
|Total current assets||166,319||63,035|
|Property and equipment, net||10,068||6,778|
|Intangible assets, net||106,842||106,842|
|Deferred offering costs||—||511|
|Liabilities, Redeemable Convertible Preferred Stock and Stockholders'|
|Current portion of contingent consideration||—||1,296|
|Current portion of deferred revenue||1,042||—|
|Total current liabilities||9,959||12,085|
|Long-term portion of contingent consideration||118,211||79,713|
|Deferred revenue, net of current portion||44,024||—|
|Deferred tax liabilities||913||6,039|
|Deferred rent, net of current portion||2,064||1,329|
|Redeemable convertible preferred stock (Series A, B and C)||—||192,896|
|Stockholders’ equity (deficit):|
|Additional paid-in capital||369,614||55,204|
|Accumulated other comprehensive income (loss)||(76||)||79|
|Total stockholders' equity (deficit)||129,417||(93,515||)|
|Total liabilities, redeemable convertible preferred|
|stock and stockholders’ equity (deficit)||$||304,588||$||198,547|
|Contacts for Translate Bio|
|Teri Dahlman||Maura Gavaghan|
Source: Translate Bio, Inc.