- Net cash used in operating and investing activities was $20.7 million in the fourth quarter and $63.8 million for the full year of 2015; net proceeds of $128.6 million raised through January 2016 public equity offering added to $370.6 million year-end cash position provides strong foundation to advance a robust clinical pipeline
- Announced PRONTO, a global registration-directed Phase 2b trial of NEOD001 in previously-treated patients with AL amyloidosis and persistent cardiac dysfunction
- Presented positive Phase 1/2 multiple ascending dose results for NEOD001 at ASCO and EHA, and published the data in the Journal of Clinical Oncology; expansion cohort data expected in 2Q16
- Presented Phase 1 single ascending dose results for PRX002 at MDS demonstrating a robust, rapid, and dose- and time-dependent, statistically significant mean reduction in levels of free serum alpha-synuclein of up to 96% after a single dose
- Initiated Phase 1 single ascending dose study of PRX003 in healthy volunteers; results expected 2Q16
Dr. Schenk continued, "We look forward to the year ahead with milestones expected in each of our clinical stage programs, advancing our objective to bring novel disease-modifying therapies to patients."Full Year 2015 and Recent Highlights: NEOD001 is a monoclonal antibody for the potential treatment of AL amyloidosis:
- Published results from the Phase 1/2 clinical trial of NEOD001 in patients with AL amyloidosis and persistent organ dysfunction in the peer-reviewed Journal of Clinical Oncology. The paper is entitled, "First-in-Human Phase 1/2 Study of NEOD001 in Patients with Light Chain Amyloidosis and Persistent Organ Dysfunction".
- Announced plans to initiate PRONTO in October 2015, a randomized, double-blind global registration-directed Phase 2b trial of NEOD001 in previously-treated patients with AL amyloidosis and persistent cardiac dysfunction.
- At oral presentations in June 2015 at the American Society for Clinical Oncology Annual Meeting (ASCO) and the 20 th Congress of the European Hematology Association (EHA), reported clinical data from the multiple ascending dose portion of a Phase 1/2 trial demonstrating NEOD001-treated patients achieved more than double the cardiac and renal biomarker responses when compared to historical data in patients treated solely with off-label standard of care.
- Added a high-dose cohort to the Phase 1 multiple ascending dose study of PRX002 in patients with Parkinson's disease. The decision to add an additional cohort of patients is intended to inform the design and dosing levels of future PRX002 clinical studies.
- In a late-breaking session at the 19th International Congress of Parkinson's Disease and Movement Disorders (MDS), presented clinical results from a Phase 1 study of PRX002 in healthy volunteers demonstrating that PRX002 was safe and well tolerated at all doses. In addition, PRX002 demonstrated robust, rapid, and dose- and time-dependent, statistically significant mean reduction in levels of free serum alpha-synuclein of up to 96% after a single dose of PRX002.
- Announced an agreement with The Michael J. Fox Foundation for Parkinson's Research to accelerate the discovery and development of novel biomarkers for Parkinson's disease that facilitate therapeutic approaches targeting alpha-synuclein, including PRX002.
- Initiated a Phase 1 single ascending dose study for PRX003 in healthy volunteers.
- Presented preclinical results from a series of novel, conformation-specific protein immunotherapy antibodies that selectively bind to amyloidogenic (diseased) forms of the transthyretin (ATTR) protein at the First European Congress on Hereditary TTR Amyloidosis.
- Executed two public offerings that raised aggregate net proceeds of $131.5 million through the issuance of 3,795,000 ordinary shares in April 2015 as well as aggregate net proceeds of $128.6 million through the issuance of 2,587,500 ordinary shares in January 2016.
- Expanded the Board of Directors with the appointment of a seasoned executive, Anders Härfstrand, MD, PhD, who brings strategic expertise in leading both entrepreneurial and large commercial organizations.
- Expect topline results from the expansion cohort of the Phase 1/2 study in the second quarter of 2016.
- Expect to complete enrollment in the Phase 3 VITAL Amyloidosis Study in the second quarter of 2017.
- Expect topline results from the Phase 2b PRONTO trial in late 2017 - early 2018.
- Expect topline results from Phase 1 multiple ascending dose study in patients with Parkinson's disease in the fourth quarter of 2016. The study will remain blinded to us until completion, which we expect to occur following completion of the newly added dose cohort follow-up period.
- Expect topline results from Phase 1 single ascending dose study in healthy volunteers in the second quarter of 2016.
- Expect to initiate Phase 1 multiple ascending dose, proof-of-biology study in patients with psoriasis in the second quarter of 2016.
Research and development (R&D) expenses totaled $17.9 million and $58.4 million for the fourth quarter and full year of 2015, respectively, as compared to $10.1 million and $38.5 million for the fourth quarter and full year of 2014, respectively. The increase in R&D expenses was primarily due to increased external expenses related to clinical trials and increased personnel cost. R&D expenses included non-cash share-based compensation expense of $1.3 million and $4.3 million for the fourth quarter and full year of 2015, respectively, as compared to $0.6 million and $2.3 million for the fourth quarter and full year of 2014, respectively.General and administrative (G&A) expenses totaled $6.6 million and $23.1 million for the fourth quarter and full year of 2015, respectively, as compared to $5.0 million and $19.1 million for fourth quarter and full year of 2014, respectively. The increase in G&A expenses for the fourth quarter and full year was primarily due to increases in personnel costs and legal expenses. G&A expenses included non-cash share-based compensation expense of $1.9 million and $6.1 million in the fourth quarter and full year of 2015, respectively, as compared to $0.8 million and $3.3 million in the fourth quarter and full year of 2014, respectively. Total non-cash share-based compensation expense was $3.3 million and $10.4 million for the fourth quarter and full year of 2015, respectively, as compared to $1.4 million and $5.6 million for the fourth quarter and full year of 2014, respectively. As of December 31, 2015, Prothena had $370.6 million in cash and cash equivalents, which does not include the $128.6 million in net proceeds received through the January 2016 equity offering, and no debt. As of February 12, 2016, Prothena had approximately 34.3 million ordinary shares outstanding, which includes the issuance of approximately 2.6 million ordinary shares as part of the January 2016 offering.
The Company expects the full year 2016 net cash burn from operating and investing activities to be $105 to $115 million, ending the year with approximately $388 million in cash (mid-point). The estimated full year 2016 net cash burn from operating and investing activities is primarily driven by an estimated net loss of $132 to $149 million, which includes an estimated $18 million of non-cash share-based compensation expense.Upcoming Investor Conferences Members of the senior management team will present and participate in investor meetings at the following upcoming investor conferences:
- RBC Capital Markets 2016 Global Healthcare Conference on February 23, 2016 at 10:00 AM ET in New York, NY.
- Cowen and Company 36th Annual Health Care Conference on March 9, 2016 at 9:20 AM ET in Boston, MA.
- Future Leaders in the Biotech Industry on March 11, 2016 at 10:00 AM ET in New York, NY.
- Barclays Global Healthcare Conference on March 16, 2016 at 2:05 PM ET in Miami, FL.
To access the call via dial-in, please dial (877) 887-5215 (U.S. toll free) or (315) 625-3069 (international) five minutes prior to the start time and refer to conference ID number 33151950. A replay of the call will be available until February 25, 2016 via dial-in at (855) 859-2056 (U.S. toll free) or (404) 537-3406 (international), Conference ID Number 33151950.Forward-looking Statements This press release contains forward-looking statements. These statements relate to, among other things, whether our cash position can fund advancement of our clinical pipeline; whether we can advance on our objective of bringing disease-modifying therapies to patients; the timing of reporting results from the expansion cohort of our Phase 1/2 study, completing enrollment in The VITAL Amyloidosis Study, and reporting results from the PRONTO trial, all for NEOD001; the timing of reporting data from the Phase 1 multiple ascending dose study for PRX002; the timing of reporting data from the Phase 1 multiple ascending dose study and initiating a Phase 1 multiple ascending dose proof-of-biology study for PRX003; our anticipated net cash burn from operating and investing activities for 2016 and expected cash balance at the end of 2016; and our estimated net loss and non-cash share-based compensation expense for 2016. These statements are based on estimates, projections and assumptions that may prove not to be accurate, and actual results could differ materially from those anticipated due to known and unknown risks, uncertainties and other factors, including but not limited to the risks, uncertainties and other factors described in the "Risk Factors" sections of our Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 13, 2015, our subsequent Quarterly Reports on Form 10-Q filed with the SEC and our Annual Report on Form 10-K to be filed with the SEC for our fiscal year 2015. Prothena undertakes no obligation to update publicly any forward-looking statements contained in this press release as a result of new information, future events or changes in Prothena's expectations. PROTHENA CORPORATION PLC CONSOLIDATED STATEMENTS OF OPERATIONS (unaudited - amounts in thousands except per share data)
|Three Months Ended December 31,||Twelve Months Ended December 31,|
|Research and development||17,890||10,107||58,439||38,452|
|General and administrative||6,629||5,011||23,105||19,051|
|Total operating expenses||24,519||15,118||81,544||57,503|
|Loss from operations||(24,212||)||(13,105||)||(79,937||)||(6,649||)|
|Other income, net||57||118||26||310|
|Loss before income taxes||(24,155||)||(12,987||)||(79,911||)||(6,339||)|
|Provision for income taxes||2||123||701||811|
|Basic and diluted net loss per share||$||(0.76||)||$||(0.48||)||$||(2.66||)||$||(0.29||)|
|Shares used to compute basic and diluted net loss per share||31,611||27,384||30,326||24,672|
|December 31,||December 31,|
|Cash and cash equivalents||$||370,586||$||293,579|
|Other current assets||6,817||5,529|
|Total current assets||377,403||299,108|
|Property and equipment, net||3,862||3,121|
|Total non-current assets||7,833||5,008|
|Liabilities and Shareholders' Equity|
|Accrued research and development||12,794||2,285|
|Other current liabilities||9,422||9,754|
|Total current liabilities||22,216||12,039|
|Total shareholders' equity||360,669||289,889|
|Total liabilities and shareholders' equity||$||385,236||$||304,116|