powerhouse : FibroGEn Inc. - der Neue Star am Biotech Himmel?
"FibroGen made excellent progress in 2014 on our lead clinical programs in anemia, fibrosis, and cancer," said Thomas Neff, chief executive officer of FibroGen. "We and our partners in the global development of roxadustat reached agreement on final protocols and are enrolling seven Phase 3 clinical trials of roxadustat for anemia in chronic kidney disease (CKD) in the US, Europe, and Asia Pacific, excluding Japan and China. Clinical trials of idiopathic pulmonary fibrosis and Stage 3 pancreatic cancer are on track, and we are now expanding our fibrosis program to include Duchenne muscular dystrophy."
"We ended 2014 with cash, cash equivalents, investments, and receivables of $346.8 million. As part of this amount, we received net proceeds of $171.8 million from our November 2014 initial public offering."
"In 2015, we expect to reach the cap on shared costs agreed upon with AstraZeneca for roxadustat CKD anemia development costs for the territories outside of China, after which, the costs we incur are reimbursed by our partners, as is described elsewhere in this press release."
"In addition, we expect non-contingent license and milestone payments of nearly $200 million in the next 15 months. We remain confident that our roxadustat CKD anemia program is fully funded through the planned Phase 3 clinical development program."
Recent Development Update
Roxadustat in Anemia of Chronic Kidney Disease
Program remains on schedule to submit regulatory filings in 2016 for China and 2018 for U.S.
Launch of seven Phase 3 studies for U.S. and European filing through collective efforts of FibroGen and its partners, AstraZeneca and Astellas.
Phase 3 program expected to enroll approximately 7,300 dialysis and pre-dialysis patients.
Completion of two carcinogenicity studies, one in rats and one in mice, and two independent pathology reviews of these studies. Final reports were audited and approved by all parties. The reports showed no carcinogenic effect of roxadustat.
FG-3019 in Idiopathic Pulmonary Fibrosis
Study 067 - Ongoing Phase 2b randomized, placebo-controlled study of FG-3019 in patients with idiopathic pulmonary fibrosis.
Protocol was amended to include subjects previously treated with recently approved IPF therapies.
Study has been expanded to include certain European and former Commonwealth countries.
FG-3019 in Pancreatic Cancer
Study 069 - Commenced enrollment in Phase 2a randomized open label study of FG-3019 in patients with Stage 3 unresectable pancreatic cancer.
The endpoint of this randomized trial is complete surgical tumor removal in stage 3 pancreatic cancer patients who had inoperable tumors at study entry, thereby improving the prognosis for survival.
FG-3019 in Duchenne Muscular Dystrophy (DMD)
Supportive review by TREAT-NMD Advisory Committee for Therapeutics (TACT) of proposed FG-3019 clinical plan was completed in December 2014. A summary of this review is available on their website.
A DMD advisory board, comprising US and EU key opinion leaders, expressed support for investigational studies of FG-3019 as a potential treatment of DMD during its March 2015 meeting. Based on the CTGF mechanism of action on muscle differentiation and fibrosis, they suggested we evaluate the effect of FG-3019 on decline of lung capacity (due to decline in diaphragm function) and on progression of upper body muscle pathology and functional loss.
FibroGen plans to commence patient enrollment in the second half of 2015.
China Manufacturing Certification
In August 2014, the Beijing CFDA completed inspections and issued the Pharmaceutical Production Permit (PPP) to FibroGen China. The permit certifies that the facility was built and validated to GMP standards, and is a regulatory pre-requisite to starting the GMP NDA registration campaign for FG-4592 (roxadustat) in China.
Financial Results (unaudited)
Revenue, Operating Expenses, Net Loss, and EPS for the year ended December 31, 2014
Total revenue was $137.6 million.
Total operating expenses were $187.7 million, which includes $23.3 million of non-cash items.
Net loss was $59.5 million.
Pro forma loss per share was $1.01 based on 59.0 million shares outstanding at December 31, 2014.
Cost-Sharing Arrangements: FibroGen has two cost sharing arrangements with its collaboration partners, Astellas and AstraZeneca, for roxadustat.
Under the Astellas arrangement, Astellas pays 50% of European and U.S. development costs, and 100% of Japan development costs incurred.
Under the AstraZeneca arrangement, FibroGen's total funding obligations for roxadustat outside China are limited to $116.5 million. FibroGen and AstraZeneca are each responsible for 50% of roxadustat development costs not reimbursed by Astellas, until the cap is reached.
Of the $116.5 million cap under the AstraZeneca arrangement, $46.8 million has been incurred and $69.7 million remained as of December 31, 2014.
Based on current internal and partner projections, FibroGen expects to reach this $116.5 million cap in the fourth quarter of 2015.
After the cap is reached, Astellas and AstraZeneca will be responsible for funding roxadustat development in CKD through launch for all territories outside of China.
Cash, Cash Equivalents, Investments, and Receivables
At December 31, 2014, FibroGen's total cash balances were $346.8 million.
Cash balance includes $171.8 million in net proceeds from the Company's initial public offering and side by side private placement of common stock to AstraZeneca.