On February 26, 2016 Bristol-Myers Squibb Company (NYSE: BMY) reported that the Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Opdivo (nivolumab) for two new indications – adults with locally advanced or metastatic non-small cell lung cancer (NSCLC) after prior chemotherapy, and adults with advanced renal cell carcinoma (RCC) after prior therapy (Press release, Bristol-Myers Squibb, FEB 26, 2016, View Source [SID:1234509217]). Schedule your 30 min Free 1stOncology Demo! Both indications are supported by Phase 3 studies in which Opdivo demonstrated a survival benefit versus a standard of care. The CHMP positive opinions will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). Opdivo is already approved by the EC for advanced melanoma and previously treated advanced squamous NSCLC.
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Michael Giordano, M.D., senior vice president, head of Development, Oncology, Bristol-Myers Squibb, commented, "We are committed to advancing our mission to make Opdivo available to a broader range of patients with a wide range of cancers who are in critical need of new treatment options. Today’s two positive CHMP opinions are important achievements and mean we are closer to reaching this goal for those with advanced non-squamous non-small cell lung cancer and renal cell carcinoma. We look forward to the European Commission’s decision and the opportunity to bring an additional treatment option to these patients as quickly as possible."
In lung cancer, the CHMP adopted the positive opinion based on a review of the global Phase 3 study, CheckMate -057, which evaluated the survival of patients with non-squamous NSCLC who had progressed during or after one prior platinum doublet-based chemotherapy regimen. In the trial, Opdivo demonstrated superior overall survival (OS) in previously treated metastatic non-squamous NSCLC compared to chemotherapy, with a 27% reduction in the risk of death (HR: 0.73 [95% CI: 0.59, 0.89; p=0.0015]), based on a prespecified interim analysis. The median OS was 12.2 months in the Opdivo arm (95% CI: 9.7, 15.0) and 9.4 months in the docetaxel arm (95% CI: 8.0, 10.7). Fifty-one percent of patients were alive at one year in the Opdivo arm (95% CI: 45-56) vs. 39% in the docetaxel arm (95% CI: 33-45). The safety profile of Opdivo in CheckMate -057 was consistent with prior studies. In the overall patient population, which included both PD-L1 expressors and non-expressors, the most frequent serious adverse reactions in at least 2% of patients receiving Opdivo were pneumonia, pulmonary embolism, dyspnea, pleural effusions and respiratory failure. The most common adverse reactions in patients treated with Opdivo (reported in >20% of patients) were fatigue (49%), musculoskeletal pain (36%), cough (30%), decreased appetite (29%) and constipation (23%).
In renal cell carcinoma, the CHMP adopted the positive opinion based on a review of the Phase 3 study, CheckMate -025, which evaluated Opdivo versus everolimus in patients with advanced clear-cell RCC after prior therapy, with OS as the primary endpoint. Patients treated with Opdivo in this study achieved a more than five month improvement in OS with median OS of 25 months for Opdivo and 19.6 months for everolimus (hazard ratio: 0.73; [98.5% CI, 0.57-0.93; p=0.0018]), with OS benefit seen regardless of PD-L1 expression. Opdivo is the first and only anti-PD-1 therapy to demonstrate a significant survival benefit in this population through a randomized Phase 3 study. In addition, patients treated with Opdivo also experienced a significant improvement in their health-related quality of life and had significantly lower symptom burden compared to patients receiving everolimus. The safety profile of Opdivo in CheckMate -025 was consistent with prior studies. Serious adverse events occurred in 47% of patients receiving Opdivo. The most frequent serious adverse reactions reported in at least 2% of patients receiving Opdivo were acute kidney injury, pleural effusion, pneumonia, diarrhea, and hypercalcemia. In the study, the most common adverse reactions in patients receiving Opdivo versus everolimus (reported in >20% of patients) were asthenic conditions (56% vs. 57%), cough (34% vs. 38%), nausea (28% vs. 29%), rash (28% vs. 36%), dyspnea (27% vs. 31%), diarrhea (25% vs. 32%), constipation (23% vs. 18%), decreased appetite (23% vs. 30%), back pain (21% vs. 16%), and arthralgia (20% vs. 14%).
Clinical results from CheckMate -057 and CheckMate -025 were presented at the 2015 European Cancer Congress, and published in The New England Journal of Medicine.
About Lung Cancer
Lung cancer is the leading cause of cancer deaths globally, resulting in more than 1.5 million deaths each year, according to the World Health Organization. Non-small cell lung cancer (NSCLC) is one of the most common types of the disease and accounts for approximately 85% of cases. About 25% to 30% of all lung cancers are squamous cell carcinomas, and non-squamous NSCLC accounts for approximately 50% to 65% of all lung cancer cases. Survival rates vary depending on the stage and type of the cancer when it is diagnosed. Globally, the five-year survival rate for Stage I NSCLC is between 47% and 50%; for Stage IV NSCLC, the five-year survival rate drops to 2%.
About Renal Cell Carcinoma
Renal cell carcinoma (RCC) is the most common type of kidney cancer in adults, accounting for more than 100,000 deaths worldwide each year. Clear-cell RCC is the most prevalent type of RCC and constitutes 80% to 90% of all cases. RCC is approximately twice as common in men as in women, with the highest rates of the disease in North America and Europe. Globally, the five-year survival rate for those diagnosed with metastatic, or advanced kidney cancer, is 12.1%.
Bristol-Myers Squibb & Immuno-Oncology: Advancing Oncology Research
At Bristol-Myers Squibb, we have a vision for the future of cancer care that is focused on Immuno-Oncology, now considered a major treatment choice alongside surgery, radiation, chemotherapy and targeted therapies for certain types of cancer.
We have a comprehensive clinical portfolio of investigational and approved Immuno-Oncology agents, many of which were discovered and developed by our scientists. Our ongoing Immuno-Oncology clinical program is looking at broad patient populations, across multiple solid tumors and hematologic malignancies, and lines of therapy and histologies, with the intent of powering our trials for overall survival and other important measures like durability of response. We pioneered the research leading to the first regulatory approval for the combination of two Immuno-Oncology agents, and continue to study the role of combinations in cancer.
We are also investigating other immune system pathways in the treatment of cancer including CTLA-4, CD-137, KIR, SLAMF7, PD-1, GITR, CSF1R, IDO, and LAG-3. These pathways may lead to potential new treatment options – in combination or monotherapy – to help patients fight different types of cancers.
Our collaboration with academia, as well as small and large biotech companies is responsible for researching the potential Immuno-Oncology and non-Immuno-Oncology combinations, with the goal of providing new treatment options in clinical practice.
At Bristol-Myers Squibb, we are committed to changing survival expectations in hard-to-treat cancers and the way patients live with cancer.
About Opdivo
Cancer cells may exploit "regulatory" pathways, such as checkpoint pathways, to hide from the immune system and shield the tumor from immune attack. Opdivo is a PD-1 immune checkpoint inhibitor that binds to the checkpoint receptor PD-1 expressed on activated T-cells, and blocks the binding of PD-L1 and PD-L2, preventing the PD-1 pathway’s suppressive signaling on the immune system, including the interference with an anti-tumor immune response.
Opdivo’s broad global development program is based on Bristol-Myers Squibb’s understanding of the biology behind Immuno-Oncology. Our company is at the forefront of researching the potential of Immuno-Oncology to extend survival in hard-to-treat cancers. This scientific expertise serves as the basis for the Opdivo development program, which includes a broad range of Phase 3 clinical trials evaluating overall survival as the primary endpoint across a variety of tumor types. The Opdivo trials have also contributed toward the clinical and scientific understanding of the role of biomarkers and how patients may benefit from Opdivo across the continuum of PD-L1 expression. To date, the Opdivo clinical development program has enrolled more than 18,000 patients.
Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world in July 2014, and currently has regulatory approval in 46 countries including the United States, Japan, and in the European Union.
U.S. FDA APPROVED INDICATIONS
OPDIVO (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
IMPORTANT SAFETY INFORMATION
Immune-Mediated Pneumonitis
Immune-mediated pneumonitis, including fatal cases, occurred with OPDIVO treatment. Across the clinical trial experience with solid tumors, fatal immune-mediated pneumonitis occurred with OPDIVO. Monitor patients for signs with radiographic imaging and symptoms of pneumonitis. Administer corticosteroids for Grade 2 or greater pneumonitis. Permanently discontinue for Grade 3 or 4 and withhold until resolution for Grade 2. In Checkmate 057, immune-mediated pneumonitis, including interstitial lung disease, occurred in 3.4% (10/287) of patients: Grade 3 (n=5), Grade 2 (n=2), and Grade 1 (n=3). In Checkmate 025, pneumonitis, including interstitial lung disease, occurred in 5% (21/406) of patients receiving OPDIVO and 18% (73/397) of patients receiving everolimus. Immune-mediated pneumonitis occurred in 4.4% (18/406) of patients receiving OPDIVO: Grade 4 (n=1), Grade 3 (n=4), Grade 2 (n=12), and Grade 1 (n=1).
Immune-Mediated Colitis
Immune-mediated colitis can occur with OPDIVO treatment. Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 (of more than 5 days duration), 3, or 4 colitis. As a single agent, withhold OPDIVO for Grade 2 or 3 and permanently discontinue for Grade 4 or recurrent colitis upon restarting OPDIVO. In Checkmate 057, diarrhea or colitis occurred in 17% (50/287) of patients receiving OPDIVO. Immune-mediated colitis occurred in 2.4% (7/287) of patients: Grade 3 (n=3), Grade 2 (n=2), and Grade 1 (n=2). In Checkmate 025, diarrhea or colitis occurred in 25% (100/406) of patients receiving OPDIVO and 32% (126/397) of patients receiving everolimus. Immune-mediated diarrhea or colitis occurred in 3.2% (13/406) of patients receiving OPDIVO: Grade 3 (n=5), Grade 2 (n=7), and Grade 1 (n=1).
Immune-Mediated Hepatitis
Immune-mediated hepatitis can occur with OPDIVO treatment. Monitor patients for abnormal liver tests prior to and periodically during treatment. Administer corticosteroids for Grade 2 or greater transaminase elevations. Withhold for Grade 2 and permanently discontinue for Grade 3 or 4 immune-mediated hepatitis. In Checkmate 057, one patient (0.3%) developed immune-mediated hepatitis. In Checkmate 025, there was an increased incidence of liver test abnormalities compared to baseline in AST (33% vs 39%), alkaline phosphatase (32% vs 32%), ALT (22% vs 31%), and total bilirubin (9% vs 3.5%) in the OPDIVO and everolimus arms, respectively. Immune-mediated hepatitis requiring systemic immunosuppression occurred in 1.5% (6/406) of patients receiving OPDIVO: Grade 3 (n=5) and Grade 2 (n=1).
Immune-Mediated Endocrinopathies
Hypophysitis, adrenal insufficiency, thyroid disorders, and type 1 diabetes mellitus can occur with OPDIVO treatment. Monitor patients for signs and symptoms of hypophysitis, signs and symptoms of adrenal insufficiency during and after treatment, thyroid function prior to and periodically during treatment, and hyperglycemia. Administer corticosteroids for Grade 2 or greater hypophysitis. Withhold for Grade 2 or 3 and permanently discontinue for Grade 4 hypophysitis. Administer corticosteroids for Grade 3 or 4 adrenal insufficiency. Withhold for Grade 2 and permanently discontinue for Grade 3 or 4 adrenal insufficiency. Administer hormone-replacement therapy for hypothyroidism. Initiate medical management for control of hyperthyroidism. Administer insulin for type 1 diabetes. Withhold OPDIVO for Grade 3 and permanently discontinue for Grade 4 hyperglycemia.
In Checkmate 025, hypophysitis occurred in 0.5% (2/406) of patients receiving OPDIVO: Grade 3 (n=1) and Grade 1 (n=1). In Checkmate 057, 0.3% (1/287) of OPDIVO-treated patients developed adrenal insufficiency. In Checkmate 025, adrenal insufficiency occurred in 2.0% (8/406) of patients receiving OPDIVO: Grade 3 (n=3), Grade 2 (n=4), and Grade 1 (n=1). In Checkmate 057, Grade 1 or 2 hypothyroidism, including thyroiditis, occurred in 7% (20/287) and elevated thyroid stimulating hormone occurred in 17% of patients receiving OPDIVO. Grade 1 or 2 hyperthyroidism occurred in 1.4% (4/287) of patients. In Checkmate 025, thyroid disease occurred in 11% (43/406) of patients receiving OPDIVO, including one Grade 3 event, and in 3.0% (12/397) of patients receiving everolimus. Hypothyroidism/thyroiditis occurred in 8% (33/406) of patients receiving OPDIVO: Grade 3 (n=2), Grade 2 (n=17), and Grade 1 (n=14). Hyperthyroidism occurred in 2.5% (10/406) of patients receiving OPDIVO: Grade 2 (n=5) and Grade 1 (n=5). In Checkmate 025, hyperglycemic adverse events occurred in 9% (37/406) patients. Diabetes mellitus or diabetic ketoacidosis occurred in 1.5% (6/406) of patients receiving OPDIVO: Grade 3 (n=3), Grade 2 (n=2), and Grade 1 (n=1).
Immune-Mediated Nephritis and Renal Dysfunction
Immune-mediated nephritis can occur with OPDIVO treatment. Monitor patients for elevated serum creatinine prior to and periodically during treatment. For Grade 2 or 3 increased serum creatinine, withhold and administer corticosteroids; if worsening or no improvement occurs, permanently discontinue. Administer corticosteroids for Grade 4 serum creatinine elevation and permanently discontinue. In Checkmate 057, Grade 2 immune-mediated renal dysfunction occurred in 0.3% (1/287) of patients receiving OPDIVO. In Checkmate 025, renal injury occurred in 7% (27/406) of patients receiving OPDIVO and 3.0% (12/397) of patients receiving everolimus. Immune-mediated nephritis and renal dysfunction occurred in 3.2% (13/406) of patients receiving OPDIVO: Grade 5 (n=1), Grade 4 (n=1), Grade 3 (n=5), and Grade 2 (n=6).
Immune-Mediated Rash
Immune-mediated rash can occur with OPDIVO treatment. Severe rash (including rare cases of fatal toxic epidermal necrolysis) occurred in the clinical program of OPDIVO. Monitor patients for rash. Administer corticosteroids for Grade 3 or 4 rash. Withhold for Grade 3 and permanently discontinue for Grade 4. In Checkmate 057, immune-mediated rash occurred in 6% (17/287) of patients receiving OPDIVO including four Grade 3 cases. In Checkmate 025, rash occurred in 28% (112/406) of patients receiving OPDIVO and 36% (143/397) of patients receiving everolimus. Immune-mediated rash, defined as a rash treated with systemic or topical corticosteroids, occurred in 7% (30/406) of patients receiving OPDIVO: Grade 3 (n=4), Grade 2 (n=7), and Grade 1 (n=19).
Immune-Mediated Encephalitis
Immune-mediated encephalitis can occur with OPDIVO treatment. Withhold OPDIVO in patients with new-onset moderate to severe neurologic signs or symptoms and evaluate to rule out other causes. If other etiologies are ruled out, administer corticosteroids and permanently discontinue OPDIVO for immune-mediated encephalitis. In Checkmate 057, fatal limbic encephalitis occurred in one patient (0.3%) receiving OPDIVO.
Other Immune-Mediated Adverse Reactions
Based on the severity of adverse reaction, permanently discontinue or withhold treatment, administer high-dose corticosteroids, and, if appropriate, initiate hormone-replacement therapy. In < 1.0% of patients receiving OPDIVO, the following clinically significant, immune-mediated adverse reactions occurred: uveitis, pancreatitis, facial and abducens nerve paresis, demyelination, polymyalgia rheumatica, autoimmune neuropathy, Guillain-Barré syndrome, hypopituitarism, systemic inflammatory response syndrome, gastritis, duodenitis, and sarcoidosis. Across clinical trials of OPDIVO as a single agent administered at doses of 3 mg/kg and 10 mg/kg, additional clinically significant, immune-mediated adverse reactions were identified: motor dysfunction, vasculitis, and myasthenic syndrome.
Infusion Reactions
Severe infusion reactions have been reported in <1.0% of patients in clinical trials of OPDIVO. Discontinue OPDIVO in patients with Grade 3 or 4 infusion reactions. Interrupt or slow the rate of infusion in patients with Grade 1 or 2. In Checkmate 057, Grade 2 infusion reactions requiring corticosteroids occurred in 1.0% (3/287) of patients receiving OPDIVO. In Checkmate 025, hypersensitivity/infusion-related reactions occurred in 6% (25/406) of patients receiving OPDIVO and 1.0% (4/397) of patients receiving everolimus.
Embryo-fetal Toxicity
Based on its mechanism of action, OPDIVO can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with an OPDIVO- containing regimen and for at least 5 months after the last dose of OPDIVO.
Lactation
It is not known whether OPDIVO is present in human milk. Because many drugs, including antibodies, are excreted in human milk and because of the potential for serious adverse reactions in nursing infants from an OPDIVO-containing regimen, advise women to discontinue breastfeeding during treatment.
Serious Adverse Reactions
In Checkmate 057, serious adverse reactions occurred in 47% of patients receiving OPDIVO. The most frequent serious adverse reactions reported in ≥2% of patients were pneumonia, pulmonary embolism, dyspnea, pleural effusion, and respiratory failure. In Checkmate 025, serious adverse reactions occurred in 47% of patients receiving OPDIVO. The most frequent serious adverse reactions reported in ≥2% of patients were acute kidney injury, pleural effusion, pneumonia, diarrhea, and hypercalcemia.
Common Adverse Reactions
In Checkmate 057, the most common adverse reactions (≥20%) reported with OPDIVO were fatigue (49%), musculoskeletal pain (36%), cough (30%), decreased appetite (29%), and constipation (23%). In Checkmate 025, the most common adverse reactions (≥20%) reported in patients receiving OPDIVO vs everolimus were asthenic conditions (56% vs 57%), cough (34% vs 38%), nausea (28% vs 29%), rash (28% vs 36%), dyspnea (27% vs 31%), diarrhea (25% vs 32%), constipation (23% vs 18%), decreased appetite (23% vs 30%), back pain (21% vs 16%), and arthralgia (20% vs 14%).
Year: 2016
BioMarin Announces Fourth Quarter and Full Year 2015 Financial Results
On February 25, 2016 BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) reported financial results for both the fourth quarter and year ended December 31, 2015 (Press release, BioMarin, FEB 25, 2016, View Source [SID:1234509260]). For the quarter ended December 31, 2015, Non-GAAP net loss was $70.0 million, compared to non-GAAP net loss of $10.7 million for the fourth quarter of 2014. For the year ended December 31, 2015, non-GAAP net loss was $142.6 million, compared to non-GAAP net loss of $25.7 million for the year ended December 31, 2014. The increased non-GAAP net loss for the year ended December 31, 2015, compared to the prior year, is primarily due to increased research and development and selling, general and administrative expenses, partially offset by increased revenues from the global launch of Vimizim and strong Kuvan sales.
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GAAP net income was $68.6 million, or $0.43 per basic and $0.39 diluted share, respectively, for the fourth quarter of 2015, compared to GAAP net loss of $69.8 million, or $0.47 per basic and diluted share, for the fourth quarter of 2014. GAAP net income for the quarter included a $369.5 million gain on the sale of intangible assets due to the sale of talazoparib to Medivation, Inc. and a $47.9 million credit to contingent consideration expense primarily due to the U.S. FDA complete response letter for Kyndrisa. The complete response letter triggered a reversal of previously accrued Contingent Value Rights (CVR) related to the potential $80 million payment to former Prosensa shareholders for U.S. FDA approval prior to May 15, 2016. These gains and credits were partially offset by a $198.7 million impairment of intangible asset charge related to the decline in value of the U.S. rights to Kyndrisa due to the U.S. FDA complete response letter for Kyndrisa and increased research and development and selling, general and administrative expenses. GAAP net loss for the year ended December 31, 2015 was $171.8 million, or $1.07 per basic and diluted share, compared to GAAP net loss of $134.0 million, or $0.92 per basic and diluted share for the year ended December 31, 2014.
Total BioMarin Revenue was $889.9 million for the year ended December 31, 2015 an increase of 18.8% compared to the same period in 2014. Fourth quarter 2015 Total BioMarin Revenue of $227.9 million decreased 1.0% due to the timing of government orders impacting Naglazyme revenue. The increase in Total BioMarin Revenue for full-year 2015 was driven by the continued global launch of Vimizim and growth in the number of Kuvan patients on therapy. The number of patients being treated with Vimizim increased 10% quarter to quarter in the fourth quarter compared to the third quarter of 2015. Sales of Vimizim in 2015 were recorded in 13 new countries to a total of 33 countries through year-end and totaled $228.1 million for the full year. Kuvan Net Product Revenue increased 17.9% to $239.3 million driven primarily by patient number increases and high rates of compliance. At year-end 2015, commercial patients on Kuvan increased 15.8% year over year.
As of December 31, 2015, BioMarin had cash, cash equivalents and investments totaling $1,018.3 million, as compared to $1,043.1 million on December 31, 2014.
"BioMarin is entering 2016 from a position of strength as supported by four factors. First, we expect that our established and growing commercial business may generate over one billion dollars in revenues this year and believe it can grow to $1.5 billion by 2020. Second, data readouts for cerliponase alfa and pegvaliase may potentially lead to two new product filings later this year and two potential new product launches in 2017. Third, we have two potential $1 billion opportunities in development with vosoritide and BMN 270 for hemophilia A. And fourth, we expect to manage this growing business with the goal of achieving non-GAAP break-even or better in 2017 regardless of the regulatory outcome of Kyndrisa in Europe," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "We look forward to hosting our annual R&D Day on April 20th in New York where we will share one year data with vosoritide for achondroplasia as well as an update on our gene therapy program with BMN 270 for the treatment of hemophilia A. In addition, in the second quarter we expect to receive an opinion from the Committee for Medicinal Products (CHMP) on a potential approval of Kyndrisa in Europe. Kyndrisa is under regulatory review in the E.U., where there is currently no approved treatment option for children with Duchenne muscular dystrophy amenable to exon 51 skipping."
Net Product Revenue (in millions, unaudited)
Total Revenue
Three Months Ended December 31, Twelve Months Ended December 31,
2015 2014 $ Change % Change 2015 2014 $ Change % Change
Vimizim (1) $ 58.5 $ 36.9 $ 21.6 58.5 % $ 228.1 $ 77.3 $ 150.8 195.1 %
Naglazyme (1) 59.7 88.5 (28.8 ) (32.5 )% 303.1 334.4 (31.3 ) (9.4 )%
Kuvan 64.8 57.4 7.4 12.9 % 239.3 203.0 36.3 17.9 %
Aldurazyme 39.0 40.9 (1.9 ) (4.6 )% 98.0 105.6 (7.6 ) (7.2 )%
Firdapse 4.4 4.1 0.3 7.3 % 16.0 18.1 (2.1 ) (11.6 )%
Net product revenues 226.4 227.8 (1.4 ) (0.6 )% 884.5 738.4 146.1 19.8 %
Collaborative agreement revenues 0.2 0.3 (0.1 ) 1.0 1.6 (0.6 )
Royalty, license and other revenues 1.3 2.0 (0.7 ) 4.4 9.3 (4.9 )
Total BioMarin revenues $ 227.9 $ 230.1 $ (2.2 ) (1.0 )% $ 889.9 $ 749.3 $ 140.6 18.8 %
(1) Vimizim and Naglazyme revenues experience quarterly fluctuations due to the timing of government ordering patterns in certain countries. The Company does not believe these fluctuations reflect a change in underlying demand.
Reconciliation of Aldurazyme Revenues
Three Months Ended December 31, Twelve Months Ended December 31,
2015 2014 $ Change % Change 2015 2014 $ Change % Change
Aldurazyme revenue reported by Genzyme $ 54.1 $ 56.3 $ (2.2 ) (3.9 )% $ 217.8 $ 228.8 $ (11.0 ) (4.8 )%
Three Months Ended December 31, Twelve Months Ended December 31,
2015 2014 $ Change 2015 2014 $ Change
Royalties earned from Genzyme $ 26.7 $ 27.9 $ (1.2 ) $ 95.8 $ 97.0 $ (1.2 )
Net product transfer revenues (2) 12.3 13.0 (0.7 ) 2.2 8.6 (6.4 )
Total Aldurazyme net product revenues $ 39.0 $ 40.9 $ (1.9 ) $ 98.0 $ 105.6 $ (7.6 )
(2) To the extent units shipped to third party customers by Genzyme exceed BioMarin inventory transfers to Genzyme, BioMarin will record a decrease in net product revenue from the royalty payable to BioMarin for the amount of previously recognized product transfer revenue. If BioMarin inventory transfers exceed units shipped to third party customers by Genzyme, BioMarin will record incremental net product transfer revenue for the period. Positive net product transfer revenues result in the period if BioMarin transferred more units to Genzyme than Genzyme sold to third-party customers.
2016 Financial Guidance
Revenue Guidance ($ in millions)
Item
2016 Guidance
Total BioMarin Revenues $1,050 to $1,100
Vimizim Net Product Revenue $300 to $330
Naglazyme Net Product Revenue $290 to $320
Kuvan Net Product Revenue $320 to $350
Select Income Statement Guidance ($ in millions, except percentages)
Item
2016 Guidance
Cost of Sales (% of Total Revenue) 18.0% to 19.0%
Selling, General and Admin. Expense $470 to $490
Research and Development Expense $680 to $720
Non – GAAP Net Loss $(75) to $(100)
GAAP Net Loss $(400) to $(430)
Anticipated Milestones in 1H16
Cerliponase alfa for CLN2, late-infantile form of Batten disease: Complete results from the Phase 1/2 study of cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1), for the treatment of patients with late-infantile neuronal ceroid lipofuscinosis type 2 (NCL-2), a form of Batten disease will be announced at the WORLD LSD Symposium on March 2, 2016. If data are supportive, the Company plans to submit in the U.S. and E.U. for regulatory approval mid-year 2016.
Pegvaliase for phenylketonuria (PKU): The Company expects to share top-line results from this study in the first quarter of 2016 and, if the data are supportive, submit a Biologics License Application (BLA) to U.S. FDA in the second half of 2016.
BMN 270 gene therapy product for hemophilia A: In the fourth quarter of 2015, the first patient was dosed in a Phase 1/2 trial with BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A. BMN 270 is an AAV-factor VIII vector, designed to restore factor VIII plasma concentrations, essential for blood clotting in patients with hemophilia A. Subjects in that study are now being dosed with the third highest dose in this dose ranging safety study. BioMarin will provide a program update at the R&D Day in April 2016.
Vosoritide for achondroplasia: In June 2015, the Company published results from the Phase 2 study showing a 50 percent in mean annualized growth velocity (speed at which growth in children occurs) in the cohort of 10 patients receiving a 15 µg/kg dose of vosoritide daily for six months compared with their own pre-treatment growth velocity (P-value= 0.01). In addition, to support further exploration of a dose that may enable "catch-up" growth in the event of delayed treatment, a fourth cohort with 30 micrograms per kilogram daily completed enrollment in the fourth arm of the Phase 2 study. BioMarin will provide 12-month results with vosoritide at the 15 µg/kg dose, preliminary safety update on the 30 µg/kg dose and an update on Phase 3 plans at the R&D Day in April 2016.
Kyndrisa (drisapersen) for Duchenne muscular dystrophy: The Committee for Medicinal Products (CHMP), the arm of the European Medicines Agency that is currently reviewing the Marketing Authorization Application for Kyndrisa, is expected to provide an opinion on the application in the second quarter of 2016. If the CHMP provides a positive opinion, Kyndrisa could potentially be approved in the E.U. in the second half of 2016.
Reveglucosidase alfa for Pompe disease: In January 2016, the Company shared interim results from the single-arm Phase 2/3 trial with patients previously treated with alglucosidase alfa who were then switched to treatment with reveglucosidase alfa. The primary endpoint of the study showed an improvement from baseline in the respiratory parameter Maximal Inspiratory Pressure (MIP) as well as the secondary endpoint 6 minute walk test. The Company is currently determining next steps for the program.
Other Corporate Achievements in 2015
October 1, 2015, BioMarin to Acquire Global Rights to PKU Franchise from Merck Serono
BioMarin and Merck Serono announced that BioMarin will acquire all global rights to Kuvan (sapropterin dihydrochloride), excluding Japan, and pegvaliase from Merck Serono (Merck). Under the terms of the agreement, BioMarin provided Merck with an upfront payment of $371.8 million. An additional €60 million in milestones will be paid to Merck if combined sales of Kuvan and pegvaliase reach undisclosed cumulative sales thresholds. In addition, €125 million will be paid to Merck conditional on the achievement of undisclosed regulatory milestones related to pegvaliase. Previously, BioMarin had exclusive rights to Kuvan in the United States and Canada and to pegvaliase in the United States and Japan. Under the terms of the transaction, BioMarin will now have exclusive worldwide rights to Kuvan and pegvaliase with the exception of Kuvan in Japan. Approved in 2007 in the U.S., Kuvan is a commercialized product for the treatment of patients with phenylketonuria (PKU). Pegvaliase is currently in registration-enabling pivotal studies as a potential therapeutic option for adult patients with phenylketonuria. With the potential approval of pegvaliase, the two products combined would expand and globalize BioMarin’s leadership position by offering a wider range of treatment options to patients worldwide with PKU.
August 24, 2015, Talazoparib acquired by Medivation, Inc.:
Medivation, Inc. and BioMarin Pharmaceutical Inc. announced that BioMarin entered into a definitive agreement to sell Medivation all worldwide rights to talazoparib (formerly referred to as BMN 673), a highly-potent, orally available poly ADP ribose polymerase (PARP) inhibitor currently in a Phase 3 study for the treatment of patients with deleterious germline BRCA 1 or BRCA 2 mutations and locally advanced and/or metastatic breast cancer. The transaction closed on October 6, 2015 and as a result, Medivation is now responsible for all research, development, regulatory and commercialization activities for all indications on a global basis. Under the terms of the agreement, Medivation paid BioMarin $410 million upfront, and will pay up to an additional $160 million upon the achievement of regulatory and sales-based milestones and mid-single digit royalties for talazoparib.
8-K – Current report
On February 25, 2016 Peregrine Pharmaceuticals, Inc. (NASDAQ: PPHM) (NASDAQ: PPHMP), a biopharmaceutical company focused on developing therapeutics to stimulate the body’s immune system to fight cancer, that it is discontinuing the company’s Phase III SUNRISE trial of bavituximab in patients with previously treated locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) (Filing, 8-K, Peregrine Pharmaceuticals, FEB 25, 2016, View Source [SID:1234509235]). The decision to stop the trial was based on the recommendation of the study’s Independent Data Monitoring Committee (IDMC) following a pre-specified interim analysis performed after 33% of targeted overall events (patient deaths) in the study were reached. Results of the analysis demonstrated that the bavituximab plus docetaxel group did not show a sufficient improvement in overall survival as compared to the docetaxel group to warrant continuation of the study. The interim analysis showed that the bavituximab combination group is performing as expected according to the original trial assumptions in terms of overall survival, while the docetaxel group is dramatically outperforming overall survival expectations based on the original trial assumptions and as compared to recently published studies.
"Let me start by taking this opportunity to thank all of the patients, their families, and the physicians who participated in the SUNRISE trial. While we are deeply disappointed by this early outcome from the SUNRISE trial, we plan to take a deliberate and detailed approach in reviewing and verifying all available data from the trial in order to understand what subgroups or other patient characteristics may have impacted the performance of the study. While we perform this analysis, we plan to put our other chemotherapy combination studies on hold until we have a clear understanding of the SUNRISE study results," said Steven W. King, president and chief executive officer of Peregrine. "While this is an unexpected and disappointing setback for the bavituximab chemotherapy combination clinical program, we have not seen anything in this trial result that diminishes our enthusiasm for advancing our immuno-oncology (I-O) combination trials. The I-O combination studies are based on different mechanistic synergies that are clearly separate from the chemotherapy combination being evaluated in the SUNRISE study. In addition, it is important to note that in no way do these results have any impact on our contract manufacturing business conducted through our wholly owned subsidiary, Avid Bioservices. This business has shown consistent revenue growth and has been instrumental in maintaining a strong cash position and our plan is to continue growing this business."
As of February 1, 2016, Avid Bioservices had a revenue backlog in excess of $58 million under committed contracts from existing clients. In addition, Peregrine had $67.5 million in cash and equivalents as of January 31, 2016.
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Keryx Biopharmaceuticals Announces Fourth Quarter and Year-End 2015 Financial Results
On February 25, 2016 Keryx Biopharmaceuticals, Inc. (Nasdaq:KERX), a biopharmaceutical company focused on bringing innovative medicines to market for people with renal disease, reported its financial results for the fourth quarter and year ended December 31, 2015 (Press release, Keryx Biopharmaceuticals, FEB 25, 2016, View Source;p=RssLanding&cat=news&id=2143160 [SID:1234509231]). The company also reviewed its commercialization progress with Auryxia (ferric citrate), upcoming milestones and selected 2016 financial guidance.
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"As we enter 2016, the fundamentals of Auryxia are solid, and we plan to build on that foundation to advance our launch in the U.S.," said Greg Madison, chief executive officer of Keryx. "Importantly, the data readout expected in early second quarter from the ferric citrate phase 3 label expansion trial will be an important marker of our efforts to help people with pre-dialysis chronic kidney disease. Specifically, we believe that ferric citrate – which, through its novel mechanism of action, delivers iron orally through the body’s natural absorption process – could be the first FDA-approved oral medicine to treat iron deficiency anemia (IDA) in this patient population."
FOURTH QUARTER 2015 AND RECENT BUSINESS HIGHLIGHTS
Auryxia (ferric citrate) Commercialization
Auryxia net U.S. product sales for the fourth quarter of 2015 were $4.8 million, based on approximately 7,850 prescriptions, an increase of 46 percent from the third quarter. For the full year 2015, Auryxia net U.S. product sales were $10.1 million, representing greater than 18,000 prescriptions.
Cumulative target physicians who have written a prescription for Auryxia increased more than 25 percent from the third quarter of 2015.
Keryx completed its sales force expansion and now will have 95 sales representatives calling on target prescribers. The expansion enables increased reach and frequency of contact with physicians, dieticians and the entire dialysis care team.
Product Expansion Opportunities
Pivotal Phase 3 Trial Aimed at Increasing the Number of Adults Eligible for Treatment with Ferric Citrate
The 24-week pivotal phase 3 trial evaluating ferric citrate for the treatment of IDA in patients with stages 3-5 CKD completed in January, as planned. Early in the second quarter of 2016, Keryx expects to announce topline safety and efficacy results. If the results are successful, Keryx intends to submit a regulatory application for approval to the U.S. FDA in the third quarter of 2016, and submit the data for presentation at a fourth quarter 2016 medical conference.
Potential Geographic Expansion
Keryx is seeking potential partners to make Fexeric (ferric citrate) available to patients in Europe.
Fourth Quarter and Year Ended December 31, 2015 Financial Results
"In the fourth quarter of 2015, we strengthened our financial position through a re-alignment of our cost structure and an infusion of capital, which we expect will take the Auryxia franchise to cash flow positive," said Scott Holmes, chief financial officer of Keryx. "For 2016, we expect prescription volume to increase between 20 percent and 35 percent on a sequential quarter basis, ramping as we realize the full impact of our expanded sales force. As we progress through 2016, we are committed to maintaining fiscal discipline, while advancing our business and supporting the continued growth of Auryxia."
At December 31, 2015, the company had cash and cash equivalents of $200.3 million.
Total revenues for the quarter ended December 31, 2015 were approximately $5.8 million, compared to $0.6 million during the same period in 2014. Total revenues for the quarter consisted of Auryxia net U.S. product sales of $4.8 million, and license revenue of $1.0 million associated with royalties received on ferric citrate net sales from Keryx’s Japanese partner. For the year ended 2015, total revenues were $13.7 million, including $10.1 million of Auryxia net U.S. product sales.
Cost of goods sold for the quarter ended December 31, 2015 was $1.1 million. Cost of goods sold for the full year 2015 was $4.5 million, which included $2.6 million related to manufacturing charges incurred as a result of not fully utilizing planned production at certain of the company’s third party manufacturers as reported in the third quarter.
Research and development expenses for the quarter ended December 31, 2015 were $8.0 million compared to $5.8 million during the same period in 2014. The increase was primarily due to an increase in costs associated with our medical affairs efforts in support of Auryxia. For the full year 2015, total research and development expenses were $36.7 million compared to $51.5 million in 2014.
Selling, general and administrative expenses for the quarter ended December 31, 2015 were $21.6 million, as compared to $34.1 million during the same period in 2014. The decrease was related to a $10.5 million decrease in non-cash stock-based compensation expense compared to the prior period, primarily related to expense recognized in connection with the first commercial sale of Auryxia in 2014. For the full year 2015, total selling, general and administrative expenses were $81.4 million compared to $70.1 million in 2014.
Net loss for the fourth quarter ended December 31, 2015 was $37.8 million, or $0.36 per share, compared to a net loss of $40.3 million, or $0.44 per share, for the comparable quarter in 2014. For the full year 2015, net loss was $123.1 million or $1.19 per share compared to a net loss of $111.5 million, or $1.23 per share in 2014.
2016 Financial Guidance
This section contains forward-looking guidance about the financial outlook for Keryx Biopharmaceuticals
Auryxia net U.S. product sales: Keryx expects full year 2016 Auryxia net U.S. product sales to be in the range of $31 to $34 million. The company expects sales to ramp throughout the year, as it realizes the full impact of its expanded sales force.
Cash operating expenses: Keryx reiterated its cash operating expenses in 2016 will be in the range of $87 million to $92 million. Cash operating expense guidance excludes cost of goods sold, license expenses, and other non-cash expenses.*
* Please refer to the section below titled "Use of Non-GAAP Financial Measures" for information about Keryx’s use of non-GAAP financial measures.
8-K – Current report
On February 25, 2016 Geron Corporation (Nasdaq: GERN) reported financial results for the fourth quarter and year ended December 31, 2015 and recent events (Filing, Q4/Annual, Geron, 2015, FEB 25, 2016, View Source [SID:1234509230]).
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Fourth Quarter and Year-End 2015 Results
For 2015, the company reported net income of $46,000, or $0.00 per share, compared to a net loss of $35.7 million, or $(0.23) per share, for 2014. For the fourth quarter of 2015, the company reported a net loss of $8.5 million, or $(0.05) per share, compared to a net loss of $8.9 million, or $(0.06) per share, for the comparable 2014 period. The company ended 2015 with $146.7 million in cash and investments.
Revenues for 2015 were $36.4 million compared to $1.2 million for 2014. Revenues for the fourth quarter of 2015 were $220,000 compared to $178,000 for the comparable 2014 period. Revenues for 2015 included the full recognition of the $35.0 million upfront payment from Janssen Biotech, Inc. (Janssen) as collaboration revenue upon the company’s transfer of the imetelstat license rights and completion of technology transfer-related activities outlined under the imetelstat collaboration agreement with Janssen in the third quarter of 2015. The upfront cash payment was received in December 2014 and recorded as deferred revenue at that time.
Total operating expenses for 2015 were $36.9 million compared to $37.5 million for 2014. Total operating expenses for the fourth quarter of 2015 were $8.9 million compared to $9.2 million for the comparable 2014 period. Operating expenses for 2015 included restructuring charges of $1.3 million in connection with the company’s organizational resizing announced in March 2015.
Research and development expenses for 2015 were $17.8 million compared to $20.7 million for 2014. Research and development expenses for the fourth quarter of 2015 were $4.0 million compared to $4.4 million for the comparable 2014 period. The decrease in research and development expenses in 2015 compared to 2014 primarily reflects the net result of lower personnel related costs due to the organizational resizing and reduced manufacturing costs for imetelstat drug product, partially offset by higher costs for the clinical development of imetelstat in collaboration with Janssen.
General and administrative expenses for 2015 were $17.8 million compared to $16.8 million for 2014. General and administrative expenses for the fourth quarter of 2015 were $4.9 million compared to $4.8 million for the comparable 2014 period. The increase in general and administrative expenses in 2015 compared to 2014 primarily reflects higher non-cash stock-based compensation expense.
Interest and other income for 2015 was $677,000 compared to $373,000 for 2014. Interest and other income for the fourth quarter of 2015 was $196,000 compared to $100,000 for the comparable 2014 period. The increase in interest and other income for 2015 compared to 2014 primarily reflects higher cash and investment balances with the receipt of the $35.0 million upfront payment from Janssen and higher yields on the company’s marketable securities portfolio.
Company Events
Publications and Presentations
● The New England Journal of Medicine (NEJM) published two papers in which data from clinical studies of two hematologic myeloid malignancies, essential thrombocythemia (ET) and myelofibrosis (MF), suggest imetelstat may have disease-modifying activity by inhibiting the malignant progenitor cell clones responsible for the underlying diseases in a relatively select manner. The papers are available online in the September 3rd issue at www.NEJM.org.
● Three presentations describing clinical and non-clinical data on imetelstat were made at the 57th annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2015:
○ Telomerase Inhibitor Imetelstat Therapy in Refractory Anemia with Ring Sideroblasts with or without Thrombocytosis (Abstract #55; oral presentation)
Data were presented for safety and efficacy as of the May 10, 2015 data cut. Nine patients with a form of the myelodysplastic syndromes (MDS) known as refractory anemia with ring sideroblasts (MDS-RARS) were enrolled in the study cohort, classified as having either intermediate-1 or intermediate-2 risk disease by the International Prognostic Scoring System (IPSS). Six of nine (66.7%) patients had prior treatment with erythropoiesis stimulating agents (ESAs).
Three of the eight (37.5%) patients who were dependent on red blood cell transfusions at study entry became transfusion independent, defined as not requiring transfusions for at least eight weeks. The median duration of transfusion independence was 28 weeks (range: nine weeks to 37 weeks).
Adverse events were similar in nature to the adverse events reported in the MF clinical study published in the NEJM.
○ Dynamics of Mutations in Patients with ET Treated with Imetelstat (Abstract #57; oral presentation)
Data from further mutational analyses of patient samples from the clinical study in ET showed that imetelstat treatment suppressed allele burdens of multiple gene mutations in addition to the JAK2V617F, CALR and MPL mutations.
○ Activity of the Telomerase Inhibitor GRN163L (Imetelstat) on Acute Myeloblastic Leukemia Blasts Is Enhanced by DNA Methyltransferase Inhibitors Irrespective of TERT Promoter Methylation Status (Abstract #1267; poster presentation)
Data from an in vitro study showed that imetelstat has activity against samples derived from patients with high risk leukemias, and that activity was enhanced by the demethylating agent 5-azacytadine, which is currently used in the treatment of some patients with high risk MDS or acute myeloid leukemia.
Clinical Development by Janssen
● IMbarkTM. In September 2015, the first patient was dosed in a Phase 2 clinical trial to evaluate imetelstat in patients with MF. The trial, referred to as IMbarkTM, will assess the efficacy, safety and tolerability of two dose levels of single-agent imetelstat and is designed to enroll approximately 200 patients (approximately 100 patients per dosing arm) with intermediate-2 or high risk MF, as defined by the Dynamic International Prognostic Scoring System, who have relapsed after or are refractory to Janus Kinase (JAK) inhibitor treatment. Patients will be assigned randomly, on a 1:1 ratio, to one of two dosing arms – 9.4 mg/kg or 4.7 mg/kg every three weeks. Patients will be blinded to the dosing arm assignment. Dose reductions for adverse events are allowed and will follow protocol-specified algorithms. An internal review of data from the trial is planned after approximately 20 patients per arm have been randomized and followed for at least 12 weeks in order to assess the adequacy of one or both of the initial dosing arms. As a result of this internal review, which is expected to occur in the second half of 2016, one or both dosing arms could continue as planned, be stopped or modified, or alternative doses could be selected.
The co-primary efficacy endpoints for IMbarkTM are spleen response rate and symptom response rate. Spleen response rate is defined as the percentage of patients who achieve ≥ 35% reduction in spleen volume from baseline at the Week 24 visit, as measured by imaging scans and assessed at a central imaging facility and by an Independent Review Committee. Symptom response rate is defined as the percentage of patients who have ≥ 50% reduction in Total Symptom Scores from baseline at the Week 24 visit, based on patient-reported outcomes on a modified Myelofibrosis Symptom Assessment Form version 2.0 electronic diary. The primary efficacy analysis of the co-primary endpoints will occur after all treated patients have been followed for at least 24 weeks, and the data cut for this analysis is expected to occur in the second half of 2017. Formal clinical data from this trial is expected to be presented at a medical conference to be determined in the future.
Further information about the trial, including participating medical centers around the world, can be found at View Source
● IMergeTM. In January 2016, the first patient was dosed in a Phase 2/3 clinical trial to evaluate imetelstat in patients with MDS. The trial, referred to as IMergeTM, will evaluate imetelstat in transfusion dependent patients with IPSS Low or Intermediate-1 risk MDS who have relapsed after or are refractory to prior treatment with an ESA.
As designed, the trial consists of two parts, and a total of approximately 200 patients are expected to be enrolled. Part 1 of the trial is planned as a Phase 2, open-label, single-arm design to assess the efficacy and safety of imetelstat. Up to 30 patients are expected to be enrolled in Part 1, all of whom will receive imetelstat and be followed for safety, hematologic improvement and reduction in transfusion requirement. Before proceeding to Part 2, the data from Part 1 must support a positive assessment of the benefit/risk profile of imetelstat in these patients. The internal review of data from Part 1 to support advancing to Part 2 is expected to occur in the second half of 2016. Part 2 of the trial is planned as a Phase 3 double-blind, randomized, placebo-controlled design to compare the efficacy of imetelstat against placebo. Approximately 170 patients are expected to be enrolled in Part 2, who will be assigned randomly, in a 2:1 ratio, to receive either imetelstat or placebo.
The primary efficacy endpoint is designed to be the rate of red blood cell transfusion-independence lasting at least eight weeks, defined as the proportion of patients without any red blood cell transfusion during any consecutive eight weeks since entry to the trial. A primary efficacy analysis is planned to occur 12 months after the last patient is enrolled.
Further information about the trial, including participating medical centers around the world, can be found at View Source
Regulatory Designations
● The United States Food and Drug Administration has granted orphan drug designation to imetelstat for the treatment of MF and for the treatment of MDS. In addition, the European Medicine Agency has granted orphan drug designation to imetelstat for the treatment of MF.