Amgen Inc. (NASDAQ:AMGN) boosted its revenue guidance for 2015 as it prepares to launch products in its new cardiovascular business and anticipates regulatory actions in its cancer franchise.
EVP of R&D Sean Harper said during Amgen's 1Q15 earnings call Tuesday that this month's launch of chronic heart failure drug Corlanor ivabradine gives the company an opportunity to build a presence in the U.S. cardiovascular market ahead of its anticipated launch of Repatha evolocumab to treat high cholesterol.
FDA approved Corlanor, a selective If channel inhibitor, last week to treat CHF. Repatha, a human mAb against PCSK9, has an Aug. 27 PDUFA date.
Amgen said 1Q15 revenues grew 11% to $5 billion from $4.5 billion in 1Q14, beating the Street's estimate of $4.9 billion. The company also reported non-GAAP EPS of $2.48, up 33% from $1.87 in 1Q14 and well above analysts' estimates of $2.10.
Revenue growth was driven by increased sales of Enbrel etanercept, Prolia denosumab, Epogen epoetin alfa, Sensipar cinacalcet, Xgeva denosumab and Kyprolis carfilzomib. Amgen raised its 2015 revenue guidance to $20.9-$21.3 billion from $20.8-$21.3 billion and its non-GAAP EPS guidance to $9.35-$9.65 from $9.05 to $9.40.
R&D spending fell by $138 million (14%) year over year as the company aims to reduce incremental spending by $400 million in 2015, including from SG&A. "You can expect that a substantial share of what we save in SG&A will be reinvested towards the launches," said CFO David Meline. At the same time, he said, clinical development would increase R&D spending over the rest of the year.
The company has an April 29 FDA panel review for talimogene laherparepvec to treat metastatic melanoma and a July PDUFA date for an sNDA for Kyprolis in multiple myeloma patients who have received at least one prior therapy. Kyprolis is under accelerated review in Europe for the same indication.
Amgen plans to submit a BLA and MAA for brodalumab to treat moderate to severe plaque psoriasis mid-year. The company said it ended a Phase II trial of the human IgG2 mAb against IL-17 receptor (IL17R; IL17RA) in asthma after a futility assessment.
Amgen also plans to submit regulatory applications next half for AMG 416 in the U.S. and EU to treat secondary hyperparathyroidism. AMG 416 is a peptide calcimimetic that acts on the parathyroid gland to reduce production of PTH.
The company said it will submit an application for ABP 501, its biosimilar of Humira adalimumab from AbbVie Inc. (NYSE:ABBV). Its launch is planned for 2017.
Amgen, which reported its numbers after market close, was up $2.49 to $168.46 on Tuesday.
Mylan N.V. (NASDAQ:MYL) rose $6.03 to $74.07 on Tuesday after Teva Pharmaceutical Industries Ltd. (NYSE:TEVA) proposed to acquire the company for $82 per share, or $40.1 billion in cash and stock. Teva's offer is a 23% premium to Mylan's close of $66.83 on April 16, before Mylan released a statement addressing rumors of a possible takeout.
In its April 17 statement, Mylan said the two generics businesses have "significant overlap" and the combination would likely violate anti-trust regulations. Teva on Tuesday said the combined company would benefit from a "more efficient and advanced infrastructure" and would have the "broadest portfolio in the industry." Mylan did not respond to inquiries. Teva declined to comment on potential anti-trust issues.
Also on Tuesday, Perrigo Co. plc (NYSE:PRGO; Tel Aviv:PRGO) said its board unanimously rejected a takeout bid from Mylan for $205 per share, or $28.9 billion. Perrigo said the offer "substantially undervalues" the company. Perrigo's rejection came after market close; the stock lost $5.26 to $192.82 on Tuesday (see BioCentury Extra, April 8).
Earlier this year, Mylan completed its acquisition of the ex-U.S. developed markets branded generics business of Abbott Laboratories (NYSE:ABT) and moved its tax domicile to the Netherlands (see BioCentury Extra, July 14, 2014).
Pharmacyclics Inc. (NASDAQ:PCYC) became the latest company to face a patent challenge from hedge fund manager Kyle Bass, joining Jazz Pharmaceuticals plc (NASDAQ:JAZZ), Shire plc (LSE:SHP; NASDAQ:SHPG) and Acorda Therapeutics Inc. (NASDAQ:ACOR).
Coalition for Affordable Drugs IV, an entity associated with Bass, filed an inter partes review (IPR) on Monday with the U.S. Patent and Trademark Office challenging U.S. Patent No. 8,754,090, which covers Imbruvica ibrutinib and expires in 2031. The '090 patent is one of 13 Orange Book-listed patents covering Imbruvica and the last to expire; the remainder expire in 2026.
According to the petition, the patent's claims covering a method for treating mantle cell lymphoma (MCL) with ibrutinib in patients who have already received one prior therapy were "anticipated by a prior art document." If the petition is accepted, the patent office would be expected to issue a final determination in 12 months, but could extend review for an additional six months.
Imbruvica has accelerated approval from FDA to treat MCL patients who have received one prior therapy. FDA also has approved the Bruton's tyrosine kinase (Btk) inhibitor for Waldenstrom's macroglobulinemia (WM), chronic lymphocytic leukemia (CLL) in patients with 17p deletion and CLL in patients who have received one prior therapy.
Last year, Pharmacyclics reported net product revenue of $492 million from Imbruvica.
AbbVie Inc. (NYSE:ABBV) expects to close its acquisition of Pharmacyclics this quarter, after which it will share U.S. rights to Imbruvica with the Janssen Biotech Inc. unit of Johnson & Johnson (NYSE:JNJ) under a 2011 deal. Janssen holds ex-U.S. rights to the drug. Consensus estimates project global sales of $8.3 billion for Imbruvica in 2025 (see BioCentury, March 16).
Coalition for Affordable Drugs IV is a subsidiary of Hayman Credes Master Fund, which is operated by Hayman Capital Management. Bass is the founder and chief investment officer of Hayman Capital.
BIO President and CEO Jim Greenwood criticized the first Bass petition against Acorda as a "cynical short-selling strategy against innovative biotech companies." Bass challenged the legitimacy of multiple sclerosis drug Ampyra dalfampridine.
Bass also is challenging patents for narcolepsy drug Xyrem sodium oxybate from Jazz and short bowel syndrome therapy Gattex teduglutide and ulcerative colitis (UC) drug Lialda mesalamine from Shire (see BioCentury Extra, April 3).
News of Bass' patent challenge emerged before market close on Monday. AbbVie gained $0.07 to $63.61 on Tuesday; it had gained $1.25 on Monday. J&J picked up $0.09 to $100.30 on Tuesday, after rising $0.63 on Monday.
Actelion Ltd. (SIX:ATLN) gained CHF6.10 to CHF123 on Tuesday after reporting earnings and raising its 2015 guidance. The company now expects core earnings growth in the low double digits, at constant exchange rates and excluding benefits derived from the reversals of U.S. rebates on its drugs. In February, it had expected low single-digit growth.
The company reported CHF95 million ($100 million) in sales of Opsumit macitentan in 1Q15, up from CHF68 million ($73.1 million) in 4Q14. The tissue-targeting endothelin receptor antagonist to treat pulmonary arterial hypertension (PAH) generated CHF180 million ($193.4 million) in sales during 2014, its first full year on the market.
Actelion's total 1Q15 sales were CHF515 million ($541.9 million), up 12% using constant exchange rates from CHF450 million ($511.7 million) excluding rebate reversals, in 1Q14.
Actelion reported diluted core EPS of CHF1.61, up CHF0.14 (12%) from CHF1.46 in 1Q14 and well above analysts' consensus estimate of CHF1.35.
MaxCyte Inc. (Gaithersburg, Md.) partnered with The Johns Hopkins University (Baltimore, Md.) to develop chimeric antigen receptor (CAR) T cell therapies to treat solid tumors based on MaxCyte's mRNA technology that results in transient expression of the CAR molecule, which MaxCyte hopes will lead to an improved safety profile.
The first program will be a mesothelin-targeted CAR, for which MaxCyte expects to submit an IND next year. Other preclinical targets and financial terms are undisclosed.
MaxCyte will provide technology to manufacture the mesothelin-targeted therapy, will own data from a planned trial, and will retain rights to the therapy. JHU will manage patients and manufacture the therapies onsite.
MaxCyte President and CEO Douglas Doerfler told BioCentury that producing mRNA-expressed CARs does not require selection or expansion of patients' cells, which could shorten manufacturing time compared to viral expression methods.
He said the process produces about eight doses of cells that temporarily express the CAR molecule, which allows the company to discontinue therapy if on-target, off-tumor toxicity develops.
Data in a Sunday presentation at the American Association for Cancer Research meeting in Philadelphia showed no complete or partial responders among five patients receiving CART-meso, a CAR directed against mesothelin from Novartis AG (NYSE:NVS; SIX:NOVN) and the University of Pennsylvania. There were some signs of efficacy, such as "the clearing of malignant cells in the pleural fluid of one patient" and "radiological and clinical evidence of stable to decreased burden of disease in one patient," according to an abstract from the UPenn presentation (see BioCentury Extra, April 20).
Advisory firm Avalere Health (Washington, D.C.) said Tanisha Carino, EVP of its life sciences unit, will leave the company to lead U.S. public policy at GlaxoSmithKline plc (LSE:GSK; NYSE:GSK). Avalere named Nancy McGee EVP of the life sciences unit. McGee was COO of the Lash Group.
EUSA Pharma (Hemel Hempstead, U.K.) named Lee Morley CEO. Founding CEO Bryan Morton will become executive chairman. Morley was VP and head of commercial at Jazz Pharmaceuticals plc (Dublin, Ireland), which acquired EUSA in 2012. The newly re-established EUSA acquired rights to several specialty hospital products from Jazz last week.
Regenerative therapy play TissueGene Inc. (Rockville, Md.) named Robert Newman as COO. Newman was SVP of manufacturing, program management, and clinical operations at Ziopharm Oncology Inc. (NASDAQ:ZIOP).
Cardiovascular company Heart Metabolics Ltd. (Dublin, Ireland) named William Daly president and CEO and Gregory Ayers CMO. Daly was SVP of operations and business development at Puma Biotechnology Inc. (NYSE:PBYI) and Ayers was a venture partner with MPM Capital.
Cristal Therapeutics (Maastricht, the Netherlands) hired Jeroen Tonnaer as CBO. Tonnaer was head of the European external scientific affairs team at Merck & Co. Inc. (NYSE:MRK).
Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) released updated 12-month data from an ongoing Phase II open-label extension study of patisiran to treat familial amyloidotic polyneuropathy (FAP). Alnylam said patisiran-treated patients who reached the 12-month endpoint (n=20) had a mean 2.5-point decrease in modified Neuropathy Impairment Score (mNIS+7). The company estimated that untreated FAP patients with similar baseline characteristics typically experience a 13- to 18-point increase in mNIS+7 after 12 months, based on historical data. Alnylam presented the 12-month data at this week's American Academy of Neurology meeting.
The company also released complete six-month results from the trial, showing that patients receiving patisiran had a mean 1.4-point decrease in mNIS+7. Alnylam said the updated data are consistent with previously announced results (see BioCentury, Oct. 20, 2014).
Patisiran is an RNAi therapeutic targeting the transthyretin (TTR) gene using second-generation lipid nanoparticle technology. There are no approved treatments in the U.S. for FAP, though Pfizer's TTR stabilizer Vyndaqel tafamidis is approved in the EU.
Alnylam plans to release an 18-month update of its Phase II results in late 2015 and is enrolling patients in a Phase III trial.
Alnylam's shares gained $2.02 to $120.70 on Tuesday.
Dimension Therapeutics Inc. (Cambridge, Mass.) raised $65 million in a series B round led by new investor New Leaf Venture Partners. Other new investors included Jennison Associates; Partner Fund Management; RA Capital; Rock Springs Capital; Tourbillon Global Ventures, and an undisclosed life sciences investor. Existing investors Fidelity Biosciences and OrbiMed also participated.
Dimension is developing gene therapies for rare diseases using its adeno-associated virus (AAV) platform. The company plans to start a clinical trial in 2H15 of its lead candidate, an as-yet-unnamed AAV delivering wild type factor IX, to treat hemophilia B.
In June 2014, Dimension closed a $30 million series A round from Fidelity and OrbiMed. It also partnered with Bayer AG (Xetra:BAYN) to develop and commercialize a gene therapy for hemophilia A (see BioCentury, June 30, 2014).
New Leaf's Mike Dybbs joined Dimension's board.
FDA issued final guidance on clinical trial endpoints to support efficacy claims and approval of treatments for advanced and metastatic non-small cell lung cancer (NSCLC).
The guidance is in line with the agency's 2011 draft guidance, which said overall survival (OS) should be the standard clinical benefit endpoint used to establish efficacy for NSCLC treatments, but that other endpoints, including progression-free survival (PFS), can be considered for regulatory decisions based on the patient population and compound's risk-benefit profile (see BioCentury Extra, June 20, 2011).
The final guidance says PFS may be appropriate as a primary efficacy endpoint to support approval if a trial is "designed to demonstrate a large magnitude for the treatment effect as measured by both the hazard ratio and absolute difference in median PFS" as well as an acceptable risk-benefit profile. FDA said sponsors should justify the use of PFS as a primary efficacy endpoint and the "magnitude of PFS effect considered likely to predict OS or represent clinical benefit."
Rep. Fred Upton (R-Mich.) said Tuesday he hopes to introduce a 21st Century Cures bill by the end of April. Upton, the chairman of the House Energy & Commerce Committee, said his staff and the staff of Rep. Diana DeGette (D-Colo.) "have spent countless hours negotiating over the last few months on policy language." Upton said he is "hopeful that we'll have an agreement by the end of the month and the legislative phase can swiftly progress."
E&C Committee Republicans released a discussion draft of the bill in January that has not been supported by any Democrats (see BioCentury, Feb. 9).
The Patient-Centered Outcomes Research Institute approved awards totaling more than $120 million to fund 34 comparative effectiveness research (CER) studies.
In the second round of awards to be granted through its Pragmatic Clinical Studies Initiative, PCORI will award about $58.5 million to fund five studies comparing different approaches to delivering care for breast cancer, Crohn's disease, fractures in older adults and treatments for children with bipolar disorder. The awards range from $7.9 million to $14 million each.
PCORI awarded the remaining $61.6 million to fund 29 studies that will compare approaches to improve outcomes for conditions including opioid addiction, arthritis, stroke, Parkinson's disease (PD), leukemia and chronic kidney disease.
PCORI has awarded $854.6 million for 399 projects since 2012. In February, it awarded $64.1 million to fund five CER studies under the Pragmatic Clinical Studies Initiative (see BioCentury Extra, Feb. 24).
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