Sandoz, a unit of Novartis AG (NYSE:NVS; SIX:NOVN), said FDA has accepted filing a BLA for a biosimilar version of Neupogen filgrastim G-CSF from Amgen Inc. (NASDAQ:AMGEN). Sandoz is the first company to announce FDA's acceptance of a biosimilar application.
Sandoz's filgrastim biosimilar is marketed as Zarzio in more than 40 countries, is the "number one biosimilar filgrastim globally and is the leading daily G-CSF in Europe with 30% volume market share," according to the company.
"Filgrastim is a relatively simple biologic about which a lot is known, and experience from other countries indicate that it will change the market dynamics for health plans, providers, and manufacturers when it becomes available as a biosimilar in the U.S.," Gillian Woollett, SVP at Avalere Health, told BioCentury.
Woollett said approval of the Sandoz application could come as early as 1Q15. She added a potential approval will provide evidence of whether biosimilars "will provide competition in the specialty drug market, increase access for patients and enable payers to hold down costs."
EMA accepted for review an MAA from GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) for its RTS,S malaria vaccine -- moving the product closer to being the first vaccine approved to prevent malaria infection in infants and children. There are currently no approved vaccines for malaria. A positive EMA opinion on the MAA would be the basis for regulatory applications for the malaria vaccine in countries in sub-Saharan Africa, which accounts for about 90% of annual malaria deaths. GSK said it does not expect a decision on the vaccine until next year. Pending EMA's decision, GSK said the World Health Organization also could issue an immunization policy recommendation for RTS,S by the end of 2015.
GSK is developing RTS,S -- a vaccine that targets the circumsporozoite protein (CSP) sequence of Plasmodium falciparum and is combined with the AS01 adjuvant -- with the Program for Appropriate Technology in Health (PATH) Malaria Vaccine Initiative (MVI). The pharma's malaria vaccine team discussed developing RTS,S and the regulatory process in an interview last year on BioCentury This Week television (see BioCentury This Week , Sept. 22, 2013).
AS01 contains the QS-21 Stimulon adjuvant from Agenus Inc. (NASDAQ:AGEN). The RTS,S MAA submission triggered an undisclosed milestone payment to Agenus, which is also eligible for low single-digit royalties. Agenus separately reported 2Q14 financial results that beat the Street on Thursday, including revenues of $3.1 million. The stock was up $0.34 (11%) to $3.39 on the day.
Bristol-Myers Squibb Co. (NYSE:BMY) and Ono Pharmaceutical Co. Ltd. (Tokyo:4528) partnered to jointly develop and commercialize multiple products, including Opdivo nivolumab and Yervoy ipilimumab, in Japan, South Korea and Taiwan. The deal also includes early stage BMS oncology compounds lirilumab (BMS-986015; IPH2102), urelumab (BMS-663513) and BMS-986016. The partners will jointly pursue development of monotherapy and combination regimens of the immunotherapies.
Under the deal, the partners will share equally development costs and commercial profits when Opdivo is used in combination with any BMS product. BMS will fund the majority of development costs and receive the majority of commercial profits when one of its compounds is used as a monotherapy or two of its compounds are used in combination. Ono will fund the majority of development costs and receive the majority of commercial profits when Opdivo is used as a single agent.
Opdivo, a human IgG4 mAb against programmed cell death 1 (PDCD1; PD-1; CD279), is approved in Japan to treat unresectable melanoma, while Yervoy, a human mAb against CTLA-4 receptor, is approved in Taiwan to treat advanced melanoma. Prior to this deal, BMS had worldwide rights to Opdivo excluding Japan, South Korea and Taiwan, where Ono retained rights. Urelumab, a mAb agonist of tumor necrosis factor (TNF) receptor superfamily member 9 (TNFRSF9; 4-1BB, CD137), and BMS-986016, an anti-lymphocyte-activation gene 3 (LAG3; CD223) mAb, are in Phase I testing. Lirilumab, a human anti-killer cell immunoglobulin-like receptors (KIR) mAb, is in Phase II testing as maintenance treatment for acute myelogenous leukemia (AML). BMS has exclusive, worldwide rights to lirilumab from Innate Pharma S.A. (Euronext:IPH).
Separately on Thursday, BMS reported 2Q14 financial results with non-GAAP EPS of $0.48 on revenues of $3.89 billion. The Street was expecting EPS of $0.44 on revenues of $3.85 billion. Yervoy sales were up 38% to $321 million in the quarter. BMS also lowered its 2014 non-GAAP EPS guidance to $1.50-$1.60 from $1.70-$1.80.
Comments by a Gilead Sciences Inc. (NASDAQ:GILD) executive during the company's 2Q14 earnings call suggest the company may not price HCV therapeutic ledipasvir (GS-5885) as high as Sovaldi sofosbuvir. EVP of Commercial Operations Paul Carter said Gilead hasn't finalized pricing for its fixed-dose combination of Sovaldi and ledipasvir, but that the company feels the "majority of the value" of the combination is in Sovaldi. The combination is under Priority Review at FDA with an Oct. 10 PDUFA date and is also under accelerated assessment in the EU.
Gilead, which released its earnings after market close on Wednesday, reported 2Q14 financial results that beat the Street, including $3.5 billion in Sovaldi sales (see BioCentury Extra, July 23).
On Thursday, Express Scripts Holding Co. (NASDAQ:ESRX) noted in a blog post that new HCV therapies are expected to come to market later this year, and that patients who are asymptomatic may be able to wait until additional options are available.
Earlier this year, Express Scripts said it would ask plan sponsors to "stand side-by-side" with the pharmacy benefit manager to encourage Gilead to lower Sovaldi's price. Sovaldi has a wholesale acquisition cost of $84,000 for a 12-week course of therapy (see BioCentury Extra, April 8).
Sarepta Therapeutics Inc. (NASDAQ:SRPT) disclosed in an SEC filing that Arthur Krieg was terminated as SVP and CSO on July 22. Krieg, co-founder and formerly CEO of RaNA Therapeutics Inc. (Cambridge, Mass.), joined Sarepta in January to run the company's drug discovery and early stage research programs. CEO Chris Garabedian declined to comment on Krieg's termination and said the company does not currently have a replacement. Garabedian did say Sarepta hopes that a future CSO "will have desires that align with fully utilizing the technologies available to our company."
Sarepta was off $0.72 to $20.63 on Thursday.
Celgene Corp. (NASDAQ:CELG) reported 2Q14 earnings that beat the Street on Thursday and raised its full-year EPS and revenue guidance. The company reported 2Q14 adjusted diluted EPS of $0.90, beating by a penny the Street's $0.89 estimate and up from $0.76 in 2Q13. Total revenues in the quarter were $1.87 billion, above the Street's $1.85 billion estimate and up 17% from $1.6 billion in 2Q13.
Sales of Otezla apremilast were $5 million in the quarter; Celgene launched the drug in the U.S. in early April to treat psoriatic arthritis. Analysts expect the drug could hit sales of $2.1 billion by 2018. Otezla is under FDA review to treat moderate to severe psoriasis, with a Sept. 23 PDUFA date, and is under review in the EU for moderate to severe psoriasis and psoriatic arthritis. Celgene also disclosed that FDA accepted for review an sNDA for Revlimid lenalidomide to treat newly diagnosed multiple myeloma. The PDUFA date is Feb. 22, 2015.
Additionally, Celgene raised its full-year guidance and now expects 2014 adjusted diluted EPS of $3.60-$3.65 on total revenues of $7.6 billion. The company previously expected 2014 EPS of $3.50-$3.60 on total revenues of $7.5 billion. The Street was expecting full-year EPS of $3.67 on revenues of $7.54 billion.
Celgene was off $2.93 to $86.19 on Thursday.
Cancer immunotherapy company Immune Design Corp. (NASDAQ:IMDZ) and biosimilar play Pfenex Inc. (NYSE:PFNX) priced their IPOs on Thursday. Immune Design raised $60 million through the sale of a bumped-up number of shares at the low end of its proposed range. Immune Design sold 5 million shares at $12, which values that company at $190.5 million. Last week, the company said it planned to sell 4.7 million shares at $12-$14. Jefferies; Leerink Partners; and Wells Fargo are underwriters.
Immune Design has several immunotherapies in Phase I testing for solid tumors or Merkel cell carcinoma, including G100 and CMB305. G100 is a toll-like receptor 4 (TLR4) agonist, and CMB305 is a combination of the company's LV305 and G305, which are both also in Phase I testing as single agents. LV305 is a lentiviral vector engineered to deliver antigen-encoding nucleic acids directly to dendritic cells in vivo. G305 is an immunotherapy comprised of recombinant cancer/testis antigen 1B (CTAG1B; NY-ESO-1) and synthetic glucopyranosyl lipid A (GLA) adjuvant.
Pfenex raised $50 million through the sale of a bumped-up number of shares below its proposed price. The company sold 8.3 million shares at $6, which values the company at $115.4 million. Last week, Pfenex said it planned to sell 8.1 million shares at $8. William Blair; JMP Securities; and Mizuho Securities are underwriters. Pfenex's PF-582 -- a biosimilar of ophthalmic drug Lucentis ranibizumab -- is in a Phase Ib/IIa trial for wet age-related macular degeneration (AMD), with Phase III testing slated to start in mid-2015.
Immune Design was up $0.05 to $12.05 on its first day of trading Thursday. Pfenex was off $0.70 to $5.30 on its first day of trading.
Intersect ENT Inc. (NASDAQ:XENT) was up $1.92 (17%) to $12.92 on its first day of trading Thursday after raising $55 million through the sale of 5 million shares at $11 in an IPO. The $11 price valued the drug-delivery device company at $247.5 million. Earlier this month, Intersect said it planned to sell 5 million shares at $11-$13. JPMorgan; Piper Jaffray; Leerink; and Wedbush were underwriters. Intersect markets the Propel and Propel mini mometasone furoate implants to treat chronic sinusitis. The products are bioabsorbable, steroid-eluting implants. In 1Q14, the company had revenues of $7.5 million and a three-month operating loss of $4.1 million.
Infectious disease play Atox Bio Inc. (Ness Ziona, Israel) raised an undisclosed amount in the first tranche of a $23 million series E round led by new investor SR One. New investors Lundbeckfond Ventures and OrbiMed Israel also participated. Atox's AB103 has completed a Phase II trial to treat necrotizing soft tissue infections. In 2H15, the company plans to start a pivotal trial of the short peptide that modulates CD28 in the indication. Atox was spun out of Hebrew University in 2003 by its Yissum Research and Development Co. Ltd. tech transfer arm.
Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) reported 2Q14 earnings that beat the Street on Thursday and separately said EMA accepted and granted accelerated assessment of an MAA for asfotase alfa to treat hypophosphatasia (HPP). The company expects to complete a rolling BLA submission for the product -- a fusion protein incorporating the catalytic domain of human tissue non-specific alkaline phosphatase (TNSALP) and a bone-targeting peptide -- this fall. HPP is an inherited metabolic disease characterized by defective bone mineralization. An accelerated assessment shortens the review period to 150 days from 210.
Separately, Alexion reported 2Q14 non-GAAP diluted EPS of $1.12, beating the Street's $1.07 estimate and up from $0.73 in 2Q13. Sales of Soliris eculizumab, Alexion's sole marketed product, were $512.5 million, up 38% from the prior year's quarter and topping the Street's $510.3 million estimate. Soliris, a humanized mAb targeting complement 5 (C5), is approved in the U.S., EU, Japan and other countries to treat atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria (PNH).
Alexion raised its 2014 guidances and said it now expects a full-year non-GAAP EPS of $4.95-$5.05 on $2.18-$2.2 billion in revenues. Previously, the company expected EPS of $4.75-$4.85 on $2.15-$2.17 billion in revenues. The Street was expecting full-year EPS of $4.86 on $2.18 billion in revenues.
Alexion was off $5.60 to $167.48 on Thursday.
ContraFect Corp. (Yonkers, N.Y.) again amended its IPO and now plans to sell 5.5 million units at $5-$6 on NASDAQ. At the $5.50 midpoint, the company would raise $30 million. Each unit comprises a share, a class A warrant to purchase a share at $4.80 and a class B warrant to purchase half a share, with each whole warrant exercisable at $4. Earlier this month, ContraFect said it planned to sell 3.6 million units at the same price range. The company filed to raise up to $23 million in the offering in April. Maxim Group is the underwriter.
ContraFect is developing a class of enzymes called lysins, which break open the cell walls of Gram-positive bacteria, along with combinations of mAbs that target virulence factors. The company's CF-301, a lysin targeting Staphylococcus aureus, is in preclinical testing, with Phase I testing slated to start next year. CF-404, a combination of three human mAbs against influenza, is in preclinical testing for seasonal and pandemic influenza infections, with Phase I testing slated to begin in 2016 (see BioCentury, July 4, 2011).
Lantheus Holdings Inc. (North Billerica, Mass.) amended its planned IPO on NASDAQ and now plans to sell 9.3 million shares at $12-$15. At the $13.50 midpoint, the company would raise $125 million and be valued at $369.1 million. Lantheus filed to raise up to $125 million in the offering in June. Citigroup; Jefferies; RBC Capital Markets; Wells Fargo; and Baird are underwriters. Lantheus is the parent company of Lantheus Medical Imaging Inc., which develops, manufactures and markets diagnostic medical imaging agents and products. Lantheus had 2013 revenues of $283.7 million.
The U.S. Centers for Disease Control and Prevention said Michael Farrell resigned as team lead for the Bioterror Rapid Response and Advanced Technology (BRRAT) Laboratory following an incident last month in which laboratory personnel were potentially, unintentionally exposed to viable anthrax. Farrell had held the position since 2009.
Separately on Thursday, the CDC said it formed an external laboratory safety workgroup to provide advice on areas including corrective actions for CDC labs; identifying potential weaknesses and necessary safeguards based on experiences of non-CDC labs; and implementing additional safeguards. The group will meet for the first time in early August.
Crenezumab from the Genentech unit of Roche (SIX:ROG; OTCQX:RHHBY) missed the primary endpoint in the Phase II ABBY trial and missed a secondary endpoint in the Phase II BLAZE trial. The July 16 issue of BioCentury Extra mischaracterized the BLAZE results.