The Sandoz unit of Novartis AG (NYSE:NVS; SIX:NOVN) launched Zarxio filgrastim-sndz in the U.S. at a 15% discount to its reference drug, Neupogen filgrastim G-CSF from Amgen Inc. (NASDAQ:AMGN). Zarxio is the first biosimilar approved and marketed in the U.S.
Novartis spokesperson Eric Althoff said Zarxio's wholesale acquisition cost (WAC) is $275.66 for a 300 ug/0.5 mL dose and $438.98 for a 480 ug/0.8 mL dose. The WAC for equivalent doses of Neupogen is $324.30 and $516.45, respectively.
FDA approved Zarxio on March 6. A July ruling by the U.S. Court of Appeals for the Federal Circuit (CAFC) had barred Sandoz from launching the drug until after Sept. 2 (see BioCentury Extra, Sept. 2).
Amgen and Sandoz filed separate petitions with the CAFC last month for a rehearing en banc. Each party's response to the petitions is due Sept. 8.
The University of Texas MD Anderson Cancer Center (Houston, Texas) and Cellectis S.A. (Euronext:ALCLS; NASDAQ:CLLS) entered a seven-year collaboration to co-develop four allogeneic chimeric antigen receptor T cell therapies from Cellectis to treat liquid tumors. Cellectis' candidates are expected to enter clinical testing within the next 12 months.
The partners will develop UCARTCS1, UCART22 and UCART38 to treat T cell acute lymphocytic leukemia (ALL). UCARTCS1 targets SLAM family member 7 (SLAMF7; CS1), UCART22 targets CD22 and UCART38 targets CD38.
MD Anderson and Cellectis also will develop UCART123, a CAR T cell therapy targeting interleukin-3 (IL-3) receptor alpha (CD123), to treat blastic plasmacytoid dendritic cell neoplasm (BPDCN). Cellectis already has partnered with Weill Cornell Medical College (New York, N.Y.) to develop UCART123 to treat acute myelogenous leukemia (AML) (see BioCentury Innovations, June 25).
Cellectis will provide research funding to MD Anderson.
In January, MD Anderson granted Ziopharm Oncology Inc. (NASDAQ:ZIOP) and Intrexon Corp. (NYSE:XON) an exclusive license to CAR T cell technologies that use Sleeping Beauty, a non-viral, plasmid-based gene transfer system (see BioCentury Extra, Jan. 14).
Amgen Inc. (NASDAQ:AGMN) priced Repatha evolocumab in several European countries. The drug will cost L340.20 ($524.42) for a 28-day supply in the U.K., or about $6,800 a year, compared to EUR 7,293 ($8,180) in Austria and EUR 7,825 ($8,777) in Finland.
Amgen priced the PCSK9 inhibitor at $14,100 annually in the U.S. (see BioCentury Extra, Aug. 27).
Praluent alirocumab, a competing PCSK9 inhibitor from Sanofi (Euronext:SAN; NYSE:SNY) and Regeneron Pharmaceuticals Inc. (NASDAQ:REGN), was priced at $14,600 a year in the U.S. (see BioCentury Extra, July 24).
Amgen spokesperson Kristen Davis told BioCentury that "prices across all European countries have not yet been finalized but the cost of a medicine reflects the unique healthcare environment and marketplace of a specific country or region."
Acadia Pharmaceuticals Inc. (NASDAQ:ACAD) submitted an NDA to FDA and requested Priority Review for Nuplazid pimavanserin to treat Parkinson's disease psychosis. The small molecule serotonin (5-HT2A) receptor inverse agonist has breakthrough therapy designation for the indication.
Acadia had hoped to submit the NDA in 1Q15, but delayed the process to prepare its commercial manufacturing systems (see BioCentury Extra, March 11).
Also on Thursday, Acadia named Steve Davis president and CEO. Davis had been interim CEO since Uli Hacksell retired in March.
Acadia gained $1.06 to $39.13 on Thursday.
Kyle Bass' Coalition for Affordable Drugs filed three new inter partes review (IPR) petitions challenging patents covering Ampyra dalfampridine from Acorda Therapeutics Inc. (NASDAQ:ACOR) on grounds of obviousness.
Two of the petitions challenge U.S. Patents Nos. 8,007,826 and 8,663,685. Last week, the Patent Trial and Appeal Board denied separate IPR petitions from Bass against those two patents (see BioCentury Extra, Aug. 27).
The third petition challenges U.S. Patent No. 8,440,703.
The U.K.'s NICE issued draft guidance conditionally recommending Vimizim elosulfase alfa from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) to treat mucopolysaccharidosis IVA (MPS IVA; Morquio's syndrome), and issued final guidance recommending against use of Otezla apremilast from Celgene Corp. (NASDAQ:CELG) to treat chronic plaque psoriasis.
NICE required that BioMarin develop a protocol outlining clinical criteria for starting and stopping treatment with Vimizim, collaborate with patient organizations and NHS England to collect real-world data on the drug's use, and continue to support the MPS IVa registry.
In June, NICE's Highly Specialised Technologies (HST) Evaluation Committee requested additional information on Vimizim's cost-effectiveness and said it was "minded not to recommend" the recombinant human N-acetylgalactosamine-6-sulfatase. The European Commission approved Vimizim last year (see BioCentury Extra, June 4).
NICE estimated that Vimizim's average annual cost is L394,680 ($608,400). BioMarin proposed a patient access scheme to provide the drug at an undisclosed discount.
Comments on the Vimizim guidance are due Sept. 23.
NICE's final draft guidance for Otezla was in line with previous draft guidance recommending against its use to treat moderate to severe chronic plaque psoriasis in adults who have not responded to systemic therapy or when systemic therapy is contraindicated or not tolerated.
NICE said there was insufficient evidence showing Otezla slows disease progression compared to tumor necrosis factor (TNF) alpha inhibitors. The oral phosphodiesterase-4 (PDE-4) inhibitor was approved in the EU in January to treat active psoriatic arthritis and moderate to severe chronic plaque psoriasis in adults.
BioMarin was off $2.81 to $126.45 on Thursday, while Celgene slipped $1.93 to $117.65.
UCB Group (Euronext:UCB) is selling U.S. generics subsidiary Kremers Urban Pharmaceuticals Inc. to Lannett Co. Inc. (NYSE:LCI) for $1.2 billion in cash. The parties expect the deal to close in 4Q15. Lazard and BNP Paribas are advising UCB, and Covington & Burling is its counsel. UCB gained EUR 1.76 to EUR 71.20 on Thursday.
Gene therapy play Genenta Science s.r.l. (Milan, Italy) named Tiziana Mattioni COO. Mattioni was director and global program leader in oncology at Merck KGaA (Xetra:MRK).
Newco Faraday Pharmaceuticals Inc. (Seattle, Wash.) named Stephen Hill CEO. Hill was president and CEO of Targacept Inc., which merged with Catalyst Biosciences Inc. (NASDAQ:CBIO).
Data from a Phase I trial published in Science Translational Medicine showed that chimeric antigen receptor T cell therapy CTL019 led to a 57% overall response rate among 14 patients with chronic lymphocytic leukemia (CLL), including three patients whose remissions have lasted at least 53 months.
The University of Pennsylvania and Novartis AG (NYSE:NVS; SIX:NOVN) partnered in 2012 to develop the treatment. UPenn had published preliminary results from the trial in 2011 (see BioCentury, Aug. 13, 2012).
UPenn said eight of 14 heavily pretreated CLL patients had responses, including four complete remissions and four partial responses. Among those with CRs, one died of an infection after removal of a basal cell carcinoma while in remission.
UPenn said T cell expansion was less robust in the six patients who did not respond to the therapy.
CTL019 consists of autologous T cells loaded with a lentiviral vector expressing CART-19, which consists of a cancer antigen-binding domain targeting CD19 linked to CD3zeta/CD137 immunostimulatory domains.
Geron Corp. (NASDAQ:GERN) climbed $0.40 (13%) to $3.45 on Thursday after data from two Phase II trials of imetelstat (GRN163L) to treat essential thrombocythemia (ET) and myelofibrosis (MF) were published in The New England Journal of Medicine.
The ET data were in line with previously reported results. The 18-patient trial in ET patients who were refractory to or intolerant of conventional therapies showed imetelstat led to a hematologic response rate of 100% and a complete response rate of 89%.
The MF data were consistent with results presented at the American Society of Hematology (ASH) meeting last year. The 33-patient trial in patients with intermediate-2 or high-risk MF showed imetelstat led to a 21% response rate, including four CRs and three partial responses. Six of the seven patients who responded to treatment remained in remission after a median of 9.9 months.
Three of the four complete responders had splicing factor 3B subunit 1 (SF3B1) or U2 small nuclear RNA auxiliary factor 1 (U2AF1) mutations. Response rates were also higher in patients with Janus kinase 2 (JAK2) mutations and in patients who did not have additional sex combs like 1 (ASXL1) mutations.
In November 2014, Geron granted the Janssen Biotech Inc. unit of Johnson & Johnson (NYSE:JNJ) exclusive, worldwide rights to develop and commercialize the oligonucleotide that competitively inhibits telomerase activity. Janssen began the Phase II IMbark study in July to treat MF, and plans to start a Phase II trial by YE15 in myelodysplastic syndrome (MDS) (see BioCentury Extra, Nov. 14, 2014).
California's Senate and Assembly both passed the California Right To Try Act (AB 159), a bill that would allow drug manufacturers and physicians to provide eligible terminally ill patients with investigational therapies that have completed a Phase I trial but have not been approved by FDA. Gov. Jerry Brown has 12 days to sign or veto the bill.
The Assembly originally passed the bill in May. This week, the Senate passed an amended version and returned it to the Assembly, which again passed the bill (see BioCentury Extra, May 19).
The Goldwater Institute said 24 other states have right-to-try laws. The institute drafted model legislation for the state bills (see BioCentury, June 30, 2014).
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