Chiesi Farmaceutici S.p.A (Parma, Italy) has filed a pricing dossier for Glybera alipogene tiparvovec with Germany's Federal Joint Committee (G-BA) that pegs the price of the gene therapy at over EUR 1 million. Chiesi has commercial rights to Glybera in Europe from uniQure N.V. (NASDAQ:QURE).
In the dossier, Chiesi seeks a retail price of EUR 53,000 ($65,625) per vial, or about EUR 1.1 million ($1.4 million) for a 62.5 kg patient who would require 21 vials.
Glybera is an adeno-associated virus (AAV) vector encoding the lipoprotein lipase (LPL) gene that was approved in Europe in 2012 to treat LPL deficiency in patients with recurring, acute pancreatitis, a rare disorder.
At the time of approval, uniQure said it planned to price the drug at EUR 250,000 ($322,625) per year of duration of clinical benefit, which was estimated at five years. The company also said it was considering a longer-term annuity pricing model, with an upfront payment followed by annual installments based on the duration of benefit.
However, under the subsequent 2013 deal, Chiesi is responsible for setting the price in Europe. Chiesi also has commercialization rights in Brazil, Mexico, Pakistan, Turkey, the Commonwealth of Independent States (CIS) and China. It did not reply to requests for comment in time for publication (see BioCentury, Sept. 2 , 2013).
uniQure shares jumped $2.32 (19%) to $14.82 on Friday.
Bristol-Myers Squibb Co. (NYSE:BMY) said FDA issued a complete response letter for daclatasvir on Wednesday, requesting additional safety and efficacy data for the HCV drug in combination with other antiviral agents. BMS said its NDA for the selective HCV NS5A protein inhibitor focused on its combination with asunaprevir, an HCV NS3 protease inhibitor. Last month, BMS withdrew an NDA for asunaprevir and said it would no longer pursue approval of a combination of the two direct-acting antivirals (see BioCentury Extra, Oct. 7).
Company spokesperson Carrie Fernandez told BioCentury BMS anticipates resubmitting the NDA with additional data from completed and ongoing trials without performing additional studies, although it is still discussing with FDA the scope of the data necessary for resubmission. She declined to say which new combinations BMS might include in its resubmission, or give a specific timeline for resubmission.
In August, the European Commission approved daclatasvir as Daklinza to treat HCV genotypes 1, 2, 3 and 4. The drug's European label recommends its use to treat genotypes 1 and 4 with or without compensated cirrhosis in combination with Sovaldi sofosbuvir from Gilead Sciences Inc. (NASDAQ:GILD); HCV genotype 3 with compensated cirrhosis or previous HCV treatment in combination with Sovaldi and ribavirin; and HCV genotype 4 in combination with ribavirin and peginterferon alfa (see BioCentury Extra, Aug. 27).
Japanese regulators approved daclatasvir in July to treat chronic HCV genotype 1 infection with and without compensated cirrhosis.
Fernandez said BMS's daclatasvir program is focused on difficult-to-treat populations including pre- and post-liver transplant patients, HCV patients co-infected with HIV, genotype 3 patients and cirrhotic patients.
Astellas Pharma Inc. (Tokyo:4503) said a pooled analysis showed that patients receiving fidaxomicin to treat Clostridium difficile infection (CDI) had reduced mortality, recurrence rates and healthcare costs compared with standard-of-care (SOC) vancomycin and metronidazole.
The real-world study data came from five U.K. hospitals that began giving patients fidaxomicin as first-line treatment for CDI in 2012. Data were presented Wednesday at the Federation of Infection Societies (FIS) conference.
Astellas said 28-day all-cause mortality was 7.6% in the fidaxomicin group compared to 23.3% for patients receiving SOC. Infection recurrence rates within 12 months were 2.8% in the fidaxomicin group vs. 10.6% for SOC. No patients receiving fidaxomicin had second recurrences, while among those receiving SOC who had already had one recurrence, 23.8% had a second recurrence.
Astellas said that for every 50 infections, fidaxomicin led to six fewer recurrences and saved NHS L40,841 ($63,879). The company said 300,000 cases of CDI occur in Europe annually.
In 2012, NICE concluded in its summary of clinical evidence that fidaxomicin was non-inferior to vancomycin, but did not formally recommend fidaxomicin. Astellas did not respond to questions about whether the company would use the data to seek NICE's recommendation (see BioCentury Extra, July 13, 2012).
Astellas has rights to the macrocyclic narrow-spectrum antibiotic in the EU, Japan and territories in the Middle East, Africa and Commonwealth of Independent States from Optimer Pharmaceuticals Inc., which Cubist Pharmaceuticals Inc. (NASDAQ:CBST) acquired in 2013 (see BioCentury, Aug. 5, 2013). The drug is approved to treat C. difficile-associated diarrhea in the U.S., where it is sold by Cubist as Dificid, and in the EU, where Astellas sells it as Dificlir. Cubist added $0.69 to $75.65 on Wednesday.
Sequencing analytics play and University of Oxford spin-out Genomics Ltd. (Oxford, U.K.) raised L10.3 million ($16.1 million) in its "first significant" round of funding, according to CEO John Colenutt. New investors Invesco Perpetual; Lansdowne Partners; Woodford Investment Management and the Wylie Family Trust participated in the untranched round alongside existing shareholders IP Group and the University of Oxford.
Founded by Oxford statistics professors Peter Donnelly and Gil McVean, Genomics plans to partner with pharmaceutical companies interested in using its genome sequencing data analytics platform during drug development.
Woodford founder and former Invesco fund manager Neil Woodford is Genomics' chairman. He also co-founded sequencing equipment maker Oxford Nanopore Technologies Ltd. (Oxford, U.K.), another Oxford spin-out. Oxford Nanopore raised L35 million ($58.8 million) in August (see BioCentury Extra, Aug. 12).
Kite Pharma Inc. (NASDAQ:KITE) said it plans to raise up to $138 million in a follow-on underwritten by Jefferies; Credit Suisse; Cowen; and Stifel. Kite raised $146.6 million through the sale of 8.6 million shares at $17 in an IPO in June.
Kite's lead program is KTE-C19, a therapy comprising autologous T cells genetically modified to express a chimeric antigen receptor (CAR) against CD19. Next year, the company plans to start Phase II trials of KTE-C19 to treat relapsed/refractory mantle cell lymphoma (MCL), chronic lymphocytic leukemia (CLL) and acute lymphoblastic leukemia (ALL), as well as a Phase I/II trial to treat relapsed or refractory diffuse large B cell lymphoma (DLBCL).
Last month, data published in The Lancet showed that patients receiving KTE-C19 had a 70% complete response rate (CRR) in a Phase I trial to treat relapsed or refractory ALL (see BioCentury Extra, Oct. 13).
Kite, which made the announcement after market close, slipped $1.24 to $42.96 on Wednesday.
S1 Biopharma Inc. (New York, N.Y.) amended its IPO on NASDAQ and plans to sell 1.75 million units at $12-$14. At $13, the company would raise $22.8 million and be valued at $105.7 million. Each unit comprises one share and one each of series A and series B warrants to purchase an additional share at the IPO price. The series A warrants expire in 2019 and the series B warrants expire in 2016. MLV and Northland Securities are underwriters.
S1 filed to raise up to $40.3 million on Oct. 1. Last month, the company amended its IPO to sell 2.8 million shares, without warrants, at $12-$14. The company is classified as an "emerging growth company" under the Jumpstart Our Business Start-Ups (JOBS) Act of 2012.
S1 plans to begin Phase IIb trials of lead candidate Lorexys to treat hypoactive sexual desire disorder (HSDD) in women in 1H15, and expects interim data from the first study by YE15. Lorexys is an oral fixed-dose enteric formulation of bupropion and trazodone.
Singapore's Agency for Science, Technology and Research launched its Diagnostics Development (DxD) Hub to speed the development and approval of new diagnostics by connecting clinicians, researchers and entrepreneurs with industry professionals.
DxD is the first of four innovation clusters Singapore expects to fund under its S$200 million ($153.5 million) Innovation Cluster Program. It has planned other clusters addressing speech and language technologies, "membranes" and additive manufacturing, but has released few details.
DxD's launch partners include local healthcare and academic centers SingHealth, National University Health System, National Healthcare Group and Singapore Clinical Research Institute. DXD's industry partners include BGI (Shenzhen, China), Johnson & Johnson (NYSE:JNJ) and Thermo Fisher Scientific Inc. (NYSE:TMO).