Amgen Inc. (NASDAQ:AMGN) said it will launch leukemia drug Blincyto blinatumomab on Thursday with a price of $3,178.57 per single-use vial, or $89,000 for a 28-day treatment cycle.
Amgen spokesperson Lori Melancon said nearly all of the patients in a Phase II trial received two cycles of the drug, which would cost $178,000. Blincyto's label says patients could receive up to three additional cycles for consolidation treatment.
On Dec. 3, FDA approved the bispecific T cell engager (BiTE) against CD19 to treat Philadelphia chromosome-negative precursor B cell acute lymphoblastic leukemia (ALL). The Orphan Drug's approval came more than five months ahead of its May 19, 2015, PDUFA date (see BioCentury Extra, Dec. 3).
Melancon said Amgen's Onyx Pharmaceuticals Inc. subsidiary will commercialize Blincyto in the U.S.
Shire plc (LSE:SHP; NASDAQ:SHPG) secured $2.1 billion in a revolving loan agreement amid speculation it was preparing to make an acquisition.
Both Shire and NPS Pharmaceuticals Inc. (NASDAQ:NPSP) declined to comment on media reports that Shire was mulling a bid for NPS, whose shares rose $5.63 (18%) to $36.10 on Wednesday, giving it a market cap of $3.9 billion.
Shire closed the third quarter with $468 million in cash. The company added another $1.6 billion in October as a breakup fee following the termination of a deal in which AbbVie Inc. (NYSE:ABBV) would have acquired Shire for $53.8 billion (see BioCentury Extra, Oct. 20).
On May 30, prior to the announcement of the failed AbbVie-Shire deal, NPS jumped $3.68 (13%) to $31.13 amid rumors that Shire was preparing a bid that would value NPS at more than $4 billion. NPS later denied Shire had approached it (see BioCentury Extra, May 30).
NPS posted $67.9 million in sales of Gattex teduglutide, known in Europe as Revestive, for short bowel syndrome (SBS) for the nine months ended Sept. 30. The company also received royalties of $89.5 million for the period, mainly from Sensipar cinacalcet. Its Natpara recombinant human parathyroid hormone 1-84 (PTH) is under FDA review to treat hypoparathyroidism, with a Jan. 24 PDUFA date.
Shire gained $6.49 to $215.25 on NASDAQ; in London, it closed up 32p to 4,522p.
Cancer immunotherapy play Flexus Biosciences Inc. (San Carlos, Calif.) said it has raised $38 million in two rounds of funding. Kleiner Perkins Caufield & Byers incubated the company and led its $13 million series A in October 2013, while The Column Group led its $25 million series B. The latter round, in which KPCB and Celgene Corp. (NASDAQ:CELG) also participated, included a tranche in July and a second installment due in January 2015.
The company has in-licensed FLX925, which inhibits FMS-like tyrosine kinase 3 (FLT3; CD135) and cyclin dependent kinases 4 (CDK4) and 6 (CDK6), from Amgen Inc. (NASDAQ:AMGN). Flexus submitted an IND for FLX925 to treat acute myelogenous leukemia (AML) in November, and expects to begin a Phase I trial in early 2015. Flexus has two undisclosed immunotherapy programs at the discovery and preclinical stages.
Flexus uses its Agents for the Reversal of Tumor Immunosuppression (ARTIS) platform to discover and develop small molecules that modulate tumor-infiltrating regulatory T cells. Next quarter, the company plans to select a clinical candidate, an inhibitor of indoleamine 2,3-dioxygenase 1 (IDO1). The company plans to begin a clinical trial by 4Q15.
Incyte Corp. (NASDAQ:INCY) has begun a Phase I/II trial of its IDO1 inhibitor, INCB024360 (INCB24360), in combination with Keytruda pembrolizumab, a humanized IgG4 mAb against PD-1 receptor (PDCD1; PD-1; CD279) from Merck & Co. Inc. (NYSE:MRK).
Flexus plans to investigate combinations of its candidates with other checkpoint inhibitors as soon as 2016, and is pursuing partnerships.
Former Amgen executive Terry Rosen is Flexus' CEO. KPCB's Beth Seidenberg is chairperson of its board, which also includes The Column Group's David Goeddel; William Rieflin, CEO of NGM Pharmaceuticals Inc. (South San Francisco, Calif.); Flexus President and Head of R&D Juan Jaen; and Rosen.
Cerus Corp. (NASDAQ:CERS) jumped $1.08 (23%) to $5.86 on Wednesday after FDA approved the company's Intercept Blood System for plasma. The plasma pathogen inactivation system using the light-activated compound amotosalen is designed to reduce the risk of transfusion-transmitted infections.
CEO William Greenman said the company will need "a couple of months" to label and ship kits containing the system to the U.S. The system is already available in the U.S. under an investigational device exemption (IDE) for the treatment of convalescent plasma collected from Ebola disease survivors for passive immune therapy. Cerus markets the system, as well as the Intercept Blood System for platelets, in Europe, the Commonwealth of Independent States (CIS) and the Middle East.
A PMA for the Intercept Blood System for platelets, a similar plasma pathogen inactivation system that uses light-activated molecules to crosslink DNA and RNA to prevent pathogen replication, is under FDA review. The platelet system is available under an expanded access IDE for use in the regions of the U.S. with outbreaks of chikungunya and dengue virus.
Halozyme Therapeutics Inc. (NASDAQ:HALO) added $1.01 (13%) to $8.65 on Wednesday after announcing a collaboration with the Janssen unit of Johnson & Johnson (NYSE:JNJ) to use the biotech's Hylenex (rHuPH20) to increase subcutaneous delivery of five of the pharma's compounds. Hylenex is a recombinant human PH20 hyaluronidase enzyme that enhances dispersion and absorption of drugs by temporarily degrading hyaluronan, a component of the extracellular matrix.
Janssen will receive exclusive, worldwide rights to develop and commercialize any products that arise from the collaboration. Halozyme is receiving $15 million up front and is eligible for up to $566 million in pre-specified development, regulatory and sales milestones, plus mid-single digit royalties.
The companies did not disclose the Janssen compounds, but Halozyme spokesperson Julie Normart said they include both approved drugs and investigational compounds.
Halozyme's other Hylenex deals include Roche (SIX:ROG; OTCQX:RHHBY) and Baxter International Inc. (NYSE:BAX). Roche markets subcutaneous formulations of Herceptin trastuzumab and MabThera rituximab using Hylenex in Europe. Baxter markets HyQvia -- a subcutaneous formulation of Baxter's Gammagard immune globulin using Hylenex -- in Europe and the U.S.
Pozen Inc. (NASDAQ:POZN) lost $0.93 (10%) to $8.10 after FDA issued a second complete response letter for the company's Yosprala combinations of aspirin and omeprazole for the secondary prevention of cardiovascular disease in patients at risk for aspirin-induced gastric ulcers.
The company said FDA used the same language from its first CRL in April, citing "inspection deficiencies" at the facility of a foreign third-party manufacturer of an active ingredient (see BioCentury Extra, April 28).
Pozen said the agency has not conducted a new inspection of the facility since April, and noted the supplier provided the agency with a plan to address the deficiencies in May. It declined to disclose the manufacturer or its location.
The company said it will request a meeting with FDA to discuss a path forward.
On Dec. 1, Pozen lost $1.26 (14%) to $7.63 after Sanofi returned U.S. rights to PA32540 and PA8140, which would be marketed as Yosprala. The candidates are tablets comprising low- or high-dose aspirin and an immediate-release coating of 40 mg omeprazole, a proton pump inhibitor (PPI). PA32540 contains 325 mg aspirin and PA8140 contains 81 mg aspirin.
Apotex Inc. (Toronto, Ontario) said FDA accepted for filing a regulatory application for a biosimilar of pegfilgrastim. The application was submitted under the 351(k) abbreviated approval pathway under the Biosimilar Price Competition and Innovation Act (BPCIA).
Pegfilgrastim is a biosimilar version of Neulasta pegylated G-CSF from Amgen Inc. (NASDAQ:AMGN), a long-acting treatment for neutropenia in cancer patients.
Apotex acquired rights to the biosimilar from Intas Pharmaceuticals Ltd. (Ahmedabad, India) in 2009.
In July, FDA accepted a BLA for EP2006, a biosimilar version of Neupogen filgrastim G-CSF from the Sandoz unit of Novartis AG (NYSE:NVS; SIX:NOVN). Amgen also markets Neupogen to treat neutropenia (see BioCentury Extra, Dec. 8).
In August, Celltrion Inc. (KOSDAQ:068270) submitted an application to FDA for Remsima infliximab, a biosimilar of Remicade from Johnson & Johnson (NYSE:JNJ) and Merck & Co. Inc. (NYSE: MRK) (see BioCentury Extra, Aug. 11).
H. Lundbeck A/S (CSE:LUN) said it will cease development of desmoteplase (DPSA) to treat acute ischemic stroke.
The compound, which has been in development for more than a decade and failed a Phase III study in 2007, received its latest clinical setback in June when it missed the primary endpoint in the Phase III DIAS-3 trial. Treatment with desmoteplase given 3-9 hours from onset of stroke symptoms did not lead to greater proportion of patients with a modified Rankin Scale (mRS) score of 0-2 points at day 90 vs. placebo (51.3% vs. 49.8%) (see BioCentury Extra, June 27).
Lundbeck will take a write-down of DKK309 million ($51.7 million) this quarter. It narrowed its 2014 core EBIT guidance to DKK1.1-DKK1.3 billion ($184-$217.5 million), from DKK0.9-DKK1.4 billion ($150.6-$234.2 million). The company said it will explore alternatives for the compound, including divestiture.
Desmoteplase, a genetically engineered fibrin-dependent salivary plasminogen activator from the vampire bat Desmodus rotundus, has Fast Track designation in the U.S. to treat ischemic stroke. Lundbeck licensed rights from Paion AG (Xetra:PA8) in 2005 for EUR 15 million ($17.8 million) up front and up to EUR 50 million ($59.5 million) in milestones plus royalities, and then paid EUR 20.1 million ($24.7 million) in cash for all remaining rights in 2012. The biotech had acquired the asset from Schering AG in 2000.
Silicon Valley entrepreneur Sean Parker pledged $24 million over the next two years to establish a research center at the Stanford University School of Medicine that aims to discover lasting cures for allergies. Parker co-founded file-sharing service Napster Inc. and was president of Facebook Inc.
The Sean N. Parker Center for Allergy Research will focus on understanding mechanisms of the immune system and the development of "rationally-based" therapies using laboratory and computational research. It will be led by Kari Nadeau, associate professor of pediatrics at the medical school and immunologist at the Lucile Packard Children's Hospital Stanford and Stanford Health Care.
On a conference call, Nadeau said the center would focus on using new knowledge of the immune system to create "long-lasting cures," and highlighted food allergies as a pressing unmet need. The center's research on immune dysfunctions also could include conditions such as asthma, eczema, eosinophilic disorders, drug allergies and gastroenterological diseases.
In its first year, the center aims to start proof of concept clinical trials. It will focus on training the next generation of allergy immunologists during its second year.
Of the $24 million, $4 million will be dedicated to a dollar-for-dollar challenge match for other new gifts to the center.
The U.K. will launch a "Precision Medicine Catapult" next year as part of the government's commitment to spend L5.9 billion ($9.3 billion) on its science and innovation strategy from 2015-21.
The plan, released on Wednesday, also pledges to create a National Formulation Centre to help U.K. companies develop medicines and chemicals that are deemed too high risk or technically challenging.
The Catapult centers aim to bring universities and businesses together to spur innovation and support emerging companies. The U.K. already has seven catapults, including one dedicated to cell therapies.
Of the L5.9 billion, L2.9 billion will support large-scale investments including in big data and manufacturing, and L3 billion will be spent on individual research projects and facilities.
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