Sanofi (Euronext:SAN; NYSE:SNY) and UCB Group (Euronext:UCB) partnered to jointly discover and develop oral small molecules targeting undisclosed pathways to treat immune-mediated diseases. The partners will use an approach developed by UCB to identify modulators of the pathway, for which the partners said biologics "have proven highly efficacious." UCB said the partners expect to identify a lead preclinical compound "shortly." UCB is eligible to receive more than EUR 100 million ($139 million) from Sanofi in an undisclosed upfront and milestone payments. The partners will split costs and profits evenly.
UCB was up EUR 0.05 to EUR 58.41 on Tuesday.
Late Tuesday, infectious disease company Chimerix Inc. (NASDAQ:CMRX) said it and FDA agreed on an open-label pilot trial to provide access to the biotech's brincidofovir (CMX001) to treat adenovirus infections in immunocompromised patients. Chimerix said it is also working with the agency on the design of a pivotal Phase III trial that would be a continuation of the open-label trial, which will enroll 20 patients. The first patient in the trial will be pediatric cancer patient Josh Hardy, who developed an adenovirus infection after a bone marrow transplant. An IV drug marketed for adenovirus infections led to kidney damage, and Hardy's doctors at St. Jude's Children's Research Hospital petitioned Chimerix for compassionate-use access to the company's oral brincidofovir.
Chimerix President and CEO Kenneth Moch had originally said making brincidofovir available outside of the company's trials "has the potential to slow or derail" Chimerix's ability to make the compound "available as soon as possible" to the thousands of patients each year who might benefit from it." Hardy's family's subsequently launched a media campaign seeking compassionate-use access to the compound. Chimerix said Hardy's story "helped accelerate a discussion" between FDA and the company on the "need for additional clinical development" for brincidofovir in adenovirus infection.
Brincidofovir is in the Phase III SUPPRESS trial for cytomegalovirus (CMV) infection in adults. Chimerix also completed an exploratory Phase II trial as preemptive therapy for adenovirus infection.
The company was up $1.20 to $20.80 on Tuesday. The company announced the agreement with FDA after market close.
Sen. Joe Manchin (D-W.Va.) became the second senator to call for HHS Secretary Kathleen Sebelius to overturn FDA's approval of Zohydro ER hydrocodone bitartrate from Zogenix Inc. (NASDAQ:ZGNX) because of the pain drug's "high potential for abuse." Last week, Sen. Charles Schumer (D-N.Y.) also sent a letter to Sebelius asking HHS to overturn Zohydro's approval. HHS said it will respond.
FDA approved Zohydro -- a non-abuse-deterrent, controlled-release formulation of hydrocodone -- in October to manage chronic pain severe enough to require a continuous, around-the-clock opioid analgesic when alternatives are inadequate. An advisory committee had recommended against approval due to concerns about the effectiveness of the classwide REMS for extended-release and long-acting opioids; the panel's prime concern was Zohydro's abuse potential. Zogenix launched Zohydro in select pharmacies last week (see BioCentury, Dec. 17, 2012).
Other legislators, patients groups, addiction centers and state attorney generals have also called on FDA Commissioner Margaret Hamburg to reconsider the agency's approval of Zohydro or to set a rigorous timeline for the product to be reformulated to be abuse deterrent. FDA said it will "review and respond directly to those that sent" letters.
Sebelius has overruled FDA once before. In 2011, she overruled FDA's recommendation for unrestricted over-the-counter use of emergency contraceptive Plan B One-Step levonorgestrel from Teva Pharmaceutical Industries Ltd. (NYSE:TEVA). A district court overturned Sebelius' decision last year (see BioCentury, April 8, 2013).
Bayer AG (Xetra:BAYN) and partner Onyx Pharmaceuticals Inc. said twice-daily oral Nexavar sorafenib as adjuvant treatment missed the primary endpoint of improving recurrence-free survival vs. placebo in the Phase III STORM trial to prevent recurrence of hepatocellular carcinoma (HCC). The double-blind, international trial enrolled about 1,100 HCC patients who had no detectable disease after surgical resection or local tumor ablation.
Nexavar is approved in more than 100 countries to treat unresectable HCC and advanced renal cell carcinoma (RCC). In the U.S., the inhibitor of CRAF (RAF1) and multiple receptor tyrosine kinases is also approved for locally recurrent or metastatic, progressive, differentiated thyroid carcinoma that is refractory to radioactive iodine treatment.
Bayer and Onyx, now part of Amgen Inc. (NASDAQ:AMGN), have a worldwide co-development agreement for Nexavar outside of Japan, where Bayer owns rights. Amgen was off $1.13 to $124.27 on Tuesday.
OxiGene Inc. (NASDAQ:OXGN) said IV Zybrestat fosbretabulin plus Avastin bevacizumab every three weeks met the primary endpoint of improving progression-free survival (PFS) vs. Avastin alone in the Phase II GOG 186I trial to treat recurrent ovarian cancer (HR=0.685, p<0.05). The combination missed the secondary endpoint of improving objective response rate (ORR) vs. Avastin alone. The open-label, U.S. trial enrolled 107 patients with platinum-sensitive and -resistant recurrent or persistent epithelial ovarian, fallopian tube or primary peritoneal carcinoma. The Gynecologic Oncology Group (GOG) conducted the trial, which was sponsored by NIH's National Cancer Institute (NCI). OxiGene said it is evaluating next steps for the combination, but could not be reached for details.
The company released the data while the market was open on Tuesday and gained $0.36 (17%) to $2.42 on the day. The stock jumped an additional $3.51 (145%) to $5.93 in early after-hours trading after OxiGene issued an updated press release that corrected an error in a headline that said the combination decreased PFS.
The Genentech Inc. unit of Roche (SIX:ROG; OTCQX:RHHBY) markets Avastin in the U.S., while Roche markets it elsewhere.
Glycotope GmbH (Berlin, Germany) raised EUR 55 million ($76.4 million) in a venture round from existing investors Jossa Arznei GmbH and Eckert Life Science Accelerator. Glycotope uses its glycosylation technologies to enhance the biological activity of both new and marketed therapeutic antibodies through the optimization of the proteins' glycosylation structures.
The company has two products in Phase IIb trials: PankoMab-GEX, a humanized IgG1 antibody targeting glycoepitope and mucin 1(MUC1; CD227), for maintenance treatment of ovarian carcinoma; and CetuGEX, a human version of cetuximab, a chimeric mAb against EGFR, to treat squamous cell carcinoma of the head and neck (SCCHN). Glycotope expects to complete the trials by the end of 2016.
The ImClone Systems Inc. subsidiary of Eli Lilly and Co. (NYSE:LLY) markets Erbitux cetuximab in North America. Merck KGaA (Xetra:MRK) markets it elsewhere.
Lumena Pharmaceuticals Inc. (San Diego, Calif.) secured $45 million in a series B round led by new investor New Enterprise Associates. New investors Adage Capital Management and RA Capital Management also participated, along with existing investors Pappas Ventures; RiverVest Venture Partners; and Alta Partners. NEA's Ed Mathers will join Lumena's board.
Lumena's LUM001 is in Phase II testing to treat primary biliary cirrhosis (PBC) in adults and Alagille syndrome in children. Lumena has exclusive, worldwide rights to the inhibitor of solute carrier family 10 sodium-dependent bile acid transporter member 2 (SLC10A2; ASBT; IBAT) from Pfizer Inc. (NYSE:PFE), which gained the compound through its 2003 acquisition of Pharmacia Corp.
Exosome Diagnostics Inc. (New York, N.Y.) raised $27 million in the initial close of a series B round co-led by new investors Qiagen N.V. (Xetra:QIA; NASDAQ:QGEN); and Arcus Ventures. New investors Tiger Partners; CD Ventures; and Monashee Capital also participated, along with existing investors NGN Capital and Forbion Capital Partners. Exosome said it could raise up to $40 million in the round. Arcus' Steven Soignet will join Exosome's board.
Exosome and Qiagen are partnered to develop and commercialize co-branded kits to capture and process RNA and DNA from biofluid exosomes and other microvesicles. In January, the partners expanded the deal to include development of a blood-based, molecular in vitro diagnostic based on exosomes to detect mutations of an undisclosed gene associated with non-small cell lung cancer (NSCLC) and other malignancies.
This year, Exosome also plans to launch its EXO106 prostate cancer diagnostic in the U.S to reduce unnecessary biopsies in patients with elevated levels of prostate-specific antigen (KLK3; PSA). EXO106 detects an mRNA signature in urine and will be available through the company's CLIA-certified lab.
TG Therapeutics Inc. (NASDAQ:TGTX) raised $18.1 million through the sale of 2.7 million shares at $6.71 in a private placement to JPMorgan Asset Management's Global Healthcare Fund. Ladenburg Thalmann underwrote the offering. TG's ublituximab (TG-1101) is in an open-label Phase II trial to treat chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL) in combination with Imbruvica ibrutinib from Pharmacyclics Inc. (NASDAQ:PCYC) and Johnson & Johnson (NYSE:JNJ). Ublituximab is a chimeric mAb against CD20.
TG announced the offering after market close on Tuesday. The company was up $0.11 to $6.71 on the day.
Versartis Inc. (Redwood City, Calif.) amended its IPO on NASDAQ and now plans to sell 4.6 million shares at $16-$19. At the $17.50 midpoint, the company would raise $80.5 million and be valued at $382.5 million. Morgan Stanley; Citigroup; Cowen; and Canaccord are underwriters. Last month, Versartis raised $55 million in a series E round and filed to raise up to $80 million in the IPO (see BioCentury Extra, Feb. 18).
Versartis is developing VRS-317, a once-monthly formulation of recombinant human growth hormone (rhGH), to treat growth hormone deficiency (GHD). The product is in the Phase Ib/IIa VERTICAL trial in children, with top-line data expected in June and a Phase III trial expected to start early next year. VRS-317 uses recombinant XTEN technology from drug delivery company Amunix Operating Inc. (Mountain View, Calif.). Versartis is a 50/50 JV between Amunix and Index Ventures.
Vital Therapies Inc. (San Diego, Calif.) revitalized plans for an IPO on NASDAQ. The company postponed its IPO in November, citing market conditions. On Tuesday, Vital filed an S-1A that no longer contains a share price or proposed number of shares. BofA Merrill Lynch; Credit Suisse; William Blair; and Canaccord are listed as underwriters.
Before postponing its IPO, Vital had planned to sell 4.4 million shares at $16-$18. At the $17 midpoint, the company would have raised $74.8 million and been valued at $277.4 million.
Vital's human liver cell-based extracorporeal liver assist device (ELAD) is in the Phase III trial VTI-208 trial to treat alcohol-induced liver decompensation (AILD), with data expected in 1H15. This half, Vital also plans to start the Phase III VTI-210 trial for severe acute alcoholic hepatitis (AAH). The device is a bio-artificial liver system comprising a cartridge of membranes separating immortalized human C3A hepatoblastoma cells from a patient's plasma, which circulates through the device.
Europe's Innovative Medicines Initiative launched the GETREAL project to help companies generate real-world data for payers during the drug development process, along with the clinical data necessary for regulatory approval. Topics for the project include designing clinical trials that generate real-world effectiveness data and developing tools and methods that measure a drug's effectiveness during a trial in a way that reflects clinical practice. Other stakeholders -- including patient groups, health technology assessment (HTA) agencies and payers -- will also provide input and will be part of a network of "healthcare decision makers."
The consortium members include 15 biotech and pharma companies from the European Federation of Pharmaceutical Industries and Associations (EFPIA), as well as universities across Europe. The project has a budget of EUR 16.3 million ($22.7 million), including EUR 8 million ($11.1 million) from IMI and EUR 6 million ($8.3 million) in in-kind contributions from consortium members.
EMA and European reimbursement authorities have committed to more upfront discussion with companies to inform development programs for both approval and reimbursement purposes, though some stakeholders have said that parallel scientific advice is unlikely to result in true harmonization of data requirements (see BioCentury, Dec. 23, 2013).
The U.S. Senate passed the Kids First Research Act (H.R. 2019), which would eliminate the options for taxpayers to fund presidential campaigns and party conventions and create a pediatric research fund managed by NIH's Common Fund. The bill authorizes $13 million per year through FY24 for the research fund. Some legislators and pediatric research advocates have expressed opposition to the bill, noting that the fund does little to offset sequestration and that the funds would still be subject to NIH's existing spending caps and would not provide the agency any additional funding beyond its authorized budget, instead diverting funds from other NIH programs.
According to an NIH report, the agency spent about $3.7 billion of its FY13 post-sequester budget of $29.2 billion on pediatric research funding. In FY14, NIH projects it will spend $3.8 billion of its $30.1 billion budget on pediatric research.
H.R. 2019 now goes to President Obama. The White House does not have a statement of administrative policy on the bill.
The American Society of Clinical Oncology projected annual costs for cancer care in the U.S. will increase to more than $173 billion in 2020, up from $104 billion in 2006, in part due to the cost of new therapies. In an annual report on trends in cancer care in the U.S., ASCO said oncologists need to reduce the use of unnecessary or ineffective cancer treatments, tests and procedures. The group also said patients, payers and providers should work together on how to define and measure value in cancer care. The U.S. cancer care system will also be strained by an increase in the number of cancer patients coupled with fewer oncologists, according to the report.
Among other recommendations, the report said instability in U.S. federal payment systems needs to be reduced, including through the repeal of the Medicare physician sustainable growth rate (SGR) formula -- a measure used by Medicare to calculate annual adjustments in payment rates to physicians. Congress has repeatedly acted to prevent any cuts to physician payments resulting from the SGR through one year "doc fixes." The latest doc fix expires on March 31, and Congress must repeal the SGR or pass another delay by then to avoid Medicare physician pay cuts (see BioCentury Extra, Feb. 7).
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