U.S. Senate Majority Leader Mitch McConnell (R-Ky.) has set in motion a procedure that could lead to a vote to confirm Robert Califf as FDA commissioner. On Thursday, McConnell scheduled a vote for Feb. 22 on a cloture motion that would cut off debate on the confirmation. If at least 60 Senators vote for the motion, Califf could be confirmed despite holds placed on the confirmation by Sens. Edward Markey (D-Mass.) and Bernie Sanders (I-Vt.), as well as a threat by Sen. Joe Manchin (D-W.Va.) to filibuster the vote.
Markey said he was disappointed by McConnell's decision and said he will continue to use the confirmation process as a tool to express opposition to FDA's opioid drug approval process.
Prior to McConnell scheduling the cloture vote, Sen. Lisa Murkowski (R-Alaska) lifted a hold she placed on Robert Califf's nomination as FDA commissioner. In a statement, Murkowski said FDA has addressed the concerns that led her to block the confirmation vote. The agency provided her with "technical drafting assistance on legislative language that would effectively mandate" labeling of genetically modified salmon, Murkowski said.
FDA approved genetically engineered AquAdvantage Salmon from AquaBounty Technologies Inc. (LSE:ABTU) in November 2015, but is barred from allowing the fish's sale until the agency publishes guidance on labeling to inform consumers of the product's content (see BioCentury Extra, Nov. 20, 2015).
Also Thursday, Manchin introduced a bill designed to spur FDA's involvement in limiting opioid addiction. The bill would add language to the agency's mission statement, reading: "The FDA is also responsible for protecting the public health by strongly considering the danger of addiction and overdose death associated with prescription opioid medications when approving these medications and when regulating the manufacturing, marketing, and distribution of opioid medications."
Shire plc (LSE:SHP; NASDAQ:SHPG) CEO Flemming Ornskov said the company is on track to complete its acquisition of Baxalta Inc. (NYSE:BXLT) mid-year, and plans to focus in the near term on integration of Baxalta and hereditary angioedema (HAE) play Dyax Corp. The comments came on a conference call Thursday to discuss Shire's 4Q15 and 2015 financial results.
"We are a company that constantly looks both at tuck-ins on the product side and will continue to do that, and we also look at M&A," noted Ornskov. "But I think the focus right now is on the integration."
Shire is acquiring Baxalta in a cash and stock deal that values Baxalta at about $32 billion. In January, Shire completed its acquisition of Dyax for $5.9 billion up front and $646 million in a contingent value right (CVR) tied to approval of Dyax's SHP643 (DX-2930). Ornskov said the integrations would be "the two largest we've ever done" (see BioCentury, Jan. 18).
CFO Jeffrey Poulton said Shire also intends to use cash flow to pay down debt. By YE17, Poulton said Shire expects to be in a position to "have multiple options to consider in terms of how to deploy cash," which he said could include M&A or share buybacks.
Ornskov also hinted that Shire's integration process may include hiving off products from the combined company. "We'll take a look at all the franchises and just make sure we think that they're all franchises that should stay with us going forward," he said on the call.
In 2015, Shire reported $6.4 billion in revenues, up from $6 billion in 2014, and non-GAAP diluted earnings per ADS of $11.68, up from $10.60 in 2014. The Street was expecting full-year revenues of $6.4 billion and earnings per ADS of $11.57.
Shire also introduced 2016 guidance. Excluding the Baxalta acquisition, the company said it expects to generate double digit top-line growth in 2016, including 13-17% growth from 2015 in product sales on a constant currency basis. Poulton said the company expects to update its guidance after the Baxalta deal is completed.
On Thursday, Shire lost 61p to 3,507p in London and shed $2.93 to $151 on NASDAQ.
Aegerion Pharmaceuticals Inc. (NASDAQ:AEGR) said it will cut costs and reduce global staff by 25% as its hypercholesterolemia treatment Juxtapid lomitapide faces increased competition.
The company said in January it expected Juxtapid sales to be $37.5-$39.5 million in 4Q15 and $211-$213 million for the year. In November, Aegerion said switches to PCSK9 inhibitors were higher than expected (see BioCentury Extra, Nov. 10, 2015).
Aegerion said it would have about 230 employees after the cuts, which it expects to complete next quarter.
Both Juxtapid and PCSK9 inhibitor Repatha evolocumab from Amgen Inc. (NASDAQ:AMGN) are approved to treat homozygous familial hypercholesterolemia (HoFH). Amgen launched Repatha in September. It has yet to break out sales of the drug.
PCSK9 inhibitor Praluent alirocumab from Sanofi (Euronext:SAN; NYSE:SNY) and Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) is approved to treat heterozygous familial hypercholesterolemia (HeFH) and atherosclerotic cardiovascular disease; its label does not include HoFH. On Tuesday, Regeneron said Praluent sales were $7 million in 4Q15 and $11 million for the year. The companies launched Praluent in July.
Juxtapid is a small molecule microsomal triglyceride transfer protein (MTTP; MTP) inhibitor.
Aegerion shed $0.22 to $6.18 on Thursday, touching a 52-week low of $6.09. It traded above $100 in October 2013.
FDA's Gastrointestinal Drugs Advisory Committee will meet on April 7 to discuss an NDA from Intercept Pharmaceuticals Inc. (NASDAQ:ICPT) for obeticholic acid (DSP-1747) to treat primary biliary cholangitis (PBC).
The company is seeking approval of the oral farnesoid X receptor (FXR; NR1H4) to treat PBC in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA. Its PDUFA date is May 29.
In September 2015, Intercept began the Phase III REGENERATE trial of OCA to treat non-alcoholic steatohepatitis (NASH).
Intercept slipped $1.45 to $94.29 on Thursday.
The Sandoz unit of Novartis AG (NYSE:NVS; SIX:NOVN) said EMA accepted an MAA for LA-EP2006, its proposed biosimilar of Neulasta pegfilgrastim from Amgen Inc. (NASDAQ:AMGN), to treat chemotherapy-induced neutropenia. In November, FDA accepted for review a BLA for LA-EP2006, a pegylated granulocyte colony-stimulating factor (G-CSF) (see BioCentury Extra, Nov. 18, 2015).
Worldwide sales of Neulasta were $4.7 billion in 2015, including $824 million outside the U.S.
Sandoz markets a biosimilar of Amgen's Neupogen filgrastim as Zarzio in the EU and Zarxio in the U.S. It is the only FDA-approved biosimilar.
Incyte Corp. (NASDAQ:INCY) reported 4Q15 and 2015 revenues that beat expectations but fell $6.80 to $65.51 on Thursday after it said it would discontinue all ongoing trials of myelofibrosis drug Jakafi ruxolitinib in solid tumors, including the Phase III JANUS 1 and JANUS 2 trials of Jakafi plus Xeloda capecitabine as a second-line therapy for advanced or metastatic pancreatic cancer.
The biotech said a planned interim analysis of JANUS 1 "did not show a sufficient level of efficacy to warrant continuation," following a similar outcome in metastatic colorectal cancer (mCRC). Last month, Incyte said it would stop early a substudy of a Phase II trial of Jakafi plus Stivarga regorafenib for mCRC after a planned interim analysis. Incyte will now discontinue that entire Phase II study (see BioCentury Extra, Jan. 27).
Incyte also will discontinue Phase II trials of the oral Janus kinase-1 (JAK-1) and JAK-2 inhibitor in breast and lung cancer, as well as a dose-finding trial of selective JAK-1 inhibitor INCB39110 as first-line therapy for metastatic pancreatic cancer.
The company said it conducted the trials to see whether JAK inhibition could be used to treat solid tumors with high levels of systemic inflammation. Incyte plans to continue ongoing trials of Jakafi and its selective JAK-1 inhibitors, including INCB39110, in hematology indications. The biotech also will continue ongoing trials of selective JAK-1 inhibitors in solid tumors that are "based on different hypotheses," including ongoing studies evaluating the "therapeutic utility of JAK-1 inhibition based on its effects on the tumor microenvironment."
At least one other JAK-1 and JAK-2 inhibitor is in the clinic to treat solid tumors. According to ClinicalTrials.gov, momelotinib (formerly CYT387) from Gilead Sciences Inc. (NASDAQ:GILD) is in a Phase III trial in combination with Abraxane nab-paclitaxel and gemcitabine for previously untreated metastatic pancreatic cancer. Gilead acquired the compound when it bought YM BioSciences in 2012 (see BioCentury, Dec. 17, 2012).
In 4Q15, Incyte's total revenues were $243.9 million, up from $124 million in 4Q14 and beating the $225.9 million consensus estimate. Its diluted EPS was $0.29, up from a loss of $0.22 in 4Q14 and beating the $0.09 consensus.
For the year, revenues were $753.8 million, up from $511.5 million in 2014 and topping the $735.5 million consensus. Its 2015 diluted EPS was $0.03, up from a loss of $0.29 in 2014 and beating the consensus estimate of a $0.19 loss.
Incyte also introduced 2016 guidance of $800-$815 million in Jakafi net product revenues. Novartis AG (NYSE:NVS; SIX:NOVN) holds exclusive, ex-U.S. rights to ruxolitinib, which it markets as Jakavi.
Gene therapy company AveXis Inc. (NASDAQ:AVXS) and respiratory company Proteostasis Therapeutics Inc. (NASDAQ:PTI) both posted losses in their first day of trading Thursday after pricing IPOs.
AveXis fell $1.95 (10%) to $18.05 after it raised $95 million through the sale of 4.8 million shares at $20, the midpoint of its proposed range. Underwriters were Goldman Sachs, Jefferies, BMO Capital Markets and Chardan. The IPO price valued AveXis at $448 million. Earlier this month, AveXis said it hoped to sell 4.3 million shares. At $20, it would have raised $85 million and been valued at $438 million. At Thursday's close, its market cap was $404.3 million. In 1H17, AveXis hopes to begin pivotal studies of AVXS-101 to treat spinal muscular atrophy (SMA) type 1. The therapy, which uses NAV rAAV9 vector technology from RegenxBio Inc. (NASDAQ:RGNX) to deliver the survival motor neuron (SMN) gene, has Orphan Drug designation from FDA to treat all types of SMA, and Fast Track designation to treat SMA type 1.
Proteostasis sank $1.36 (17%) to $6.64 after raising $50 million through the sale of 6.3 million shares at $8, giving it a valuation of $152.9 million. Underwriters were Leerink, RBC Capital Markets, Baird and H.C. Wainwright. Proteostasis said earlier this month it hoped to sell 3.9 million shares at $12-14. At $13, it would have raised $50.1 million and been valued at $217.2 million. At Thursday's close, its market cap was $126.9 million. The company plans to begin a Phase I trial this quarter of lead candidate PTI-428, a cystic fibrosis transmembrane conductance regulator (CTFR) amplifier to treat cystic fibrosis.
M3 Biotechnology Inc. (Seattle, Wash.) raised $10 million in an oversubscribed series A round. Brothers Bruce and Michael Montgomery invested alongside W-Fund, WRF Capital, John Fluke and Dolby Family Ventures.
CEO Leen Kawas told BioCentury the company plans to begin clinical studies by YE16 of lead candidate MM-201, an oral small molecule mimetic of a neurotrophic factor to treat neurodegenerative diseases.
The Montgomery brothers will join M3's board.
HHS Secretary Sylvia Burwell told members of the U.S. House Ways and Means Committee that HHS is "continuing to try and pursue every administrative option" to combat high drug prices, and is considering issuing guidelines on when NIH may exercise its march-in authority. The comment came in response to a question from Rep. Lloyd Doggett (D-Texas) during a committee hearing on Wednesday to discuss President Obama's FY17 budget request for HHS.
Last month, 50 House Democrats including Doggett sent a letter to Burwell and NIH Director Francis Collins urging NIH to issue guidelines on when it would exercise its march-in rights under the Bayh-Dole Act. Under the act, NIH may require the holder of a patent developed using federally funded research to license the patent to other parties to "promote the utilization of inventions arising from federally supported research or development."
"We want pharmaceutical manufacturers to have the certainty of clear guidelines that indicate when march-in rights apply," the Representatives wrote. "With adequate guidance, pharmaceutical companies should be able to make better-informed pricing decisions."
NIH has never exercised its march-in rights.
Doggett asked Burwell for assurance that HHS is giving thorough consideration to the letter. "It is essential that the administration use every tool at its disposal to prevent price gouging," he said. Burwell told Doggett HHS is considering the letter's request while "looking at a wide array" of measures.
Obama's FY17 budget request contains drug pricing measures that include granting the HHS Secretary negotiating power for high-cost drugs and biologics covered under Medicare Part D. There is little chance the full budget will be enacted, but the proposals add to the list of legislative options the next administration and Congress may consider (see BioCentury Extra, Feb. 9).
Cancer Research UK (London, U.K.) released its response to a proposal to incorporate the Cancer Drugs Funds into the drug appraisal process of the U.K.'s NICE. The organization supported the proposal, but cited additional considerations for NICE and NHS.
CRUK said NICE decisions should be transparent and that NICE should be flexible in its evidence requirements and timelines for cancer drug approvals, particularly for rare cancers. Under the new program, drugs that require additional evidence before commissioning would receive CDF funding for up to two years, which CRUK said may be insufficient for some diseases.
The organization also noted the potential effect of the proposal on patients in Wales and Northern Ireland, which do not have dedicated CDFs, and the need for sufficient resources at NICE to meet the increased demand.
NHS and NICE released their proposed modifications to the fund in November 2015. NICE would conduct reviews of all new cancer drugs under the new system, issuing a recommendation for or against routine commissioning of the drug. It could also recommend funding a drug out of the CDF budget while additional real-world data are collected (see BioCentury Extra, Nov. 19, 2015).
The CDF was launched in 2011 to cover cancer drugs not recommended by NICE. The fund is set to run through March 2016.
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