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Gene therapy company bluebird bio Inc. (NASDAQ:BLUE) raised $101 million in an IPO through the sale of a bumped-up number of shares above its proposed price range. The company sold 5.9 million shares at $17, which values the company at $387.8 million. Earlier this month, bluebird had amended its IPO and said it planned to sell 5 million shares at $14-$16. The shares are slated to start trading on Wednesday. JPMorgan; BofA Merrill Lynch; Cowen; Canaccord; and Wedbush are underwriters.
Late this year, bluebird plans to start Phase II/III testing of Lenti-D to treat childhood cerebral adrenoleukodystrophy (ALD). Lenti-D is a hematopoietic stem cell therapy with a lentiviral vector encoding the wild-type gene that encodes the ALD protein.
bluebird went public during the first real IPO window since 2000. Prior to the company's IPO, sixteen companies had gone out since the start of the year, raising almost $1 billion (see BioCentury, June 17).
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Exact Sciences Corp. (NASDAQ:EXAS) raised $67.9 million through the sale of 5.5 million shares at $12.35 in a follow-on underwritten by Jefferies; Baird; William Blair; Canaccord; and Mizuho. Exact Sciences proposed the follow-on late Monday, when its share price was $12.59. On Tuesday, the company was up $0.24 to $12.83.
Last week, Exact Sciences said it submitted the final module of a PMA to FDA for Cologuard as a colorectal cancer screening test. Cologuard is a non-invasive stool DNA test that uses a multiplexed quantitative Invader assay for the simultaneous detection of methylated and unmethylated sequences in the promoter region of the vimentin (VIM) gene (see BioCentury Extra, June 10).
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Prosensa B.V. (Leiden, the Netherlands) amended its IPO and now plans to sell 5 million shares at $11-$13. At the $12 midpoint, Prosensa would raise $60 million and be valued at $408 million. JPMorgan; Citigroup; Leerink; Wedbush; KBC Securities; and Trout Capital are underwriters. In May, Prosensa proposed to raise up to $60 million in the offering.
Prosensa is one of three companies vying to gain the first approved drug for Duchenne muscular dystrophy (DMD). The company's drisapersen (PRO051) is in Phase III testing, with data expected in 4Q13. GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) has exclusive, worldwide rights from Prosensa under a 2009 deal to develop and commercialize the antisense oligoribonucleotide that induces exon 51 skipping on the dystrophin gene (see BioCentury, June 3).
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FDA issued a drug safety communication warning that two patients died 3-4 days after receiving an appropriate dose of schizophrenia drug Zyprexa Relprevv olanzapine from Eli Lilly and Co. (NYSE:LLY). The long-acting antipsychotic is approved with a REMS that requires patients be continuously monitored at a REMS-certified health care facility for at least three hours following an injection and be accompanied home from the facility. The drug's label also includes warnings about the risk of post-injection delirium sedation syndrome, a condition in which the drug enters the blood stream too fast following an intramuscular injection and causes elevated blood levels. According to the agency, the two patients had "very high olanzapine blood levels." FDA said it is providing information to health care professionals while it continues to investigate the unexplained deaths.
Lilly said based on available information it is unable to conclude whether the two deaths were related to administration of Zyprexa Relprevv, but the pharma said it remains confident in the drug's benefit-risk profile. Lilly reported $1.7 billion in 2012 sales for its Zyprexa franchise; it does not break out sales from the franchise, which includes an oral formulation of the monoaminergic receptor antagonist marketed as Zyprexa. The oral formulation lost EU and U.S. patent protection in 2011.
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Pharming Group N.V. (Euronext:PHARM) and partner Santarus Inc. (NASDAQ:SNTS) said FDA accepted for review a resubmitted BLA for Ruconest conestat alfa to treat acute attacks of hereditary angioedema (HAE). The PDUFA date is April 16, 2014. The partners said FDA plans to hold a meeting of its Blood Products Advisory Committee to discuss Ruconest, but the meeting hasn't been scheduled. FDA refused to file a BLA for the product in 2011 (see BioCentury Extra, April 17).
The BLA acceptance triggered a $5 million milestone to Pharming from Santarus under a 2010 deal granting Santarus exclusive, North American commercialization rights to Ruconest. The recombinant human complement 1 (C1) esterase is already marketed in the EU for HAE. Santarus was up $0.76 to $22.28 on Tuesday. Pharming was off EUR 0.01 to EUR 0.14.
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Royalty Pharma (New York, N.Y.) withdrew a request for judicial review of a decision by the Irish Takeover Panel regarding its offer to acquire Elan Corp. plc (NYSE:ELN), ending a four-month public spat between the companies. Royalty was seeking to acquire Elan for $13 per share in cash plus a contingent value right (CVR) worth up to $2.50, which would have valued Elan at up to $7.9 billion. Royalty's decision officially ends its hostile bid to acquire Elan, though Elan on Friday announced a formal sales process and said its financial advisors contacted Royalty's financial advisors to invite Royalty to participate in the process.
On Monday, Royalty's bid for Elan lapsed after Elan shareholders approved the biotech's proposal to repurchase $200 million in shares; shareholders rejected three other proposals from Elan. Royalty was seeking to make its offer contingent on Elan shareholders rejecting only two proposals: the acquisition of rare disease company AOP Orphan Pharmaceuticals AG (Vienna, Austria); and Elan's proposal to pay Theravance Inc. (NASDAQ:THRX) $1 billion for a 21% interest in royalties Theravance is eligible to receive from partner GlaxoSmithKline plc (NSE:GSK; NYSE:GSK) for four respiratory programs. The Irish High Court was scheduled to start proceedings on Wednesday to review the decision by the Irish Takeover Panel that Royalty's offer remained contingent on Elan shareholders rejecting all four deals, including the share buyback (see BioCentury Extra, June 17).
Royalty, which declined to comment, is barred under Irish takeover rules from making another unsolicited bid for Elan for a year. Elan was up $0.29 to $13.78 on Tuesday.
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Daiichi Sankyo Co. Ltd. (Tokyo:4568; Osaka:4568) submitted an NDA to Japan's Ministry of Health, Labor and Welfare for prasugrel to treat patients with ischemic heart disease undergoing percutaneous coronary intervention (PCI). Daiichi Sankyo and Eli Lilly and Co. (NYSE:LLY) market the thienopyridine P2Y12 receptor antagonist as Effient to reduce the rate of thrombotic cardiovascular events in patients with acute coronary syndrome (ACS) who are to be managed with PCI. The product is approved in more than 70 countries, including the U.S. and EU.
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RedHill Biopharma Ltd. (Tel Aviv:RDHL; NASDAQ:RDHL) said FDA accepted for review an NDA for RHB-103 to treat acute migraine. The PDUFA date is Feb. 3, 2014. The NDA was submitted under section 505(b)(2) of the Food, Drug and Cosmetic Act, which allows sponsors to reference data on safety and efficacy from the scientific literature or from previously approved products. RedHill has exclusive, worldwide rights to co-develop and commercialize RHB-103 from IntelGenx Corp. (TSX-V:IGX; OTCBB:IGXT) under a 2010 deal. The product is a fast-dissolving thin-film formulation of rizatriptan delivered using IntelGenx's VersaFilm oral disintegrating film.
RedHill was down NIS0.14 to NIS3.70 in Tel Aviv on Tuesday. On NASDAQ, the stock was up $0.55 to $10.70. IntelGenx was up C$0.03 to C$0.61 on Tuesday. On the OTC Bulletin Board, the stock was up $0.04 to $0.61.
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FDA's Cardiovascular and Renal Drugs Advisory Committee will meet on Aug. 5 to discuss an NDA from Otsuka Pharmaceutical Co. Ltd. (Tokyo, Japan) for tolvaptan to slow kidney disease in adults with rapidly progressing autosomal dominant polycystic kidney disease. The product is under Priority Review, with a Sept. 1 PDUFA date. The vasopressin 2 (V2) receptor antagonist is approved as Samsca in the EU to treat hyponatremia secondary to syndrome of inappropriate antidiuretic hormone secretion (SIADH) in adults. The drug is approved in the U.S. to treat hypervolemic and euvolemic hyponatremia.
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NIH's National Center for Advancing Translational Science (NCATS) awarded $12.7 million to nine academic research groups to repurpose compounds from pharma partners under a pilot initiative. The groups will evaluate the selected compounds, all of which have cleared safety and toxicity hurdles through at least Phase I testing, for effectiveness against eight diseases or conditions for which the compounds have previously been unexplored. Last year, a group of pharmas that includes AstraZeneca plc (LSE:AZN; NYSE:AZN), Eli Lilly and Co. (NYSE:LLY), Johnson & Johnson (NYSE:JNJ), Pfizer Inc. (NYSE:PFE) and Sanofi (Euronext:SAN; NYSE:SNY) partnered with NCATS to provide academic researchers access to 58 compounds for repurposing (see BioCentury Extra, June 12, 2012).
The projects under the pilot initiative include Lilly's LY500307, an estrogen receptor-b agonist for schizophrenia; AstraZeneca's saracatinib (AZD0530), a dual inhibitor of c-src and BCR-ABL tyrosine kinase for Alzheimer's disease; ZD4054, an endothelin A receptor antagonist for peripheral artery disease (PAD); and Pfizer's PF-03463275, a glycine transporter type 1 (GlyT1; SLC6A9) inhibitor for schizophrenia.
Other projects will focus on evaluating a Pfizer compound for alcoholism; a Sanofi compound for calcific aortic valve stenosis; a J&J compound for smoking cessation; a Sanofi compound for Duchenne muscular dystrophy (DMD); and an AstraZeneca compound for lymphangioleiomyomatosis (LAM), a rare disease that causes the lungs of women in their thirties and forties to develop cysts that destroy lung function.
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FDA is holding a public workshop on July 23 to discuss preclinical methods to assess arrhythmia risk including the risk of torsades de pointes during the drug development process as an alternative to clinical thorough QT studies. The workshop will focus on assessing the use of ion channel currents as a screening method to evaluate the proarrhythmic potential of therapeutic candidates. The workshop is cosponsored by the International Life Sciences Institute's Health and Environmental Sciences Institute, as well as the Cardiac Safety Research Consortium. FDA formed the consortium -- which supports research evaluating cardiac safety or medical products -- with Duke University in 2006 under the agency's Critical Path initiative to promote public-private partnerships.
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The American Society of Clinical Oncology and more than 50 other advocacy organizations sent a letter to HHS and the U.S. Department of Labor requesting regulations or guidance by January on a provision of the Affordable Care Act that requires insurers to cover routine medical costs for patients participating in clinical trials. In the letter, the groups said a list of frequently asked questions on the implementation of ACA that was released by the departments in April suggests the departments "do not expect to issue regulations in the near future" on the implementation of the provision requiring coverage of trial expenses. The provision is slated to take effect in January.
The groups also outlined a number of issues with the implementation of the provision that they believe should be addressed by rulemaking or guidance. The groups want HHS and the Department of Labor to include explicit safeguards to ensure that "routine costs" includes the prevention, detection, and treatment of complications arising from clinical trials, and also to include regulations that prevent insurance companies from requiring patients to travel "unreasonable" distances to enroll in a trial with an in-network provider.
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Japan's Ministry of Health, Labor and Welfare (MHLW) last week withdrew its recommendation of HPV vaccination pending further review of safety data related to systemic pain, according to GlaxoSmithKline plc (LSE:GSK; NYSE:GSK). MHLW-approved HPV vaccines include Cervarix from GSK and Gardasil from Merck & Co. Inc. (NYSE:MRK).
GSK told BioCentury that there is "no evidence" that systemic pain is related to Cervarix administration, but that the company is providing information to MHLW. The pharma added that despite the recommendation, the Japanese government is continuing to pay for HPV vaccinations. Merck did not provide comment in time for publication, while MHLW could not be reached for details.
Cervarix had 2012 worldwide sales of L270 million ($436.5 million), while Gardasil had sales of $1.6 billion. The vaccines are approved to prevent cervical cancer and precancerous lesions caused by HPV infection.
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EMA's Pharmacovigilance Risk Assessment Committee (PRAC) is in the process of drafting a consultation that will lay out the rules of procedure for public hearings on the safety of marketed drugs or classes of medicines. The hearings, which will allow the public to engage with the agency on issues related to drug safety, are part of pharmacovigilance legislation that came into effect last July. EMA expects the consultation to be published before year end.
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Adamas Pharmaceuticals Inc. (Emeryville, Calif.) said once-daily doses of oral ADS-5102 improved levodopa-induced dyskinesia in the Phase II/III EASED trial in patients with Parkinson's disease (PD). Specifically, once-daily 340 and 420 mg doses of oral ADS-5102 met the primary endpoint of improving levodopa-induced dyskinesia as measured by the mean change in Unified Dyskinesia Rating Scale (UDysRS) total score from baseline to week eight vs. placebo (p=0.005 and p=0.013, respectively). The once-daily 260 mg dose of ADS-5102 led to a non-significant improvement on the endpoint (p=0.159). The double-blind, U.S. trial enrolled 83 patients with PD. Data were presented at the Parkinson's Disease and Movement Disorders meeting in Sydney.
Adamas said it plans to review the data and discuss next steps with FDA "very soon," but declined to disclose a specific time frame. ADS-5102 is an oral extended-release formulation of amantadine.
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Two groups of academic researchers published data on Tuesday in the Annals of Internal Medicine from a pair of independent reviews analyzing the safety and efficacy of InFuse Bone Graft from Medtronic Inc. (NYSE:MDT), which is used in spinal fusion surgery. Based on an analysis of individual-patient data from 13 randomized controlled trials sponsored by Medtronic and 31 cohort studies, researchers at Oregon Health & Sciences University concluded that InFuse "has no proven clinical advantage over [iliac crest] bone graft and may be associated with important harms." The Oregon researchers also found that InFuse was associated with an increased cancer risk at 24 months, but event rates were low and cancer was heterogeneous. The researchers concluded that early journal publications misrepresented the effectiveness and harms through selective reporting, duplicate publication and underreporting.
In a separate analysis, researchers at the University of York reported data from an analysis of individual-patient data from 11 randomized controlled trials sponsored by Medtronic and one other eligible trial and concluded that InFuse "increases fusion rates, reduces pain by a clinically insignificant amount, and increases early postsurgical pain" vs. iliac crest bone graft at 24 months. Furthermore, cancer was almost twice as common with InFuse compared to iliac crest bone graft, but the small number of events precluded a definite conclusion. Medtronic said it has funded a retrospective analysis of a national payer database to investigate the incidence of cancer in real-world use of InFuse; the analysis is slated to be presented next half.
In 2011, Medtronic commissioned Yale University to review the safety and efficacy of InFuse following reports published in The Spine Journal in June 2011 alleging that the company failed to disclose financial ties to medical researchers involved in InFuse trials. The reports also claimed those same researchers failed to report the risk of complications associated with InFuse. Yale subsequently commissioned the Oregon and York researchers to conduct separate analyses (see BioCentury Extra, Aug. 4, 2011).
Medtronic has said sales of InFuse, an implantable material containing bone morphogenic protein 2 (BMP2), have declined due to the Spine article, but the company has not broken out sales of the product since then. Medtronic was off $0.59 to $52.92 on Tuesday.