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Gene therapy company bluebird bio (NASDAQ:BLUE) climbed $9.91 (58%) to $26.91 in its trading debut Wednesday. On Tuesday, the company raised $101 million through the sale of 5.9 million shares at $17 in an IPO, valuing bluebird at $387.8 million. With Wednesday's gains, the company is now valued at $613.8 million (see BioCentury Extra, June 18).
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 for the ALD protein.
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Intercept Pharmaceuticals Inc. (NASDAQ:ICPT) raised $57.1 million through the sale of 1.7 million shares at $33.01 in a follow-on underwritten by BofA Merrill Lynch; Citigroup; BMO Capital Markets; Needham; Wedbush; and Janney Montgomery Scott. Intercept proposed to raise up to $63.3 million through the sale of up to 2 million shares in the offering on June 10, when its share price was $33.65. On Wednesday, Intercept was up $5.40 (16%) to $38.41.
Intercept's obeticholic acid (OCA) is in the international Phase III POISE trial to treat primary biliary cirrhosis, with data expected in 2Q14. The company raised $86.3 million in an IPO last October.
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Stem cell company ReproCell Inc. (JASDAQ:4978) raised Y2.1 billion ($21.7 million) through the sale of 642,000 shares at Y3,200 in an IPO on the JASDAQ Growth Market of the Osaka Securities Exchange. The price values ReproCell at Y26.6 billion ($280.5 million). SMBC Nikko Securities and seven other firms are underwriters. The shares are slated to start trading on June 26.
ReproCell markets induced pluripotent stem cell (iPSC)-derived cardiomyocytes and neurons for drug discovery. The company also markets media and reagents for embryonic stem cells and iPSCs.
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Biodel Inc. (NASDAQ:BIOD) raised $19.5 million through the sale of 4.5 million shares at $4.35 in a follow-on underwritten by William Blair and Ladenburg Thalmann. Biodel proposed the offering late Tuesday, when its share price was $4.76. On Wednesday, Biodel was off $0.41 to $4.35.
The company's BIOD-123, an ultra-rapid-acting formulation of recombinant human insulin, is in Phase II testing for Type I diabetes, with data expected next quarter.
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Progenics Pharmaceuticals Inc. (NASDAQ:PGNX) proposed late Wednesday a follow-on underwritten by Jefferies. Earlier this month, partner Salix Pharmaceuticals Inc. (NASDAQ:SLXP) said FDA will convene an advisory committee meeting to discuss Salix's appeal of a complete response letter for subcutaneous Relistor methylnaltrexone to treat opioid-induced constipation (OIC) in patients with chronic pain. Relistor is already approved in more than 55 countries, including the U.S., to treat OIC in patients receiving palliative care when response to laxative therapy has not been sufficient. Salix has worldwide rights to the peripheral mu opioid receptor (OPRM1; MOR) antagonist from Progenics (see BioCentury Extra, June 11).
Progenics was off a penny to $4.80 on Wednesday.
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Iroko Pharmaceuticals LLC (Philadelphia, Pa.) filed to raise up to $145 million in an IPO underwritten by Jefferies; William Blair; and Canaccord. In March, Iroko said FDA accepted for review an NDA for Zorvolex submicron diclofenac to treat mild to moderate acute pain in adults. Iroko submitted the NDA in February; a standard 10-month review would place the PDUFA date in December, though the specific PDUFA date is not disclosed. In April, Iroko submitted an NDA for Tiforbex submicron indomethacin to treat acute pain in adults.
Zorvolex is a nanoformulation of the NSAID diclofenac, and Tiforbex is a nanoformulation of the NSAID indomethacin. Both use Iroko's SoluMatrix nanoformulation technology. Iroko is seeking approval of both products 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.
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Bristol-Myers Squibb Co. (NYSE:BMY) and partner AstraZeneca plc (LSE:AZN; NYSE:AZN) said once-daily Onglyza saxagliptin missed the primary efficacy endpoint in the Phase IV SAVOR-TIMI-53 cardiovascular outcomes trial in 16,500 Type II diabetics who had either a history of established cardiovascular disease or multiple risk factors. Specifically, Onglyza was not superior to placebo in reducing a composite of cardiovascular death, non-fatal myocardial infarction (MI) or non-fatal ischemic stroke. Onglyza did meet the primary safety endpoint of non-inferiority to placebo.
Bristol-Myers said the trial was designed to fulfill a postmarketing requirement requested by FDA for the oral dipeptidyl peptidase-4 (DPP-4) inhibitor. The pharma said the partners plan to share the full data with regulatory authorities, but declined to disclose details. AstraZeneca could not be reached for comment.
Onglyza is approved to treat Type II diabetes in 86 countries, including the U.S. and countries in the EU. Bristol-Myers and AstraZeneca partnered in 2007 to develop and commercialize Onglyza. Otsuka Pharmaceutical Co. Ltd. (Tokyo, Japan) has rights in Japan.
Data from the TECOS CV outcomes trial of Januvia sitagliptin, a competing DPP-4 inhibitor from Merck & Co. Inc. (NYSE:MRK), are expected next year.
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ALK-Abello A/S (CSE:ALK-B) said once-daily Mitizax for one year met the primary endpoint in the Phase III MERIT (MT-06) trial to treat house dust mite-induced allergic rhinitis. Mitizax -- a House Dust Mite Allergy Immunotherapy Tablet (HDM AIT) -- reduced average total combined rhinitis symptom and medication use score during the last eight weeks of treatment vs. placebo (p<0.01). The double-blind, European trial enrolled 992 patients.
MERIT is the first of two Phase III trials of HDM AIT conducted by ALK-Abello to treat house dust mite-induced respiratory diseases. Data from the Phase III MITRA trial to treat house dust mite-induced allergic asthma are expected by the end of 3Q13. ALK-Abello plans to submit a European regulatory application for the tablet-based vaccine against biological house dust mite allergen in 2014. ALK-Abello gained DKK22 to DKK437 on Wednesday.
Torii Pharmaceutical Co. Ltd. (Tokyo:4551) has exclusive Japanese rights to HDM AIT from ALK-Abello, while Merck & Co. Inc. (NYSE:MRK) has exclusive North American rights. Torii is conducting a pair of Japanese Phase II/III trials, while Merck is conducting a Phase IIb trial and planning a Phase III trial.
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Paladin Labs Inc. (TSX:PLB) said FDA accepted and granted Priority Review to an NDA for Impavido miltefosine to treat leishmaniasis. The PDUFA date is Dec. 19. Paladin said it is eligible for a Priority Review voucher if FDA approves the application. The transferrable voucher entitles the bearer to priority FDA review for another drug. The voucher program was started in 2007 to create incentives to develop drugs for neglected tropical diseases (see BioCentury, Aug. 8, 2011).
Impavido is already marketed for leishmaniasis in Europe, India and Central and South America. Paladin acquired the alkylphosphocholine analog that inhibits CTP phosphocholine cytidylyl transferase from Aeterna Zentaris Inc. (TSX:AEZ; NASDAQ:AEZS) in 2008.
Paladin, which announced the news late Wednesday, was up C$0.60 to C$52.50. Aeterna was unchanged at C$1.92 on the day. On NASDAQ, the company was down $0.03 to $1.87.
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Amarin Corp. plc (NASDAQ:AMRN) said FDA notified the company that an advisory committee will meet on Oct. 16 to discuss an sNDA for hypertriglyceridemia drug Vascepa icosapent ethyl. The company is seeking to expand the drug's label to include treatment of adult patients with high triglycerides -- defined as triglyceride levels greater than or equal to 200 mg/dL and less than 500 mg/dL -- with mixed dyslipidemia. The PDUFA date for the sNDA is Dec. 20.
Amarin markets the more than 96% pure ethyl ester of eicosapentaenoic acid (ethyl-EPA) in the U.S. as an adjunct to diet to reduce triglyceride levels in adults with severe hypertriglyceridemia, defined as triglyceride levels of greater than or equal to 500 mg/dL. On Wednesday, Amarin was off $0.13 to $6.47.
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Life Technologies Corp. (NASDAQ:LIFE) granted Suzhou Ribo Life Sciences Co. Ltd. (Kunshan, China) exclusive rights to use Invivofectamine Rx delivery technology to develop small interfering RNA (siRNA) therapeutics in China. The first program will focus on HBV infection. Life Tech is eligible to receive undisclosed milestones plus royalties for each product developed under the deal. Invivofectamine Rx uses an undisclosed chemical structure to increase potency and decrease toxicity of RNA interference (RNAi) compounds, which are delivered in vivo into liver cells. Suzhou Ribo develops nucleic acid drugs and products based on RNAi technology for the Chinese market.
Life Tech, which is being acquired by Thermo Fisher Scientific Inc. (NYSE:TMO), was off $0.06 to $74.21 on Wednesday.
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Daiichi Sankyo Co. Ltd. (Tokyo:4568; Osaka:4568) said its Kitasato Daiichi Sankyo Vaccine Co. Ltd. subsidiary submitted an NDA to Japan's Ministry of Health, Labor and Welfare (MHLW) for a cell culture-based pandemic influenza vaccine. Kitasato Daiichi received a grant from the ministry in August 2011 to develop the vaccine, as well as a cell culture vaccine production facility. Daiichi could not provide details in time for publication.
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Galecto Biotech AB (Copenhagen, Denmark) appointed Magnus Persson as chairman. He succeeds Kjell Stenberg, who remains as a director. Persson, a co-founder of Aerocrine AB (SSE:AERO), was a partner at HealthCap Partners and managing partner at The Column Group.
Galecto is developing galectin-3 (LGALS3) inhibitors that target both macrophages and fibroblasts. The company's TD139 is in preclinical testing to treat idiopathic pulmonary fibrosis (IPF) (see BioCentury, May 14, 2012).
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Republicans on the U.S. Senate Committee on Health, Education, Labor and Pensions sent a letter to FDA Commissioner Margaret Hamburg asking "why and under what authority" the agency is publicizing new health insurance exchanges under the Affordable Care Act. According to the letter, FDA sent an email earlier this month to "families, individuals, small businesses and clinicians" promoting open enrollment in the new exchanges. The senators expressed concern that the email was an attempt by HHS to "avoid congressional limitations on implementation expenditures" for ACA, noting that the exchanges are outside of FDA's mission.
The senators are seeking information on FDA's legal authority to promote the implementation of ACA and the new health insurance exchanges and how the activities further the agency's mission, as well as details on the total costs FDA has spent during FY13 on promoting ACA implementation. The senators are seeking a response by June 26. FDA could not be reached for comment.
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The American Medical Association voted at its annual meeting to adopt policy that recognizes obesity as a disease requiring medical intervention to treat and prevent. The policy is in contrast to advice from an AMA committee that recommended against considering obesity a disease because the method of diagnosing obesity -- body mass index (BMI) -- has "existing limitations," and because it is "unclear" that recognizing obesity as a disease instead of a condition or disorder will improve health outcomes.
AMA also voted to "revisit the topic of biosimilars" and to study "emerging issues that are relevant for such products under the current abbreviated pathway for approval," though the association did not provide a timeline or details. Additionally, the association adopted a policy encouraging companies, laboratories, researchers and providers to publicly share data and the clinical significance of genetic variants, as well as a policy supporting FDA oversight and regulation of facilities that compound sterile drug products without receiving a prescription prior to compounding.
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Legislators introduced a bicameral bill that would require Medicare Part D to cover prescription drugs to treat obesity. The Treat and Reduce Obesity Act also includes measures to encourage and expand access to behavioral counseling for obesity. Currently, Medicare covers obesity surgery and counseling, but not drug treatments. Sens. Lisa Murkowski (R-Alaska) and Tom Carper (D-Del.) introduced the bill into the Senate, and Reps. Bill Cassidy (R-La.) and Rob Kind (D-Wis.) introduced the bill into the House of Representatives.