The Daily Extra

Arena raises $60.5M in follow-on

Wed, 16 May 2012 17:45:40 -0700

Arena Pharmaceuticals Inc. (NASDAQ:ARNA) raised $60.5 million through the sale of 11 million shares at $5.50 in a follow-on underwritten by Jefferies; Piper Jaffray; and BMO Capital Markets. The company proposed the offering late Tuesday, when its share price was $6.06. Arena was off $0.39 to $5.67 on Wednesday.

On Friday, an FDA advisory committee voted 18-4, with one abstention, that the benefit-risk profile for Arena's lorcaserin supports approval to treat obesity. The PDUFA date for the serotonin (5-HT2C) receptor agonist is June 27 (see BioCentury, May 14).

Amgen's anti-HGF/SF mAb improves OS in high c-MET patients

Wed, 16 May 2012 17:45:40 -0700

Amgen Inc. (NASDAQ:AMGN) said rilotumumab plus chemotherapy significantly improved median overall survival vs. chemotherapy alone in patients with gastric tumors exhibiting high c-MET expression (11.1 vs. 5.7 months, p=0.012). The results came from an exploratory analysis of a subset of 90 patients in a completed Phase II trial of the mAb against human hepatocyte growth factor/scatter factor. In patients with low c-MET expression, rilotumumab plus chemotherapy was associated with a trend towards unfavorable OS compared to chemotherapy alone. Based on the results, Amgen plans to move rilotumumab into Phase III testing this year to treat advanced gastric and gastroesophageal junction cancer in patients whose tumors have high c-MET expression. Data will be presented at the American Society of Clinical Oncology meeting in Chicago in June.

Amgen defined high c-MET expression as more than 50% of tumor cells testing positive for the c-MET protein. Last year, Amgen reported data from all 121 patients in the Phase II trial showing that rilotumumab plus chemotherapy led to median progression-free survival of 5.6 months and OS of 11.1 months, compared to PFS of 4.2 months and OS of 8.9 months for chemotherapy alone.

Earlier this year, Amgen and Dako A/S (Glostrup, Denmark) partnered to develop and evaluate the use of a companion diagnostic in the development of rilotumumab. Amgen was up $0.30 to $70.98 on Wednesday.

Researchers question concept of raising HDL to reduce MI risk

Wed, 16 May 2012 17:45:40 -0700

Some genetic variants that increase plasma HDL-C do not also reduce the risk of myocardial infarction, according to a study published in The Lancet. In the paper, researchers from the Massachusetts General Hospital and colleagues said the data challenge the concept that raising HDL-C will translate into reductions in MI risk.

In an analysis of 20,913 MI cases and 95,407 controls, carriers of the LIPG 396Ser allele had significantly higher HDL-C but similar levels of other lipid and non-lipid risk factors for MI compared with non-carriers. The researchers said the difference in HDL-C was expected to decrease MI risk by 13%, but they instead found no association between the allele and MI risk. Additionally, the researchers said a genetic score combining 14 genetic variants related to HDL-C failed to show an association with MI risk.

The study was funded by NIH, the Wellcome Trust, the EU, the British Heart Foundation and the German Federal Ministry of Education and Research.

Piramal to acquire healthcare analytics firm

Wed, 16 May 2012 17:45:40 -0700

Piramal Healthcare Ltd. (BSE:500302; NSE:PIRHEALTH) will acquire healthcare information and analytics company Decision Resources Group (Burlington, Mass.) for about $635 million. Decision Resources, which offers services to forecast trends in drug and medical device markets, expects $160 million in 2012 revenue. The deal is expected to close by June 30.

Piramal said the acquisition is part of the Indian company's strategy to buy global growth businesses with sustainable returns following the 2010 sale of its domestic formulation business to Abbott Laboratories (NYSE:ABT) for $3.8 billion. Last month, Piramal's Piramal Imaging S.A. subsidiary said it will acquire worldwide rights to the PET imaging tracer assets of Bayer AG (Xetra:BAYN). Piramal had revenues of $450 million in FY12 (see BioCentury Extra, April 16).

Piramal was off Rs1.35 to Rs428.40 on Wednesday.

OraSure gains after FDA panel backs OTC HIV test

Wed, 16 May 2012 17:45:40 -0700

OraSure Technologies Inc. (NASDAQ:OSUR) gained $1.85 (20%) to $10.95 on Wednesday after FDA's Blood Products Advisory Committee voted 17-0 late Tuesday that data show the OraQuick In-Home HIV test is safe and effective. The panel also unanimously voted that the test's projected benefits outweigh the potential risks of false positive and false negative results. The test is an OTC version of OraSure's OraQuick Advance Rapid HIV-1/2 point-of-care test. OraQuick Advance is an oral fluid, finger-stick or venipuncture whole blood or plasma specimen HIV-1 and HIV-2 antibody diagnostic. FDA said if the OTC test is approved, it would be the first OTC home-use test kit for any infectious agent.

SEC investigating Dendreon

Wed, 16 May 2012 17:45:40 -0700

Dendreon Corp. (NASDAQ:DNDN) disclosed in a 10Q filing that the SEC is investigating the company. Dendreon said it does not know the specific focus of the investigation, but believes it may be related to statements and predictions the company made about the launch of prostate cancer therapy Provenge sipuleucel-T. Dendreon said it is cooperating with the SEC. Last August, Dendreon pulled its 2011 revenue guidance of $350-$400 million as a result of lower-than-expected uptake of Provenge, which is the company's only drug on the market (see BioCentury, Aug. 8, 2011).

Dendreon is also a defendant in a securities class action suit in the U.S. District Court for the Western District of Washington related to the Provenge launch. Dendreon was down $0.22 to $8.62 on Wednesday.

Ra raises $8.6M in second tranche of series A

Wed, 16 May 2012 17:45:40 -0700

Ra Pharmaceuticals Inc. (Cambridge, Mass.) raised $8.6 million in the second tranche of a planned $27 million series A round. Ra raised $10.3 million in the first tranche in February 2010. New Enterprise Associates led the tranche, with participation from Morgenthaler Ventures; Novartis Venture Funds; and Amgen Ventures.

Ra is developing peptide-like molecules called cyclomimetics that the company said could have clinical utility as oral alternatives for marketed injectable biologics and could address previously undruggable targets, such as intracellular protein-protein interactions. The company is initially developing cyclomimetics against plasma kallikrein to treat hereditary angioedema (HAE).

MEDCAC provides input on CED process

Wed, 16 May 2012 17:45:40 -0700

CMS's Medicare Evidence Development & Coverage Advisory Committee voted 14-0 that an evidentiary threshold can be defined to trigger a review of coverage with evidence development for a product or service to determine if the coverage should be stopped, continued or modified. CMS uses CED to provide coverage that is contingent on some form of new clinical data collection in the Medicare population. CMS convened the panel to provide feedback on the characteristics of evidence that are appropriate for the CED process.

The panel felt a CED should be reviewed when sufficient data has been gathered to address the question of whether or not the product or service is "reasonable and necessary," and that the specific threshold should be determined when the CED is first issued.

The panel voted 13-1 that an evidentiary threshold can be defined to invoke CED but felt the level of required evidence would vary depending on the severity of disease, the level of unmet need and safety profile of the product or service.

CMS said it would consider the panel's feedback as it drafts new guidance on the CED process. Last November, CMS began reviewing potential improvements to the CED process and solicited public comments (see BioCentury, Nov. 21, 2011).

Study shows FDA faster at reviews than EMA, Health Canada

Wed, 16 May 2012 17:45:40 -0700

FDA reviewed novel therapeutics significantly faster than EMA and Health Canada during 2001-10, and the majority of those products were first approved in the U.S., according to a study by Yale University researchers. The median length of time to complete the first review was 303 days for applications approved by FDA, compared with 366 days for EMA and 352 days for Health Canada (p<0.0001). The median total review time, which includes multiple review cycles, was also significantly shorter at FDA compared with both EMA and Health Canada (322 days vs. 366 and 393; p=0.002).

For products approved in the U.S. and Europe, 64% were first approved in the U.S. For products approved in the U.S. and Canada, 86% were first approved in the U.S. EMA did approve 96% of products after just one review cycle, compared to 62% of products approved by FDA and 69% of products approved by Health Canada. Data were published in the New England Journal of Medicine.

The authors said the results contradict recent criticisms of FDA's review speed and raise questions about whether the speed of the review process should be an emphasis of PDUFA reauthorization. The study comes as congress is considering PDUFA V legislation (see BioCentury Extra, May 14).

Harkin adds patient input requirement to PDUFA bill

Wed, 16 May 2012 17:45:40 -0700

Sen. Tom Harkin (D-Iowa) introduced a provision to the PDUFA reauthorization bill that would require HHS to include patient input during product development and regulatory discussions. The bill is under consideration by the full U.S. Senate. A vote has yet to be scheduled.

GSK to acquire Cellzome

Tue, 15 May 2012 00:00:00 -0700

GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) will acquire the 80.02% of proteomics company Cellzome AG (Heidelberg, Germany) it does not already own for L61 million ($98 million) in cash. GSK said Cellzome's proteomic mass spectrometry and screening capabilities will allow the pharma to reduce compound attrition during development. GSK and Cellzome are partnered in two inflammatory disease deals: one in 2008 to develop kinase-targeted small molecules using Cellzome's Kinobeads screening and profiling technology; and another in 2010 to use Cellzome's Episphere technology to discover small molecules against epigenetic targets. GSK and Cellzome plan to spin out assets that GSK does not plan to progress, but the companies did not disclose details on specific programs. The deal is expected to close on May 21.

NICE reverses prior decision, backs Zytiga

Tue, 15 May 2012 00:00:00 -0700

The U.K.'s NICE issued draft guidance late Tuesday recommending Zytiga abiraterone from Johnson & Johnson (NYSE:JNJ) in combination with prednisone or prednisolone in patients with metastatic, castration-resistant prostate cancer (CRPC) that has progressed on or after docetaxel-containing therapy -- the drug's approved indication. NICE reversed a prior negative decision after J&J submitted a revised patient access scheme, as well as information on the patients who are expected to benefit most from Zytiga and clarification on how many patients could receive the drug. NICE said Zytiga now meets the criteria for a life-extending, end-of-life treatment, with an incremental cost-effectiveness ratio (ICER) less than L50,000 ($80,370) per quality-adjusted life year (QALY). In February, NICE said Zytiga did not meet the life-extending, end-of-life treatment criteria and would have an ICER of L63,200 ($99,053) per QALY (see BioCentury Extra, Feb. 1). J&J gained the inhibitor of steroidal enzyme 17 alpha-hydroxylase/C17, 20 lyase (CYP17) through its 2009 acquisition of Cougar Biotechnology Inc., which received rights to abiraterone from BTG plc (LSE:BGC). BTG was up 2.80p to 373.10p on Tuesday.

Merck KGaA details savings targets for biotech cuts

Tue, 15 May 2012 00:00:00 -0700

Merck KGaA (Xetra:MRK) hopes to achieve EUR 300 million ($386.6 million) in net cost savings by 2014 through the previously announced restructuring of its Merck Serono biotech division. The target, announced Tuesday along with the group's 1Q12 financial results, follows last month's news that the pharma will shutter Merck Serono's headquarters in Geneva, Switzerland, and eliminate 500 (40%) of the 1,250 positions at the site. Merck plans to rebuild the division's pipeline through internal research and early in-licensing, and increase the division's focus on biologics. Merck Serono also will shift investments to regions outside of Europe and potentially expand its footprint in cancer. The division's sales are expected to grow 2-6% from EUR 5.6 billion ($7.3 billion) in 2011 to EUR 5.7-EUR 5.9 billion ($7.3-7.6 billion) in 2014. EBITDA before one-time items is expected to grow 15-21% by 2014 to EUR 1.8-EUR 1.9 billion ($2.3-$2.4 billion). Merck Serono's 1Q12 sales grew 5% to EUR 1.4 billion ($1.9 billion), with EBITDA before one-time items declining 2% to EUR 394 million ($525.6 million). Merck KGaA added that it will not consider "major portfolio divestments" or "transformational acquisitions" before completing the restructuring. The pharma did say it will continue to do "bolt-on" acquisitions. Revenues for the group rose 3% to EUR 2.6 billion ($3.5 billion). EPS before one-time items was EUR 1.67, off 13% from EUR 1.91 in 1Q11 (see BioCentury, March 19).

Scil, Ono in protein discovery deal

Tue, 15 May 2012 00:00:00 -0700

Scil Proteins GmbH (Halle, Germany) partnered with Ono Pharmaceutical Co. Ltd. (Tokyo:4528; Osaka:4528) to discover and develop therapeutics from Scil's Affilin ubiquitin-based protein libraries. Scil will select and characterize Affilin candidates against targets provided by Ono, which will be responsible for further development and commercialization. Scil will receive research funding, milestone payments and royalties.

Cytos raises $32.6 million

Tue, 15 May 2012 00:00:00 -0700

Cytos Biotechnology AG (SIX:CYTN) raised CHF30.4 million ($32.6 million) in an equity and debt financing led by venBio, with participation from Amgen Inc. (NASDAQ:AMGN); Abingworth; and Aisling Capital. The financing comprises CHF23.8 million ($25.5 million) through the sale of 12.7 million shares at CHF 1.87 and CHF6.6 million ($7.1 million) in a first tranche of secured convertible notes. The share price is a 4% premium to Cytos' close of CHF1.79 on March 20, before the offering was announced. The notes convert at CHF2.24. The investors will also receive warrants to purchase shares exercisable at CHF2.24. Cytos is eligible for an equal second tranche of notes subject to milestones related to asthma product CYT003-QbG10. Last week, Cytos raised CHF3.2 million ($3.4 million) through the sale of 1.7 million shares in a rights offering. Shareholders were eligible to purchase seven shares for every 15 held. Cytos plans to begin a Phase IIb trial of CYT003-QbG10 in patients with allergic asthma next half. The virus like particle (VLP) containing an oligonucleotide agonist of toll-like receptor 9 (TLR9) has completed three Phase II trials to treat allergic asthma and allergic rhinitis (see BioCentury, March 26).

Newron up as Zambon exercises PD option

Tue, 15 May 2012 00:00:00 -0700

Newron Pharmaceuticals S.p.A. (SIX:NWRN) jumped CHF1.50 (37%) to CHF5.51 on Tuesday after Zambon Co. S.p.A. (Bresso, Italy) exercised its option to license exclusive rights to develop and commercialize Parkinson's disease therapy safinamide worldwide, excluding Asian territories where Meiji Seika Pharma Co. Ltd. (Tokyo, Japan) has rights. The move came as Newron said the product showed "substantial evidence" of efficacy in two Phase III trials in both early and advanced PD patients, but did not disclose detailed data. Full data will be presented at upcoming scientific meetings. Worldwide regulatory submissions are slated for next year (see BioCentury Extra, May 14).

Arena planning follow-on after panel backs lorcaserin

Tue, 15 May 2012 00:00:00 -0700

Arena Pharmaceuticals Inc. (NASDAQ:ARNA) said late Tuesday it is planning a follow-on underwritten by Jefferies; Piper Jaffray; and BMO Capital Markets. On Friday, an FDA advisory committee voted 18-4, with one abstention, that the benefit-risk profile for Arena's lorcaserin supports approval to treat obesity. The PDUFA date for the serotonin (5-HT2C) receptor agonist is June 27 (see BioCentury, May 14). Arena, which made the announcement after market close, was off $0.55 to $6.06 on Tuesday.

Pfizer's Torisel misses second-line RCC endpoint

Tue, 15 May 2012 00:00:00 -0700

Pfizer Inc. (NYSE:PFE) said IV Torisel temsirolimus missed the primary endpoint of progression-free survival (PFS) vs. oral Nexavar sorafenib in the Phase III INTORSECT trial to treat advanced renal cell carcinoma in patients who failed first-line treatment with Sutent sunitinib. Additionally, Pfizer said data for the secondary overall survival (OS) endpoint showed significance favoring Nexavar. The open-label, international trial enrolled 512 patients. Pfizer markets Torisel in the U.S. treat advanced RCC; the label does not specify a line of treatment. In the EU, Torisel is marketed for first-line treatment of advanced RCC and to treat mantle cell lymphoma (MCL). Pfizer markets Sutent, while Bayer AG (Xetra:BAYN) and Onyx Pharmaceuticals Inc. (NASDAQ:ONXX) market Nexavar. Torisel is a mammalian target of rapamycin (mTOR; FRAP; RAFT1) inhibitor. Nexavar is an inhibitor of RAF1 and multiple receptor tyrosine kinases.

HHS announces national AD plan

Tue, 15 May 2012 00:00:00 -0700

HHS published a national plan to develop new prevention and treatment approaches for Alzheimer's disease by 2025. The Obama administration's proposed FY13 budget provides a $100 million increase over the amount allocated in FY12 to support the plan. The increase includes $80 million for AD research and $1.3 million to improve data collection. In February, the Obama administration announced it will immediately dedicate an additional $50 million from its FY12 budget for AD research (see BioCentury Extra, Feb. 7). The plan includes a $16 million commitment to help fund a five-year, $100 million prevention trial evaluating crenezumab from Genentech Inc. in about 300 people with a rare genetic mutation that creates a high risk for AD. The non-profit Banner Alzheimer's Institute will also provide $15 million for the trial, while Genentech will fund the remainder of the trial's cost. Data from the trial will be shared publicly. Genentech has exclusive, worldwide rights the product from AC Immune S.A. (Lausanne, Switzerland). The humanized mAb against beta amyloid is in Phase II testing in mild to moderate AD patients. Genentech is a unit of Roche (SIX:ROG; OTCQX:RHHBY). NIH also awarded $7.9 million to researchers at the University of Washington and Veterans Affairs Puget Sound for an 18-month U.S. trial of an insulin nasal spray in 240 patients with mild cognitive impairment (MCI) and mild to moderate Alzheimer's dementia.

FDA, WHO partner for vaccine research

Tue, 15 May 2012 00:00:00 -0700

FDA's Center for Biologics Evaluation and Research (CDER) partnered with the World Health Organization to research new vaccine trial designs and ways to enhance postmarketing surveillance of vaccine safety. CDER plans to provide WHO with up to $2 million in FY12, with the possibility to extend the funding for four additional years.

Intercell raises EUR 35.2 million

Mon, 14 May 2012 00:00:00 -0700

Intercell AG (VSE:ICLL; OTCQX:INRLY) raised EUR 35.2 million ($45.3 million) in a debt and equity financing. The financing comprised EUR 20 million ($25.8 million) in a secured loan from BB Biotech; and EUR 15.2 million ($19.5 million) in a private placement of 6.6 million shares at EUR 2.30 to BB Biotech; Novartis AG (NYSE:NVS; SIX:NOVN); and healthcare and institutional investors. Intercell was up EUR 0.11 to EUR 2.38 on Monday.

Ventrus gains on Phase III data

Mon, 14 May 2012 00:00:00 -0700

Ventrus Biosciences Inc. (NASDAQ:VTUS) gained $1.62 (15%) to $12.58 on Monday after the company's topical formulation of diltiazem hydrochloride (VEN 307) met the primary endpoint in a Phase III trial to treat anal fissures. The double-blind, European trial enrolled 465 patients. Ventrus plans to request a meeting with FDA to discuss the results, as well as next steps, including a second Phase III trial slated to start next half. Ventrus hopes to seek approval under section 505(b)(2) of the Food, Drug and Cosmetic Act, which allows sponsors to reference data on safety and efficacy from scientific literature or from previously approved products.

Mologen up on mCRC data

Mon, 14 May 2012 00:00:00 -0700

Mologen AG (Xetra:MGN) jumped EUR 1.90 (20%) to EUR 11.40 on Monday after twice-weekly MGN1703 as maintenance therapy met the primary endpoint of median progression-free survival vs. placebo in the Phase II IMPACT trial to treat metastatic colorectal cancer (mCRC). Specifically, median PFS in an undisclosed predefined target population of 46 patients showed that MGN1703 more than doubled median PFS vs. placebo (p<0.02). Based on the data, Mologen prematurely stopped enrollment in the trial at 58 patients. Overall survival (OS) data will continue to be collected. The trial had planned to enroll 129 patients who had disease stabilization or partial or complete remission of their cancer after receiving first-line therapy with chemotherapy plus Avastin bevacizumab for 4.5-6 months. MGN1703 is a toll-like receptor 9 (TLR9) agonist based on the company's double Stem Loop Immunomodulator (dSLIM) technology. Mologen plans to discuss next steps with FDA and EMA. The company also plans to start a Phase II trial of MGN1703 to treat non-small cell lung cancer (NSCLC) this year. Genentech Inc., a unit of Roche (SIX:ROG; OTCQX:RHHBY), markets Avastin in the U.S., while Roche markets it elsewhere.

H. Lundbeck gains on depression data

Mon, 14 May 2012 00:00:00 -0700

H. Lundbeck A/S (CSE:LUN) gained DKK9.60 to DKK118.60 on Monday after Lu AA21004 -- a serotonin modulator and stimulator -- significantly improved depression rating scores vs. placebo in three identical Phase III trials. The trials measured the change in Montgomery-Asberg Depression Rating Scale (MADRS) scores from baseline to week eight. H. Lundbeck and partner Takeda Pharmaceutical Co. Ltd. (Tokyo:4502) plan to submit an NDA to FDA for Lu AA21004 to treat major depressive disorder (MDD) in 2H12. Next half, Lundbeck plans to submit an MAA to EMA and an NDS to Health Canada. The trials enrolled 1,676 patients aged 18-75.

Paion reports Phase II data for remimazolam

Mon, 14 May 2012 00:00:00 -0700

Paion AG (Xetra:PA8) said all 75 evaluable patients undergoing surgery who received IV remimazolam for the induction of general anesthesia achieved loss of consciousness and underwent successful intubation in a Phase II trial. The open-label, Japanese Phase II trial enrolled 85 patients aged 20 and older who were receiving surgery under general anesthesia. Paion said it is seeking a partner outside of Japan to advance remimazolam into Phase III testing for procedural sedation. Ono Pharmaceutical Co. Ltd. (Tokyo:4528; Osaka:4528) has rights in Japan from Paion to develop and commercialize the short-acting GABA A receptor modulator. Paion gained EUR 0.11 (13%) to EUR 0.91.

Zambon exercises option for Newron's safinamide

Mon, 14 May 2012 00:00:00 -0700

Zambon Co. S.p.A. (Bresso, Italy) exercised its option from Newron Pharmaceuticals S.p.A. (SIX:NWRN) to license exclusive rights to develop and commercialize Parkinson's disease therapy safinamide worldwide, excluding Asian territories where Meiji Seika Pharma Co. Ltd. (Tokyo, Japan) has rights. Newron said on Monday that the product showed "substantial evidence" of efficacy in two Phase III trials in both early and advanced PD patients, but did not disclose detailed data. The company said full data would be presented at upcoming scientific meetings. Worldwide regulatory submissions are slated for next year. Last month, Zambon paid EUR 5 million ($6.3 million) for the option and a 9.1% stake in Newron. Newron will receive an additional EUR 5 million exercise fee and is eligible for milestones and double-digit royalties. Zambon will cover all development and regulatory costs for safinamide. Zambon will be eligible for one seat on Newron's board. The product is a alpha-aminoamide derivative that acts as a reversible monoamine oxidase B (MAO-B) and dopamine reuptake inhibitor. On April 17, the Merck Serono S.A. division of Merck KGaA (Xetra:MRK) returned rights to safinamide. Separately, Newron said founder Luca Benatti will step down as CEO and a director to pursue other opportunities. CFO Stefan Weber will succeed him. Newron, which made the announcement after market close, gained CHF0.24 to CHF4.01 on Monday.

Pharmas provide $23.1M for early stage research

Mon, 14 May 2012 00:00:00 -0700

A group of six pharmas will provide L14.4 million ($23.1 million) to fund a research consortium at the University of Dundee for an additional four years. The Division of Signal Transduction Therapy (DSTT) consortium, which was founded in 1998, is studying cell signaling and developing inhibitors of kinases and phosphatases to treat diseases such as cancer, arthritis and lupus. The pharmas share access to unpublished results, technology, know-how and reagents in the laboratories participating in the consortium, and also have first rights to license IP. The pharma companies are: AstraZeneca plc (LSE:AZN; NYSE:AZN), Boehringer Ingelheim GmbH (Ingelheim, Germany), GlaxoSmithKline plc (LSE:GSK; NYSE:GSK), Johnson & Johnson (NYSE:JNJ), the Merck Serono division of Merck KGaA (Xetra:MRK), and Pfizer Inc. (NYSE:PFE).

FDA concludes Gilenya safety review

Mon, 14 May 2012 00:00:00 -0700

FDA completed its safety review of Gilenya fingolimod and said it could not definitively conclude that the product contributed to recent deaths of patients receiving the multiple sclerosis drug, including a patient who died within 24 hours after the first dose. However, FDA said it remains concerned about Gilenya's cardiovascular effects after the first dose; the agency now contraindicates the Novartis AG (NYSE:NVS; SIX:NOVN) drug in patients with certain preexisting or recent heart conditions or stroke, or who are taking certain antiarrhythmic medications. Those include patients who in the last six months had a myocardial infarction, unstable angina, stroke, transient ischemic attack, decompensated heart failure or heart failure. FDA recommends hourly pulse and blood pressure monitoring for patients starting Gilenya. Novartis initially announced the label changes in April (see BioCentury Extra, April 20).

F-star names Haurum CEO, Dancer COO

Mon, 14 May 2012 00:00:00 -0700

Antibody company F-star Biotechnologische Forschungs und Entwicklungs GmbH (Vienna, Austria) hired John Haurum as CEO. Haurum is the former VP of research at ImClone Systems Inc., which Eli Lilly and Co. (NYSE:LLY) acquired 2008. He succeeds Kevin FitzGerald, who departed. F-star also hired Jane Dancer as COO and Sharon Grimster as VP of development. Dancer was formerly VP of business development at Cellzome AG (Heidelberg, Germany). Grimster is a former VP at Antisoma plc (LSE:ASM).

Medicis prices $450M note deal

Mon, 14 May 2012 00:00:00 -0700

Medicis Pharmaceutical Corp. (NYSE:MRX) raised $450 million in a bumped-up offering of senior convertible notes. The notes bear 1.375% interest and mature on June 1, 2017. They initially covert at $47.07, which is a 22% premium to Medicis' close of $38.52 last Wednesday, before the company proposed to raise $400 million in the offering. Deutsche Bank and JPMorgan were underwriters. Medicis markets dermatology and cosmetic products. Medicis was unchanged at $37.91 on Monday.

CBO: Senate PDUFA bill would provide $6.4B in user fees

Mon, 14 May 2012 00:00:00 -0700

The U.S. Senate version of PDUFA reauthorization legislation (S. 2516) would provide FDA with about $6.4 billion in user fees over 2013-17, according to a report from the Congressional Budget Office. The estimate includes about $128 million from new biosimilar user fees and about $1.6 billion from new generics user fees. Additionally, CBO said the bill's provisions to reduce marketing barriers for generics and biologics would reduce spending for mandatory health programs by $753 million over 2013-22. Those provisions include preventing branded manufacturers from using a product's REMS as justification for refusing to sell to manufacturers that want to use the branded product in the development of a generic or biosimilar. S. 2516 has been sent to the full Senate. Last week, the U.S. House Energy and Commerce Committee voted to send a version of PDUFA reauthorization to the full House. The House bill is now more closely aligned with the Senate PDUFA bill than previous versions. Changes that reflect the Senate bill include dropping sections that would add jobs creation and economic development language to FDA's mission and adding a section that would create an expedited development pathway for breakthrough drugs.

Arena soars on lorcaserin vote

Fri, 11 May 2012 00:00:00 -0700

Arena Pharmaceuticals Inc. (NASDAQ:ARNA) jumped $2.70 (74%) to $6.36 on Friday after an FDA advisory committee voted 18-4, with one abstention, that the benefit-risk profile for Arena's lorcaserin supports approval to treat obesity. The PDUFA date for the serotonin (5-HT2C) receptor agonist is June 27. The stock move represents an increase in market cap of $492.8 million. Arena's market cap is now $1.2 billion (see BioCentury Extra, May 10). Shares of obesity companies Vivus Inc. (NASDAQ:VVUS) and Orexigen Therapeutics Inc. (NASDAQ:OREX) also rose on Friday. Orexigen jumped $0.64 (19%) to $3.99, while Vivus was up $2.01 to $24.70. Orexigen is designing a cardiovascular outcomes trial for Contrave, a fixed-dose combination of naltrexone and bupropion, in response to a 2011 complete response letter. Vivus' Qnexa phentermine/topiramate is under review with a July 17 PDUFA date. In February, an agency panel voted 20-2 that the benefit-risk profile for Qnexa supports approval (see BioCentury Extra, April 9).

Prolor raises $32.5 million in follow-on

Fri, 11 May 2012 00:00:00 -0700

Prolor Biotech Inc. (NYSE-A:PBTH; Tel Aviv:PBTH) raised $32.5 million through the sale of 6.5 million shares at $5 in a follow-on underwritten by Jefferies; Ladenburg Thalmann; and Oppenheimer. The company proposed the offering late Thursday, when its share price was $5.69. Prolor was down $0.45 to $5.24 on Friday. Prolor's lead product, HGH-CTP (MOD-4023), is in Phase II testing to treat growth hormone deficiency (GHD). HGH-CTP is long-acting human growth hormone (hGH) combined with the company's carboxyl terminal peptide (CTP) technology.

OncoMed files for IPO

Fri, 11 May 2012 00:00:00 -0700

OncoMed Pharmaceuticals Inc. (Redwood City, Calif.) proposed to raise $115 million in an IPO underwritten by Jefferies; Leerink; Piper Jaffray; and BMO Capital Markets. OncoMed's clinical pipeline includes: demcizumab (OMP-21M18), a human mAb targeting delta-like 4 (DLL4), in Phase Ib testing for non-small cell lung cancer (NSCLC) and pancreatic cancer; OMP-18R5, a Wnt pathway antibody against frizzled homolog 7 (FZD7) in Phase I testing for solid tumors; and OMP-59R5, a mAb that binds to Notch 2 (NOTCH2) and NOTCH3, in Phase I testing for advanced solid tumors. OncoMed plans to begin a Phase Ib/II trial of OMP-59R5 this year. Bayer AG (Xetra:BAYN) has an option to license OMP-18R5. GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) has an option to license OMP-59R5.

FDA panel votes in favor of Quad for HIV

Fri, 11 May 2012 00:00:00 -0700

FDA's Antiviral Drugs Advisory Committee voted 13-1 that the benefit-risk profile of the fixed-dose Quad regimen from Gilead Sciences Inc. (NASDAQ:GILD) supports approval to treat HIV-1 infection in treatment-naive adults. The PDUFA date is Aug. 27. The combination consists of elvitegravir, cobicistat and Truvada emtricitabine/tenofovir. Gilead's elvitegravir, an HIV integrase inhibitor, and cobicistat, an inhibitor of cytochrome P450 3A (CYP3A), are in Phase III testing. Gilead markets Truvada. Gilead was up $0.59 to $51.84 on Friday.

Takeda's vedolizumab meets Crohn's endpoint

Fri, 11 May 2012 00:00:00 -0700

Takeda Pharmaceutical Co. Ltd. (Tokyo:4502) said vedolizumab met two of three co-primary endpoints in the Phase III GEMINI II trial to treat moderate to severe Crohn's disease (CD). The humanized mAb against integrin alpha(4)beta(7) led to a significantly greater proportion of patients in clinical remission at week six (induction phase) and at week 52 (maintenance phase) vs. placebo. However, vedolizumab missed the co-primary endpoint of improving enhanced response rate in the induction phase vs. placebo. Takeda said enhanced response rate is a clinical measure that includes observable changes in disease and patient reported outcomes. The trial enrolled 1,115 patients with moderately to severely active CD who have failed one or more conventional therapy, including tumor necrosis factor (TNF) alpha antagonists. In February, Takeda reported data from the Phase III GEMINI I trial in patients with ulcerative colitis (UC) showing that vedolizumab met the co-primary endpoints of a greater proportion of patients with a clinical response/remission in the induction and maintenance phases of the trial vs. placebo. Takeda plans to submit a BLA and MAA to FDA and EMA, respectively, for vedolizumab during its fiscal year 2013 ending March 30, 2014. The pharma said that because the CD data are still being analyzed, it has not yet been determined whether the initial regulatory submission will be for just UC or for both UC and CD.

Cytochroma reports data for vitamin D insufficiency

Fri, 11 May 2012 00:00:00 -0700

Cytochroma Inc. (Markham, Ontario) said CTAP101 significantly reduced mean intact parathyroid hormone (PTH) levels from baseline to week six vs. placebo in a Phase IIb trial to treat vitamin D insufficiency in patients with secondary hyperparathyroidism and stage III or IV chronic kidney disease (CKD). A 90 ug oral dose of the modified-release formulation of 25-hydroxyvitamin D reduced mean intact PTH levels by 39% compared to a 17% increase for placebo (p<0.005). The double-blind, U.S. trial enrolled 78 patients. Phase III testing is slated to start this year Data were presented at the National Kidney Foundation meeting in Washington.

Daiichi discontinues anorexia program

Fri, 11 May 2012 00:00:00 -0700

Daiichi Sankyo Co. Ltd. (Tokyo:4568; Osaka:4568) said it discontinued development of SUN11031, a human ghrelin that was in Phase III testing in Japan to treat anorexia nervosa. The pharma said that the "expected goal was not achieved," but did not disclose details. Last year, Daiichi Sankyo discontinued development of SUN11031 to treat cachexia associated with chronic obstructive pulmonary disease (COPD) in the U.S. and EU, where the compound was in Phase II testing, but also did not disclose details.

NICE rethinking value of long-term benefit

Fri, 11 May 2012 00:00:00 -0700

The U.K.'s NICE is asking for comments on an advisory panel's recommendation to increase the value attached to a drug's long-term benefit when calculating cost-effectiveness estimates per quality-adjusted life year (QALY). According to a report issued Friday, members voted 19-1 at a meeting in November that NICE should discount future costs and benefits differently. NICE currently applies a 3.5% discount rate to a drug's costs and benefits. The panel agreed the discount rate applied to costs should remain constant, but felt rates for future benefits could be varied. For example, panelists suggested that drugs with a high initial cost, but a benefit of more than 30 years, or those providing a total cure, could be discounted at a lower rate than drugs with a shorter benefit. Some panelists also suggested that discounting tends to favor shorter over longer term benefits, and this discriminates against preventive and other public health programs. The council conducted a mock appraisal of a fictional drug for pediatric polyarteritis nodosa, a rare condition affecting about 50 patients per year. Under the scenario, the drug was curative in about 77% of the population, and patients lived more than 60 years if they received treatment. At a 3.5% discount rate, the council assigned the drug a cost per QALY of about L57,000 ($92,000). If the discount rate were dropped to 0%, the cost per QALY would be L24,000 ($38,700). Comments on the report are due by June 8.

Panel backs Truvada for HIV-1 prophylaxis