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BioCentury Extra
As published Thursday, November 20, 2014 6:56 PM PST

  • Clovis reports interim Phase II in PARP race

    Clovis Oncology Inc. (NASDAQ:CLVS) reported initial data from the Phase II ARIEL2 trial of rucaparib (CO-338) to treat platinum-sensitive, relapsed ovarian cancer. The biotech suggested combining tumor BRCA1/2 mutation status with genomic loss of heterozygosity (LOH) analysis may help predict which patients are likely to benefit from the oral inhibitor of poly(ADP-ribose) polymerase-1 (PARP-1) and PARP-2.

    Patients were identified as BRCA mutant, wild-type BRCA with high genomic LOH or biomarker negative. Patients with the BRCA mutation had a 70% overall response rate compared to 40% in wild-type BRCA patients with high genomic LOH and 8% in biomarker negative patients. Overall response was defined by RECIST criteria and a CA-125 response.

    Updated results from ARIEL2 will be presented in 1H15. The study results will be used to evaluate efficacy based on tumor signature in the ARIEL3 Phase III study, which has started enrollment.

    Clovis has exclusive, worldwide rights to rucaparib from Pfizer Inc. (NYSE:PFE). The compound has Orphan Drug designation in the U.S. and EU to treat ovarian cancer.

    Competitor AstraZeneca plc (LSE:AZN; NYSE:AZN) has Lynparza olaparib in Phase Ib/II testing for heavily pretreated ovarian cancer. Interim data showed the PARP inhibitor in combination with paclitaxel and carboplatin led to an ORR of 66% in patients with or without BRCA mutations.

    Lynparza has a Jan. 3 PDUFA date as a maintenance treatment for platinum-sensitive relapsed ovarian cancer in patients with germline BRCA mutations who are responding to platinum-based chemotherapy. The compound also received positive backing from EMA's CHMP as monotherapy for maintenance treatment of relapsed, platinum-sensitive epithelial ovarian, fallopian tube or primary peritoneal cancer in patients with BRCA mutations who have responded to platinum-based chemotherapy.

    Clovis was up $2.51 to $50.79 on the day. AstraZeneca was off $1.17 to $74.21.

  • Sanofi says new products will beat consensus by EUR 5B

    On its annual analyst call Thursday, Sanofi (Euronext:SAN; NYSE:SNY) said it plans to launch 18 new products by 2020 with cumulative sales of EUR 30 billion. Interim CEO and Chairman Serge Weinberg said the projection is EUR 5 billion over the analyst consensus sales estimates of EUR 25 billion for the new products over that period.

    The new products include one animal health product, five vaccines, four drugs approved in 2014 and eight drug candidates in registration, Phase III or Phase II.

    Weinberg also said Sanofi would "enhance" its strategic priorities across its different businesses, including pharmaceuticals, consumer health, vaccines and animal health. However, the company's board has already said large acquisitions are off the table (see Biocentury, Nov. 17).

    "We are reviewing ways to strengthen these positions," said Weinberg. "We are trying to figure out more actively where we can build on these franchises."

    Weinberg took over as interim chief executive on Oct. 29 after the board removed Christopher Viehbacher. The pharma's shares were down $1.17 to $74.21 in New York, and were off EUR 1.81 to EUR 75.02 on Euronext.

  • FDA approves Purdue's abuse-resistant hydrocodone

    FDA approved Hysingla ER hydrocodone bitartrate from Purdue Pharma L.P. (Stamford, Conn.) to treat chronic pain for which alternative treatment options are inadequate. Hysingla is a once-daily, abuse-deterrent extended-release formulation of the opioid that received Priority Review.

    Purdue said Hysingla is the first formulation of hydrocodone FDA recognizes as abuse-deterrent. In its approval announcement, the agency said the drug's label is consistent with its 2013 draft guidance addressing abuse-deterrent opioids (see BioCentury, Jan. 9, 2013).

    FDA said it expects the drug's properties to reduce but not prevent abuse. The tablets are difficult to crush, break or dissolve, and form a viscous hydrogel that is difficult to prepare for injection, but the agency acknowledged that abuse via inhalation, ingestion or injection is still possible.

    FDA based its approval on a Phase III trial to treat patients with chronic low back pain, as well as data from other studies demonstrating abuse deterrence. The agency is requiring post-marketing surveillance studies to assess the effects of the abuse-deterrent features.

    Purdue expects to launch Hysingla ER early next year.

    Zogenix Inc. (NASDAQ:ZGNX) was unchanged at $1.21 on Thursday. Its Zohydro ER is the only other FDA approved hydrocodone-only oral drug, but lacks abuse deterrence technology.

  • MM-398 put on Fast Track for pancreatic cancer

    PharmEngine Inc. (GreTai:4162) and partner Merrimack Pharmaceuticals Inc. (NASDAQ:MACK) said FDA granted Fast Track designation to their MM-398 to treat metastatic pancreatic cancer in patients previously treated with gemcitabine. Merrimack plans to begin a rolling NDA submission by year end.

    Merrimack has exclusive, worldwide rights to the nanoparticle liposome formulation of irinotecan from PharmaEngine outside of Taiwan, where PharmaEngine retains rights.

    In September, Merrimack granted Baxter International Inc. (NYSE:BAX) exclusive rights to MM-398 outside of the U.S. and Taiwan (see BioCentury Extra, Sept. 24).

    Merrimack announced the news before U.S. market open Wednesday, and closed up $0.22 to $8.94 on Wednesday. PharmaEngine gained NT$15.50 to NT$242 in Taiwan on Thursday, giving it a market cap of NT$24.4 billion ($793.2 million).

  • St. Jude, UCL gene therapy cuts bleeding in hemo B trial

    Patients receiving scAAV2/8-LP1-hFIXco, a gene therapy developed by St. Jude Children's Research Hospital and University College London (UCL), maintained higher Factor IX levels and had markedly fewer spontaneous bleeding episodes in a Phase I dose-escalation trial to treat severe hemophilia B. The New England Journal of Medicine published the results on Nov. 20.

    Six patients in the study's high-dose group who received single infusions of the modified adeno-associated virus (AAV) 8 vector encoding the Factor IX gene achieved circulating Factor IX levels averaging 5.1% of normal after one year, up from a baseline of <1%. All 10 patients receiving infusions had Factor IX levels 1-6% of normal, and maintained elevated levels for a median of 3.2 years.

    Investigators reported that bleeding episodes decreased 90% (p=0.009) and 94% (p=0.03) for the full group and high-dose subgroup, respectively. Use of Factor IX replacement therapy declined in the year after infusion by an average of 92% (p=0.002) for the entire group and by 96% (p=0.03) for the high-dose subgroup.

    Baxter International Inc. (NYSE:BAX), uniQure N.V. (NASDAQ:QURE) and Spark Therapeutics LLC (Philadelphia, Pa.) also have adeno-associated virus vector-based gene therapies encoding the Factor IX gene. Baxter's BAX 335, uniQure's AMT-060 and Spark's AAV8-hFIX19 are all in Phase I/II testing to treat hemophilia B. uniQure predecessor Amsterdam Molecular Therapeutics licensed AMT-060 from St. Jude.

  • BioNano raises $53M in series C

    BioNano Genomics Inc. (San Diego, Calif.) raised $53 million in an untranched series C round led by new investors Legend Capital and Novartis Venture Fund. Other new investors included Federated Kaufmann Fund and Monashee Investment Management. Existing investors Domain Associates; Battelle Ventures; and Gund Investment also participated.

    BioNano sells the Irys genome mapping system, which uses single molecule imaging to visualize long nucleic acids, allowing researchers and clinicians to detect structural variations.

    BioNano launched the Irys System in the U.S. and China in 2012. It raised $10 million in a series B round in 2013.

  • Neothetics loses ground after $65.1M IPO

    Neothetics Inc. (NASDAQ:NEOT) fell $2 (14%) to $12 in its first day of trading on Thursday after it raised $65.1 million through the sale of 4.7 million shares at $14 in an IPO. The deal valued Neothetics at $190.7 million. Piper Jaffray; Guggenheim Securities; and Needham are underwriters.

    Next half, Neothetics expects to begin two Phase III trials of LIPO-202, a subcutaneous formulation of salmeterol, to reduce central abdominal bulging due to subcutaneous fat in non-obese patients. It expects top-line data by YE15, and plans to submit an NDA to FDA for the long-acting adrenergic receptor beta 2 agonist (LABA) in 2H16.

    Neothetics filed to raise up to $63.3 million last month.

  • Rockwell dips after $58.5M follow-on

    Rockwell Medical Inc. (NASDAQ:RMTI) lost $0.90 to $8.91 on Thursday after it raised $58.5 million through the sale of 6.5 million shares at $9 in a follow-on underwritten by BofA Merrill Lynch; Stifel; Summer Street; Craig-Hallum; Chardan Capital; and LifeSci Capital. The company proposed to raise $55 million in the offering after market close on Monday, when its share price was $10.58.

    Rockwell's lead compound, Triferic soluble ferric pyrophosphate citrate, is under FDA review to treat iron deficiency in chronic kidney disease (CKD) patients receiving hemodialysis. The soluble form of iron in liquid bicarbonate has a Jan. 24 PDUFA date.

  • Trevena planning $40M follow-on

    Neurology company Trevena Inc. (NASDAQ:TRVN) proposed to raise $40 million in a follow-on underwritten by Barclays; Cowen; Jefferies; JMP; and Needham.

    Trevena is developing GPCR ligands that activate or block specific signaling pathways mediated through individual GPCRs.

    Earlier this week, the company's TRV130, a G protein-biased mu opioid receptor (OPRM1; MOR) ligand to treat moderate to severe acute postoperative pain, met the primary endpoint in a Phase II study. Trevena plans to begin a second Phase II trial to treat soft tissue pain by year end, with data expected in mid-2015.

    In January, it began a Phase IIb trial of TRV027 to treat acute heart failure (AHF). The compound is a dual-acting ligand that inhibits angiotensin-mediated G-protein signaling and stimulates beta arrestin signaling via the angiotensin II type 1 (AT1) receptor (AGTR1).

    Trevena shed $0.36 to $5.23 on Thursday.

  • Celsus planning $46M follow-on

    Inflammation and dermatology play Celsus Therapeutics plc (NASDAQ:CLTX) intends to raise up to $46 million in a follow-on underwritten by Cowen; Piper Jaffray; and MTS. The offering will comprise American Depositary Shares, worth 10 common shares each. The company raised $9.2 million in an ADS offering in January.

    Celsus intends to submit an IND for MRX-6 cream 2%, a topical multifunctional anti-inflammatory compound that inhibits phospholipase A2 (PLA2), to treat atopic dermatitis.

    Celsus closed down $0.04 to $5.32 before proposing the offering late Thursday.

  • EyeGate downsizes proposed IPO

    Ophthalmology company EyeGate Pharmaceuticals Inc. (Waltham, Mass.) amended terms for its proposed IPO on NASDAQ underwritten by Aegis Capital Corp. The company now plans to sell 1.4 million shares at $6-$8. At $7, EyeGate would raise $10 million and be valued at $48.1 million. EyeGate filed to raise up to $28.8 million in July.

    By 2Q15, EyeGate plans to start a confirmatory Phase III trial of EGP-437, a dexamethasone phosphate opthalmic solution delivered by the EyeGate II Ocular Drug Delivery System, to treat non-infectious anterior segment uveitis.

  • IMS: Global drug spending peak in 2014

    In its annual "Global Outlook for Medicines" report published Thursday, the IMS Institute for Healthcare Informatics said it expects global drug spending to reach about $1.3 trillion by 2018 from $989 billion in 2013.

    The institute anticipates the launch of new drugs, fewer patent expirations and greater access to healthcare will push spending to increase at a compound annual growth rate of 4-7% over the next five years compared to 5.2% in the last five.

    According to IMS, the absolute global spend for pharmaceuticals will increase by $305-$335 billion on a constant dollar basis by 2018, compared to $230-$260 billion for the five years through 2017 covered in the firm's 2013 report.

    The report notes drug spending will spike at $70 billion this year on the back of outlays in the U.S., five major European countries and Japan, followed by more moderate growth. U.S. spending will maintain a 5-8% CAGR over five years, while emerging markets will provide nearly half of absolute growth by 2018, the institute said.

    The numbers do not reflect the impact of rebates, other discounting schemes and taxes, which IMS said would lower net revenues by $60-$80 billion over the five years, amounting to 25% of the growth forecast.

    IMS forecasts that emerging markets will account for 27-30% of global spending in 2018, up from 24.5% in 2013. Population growth and increased access to healthcare are the drivers of these markets, as non-branded medicines, primarily generics, will account for 83% of the growth in drug spend.

    The institute expects the fastest growth to occur in China, where it projects per capita spending to rise by more than 70%. Total drug outlays are projected to reach $155-$185 billion by 2018. IMS noted China is the world's second largest pharmaceutical market, as large as the top five European markets combined.

    Indeed, IMS anticipates that European spending for drugs will remain flat., and may decrease in France and Spain as a result of policies designed to rein in spending.

    The institute forecasts spending on cancer drugs to reach $100 billion in 2018, compared to $65 billion in 2013, and anticipates $100 billion in total spending on HCV treatments from 2014-18.

  • EMA updates conflict-of-interest policy

    EMA published its revised conflict-of-interest policy, clarifying which levels of conflict are tied to certain restrictions upon who can serve as experts or scientific committee members.

    The new policy precludes in perpetuity former pharma executives from serving in agency activities for any medical product for which the company holds marketing authorization. The "lifetime non-involvement" stipulation also prevents employees who had a "lead role" in the development of a product from participating in agency activities related to that product.

    For most conflicts, the policy specifies a three-year cooling-off period after the interest terminates, with restrictions decreasing over time.

    The policy does not require a cooling-off period for conflicts of purely financial interest and those related to grants or family connections.

    The revised policy was endorsed by EMA's management board in March 2014 and goes into effect on Jan. 30, 2015. It supersedes a 2012 policy.

  • U.K. launches review to speed patient access

    The U.K. government said it will launch a review of its drug, device and diagnostics development process in order to speed up patient access to new therapies.

    The Innovative Medicines and MedTech Review will explore ways in which precision medicine and new technologies are leading to faster drug development, examine how companies and regulatory agencies can share data in a faster review process, and study the role of charities and patient groups in increasing access to new treatments.

    The Association of the British Pharmaceutical Industry (ABPI), the BioIndustry Association and NICE all released statements welcoming the initiative. ABPI noted the review "needs to be fully joined up with other initiatives such as those that impact NICE," which it said needs "to change the way it evaluates new medicines and align with the changing regulatory model."

    NICE noted it already had proposed creating an office of innovation to provide a "flight path" for companies to take new products through to adoption by the NHS (see BioCentury Extra, Sept. 17).

    The agency also reiterated its call for "more productive" risk-sharing agreements with drug sponsors and NHS that are tied to evidence of value collected under NHS England's "commissioning through evaluation" program.

    The Department of Health did not release a timeline for the review process.

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