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BioCentury Extra
As published Monday, July 21, 2014 6:23 PM PST


  • Cerecor raises $15 million in B round

    Cerecor Inc. (Baltimore, Md.) raised $15 million in the first tranche of a planned $32 million series B round. New investors New Enterprise Associates; Apple Tree Partners; and MPM Capital led the tranche. Cerecor's CERC-301 is in Phase II testing as adjunctive therapy for major depressive disorder (MDD), with data expected by year end. Cerecor has exclusive, worldwide rights to develop and commercialize the small molecule NMDA receptor NR2B subtype (GRIN2B; NR2B) antagonist from Merck & Co. Inc. (NYSE:MRK).

    NEA's James Barrett and Frank Torti, MPM's Luke Evnin and Apple Tree's Behshad Sheldon and Mayukh Sukhatme joined Cerecor's board.

  • TB Alliance's high-dose regimen meets in Phase IIb

    The not-for-profit Global Alliance for TB Drug Development said once-daily 200 mg PA-824 in combination with moxifloxacin and pyrazinamide -- what the alliance calls the PaMZ TB regimen -- met the primary endpoint vs. once-daily Rifafour rifampicin/isoniazid/ethambutol/pyrazinamide in the Phase IIb NC-002 trial to treat tuberculosis (TB) infection. The dose of PA-824 in combination with moxifloxacin and pyrazinamide led to a greater average daily reduction in colony forming units (CFUs) counts over eight weeks vs. once-daily Rifafour from Sanofi (Euronext:SAN; NYSE:SNY) (p<0.05). A 100 mg dose of PA-824 in combination with moxifloxacin and pyrazinamide missed the primary endpoint vs. Rifafour.

    The open-label, South African and Tanzanian trial enrolled 181 patients with drug-sensitive TB infection. In the trial, 20% of patients were also co-infected with HIV. The alliance said there was no interference or side effects between the PaMZ regimen and the antiretroviral therapies being taken by co-infected patients. Data were presented at the International AIDS Conference in Melbourne.

    By year end, the TB Alliance plans to start the international Phase III STAND trial to evaluate the regimen for drug-susceptible TB and multidrug-resistant TB (MDR-TB). PA-824 is a nitroimidazopyran, moxifloxacin is a fluoroquinolone antibiotic and pyrazinamide is an off-patent TB drug. The alliance has exclusive, worldwide rights to PA-824 from Chiron Corp., now part of Novartis AG (NYSE:NVS; SIX:NOVN); and to moxifloxacin for TB from Bayer AG (Xetra:BAYN).

  • Right-to-try bill moves through Michigan legislature

    The Michigan Senate will vote on Aug. 13 whether to pass a right-to-try (RTT) bill (SB001). The bill, which the Michigan Senate Health Policy Committee approved last Wednesday, would allow eligible terminally ill patients access to investigational products that have completed a Phase I trial but have not been approved by FDA. Under the proposed law, manufacturers may either provide the product for free or require patients to pay.

    The Michigan House of Representatives version of the bill (HB56751) was introduced and referred to the House Committee on Health Policy in June.

    Similar legislation has been passed in Colorado, Louisiana and Missouri, and RTT bills are moving through the legislatures of at least two other states. New Jersey state representatives introduced a version in June and the Arizona legislature has voted to submit an RTT law to voters as a ballot initiative in November. To benefit patients, RTT laws must withstand possible legal challenges, and biopharma companies must be willing to rely on state law to bypass FDA oversight (see BioCentury, June 30).

    BioCentury This Week TV delved into the RTT issue in its June 15 edition with Lucy Caldwell, communications director of the Goldwater Institute; Diane Dorman, VP for Public Policy at the National Organization for Rare Disorders (NORD); and Sjaak Vink, CEO of myTomorrows (see BioCentury This Week, June 15).

  • CVS joins PBMs in questioning Sovaldi cost

    Pharmacy benefit manager CVS Caremark Corp. (NYSE:CVS) has joined the debate over the price of HCV drug Sovaldi sofosbuvir from Gilead Sciences Inc. (NASDAQ:GILD). In an article in the Journal of the American Medical Association, CVS CMO Troyen Brennan and CSO William Shrank stressed the need for "effective approaches to control costs for high-priced medications" such as Sovaldi. They noted that Sovaldi is a huge outlier among specialty medications due to its high cost and large population eligible for treatment. The authors estimated there may be as many as 3 million HCV patients eligible for sofosbuvir in the U.S.

    CVS' comments echo those of another PBM, Express Scripts Holding Co. (NASDAQ:ESRX). In April, Express Scripts said it would ask plan sponsors to "stand side-by-side" with the PBM to encourage Gilead to provide Sovaldi at a "more reasonable price." The wholesale acquisition cost (WAC) for a 12-week course of Sovaldi is $84,000.

    Earlier this month, two members of the Senate Finance Committee requested pricing information for Sovaldi from Gilead including an explanation of the difference between the drug's current cost and a lower cost projected by its original developer. Gilead gained Sovaldi through its 2012 acquisition of originator Pharmasset Inc. (see BioCentury Extra, July 11, 2014).

    Stakeholders including National Coalition on Health Care President and CEO John Rother and America's Health Insurance Plans spokesperson Brendan Buck discussed the cost of providing access to Sovaldi and how to price specialty drugs on the July 13 episode of BioCentury This Week television (see BioCentury This Week, July 13).

  • HHS issues interpretative rule on 340B and Orphan drugs

    As expected, HHS's Health Resources and Services Administration (HRSA) issued an interpretative rule on Monday that states that Orphan drugs can be discounted under Medicare's 340B program when used for non-Orphan indications. The 340B program requires manufacturers to deeply discount outpatient drugs to hospitals and clinics bearing the brunt of healthcare for low income and other special populations. In 2010, Congress amended the program to exclude Orphan drugs from discounting, but the law was unclear on whether the exclusion applied when the drugs were used for non-Orphan indications.

    A July 2013 final rule from HHS said Orphan drugs were to be discounted when used in non-Orphan indications. The Pharmaceutical Research and Manufacturers of America subsequently challenged the final rule, and in May, the U.S. District Court for the District of Columbia vacated the final rule because HHS lacked the authority to issue it. Despite the ruling, last month, HRSA said it would continue to enforce the 2013 final rule and its interpretation of the statue for Orphan drugs, noting the court did not rule on the agency's interpretation of the statute (see BioCentury Extra, June 18).

    Following the May ruling, PhRMA filed a motion asking the court to either require HHS provide "additional briefing" on whether the vacated final rule could exist as an interpretive rule, or to issue a judgment vacating the final rule both because HHS lacked the authority to issue the rule and because the final rule could not exist as an interpretative rule. An interpretative rule articulates an agency's interpretation of a statute but does not create rights or requirements beyond those explicit in the statute.

    In in its May ruling, the court said it was "inclined to think" HHS's final rule could not survive as an interpretative rule because of its legal consequences for 340B hospitals and drug manufacturers. On Monday, PhRMA requested an expedited decision from the court.

  • India wants price controls on cancer generics

    India's National Pharmaceutical Pricing Authority (NPPA) requested pricing information on generic cancer drugs from six pharma companies so the agency can fix the products' ceiling prices. The products, which are included on the country's national list of essential medicines, include Refolinon folinic acid from Pfizer Inc. (NYSE:PFE); cyclosporine from Biocon Ltd. (NSE:BIOCON; BSE:BIOCON), Ranbaxy Laboratories Ltd. (NSE:RANBAXY; BSE:500359) and Cipla Ltd. (Mumbai, India); mercaptopurine from Cipla; and cyclophosphamide, marketed as Endoxan by Drugs Dropship (Haryana, India) and as Cycloxan by Biochem Pharmaceutical Industries Ltd., which is part of Zydus Cadila Group (NSE:CADILAHC; BSE:532321).

    The agency requested the information by Thursday.

    Earlier this month, NPPA capped the prices of 50 cardiovascular and diabetes drugs, including some not listed on the essential medicines list. Under India's Drug Price Control Order, the government can fix the ceiling price or retail price of any drug in "extraordinary circumstances" if it considers the action necessary in the public interest (see BioCentury Extra, July 14).

  • FDA to discuss best practices for developing biomarkers

    FDA will explore scientific and regulatory approaches to biomarker development, including their acceptance and utility in drug development programs, during a public meeting on Sept. 5. The meeting will focus on identifying challenges and best practices for biomarker-based development programs, including co-development of in vitro diagnostic devices and use of biomarkers as outcome measures in clinical trials. The meeting satisfies an FDA commitment under PDUFA V.

  • Marletta to leave as TSRI president and CEO

    The Scripps Research Institute Chairman Richard Gephardt announced on Monday that President and CEO Michael Marletta "has indicated his desire to leave" Scripps. Gephardt said in a statement the board is working with Marletta on a transition plan.

    On June 18, Scripps and the University of Southern California announced discussions on a potential partnership between the organizations. Media outlets said TSRI was in talks to be acquired by the university and that TSRI faculty opposed such a takeout. Later that month, TSRI said it was on "secure financial footing in the near-term" but that it was reviewing its options. On July 9 TSRI said talks with USC were mutually terminated.

  • Allergan beats Street, to cut 13% of workforce

    Allergan Inc. (NYSE:AGN) reported 2Q14 financial results on Monday that beat the Street but said the company will reduce headcount by about 1,500 (13%) to about 10,200. The cuts include about 700 R&D positions and about 250 positions from the company's sales force. Allergan said all R&D programs that are in the clinic will continue; however, the company cut earlier stage programs, including a preclinical acne program and Phase I programs in rosacea and glaucoma. The company will have about 1,450 R&D positions and 1,450 sales positions after the cuts. In addition to the staff cuts, Allergan said it will eliminate 250 vacant positions. The company said the cuts will generate pre-tax savings of about $475 million in 2015.

    Allergan reported 2Q14 non-GAAP diluted EPS of $1.51, beating the Street's $1.44 estimate and up from $1.22 in 2Q13. Revenues in the quarter grew 16% to $1.83 billion, beating the Street's $1.77 billion estimate. The company said it now expects 2014 net sales of $6.9-$7.1 billion and non-GAAP diluted EPS of $5.74-$5.80. Previously, Allergan said it expected 2014 sales of $6.8-$7 billion and full-year EPS of $5.64-$5.73.

    Last month, Valeant Pharmaceuticals International Inc. (TSX:VRX; NYSE:VRX) made a hostile tender offer to acquire Allergan for $72 in cash and 0.83 Valeant shares per Allergan share. The company's board rejected the offer, which values Allergan at around $51.6 billion based on Valeant's close on Friday of $121.97. The offer is set to expire on Aug. 15.

    Allergan was up $3.74 to $171.14 on Monday.

  • Auris, Avalanche, Tobira, Loxo set IPO terms

    Audiology company Auris Medical AG (Zug, Switzerland), ophthalmic play Avalanche Biotechnologies Inc. (Menlo Park, Calif.), infectious disease company Tobira Therapeutics Inc. (San Francisco, Calif.) and Loxo Oncology Inc. (Stamford, Conn.) amended NASDAQ IPOs on Monday.

    Auris now plans to sell 6.9 million shares at $10-$12. At the $11 midpoint, the company would raise $75.9 million and be valued at $283.2 million. Last month, Auris filed to raise up to $86.3 million. Jefferies; Leerink Partners; JMP Securities; and Needham are underwriters. The company's esketamine (AM-101) is in the North American Phase III TACTT2 trial and the European Phase III TACTT3 trial for acute inner ear tinnitus, with top-line data expected in early 2016. Esketamine is an intratympanically injected non-competitive antagonist of NMDA receptors. Next quarter, Auris plans to begin Phase III testing of its AM-111 to treat acute inner ear hearing loss. A selective c-jun N-terminal kinase (JNK) inhibitor, AM-111 is partnered with Xigen S.A. (Epalinges, Switzerland).

    Avalanche now plans to sell 5.4 million shares at $13-$15. At the $14 midpoint, the company would raise $75.6 million and be valued at $292.4 million. Partner Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) agreed to purchase about $10 million in Avalanche shares in a concurrent private placement. In May, the companies partnered to discover, develop and commercialize gene therapy products to treat ophthalmologic diseases. Last month, Avalanche filed to raise up to $86.3 million in the IPO. Jefferies; Cowen; Piper Jaffray; and William Blair are underwriters. Avalanche plans to report Phase IIa data of AVA-101 to treat wet age-related macular degeneration (AMD) in mid-2015. AVA-101 is an adeno-associated virus (AAV) vector-based therapy that delivers soluble vascular endothelial growth factor (VEGF) receptor 1 (sFLT1; sVEGFR-1), a VEGF inhibitor.

    Tobira now plans to sell 4.6 million shares at $12-$14. At the $13 midpoint, the company would raise $60 million and be valued at $142.7 million. Last month, Tobira filed to raise up to $69 million. BMO Capital Markets; JMP Securities; Oppenheimer; and Nomura are underwriters. Tobira's cenicriviroc has completed a Phase IIb trial to treat HIV infection, but Tobira plans to advance the compound for the indication with a partner or with non-dilutive financing. This half, Tobira plans to start a Phase II trial of the dual CC chemokine receptor 5 (CCR5; CD195) and CCR2 antagonist to treat non-alcoholic steatohepatitis (NASH). Tobira has exclusive, worldwide rights to cenicriviroc from Takeda Pharmaceutical Co. Ltd. (Tokyo:4502).

    Loxo plans to sell 4.4 million shares at $12-$14. At $13, the company would raise $57 million and be valued at $195.8 million. Last month, Loxo filed to raise up to $69 million. Cowen; Stifel; Oppenheimer; and JMP Securities are underwriters. Loxo's LOXO-101 is in Phase I testing to treat advanced solid tumors. Data are expected early next year. Loxo has exclusive, worldwide rights to the neurotrophic tyrosine kinase receptor 1 (NTRK1;TrkA) inhibitor from Array BioPharma Inc. (NASDAQ:ARRY). Loxo raised $24 million in a series B in May (see BioCentury Extra, May 6).

  • FDA panel to discuss NPS's Natpara

    FDA's Endocrinologic and Metabolic Drug Products Advisory Committee will meet on Sept. 12 to discuss a BLA from NPS Pharmaceuticals Inc. (NASDAQ:NPSP) for Natpara to treat hypoparathyroidism. The PDUFA date is Oct. 24. The product is a recombinant human parathyroid hormone 1-84 (PTH).

    NPS was up $0.20 to $29.29 on Monday.


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