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BioCentury Extra
As published Thursday, May 21, 2015 6:15 PM PST

  • FDA funding added to 21st Century Cures Act

    The U.S. House Energy & Commerce Committee passed the 21st Century Cures Act (H.R. 6) on Thursday by a 51-0 vote following the last minute addition of FDA funding provisions. The bill includes a "Cures Innovation Fund" that provides FDA $110 million per year for five years. The $550 million total falls short of the $960 million FDA estimates it will cost to implement the bill, but is nonetheless a major achievement for FDA because prior versions did not appropriate additional money for the agency.

    The bill also includes requests to the House Appropriations Committee to appropriate additional funding to FDA, and a provision exempting user fees from sequestration.

    The E&C Committee agreed to measures intended to offset the bill's costs, which include a $10 billion five-year NIH "Innovation Fund." The offset measures include a change to the timing of Medicare's monthly reinsurance pre-payments to Medicare Advantage Part D prescription drug sponsors so that the federal government, rather than insurance companies, keeps interest revenue. This change is expected to generate $5-$7 billion. Other offsets include sales of oil from the Strategic Petroleum Reserve pegged at $5.2 billion, and $2.8 billion in savings from limiting Medicaid spending to Medicare payment levels on certain durable medical equipment.

    In remarks to rare disease advocates a few hours after the committee vote, E&C Chairman Rep. Fred Upton (R-Mich.) predicted that the full House will pass H.R. 6 this summer with 300-350 votes. Upton said he expects the Senate to pass a much more modest medical innovation bill. Upton said he hopes the two bills would be reconciled into a single bill that can be sent with bipartisan support to President Obama.

  • G-BA's Glybera assessment in line with uniQure's expectations

    Germany's Federal Joint Committee (G-BA) issued a final benefit assessment stating that the additional benefit was not quantifiable from gene therapy Glybera alipogene tiparvovec from uniQure N.V. (NASDAQ:QURE) to treat lipoprotein lipase (LPL) deficiency. uniQure chief commercial officer Hans Rohde told BioCentury the decision was in line with the benefit assessment that uniQure had requested and will allow the company to proceed with pricing negotiations in Germany, which are expected to last three to six months. G-BA had postponed its assessment of Glybera earlier this month (see BioCentury Extra, May 7).

    "This is what had been requested based on the number of patients we had and the lack of statistically significant data we could provide," he said, adding that uniQure believes the decision will not negatively affect pricing negotiations.

    In November, partner Chiesi Farmaceutici S.p.A (Parma, Italy) filed a pricing dossier for Glybera with G-BA that pegged the price of the gene therapy at EUR 1.1 million ($1.4 million) for a 62.5 kg patient (see BioCentury Extra, Nov. 24, 2014).

    G-BA will re-evaluate Glybera's benefit on June 1, 2016. The agency did not request specific safety or efficacy data from uniQure. Rather, it will review data already required by EMA on outcomes in patients treated with Glybera.

    Glybera is an adeno-associated virus (AAV) vector carrying the lipoprotein lipase (LPL) gene. It was approved in Europe in 2012 to treat LPL deficiency in patients with recurring, acute pancreatitis, a rare disorder. Chiesi expects the first patient in Europe to receive Glybera in mid-2015.

    uniQure lost $1.22 to $26.66 on Thursday.

  • bluebird climbs on first LentiGlobin sickle cell data

    bluebird bio Inc. (NASDAQ:BLUE) climbed $7.23 to $172.94 on Thursday after the company disclosed the first efficacy data for a sickle cell disease (SCD) patient treated with its LentiGlobin BB305 gene therapy. The data from the ongoing Phase I/II French HGB-205 study were included in an abstract published ahead of next month's European Hematology Association (EHA) meeting.

    Sickle cell is caused by a point mutation in the beta globin gene that leads red blood cells to make abnormal hemoglobin, which polymerizes when deoxygenated, distorting the cells. LentiGlobin BB305 comprises autologous CD34+ stem cells transduced ex vivo with a lentiviral vector delivering the human beta globin gene. The therapy is given via autologous hematopoietic stem cell transplantation (HSCT).

    At 4.5 months post-transplant, bluebird said 24% of the patient's hemoglobin contained the globin gene delivered by the therapy, up from 10% at 3 months post-transplant. At 4.5 months, 32% of the patient's total hemoglobin was what bluebird called anti-sickling hemoglobin.

    According to bluebird, sickle cell patients with red blood cells containing at least 30% anti-sickling hemoglobin have the "potential to reduce or eliminate the serious and life-threatening events with SCD."

    bluebird said the sickle cell patient was receiving chronic transfusions when enrolled, but has not received a transfusion since day 88 post-transplant. It took the SCD patient about a month for the transplant to engraft, and the patient has not had any post-transplant hospitalizations for complications related to sickle cell disease.

    The EHA abstract also includes an update on two beta thalassemia major patients treated with LentiGlobin showing those patients remain transfusion independent at 14 months and 11 months post-transplant.

    bluebird said Tuesday it had reached "general agreement" with U.S. and EU regulatory agencies on pathways that could result in accelerated approval of LentiGlobin for beta thalassemia major (see BioCentury Extra, May 19).

    bluebird has added $543 million in market cap since Monday's close, giving it a valuation of $5.7 billion at Thursday's close.

  • Sarilumab meets Phase III RA endpoints

    Sanofi (Euronext:SAN; NYSE:SNY) and Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) said sarilumab (REGN88, SAR153191), a human mAb against the IL-6 receptor, met both co-primary efficacy endpoints in the Phase III SARIL-RA-TARGET trial to treat rheumatoid arthritis (RA). The partners intend to submit a BLA to FDA in 4Q15.

    The trial compared 200 and 150 mg doses of sarilumab plus non-biologic disease modifying anti-rheumatic drugs (DMARDs) vs. placebo plus DMARDs in 546 RA patients who responded inadequately to, or were intolerant of, TNF-alpha inhibitors.

    Both doses improved signs and symptoms of RA at 24 weeks, a co-primary endpoint that was measured by the number of patients achieving ARC20 improvement. In the high- and low-dose arms, 61% and 56% of patients achieved an ARC20 vs. 34% in the placebo arm (p<0.001 for both doses).

    Sarilumab also met the second co-primary endpoint of improvement in physical function at 12 weeks, measured by change from baseline vs. placebo of the Health Assessment Question-Disability Index (HAQ-DI) (p<0.001 for both doses).

    The most common adverse events were infections, injection site reactions and decreased neutrophil counts.

    The companies also said sarilumab met the primary endpoints in two other Phase III studies. There were no technical failures of the sarilumab autoinjector among 217 patients in the SARIL-RA-EASY study, and no "clinically meaningful" differences in rates of serious AEs or infections among 202 patients receiving sarilumab vs. Actemra tocilizumab in the SARIL-RA-ASCERTAIN study. Actemra is a humanized mAb against the IL-6 receptor marketed by Roche (SIX:ROG; OTCQX:RHHBY) to treat RA.

    The SARIL-RA-TARGET trial assessed subcutaneous injections of each dose of sarilumab, administered every other week. Subcutaneous Actemra can be dosed as often as weekly (see BioCentury, Dec. 16, 2013).

    Sanofi and Regeneron partnered to develop and commercialize sarilumab in 2007.

    Regeneron gained $3.23 to $516 on Thursday.

  • Calithera gains on Phase I leukemia data

    Calithera Biosciences Inc. (NASDAQ:CALA) rose $1.17 (11%) to $12.02 on Thursday after it said one patient had a complete response among 15 efficacy-evaluable patients in a Phase I trial of CB-839 to treat relapsed or refractory acute myelogenous leukemia (AML). The company expects data from the study to be presented next month at the European Hematology Association meeting in Vienna.

    Calithera said the patient showed a CR in the bone marrow with incomplete recovery of peripheral counts after six treatment cycles of 21 days each. Patients in the study received doses of 100-1,000 mg three times daily.

    The company said five (33%) of the patients had stable disease for 4-10 treatment cycles. There were no dose-limiting toxicities.

    Calithera expects solid tumor data from Phase I trials of the oral selective glutaminase (GLS) inhibitor to be presented May 30 at the American Society for Clinical Oncology meeting in Chicago.

  • Follow-on roundup: Momenta, Dicerna

    Momenta Pharmaceuticals Inc. (NASDAQ:MNTA) and Dicerna Pharmaceuticals Inc. (NASDAQ:DRNA) raised a combined $186.6 million in follow-ons.

    Biosimilars and complex generics developer Momenta raised $137.8 million through the sale of 7.3 million shares at $19. Underwriters were Goldman Sachs; JPMorgan; and Stifel. The company proposed the offering Monday after market close, when its shares were at $20.22. Momenta slipped $0.02 Wednesday after pricing the offering post-market on Tuesday; it regained $0.02 to $19.31 on Thursday.

    Last month, FDA approved Glatopa glatiramer acetate from Momenta and partner Sandoz, a unit of Novartis AG (NYSE:NVS; SIX:NOVN). Glatopa is a generic version of Copaxone from Teva Pharmaceutical Industries Ltd. (NYSE:TEVA). Momenta and Sandoz would risk being liable for damages if they were to launch Glatopa ahead of either the expiration of Teva's Copaxone patent on Sept. 1 or a decision by the Court of Appeals for the Federal Circuit on pending IP litigation (see BioCentury Extra, April 16).

    RNAi oncology company Dicerna raised $48.8 million through the sale of 2.8 million shares at $17.75. Jefferies; Leerink; Cowen; and Stifel were underwriters. Dicerna proposed the offering Monday after market close, when its shares were valued at $20.27. It priced the follow-on late Wednesday, and dipped $2.38 (13%) to $16.31 on Thursday.

    Dicerna expects data this year from a Phase I trial of DCR-MYC to treat multiple cancers. The dicer substrate short interfering RNA (siRNA) targeting c-Myc is in Phase Ib/II testing to treat hepatocellular carcinoma (HCC).

  • Ablynx raises EUR 100M in bond issue

    Ablynx N.V. (Euronext:ABLX) raised EUR 100 million ($113.9 million) in a private placement of convertible bonds with investors outside the U.S. The bonds mature on May 27, 2020, bear 3.25% interest and convert into stock at EUR 12.93, a 27% premium to Ablynx's Wednesday close of EUR 10.16.

    Ablynx develops Nanobodies, therapeutic proteins based on single-domain antibody fragments. In 2H15, the company plans to begin a Phase III trial of caplacizumab (anti-vWF Nanobody), a therapy comprising Nanobodies targeting von Willebrand factor, to treat thrombotic thrombocytopenic purpura (TTP).

    Earlier this week, the company partnered with the Genzyme Corp. unit of Sanofi (Euronext:SAN; NYSE:SNY) to develop Nanobodies to treat multiple sclerosis (see BioCentury Extra, May 18).

    Ablynx gained EUR 0.04 to EUR 10.20 on Thursday.

  • ASCO proposes cancer payment reforms

    The American Society of Clinical Oncology released an updated proposal to reform payments for cancer care, expanding on a payment model proposed last year.

    The model unveiled last May included five bundled payments intended to compensate physicians for services they currently provide but for which they are not reimbursed. The prior model did not address how practices should be paid for drugs (see BioCentury, June 2, 2014).

    The new proposal includes three distinct payment models that would allow practices to transition towards bundled payments. The most advanced model would include bundled payments that would also cover the cost of drugs.

    The basic Patient-Centered Oncology Payment (PCOP) would provide $750 per new patient for treatment planning, $200 per month during treatment, $50 per month during active monitoring and for up to six months after the end of treatment. It also would provide $100 per month during treatment in a clinical trial and for six months afterward. In addition to the new payments, oncology practices would continue to bill for evaluation and management services, chemotherapy infusions and drugs.

    The second PCOP option would replace the 58 current billing codes with fewer than a dozen new codes in three categories. A new patient payment category would cover diagnosis, treatment planning and education and support services prior to the start of treatment. Diagnostic tests would still be billed separately. A treatment month category would cover practice costs including office visits, patient contacts and administration of chemotherapy, though the cost of drugs, tests and imaging would still be billed separately. An active monitoring category would cover costs incurred during the time a patient is off treatment but still being monitored for progression.

    The final PCOP option consists of monthly bundled payments for practice services as well as other costs that may include drugs, testing, imaging, emergency room visits and hospitalizations. The payments would be administered as budgets and adjusted based on risks such as treatment complications and increased drug prices.

    In return for payments received under PCOP, practices would be accountable for helping patients avoid treatment complications that require ER visits and hospitalizations, following evidence-based guidelines for drugs, lab testing and imaging studies, and providing end-of-life support.

    ASCO estimated that savings to payers would offset their additional payments to practices, which would be able to maintain lower rates of emergency room visits and hospitalizations and better ensure the appropriate use of drugs and tests. ASCO estimated that payers would see a net reduction of at least 4% in total spending if all practices participated in PCOP models.

    The proposal is open for comments until July 20.

  • House passes R&D tax credit

    The U.S. House of Representatives voted 274-145 to pass legislation that would increase and make permanent the R&D tax credit. H.R. 880, the American Research and Competitiveness Act, would increase the tax credit to 20% from 14%. The bill will now go to the U.S. Senate.

    On Tuesday, the Obama Administration said it "strongly opposes" the bill because it does not include offsets and would add $180 billion to the deficit over the next 10 years. The administration said that if President Obama were presented with the bill, his senior advisors would recommend that he veto it.

    The Biotechnology Industry Organization (BIO) expressed strong support for the bill.

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