Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) raised its 2014 guidance after reaching an agreement with the French government over reimbursement for Orphan drug Soliris eculizumab. The company is not disclosing details of the agreement or the list price of Soliris in France, but said the agreement covers both atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria (PNH) -- the drug's two approved indications. Alexion said the agreement also covers Soliris shipments prior to 2014, and as a result the company expects to record about $88 million in additional Soliris sales in 1Q14. Alexion said it now expects 2014 non-GAAP EPS of $4.37-$4.47 on full-year revenues of $2.15-$2.17 billion. In January, the company said it expected 2014 EPS of $3.70-$3.80 on $2-$2.02 billion in revenues.
The French reimbursement agreement comes less than a week after the U.K.'s NICE requested information from Alexion on the development costs for Soliris for aHUS, as well as an explanation of how those costs relate to the company's proposed price for Soliris for the indication. In the U.K., Soliris costs L3,150 ($5,266) per vial, which translates to an estimated cost of L252,000-L340,200 ($421,243-$568,678) per patient for the first year of treatment, depending on the indication. A maintenance dose is estimated to cost L245,700-L327,600 ($410,712-547,616) per year (see BioCentury Extra, March 3).
Alexion was up $11.95 to $180 on Monday.
CMS Administrator Marilyn Tavenner said that because of the "complexities of these issues and stakeholder input," the agency does not plan to finalize some of the changes to Medicare Part D proposed in January, according to a letter Tavenner sent to legislators. The proposals that CMS will not finalize include: eliminating two of the six protected classes for which plans must provide "substantially all" approved drugs; limiting the number of plans a given insurance company can offer in a single region; and allowing CMS to intervene in negotiations between payers and pharmacies, which would expand preferred pharmacy networks. In her letter, Tavenner said CMS will "engage in further stakeholder input before advancing some or all of the changes" in the future.
Since CMS issued the proposal, legislators and organizations including patient groups, health insurers and the Biotechnology Industry Organization (BIO) and Pharmaceutical Research and Manufacturers of America (PhRMA) have pressured Tavenner and HHS Secretary Kathleen Sebelius to withdraw or not implement the proposed changes (see BioCentury, Feb. 17).
Tavenner said CMS does plan to finalize some measures, including changes to antifraud provisions that strengthen standards for prescribers.
Douglas Hoey, CEO of the National Community Pharmacists Association; Chris Holt, director of health care policy at the American Action Forum; Edith Rosato, CEO of the Academy of Managed Care Pharmacy; and Andrew Sperling, director of federal legislative advocacy at the National Alliance on Mental Illness discussed the proposed Part D changes on the March 9 edition of BioCentury This Week television (see BioCentury This Week, March 9).
Efficacy endpoints for trials evaluating treatments for chronic fatigue syndrome (CFS) should include patient-reported symptoms and "reflect the claimed clinical benefit related to how a patient feels or functions," according to draft guidance FDA released on Monday. The guidance notes that "FDA is not aware of existing [patient-reported outcome] instruments or set of instruments optimal for measurement of fatigue or other symptoms" and will consider novel measures or tools that have been developed and evaluated for other conditions. Other efficacy endpoints could include exercise capacity, post-exertional malaise and health-related quality of life, according to the guidance.
Additionally, the guidance notes that FDA does not recognize a definition or diagnostic criteria for CFS, and sponsors can thus use "any case definition or criteria" to define a patient population. Sponsors should provide justification of their definition, and trials should exclude patients with potentially confounding diseases. The guidance also recommends two 24-week, placebo-controlled trials and the collection of long-term safety data.
FDA has not previously issued guidance on CFS, for which there are no approved treatments. The new draft guidance incorporates feedback from stakeholders at a Patient-Focused Drug Development meeting held last year that was focused on CFS. During that meeting, stakeholders stressed the heterogeneity of symptoms and the need to identify specific CFS populations and subgroups for drug development (see BioCentury, May 6, 2013).
Comments on the draft guidance are due 60 days from publication in the Federal Register, which is expected Tuesday.
Members of the California Technology Assessment Forum (CTAF) said on Monday that there is adequate evidence to support the superiority of Sovaldi sofosbuvir from Gilead Sciences Inc. (NASDAQ:GILD) and Olysio simeprevir from Johnson & Johnson (NYSE:JNJ) to standard of care, but said the benefits do not justify the high cost of the HCV drugs.
Based on meta-analyses conducted by the not-for-profit Institute for Clinical and Economic Review (ICER), the CTAF panel said Sovaldi or Olysio plus peginterferon and ribavirin were superior to a first-generation protease inhibitor added to peginterferon and ribavirin in patients with HCV genotype 1 infection. The panel also said Sovaldi plus ribavirin was superior to peginterferon plus ribavirin alone in patients with HCV genotype 2 and genotype 3 infections. Sovaldi is a single isomer of a nucleotide analog HCV NS5B polymerase inhibitor. Olysio is an HCV NS3/4A protease inhibitor.
However, most panel members felt that the improved efficacy of Sovaldi or Olysio compared to use of a first-generation protease inhibitor wasn't sufficient to justify their high costs. As a result, many of the panel members recommended that Sovaldi and Olysio only be used to treat the sickest HCV patients, including those with cirrhosis and other disease complications like hepatocellular carcinoma (HCC) or advanced liver fibrosis.
The wholesale acquisition cost (WAC) for a 28-day supply of Sovaldi is $28,000, while the WAC for a 28-day supply of Olysio is $22,120. ICER calculated that the treatment regimens would cost $70,000-$100,000 per course of therapy. The first-generation protease inhibitors are Incivek telaprevir from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) and Victrelis boceprevir from Merck & Co. Inc. (NYSE:MRK). The WAC for a 28-day supply of Incivek is $22,051, and the WAC for a 28-day supply of Victrelis is $6,687.
CTAF conducts and discusses technology assessments to help patients, clinicians, insurers and policy makers interpret and use evidence to improve the quality and value of healthcare. CTAF was funded and managed by the Blue Shield of California Foundation, but is now part of ICER.
HHS's Agency for Healthcare Research and Quality (AHRQ) released a draft topic refinement outlining the key questions and scope of a potential technology assessment on therapeutic options for obesity, including surgery, drugs and lifestyle interventions. CMS requested the topic refinement -- the first step in a potential technology assessment by AHRQ. CMS uses technology assessments to inform national coverage policies.
The two questions in the draft topic refinement are: what is the comparative effectiveness of interventions for obesity as evaluated by outcomes that include weight and obesity-related conditions; and how well does treatment-induced reduction in body mass index (BMI) predict obesity-related outcomes. Comments are due March 20.
FDA Commissioner Margaret Hamburg is scheduled to testify on Thursday at a hearing of the U.S. Senate Health, Education, Labor and Pensions (HELP) Committee to discuss FDA's oversight of the food and medical product supply. The hearing will focus on the agency's implementation of reforms mandated by the FDA Safety and Innovation Act (FDASIA), as well as the agency's current priorities and initiatives.
Cancer company Cerulean Pharma Inc. (Cambridge, Mass.) filed to raise up to $75 million in an IPO on NASDAQ underwritten by Leerink Partners; Canaccord; JMP Securities; and Wedbush PacGrow. Cerulean's CRLX101 is in a two-part Phase II trial in ovarian cancer patients who had progressed on prior lines of cytotoxic chemotherapy. In January, Cerulean said CRLX101 met the primary endpoint in the first part of the trial of four or more of 29 patients achieving six months of progression-free survival (PFS).
CRLX101 is also in Phase Ib/II testing for relapsed renal cell carcinoma (RCC), with a Phase II trial slated to start next half; and in Phase Ib/II testing for neoadjuvant rectal cancer. Cerulean has exclusive, worldwide rights to develop and commercialize the dual inhibitor of topoisomerase I (TOP1) and hypoxia-inducible factor 1 alpha (HIF1A; HIF1-alpha) from Calando Pharmaceuticals Inc. (Pasadena, Calif.), a subsidiary of Arrowhead Research Corp. (NASDAQ:ARWR).
Kythera Biopharmaceuticals Inc. (NASDAQ:KYTH) is reacquiring rights outside of the U.S. and Canada to ATX-101 from partner Bayer AG (Xetra:BAYN). The synthetic sodium deoxycholate has completed Phase III testing to reduce submental (under chin) fat. Kythera -- which granted Bayer exclusive rights to co-develop and commercialize ATX-101 outside the U.S. and Canada in 2010 -- will now hold full global rights to the compound. The company plans to submit an NDA to FDA next quarter, with ex-U.S. regulatory submissions slated for the next 12 months. Bayer will receive $33 million in Kythera stock and a $51 million unsecured promissory note due March 2024. Bayer also is eligible for up to $123.8 million in ex-U.S. and ex-Canadian sales milestones.
Kythera announced the news after market close on Monday. The company was off $0.49 to $44.53 on the day.
Keryx Biopharmaceuticals Inc. (NASDAQ:KERX) submitted an MAA to EMA for Zerenex ferric citrate to treat hyperphosphatemia in dialysis dependent and non-dialysis dependent patients with chronic kidney disease (CKD). The product is under FDA review for CKD in dialysis patients, with a June 7 PDUFA date. In January, Japan approved the oral ferric iron-based phosphate binder as Riona. Keryx has worldwide rights to the product, including Japan but excluding the rest of the Asia Pacific region, from Panion & BF Biotech Inc. (Taipei, Taiwan).
Keryx was off $0.28 to $14.99 on Monday.
Janssen Pharmaceuticals Inc. will provide Alector LLC (San Francisco, Calif.) undisclosed funding for research of an undisclosed Alzheimer's disease target from Alector through proof-of-concept disease models. Janssen, a unit of Johnson & Johnson (NYSE:JNJ), will have a "time-limited option to negotiate a commercial agreement." Alector is located at Janssen Labs @QB3, a San Francisco incubator owned by the California Institute for Quantitative Biosciences (QB3).
Alector debuted last year with a non-exclusive license to yeast-based antibody discovery technology from Adimab LLC (Lebanon, N.H.) and undisclosed funding from Polaris Venture Partners and OrbiMed Advisors to develop human mAbs for four undisclosed targets for AD, dementia and neurodegeneration (see BioCentury Extra, Oct. 31, 2013).
Trophos S.A. (Marseille, France) said once-daily oral olesoxime (TRO19622) prevented loss of motor function as measured by the change from baseline in Motor Function Measure (MFM) score at two years -- the primary endpoint -- compared to placebo in a Phase II/III trial to treat spinal muscular atrophy (SMA). The company said placebo patients showed a progressive loss of motor function typical for the disease. The double-blind, European trial enrolled 165 patients ages three to 25 with type II or non-ambulatory type III SMA. Trophos said it plans to submit U.S. and EU regulatory applications for the product in the indication, but declined to disclose a time frame. Olesoxime is a small molecule with cholesterol-like structure that interacts with the mitochondrial permeability transition pore (MPTP).
Torii Pharmaceutical Co. Ltd. (Tokyo:4551) said TO-203 met the primary endpoint of reducing Total Combined Rhinitis Score (TCRS) vs. placebo in a Japanese Phase II/III trial in patients with house dust mite-induced allergic rhinitis. Torii is also conducting a Japanese Phase II/III trial with TO-203 in patients with dust mite-induced allergic asthma, with data expected in "the coming months." Torii said the results from both trials will determine whether the company submits a Japanese NDA for the tablet-based sublingual allergen immunotherapy.
Torii has exclusive Japanese rights to TO-203 from ALK-Abello A/S (CSE:ALK-B), which plans to submit a European regulatory application for the product under the name Mitizax this year. Merck & Co. Inc. (NYSE:MRK) has exclusive North American rights, where the product is known as MK-8237. Last week, Merck said MK-8237 met the primary endpoint in a Phase IIb trial for house dust mite-induced allergic rhinitis (see BioCentury Extra, March 5).
ALK-Abello was up DKK5 to DKK685 on Monday.
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