Alexion ups guidance
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.
POLITICS & POLICY
CMS backpedals on Part D changes
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.
Sponsors for CFS treatments should focus on symptoms
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symptoms and "reflect the claimed clinical benefit related to how a patient feels or functions," according to draft guidance FDA released on Monday.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.
Group says HCV drugs effective, but lack value
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.
CMS assessing obesity treatments
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.
Hamburg to testify at Senate HELP hearing
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.
Cerulean files IPO
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).
Kythera reacquires global rights to ATX-101
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.
Keryx submits Zerenex MAA
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).
J&J to fund Alector research for AD target
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).
Trophos reports data for muscular atrophy compound
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 reports Japanese data for dust mite immunotherapy
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.