FDA's Arthritis Advisory Committee voted 21-3 to support approval of CT-P13 from Celltrion Inc. (KOSDAQ:068270), a biosimilar of Remicade infliximab from Johnson & Johnson (NYSE:JNJ), to treat seven of the eight indications for which Remicade is approved. No biosimilar mAb has received FDA approval.
Remicade is approved for rheumatoid arthritis, ankylosing spondylitis, adult and pediatric Crohn's disease, adult and pediatric ulcerative colitis (UC), psoriatic arthritis and plaque psoriasis. Celltrion is seeking extrapolation of CT-P13 across all except pediatric UC, for which Remicade has Orphan Drug designation.
Celltrion's BLA included clinical studies in which CT-P13 showed similar efficacy and safety to Remicade in RA and ankylosing spondylitis. The BLA also included analytical and pharmacokinetic data demonstrating CT-P13's chemical and biologic similarity to Remicade.
The panel members who supported approval across the indications said based on the "totality" of the data for CT-P13, Celltrion had demonstrated CT-P13's biosimilarity to Remicade. Other panelists were less convinced of CT-P13's biosimilarity for the Crohn's and UC indications, and wanted to wait until data are available in 4Q16 from an ongoing non-inferiority Phase III study of CT-P13 in Crohn's.
CT-P13 had a biosimilar user fee goal of June 8, 2015. An initial advisory committee meeting to discuss the BLA in March 2015 was postponed.
Immunology company Alder Biopharmaceuticals Inc. (NASDAQ:ALDR) hired James Bucher as SVP and general counsel. Bucher was VP of corporate legal affairs and corporate compliance officer at Exelixis Inc. (NASDAQ:EXEL).
Medgenics Inc. (NYSE-M:MDGN) promoted VP of Finance and Investor Relations Brian Piper to CFO. He replaces John Leaman, who left the hematology company.
Berg LLC (Framingham, Mass.) hired Jason Haddock as CFO and COO of the cancer company. He was head of finance for worldwide commercialization and worldwide medical at Bristol-Myers Squibb Co. (NYSE:BMY).
Infectious disease company Amplyx Pharmaceuticals Inc. (San Diego, Calif.) named Michael Hodges CMO. He was CMO and head of drug development at Santaris Pharma A/S, which Roche (SIX:ROG; OTCQX:RHHBY) acquired.
Actelion Ltd. (SIX:ATLN) reported 2015 product sales of CHF2.05 billion ($2.06 billion), crossing the CHF2 billion ($2.02 billion) threshold for the first time. Sales were up 7% at constant exchange rates from CHF1.96 billion ($1.97 billion) in 2014.
Actelion also said it expects core operating income to grow in 2016 in the low single-digit percentage range, assuming constant currency. Core EPS in 2015 was CHF6.16, up 15% at constant exchange rates from CHF5.58 in 2014.
Sales of pulmonary arterial hypertension drug Opsumit macitentan were CHF516 million ($519.9 million) in 2015, up from CHF179 million ($180.3 million) in 2014. Actelion launched the drug in late 2013. Sales of PAH drug Tracleer bosentan were CHF1.2 billion ($1.2 billion) in 2015, down 11% in constant currencies from CHF1.4 billion in 2014, excluding rebate reversals. Actelion said Tracleer sales fell due to "sharply decreased enrollment of new patients" in countries where Opsumit is available.
"Doctors can now treat their PAH patients with at least one of our drugs or with a combination of our drugs through the entire course of the disease," noted CEO Jean-Paul Clozel on a conference call Tuesday. Last month, Actelion launched PAH drug Uptravi selexipag in the U.S., while EMA's CHMP recommended Uptravi's approval.
Clozel said Actelion is focused on growing beyond PAH, calling the company's pipeline of candidates for rare or severe diseases "the base of our second wave of growth." Actelion's Phase III programs include cadazolid, a chimeric quinolonyl-oxazolidinone antibiotic in development to treat Clostridium difficile-associated diarrhea (CDAD); and ponesimod (ACT-128800), a sphingosine-1-phosphate receptor 1 (S1PR1; S1P1; EDG1) agonist for relapsing multiple sclerosis (MS).
Clozel and EVP and CFO Andre Muller said on the call that Actelion intends to be disciplined with regard to M&A. Muller called the dealmaking environment is "highly competitive" and said other cash-rich buyers may be "a little more desperate" than Actelion. Last year, hyperkalemia play ZS Pharma Inc. said it had discussed a deal with Actelion. AstraZeneca plc (LSE:AZN; NYSE:AZN) later acquired ZS Pharma for $2.7 billion in cash (see BioCentury Extra, Nov. 6, 2015).
Actelion gained CHF1.30 to CHF123.80 on Tuesday.
Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) shed $24.56 to $365.97 on Tuesday after reporting 4Q15 and 2015 financial results that missed expectations and guiding to slower growth of 2016 U.S. sales of ophthalmic drug Eylea aflibercept.
Regeneron's 4Q15 total revenues were $1.1 billion, up 37% from $802 million in 4Q14 but short of the $1.2 billion consensus estimate. Full-year revenues were $4.1 billion, up 46% from $2.8 billion in 2014 but shy of the $4.2 billion consensus.
Sales of LDL-lowering therapy Praluent alirocumab were $7 million in 4Q15 and $11 million for the year. Regeneron and partner Sanofi (Euronext:SAN; NYSE:SNY) launched the PCSK9 inhibitor in the U.S. in July and in the EU in 4Q15.
Regeneron reported non-GAAP diluted EPS in 4Q15 of $2.83, up slightly from $2.79 in 4Q14 but missing the $3.36 Street estimate. Full-year non-GAAP diluted EPS was $12.07, up from $10 in 2014 but below the $12.57 consensus.
Regeneron reiterated overall 2016 guidance first introduced last month at the JPMorgan Healthcare Conference. The company said Tuesday it expects U.S. net product sales of Eylea to grow by about 20% over 2015. Net U.S. Eylea sales grew 54% to $2.7 billion in 2015, from $1.7 billion in 2014.
President Obama's FY17 budget request includes several programs intended to reduce the cost of pharmaceuticals, including allowing Medicaid to negotiate prices for high-cost drugs and establishing transparency and reporting requirements in drug pricing. The White House released the proposed budget Tuesday, and HHS released a summary of its proposed outlays. While there is little chance the full budget will be enacted, the proposals add to the list of legislative options the next administration and Congress may consider.
The budget would grant the HHS Secretary negotiating power for high-cost drugs and biologics covered under Medicare Part D, as well as require manufacturers to submit additional cost and clinical data for certain high cost drugs. In a pricing transparency measure, manufacturers would be required to disclose R&D costs, payer discounts and production costs. Additional measures would prohibit "pay-for-delay" deals and reduce the exclusivity period for biologics to seven years from 12. HHS said the measures would save Medicare more than $19 billion over 10 years.
BIO released a statement critical of the budget, saying its proposals would "severely restrict the ability of innovative biomedical companies to recoup their enormous investments in innovation."
The budget would provide NIH with $33.1 billion for FY17, a 3% increase from FY16. The boost includes $680 million to support the National Cancer Moonshot initiative. FDA would receive $75 million for the Moonshot program. Last week, Obama asked for $1 billion, including $755 million for NIH and FDA, for the Moonshot (see BioCentury Extra, Feb. 1).
It would provide $309 million to NIH to support development of the Precision Medicine Initiative, including $300 million for its research cohort 1 million volunteers. The allocation is a $107 million bump from FY16. Obama announced the initiative last year (see BioCentury Extra, Jan. 30, 2015).
The budget also would also give $195 million to the Brain Research Advancing Innovative Neurotechnologies (BRAIN) Initiative, a $45 million increase over FY16.
The proposal would grant FDA $5.1 billion for FY17, an 8% increase over FY16. FDA set a performance target to review 90% of ANDAs received in FY17 with ten months, up from its current target of 75% of ANDAs within 15 months.
U.S. Senate Health, Education, Labor and Pensions (HELP) Committee Chairman Lamar Alexander (R-Tenn.) said he is willing to consider creating an Innovation Projects Fund that would give the NIH director "authority and ability to harness extraordinary opportunities" for five high priority NIH initiatives. Alexander said discussion of how to pay for the Innovation Fund, including the use of mandatory funding, would need to occur on the Senate floor rather than in committee.
The comment came in prepared remarks ahead of a Tuesday hearing to mark up a set of seven bipartisan bills introduced as part of the Senate's companion legislation to the 21st Century Cures Act, which the House of Representatives passed last summer. The House bill included two mandatory five-year funds: an $8.8 billion NIH Innovation Fund and a $550 million FDA Cures Fund. Alexander did not mention an FDA innovation fund on Tuesday. His proposed NIH fund would cover a narrower range of activities than the one in the 21st Century Cures Act, and would likely receive less funding (see BioCentury Extra, July 10, 2015).
"We may be able to support these initiatives in our normal appropriations process but I am willing to consider using mandatory funding for these initiatives," said Alexander. He named five programs, including the Precision Medicine Initiative and the National Cancer Moonshot initiative, that could receive funding.
He noted that the HELP committee would have to offset any increases in mandatory funding for the NIH initiatives by cutting existing mandatory funding elsewhere. "As a practical matter, there are not bipartisan ways to pay for an innovation fund through mandatory funding in this committee's jurisdiction," added Alexander.
In January, Alexander said the HELP committee would take an a la carte approach as it drafts new companion legislation to the 21st Century Cures Act, rather than bundling provisions into a single innovation bill (see BioCentury Extra, Jan. 19).
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