FDA extended to May 26 from Feb. 26 the PDUFA date for eteplirsen (AVI-4658) from Sarepta Therapeutics Inc. (NASDAQ:SRPT) to treat Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Sarepta fell $1.48 (12%) to $10.74 on Monday.
Sarepta said it submitted four-year clinical effectiveness data to FDA in January that the agency considers to be a major amendment to the NDA. The company added six-minute walk test (6MWT) and loss of ambulation data compared to historical controls.
Last month, FDA postponed a scheduled meeting of its Peripheral and Central Nervous System Drugs Advisory Committee to discuss the eteplirsen NDA, citing forecasts of inclement weather in Washington, D.C. FDA has not yet rescheduled the meeting (see BioCentury Extra, Jan. 20).
Eteplirsen is a phosphorodiamidate morpholino oligomer (PMO) that induces skipping of exon 51 in dystrophin mRNA.
The U.K.'s NICE issued draft guidance recommending against the use of Praluent alirocumab from Sanofi (Euronext:SAN; NYSE:SNY) and partner Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) alone or in combination with lipid-lowering therapies to treat primary hypercholesterolemia or mixed dyslipidemia.
NICE expressed concern that Praluent had not been compared with the combination of a statin and Zetia ezetimibe from Merck & Co. Inc. (NYSE:MRK) in patients with non-familial hypercholesterolemia. The committee also said that although it was "reasonable to infer that alirocumab would reduce cardiovascular events, the extent of this reduction was uncertain." Sanofi expects results in 2017 from a cardiovascular outcomes trial (CVOT) of Praluent.
Additionally, NICE said that even with an undisclosed discount under a patient access scheme, most of the "value-for-money analyses resulted in ICERs that exceeded the range normally considered to be a cost-effective use of NHS resources."
Comments on the Praluent draft guidance are due Feb. 29.
In a reversal of opinion, NICE now recommends a specific dose of rival PCSK9 inhibitor Repatha evolocumab from Amgen Inc. to treat high cholesterol in some patients. In November, NICE rebuffed Repatha, citing a lack of CV outcomes data (see BioCentury Extra, Nov. 18, 2015).
In updated guidance, NICE now backs a 140 mg dose of Repatha every two weeks alone or in combination with other lipid-lowering therapies as an option in some patients with primary non-familial hypercholesterolemia, heterozygous familial hypercholesterolemia (HeFH) or mixed dyslipidaemia with progressive, symptomatic cardiovascular disease (CVD), as well as certain severe HeFH patients without CVD who meet other conditions. The recommendations are contingent on Amgen providing Repatha at a discount under a patient access scheme. Comments on the new Repatha draft guidance are due Feb. 26.
On Monday, Amgen was down $2.04 to $143. Regeneron fell $6.08 to $390.53. Both declines were less steep than the 4.4% fall on the BioCentury 100 index.
Cigna Corp. (NYSE:CI) said Monday that it struck an outcomes-based pricing deal with Novartis AG (NYSE:NVS; SIX:NOVN) for heart failure drug Entresto sacubitril/valsartan.
Cigna said the agreement ties financial terms to improvement in health based on a primary metric of reduction in the proportion of customers with heart failure hospitalizations.
FDA approved Entresto in July 2015 to reduce the risk of cardiovascular death and hospitalization for heart failure in chronic heart failure patients with reduced ejection fraction. The drug was superior to SOC enalapril in the Phase III PARADIGM-HF trial, reducing the risk of death from CV causes by 20% and reducing hospitalizations due to heart failure by 21%.
On its 4Q15 earnings call last month, Novartis said it had agreed to outcomes-based deals for Entresto with two U.S. payers, and that both deals included an undisclosed base rebate that will go up or down depending on the drug's performance (see BioCentury, Feb. 1).
Novartis spokesperson Eric Althoff confirmed to BioCentury on Monday that Cigna and Aetna Inc. (NYSE:AET) were the two payers (see BioCentury Extra, Jan. 29).
Pfizer Inc. (NYSE:PFE) named the management team that is to lead the company after it completes its merger with Allergan plc (NYSE:AGN).
Pfizer intends to combine its Global Innovative Pharma (GIP) and Vaccines, Oncology and Consumer (VOC) businesses as the merger is completed. It said VOC Group President Albert Bourla will become GIP Group President and lead the combined team. Current Group President of GIP Geno Germano will leave the company.
Bill Meury, Allergan's EVP and president of branded pharma, will become group president of Pfizer's new Global Specialty and Consumer Brands segment. The division will include Pfizer's Consumer Healthcare unit and Allergan's ophthalmology and aesthetics businesses, including both therapeutic and cosmetic Botox onabotulinumtoxinA.
The company said in November 2015 that Ian Read will remain chairman and CEO of the merged company, while Allergan CEO Brent Saunders will be president and COO (see BioCentury Extra, Nov. 23, 2015).
Bourla and Meury will report to Saunders, as will Pfizer EVP and President of Global Supply Tony Maddaluna; EVP of Strategy, Portfolio and Commercial Operations Laurie Olson; and Group President of Global Established Pharma John Young.
Several top Pfizer executives will continue to report to Read. They include EVP and CFO Frank D'Amelio; President of Worldwide R&D Mikael Dolsten; EVP of Worldwide Human Resources Chuck Hill; EVP and Chief Compliance and Risk Officer Rady Johnson; EVP and General Counsel Doug Lankler; EVP and CMO Freda Lewis-Hall; and EVP of Corporate Affairs Sally Susman.
Protein degradation newco C4 Therapeutics Inc. (Cambridge, Mass.) hired Andrew Phillips as CSO. He was senior director of the center for development of therapeutics at the Broad Institute of MIT and Harvard.
Achillion Pharmaceuticals Inc. (NASDAQ:ACHN), which is developing complement factor D (CFD; adipsin) inhibitors for ultra-rare diseases, named Martha Manning EVP and general counsel, and Amy Jennings SVP of regulatory affairs. Manning was general counsel at iCeutica Inc (Philadelphia, Pa.). Jennings was head of U.S. regulatory for diabetes at Sanofi (Euronext:SAN; NYSE:SNY).
Immuno-oncology play Lion Biotechnologies Inc. (NASDAQ:LBIO) named Steven Fischkoff CMO. He was VP of clinical and medical affairs at the Celgene Cellular Therapeutics division of Celgene Corp. (NASDAQ:CELG).
Regenerative medicine company Osiris Therapeutics Inc. (NASDAQ:OSIR) said CEO Lode Debrabandere resigned. CBO Dwayne Montgomery will be interim CEO. Osiris also promoted VP and General Manager of Wound Care Frank Czworka to COO.
CTI BioPharma Corp. (NASDAQ:CTIC; Milan:CTIC) fell $0.68 (61%) to $0.44 on Monday after it said FDA placed a partial clinical hold on on pacritinib (BAX 2201), citing "excess mortality and other adverse events" in the Phase III PERSIST-1 study. Last month, CTI and partner Baxalta Inc. (NASDAQ:BXLT) completed submission of a rolling NDA to FDA for pacritinib to treat patients with intermediate- and high-risk myelofibrosis (MF) with low platelet counts of <50,000/uL. PERSIST-1 was included in the NDA.
CTI said FDA observed an increased risk of adverse events in pacritinib-treated patients vs. control in the trial, with excess mortality most evident during a crossover period in which control-treated patients could switch to pacritinib.
Under the hold, patients already receiving the orally available dual inhibitor of Janus kinase-2 (JAK-2) and FMS-like tyrosine kinase 3 (FLT3; CD135) may continue to receive the therapy. No new patients are to receive the therapy, and pacritinib studies are not to begin crossover periods or enroll new patients.
CTI has completed enrollment in a second Phase III study, PERSIST-2, which is evaluating pacritinib in MF patients whose platelet counts are ≤100,000/uL. The company did not respond to inquiries.
In March 2015, CTI said pacritinib met PERSIST-1's primary endpoint to treat MF. CTI released a subgroup analysis in June 2015 showing efficacy in patients with platelet counts of <50,000/uL. Those patients cannot be treated with Jakafi ruxolitinib from Incyte Corp. (NASDAQ:INCY), an approved oral JAK-1 and JAK-2 inhibitor that can cause thrombocytopenia (see BioCentury Extra, June 1, 2015).
Shire plc (LSE:SHP; NASDAQ:SHPG) is merging with Baxalta.
BioCryst Pharmaceuticals Inc. (NASDAQ:BCRX) crumbled $4.36 (71%) to $1.78 on Monday after its oral avoralstat (BCX4161) missed the primary endpoint in the Phase II/III OPuS-2 trial to prevent acute attacks in patients with hereditary angioedema (HAE). Avoralstat failed to significantly reduce angioedema attack frequency vs. placebo. Patients in the double-blind trial received 300 or 500 mg avoralstat thrice daily or placebo for 12 weeks.
The company said high-dose avoralstat showed significant improvements vs. placebo on the secondary endpoints of duration of attacks and Angioedema Quality of Life total score.
According to BioCryst, "meaningfully better exposure is needed for avoralstat," a selective inhibitor of plasma kallikrein that subsequently suppresses bradykinin production.
The company expects data mid-year from a bioavailability study in healthy volunteers of a solid dosage form of avoralstat that is intended to achieve higher exposure and have twice daily dosing. BioCryst said it will not pursue further development of the liquid formulation of avoralstat tested in OPUS-2.
BioCryst also has BCX7353, a second-generation kallikrein inhibitor that the company said could provide higher exposure levels. The company expects data by YE16 from the dose-ranging APeX-1 study of BCX7353 in HAE patients.
BIO unveiled a one-minute advertisement and a new website stressing the value of biomedical innovation. Last month, Ron Cohen, president and CEO of Acorda Therapeutics Inc. (NASDAQ:ACOR) and chairman of BIO, told BioCentury the trade group would soon begin a media and lobbying campaign emphasizing the value of biopharmaceuticals and the high costs of other healthcare products and services (see BioCentury, Jan. 18).
A BIO spokesperson told BioCentury the organization expects to spend "in the mid-to-high six figures" on this stage of the campaign, and said the campaign's website will include "multiple voices" including those of patients and other stakeholders.
Also on Monday, PhRMA launched the latest advertisement from its own campaign highlighting the value of innovation.
In February 2015, Mersana Therapeutics Inc. (Cambridge, Mass.) raised $35 million in a series B round. The Feb. 3, 2016, BioCentury Extra misstated the financing history.
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