Novartis AG (NYSE:NVS; SIX:NOVN) on Tuesday said it remains on track to submit a BLA to FDA early next year for its CTL019 chimeric antigen receptor (CAR) T cell therapy to treat pediatric acute lymphoblastic leukemia (ALL). A second submission for diffuse large B cell lymphoma (DLBCL) is slated for 2H17.
On a conference call to announce 3Q16 earnings, CEO Joe Jimenez said the company's August decision to integrate its stand-alone cell and gene therapy unit into its oncology business would lower overhead while improving the unit's development, manufacturing and commercial capabilities as CTL019 prepares to launch.
Novartis also reported accelerating biopharmaceutical sales at its Sandoz unit, growing to $262 million in 3Q16, up 41% from 3Q15 at constant exchange rates. The Sandoz biopharma division includes both Glatopa glatiramer acetate, a generic version of multiple sclerosis drug Copaxone from Teva Pharmaceutical Industries Ltd. (NYSE:TEVA); and Zarxio filgrastim-sndz, a biosimilar of neutropenia drug Neupogen from Amgen Inc. (NASDAQ:AMGN).
Novartis said total sales of Zarxio now exceed $100 million since its September 2015 launch in the U.S. The pharma said Glatopa, developed with Momenta Pharmaceuticals Inc. (NASDAQ:MNTA) and launched in June 2015, now accounts for 40% of the 20 mg daily glatiramer acetate market in the U.S. Teva also markets a 40 mg thrice-weekly formulation of Copaxone.
Novartis expects a stream of pivotal data for 10 candidates it believes could be blockbusters, including readouts by year end for candidates in advanced macular degeneration and to prevent migraine; by the end of 2017 for three additional programs; and for 5 other products by the end of 2019.
Among the company's existing growth drivers, autoimmune drug Cosentyx secukinumab posted sales of $301 million in 3Q16 and $737 million for the first nine months of 2016. MS drug Gilenya fingolimod recorded a 15% sales gain at constant exchange rates to $790 million in 3Q16 for a nine-month total of $2.3 billion.
For the quarter, Novartis reported core EPS of $1.23 on total revenues of $12.13 billion, compared with the consensus estimate of $1.19 per share on $12.23 billion in revenues.
Portola Pharmaceuticals Inc. (NASDAQ:PTLA) said it submitted an NDA to FDA for oral Factor Xa inhibitor betrixaban (PRT054021) as extended-duration prophylaxis of venous thromboembolism (VTE) in acute medically ill patients with risk factors for VTE. Betrixaban has Fast Track designation from FDA in the indication.
In March, Portola reported data from the Phase III APEX trial showing that betrixaban failed to show superiority to standard of care enoxaparin to prevent VTE in acute medically ill patients. The trial's design required that significant superiority be shown in the trial's first cohort in order to evaluate two larger cohorts sequentially. Betrixaban narrowly missed statistical significance in the first cohort. Despite the miss, Portola conducted an exploratory analysis showing that betrixaban met the superiority endpoint in the larger cohorts, including the overall study population (see BioCentury, April 4).
The company also plans to submit an MAA to EMA this year.
Portola gained $0.26 to $19.76 on Tuesday.
Heat Biologics Inc. (NASDAQ:HTBX) and the University of Miami said they are collaborating to develop vaccines targeting the Zika virus and other infectious diseases based on Heat's heat shock 90 kDa protein beta 1 (Hsp90B1; GP96; GRP94) vaccine platform. Heat CEO Jeff Wolf told BioCentury that using GP96 to induce an immune response in the placenta could allow the partners to develop the first vaccine to provide fetal protection against Zika.
The partners seek to genetically modify cells to stimulate release of GP96 in order to activate dendritic cells and induce an immune response, which can be directed against cancer or infectious diseases. In 2013, researchers at the university led by Natasa Strbo showed a GP96 vaccine generated an immune response in the mucosa, and led to a 73% reduction in the risk of viral infection in macaques challenged with simian immunodeficiency virus (SIV). The Journal of Immunology published the results.
Wolf said that basic research for the Zika vaccine will take place at Strbo's lab and Heat will perform translational work.
Heat has formed a subsidiary, Zolovax Inc., to exclusively develop GP96-based vaccines targeting Zika, HIV, and other infectious diseases. Heat has GP96-based vaccines in clinical development for lung and bladder cancer. Wolf declined to disclose milestones for the Zika program.
Heat added $0.04 to $1.34 on Tuesday.
Dipexium Pharmaceuticals Inc. (NASDAQ:DPRX) plummeted $9.95 (78%) to $2.80 after Locilex pexiganan cream 0.8% missed the primary endpoints in the identical Phase III OneStep-1 and OneStep-2 studies to treat mild infections of diabetic foot ulcers. Locilex failed to show superiority to vehicle plus standard wound care at inducing clinical responses.
Locilex also failed to lead to a higher rate of bacterial eradication, missing a secondary endpoint, and did not lead to a "meaningful difference" in wound closure rate, the company said.
Dipexium is evaluating a path forward that may include other indications. Locilex is a 22-amino acid linear peptide that disrupts bacterial cell membrane permeability.
Merck & Co. Inc. (NYSE:MRK), Eli Lilly and Co. (NYSE:LLY) and Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) all reported 3Q16 earnings and corporate updates on Tuesday.
Merck raised its full-year 2016 guidance of adjusted EPS to $3.71-$3.78 from $3.67-$3.77. The company posted 3Q16 adjusted EPS of $1.07 on $10.54 billion in sales, topping consensus estimates of $0.99 per share on sales of $10.18 billion. Merck's 3Q16 earnings report came less than a day after FDA approved Merck's PD-1 inhibitor Keytruda pembrolizumab as a first-line therapy for non-small cell lung cancer (NSCLC) in patients whose tumors express PD-L1 at ≥50%. The drug is also approved to treat melanoma, and has accelerated approval in head and neck cancer (see BioCentury Extra, Oct. 24).
Sales of Keytruda were $356 million during the quarter. On a conference call Tuesday, EVP and President of Merck Research Laboratories Roger Perlmutter said the company anticipates more regulatory submissions this quarter and next year for Keytruda, and said recent data from a Phase III study in urothelial cancer were "registration-worthy" (see BioCentury Extra, Oct. 21).
Along with its earnings, Lilly said the timeline has slowed for an anticipated readout of its Phase III study of anti-calcitonin gene-related peptide (CGRP) mAb galcanezumab (LY2951742) to prevent cluster headaches. The company now expects data in 2018, rather than this year. On a conference call, Lilly Bio-Medicines President and incoming Lilly CEO Dave Ricks said the delay is related to a longer-than-expected amount of time to accrue the necessary cluster headache events in the trial, and added that the company still expects data in 2017 from the candidate's Phase III program to treat migraines. Lilly also still anticipates top-line data this year from its Phase III EXPEDITION3 study of Alzheimer's disease candidate solanezumab (LY2062430), a major catalyst for the company (see BioCentury, Oct. 24).
Vertex said it plans to begin Phase II studies by YE16 of triple-combination regimens to treat cystic fibrosis, including trials involving next-generation CF transmembrane conductance regulator (CFTR) correctors VX-440 and VX-152, each in combination with Kalydeco ivacaftor and tezacaftor (VX-661). It expects data from both studies in 2H17. Vertex also plans to start a Phase I trial this year of another next-generation corrector, VX-659, and hopes to begin clinical studies of a fourth corrector next year. The company reported 3Q16 CF product revenues of $410 million, including $234 million for Orkambi lumacaftor/ivacaftor and $176 million for Kalydeco.
Merck gained $1.20 to $61.95 on Tuesday, while Lilly added $0.18 to $77.75.
Vertex slipped $0.02 to $78.71. It reported earnings after the bell; the stock then added $4.90 to $83.61 in early after-hours trading.
Perceptive Advisors LLC closed its Perceptive Credit Opportunities Fund at $323 million, exceeding its $300 million target. The fund will provide debt financing to companies in the therapeutics, medical device, diagnostics, life science research and healthcare IT sectors, investing $10-$50 million per company. Its LPs include undisclosed endowments, family offices and institutional investors.
Regenerative medicine company Pluristem Therapeutics Inc. (NASDAQ:PSTI; Tel Aviv:PSTI) said China's Innovative Medical Management Co. Ltd. (SZSE:002173) will invest about $30 million in the company through its purchase of 16.9 million shares at $1.77. Innovative Medical will also receive 4.4 million five-year warrants exercisable at $2.50.
Additionally, Innovative Medical will have one seat on Pluristem's board as long as it holds at least 12.5% of the company's issued and outstanding stock.
Pluristem hopes to begin a Phase III trial next year of PLX-PAD cells to treat critical limb ischemia. The therapy consists of allogeneic, placental-derived expanded (PLX) cells.
On Tuesday, Pluristem rose $0.12 to $1.64 on NASDAQ and gained NIS42 to NIS617 in Tel Aviv. The NASDAQ close values the company at $132.4 million based on 80.7 million shares outstanding on Aug. 31.
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