Esperion Therapeutics Inc. (NASDAQ:ESPR) said both doses of its ETC-1002 met the primary endpoint compared to ezetimibe in a Phase IIb study to treat patients with hypercholesterolemia, with or without statin intolerance. Ezetimibe, which inhibits absorption of cholesterol from the intestine, is marketed as Zetia by Merck & Co. Inc. (NYSE:MRK).
ETC-1002, an AMP-activated protein kinase (AMPK) activator and ATP citrate lyase (ACL) inhibitor, led to LDL-cholesterol lowering of 30% at the 180 mg dose (p<0.0001) and 27% at the 120 mg dose (p=0.0008) after 12 weeks vs. 21% with ezetimibe.
The effect was enhanced in patients who received ETC-1002 plus ezetimibe; they experienced LDL-C reductions of 48% at the 180 mg dose (p<0.0001) and 43% at the 120 mg dose (p<0.0001).
Esperion plans to begin Phase III trials by the end of 2015, with regulatory submissions in mid-2018.
The company's shares have gained 51% between its close of $16.17 on Sept. 23 and Wednesday's close of $24.42. In postmarket trading after Esperion announced the Phase IIb data on Wednesday, the stock gained another $8.08 (33%) to $32.50.
HBV play OnCore Biopharma Inc. (Doylestown, Pa.) acquired infectious disease company Enantigen Therapeutics Inc. (Doylestown, Pa.) in an all-cash deal of undisclosed size. OnCore gains two HBV programs, an s-antigen secretion inhibitor and a capsid assembly inhibitor. OnCore CEO Patrick Higgins declined to give the stages of development of the two programs.
OnCore was founded by a group of former Pharmasset Inc. executives, including one credited with inventing Sovaldi sofosbuvir before the biotech was acquired by Gilead Sciences Inc. (NASDAQ:GILD). Last month, OnCore licensed the sangamide-based cyclophilin inhibitor NVP018 from NeuroVive Pharmaceutical AB (SSE:NVP) (see BioCentury, Sept. 15).
OnCore says it intends to create the first oral HBV cure. The company plans to bring multiple combination therapies into the clinic next year. It announced a venture round of undisclosed size in August.
Enanta Pharmaceuticals Inc. (NASDAQ:ENTA) saidNovartis AG (NYSE:NVS; SIX:NOVN) will return rights to the biotech's EDP-239, an HCV non-structural protein 5A (NS5A) inhibitor that is in Phase I testing, as the pharma exits the HCV business.
Novartis acquired exclusive, worldwide rights to EDP-239 in February 2012. Enanta received $34 million up front and an $11 million milestone payment in January 2013.
Enanta's HCV portfolio also includes a cyclophilin inhibitor program and a nucleotide polymerase inhibitor program, both of which are in preclinical development. Enanta was up $0.72 to $40.29 on Wednesday.
Regenerative medicine company Mesoblast Ltd. (ASX:MSB; Pink:MBLTY) said partner JCR Pharmaceuticals Co. Ltd. (Tokyo:4552) is seeking Japanese approval of JR-031 to treat graft-versus-host disease (GvHD) in children and adults. JR-031, which consists of allogeneic adult mesenchymal precursor cells (MPCs) used to expand cord blood by up to 40-fold, has Orphan Drug status in Japan and will undergo Priority Review. If JR-031 is approved, Mesoblast said it would be the first allogeneic cell-based product available in Japan. The Australian company is eligible for regulatory and sales milestones as well as royalties on sales. Mesoblast has worldwide rights to develop JR-031 in all indications except for hematologic indications in Japan.
Mesoblast plans to submit an NDA in the U.S. in 2016 for approval of JR-031 to treat children with steroid-refractory acute GVHD. Its shares closed down down A$0.04 to A$4.28 in Sydney on Wednesday; JCR closed down Y62 to Y2,660.
Zogenix Inc. (NASDAQ:ZGNX) submitted an sNDA to FDA for a capsule formulation of Zohydro ER hydrocodone bitartate designed to deter abuse by injection or nasal administration.
Zohydro, the lone approved hydrocodone-only oral drug, has faced criticism from lawmakers for its lack of abuse deterrence technology. The company expects a decision on the new formulation by FDA in 1Q15. If the sNDA is approved, Zogenix plans to begin marketing the new formulation in place of the currently marketed one beginning in 2Q15.
Zogenix also is developing an abuse-deterrent tablet formulation of hydrocodone designed to maintain the drug's extended release properties when crushed or chewed. The company plans to file a separate NDA for the tablet formulation in 1H16.
Portola Pharmaceuticals Inc. (NASDAQ:PTLA) gained $4.04 (16%) to $29.32 on Wednesday after its andexanet alfa met the primary and secondary endpoints in a Phase III study to reverse the anticoagulant activity of Eliquis apixaban from Bristol-Myers Squibb Co. (NYSE:BMY) and Pfizer Inc. (NYSE:PFE).
Andexanet alfa, a recombinant protein that reverses the anticoagulant activity of Factor Xa inhibitors, has breakthrough therapy designation from FDA. In the first of two Phase III ANNEXA-A studies, data showed that 400 mg of andexanet alfa immediately and significantly reversed the anticoagulation activity of Eliquis.
Portola will present full trial data at the American Heart Association meeting in November. Portola expects to submit a BLA to FDA in late 2015 seeking accelerated approval.
The company also proposed a $160 million follow-on underwritten by Morgan Stanley and Credit Suisse.
Ardelyx Inc. (NASDAQ:ARDX) jumped $4.79 (34%) to $19 on Wednesday after 50 mg tenapanor (RDX5791/AZD1722) met the primary endpoint of increasing complete spontaneous bowel movement (CSBM) responder rate in a Phase IIb trial to treat constipation predominant irritable bowel syndrome (IBS-C).
The small molecule inhibitor of non-absorbed solute carrier family 9 sodium hydrogen exchanger member 3 (SLC9A3; NHE3) resulted in a 60.7% CSBM responder rate in patients receiving 50 mg tenapanor twice daily vs. 33.7% in those receiving placebo over 12 weeks. Two other dosages tested -- 5 mg and 20 mg -- missed the primary endpoint.
Ardelyx and partner AstraZeneca plc (LSE:AZN; NYSE:AZN) are also investigating the compound in a Phase IIb trial to treat hyperphosphatemia in end-stage renal disease (ESRD) patients on hemodialysis, with data expected in 1H15; and a Phase IIa trial to treat chronic kidney disease (CKD) patients with Type II diabetes, with data expected in 2H15. AZ has exclusive, worldwide rights to tenapanor under an October 2012 deal covering Ardelyx's NHE3 inhibitor programs.
Erytech Pharma S.A. (Euronext:ERYP) climbed EUR 3.33 (13%) to EUR 29.50 on Wednesday after it said Graspa met the co-primary endpoints in the GRASPIVOTALL Phase III trial to treat children and adults with relapsed or refractory acute lymphoblastic leukemia (ALL).
The 80 patient study compared the L-asparaginase encapsulated in red blood cells to non-encapsulated L-asparaginase, in combination with standard chemotherapy. The co-primary endpoints were a statistically significant reduction in allergic reactions and a statistically significant increase in the duration of circulating asparaginase activity.
None of the Graspa patients experienced an allergic reaction vs. 42% of those treated with non-encapsulated L-asparaginase. In Graspa-treated patients, asparaginase activity was above 100 IU/l for an average of 20.5 days when given one to two injections in the first month, compared to 9.2 days in subjects given up to eight injections of the control.
Erytech plans to submit an MAA to EMA in 1H15.
LSP-Life Sciences Partners has raised the first EUR 80 million ($101.4 million) of a planned EUR 150 million fund. The firm said it intends to invest its new Fund 5 in 10-15 private companies with "innovative medicines and medical technologies," primarily in Europe but also in the U.S.
Both new and existing institutional and private investors participated in the new fund. LSP said an unidentified large pharma invested in Fund 5, as well as its predecessor Fund 4, which closed in 2009 at just above EUR 90 million. Pfizer Inc. (NYSE:PFE) and GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) have previously invested in LSP's funds (see BioCentury, March 31).
LSP said it has generated 15 exits in the last 22 months, and has exited 55 of the more than 75 investments in its history. Among recent exits are Okairos AG (Basel, Switzerland), acquired by GlaxoSmithKline for EUR 250 million in 2013; and Activaero GmbH (Gemuenden, Germany), acquired by Vectura Group plc (LSE:VEC) for EUR 130 million in March. On the IPO front, portfolio company Celladon Corp. (NASDAQ:CLDN) went public in January. The firm claims an average annual internal rate of return (IRR) of 25% over 25 years.
Shares of several Ebola plays settled back Wednesday from double-digit gains in after-hours trading on Tuesday's news the CDC and the Texas Department of State Health Services announced the first U.S. diagnosis of an Ebola infection.
The agencies said a patient in a Texas hospital was placed into isolation at Texas Health Presbyterian Hospital after admission Sunday. The patient traveled from Liberia to the U.S. on Sept. 20, but did not exhibit symptoms until Sept. 24.
The media relations office of Texas health services did not respond to questions about how the patient was being treated.
In Wednesday trading, Tekmira Pharmaceuticals Corp. (NASDAQ:TKMR) still finished up 18%, gaining $3.85 to $24.99. NewLink Genetics Corp. (NASDAQ:NLNK) was up $1.53 to $22.95. BioCryst Pharmaceuticals Inc. (NASDAQ:BCRX) gained $0.37 to $10.15. Sarepta Therapeutics Inc. (NASDAQ:SRPT) was up $0.78 to $21.88.
VBL Therapeutics Ltd. (NASDAQ:VBLT) raised $40 million through the sale of 6.7 million shares at $6 in an IPO underwritten by Deutsche Bank, JMP and Oppenheimer. This is the company's second attempt at an IPO. Its August IPO was terminated by the underwriters when an investor did not make payments for shares it had agreed to purchase. At that time, the company was offering 5.4 million shares at $12 (see BioCentury Extra, August 8).
In 1H15, VBL expects to start Phase III testing of its VB-111 gene-based dual anti-angiogenic and vascular disruptive agent (VDA) to treat recurrent glioblastoma.
FibroGen Inc. (San Francisco, Calif.) filed to raise up to $125 million in an IPO on NASDAQ underwritten by Goldman, Sachs; Citigroup; Leerink; RBC; Stifel; and William Blair.
FibroGen's lead compound is roxadustat (FG-4592, ASP1517), a small molecule inhibitor of hypoxia-inducible factor prolyl hydroxylase (HIF-PH; EGLN) in Phase III testing to treat anemia in chronic kidney disease. Astellas has rights to roxadustat from FibroGen in Japan, Europe, the Commonwealth of Independent States (CIS), the Middle East and South Africa. FibroGen and AstraZeneca partnered to develop and commercialize roxadustat worldwide, excluding the countries in which Astellas already has rights.
FibroGen is also developing FG-3019, a human mAb against connective tissue growth factor (CTGF) in Phase II testing to treat idiopathic pulmonary fibrosis (IPF), pancreatic cancer and liver fibrosis.
S1 Biopharma Inc. (Jersey City, N.J.) proposed to raise up to $40.3 million in an IPO on NASDAQ underwritten by MLV. The biotech's lead product is lorexys, an oral fixed-dose enteric formulation of bupropion and trazodone to treat hypoactive sexual desire disorder (HSDD) in women. The company expects to begin Phase IIb testing next year.
NantHealth, a subsidiary of Patrick Soon-Shiong's NantWorks LLC (Los Angeles, Calif.), raised $250 million from the Kuwait Investment Authority (KIA) in the final close of its series B, bringing to $320 million the total raised in the round.
Patrick Soon-Shiong was the executive chairman and CEO of Abraxis Bioscience Inc., which was acquired by Celgene Corp. (NASDAQ:CELG) in 2010.
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