Intercept Pharmaceuticals Inc. (NASDAQ:ICPT) gained $49.31 (29%) to $220 in after hours-trading on Thursday after it said FDA granted breakthrough therapy designation to its OCA obeticholic acid (DSP-1747) to treat non-alcoholic steatohepatitis (NASH) with liver fibrosis.
Intercept said the designation was based on data from two Phase II trials of the oral farnesoid X receptor (FXR; NR1H4) agonist in NASH and non-alcoholic fatty liver disease (NAFLD). In the 283-patient FLINT trial, 45% of NASH patients given OCA had improved liver histology at 72 weeks vs. 21% of patients given placebo (p=0.0002). Intercept said the second trial, in 64 NAFLD patients, assessed the effect of OCA treatment on insulin sensitivity (see BioCentury Extra, Nov. 7, 2014).
The company said the designation will help speed the design of its Phase III program, which it expects to start this half (see BioCentury Extra, Jan. 13).
Intercept gained $1.13 to $170.69 before the news was announced after market close.
Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) said principal founder Leonard Bell will retire as CEO, effective April 1, while remaining chairman.
COO David Hallal, who joined Alexion in 2006, will succeed Bell as CEO. Hallal took a board seat and was promoted to COO from EVP and chief commercial officer last September. He previously held commercial roles at OSI Eyetech Inc., Biogen Idec Inc. (NASDAQ:BIIB) and Amgen Inc. (NASDAQ:AMGN).
Bell, who has been CEO since Alexion's inception in 1992, took over as chairman in October following the passing of Max Link (see BioCentury Extra, Oct. 9).
Separately, Alexion reported 4Q14 non-GAAP EPS of $1.30, up 49% from $0.87 in the prior year's quarter and beating the Street estimate by a penny. 4Q sales of Soliris eculizumab rose 36% to $599 million. The humanized mAb targeting complement 5 (C5) is approved for paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). The company said it expects non-GAAP EPS of $5.60-$5.80 in 2015, with sales of $2.55-$2.6 billion. The Street was expecting 2015 EPS of $5.91 on sales of $2.7 billion.
EVP and CFO Vikas Sinha noted during Thursday's earnings call that Alexion's guidance suffered from negative effects of foreign exchange rates, but that the company expects its 2015 pre-tax operating margins to be similar to its 2014 margins. The company also revised its non-GAAP tax rate to 7-9%, lower than its previous forecast of 13-15%.
Alexion picked up $10.27 to $188.05 on Thursday.
Biogen Idec Inc. (NASDAQ:BIIB) started a collaboration with Fondazione Telethon (Milano, Italy) and Ospedale San Raffaele (Milano, Italy) to develop gene therapies to treat hemophilia A and B.
Biogen will pay the San Raffaele-Telethon Institute for Gene Therapy (TIGET) $5 million up front and will receive options to license global development and commercialization rights to two hemophilia programs, one in hemophilia A and one in hemophilia B, after clinical proof-of-concept trials. TIGET is eligible for undisclosed development milestones. Biogen will fund the research.
Biogen spokesperson Lee-Ann Murphy said it is too soon to say when the programs will enter the clinic.
The programs use TIGET's lentiviral technology, which is administered directly into the body and specifically targets liver cells. Murphy said Biogen will use the technology to make the body's liver cells produce coagulation factors that are missing in the diseases. Hemophilia A results from a deficiency in Factor VIII, hemophilia B from deficiency in Factor IX.
Murphy said lentiviral gene transfer has two advantages over adeno-associated viral (AAV) vectors: the vector integrates into the host genome, offering the possibility of longer-lasting gene expression; and the vector can carry large cargo, which she said is especially important for delivering Factor VIII.
Baxter International Inc. (NYSE:BAX), uniQure N.V. (NASDAQ:QURE) and Spark Therapeutics Inc. (Philadelphia, Pa.) all have AAV vector-based gene therapies encoding the Factor IX gene. Baxter's BAX 335, uniQure's AMT-060 and Spark's AAV8-hFIX19 are all in or entering Phase I/II testing to treat hemophilia B.
Recombinant blood factor therapies are the SOC in hemophilia, but some patients develop neutralizing antibodies that inhibit the exogenous factors. Gene therapies offer a potential solution (see BioCentury, Sept. 29, 2014).
Last year, FDA approved two long-acting factors from Biogen to treat hemophilia: Alprolix for hemophilia B, and rFVIIIFc for hemophilia A. Alprolix and rFVIIFc are recombinant fusion proteins consisting of human coagulation Factor IX and Factor VIII, respectively, attached to the Fc domain of human IgG1. Biogen is partnered with Swedish Orphan Biovitrum AB (SSE:SOBI) for both.
Biogen gained $2.07 to $353.25 on Thursday.
Symphogen A/S (Copenhagen, Denmark) regained exclusive, worldwide rights to Sym004 from Merck KGaA (Xetra:MRK), unwinding a 2012 deal. Both Merck and Symphogen said the decision was not related to new safety or efficacy results related to Sym004.
The combination of two anti-EGFR mAbs against two distinct and non-overlapping epitopes in the extracellular domain III of EGFR is in a Phase IIb trial to treat metastatic colorectal cancer (mCRC). It is also in a Phase Ib trial to treat non-small cell lung cancer (NSCLC) and a Japanese Phase I trial in patients with solid tumors. Symphogen will continue the Phase IIb trial in mCRC but will not pursue the Phase Ib trial in NSCLC. Merck will complete the Japanese Phase I trial, after which it will transfer data to Symphogen.
Symphogen said it has more than EUR 70 million ($78.7 million) to pursue its development programs. Under the deal with Merck, Symphogen received EUR 20 million ($25.2 million) up front and was eligible for up to EUR 475 million ($597.4 million) in milestones, plus royalties. Symphogen said it received three undisclosed milestone payments from Merck last year. Merck and Symphogen both declined to disclose details (see BioCentury Extra, Sept. 6, 2012).
FDA approved an sNDA for Imbruvica ibrutinib from Pharmacyclics Inc. (NASDAQ:PCYC) and the Janssen Biotech Inc. unit of Johnson & Johnson (NYSE:JNJ) to treat Waldenstrom's macroglobulinemia, well ahead of its April 17 PDUFA date. The drug had breakthrough designation from FDA. Pharmacyclics said Imbruvica is the only FDA-approved treatment for Waldenstrom's, a form of B cell lymphoma.
The agency granted Imbruvica full approval last July as a first-line therapy to treat chronic lymphocytic leukemia (CLL) in patients with 17p deletion and to treat CLL in patients who have received one prior therapy. It received accelerated approval in November 2013 to treat mantle cell lymphoma (MCL) (see BioCentury Extra, July 28, 2014).
Pharmacyclics said this month it expects Imbruvica's U.S. sales to double in 2015, reaching $1 billion on increased sales in existing indications plus the "imminent" approval in Waldenstrom's (see BioCentury Extra, Jan. 13).
Pharmacyclics and Janssen share U.S. rights to the Bruton's tyrosine kinase (Btk) inhibitor that covalently binds to cysteine residue 481, while Janssen has ex-U.S. rights under a 2011 deal.
Pharmacyclics gained $2.06 to $167.47 on Thursday.
Biotechs continued to raise money as a strong month of fundraising nears its end. Since market close on Wednesday, Omeros Corp. (NASDAQ:OMER), Neovasc Inc. (TSX:NVC; NASDAQ:NVCN), Mirati Therapeutics Inc. (NASDAQ:MRTX) and Xenoport Inc. (NASDAQ:XNPT) have raised more than $280 million in follow-on offerings and a note deal.
Ophthalmic, inflammation and CNS play Omeros raised $75 million through the sale 3 million shares at $20.03 and 749,250 warrants at $20.02. Each seven-year warrant is exercisable at $0.01 per share. Wedbush PacGrow Life Sciences; Needham; WBB Securities; Maxim Group; and MLV were underwriters. Omeros gained $3.41 (17%) to $23.44 on Thursday after proposing the offering after market close Wednesday.
Cardiovascular device developer Neovasc raised $63.6 million through the sale of 8.8 million shares at $7.19. Shareholders including directors, officers, and employees sold another 1.7 million shares. Leerink; Canaccord Genuity; JMP Securities; and Ladenburg Thalmann were underwriters. Neovasc proposed the offering after market close on Monday, when its shares were at $8.71. The company added $1.81 (25%) to $9 on NASDAQ and $2.62 (30%) to $11.49 in Toronto on Thursday.
Oncology drug developer Mirati raised $45 million through the sale of 2.3 million shares at $20. Underwriters included Citigroup; Jefferies; and Leerink. Mirati proposed the offering after market Wednesday, and gained $0.83 to $21.29 on Thursday.
CNS play Xenoport raised $100 million through the sale of convertible senior notes, which bear 2.5% percent interest, mature in 2022 and initially convert at $10.72. Xenoport gained $0.14 to $8.89 on Thursday after proposing the note deal after market Wednesday.
Diagnostics company Atlas Genetics Ltd. (Trowbridge, U.K.) raised $20 million in a series C round from new investor RMI Partners, a subsidiary of Rusnano, and existing investors Novartis Venture Funds; Consort Medical plc; Johnson & Johnson Development Corp. (JJDC), the venture arm of Johnson & Johnson (NYSE:JNJ); LSP; BB Biotech Ventures; and South West Ventures Fund.
The company expects to launch its Atlas io system in Europe this year as a diagnostic for chlamydia. The point-of-care diagnostic detects nucleic acids present in a variety of bacterial infections including chlamydia and methicillin-resistant Staphylococcus aureus (MRSA) infection. The company is planning U.S. clinical trials of the system.
Atlas received the final tranche of its L16.9 million ($28.5 million) series B round in April 2014.
Sen. Lamar Alexander (R-Tenn.), chairman of the U.S. Senate Health, Education, Labor and Pensions (HELP) Committee, and Sen. Richard Burr (R-N.C.) released the Innovation for Healthier Americans report on Thursday. The report is intended to identify opportunities for reform at NIH and FDA. Its publication marked the start of a Senate HELP Committee initiative, led by Alexander and ranking Democrat Sen. Patty Murray (D-Wash.), to examine the development and review process for drugs and medical devices and introduce legislation to address inefficiencies. Comments are due Feb. 23.
The report followed Tuesday's release of a discussion draft of 21st Century Cures legislation by the U.S. House Energy & Commerce Committee proposing changes to FDA's regulation of drugs, medical devices, clinical research and reimbursement (see BioCentury Extra, Jan. 27).
Alexander and Burr called for feedback on how to facilitate adoption of innovative clinical trial designs that would speed development and reduce costs.
The report questioned whether FDA should update its standards for safety and efficacy and whether FDA is adequately prepared to review a wide range of new technologies. It highlighted initiatives at FDA and NIH dedicated to accelerating the medical product review process and called for feedback on identifying measurable outcomes from these initiatives.
The report also asks whether there are opportunities for FDA to collaborate with international regulatory partners and expedite approvals for products that have been reviewed and approved abroad.
House Energy & Commerce Committee Chairman Rep. Fred Upton (R-Mich.) issued a statement expressing support for the report.
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