Amgen Inc. (NASDAQ:AMGN) submitted a BLA to FDA for evolocumab (AMG 145), a human mAb against proprotein convertase subtilisin/kexin type 9 (PCSK9), to treat high cholesterol. The application -- the first in the U.S. for a PCSK9 program -- includes data from 10 Phase III trials. Separately, Amgen said evolocumab plus a statin met the co-primary endpoints vs. placebo in the Japanese Phase III YUKAWA-2 trial, which enrolled 404 patients with high cardiovascular risk and high cholesterol. Amgen said it plans to submit a regulatory application in the EU this quarter, with a submission in Japan slated for next year.
Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) and partner Sanofi (Euronext:SAN; NYSE:SNY) are not far behind Amgen in the PCSK9 race, with plans to submit a BLA to FDA for alirocumab by year end. The partners plan to use a Priority Review voucher for the human mAb targeting PCSK9, which could help them close the gap with Amgen (see BioCentury, Aug. 4).
Amgen was up $0.86 to $138.69 on Thursday. Regeneron was off $3.09 to $346.92.
The U.K.'s NICE issued draft guidance concerning the cost effectiveness of Type II diabetes drug Jardiance empagliflozin (BI-10773) from partners Boehringer Ingelheim GmbH (Ingelheim, Germany) and Eli Lilly and Co. (NYSE:LLY).
NICE said Boehringer's economic model for the drug's cost effectiveness is "inherently flawed," preventing NICE from comparing it with recommended treatments. NICE asked Boehringer to deliver a revised estimation in advance of a second appraisal committee meeting Sept. 24. The committee estimated the annual cost of the drug at L470.30 ($779.29). NICE also requested revised sensitivity analyses.
The European Commission approved the oral, once-daily sodium-glucose co-transporter 2 (SGLT2) inhibitor in May. Jardiance was approved in the U.S earlier this month.
Comments on NICE's draft guidance are due Sept. 18.
Luye Pharma Group Ltd. (HKSE:2186) will acquire a 58% stake in Beijing Jialin Pharmaceutical Co. Ltd. from Beijing Jialin shareholders Mylin Holding Group Co. Ltd. (Beijing, China) and Beijing CITIC in a tranched RMB3.7 billion ($599 million) cash deal. Mylin will ask the other Beijing Jialin shareholders to sell Luye their remaining 42% interest.
Beijing Jialin develops and markets pharmaceuticals in China for cardiovascular diseases and cancer. The company had 2013 revenues of RMB1.1 billion ($185 million). Luye said the deal will supplement its existing cardiovascular product portfolio and sales and marketing coverage. Luye will hold a special general meeting to seek shareholder approval.
Luye develops and commercializes small molecules, biologics and traditional Chinese medicine, primarily in China. The company raised HK$4 billion ($509.8 million) in a Hong Kong IPO last month (see BioCentury Extra, July 9).
BTG plc (LSE:BTG) and SciClone Pharmaceuticals Inc. (NASDAQ:SCLN) said the China Food and Drug Administration (CFDA) approved BTG's DC Bead to treat patients with malignant hypervascularized tumors like hepatocellular carcinoma (HCC). DC Bead is a drug-eluting bead for controlled release of doxorubicin or irinotecan. SciClone has exclusive Chinese commercialization rights to the product.
BTG was off 16p to 645p on Thursday. SciClone was off $0.16 to $6.63 on the day.
Chugai Pharmaceutical Co. Ltd. (Tokyo:4519) said it and majority owner Roche (SIX:ROG; OTCQX:RHHBY) amended an agreement covering the geographic regions and timing of Roche's first refusal rights to Chugai compounds. Under the amended deal, Roche has worldwide first refusal rights to Chugai compounds, excluding Japan, South Korea and Taiwan. The pharma also will have first refusal rights to Chugai compounds at early proof of concept. The companies did not change Chugai's right of first refusal to Roche's products in Japan.
As of June 30, Roche held a 61.5% stake in Chugai.
NHS England said it will provide the Cancer Drugs Fund (CDF) with an additional L160 million ($265.1 million) over two years. The fund is intended to give patients access to drugs that would not otherwise be available on the NHS.
CDF will also now re-evaluate and remove coverage of the least effective cancer drugs. CDF said drugs it deems excessively expensive could be removed from the national list of covered treatments unless their prices are adjusted. The list includes drugs appraised by the U.K.'s NICE but not recommended based on cost-effectiveness, as well as drugs not yet appraised by NICE.
CDF will not re-evaluate drugs that are the only proven treatments for particular cancers. CDF also plans to conduct a confidential analysis of drug costs and patient benefits.
NHS's bump will expand the fund's annual budget by 40% to L280 million ($464 million). Launched in 2010, CDF is funded until 2016.
The U.S. District Court for the District of Columbia upheld an interpretive rule allowing discounts for Orphan drugs under Medicare's 340B program when used for non-Orphan indications. The Pharmaceutical Research and Manufacturers of America (PhRMA) had asked the court to vacate the rule, which was issued in July by HHS's Health Resources and Services Administration (HRSA).
The 340B program requires manufacturers to deeply discount outpatient drugs to hospitals and clinics that provide healthcare for low income and other special populations. In 2010, Congress amended 340B to exclude Orphan drugs from discounting, but the law was unclear on whether the exclusion applied when the drugs were used for non-Orphan indications.
The Pharmaceutical Research and Manufacturers of America and Biotechnology Industry Organization voiced concerns about FDA's June draft guidance on how companies can distribute journal articles that discuss new risk information for approved products.
Both stressed manufacturers' First Amendment rights to distribute truthful, non-misleading information. PhRMA urged FDA to allow dissemination of new information about approved drugs' efficacy as well as risk, using comparable standards of substantiation for both.
BIO requested clarifications of procedures related to verbal statements that may contradict product labels, as well as financial disclosures. BIO also asked FDA to clarify recommendations on appropriate data sources. BIO requested that open-label extensions of clinical trials be considered acceptable sources of data, and wanted to know what FDA considers a pharmacoepidemiologic study.
Comments on the draft guidance were due Monday.
Repros Therapeutics Inc. (NASDAQ:RPRX) jumped $3.37 (19%) to $21.58 on Thursday after reporting data from the Phase III ZA-305 trial of Androxal enclomiphene to treat secondary hypogonadism. In the trial, 65.9% of patients receiving Androxal achieved a 24-hour average testosterone level in the normal range with a mean sperm concentration of 10 million/mL or greater -- a co-primary endpoint -- vs. 33.3% for AndroGel 1.62% testosterone gel from AbbVie Inc. (NYSE:ABBV) (p=0.0047) and 7.3% for placebo (p<0.0001).
Androxal led to a 14% increase in mean sperm concentration from baseline to week 16 -- also a co-primary endpoint. The increase was non-significant from baseline, but was significant compared to a 30% reduction in sperm concentration at week 16 for AndroGel (p=0.0004) and an increase of 7% for placebo (p=0.2042).
By year end, Repros plans to submit an NDA to FDA for Androxal in the indication. The compound is a trans-isomer of clomiphene citrate.
The Wellcome Trust, the Medical Research Council and the U.K.'s Department for International Development provided a L2.8 million ($4.6 million) grant to fund trials of an Ebola vaccine candidate from GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) and NIH's National Institute of Allergy and Infectious Diseases (NIAID). The candidate is based on chimp adenovirus type 3 (ChAd3), an attenuated strain of chimpanzee cold virus.
Trials could begin next month if the candidate receives ethical and regulatory approvals. Studies will take place in the U.S., U.K., and West Africa. GSK will simultaneously manufacture 10,000 additional doses for the World Health Organization to create an emergency immunization program.
Separately, the World Health Organization (WHO) released a roadmap for international response operations including fulfilling the needs of treatment and management centers, social mobilization and safe burials. It also will map disease hotspots and present epidemiological data as the outbreak evolves.
WHO endorsed investigational Ebola compounds that have not yet been evaluated for safety and efficacy in humans (see BioCentury Extra, Aug 12).
OncoMed Pharmaceuticals Inc. (NASDAQ:OMED) said FDA lifted a partial hold on the company's Phase Ib trials of vantictumab (OMP-18R5), a human HuCAL mAb targeting the Wnt signaling pathway. The agency placed the hold after the company halted the trial due to mild to moderate bone-related adverse events. According to OncoMed, FDA lifted the partial hold following a review of safety and efficacy data and revised study protocols submitted by the company with modified dosing regimens, risk mitigation measures, and modified enrollment criteria. OncoMed said FDA's partial hold of Fzd8-Fc (OMP-54F28), which also targets the Wnt pathway, is still in place.
OncoMed closed up $1.95 to $21.58 on Thursday on the news. Despite the move, the company is still below its close of $24.90 on June 12, before it halted enrollment and dosing in trials of vantictumab and Fzd8-Fc (see BioCentury Extra, June 13).
OncoMed and Bayer AG (Xetra:BAYN) are partnered to discover and develop antibodies, proteins and small molecules targeting the Wnt pathway for cancer. Bayer has an option to exclusively license vantictumab and Fzd8-Fc at any point up to the completion of Phase I testing under the deal.
Orexo AB (SSE:ORX; OTCQX:ORXOY) is raising an undisclosed amount in a private placement with Swedish and international institutional and strategic investors. Danske Bank is the sole lead manager and bookrunner.
Orexo said the placement will include 1.1 million treasury shares, which alone would raise about SEK160.6 million ($23.4 million) based on the company's Thursday close of SEK143.25, before the offering was announced. The pricing and number of shares to be issued are expected to be determined before trading on Friday. Orexo's Zubsolv buprenorphine/naloxone, a sublingual tablet formulation of buprenorphine and naloxone, is marketed in the U.S. to treat opioid dependence.
Orexo was off SEK4.25 on Thursday.
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