Gilead Sciences Inc. (NASDAQ:GILD) reported 2Q14 financial results that beat the Street, including $3.5 billion in sales of HCV drug Sovaldi sofosbuvir. The Street was expecting $2.5-$2.6 billion in Sovaldi sales in the quarter. About $3 billion of Sovaldi's 2Q14 sales were in the U.S., where 65% of its usage is in patients with HCV genotype 1 infection. According to Gilead, Sovaldi has been prescribed for more than 80,000 patients in the U.S. and Europe through June 30 since its launch; the company launched Sovaldi in the U.S. in December and in Europe earlier this year.
Gilead reported 2Q14 non-GAAP diluted EPS of $2.36, beating the Street's estimate of $1.79 and up from $0.50 in 2Q13. Revenues for the quarter were $6.5 billion, beating the Street's $5.9 billion estimate and up from $2.8 billion in 2Q13. Gilead also for the first time provided 2014 net product sales guidance that accounts for Sovaldi sales. The company said it expects full-year sales of $21-$23 billion. Previously, the company guided to full-year sales of $11.3-$11.5 billion, not including Sovaldi sales.
During the quarter, Gilead also repurchased about 15.2 million shares for about $1.2 billion. The company has about $1.7 billion remaining under an existing repurchase program. A new $5 billion repurchase program is slated to start once the existing program is completed, which is expected in September.
Gilead was up $1.01 to $90.34 on Wednesday. The company released its earnings after market close. Separately, FDA approved Gilead's Zydelig idelalisib for three blood cancers during market hours.
Biogen Idec Inc. (NASDAQ:BIIB) gained $33.93 (11%) to $337.60 on Wednesday after reporting 2Q14 financial results that beat consensus EPS and sales estimates. The move translates to a gain of $8 billion in market cap for a closing valuation of $79.7 billion. Non-GAAP diluted EPS grew 52% to $3.49 from 2Q13 and beat by $0.66 the Street's $2.83 estimate. Revenues in the quarter rose 40% to $2.4 billion from $1.7 billion in 2Q13 and beat the Street's estimate of $2.2 billion.
Revenues of Tecfidera dimethyl fumarate skyrocketed over 260% to $700.4 million in 2Q14, including $585 million in U.S. sales and $115 million in ex-U.S. sales, compared to the same quarter last year. The company launched the multiple sclerosis drug in the U.S. in April 2013, and the European Commission approved the oral dimethyl fumarate that activates the NF-E2-related factor 2 (Nrf2) pathway in February. Biogen Idec also raised its guidance and now expects 2014 non-GAAP diluted EPS of $12.90-$13.10 on revenue growth of 38-41%. In April, Biogen said it expected 2014 EPS of $11.35-$11.45 on revenue growth of 26-28%. The company reported 2013 revenues of $6.9 billion.
On the pipeline front, Biogen Idec said the EC approved Plegridy peginterferon beta-1a (BIIB017) to treat relapsing-remitting MS (RRMS). The company plans to launch the product "in the coming weeks." A BLA for the pegylated interferon (IFN) beta-1a is under FDA review for relapsing forms of MS, with a decision expected this half. Biogen also announced that Japan's Ministry of Health, Labor and Welfare (MHLW) approved Alprolix rFIXFc for hemophilia B. FDA and Health Canada approved the recombinant fusion protein consisting of human coagulation Factor IX attached to the Fc domain of human IgG1 in March. Biogen is partnered with Swedish Orphan Biovitrum AB (SSE:SOBI) for Alprolix under an amended 2006 deal. On Wednesday, Swedish Orphan was off SEK0.40 to SEK84.30.
GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) reported 2Q14 financial results on Wednesday that missed consensus EPS and sales estimates; the pharma also lowered its 2014 guidance. The pharma's 2Q14 core EPS dropped 12% to 19.1p and missed the Street's 21.35p estimate, while sales in the quarter were off 4% to L5.6 billion ($9.5 billion), missing the Street's L5.8 billion estimate. Pharmaceutical sales were down 6% to L3.8 billion ($6.4 billion) compared to 2Q13.
Pharmaceutical and vaccines sales growth in emerging markets was adversely affected by about four percentage points, the pharma said, due to the ongoing investigation in China regarding allegations of bribery and tax crimes. Total sales in China were down 25% in the quarter to L129 million ($219.6 million) (see BioCentury Extra, July 11, 2013).
GSK disclosed that it has started a process to divest U.S. and European products in its established products portfolio with total sales of around L1 billion ($1.7 billion). The pharma expects to close a deal this year.
GSK also lowered its 2014 guidance and now expects core EPS to be "broadly similar" to core EPS of 108.4p in 2013 at constant exchange rates and excluding any divestments. In April, GSK said it expected 2014 core EPS growth guidance of 4-8%. All percent changes assume constant currency.
GSK was off 73.50p (5%) to 1,481p on Wednesday. The pharma was off $3.18 (6%) to $50.04 on the NYSE.
Sofinnova Ventures raised $500 million for Sofinnova Venture Partners IX, its ninth fund and second life sciences-only fund. The firm was targeting $425 million for the fund, which was oversubscribed. Sofinnova plans to invest the fund in 20-22 therapeutics companies primarily in the U.S. but also in Europe. The firm said it will invest the fund in companies developing specialty pharmaceuticals and Orphan disease products.
Sofinnova raised $440 million for its eighth fund and first life sciences-only fund in 2011. The firm's portfolio includes biosimilar play Coherus BioSciences Inc. (Redwood City, Calif.), gene therapy company Spark Therapeutics LLC (Philadelphia, Pa.) and cancer company NuCana BioMed Ltd. (Edinburgh, U.K.).
Puma Biotechnology Inc. (NYSE:PBYI) jumped $174.40 (295%) to $233.43 on Wednesday after the company reported top-line data for neratinib (PB272) as adjuvant treatment of HER2-positive breast cancer late Tuesday and separately said it will be responsible for more trial costs for the product but will owe lower royalty rates to partner Pfizer Inc. (NYSE:PFE). Puma's Wednesday move translates to a gain in market cap of $5.3 billion for a closing valuation of $7 billion. With the move, Puma is also now above its close of $139.92 on Jan. 21, before the company slid after reporting detailed efficacy and safety data for the oral inhibitor of HER1, HER2 and HER4 kinases from the adaptive Phase II I-SPY 2 trial evaluating neoadjuvant breast cancer therapies (see BioCentury Extra, July 22).
Remiges Ventures has raised $70 million of the targeted $150 million in its Remiges BioPharma Fund L.P., according to an SEC filing. The firm will invest in early stage companies in the U.S. and Europe that are developing therapeutics "with a definable commercial opportunity in the U.S. market." Taiho Pharmaceutical Co. Ltd. (Tokyo, Japan) and Sumitomo Dainippon Pharma Co. Ltd. (Tokyo:4506) are each investing $30 million in the fund. Remiges, which could not be reached for comment, has offices in Cambridge, Mass., and Tokyo.
Taro Inaba is managing director at Remiges, and Walter Olesiak is a partner. Inaba was formerly president and CEO of Mitsui Ventures, and Olesiak was an investment partner at Mitsui.
La Jolla Pharmaceutical Co. (NASDAQ:LJPC) raised $50.4 million through the sale of 4.8 million shares at $10.50 in a follow-on underwritten by Jefferies; Chardan Capital Markets; H.C. Wainwright; LifeSci Capital; and Noble Financial Group. La Jolla proposed the follow-on late Tuesday, when its share price was $10.95.
The company's lead product is GCS-100, a galectin-3 (LGALS3) inhibitor in Phase II testing for chronic kidney disease (CKD). La Jolla gained $7.06 (65%) to $17.96 on March 11 after reporting data from a Phase IIa trial showing GCS-100 met the primary endpoint of improving estimated glomerular filtration rate vs. placebo in CKD patients.
La Jolla was up $0.51 to $11.46 on Wednesday.
Biomarker discovery company KineMed Inc. (Emeryville, Calif.) withdrew its IPO on NASDAQ, citing "market conditions." The company declined to comment on future financing plans. Last month, KineMed amended its IPO to sell 4.5 million shares at $6.50-$7.50. At the $7 midpoint, the company would have raised $31.5 million and been valued at $113.8 million. In January, the company filed to raise up to $50.9 million, with H.C. Wainwright; MKM Dillon; and Dawson James Securities as underwriters. KineMed replaced the underwriters with Feltl; MLV; and Laidlaw. In 1Q14, the company reported $1.4 million in revenues and had a three-month operating loss of $1.7 million. As of March 31, KineMed had $7.3 million in cash.
KineMed is using its biomarker platform technology for drug development, medical diagnostics and to pair drugs with biomarker tests. The company has signed about 100 biomarker collaborations with companies including Pfizer Inc. (NYSE:PFE) and AstraZeneca plc (LSE:AZN; NYSE:AZN), as well as collaborations with not-for-profits and government agencies. KineMed had 2013 revenues of $6.9 million.
FDA approved Zydelig idelalisib from Gilead Sciences Inc. (NASDAQ:GILD) for three blood cancers on Wednesday. The agency granted full approval to Zydelig in combination with rituximab to treat relapsed chronic lymphocytic leukemia (CLL), for which the compound has breakthrough therapy designation. FDA granted accelerated approval to the small molecule inhibitor of phosphoinositide 3-kinase (PI3K) delta as monotherapy to treat relapsed follicular B cell non-Hodgkin's lymphoma (FL) and relapsed small lymphocytic lymphoma (SLL) -- both types of indolent NHL. The accelerated approvals are based on the single-arm Phase II Study 101-09, where Zydelig led to an overall response rate (ORR) of 54% in 72 FL patients and 58% in 26 SLL patients (see BioCentury Extra, Dec. 9, 2013).
Zydelig's label includes a boxed warning on the risk of fatal and serious toxicities, including liver toxicity, diarrhea, colon and lung inflammation and intestinal perforation. The drug was also approved with a REMS that includes a communication plan. Gilead said it plans to launch the drug "as soon as possible" with a wholesale acquisition cost of $7,200 for a one-month supply. According to Zydelig's label, in clinical trials the median duration of exposure to Zydelig was five months in CLL patients and 6.1 months in indolent NHL patients.
Gilead was up $1.01 to $90.34 on Wednesday. The Zydelig approval news came during market hours. Separately, the company reported 2Q14 earnings after market close.
Biogen Idec Inc. (NASDAQ:BIIB) and the Genentech Inc. unit of Roche (SIX:ROG; OTCQX:RHHBY) co-market rituximab as Rituxan in the U.S. Roche markets it as MabThera elsewhere.
FDA approved an NDA for Targiniq ER oxycodone/naloxone from Purdue Pharma L.P. (Stamford, Conn.) to treat pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate. Targiniq ER is an abuse-deterrent, extended-release/long-acting combination of oxycodone and naloxone. Purdue declined to disclose launch plans, including pricing.
FDA is requiring postmarketing studies for Targiniq ER to assess the risks of misuse, abuse, increased sensitivity to pain, addiction, overdose and death associated with long-term use, as well as studies to further assess the effects of the drug's abuse-deterrent features. FDA said Targiniq ER is the second ER/LA opioid analgesic with abuse-deterrent labeling consistent with draft guidance the agency issued last year. The product is already marketed in Canada, Europe and Australia (see BioCentury Extra, Jan. 9, 2013).
Purdue markets the only other pain drug in the U.S. with abuse-deterrent labeling, a reformulated version of OxyContin oxycodone.
ArmaGen Technologies Inc. (Calabasas, Calif.) granted Shire plc (LSE:SHP; NASDAQ:SHPG) exclusive, worldwide rights to develop and commercialize AGT-182, which is in development to treat mucopolysaccharidosis type II (MPS-II, Hunter's syndrome). ArmaGen will receive $15 million up front in cash and equity. The company is also eligible for up to $210 million in an additional equity investment, R&D funding and milestones, plus up to double-digit royalties. ArmaGen will conduct a Phase I/II trial of AGT-182, after which Shire will be responsible for further development and commercialization. ArmaGen expects to start the Phase I/II trial by year end.
AGT-182 is in development for both CNS and somatic manifestations of Hunter's syndrome, a rare lysosomal storage disorder caused by inadequate activity of the enzyme iduronate-2-sulfatase (IDS). AGT-182 is an enzyme replacement therapy comprised of IDS formulated with ArmaGen's Trojan horse technology. The technology creates a fusion protein of a therapeutic compound and a molecule that targets surface receptors on endothelial cells to facilitate transport across the blood-brain barrier.
AbbVie Inc. (NYSE:ABBV) is acquiring Shire for about L31.4 billion ($53.8 billion) in a cash and stock deal slated to close next quarter (see BioCentury Extra, July 18).
FDA approved an NDA from Eagle Pharmaceuticals Inc. (NASDAQ:EGRX) for Ryanodex dantrolene to treat malignant hyperthermia. The company said it plans to launch the drug next month at a wholesale acquisition cost of $2,300 for a 250 mg vial. Eagle told BioCentury that one vial of Ryanodex "is sufficient to provide a loading dose for patients up to 100 kg. The average total dosage required to treat a patient during an MH episode typically requires one-to-three vials, depending on the patient's weight and condition severity."
Ryanodex is a lyophilized formulation of the muscle relaxant dantrolene, which reverses the signs of malignant hyperthermia. Eagle said Ryanodex can be reconstituted in less than one minute compared to the 15-20 minutes required for marketed formulations of dantrolene.
Malignant hyperthermia is a potentially fatal, rare, inherited disorder characterized by a rapid heart rate, high body temperature and blood pressure and increased acid levels. The disorder is usually associated with the administration of certain anesthetics or succinylcholine, which triggers increased levels of calcium in the skeletal muscle.
Eagle was down $0.46 to $12.61 on Wednesday.
UCB Group (Euronext:UCB) said adjunctive treatment with once-daily brivaracetam for 12 weeks met the FDA-defined and EMA-defined primary endpoints vs. placebo in the Phase III N01358 trial to treat partial-onset seizures in epilepsy patients. The FDA-defined endpoint was percent reduction in partial-onset seizure frequency from baseline to day 28, and the EMA-defined endpoint was a 50% responder rate for partial-onset seizure frequency at day 28. The double-blind, international trial enrolled 768 patients with partial-onset seizures not fully controlled despite treatment with one to two concomitant antiepileptic drugs.
Early next year, UCB plans to submit an NDA to FDA and an MAA to EMA for brivaracetam as adjunctive treatment of partial-onset seizures in adult epilepsy patients. The product is a high affinity synaptic vesicle protein (SV2A) ligand.
UCB was up EUR 1.46 to EUR 66.97 on Wednesday.
Inovio Pharmaceuticals Inc. (NYSE-M:INO) gained $1.96 (18%) to $13.10 on Wednesday after reporting that intramuscular VGX-3100 followed by electroporation with Inovio's Cellectra device met the primary endpoint vs. placebo in a Phase II trial to treat cervical intraepithelial neoplasia (CIN) associated with HPV types 16 or 18. VGX-3100 followed by electroporation at weeks 0, 4 and 12 led to a greater proportion of patients with histopathological regression of cervical dysplasia to CIN 1 or no disease at week 36 vs. placebo (49.5% vs. 30.6%, p<0.025). The double-blind, international trial enrolled 143 women with biopsy-proven CIN grade 2/3. Data were presented at the International Society of DNA Vaccines meeting in San Diego.
Inovio said the trial will guide the advancement of VGX-3100 for precancerous dysplasias as well as HPV-associated cervical, head and neck and anogenital cancers. The company said it will determine next steps after completing data review, but declined to provide a timeline. VGX-3100 is a DNA-based therapeutic vaccine targeting the E6 and E7 proteins of HPV 16 and 18.
Data from a pair of in vitro studies published in Science Translational Medicine on Wednesday show cystic fibrosis drug Kalydeco ivacaftor from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) reduced the correction efficacy of the company's lumacaftor (VX-809) in human cells with two copies of the delta F508 mutation in the CF transmembrane conductance regulator (CFTR) gene. In a statement, Vertex said while the "preclinical experiments represent interesting cell biology that can inform our research efforts, what doctors and patients care about -- and what we're focused on -- is the benefit the combination has on people with CF."
The published studies showed that in cells with two copies of the delta F508 CFTR mutation, Kalydeco impaired the biochemical stability of the mutant CFTR, leading to reduced expression of lumacaftor-corrected delta F508 CFTR protein on the plasma membrane. One study was conducted by researchers at McGill University and colleagues. The second was conducted by researchers at the University of North Carolina at Chapel Hill. Lumacaftor is a small molecule CFTR corrector that works by partially restoring the defects caused by the delta F508 CFTR mutation to increase functional expression of the protein. Kalydeco is a small molecule potentiator of CFTR that works by improving gating defects caused by CFTR mutations.
In June, Vertex reported data from the identical, double-blind, international Phase III TRAFFIC and TRANSPORT trials in CF patients who have two copies of the delta F508 CFTR mutation showing that lumacaftor plus Kalydeco met the primary endpoint in both trials. Next quarter, the company plans to submit an NDA to FDA and MAA to EMA for the combination. According to Vertex, the delta F508 CFTR mutation is the most prevalent genetic mutation that causes CF (see BioCentury, June 30).
Kalydeco is approved in the U.S., Europe, Canada, Australia and New Zealand to treat CF in patients 6 years of age and older who have at least one copy of the G551D mutation in the CFTR gene. Kalydeco also is approved in the U.S. to treat CF in patients ages 6 years of age and older who have one of eight additional mutations in the CFTR gene, not including the delta F508 mutation.
Vertex was up $0.40 to $98.14 on Wednesday.
Reinstating Medicare rebates would save an estimated $141.2 billion over 10 years, according to a report released by Social Security Works the Medicare Rights Center. The report outlines policy options to cut costs for Medicare, including restoring Medicare prescription drug rebates and allowing Medicare to negotiate drug prices for Part D.
The advocacy groups noted that many strategies lawmakers have proposed to cut Medicare costs involve slashing benefits or shifting costs to beneficiaries, and instead recommended that the federal government secure savings by lowering drug costs. During a conference call with reporters, Rep. Henry Waxman (D-Calif.) -- who has also called for drug rebates for Medicare enrollees as well as discounts for Part D drugs -- pointed to the emergence of blockbuster HCV drugs like Sovaldi sofosbuvir from Gilead Sciences Inc. (NASDAQ:GILD) as a cause for concern and questioned the sustainability of rising prescription drug costs. Waxman said Gilead has not responded to requests from Congress for pricing justification of the drug.
Last month, Waxman and Rep. Diana DeGette (D-Colo.) requested that the House Energy and Commerce Committee hold a hearing to discuss the HCV drug and its costs to seniors, taxpayers, Medicare Part D drug plans and the Medicare program. Sovaldi has a current wholesale acquisition cost of $84,000 for a 12-week course (see BioCentury Extra, June 19).
Other proposals in the report include securing better discounts from manufacturers to close the Part D doughnut hole and promoting cost-effective prescribing of expensive Part B drugs by reducing the percentage at which drugs are reimbursed under the outpatient benefit. The report suggests reducing the benefit to 103% from 106%.
HHS Secretary Sylvia Burwell hired Leslie Dach as senior counselor, a new position. He will work on policy issues and strategic initiatives and will help with the second open enrollment period for health insurance through the health exchanges. Dach was EVP of corporate affairs at Walmart Stores Inc. (NYSE:WMT). The second open enrollment period is slated to start Nov. 15.
Thirteen states and the District of Columbia are running their own exchanges, while the federal government is facilitating or running the exchanges in the remaining states. Despite glitches and technical problems with the exchanges during the first open enrollment period, the White House said 8 million people enrolled in health plans through the exchanges. The first open enrollment period ended March 31.
Stakeholders requested that FDA allow pharmaceutical companies to share more information to physicians about real-world drug usage so they can make more informed choices to provide better patient care. During a Tuesday hearing held as part of the U.S. House of Representatives Energy and Commerce Committee's Path to 21st Century Cures initiative, stakeholders discussed the discovery, development, and delivery cycle and the importance of transparent collaboration to bring better treatments to patients sooner.
Subcommittee Chairman Joseph Pitts acknowledged that discovery of the risks and benefits of a drug does not end with FDA approval and is often the beginning of learning about different uses for drugs, including other indications. Moreover, real-world usage brings out additional information on safety and efficacy, and "ensuring that this knowledge is shared widely among providers, patients, and researchers is critical."
In addition, the ecosystem that has supported innovation in the U.S. is "being eroded by an increasingly costly and cumbersome risk-averse culture," said Michael Mussallem, chairman and CEO of Edwards Lifesciences.
The Pharmaceutical Research and Manufacturers of America said in a whitepaper that most patients are likely not aware that FDA regulates the medical and scientific information that biopharmaceutical companies may share with healthcare professionals, and thus FDA's regulations have a direct impact on patient care. "Congress should press the FDA to revise its regulations to allow companies to share truthful, scientifically accurate, and data-driven information with healthcare professionals to inform treatment decisions," PhRMA said.
"The key here is to make sure that we have an appropriate balance between protecting patient information, and we believe very strongly in that, but we also don't want to erect barriers to having access to that data," stressed Mary Grealy, president of the Healthcare Leadership Council.
Kathy Hudson, deputy director for science, outreach, and policy at NIH, Rep. Diana DeGette (D-Colo.) and Rep. Fred Upton (R-Mich.) described the initiative's goals on the June 1 edition of BioCentury This Week television (see BioCentury This Week, June 1, 2014 2013).