AstraZeneca plc (LSE:AZN; NYSE:AZN) will acquire the respiratory disease business of Almirall S.A. (Madrid:ALM) for $875 million up front and up to $1.2 billion in milestones. The business had 1H14 sales of EUR 123.5 million ($168.6 million). The deal is expected to close by year end.
AZ will gain Almirall's chronic obstructive pulmonary disease (COPD) drug Eklira Genuair aclidinium bromide, an inhalable long-acting, selective M2 and M3 muscarinic receptor antagonist delivered using Almirall's Genuair inhaler; and Almirall's aclidinium bromide/formoterol fumarate (LAS40464), a fixed-dose combination of aclidinium bromide with a long acting beta agonist (LABA) that is under review in the EU for COPD.
The pharma also will gain Almirall's abediterol (LAS100977), a once-daily LABA inhaled bronchodilator in Phase II testing; LAS190792, a dual long-acting muscarinic antagonist beta 2 agonist (MABA) in Phase I testing, along with two preclinical MABAs; and undisclosed preclinical programs. Additionally, AZ is gaining the Almirall Sofotec GmbH subsidiary of Almirall, which develops device technologies including the Genuair inhaler.
AZ's marketed respiratory products include Symbicort budesonide/formoterol, a fixed-dose corticosteroid/LABA combo, and asthma drug Pulmicort budesonide turbuhaler. The pharma made steps to bolster its respiratory pipeline by acquiring respiratory company Pearl Therapeutics Inc. last year.
Almirall was up EUR 0.85 to EUR 11.60 on Wednesday. The company separately reported 1H14 financial results after market close in Spain on Tuesday. Total revenues for the half were EUR 433.4 million ($591.6 million), up from EUR 410.4 million ($534.3 million) in 1H13.
Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) and partner Sanofi (Euronext:SAN; NYSE:SNY) said subcutaneous alirocumab (REGN727) met the primary endpoint in nine Phase III trials of the human mAb targeting proprotein convertase subtilisin/kexin type 9 (PCSK9). The partners aren't yet reporting detailed data from the trials. In eight trials, the primary endpoint was a statistically significant reduction in LDL-C from baseline to week 24 vs. placebo or an active comparator. Four of the trials enrolled patients with hypercholesterolemia and a high cardiovascular risk not adequately controlled with lipid-lowering therapy; two of the trials enrolled patients with heterozygous familial hypercholesterolemia (heFH) not adequately controlled with lipid-lowering therapy; and one of the trials enrolled patients with primary hypercholesterolemia with a history of intolerance to at least two statins and a moderate to very high CV risk.
In the ninth trial -- the 2,341-patient ODYSSEY LONG TERM trial -- alirocumab met the primary endpoint of reductions in LDL-C at week 24. Additionally, a post hoc analysis showed a significantly lower rate of adjudicated major CV events in the alirocumab arm compared to the placebo arm (p<0.05). The trial enrolled hypercholesterolemia patients with high CV risk not adequately controlled with lipid-lowering therapy.
Last year, alirocumab met the primary endpoint in the Phase III ODYSSEY MONO trial in patients with primary hypercholesterolemia and moderate CV risk. The companies expect data from three other Phase III trials in 2015 or later (see BioCentury Extra, Oct. 16, 2013).
Sanofi and Regeneron plan to submit a BLA to FDA and an MAA to EMA for alirocumab to treat hypercholesterolemia by year end. The partners acquired a Priority Review voucher from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) that they plan to use for alirocumab (see below).
Regeneron -- which also saw a U.S. label expansion for ophthalmic drug Eylea aflibercept on Wednesday -- was up $17.67 to $322.18. The move translates to a gain in market cap of $1.8 billion for a closing valuation of $32.5 billion.
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) said it sold a Priority Review voucher to Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) and partner Sanofi (Euronext:SAN; NYSE:SNY) for $67.5 million late Wednesday and separately reported 2Q14 financial results. BioMarin gained the voucher earlier this year following FDA's approval of the company's Vimizim elosulfase alfa to treat mucopolysaccharidosis IVA (MPS-IVA, Morquio's syndrome). BioMarin was the first company to receive a voucher under the agency's Rare Pediatric Disease Priority Review Voucher program, which was created by PDUFA V to encourage the development of new drugs and biologics to prevent and treat rare pediatric diseases (see BioCentury Extra, Feb. 7).
Sanofi and Regeneron are equally splitting the cost of the voucher and said they plan to use it to obtain Priority Review for a BLA for alirocumab to treat primary hypercholesterolemia. The partners plan to submit the application for the human mAb targeting proprotein convertase subtilisin/kexin type 9 (PCSK9) to FDA by year end.
Separately, BioMarin reported 2Q14 revenues of $191.7 million, up from $136.8 million in the prior year's quarter and beating the Street's $159 million estimate. Revenues included $14.3 million in sales of Vimizim, which BioMarin launched in the U.S. in February. The company reported a non-GAAP diluted EPS in the quarter of $0.06.
BioMarin was up $0.06 to $21.09 on Wednesday. News of the voucher sale as well as the company's 2Q14 financial results came after market close.
Pfizer Inc. (NYSE:PFE) will acquire the marketed vaccine portfolio of Baxter International Inc. (NYSE:BAX) for $635 million in cash. Baxter's marketed vaccines are NeisVac-C, a group C meningococcal polysaccharide conjugate vaccine; and FSME-IMMUN, also known as TicoVac, a tick-borne encephalitis vaccine. The vaccines are marketed in ex-U.S. territories, including the EU. Pfizer will also acquire a portion of Baxter's facility in Orth, Austria, where the vaccines are manufactured.
The products will complement Pfizer's marketed Prevnar family of pneumococcal vaccines. The deal is slated to close by year end. Credit Suisse advised Pfizer.
FDA approved Eylea aflibercept from Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) to treat diabetic macular edema (DME). The drug is already approved in the U.S. to treat wet age-related macular degeneration (AMD) and macular edema following central retinal vein occlusion (CRVO).
Eylea is also approved for wet AMD in the EU, Japan, Australia and several other countries and in Japan and the EU for macular edema following CRVO. EMA's CHMP backed approval of Eylea for DME in June. Bayer AG (Xetra:BAYN) has ex-U.S. rights to the product from Regeneron, which was up $17.67 to $322.18 on Wednesday.
Separately on Wednesday, Regeneron and partner Sanofi (Euronext:SAN; NYSE:SNY) reported data from nine Phase III trials of alirocumab and acquired a Priority Review voucher they plan to use for the human mAb targeting proprotein convertase subtilisin/kexin type 9 (PCSK9).
Theraclone Sciences Inc. (Seattle, Wash.) granted Gilead Sciences Inc. (NASDAQ:GILD) exclusive rights to develop and commercialize products incorporating Theraclone's preclinical broadly neutralizing HIV antibodies (bNAbs) for therapeutic applications. Theraclone received an undisclosed upfront payment and is eligible to receive undisclosed development and commercialization milestones. The deal includes bNAbs targeting a glycan-dependent epitope on the pre-fusion conformation of HIV gp41 on cleaved HIV env trimers as well as bNAbs targeting a variety of epitopes on HIV gp120, including PG9 and PG16.
The Scripps Research Institute and the International AIDS Vaccine Initiative (IAVI) discovered the bNAbs using Theraclone's I-STAR antibody discovery technology. IAVI has rights to develop HIV vaccines based on the bNAbs. Gilead declined to comment on the deal.
Theraclone is partnered with IAVI under an expanded 2008 collaboration to discover HIV-neutralizing antibodies. In 2009, the partners identified PG9 and PG16, which target a variable region of gp120 that is exposed only when three molecules of the viral gp120 protein form a trimeric complex (see SciBX: Science-Business eXchange, Sept. 17, 2009).
Gilead was up $0.80 to $93.79 on Wednesday.
FDA reviewers said the potential benefits of hematology product HyQvia from Baxter International Inc. (NYSE:BAX) must be weighed against unknown long-term risks of antibodies generated against recombinant human PH20 hyaluronidase (rHuPH20), one of the product's components. HyQvia is a subcutaneous formulation of IV Gammagard, an IgG antibodies plasma-based therapy, and rHuPH20 from Halozyme Therapeutics Inc. (NASDAQ:HALO). The product is under review as replacement therapy to treat adults with primary immunodeficiency associated with defects in humoral immunity.
The reviewers said although HyQvia's short-term risks in clinical trials are acceptable, it is unknown whether PH20 antibodies developed as a result of HyQvia will penetrate tissues in testes, bowel and brain and cause adverse events such as infertility and inflammation in the bowel and brain. They said a pharmacovigilance plan to monitor such risks may not be feasible "as many years of subject and offspring follow up may be required." The reviewers also noted the main advantage of HyQvia compared to immune globulin without rHuPH20 is the lower frequency of dosing, and patients already have access to a variety of immune globulin products without exposure to rHuPH20. The comments came in briefing documents released ahead of Thursday's meeting of FDA's Blood Products Advisory Committee to discuss the resubmitted BLA for HyQvia.
Baxter expects a decision this quarter, but the exact PDUFA date is undisclosed. The company received a complete response letter from FDA in August 2012 in which the agency requested additional preclinical data to address concerns about antibodies against rHuPH20.
Halozyme was off $0.29 to $9.22 on Wednesday.
Dendreon Corp. (NASDAQ:DNDN) hired W. Thomas Amick as president and CEO. He will also sit on the board of directors. He succeeds John Johnson, who announced his departure in June. Amick is former chairman and CEO of Discovery Laboratories Inc. (NASDAQ:DSCO). Dendreon, which made the announcement after marbut ket close, was unchanged at $2.10 on Wednesday.
Amgen Inc. (NASDAQ:AMGN) jumped as much as $7.93 on Wednesday -- hitting an all-time high of $131.24 -- before closing the day up $6.70 to $130.01 after reporting 2Q14 financial results late Tuesday that beat the Street and unveiling plans to reduce headcount by 12-15% through 2015. Amgen's Wednesday move translates to a gain in market cap of $5.1 billion for a closing valuation of $98.4 billion. The company said it expects the headcount reductions to improve cost structure and reduce layers of management (see BioCentury Extra, July 29).
Hospira Inc. (NYSE:HSP) gained $4.31 to $56.21 on Wednesday after reporting 2Q14 financial results that beat the Street. Adjusted diluted EPS for the quarter was $0.72, beating the Street's $0.57 estimate and up from $0.55 in 2Q13. Hospira reported net sales of $1.14 billion for the quarter, beating the Street's estimate of $1.06 billion and up from $1.03 billion in 2Q13. The company increased its full-year guidance and now expects adjusted diluted EPS of $2.30-$2.50 on 6-9% in sales growth compared to 2013. The company previously forecast a change in net sales of -2% to +3% on a constant currency basis over 2013 and adjusted diluted EPS of $2-$2.25. The Street was expecting $2.22 in full-year EPS.
Hospira disclosed during its earnings call that it plans to submit a regulatory application to FDA for Retacrit epoetin zeta, a biosimilar of Epogen epoetin alfa from Amgen Inc. (NASDAQ:AMGN), later this year or early next year. Hospira already markets Retacrit in Europe to treat anemia associated with chronic renal failure and chemotherapy.
The company declined to comment on the potential for a tax inversion deal. Earlier this week, Hospira was rumored to be in acquisition talks with the medical nutrition unit of food product conglomerate Danone (Euronext:BN) (see BioCentury Extra, July 28).
Women's healthcare company TherapeuticsMD Inc. (NYSE:TXMD) raised $40.2 million through the sale of 8.6 million shares at $4.67 in a follow-on underwritten by Goldman Sachs and Noble Capital Markets. TherapeuticsMD proposed the follow-on late Tuesday, when its share price was $4.55.
TherapeuticsMD is developing a pipeline of hormone therapy products for menopause-related hormone deficiencies, with two products in Phase III testing: TX 12-001HR, an oral combination of estradiol and progesterone; and TX-002HR, an oral progesterone. The company also markets branded and generic prescription prenatal vitamins, as well as OTC vitamins and cosmetics.
TherapeuticsMD was up $0.45 (10%) to $5.12 on Wednesday.
Cancer molecular diagnostic company Epic Sciences Inc. (La Jolla, Calif.) raised $30 million in a series C round from new investors RusnanoMedInvest and Arcus Ventures, along with existing investors Domain Associates; Roche Venture Fund; Pfizer Venture Investments. Undisclosed individual investors also participated.
Epic's circulating tumor cell (CTC) technology uses protein and fluorescence in situ hybridization (FISH) markers instead of physical size to separate CTCs from blood samples, which the company believes will prevent destruction of cells and provide more accurate cell counts. Epic's internal pipeline includes diagnostics to help guide treatment and monitor patients' response. The company also has partnerships to develop companion diagnostics for cancer therapeutics with 17 companies. Epic was spun out of the Scripps Research Institute in 2008.
Separately on Wednesday, Epic appointed Gregory Lucier chairman. Lucier was chairman and CEO of Life Technologies Corp., now part of Thermo Fisher Scientific Inc. (NYSE:TMO).
The U.S. Pharmacopeial Convention said its quality standards "may be helpful for informing FDA's evaluation" of the similarity between a proposed biosimilar and its reference product. The remark came in USP's comments on May FDA draft guidance on pharmacological data requirements for biosimilars. USP said although its standards "are not directly pertinent to the assessment of clinically meaningful differences" between the proposed biosimilar and reference product, they may help FDA assess similarity on "certain key quality attributes." USP noted that only FDA has the authority to determine whether two drugs are similar or interchangeable.
The draft guidance states that FDA will characterize its assessment of biosimilarity into one of four levels -- not similar, similar, highly similar or highly similar with a fingerprint-like similarity -- depending on the type, nature and extent of any structural and functional differences revealed (see BioCentury Extra, May 13).
USP is a nonprofit body that sets standards for the identity, strength, quality and purity of medicines and is a member of a council that negotiates non-proprietary names with manufacturers.
Earlier this month, the Sandoz unit of Novartis AG (NYSE:NVS; SIX:NOVN) said the agency accepted a BLA for a biosimilar version of Neupogen filgrastim G-CSF from Amgen Inc. (NASDAQ:AMGN) -- the first publicly disclosed FDA acceptance of a biosimilars application (see BioCentury, July 28).
The World Health Organization published a draft proposal for a global system to assign "biological qualifiers" to biologics and biosimilars. A biological qualifier is a random four-letter code assigned to a biologic manufactured at a specified site. WHO said the scheme would be voluntary for each regulatory authority and applicable retroactively. The qualifier would not be part of a biologic's international non-proprietary name (INN), although WHO's INN expert group would oversee the scheme.
WHO said the proposed scheme is intended to avoid separate national qualifier systems. Comments on the draft are due Sept. 19.
New enrollees will likely face significant challenges during open enrollment for Affordable Care Act exchanges unless CMS improves its processes, according to William Woods, director of acquisition and sourcing management at the U.S. Government Accountability Office. According to Woods, despite $840 million allocated to develop Healthcare.gov and its supporting systems, inconsistent contractor oversight within the program office and "unclear" roles and responsibilities led to confusion about who had the authority to approve contractor requests for additional work. Woods identified about 40 instances during the development of the federally facilitated marketplace in which CMS staff "inappropriately" authorized contractors to spend more than $30 million.
The comments came in testimony released ahead of a Thursday meeting of the House Energy and Commerce Committee's subcommittee on oversight and investigations to discuss the implementation of the ACA.
Andy Slavitt, CMS principal deputy administrator, said in his testimony for the meeting that the agency is building on its first year of ACA implementation by improving information, plan options and affordability of the "shopping experience." He said the agency is addressing the "execution and technology lessons" learned during the first open enrollment period and is continuing to automate marketplace's back-end systems. According to Slavitt, two new roles -- administrator and secretary -- were created to "provide clear accountability and visibility." HHS Secretary Sylvia Mathews Burwell hired Slavitt late last month to oversee policy and operational coordination for Medicare, Medicaid and the Children's Health Insurance Program (CHIP) (see BioCentury Extra, June 20).
FDA's Center for Drug Evaluation and Research published a Manual of Policies and Procedures outlining CDER actions from the time a product receives breakthrough therapy designation until a marketing application has been submitted. According to the manual, CDER should review the status of breakthrough therapy programs every three to six months, and may consider rescinding the designation if the criteria for a breakthrough therapy are no longer met.
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