Nogra Pharma Ltd. (Dublin, Ireland) granted Celgene Corp. (NASDAQ:CELG) exclusive, worldwide rights to develop and commercialize GED-0301 to treat moderate to severe Crohn's disease and other gastrointestinal indications. Nogra (formerly Giuliani S.p.A.) will receive $710 million up front and is eligible for up to $1.9 billion in milestones, plus tiered single-digit royalties. GED-0301, an oral antisense oligonucleotide targeting SMAD family member 7 (MADH7; SMAD7), has completed a Phase II trial for active Crohn's. Celgene plans to start a Phase III program by year end.
Celgene was off $3.57 to $141.25 on Thursday. Separately, the company reported 1Q14 adjusted diluted EPS that beat the Street on revenues that just missed the Street (see below).
Celgene Corp. (NASDAQ:CELG) reported 1Q14 adjusted diluted EPS that just beat the Street on revenues that fell shy of the consensus estimate. Revenues for the quarter were $1.73 billion, below the $1.77 billion the Street was expecting but up from $1.5 billion in 1Q13. Sales of multiple myeloma drug Revlimid lenalidomide were $1.14 billion, up 14% from $1 billion in 1Q13. The consensus estimate for Revlimid sales was $1.16 billion. Celgene reported 1Q14 EPS of $1.67, beating the Street's estimate of $1.65 and up from $1.37 in 1Q13.
Celgene reiterated its 2014 guidance of adjusted diluted EPS of $7-$7.20 on $7.5 billion in revenues. The Street was expecting EPS of $7.29 on $7.6 billion in revenues. During the quarter, the company repurchased 10.7 million shares for about $1.6 billion. Celgene has about $407 million remaining under an existing stock repurchase plan, and the company's board approved a new $4 billion stock repurchase program.
Celgene was off $3.57 to $141.25 on Thursday.
Sarepta Therapeutics Inc. (NASDAQ:SRPT) raised $100.7 million through the sale of 2.7 million shares at $38 in a follow-on underwritten by BofA Merrill Lynch; Morgan Stanley; Deutsche Bank; Baird; William Blair; and Canaccord. Sarepta proposed to raise up to $100 million in the offering on Tuesday, when its share price was $38.79.
On Monday, Sarepta jumped $9.58 (39%) to $33.98 after the company said it plans to submit an NDA to FDA for eteplirsen (AVI-4658) to treat Duchenne muscular dystrophy (DMD) by year end. In November, the company fell after saying approval of eteplirsen could be delayed by at least two years (see BioCentury Extra, April 21).
Sarepta was off $0.72 to $38.19 on Thursday.
Agios Pharmaceuticals Inc. (NASDAQ:AGIO) raised $88 million through the sale of 2 million shares at $44 in a follow-on underwritten by JPMorgan; Goldman Sachs; Cowen; and Leerink. Agios filed to raise up to $75 million in the offering on April 9, when its share price was $48.42. On Monday, the company amended the offering and said it planned to sell 2 million shares.
Agios' two most advanced programs are AG-221, an inhibitor of mutated isocitrate dehydrogenase 2 (IDH2), and AG-120, an inhibitor of mutated IDH1. Both are in Phase I testing. Celgene Corp. (NASDAQ:CELG) has the exclusive option to license worldwide rights to AG-221 and ex-U.S. rights to AG-120 at the end of Phase I testing. Earlier this month, Agios also started Phase I testing of AG-348 to treat pyruvate kinase deficiency, a rare form of hereditary hemolytic anemia. AG-348 is an oral small molecule activator of pyruvate kinase R.
Agios was off $2.76 to $43.58 on Thursday.
Juno Therapeutics Inc. (Seattle, Wash.) raised $31 million in the final tranche of a series A round, bringing the total raised in the round to $176 million. The cancer immunotherapy company initially raised $120 million in the A round in December and in January expanded the round to $145 million. Juno declined to disclose who participated in the final tranche. The company also hired Mark Frohlich as EVP of R&D. He was EVP of R&D and CMO at cancer immunotherapy company Dendreon Corp. (NASDAQ:DNDN).
Earlier this month, patient deaths prompted Memorial Sloan-Kettering Cancer Center, Juno's partner, to briefly halt enrollment and update inclusion criteria for its CD19 directed chimeric antigen receptor T cell therapy. But Juno and other CAR players don't think this will affect development of similar therapies (see BioCentury, April 21).
CRISPR Therapeutics (Basel, Switzerland) secured $25 million in a series A round from Versant Ventures. CRISPR is using genome editing technology based on the CRISPR (clustered, regularly interspaced short palindromic repeats)-associated bacterial endonuclease Cas9 system to develop therapeutic candidates for serious genetic diseases. The company declined to disclose specific indications or genetic targets it will pursue. CRISPR-Cas9 is a newly identified bacterial defense system that can induce site-specific mutations.
In November, Editas Medicine (Cambridge, Mass.), another genome editing company using CRISPR-Cas9, secured $43 million in a tranched series A round (see BioCentury, Dec. 9, 2013).
Arch Venture Partners is looking to raise $250 million for Arch Venture Fund VIII LP, according to an SEC filing. The firm, which could not be reached for comment, closed its seventh fund with $400 million in 2007 and has nearly $1.5 billion under management. Arch invests in early stage life sciences, information technology and physical sciences companies. The firm's portfolio includes RNAi company Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) and cancer immunotherapy play Juno Therapeutics Inc. (Seattle, Wash.), which closed its A round with $176 million on Thursday. (see above)
Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) reported 1Q14 earnings on Thursday that beat the consensus EPS and revenue estimates and disclosed that it began submission to FDA of a rolling BLA for asfotase alfa (ENB-0040) to treat hypophosphatasia (HPP). Asfotase alfa has breakthrough therapy designation from FDA for HPP, an inherited metabolic disease characterized by defective bone mineralization.
Net sales in the quarter were $566.6 million, up 67% from $338.9 million in 1Q13 and beating the Street's $560.2 million estimate. The company's sole product is Orphan drug Soliris eculizumab, which the company markets in almost 50 countries for atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria (PNH). Non-GAAP diluted EPS was $1.53, up from $0.65 the prior year and topping the Street's $1.26 estimate.
Alexion raised its 2014 non-GAAP diluted EPS guidance and said it now expects full-year EPS of $4.75-$4.85 on revenues of $2.15-$2.17 billion. In March the company guided to 2014 EPS of $4.37-$4.47 on the same revenues. The Street was expecting full-year EPS of $4.49 on $2.2 billion in revenues.
Alexion was up $3.78 to $156.01 on Thursday.
AstraZeneca plc (LSE:AZN; NYSE:AZN) reported 1Q14 financial results on Thursday and reemphasized its commitment to its core research areas of cardiovascular/metabolic, cancer, and respiratory/inflammation as well as its plans to seek partners for its neurology and infectious disease programs. Last year, the pharma said R&D and business development in neurology and infectious diseases would be "opportunity-driven." On a conference call to discuss the pharma's 1Q14 financial results, CEO Pascal Soriot stressed that he was not talking about divesting the units. The comment came in response to a question about a Financial Times article speculating that a spinout or divestments of AZ's neurology and infectious diseases businesses could be worth $15 billion. Soriot did not comment on media reports of aborted talks for an acquisition by Pfizer Inc. (NYSE:PFE) (see BioCentury Extra, April 21).
First quarter revenues were $6.42 billion, up 3% from $6.39 billion in 1Q13. The Street was expecting $6.32 billion. Core EPS was $1.17 for 1Q14, a nickel below the Street's $1.23 estimate and down 11% from $1.41 in 1Q13. The pharma reiterated its 2014 guidance of a low- to mid- single digit percentage decline in revenue and a percentage decline in core EPS in the teens. All percent changes are at constant exchange rates.
AstraZeneca also said FDA granted breakthrough therapy designation to AZD9291 to treat metastatic EGFR T790M mutation-positive non-small cell lung cancer (NSCLC) that progressed during treatment with an EGFR tyrosine kinase inhibitor (TKI). The pharma plans to start Phase III testing this year.
Hydra Biosciences Inc. (Cambridge, Mass.) partnered with Boehringer Ingelheim GmbH (Ingelheim, Germany) to develop small molecule inhibitors against transient receptor potential cation channel subfamily C member 4 (TRPC4) and TRPC5 for CNS diseases and disorders. Hydra and Boehringer will jointly identify and advance candidate inhibitors through preclinical development, after which Boehringer will be responsible for global development and commercialization. Hydra will receive an undisclosed upfront payment and research funding and is eligible for milestones, plus tiered royalties.
The board of Biocon Ltd. (NSE:BIOCON; BSE:BIOCON) promoted Arun Chandavarkar to CEO, joint managing director and a director. Chandavarkar is COO at the company. Biocon Chairman and founder Kiran Mazumdar-Shaw is also a managing director. Biocon could not be reached for details, including whether CEO is a new position and whether Chandavarkar will remain COO.
The Indian biopharmaceutical company also reported fourth quarter and full year FY14 financial results on Thursday, including full year EPS of Rs20.7 on FY14 revenues of Rs29.3 billion ($486.9 million). Biocon made both announcements after market close.
Hyperion Therapeutics Inc. (NASDAQ:HPTX) will acquire the Andromeda Biotech Ltd. diabetes subsidiary of Clal Biotechnology Industries Ltd. (Tel Aviv:CBI) in a cash and stock deal. Clal will receive an upfront payment of $12.5 million in cash and 312,869 Hyperion shares, valued at $8 million based on Hyperion's close of $25.61 on Wednesday, before the deal was announced. Clal is also eligible for up to $550 million in milestones, plus 10-25% royalties on DiaPep277 sales.
DiaPep277 is in Phase III testing for new onset Type I diabetes, an Orphan indication with about 35,000 adults diagnosed annually in the U.S. and Europe. Data are expected in 1Q15. In February, Andromeda paid Teva Pharmaceutical Industries Ltd. (NYSE:TEVA) about $72 million to reacquire exclusive, worldwide rights to commercialize the peptide comprising 24 amino acids derived from the sequence of heat shock protein 60 (Hsp60). Andromeda has rights to DiaPep277 from DeveloGen AG, which Evotec AG (Xetra:EVT) acquired (see BioCentury Extra, Feb. 24).
The boards of Hyperion and Clal approved the deal, which is slated to close this quarter. On Thursday, Hyperion was off $0.08 to $25.53. Clal was down NIS0.85 to NIS11.40.
OncoFusion Therapeutics Inc. (Ann Arbor, Mich.) granted Medivation Inc. (NASDAQ:MDVN) access to OncoFusion's preclinical library of small molecule inhibitors of the BET bromodomain family. Medivation will have exclusive, worldwide rights to develop and commercialize selected compounds. OncoFusion is eligible to receive undisclosed upfront payments and milestones, plus royalties. The partners could not be reached for details.
In a paper published in Nature on Wednesday, a BET bromodomain inhibitor significantly reduced tumor volume and weight vs. Xtandi enzalutamide in a mouse model of metastatic castration-resistant prostate cancer (CRPC). OncoFusion co-founder Arul Chinnaiyan is corresponding author of the article.
Medivation was off $2.32 to $57.72 on Thursday. The company markets Xtandi with partner Astellas Pharma Inc. (Tokyo:4503).
The not-for-profit Global Alliance for TB Drug Development granted Shanghai Fosun Pharmaceutical Group Co. Ltd. (Shanghai:600196; HKSE:2196) exclusive rights to develop and commercialize the alliance's PaMZ tuberculosis regimen in China, Taiwan, Hong Kong and Macau. The alliance will receive an undisclosed upfront payment and is eligible for an undisclosed milestone, plus royalties. By year end, the alliance is aiming to start the international Phase III STAND trial to evaluate the regimen for drug-susceptible TB and multidrug-resistant TB (MDR-TB). The Bill & Melinda Gates Foundation will provide "significant" funding for the trial, which is expected to cost $58-$65 million.
The regimen comprises PA-824, a nitroimidazopyran; moxifloxacin, a fluoroquinolone antibiotic; and pyrazinamide, an off-patent TB drug. The TB Alliance has exclusive, worldwide rights to PA-824 from Chiron Corp., now part of Novartis AG (NYSE:NVS; SIX:NOVN); and to moxifloxacin for TB from Bayer AG (Xetra:BAYN).
FDA proposed an expedited access program for medical devices, including diagnostics, that treat or diagnose a life-threatening or irreversibly debilitating disease or condition with no approved alternative treatment or diagnostic and are reviewed as a PMA. According to draft guidance for the proposed Expedited Access PMA (EAP) program, FDA may accept less certainty on the benefit-risk profile of products in the program at the time of approval but will require postmarket confirmatory data and will work with sponsors to develop a plan outlining what data will be collected pre and postmarket. FDA said it may also require less manufacturing information or forgo inspection of some manufacturing sites until after approval. The agency said it expects 6-12 requests for EAP annually and expects most devices to enter the program when in preclinical development. Comments on the draft guidance are due July 22.
FDA will hold a public meeting on June 10 to discuss neurological manifestations of inborn errors of metabolism, such as lysosomal storage disorders Gaucher's and Niemann-Pick disease. The meeting is part of FDA's Patient-Focused Drug Development initiative, under which FDA is holding public meetings for 20 diseases to discuss the impact of the disease, the measures of benefit that matter most to patients and the adequacy of existing treatment options.
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