Celgene fronts $710M for Crohn's compound
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 tops Street EPS estimate
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.
Sarepta raises $100.7 million in follow-on
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.
Agios raises $88 million in follow-on
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.
Juno closes A round with $176 million
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).
Genome editing play CRISPR secures $25 million
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.
Arch planning $250 million eighth fund
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 beats Street, starts asfotase alfa BLA
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.
AstraZeneca reports 1Q14 results
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).
Hydra, Boehringer in CNS deal
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.
Biocon promotes Chandavarkar to CEO
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.
Hyperion to acquire Clal's diabetes unit
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.
Medivation, OncoFusion in bromodomain deal
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.
TB Alliance grants Fosun Chinese rights to TB regimen
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.
POLITICS & POLICY
FDA proposes expedited access program for devices
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 holding meeting for inborn errors of metabolism
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.