Aerie Pharmaceuticals Inc., of Irvine, Calif., and Ramot at Tel Aviv University Ltd., of Tel Aviv, Israel, said they inked a research collaborative and license agreement for a preclinical anti-beta amyloid small-molecule candidate for neuroprotection and dry age-related macular degeneration. Under the terms, Aerie will undertake all research and development programs, and Ramot will receive development milestone payments and royalties on successful commercialization of products that result from the collaboration. The agreement will last one year with an option for Aerie to obtain an exclusive license from Ramot for further development of the candidate for all ophthalmic indications, including front and back of the eye. Financial details of the agreement were not disclosed.
Amyris Inc., of Emeryville, Calif., and the Infectious Disease Research Institute agreed to explore testing and development of an Amyris material for use in adjuvant formulations to enhance the effectiveness of vaccines. Amyris will provide its materials for testing new adjuvant formulations, and the organizations will work together to explore their use to improve the global supply of important vaccines. Financial terms were not disclosed.
Asterias Biotherapeutics Inc., of Menlo Park, Calif., said data from preclinical safety studies of its investigational drug, AST-OPC1, were published in Regenerative Medicine. "Preclinical Safety of hESC-Derived Oligodendrocyte Progenitors Supporting Clinical Trials in Spinal Cord Injury" outlines the results that were submitted to the FDA in an investigational new drug application. AST-OPC1 is in a phase I/IIa dose-escalation trial for complete cervical spinal cord injury.
Asuragen Inc., of Austin, Texas, said it entered a collaboration with Illumina Inc., of San Diego, to establish a framework for the development and commercialization of sequencing-based companion diagnostics in partnership with biotech and pharmaceutical companies. Terms were not disclosed.
Catalyst Biosciences Inc., of South San Francisco, said it has begun a project to develop a generic version of Sabril (vigabatrin, Sanofi S.A.), which is marketed by Lundbeck Inc., part of H. Lundbeck A/S, of Valby, Denmark, in the U.S. to treat infantile spasms and complex partial seizures.
Celsion Corp., of Lawrenceville, N.J., said it has finished the integration of Egen Inc., which it acquired in June 2014. Celsion has consolidated all early stage and preclinical candidates at its facility in Huntsville, Ala., and has organized all clinical development, commercialization (which includes early access programs for Thermodox), business development and administrative functions at its Lawrenceville, N.J. headquarters. Celsion said it expects a 15 percent to 20 percent reduction in personnel and related annual operational costs from these changes. In addition Celsion said it is lining up opportunities to improve GEN-1 IL-12-based immunotherapy and is structuring clinical development to capitalize on previous proof-of-concept clinical data.
Checkpoint Therapeutics Inc., of New York, said it filed a registration statement on Form 10 with the SEC under the Securities Exchange Act of 1934 to become a reporting company. Checkpoint is an immune-oncology biopharma that concentrates on acquiring, developing and commercializing non-chemotherapeutic, immune-enhanced combination treatments for those with solid tumor cancers.
Chugai Pharmaceutical Co. Ltd., of Tokyo, and Roche Group, of Basel, Switzerland, said that Roche unit, Genentech Inc., of South San Francisco, filed a new drug application (NDA) with the FDA for alectinib to treat patients with anaplastic lymphoma kinase (ALK)-positive advanced non-small-cell lung cancer (NSCLC) who progressed on or are intolerant to crizotinib (Xalkori, Pfizer Inc.). The agency accepted the filing and granted priority review, setting a PDUFA date of March 4, 2016. The FDA previously granted breakthrough therapy designation for the ALK inhibitor. Chugai markets alectinib, branded Alecensa, in Japan in the NSCLC indication. Rights to other countries, including the U.S. and EU nations, were outlicensed to Roche, which holds a 62 percent stake in the Japanese pharma. The NDA for alectinib includes data from two phase II studies. ALEX, an ongoing randomized phase III study, is comparing alectinib to crizotinib as first-line treatment for people with advanced NSCLC whose tumors were characterized as ALK-positive by a companion immunohistochemistry test developed by Roche Diagnostics.
Epirus Biopharmaceuticals Inc., of Boston, said it entered an agreement for the acquisition of Bioceros Holding B.V., of Utrecht, the Netherlands, enabling the expansion of its biosimilar pipeline and vertical integration product development capabilities. Epirus will pay $14.1 million in cash and stock in installments over a one-year period. Bioceros is focused on the development of MAbs and generation of GMP-ready cell lines. Using this platform, Epirus will expand its pipeline with the addition of three preclinical product candidates: BOW080, a proposed biosimilar to eculizumab (reference biologic Soliris, Alexion Pharmaceuticals); BOW090, a proposed biosimilar to ustekinumab (reference biologic Stelara, Janssen); and BOW100, a proposed biosimilar to golimumab (reference biologic Simponi, Janssen).
Evotec AG, of Hamburg, Germany, agreed to a four-year research collaboration with Pfizer Inc., of New York, in the field of tissue fibrosis. Scientists at the companies plan to explore potential mechanisms as targets to treat multi-organ fibrosis. Evotec will contribute its drug discovery platform while Pfizer will provide technologies, industrial scope and drug development and marketing expertise. Pfizer will make an undisclosed up-front and potential development and sales milestone payments to Evotec. Other terms were not disclosed.
Ibio Inc., of Newark, Del., said it has been awarded a grant to fund further development of its therapeutic product for treatment of fibrotic diseases from the National Heart, Lung, and Blood Institute under its Small Business Technology Transfer program. The company is extending the research of Feghali-Bostwick, of the Medical University of South Carolina (MUSC), to develop products for the treatment of a range of fibrotic diseases including idiopathic pulmonary fibrosis and systemic sclerosis/scleroderma. IBIO-CFB03, produced using the company's Ibiolaunch gene expression platform, is the first product candidate from this program being advanced for clinical development. The company is sponsoring research at both MUSC and Novici Biotech LLC in a coordinated program focused on IBIO-CFB03 and potentially other product candidates.
Janssen-Cilag International NV, of Beerse, Belgium, part of Johnson & Johnson, submitted a marketing authorization application to the EMA for daratumumab, its human anti-CD38 monoclonal antibody candidate, to treat patients with relapsed and refractory multiple myeloma. The submission was based on data from the phase II MMY2002 (SIRIUS) monotherapy study, the phase I/II GEN501 monotherapy study and three supportive studies. Daratumumab was previously granted orphan drug status by the EMA to treat plasma cell myeloma. The EMA submission followed this month's acceptance by the FDA of the biologics license application for daratumumab in the U.S. under priority review. Janssen holds an exclusive global license to develop, manufacture and commercialize daratumumab from Genmab AS, of Copenhagen, Denmark. (See BioWorld Today, Aug. 31, 2012, and March 7, 2014.)
Immunocore Ltd., of Oxford, UK, said its lead product, IMCgp100, was accepted in the EMA's adaptive pathways pilot program, designed to help provide patients with timely access to medicines that treat serious conditions with a high unmet medical need. Immunocore plans to seek conditional approval for IMCgp100 to treat patients with metastatic uveal melanoma. Conversion to full approval would be subject to completion of a phase II trial in the indication, plus long-term follow-up data. (See BioWorld Today, July 17, 2015.)
Medgenics Inc., of Philadelphia, acquired Neurofix Therapeutics LLC, which developed NFC-1, a nonstimulant metabotropic glutamate receptor (mGluR) neuromodulator entering phase II/III study to treat mGluR network mutation-positive attention deficit hyperactivity disorder (mGluR+ ADHD) and neuropsychiatric symptoms resulting from the related rare genetic disorder, 22q11.2 deletion syndrome. Medgenics acquired the outstanding shares of Neurofix for $2 million in cash up front, undisclosed performance-based milestone payments and sales royalties.
Opko Health Inc., of Miami, said it inked a global license deal with The Scripps Research Institute (TSRI) for the development and commercialization of lipoprotein signal peptidase inhibitors developed and discovered using a high-throughput screening technology funded and exclusively licensed by Opko from TSRI. Terms were not disclosed.
Pacira Pharmaceuticals Inc., of Parsippany, N.J., filed suit against the FDA seeking to exercise its rights to communicate truthful and non-misleading information about its flagship product, Exparel (bupivacaine liposome injectable suspension), which was approved in 2011 for postsurgical analgesia. In September 2014, the FDA issued Pacira a warning letter related to certain promotional materials. Pacira said it took certain actions to address immediate FDA concerns and presented the agency with evidence defending its marketing practices, requesting multiple follow-up discussions. Six weeks ago, the agency issued a close-out letter on the matter, according to Pacira. The complaint describes the company's position that the FDA violated the Administrative Procedure Act, the Fifth Amendment, its own guidance documents and precedent by threatening enforcement action for the promotion of Exparel that Pacira said is consistent with the approved indication. The lawsuit also alleges that the FDA violated the First Amendment rights of Pacira and co-plaintiffs by restricting the exchange of truthful and non-misleading speech. As part of the complaint, filed in the U.S. District Court for the Southern District of New York, Pacira is seeking declaratory relief and a preliminary and/or permanent injunction preventing the FDA and other defendants from taking any action to violate the company's rights. (See BioWorld Today, Jan. 18, 2011.)
Pernix Therapeutics Holdings Inc., of Morristown, N.J., said a series of studies of ZX007, its next-generation extended-release formulation of hydrocodone bitartrate developed in coordination with Altus Formulation Inc., of Mirabel, Quebec, showed the tablet resisted both physical manipulation and extraction with a variety of typical household solvents commonly used by opioid abusers. Pernix said success of the studies supported continued evaluation of the formulation in human abuse liability studies. The analysis was accepted as a late-breaking abstract for presentation as a poster at Pain Week in Las Vegas.
Pluristem Therapeutics Inc., of Haifa, Israel, said the FDA indicated that data the company presented from preclinical tests of its hematopoietic stem cell therapy PLX-R18 would support an investigational new drug application. The company also showed the regulator the design of its proposed phase I study, which it now anticipates starting in the U.S. in early 2016. PLX-R18, the company's second cell therapy product in development, is being studied as a potential treatment of incomplete hematopoietic recovery following hematopoietic cell transplantation. PLX-R18 is also being developed, in partnership with the National Institutes of Health, as a potential treatment for acute radiation syndrome. (See BioWorld Today, May 19, 2015.)
Redhill Biopharma Ltd., of Tel-Aviv, Israel, reported that the National Cancer Institute awarded the company a $2 million SBIR grant to support its planned phase II study of the sphingosine kinase-2 (SK2) selective inhibitor Yeliva (ABC-294640) as a possible treatment of refractory or relapsed multiple myeloma. The grant covers a three year period and was awarded to Apogee Biotechnology Corp., of Hummelstown, Pa. – from which Redhill acquired rights to the candidate in March – in conjunction with Duke University. Redhill expects the open-label, dose escalation study to begin by the end of this year. On Wednesday, the company reported supportive data from the first phase III study of RHB-105 for eradication of H. pylori. (See BioWorld Today, June 16, 2015.)
Rigel Pharmaceuticals Inc., of South San Francisco, reported forging an exclusive global license agreement with a subsidiary of Malvern, Pa.-based Aclaris Therapeutics Inc., which will develop and commercialize specified Rigel JAK inhibitors for the treatment of alopecia areata and other dermatological conditions. Under terms of the agreement, Rigel will receive an up-front payment of $8 million, and will be eligible to receive various milestone payments of up to $90 million based on global development and multiple indications, as well as tiered royalties on any future sales of these compounds. The Aclaris subsidiary, Aclaris Therapeutics International Ltd., will assume responsibility for the continued development of the covered compounds.
Santhera Pharmaceuticals AG, of Liestal, Switzerland, said the European Commission granted marketing authorization for Raxone (idebenone), as the first approved treatment available in all 28 member states of the EU, Norway, Iceland and Liechtenstein for the treatment of visual impairment in adolescent and adult patients with Leber's hereditary optic neuropathy, a rare, inherited mitochondrial disease that usually leads to rapid and permanent blindness if untreated. Approval of Raxone, a synthetic short-chain benzoquinone and a co-factor for the enzyme NAD(P)H:quinone oxidoreductase, was based on efficacy data from a the RHODOS trial and an open-label expanded access program, in which the drug demonstrated an ability to mitigate or reverse vision loss. The EMA's Committee for Human Medicinal Products voted in favor of approval in June. (See BioWorld Today, June 29, 2015.)
Spotlight Innovation Inc., of West Des Moines, Iowa, reported that its subsidiary, Celtic Biotech, has entered an exclusive patent license agreement, applicable to North America, with Sao Paulo, Brazil-based Instituto Butantan for the use of Crotamine, a neurotoxin derived from the venom of the South American rattlesnake Crotalus durissus, as a potential imaging agent for cancer and gene therapy delivery vector. Celtic intends to run validation studies with the intent to move into phase I studies studying Crotamine as a positron emission tomography radiopharmaceutical imaging agent for lung cancer tumors. Financial terms were not disclosed.
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan, reported that the FDA has granted priority review status to the new drug application for ixazomib, the first investigational oral proteasome inhibitor for the treatment of patients with relapsed or refractory multiple myeloma. Patients in the company's pivotal phase III trial, TOURMALINE-MM1, continue to be treated to progression in this trial and will be evaluated for long-term outcomes. (See BioWorld Today, Feb. 5, 2014.)
Vaccinogen Inc., of Baltimore, struck an exclusive option agreement with Ireland's Dublin City University that provides the company with an exclusive two-year option to evaluate and acquire a high-throughput, multiplex, analysis platform, called DiCAST for Direct Clone Analysis and Selection Technology. Vaccinogen said that it intends to use the platform to expand and accelerate its human monoclonal antibody program, screening patient-derived biological samples acquired after gaining anti-cancer immunity. Vaccinogen also expects to use the system to identify immunologically relevant data from the biological samples to develop cancer vaccines and immunotherapies. In conjunction with the agreement, the scientific team that developed the platform, including the lead inventor, Paul Leonard, has joined Vaccinogen and will lead the company's operations in Ireland. Financial terms of the deal were not disclosed.
Visterra Inc., of Cambridge, Mass., and Serum Institute of India Ltd. entered a license agreement for the development, manufacture and commercialization of VIS-513, Visterra's preclinical humanized monoclonal antibody dengue vaccine in the Indian subcontinent countries. The vaccine is designed to bind and neutralize all four serotypes of dengue virus and was engineered using Visterra's Hierotope technology. Under terms of the agreement, Serum Institute's license covers territories including India, Pakistan, Bangladesh, Nepal, Bhutan, Maldives and Sri Lanka. The institute will pay Visterra $5 million up front plus up to $34 million based on the achievement of certain development and commercial milestones. Additionally, should VIS-513 be commercialized, Visterra would be eligible to receive tiered, double-digit royalties based on net sales in the licensed territories. Serum Institute will fund and be responsible for clinical development and the filing of regulatory applications in the countries covered.
Xenetic Biosciences Inc., of Lexington, Mass., reported that its R&D, license and supply agreements with Baxter Healthcare SA and Baxter Healthcare Corp. have made the post-spinout leap, and are now assigned to Baxalta Inc. Xenetic has an exclusive deals with Baxalta to develop products with an extended half-life of certain proteins and molecules using its Polyxen technology.