Acucela Inc., of Seattle, and Youhealth Eyetech Inc., of San Diego, have agreed on an exclusive option from Youhealth for lanosterol technology licensed from the Regents of the University of California, San Diego, for the non-surgical pharmacologic treatment of cataracts. Lanosterol, a naturally occurring compound found in the human body, has shown promise for this condition in preclinical studies. It has been identified as a key molecule in the prevention of lens protein aggregation and lens opacification. Acucela will evaluate lanosterol as a treatment for mild cataracts with an option to fully develop and commercialize the compound globally with the exception of Greater China including China, Taiwan and Hong Kong. The use of lanosterol in other ophthalmic conditions such as presbyopia may be investigated by Acucela in the future. Youhealth's parent company, Guangzhou Kang Rui Biological Pharmaceutical Technology Co. Ltd., of Guangzhou, China, will be responsible for lanosterol's clinical development and commercialization in Greater China including China, Taiwan and Hong Kong.

Allergan plc, of Dublin, filed an abbreviated new drug application (ANDA) with the FDA seeking approval to market Paclitaxel Protein-Bound Particles for Injectable Suspension, 100 mg/vial, a generic version of Abraxane (nab-paclitaxel, Celgene Corp.), which is indicated for the treatment of metastatic breast cancer, after failure of combination chemotherapy for metastatic disease or relapse within six months of adjuvant chemotherapy; locally advanced or metastatic non-small-cell lung cancer, as first-line treatment in combination with carboplatin, in patients who are not candidates for curative surgery or radiation therapy; and metastatic adenocarcinoma of the pancreas as first-line treatment, in combination with gemcitabine. Allergan said it is the first applicant to file an ANDA for this generic.

The American Pharmacists Association House of Delegates voted to adopt new policy on the labeling and measurement of oral liquid medications at its annual meeting in Baltimore, supporting a move away from dosing cups and teaspoons in favor of oral syringes and cups that use the milliliter (mL) as the standard unit of measure for prescribing and measuring oral liquid medications.

Aradigm Corp., of Hayward, Calif., said the EMA has approved its request to review Pulmaquin, Aradigm's investigational inhaled liposomal ciprofloxacin product, under the Centralised Authorisation Procedure drug review process. The request was granted because, the company said, Pulmaquin represents a significant technical innovation for the treatment of non-cystic fibrosis bronchiectasis associated with chronic Pseudomonas aeruginosa infection.

Bayer Healthcare Pharmaceuticals Inc., part of Bayer AG, of Leverkusen, Germany, said the FDA approved Kovaltry Antihemophilic Factor (recombinant), an unmodified, full-length factor VIII compound for the treatment of hemophilia A in children and adults. The approval is based on results from the LEOPOLD (Long-Term Efficacy Open-Label Program in Severe Hemophilia A Disease) clinical trials, which supported the approval for routine prophylaxis to reduce the frequency of bleeding episodes. Kovaltry can be used two or three times a week in adolescents and adults, and two or three times a week or every other day in children. Bayer's established hemophilia A therapy, Kogenate, brought in global sales of about €1.15 billion (US$1.30 billion) in 2015. Bayer also is working with Cambridge, Mass.-based Dimension Therapeutics Inc. to develop a hemophilia gene therapy. (See BioWorld Today, June 24, 2014.)

Biota Pharmaceuticals Inc., of Atlanta, completed the sale of assets related to its broad spectrum antibiotic program to a newly formed subsidiary of Spero Therapeutics LLC, a Cambridge, Mass.-based company founded to develop therapies for the treatment of bacterial infections. Financial terms of the transaction were not disclosed. Biota CEO Joseph Patti said that the transaction would allow his company to "fully focus our resources on our clinical-stage direct antiviral programs aimed at addressing infections with limited therapeutic options."

Capricor Therapeutics Inc., of Los Angeles, said the California Institute for Regenerative Medicine approved a grant of approximately $3.38 million to support its phase I/II HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) trial investigating cardiac cell therapy CAP-1002 in patients with Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. Capricor also said it completed enrollment of the pre-specified first patient cohort in the HOPE-Duchenne trial, with top-line data expected in the first quarter of 2017. (See BioWorld Today, March 3, 2016.)

Cellectis SA, of Paris, said that it is developing a new class of monoclonal antibodies targeting PD-1 that promote the recovery of T-cells from exhaustion through a new mechanism of action. The company plans to combine these PD-1 MAbs with its gene-edited UCART product candidates to enhance their activity and increase their half-life. The project comes by way of a new research and licensing agreement with Mabquest SA, a recently formed biotech company based in Pully, Switzerland. The deal includes a collaboration phase funded by Cellectis whereby Cellectis and Mabquest will jointly pursue preclinical research on several candidate antibodies; and a clinical development and commercialization phase of the best selected antibodies which will be led by Cellectis. Financial details of the transaction were not disclosed.

Duarte, Calif.-based City of Hope said that a research team led by Yanhong Shi, director of the hospital's division of stem cell biology research, has received a $7.4 million grant from the California Institute for Regenerative Medicine to develop a new treatment for Canavan disease, a rare and fatal neurological disease that afflicts infants. About 12 percent of all Canavan disease cases in the U.S. are in California.

Cleveland Biolabs Inc., of Buffalo, N.Y., said the FDA will require an in vivo study to establish bio-comparability between the drug formulation of entolimod proposed for use as a radiation countermeasure under its pre-Emergency Use Authorization (pre-EUA) and the drug formulation used in previously conducted preclinical and clinical studies. The FDA said further review of the pre-EUA dossier would not proceed until the bio-comparability data were evaluated by the agency. The company said design of the biocomparability study is under way and will require the FDA's endorsement before the study is executed. The company provided the update following the receipt of minutes from a meeting with the FDA. (See BioWorld Today, Jan. 24, 2014.)

Dimension Therapeutics Inc., of Cambridge, Mass., said that the EMA's Committee for Orphan Medicinal Products recommended giving its adeno-associated virus gene therapy, DTX301, orphan status for the treatment of ornithine transcarbamylase (OTC) deficiency. Preclinical studies have shown the therapy to normalize levels of urinary orotic acid, a marker of ammonia metabolism, which if elevated, can cause serious neurological deficiencies in patients. Dimension has completed candidate selection in its DTX301 program and plans to submit an investigational new drug application with the FDA in the second half of 2016.

Elite Pharmaceuticals Inc., of Northvale, N.J., gained FDA acceptance of its new drug application for Sequestox (ELI-200), the company's lead opioid abuse-deterrent candidate for the management of moderate to severe pain where the use of an opioid analgesic is appropriate. The agency has set a PDUFA date of of July 14 for the drug. Sequestox is an immediate-release oxycodone hydrochloride containing sequestered naltrexone which incorporates 5 mg, 10 mg, 15 mg, 20 mg and 30 mg doses of oxycodone into capsules.

Glaxosmithkline plc (GSK), of London, and Miltenyi Biotec Inc., of San Diego, formed a strategic collaboration to optimize the manufacturing and delivery of cell and gene therapy-based treatments in oncology and rare diseases. Miltenyi Biotec will help to integrate greater automation and high-tech processing technology into GSK's current cell and gene therapy R&D manufacturing capabilities with the goal of further industrializing cell and gene therapy and overcoming manufacturing and scale-up constraints associated with existing processes. The collaboration includes the application of technologies to advance the discovery of chimeric antigen-receptor T-cell (CAR-T) based therapeutics for defined oncology targets. GSK said the effort will supplement its existing preclinical CAR-T portfolio. Terms were not disclosed.

Huawei, of Shenzhen, China, an information and communications technology firm, and Wuxi Apptec, of Shanghai, said they signed a framework agreement to support the China Precision Medicine Initiative through the creation of a precision medicine cloud platform.

Kite Pharma Inc., of Santa Monica, Calif., said it entered a clinical trial collaboration with Genentech Inc., a unit of Basel, Switzerland-based Roche AG, to evaluate the safety and efficacy of CAR T-cell immunotherapy KTE-C19 in combination with PD-L1-targeting antibody atezolizumab (also known as MPDL3280A) in patients with refractory, aggressive non-Hodgkin's lymphoma. A phase Ib/II study is set to begin this year. Terms were not disclosed.

Merz Pharma Group, of Frankfurt, said the neurotoxin Bocouture was approved by European regulatory authorities for an expanded indication to treat upper facial lines, including horizontal frown lines, lateral periorbital lines and glabellar frown lines.

Mesoblast Ltd., of Melbourne, Australia, said it exclusively licensed technology developed at Harvard Medical School that can modify mesenchymal lineage adult stem cells (MLCs) to enhance their natural homing properties to sites of excessive inflammation. Called ex vivo fucosylation, that technology has modified MLCs capable of inducing durable reversal of type 1 diabetes in a preclinical study, the company reported. Results showed the cell targeting technology increased by threefold the number of MLCs reaching the inflamed pancreas in autoimmune diabetic mice following intravenous infusion, compared with unmodified MLCs.

Novogen Ltd., of Sydney, said it received a cash refund of $2.8 million from the Australian Taxation Office under the R&D Tax Incentive Program for the financial year ending June 30, 2015. Novogen said the funding will support advancing Cantrixil (TRXE-002-1), its lead superbenzopyran candidate, into phase I testing this year.

Orexo AB, of Uppsala, Sweden, said Astrazeneca plc, of London, acquired all rights to its leukotriene C4 synthase inhibitor program (OX-CLI project) for MUSD 5 in conjunction with their collaboration and option agreement, after the program advanced into preclinical development with an identified development candidate. Based on a new chemical entity, the OX-CLI project aims to develop a treatment for respiratory disorders such as asthma and chronic obstructive pulmonary disease. In 2013, Astrazeneca assumed responsibility to fund R&D activities, and Orexo is set to receive a payment of $5 million for rights to OX-CLI, as well as future milestone payments based on defined development and commercial objectives. Orexo also is entitled to a tiered single-digit royalty on net revenues from product sales based on the OX-CLI program.

Prothena Corp. plc, of Dublin, disclosed the publication of preclinical data demonstrating that the company's conformation-specific antibodies developed against misfolded transthyretin (TTR) bind to and facilitate in vitro cellular uptake of amyloidogenic forms of TTR. The research was published online in Amyloid. The published research describes monoclonal antibodies that specifically bind to misfolded forms of the TTR protein, leaving the native form of the protein unaffected.

Reset Therapeutics Inc., of South San Francisco, inked a collaboration with an affiliate of Alkermes plc, of Dublin, to discover, develop and commercialize orexin receptor modulators from Reset's drug discovery platform. The orexin system is a modulator of wakefulness, appetite and mood, and its dysregulation is associated with central nervous system disorders such as narcolepsy and depression. Alkermes gained an exclusive, worldwide option to license orexin modulators resulting from the collaboration in return for an undisclosed up-front fee and equity investment in Reset, which also is eligible for development, regulatory and sales-based milestone payments as well as royalties on product sales. Reset is responsible for the development of molecules arising from the collaboration through the completion of initial phase II studies. Additional terms were not disclosed.

Soligenix Inc., of Princeton, N.J., said the National Institute of Allergy and Infectious Diseases (NIAID) exercised an option to accelerate regulatory activities with the FDA pertaining to the company's heat stable ricin toxin vaccine, Rivax. The exercised option will provide Soligenix with an additional $660,000 in funding. If all contract options are exercised, the company stands to receive up to $24.7 million to support the preclinical, manufacturing and clinical development activities necessary to advance heat stable Rivax with the FDA. The overall objective of the NIAID contract is advancement of the company's thermostabilization technology, Thermovax, in combination with Rivax, as a medical countermeasure to prevent the effects of ricin exposure.

Thrombogenics NV, of Leuven, Belgium, signed a global and exclusive license agreement with Galapagos NV, of Mechelen, Belgium, to develop and commercialize integrin antagonists for the treatment of diabetic eye disease. Thrombogenics, which gained access to a collection of integrin antagonists developed by Galapagos, said it plans to explore and characterize the assets using validated preclinical models of diabetic eye disease, with the goal of developing a small-molecule integrin antagonist to treat a broad range of patients with diabetic retinopathy, with or without diabetic macular edema. Thrombogenics designated the most advanced integrin antagonist candidate as THR-687. Thrombogenics is set to pay a €1 million (US$1.1 million) up-front technology transfer payment to Galapagos, which is also entitled to development and commercial milestone payments plus royalties on net sales of products emerging from its library of integrin antagonists.

Zambon SpA, of Milan, Italy, concluded a strategic agreement with US Worldmeds for the U.S. commercialization of Xadago (safinamide) to treat Parkinson's disease. In 2012, Zambon gained global marketing rights to safinamide outside Asia and Japan from developer Newron Pharmaceuticals SpA, also of Milan. Zambon is commercializing safinamide, branded Xadago, in the EU and Switzerland, following approval by regulatory authorities last year. The new drug application for safinamide is under review by the FDA with a PDUFA date of March 29. Under terms of the U.S. sublicense, US Worldmeds agreed to pay Zambon undisclosed up-front, regulatory and commercial milestone payments and royalties on product sales. Newron also is entitled to a milestone payment upon FDA approval and a share of up-front, milestone and royalty payments made by US Worldmeds. Zambon said US Worldmeds plans to dedicate more than 60 sales representatives to the U.S. launch of Xadago. (See BioWorld Today, April 6, 2012, and Dec. 30, 2014.)

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