Amicus Therapeutics Inc., of Cranbury, N.J., said it submitted an NDA to Japan's PMDA for chaperone therapy migalastat in Fabry disease. The application includes data from two pivotal phase III studies as well as a phase I study evaluating the pharmacokinetics of migalastat in Japanese volunteers. The drug has orphan designation in Japan, which makes it eligible for priority review as well as 10 years of market exclusivity, if approved. Based on the priority review timeline, Amicus expects a decision from the PMDA in the first half of 2018. Migalastat previously gained approval as Galafold in the EU.

Arrien Pharmaceuticals LLC, of Salt Lake City, said an unnamed U.S.-based pharmaceutical company licensed the worldwide rights to its ROR-γt targeting compounds, including ARN-6039, a phase I drug candidate for the treatment of moderate to severe psoriasis and potentially other autoimmune disorders. Arrien will receive an undisclosed up-front license fee with potential for future development and sales milestones and royalties on net sales. The safety, tolerability and pharmacokinetics of ARN-6039 were established in a phase I trial of single ascending doses in healthy adult subjects.

Cancer Research Technology Ltd., of London, signed an extended deal with Merck KGaA, of, Darmstadt, Germany, to discover new cancer drugs targeting the Hippo pathway. The extension follows a one-year partnership to better understand the role of the Hippo pathway in cancer and how best to target the pathway. CRT, the commercial arm of Cancer Research UK, will receive undisclosed royalties and milestone payments from drugs developed under the collaboration.

Catalyst Biosciences Inc., of South San Francisco, said the European Commission granted orphan designation to CB-2679d/SU304, a next-generation coagulation factor IX variant, for hemophilia B. Collaborator ISU Abxis Co. Ltd., of South Korea, completed dosing in the first of up to five patient cohorts in a phase I/II study in severe hemophilia. Data are expected by the end of this year.

Evotec AG, of Hamburg, Germany, disclosed a collaboration with Censo Biotechnologies Ltd., of Midlothian, U.K., to source and provide patient-derived induced pluripotent stem cells (iPSC) to support Evotec's iPSC platform. Censo will provide its sourcing and reprogramming technologies and expertise to create a high-quality, bespoke repository of hundreds of patient-derived iPS cell lines, covering multiple diseases with an initial focus on CNS diseases. The contract will run for an initial period of two years. Financial terms were not disclosed.

Global Blood Therapeutics Inc., of South San Francisco, said the EMA determined that GBT-440 for sickle cell disease is eligible for its Priority Medicines, or PRIME, program, which provides early and proactive EMA support for products with the potential to address unmet medical needs. GBT-440, which is designed as an oral, once-daily therapy and works by increasing hemoglobin's affinity for oxygen, is in an ongoing phase I/II trial.

Heptares Therapeutics, of London, a subsidiary of Sosei Group Corp., said it launched a research collaboration under its ORBIT initiative with New York University School of Medicine. It will support a multiyear program with NYU's accelerator group, the Office of Therapeutics Alliances and the lab of Dimitris Placantonakis, an expert on the pathology and treatment of brain tumors, at the Neurosurgical Laboratory for Stem Cell Research, in the Department of Neurosurgery at the NYU School of Medicine. Research will focus on the discovery of molecules that selectively modulate a G protein-coupled receptor implicated in the formation and progression of glioblastoma multiforme, an aggressive brain cancer. Under the terms, Heptares and NYU will jointly fund the discovery phase, and Heptares has an exclusive option to license IP relevant to the target and to take any promising compounds further through development and potentially to commercialization. NYU is eligible to receive milestone payments and royalties.

Janssen Research & Development LLC, of Raritan, N.J., a unit of Johnson & Johnson, said the FDA accepted for priority review a supplemental NDA for Xarelto (rivaroxaban) to include a 10-mg once-daily dose for reducing the risk of venous thromboembolism (VTE) after at least six months of standard anticoagulant therapy. The application was based on data from EINSTEIN CHOICE, a study that showed two doses of Xarelto (10 mg and 20 mg) was superior to aspirin in reducing the risk of recurrent VTE, with comparable rates of major bleeding.

Kempharm Inc., of Coralville, Iowa, said it's developing KP484, a super-extended release d-threo-methylphenidate (d-MPH) for the treatment of attention deficit hyperactivity disorder (ADHD). The prodrug was discovered during the phase I trial testing KP415, the company's other prodrug of d-MPH. Kempharm plans to file an IND application for KP484 as early as the third quarter of 2017 and said it plans to use data from the KP415 to expedite development of KP484, potentially submitting an NDA as soon as 2019. After the end-of-phase I meeting with the FDA for KP415, Kempharm said it anticipates starting a pivotal efficacy trial testing KP415 in patients with ADHD in the second half of 2017 and remains on schedule to submit an NDA as soon as late 2018.

Kite Pharma Inc., of Santa Monica, Calif., said the U.S. Patent and Trademark Office provided a notice of intent to issue an Ex Parte Reexamination Certificate that confirms the patentability of amended claims presented in U.S. Patent No. 7,741,465, known as the Eshhar '465 patent. The patent was licensed by Kite in 2013 and covers its CAR-T treatment axicabtagene ciloleucel, which is currently under review by the FDA for the treatment of aggressive non-Hodgkin's lymphoma with a PDUFA target action date of Nov. 29.

Mateon Therapeutics Inc., of South San Francisco, reported preclinical data showing that vascular-disrupting agent CA4P in combination with a checkpoint inhibitor significantly reduced tumor size in a CT-26 colon cancer animal model. The company said CA4P increases the effects of checkpoint inhibitors because it rapidly causes tumor cell death, which likely increases tumor antigen presentation and T-cell activation and the overall immunologic response. Animals in the combination CA4P and anti-CTLA-4 antibody treatment group continue to show declines in tumor volume beyond day 14. CA4P currently is in phase II/III testing in platinum-resistant ovarian cancer.

Merlion Pharmaceuticals Pte Ltd., of Singapore, said its joint project with the Defence Science and Technology Laboratory, U.K., was awarded a grant from the U.S. Defense Threat Reduction Agency Chemical and Biological Defense Program. The multiyear, multiphase project will study the impact of Merlion's fluoroquinolone, finafloxacin, for the treatment of infections caused by the biological threat agent Burkholderia pseudomallei. In addition, the broad-spectrum efficacy of finafloxacin against other intracellular biothreat agents such as Francisella tularensis and Yersinia pestis and other multidrug-resistant pathogens of clinical significance will be investigated in further detail.

Mithra Pharmaceuticals SA, of Liege, Belgium, signed a license and supply agreement with Fuji-City, Japan-based Fuji Pharma Co. Ltd., for the commercialization of Donesta, a hormone therapy based on Estetrol, in Japan and the Association of Southeast Asian Nations. Mithra will get €1 million (US$1.14 million) upfront and is eligible for development, regulatory and commercialization milestones in the low double-digit million euros. The companies will equally fund the development of Donesta phase III program in Japanese subjects in the hormone therapy indication. A dose-finding phase II study is currently ongoing with top-line results expected late in the first quarter of 2018.

Opiant Pharmaceuticals Inc., of Santa Monica, Calif., said it inked a global, exclusive licensing agreement gaining access to San Diego-based Aegis Therapeutics LLC's Intravail drug delivery technology for all of Opiant's opioid antagonist compounds. Financial terms were not disclosed.

Profounda Inc., of Orlando, Fla., said the FDA granted orphan designation to miltefosine to treat granulomatous amebic encephalitis. Profounda licensed the drug, an alkyl-lysophospholipid analogue branded Impavido, from Knight Therapeutics Inc., of Princeton, N.J. It previously gained approval for visceral, mucosal and cutaneous leishmaniasis.

Scholar Rock Inc., of Cambridge, Mass., reported preclinical data for RK-015, an inhibitor of the supracellular activation of myostatin, to be presented at the Cure SMA Annual Conference in Orlando, Fla. SRK-015 specifically targets the activation of latent forms of myostatin, but doesn't inhibit GDF11 or Activin A that are structurally similar to myostatin. The drug increased lean body mass in non-human primates, increasing muscles with a high proportion of fast-twitch fibers, which are particularly affected in spinal muscular atrophy (SMA). In a mouse model of SMA, inhibition of myostatin activation, in combination with a therapy that targets the underlying genetic defect, significantly improved the strength of the gastrocnemius relative to treatment with the targeted therapy alone.