Advancells Pvt Ltd., of Noida, India, said it reversed multiple sclerosis using adult stem cells and regenerative medicine in the pilot patient of a planned trial. The first patient, diagnosed in 2010 and having had multiple relapses of the disease in the past seven years, could, after treatment, walk better, climb stairs (which he was unable to do after 2012) and even go on a treadmill, the company said. Advancells added that it will take about three months to review changes as shown on magnetic resonance imaging of the patient.
Agilis Biotherapeutics Inc., of Cambridge, Mass., and Tokyo-based Gene Therapy Research Institution Co. Ltd. established a manufacturing and collaboration partnership joint venture to advance adeno-associated virus gene therapies. The joint venture, which will be headquartered in Japan, will focus on developing therapies for diseases of the central nervous system.
India's Central Drugs Standard Control Organisation (CDSCO) and the Indian Pharmacopoeia Commission will hold the third regional workshop on pharmacovigilance Sept. 5 at the CDSCO zonal office in Hyderabad, India. The workshop is part of a nationwide pharmacovigilance program that began in 2010 to better monitor adverse drug reactions. An amendment passed last year to the Drugs & Cosmetics Rules requires the marketing authorization holders for drugs in India to implement a properly designed pharmacovigilance system at their site. The workshop will bring together drug regulators and pharma professionals/experts to discuss the basics of pharmacovigilance in India and the establishment of appropriate systems, according to the CDSCO. Registration is due by Aug. 31.
Cytokinetics Inc., of South San Francisco, said the phase II trial of omecamtiv mecarbil in Japanese patients with heart failure has met its pharmacokinetic primary endpoint and demonstrated statistically significant improvements in systolic ejection time, a secondary endpoint. Omecamtiv mecarbil, a novel investigational cardiac myosin activator that increases cardiac contractility, is being developed by Amgen Inc., of Thousand Oaks, Calif., in collaboration with Cytokinetics for the potential treatment of heart failure. Cytokinetics is eligible to earn a $10 million milestone payment from Amgen upon the first dosing of a patient in Japan in GALACTIC-HF, the phase III cardiovascular outcomes trial.
Enzychem Lifesciences Corp., of Seoul, South Korea, said the FDA granted the company an IND to start a phase II dose-escalation trial testing the ability of EC-18, a synthetic palmitoyl-2-linoleoyl-3-acetyl-rac-glycerol (mosedipimod), to reduce chemoradiation-induced oral mucositis in 84 patients with head and neck cancers. The trial will measure the incidence of severe oral mucositis, defined as WHO grades 3 or 4, over the seven-week treatment period.
Hitgen Ltd., of Chengdu, China, entered a multitarget, multiyear collaboration with LEO Pharma A/S, of Ballerup, Denmark, to discover small-molecule leads for multiple therapeutic targets of interest to LEO. According to the agreement, Hitgen will use its platform based on DNA-encoded library design, synthesis and screening to discover leads, which will be licensed exclusively to LEO. Under the terms, Hitgen will receive an up-front payment and will be eligible for milestone payments from LEO. Details of the financial arrangement were not disclosed.
Isogenica Ltd., of Cambridge, U.K., signed a new licensing deal with Takeda Pharmaceutical Co. Ltd., of Osaka, Japan. Under the terms, Isogenica has granted Takeda licenses to its family of llamdA VHH single-domain antibody libraries for the discovery, development and commercialization of therapeutic products derived from those libraries. Isogenica is entitled to an up-front and annual license payments. If antibodies are advanced into development, Isogenica is entitled to further license fees, milestones and royalties, though the exact terms were not disclosed.
Lumosa Therapeutics Co. Ltd., of Taipei, Taiwan, said it enrolled the first subject for the phase I trial of small-molecule candidate LT-3001 for acute ischemic stroke. Up to 80 healthy volunteers will be enrolled in the double-blind, placebo-controlled, single ascending-dose study to evaluate the safety and pharmacokinetics of LT-3001, results of which will determine the doses for subsequent clinical studies in stroke patients. The trial is expected to be completed in the first quarter of 2018. In nonhuman primate stroke models, LT-3001 showed an apparent wider therapeutic time window and a better safety profile than those reported for recombinant tissue plasminogen activator, or rtPA. LT-3001 is a peptide and small-molecule combination designed to induce thrombolysis and dissolve blood clots while limiting oxidative stress and protecting neurons from endothelial cell damage and reperfusion injury.
Molecular Templates Inc., of Austin, Texas, and Takeda Pharmaceutical Co. Ltd., of Osaka, Japan, said they are collaborating on oncology drug discovery programs directing Molecular Templates' engineered toxin bodies technology platform to potential therapeutic targets provided by Takeda through a joint scientific committee. Under the terms, Takeda will make an equity investment and Molecular Templates is eligible to receive up-front payments, development and commercial milestone payments, plus royalties on the sales of commercial products developed through the collaboration. Takeda also will also appoint a director to Molecular Templates' board.
MT Pharma America Inc., of Jersey City, N.J., changed its corporate name to Mitsubishi Tanabe Pharma America Inc. It remains a wholly owned subsidiary of Osaka, Japan-based Mitsubishi Tanabe Pharma Corp.
Propanc Biopharma Inc., of Melbourne, Australia, said a safety margin to support a starting dose for PRP in first-in-human studies was confirmed after no treatment-related findings were reported by pathologists upon completion of a GLP-compliant 28-day repeat-dose toxicity study with PRP, a solution for once-daily intravenous administration of a combination of two pancreatic proenzymes trypsinogen and chymotrypsinogen.
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan, inked a cardiovascular development partnership with Cardurion Pharmaceuticals LLC, of Cambridge, Mass., which is developing therapeutics to treat of heart failure and other cardiovascular diseases. Financial terms were not disclosed.
Theratechnologies Inc., of Montreal, said the FDA completed the pre-license inspection of the Shanghai-based Wuxi Biologics Inc. facility where ibalizumab will be manufactured. The inspection was carried out from July 17, 2017, to Aug. 2, 2017, and finished with no critical findings. The FDA has made some observations, and Wuxi said it is committed to complete all follow-up actions as soon as possible, which is not expected to impact the review timelines of the ibalizumab biologics license application, the company said. Ibalizumab bears a PDUFA date of Jan. 3, 2018. The drug is a humanized monoclonal antibody being developed for the treatment of multidrug-resistant HIV-1 infection. (See BioWorld Today, March 4, 2015.)
Toray Group, a chemical firm in Japan, and QB3@953, of San Francisco, a life science incubator, reported that they formed a partnership to pursue drug development. Toray said its goal is to develop small-molecule drugs to treat chronic itch, pain and neurodegenerative disease through academic collaborations. Financial terms were not disclosed.
Middle East respiratory syndrome coronavirus (MERS-CoV) is an ongoing public health threat. Though cases of the illness are rare, the virus kills about a third of the individuals it infects, and infections are continuously re-seeded from its animal reservoir in dromedary camels. Vaccine efforts have focused on vaccinating in a way that leads to a durable antibody response. Researchers from the University of Iowa, the Chinese Guangzhou Medical Center, and the Saudi Arabian King Faisal Research Center have instead focused on identifying durable T-cell responses to the virus. The authors studied the serum of MERS survivors and found that antibody and helper T-cell responses correlated with disease severity, but killer T-cell responses did not. Killer T-cell responses were also more long-lasting than antibody responses, which were often transient. The author suggested that T-cell responses "may be useful for predicting prognosis, monitoring vaccine efficacy, and identifying MERS patients with mild disease in epidemiological studies." Their work appeared in the Aug. 2, 2017, issue of Science Translational Medicine.