2-BBB Medicines BV, of Leiden, the Netherlands, said it set up a Taiwan-based joint venture with TTY Biopharm Co. Ltd., of Taipei, Taiwan, for the further clinical and commercial development of its product candidate designed to treat neuroinflammation, 2B3-201, initially focused on patients with acute multiple sclerosis relapses. The capital of the joint venture company was set at NT360 million (US$11.7 million) and TTY will invest NT240 million in cash to obtain a 66.67 percent stake. 2-BBB will obtain a 33.33 percent stake in the new company for the exclusive 2B3-201-related patent rights and results valued at NT120 million. Furthermore, TTY will put another NT260 million into TTY’s dedicated commercial-scale manufacturing infrastructure for 2B3-201, made available to the joint venture under an exclusive manufacturing agreement.
CASI Pharmaceuticals Inc., of Rockville, Md., said a decision was reached to stop further patient enrollment in the phase II open-label study of ENMD-2076 in previously treated locally advanced or metastatic triple-negative breast cancer (TNBC) conducted at the University of Colorado, Denver, and the Indiana University Melvin and Bren Simon Cancer Center. The two-stage study targeted an accrual of 35 patients and was expanded to take into consideration those patients who may not be evaluable for efficacy analysis as per the protocol. The uncommon tumor type has proved a challenging setting to recruit, the company said, and a total of 41 patients have been enrolled, which has been determined to be sufficient to assess the potential efficacy of ENMD-2076. CASI continues to collect and evaluate data in those patients enrolled in the study, and the biomarker program evaluating potential markers that could be predictive of a response to ENMD-2076 is ongoing. In addition, the ongoing phase IIa study in China in patients with TNBC continues to accrue patients in the dose-escalation phase, the primary objective of which is to determine the recommended phase II dose in Chinese study subjects. That phase is expected to reach the full accrual target in the third quarter of this year. ENMD-2076 is an Aurora A kinase and tyrosine kinase inhibitor.
Catalyst Biosciences Inc., of South San Francisco, said it achieved a milestone under its collaboration with Isu Abxis Co. Ltd., of South Korea, to advance the factor IX program toward its first clinical trial. Completion of the CB 2679d/ISU304 toxicology studies supported the recent IND approval by the Korean Ministry of Food and Drug Safety and triggered a milestone payment to Catalyst. CB 2679d/ISU304, a next-generation coagulation factor IX variant, has demonstrated the potential to normalize human factor IX levels with a daily subcutaneous injection in preclinical studies. Isu Abxis plans to initiate a phase I/II study with single and multiple subcutaneous injections in patients with severe hemophilia B in the second quarter.
Researchers have shown that the heart, brain and kidneys cooperate to activate the immune system in response to pressure overload, attempting to ward off heart failure. A team from the Japanese Chiba University and Jichi Medical University investigated the systemic response to cardiac pressure overload, a precursor of heart failure. The researchers showed that multiple organs interacted in the adaptive response, and that beta-2 adrenergic receptor signaling was an essential component of that response. Beta-adrenergic blockers are standard treatment to prevent heart failure. The authors wrote that their beneficial effects, which are clearly established, are due to their effects on beta-1 receptors, and concluded that “our findings highlight the need to re-evaluate the effects of pharmacological adrenergic receptor intervention at the multisystem and multi-organ levels.” Their work appeared in the April 10, 2017, issue of Nature Medicine.
China Cord Blood Corp., of Hong Kong, rejected a bid by Golden Meditech Holdings Ltd. to take it private. The company provides cord blood collection, laboratory testing, hematopoietic stem cell processing and stem cell storage services. Its board unanimously adopted a recommendation to terminate negotiation on the proposal advanced by a special committee appointed to review the offer. Company shares (NYSE:CO) rose 15.1 percent to close at $8.05 on Thursday.
Clinuvel Pharmaceuticals Ltd., of Melbourne, Australia, said it reached agreement with the German National Association of Statutory Health Insurance Funds for the treatment of erythropoietic protoporphyria (EPP) patients with its drug Scenesse (afamelanotide 16 mg). The company adopted a uniform global pricing policy and the annual costs of therapy range between €56,404 (US$59,864) and €84,606 (US$89,796) per EPP patient per annum.
Repeat problems and hundreds of complaints landed Mumbai, India-based Indoco Remedies Ltd. an FDA warning letter. A September inspection revealed that the contract manufacturer based in Verna, India, had received more than 1,500 complaints from 2012 through last August about leaking, empty and under-filled bottles of sterile drug solutions. The company’s investigation attributed the persistent problem on a malfunction with a filling machine. Operators intervene when they spot defects, but the defects are not easily detected. Thus, the line has produced finished, capped units with nonintegral container-closures and, in some cases, cracks develop in the bottles days later, compromising the quality, integrity and sterility of the contents. Although Indoco has tried to address the problem in various ways over the past four years, it has continued to receive a large number of complaints about the bottles. Furthermore, the company’s threshold for initiating an investigation is based on the number of complaints received per batch rather than the criticality of the complaint, according to the letter. “Any complaint of lost integrity of your sterile drugs should be considered as a serious problem . . . and trigger an appropriate investigation,” the FDA said in the letter. In addition, the investigator found no evidence that Indoco had notified its customer, as required under its quality agreement, of complaints that could necessitate the filing of field alert reports. The letter also noted that a 2011 inspection revealed numerous complaints about leakage or crusty residue on bottles of some of the same drug solutions cited in more recent complaints.
Mundipharma International Ltd., of Cambridge, U.K., said it inked a strategic partnership with Kolon Life Science Inc., of South Korea, to market Invossa, Kolon’s cell-medicated gene therapy for osteoarthritis. Kolon anticipates the treatment will be approved by South Korea’s Ministry of Food and Drug Safety in the second quarter. Under the terms, Mundipharma will handle marketing and distribution in South Korea, while Kolon will cover the general practitioner market. Financial terms were not disclosed.
Prima Biomed Ltd., of Sydney, Australia, received a cash rebate of A$492,144 (US$ 368,399) from the Australian Federal Government’s research and development tax incentive program. The rebate was provided for expenditures incurred on eligible activities conducted in the 2016 fiscal year, mainly related to the company’s TACTI-mel trial, a phase I study in melanoma using its lead compound IMP-321, conducted in Australia. IMP-321 is described as a first-in-class antigen-presenting cell activator based on the immune checkpoint LAG-3.
Decreased levels of cohesin expression in the developing brain led to greater synaptic complexity, more immature synapses and greater levels of anxiety in mouse models of the neurodevelopmental disorder Cornelia de Lange syndrome. Cohesin is part of a protein complex with epigenetic functions, controlling chromosome structure and function. In their experiments, researchers from the Japanese Osaka University showed that deleting the Smc3 gene, which codes for cohesion, in mice resulted in different symptoms of Cornelia de Lange syndrome depending on the cell types that lacked the gene. Their work appeared in the April 13, 2017, online issue of the Journal of Experimental Medicine.
Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan, reported top-line results from a phase III study testing dasotraline (SEP-225289), a dopamine norepinephrine reuptake inhibitor, in children, ages 6 to 12, with attention deficit hyperactivity disorder (ADHD). The trial met the primary endpoint, defined as change from baseline at day 15 in ADHD symptoms as measured by mean SKAMP-Combined Score obtained from an average of seven assessments collected across the 12-hour laboratory classroom day (12-24 hours post-dose). Dasotraline 4 mg/day demonstrated sustained efficacy over the 12- to 24-hour time period post-dose and was associated with an acceptable safety profile over the two-week study duration. Based on the results, Sunovion Pharmaceuticals Inc., a U.S. subsidiary of Sumitomo Dainippon, plans to submit an NDA to the FDA this year for adult and pediatric populations.
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan, said it formed a strategic collaboration with Nubiyota LLC, of Teaneck, N.J., to develop new products via Nubiyota’s Microbial Ecosystem Therapeutic platform. The companies aim to advance oral microbial consortia products for unmet gastroenterology indications. Takeda will pay Nubiyota an up-front payment and Nubiyota will be eligible for success-based development, regulatory and commercial milestones and tiered royalties based on net sales. Further terms of the agreement were not disclosed.
Transgene SA, of Strasbourg, France, said the first patient with soft tissue sarcoma (STS) was treated in the phase II part of the METROmaJX phase I/II trial testing the tolerability and efficacy of the co-administration of Pexa-Vec with metronomic cyclophosphamide (low doses given with high frequency) in patients with advanced solid tumors such as breast cancer and STS. In part one, the combination demonstrated a satisfactory tolerability profile. The second part of the open-label trial will enroll patients with STS and HER2-negative-breast cancer and will primarily assess antitumor efficacy. Pexa-Vec is a GM-CSF-expressing vaccinia-derived oncolytic virus co-developed by Transgene and Sillajen Co. Ltd., of South Korea.