Eisai Co. Ltd., of Tokyo, said it completed construction of the 5th manufacturing building at the Kawashima Industrial Park located in Gifu Prefecture, Japan. The facility is specifically designated for formulating anti-cancer drugs and the company plans to manufacture its in-house discovered and developed anticancer agent Lenvima at this facility.
Enesi Pharma Ltd., of Oxford, U.K., said it is collaborating with Robin Shattock, a leading infectious disease and vaccine expert, and his group at Imperial College London, to develop RNA vaccines, including against SARS-CoV-2, that are stable at ambient temperatures. This could minimize or eliminate the cold chain requirements for global deployment and mass vaccination programs with RNA-based vaccines.
Eyepoint Pharmaceuticals Inc., of Watertown, Mass., said it entered a royalty monetization agreement with SWK Holdings Corp. for royalties payable to the company under its license agreement with Atlanta-based Alimera Sciences Inc. for retinal diseases drug Iluvien. It received a one-time $16.5 million payment from SWK and, in return, SWK is entitled to receive future royalties payable under the Alimera agreement. The company applied $15 million of net proceeds against existing long-term debt obligations with CRG Servicing LLC. The remaining $1.5 million will be used to advance product pipeline programs. The transaction will also result in a reduction of annual interest payments of approximately $1.7 million.
Gyroscope Therapeutics Ltd., of London, said it entered a sponsored research agreement with the University of Pennsylvania and the Penn Center for Advanced Retinal and Ocular Therapeutics (CAROT) to develop gene therapies for serious eye diseases. The company has an exclusive option to the intellectual property associated with, and arising from, the research conducted under the agreement. A team of researchers from CAROT and Gyroscope will work together to explore specific gene therapy targets for glaucoma, optic neuritis and retinitis pigmentosa.
The Institute for Clinical and Economic Review (ICER) issued a final evidence report assessing the comparative clinical effectiveness and value of nadofaragene firadenovec (Adstiladrin, FKD Therapies Oy and Fergene LLC) and oportuzumab monatox (Vicineum, Sesen Bio Inc.) to treat non-muscle invasive bladder cancer (NMIBC) that does not respond to Bacillus Calmette-Guerin (BCG) intravesical therapy. The findings were reviewed at the November 2020 public meeting of the Midwest Comparative Effectiveness Public Advisory Council (CEPAC), one of ICER’s three independent evidence appraisal committees, where panelists evaluated the clinical evidence for these treatments across adults with BCG-unresponsive NMIBC with carcinoma in situ and those with BCG-unresponsive NMIBC with high-grade papillary disease or superficial invasion but without carcinoma in situ. For both populations, most panelists concluded that evidence was adequate to show that both therapies offer a net benefit over best supportive care. However, they found that the evidence was not adequate to compare the treatments against each other or to show that either provides a benefit over gemcitabine, with or without docetaxel. For the population without carcinoma in situ, most panelists agreed the evidence was not adequate to show a benefit for either therapy over systemic Keytruda (pembrolizumab, Merck and Co. Inc.). Because neither drug is approved by the FDA and has no U.S. commercial price, the Midwest CEPAC did not vote on the long-term value of the treatments. Based on estimates derived from single-arm trials of patient benefits from delay of metastasis and need for cystectomy, ICER calculated annual health benefit price benchmark ranges of approximately $121,000 to $201,000 for nadofaragene firadenovec and $93,000 to $162,000 for oportuzumab monatox.
Khondrion BV, of Nijmegen, the Netherlands, said it joined a consortium of universities, academic hospitals and industrial partners seeking to understand the impact of metabolomic processes on the severity of COVID-19 in patients. The consortium, which received a €1 million (US$1.2 million) grant from the foundation Health~Holland (Top Sector Life Sciences & Health), plans to investigate the metabolic fingerprints of 5,000 to 7,000 patients with COVID-19 to identify markers that could predict the severity of symptoms in those who contract the virus. The consortium will test the effects of existing and new drugs and seek to optimize the diets and dietary supplements of patients. Its investigation will include Khondrion’s lead asset, sonlicromanol, which may show potential as a repurposed treatment for the prostaglandin E2-driven inflammatory consequences underlying severe COVID-19 disease.
Macrogenics Inc., of Rockville, Md., said it agreed to a research collaboration and global license to develop a preclinical bispecific molecule with Janssen Biotech Inc., a unit of Johnson & Johnson, of New Brunswick, N.J., using the Macrogenics Dart platform to enable simultaneous targeting of two targets, which were not disclosed, in a therapeutic area outside oncology. Janssen agreed to pay Macrogenics $20 million up front and to fund all expenses. Macrogenics also is eligible for up to $312 million in potential milestone payments and tiered royalties on global sales.
Medexus Pharmaceuticals Inc., of Toronto, said it formed an exclusive license with Ethypharm SA, of Saint-Cloud, France, covering registration and commercialization of triamcinolone hexacetonide injectable suspension 20 mg/mL in the U.S. The corticosteroid is indicated for intra-articular, intrasynovial or periarticular use in adults and adolescents to treat symptoms of sub-acute and chronic inflammatory joint diseases, including rheumatoid arthritis, juvenile idiopathic arthritis, osteoarthritis and post-traumatic arthritis. Medexus agreed to make an up-front payment to Ethypharm, which was not disclosed, along with milestone payments linked to FDA approval, commercial launch and sales targets. Medexus also will pay a double-digit royalty to Ethypharm on net U.S. sales.
Moderna Inc., of Cambridge, Mass., said the EC exercised its option to purchase 80 million additional doses of mRNA-1273, the company’s COVID-19 vaccine candidate, bringing the EC’s confirmed order commitment to 160 million doses. The first deliveries to European countries from Modena’s dedicated European supply chain are expected to begin early in 2021, subject to regulatory approval by the EMA, whose scientific committee for human medicines plans to meet Jan. 6, 2021, to discuss the candidate.
Ose Immunotherapeutics SA, of Nantes, France, said it received €5.2 million (US$6.4 million) under the PSPC-COVID call for projects operated on behalf of the French government by Bpifrance as part of the Programme d’investissements d’avenir to support the company’s development program on Covepit, its multitarget prophylactic vaccine against COVID-19. The funds are expected to help accelerate the candidate’s development, including initiation of a phase I/II trial expected to begin in the first quarter of 2021. Preclinical studies included a human ex vivo study that compared 120 convalescent COVID-19 patients to unexposed subjects and enabled OSE to identify T memory immuno-dominant epitopes following infection with COVID-19 that were selected for their immunogenicity potential. Funds from the French State also will support production of a clinical batch according to GMP standards.
Scancell Holdings plc, of Oxford, U.K., said it selected COVID-19 vaccine candidate, SN-14, which targets both the SARS-CoV-2 nucleocapsid (N) protein and the key receptor-binding domain of the spike (S) protein, for additional development and human trials. SN-14, which is based on a modification of Scancell’s Immunobody DNA vaccine technology, was selected from among 15 internal candidates with various N and S components that were evaluated for T cell and antibody responses. SN-14 reproducibly elicited high-titer anti-S virus neutralizing antibodies together with high avidity T cells against both proteins. Scancell said the candidate offers the potential to remain effective against new variants of coronavirus and uses the company’s Avidimab technology to enhance the prospect of long-term protection and immunological memory, along with inherent advantages of stable storage and simpler manufacturing processes for DNA vaccines.
Ultragenyx Pharmaceutical Inc., of Novato, Calif., Mereo Biopharma Group plc, of London, said they formed a license and collaboration pact for setrusumab, an anti-sclerostin fully human monoclonal antibody to treat osteogenesis imperfecta (OI). Ultragenyx agreed to make an up-front payment of $50 million to Mereo and to fund global development of the program until approval. Ultragenyx also will pay Mereo up to $254 million upon achievement of certain clinical, regulatory and commercial milestones along with tiered double-digit percentage royalties to Mereo on net sales outside Europe. Mereo will pay a fixed double-digit percentage royalty to Ultragenyx on net sales in Europe. Under terms of its 2015 agreement with Novartis AG, of Basel, Switzerland, Mereo also will pay the pharma a percentage of proceeds, receiving a substantial majority of the payments from Ultragenyx. Data from a phase IIb trial of the candidate conducted by Mereo showed a dose-dependent increase in bone formation, density and strength in adults with OI. The companies plan to expand and initially prioritize the agent’s development to treat children with OI, including the conduct of a pediatric phase II/III study, expected to begin in 2021, to determine an optimal dose based on increases in collagen production using serum P1NP levels and an acceptable safety profile before transitioning into a pivotal phase III that will evaluate fracture reduction over an estimated 15 to 24 months as the primary endpoint, pending regulatory review. A separate pivotal study is planned in adults with OI.
Y-mabs Therapeutics Inc., of New York, said it agreed to a license with Sciclone Pharmaceuticals International Ltd., of Foster City, Calif., as exclusive co-development and commercialization partner in China for the antibodies Danyelza (naxitamab-gqgk), approved by the FDA on Nov. 25, 2020, to treat people with relapsed/refractory high-risk neuroblastoma and, if approved, omburtamab to treat children with central nervous system/leptomeningeal metastasis from neuroblastoma. The license agreement covers Greater China, including Mainland China, Taiwan, Hong Kong and Macau, where Sciclone agreed to employ its development, sales, marketing and regulatory expertise to commercialize the antibodies. Sciclone is set to pay Y-mabs $20 million up front with the potential for up to $100 million in additional development, regulatory and sales milestone payments for both programs as well as double-digit royalties on net sales of the agents in the territory. Y-mabs plans to resubmit the BLA for omburtamab to the FDA by early 2021 to address the agency’s refuse to file letter in October 2020 requesting additional data on certain parts of the CMC and clinical modules.