On January 4, 2018 CureVac AG, a leading clinical-stage biopharmaceutical company focused on the development of pioneering mRNA therapeutics, and Arcturus Therapeutics Ltd. (NASDAQ:ARCT), an RNA medicines company, reported they have entered into a broad strategic collaboration to jointly discover, develop and commercialize novel messenger RNA (mRNA) therapeutics (Press release, CureVac, JAN 4, 2018, View Source [SID1234522914]).

Under the agreement, the companies will collaborate to develop up to four molecular therapy products for rare diseases using Curevac’s optimized natural mRNA sequence (RNAoptimizer) and Arcturus’s lipid-mediated nucleic acid delivery system (LUNAR). The agreement focuses on developing mRNA therapeutics for enzyme replacement and antibody generation. Development costs will be shared between the companies, with plans to co-commercialize products in the future under a profit sharing arrangement. The first mRNA therapy to be jointly developed and potentially commercialized by the companies will target ornithine transcarbamylase (OTC) deficiency, a genetic disease characterized by the accumulation of ammonia in the blood. The collaboration also grants CureVac access to the full suite of Arcturus’s lipid-mediated delivery intellectual property to enable the development of additional mRNA product candidates.

"This collaboration for up to four products establishes a sound relationship with Arcturus, which we believe is one of the leaders in developing lipid-mediated delivery systems for mRNA molecules," said Ingmar Hoerr, Ph.D., co-founder and CEO of CureVac. "Just as important, we are excited to have secured access to Arcturus’s leading intellectual property rights for future product development in molecular therapies. This partnership combines both companies’ technology platforms with the expertise necessary to develop the next generation of therapeutics based on the considerable potential of mRNA."

"We are thrilled to combine Arcturus’s platform technologies and expertise with CureVac’s recognized capabilities in mRNA construct optimization and GMP manufacturing to co-develop messenger RNA medicines for patients in need," said Joseph Payne, President and CEO of Arcturus. "We believe our collaboration with CureVac has the potential to help reduce costs, mitigate manufacturing risks, and accelerate our timelines for ushering quality mRNA medicines into the clinic."

On January 4, 2018 Rasna Therapeutics, Inc. (OTCQX: RASP), a clinical stage biotechnology company focused on the development of disease-modifying drugs for hematological malignancies, reported follow up Phase II clinical data showing that 4 out of 9 (44%) evaluable AML patients carrying the NPM1 gene mutation treated with actinomycin D ("Act D"), achieved complete remission (CR) (Press release, Rasna Therapeutics, JAN 4, 2018, View Source [SID1234522893]). Treatment with Act D was well-tolerated except that patients experienced oral mucositis as the major toxicity. Rasna Therapeutics has developed a proprietary nanoparticle based formulation of Act D (RASP-101), which is anticipated to maximize efficacy while minimizing oral mucositis. RASP-101 could potentially be a first-in-class modality for treatment of NPM1-mutated acute myeloid leukemia (AML).
NPM1-mutated AML is a specific genetic leukemia entity that accounts for approximately one-third cases of AML in adults. As we reported previously, intravenous treatment of refractory or relapsed NPM1-mutated AML patients with Act D (12.5 µg or 15 µg/day) for 5 consecutive days produced hematological complete response in some of them (Falini et al., N Eng J Med. 373: 12, 2015).

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In a follow-up phase II clinical study (ActD-AML-PG01, EudraCT 2014-000693-18) in refractory/relapse (R/R) AML patients carrying NPM1 gene mutation, treatment with Act D at 15 µg/kg/day for 5 days every 28 days induced CR in 4 out of the 9 evaluable patients (44.4%) with only 1 or 2 cycles of therapy. Three out of the 4 (75%) patients who obtained CR relapsed after 3, 5 and 7 months, respectively. One patient underwent haploidentical allogeneic PBSC transplantation at 3 months after CR achievement and is alive in molecular CR (MRD-negative) after 24 months.

"The precise mechanism of action of Act D in NPM1-mutated AML is still not clearly understood. Follow up data confirms our earlier findings and further support the use of Act D to induce complete hematological remissions with possible long-term molecular responses in NPM1-mutated AML patients. To note, our first previously reported NPM1-mutated AML patient (resistant to hypomethylating therapy) treated with Act D remains in molecular remission at over 3 years since CR achievement," said Dr. Brunangelo Falini, a member of the scientific advisory board of Rasna Therapeutics, Inc.

"We have developed a proprietary formulated Act D (RASP-101), which we anticipate will produce a superior clinical outcome with improved efficacy and safety profile. Emerging data from the multicenter clinical studies will allow us to develop a biomarker strategy to select responsive patients and improve clinical outcome for this unmet clinical need" commented Alessandro Padova, Chairman of Rasna.

About Actinomycin D (Dactinomycin)
Actinomycin D, also known as dactinomycin, a cytotoxic antibiotic produced by Streptomyces parvullus, was approved for medical use in the United States in 1964. It is on the World Health Organization’s List of Essential Medicines, the most effective and safe medicines needed in a health system. It is believed to work by blocking RNA synthesis. The drug has been used to treat a number of types of cancers, including Wilms tumor, rhabdomyosarcoma, Ewing’s sarcoma, trophoblastic neoplasm, testicular cancer, and certain types of ovarian cancer.

About Dr. Brunangelo Falini, M.D.
Brunangelo Falini is the head of the Institute of Hematology and Hemopoietic Stem Cell Transplantation at the University of Perugia, Perugia, Italy. His research activity has mainly focused on the genetic characterization of lymphomas and leukemias using monoclonal antibodies and, more recently, NGS technologies. He led the research group who discovered NPM1 mutations in AML in 2005 and the BRAF-V600E mutation in hairy cell leukemia in 2011. Both these seminal discoveries have already translated into a better diagnosis and therapy of patients affected by these hematological malignancies. Dr. Falini is the recipient of numerous prestigious prizes, including the "Josè Carreras Award" from EHA (Free EHA Whitepaper) (Barcelona, 2010), the "Leopold Griffuel Prize" from ARC (Paris, 2015) and the "Prize for Excellence in Medicine" from the American-Italian Cancer Foundation (New York, 2017).

On January 4, 2018 CureVac AG, a leading clinical-stage biopharmaceutical company focused on the development of pioneering mRNA therapeutics, and Arcturus Therapeutics Ltd. (NASDAQ:ARCT), an RNA medicines company, reported they have entered into a broad strategic collaboration to jointly discover, develop and commercialize novel messenger RNA (mRNA) therapeutics (Press release, CureVac, JAN 4, 2018, View Source [SID1234522914]).

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Under the agreement, the companies will collaborate to develop up to four molecular therapy products for rare diseases using Curevac’s optimized natural mRNA sequence (RNAoptimizer) and Arcturus’s lipid-mediated nucleic acid delivery system (LUNAR). The agreement focuses on developing mRNA therapeutics for enzyme replacement and antibody generation. Development costs will be shared between the companies, with plans to co-commercialize products in the future under a profit sharing arrangement. The first mRNA therapy to be jointly developed and potentially commercialized by the companies will target ornithine transcarbamylase (OTC) deficiency, a genetic disease characterized by the accumulation of ammonia in the blood. The collaboration also grants CureVac access to the full suite of Arcturus’s lipid-mediated delivery intellectual property to enable the development of additional mRNA product candidates.

"This collaboration for up to four products establishes a sound relationship with Arcturus, which we believe is one of the leaders in developing lipid-mediated delivery systems for mRNA molecules," said Ingmar Hoerr, Ph.D., co-founder and CEO of CureVac. "Just as important, we are excited to have secured access to Arcturus’s leading intellectual property rights for future product development in molecular therapies. This partnership combines both companies’ technology platforms with the expertise necessary to develop the next generation of therapeutics based on the considerable potential of mRNA."

"We are thrilled to combine Arcturus’s platform technologies and expertise with CureVac’s recognized capabilities in mRNA construct optimization and GMP manufacturing to co-develop messenger RNA medicines for patients in need," said Joseph Payne, President and CEO of Arcturus. "We believe our collaboration with CureVac has the potential to help reduce costs, mitigate manufacturing risks, and accelerate our timelines for ushering quality mRNA medicines into the clinic."

On January 4, 2018 Sangamo Therapeutics, Inc. (NASDAQ: SGMO) reported that Dr. Sandy Macrae, CEO of Sangamo, will present at the 36th Annual J.P. Morgan Healthcare Conference on Wednesday, January 10th at 4:00 p.m. PT. Dr. Macrae’s presentation will be followed by a separate Q&A session starting at 4:30 p.m. PT (Press release, Sangamo Therapeutics, JAN 4, 2018, View Source [SID1234522912]). The conference is being held in San Francisco, CA from January 8-11, 2018.

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The presentation and Q&A session will be webcast live and may be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. The presentation will be archived on the Sangamo website for two weeks after the event.

On January 4, 2018 AvantGen reported the successful completion of a first cancer therapeutic antibody discovery program for Tanabe Research Laboratories USA, Inc. (TRL) and the initiation of a second program (Press release, AvantGen, JAN 4, 2018, View Source [SID1234522916]).

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Under the terms of the collaboration, AvantGen is responsible for generating novel antibodies to specified cancer targets using its proprietary human antibody yeast display technology platform. TRL has worldwide rights to develop and commercialize antibodies discovered by AvantGen. AvantGen receives upfront payments and is eligible to receive milestone payments and royalties associated with the development and sale of any products derived from the collaboration.

"AvantGen’s antibody discovery and optimization services have been extensively validated against hundreds of targets primarily through numerous NIH SBIR I and II funded projects. Since the recent introduction of our new, state-of-the-art human antibody discovery and optimization platform, this second project with TRL represents the fifteenth de novo antibody discovery project we have initiated specifically for corporate partners," said Xiaomin Fan, Ph.D., founder and president of AvantGen. "The platform utilizes advantageous technologies, including a robust yeast display system, large natural human antibody database and fully human antibody libraries, for the generation of antibodies to meet challenging design goals for therapeutic, diagnostic and research tool purposes."

"The therapeutic antibody discovery program conducted by AvantGen was highly collaborative and was accomplished faster than expected, without the need to further optimize the antibodies in order to meet TRL’s design goals," said Roland Newman, Ph.D., chief scientific officer for TRL. "We look forward to working together on the second program and others in the future."