Globenewswire | May 19, 2023
Mustang Bio, Inc. a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers and rare genetic diseases, today announced a strategic update, including anticipated milestones for 2023. Mustang intends to optimize the allocation of its resources and focus on MB-106, MB-109, and in vivo CAR T platform technology. Additionally, Mustang announced a partnership with uBriGene (Boston) Biosciences Inc. (“uBriGene”), the U.S. subsidiary of uBriGene Group, a leading cell and gene therapy contract development and manufacturing organization (“CDMO”), which includes the sale of the Company’s development, manufacturing and analytical testing facility in Worcester, Massachusetts to uBriGene.
Under the terms of an asset purchase agreement between Mustang and uBriGene, uBriGene will acquire Mustang’s state-of-the-art clinical- and commercial-scale cell and gene therapy manufacturing facility in Worcester, Massachusetts, for a total consideration of $11 million. This consideration includes $6 million payable upfront plus an additional $5 million payable upon Mustang raising $10 million in gross proceeds from equity raises following the closing of the transaction. The closing of the transaction is subject to the satisfaction of certain conditions, including approval of transfer of the Company’s lease to uBriGene by the owner of the building (an affiliate of the University of Massachusetts Chan Medical School) and the acceptance of offers of employment with uBriGene or its affiliates by certain key current Mustang employees. Subject to satisfaction of conditions, the Company expects the transactions to close in June 2023.
Subject to closing, the parties will enter into a manufacturing supply agreement, under which uBriGene will manufacture Mustang’s lead product candidates, including continuing to support MB-106 manufacturing for the ongoing multi-center Phase 1/2 trial.
Mustang’s Worcester facility is a 27,000 square foot, cutting edge cGMP facility supporting process development, manufacturing and analytical testing, designed with the flexibility to expand and support various cell and gene therapy production requirements and capacities. uBriGene intends to expand the Worcester site’s capabilities while leveraging Mustang’s experienced staff and robust quality and operating systems to manufacture a broader portfolio of advanced modalities. uBriGene will also offer their expertise in preclinical research services and late-stage and commercial manufacturing of advanced therapy products with respect to product and process characterization, and regulatory inspections.
Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, commented, “We are very pleased to have found a great partner for the manufacturing of our CAR T cell and gene therapies, and we believe that this strategic partnership with uBriGene will meet our portfolio manufacturing needs to reach critical upcoming data inflection points, while extending our cash runway. I want to thank our manufacturing team for their dedication in building and growing our Worcester facility since it opened in 2018. While we are optimizing our resources at Mustang, we look forward to continuing to work with many of our colleagues in this new capacity, as our CDMO.”
“This acquisition is important to uBriGene’s commitment to support the development, clinical, and commercial supply of cell and gene therapies to sustain industry demand and provide new CDMO options,” said Alex Chen, President of uBriGene. “We look forward to working together with the University of Massachusetts Chan Medical School and local biotechnology companies to continue to advance the manufacturing ecosystem in the Greater Boston region. This partnership enables us to expand rapidly to create a North American presence and offer the same high-quality cell and gene therapy development and manufacturing capabilities for the U.S. that we currently provide in Asia, including to support Mustang Bio’s lead clinical-stage CAR-T program.”
Mustang Bio Strategic Portfolio Updates
CAR T Cell Therapies
After a review of its portfolio of product candidates to determine the future strategy of its programs and the proper allocation of its resources, Mustang will discontinue development of its MB-102, its CD123-targeted CAR T cell therapy, as well as its HER2-, CS1- and PSCA-targeted CAR T cell therapy programs, comprising a portion of the Company’s portfolio of CAR T cell therapies being developed by the Company in partnership with City of Hope.
Mustang will continue to work with Fred Hutchinson Cancer Center to develop MB-106 and with Mayo Clinic to develop its in vivo CAR T platform technology. Mustang will also continue to work with City of Hope and with Nationwide Children’s Hospital on the development of MB-109 (MB-101 CAR T cell therapy targeting IL13Rα2 on malignant glioma cells + MB-108 oncolytic virus to potentially make these tumors more susceptible to killing by the CAR-T cells).
Additionally, based on a review of the data from the investigator-sponsored clinical trials of the gene therapy for X-linked severe combined immunodeficiency (“XSCID”) that has been licensed to Mustang Bio, enrollment to these trials has been paused. We await data from new investigator-sponsored trials being planned by our partners that will test a modified version of the current lentiviral vector prior to initiating multicenter Mustang-sponsored trials in both the newborn and previously transplanted patient populations. No safety concerns in the trials utilizing the current vector have been noted to date and no insertional mutagenesis or malignancy has been detected in either of the two investigator-sponsored trials. However, Mustang has made the decision to delay initiating its own sponsored trials out of an abundance of caution, and once we have had the opportunity to review the emerging data from the planned trials utilizing the modified vector, Mustang expects to provide more information on timelines
About Mustang Bio
Mustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR-T therapies across multiple cancers, as well as lentiviral gene therapies for severe combined immunodeficiency. Mustang’s common stock is registered under the Securities Exchange Act of 1934, as amended, and Mustang files periodic reports with the U.S. Securities and Exchange Commission (“SEC”). Mustang was founded by Fortress Biotech, Inc.
INDUSTRIAL IMPACT, MEDICAL
Accesswire | March 24, 2023
Moderna, Inc. a biotechnology company pioneering messenger RNA therapeutics and vaccines, and Generation Bio Co. a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, today announced that the two companies have entered into a strategic collaboration to combine Moderna's biological and technical expertise with core technologies of Generation Bio's non-viral genetic medicine platform. The collaboration aims to expand the application of each company's platform by developing novel nucleic acid therapeutics, including those capable of reaching immune cells, to accelerate their respective pipelines of non-viral genetic medicines.
"Moderna continues to invest in innovative technology to enable us to develop a breadth of transformative medicines for patients," said Rose Loughlin, Ph.D., Moderna's Senior Vice President for Research and Early Development. "Through this collaboration, which builds on Generation Bio's non-viral genetic medicines platform, we have the potential to target immune cells with diverse nucleic acid cargos and the liver for gene replacement. We are excited to have Generation Bio as our partner as we continue to broaden our therapeutic pipeline and extend the potential benefit of nucleic acid therapeutics to more patients."
"Non-viral DNA therapeutics may offer durable, redosable, titratable genetic medicines to patients suffering from rare and prevalent diseases on a global scale," said Phillip Samayoa, Ph.D., Chief Strategy Officer of Generation Bio. "This collaboration represents a foundational investment in our platform science, both deepening our pipeline of rare and prevalent liver disease programs beyond hemophilia A and accelerating our work to reach outside of the liver with nucleic acid therapies. We are thrilled to collaborate with Moderna to extend genetic medicines to new tissues and cell types through the joint development of novel targeting for our stealth ctLNPs to reach immune cells."
About the Collaboration
Under the terms of the agreement, Moderna may advance two immune cell programs, each of which may use a jointly developed ctLNP to deliver ceDNA. In addition, Moderna may advance two liver programs, each of which may use a liver-targeted ctLNP developed by Generation Bio to deliver ceDNA. Moderna retains an option to license a third program for either immune cells or the liver.
Generation Bio will receive a $40 million upfront cash payment and a $36 million equity investment issued at a premium over recent share prices. Moderna will fund all collaboration work, including a research pre-payment. Generation Bio is also eligible for future development, regulatory and commercial milestone payments, as well as royalties on global net sales of liver-targeted and immune cell-targeted products commercialized under the agreement. The agreement additionally provides Moderna with the right, subject to certain terms and conditions, to purchase additional shares of common stock in connection with a future equity financing by Generation Bio.
Further, Moderna and Generation Bio will both leverage collaboration research to continue to advance in vivo immune cell targeting as a new class of genetic medicines, with downstream economics on products utilizing such technology. Generation Bio is eligible to receive certain exclusivity fees as well as potential development and regulatory milestones and royalties on products that Moderna advances using ctLNP technology developed under the collaboration.
In over 10 years since its inception, Moderna has transformed from a research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across seven modalities, a broad intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows for rapid clinical and commercial production at scale. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Moderna's capabilities have come together to allow the authorized use and approval of one of the earliest and most effective vaccines against the COVID-19 pandemic.
Moderna's mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Moderna has been named a top biopharmaceutical employer by Science for the past eight years.
Puma Biotechnology, Inc. | March 16, 2023
Puma Biotechnology, Inc., a biopharmaceutical company, recently announced the online publication of the Phase II TBCRC041 randomized clinical trial results in JAMA Oncology. The trial investigated the use of alisertib, an adenosine triphosphate–competitive and reversible inhibitor of aurora kinase A, alone or in combination with fulvestrant in postmenopausal women with endocrine-resistant, HER2-negative metastatic breast cancer who were previously treated with fulvestrant.
The Phase II randomized clinical trial was conducted via the Translational Breast Cancer Research Consortium and included patients previously treated with CDK 4/6 inhibitors and everolimus.
The trial enrolled 91 evaluable patients, with baseline characteristics balanced between the two arms of the trial. However, more patients in the alisertib plus fulvestrant arm had previously received chemotherapy for metastatic disease (47.8% in the alisertib alone arm vs. 68.9% in the alisertib plus fulvestrant arm). In each arm of the trial, all patients had earlier been treated with CDK 4/6 inhibitors. Everolimus was previously administered to 37% of patients in the alisertib alone arm and 57.8% in the alisertib plus fulvestrant arm.
The trial's efficacy results indicated that nine partial responses were observed in the 46 evaluable patients in the alisertib alone arm, leading to an overall response rate of 19.6%. The median duration of response was 15.1 months, with a clinical benefit rate of 41.3% at 24 weeks. The projected median progression-free survival (PFS) was 5.6 months. Nine of the 45 evaluable patients in the alisertib plus fulvestrant arm of the study responded, for an overall response rate of 20.0%. There was one patient who had a complete response and eight patients who showed partial responses. The median duration of response was 8.5 months, with a clinical benefit rate of 28.9% at 24 weeks. The median PFS was expected to be 5.4 months.
The most prevalent grade 3 or higher adverse events in the alisertib alone arm of the trial were neutropenia (43.4%), anemia (19.6%) and leukopenia (17.4%). The most prevalent grade 3 or higher adverse events in the alisertib plus fulvestrant arm of the study were neutropenia (42.2%), leukopenia (31.1%), lymphopenia (15.6%), fatigue (11.1%), and anemia (8.9%).
About Puma Biotechnology, Inc.
Puma Biotechnology, Inc. is a leading biopharmaceutical company specializing in the development and commercialization of innovative treatments for cancer. Its flagship product is Nerlynx (neratinib), an oral tyrosine kinase inhibitor approved by the USFDA for the extended adjuvant treatment of HER2-positive early-stage breast cancer. In addition to Nerlynx, it has a robust pipeline of product candidates in various stages of development. The company is firmly committed to research and development and collaborates with leading academic institutions and research organizations to advance the understanding of cancer and develop new therapies.