Industrial Impact

SIGA Announces Oncology Collaboration with KaliVir Immunotherapeutics

SIGA Technologies Inc. | July 16, 2022 | Read time : 2 min

SIGA Announces
SIGA Technologies, Inc. a commercial-stage pharmaceutical company focused on the health security market, today announced a collaboration with KaliVir Immunotherapeutics to make TPOXX® available for use with KaliVir’s proprietary oncolytic vaccinia immunotherapy platform. This novel oncolytic platform includes multiple proprietary genetic modifications that can be combined to generate a unique oncolytic virus that has been optimized for systemic delivery and anti-tumor immune stimulation. Under this partnership, SIGA is providing its TPOXX oral capsules to support future clinical programs.

KaliVir is an innovator in the creation of oncolytic viral immunotherapies, and we are excited to enter into this collaboration with them. TPOXX is a powerful antiviral drug to vaccinia and allows the safe use of higher doses of vaccinia vectors; there is also the potential it could increase immunotherapeutic outcomes. This collaboration helps bring new levels of assurance to physicians, regulators, and especially patients receiving these promising investigational therapies.”

Dr. Phil Gomez, CEO of SIGA

“We are pleased to announce this collaboration with SIGA Technologies,” said Helena Chaye, Ph.D., J.D., CEO of KaliVir. “Pairing oncolytic immunotherapies with an effective antiviral agent is a critical part of the development of new treatments, and we look forward to enhancing our groundbreaking oncolytic immunotherapy programs with the support of SIGA’s TPOXX.”

On July 13, 2018, the U.S. Food and Drug Administration (FDA) approved oral TPOXX for the treatment of smallpox to mitigate the impact of a potential outbreak or bioterror attack. In preclinical studies, TPOXX has been shown to be active against most orthopoxviruses, including vaccinia The unique mechanism of action of TPOXX coupled with published efficacy in animal studies, make it an important addition to development programs focused on vaccinia-based cancer therapies. In 2020, SIGA entered into numerous collaborations, including a partnership with Turnstone Biologics to supply TPOXX to support Turnstone’s clinical oncolytic vaccinia immunotherapy programs. In 2021, SIGA entered into a preclinical research collaboration with Bioarchitech to investigate TPOXX enabling higher doses of vaccinia vectors when used in combination with Bioarchitech’s oncolytic vaccinia-based immunotherapy platform.

SIGA Technologies, Inc. is a commercial-stage pharmaceutical company focused on the health security market. Health security comprises countermeasures for biological, chemical, radiological and nuclear attacks (biodefense market), vaccines and therapies for emerging infectious diseases, and health preparedness. Our lead product is TPOXX®, also known as tecovirimat and ST-246®, an orally administered and IV formulation antiviral drug for the treatment of human smallpox disease caused by variola virus. TPOXX is a novel small-molecule drug and the US maintains a supply of TPOXX under Project BioShield. The oral formulation of TPOXX was approved by the FDA for the treatment of smallpox in 2018. The full label is available by clicking here. Oral tecovirimat received approval from the European Medicines Agency (EMA) in 2022. The EMA approval includes labeling for oral tecovirimat indicating its use for the treatment of smallpox, monkeypox, cowpox, and vaccinia complications following vaccination against smallpox. The full label is available by clicking here. In September 2018, SIGA signed a contract with the Biomedical Advanced Research and Development Authority (BARDA), part of the office of the Assistant Secretary for Preparedness and Response within the U.S. Department of Health and Human Services, for additional procurement and development related to both oral and intravenous formulations of TPOXX. 

KaliVir Immunotherapeutics is a privately held biotech company developing cutting-edge, next-generation oncolytic viral immunotherapy programs. The company has developed a unique vaccinia virus-based platform that can generate potent novel oncolytic vaccinia viruses with modifications to maximize viral replication and to enhance intravenous delivery and spread (Vaccinia Enhanced Template “VET” Platform). VET™ platform utilizes the large transgene capacity of the vaccinia virus to deliver therapeutics matched to tumor immunophenotypes to stimulate patients’ immune systems and modify the tumor microenvironment. KaliVir’s oncolytic product candidates are designed to be safe, potent and systemically deliverable to treat cancer patients across multiple tumor types. KaliVir is in the process of advancing multiple therapeutic candidates toward the clinic.


Bioprinting with living human cells is transforming healthcare as we know it. From Slurpee-like substances to molding 3D tissues, explore the cutting-edge world of 3D bioprinting. Dr. Mark Skylar-Scott from Stanford University discusses the fascinating realm of 3D bioprinting, a groundbreaking approach that goes beyond traditional plastic printing.


Bioprinting with living human cells is transforming healthcare as we know it. From Slurpee-like substances to molding 3D tissues, explore the cutting-edge world of 3D bioprinting. Dr. Mark Skylar-Scott from Stanford University discusses the fascinating realm of 3D bioprinting, a groundbreaking approach that goes beyond traditional plastic printing.

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Exacta Bioscience and Ginkgo Bioworks Announce Partnership to Optimize Exacta's Crop Protection Product

prnewswire | September 01, 2023

Exacta Bioscience, a biotech company that harnesses the power of nature and its microorganisms for crop protection, and Ginkgo Bioworks which is building the leading platform for cell programming and biosecurity, today announced that Exacta is leveraging Ginkgo's end-to-end agricultural R&D services to run a fermentation optimization and scale-up program to enhance one of its crop protection products, FitoRoot®, paving the way for large-scale commercialization. FitoRoot, a biostimulant product composed of three Bacillus strains, is designed to help crops better absorb water, nutrients and strengthen their defenses against abiotic stress. This can lead to overall better crop health, improved yield and increased resistance to stress conditions such as drought or extreme temperatures. Through this partnership, Exacta will use Ginkgo's fermentation and formulation optimization services to reduce the cost of goods to make its FitoRoot technology more competitive. Accordingly, FitoRoot is currently marketed in Chile and is expected to expand to other Latin American markets, with a planned launch in 2023 by Adama Chile, who is driving biological adoption in the region. Adama Ltd is one of the world's leading crop protection companies. "We chose Ginkgo because their deep expertise in agricultural product development and optimization positions us to make one of our key crop protection products, FitoRoot, more accessible," said José Bustos, CEO at Exacta Bioscience. "We're proud of the progress Exacta has made, and this partnership marks a vital step toward accelerating our efforts to assure the quality and availability of one of our products and creating a stable and safer ecosystem." "Exacta Bioscience's emphasis on microbial solutions in crop health brings more sustainable and differentiated solutions to farmers throughout the Americas," said Magalie Guilhabert, Head of Ag Biologicals, Ginkgo Bioworks. "Together, we can continue to push the boundaries of agricultural innovation through biotechnological solutions that promote a more sustainable future for our food systems." Ginkgo's end-to-end agricultural services can help R&D teams develop more effective biological products across a variety of applications. Find out more. About Ginkgo Bioworks Ginkgo Bioworks is the leading horizontal platform for cell programming, providing flexible, end-to-end services that solve challenges for organizations across diverse markets, from food and agriculture to pharmaceuticals to industrial and specialty chemicals. Ginkgo's biosecurity and public health unit, Concentric by Ginkgo, is building global infrastructure for biosecurity to empower governments, communities, and public health leaders to prevent, detect, and respond to a wide variety of biological threats. About Exacta Bioscience Exacta Bioscience is a pioneering biotech company that uses microorganisms to create innovative bioproducts for agriculture. Their nature-inspired solutions support a future without chemical pesticides and antibiotics. With extensive knowledge of microorganisms and their effects on crops and soil, Exacta tackles modern farming challenges. Their expertise is in formulating microorganism mixtures, providing a wide range of bioproducts, specifically for crop protection. They are dedicated to delivering effective, stable, and safe crop protection and soil-enhancing products. They invite everyone to join in advancing sustainable practices for a healthier future.

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Medical, Industry Outlook

Mustang Bio Announces First Data from Ongoing Multicenter Phase 1/2 Clinical Trial Evaluating MB-106 CAR-T Cell Therapy

Globenewswire | August 17, 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 the first data from the indolent lymphoma cohort of the Company’s ongoing multicenter Phase 1/2 clinical trial evaluating MB-106, a first-in-class CD20-targeted, autologous CAR-T cell therapy for the treatment of relapsed or refractory B-cell non-Hodgkin lymphomas (“B-NHL”) and chronic lymphocytic leukemia (“CLL”), demonstrating clinical responses as well as safety and efficacy consistent with the ongoing Phase 1/2 clinical trial taking place at Fred Hutchinson Cancer Center (“Fred Hutch”). Initial data were presented by Mazyar Shadman, M.D., M.P.H., Study Chair, Associate Professor and physician at Fred Hutch and University of Washington, at the 5th International Workshop on CAR-T and Immunotherapies (“iwCAR-T”). Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, “We are encouraged that the first data from the multicenter trial of our lead candidate, MB-106, show clinical responses and that the trial is on track to achieve results consistent with those from the ongoing trial taking place at Fred Hutch. Overall, MB-106 continues to exhibit high efficacy and a favorable safety profile compared to currently approved autologous CAR-Ts. We expect to provide an additional update on dose escalation and report response data at a major medical meeting later this year.” The multicenter study data show clinical responses in four of four patients with relapsed or refractory indolent NHL at the starting dose of 3.3 x 106 CAR-T cells/kg, a dose comparable to that employed for the majority of the indolent lymphoma patients in the Fred Hutch trial. The multicenter data also show persistence of CAR-T cells at 6+ months and favorable safety data with only Grade 1 cytokine release syndrome (“CRS”) reported to date. Two patients with follicular lymphoma had complete response (“CR”) by both PET-CT and bone marrow, one of whom had been previously treated with a CD19-directed CAR-T. A third patient, with a diagnosis of Waldenstrom macroglobulinemia (“WM”), who had nine prior treatments and high disease burden, achieved complete metabolic response by PET-CT, morphologic clearance of lymphoma in bone marrow, and resolution of the IgM monoclonal protein. The fourth patient, with a diagnosis of hairy cell leukemia variant, who had been heavily transfusion dependent, continues to have stable disease with decreased disease in his bone marrow and achieved complete transfusion independence, which is ongoing at 6+ months. “MB-106 continues to show potential as an immunotherapy option for patients with a wide range of hematologic malignancies, including patients previously treated with CD19-directed CAR-T cell therapy,” said Mazyar Shadman, M.D., M.P.H., who holds the Innovators Network Endowed Chair at Fred Hutch and is the Study Chair, as well as Associate Professor and physician at Fred Hutch and University of Washington. “We are excited that the first data from the expanded evaluation of MB-106 are similar in safety as what we’ve seen to date in the ongoing Phase 1/2 clinical trial at Fred Hutch. Additionally, the data from the ongoing clinical trial at Fred Hutch continue to demonstrate a high rate of complete and durable responses.” Dr. Shadman also presented data from the ongoing Fred Hutch Phase 1/2 clinical trial, specific to two B-NHL cohorts, follicular lymphoma (“FL”) and WM. In the FL data cohort (n=20), an overall response rate (“ORR”) of 95% was seen, of which 80% of patients experienced a CR, and 15% had a partial response. The CR patients include a patient who was previously treated with a CD19-directed CAR-T cell therapy. Of the six patients who experienced CRS, only one had Grade 2. Ten patients continue to experience CR for more than 10 months, four patients have experienced CR for more than two years (all ongoing), and the first patient enrolled has sustained CR for more than 3 years. In the WM cohort (n=6), all of whom had received prior Bruton tyrosine kinase inhibitor, two patients experienced CR, one of whom continues to be in CR at more than 22 months. No patients experienced CRS or immune effector cell-associated neurotoxicity syndrome over Grade 2. None of the six WM patients have needed to start new therapy for their disease. As previously reported, Mustang plans to treat patients with WM in the Phase 1 portion of its multicenter clinical trial to support a fast-to-market Phase 2 strategy for this indication and received Orphan Drug Designation from the U.S. Food and Drug Administration (“FDA”). There is currently no FDA-approved CAR-T cell therapy for WM, and the first patient in the pivotal Phase 2 WM trial is expected to be treated in mid-2024, which could enable top-line data as early as mid-2026. By the end of 2023, we anticipate confirming this strategy at an end-of-Phase 1 meeting with the US Food and Drug Administration (FDA). Furthermore, Mustang anticipates requesting regenerative medicine advanced therapy (RMAT) designation for WM from the FDA in 2024. Finally, data from the Fred Hutch clinical trial also support the potential of MB-106 to be administered as outpatient therapy and provide a best-in-class immunotherapy option for patients treated previously with CD19-directed CAR-T cell therapy. About Mustang’s Multicenter MB-106 Phase 1/2 Clinical Trial The five-center Phase 1/2 clinical trial is a three-arm study targeting CLL and B-NHL, including FL, diffuse large B-cell lymphoma and mantle cell lymphoma. We anticipate adding a sixth center by the end of 2023. The Mustang-sponsored multicenter clinical trial is using the same lentiviral vector as the Fred Hutch-sponsored single-center trial. Included in the eligibility criteria are patients who have relapsed after treatment with CD19 CAR-T cell therapy. Additionally, the FL arm will evaluate other indolent histologies including Waldenstrom macroglobulinemia, a rare type of B-NHL for which the U.S. Food and Drug Administration granted MB-106 Orphan Drug Designation. Patients will be enrolled in one of three arms, based on their primary diagnosis; escalating MB-106 dose levels will be tested independently in each arm using a 3+3 design. A total of up to 18 patients are anticipated to be treated in each Phase 1 arm, including six patients at the maximum tolerated dose in each independent arm. Safety of each dose level will be reviewed for each arm until the maximum tolerated dose has been reached and the recommended Phase 2 dose (“RP2D”) has been established for each arm. An assessment of the safety and tolerability of the dose will be made by the Safety Review Committee based on the data from the 28-day dose-limiting toxicity observation period. In Phase 2, specific arms of relapsed or refractory CD20-positive B-cell hematologic malignancies will be treated with MB-106 at the respective RP2D for each arm. The two top priorities are WM and diffuse large B-cell lymphoma (DLBCL) relapsed from prior CD19 CAR-T therapy. Each arm will initially include up to 20 patients. Based on the results of the interim analysis, up to an additional 51 patients may be added to each of the arms. About MB-106 (CD20-targeted autologous CAR-T Cell Therapy) CD20 is a membrane-embedded surface molecule which plays a role in the differentiation of B-cells into plasma cells. The CAR-T was developed by Mustang’s research collaborator, Fred Hutch, in the laboratories of the late Oliver Press, M.D., Ph.D., and Brian Till, M.D., Associate Professor in the Clinical Research Division at Fred Hutch, and was exclusively licensed to Mustang in 2017. The lentiviral vector drug substance used to transduce patients’ cells to create the MB-106 drug product produced at Fred Hutch has been optimized as a third-generation CAR derived from a fully human antibody. MB-106 is currently in a Phase 1/2 open-label, dose-escalation trial at Fred Hutch in patients with B-NHLs and CLL. The same lentiviral vector drug substance produced at Fred Hutch is used to transduce patients’ cells to create the MB-106 drug product produced at Mustang Bio’s Worcester, MA, cell processing facility for administration in the multicenter Phase 1/2 clinical trial under Mustang Bio’s IND. It should be noted that Mustang Bio has introduced minor improvements to its cell processing to facilitate eventual commercial launch of the product. In addition, prior to commercial launch, Mustang Bio will replace the Fred Hutch lentiviral vector drug substance with vector produced at a commercial manufacturer. Additional information on the trials can be found at using the identifier NCT05360238 for the multicenter trial and NCT03277729 for the ongoing trial at Fred Hutch. On May 18, 2023 Mustang Bio entered into an Asset Purchase Agreement, as amended by the First Amendment, dated as of June 29, 2023 and a Second Amendment, dated as of July 28, 2023 pursuant to which it agreed to sell its assets primarily pertaining to the manufacturing and production of cell and gene therapies located at its cell processing facility in Worcester, MA; and, subject to the satisfaction of certain conditions, its leasehold interest in that facility. Concurrent with the Second Amendment, Mustang closed the transaction under the terms of the amended asset purchase agreement and entered into manufacturing services agreements with the purchaser to provide for the continued production of the MB-106 drug product. For additional information, please refer to the Form 8-Ks filed by Mustang Bio with the U.S. Securities and Exchange Commission (“SEC”) on May 22, 2023, June 30, 2023 and July 31, 2023. 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 difficult-to-treat cancers 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.

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Medical, Industry Outlook

QurAlis and Unlearn Announce Collaboration to Accelerate and Optimize ALS Clinical Trials With Generative Artificial Intelligence Technologies

PRNewswire | June 28, 2023

QurAlis Corporation, a clinical-stage biotechnology company developing breakthrough precision medicines for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases with genetically validated targets, and Unlearn, a pioneering technology company innovating machine learning to revolutionize medical research, today announced they have entered into a collaboration to accelerate and optimize QurAlis' clinical program in ALS with Unlearn's advanced generative artificial intelligence (AI) technology. Unlearn develops digital twins of clinical trial patients that are predictions of individual health outcomes under the control treatment over time. Digital twins are employed in randomized controlled trials (RCTs) called TwinRCTs to run more efficient trials that produce regulatory-suitable evidence. "Advances in machine learning and AI make it possible to enhance trial power to detect a positive result when one truly exists while controlling for Type-1 error and significantly shorten timelines without introducing bias into the study," said Kasper Roet, Ph.D., founder and chief executive officer (CEO) of QurAlis. "We are excited to partner with Unlearn to help advance our clinical program with AI and other innovative technologies to generate evidence suitable for supporting regulatory decisions and help speed new, lifesaving precision medicines to patients with ALS and other neurodegenerative diseases." The collaboration aims to reduce variability and increase the study power in QurAlis' clinical trials for QRL-201 and QRL-101, the Company's lead product candidates in ALS. Unlearn's patented machine learning models are trained on existing clinical data. After validation, they are used to generate digital twins from baseline data for each patient enrolled in a TwinRCT, regardless of their randomization assignment. Prognostic scores derived from digital twins are incorporated into the trial's primary analysis to precisely estimate treatment effects and control for Type-1 error. "By using machine learning to leverage the wealth of existing patient data from completed clinical trials, our technology significantly shortens typical timelines by months while generating evidence suitable for supporting regulatory decisions," said Charles Fisher, Ph.D., founder and CEO of Unlearn. "The AI technology we're developing today will revolutionize the future of clinical research." QRL-201 is a first-in-class therapeutic product candidate aiming to restore STATHMIN-2 (STMN2) expression in ALS patients. STATHMIN-2 is a well-validated protein important for neural repair and axonal stability, the expression of which is significantly decreased in nearly all ALS patients. QRL-201 rescues STMN2 loss of function in QurAlis ALS patient-derived motor neuron disease models in the presence of TDP-43 pathology. QRL-201 recently entered the clinic in the first-ever clinical trial to evaluate a therapy that rescues STMN2 in people with ALS. QRL-201 is the second program in QurAlis' pipeline to enter the clinic recently. In December 2022, QurAlis announced the Company initiated dosing of QRL-101 in a first-in-human Phase 1 clinical trial (NCT05667779). QRL-101 is a first-in-class selective Kv7.2/7.3 ion channel opener for the treatment of hyperexcitability-induced disease progression in ALS. About QurAlis Corporation QurAlis is trailblazing the path to conquering amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases with genetically validated targets with next-generation precision medicines. QurAlis' proprietary platforms and unique biomarkers enable the design and development of drugs that act directly on disease-causing genetic alterations. Founded by an internationally recognized team of neurodegenerative biologists from Harvard Medical School and Harvard University, QurAlis is advancing a deep pipeline of antisense oligonucleotides and small molecule programs including addressing sub-forms of ALS that account for the majority of ALS patients. About Unlearn Unlearn is a San Francisco-based technology company pioneering AI-generated digital twins that forecast health outcomes for individual patients. Founded in 2017, Unlearn brings together a team of world-class innovators at the intersection of artificial intelligence, clinical, and regulatory science to transform the future of medicine through generative AI. Unlearn's technology is regulatory-qualified and used by leading global pharmaceutical companies to run AI-powered clinical trials that reach full enrollment faster and bring new treatments to patients sooner.

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