On February 3, 2020 GlaxoSmithKline plc reported that the European Medicines Agency (EMA) validated the marketing authorisation application (MAA) for belantamab mafodotin for the treatment of patients with relapsed or refractory multiple myeloma whose prior therapy included an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody (Press release, GlaxoSmithKline, FEB 3, 2020, View Source [SID1234553759]). Belantamab mafodotin was accepted for accelerated assessment by the EMA’s Committee for Human Medicinal Products (CHMP).

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Accelerated assessment is granted if the CHMP determines the treatment is of major interest from a public health perspective and represents a therapeutic innovation. Validation of the MAA confirms that the submission is accepted and begins the formal review process by the CHMP.

The MAA is based on data from the pivotal DREAMM-2 (DRiving Excellence in Approaches to Multiple Myeloma) study. Full results from the study, recently published in The Lancet Oncology, demonstrated a 31% overall response rate (ORR) with a 2.5 mg/kg regimen of single-agent belantamab mafodotin in heavily pre-treated patients with multiple myeloma who were refractory to an immunomodulatory drug and a proteasome inhibitor and were refractory and/or intolerant to an anti-CD38 antibody. The safety and tolerability profile was consistent with previously reported data on belantamab mafodotin. [1]

More than 48,000 people in the European Union were diagnosed with multiple myeloma in 2018.[2] Belantamab mafodotin was granted PRIME designation in 2017 by the EMA, a programme that is intended to facilitate development of investigational medicines that have shown clinical promise for conditions where there is significant unmet need.

About multiple myeloma
Multiple myeloma is the second most common blood cancer and is generally considered treatable, but not curable.[3] Research into new therapies is needed as multiple myeloma commonly becomes refractory to available treatments.[4]

About B-cell maturation antigen (BCMA)
The normal function of BCMA is to promote plasma cell survival by transduction of signals from two known ligands, BAFF (B-cell activating factor) and APRIL (a proliferation-inducing ligand). This pathway has been shown to be important for myeloma cell growth and survival. BCMA expression is limited to B cells at later stages of development. BCMA is expressed at varying levels in myeloma patients and BCMA membrane expression is universally detected in myeloma cell lines.[5]

About the DREAMM clinical trial programme for belantamab mafodotin (GSK2857916)
Belantamab mafodotin is an investigational immunoconjugate comprising a humanised anti-B cell maturation antigen (BCMA) monoclonal antibody conjugated to the cytotoxic agent auristatin F via non-cleavable linker. The drug linker technology is licensed from Seattle Genetics; monoclonal antibody is produced using POTELLIGENT Technology licensed from BioWa.

Belantamab mafodotin is not currently approved for use anywhere in the world.

Trial Name

GSK ID/NCT ID

Status

Design

DREAMM-1

117159/ NCT02064387

Completed

A Phase I Open-label Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics, Immunogenicity and Clinical Activity of Belantamab Mafodotin (GSK2857916) in Subjects with Relapsed/Refractory Multiple Myeloma and Other Advanced Hematologic Malignancies Expressing BCMA

DREAMM-2

205678/ NCT03525678

Active, not recruiting

A Phase II Study to Investigate the Efficacy and Safety of Two Doses of Belantamab Mafodotin (GSK2857916) in Subjects with Relapsed/Refractory Multiple Myeloma Who are Refractory to a Proteasome Inhibitor and an Immunomodulatory Agent and Have Failed Prior Treatment with an Anti-CD38 Antibody

DREAMM-3

207495

Planned

A Phase III Open-Label, Randomized Study to Evaluate the Efficacy and Safety of Belantamab Mafodotin (GSK2857916) Compared to Pomalidomide plus low-dose Dexamethasone (Pom/Dex) in Participants with Relapsed/Refractory Multiple Myeloma

DREAMM-4

205207/ NCT03848845

Recruiting

A Phase I/II Single Arm Open-Label Study to Explore Safety and Clinical Activity of Belantamab Mafodotin (GSK2857916) Administered in Combination with Pembrolizumab in Subjects with Relapsed/Refractory Multiple Myeloma

DREAMM-5

208887/

NCT04126200

Recruiting

A Phase I/II, Randomized, Open-label Platform Study of Belantamab Mafodotin (GSK2857916) with Innovative Combination Anti-Cancer Treatments in Participants with Relapsed/Refractory Multiple Myeloma

DREAMM-6

207497/ NCT03544281

Recruiting

A Phase I/II Randomized Study to Evaluate Safety, Tolerability and Clinical Activity of Belantamab Mafodotin (GSK2857916) Administered in Combination with Lenalidomide plus Dexamethasone (Arm A), or in Combination with Bortezomib plus Dexamethasone (Arm B) in Subjects with Relapsed/Refractory Multiple Myeloma

DREAMM-7

207503

Planned

A Phase III Study of Belantamab Mafodotin (GSK2857916) Administered in Combination with Bortezomib plus Dexamethasone versus Daratumumab, Bortezomib, and Dexamethasone in Participants with Relapsed/Refractory Multiple Myeloma

DREAMM-8

207499

Planned

A Phase III, Multicentre, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of Belantamab Mafodotin (GSK2857916) in Combination with Pomalidomide plus Low-Dose Dexamethasone (BPd) versus Pomalidomide plus Bortezomib and Low-Dose Dexamethasone (PVd) in Participants with Relapsed/Refractory Multiple Myeloma

DREAMM-9

209664/ NCT04091126

Recruiting

A Phase III Study of Belantamab Mafodotin (GSK2857916) Administered in Combination with Bortezomib plus Lenalidomide and Low-Dose Dexamethasone (VRd) vs. VRd in Participants with Newly Diagnosed Multiple Myeloma who are Ineligible for Transplant

DREAMM-10

207500

Planned

A Phase III Study of Belantamab Mafodotin (GSK2857916) Administered in Combination with a Novel Agent versus SoC

ISS / GSK Co-Sponsored Study

209418/ NCT03715478

Recruiting

A Phase I/II Dose-escalation and Dose-expansion Study of Belantamab Mafodotin (GSK2857916) Administered in Combination with Pomalidomide plus Low-dose Dexamethasone in Patients with Relapsed/Refractory Multiple Myeloma Who Have Received Two or More Prior Lines of Therapy That Must Have Included Lenalidomide and a Proteasome Inhibitor

GSK in Oncology
GSK is focused on maximising patient survival through transformational medicines. GSK’s pipeline is focused on immuno-oncology, cell therapy, cancer epigenetics, and synthetic lethality. Our goal is to achieve a sustainable flow of new treatments based on a diversified portfolio of investigational medicines utilising modalities such as small molecules, antibodies, antibody drug conjugates and cells, either alone or in combination.

On February 3, 2020 Catalent, Inc. (NYSE: CTLT), the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, gene therapies, and consumer health products, and MaSTherCell Global, Inc., a technology-focused cell and gene therapy contract development and manufacturing organization to leading cell therapy innovators, reported that Catalent has agreed to acquire MaSTherCell Global, a company backed by Great Point Partners, SFPI-FPIM, and Orgenesis Inc. (NASDAQ:ORGS), for an aggregate of $315 million in cash, subject to a customary adjustments (Press release, Catalent, FEB 3, 2020, https://www.catalent.com/catalent-news/catalent-to-acquire-leading-cell-therapy-company-masthercell-global/ [SID1234553778]).

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"MaSTherCell extends our leadership position in the biotech industry, complements our leading gene therapy offering, and allows us to deliver comprehensive development, manufacturing, analytical, fill-finish, and clinical supply solutions for innovators across the large molecule space," said John Chiminski, Catalent’s Chair & Chief Executive Officer. "Both autologous and allogeneic cell therapies provide important new treatment options, with a rising number expected to gain regulatory approval over the coming years. Catalent is well-positioned to combine MaSTherCell’s expert teams and capabilities with our extensive resources and experience in scaling new platforms, and to help MaSTherCell build-out its development and commercial manufacturing capabilities."

Founded in 2011, MaSTherCell has a 25,000 square-foot facility in Gosselies, Belgium providing clinical services, and construction is in progress on a dedicated 60,000 square-foot adjacent commercial-scale production and fill-finish facility, which is scheduled to open in the fall of 2021 and ultimately add over 250 new jobs to the current team of over 240. Its 32,000 square-foot U.S. facility in Houston, Texas, upon completion of validation activities, will focus on development-scale projects and will employ a team of over 50 experts before the end of the year. MaSTherCell has a growing customer base and offers a unique portfolio of capabilities and technologies that include the development and manufacture of autologous and allogeneic cell therapies, as well as a variety of related analytical services. Its experience includes therapies based on chimeric antibody receptor-engineered T cells (CAR-T), T cell receptors (TCR), tumor-infiltrating lymphocytes (TIL), and mesenchymal stem cells (MSC).

Darren Head, MaSTherCell’s Chief Executive Officer, commented, "MaSTherCell has built comprehensive capabilities to address the complexities of cell therapy production. Like Catalent, our experience has taught us the importance of technology selection, advanced know-how, and quality and regulatory expertise for successful cell therapy programs that are aimed at producing life-altering treatments. We are excited to join the industry-leading team at Catalent and work together to scale-up our business. This combination will strengthen our service offering to our customers and, in the end, benefit patients who need innovative life-saving therapies. Catalent’s backing will position MaSTherCell for growth and enhance Catalent’s existing presence in Belgium."

Vered Caplan, Chief Executive Officer of Orgenesis, stated, "We are very proud of the developments and accomplishments at MaSTherCell, which has become a best-in-class contract development and manufacturing organization (CDMO) servicing many of the leading cell and gene therapy companies. Since we completed our acquisition of MaSTherCell in 2015, the business has grown along with this burgeoning industry. With Catalent’s expertise and resources, we believe MaSTherCell will be well positioned to continue on its current trajectory in order to meet the evolving needs of the industry."

Noah Rhodes, Managing Director at Great Point Partners, commented, "We identified MaSTherCell as the leading provider of development and manufacturing services to cell and gene therapy companies, and have enjoyed watching the company and its team continue to flourish. We cannot think of a better partner for MaSTherCell than Catalent, as the combined company will be a leading vertically-integrated provider able to serve customers and patients on a global basis."

Upon completion of the transaction, which is expected in the current, third quarter of Catalent’s 2020 fiscal year and is subject to customary closing conditions, MaSTherCell’s 240 talented employees, including its outstanding leadership team, will become part of the Catalent Biologics business.

The acquisition is an all-cash transaction, which Catalent expects to finance with either a partial drawn down of its revolving credit facility or the proceeds from future capital-raising activity. As part these activities, Catalent has obtained $200 million of committed financing, subject to customary conditions, from JPMorgan Chase Bank N.A. It should be noted, however, that the agreement governing the acquisition is not contingent on any financing activity. Catalent intends to file a Current Report on Form 8-K with the Securities and Exchange Commission, which will have further details concerning the acquisition and the committed financing.

On February 3, 2020 CTI BioPharma Corp. (Nasdaq: CTIC) reported that following a meeting with the U.S. Food and Drug Administration ("FDA" or "the Agency"), CTI has reached agreement on an accelerated approval pathway for pacritinib for the treatment of myelofibrosis patients with severe thrombocytopenia (platelet counts <50,000/µL) (Press release, CTI BioPharma, FEB 3, 2020, View Source [SID1234553794]). CTI will be amending the PACIFICA pivotal Phase 3 trial protocol to allow for the primary analysis of SVR rates on the first 168 patients, with an end-of-study analysis of TSS and OS following the full enrollment of 348 patients. If the primary endpoint of SVR is met following the planned review of data from the first 168 patients, CTI intends to submit a New Drug Application (NDA) under the FDA’s subpart H regulations, subject to review of all available efficacy and safety data. Conversion to a regular approval of pacritinib would be anticipated following the successful end-of-study assessment of the secondary efficacy endpoints, and the completion of post-marketing requirements.

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"Since the initiation of the PACIFICA trial in September 2019, we have been working diligently with the FDA to identify an expedited approval pathway for pacritinib for the treatment of myelofibrosis patients with severe thrombocytopenia," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI Biopharma. "Severely thrombocytopenic myelofibrosis patients (platelet counts <50,000/µL) have reduced survival and very limited therapeutic options. Pacritinib has now demonstrated clinical benefit in this population in three clinical trials, including two prior randomized Phase 3 studies, so we believe that pacritinib has the potential to change the treatment paradigm in this area of serious unmet medical need."

Based on the new trial design, CTI expects to report primary SVR data by the end of 2021, with a potential NDA filing in early 2022 if the SVR data is positive. Final study efficacy data is expected in 2023.

Concurrent with this press release, CTI is announcing a $60 million rights offering. For further details, see the concurrent press release relating to the rights offering.

About Myelofibrosis and Severe Thrombocytopenia
Myelofibrosis is a type of bone marrow cancer that results in formation of fibrous scar tissue and can lead to severe anemia, weakness, fatigue and an enlarged spleen and liver. Patients with severe thrombocytopenia are estimated to make up more than one-third of patients treated for myelofibrosis, or approximately 17,000 people. Severe thrombocytopenia, defined as blood platelet counts of less than 50,000 per microliter, has been shown to result in overall survival rates of just 15 months. Thrombocytopenia in patients with myelofibrosis is associated with the underlying disease but has also been shown to correlate with treatment with ruxolitinib, which can lead to dose reductions, and as a result, may potentially reduce clinical benefit. Survival in patients who have discontinued ruxolitinib therapy is further compromised, with an average overall survival of seven to 14 months. There are currently no approved therapies available to treat myelofibrosis patients with severe thrombocytopenia or patients who have failed ruxolitinib treatment, thereby making this a significant unmet medical need.

About Pacritinib
Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. The JAK family of enzymes is a central component in signal transduction pathways, which are critical to normal blood cell growth and development, as well as inflammatory cytokine expression and immune responses. Mutations in these kinases have been shown to be directly related to the development of a variety of blood-related cancers, including myeloproliferative neoplasms, leukemia and lymphoma. In addition to myelofibrosis, the kinase profile of pacritinib suggests its potential therapeutic utility in conditions such as acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), and chronic lymphocytic leukemia (CLL), due to its inhibition of c-fms, IRAK1, JAK2 and FLT3.

On February 3, 2020 AskAt reported that it received a notice of allowance dated January 22, 2020 from the Mexican Institute of Industrial Property (IMPI: Instituto Mexicano de la Propiedad Industrial) in connection with the Company’s Mexican Patent Application No. MX/a/2014/004566, a formulation patent for its COX-2 inhibitor (AAT-076) (Press release, AskAt, FEB 3, 2020, View Source [SID1234553760]).

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On February 3, 2020 Genprex, Inc.("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company utilizing a unique, non-viral proprietary platform designed to deliver tumor suppressor genes to cancer cells, reported the Company will present at the Noble Capital Markets 16th Annual Investor Conference on Monday, February 17, 2020 at 1:30 p.m. EST (Press release, Genprex, FEB 3, 2020, View Source [SID1234553779]). The conference is being held on February 16-18, 2020 at the Seminole Hard Rock Hotel & Casino in Hollywood, Florida.

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Event: NobleCon16 – Noble Capital Markets’ 16th Annual Investor Conference

Presentation Date: Monday, February 17, 2020

Presentation Time: 1:30 p.m. EST – Terrace Ballroom A

Location: Seminole Hard Rock Hotel & Casino – Hollywood, FL

Mr. Rodney Varner, Chief Executive Officer of Genprex, will deliver an overview of the Company and provide updates on its product pipeline, including the recent Fast Track Designation received from the Food and Drug Administration for its Oncoprex immunogene therapy in combination with AstraZeneca’s Tagrisso. To arrange a one-on-one meeting with the Company, please email [email protected].

A high-definition video webcast of Mr. Varner’s presentation will be available for playback at the link below approximately 24 hours following his live address, and it will be archived for 90 days following the conference:

Presentation Webcast Playback: http://bit.ly/2S5AR7Z

This is the 16th year Noble Capital Markets has hosted "NobleCon" and has invited emerging growth companies to present to and meet with attending investors. NobleCon will feature approximately 125 small growth companies presenting across four tracks and will be broadening access to investors worldwide by hosting the conference site on Channelchek.com, its web-based market platform that emphasizes emerging growth companies.