On October 1, 2020 Imago BioSciences, Inc. ("Imago") a clinical-stage biopharmaceutical company developing innovative treatments for myeloid diseases, reported that the first patient has been dosed in the first Imago-sponsored Phase 2b clinical trial of bomedemstat (IMG-7289) for the treatment of essential thrombocythemia (ET) (Press release, Imago BioSciences, OCT 1, 2020, View Source [SID1234567853]).

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"We are excited to start this study. There are many patients with essential thrombocythemia in need of alternatives to the current standards of care. Bomedemstat has the potential to serve as a new option for such patients," said Hugh Young Rienhoff, Jr. MD, CEO, Imago BioSciences.

This Phase 2b multi-center, open-label study is designed to assess the safety, efficacy, and pharmacodynamics of bomedemstat, an oral inhibitor of the epigenetic enzyme lysine-specific demethylase 1 (LSD1) (www.clinicaltrials.gov Identifier NCT04254978). Eligible patients aged 18 or older with ET who have failed at least one standard therapy and require treatment in order to lower their platelet count will be considered for participation in this study. Exploratory assessments include the serial measurement of mutant allele frequencies and changing plasma cytokine profiles. The trial is being conducted in the United States, the United Kingdom, Europe, New Zealand, and Australia.

About Bomedemstat (IMG-7289)

In addition to the ET study, bomedemstat is being evaluated in an open-label Phase 2 clinical trial for the treatment of advanced myelofibrosis (MF), a bone marrow cancer that interferes with the production of blood cells. MF patients who are resistant to, intolerant of, or ineligible for a Janus Kinase (JAK) inhibitor are eligible for the study of bomedemstat as monotherapy. The endpoints include spleen volume reduction and symptom improvement at 12 and 24 weeks of treatment. Preliminary results have been presented at meetings of the European Hematology Association (EHA) (Free EHA Whitepaper) and the American Society of Hematology (ASH) (Free ASH Whitepaper).

Bomedemstat is an orally available small molecule discovered and developed by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme shown to be vital in cancer stem/progenitor cells, particularly neoplastic bone marrow cells. In non-clinical studies, bomedemstat demonstrated robust in vivo anti-tumor efficacy across a range of myeloid malignancies as a single agent and in combination with other chemotherapeutic agents. Bomedemstat is an investigational agent currently being evaluated in ongoing clinical trials (ClinicalTrials.gov Identifier: NCT03136185, NCT04262141 and NCT04081220). Bomedemstat has FDA Orphan Drug and Fast Track Designation for the treatment of myelofibrosis and essential thrombocythemia, Orphan Drug Designation for treatment of acute myeloid leukemia and PRIME designation by the European Medicines Agency for the treatment of MF.

On October 1, 2020 Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO) reported that the open-label Expanded Access Program, sEAPort, for eligible U.S. patients, is formally open (Press release, Oncopeptides, OCT 1, 2020, View Source [SID1234567852]). Melflufen (INN melphalan flufenamide), is currently being evaluated in several clinical studies as a treatment for patients with triple-class refractory multiple myeloma. The sEAPort program is available to adults, age 18 and older, who have received at least two prior lines of therapy and whose multiple myeloma is refractory to at least one proteasome inhibitor, one immunomodulatory drug and one anti-CD38 monoclonal antibody, (i.e., triple-class refractory multiple myeloma patients).

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The Expanded Access Program was initiated following the Company´s June 30 submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration, FDA, for accelerated approval of melflufen in combination with dexamethasone for the treatment of adult patients with triple-class refractory multiple myeloma. The NDA and the sEAPort program are primarily supported by data from the pivotal phase 2 HORIZON study, which demonstrates that melflufen in combination with dexamethasone, has a potential to provide a therapeutic option for patients with relapsed refractory multiple myeloma who are hard to treat and have a poor prognosis, including patients with triple-class refractory multiple myeloma and patients with extramedullary disease.

"Despite therapeutic advances, multiple myeloma remains incurable," said Paula O’Connor, U.S. Head of Medical Affairs at Oncopeptides. "There is an urgent need for more therapies as patients become multi-resistant earlier in their treatment journey. Our Expanded Access Program enables us to provide access to melflufen as a potential treatment for eligible patients while our application is under review by the U.S. Food and Drug Administration."

EAPs are designed to provide patients living with serious or life-threatening conditions access to investigational medicines when no comparable or satisfactory treatment options are available, alternative therapies have been exhausted or the patient is ineligible for ongoing interventional trials.

Forty to fifty medical sites in the U.S. are expected to enroll 100-200 patients in the sEAPort program.

This information was submitted for publication at 08.00 (CET), October 1, 2020.

About melflufen
Melflufen (INN melphalan flufenamide) is a first in class peptide-drug conjugate (PDC) that targets aminopeptidases and rapidly releases alkylating agents into tumor cells. Melflufen is rapidly taken up by myeloma cells due to its high lipophilicity and is immediately hydrolyzed by peptidases to release an entrapped hydrophilic alkylator payload. Aminopeptidases are overexpressed in tumor cells and are even more pronounced in advanced cancers and tumors with a high mutational burden. In vitro, melflufen is 50-fold more potent in myeloma cells than the alkylator payload itself due to the increased intracellular alkylator concentration. Melflufen displays cytotoxic activity against myeloma cell lines resistant to other treatments, including alkylators, and has also demonstrated inhibition of DNA repair induction and angiogenesis in preclinical studies. In the pivotal phase 2 HORIZON study melflufen plus dexamethasone demonstrated encouraging efficacy and a clinically manageable safety profile in heavily pretreated patients with relapsed refractory multiple myeloma, with primarily hematologic Adverse Events (AE) and a low incidence of non-hematologic AEs.

On October 1, 2020 Vaccibody AS, a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel immunotherapies, reported that it has entered into an exclusive worldwide license and collaboration agreement with Genentech, a member of the Roche Group, for the development and commercialization of DNA-based individualized neoantigen vaccines for the treatment of cancers (Press release, Vaccibody, OCT 1, 2020, View Source [SID1234567850]). Vaccibody will conduct development through the end of Phase 1b and Genentech will be responsible for development and commercialization thereafter. The transaction will combine Genentech’s global cancer immunotherapy research, development and commercial leadership with Vaccibody’s targeted DNA-based vaccine platform to realize a potential new treatment paradigm of individualized cancer vaccines.

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Michael Engsig, CEO of Vaccibody, said: "We are very excited to have entered into this transformative agreement that marks the start of a new era for Vaccibody. Genentech is widely recognized as one of the foremost leaders in leveraging the immune system to develop therapies for cancer and is a scientific pioneer within the neoantigen cancer vaccine space. They are therefore the partner of choice for the further development and commercialization of our innovative next-generation cancer vaccine platform for generating individualized therapies."

Under the terms of the agreement, Vaccibody will receive USD 200 million in initial upfront and near-term payments. Additionally, Vaccibody will be eligible to receive up to a further USD 515 million in potential payments and milestones, plus low double-digit tiered royalties on sales of commercialized products arising from the partnership. Following completion of the Phase 1b study, Genentech will have responsibility and bear all costs for clinical, regulatory, manufacturing and commercialization activities.

James Sabry, Global Head of Roche Pharma Partnering said: "We are committed to evaluating emerging classes of cancer immunotherapies, and we believe targeting neoantigens has the 2 potential to transform the treatment landscape for many types of cancers. We are pleased to collaborate with Vaccibody to help realize the full potential of its individualized neoantigen vaccine technology with the shared goal of broadening the number of people who may benefit from immunotherapy treatment."

Through this partnership, Genentech and Vaccibody will progress Vaccibody’s investigational product, VB10.NEO, into clinical trials in the U.S. and in Europe. VB10.NEO, an individualized DNA-based neoantigen vaccine, uniquely targets encoded antigens to antigen presenting cells which are essential for generating potent T cell responses required for cancer therapy. The vaccine is designed to be produced on-demand according to the neoantigen profile of an individual patient. Neoantigens are proteins generated by tumor-specific mutations not present in normal tissues, and are thus an attractive target for cancer immunotherapy as they may be recognized as foreign by the immune system.

"It is widely believed that the clinical use of cancer vaccines has been limited by the ability to efficiently present the antigens to the immune system and the limited insight into what constitutes clinically relevant antigens. Vaccibody’s immunotherapy platform has been shown to address those challenges with preclinical and clinical data indicating induction of unique CD4+ and importantly CD8+ tumor-specific T cell responses against selected antigens essential for clinical responses," said Agnete B. Fredriksen, Co-Founder, President & Chief Scientific Officer of Vaccibody.

The consummation of the transactions contemplated by the agreement is subject to customary closing conditions, including the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act, as amended, and is expected to occur in the second half of 2020.

Vaccibody future R&D focus and strategy (also see separate press release, issued today for further detail) The payments and milestones received under the terms of this licensing agreement will enable Vaccibody to accelerate and expand its other internal R&D programs under the Vaccibody technology platform. As further detailed in the accompanying press release today, Vaccibody will focus its R&D efforts on further development of VB10.16 and explore other shared neoantigen cancer vaccines; infectious diseases vaccines and in addition, leverage its in-house expertise and deep know-how to develop novel immunotherapeutic products in new strategic areas. Webcast Michael Engsig, CEO, and Agnete B. Fredriksen, President & Chief Scientific Officer, will host a webcast on October 01, 2020 at 4 p.m. CET to discuss the partnership with Genentech and 3 provide an update on Vaccibody’s future R&D focus and strategy. A presentation will be available on Vaccibody’s website, www.vaccibody.com, before the webcast.

About VB10.NEO
VB10.NEO, is a proprietary therapeutic DNA vaccine which uses the patient’s own neoantigens for the personalized treatment of cancer patients. VB N-01, a Phase I/IIa neoantigen clinical trial is being conducted for patients with locally advanced or metastatic melanoma, non-small cell lung carcinoma, clear renal cell carcinoma as well as urothelial cancer or squamous cell carcinoma of the head and neck. The drug candidate has demonstrated positive initial responses in patients. VB10.NEO has demonstrated the ability to induce strong tumor specific immune responses which leads to clinical responses in several patients with locally advanced or metastatic disease. Interim results from Phase I/IIa clinical trial suggests a clear link between selection of high quality neoepitopes, generation of strong neoepitope-specific CD8+ T cell responses and clinical responses. VB10.NEO is exclusively licensed to Genentech.

The VB N-01 clinical trial is a multi-centre, open-label clinical trial. The trial is fully enrolled. The clinical trial has the ClinicalTrials.gov Identifier: NCT03548467.