On August 16, 2021 VBL Therapeutics (Nasdaq: VBLT) reported financial results for the second quarter ended June 30, 2021, and provided a corporate update (Press release, VBL Therapeutics, AUG 16, 2021, View Source [SID1234586640]).

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"With $57.2 million in cash that is projected to fund our operations until year-end 2023, we are excited to advance our OVAL study, with PFS as its additional primary endpoint, towards clinical readout in the second half of 2022," said Dror Harats, M.D., Chief Executive Officer of VBL. "Following the FDA request for additional technical production data on VB-111, we prepared and submitted the requested information in early August and are currently awaiting agency guidance. With several important milestones anticipated through the rest of 2021, we look forward to keeping investors apprised of our progress."

Second Quarter and Recent Corporate Highlights

VB-111

●In June, VBL presented an update on the progress of the OVAL Phase 3 registration-enabling study of VB-111 in ovarian cancer at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The presentation announced an amendment to the primary endpoint in the OVAL study to include a second, separate primary endpoint of PFS in addition to the original primary endpoint of overall survival (OS).
●As part of VBL’s discussion with the Chemistry, Manufacturing, and Controls (CMC) group of the FDA on VB-111 production, it was agreed that VBL would provide additional documentation on new batches to be used in the OVAL study. VBL prepared and submitted the requested documentation to the FDA in early August and is currently awaiting agency guidance.
●As a precautionary step to preserve supply of FDA-approved batches, in June, VBL voluntarily paused enrollment of new U.S. patients in the OVAL study. Existing patients enrolled in the United States continue on protocol and enrollment continues in Europe, Israel, and in recently opened sites in Japan.
Corporate

In April, VBL closed a public offering raising net proceeds of $26.4 million. The Company’s cash position is expected to fund operations until year-end 2023, through the readout from the OVAL study and potential BLA submission for VB-111 in ovarian cancer.
●In July, VBL announced the appointments of Alison Finger and Michael Rice to its Board of Directors.
●In July, the planned succession for Chairmanship of VBL’s Board of Directors was completed. Marc Kozin, who joined the Board as Vice Chairman in October 2020 was appointed Chairman. Former Chairman, Dr. Bennett Shapiro, stepped down as Chairman but will remain a Director.
Financial Results for the Second Quarter 2021

●As of June 30, 2021, VBL had cash, cash equivalents, short-term bank deposits and restricted bank deposits totaling $57.2 million. In April 2021, VBL raised net proceeds of $26.4 million in a public offering of shares and pre-funded warrants (including partial exercise of the underwriters’ overallotment option). VBL expects that its cash, cash equivalents and short-term bank deposits will be sufficient to fund operating expenses and capital expenditure requirements until year-end 2023.
●Revenues for the second quarter 2021 were $188 thousand, as compared to $158 thousand in the comparable period in 2020.
●R&D expenses, net, were $6.6 million for the second quarter compared to $4.7 million in the comparable period in 2020. This increase is due to the clinical development activity of VB-111 for ovarian cancer, in addition to the advancement of VB-601 toward Investigational New Drug Application (IND) submission.
G&A expenses were $1.5 million for the second quarter compared to $1.3 million in the comparable period in 2020.
●VBL reported a net loss for the quarter ended June 30, 2021, of $8.0 million, or ($0.12) per basic share, compared to a net loss of $5.8 million, or ($0.14) per basic share, in the comparable period in 2020.

The live webcast will be available online and may be accessed from the "Events and Presentation" page of VBL’s website. A replay of the webcast will be available beginning approximately one hour after the conclusion of the call and will remain available for at least 30 days thereafter.

On August 16, 2021 Jubilant Therapeutics Inc., a biopharmaceutical company advancing small molecule precision therapeutics to address unmet medical needs in oncology and autoimmune diseases, reported the appointments of Leila Alland, M.D. to its Board of Directors, and Jeremy Barton, M.D. as Strategic Advisor and Interim Chief Medical Officer (Press release, Jubilant Therapeutics, AUG 16, 2021, View Source [SID1234586656]).

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Jubilant Therapeutics Strengthens Board of Directors with Addition of Leila Alland, M.D. and Announces Appointment of Jeremy Barton, M.D., as Strategic Advisor and Interim CMO
Jubilant Therapeutics Strengthens Board of Directors with Addition of Leila Alland, M.D. and Announces Appointment of Jeremy Barton, M.D., as Strategic Advisor and Interim CMO
"It is a pleasure to welcome Dr. Alland to the Jubilant Therapeutics board and Dr. Barton to the team," said Hari S Bhartia, Chairman, Jubilant Therapeutics Inc. "They join at an exciting time as we progress towards the clinic and I look forward to their engagement and support in guiding our objectives."

"Dr. Alland and Dr. Barton’s combined experience in strategic oncology drug development will help propel the company towards our goal to bring innovative small molecule modulators to patients with cancer," said Syed Kazmi, Ph.D., President and Chief Executive Officer of Jubilant Therapeutics Inc.

"The Jubilant Therapeutics team is making great strides in developing precision oncology therapeutics and I am honored to be joining their Board and supporting them in advancing their exciting pipeline into the clinic," said Leila Alland, M.D.

Dr. Alland currently serves as Chief Medical Officer of PMV Pharmaceuticals where she is responsible for leading the company’s clinical stage precision anticancer therapies. Previously, she served as Chief Medical Officer at Affimed and Tarveda Therapeutics and held leadership positions at AstraZeneca, Bristol-Myers Squibb, Novartis and Schering-Plough. Dr. Alland completed her fellowship in pediatric hematology-oncology at the New York Hospital and Memorial Sloan-Kettering Cancer Center and served as Assistant Professor of Pediatrics at Albert Einstein College of Medicine, where she was awarded the James S. McDonnell Foundation Scholar Award and pursued basic cancer research while also caring for children with cancer and blood disorders.

"I am excited to be working with Jubilant Therapeutics and contributing to advancing their pipeline of novel therapeutics to address unmet needs in patients with cancer and autoimmune diseases," said Jeremy Barton, M.D.

Dr. Barton brings to Jubilant Therapeutics over 29 years of experience across all phases of oncology drug development from major pharmaceutical companies in Switzerland, the United Kingdom and the United States. He currently works as a consultant advising biotech companies on strategic oncology drug development. Previously, Dr. Barton served as Chief Medical Officer at Biogen Idec, eFFECTOR Therapeutics, and Mirati Therapeutics. He served as the Head of early Oncology Development at Pfizer for where he oversaw the transition of all oncology compounds from research to IND and then to proof-of-concept studies. Dr. Barton is Board Certified in Internal Medicine and Clinical Oncology, a Member of the Royal College of Physicians, a Fellow of the Royal College of Radiologists (Clinical Oncology) and a Member of Faculty of Pharmaceutical Medicine. He earned his Master’s in Physiology from Oxford University, and his M.D. degree at University College Hospital Medical School in London.

On August 16, 2021 Biosight Ltd., a pharmaceutical development company developing innovative therapeutics for hematological malignancies and disorders, reported the initiation of a Phase 2 trial to evaluate aspacytarabine (BST-236), Biosight’s proprietary antimetabolite, as a second line treatment for patients with relapsed or refractory myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) (Press release, Advaxis, AUG 16, 2021, View Source [SID1234586688]). The investigator sponsored trial will be led by Dr. Pierre Fenaux of the Groupe Francophone des Myélodysplasies (GFM), the French Study Group of the European Myelodysplastic Syndromes (MDS) Cooperative Group (EMSCO). GFM is a non-profit organization comprised of most French hematology centers that conducts and sponsors clinical trials and translational research and coordinates diagnostic and therapeutic guidelines for MDS.

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"This new trial is an important step forward in expanding the reach of aspacytarabine to a broader patient population, addressing the unmet needs in the treatment of relapsed/refractory AML and MDS," said Dr. Ruth Ben Yakar, Chief Executive Officer of Biosight. "Initiating an additional trial furthers our clinical momentum, building on updated, encouraging data from our ongoing first-line Phase 2b study presented at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and the receipt of Orphan Medicinal Product Designation for the European Medicines Agency. GFM is an ideal partner to expand the clinical evaluation into MDS with their extensive network of hematology centers, including registered centers of excellence, and we look forward to applying their expertise in clinical trials, treatments and diagnostics as we advance this Phase 2 trial."

Professor Pierre Fenaux, M.D., Ph.D., Head of Hematology at Hospital Saint Louis in Paris, and founding member and Chairman of GFM, said, "The first-line Phase 2b data that were presented at ASCO (Free ASCO Whitepaper), with demonstrated efficacy across key measures including encouraging complete remission and negative minimal residual disease rates, duration of response and overall survival, further increase my conviction that aspacytarabine may serve as a more tolerable, end effective standard of care treatment for patients with both AML and MDS. We are thrilled to be collaborating with Biosight to advance this potentially transformative treatment for relapsed or refractory MDS and AML patients who currently are faced with poor prognoses and no effective standard of care treatment."

About Aspacytarabine (BST-236)

Aspacytarabine is a novel proprietary anti-metabolite. It is composed of cytarabine covalently bound to asparagine, acting as a pro-drug of cytarabine. Cytarabine serves as the backbone of AML therapy for over 45 years due to its superior efficacy, however, it is associated with severe bone marrow, gastrointestinal, and neurological toxicities, which significantly limit its use, especially in older and medically compromised patients. Due to its unique pharmacokinetics and metabolism, aspacytarabine enables high-dose therapy with lower systemic exposure to free cytarabine and relative sparing of normal tissues. As such, aspacytarabine may serve as a new therapy for AML and other hematological malignancies and disorders, including for older adults who are unfit for intensive therapy.

Aspacytarabine was granted FDA Fast Track Designation for treatment of AML patients unfit for standard chemotherapy, and FDA and EMA Orphan Drug Designations, which entitle Biosight to seven and ten years of market exclusivity in the U.S. and Europe, respectively, upon aspacytarabine marketing approval for the treatment of AML in each territory.

Interim results from an ongoing Phase 2b study evaluating aspacytarabine as a single-agent first-line AML therapy demonstrate safety and single-agent activity, and additional studies are launched to evaluate aspacytarabine as a second line treatment for patients with relapsed or refractory MDS or AML. For more information regarding the Phase 2b clinical study of BST-236, please visit www.clinicaltrials.gov.

On August 16, 2021 Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") reported that it has obtained manufacturing and marketing approval for the EZH2 inhibitor "Tazverik Tablets 200 mg" (tazemetostat hydrobromide) in Japan with the indication of relapsed or refractory EZH2 gene mutation-positive follicular lymphoma (only when standard treatment is not applicable) (Press release, Eisai, AUG 16, 2021, View Source [SID1234586585]).

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This approval is based on the results of a multicenter, open-label, single-arm clinical phase II trial (Study 206)1 in Japan conducted by Eisai and other studies2 conducted by Epizyme, Inc. (Headquarters: Massachusetts, United States) outside Japan. Study 206 enrolled patients with EZH2 gene mutation-positive, primarily follicular lymphoma, who had relapsed or were refractory. The primary endpoint of this study was objective response rate (ORR), and secondary endpoints included safety. This study achieved the primary endpoint target and exceeded a prespecified tumor response threshold with statistical significance: ORR in patients with EZH2 mutation-positive relapsed or refractory follicular lymphoma (n=17) was 76.5% (90% confidence interval (CI): 53.9-91.5) as measured by independent review. Treatment-emergent adverse events (incidence of 25% or more) observed in this study were dysgeusia (52.9%), nasopharyngitis (35.3%), lymphopenia (29.4%) and blood creatine phosphokinase increased (29.4%). Eisai will conduct a post-marketing special use results survey (all-case surveillance) in all patients who are administered "Tazverik" until a pre-determined number of patients has been reached in accordance with an approval condition imposed by the MHLW.

Created by utilizing Epizyme’s proprietary product platform, "Tazverik" is a first-in-class small molecule inhibitor of the epigenetic enzyme EZH2. It is one of the histone methyltransferases in the epigenetics-related protein group, and is thought to regulate the expression of cancer-related genes and suppress the growth of cancer cells by specifically targeting EZH2, which contributes to the cancer growth process.3 Eisai is responsible for the development and commercialization of this agent in Japan, while Epizyme, Inc. is responsible for all regions outside of Japan. In the United States, "Tazverik" received accelerated approval for the indication of epithelioid sarcoma in January 2020, and follicular lymphoma in June 2020 (Notes to editors 1).

Follicular lymphoma is a low-grade B-cell lymphoma that accounts for 10-20% of non-Hodgkin’s lymphomas. Follicular lymphoma is generally indolent and sensitive to chemotherapy. However, development of a new treatment strategy is required for follicular lymphoma which still remains difficult to cure, as recurrence often occurs repeatedly. 7-27% of follicular lymphomas are reported to have gain-of-function mutations in the EZH2 gene,4,5 and it is estimated that there are approximately 600 to 2,400 patients with follicular lymphoma with EZH2 gene mutations in Japan. A companion diagnostic test for EZH2 gene mutations, "cobas EZH2 Mutation Test" by Roche Diagnostics K.K. (Headquarters: Tokyo) was approved in May 2021.
　

Eisai aims to make continuous efforts to meet the diversified needs of and increase the benefits provided to patients with cancer, their families, and healthcare professionals, by delivering "Tazverik" as a new treatment option for EZH2 gene mutation-positive follicular lymphoma.　

1. About "Tazverik Tablets 200 mg" (tazemetostat hydrobromide, Development Code: E7438, Epizyme, Inc.’s Development Code: EPZ-6438)

"Tazverik" is a first-in-class, oral small molecule inhibitor that targets EZH2. Eisai and Epizyme, Inc. have conducted joint research and development with utilizing Epizyme, Inc.’s proprietary product platform, based on the alliance agreement concluded in March 2011 targeting EZH2 for research, development, and sales. This agent selectively inhibits EZH2 in a competitive matter with S-adenosylmethionine (a methyl group donor) to suppress methylation of H3K27. Due to the alteration in the alliance agreement between the two companies in March 2015, Eisai is responsible for development and commercialization of this agent in Japan, while Epizyme, Inc. is responsible for all regions outside Japan.

At the Kawashima Industrial Park located in Gifu Prefecture, Japan, the production site for this agent in Japan, Eisai has successfully developed the innovative pharmaceutical manufacturing technology so-called "Continuous Manufacturing", and has applied it to production. Continuous Manufacturing contributes to quality improvement and stable supply of pharmaceutical products by integrating manufacturing automation and real-time quality monitoring technology. In addition, Continuous Manufacturing is a technology that is attracting attention as a new pharmaceutical manufacturing method that can achieve high production efficiency with space saving and energy saving, and has a low environmental load. Eisai was one of the first to adopt Continuous Manufacturing system (CTS-MiGRA) and applied this technology to the production of this agent.

The accelerated approval was granted for this agent in January 2020 in the United States with the indication of "adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection". Also, in June of the same year, the accelerated approval was granted for this agent with the indication of "adult relapsed / refractory follicular lymphoma who had at least 2 regimens of prior treatment and whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test", and of "adult relapsed / refractory follicular lymphoma for which there are no satisfactory alternative treatment options".

2. About epigenetics

Epigenetics is a branch of science that studies the mechanism for the acquired activation/inactivation of gene function and seeks to determine how gene function is inherited through cell division, irrespective of DNA base sequence alteration. Examples of modification that lead to the regulation of gene expression include methylation of DNA and modifications of histone (methylation, acetylation, phosphorylation, etc.).

3. About EZH2

EZH2 is one of the histone methyltransferases within a larger class of epigenetics-related proteins, and specifically catalyzes the methylation of histone H3 at lysine 27 (H3K27), thus controlling expression of various genes. It is indicated that an increase in methylation of H3K27 caused by EZH2 gain-of-function mutation, overexpression, or the dysfunction of EZH2 suppressive factors plays an important role in carcinogenesis.

1 Shinya Rai, et al. Phase 2 Study of Tazemetostat in Japanese patients with Relapsed or Refractory EZH2 mutation-positive B-cell Non-Hodgkin’s Lymphoma. AACR (Free AACR Whitepaper) Meet. 2021, CT176.

2 Franck Morschhauser, et al. Tazemetostat for patients with relapsed or refractory follicular lymphoma: an open-label, single-arm, multicentre, phase 2 trial. The Lancet Oncology. 2020 Nov; 21(11):1433-1442.

3 Sarah K. Knutson, Satoshi Kawano, Yukinori Minoshima, et al. Selective Inhibition of EZH2 by EPZ-6438 Leads to Potent Antitumor Activity in EZH2-Mutant Non-Hodgkin Lymphoma. Molecular Cancer Therapeutics. 2014 Apr; 13(4):842–854.

4 Csaba Bödör, et al. EZH2 mutations are frequent and represent an early event in follicular lymphoma. Blood, 2013 Oct; 122(18), 3165-3168.

5 Ryan D. Morin, et al. Somatic mutation of EZH2 (Y641) in Follicular and Diffuse Large B-cell Lymphomas of Germinal Center Origin. Nat Genet. 2010 February; 42(2): 181–185.

On August 16, 2021 Carrick Therapeutics, an oncology-focused biopharmaceutical company discovering and developing highly differentiated therapies, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designations to samuraciclib in combination with fulvestrant for CDK4/6i resistant HR+, HER2- advanced breast cancer and samuraciclib in combination with chemotherapy for the treatment of locally advanced or metastatic triple negative breast cancer (TNBC) (Press release, Carrick Therapeutics, AUG 16, 2021, View Source [SID1234586624]).

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"The FDA’s decision to grant Fast Track designations for both samuraciclib combinations underscores the urgent need for innovative therapies that can significantly improve HR+, HER2- advanced breast cancer and locally advanced or metastatic TNBC patient outcomes," said Tim Pearson, Chief Executive Officer of Carrick Therapeutics. "This is a meaningful milestone for our development in samuraciclib as we work to advance innovative combination treatment approaches for patients who have few treatment options available today."

The FDA’s Fast Track program is designed to facilitate and expedite the development of investigational treatments that demonstrate a potential to address unmet medical needs in serious or life-threatening conditions. Programs with Fast Track designation can benefit from early and frequent communication with the FDA in addition to a rolling submission of the marketing application.

Samuraciclib in combination with fulvestrant is currently being evaluated in a Phase 2a study for CDK4/6i resistant HR+, HER2- metastatic breast cancer and the Company expects to present the new data from the ongoing study at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021 in September. Meanwhile, samuraciclib in combination with chemotherapy for the treatment of treatment of TNBC is currently undergoing IND-enabling studies.

About Fast Track Designation
Fast Track is a process designed to facilitate the development, and expedite the review of, drugs to treat serious conditions that address an unmet medical need, by providing a therapy where none exists or providing a therapy which may be potentially better and shows some advantage over available therapy. Fast Track designation includes opportunities for more frequent meetings with the FDA to discuss trial design, development plans, data needed to support drug approval, submission of a New Drug Application (NDA) on a rolling basis, and eligibility for accelerated approval and priority review, if relevant criteria are met.

Visit the FDA’s website for more information on Fast Track Designation.

About Samuraciclib (CT7001)
Samuraciclib is the most advanced oral CDK7 inhibitor in clinical development. Inhibiting CDK7 is a promising therapeutic strategy in cancer as CDK7 regulates the transcription of cancer-causing genes, promotes uncontrolled cell cycle progression and resistance to anti-hormone therapy. Samuraciclib has demonstrated a favorable safety profile and encouraging efficacy in early clinical studies. Samuraciclib has been granted Fast Track designations from the U.S. Food and Drug Administration (FDA) for use in combination with fulvestrant for the treatment of CDK4/6i resistant HR+, HER2- advanced breast cancer and samuraciclib in combination with chemotherapy for the treatment of locally advanced or metastatic triple negative breast cancer (TNBC).