On August 21, 2020 Ono Pharmaceutical Co., Ltd. reported that ONO received a supplemental approval for Velexbru (generic name: tirabrutinib hydrochloride) Tablet 80mg ("Velexbru"), a Bruton’s tyrosine kinase ("BTK") inhibitor, in Japan for additional indication of Waldenstrom macroglobulinemia and lymphoplasmacytic lymphoma, for a partial change in approved items of the manufacturing and marketing approval (Press release, Ono, AUG 21, 2020, View Source [SID1234584587]).

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This approval is based on the result from a multi-center, open-label, single-arm Phase II study (ONO4059-05), evaluating Velexbru in patients with previously untreated, or relapsed or refractory Waldenstrom macroglobulinemia (WM) and lymphoplasmacytic lymphoma (LPL). In 27 patients who received tirabrutinib (untreated 18 patients and relapsed or refractory 9 patients) in this study, the overall response rate (partial response or greater) assessed by an independent review committee (IRC), a primary endpoint, was 88.9% (16/18 patients) (95% CI: 65.3 – 98.6) in the untreated group, and 88.9% (8/9 patients) (95% CI: 51.8 – 99.7) in the relapsed/refractory group. The secondary endpoints of progression-free survival (PFS) and overall survival (OS) were 100% at 6 months both in the untreated group and relapsed/refractory group. The most commonly observed grade ≥3 adverse events (AEs) were neutropenia and lymphopenia (11.1% each), and leukopenia (7.4%).

With this approval, Velexbru becomes the first BTK inhibitor approved for the treatment of patients with previously untreated, relapsed or refractory WM and LPL in Japan. Velexbru was designated as an orphan drug for the indication of WM and LPL by the Ministry of Health, Labour and Welfare (MHLW) in Japan on November 19, 2019.

About Waldenstrom macroglobulinemia ("WM") and lymphoplasmacytic lymphoma ("LPL") WM and LPL are one of the malignant lymphomas and are classified as "indolent lymphoma" which means one with relatively slow progression*1. It is estimated that there are approximately 240 new cases* 2,3 with LPL per year in Japan. WM and LPL generally grow and spread slowly in the clinical course, with a median survival of more than 5 years, but these are intractable diseases that cannot be cured with existing therapies*4. In Japan, standard treatment has not been established in patients with untreated relapsed or refractory WM and LPL, so a new treatment option is expected for these patient populations.

About ONO-4059-05 Study This study is a multi-center, open-label, single-arm Phase II study, evaluating the efficacy and safety of a monotherapy with Velexbru in patients with previously untreated, or relapsed or refractory WM and LPL. In this study, 27 patients were recruited (untreated 18 patients and relapsed or refractory 9 patients). Patients received Velexbru 480 mg (fasted) once daily and were treated until disease progression or unacceptable toxicity. The primary endpoint of this study is the overall response rate (partial response or greater) assessed by an independent review committee (IRC). The secondary endpoints are progression-free survival (PFS) and overall survival (OS).

About Velexbru Velexbru (tirabrutinib hydrochloride), discovered and developed by ONO, is a highly selective, oral BTK inhibitor and has been developed for the treatment in patients with B-cell tumors and autoimmune diseases in Japan. B cell receptor ("BCR") signaling plays a core role in the survival, activation, proliferation, maturation and differentiation of B cell lymphocyte. The BCR signaling pathway is known to be permanently activated, particularly B cell non-Hodgkin lymphoma (B-NHL) and chronic lymphocytic leukemia (CLL). Velexbru is expected to have a therapeutic effect because it inhibits BTK, a mediator located downstream of BCR. In December 2014, ONO out-licensed Tirabrutinib to Gilead Sciences, Inc. (Gilead) to allow Gilead the right to develop and commercialize the product in all countries of the world, except Japan, South Korea, Taiwan, China and ASEAN countries where ONO retains the development and commercialization rights of the product. In Japan, Velexbru was approved in March 2020 and launched in May 2020 for the treatment of relapsed or refractory primary central nervous system lymphoma (PCNSL).

On August 21, 2020 Affibody Medical AB (publ) ("Affibody" or "the Company"), a Swedish biotechcompany focused on developing next generation biopharmaceuticals based on its unique proprietary technology platforms: Affibody molecules and Albumod, reported its Second Quarter Report for 2020 (Press release, Affibody, AUG 21, 2020, View Source [SID1234575697]).

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Financial Highlights

Revenue for the 2nd Quarter 2020 amounted to SEK 109.6 (217.0) m, and to 119.2 (247.0) m for the full six-month period
Operating result for the quarter amounted to SEK 9.5 (153.2) m, and to -48.6 (128.3) m for the full six-month period
Net result for the quarter amounted to SEK 8.1 (147.6) m, and to -50.9 (122.9) m for the full six-month period
Cash flow for the quarter amounted to SEK -18.9 (315.5) m, and to -97.2 (326.1) m for the full six-month period
Cash and cash equivalents at the end of the period amounted to SEK 277.5 (417.1) m
Significant Events during period

On April 30, 2020 we announced that Daewoong, a South Korea-based pharmaceutical company, had exercised an option under the collaboration related to a half-life extended biotherapeutics product
On May 15, 2020, we and Inmagene Biopharmaceuticals announced a strategic partnership to develop and commercialize ABY-035, a bispecific molecule targeting Interleukin-17A (IL-17), for multiple auto-immune diseases. Inmagene will be responsible for commercialization in mainland China, Hong Kong, Taiwan, and Macau (Greater China), and South Korea, as well as development activities in the Asia Pacific region, excluding Japan. Affibody will retain global commercial rights outside of Greater China and South Korea. The partners will work together to enroll patients into global registrational trials to support Biologics License Applications (BLAs) in multiple indications worldwide. Under the terms of the agreement, Affibody will receive a $10 million upfront payment and is eligible to receive up to $215.5 million in additional regulatory and sales milestones, plus royalties on sales in Inmagene’s commercialization territory.
On June 15, 2020 we announced positive top-line data from our Phase 2, 52-week trial investigating the novel bispecific IL-17A inhibitor ABY-035 in patients with moderate-to-severe psoriasis ("AFFIRM-35").
On June 15, 2020 we announced the completion of the ABY-039 Phase 1 trial and the termination of the ABY-039 program, our FcRn inhibitor, due to tolerability observations that would limit the target product profile of subcutaneous high dose once monthly maintentance injections. Based on these observations Alexion has terminated the co-development agreement with Affibody.
Significant Events during the rest of the Year

An EGM on February 17, 2020 elected José Suarez as Board member.
Affibody is a clinical stage biopharmaceutical company with a broad product pipeline focused on developing innovative bi- and multi-specific next generation biopharmaceuticals based on its unique proprietary technology platforms: Affibody molecules and Albumod.

The company operates a focused experimental medicine model and currently has two clinical stage programs. The first is a therapeutic program that targets psoriasis. The second program is a diagnostic imaging program that is directed primarily towards metastatic breast cancer.

Affibody AB is a holding of Patricia Industries.

On August 21, 2020 Tiziana Life Sciences plc (Nasdaq: TLSA; AIM: TILS) ("Tiziana" or the "Company"), a biotechnology company focused on innovative therapeutics for oncology, inflammation and infectious diseases, reported that the United States Patent and Trademark Office ("USPTO") has granted a patent on use of Milciclib in combination with tyrosine kinase inhibitors (TKIs) such as Sorafenib (Nexavar), Regorafenib (Stivarga) and Lenvatinib (Lenvima) for the treatment of hepatocellular carcinoma (HCC) and other cancers in humans (Press release, Tiziana Life Sciences, AUG 21, 2020, View Source [SID1234568595]). This patent will be published by the USPTO on 1 September 2020 as Patent No. 10,758,541 (Inventor: Kunwar Shailubhai). Like most human cancers, HCC is a complex multi-factorial cancer with multiple underlying mechanisms causing enormous heterogeneity in patient populations. Consequently, patients with HCC often develop resistance towards the monotherapies of existing therapeutics. Thus, there is an urgent need for combination drug treatment approaches targeting different mechanisms to achieve better clinical outcomes.

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Recently, the Company presented two posters on clinical evaluation of Milciclib at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 (ASCO2020). The poster on Phase 2a clinical evaluation of Milciclib, a broad-spectrum inhibitor of cyclin dependent kinases, indicated that the treatment was well-tolerated, and it produced encouraging clinical activity in sorafenib-resistant patients of HCC(1). The second poster was on the evaluation of Milciclib in combination with Regorafenib, a specific TKI drug, in liver transplant patients with HCC recurrence in the MiHRCO (Milciclib and Half Regorafenib CO administration) trial. The combination treatment was safe and produced promising clinical response(2) in these delicate and difficult to treat patients. Additionally, the Company earlier reported data from an animal study suggesting that the combination of Milciclib with Sorafenib, both acting via different mechanisms, suppressed expression of protooncogene c-Myc to produce pronounced synergistic anti-HCC activity(3).

Dr. Kunwar Shailubhai, CEO & CSO of Tiziana Lifesciences, commented, "Advanced cases of patients with HCC have limited therapeutic options because of the heterogeneity of the multiple mechanisms underlying the development of drug resistance and limited clinical responses. Thus, combination of drugs with different mechanism of actions are necessary to achieve superior clinical outcome. We are delighted that we now have this key patent on use of Milciclib in combination with other HCC drugs, including a TKI. Issuance of this patent strengthens our clinical strategy as we move forward with the combination of Milciclib and a TKI for the clinical evaluation of advanced cases of HCC as well as in patients with recurrent HCC after liver transplantation."

The person who arranged for the release of this announcement was Dr Kunwar Shailubhai, Chief Executive Officer and Chief Scientific Officer of the Company

Cited References:

1. Villa, E., Piscaglia, F., Geva, R., Dalecos, G., Papatheodoridis, G., Ciomei, M., Davite, C., Crivori, P., Palejwala, V., Jacob, J., Hamzeh, F., Shailubhai, K., Santoro, A., and A., S. (2020) Phase 2a Safety and Efficacy of Milciclib, a Pan-Cyclin Dependent Kinase Inhibitor, in Unresectable, Sorafenib-Refractory or -Intolerant Hepatocellular Carcinoma Patients. ASCO (Free ASCO Whitepaper) Abstract #29856

2. Pivetti, A., Di Marco, L., Bristot, L., Milosa, F., Maria Critelli, R., De Maria, N., Di Benedetto, F., Palejwala, V., Jacob, J., Shailubhai, K., and Villa, E. (2020) Safety and Clinical Activity of Combination Treatment with Regorafenib and Milciclib in Liver Transplant Patients with Hepatocellular Carcinoma Recurrence. ASCO (Free ASCO Whitepaper) Abstract #307309

3. Jindal, A., Palejwala, V. a., and Shailubhai, K. (2018) Oral Treatment with Milciclib Either Alone or in Combination with Sorafenib Inhibited Tumor Growth in an Orthotopic Model of Hepatocellular Carcinoma. Hepatology 68, 879A

About Milciclib (TZLS-201)

Milciclib (PHA-848125AC) is a small molecule inhibitor of several cyclin dependent kinases such as CDK1, CDK2, CDK4, CDK5 and CDK7. CDKs are serine threonine kinases that play crucial roles in progression of the cell cycle from G1 to S phase. Overexpression of CDKs and other downstream signaling pathways that regulate cell cycles have been frequently found to be associated with development of resistance towards chemotherapies. In a phase I study, oral treatment with Milciclib was found to be well-tolerated and the drug showed promising clinical responses in patients with advanced solid malignancies such as in NSCLC, pancreatic and colon cancer, thymic carcinoma and thymoma.

On August 21, 2020 Prescient reported that it is focused on developing novel, personalised therapies against a range of cancers (Press release, Prescient Therapeutics, AUG 21, 2020, View Source;utm_medium=rss&utm_campaign=health-kick-podcast-chemos-diminishing-role-the-personalised-approach-to-cancer-treatment-and-ptxs-car-t-innovations [SID1234567639]).

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The company’s universal CAR-T and targeted therapies are both personalised medicine approaches that seek to improve patient outcomes and provide new tools for clinicians in combating cancer.

Prescient has a broad pipeline of personalised cancer treatments, comprising CAR-T and targeted therapies, and spanning a range of different cancers.

The company made headlines earlier this month with news of its partnership with global cancer heavyweight Peter MacCallum Cancer Centre to develop new treatments.

As of August 21, 2020, NantKwest, Inc. (the "Company") reported that it entered into a definitive collaboration agreement (the "Collaboration Agreement") with ImmunityBio, Inc., an affiliate of the Company, to pursue collaborative joint development, manufacturing and marketing of certain Covid-19 therapeutics and vaccines (Filing, 8-K, NantKwest, AUG 21, 2020, View Source [SID1234563975]). The terms of the Collaboration Agreement supersede and replace the terms of the binding term sheet executed on May 22, 2020 as previously disclosed. Through their efforts, the parties agreed to jointly develop ceNK, haNK, mesenchymal stem cells (MSC), adenovirus constructs (Ad5), and N-803, a novel IL-15 cytokine superagonist, for the prevention and treatment of SARS-CoV-2 viral infections and associated conditions in humans, including without limitation, COVID-19. NantKwest will contribute the ceNK, haNK, MSC and certain of its manufacturing capabilities and ImmunityBio will contribute Ad5 and N-803. Ad5 will be developed as a vaccine, and ceNK, haNK, MSC and N-803 will each be developed as a therapeutic for treating COVID-19 at various stages of infection.

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From and after the effective date of the Collaboration Agreement, the parties will share equally in all costs relating to developing and manufacturing of the product candidates globally. With the exception of N-803, NantKwest will be primarily responsible for the manufacture of the products. Each party will be responsible for the regulatory affairs and the commercialization relating to its contributed products. The global net profits from the collaboration products will be shared 60% / 40% in favor of the party contributing the product on which the sales are based except if the parties mutually agree because of certain circumstances. All net profits from sales of combined collaboration products will be shared equally. This collaboration will be supervised by the joint steering committee which will be comprised of an equal number of representatives from both parties.

The foregoing description of the Collaboration Agreement does not purport to be complete and is subject to, and qualified in its entirety, by the full text of the Collaboration Agreement, a copy of which will be filed as an exhibit to the Company’s Quarterly Report on Form 10-Q for the period ended September 30, 2020, the terms of which are incorporated herein by reference.