On December 29, 2021 The Janssen Pharmaceutical Companies of Johnson & Johnson reported the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval of teclistamab for the treatment of patients with relapsed or refractory (R/R) multiple myeloma (Press release, Johnson & Johnson, DEC 29, 2021, View Source [SID1234597842]). Teclistamab is an investigational, off-the-shelf, T-cell redirecting, bispecific antibody targeting both B-cell maturation antigen (BCMA) and CD3.

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"Despite all the gains that have been made in treating multiple myeloma, the unmet need still remains very high. Our relentless pursuit of treatments for this disease continues with the same sense of urgency that we have always had," said Peter Lebowitz, M.D., Ph.D., Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC. "We look forward to working with the FDA in their review of our teclistamab submission."

The BLA submission for teclistamab is supported by data from MajesTEC-1 (NCT04557098, NCT03145181), an open-label, multicenter clinical trial evaluating the safety and efficacy of teclistamab in adults with R/R multiple myeloma. In the study, investigators assessed efficacy outcomes, including overall response rate, very good partial response and complete response using International Myeloma Working Group (IMWG) criteria, as well as the safety profile of teclistamab. Updated MajesTEC-1 data were recently presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting.1

"The deep expertise, creativity and persistence of the entire Janssen R&D organization enabled the expeditious advancement of teclistamab for multiple myeloma," said Mathai Mammen, M.D., Ph.D., Global Head, Janssen Research & Development, Johnson & Johnson. "Today’s submission is another important step in our commitment to bring to patients truly transformational medicines that profoundly impact their health."

Multiple myeloma is an incurable blood cancer that affects white blood cells called plasma cells, which are found in the bone marrow and normally make antibodies which fight infection.2,3 When these plasma cells become malignant and develop into multiple myeloma, these myeloma cells proliferate and replace normal cells in the bone marrow. In 2021, it is estimated that nearly 35,000 people will be diagnosed and more than 12,000 will die from this disease in the U.S.4 While some patients with multiple myeloma initially have no symptoms, many patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems or infections.5

About Teclistamab
Teclistamab is an investigational, off-the-shelf, T-cell redirecting, bispecific antibody targeting both BCMA (B-cell maturation antigen) and CD3, the T-cell receptor. BCMA is expressed at high levels on multiple myeloma cells.6,7,8,9,10 Teclistamab redirects CD3-positive T-cells to BCMA-expressing myeloma cells to induce killing of tumor cells.5

Teclistamab is currently being evaluated in several monotherapy and combination studies. In 2020, the European Commission and the U.S. FDA each granted teclistamab Orphan Drug Designation for the treatment of multiple myeloma. In January 2021 and June 2021, teclistamab received a PRIority MEdicines (PRIME) designation by the European Medicines Agency (EMA) and Breakthrough Therapy Designation (BTD) by the U.S. FDA, respectively. PRIME offers enhanced interaction and early dialogue to optimize drug development plans and speed up evaluation of cutting-edge, scientific advances that target a high unmet medical need.11 The U.S. FDA grants BTD to expedite the development and regulatory review of an investigational medicine that is intended to treat a serious or life-threatening condition and is based on preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.12

On December 29, 2021 AstraZeneca reported that it has closed a global development and commercialisation agreement with Ionis Pharmaceuticals, Inc. (Ionis) for eplontersen, formerly known as IONIS-TTR-LRX (Press release, AstraZeneca, DEC 29, 2021, View Source [SID1234597822]).

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The companies will jointly develop and commercialise eplontersen in the US, while AstraZeneca will develop and commercialise it in the rest of the world, except in Latin America.

Financial considerations
Under the terms of the agreement, the upfront payment from AstraZeneca to Ionis is $200m. AstraZeneca will make additional conditional payments of up to $485m following regulatory approvals. It will also pay up to $2.9bn of sales-related milestones based on sales thresholds between $500m and $6bn, plus royalties in the range of low double-digit to mid-twenties percentage depending on the region. The collaboration includes territory-specific development, commercial and medical affairs cost-sharing provisions.

The transaction will be funded with cash and is expected to be neutral to Core earnings in 2021. It will be accounted for as an intangible asset acquisition, recognised initially at the upfront amount, with any potential future milestone payments capitalised into the intangible asset as they are recognised.

Ionis will continue to manufacture and supply eplontersen for the existing clinical studies and process qualification. AstraZeneca will be responsible for commercial supply, with transition timing to be agreed by both parties. AstraZeneca will book all sales generated under the agreement.

The transaction does not impact AstraZeneca’s financial guidance for 2021.

Notes

Eplontersen
Eplontersen is a ligand-conjugated antisense (LICA) investigational medicine designed to reduce the production of transthyretin, or TTR protein, to treat all types of TTR amyloidosis (ATTR), a systemic, progressive and fatal disease.

TTR Amyloidosis (ATTR)
Cardiomyopathy and polyneuropathy due to ATTR are caused by aging or genetic mutations resulting in misfolded TTR protein and accumulation as amyloid fibrils in the cardiac myocardium and peripheral nerves, respectively. In patients with ATTR, both the mutant and wild type TTR protein builds up as fibrils in tissues, such as the peripheral nerves, heart, gastrointestinal system, eyes, kidneys, central nervous system, thyroid and bone marrow. The presence of TTR fibrils interferes with the normal functions of these tissues. As the TTR protein fibrils enlarge, more tissue damage occurs and the disease worsens, resulting in poor quality of life and eventually death. Worldwide, there are an estimated 300,000 – 500,000 patients with amyloid transthyretin cardiomyopathy (ATTR-CM)1,2 and 10,000 – 40,000 patients with amyloid transthyretin polyneuropathy (ATTR-PN).3

On December 29, 2021 Aptose Biosciences Inc. ("Aptose") (NASDAQ: APTO, TSX: APS) reported that the company will participate in two separate biotech events in January 2022: the H.C. Wainwright BIOCONNECT Virtual Conference and the 11th Annual LifeSci Partners Corporate Access Event (Press release, Aptose Biosciences, DEC 29, 2021, View Source [SID1234597824]). The Aptose management team will be hosting investor meetings during both events.

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Details of the events are as follows:

Event: H.C. Wainwright BIOCONNECT Virtual Conference
Date: January 10-13, 2022
Time: Presentations available starting at 7:00 a.m., ET on January 10, 2022
Webcast: Accessible for registered conference attendees via the conference’s virtual platform.

Event: 11th Annual LifeSci Partners Corporate Access Event
Date: January 5-7, 2022
1×1 Meeting
Requests: Register and submit 1×1 meeting requests here.

On December 29, 2021 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CFBI), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address inflammatory, cancer and liver diseases, reported the Company is filing new patent applications in several countries for the treatment of all advanced solid tumors based on new clinical data showing that a patient with advanced hepatocellular carcinoma (HCC), the most common form of liver cancer, was cleared of all tumor lesions following treatment with Namodenoson, an A3 adenosine receptor (A3AR) ligand (Press release, Can-Fite BioPharma, DEC 29, 2021, View Source [SID1234597825]). These pending patent applications are the latest in Can-Fite’s growing IP portfolio covering its platform technology, as well as its drug candidate Namodenoson and its use in a variety of advanced cancers.

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Can-Fite’s Namodenoson, a small orally bioavailable drug that binds with high affinity and selectivity to A3AR, was recently found to clear all cancer lesions in a patient treated under an Open Label Extension program of its concluded Phase II study for the treatment of HCC. In the first quarter of 2022, Can-Fite expects to commence patient enrollment in its pivotal Phase III trial for Namodenoson in the treatment of patients with advanced HCC with underlying Child Pugh B7 (CPB7) cirrhosis to support a New Drug Application (NDA) submission and approval.

"Can-Fite’s IP portfolio includes about 200 patents and pending patent applications in 16 patent families. Given the very strong new data on Namodenoson’s efficacy in advanced HCC, these latest patent filings seek to further fortify our IP position with respect to Namodenoson in liver cancer and also expand our pending claims for advanced disease in other cancer indications", stated Can-Fite’s Chairman, Dr. Ilan Cohn, a Senior and Founding Partner at Cohn De Vries Stadler & Co., a leading IP firm.

About Namodenoson

Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson was evaluated in Phase II trials for two indications, as a second line treatment for hepatocellular carcinoma, and as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug.

On December 29, 2021 ImmunoGen, Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported that Mark Enyedy, President and CEO, will present at the upcoming 40th Annual J.P. Morgan Virtual Healthcare Conference (Press release, ImmunoGen, DEC 29, 2021, View Source [SID1234597826]). The presentation is scheduled for 9:00am ET on January 12, 2022.

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Following the presentation, Mr. Enyedy will be joined by other members of ImmunoGen’s management team for a question-and-answer session at 9:20am ET.

A webcast of the presentation and question-and-answer session will be accessible live through the "Investors & Media" section of the Company’s website, www.immunogen.com; a replay will be available in the same location.